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 [...]
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. [...]
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 [...]
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 [...]
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 [...]
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 [...]
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 [...]
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 [...]
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 [...]
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 [...]
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 [...]
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 is in its 'final pivotal study' for its new drug, bardoxolone. In the trials, the drug has showed promising results to reverse kidney damage. Yahoo Finance sits down with the CEO of Reata, Warren Huff, who founded the company in 2002, to discuss the plans to launch the product in the near future. [...]
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, [...]
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 [...]
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 [...]
Nick Vita, CEO and co-founder of medical cannabis company Columbia Care, talks about being acquired by Canaccord Genuity Growth Corp. and what it will allow the company to do.
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 [...]
