Orchard Therapeutics Announces Multiple Presentations from across its Late-stage Neurometabolic Portfolio at SSIEM 2024

Company Overview - Orchard Therapeutics, recently acquired by Kyowa Kirin, aims to accelerate the delivery of new gene therapies globally [1] - The company focuses on hematopoietic stem cell (HSC) gene therapy to treat genetic and severe diseases [23][24] - Orchard has a pipeline of HSC gene therapies targeting serious diseases with limited treatment options [24] Upcoming Presentations - Orchard will present nine studies from its late-stage neurometabolic HSC gene therapy portfolio at the SSIEM 2024 Annual Symposium in Porto, Portugal, from September 3-6 [1] - Key presentations include a proof-of-concept study on OTL-203 for Hurler syndrome and an encore presentation on atidarsagene autotemcel (Lenmeldy) for metachromatic leukodystrophy (MLD) [2][3] Specific Therapies - OTL-203 is an investigational therapy for the Hurler subtype of mucopolysaccharidosis type I (MPS-IH), currently in a randomized clinical trial [22] - Lenmeldy (atidarsagene autotemcel) is the only approved therapy in the U.S. for treating children with pre-symptomatic and early-symptomatic forms of MLD [5][7] Regulatory Designations - OTL-203 has received Rare Pediatric Disease and Fast Track designations from the U.S. FDA, as well as PRIME status from the EMA [22] - Lenmeldy has been approved in Europe as Libmeldy and is recognized by various regulatory agencies [6]