gene therapy
CRISPR Gene Editing Therapy Can Be Administered Twice, Intellia Study Suggests
CRISPR, gene editing, Intellia Therapeutics, transthyretin amyloidosis, gene therapy, re-dosing
Sarepta Therapeutics: Next Steps After Full FDA Approval for Duchenne Muscular Dystrophy Gene Therapy
Sarepta Therapeutics, Duchenne muscular dystrophy, gene therapy, FDA approval, Elevidys, microdystrophin, ambulatory patients, non-ambulatory patients, pipeline development, rare diseases, genetic medicine.
Exsilio Therapeutics Launches with $82 Million to Develop mRNA-Based Genomic Medicines Under Tal Zaks’ Leadership
Exsilio Therapeutics, mRNA, genomic medicines, Tal Zaks, gene therapy, gene editing, lipid nanoparticle delivery, redosing, safety profile, gene therapy approval
Exsilio Emerges from Stealth with $82 Million to Revolutionize Gene Therapy
Exsilio Therapeutics, Gene Therapy, Novel Approach, mRNA, Lipid Nanoparticle Delivery, Biotech Company, Genetic Medicine
FDA Expands Approval of Sarepta’s Gene Therapy for Duchenne Muscular Dystrophy to Include Older Boys and Non-Ambulatory Patients
FDA, Sarepta, Duchenne Muscular Dystrophy, Gene Therapy, Elevidys, Ambulatory, Non-Ambulatory, Expanded Approval
Kyverna’s CAR-T Relapse in Lupus Dents Autoimmune Hopes Amid Biotech Gold Rush
Kyverna, CAR-T, Lupus, Autoimmune Disease, Biotech, Gold Rush, Relapse, Clinical Trials, Gene Therapy
Taysha Gene Therapies Announces Positive Results for TSHA-102 in Rett Syndrome Trials
Taysha Gene Therapies, TSHA-102, Rett Syndrome, Gene Therapy, Motor Skills, Communication, Autonomic Function, Seizures
FDA’s Operation Warp Speed for Rare Diseases: First Five Participants Announced
FDA, Operation Warp Speed, Rare Diseases, START Program, Gene Therapies, Neurodegenerative Conditions
Bionova Scientific Expands into Plasmid DNA Services for Cell and Gene Therapy Developers
Bionova Scientific, Plasmid DNA, Cell and Gene Therapy, CDMO, Asahi Kasei Group, Biopharmaceuticals, Biologics, Texas Facility
Biotech Faces a Reckoning: The Challenges and Shifts in Cell Therapies
cell therapy, biotech, gene therapy, autoimmune diseases, manufacturing challenges, market acceptance, pharmaceutical industry