Homology Medicines Provides Update From Gene Therapy Trials

Homology Medicines Inc (NASDAQ:FIXX) expects to provide initial data from the pheEDIT trial of HMI-103 for phenylketonuria (PKU) in mid-year 2023. There are nine active clinical trial sites, with more expected to be initiated throughout 2023.
The juMPStart Phase 1 gene therapy trial for Hunter syndrome (MPS II) has five clinical sites in the U.S. and Canada, with more expected to be initiated, and initial data is expected in 2H of 2023.
Homology shared new preclinical data supporting the immunosuppression regimen incorporated in the pheEDIT and juMPStart trials.
Related: Homology Medicines Pauses Enrollment For Rare Inherited Disorder Study.
In non-human primates (NHPs), using a prophylactic T-cell inhibitor combined with steroids reduced the neutralizing antibody response to the AAVHSC vector and increased mRNA expression, compared to NHPs not receiving the regimen and to those receiving each agent alone.
Homology shared preclinical potency data for a gene-editing candidate, HMI-103, designed to use homologous recombination to integrate the PAH gene and a liver-specific promoter into the genome and to maximize PAH expression in all transduced liver cells.
In the preclinical PKU model, the murine surrogate of HMI-103 was ten times more potent than the non-integrating gene therapy vector HMI-102.
In 2023, Homology plans to progress its pipeline of genetic medicines, including conducting IND-enabling studies of HMI-104, a one-time GTx-mAb candidate for paroxysmal nocturnal hemoglobinuria.
The company is also focused on efforts to partner the HMI-204 gene therapy candidate for metachromatic leukodystrophy.
Price Action: FIXX shares closed higher by 8.33% at $1.30 on Wednesday.

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