Clients in the Media

/Clients in the Media

November 20, 2019 | SQZ Biotech

“Squeezing” Cells Could Transform Cell Therapy Manufacturing

Manufacturing today’s cell therapies remains expensive and complex due to the need for viruses or electric shocks to engineer patient cells. Yescarta®, for example, one of the first CAR-T therapies approved for sale, takes 3-4 weeks to reach patients and has a price tag of $373,000.00. With CAR-T, expanding out a patient’s T-cells and transducing them with a virus is the most expensive and time-consuming step. That’s according to Armon Sharei, PhD, the 32-year-old CEO of SQZ Biotech, whose cell engineering platform was named as a top 10 world-changing technology by Scientific American in 2014.

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November 18, 2019 | Reata Pharmaceuticals

Plano-based drug developer Reata raises $505 million, expects to double to 400 employees by end of 2020

Plano-based drug developer Reata Pharmaceuticals Inc. raised more than $505 million in a stock sale Monday to speed its transition from a clinical-stage biotech startup to a company that makes treatments for life-threatening diseases. The publicly-traded company will use the money to register and commercialize its first two treatments: one for Alport syndrome, a genetic disease that can cause kidney disease and failure, and the other for Friedreich’s ataxia, a genetic disease that often begins in childhood and leads to brain and nervous system damage.

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November 18, 2019 | Arrowhead Pharmaceuticals

Novel RNA interference agents improve lipid profiles in healthy volunteers

Two novel RNA interference agents, one targeting apolipoprotein C-III and one targeting angiopoietin-like protein 3, improved various lipid parameters in early studies with healthy volunteers, according to new data presented at the American Heart Association Scientific Sessions. One agent targeting apolipoprotein C-III (ARO-APOC3, Arrowhead) was associated with reductions in serum APOC3 and improvements in other lipids, while another agent targeting ANGPTL3 (ARO-ANG3, Arrowhead Pharmaceuticals) was associated with reductions in ANGPTL3 and improvements in other lipids, researchers reported.

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November 18, 2019 | Valneva

Chikungunya Vaccine Candidate Delivered Excellent Study Results

Valneva chikungunya vaccine candidate VLA1553 was generally safe in all dose groups and the local tolerability profile was excellent.

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November 16, 2019 | Redhill Biopharma

FDA Approves Talicia To Treat Superbug Linked To Stomach Cancer

The FDA’s decision to approve Talicia could have far-reaching impact, as this bug infects millions of American adults a year and is one of the strongest known causes of stomach cancer. Talicia is a three-drug combination of omeprazole, a proton pump inhibitor, amoxicillin, a penicillin-class antibacterial, and rifabutin, a rifamycin antibacterial. It is indicated for the treatment of H. pylori infection in adults.

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November 13, 2019 | Vivex Biologics

Tissue Donor Tuly Gonzalez’s Legacy Continues Through The 8 Lives She Impacted

Family and friends gathered at the University of Miami Life Science and Technology Park Wednesday morning to remember Tuly Gonzalez on what would have been her 32nd birthday. She died 11 years ago but the gathering was not just to remember her, but to also celebrate the lives she has impacted though her donation.

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November 12, 2019 | Durect

Novel therapy safe, effective in acute, severe alcoholic hepatitis

In this exclusive video from The Liver Meeting 2019, Tarek Hassanein, MD, FAASLD, professor of medicine at UC San Diego Health and medical director of Southern California GI and Liver Centers, discusses results of a phase 2a study, which demonstrated that DUR-928 was well-tolerated and effective in patients with acute alcoholic hepatitis.

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November 12, 2019 | Durect

AASLD: Durect’s alcoholic hepatitis med repairs liver, cuts mortality in phase 2

Hepatitis used to be the talk of the town at The Liver Meeting, the annual gathering of the American Association for the Study of Liver Diseases. These days, the conversation is turning to nonalcoholic steatohepatitis (NASH), but its cousin alcoholic hepatitis (AH) isn’t getting much attention. Durect is trying to change that.

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November 12, 2019 | Celyad

First Results Out for Off-the-Shelf CAR-T Therapy in Solid Tumors

The interim results come from an ongoing phase I trial in patients with colorectal cancer. Of the 12 patients given Celyad’s CAR T-cell immunotherapy so far, six have seen their tumors shrink. The therapy has also shown no evidence of causing graft-versus-host disease, a condition where foreign immune cells attack the host body. Celyad’s CAR T-cell immunotherapy is aimed to overcome the drawbacks of CAR T-cell immunotherapies already in the market such as Kymriah and Yescarta.

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November 11, 2019 | Reata Pharmaceuticals

Reata’s kidney med bardoxolone delivers in phase 3, teeing up FDA filing

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November 11, 2019 | Adverum

Gene therapy for wet AMD ‘on the horizon’

In the OPTIC trial, patients with wet age-related macular degeneration who were treated with ADVM-022, an intravitreal gene therapy, did not require additional injections following treatment, according to a presenter at the American Academy of Ophthalmology annual meeting.

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November 10, 2019 | Angion

Angion’s kidney therapy advances

The 28-patient study showed that Angion’s drug improved kidney function, based on patients’ estimated glomerular filtration rate (eGFR), a key renal function metric.

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November 8, 2019 | Arrowhead Pharmaceuticals

Why It Could Soon Be Time To Take Profits In This Highflying Biotech Stock

Arrowhead Pharmaceuticals (ARWR) and Johnson & Johnson (JNJ) said Friday they eradicated nearly all traces of hepatitis B virus in a Phase 2 study — sending Arrowhead stock careening toward a record high. On the stock market today, Arrowhead stock jumped 6.3% to 42.10. On Monday, shares hit a record high in intraday action of 42.29. Arrowhead stock broke out on Oct. 17, topping a buy point at 36.90 out of a cup base. Now, the stock is roughly 14% extended. CAN SLIM rules for investing suggest taking some profits when a stock rises 20%-25% above its buy point.

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November 5, 2019 | ProQR

Vision Tech: Several Gene Therapies for Blindness Reach Clinical Trials

ProQR Therapeutics is a Netherlands-based company currently researching RNA therapies to inhibit or counteract the harmful effects of the defective genes associated with the above retinal diseases. How does that work? As you may recall from biology class, genes are the building blocks for the proteins that build and maintain our bodies.

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November 5, 2019 | Synaptive

Robotic Microscope Helping Surgeons During Delicate Brain Tumor Removal Procedures

There is amazing new technology that’s helping surgeons perform the most delicate of all operations — brain surgery. It saved the life of a Westchester County teenager whose headaches were from a brain tumor, CBS2’s Dr. Max Gomez reported Tuesday.

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November 4, 2019 | Synaptive

How Synaptive Medical is Transforming Neurosurgery

Neurosurgery is often thought of as one of the most difficult medical disciplines to pursue. Surgeons are tasked with breaching the multiple protective layers of the brain then navigating through an intricate web of fibers, tissues, blood vessels and fluid to extract a tumor or implant a device — all this without harming the delicate structures within.

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November 1, 2019 | Celyad

Cell and Gene Therapies Necessitate New Lot Release Test Methods

Product-release testing is required for all drug products to ensure their quality and safety. For most conventional small-molecule and biologic drugs, batches are held until the release testing results are received and confirm the product is within specifications. Cell and gene therapies and some of their components (e.g., viral vectors), however, often must be used in further processing or administered to the patient within a short timeframe.

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November 1, 2019 | Arrowhead Pharmaceuticals

This Biotech Stock With A 221% Gain In 2019 Is Ready For ‘Prime Time’

Arrowhead Pharmaceuticals (ARWR) is using a technology called RNAi to tackle two colossal hurdles in treating genetic diseases. And it’s helping Arrowhead stock to finally realize profits after the biotech company operated for years in the red. RNAi, or RNA interference, is a method of silencing genes. Some genetic diseases cause the overproduction of troublesome proteins.

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October 28, 2019 | Ceravance

What Makes A Biopharma Serial Entrepreneur Tick? Part 1

Biopharmaceutical executives, especially those who have founded their own companies, never cease to amaze me. Why? Well for starters, the process of starting with nothing but an idea and then pulling together all the necessary resources to build a company capable of developing and delivering a product to patients is an amazing logistical challenge. It requires someone capable of big-picture thinking. Someone confident in being able to recruit top talent to join them on their journey.

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October 24, 2019 | Celyad

Broadening the Applicability of CAR-T Immunotherapy to Treat the Untreatable

In the early 2000s, chimeric antigen receptor T (CAR-T) cells emerged on the scene with promise as a revolutionary cancer treatment. However, several key challenges and, in particular, downsides related to manufacturing, continue to hamper broad adoption of this therapeutic approach. In addition, existing CAR-T cell therapies are based on autologous approaches (i.e. using the own patient’s cells), meaning that product has to be individually made for each patient. This presents a true challenge in terms of scalability and having the product available for a large patient population.

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October 23, 2019 | ProQR

ProQR announces rare pediatric disease designation, phase 1/2 results for sepofarsen

Sepofarsen has received rare pediatric disease designation from the FDA for the treatment of Leber’s congenital amaurosis 10, according to a press release from ProQR Therapeutics. Sepofarsen, an RNA-based oligonucleotide, is designed to address the underlying cause of LCA10 in the CEP290 gene. With the designation, the drug will receive priority review by the FDA, and it could qualify ProQR for a voucher redeemable for priority FDA review of a subsequent marketing application for a different product, the release said.

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October 22, 2019 | Cidara Therapeutics

Four new drugs may help fight the growing threat of fatal fungi

At a medical mycology meeting here earlier this month, scientists presented promising phase II clinical data for rezafungin, a new member of an existing class called the echinocandins developed by Cidara Therapeutics in San Diego.

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October 18, 2019 | Arrowhead Pharmaceuticals

Biotech Says It Hurdled A Genetic Barrier, But Shares Gave Up A Breakout

Arrowhead Pharmaceuticals stock flirted with a breakout Friday after the biotech company said it’s experimenting with drugs that will “silence” multiple troublesome genes simultaneously. Previously, that was impossible, Chief Executive Christopher Anzalone said late Thursday on CNBC’s Mad Money. Arrowhead Pharmaceuticals (ARWR) is among a slew of biotech companies using technology called RNA interference to silence genes responsible for creating problematic proteins.

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October 18, 2019 | Reata Pharmaceuticals

Reata CEO on positive trial results for drug to treat Friedreich’s ataxia

Warren Huff, Reata Pharma CEO, joins CNBC’s “Power Lunch” team to discuss his company’s positive clinical trial results for a drug to treat the neuromuscular disease Friedreich’s ataxia.

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October 18, 2019 | Valneva

Lyme Disease Vaccine

If the fear of ticks prevents you from enjoying the great outdoors, there might be some good news on the horizon. Thomas Lingelbach with Valneva has been working on a new vaccine designed to ward off Lyme disease.

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October 18, 2019 | Orasis Pharmaceuticals

Eye Drops To Reverse Vision Loss One Step Closer To Market

Eye drops that can reverse poor vision? It may sound like science fiction, but one Israeli company is aiming to bring this product to market. The drops passed their Phase 2b clinical trial earlier this month, meaning they’ve proven to improve farsightedness, the inability to see or read nearby objects, and are highly tolerable. Although results have yet to be released, Israel-based Orasis Pharmaceuticals, revealed details last week about the latest results of the Phase 2b study for their CSF-1 eye drops.

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October 17, 2019 | Arrowhead Pharmaceuticals

Arrowhead CEO breaks down drug partnerships with Johnson & Johnson and Amgen

Jim Cramer chats with Arrowhead Pharmaceuticals CEO Christopher Anzalone about projects the company is working on to silence genes that cause rare genetic disorders.

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October 16, 2019 | Arrowhead Pharmaceuticals

Pharma’s roller-coaster relationship with RNA therapies

Fortunes can shift precipitously in the drug-discovery world. At the start of the twenty-first century, all eyes were focused on a powerful gene-silencing technology called RNA interference (RNAi), and many companies saw the almost limitless potential of harnessing the tool to manipulate genes implicated in diseases.

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October 11, 2019 | Kitov

Kitov updates on US Consensi launch

Kitov Pharmaceuticals today announced its combination drug to treat osteoarthritis pain and hypertension.

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October 11, 2019 | RedHill Biopharma

RedHill Biopharma contends with Crohn’s

RedHill Biopharma Ltd. announced today the full Week 52 results for all subjects in the Phase 3 randomized, controlled study of RHB-104 in Crohn’s disease (MAP US study) and supportive topline results from the open-label extension Phase 3 study (MAP US2 study).

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October 11, 2019 | ProQR

RNA Therapy Improves Vision in Untreatable Genetic Blindness

A phase I/II trial run by the Dutch company ProQR has found that its RNA therapy could significantly improve the vision of people with Leber’s congenital amaurosis, a rare genetic disease for which there is no treatment.

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October 10, 2019 | Reata Pharmaceuticals

AbbVie exits $850M Nrf2 deal with Reata, recouping $330M

After a nine-year run, AbbVie is calling it quits on its Reata Pharmaceuticals partnership. The Big Pharma, which ponied up more than $800 million for the rights to a set of Nrf2 activators for kidney disease and autoimmune disease, is now handing those rights back for $330 million in cash.

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October 10, 2019 | Reata Pharmaceuticals

Reata buys drug rights back from AbbVie for $330M before readout

Reata Pharmaceuticals said Thursday it will pay AbbVie $330 million over the next two years to re-acquire most of the ex-U.S. rights for its leading drug candidates.

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October 9, 2019 | Motus GI

Colonoscopy player announces first commercial placements of device

Motus GI, a medical technology company focussing on colonoscopy, has announced the first commercial placements of its Pure-Vu System as part of its initial U.S. market launch targeting early adopter hospitals.

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October 9, 2019 | Cerevance

Improving How Neurological Drugs are Developed Using Cell-Type-Specific Analysis

Cerevance, a pharmaceutical company focusing on central nervous system diseases, has developed a new process for peering into the gene expression of specific brain cells. Their Nuclear Enriched Transcript Sort sequencing (NETSseq) platform can leverage a technique to specifically sort and isolate specific types of brain cells to more closely study each type.

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October 8, 2019 | Alkahest

AI And Machine Learning Help Unravel The Biology Of Alzheimer’s

Alkahest is a clinical stage company with multiple therapeutic candidates in Phase 2 trials. The company is hoping to develop a treatment for patients with Alzheimer’s but is taking a different path. Alkahest is looking at the plasma proteome and, specifically, how it changes with age.

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October 4, 2019 | Aerami Therapeutics

‘Smart’ Inhaler Can Make Insulin Delivery Quick and Painless

One of the main barriers to medication adherence among diabetics is the painful, constant administration of insulin using a needle and syringes. Moreover, subcutaneous insulin administration has long been admonished for its slow onset of action. People living with this chronic condition need a convenient and efficient method of administering this crucial hormone. Biopharmaceutical company Aerami Therapeutics (formerly Dance Biopharm) may have a solution that changes the way insulin is delivered.

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October 3, 2019 | Electrocore

Spring resident finds hope in new migraine treatment device

Spring resident Leah Cravens wanted to have a baby. With her husband diagnosed with cystic fibrosis, rendering him infertile, an in vitro fertilization was her only choice. The problem? Chronic migraines have been plaguing Cravens for more than 20 years, the medicine for which would be unsafe during her pregnancy.

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October 2, 2019 | Valneva

Lyme Vaccines Show New Promise, and Face Old Challenges

Jessica Reeder worries about ticks and the diseases they carry. She had Lyme disease, her brother had Lyme disease, and every fall her children come home from school with notes reminding parents to do a nightly tick check. Even in Philadelphia, where Reeder lives, the bloodsuckers lurk in the woods around the playground. Her family used to go tent camping with friends, but they stopped after a few people in the group contracted Lyme.

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October 2, 2019 | ArQule

Marc Schegerin, chief financial officer of ArQule, on its breakout year and its MOSAIC trial

Marc Schegerin, chief financial officer of ArQule, discusses ArQule’s breakout year including data showing clinical efficacy of ARQ 531 in CLL and the initiation of its MOSAIC trial

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October 2, 2019 |

Drug company’s Bay Area exodus: All about a new CEO, the right talent — and getting out of founder’s living room

Dance BioPharma has joined the Bay Area exodus, but its story is unlike some of the more high-profile departures.

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September 30, 2019 | Orchestra BioMed

TCT 2019 – Orchestra data could lead to a duet

Data on the company’s pacemaker-neurostimulator combo are probably good enough to permit further development – but first the company wants to hook a partner.

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September 26, 2019 | Diasome

Could Aiming Insulin at the Liver Be an Answer to Using Less?

Amid the debate about why insulin costs so much lies a second question: if the hormone can be delivered more efficiently, will people with diabetes need less insulin? It’s not just a question that’s come up in the era of the artificial pancreas, as people with type 1 diabetes (T1D) work with technology that uses an algorithm to anticipate their blood glucose levels and make constant adjustments to their insulin needs. A company called Diasome is pushing the idea further, by trying to take insulin to the place where it would go if created by islet cells and released by a human pancreas: straight to the liver.

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September 25, 2019 | Rocket Pharma

Rocket Pharma Q&A: efficacy of gene therapy in Fanconi anaemia

Due to the need for chemotherapy and the risks caused by allogeneic stem cell transplants in Fanconi anaemia patients, Rocket Pharma has developed gene therapy candidate RP-L102, which doesn’t need conditioning and corrects the gene mutation underlying Fanconi. Rocket CEO Guarav Shah explains RP-L102’s mechanism of action, as well as how the company is working to improve its cell culturing methods and overcome continuing risks of head and neck cancer.

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September 25, 2019 | Kitov Pharmaceuticals

Overcoming Cancer Drug Resistance with A Bispecific Inhibitor

Drug resistance presents a significant challenge to oncologists. Therapies that were once capable of destroying the tumor are rendered ineffective once one resistant cancer cell begins to multiply. Part of the challenge of developing effective cancer therapies is tackling the multiple pathways by which drug resistance arises. Isaac Israel, CEO of Kitov Pharmaceuticals, and his team are hoping to rise to this challenge. Results from recent preclinical studies suggest that their small molecule drug, NT-219, can effectively block two signaling proteins that function in two different resistance pathways.

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September 25, 2019 | Aerami Therapeutics

Dance Biopharm rebrands to Aerami Therapeutics

Dance Biopharm today said it rebranded as Aerami Therapeutics and will move its headquarters to Durham, N.C. The rebranding comes as the company seeks strategic partners for its Dance 501 inhaled human insulin. The company also hopes the rebranding will help progress into pivotal registration studies and expand to include inhaled therapeutics for endocrinology diseases.

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September 25, 2019 | Aerami Therapeutics

Diabetes relief from an inhaler? Durham company says yes

In the not very distant future, Type 2 diabetes patients may have an option to inhale insulin that acts faster and lasts longer than Lilly’s injected insulin Humalog or its generic, lispro, the current standard treatment. Durham-based Aerami Therapeutics, which changed its name from Dance Biopharm Holdings Inc. on Wednesday, reports that data from its Phase 2 clinical study of Dance 501, a novel gentle mist formulation of human insulin administered with its smart inhaler, showed it had comparable glucodynamic properties to injected insulin, but delivered a faster onset of action. It had no safety issues in the trial.

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September 25, 2019 | Diasome

Novel liver-targeted insulin comparable to insulin lispro in type 1 diabetes

In reporting findings from the phase 2b Insulin Liver Effect (ISLE-1) trial, the researchers also found that participants with a high baseline HbA1c assigned to hepatocyte-directed vesicle (HDV) insulin lispro experienced similar HbA1c reductions while using 25% less mealtime insulin with less hypoglycemia. HDV technology is designed to be mixed with any commercially available insulin to improve glycemic response, according to Diasome Pharmaceuticals.

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September 24, 2019 | Valneva

Inner Workings: Lyme disease vaccines face familiar challenges, both societal and scientific

Just over 20 years ago, a Lyme disease vaccine called LYMErix was approved for sale in the United States. Researchers designed the vaccine to prevent the transmission of the tick-borne pathogen Borellia burgdorferi, which spurs a bacterial infection that can cause fever, headaches, and joint pain if left untreated.

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September 20, 2019 | Durect

Alcoholic hepatitis drug candidate shows ‘life-saving potential’

Data from a phase 2a clinical trial of DUR-928, an endogenous, small-molecule new chemical entity, for patients with alcoholic hepatitis demonstrated that the treatment led to greater reductions from baseline in bilirubin and MELD and improved Lille scores compared with a historical control group from a University of Louisville alcoholic hepatitis study, according to a company press release.

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September 19, 2019 | Nurix Therapeutics

A Therapeutic Approach Built on Protein Degradation

Nurix Therapeutics is harnessing the body’s natural process for controlling protein levels to target and degrade proteins that drive cancer and other diseases. Its small molecule therapies control key enzymes responsible for protein breakdown and can be used to modulate the levels of proteins within cells. The company believes it can leverage the approach to treat a range of diseases. We spoke to Arthur Sands, CEO of Nurix, about the company’s discovery platform, its pipeline of cancer therapies, and its recent collaboration with Gilead Sciences to discover and develop drug candidates against up to five targets.

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September 12, 2019 | Nurix

Getting Higher-Quality Hits out of DNA-Encoded Libraries

DNA-encoded libraries are quickly surpassing traditional high throughput screens as industry’s first stop for hit generation. Now, developers are taking the technology up a notch to get more tractable hits in the output. DNA-encoded library (DEL) screens blow conventional high throughput screening techniques out of the water when it comes to library size and diversity, enabling screening of millions more compounds without sacrificing time or cost.

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September 9, 2019 | Oncocyte

OncoCyte Adds Lung Cancer Assay Resurrected by Razor After Previous Sale to Life Tech

OncoCyte provided additional details about the history of the Razor test, its timeline for making it commercially available, and plans for a follow-on study.
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September 5, 2019 | Oncocyte

OncoCyte Spells Out the Acquisition Plan for Razor Genomics

OncoCyte’s newly appointed CEO Ronald Andrews is making good on a promise for more transparency in the company. Investors and analysts witnessed this new level of transparency first hand during a detailed conference call the Alameda, CA-based company held concerning its plan for acquiring Razor Genomics.

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September 5, 2019 | Orchestra BioMed

Orchestra BioMed wins CE Mark for BackBeat hypertension neurostim, names CMO

Orchestra BioMed said yesterday that it received the CE Mark for its Moderato implantable pulse generator system for BackBeat cardiac neuromodulation therapy (CNT), just nine days after naming a new chief medical officer. BackBeat therapy is a bioelectronic treatment designed to lower blood pressure while modulating the autonomic nervous system. The Moderato implantable device is designed to deliver BackBeat CNT and provide standard pacemaker functions.

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September 4, 2019 | Orchestra BioMed

Pacemaker-Like Implant Cleared in Europe to Lower Blood Pressure

Orchestra BioMed, a company out of New Hope, Pennsylvania, won the European CE Mark of approval for the Moderato implantable pulse generator which delivers Orchestra’s unique BackBeat Cardiac Neuromodulation Therapy (CNT) to treat hypertension. High blood pressure is typically treated using drugs, but BackBeat allows even existing cardiac implants to deliver therapy in a novel way by modulating the bioelectronic signals associated with blood pressure control.

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September 4, 2019 | Labiotech

Overcoming Clinical Setbacks to Fight Tropical Diseases

Sometimes things don’t go your way in the biotech industry. The CEO of Valneva, Thomas Lingelbach, shared his experience of migrating from big pharma to biotech, and how his first biotech company weathered phase III failures to develop vaccines for neglected diseases.

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September 4, 2019 | Columbia Care

With pancreatic cancer, what Stephen needs is legalised cannabis

A year into his cancer treatment, Stephen heard about the benefits of medical marijuana and CBD oil, but it has proven difficult to get.

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September 4, 2019 | Valneva

How Valneva are changing the approach to vaccine clinical trials

You’ve invested millions of dollars, spent over a decade conducting trials and it’s only when you reach the final phase that you realize your product doesn’t even work. That can be the unfortunate reality for vaccine development. Few understand this better than Thomas Lingelbach, CEO of Valneva, a company focused on the development of innovative vaccines in areas of unmet medical need. With over twenty years of experience in the industry, having worked for Chiron, Novartis and Intercell, Thomas has overseen numerous vaccines go from bench to market.

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September 3, 2019 | Anima Biotech

https://www.genengnews.com/get-the-gen-digital-magazine/

For Anima Biotech, it’s all in the translation—that is, mRNA translation. Drug development and small molecule discovery have long targeted small binding pockets or catalytic sites on proteins’ surfaces. This approach has yielded many therapeutic molecules as well as substantial returns on investment, but it is not without its pitfalls.

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August 29, 2019 | Krystal Biotech

How Krystal Biotech Went From Founding To IPO In 18 Months

Chairman and CEO Krish Krishnan is an experienced biotech executive. But even he didn’t expect to go from self-funding Krystal Biotech to IPO in 18 months. The Pittsburgh-based company, which Krishnan co-founded with his wife and COO Suma Krishnan, is working to develop to develop treatments for rare, orphan skin diseases caused by the absence of, or a mutation in, a single gene.

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August 27, 2019 | Diasome

Using Social Listening In The Design Of A Diabetes Clinical Trial

Social media has become increasingly important in the biopharma space as it not only allows emerging clinical-stage companies to efficiently increase their visibility online by posting interesting content, but it also provides a unique platform to engage with members of specific disease communities. By listening to these members’ voices online, valuable insight can be gained about the direction in which a company should go.

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August 27, 2019 | Anima Biotech

Seeking Small Molecule Drugs to Control mRNA Translation

Wouldn’t it be amazing to be able to see a protein being made in a cell in real time? And wouldn’t it be even better if you could use that capability to discover new drugs to previously “undruggable” targets, creating new medicines? Well that’s exactly what Anima Biotech is aiming to do. “We are going after mRNA translation to specifically target protein production using small molecules,” Yochi Slonim, co-founder and CEO of Anima, told BioSpace.

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August 22, 2019 | Valneva

Can a new Lyme disease vaccine overcome a history of distrust and failure?

As the threat of Lyme disease grows and fears surrounding it spread faster than the ticks that carry the infection, researchers are developing two vaccine or vaccine-like approaches to prevent this increasingly problematic disease. But don’t expect to get one soon. They are at least three to five years away from clinical use, according to their developers.

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August 20, 2019 | Krystal Biotech

Topical Gene Therapy Shows Promise for DEB

A novel topical gene therapy, developed by Krystal Biotech, shows promise for treating wounds of patients with dystrophic epidermolysis bullosa (DEB). The therapy KB103 targets the type VII collagen gene (COL7A1), missing in patients with DEB, to improve wound closure and skin cohesion.

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August 20, 2019 | Alkahest

Old made new: Alkahest plasma fraction research entering another phase II

Chief Commercial and Strategy Officer Elizabeth Jeffords told BioWorld that the endpoints in Alkahest Inc.’s just-begun phase II trial with human plasma fraction GRF-6021 will not only test how well patients bounce back from hip or knee arthroplasty but “could absolutely support the work in cognitive and neurodegenerative indications as well,” where data rolled out earlier this month.

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August 15, 2019 | Samumed

In conversation with Samumed CEO Osman Kibar: Drugging Wnt, restoring youth and unconventional capital

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August 14, 2019 | Catalyst Biosciences

Straight A’s for MarzAA

Last month at the 2019 Congress of the International Society on Thrombosis and Haemostasis (ISTH), Catalyst Biosciences Inc. presented Phase 2 data regarding its subcutaneous (SQ) Factor VIIa (FVIIa) variant marzeptacog alfa (activated) (MarzAA) for prophylaxis in patients with hemophilia A or B with inhibitors. The study met its primary endpoint of significantly reducing the annualized bleed rate, as well as all secondary endpoints of safety, tolerability and lack of anti-drug antibody or inhibitor formation.

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August 12, 2019 | ProQR

ProQR’s retinitis pigmentosa candidate gets investigational NDA clearance

The FDA has cleared an investigational new drug application for QR-1123, an investigational oligonucleotide treatment candidate for vision loss with autosomal dominant retinitis pigmentosa due to the P23H mutation in the rhodopsin gene, according to a press release from ProQR Therapeutics.

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August 9, 2019 | OSE Immunotherapeutics

Eat this, don’t eat that: CD47 companies’ first hurdle

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August 8, 2019 | Arena Pharmaceuticals

Inside the Arena Makeover | Kevin Lind, CFO, Arena Pharmaceuticals

Up until about three years ago Kevin Lind would likely have been identified as yet another gifted private equity executive – capable of issuing business remedies from the tip of his tongue. At least in the minds of his CFO peers, who are accustomed to listening to PE pundits routinely hand down such remedies for ailing businesses. For Lind the CFO office at Arena Pharmaceuticals is a game changer, where he no longer hands down remedies but serves as a finance leader – an individual tasked with summoning others forward and building trust across an organization even as he or she sometimes completes necessary layoffs.

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August 7, 2019 | Alkahest

Initial Reports Positive for Plasma Treatment of Alzheimer Disease

Officials are optimistic about the initial results of a phase 2 clinical trial for a new plasma-based treatment for mild-to-moderate Alzheimer disease (AD). California-based Alkahest announced that GRF6019, a proprietary plasma fraction comprised of about 400 proteins, was both safe and well-tolerated with no cognitive decline as a treatment for AD.

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August 5, 2019 | Alkahest

Alzheimer’s patients didn’t decline after getting a cocktail derived from young blood, but big questions remain

Alzheimer’s patients who received an experimental protein cocktail derived from young blood plasma maintained their performance on measures of cognition and function after six months, the biotech company behind the therapy, Alkahest, said on Monday.

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August 1, 2019 | Neubase

NeuBase takes antisense oligos to more targets in more places

Newcomer NeuBase Therapeutics is re-engineering antisense oligos to access a broader range of targets, while simultaneously solving the modality’s dosing and delivery issues. With three approvals since the start of 2016, antisense oligonucleotides (ASOs) have started to hit their stride, yet first-generation technologies are limited on multiple fronts.

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July 29, 2019 | Alkahest

Oral AMD treatment meets phase 2 endpoints

At the American Society of Retina Specialists meeting, Michael Stewart, MD, chairman and professor of ophthalmology at the Mayo Clinic of Jacksonville, Florida, discusses phase 2 results of AKST4290 (Alkahest), an orally administered small molecule CCR3 inhibitor.

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July 29, 2019 | Alkahest

Alkahest Phase 2a Study AKST4290-202 in Refractory Wet AMD Meets Primary and Secondary Endpoints

Alkahest announced that its phase 2a trial of AKST4290, an orally-administered small molecule CCR3 inhibitor, met the primary endpoint of achieving an increase in best corrected visual acuity (BCVA) in patients with refractory wet or neovascular age-related macular degeneration (nAMD). AKST4290 was also found to be safe and well-tolerated, meeting the secondary endpoint of the trial.

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July 25, 2019 | electroCore

Anthony Fiorino, MD, PhD: Using nVNS in Various Migraine Populations

At the 2019 American Headache Society Annual Meeting, July 11-14, in Philadelphia, Pennsylvania, one of the topics of focus among the newly approved therapies for migraine was the use of devices to treat the condition acutely. As such, a number of companies presented data on neuromodulation and neurostimulation devices—including electroCore, which has undergone the development of noninvasive vagus nerve stimulation (nVNS) to treat migraine, episodic cluster headache, and is currently being studied in post-traumatic headache.

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July 15, 2019 | Oncocyte

Oncocyte CEO, Ronnie Andrews On Developing New Ways To Test For Lung Cancer

Watch “Ronnie Andrews On Developing New Ways To Test For Lung Cancer”, an archived episode of The Watch List originally aired 07/15/2019 on the TD Ameritrade Network.

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July 12, 2019 | Altimmune

Not just a vaccine company: How this Gaithersburg biotech CEO plans to grow the business

The Gaitherburg biotech’s CEO, not yet a year into his tenure, sees the prospect of more acquisitions or licensing deals going forward.

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July 12, 2019 | Check-Cap

Positive results of a prep-free colon cancer screening device reported

Check-Cap announced positive final results from its completed post-CE approval study evaluating the efficacy and safety of the C-Scan system. “The final results from the post-CE approval study confirm the potential clinical value of the C-Scan system. The results also demonstrate an advancement in the detection of larger polyps, generally considered to have higher potential for malignant transformation,” Nadir Arber, MD, MSc, MHA, director of the Integrated Cancer Prevention Center at the Tel-Aviv Sourasky Medical Center, said in a press release.

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July 12, 2019 | electroCore

Interview with the CEO: electroCore, Inc. (NASDAQ:ECOR)

Francis R. Amato is Chief Executive Officer of electroCore, Inc. Prior to being the Chief Executive Officer and joining electroCore in 2012, Mr. Amato spent 22 years in the pharmaceutical industry, most recently at Merck, where he served as Vice President of the Specialty Commercial Operations Group within Global Human Health.

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July 12, 2019 | Orchestra BioMed

Encore: Orchestra BioMed quickly nets another $34M for its cardiovascular pipeline

Hot on the heels of inking an international distribution deal with Terumo, Orchestra BioMed has secured $34 million in new funding for its devices aimed at high blood pressure and blocked arteries. The series B-1 raise, and its previous series B round, also provide for follow-on investments of up to $57 million in the company. The latest preferred stock financing was led by Perceptive Advisors, RTW Investments and Soleus Capital, alongside additional backing from Terumo and previous investor SternAegis Ventures.

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July 11, 2019 | ProQR

ProQR: RNA Therapy Has Potential to Restore Vision in Patients With Usher Syndrome

Usher syndrome is an inherited retinal degeneration that affects hearing, balance, and vision. Usher syndrome caused by USH2A mutations is one of the most common causes of retinitis pigmentosa (RP) with syndromic features. Currently there is no treatment for the sensory deficits caused by Usher syndrome.

In an attempt to fill this void, QR-421a is an antisense oligonucleotide (ASO) and designed to specifically target mutations in exon 13 of the USH2A gene for the treatment of patients with RP.

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July 11, 2019 | Orchestra BioMed

Orchestra Biomed raises $34M to advance circulatory solutions

New Hope, Pa.-based Orchestra Biomed Inc. scooped up $34 million in a series B-1 preferred stock financing led by Perceive Advisors, RTW Investments and Soleus Capital. The funds will be used to advance development of the company’s Backbeat cardiac neuromodulation therapy (CNT) system, to support commitments to Orchestra’s strategic global partnership with Tokyo-based Terumo Corp. for the development and marketing of the Virtue sirolimus-eluting balloon (SEB) and to grow the company’s product pipeline and pursue future collaborations.

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July 2, 2019 | SQZ Biotech

The Cell Therapy Discovery About To Disrupt Clinical Trials

Armon Sharei developed an interest in novel cell therapies while pursuing his PhD in chemical engineering at MIT. While pursuing that interest, he made a discovery that today is the foundation of SQZ Biotech, a cell-therapy company where he serves as CEO. Sharei discovered a new method of inserting materials into cells more effectively than anything that currently existed. His discovery is now opening doors to what researchers can force cells to do, and, as a result, what cells can do for patients.

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June 27, 2019 | Daily Mail

My sore throat turned out to be a brain tumor: Girl, 19, describes shock diagnosis that required innovative new surgical technique to remove the lump

For years, Christina Giuffrida had been experiencing an on-again, off-again sore throat. The 19-year-old from Yonkers, New York, also had slight balance issues and would struggle to hear people speaking at normal volumes. One doctor told her and her parents, it was a problem with her eyesight. Another said it was a flare-up from a previous mononucleosis infection or allergies.

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June 26, 2019 | Cerevance

It’s time to find new targets for brain diseases instead of just pursuing old ones

Ifirst learned about drug discovery and development in the mid-1990s when two of my sons were diagnosed with a brutal, untreatable genetic disease called ataxia-telangiectasia (A-T). Children with A‑T struggle with immune deficiency and lung problems, and have an extremely high cancer risk.

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June 24, 2019 | Krystal Biotech

With phase 2 update, Krystal Biotech eyes phase 3 for rare skin disease gene therapy

Krystal Biotech unveiled data from a small phase 2 study showing its topical gene therapy closed the majority of wounds in patients with a rare skin disorder. The company is waiting on data for how long the wounds stayed closed after treatment before it moves the program into phase 3. It expects to start the pivotal trial by the end of the year.

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June 24, 2019 | Krystal Biotech

Gene therapy biotech sees its stock rocket higher on promising results for rare cases of butterfly disease

Shares of Krystal Biotech took off this morning $KRYS after the little biotech reported promising results from its gene therapy to treat a rare skin disease called epidermolysis bullosa.

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June 21, 2019 | Arena Pharmaceuticals

First Patient Dosed in Phase 3 Trial of Etrasimod for Moderate to Severe UC

The Phase 3 trial ELEVATE UC 52 evaluating Arena Pharmaceuticals’ investigational therapy etrasimod for patients with moderately to severely active ulcerative colitis has dosed its first patient. “We are pleased to enroll the first patient in the ELEVATE UC trial, supporting etrasimod’s potential as an important future therapy for ulcerative colitis,” Darshan Anandu, MD, of G.I. Specialists of Houston, said in a press release.

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June 19, 2019 | Nurix

With $45M Nurix Deal, Gilead Enters Crowded Protein Degradation Field

Gilead Sciences is turning to Nurix Therapeutics in an effort to discover new drugs that harness the cellular machinery our bodies use to dispose of damaged or harmful proteins.

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June 19, 2019 | Nurix

Why Gilead could pay more than $2 billion in deal with this small S.F. biotech

Founded by three professors from UC Berkeley and UCSF, Nurix’s protein-degradation approach to cancer, and potentially other diseases, could stop cancer in its tracks.

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June 18, 2019 | electroCore

The Differences Between Device and Drug Studies

Device and drug trials pose a variety of challenges; they are, after all, completely different in study design, have varying regulatory pathways, as well as differing executional challenges. In this interview, Tony Fiorino, Chief Medical Officer of electroCore, will discuss challenges he has faced with device studies, and will elaborate on his experiences about the differences between device and drug trials.

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June 17, 2019 | ArQule

Arqule sees strength in early BTK inhibitor data; shares rise on phase I CLL news

Shares of Arqule Inc. (NASDAQ:ARQL) hit a 52-week high Friday on news that the company’s Bruton’s tyrosine kinase (BTK) inhibitor, ARQ-531, achieved partial responses in four of six heavily pretreated people with relapsed/refractory (r/r) chronic lymphocytic leukemia (CLL) during an ongoing phase I trial.

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June 13, 2019 | Orchestra BioMed

Terumo plans to take Orchestra BioMed’s sirolimus balloon international in $35M deal

Orchestra BioMed is partnering up with Terumo to help develop and commercialize its sirolimus-eluting balloon in both coronary and peripheral cardiovascular interventions. The Virtue angioplasty system is designed to deliver a bioabsorbable, sustained-release formulation of sirolimus directly into the artery wall. The drug helps prevent the vessel from narrowing and reclogging following the placement of a stent or other intervention, a process known as restenosis.

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June 12, 2019 | eGenesis

A CRISPR startup is testing pig organs in monkeys to see if they’re safe for us

Gene-editing company eGenesis is now carrying out experiments to help solve a critical shortage of human organs available for transplant.

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June 11, 2019 | Celyad

CAR-T Therapy Development on the Rise

Now, the biotech company Celyad is looking for a non-gene-edited approach to create allogeneic CAR-T cells using RNAi. Last October, Celyad announced a collaboration with shRNA experts Dharmacon, a Horizon Discovery company, to use their optimized shRNA technology to create CAR-T therapies.

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June 5, 2019 | Arena Pharmaceuticals

Arena Pharmaceuticals’ Selective CB2 Receptor Activator Drug Candidate Fights Pain in Irritable Bowel Syndrome

For about 12 percent of the world’s population living with the bowel movement disorder, irritable bowel syndrome, abdominal pain is a part of everyday life. While there are drugs available to treat symptoms like constipation and diarrhea, there is one complication that few irritable bowel syndrome drugs treat effectively. “What’s common to all irritable bowel syndrome patients is the presence of abdominal pain, and it is probably the aspect of the condition that is most difficult to treat,” said Dr. Preston Klassen, Head of Research and Development and Chief Medical Officer at Arena Pharmaceuticals.

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June 4, 2019 | Orchestra BioMed

FDA Approvals

Virtue Sirolimus-Eluting Balloon (SEB) for the treatment of coronary in-stent restenosis (ISR) secured breakthrough device designation from the FDA. Virtue SEB is a drug/device combination product delivering sustained-release bioabsorbable nanoparticle-encapsulated Sirolimus for preventing restenosis directly to the artery during balloon angioplasty, without the need for a coating.

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June 3, 2019 | Check-Cap

Colon Cancer Screening without the Prep

Alex Ovadia, CEO of Check-Cap, talks about the company’s ingestible imaging capsule-based system for colorectal cancer screening.

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May 31, 2019 | eGenesis

Gene Editing Startups Fabricate Industrial-Grade CRISPR Tools

Porcine xenotransplantation research was halted in the 1990s because of the risk of transmitting porcine endogenous retrovirus (PERV) to patients during organ transplants. To help resurrect the field, genomic pioneer George Church, PhD, and researcher Luhan Yang, PhD, co-foundeed eGenesis, a company devoted to developing human-compatible organs, tissues, and cells. eGenesis is working to make porcine tissue PERV-free and as human-like as possible to enable life-saving medical interventions.

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May 23, 2019 | Abivax

ABX464 as a Treatment for Ulcerative Colitis

An open-label study has found data that could support the use of ABX464 as a treatment for moderate to severe ulcerative colitis (UC).  Investigators presented information from the ABX464-102 phase 2a study, which found that almost all patients demonstrated clinical response and nearly half were in clinical remission, at Digestive Disease Week 2019 in San Diego, CA

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May 22, 2019 | mRNA and Artificial Intelligence: An Interview with Anima Biotech’s Yochi Slonim

mRNA and Artificial Intelligence: An Interview with Anima Biotech’s Yochi Slonim

In the life sciences industry, there is a wide variety of directions that companies can go. Many high-profile biopharma companies have a focus on messenger RNA (mRNA), and some are broadening their horizons by taking an interest in artificial intelligence. One company, Anima Biotech, has a unique approach to both.

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May 22, 2019 | Reata Pharmaceuticals

Reata’s drug, Bardoxolone, shows promising results for chronic kidney disease

Reata is in its ‘final pivotal study’ for its new drug, bardoxolone. In the trials, the drug has showed promising results to reverse kidney damage. Yahoo Finance sits down with the CEO of Reata, Warren Huff, who founded the company in 2002, to discuss the plans to launch the product in the near future.

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May 20, 2019 | Reata Pharmaceuticals

Reata’s big test approaches

Reata Pharmaceuticals has shown that perseverance can pay off. Its lead project, bardoxolone, which is now focused on rare kidney diseases, is forecast to sell $1.3bn in 2024 after earlier flopping in a broader indication. The company should soon find out whether bardoxolone is approvable, with phase III data in the project’s first indication, Alport syndrome, due in the second half of the year. And Reata and analysts alike are confident after positive results from the phase II portion of the same study.

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May 14, 2019 | Noxopharm

A booster for immuno-oncology drugs

Touting a new, more robust class of immune-oncology drugs to date, Noxopharm has released data from the first series of preclinical studies confirming that idronoxil (IDX), the active ingredient in the company’s anti-cancer drug candidate, Veyonda, not only activates, but potentially boosts the function of current immuno-oncology drugs.

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May 13, 2019 | ProQR

The Eye Travels Wide: ProQR’s Bid For Leadership In The Genetics Of Sight

How did a youthful vendor of IT services, faced with a personal exposure to crippling illness, attract some of the best minds in biotech to launch a company with a then-untested RNA technology that today is a contender for leadership in the rarified space of inherited retinal disease? The answer may lie in the ambitiously definitive name that 36-year-old founder Daniel de Boer gave to his enterprise with a mission: ProQR – an active synonym for finding cures.

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May 13, 2019 | Vyome Therapeutics

Bactericidal, antibiotic acne topical treatment reduces inflammatory lesions

Vyome Therapeutics reported results from two clinical trials of its anti-acne candidate VB-1953; the bactericidal, antibiotic topical gel kills sensitive and resistant strains of C.acnes infection, and has been shown to reduce inflammation, at the Society for Investigative Dermatology annual meeting in Chicago.

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May 10, 2019 | Celyad

All To Play For: CAR-T Specialist Celyad’s Big Year

Celyad’s leadership team talks to Scrip about the critical nature of the coming months for its lead cancer therapy product, and CAR-T market differentiation.

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May 3, 2019 | Pluristem

NASA teams up with Israeli biotech firm to work on solving long duration spaceflight

It is one of the biggest hurdles NASA must overcome before astronauts embark on long duration space travel to Mars or building permanent settlements on the moon: how to minimize the heavy toll exacted on even the most physically fit human bodies in zero gravity. So to help ensure astronauts don’t arrive at their destination too weak to explore, the space agency has teamed up with Pluristem, an Israeli biotech company, to investigate whether injecting them with cells derived from a mother’s nutrient-rich placenta can increase muscle volume — a process now in trials on Earth for the elderly.

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May 2, 2019 | Columbia Care

Columbia Care Begins Trading on NEO Exchange in Canada

Nick Vita, CEO and co-founder of medical cannabis company Columbia Care, talks about being acquired by Canaccord Genuity Growth Corp. and what it will allow the company to do.

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May 1, 2019 | Noxopharm

From Academia To Pharma CEO: Challenges And Lessons Learned

A growing number of researchers are transitioning from the traditional academic route to entrepreneurship. In doing so, these individuals often find themselves in unexpected leadership roles. Prior to Noxopharm, I spent a significant amount of time as a medical researcher at The University of Sydney and developed a scientific hypothesis that led me to enter the biotechnology industry and ultimately found several companies, serving as their CEO.

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April 29, 2019 | Synaptive Medical

Canadian hospital, vendor collaborate on ‘head-only’ MRI

Synaptive Medical is developing a magnetic resonance imaging system that aims to provide the imaging technology directly at the point of care. The Nova Scotia Halifax Authority is collaborating on the project to develop the head-only MRI—called Evry—with Synaptive Medical, a designer of surgical planning and navigation technologies. The technology will be worked on and tested at the QEII Health Sciences Centre in Halifax, Nova Scotia.

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April 24, 2019 | Arrowhead Pharmaceutical

Arrowhead starts triple-combo hepatitis B test, triggering J&J payday

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April 24, 2019 | Orchestra BioMed

Virtue sirolimus-eluting balloon granted breakthrough designation for coronary ISR

Pennsylvania-based health company Orchestra BioMed has obtained FDA breakthrough device designation for its Virtue sirolimus-eluting balloon (SEB), according to a statement issued April 24. Virtue SEB, a drug/device combination product that delivers sustained-released bioabsorbable nanoparticle-encapsulated sirolimus directly to a patient’s artery during balloon angioplasty, works without the need for a drug coating, Orchestra said. It’s intended for use in individuals with coronary in-stent restenosis (ISR), a complication that comprises more than 10% of total interventional cardiology procedures.

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April 18, 2019 | Diasome

Diasome’s Liver Targeted Insulin

We spoke with Robert Geho, CEO of Diasome Pharmaceuticals, Inc. — www.diasome.com — about its nanotechnology additive that delivers insulin to the liver where it can substantially improve T1D glucose control.

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April 16, 2019 | Cerevance

Cerevance gains a new drug discovery chief as another AstraZeneca executive jumps ship

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April 11, 2019 | Oxurion

Oxurion CEO discusses expertise in treating diabetic eye disease and the company’s robust pipeline

Patrik De Haes, MD is CEO of Oxurion (Oxurion.com). Their first major success was cardiac drug tPA; today, Jetrea is their first commercialized product for diabetic eye disease, with three new molecules for diabetic eye disease in development.

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April 11, 2019 | Angion

Angion Biomedica Nears End of Clinical Trials for Organ-Repairing Molecule

Repairing a damaged organ in order to allow an individual to have a healthier life, as well as a greater quality of life, could soon be a reality as New York-based Angion Biomedica Corp.moves toward completing clinical trials of its lead asset.

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April 11, 2019 | Nurix

Nurix: Two Sides Of Protein Degradation

Nurix is flipping the concept of targeted protein degradation on its head. In addition to the usual approach of inducing target destruction, the company has technology that can block E3 ligase activity and increase a protein’s levels. The latter is the basis of its preclinical immuno-oncology partnership with Celgene Corp.

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April 10, 2019 | Check-Cap

Check-Cap Tests Colorectal Cancer-Screening Capsule in New Study

Earlier this month, several people took a pill that might have the ability to prevent colorectal cancer. These individuals are participants of a new pilot study launched by Check-Cap, a medical diagnostics company intent on developing the world’s first ingestible imaging capsule for colorectal cancer screening.

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April 8, 2019 | Isofol

Fight Colon Cancer

In this episode, Dr. Gabrail and Carrie discuss colon cancer with special guest Anders Rabbe the CEO of Isofol.

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April 4, 2019 | Celyad

Second Shot for shRNA

Having long been demoted to tool compound status, shRNA is gaining renewed attention as a therapeutic modality by offering a simpler path to creating next-generation cell therapies than gene editing. Celyad has jumped in, using Horizon Discovery’s shRNA platform to launch a new arm of its CAR T cell pipeline.

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April 3, 2019 | eGenesis

Do animals hold the key to the global organ shortage?

Scientist Wenning Qin holds up a Petri dish, carefully sloshes around the pink liquid inside, and slides it under a microscope. Some identical tiny slashes come into focus. These cells, she explains, are derived from the ear of a pig. And they may contain the future of animal to human organ transplantation.Click edit button to change this text.

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April 2, 2019 | Reata Pharmaceuticals

Power Lunch: Reata CEO on drug that shows signs of reversing kidney damage

Reata CEO Warren Huff discusses his company’s new drug, which has shown signs it can reverse kidney damage.

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April 1, 2019 | Vyome Therapeutics

Companies To Watch: Vyome Therapeutics

Transitions challenge every company, but maybe biopharmas most of all. Drug development consists of a series of big steps, each one bringing a dramatic increase in the scale of a company’s expertise, operations, and risk-taking. Vyome is reaching the cusp of an upcoming Phase 2b trial, and it faces its greatest transition so far as a small company. With roots in India but new headquarters in Princeton, NJ, Vyome must now play for much higher stakes in focusing its dermatology portfolio on the U.S. market.

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March 27, 2019 | OSE Immunotherapeutics

Artificial Intelligence Could Beat Tumors Resistant to Immunotherapy

The French company OSE Immunotherapeutics has signed a partnership to use artificial intelligence to develop treatments for tumors that don’t respond to checkpoint inhibitor drugs.

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March 27, 2019 | Samumed

The Rheumatologist

SM04690 is a small-molecule inhibitor of the Wnt pathway currently in clinical trials to evaluate its use managing knee osteoarthritis (OA). The treatment is an intra-articular injection being developed as a disease-modifying osteoarthritis drug (DMOAD). Preclinical data suggest this agent has a dual action mechanism, with three joint health effects: cartilage generation, slowing of cartilage breakdown and inflammation reduction. Presently, no DMOADs are approved by the U.S. Food and Drug Administration.

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March 25, 2019 | OSE Immunotherapeutics

Bispecific antibodies a new hot rod on the anti-cancer circuit

Next-gen bispecific antibodies (BsAbs) are on the move. The blockbuster potential of the class has galvanized biopharma, with deals and assets racing to capture the pole position as the entire field gains speed.

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March 20, 2019 | DalCor

DalCor Keeps Faith In CETP Biomarker, Despite No Dice For Merck’s REVEAL Genetic Analysis

Hopes for a precision approach for the vast cardiovascular disease population live on – past failed studies of HDL-raising drugs and lack of evidence for genetic typing in Merck’s latest analysis of anacetrapib in REVEAL.

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March 20, 2019 | DalCor

ACC: Merck anacetrapib reveal bodes neutral at best for DalCor’s dalcetrapib

Pfizer, Roche, Lilly and finally, Merck. One by one, they ended their CETP inhibitor programs, giving up the ghost on a once-vaunted class of cholesterol-targeting drugs thought to be a potential game-changer for heart disease. In spite of the mass exodus, little DalCor Pharmaceuticals emerged in 2015, with the Roche castoff dalcetrapib and a goal to succeed where the heavy hitters of Big Pharma had failed.

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March 20, 2019 | The New Orleans Advocate

Tulane working on new stroke drugs that tweak brain chemistry

“Drugs currently on the market to treat hypertension work on the blood vessels by either dilating them, reducing pressure in them by eliminating salt and water or slowing down the heart rate,” said Bruno Besse, M.D., medical director of the French biotech company Quantum Genomics, which developed the drug.

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March 18, 2019 | ProQR

ProQR Therapeutics sets goals for 2023

ProQR Therapeutics, a company focused on treating rare diseases, announced plans to advance its pipeline of RNA medicines to treat inherited retinal diseases to include two fully approved commercial products in Europe and the U.S., three late-stage clinical therapies and seven early-stage therapies by 2023.

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March 8, 2019 | Diasome

Oral Insulin Innovation

While clinical data suggest an oral insulin product may finally be within reach, the technology struggles to compete with injectables on price and efficacy. A handful of academics and companies are pushing preclinical innovation in formulation and device design, aiming to solve the commercial conundrum.

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March 7, 2019 | Krystal Biotech

Krystal moves towards ‘full integration’ with gene therapy facility opening

Krystal opens cGMP facility for the clinical and commercial manufacture of its lead drug candidate, KB103, and plans to open its second in 2020.

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March 4, 2019 | Alkahest

As the FDA warns against ‘young blood,’ an ambitious Silicon Valley startup tests a cocktail for Alzheimer’s

The executives at Alkahest have heard plenty of vampire jokes. They are, after all, turning to blood from young donors in search of an elixir for diseases associated with aging. And yes, they’re in Silicon Valley. But this five-year-old biotech company led by Genentech alums is doing serious science.

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March 4, 2019 | ProQR

RNA EDITING – New Editing Technology Enables Body to Repair its Own RNA

An in-house novel RNA-editing technology called Axiomer® may be the treatment answer for people with rare genetic conditions. Axiomer utilizes a person’s own RNA repair mechanism. It can be used to attract this mechanism to a specific mutation in the RNA, where it alters a single nucleotide to repair the mutation and restore the broken or missing protein.

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February 28, 2019 | Abpro

Abpro, NJCTTQ To Develop Bispecific Antibodies in Immuno-oncology

Antibody company Abpro Corp. (Woburn, Mass.) has partnered with Nanjing Chia Tai Tianqing Pharmaceutical Co. Ltd. (NJCTTQ) to use Abpro’s DiversImmune antibody discovery platform to develop multiple bispecific antibodies, including T cell engagers, to treat cancer.

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February 28, 2019 | Abpro

Biotech Bispecifics player Abpro teams up with China’s NJCTTQ in biobucks deal worth up to $4B

Abpro is partnering with China-based NJCTTQ to develop new bispecific antibodies using its antibody discovery platform. Under the agreement, Abpro could collect up to $4 billion, which includes $60 million in “near-term R&D funding,” as well as milestone payments and royalties.

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February 25, 2019 | Orchard Therapeutics

Orchard Achieves Good Survival with Cell Therapy for Rare Immune Deficiency

The patients had a genetic disease called adenosine deaminase–deficient severe combined immune deficiency (ADA-SCID) — also known as bubble boy syndrome. Caused by a mutation on a vital gene, the disease weakens the immune system to the point where patients are very vulnerable to day-to-day infections.

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February 21, 2019 | Pluristem

Brainstorm Health: Stem Cells in Space, CVS Stock Plunge, Merck Setback

Israel-based Pluristem Therapeutics is hoping to do via a collaboration with NASA. The biotech, which focuses on developing stem cell therapy products, is partnering with the space agency’s Ames Research center to see whether or not its experimental PLX cell therapies could help treat and prevent various medical conditions that may arise during space missions, according to a press release.

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February 20, 2019 | Pluristem

Stem cell therapies for space missions? Pluristem joins forces with NASA to combat space-related ailments

When in space, your inner machinery can go awry — ask astronaut Scott Kelly. The assault of protracted radiation and microgravity can culminate in progressive bone and muscle loss and vex the immune system. Placenta-based stem cells may be the answer to limiting that damage, and Israel’s Pluristem is working with NASA to develop such an antidote.

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February 15, 2019 | Orchard Therapeutics

Orchard Looks toward Harvesting Expanded Gene Therapy Pipeline – Global Genes

It was about ten months ago that Orchard Therapeutics acquired the gene therapy portfolio of GSK, expanding its pipeline and giving it its first approved therapy. We spoke to Orchard CEO Mark Rothera about his company’s approach to gene therapy, its pipeline, and what he sees as the biggest challenges as Orchard moves towards commercializing

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February 8, 2019 | ImmusanT

Bring on the gluten: Vaccine offers celiac relief

NEW YORK (NBC News) A peek inside the Friednman family’s kitchen leaves one seeing double. There are two microwaves, two toasters and two different pantries. It’s how the family separates the family’s meals from the gluten-free foods necessary for their 15-year-old son Tyler, who has celiac disease. “He can go in there and get a snack and not have to read every label and worry about things,” Wendy Friedman says.

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January 31, 2019 | DalCor

The Blockbuster Is Dead. Long Live the Blockbuster!

“The definition of blockbuster is changing,” Fouzia Laghrissi-Thode, MD, chief executive officer of DalCor Pharmaceuticals, tells GEN. “Now it’s not necessarily just a dollar sign [in front of a revenue figure] that determines a blockbuster.” As drug development and regulatory reviews become more efficient, she suggests, lesser revenues can generate blockbuster-scale returns on investment.

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January 31, 2019 | OncoCyte

OncoCyte soars after promising results from its cancer detection tests

Biotech company OncoCyte is using a liquid biopsy to detect lung cancer that is showing promising results. Yahoo Finance’s Alexis Christoforous speaks to William Annett, the CEO of Oncocyte.

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January 30, 2019 | Celyad

These pricey new cancer therapies are custom-made. Can they ever be more mainstream?

It’s the ultimate example of personalized medicine — for some cancer patients who have run out of options, scientists can extract their immune cells and genetically re-engineer them to fight against the tumors.

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January 29, 2019 | OncoCyte

Liquid biopsy maker OncoCyte shares skyrocket on validation data

A set of positive results validating OncoCyte’s liquid biopsy test has bolstered its stock $OCX, up 221% at press time. DetermaVu scored on both sensitivity — capacity to detect true positives — and specificity, the ability to snuff out negatives, in a blinded trial that involved 250 blood samples from lung cancer patients.

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January 29, 2019 | OncoCyte

Tiny Health Stock Triples After Lung Cancer Test Wins Wall Street Praise

Micro-cap diagnostic company OncoCyte Corp tripled intraday after announcing break-through results for its lung cancer test that won praise from Wall Street. The company’s R&D validation study of DetermaVu, a non-invasive blood test, “significantly outperforms any other previous blinded study of its kind and signifies the first blood based test on the market to use strictly biomarkers for cancer detection,” Janney analyst Paul Knight proclaimed in a note upgrading his rating of shares to buy from neutral.

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January 18, 2019 | Isofol

Isofol to the rescue in mCRC as folate-based candidate seeks to boost SOC

Isofol Medical AB, a small Swedish company founded a decade ago, is seeking to improve the standard of care (SOC) for metastatic colorectal cancer (mCRC), an oncology indication that receives far less attention than metastatic forms of more prevalent breast, prostate and lung cancers, said CEO Anders Rabbe.

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January 17, 2019 | Alkahest

The CEO of a startup aimed at harnessing the benefits of young blood shares his real plan to beat aging

Instead of opening up a clinic to perform transfusions of young blood, Alkahest researchers aim to develop drugs for age-related diseases which are inspired by their work with plasma.

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January 16, 2019 | SQZ Biotech

Drug companies chase deals for cell and gene therapies, biotech’s ‘next wave’

Meanwhile, Novartis’s crosstown rival Roche Holding AG has enlisted privately held SQZ for up to $1 billion in milestone payments. SQZ created a device that constricts cells to the point where small holes are formed in their outer membranes, allowing genetic material to pass through. The powered-up cells are then able to recognize antigens on a cancer cell, prompting an immune response.

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January 16, 2019 | Valneva

Valneva looking to buy small, overlooked vaccines from Big Pharma: CEO

Vaccine maker Valneva has some experience pumping up a Big Pharma also-ran, and now it’s looking for more.

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January 10, 2019 | NeuBase Therapeutics

For tiny biotechs, J.P. Morgan is a big opportunity — and a big cost

The company, which is working on drugs based on antisense oligonucleotides to treat genetic conditions, announced a reverse merger with Ohr Pharmaceutical on Jan. 3, which would allow its stock to trade publicly.

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January 3, 2019 | NeuBase Therapeutics

Gene silencing company NeuBase Therapeutics to reverse merge its way onto Nasdaq via troubled microcap Ohr

Dietrich Stephan, one of the pioneers in the field of precision medicine, was in process of raising venture funds for his gene-silencing company NeuBase Therapeutics when he was introduced to the team at the struggling eye drug developer Ohr Pharmaceutical $OHRP.

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January 1, 2019 | Columbia Care

CDB, THC In Medical Marijuana Seen As New Tool In Fight Against Opioid Addiction

CBD oil falls into a gray area that might be legal in some states now because it is not psychoactive.

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January 1, 2019 | ImmusanT

Modifying the Immune Response in Celiac

Leslie Williams, CEO of ImmusanT, discusses the company’s research for developing a disease-modifying therapeutic for patients with celiac disease.

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December 14, 2018 | Check-Cap

Colorectal Cancer Screening Pill to Be in US Pilot Study

Check-Cap received CE mark for its C-Scan Capsule in January of this year, now the Isfiya, Israel-based company is setting its sights on the U.S. market.

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December 13, 2018 | Redhill Biopharma

H. pylori drug achieves positive results in phase 3 study

Talicia, a novel drug for the treatment of Helicobacter pylori, achieved positive top-line results in a phase 3 study, according to a press release from RedHill Biopharma, the manufacturer.

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December 13, 2018 | Orchard Therapeutics

Orchard Therapeutics to Build Out Gene Therapy Manufacturing Site in Fremont, CA

Orchard Therapeutics said it will build out a gene therapy manufacturing facility in Fremont, CA, that will employ 100 people and expand its manufacturing capabilities to support plans for an expanded pipeline.

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December 13, 2018 | Check-Cap

Check-Cap wins conditional IDE approval for C-Scan pilot study

Check-Cap (NSDQ:CHEK) said today it won conditional approval from the FDA for its investigational device exemption application to launch a pilot study of its C-Scan system. The Israel-based company’s C-Scan system is designed as an alternative to standard colon cancer screening methods.

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December 13, 2018 | Orchard Therapeutics

Orchard Expects to Hire 100, Expands Gene Therapy Manufacturing in Bay Area

Orchard Therapeutics is expanding its physical footprint in California. The U.K.-based company signed a long-term lease to build out a gene therapy manufacturing facility in Fremont, Calif. The 150,000-square-foot facility will add to the company’s presence in the Bay Area.

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December 11, 2018 | Oxurion

Robust Pipeline for Treatments for Diabetic Eye Disease with Dr. Patrik De Haes Oxurion

Dr. Patrik De Haes, CEO, Oxurion discusses the need for new treatments for diabetic eye disease, a leading cause of blindness in people of working age worldwide.

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December 11, 2018 | Brainsway

Transcranial Magnetic Brain Stimulation to Treat Depression and OCD: Interview with Stanford’s Dr. Nolan Williams

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December 7, 2018 | Orchard Therapeutics

Orchard’s Gene Therapy for Rare Immune Disease Works for Over a Year

A single dose of a gene therapy developed by Orchard Therapeutics can restore immune function for at least a year in patients with X-linked chronic granulomatous disease (X-CGD).

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December 6, 2018 | Alkahest

Alkahest doses first patient in Phase II Parkinson’s drug trial

Alkahest has dosed the first subject in a Phase II clinical trial of GRF6021 to treat Parkinson’s disease associated with mild cognitive impairment or dementia.

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December 3, 2018 | Oxurion

Novel treatments in pipeline for diabetic eye disease

Patrik De Haes, MD, CEO of Oxurion NV, shares the latest updates in clinical studies at the OIS@AAO meeting in Chicago, Oct. 2018.

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December 3, 2018 | Redhill Biopharma

Redhill Planning NDA For H. Pylori Antibiotic Following Second Phase III Readout

RedHill Biopharma Ltd. plans to submit an NDA to FDA next half for Talicia (RHB-105) as first-line treatment of Helicobacter pylori infection regardless of ulcer status after reporting that the antibiotic met the primary endpoint in the Phase III ERADICATE Hp2 trial in the indication.

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November 22, 2018 | Valneva

Valneva/Emergent BioSolutions’ Candidate Zika Vaccine Shows Early Clinical Promise

A European/US company collaboration has reported encouraging safety and immunogenicity in early clinical studies with an alum-adjuvanted Zika vaccine based on inactivated whole virus.

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November 20, 2018 | ArQule

Overcome The Challenges Of An Ultra-Rare Disease Trial

ArQule is a personalized medicine company working in the oncology space to develop treatments that target specific mutations. The Massachusetts-based company faces many challenges in this space, including one that is common to many cancer treatments.

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November 20, 2018 | Phase I Gives Hope for First Zika Vaccine

Phase I Gives Hope for First Zika Vaccine

Results from a Phase I trial showed promising safety and efficacy of a potential first vaccine against the mosquito-borne Zika virus, developed by French biotech Valneva and US biopharma Emergent BioSolutions.

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November 19, 2018 | SQZ Biotech

SQZ Biotech pushes forward with its method to squeeze drugs into cells

One Watertown, Mass.-based biotech is working to capitalize on growing momentum for cell and gene therapies — and it seems to have found its opening. The company is working on a technology that will squeeze cells to open up tiny pores in their membranes to deliver gene therapies or medicines straight into the cell. And SQZ has had a busy fall.

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November 19, 2018 | Oxurion

Oxurion’s CEO Dr. Patrik De Haes discusses three pipeline candidates for diabetic eye disease

Patrik De Haes, MD, CEO of Oxurion, discusses the company’s three disease-modifying drug candidates for the treatment of diabetic eye disease with EyeWire News at the Ophthalmology Innovation Summit in Chicago.

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November 15, 2018 | Arena Pharmaceuticals

Arena bags big deal with United for PAH drug ralinepag

Arena’s run of positive news continues, with a massive $800 million upfront licensing deal with United Therapeutics for its pulmonary arterial hypertension drug ralinepag. The global deal, which also includes $400 million in milestones and double-digit royalties, comes on the heels of well-regarded phase 2 data with the oral prostacyclin receptor agonist in PAH that caused Arena’s shares to soar when it was released last year.

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November 15, 2018 | Columbia Care

Could Pot Help Solve The Opioid Crisis?

A $238,000 federal study will follow more than 10,000 medical marijuana patients in New York over the next two years to see if their opioid use drops. Scott Jordan never thought weed would help him kick the painkillers he took for a bad back. “I smoked pot in high school,” he told BuzzFeed News. “I didn’t really believe medical marijuana was going to help me.”

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November 15, 2018 | Arena Pharmaceuticals

Arena Pharma soars on deal with United Therapeutics for hypertension drug

Shares of Arena Pharmaceuticals Inc jumped nearly 30 percent after the drug developer sold rights to develop and sell its hypertension treatment to United Therapeutics Corp, fetching it $800 million in an upfront payment. Arena Pharma said the money will help it prepare for the eventual launch of its bowel disorder drugs.

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November 15, 2018 | Arena Pharmaceuticals

United Therapeutics pays $800M-plus to grab Arena’s PhIII PAH drug

United Therapeutics $UTHR is paying an $800 million cash upfront in a big bet to stay among the leaders in pulmonary arterial hypertension, grabbing rights to Arena Pharmaceuticals’ ralinepag in the deal. And the executive crew is willing to add hundreds of millions more in milestones if they can stay on track through to an approval and commercialization.

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November 14, 2018 | Orasis Pharmaceuticals

CSF-1 drops demonstrate efficacy, safety in clinical studies for treatment of presbyopia

Elad Kedar, CEO of Orasis Pharmaceuticals, provides a company update at OIS@AAO 2018 in Chicago.

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November 12, 2018 | Quantum Genomics

Quantum Genomics’ brain-targeting blood pressure drug meets phase 2b endpoints

Quantum Genomics’ blood pressure drug firibastat, an inhibitor that targets aminopeptidase A in the brain, met its primary endpoint in a phase 2b study—which the company says paves the way for a wider phase 3 trial in more resistant hypertension.

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November 9, 2018 | Samumed

Wnt is back in drugmakers’ sights, but is it druggable?

See author Cormac Sheridan’s discussion of the biotech industry members developing therapeutics based on targeting the Wnt pathway in this issue of Nature Biotech. Included in the report is Samumed, a company developing a Wnt-targeted therapy for the treatment of Osteoarthritis and other prevalent diseases.

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November 7, 2018 | Samumed

Clinical Challenges: Can OA Be Stopped?

A small-molecule intra-articular Wnt pathway inhibitor, SM04690, is being developed and the results of a phase IIb trial were presented at the ACR meeting by Yusuf Yazici, MD, of NYU Langone School of Medicine in New York City and Samumed, LLC, in San Diego.

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November 7, 2018 | Foamix

Buoyed By Phase III Rosacea Success, Foamix Plans Commercial Transition

Market of dermatologists prescribing for rosacea and acne is very focused, so Foamix says it can manage commercialization of topical minocycline foam on its own.

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November 7, 2018 | Oxurion

Oxurion introduces new clinical portfolio at OIS

CHICAGO: At the Ophthalmology Innovation Summit at AAO, Patrik De Haes, MDCEO of Oxurion, introduces the company’s new clinical portfolio with three separate molecules in clinical trials for the treatment of diabetic macular edema.

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November 7, 2018 | Celyad

Going Public: A Guide for European Biotechs

“For Celyad, the sequence of listing first in Europe and then in the US allowed the company to first strengthen and solidify its company and clinical roots in Europe, and then broaden its investor base and support new programs by listing in the US,” Filippo Petti, CFO of Celyad, told me.

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November 7, 2018 | OSE Immunotherapeutics

Developing Therapies to Fight Cancer and Autoimmune Diseases

We recently spoke to Alexis Peyroles, CEO at OSE Immunotherapeutics to hear more about the findings of their recent study, published in Nature Communications, describing, for the first time ever, the in vivo mechanism of action of interleukin 7 receptor alpha (IL-7Rα) monoclonal antibodies, suggesting therapeutic efficacy in controlling chronic inflammation.

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November 6, 2018 | Oxurion

Oxurion and Beta Therapeutics To Develop New Heparanase Inhibitors for Treatment of dry AMD

Oxurion announced that it entered into a strategic research collaboration with Beta Therapeutics (Canberra, Australia) to develop new heparanase inhibitors for the treatment of retinal disorders with large unmet medical needs such as dry age-related macular degeneration.

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November 2, 2018 | Zosano Pharma

Researchers test patch for migraine relief

Researchers are testing a new transdermal patch from Zosano Pharma that could deliver treatment for migraines.

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October 31, 2018 | Columbia Care

Morning Rounds – Pilot study finds medical cannabis tied to lower opioid use in chronic nerve pain patients

Taking medical cannabis led 62 percent of patients with chronic nerve pain to cut down or stop their opioid use, according to a first-of-its-kind pilot study that tracked 76 individuals over nine months.

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October 29, 2018 | ProQR

ProQR licenses Ionis’ RNA drug for inherited blindness

ProQR is licensing an RNA medicine from Ionis Pharmaceuticals for the treatment of retinitis pigmentosa, a form of rare, inherited blindness. The plan is to start a phase 1/2 trial of the drug in 2019.

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October 28, 2018 | Oxurion

Patrik De Haes, MD: What’s Coming Down the Retina Pipeline

At the 2018 American Academy of Ophthalmology Annual Meeting in Chicago, IL, Patrik De Haes, MD, CEO of Oxurion (formerly ThromboGenics) spoke about several investigational molecules his company is investigating for various retina conditions.

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October 22, 2018 | Columbia Care

Expansion of Medical Marijuana Legalization

Columbia Care is the largest multi-state medical cannabis operator in the United States. With a total of 33 licenses in 13 states, the company is looking to make an impact in the global cannabis industry by partnering with Canaccord Genuity Growth. Nicholas Vita, CEO and co-founder of Columbia Care, joins Cheddar to discuss.

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October 16, 2018 | Columbia Care

Columbia Care CEO on Canada cannabis legalization

Nicholas Vita, Columbia Care CEO, discusses how Canada’s legalization of recreational marijuana on Wednesday will affect the medicinal use market and how it may influence U.S.-based regulations.

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October 15, 2018 | Krystal Biotech

Krystal Gains on Early Data for Topical Gene Therapy in Skin Blistering Disease

Krystal Biotech Inc. (NASDAQ:KRYS) added $4.32 (28%) to $20 on Monday when it reported interim data from the Phase I/II GEM trial showing that topical gene therapy KB103 met all primary efficacy and safety endpoints in two adults with recessive dystrophic epidermolysis bullosa.

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October 15, 2018 | Krystal Biotech

This Biotech Has A Topical Gene Therapy — And Just Flew 30% On A Test

Krystal Biotech (KRYS) rocketed to a record high Monday after its gene therapy showed promise as a skin disease treatment. In midday trading on the stock market today, Krystal stock soared 30.9%, near 20.50. Earlier, Krystal popped as much as 37.6% in higher-than-average volume. Krystal stock began trading around 10 in September 2017.

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October 11, 2018 | OSE Immunotherapeutics

FR104 offers control of graft-versus-host disease

In a game-changing preclinical study targeted toward controlling graft-versus-host disease (GvHD), OSE Immunotherapeutics SA has shown new efficacy results evaluating FR104 and whether a transplant takes. This study, announced Aug. 22, showed strong evidence in the value of targeting CD28 in GvHD for potential clinical applications in autoimmune diseases and transplantation.

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October 4, 2018 | Brainsway

New Treatment for Obsessive-Compulsive Disorder with Dr. Nolan Williams

Dr. Nolan Williams, Clinical Assistant Professor of Psychiatry and Behavioral Sciences at Stanford University. We speak about new treatment options for use in treatment-resistant patients with OCD, including brain stimulation. A deep TMS device was recently FDA approved for the treatment of treatment-resistant OCD.

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October 4, 2018 | Arrowhead Pharmaceuticals

J&J hands Arrowhead a plum $3.7B deal for hep B-plus 3 — corralling a possible game-changing RNAi drug

Two years after Arrowhead Pharmaceuticals $ARWR conceded that a substantial segment of their RNAi pipeline was hopelessly compromised and had to be jettisoned, crippling their stock price, the biotech is staging a major comeback.

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September 28, 2018 | Arena Pharmaceuticals

On A Roll: Arena Continues Streak Of Positive Data With Small Crohn’s Pain Study

Arena announced encouraging data from a small Phase IIa study for oloranib (APD371) in abdominal pain associated with Crohn’s disease as the company continues a streak of good news after two of its more advanced clinical-stage programs posted positive results.

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September 27, 2018 | Abivax

Twice as Splice

Abivax S.A. (Euronext:ABVX) has positioned its anti-HIV therapy ABX464 to take on inflammatory diseases after discovering the small molecule triggers RNA splicing that both halts HIV viral replication and boosts anti-inflammatory responses in HIV and ulcerative colitis. According to CEO Hartmut Ehrlich, ABX464 binds the human cap binding complex (CBC) and promotes its splicing activity.

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September 25, 2018 | Arena Pharmaceuticals

Biotech Rises On Crohn’s Disease Study For Cannabinoid Drug

Arena Pharmaceuticals (ARNA) jumped early Tuesday on promising data for its non-opioid treatment for Crohn’s disease pain. In morning trading on the stock market today, Arena stock lifted 2%, near 43.70, after earlier rising as much as 3.7%. Shares are consolidating with a buy point at 50.15.

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September 25, 2018 | Reata Pharmaceuticals

Reata’s bardoxolone hits goals in phase 2 kidney disease trial

Reata Pharmaceuticals’ bardoxolone has improved kidney function in patients with IgA nephropathy and type 1 diabetic chronic kidney disease (CKD). The phase 2 trial linked the Nrf2 activator to improvements in estimated glomerular filtration rate (eGFR), a measure of kidney damage.

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September 25, 2018 | Arena Pharmaceuticals

Arena 3 for 3 as Crohn’s drug meets proof-of-concept test

Arena Pharma’s cannabinoid receptor-targeting olorinab could help treat the pain associated with Crohn’s disease, one of the most debilitating symptoms of the inflammatory bowel disorder. Olorinab—a cannabinoid receptor type 2 (CB2) agonist—was able to reduce abdominal pain scores by at least 30% compared to baseline levels in 11 evaluable patients in the phase 2a study, which recruited 14 subjects with mild Crohn’s. Shares in the biotech rose almost 3% in after-hours trading after the results were announced.

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September 20, 2018 | Oxurion

First patient enrolled in Oxurion trial evaluating anti-PIGF treatment

The first patient has been enrolled in a phase 2 open-label multicenter study to evaluate intravitreal THR-317, an anti-PIGF antibody, for the treatment of macular telangiectasia type 1, according to a press release from Oxurion. The study will enroll 10 patients with macular edema caused by macular telangiectasia type 1 (MacTel 1), a rare disease that affects the macula and can lead to vision loss. There is no current effective treatment for MacTel 1.

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September 14, 2018 | Foamix

Foamix Raises $70M Following Phase III Acne Readout

Foamix Pharmaceuticals Ltd. (NASDAQ:FOMX) raised $70 million through the sale of 11.7 million shares at $6 in a follow-on underwritten by BofA Merrill Lynch, Cowen, Barclays and Cantor Fitzgerald. The price is a tiny discount to Foamix’s close of $6.11 on Wednesday, when it proposed the offering after market hours. The company gained $0.19 to $6.30 on Thursday, and priced the offering after market hours.

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September 13, 2018 | Arrowhead Pharmaceuticals

Looking for new mechanisms to cure hepatitis B

A genome editing tie-up between Gilead and Precision Biosciences, and promising early data with Arrowhead’s RNA interference project, are two novel approaches in hep B.

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September 11, 2018 | RedHill Biopharma

Conquering Crohn’s disease

RedHill Biopharma Ltd., which just announced encouraging results in Phase 3 studies, became involved in the development of RHB-104 in 2010 when it acquired the rights to the drug combination that had been developed by Prof. Thomas Borody, an Australian gastroenterologist. RHB-104 was originally developed based on the hypothesis that Crohn’s disease is triggered by an infection in susceptible patients.

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September 6, 2018 | Arrowhead Pharmaceuticals

Arrowhead Shares Pop on Early Clinical Data for Hepatitis B Drug

Arrowhead Pharmaceuticals on Thursday made public select initial data from an early-stage clinical study of a hepatitis B drug it’s developing, sending the company’s stock price (NASDAQ: ARWR) to a four-year high. Arrowhead shared data from a study aimed at evaluating the safety and tolerability of a drug candidate it’s developing to treat chronic hepatitis B virus (HBV). An estimated 257 million people worldwide are living with HBV infection, which can lead to cirrhosis and liver cancer, according to the World Health Organization.

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September 6, 2018 | Arrowhead Pharmaceuticals

Arrowhead’s Third-Generation RNAi Shows Initial Efficacy Against HBV

Arrowhead Pharmaceuticals Inc. (NASDAQ:ARWR) gained $5.29 (38%) to $19.39 Thursday after reporting initial data showing that monthly doses of its third-generation RNAi therapy ARO-HBV reduced circulating HBV surface antigen (HBsAg) in a broad population of patients with chronic HBV infection. The biotech added $465 million in market cap on the data.

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September 6, 2018 | Arrowhead Pharmaceuticals

This Biotech Broke Out Bullishly On Its Experimental Hepatitis B Drug

Arrowhead Pharmaceuticals (ARWR) broke out in bullish fashion Thursday on strong data for a study of its hepatitis B treatment. On the stock market today Arrowhead stock rocketed 37.5%, to 19.39, after earlier flying as much as 58.8% in high volume. Shares touched a four-year high and broke out of a consolidation with a buy point at 17.60.

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September 5, 2018 | ProQR

Here’s Why ProQR Therapeutics N.V. Is Skyrocketing Today

After reporting data from a phase 1/2 clinical trial, shares of ProQR Therapeutics N.V.(NASDAQ:PRQR), a clinical-stage biotech focused on RNA medicines that treat rare genetic diseases, rose 70% as of 12:13 p.m. EDT on Wednesday. ProQR reported results from its phase 1/2 trial that is testing its compound called QR-110 as a hopeful treatment for LCA10, which is a genetic mutation that usually leads to childhood blindness.

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September 5, 2018 | ProQR

ProQR soars as childhood blindness drug succeeds in early trial

Shares of ProQR Therapeutics NV surged 70 percent in premarket trading on Tuesday after an interim analysis of an early-stage trial showed that its experimental treatment for a rare form of childhood blindness improved vision. The Dutch drug developer said it would now stop enrollments for the small study and progress to a mid-stage trial, which is expected to start in the first half of 2019.

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September 5, 2018 | Valneva

Lyme & Reason: Battles & Breakthroughs Against Lyme Disease

It’s a battle that has been raging for decades – the battle against Lyme Disease. Over the years, progress has been made against this dreaded tick-borne illness. But with breakthroughs come even more barriers. Watch this early peek at a new Fox 5 News special on the fight that could be signaling a new chapter in the ongoing bout against Lyme.

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September 4, 2018 | Check-Cap

Check-Cap touts interim post-CE Mark C-Scan study results

Check-Cap (NSDQ:CHEK) today released interim results from the post-CE Mark approval study of its C-Scan system version 3, touting the device’s ability to detect polyps in un-prepped colons.

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September 4, 2018 | Check-Cap

Check-Cap Announces Interim Results of European Study of C-Scan System Version 3

Check-Cap Ltd. announced the interim results for its post-CE approval study of the C-Scan system Version 3, an ingestible, capsule-based device for preparation-free colorectal cancer (CRC) screening. The company said data from the multicenter clinical investigation showed promising results for detecting patients with polyps in an un-prepped colon.

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September 4, 2018 | Abivax

Abivax posts ulcerative colitis data, commits to phase 2b

Abivax has posted data from a phase 2a trial in patients with moderate-to-severe ulcerative colitis that are refractory to anti-TNF antibodies or corticosteroids. The trial linked ABX464 to improvements in mucosal healing and performance on a symptom scale, leading Abivax to commit to a phase 2b.

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September 4, 2018 | Red Hill Biopharma

RedHill Biopharma: transforming treatments for gastrointestinal diseases

GI disease-focused RedHill Biopharma wants to radically improve the treatment options for these types of disorders using antibiotic combination therapy. RedHill COO Gilead Raday discusses the pipeline, starting to focus on other therapeutic areas in addition to the GI space and how the company is dealing with challenges facing the biopharma industry.

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August 23, 2018 | Krystal Biotech

FDA Grants Rare Pediatric Disease Designation to KB105 for TGM-1 Deficient ARCI

This morning, August 23, 2018, the US Food and Drug Administration (FDA) granted a rare pediatric disease designation to Krystal Biotech’s KB105 for the treatment of patients with TGM-1-deficient autosomal recessive congenital ichthyosis (ARCI).

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August 16, 2018 | Catalyst Biosciences

Catalyst gives positive hemophilia update as 2 more subjects complete study

Two additional subjects completed Catalyst Biosciences’ ongoing hemophilia Phase 2/3 trial without any bleeding or developing any anti-drug antibodies (ADAs), the company said in a statement Wednesday.

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August 14, 2018 | Valneva

Lyme Disease Is Spreading Fast. Why Isn’t There a Vaccine?

The only vaccine was taken off the market more than 15 years ago. As reports of the disease skyrocket, researchers are working on a new one.

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August 6, 2018 | Samumed

Samumed raises $438 million for regenerative therapies

Samumed, LLC has raised $438 million, the San Diego regenerative medicine company said Monday. The new financing round brings the total to more than $650 million since Samumed’s founding, valuing the company at $12 billion, the privately held company said.

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August 6, 2018 | Reata Pharmaceuticals

Experimental Drug Keeps Father Of Two From Needing Kidney Transplant

“We’re studying a medicine called bardoxolone methyl. It’s made by a company called Reata,” explained Block. Hinerfeld was the first person with Alport Syndrome to try bardoxolone. Block said, after 12 weeks, Hinerfeld’s kidney function stopped declining and actually improved.

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August 2, 2018 | Celtaxys

Celtaxsys cystic fibrosis drug reduces key symptom in mid-stage study

Privately held Celtaxsys Inc said on Thursday a mid-stage trial testing its experimental cystic fibrosis treatment was successful in reducing a key symptom of the genetic lung disease, but did not improve lung function.

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August 2, 2018 | Celtaxsys

Celtaxsys’ cystic fibrosis drug reduces lung exacerbations in phase 2 study

Celtaxsys’ oral anti-inflammatory drug for cystic fibrosis, acebilustat, cleared a phase 2 study by demonstrating meaningful improvements in pulmonary exacerbations, or the acute worsening of symptoms such as heavy coughing or shortness of breath, combined with sharp decreases in lung function following irreversible organ damage.

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July 24, 2018 | ProQR

First Human Trial to Evaluate QR-313 for Dystrophic Epidermolysis Bullosa Initiated

While there are currently no approved treatments for the rare skin disease, there are therapies in development, such as ProQR’s RNA therapy, QR-313, which is now being evaluated in its first human clinical trial, dubbed “WINGS.”

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July 23, 2018 | Reata Pharmaceuticals

This Biotech Stock Rocketed 40% On Kidney Disease Drug Study

Reata Pharmaceuticals (RETA) skyrocketed to a record high Monday after its experimental drug crushed expectations in two groups of chronic kidney disease patients. Reata said its drug known as bardoxolone methyl benefited patients with what’s known as Alport syndrome and autosomal dominant orphan kidney disease.

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July 13, 2018 | Arrowhead Pharmaceuticals

Arrowhead Takes Flight

Shares of Pasadena’s Arrowhead Pharmaceuticals Inc. take flight.

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July 12, 2018 | Zosano

Alan Rapoport, MD

At the American Headache Society’s 60th Annual Scientific Meeting, Dr. Alan Rapoport summarizes a poster he presented regarding ZOTRIP, Zosano Pharma’s zolmitriptan patch.

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July 9, 2018 | Valneva

New Effort for Lyme Disease Vaccine Draws Early Fire

In March, Valneva SE , a company based in France, announced initial phase-one clinical trial results after testing its proposed vaccine in 180 healthy adults who took the vaccine with no serious side effects. The study also showed that the vaccine stimulated an immune response, says David Lawrence, chief financial officer of the company.

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June 28, 2018 | Arrowhead Pharmaceuticals

The 3 Best Biotech Stocks of 2018 So Far

Arrowhead Pharmaceuticals stock has soared more than 260% so far this year. The biotech’s share price marched steadily upward from the get-go in 2018, but Arrowhead really kicked into high gear in May after providing its second-quarter update to investors.

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June 27, 2018 | Columbia Care

Kent County’s First Medical Marijuana Dispensary to Open

Columbia Care is set to open Kent County’s first medical marijuana dispensary on Friday.

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June 7, 2018 | Sensorion

Treatments for the Growing Problem of Hearing Loss – Sensorion’s Nawal Ouzren

Listen up! People are starting to hear, pay attention to, and react to the growing problem of hearing loss and other conditions of the inner ear. Nawal Ouzren explains how her company, Sensorion, is working to address the issue.
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June 7, 2018 | Brainstorm Cell Therapeutics

Brainstorm Considering Right To Try For ALS Therapy

BrainStorm Cell Therapeutics Inc. (NASDAQ:BCLI) is considering offering NurOwn to treat amyotrophic lateral sclerosis under the recently enacted federal right-to-try law, BrainStorm President and CEO Chaim Lebovits told BioCentury.

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June 1, 2018 | Sensorion

Companies to Watch: Sensorion

Sensorion is a public company in France developing new medicines for diseases of the inner ear, with two compounds in the pipeline: SENS- 111 for treating acute unilateral vestibulopathy…

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May 31, 2018 | Sensorion

Is Hearing Loss a Necessary Price for Children to Pay for Life-Saving Chemotherapies

We spoke to Nawal Ouzren, CEO of Sensorion, which will participate in the FDA meeting and is developing a treatment for sudden hearing loss that may be able to help pediatric cancer patients experiencing cisplatin-induced ototoxicity.

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May 25, 2018 | ProQR

Daniel de Boer, CEO of ProQR, Explains RNA Treatment, QR-313, for DEB

In a recent interview with Daniel de Boer, CEO of ProQR, Rare Disease Report® discussed the company’s new RNA therapy, QR-313, for the treatment of dystrophic epidermolysis bullosa (DEB).

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May 21, 2018 | Rain Therapeutics

Cancer Treatment Developer Rain Therapeutics Closes $18.4 Million Series A

Rain Therapeutics Inc., a Fremont, Calif.-based developer of small molecule therapeutics for patients with cancer, has secured $18.4 million in Series A funding. Biotechnology Value Fund led the round, with participation from investors including Perceptive Advisors and Auckland UniServices Ltd.’s Inventors Fund.

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May 21, 2018 | Rain Therapeutics

Rain Therapeutics nets $18M to push lung cancer asset into phase 2

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May 11, 2018 | Catalyst Biosciences

The Case for a Daily Self-Injection to Treat Hemophilia

We spoke to Nassim Usman, CEO of Catalyst, about the company’s approach to hemophilia, how its drugs work, and why he believes his company’s pipeline may provide better alternatives for hemophilia patients.

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May 3, 2018 | Samumed

Samumed CMO Hopes To Reach Drug Approvals Via The Wnt Pathway

The clinical-stage biotech has diverse pipeline generated by its Wnt signalling pathway technology platform, and aims to get its first drug to market in 2020.

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April 19, 2018 | Celyad

“This is the First Time CAR-T Has Ever Worked on Acute Myeloid Leukemia“

Celyad has reported clinical results showing the first time a patient has fully recovered from acute myeloid leukemia thanks to a CAR-T therapy, and without the need for chemotherapy preconditioning.

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April 19, 2018 | Arena Pharmaceuticals

Bloomberg Markets: ICO Compliance, Drug Development

Amit Munshi President/CEO Arena Pharmaceuticals Inc Discussing drug development in ulcerative colitis space.

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April 17, 2018 | Arena Pharmaceuticals

Arena CEO: We have two great assets and are trying to build a company around that

Looking at the performance of Arena Pharmaceuticals with CEO Amit Munshi and CNBC’s Meg Tirrell.

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April 12, 2018 | Orchard Therapeutics

Orchard Gains GSK’S Rare Disease Gene Therapy Portfolio

GlaxoSmithKline plc (LSE:GSK; NYSE:GSK) said it will divest its rare disease gene therapy portfolio, including Strimvelis, to Orchard Therapeutics Ltd. (London, U.K.).

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April 12, 2018 | Orchard Therapeutics

UK gene therapy firm Orchard plans stock offer after GSK deal

Britain’s Orchard Therapeutics, which has already raised more than $140 million to fund its work in gene therapy, plans another private sale of shares following its acquisition of a portfolio of GlaxoSmithKline…

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April 9, 2018 | OSE Immunotherapeutics

OSE Immunotherapeutics Adds Boehringer Ingelheim To Its Pharma Partnering List

Collaborating with the German family-controlled drug maker on OSE’s novel checkpoint inhibitor aligns with French firm’s strategy of partnering its portfolio candidates.

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April 6, 2018 | Reata Pharmaceuticals

Reata Pharmaceuticals: Return & Reach Beyond

The Enterprisers: Life Science Leadership In Action. What good can come of setback? What is the value, if any, in so-called failure? Must a “failed” clinical trial wind up in the great waste bin of misbegotten science?

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April 5, 2018 | BrainStorm Cell Therapeutics

Trials for potential ALS treatment spark cautious hope

As an engineering professor at Lehigh University in Pennsylvania, Richard P. Vinci has run plenty of experiments. He specializes in figuring out what makes materials break…

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April 4, 2018 | OSE Immunotherapeutics

Boehringer, OSE tie up in I-O deal that could total more than $1.4B

Boehringer Ingelheim and OSE Immunotherapeutics are partnering to develop the latter’s checkpoint inhibitor, OSE-172, for the treatment of advanced solid tumors.

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April 4, 2018 | BrainStorm Cell Therapeutics

ALS patient hopes stem cell treatment will be “a powerful weapon”

Forty-one-year-old David Neufeglise was a healthy family man and mechanical engineer when he started noticing constant twitching in his arm…

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March 15, 2018 | Arrowhead Pharmaceuticals

RNAi Therapeutics: To the Liver … and Beyond

RNAi Therapy Components Are Surviving Degradation, Staying Potent, and Reaching More Targets Than Ever

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