ZipBio and MeiraGTx Enter into Exclusive License Agreement to Advance First-in-Class AAV Gene Therapy for Geographic Atrophy

ZipBio, a biotechnology company pioneering AI-driven protein therapeutics, announced an exclusive license agreement with MeiraGTx Holdings plc (Nasdaq: MGTX), a vertically integrated, clinical-stage ...

Potent DNA-recombination method developed for large cargo delivery in gene therapy

Delivery of therapeutic genes is essential for gene therapy. Adeno-associated viruses (AAVs) are a prime vector for carrying ...

Gene-editing therapy proves effective for Leber's hereditary optic neuropathy

A research team has successfully demonstrated the world's first gene-editing treatment for Leber's hereditary optic ...

Ultragenyx Resubmits Biologics License Application for UX111 AAV Gene Therapy to Treat Sanfilippo Syndrome Type A (MPS IIIA) to U.S. FDA

Company expects up to six-month review period per FDA guidelines New longer-term clinical data demonstrating durable positive brain biochemical ...
Technology Networks

New AAV Reference Standards Improve Gene Therapy QC

New, well-characterized AAV reference standards enable accurate measurement of full and empty capsids, improving assay ...
Nasdaq

MeiraGTx Holdings Receives RMAT Designation from FDA for AAV-GAD Gene Therapy in Parkinson's Disease

MeiraGTx Holdings plc announced that the FDA has granted its investigational gene therapy AAV-GAD the Regenerative Medicine Advanced Therapy (RMAT) designation for treating Parkinson's disease ...
PharmiWeb

Ajinomoto CELLiST Korea Launches CELLiSTTM AAV Production Supplement 1 to Boost Gene Therapy Production Efficiency

Based on Ajinomoto Group’s collaborative development with Forge Biologics, now available worldwide Incheon, Korea — December 2025 — Ajinomoto CELLiST Korea, a subsidiary of the Ajinomoto Group ...
KXAN

Andelyn Biosciences Applies the AAV Curator® Platform to Deliver Novel Gene Therapy for an Ultra-Rare NEDAMSS Disease Patient 14-Months after Diagnosis

‍COLUMBUS, Ohio, May 12, 2025 /PRNewswire/ -- Andelyn Biosciences, Inc., a leading and patient-focused cell and gene therapy Contract Development and Manufacturing Organization (CDMO), has ...
MedPage Today

Hemophilia B Gene Therapy Sustains Efficacy Over a Decade Later

At a median 13 years after a single infusion of AAV-mediated gene therapy, factor IX expression remained stable in 10 patients with severe hemophilia B. The median annualized bleeding rate decreased ...