Advancing Gene Therapy: AAVantgarde Bio Closes €61 Million Series
AAVantgarde Bio Secures €61 Million Series A Funding for Breakthrough Gene Delivery Platforms Italian-based biotechnology company AAVantgarde Bio has successfully raised €61 million ($65.1 million) in a Series A financing round, marking a significant milestone for the Italian biotech sector.
AAVantgarde specializes in developing two cutting-edge adeno-associated viral (AAV) vector platforms designed to deliver large genes effectively. The first platform, known as dual hybrid, employs DNA recombination, while the second platform, named AAV intein, utilizes protein trans-splicing. Currently, AAVantgarde is focusing on two key programs to validate these platforms.
The primary program aims to address Usher Syndrome Type 1 B associated retinitis pigmentosa (Usher1B) by leveraging the dual hybrid platform. Simultaneously, the second program targets Stargardt disease, utilizing the AAV intein platform. The funding obtained from the Series A round will support the completion of a groundbreaking first-in-human proof of concept (POC) study for Usher1B and further development of the AAV intein program. Additionally, AAVantgarde has plans to expand its pipeline and explore applications beyond ophthalmology. With the potential to deliver large genes to ocular and non-ocular tissues, AAVantgarde’s AAV-based platforms offer promising opportunities for treating various genetic conditions.
The company was co-founded by Alberto Auricchio at TIGEM (Telethon Institute of Genetics and Medicine) in Naples, Italy, and the Telethon Foundation, with a primary focus on validating the technology in the clinic for inherited retinal diseases lacking effective treatments. Natalia Misciattelli, CEO of AAVantgarde, expressed her excitement about the significant funding round, emphasizing the company’s commitment to making a difference in the lives of patients. She stated, “This funding represents a major milestone in our mission to improve patient outcomes, and we look forward to collaborating with our new and existing investors to advance our platforms and pipeline.” Misciattelli underscored the company’s dedication to addressing the profound impact of blindness associated with Usher1B syndrome and Stargardt disease.
The financing was co-led by Atlas Venture, a renowned biotechnology venture creation firm based in the United States, and Forbion, a prominent European life sciences venture capital investment firm. Longwood Fund and Sofinnova Partners, the company’s founding investor through its Sofinnova Telethon Fund, also participated in the funding round. Usher syndrome type 1B (Usher1B) is an inherited disease affecting both the retina and the inner ear. This condition arises due to mutations in the MYO7A gene. However, the therapeutic gene required to treat Usher1B is too large, measuring 6.7 kb, to fit into a standard AAV vector. Currently, approximately 20,000 patients in the United States and European Union are affected by Usher1B, experiencing deafness, vestibular dysfunction, and progressive vision loss, for which there are no approved treatments available.
Stargardt disease, the most prevalent inherited macular degeneration, typically follows an autosomal recessive pattern and is caused by mutations in the ABCA4 gene. The therapeutic gene necessary for treating Stargardt disease (ABCA4) is also too large, measuring 6.8 kb, for standard AAV vectors. Stargardt disease affects approximately 60,000-75,000 patients in the United States and European Union, and presently, there are no approved treatments for the associated blindness.
Advancing Gene Therapy
