gene therapy
MeiraGTx’s Gene Therapy Shows Significant Improvements in Motor Function and Quality of Life in Parkinson’s Disease Trial
MeiraGTx, Gene Therapy, Parkinson’s Disease, AAV-GAD, Motor Function, Quality of Life, Clinical Trial, Phase 2 Trial, Neurodegenerative Disease, Dopamine Replacement, GABA Production
MeiraGTx Announces Positive Phase 2 Data for AAV-GAD Gene Therapy in Parkinson’s Disease, Paving Way for Phase 3 Trial
MeiraGTx, AAV-GAD, Gene Therapy, Parkinson’s Disease, Phase 2 Trial, Phase 3 Trial, Neurodegenerative Disorders, Genetic Medicine
Pfizer Secures Second FDA Approval for Hemophilia Treatment in Six Months
Pfizer, FDA approval, Hemophilia, Gene Therapy, Hemophilia B, HYMPAVZI
Basecamp Research Secures $60M to Revolutionize AI Drug Discovery with Broad Institute Partnership
AI drug discovery, Basecamp Research, Broad Institute, David R. Liu, Biotechnology, Gene therapy, Artificial intelligence in biology
Purespring Therapeutics Secures £80M ($105M) Series B Funding to Advance IgAN Gene Therapy into Clinical Trials
Purespring Therapeutics, IgAN gene therapy, kidney disease, Series B funding, clinical trials, AAV gene therapy, podocytes, nephrotic syndrome, glomerular kidney disease.
GEMMABio Secures $100M Partnership to Advance Rare Disease Gene Therapies in Brazil
GEMMABio, gene therapy, Brazil, rare diseases, $100M partnership, health ministry
Astellas Expands Gene Therapy Portfolio with $2.2 Billion Deal for AviadoBio’s Frontotemporal Dementia Treatment
Astellas Pharma Inc., AviadoBio Ltd., Gene Therapy, Frontotemporal Dementia, FTD-GRN, AVB-101, Progranulin Mutations, Neurodegenerative Diseases
Gene Therapy Pioneer Jim Wilson Launches Franklin Biolabs, Expanding Genetic Medicine Services
Jim Wilson, Franklin Biolabs, Gene Therapy, Genetic Medicine Services, Rare Diseases, Adeno-Associated Viruses (AAVs)
Navigating Complex Regulatory Landscapes: The Importance of External Support for Biotech Sponsors in IRB, IBC, DMCs, and EACs
Biotech sponsors, IRB (Institutional Review Board), IBC (Institutional Biosafety Committee), DMCs (Data Monitoring Committees), EACs (Ethics Advisory Committees), Regulatory compliance, Clinical trials, Gene therapy research, Risk management
BridgeBio Cuts Gene Therapy Budget by Over $50 Million Following Disappointing Early-Phase Data
BridgeBio, gene therapy, budget cut, early-phase data, CAH treatment, BBP-631, congenital adrenal hyperplasia