April 23, 2024 — 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 accepted as an oral presentation submitted to The American Society of Gene & Cell Therapy 27th Annual Meeting (ASGCT), to be held May 7-11 in Baltimore.
The data to be presented confirms that AAV intein-mediated retinal gene therapy for Stargardt disease is effective and safe in large animal models (pig and NHP). In pigs, AAVantgarde demonstrate that lipofuscin accumulation in the retinal pigmented epithelium was reduced upon subretinal delivery of AAV-ABCA4 intein vectors. Similarly, in NHP, BaseScope analysis showed nearly total photoreceptor co-expression of mRNAs encoding both ABCA4-intein halves across an extended NHP retinal region.
We are very excited to be presenting positive Pig and NHP safety and efficacy data from our Stargardt program as an oral presentation, supporting both the efficacy and safety of AAV-ABCA4-intein vectors in relevant large animal models, and providing important insights towards the clinical translation of this platform for gene therapy of STGD1.
Prof. Alberto Auricchio
CSO of AAVantgarde
This preclinical positive data in large animal models is promising and provides hope for underserved Stargardt patients that don’t have any therapeutic options to prevent them from losing their sight.
Dr. Natalia Misciattelli
CEO of AAVantgarde
Oral Presentation details:
Presentation Date/Time: Friday May 10, 2024, 4:00pm - 4:15pm
Session title: AAV Vectors - Non-Human Primates and Large Animal Models
Session Room: Ballroom 4
Final abstract number: 297
Contact
Magda Blanco – Head of Corporate Development, AAVantgarde
Email: m.blanco@aavantgarde.com