Orchard Therapeutics Announces First Patient Randomized in Registrational Trial of OTL-203 for MPS-I Hurler Syndrome

February 5, 2024

https://www.globenewswire.com/news-release/2024/02/05/2823377/0/en/Orchard-Therapeutics-Announces-First-Patient-Randomized-in-Registrational-Trial-of-OTL-203-for-MPS-I-Hurler-Syndrome.html

Gene therapy being developed as a one-time treatment to address the underlying cause of the disease by inserting a functional copy of the human IDUA gene into a patient’s own hematopoietic stem cells

Read more at globenewswire.com

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