gene therapy
Vertex’s CASGEVY Marks Historic Milestone with First Commercial Patient Infusions, Paving the Way for 2025 Launches
CASGEVY, Vertex Pharmaceuticals, gene therapy, sickle cell disease, beta thalassemia, CRISPR/Cas9, FDA approval, commercial patient infusions, 2025 launches.
Beam Therapeutics Reports Promising Early Results in Sickle Cell Disease Base Editing Therapy, Despite Conditioning-Related Fatality
Beam Therapeutics, Sickle Cell Disease, Base Editing, Gene Therapy, BEAM-101, ESCAPE, Conditioning, Patient Death
Lexeo Therapeutics Unveils Promising Interim Data for LX1001 Gene Therapy in APOE4-Associated Alzheimer’s Disease
Lexeo Therapeutics, LX1001, Gene Therapy, Alzheimer’s Disease, APOE4, APOE2, Clinical Trial, Phase 1/2
Intellia’s CRISPR Gene Editing Therapy Shows Promising Results in Reducing HAE Attacks
Intellia Therapeutics, CRISPR Gene Editing, Hereditary Angioedema (HAE), NTLA-2002, Clinical Trials, Gene Therapy, Rare Diseases
Roche and Dyno Therapeutics Launch $1B+ Collaboration to Develop Next-Generation AAV Gene Therapy Vectors for Neurological Diseases
Roche, Dyno Therapeutics, AAV gene therapy vectors, Neurological diseases, AI-driven gene delivery, LEAP technology, Gene therapy collaboration
Roche Revisits Dyno for AAV Technology with $50M Upfront Payment
Roche, Dyno Therapeutics, AAV technology, gene therapy, neurological diseases, strategic partnership
Pfizer Announces Additional Layoffs in North Carolina Following DMD Trial Setback
Pfizer layoffs, North Carolina, Duchenne muscular dystrophy (DMD), gene therapy trial failure, cost-cutting measures
Ocuphire Pharma Acquires Opus Genetics, Expanding into Inherited Retinal Disease Gene Therapies
Ocuphire Pharma, Opus Genetics, gene therapy, inherited retinal diseases (IRDs), merger, acquisition, biopharmaceutical, ophthalmic treatments
Sangamo Therapeutics Aligns with FDA on Accelerated Approval Pathway for ST-920 Gene Therapy in Fabry Disease
Sangamo Therapeutics, FDA, Accelerated Approval Pathway, ST-920, Fabry Disease, Gene Therapy, BLA Submission
Sangamo Therapeutics Fast-Tracks Fabry Gene Therapy with FDA Accelerated Approval Pathway
Sangamo Therapeutics, Fabry disease, gene therapy, isaralgagene civaparvovec, FDA, accelerated approval pathway, ST-920, STAAR study