Business
Homology Medicines Reports Fourth Quarter and Full Year 2022 Financial Results and Recent Highlights
- On Track to Provide Initial Clinical Data from Gene Editing Trial for PKU Mid-Year with First Participant Dosed and Others in Screening - - Initial Clinical
About this update from Q32 Bio Inc.
[{"type":"text","content":"- On Track to Provide Initial Clinical Data from Gene Editing Trial for PKU Mid-Year with First Participant Dosed and Others in Screening - - Initial Clinical Data from Gene Therapy Trial for Hunter Syndrome Anticipated in Second Half of 2023; Trial Recruiting in the U.S. and Canada - - Anticipated Cash Runway Into Fourth Quarter 2024 Enables Execution Against Key Milestones - BEDFORD, Mass., March 09, 2023 (GLOBE NEWSWIRE) -- Homology Medicines, Inc. (Nasdaq: FIXX), a genetic medicines company, announced today financial results for the fourth quarter and full year ended December 31, 2022, and highlighted recent accomplishments. “We entered 2023 with strong momentum across our gene editing and gene therapy clinical trials for PKU and Hunter syndrome, and we anticipate initial data read-outs from both programs this year,” said Albert Seymour, Ph.D., President and Chief Executive Officer of Homology Medicines. “Dosing of the first patient in the pheEDIT trial for PKU marked a key milestone for Homology, and the forthcoming data will represent the first gene editing data in PKU patients. We believe the continued interest in the juMPStart trial for Hunter syndrome underscores the impact a one-time gene therapy with potential to address both the peripheral and CNS disease manifestations could have for patients and their loved ones.” Continued Dr. Seymour, “We recently shared a series of data that support these programs, including preclinical work on the targeted immunosuppression regimen being utilized in both pheEDIT and juMPStart. We also unveiled details of our optimized MLD gene therapy candidate, demonstrating its ability to cross the blood-brain-barrier in the disease model with enzyme activity expected to lead to in vivo efficacy. The third arm of our platform, GTx-mAb, continues to advance as we move HMI-104 for PNH through IND-enabling studies. We are well-positioned to execute against key milestones with anticipated funding into the fourth quarter of 2024.” Fourth Quarter 2022 and Recent Accomplishments Dosed first participant in the Phase 1 pheEDIT clinical trial evaluating in vivo nuclease-free gene editing candidate HMI-103 in adults with phenylketonuria (PKU); additional participants are in screening. Homology continues to expect initial clinical data from the trial mid-year 2023. Shared preclinical data that showed mur...