Vanda Pharmaceuticals Inc.’s VGT-1849A, a selective antisense oligonucleotide (ASO)-based JAK2 inhibitor, has been awarded orphan drug designation by the FDA for the treatment of polycythemia vera.
Ionis Pharmaceuticals Inc. CEO Brett Monia predicted that diagnoses of familial chylomicronemia syndrome (FCS) will “accelerate fairly quickly” now that the firm has gained U.S. FDA clearance for Tryngolza (olezarsen) as an adjunct to diet to reduce triglycerides in adults with the rare form of severe hypertriglyceridemia (sHTG) that can lead to life-threatening acute pancreatitis (AP).
After missing out on the glucagon-like peptide 1 obesity market, Sanofi SA is prospecting for next-generation drugs and is making a strategic equity investment in Resalis Therapeutics Srl, providing the Italian biotech with funding to take its lead program RES-010 through to phase II.
Duchenne muscular dystrophy is a severe and progressive disorder caused by mutations in the dystrophin (DMD) gene that lead to malfunction or absence of dystrophin. This protein stabilizes the sarcolemma and protects muscle cells during contraction.
As in other muco-obstructive diseases, the airways in cystic fibrosis (CF) are characterized by goblet cell and glandular hyperplasia, with overproduction of mucins MUC5 and MUC5AC, resulting in viscous mucus, respiratory blockade and recurrent infections and inflammation.
A team of scientists from the Perelman School of Medicine at the University of Pennsylvania and Aum Biotech LLC have described the development of a novel cancer immunotherapy designed to target FOXP3-positive T regulatory cells (Tregs) with a next generation of antisense oligonucleotides (ASOs), termed FOXP3 Aumsilence ASO. In contrast to previous ASOs, FOXP3 Aumsilence ASOs do not require delivery agents, and are capable of highly specific RNA silencing of previously undruggable targets.
Acurastem Inc. has secured $4 million in grant funding from the California Institute for Regenerative Medicine (CIRM) to facilitate the development of its UNC13A program toward clinical trials for amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD).
While Dyne Therapeutics Inc.’s DYNE-251 demonstrated high levels of dystrophin expression and functional improvement in boys with Duchenne muscular dystrophy who are amenable to exon 51 skipping, investors focused on three serious adverse events related to the drug, driving shares (NASDAQ:DYNE) down by 31% on Sept. 3.
Pepgen Inc. fell slightly short of its phase II dystrophin goal with PGN-EDO51 for patients with Duchenne muscular dystrophy (DMD) whose mutations are amenable to an exon 51-skipping approach, but Wall Street reacted in a big way, sending the Boston-based firm’s stock (NASDAQ:PEPG) down 33%, or $5.55, to close July 31 at $11.43.
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