Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today announced early clinical findings, including the first neurocognitive results, from its ongoing proof-of-concept (PoC) study of OTL-201, an investigational hematopoietic stem cell (HSC) gene therapy being developed for the treatment of mucopolysaccharidosis type IIIA (MPS-IIIA), also known as Sanfilippo syndrome type A.
December 12, 2022
· 12 min read