Struggling for a Cure: The Financial Roadblocks of Gene Therapy for Rare Diseases

Robin Alderman's quest for a gene therapy to cure her son Camden's rare immune deficiency has faced significant financial hurdles. London-based Orchard Therapeutics ceased its experimental treatment for Wiskott-Aldrich syndrome, leaving many families in despair. Worldwide, millions suffer from rare genetic diseases, yet financial incentives for treatments are lacking.

Robin Alderman faces a grim reality: gene therapy might cure her son Camden's rare genetic immune deficiency, but it remains out of reach.

In 2022, London-based Orchard Therapeutics halted its experimental treatment for Wiskott-Aldrich syndrome, a decision that left many families grappling for alternatives. For Robin, who has been her 21-year-old son’s advocate since birth, the news was devastating.

Approximately 350 million people worldwide are affected by rare diseases, most of which are genetic. But with each condition affecting only a tiny fraction of the population, commercial incentives for one-time gene therapy solutions are scant. Families like the Aldermans find themselves turning to DIY fundraising for potential cures, often with slim chances of success.