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Homology Medicines Reports Second Quarter 2021 Financial Results and Recent Highlights
- On Track to Report Initial Results from Phase 2 pheNIX Gene Therapy Trial for Adults with PKU in 2021- - Plans to Initiate First-Ever Gene Editing Trial for
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[{"type":"text","content":"- On Track to Report Initial Results from Phase 2 pheNIX Gene Therapy Trial for Adults with PKU in 2021- - Plans to Initiate First-Ever Gene Editing Trial for PKU with HMI-103 This Year - - Expects to Commence Clinical Trial with HMI-203 In Vivo Gene Therapy Candidate for MPS II by Year End - - Named Clinical Development Candidate from GTx-mAb Platform, HMI-104 - BEDFORD, Mass., Aug. 12, 2021 (GLOBE NEWSWIRE) -- Homology Medicines, Inc. (Nasdaq: FIXX), a clinical-stage genetic medicines company, announced today financial results for the second quarter ended June 30, 2021, and highlighted recent accomplishments. “We continue to demonstrate the breadth and depth of our genetic medicines platform with three clinical programs expected this year, which span in vivo gene therapy and gene editing trials for rare liver and CNS-based diseases,” stated Arthur Tzianabos, Ph.D., President and CEO of Homology Medicines. “With additional data from our gene therapy trial and the start of our first gene editing trial, both of which are focused on developing one-time treatments for PKU and are expected this year, we will be closer to our goal of helping adults and children living with this challenging inherited metabolic disorder. In addition, we remain on track to initiate our clinical trial for Hunter syndrome and we believe our single I.V. delivery may address both peripheral and CNS aspects of this disease.” Dr. Tzianabos added, “Our AAVHSC technology was expanded during the second quarter to include the launch of our GTx-mAb platform, a new approach using gene therapy to deliver, produce and distribute antibodies throughout the body. Preclinical proof-of-concept data gave us great confidence in selecting our C5 antibody development candidate for PNH, HMI-104, which uses another capsid from our AAVHSC portfolio. In conjunction with our C5 program, we developed a novel transfection system that has yielded up to 230 percent more vector than the current industry standard transfection system. Additional advances in our internal manufacturing platform were the subject of scientific presentations, including one describing our new formulation that will allow for a more convenient storage condition than frozen storage, which is a clear advantage for our clinical programs as well as potential commercial products.” Second Quarter 2021 and Recent Accom...