News about "Gene Therapy"

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

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 |  139

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

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 |  231

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 |  217

Ocugen and Kwangdong Finalise OCU400 Gene Therapy Deal for Retinitis Pigmentosa in Korea

Pursuant to the licence agreement, Ocugen will receive upfront license fees and near-term development milestones equaling up to USD 7.5 million. The company will be entitled to sales milestones of USD 1.5 million for every USD 15 million of sales in Korea, projected to reach USD 180 million or more in the first 10 years of commercialisation.

Gene Therapy | 17/09/2025 | By Dineshwori |  487

Sanofi Drug Earns Fast Track Status for Wet AMD in US

Sanofi is evaluating SAR402663 in a Phase 1/2 trial (NCT06660667) for neovascular AMD. The gene therapy targets abnormal blood vessel growth, reduces retinal damage, and aims to eliminate the need for frequent intravitreal injections.

Gene Therapy | 11/09/2025 | By Dineshwori |  163

Sarepta Pauses Shipments of Duchenne Gene Therapy Elevidys Amid FDA Safety Review

Sarepta Therapeutics has announced a voluntary and temporary suspension of all US shipments of its Duchenne muscular dystrophy (DMD) gene therapy, ELEVIDYS (delandistrogene moxeparvovec), following the FDA’s distribution suspension request last week.

Gene Therapy | 24/07/2025 | By Dineshwori |  428

Nvelop Therapeutics to Launch Dual Platforms for In Vivo Delivery of Next-Gen of Genetic Medicines

Nvelop Therapeutics has officially announced to develop the next generation of genetic medicines for a wide range of diseases.

Gene Therapy | 10/04/2024 | By Aishwarya |  656