FDA Rejects Rocket Pharmaceuticals’ Gene Therapy for Ultra-Rare Immune Disease, Requests Additional Manufacturing Information

FDA, Rocket Pharmaceuticals, Gene Therapy, Leukocyte Adhesion Deficiency-I (LAD-I), Manufacturing Information, Complete Response Letter

FDA Approves EPKINLY (Epcoritamab-Bysp) for Relapsed or Refractory Follicular Lymphoma

EPKINLY, epcoritamab-bysp, follicular lymphoma, FDA approval, relapsed or refractory, bispecific antibody, T-cell engaging, subcutaneous treatment

Sarepta Therapeutics: Next Steps After Full FDA Approval for Duchenne Muscular Dystrophy Gene Therapy

Sarepta Therapeutics, Duchenne muscular dystrophy, gene therapy, FDA approval, Elevidys, microdystrophin, ambulatory patients, non-ambulatory patients, pipeline development, rare diseases, genetic medicine.

Wave Life Sciences Achieves Breakthrough in Huntington’s Disease Treatment with WVE-003, Eyes Accelerated Approval

Wave Life Sciences, Huntington’s disease, WVE-003, Phase Ib/IIa study, accelerated approval, mutant huntingtin protein, allele-selective silencing, RNA medicine

Exsilio Therapeutics Launches with $82 Million to Develop mRNA-Based Genomic Medicines Under Tal Zaks’ Leadership

Exsilio Therapeutics, mRNA, genomic medicines, Tal Zaks, gene therapy, gene editing, lipid nanoparticle delivery, redosing, safety profile, gene therapy approval