FDA Backs Gene Therapy for Fatal Neurodegenerative Childhood Disease

DA Backs Gene Therapy Giant for Rare Childhood Disease

Orchard Therapeutics has received a fourth Rare Pediatric Disease Designation from the FDA for its gene therapy candidate for metachromatic leukodystrophy.

Orchard Therapeutics, based in London, has received a Rare Pediatric Disease Designation form the FDA for its gene therapy OTL-200. The treatment is for patients with metachromatic leukodystrophy (MLD), a fatal genetic disease that causes sulfur to accumulate in the body, harming the nervous system and metabolism.

Last year, the company raised £85M (€96M) in London’s largest biotech fundraising of the year to support the development of its treatment for ADA-SCID, a rare inherited immune disorder. With the fresh card, Orchard recently bought GSK’s rare disease portfolio of gene therapies — including Strimvelis, GSK’s gene therapy for ADA-SCID.

Gene therapies could be effective in treating rare genetic conditions for which there is currently no cure. However, they still face issues connected with safety and pricing, which may have led GSK to sell its portfolio. Especially after a controversial Goldman Sachs report proposed that curing patients is not a profitable business model.


Image by vchal/Shutterstock

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