Clients in the Media

/Clients in the Media

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 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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