AXV-101 / Axovia Therap - LARVOL DELTA

Axovia Therapeutics Presents New Preclinical Data for AXV-101 for the Treatment of Blindness Associated with BBS Mutations at ASGCT (GlobeNewswire) - "Axovia Therapeutics Ltd... announced the presentation of new preclinical data in two poster presentations at the American Society of Gene and Cell Therapy (ASGCT) 28th Annual Meeting, which is being held from May 13-17, 2025 in New Orleans, LA. The two poster presentations include data that support the dosing regimen and protocol for the subretinal Phase 1/2 study for the company’s lead program for Bardet-Biedl Syndrome (BBS), AXV-101, which is expected to initiate in mid-2025."

Clinical protocol • Preclinical • Bardet–Biedl Syndrome

Axovia Therapeutics Announces Three Upcoming Poster Presentations at ASGCT (GlobeNewswire) - "Two poster presentations include preclinical data that support the dosing regiment and study protocol for the subretinal clinical study for its lead program for Bardet-Biedl Syndrome (BBS), AXV-101, which the company expects to initiate in mid-2025. A third poster unveils the first preclinical proof of concept data for novelly designed, AXV-201, to treat individuals with severe obesity and very high BMIs resulting from a MC4R mutation."

New trial • Preclinical • Bardet–Biedl Syndrome • Obesity

Development of a ddPCR Assay to Quantify AXV-101 Levels for a Mouse Biodistribution Study (ASGCT 2025) - "This supports the use of the assay in measuring biodistribution of AXV-101 in toxicology study samples. Disease Focus of Abstract:Genetic Diseases"

Preclinical • Gene Therapies • Genetic Disorders

A new dosing regimen to treat Bardet-Biedl Syndrome 1 ( BBS1) retinal degeneration with AXV-101 (AAV9-BBS1) improves histological and functional photoreceptor survival in Bbs1 M390R mice. Finding the right balance between bleb number, dose per bleb and total dose per eye. (ASGCT 2025) - "That AXV-101 can sustain a high level of functional rescue over time and it is a prime candidate for first-in-human clinical trials. Disease Focus of Abstract:Eye Disorders"

Preclinical • Bardet–Biedl Syndrome • Inherited Retinal Dystrophy • Ophthalmology

A Race Against Blindness Announces $2 Million Grant to Support Sight-Saving Clinical Trial (GlobeNewswire) - "A Race Against Blindness, a nonprofit organization dedicated to funding sight-saving therapies for children, is thrilled to announce a $2.0 million grant was awarded in December 2024 to Axovia Therapeutics to support a planned clinical trial for the company’s product candidate, AXV101, aimed at combating childhood blindness due to retinitis pigmentosa (RP) caused by Bardet-Biedl Syndrome 1 (BBS1)....The grant funding provided by A Race Against Blindness to Axovia Therapeutics has enabled significant progress in nearing the anticipated Clinical Trial Application (CTA) submission, which if cleared, would allow the company to begin a clinical trial in the UK in mid-2025."

Financing • New trial • Bardet–Biedl Syndrome