Orchard Therapeutics Announces First Patient Dosed with OTL-201 Gene Therapy in Proof-of-Concept Clinical Trial for Sanfilippo Syndrome (MPS-IIIA)
Orchard Therapeutics plc - American Depositary Shares (ORTX)
Last orchard therapeutics plc - american depositary shares earnings: 2/27 07:00 am
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Source: GlobeNewswire
BOSTON and LONDON, April 27, 2020 (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today announced that the first patient has been dosed in an open-label, proof-of-concept investigational study of OTL-201, an ex vivo autologous hematopoietic stem cell (HSC) gene therapy for the treatment of mucopolysaccharidosis type IIIA (MPS-IIIA). The study is designed to evaluate safety, tolerability and clinical efficacy and is intended to enroll up to five patients between three months and 24 months of age who will be followed for three years. The study also contains a number of key secondary outcome measures such as overall survival, cognition and behavior to help inform future clinical development of HSC gene therapy in this indication. MPS-IIIA, also known as Sanfilippo syndrome type A, is a rare, inherited neurometabolic disorder caused by genetic mutations that leads to the buildup of sugar molecules called mucopolysaccharides in the body, result
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