Lawyers
Intellectual property partner Jonathan Ashtor and counsel Hilary Smith discussed Paul, Weiss’s novel collaboration with parent-formed patient advocacy nonprofit FOXG1 Research Foundation (FRF) in an interview with Managing IP. In the article, Jonathan and Hilary describe how Paul, Weiss has supported FRF’s groundbreaking efforts over the past five years to raise funds supporting the development of a gene therapy for FOXG1 syndrome—an ultra-rare neurodevelopmental disorder caused by a mutation in the FOXG1 gene—and initiate a clinical trial to evaluate the potential treatment. During that time, Paul, Weiss worked with FRF to craft a unique business model that relied on university collaborations and donations as funding sources rather than traditional pharmaceutical investment, with IP rights playing a central role in these efforts.
This approach has helped FRF avoid following the traditional drug development model, which is not necessarily the best fit for ultra-rare diseases. In some cases, the small number of affected patients means revenue opportunities may not match investors’ cost and risk of development, Jonathan and Hilary say. “The issue with treatments for rare diseases is that they get shelved and deprioritized. They may be promising, but the economics just aren’t there, leaving patients without access to these treatments,” Jonathan says. “By funding all this research and structuring it in a way that FRF has rights over IP, they are able to maintain a level of control that enables them to sponsor the clinical trial.”
» read the article (subscription required)
Related Insights
