Homology Medicines Presents on Design of pheEDIT Trial Evaluating One-Time Nuclease-Free Gene Editing Candidate HMI-103 for PKU at American Society of Human Genetics Meeting
- Additional Presentation Focused on Use of Single-Molecule, Modified Base Sequencing to Support Vector Design -
“The pheEDIT dose-escalation clinical trial is the first gene editing study for PKU, and investigational HMI-103 has the potential to treat adult and pediatric PKU with its unique dual mechanism of action designed to integrate the PAH gene and liver-specific promoter into the genome and to maximize PAH expression in all transduced liver cells,” said
The poster presentation titled, “A Phase 1, Open-Label, Dose-Escalation Study to Evaluate the Safety and Efficacy of HMI-103, a One-Time Gene-Editing Vector in Adult Participants with Classical PKU Due to PAH Deficiency,” outlined the design of the pheEDIT trial, which is:
- A Phase 1, open label, sequential dose-escalation trial evaluating safety and efficacy of a single I.V. administration of HMI-103, including tolerability and Phe levels;
- Designed to enroll three dose cohorts with up to three patients with uncontrolled PKU in each cohort; and
- Incorporating a steroid-sparing prophylactic immunosuppression regimen, including a T-cell inhibitor, to temporarily dampen potential immune response to the capsid.
A second presentation titled, “Single-Molecule, Modified Base Sequencing to Identify Frequency and Cause of rAAV Vector Breakpoints,” demonstrated that use of next-generation sequencing can lead to improved vector design by identifying the locations and frequency of potential breakpoints that can be addressed by optimizing the vectors.
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This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including, without limitation, statements regarding: the potential, safety, efficacy, and regulatory and clinical progress of our product candidates; the potential of our gene therapy and gene editing platforms, including our GTx-mAb platform; our plans and timing for the release of additional preclinical and clinical data; and our position as a leader in the development of genetic medicines. These statements are neither promises nor guarantees, but involve known and unknown risks, uncertainties and other important factors that may cause our actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements, including, but not limited to, the following: the impact of the COVID-19 pandemic on our business and operations, including our preclinical studies and clinical trials, and on general economic conditions; we have and expect to continue to incur significant losses; our need for additional funding, which may not be available; failure to identify additional product candidates and develop or commercialize marketable products; the early stage of our development efforts; potential unforeseen events during clinical trials could cause delays or other adverse consequences; risks relating to the regulatory approval process; interim, topline and preliminary data may change as more patient data become available, and are subject to audit and verification procedures that could result in material changes in the final data; our product candidates may cause serious adverse side effects; inability to maintain our collaborations, or the failure of these collaborations; our reliance on third parties, including for the manufacture of materials for our research programs, preclinical and clinical studies; failure to obtain
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Source: Homology Medicines, Inc.