Alessandro Aiuti, MD, PhD, on Durable, Clinically Meaningful Efficacy of Arsa-Cel in Metachromatic Leukodystrophy


The deputy director, clinical research, San Raffaele Telethon Institute for gene therapy, discussed long-term follow-up data of up to 12 years.

“This represents the proof of principle that ex vivo lentiviral gene therapy into stem cells can correct a lysosomal storage disorder and this has been further applied to mucopolysaccharidosis type one... and could be applicable to other lysosomal storage disorders and we're working on new diseases to bring this approach of enzyme overexpression correction to."

Atidarsagene autotemcel (arsa-cel) shows a favorable benefit-risk profile with clinically meaningful, durable reductions in disease progression in patients with early-onset metachromatic leukodystrophy (MLD). Long-term data from across 2 prospective clinical trials (n=30) and expanded access frameworks (n=9), comprising up to 12 years of follow-up, were presented at the American Society of Gene & Cell Therapy (ASGCT) 27th Annual Meeting, held May 7 to 10, 2024, in Baltimore, Maryland, by Alessandro Aiuti, MD, PhD, deputy director, clinical research, and head, Clinical Research Unit, San Raffaele Telethon Institute for gene therapy, and Group leader, Pathogenesis and therapy, primary immunodeficiencies unit, and full professor, Università Vita Salute San Raffaele, and chief of clinic, Pediatric Immunohematology Unit, San Raffaele.1

CGTLive® spoke with Aiuti to learn more about the updated data what it means for the field. He also discussed other investigations ongoing with arsa-cel for MLD as well as for other diseases, including mucopolysaccharidosis type 1. Arsa-cel was approved by the FDA under the name Lenmeldy in March 2024.2

1. Calbi V, Fumagalli F, De Mattia F, et al. AtidarsageneAutotemcel (Hematopoietic Stem Cell GeneTherapy) Preserves Cognitive and Motor Development in Metachromatic Leukodystrophy with up to 12 Years Follow-Up. Presented at: ASGCT 27th Annual Meeting, May 7-10; Baltimore, Maryland. Abstract #1
2. FDA Approves First Gene Therapy for Children with Metachromatic Leukodystrophy. News release. Orchard Therapeutics. March 18, 2024. Accessed March 18, 2024.
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