AAVantgarde announces updated NHP data from its Stargardt disease program in a poster presentation at the ARVO 2024 annual meeting

24 Apr 2024
Gene Therapy
MILAN, Italy, April 24, 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 the publication of an abstract submitted to The Association for Research in Vision and Ophthalmology Annual Meeting (ARVO), to be held May 5-9 in Seattle. “We are delighted to be presenting positive NHP safety and efficacy data from our Stargardt program demonstrating that AAV.ABCA4.intein vectors can be administered safely to NHPs under an immunosuppressive protocol designed for use in man, and at doses showing robust expression matching the pattern of endogenous ABCA4. It is encouraging to see that our AAV.ABCA4.intein product shows potential to treat retinal dystrophies, such as Stargardt Disease,” said Prof. Alberto Auricchio, CSO of AAVantgarde. Dr. Natalia Misciattelli, CEO of AAVantgarde explained that “This positive data in NHP is very encouraging as it shows the potential of our Stargardt program, and it provides hope to the underserved Stargardt patients that currently don’t have many therapeutic options available.” Poster Presentation details: Presentation Number / Posterboard Number: 6097 - B0943 Abstract Title: Safety and expression following subretinal administration of AAV.ABCA4.intein vectors in the non-human primate retina Session Number: 515 Session Title: Gene and cell therapies and other novel therapeutics II Session Date/Time: May 9, 2024 from 8:00 AM to 9:45 AM 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: Contact: Magda Blanco – Head of Corporate Development AAVantgarde Phone: +44 (0) 7522 128285 Email: m.blanco@aavantgarde.com
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