Orchard Therapeutics Announces Publication in The Lancet of Long-term Clinical Outcomes with Libmeldy for the Treatment of Children with Early-onset MLD
Administration of one-time hematopoietic stem cell (HSC) gene therapy resulted in sustained, clinically meaningful benefits by preserving cognitive function and motor development in most patients
90% overall survival with up to 7.5 years of follow-up (median 3.2 years) in 29 patients
UCLB spinout and global gene therapy leader, Orchard Therapeutics has announced the publication in The Lancet of long-term clinical outcomes evaluating the safety and efficacy of Libmeldy® (atidarsagene autotemcel) for the treatment of early-onset metachromatic leukodystrophy (MLD). Libmeldy is the only approved one-time gene therapy intended to correct the underlying cause of MLD for eligible patients in the European Union, UK, Iceland, Liechtenstein and Norway. Also known as OTL-200, it is an investigational therapy in the U.S.