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Homology Medicines Announces Presentations on HMI-203 Investigational Gene Therapy for Hunter Syndrome and Broad Applicability of AAVHSC Platform for Lysosomal Storage Disorders at the 18th Annual WORLDSymposium™ Meeting

- Data Supported juMPStart Trial Evaluating One-Time, Systemic Administration of HMI-203 in ERT-Treated Adults - - Details of HMI-203 Clinical Trial Design and Encouraging Preclinical Data. | February 10, 2022 ....

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Homology Medicines Announces First Presentation of Data with HMI-203 In Vivo Gene Therapy Development Candidate for Hunter Syndrome

This Year -
BEDFORD, Mass., Feb. 08, 2021 (GLOBE NEWSWIRE) Homology Medicines, Inc. (NASDAQ:FIXX), a clinical-stage genetic medicines company, announced today the first scientific presentation of data from IND-enabling studies with its HMI-203 gene therapy development candidate for Hunter syndrome (MPS II). The results from Homology s
in vivo approach demonstrated the potential of HMI-203 to address peripheral manifestations in the murine disease model and showed that HMI-203 crossed the blood-brain-barrier following a single I.V. administration. These data will be presented during a poster presentation at the 17
th Annual WORLD
in vivo gene therapy program for metachromatic leukodystrophy (MLD).
By leveraging our family of AAVHSC vectors to target both peripheral organs and the central and peripheral nervous systems in preclinical models of Hunter syndrome and MLD, debilitating diseases that have high unmet medical need, we also demonstrated the continue ....

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