ORTX

Orchard Therapeutics Plc

A biotechnology company that makes one-time gene therapies for rare, often fatal genetic diseases. Its approach takes a patient's own blood stem cells, fixes a faulty gene outside the body, and returns the corrected cells to the patient. Its flagship treatment, Libmeldy (Lenmeldy in the US), targets metachromatic leukodystrophy, a rare nerve-destroying disorder — the name echoes the disease's initials. Founded in 2015 by gene-therapy pioneers, the company was acquired by Japanese drugmaker Kyowa Kirin in 2024.

Orchard Therapeutics plc ADR