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Homology Medicines Initiates Clinical Trial for HMI-203, a One-Time Investigational Gene Therapy Candidate for Adults with MPS II (Hunter Syndrome)
- juMPStart Trial to Evaluate First Systemic Gene Therapy Candidate for Patients with Hunter Syndrome - BEDFORD, Mass., Oct. 18, 2021 (GLOBE NEWSWIRE) --
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[{"type":"text","content":"- juMPStart Trial to Evaluate First Systemic Gene Therapy Candidate for Patients with Hunter Syndrome - BEDFORD, Mass., Oct. 18, 2021 (GLOBE NEWSWIRE) -- Homology Medicines, Inc. (Nasdaq: FIXX), a genetic medicines company, announced today that it has initiated the Phase 1 trial for HMI-203, a one-time, in vivo gene therapy candidate for the treatment of adults with mucopolysaccharidosis type II (MPS II), or Hunter syndrome. The juMPStart trial is an open-label, dose-escalation study evaluating the safety and efficacy of a single intravenous (I.V.) administration of HMI-203. Hunter syndrome is a lysosomal storage disorder caused by mutations in the iduronate 2-sulfatase (IDS) gene leading to absent or deficient I2S enzymatic activity, which causes toxic lysosomal accumulation of glycosaminoglycans (GAGs). There are no treatments currently available that address both the peripheral organ and cognitive manifestations of the disease, and there remains a high unmet medical need for patients despite the availability of enzyme replacement therapy (ERT). “We are excited to start the Phase 1 trial for HMI-203, our investigational gene therapy for Hunter syndrome, as our team remains dedicated to improving outcomes for the patient community since our prior work developing ERT,” said Arthur Tzianabos, Ph.D., President and Chief Executive Officer of Homology Medicines. “With today’s milestone, we have also accomplished our goal of having three clinical programs by the end of 2021, with our gene therapy and gene editing clinical trials for PKU and the initiation of the juMPStart trial for Hunter syndrome. We are executing on this and our other programs, and we look forward to our continued collaboration with the MPS II community.” The juMPStart trial is expected to enroll up to 9 male patients ages 18-30 years old who have been diagnosed with Hunter syndrome and are currently receiving ERT. In addition to safety endpoints, the trial plans to measure plasma I2S activity, urinary GAG levels and other peripheral disease manifestations. The Phase 1 dose-escalation portion of the trial is designed to evaluate three doses of HMI-203 to potentially determine the optimal dose(s) for a future trial. “I am pleased to work with the Homology team, as we both have a long-held and shared commitment to the MPS II community and a focus on bringing forward ...