Nonprofit organizations are playing a big role in developing treatments for rare diseases, especially when big companies aren’t interested. Gene therapies are a key part of this, as they can tackle the root cause of many rare diseases. While gene therapies have had a tough time getting investment lately, nonprofits can help keep this field alive. One example is Fondazione Telethon (FT) in Italy, which developed the first gene therapy approved for Wiskott-Aldrich Syndrome. To keep this treatment available, FT plans to sell it and use the money to fund more research. Another nonprofit, Généthon in France, is working on 12 gene therapies for diseases like muscular dystrophy and blood disorders. They also work on improving how these therapies are made and studied. Nonprofits often partner with biotech and pharmaceutical companies to raise money for late-stage research and getting treatments to patients. For instance, Généthon’s research led to the development of Zolgensma, a gene therapy for spinal muscular atrophy, which was later approved and sold by Novartis. Frédéric Revah, CEO of Généthon, talked about how being a nonprofit allows them to take risks and focus on treatments that big companies might ignore. They get most of their funding from public donations and deals with pharmaceutical companies. Généthon is currently testing a gene therapy for Duchenne muscular dystrophy (DMD), called GNT0004. Early results show it’s safe and effective, with patients showing improved muscle function and reduced muscle damage. They plan to submit this therapy for approval in Europe in 2027. Revah also discussed how nonprofits can help patients access treatments, even for ultra-rare diseases where traditional approval processes might not make sense. He suggested that clinical trials could be used to provide access, with costs covered by public funds. This approach is starting to gain support in Europe. In France, doctors can apply for ‘Compassionate Access’ to get treatments not yet on the market, with the cost covered by regulators if approved.