Gene therapies for rare diseases are under threat. Scientists hope to save them

It was one of gene therapy’s greatest successes — and greatest disappointments.

Strimvelis is a potent treatment for a devastating genetic disorder of the immune system and one of the first gene therapies to be approved in Europe. But in 2022, the company that sold it announced that it could no longer afford to do so: Strimvelis was highly effective at treating disease, but not at turning a profit.

Now it is getting a second chance. The Italian charity Telethon Foundation in Milan has announced that it will produce Strimvelis itself. “We are making a paradigm shift,” said Francesca Pasinelli, general manager of the Telethon Foundation, in a 12 September statement announcing the decision. “We are the first non-profit organization to take on the commercialization of a gene therapy.”

The charity’s unprecedented scheme offers hope to scientists who have spent decades developing gene therapies for rare disorders, only to find that the standard way of getting those treatments to people in need — licensing them to for-profit companies — is unavailable. Other unorthodox ways to keep these efforts going...