AAVantgarde and TIGEM Publish Groundbreaking Research in Science Advances

/EIN News/ -- MILAN, March 31, 2025 (GLOBE NEWSWIRE) -- AAVantgarde Bio (AAVantgarde), a clinical-stage biotechnology company, in collaboration with the Telethon Institute of Genetics and Medicine (TIGEM), announces the publication of their preclinical research in Stargardt’s disease in Science Advances, a prestigious peer-reviewed journal. This breakthrough study underscores the companies' joint commitment to advancing gene therapy and expanding the frontiers of scientific discovery.

The paper, titled “Retinal gene therapy for Stargardt disease with dual AAV intein vectors is both safe and effective in large animal models”, presents novel findings that could significantly impact the development of an innovative therapy for Stargardt’s disease. By leveraging AAVantgarde’s proprietary adeno-associated virus (AAV) intein platform and TIGEM’s world-class expertise in genetic research, the study provides compelling evidence for the translation of this program into the clinic.

“This publication marks an important milestone in our mission to revolutionize gene therapy,” said Dr. Natalia Misciattelli, CEO of AAVantgarde. “Our collaboration with TIGEM has enabled us to push the boundaries of what is possible, bringing us one step closer to developing transformative treatments for patients with Stargardt’s disease.”

Professor Alberto Auricchio, CSO of AAVantgarde and Scientific Director at TIGEM, added, “The results of this study highlight the power of combining cutting-edge vector engineering with deep biological insights. We are excited to contribute to this field and look forward to translating these findings into tangible clinical applications.”

For more information about the publication and the groundbreaking work being conducted at AAVantgarde and TIGEM, please visit www.aavantgarde.com and https://www.tigem.it/.

About AAVantgarde

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 are being used to deliver large genes to the Company’s two lead programs in Usher 1B and Stargardt disease, two inherited retinal diseases with clear unmet need, with the platform also having the potential for applicability in non-ocular tissues. For more information, please visit: www.aavantgarde.com

Contact:
Dr. Magda Blanco – Head of Corporate Development
Email: info@aavantgarde.com