Sarepta Reported Elevidys Gene Therapy for Duchenne Muscular Dystrophy will not be used in non-ambulatory patients
ELEVIDYS (delandistrogene moxeparvovec-rokl), the only approved gene therapy for patients with Duchenne muscular dystrophy, has received a safety update from Sarepta Therapeutics, the leader...
New Positive Functional Data From The RGX-202 Affinity Duchenne® Trial Phase 1/2 Reported By Regenxbio
The Regenxbio press statement stated that the findings, which came from five patients in the AFFINITY DUCHENNE trial who were between the ages of...
PepGen Announces It Will Halt Clinical Trials of Exon 51 Skipping Study
PepGen, a clinical-stage biotechnology company advancing the next generation of oligonucleotide therapies, today announced that based on the levels of dystrophin protein measured in...
Entrada Therapeutics Receives EU Authorization to Start Exon 45 Skipping Clinical Trial in Duchenne Muscular Dystrophy
Entrada Therapeutics, today announced it has received authorization from the Health Authorities and Ethics Committees of multiple countries under the European Union Clinical Trial...
Entos Pharmaceuticals Develops Re-Dosable, Full-Length Dystrophin Gene Therapy: Fusogenix PLV Method
Entos Pharmaceuticals Inc. (Entos) is a biotech business that creates genetic therapies using its non-viral, redosable Fusogenix PLV delivery technology. CureDuchenne Ventures announced an...