Homology Medicines Announces Presentations at ACMG Annual Clinical Genetics Meeting

- Data From Company’s PKU and Hunter Syndrome Gene Therapy Programs Featured in Presentations -

BEDFORD, Mass., April 14, 2021 (GLOBE NEWSWIRE) -- Homology Medicines, Inc. (Nasdaq: FIXX), a clinical-stage genetic medicines company, announced today two presentations highlighting clinical and preclinical data from the Company’s phenylketonuria (PKU) and MPS II (Hunter syndrome) in vivo gene therapy programs, respectively, at the virtual American College of Medical Genetics and Genomics (ACMG) Annual Clinical Genetics Meeting.

“We continue to demonstrate the breadth of our growing pipeline with expanded focus on diseases of the central nervous system,” stated Albert Seymour, Ph.D., Chief Scientific Officer of Homology Medicines. “Our AAVHSC vectors have shown the ability to target peripheral organs and the central and peripheral nervous systems, and have crossed the blood-brain barrier in preclinical studies with a single I.V. dose of HMI-203, our gene therapy candidate for Hunter syndrome. We remain on track to initiate a Phase 1/2 dose-escalation clinical trial this year.”

In the poster titled, “The pheNIX Trial: First-In-Human Gene Therapy Trial for PKU Due to Phenylalanine Hydroxylase (PAH) Deficiency,” Olaf Bodamer, M.D., Ph.D., FACMG, FAAP, Park Gerald Chair in Genetics & Genomics and Associate Chief of Genetics & Genomics at Boston Children’s Hospital, and principal investigator of the pheNIX trial, presented results from the dose-escalation phase of the pheNIX trial, which is evaluating Homology’s lead gene therapy candidate, HMI-102, in adults with PKU. These data, previously presented at the New England Consortium of Metabolic Programs annual meeting in November, showed that HMI-102 was generally well-tolerated and resulted in marked reductions in phenylalanine (Phe) and the Phe-to-tyrosine (Tyr) ratio (Phe/Tyr ratio) at two doses. Recruitment is ongoing for the Phase 2 randomized, concurrently controlled, dose expansion phase of the pheNIX trial, with initial data expected this year.

In an additional poster titled, “HMI-203: Gene Therapy Development Candidate for Mucopolysaccharidosis Type II (MPS II), or Hunter Syndrome,” a single I.V. dose of HMI-203 in the adult murine model led to robust biodistribution and sustained human I2S (hI2S) enzyme expression, ameliorated paw deformities and also led to uptake of hI2S from the serum of the HMI-203-treated model in human cell lines. Importantly, these data, which were featured for the first time at the WORLDSymposium, demonstrated the potential for cell cross-correction.