Tevard Biosciences Inc. has entered into a 4-year global research collaboration with Vertex Pharmaceuticals Inc. aimed at creating new tRNA-based therapies for patients with Duchenne muscular dystrophy (DMD) caused by nonsense mutations, with options to expand into additional muscular dystrophies and a second indication.
As broader markets struggled throughout 2022, the biopharma industry’s largest and most lucrative companies ended the year in a strong position, according to BioWorld’s Biopharmaceutical Index (BBI), which showed the 22 component stocks climbed by 17.3% throughout the year. Neither the Nasdaq Biotechnology Index (NBI) nor the Dow Jones Industrial Average (DJIA) demonstrated such gains. They were down by 10.9% and 8.8%, respectively. All three indices, however, are following the same path in 2023, shooting up throughout the month of January and back down in recent weeks.
Vertex Pharmaceuticals Inc. has divulged N-(hydroxyalkyl (hetero)aryl) tetrahydrofuran carboxamides acting as sodium channel protein type 10 subunit α (SCN10A; Nav1.8) blockers reported to be useful for the treatment of pain, arrhythmia, Charcot-Marie-Tooth disease, cough, urinary incontinence and multiple sclerosis.
Several recent patents from Vertex Pharmaceuticals Inc. have disclosed sodium channel protein type 10 subunit α (SCN10A; Nav1.8) blockers reported to be useful for the treatment of pain, arrhythmia, Charcot-Marie-tooth disease, cough, urinary incontinence and multiple sclerosis.
Vertex Pharmaceuticals Inc. has received clearance from the FDA for its IND application for VX-522, a messenger ribonucleic acid (mRNA) therapy targeted at treating the underlying cause of cystic fibrosis (CF).
Vertex Pharmaceuticals Inc. and Entrada Therapeutics Inc. have entered into a global collaboration focused on discovering and developing intracellular Endosomal Escape Vehicle (EEV) therapeutics for myotonic dystrophy type 1 (DM1).
An uptick in positive clinical data reported in recent months has helped nudge the BioWorld Biopharmaceutical Index (BBI) to its highest point this year. BBI is up by 15.58%, significantly higher than the 1.36% increase recorded at the end of August, just as statistically significant clinical results began to roll in from a number of biopharma companies.
CRISPR-based cell therapies continued to gain steam Sept. 27 with the announcements of a potentially valuable big pharma collaboration and an ambitious global regulatory push.
Verve Therapeutics Inc. has packed a lot into the past few weeks. The latest is a four-year research deal with Vertex Pharmaceuticals Inc. to find and develop an in vivo gene editing program for an undisclosed liver disease. Vertex will pick up the tab for program costs as Verve does the preclinical R&D. Verve is getting an up-front $60 million from Vertex, along with a $35 million equity investment.
At the European Hematology Association's annual meeting in Vienna last week, companies reported impressive progress for the treatment of sickle cell disease.
