Gene Therapies for Rare Diseases Are Under Threat. Scientists Hope to Save Them

October 6, 2023

(Nature) – 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. (Read More)