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Orchard Therapeutics

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United Kingdom

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CPhI India 2021

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Orchard Therapeutics Unveils Details on New HSC Gene Therapy Research Programs as Part of R&D Investor Event

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Lead Product(s): OTL-204

Therapeutic Area: Neurology Product Name: OTL-204

Highest Development Status: Preclinical Product Type: Cell and Gene therapy

Partner/Sponsor/Collaborator: Not Applicable

Deal Size: Not Applicable Upfront Cash: Not Applicable

Deal Type: Not Applicable November 12, 2020

Details:

OTL-204 for GRN-FTD: Orchard’s preclinical program in GRN-FTD seeks to introduce a working copy of the GRN gene into HSCs, which can differentiate into microglia and secrete progranulin in the central nervous system, potentially correcting the underlying cause of the disease.