Homology Medicines Initiates Phase 1/2 Study for HMI-102 Gene Therapy Candidate for Adults with PKU and Expects Initial Clinical Results by Year End 2019
Homology Medicines, Inc. (FIXX)
Last homology medicines, inc. earnings: 3/12 04:10 pm
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Source: GlobeNewswire
- Enrollment in the pheNIX Study Marks First Gene Therapy Clinical Trial for PKU - BEDFORD, Mass., June 10, 2019 (GLOBE NEWSWIRE) -- Homology Medicines, Inc. (Nasdaq: FIXX), a genetic medicines company, announced today that it has commenced enrollment of the Phase 1/2 pheNIX trial for HMI-102, a one-time gene therapy development candidate for the treatment of adults with phenylketonuria (PKU). The pheNIX study is designed to evaluate the safety and efficacy of the investigational gene therapy in a randomized, concurrently-controlled, dose-escalation study. PKU is an inborn error of metabolism caused by a mutation in the PAH gene that results in a potentially toxic buildup of phenylalanine (Phe), an essential amino acid derived primarily from dietary protein. “Our early work with the clinical sites has enabled us to move expeditiously from receiving
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