EG 427 SAS has received IND clearance from the FDA for EG-110A, a gene therapy for the treatment of neurogenic detrusor overactivity in patients with spinal cord injury. A phase Ib/IIa study is being initiated.
Sarepta Therapeutics Inc. CEO Douglas Ingram said he expects “ferocious” demand for gene therapy Elevidys (delandistrogene moxeparvovec), granted full approval by the U.S. FDA for Duchenne muscular dystrophy (DMD). Shares of the Cambridge, Mass.-based firm closed June 21 at $16.72, up $37.22, or about 30% on the news.
Astellas Pharma US Inc., a U.S. affiliate of Astellas Pharma Inc., has entered into a sponsored research agreement with the University of Massachusetts Medical School (UMass Chan Medical School) to conduct research for an AAV vector-mediated gene therapy for the treatment of Alexander disease.
Opus Genetics Inc. has received $1.7 million in project-based funding from the Foundation Fighting Blindness to help advance two preclinical candidate programs for inherited retinal diseases.
The good news for Sarepta Therapeutics Inc. is bad news for Pfizer Inc. as the phase III study of its mini-dystrophin gene therapy in Duchenne muscular dystrophy (DMD) has missed its primary endpoint. Now Sarepta’s Elevidys (delandistrogene moxeparvovec), a single-dose, adeno-associated virus-based gene transfer therapy for DMD, is barreling toward a June 21 PDUFA date with the U.S. FDA as the near competition shrinks in the rearview mirror.
The FDA has granted orphan drug designation to Be Biopharma Inc.’s BE-101, a novel engineered B-cell medicine being developed for the treatment of hemophilia B.
The EMA has been sent back to the drawing board to re-evaluate PTC Therapeutics Inc.’s Duchenne muscular dystrophy therapy Translarna (ataluren), after failing to get the usual rubber stamp following its recommendation in January that the drug’s conditional approval be withdrawn.
Researchers from Purespring Therapeutics Ltd. and affiliated organizations have presented preclinical data for the adeno-associated vector (AAV) gene therapy PS-001 for the treatment of glomerular disease.
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