Clients in the Media

 A man who lives in Surprise is helping researchers come up with a treatment for a rare disease that he was diagnosed with 20 years ago. Jerry DeVries, now 61, is possibly the oldest living person with Proteus Syndrome.

Rather than picking targets based on conventional approaches, Immunome is letting a patient‘s ability to fight the disease guide target selection and therapy identification.

We are always acutely aware of investors’ appetite to participate in financing deals, and to what degree. There has been a lot of chatter about the “window closing” in 2020, but from what we can see, financing deals for good science and novel ideas are still getting done, and we’re cautiously optimistic that the environment will remain open to healthy deals at the right valuations.

Eleven drugmakers led by Pfizer and Novartis have set aside a combined $2 billion to invest in gene therapy manufacturing since 2018, according to a Reuters analysis, in a drive to better control production of the world’s priciest medicines. The full scope of Novartis’ (NOVN.S) $500 million plan, revealed to Reuters in an interview with the company’s gene therapy chief, has not been previously disclosed. It is second only to Pfizer (PFE.N), which has allocated $600 million to build its own gene therapy manufacturing plants, according to filings and interviews with industry executives.

There is a great need for transformative treatments that increase life expectancy and improve the quality of life of patients and families living with rare inherited diseases. Current therapeutic approaches, including small molecules and enzyme replacement therapies, have limitations and cannot be applied to every disease. ProQR specializes in the development of RNA therapies and is expanding its toolbox of RNA approaches to develop life-changing medicines for rare genetic disorders that are currently untreatable.

As of Nov. 21, Arrowhead stock is trading at a record high of $49 a share. It has a new slate of drugs that use slightly different molecular technology to silence disease-causing genes and is targeting a range of different diseases. Several of those drugs are now going through clinical trials with promising early results.

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.

The first robotic surgery took place in 1985 when the PUMA 560 was used in a stereotaxic operation in which computed tomography (or x-ray) was used intraoperatively to guide a robot as it inserted a needle into the brain for biopsy. In the late 1980s and early 1990s, robotic systems began to be used for laparoscopic surgery, in which a flexible optical instrument was inserted into the body and used to guide surgeons through hard-to-reach areas, from the pelvis to the chest cavity.

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.

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.

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

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.

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.

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.

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.

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.

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.

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.

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.

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.

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.

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.

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.

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.

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.

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.

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

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).

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.

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.

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.

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.

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.

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.

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.

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.

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.

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

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.

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.

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.

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.

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.

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.

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.

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.

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.

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.

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.

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.

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.

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.

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

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.

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.

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.

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.

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.

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.

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.

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.

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.

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.

OSE Immunotherapeutics S.A. and Boehringer Ingelheim GmbH are testing the SIRPA inhibitor BI 765063 in solid tumors as a monotherapy and in combination with Boehringer’s anti-PD1 mAb BI 754091. OSE Immunotherapeutics CSO Nicolas Poirier told BioCentury BI 765063 overcomes resistance to checkpoint inhibition in preclinical models by modulating macrophage activity and allowing more T cells to enter the tumor microenvironment. He said OSE has patented selective blockade of SIRPA but not SIRPG, which contributes to antitumor T cell responses.

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.

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.

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.

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.

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

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.

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.

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.

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.

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.

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.

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.

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.

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.

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.

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.

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.

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.

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.

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.

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.

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

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.

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.

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.

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

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.

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

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.

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.

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.

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.

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.

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.

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.

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.

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.

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.

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.

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.

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.

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.

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

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.

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.

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.

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.

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.

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.

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.

“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.

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.

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

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.

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.

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.

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.

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.

“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.

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.

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.

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.

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.

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.

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.

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

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.

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.

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

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.

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.

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.

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.

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.

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).

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.

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.

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.

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.

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.

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.

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.

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.”

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.

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.

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.

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.

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.

“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.

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.

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.

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.

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.

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.

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.

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.

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.

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.

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.

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.

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.

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.

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.

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.

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.

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.

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.

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.

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.

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.

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.

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.

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.

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.

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.

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).

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.

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.

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.

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.

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.

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.”

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.

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

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.

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.

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…

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).

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.

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.

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.).

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…

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.

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?

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…

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

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

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