gene therapy – Page 9

UniQure Wins First FDA RMAT in Huntington’s Disease After Strong Phase I/II Data

10 months ago talkbio0Tagged AMT-130, Clinical Trials, Composite, cUHDRS, Disease Progression, gene therapy, Huntington 's disease, Huntington 's Disease Rating Scale, Identifier, NEFL gene, Neurodegenerative Disorders, NfL, Phase, RMAT, Unified - Type of Agreement, UniQure, United States Food and Drug Administration

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

10 months ago talkbio0Tagged concerns, deficiency-I, gene therapy, LAD-I, leukocyte cell-cell adhesion, Manufacture, Rocket, United States Food and Drug Administration

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

10 months ago talkbio0Tagged Complete, control act - information, deficiency-I, gene therapy, LAD-I, leukocyte cell-cell adhesion, Manufacture, Response process, Rocket, United States Food and Drug Administration

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

10 months ago talkbio0Tagged biotech, CDMO, gene therapy, GenScript ProBio, Plants, production

GenScript ProBio, Gene Therapy, Production Plant, New Jersey, CDMO, Biotech

CRISPR Gene Editing Therapy Can Be Administered Twice, Intellia Study Suggests

10 months ago talkbio0Tagged Amyloidosis, Clustered Regularly Interspaced Short Palindromic Repeats, Gene Modification, gene therapy, Intellia, Prealbumin, re-dosing

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

10 months ago talkbio0Tagged Ambulatory - qualifier value, Approved, Duchenne, ELEVIDYS, gene therapy, Genetic Medicine, Microdystrophin, pipeline development, Unable to walk (finding), United States Food and Drug Administration

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

10 months ago talkbio0Tagged Approved, Exsilio, Gene Modification, gene therapy, genomic medicines, Lipids, nanoparticle delivery, RNA, Messenger, safety profile, Tal Zaks

Exsilio Therapeutics, mRNA, genomic medicines, Tal Zaks, gene therapy, gene editing, lipid nanoparticle delivery, redosing, safety profile, gene therapy approval