AAVantgarde presents positive preclinical data in large animal models from its Stargardt disease program in an oral presentation at the ASGCT 2024 annual meeting
April 23 2024 - 2:00AM
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.
Prof. Alberto Auricchio, CSO of
AAVantgarde stated “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.”
Dr. Natalia Misciattelli, CEO of
AAVantgarde added that “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.”
Oral Presentation details:Presentation
Date/Time: Friday May 10, 2024, 4:00pm - 4:15pmSession title: AAV
Vectors - Non-Human Primates and Large Animal ModelsSession Room:
Ballroom 4Final abstract number: 297
About AAVantgarde
BioAAVantgarde 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 AAVantgardePhone: +44 (0) 7522 128285Email:
m.blanco@aavantgarde.com |