Finance : Homology Medicines' Gene Therapy For Phenylketonuria Found Safe, Efficacy Data Mixed

Bedford, Massachusetts-based Homology announced initial Phase 1/2 data from the pheNIX trial that evaluated its HMI-102 gene therapy in patients with phenylketonuria, with safety data showing that HMI-102 was well-tolerated. The efficacy data was mixed, with the Cohort 1 patients not showing a reduction in phenylalanine through weeks 10 and 12. Additionally, the first patient dosed in Cohort 2 also showed increases in tyrosine levels of 72% and 85% at weeks one and four, respectively, which suggests increased PAH enzyme activity.

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December 18, 2019 at 08:53PM