AAVantgarde Bio’s Dual-Vector USH1B Gene Therapy Moves into Phase 1/2 Clinical Trial

AAVantgarde Bio, a gene therapy development company based in Italy, has dosed the first patient in its Phase 1/2 retinal gene therapy clinical trial for people with Usher syndrome type 1B (USH1B) which is caused by mutations in the gene MYO7A. Known as LUCE-1, the trial is taking place at the University Hospital of Campania “Luigi Vanvitelli” (Naples, Italy) with Prof. Francesca Simonelli serving as lead investigator. Two additional sites for the Phase 1/2 trial in the UK will be announced. The company plans to launch sites in the US if the emerging gene therapy moves into a pivotal, Phase 3 trial. Fifteen patients ranging from 18 to 50 years of age will be enrolled in the Phase 1/2 trial. Three doses of the USH1B gene therapy will be evaluated.

Known as AAVB-081, the gene therapy is the first clinical application of a dual-vector gene delivery system for a retinal disease. AAVB-081 uses an adeno-associated virus (AAV), which works like a vast container system, to deliver healthy copies of the MYO7A gene into retinal cells. Because the MYO7A gene is too large for a single AAV container, the gene is split into two halves and delivered in two containers. Once in the cell, the halves are recombined. The one-time treatment is contained in a tiny drop of liquid that is injected underneath the retina.

The dual-AAV technology for large gene delivery comes from the research of Prof. Alberto Auricchio at TIGEM, an international research institute based in Naples that is owned and managed by the Telethon Foundation. AAVantgarde is a spin-off of TIGEM and has an exclusive license for the AAV-dual technology for inherited retinal diseases. Usher syndrome type 1 is the most severe type of Usher syndrome. USH1B, caused by mutations in MYO7A, is the most common cause of USH1. Children with Usher syndrome type 1B are born with profound hearing loss and begin experiencing progressive vision loss from retinitis pigmentosa before puberty. They also have issues with balance. AAVantgarde says that approximately 20,000 people in the US and EU have USH1B.

AAVantgarde is also developing a gene therapy for Stargardt disease caused by mutations in ABCA4.

For more information about the USH1B gene therapy clinical trial, send a note to info@aavantgarde.com.