MILAN, Dec. 03, 2024 (GLOBE NEWSWIRE) -- AAVantgarde Bio (AAVantgarde), a clinical-stage, Italian-based international biotechnology company with two proprietary Adeno-Associated Viral (AAV) vector platforms for large gene delivery, today announces an oral presentation at the FLORetina Annual Meeting, to be held December 5-8 in Florence (Italy). At this conference, safety results at 60 days for the two first subjects with advanced retinitis pigmentosa due to Usher syndrome 1B dosed in the LUCE-1 study will be presented by Prof. Francesca Simonelli, Principal Investigator for this study. This is the first administration of a dual AAV gene therapy in Ophthalmology.
Oral Presentations details:
Presentation Date/Time: 7 December 2024, 10.50-10.54 h CET
Presenter: Prof. Francesca Simonelli
Session title: Multicentric longitudinal study in Usher Syndrome IB: disease course and implication for gene therapy
Session: Inherited retinal diseases treatments
About the AAVB-081 program
AAVB-081 is an intra-retinal AAV8-based dual hybrid product targeting MYO7A-associated Usher syndrome (USH1B). AAVantgarde’s dual hybrid platform uses two AAV8 vectors, each containing one half of an expression cassette encoding for the Myo7A gene and works at the cell nucleus level, recombining the two halves of the transgene back into a single one within the cell. This technology translates into an efficient recombination that generates therapeutically meaningful protein levels in animal models.
About the LUCE-1 Trial
LUCE-1 is a Phase 1/2 multicenter, open-label, dose escalation study investigating safety, tolerability and preliminary efficacy of 3 dose levels of dual AAV8.MYO7A (AAVB-081) administered subretinally in subjects with retinitis pigmentosa associated with Usher Syndrome Type 1B. For further information, please refer to: https://clinicaltrials.gov/study/NCT06591793?term=AAvantgarde&rank=2
About Usher syndrome type 1B
Usher syndrome type 1B (Usher1B) is an inherited disease that affects the retina and the inner ear. Usher1B is caused by mutations in the MYO7A gene. The therapeutic gene to treat Usher1B is 6.7 kb long and is therefore too large to fit inside a standard AAV vector. Approximately 20,000 patients in the U.S. and E.U. have Usher1B. These children are born deaf, have vestibular dysfunction, and begin to progressively lose vision in their first decade of life. Although there are surgical treatments available to treat deafness in these patients, there are no treatments available to treat progressive vision loss and blindness in these patients.
About AAVantgarde Bio
AAVantgarde Bio is a clinical stage, Italian headquartered, international biotechnology company that has developed two proprietary Adeno-Associated Viral (AAV) vector platforms to address the gene therapy cargo capacity limitations of AAV vectors. The AAVantgarde platforms could be used to deliver large genes to ocular and non-ocular tissues. Co-founded by Professor Alberto Auricchio at TIGEM (Telethon Institute of Genetics and Medicine) in Naples, Italy, and Telethon Foundation, AAVantgarde will initially validate the platform in the clinic in two inherited retinal diseases with clear unmet need. For more information, please visit: www.aavantgarde.com
Contact:
Magda Blanco – Head of Corporate Development AAVantgarde
Email: info@avvantgarde.com
