A future going dark. A company racing to light it.
At Forbion, we support companies advancing transformative science and work alongside them as they pursue meaningful, real-world impact. Our role extends beyond capital. We bring strategic experience, specialist networks and long-term partnership to help ambitious teams navigate the path from scientific innovation to potential new medicines.
Our Portfolio Company Spotlight Series brings that work into view: the challenges our portfolio companies are addressing, the science behind their approach and the people leading their progress.
In Episode 2, Natalia Misciattelli, PhD, CEO of AAVantgarde, introduces how the company is working to transform the prospects for people living with inherited retinal diseases.
For those affected, the progressive loss of sight reshapes every aspect of life. Despite this, many inherited retinal diseases still have no effective treatment. AAVantgarde is addressing this unmet need through next-generation gene therapies for Usher syndrome type 1B (USH1B) and Stargardt disease. Both conditions are associated with mutations in genes that are too large for conventional adeno-associated virus (AAV) vectors. AAVantgarde’s proprietary dual-hybrid and intein platforms are designed to overcome this limitation by dividing and reconstituting large therapeutic genes or proteins within retinal cells.
AAVantgarde is the first company to clinically evaluate a dual-AAV approach in the retina, establishing it as a leader in retinal large-gene delivery. Natalia discusses this clinical differentiation and the encouraging safety profile and early efficacy signals observed in the company’s emerging dataset. She also looks ahead to the work required to translate that progress into potential treatments: rigorous clinical execution, reaching enrolment milestones, analysing datasets and building the manufacturing capabilities needed to support future development and supply.
Partnership is an important part of that journey. In this interview, Natalia sheds light on the relationship the company has with its investors, including Forbion.
For us, as one of the world’s leading life sciences venture capital firms, this is what active partnership means: supporting exceptional teams and transformative science while helping companies build the capabilities needed to convert innovation into potential patient impact.