Trial Profile

A Phase I-II, Study of Autologous CD34+ Haematopoietic Stem Cells Transduced Ex Vivo with CD11b Lentiviral Vector Encoding for Human SGSH in Patients with Mucopolysaccharidosis Type IIIA (MPS IIIa, Sanfilippo Syndrome Type A)

Status: Active, no longer recruiting

Phase of Trial: Phase I/II

Latest Information Update: 04 Apr 2025

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At a glance

30 Mar 2025 Planned End Date changed from 30 Oct 2024 to 30 Oct 2026.
02 Feb 2024 According to an Orchard Therapeutics media release, an updated neurocognitive and biochemical results from this proof-of-concept study will be featured at the 20th Annual WORLDSymposium™ taking place February 4-9, 2024, in San Diego, California.
15 May 2023 According to an Orchard Therapeutics media release, New and updated biochemical and early neurocognitive results will be presented at ASGCT will include follow-up of the earliest treated patients now exceeds 2.5 year.

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