Gene Therapy May Cure Rare Diseases. But Drugmakers Have Few Incentives, Leaving Families Desperate ...Middle East

Written by LAURA UNGAR Robin Alderman faces an agonizing reality: Gene therapy might cure her son Camden’s rare, inherited immune deficiency. But it’s not available to 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 join. “We feel like we are the forgotten,” said Alderman, who’s advocated for her 21-year-old son since he was a baby. Collectively, about 350 million people worldwide suffer from rare diseases, most of which are genetic. But each of the 7,000 individual disorders affects perhaps a few in a million people or less. There’s little commercial ince

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