gene therapy
Advancements in Hematology: Shaping the Future of Blood Disorder Diagnosis and Treatment
Hematology, Blood Disorders, Sickle Cell Disease, Gene Therapy, Clinical Guidelines, Quality Improvement
Neurogene’s Rett Syndrome Trial Halted After Patient Death Linked to High-Dose Gene Therapy
Neurogene, Rett syndrome, gene therapy, AAV, clinical trial, patient death, high-dose complications
CSL Behring to Close California R&D Site Amid Shift in Gene Therapy Strategy
CSL Behring, California R&D site closure, ex vivo gene therapy, strategic repositioning, biotech industry trends
Neurogene Halts Development of Batten Gene Therapy NGN-101 Following FDA Denial
Neurogene, Batten disease, gene therapy, NGN-101, FDA denial, rare disease research
Regenxbio Advances Duchenne Muscular Dystrophy Gene Therapy to Pivotal Studies, Potentially Challenging Sarepta’s Dominance
Regenxbio, Sarepta Therapeutics, Duchenne Muscular Dystrophy (DMD), Gene Therapy, RGX-202, Elevidys, Pivotal Studies, Biologics License Application (BLA), FDA Approval
CSL Behring Closes California R&D Site, Shifts Operations to Massachusetts Amid Strategic Repositioning
CSL Behring, California R&D site closure, gene therapy, ex vivo work, strategic repositioning, Massachusetts relocation
Bluebird Bio Confronts Financial Crunch Amidst Breakeven Projections
Bluebird Bio, cash gap, breakeven point, financial stability, gene therapy, cash flow
FDA Approves PTC Therapeutics’ Kebilidi, the First Gene Therapy Delivered Directly to the Brain for AADC Deficiency
PTC Therapeutics, Kebilidi, Gene Therapy, AADC Deficiency, FDA Approval, Brain-Delivered Therapy, Rare Genetic Disorder
Neurogene’s Rett Syndrome Gene Therapy Shows Efficacy but Raises Safety Concerns
Neurogene, Rett syndrome, gene therapy, NGN-401, AAV vector, clinical trial, safety concerns, efficacy results
Emerging Therapies for Huntington’s Disease: A Review of Promising Treatments
Huntington’s disease, gene therapy, antisense oligonucleotides, stem cell therapy, disease-modifying treatments