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Homology Medicines Announces Presentations on Design of Ongoing Gene Editing and Gene Therapy Trials for PKU and Hunter Syndrome at ACMG Annual Clinical Genetics Meeting
BEDFORD, Mass., March 15, 2023 (GLOBE NEWSWIRE) -- Homology Medicines, Inc. (Nasdaq: FIXX), a genetic medicines company, announced two presentations during
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[{"type":"text","content":"BEDFORD, Mass., March 15, 2023 (GLOBE NEWSWIRE) -- Homology Medicines, Inc. (Nasdaq: FIXX), a genetic medicines company, announced two presentations during the ACMG Annual Clinical Genetics Meeting, which highlight the preclinical data that supported initiation of the Company’s Phase 1 pheEDIT gene editing trial for phenylketonuria (PKU) and the Phase 1 juMPStart gene therapy trial for Hunter syndrome (MPS II). Additionally, the presentations feature trial design, including the targeted immunosuppression regimen incorporated in both studies. The pheEDIT-focused presentation also includes preclinical potency data with HMI-103, the nuclease-free, in vivo, gene editing candidate for PKU. “Our ongoing clinical trials are supported by robust preclinical data packages that informed the design and selected dose levels. Additionally, data on the immunosuppression regimen from non-human primates demonstrated the ability to temporarily dampen the immune response, which could potentially enhance expression following vector administration,” stated Albert Seymour, Ph.D., President and Chief Executive Officer of Homology Medicines. “Related to the pheEDIT trial for PKU, data presented at ACMG showed the murine surrogate of HMI-103 was ten times more potent at reducing blood Phe levels as compared to non-integrating gene therapy vector HMI-102, which we believe relates to its dual mechanism of action of integration and episomal expression as well as additional optimizations we made to the candidate. We remain on track to share initial pheEDIT clinical data by mid-year, and anticipate sharing initial juMPstart clinical data in the second half of 2023.” Homology’s poster presentation details are as follows: Title: pheEDIT: A Phase 1, Open-Label, Dose-Escalation Safety and Efficacy Gene Editing Study Evaluating HMI-103 in Adults with Classical PKUDate and Time: Friday, March 17, 2023 at 10:30 a.m. MT Title: juMPStart: Phase 1, Open-Label, Dose-Escalation Safety and Efficacy Gene Therapy Study Evaluating HMI-203 in Adults with MPS IIDate and Time: Thursday, March 16, 2023 at 10:30 a.m. MT For more information, please visit the Publications and Presentations page on Homology’s website. About Homology Medicines, Inc.Homology Medicines, Inc. is a clinical-stage genetic medicines company dedicated to transforming the lives of patients suffering from r...