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 –

Excerpt from the Press Release:

BEDFORD, Mass., Oct. 27, 2022 (GLOBE NEWSWIRE) — Homology Medicines, Inc. (Nasdaq: FIXX), a genetic medicines company, announced today a presentation on the design of pheEDIT, a Phase 1, open-label, dose-escalation study evaluating one-time gene editing candidate HMI-103 in adults with phenylketonuria (PKU). The presentation included preclinical data demonstrating efficacy in a PKU murine model and precision of editing in a humanized murine model. During the American Society of Human Genetics (ASHG) Annual Meeting, the Company also presented data that demonstrated single-molecule, modified base sequencing can aid in the characterization, design and optimization of AAV vectors.

“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 Albert Seymour, Ph.D., President and Chief Executive Officer of Homology Medicines. “The program’s preclinical data showed long-term Phe normalization in the PKU model following a single I.V. administration, and a genome-wide integration assay demonstrated the precision of homologous recombination with no off-target integration in a humanized liver model. Together, these data supported the initiation of pheEDIT and highlight our commitment to leading the field with an unbiased approach to evaluating on-and off-target integrations.”

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;

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