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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)

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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: 07 Feb 2024

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At a glance

  • Drugs OTL 201 (Primary)
  • Indications Mucopolysaccharidosis III
  • Focus Adverse reactions; First in man; Proof of concept; Therapeutic Use

    • Most Recent Events

    • 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.
    • 03 May 2023 According to an Orchard Therapeutics media release, updated biochemical and early neurocognitive results from this trial will be presented at the 26th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT) 2023.

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