MILAN, Dec. 02, 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 announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to its lead program, AAVB-081 for the treatment of Usher Syndrome Type 1B (USH1B) retinitis pigmentosa.
“People living with USH1B are in urgent need of a therapy to prevent their progressive loss of vision leading to eventual blindness. Receiving FDA Orphan Drug Designation reinforces the unmet need of this indication and is an important milestone in the development of this program,” said Dr. Natalia Misciattelli, Chief Executive Officer of AAVantgarde.
The FDA’s Orphan Drug Designation program provides orphan status to drugs and biologics for rare diseases that meet certain criteria. Orphan Drug Designation provides various incentives including tax credits for qualified clinical trials, exemption from user fees, and the potential for seven years of market exclusivity after approval.
About 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 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, 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. AAVantgarde is initially validating its platforms in two inherited retinal diseases with clear unmet need, with its lead program in Usher syndrome type 1B already in the clinic. For more information, please visit: www.aavantgarde.com
Contact:
Dr. Magda Blanco – Head of Corporate Development, AAVantgarde
Email: info@aavantgarde.com
