Gene therapy can cure rare diseases. But drug makers have little incentive, leaving families desperate

Robin Alderman faces a painful reality: gene therapy could cure her son Camden's rare hereditary immune deficiency. But it's not for him.

In 2022, London-based Orchard Therapeutics stopped investing in an experimental treatment for the condition, Wiskott-Aldrich syndrome. And there are no gene therapy studies he can participate in.

"We feel like we've been forgotten," said Councilwoman, who has been advocating for her 21-year-old son since he was a baby.

Collectively, approximately 350 million people worldwide suffer from rare diseases, most of which are genetic. But each of the 7,000 individual conditions may affect a few in a million people ...