Homologous Drugs (NASDAQ: repair) expected to provide Preliminary data from the mid-2023 pheEDIT trial of HMI-103 in phenylketonuria (PKU). There are nine active clinical trial sites, with additional sites expected to be launched throughout 2023.
The juMPStart Phase 1 gene therapy trial for Hunter Syndrome (MPS II) has five clinical sites in the US and Canada, with additional sites expected to start, with preliminary data expected in the second half of 2023.
Homology shared new preclinical data supporting the inclusion of immunosuppressive regimens in the pheEDIT and juMPStart trials.
related: Homology Medicines Pauses Recruitment for Rare Genetic Disease Study.
In nonhuman primates (NHPs), use of prophylactic T cell inhibitors and steroids reduced neutralizing antibody responses to AAVHSC vectors compared to NHPs not receiving the regimen and NHPs receiving each drug alone Increases mRNA expression.
Homology Shares Preclinical Efficacy Data for Gene Editing Candidate HMI-103 Aimed at Integration Using Homologous Recombination BioSpace PAHs Gene and liver-specific promoters were integrated into the genome and maximized PAH expression in all transduced hepatocytes.
The murine surrogate of HMI-103 was ten-fold more potent than the non-integrating gene therapy vector HMI-102 in a preclinical PKU model.
In 2023, Homology plans to advance its gene medicines pipeline, including an IND-enabling study of HMI-104, a one-time GTx-mAb candidate for paroxysmal nocturnal hemoglobinuria.
The company is also focused on collaborating with the HMI-204 gene therapy candidate for the treatment of metachromatic leukodystrophy.
Price Action: FIXX shares closed up 8.33% at $1.30 on Wednesday.
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