SonoThera will Present New Data of Genetic Medicine Using Targeted, Ultrasound-Mediated Delivery (UMD) in Treating Duchenne Muscular Dystrophy
SonoThera will present new data at the 2025 MDA Conference demonstrating the potential of genetic medicine using targeted, Ultrasound-Mediated Delivery (UMD) in treating Duchenne...
Solid Biosciences Plans to Request a Meeting with the FDA in mid-2025 to Discuss Potential Accelerated Approval Pathways for SGT-003
Solid Biosciences, a life sciences company developing precision, next generation, genetic medicines for neuromuscular and cardiac diseases, reported financial results for the fourth quarter...
GenAssist Announces FDA Approval of New Drug Application for GEN6050X Exon 50 Skipping Therapy
On March 06, 2025, GenAssist Ltd (GenAssist), a pioneering gene-editing biotechnology company specializing in genome medicines, is thrilled to announce that it has received...
PepGen Temporarily Pauses Exon 51 Skipping Study
PepGen, a clinical-stage biotechnology company advancing the next generation of oligonucleotide therapies with the goal of transforming the treatment of severe neuromuscular and neurological...
Capricor Therapeutics Announces FDA Acceptance and Priority Review of its Biologics License Application for Deramiocel to Treat Duchenne Muscular Dystrophy
Capricor Therapeutics, a biotechnology company developing transformative cell and exosome-based therapeutics for the treatment of rare diseases, today announced the U.S. Food and Drug...