Poster Presentation: Title: Advancing AAV Capsid Engineering for Targeted Gene Therapy Using PackGene's π-Icosa Platform (AMA1046) Time: May 13th, 6-7:30pm Location: Poster Hall 2 ...

Tardieu, M., et al. (2017). Intracerebral gene therapy in children with mucopolysaccharidosis type IIIB syndrome: an uncontrolled phase 1/2 clinical trial.

JCR Pharmaceuticals has signed a licence agreement with Alexion, AstraZeneca Rare Disease, for its proprietary JUST-AAV capsids.

Ixo-vec reduced anti-VEGF injection frequency and was well tolerated in patients with wet AMD. A phase 3 pivotal trial ...

The primary safety risks associated with gene therapy include immune reactions and off-target effects in unintended organs, such as the liver, lungs, and spleen. Nevertheless, Ali and Townsend note ...

Latus Bio Announces Publication of Breakthrough Research on Novel AAV Capsid Variant for Huntington’s and Parkinson’s Disease Gene Therapy Provided by Business Wire May 20, 2025, 4:00:00 AM ...

Demonstration of 20- to 400-fold increased gene transfer capabilities of the novel AAV capsid VCAP-102 compared to existing methods, showcasing potential advancements in gene therapy efficacy.

Apertura Gene Therapy and Rett Syndrome Research Trust Collaborate to Pioneer Advanced Genetic Medicines for Rett Syndrome Using TfR1-Targeted AAV Capsid Provided by PR Newswire Apr 30, 2025, 11: ...

This study marks the first time gene therapy has shown consistent success in both children and adults with OTOF-related deafness. It also paves the way for similar therapies targeting other genes, ...

Taysha Gene Therapies, Inc. (NASDAQ:TSHA) is one of the best biotech penny stocks to buy right now. On July 1, analyst Gil Blum from Needham maintained a Buy rating on Taysha Gene Therapies, Inc.

This guide explores a faster, high-throughput approach to accurately evaluate AAV capsid content and help researchers improve efficiency and accelerate gene therapy development. Inside this technical ...