FDA approves LENMELDY, the first gene therapy for pediatric metachromatic leukodystrophy (MLD), a rare genetic disease.

Approximately 1 in 40,000 individuals in the U.S. are affected by MLD.

LENMELDY is a one-time, single-dose infusion made from the patient's own hematopoietic stem cells.

Orchard Therapeutics' CEO Bobby Gaspar views LENMELDY as a groundbreaking treatment offering new hope for children with early-onset MLD.