Hereditary Diseases
FDA Advisory Committee Approves Zevra Therapeutics’ Arimoclomol for Rare Genetic Disease Treatment
Zevra Therapeutics, Arimoclomol, FDA Advisory Committee, Niemann-Pick disease, rare genetic disease, drug approval
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
Ultragenyx Advances Gene Therapy for Glycogen Storage Disease Type Ia Following Phase 3 Success
Ultragenyx, gene therapy, glycogen storage disease type Ia, DTX401, Phase 3 study, GlucoGene, rare genetic diseases
Maze Therapeutics Secures New Partnership for Pompe Drug Development After Sanofi Deal Collapse
Maze Therapeutics, Pompe drug, partnership, Sanofi deal collapse, drug development, genetic diseases
Revolutionary Gene Therapy Advancements: Early Trial Results from Leading Biotech Companies
ASGCT24, gene therapy, early trial results, Freeline, Rocket, Creyon, biotech companies, genetic diseases, hemophilia, liver diseases, Parkinson’s disease.
Revival of Gene Editing Clinical Trials in China Post CRISPR Baby Scandal
CRISPR baby scandal, China, gene editing, clinical trials, restart, regulations, ethics, CRISPR-Cas9, genetic diseases, biotechnology.
Prime Medicine Secures FDA Approval for Pioneering Gene Editing Therapy Trial
Prime Medicine, FDA approval, gene editing, clinical trial, innovative therapy, genetic diseases
Pfizer’s Revolutionary Hemophilia B Gene Therapy Gains FDA Approval and Introduces Unique Warranty Program
Pfizer, FDA, Hemophilia B, Gene Therapy, Approval, Warranty, Treatment, Genetic Disorder, Blood Clotting, Healthcare, Biotechnology
FDA Approves Pfizer’s Hemophilia B Gene Therapy with a $3.5 Million Price Tag
Pfizer, FDA, hemophilia B, gene therapy, approval, pricing, healthcare, biotechnology, rare diseases, genetic disorders, treatment cost.