News about "Gene Therapy"

UK MHRA Approves Vyjuvek for Treatment of Dystrophic Epidermolysis Bullosa Wounds

Medicines and Healthcare products Regulatory Agency has approved Vyjuvek for treating wounds in patients with dystrophic epidermolysis bullosa, including newborns.

Gene Therapy | 18/05/2026 | By News Bureau

FDA Clears IND Application for Chengdu Origen's Gene Therapy Targeting Hypertrophic Cardiomyopathy

Chengdu Origen Biotechnology and Vanotech have received US FDA clearance to begin clinical evaluation of KHN921, a potential first-in-class gene therapy designed to address the genetic root cause of hypertrophic cardiomyopathy linked to MYBPC3 mutations.

Gene Therapy | 16/05/2026 | By News Bureau

Dyno Therapeutics Unveils Next-Generation AI-Engineered AAV Capsids for CNS and Muscle Gene Delivery

Dyno Therapeutics introduced new AAV gene delivery capsids for central nervous system and muscular applications, highlighting enhanced cross-species transduction, reduced liver targeting and scalable manufacturing potential for next-generation genetic medicines.

Gene Therapy | 16/05/2026 | By News Bureau

Regenxbio Reports Positive Pivotal Trial Results for Duchenne Gene Therapy RGX-202

Regenxbio’s investigational gene therapy RGX-202 demonstrated strong microdystrophin expression, functional gains and a favourable safety profile in Duchenne muscular dystrophy patients, supporting plans for accelerated approval and a potential commercial launch in 2027.

Gene Therapy | 15/05/2026 | By News Bureau

NIH-Funded Study Unveils Compact CRISPR System for Targeted In-Body Gene Editing

Researchers backed by the National Institutes of Health have developed an enhanced CRISPR enzyme that enables efficient gene editing and fits into viral delivery systems, advancing potential in-body therapies.

Gene Therapy | 15/04/2026 | By News Bureau |  266

Belief Pharmaceuticals' Haemophilia B Gene Therapy Approved in Macau

Macau approves Belief Pharmaceuticals’ Popedacoq gene therapy for haemophilia B, marking regional expansion after China approval and advancing access to one-time treatment solutions.

Gene Therapy | 23/03/2026 | By News Bureau

Chugai Launches Elevidys Gene Therapy for Duchenne Muscular Dystrophy in Japan

Elevidys receives conditional approval and NHI listing for ambulatory DMD patients aged three to under eight years; launch backed by Phase III EMBARK study and strengthened safety measures.

Gene Therapy | 23/02/2026 | By News Bureau |  190

Genethon Partners with AskBio on Pompe Gene Therapy

The licensing deal enables AskBio to advance the clinical development of Genethon’s investigational gene therapy for Pompe disease through an industry partnership.

Gene Therapy | 15/01/2026 | By News Bureau |  122

EMA Recommends EU Approval for Waskyra to Treat Wiskott-Aldrich Syndrome

The EMA has recommended EU marketing authorisation for Waskyra (etuvetidigene autotemcel), a gene therapy for patients aged six months and older with Wiskott-Aldrich Syndrome (WAS). The treatment uses a patient’s own modified stem cells to restore functional WAS protein.

Gene Therapy | 17/11/2025 | By Dineshwori |  243

Waters Corporation Unveils Xevo Charge Detection MS for Advanced Biomolecule Analysis

The new Xevo CDMS system by Waters Corporation enables measurement of large biomolecules to support Research and Development (R&D) in gene and protein-based therapies.

Gene Therapy | 21/10/2025 | By Dineshwori |  232