AAVantgarde Bio, an international Italian-based biotechnology company in the clinical stage with two adeno-associated virus (AAV) vector platforms for large gene delivery, has closed a €61 million ($65.1 million) Series A financing.
The amount is the largest series A round ever in Italian biotech.
AAVantgarde has two patented AAV-based large gene delivery platforms: one utilizing DNA recombination, named double hybrid; and one trans-splicing protein, named AAV intein.
AAVantgarde validated the platform in two main programmes: Usher Syndrome Type 1B associated retinitis pigmentosa (Usher1B), using a double hybrid; and Stargardt’s disease, using AAV inteins. This funding will fund the completion of the first human in-subject proof of concept (POC) study with Usher1B and the further development of the AAV intention program and admission to the clinic for Stargardt disease. The company also intends to pursue programs outside of ophthalmology.
The AAVantgarde platform can be used to deliver large genes to ocular and non-ocular tissues. Co-founded by Alberto Auricchio at TIGEM (Telethon Institute of Genetics and Medicine) in Naples, Italy, and the Telethon Foundation, AAVantgarde will initially validate the platform in the clinic in two congenital retinal diseases with clearly unmet needs.
“This funding is a watershed moment in our mission to help patients, and we are excited to work with our new investors and founders to advance our platform and channel,” said Natalia Misciattelli, CEO of AAVantgarde.
“Completing this round with a group of high caliber global life science investors is recognition of the importance of AAVantgarde’s unique technology, which will initially be used to assist patients suffering from the debilitating effects of blindness associated with Usher1B syndrome and Stargardt disease.”
The funding was co-led by US biotechnology venture creation firm Atlas Venture and European life sciences venture capital investment firm Forbion, with participation from the Longwood Fund and the company’s founding investor Sofinnova Partners via the Sofinnova Telethon Fund.
About Usher’s syndrome
Usher syndrome type 1B (Usher1B) is an inherited disease that affects the retina and inner ear. Usher1B is caused by a mutation in the MYO7A gene. The therapeutic gene to treat Usher1B is 6.7 kb long and therefore too large to fit inside a standard AAV vector.
About 20,000 patients in the US and EU have Usher1B. These children are born deaf, have vestibular dysfunction, and begin progressive loss of vision in the first decades of life. While there are surgical treatments available to treat deafness in these patients, there are no treatments available to treat vision loss and progressive blindness in these patients.
About Stargardt’s disease
Stargardt’s disease is the most common congenital macular degeneration. Inherited in the majority of cases as an autosomal recessive, Stargardt disease is caused by a mutation in the ABCA4 gene. The therapeutic gene for treating Stargardt’s disease (ABCA4) is 6.8 kb long which is too large to fit in a standard AAV vector.
Stargardt’s disease affects an estimated 60,000-75,000 patients in the US and EU. Currently, there is no treatment for blindness caused by Stargardt’s disease.
The biotech sector in Italy
AAVantgarde Bio’s Series A financing is an important milestone for the Italian company and biotechnology industry. To learn more about Italy’s thriving biotech landscape and some of the innovative companies making waves, you can explore this article.