Precision BioSciences Receives FDA Rare Pediatric Disease Designation for PBGENE-DMD for the Treatment of Duchenne Muscular Dystrophy
Precision BioSciences, a clinical stage gene editing company utilizing its novel proprietary ARCUS® platform to develop in vivo gene editing therapies for diseases with...
Capricor Therapeutics Provides Regulatory Update on Deramiocel BLA for Duchenne Muscular Dystrophy
As part of the FDA’s ongoing review, the Company has been informed that an Advisory Committee meeting is not indicated at this time. The...
Cumberland’s Ifetroban Demonstrated a 5.4% Improvement Compared to Placebo-Treated Patients
Cumberland Pharmaceuticals a specialty pharmaceutical company committed to developing new products for rare diseases, shared the latest positive results from its Phase 2 FIGHT...
Capricor Therapeutics Announces Positive 4-Year Data of Deramiocel (CAP-1002) in Duchenne Muscular Dystrophy
Capricor Therapeutics, a biotechnology company developing transformative cell and exosome-based therapeutics for rare diseases, today announced positive four-year safety and efficacy results from its...
Capricor Therapeutics Announces BMD Orphan Drug Designation and DMD Program Regulatory Development for Deramiocel (CAP-1002)
Capricor Therapeutics, a biotechnology company developing transformative cell and exosome-based therapeutics for the treatment of rare diseases, today announced that the U.S. Food and...