About us

AAVantgarde is a clinical-stage, international biotechnology company (headquartered in Italy) that has developed two proprietary adeno-associated viral (AAV) vector platforms to address the DNA cargo capacity limitations of AAV vectors

Our platforms (dual hybrid and intein-mediated) are being validated in two inherited retinal diseases (IRDs) with a high unmet need: Usher syndrome type 1B (USH1B) and Stargardt disease (STGD) respectively and have the potential to be extended into different diseases caused by a mutated large gene.

Our mission

Gene therapy utilizing AAV vectors has proven to be transformative in the treatment of genetic diseases. However, the small DNA vector payload limits their application when the size of the gene of interest is over 4.7 kb.

The cutting-edge technology used in AAVantgarde platforms are focused on solving the constraint of oversized gene delivery, opening the opportunity for genetic medicine to patients with no current therapeutic options. AAVantgarde has a clear mission to bring the next generation of genetic medicines to underserved patients with debilitating diseases.

Our history

AAVantgarde is a spin-off of Telethon Institute for Genetics and Medicine (TIGEM), an international research institute based in Naples that is owned and managed by the Telethon Foundation. AAVantgarde was founded by Professor Alberto Auricchio, a highly recognized scientist and a pioneer in the field of gene therapy.

As well as being Founder and CSO of AAVantgarde, Professor Auricchio is TIGEM Scientific Director and Professor of Medical Genetics at University “Federico II” in Naples, Italy. He is co-author of over 150 peer-reviewed publications, inventor of several patents related to the use of viral vectors for gene therapy and has received the Outstanding New Investigator Award of the American Society of Gene Therapy and the International Prize for Scientific Research “Arrigo Recordati. He is Vice-President of the ESGCT since 2023.