gene therapy
FDA’s Top Cell and Gene Therapy Leaders Exit Amid CBER Upheaval
FDA; cell therapy; gene therapy; CBER; Nicole Verdun; Rachael Anatol; Peter Marks; leadership changes; biopharma regulation
Second Patient Dies After Receiving Sarepta’s Elevidys Gene Therapy for DMD
Duchenne muscular dystrophy; gene therapy; Sarepta Therapeutics; Elevidys; acute liver failure; AAV-based gene therapy; patient death; non-ambulatory patients; FDA; risk mitigation
Sarepta Halts Duchenne Gene Therapy in Some Patients After Second Death
Sarepta Therapeutics; gene therapy; Duchenne muscular dystrophy; Elevidys; acute liver failure; non-ambulatory patients; FDA; clinical trial pause; immunosuppression; patient safety
Illuminating the CKD Patient Journey: Clinicogenomic Data Reshapes Research & Drug Discovery
chronic kidney disease; CKD; clinicogenomics; drug discovery; cell therapy; gene therapy; clinical trials; personalized medicine
Biotech Leaders Urge FDA to ‘Modernise’ Regulation to Compete with China
FDA; biotech; regulation; modernization; China; cell and gene therapy; CEO roundtable; competitiveness; innovation
Regenxbio Reports Consistent One-Year Benefit for DMD Gene Therapy RGX-202
Regenxbio; DMD; Duchenne muscular dystrophy; RGX-202; gene therapy; AFFINITY DUCHENNE trial; clinical trial; microdystrophin; biomarker; phase I/II; functional data
Sarepta’s rAAVrh74 Viral Vector Receives FDA Platform Technology Designation for Gene Therapy
Sarepta Therapeutics; FDA; platform technology designation; rAAVrh74 viral vector; gene therapy; SRP-9003; limb-girdle muscular dystrophy; rare diseases
Analysts Downplay Intellia Adverse Event, but Gene Therapy Safety Remains Under Scrutiny
Intellia Therapeutics; gene therapy; safety event; adverse event; MAGNITUDE trial; liver transaminase; stock decline; ATTR-CM; gene editing; analyst response
Patient Death in Rocket Pharmaceuticals Gene Therapy Trial Prompts FDA Clinical Hold
Rocket Pharmaceuticals; gene therapy; Danon disease; clinical hold; FDA; patient death; capillary leak syndrome; acute systemic infection; RP-A501; adverse event