gene therapy
Pfizer’s Hemophilia A Gene Therapy Candidate Achieves Phase 3 Success
Pfizer, Hemophilia A, Gene Therapy, Phase 3 Trial, Giroctocogene Fitelparvovec, Bleeding Rate Reduction
Bluebird Bio’s Fertility Support Program for Gene Therapies Rejected by HHS Inspector General
Bluebird Bio, Gene Therapy, Fertility Support, HHS Inspector General, Zynteglo, Skysona, Anti-Kickback Rules
4DMT’s Wet AMD Gene Therapy Results Disappoint Investors, Stock Plummets
4DMT, Wet AMD, Gene Therapy, Stock Performance, Clinical Trials
Vertex Pharmaceuticals Sues US Government Over Fertility Assistance Program for Gene Therapy Patients
Vertex Pharmaceuticals, gene therapy, fertility assistance program, lawsuit, US government, health insurance, fertility preservation
Lexeo Therapeutics Reports Promising Early Data for Gene Therapy LX2006 in Friedreich Ataxia Cardiomyopathy
Lexeo Therapeutics, LX2006, Gene Therapy, Friedreich Ataxia, Cardiomyopathy, Clinical Trials, Rare Genetic Disorders
uniQure Announces Positive Interim Data Update Demonstrating Slowing of Disease Progression in Phase I/II Trials of AMT-130 for Huntington’s Disease
uniQure, Huntington’s disease, AMT-130, gene therapy, Phase I/II trials, disease progression, neurofilament light protein (NfL), Unified Huntington’s Disease Rating Scale (cUHDRS), Regenerative Medicine Advanced Therapy (RMAT) designation
UniQure Wins First FDA RMAT in Huntington’s Disease After Strong Phase I/II Data
UniQure, Huntington’s disease, gene therapy, AMT-130, FDA, RMAT designation, Phase I/II clinical trials, neurodegenerative disorder, neurofilament light (NfL), disease progression, composite Unified Huntington’s Disease Rating Scale (cUHDRS).
FDA Rejects Rocket Pharmaceuticals’ Gene Therapy for Ultra-Rare Immune Disease Over Manufacturing Concerns
FDA, Rocket Pharmaceuticals, Gene Therapy, Leukocyte Adhesion Deficiency-I (LAD-I), Manufacturing Concerns
FDA Rejects Rocket Pharmaceuticals’ Gene Therapy for Ultra-Rare Immune Disease, Requests Additional Manufacturing Information
FDA, Rocket Pharmaceuticals, Gene Therapy, Leukocyte Adhesion Deficiency-I (LAD-I), Manufacturing Information, Complete Response Letter
GenScript ProBio Launches New Gene Therapy Production Plant in New Jersey
GenScript ProBio, Gene Therapy, Production Plant, New Jersey, CDMO, Biotech