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.
Flamingo Therapeutics BV and Ionis Pharmaceuticals Inc.’s FTX-001 (also known as FLM-7523) is a potential first-in-class antisense oligonucleotide (ASO) targeting the human metastasis-associated lung adenocarcinoma transcript 1 (MALAT1) long noncoding RNA (lncRNA); it is being developed for the treatment of cancer.
Geron Corp., a company founded more than three decades ago, is finally celebrating its first U.S. FDA approval. The agency cleared imetelstat, branded Rytelo, for use in transfusion-dependent anemia in adults with low- to intermediate-risk myelodysplastic syndromes, specifically those requiring four or more red blood cell units over eight weeks who have failed or no longer respond to erythropoiesis-stimulating agents (ESA) or who are not eligible for ESA treatment.
Shares of Dyne Therapeutics Inc. (NASDAQ:DYN) closed May 20 at $35.38, up $7.70, or 28%, on word of positive data from the phase I/II Achieve trial of DYNE-101 in myotonic dystrophy type 1 (DM1) and the phase I/II Deliver effort with DYNE-251 in Duchenne muscular dystrophy (DMD) who are amenable to exon 51 skipping. CEO John Cox, who joined Waltham, Mass.-based Dyne eight weeks ago, said he “couldn’t be more proud to be part of this team.” Studies are ongoing, but new data with regard to DM1 as well as DMD showed a “compelling” impact, Dyne said, plus satisfying safety profiles.
The higher of two doses of Ionis Pharmaceuticals Inc.’s drug olezarsen for treating adults with familial chylomicronemia syndrome reduced fasting triglyceride levels by 44% from baseline at the six-month mark in a phase III study, but one case of pancreatitis in the trial paints a less attractive picture than top-line data the firm reported in September.
Updated results from Stoke Therapeutics Inc.’s studies of antisense oligonucleotide STK-001 showed a reduced convulsion seizure frequency in those ages 2 to 18 with Dravet syndrome. The new data, which also showed improved cognition and behavior in participants, substantially boosted the company’s stock value and caused analysts to applaud.
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