gene therapy
Novartis Prepares to File Intrathecal Zolgensma for Older SMA Patients by Mid-2025
Zolgensma, intrathecal delivery, spinal muscular atrophy, gene therapy, older patients
Sarepta Reports Death of DMD Patient Following Elevidys Gene Therapy Treatment
Sarepta, Elevidys, gene therapy, Duchenne muscular dystrophy, patient death, acute liver failure
Lonza’s Vision for the Future: A Fireside Chat with New CEO Wolfgang Wienand
Lonza, Wolfgang Wienand, CDMO, Pharmaceutical manufacturing, Biologics, Cell and gene therapy, Small molecules, Leadership transition, Industry outlook
MeiraGTx and Hologen AI Form $430M Collaboration to Advance Parkinson’s Gene Therapy
gene therapy, Parkinson’s disease, artificial intelligence, clinical trials, biotechnology partnership
Regeneron gene therapy improves hearing in 10 children with rare form of hearing loss
Regeneron Pharmaceuticals, DB-OTO gene therapy, otoferlin (OTOF) gene, congenital hearing loss, intracochlear injection
Pfizer Discontinues Hemophilia B Gene Therapy Beqvez, Exits Gene Therapy Field
Pfizer, Beqvez, hemophilia B, gene therapy, discontinuation, portfolio exit
Bluebird Bio’s Fall from Grace: Gene Therapy Pioneer Sells for $29M After $10B Valuation
Bluebird Bio, gene therapy, private equity, Carlyle, SK Capital, financial challenges, acquisition, biotech industry
Solid Biosciences’ DMD Gene Therapy Shows Promising Early Results, Driving Stock Surge
Duchenne muscular dystrophy (DMD), gene therapy, SGT-003, microdystrophin, clinical trial, stock surge, Solid Biosciences
Voyager Therapeutics Halts ALS Gene Therapy Development Due to Off-Target Effects
SOD1 ALS gene therapy, Off-target effects, VY9323, siRNA payload, TRACER capsid, Preclinical data, Drug development delay
NHS England Approves Groundbreaking CRISPR Gene Therapy for Sickle Cell Disease
Casgevy, exagamglogene autotemcel, CRISPR gene therapy, sickle cell disease, NHS England, Vertex Pharmaceuticals, NICE approval