Akcea Therapeutics, Inc. (NASDAQ: AKCA), a majority-owned affiliate of Ionis Pharmaceuticals, Inc., and Ionis Pharmaceuticals, Inc. (NASDAQ: IONS), today announced positive topline results from the Phase 2 study of AKCEA-APOCIII-LRx in the treatment of patients with hypertriglyceridemia who are at risk for or have established cardiovascular disease (CVD). The study met the primary endpoint of significant triglyceride lowering and multiple…
Read More
A prevailing dogma is that inhibition of vascular thrombosis by antagonizing platelet integrin αIIbβ3 cannot be achieved without compromising hemostasis, thus causing serious bleeding and increased morbidity and mortality. It is speculated that these adverse outcomes result from drug-induced activating conformational changes in αIIbβ3 but direct proof is lacking. Here, we report the structure-guided design of peptide Hr10 and a…
Read More
One day, a scientist in Craig Ferris’s lab was scanning the brains of very old rats when he found that one could see, hear, smell, and feel just like the other rats, but it was walking around with basically no brain—and likely had been since birth. This rat, named R222, did have a brain. But its brain, affected by a condition called hydrocephalus, had…
Read More
Some cancer treatments don’t work, but the problem isn’t the chemicals in the drugs—it’s the shape of them. This new technology could revitalize these drugs and help them do their job. And it looks like a sea anemone. In this episode, we talk to Ke Zhang (associate professor of chemistry and chemical biology) and Dali Wang (postdoctoral fellow with the Zhang Group).
Read More
Biohaven Pharmaceutical Holding Company Ltd. (NYSE: BHVN) (“Biohaven” or the “Company”), a biopharmaceutical company with multiple late-stage neuroscience drug candidates in development, announced today that the U.S. Food and Drug Administration (FDA) has notified its collaborators at the Sean M. Healey & AMG Center for Amyotrophic Lateral Sclerosis (ALS) at Massachusetts General Hospital (MGH) that they may proceed with clinical investigation of verdiperstat,…
Read More
Genialis, a leader in applied data science for the development of precision medicines, today announced a collaborative partnership with Oncologie, an international, clinical-stage biopharma developing next-generation immunotherapies for cancer. Initial activities will focus on the use of Genialis’ Expressions software platform and analytics IP with Oncologie’s clinical and translational expertise to refine patient stratification methodologies. “We are incredibly excited to work with Genialis…
Read More
Researchers led by a team at Massachusetts Eye and Ear have identified a novel cellular entry factor for adeno-associated virus vector (AAV) types–the most commonly used viral vectors for in vivo gene therapy. AAVs are vectors-or vehicles–that are created from a virus that is made harmless by molecular engineering, and have shown promise transporting genetic therapy treatments to affected tissues.…
Read More
Patient deaths, testing concerns and “weak arguments.” Those are just a few of the issues the FDA had with Sarepta Therapeutics’s recent drug application, according to new documents released this week. The FDA offered a rare peek at its decision-making process Wednesday, releasing documents related to its initial rejection and later approval of Sarepta’s (Nasdaq: SRPT) rare disease drug Vyondys 53. Such documents are…
Read More
Moderna shares are higher on Wednesday after the Cambridge, Mass., drugmaker disclosed that it is working with the National Institutes of Health to develop a vaccine to combat coronavirus. The disease has infected hundreds of people in China. The first U.S. case of coronavirus has been diagnosed in Washington state. “Moderna’s mRNA vaccine technology could serve as a rapid and flexible…
Read More
Empirical and anecdotal evidence has associated stress with accelerated hair greying (formation of unpigmented hairs), but so far there has been little scientific validation of this link. Here we report that, in mice, acute stress leads to hair greying through the fast depletion of melanocyte stem cells. Using a combination of adrenalectomy, denervation, chemogenetics, cell ablation and knockout of the…
Read More