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Homology Medicines Announces World’s First Gene Editing Clinical Trial for PKU
- IND Clearance of pheEDIT Study to Evaluate a One-Time Dose of Investigational HMI-103 Incorporating a Novel Nuclease-Free Gene Editing Approach - - Company
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[{"type":"text","content":"- IND Clearance of pheEDIT Study to Evaluate a One-Time Dose of Investigational HMI-103 Incorporating a Novel Nuclease-Free Gene Editing Approach - - Company Provides Update on Enrollment in Ongoing pheNIX Trial of HMI-102 Gene Therapy for Adults with PKU - BEDFORD, Mass., Oct. 12, 2021 (GLOBE NEWSWIRE) -- Homology Medicines, Inc. (Nasdaq: FIXX), a genetic medicines company, announced today the pheEDIT Phase 1 clinical trial for HMI-103, a one-time, in vivo product candidate that utilizes a gene editing approach for phenylketonuria (PKU), based on the Investigational New Drug Application (IND) clearance from the U.S. Food and Drug Administration (FDA). HMI-103 will be the world’s first gene editing candidate for PKU to enter clinical trials from Homology’s dual gene therapy and gene editing technology platform, and with the launch of pheEDIT Homology moves closer to its goal of offering solutions for both adults and pediatric patients with PKU. “Today’s milestone is the culmination of our team’s tireless work to translate our gene editing technology from an academic discovery into a clinical program for people with PKU,” said Albert Seymour, Ph.D., Chief Scientific Officer of Homology Medicines. “Our positive preclinical data in the PKU model demonstrated phenotypic correction, and the precision of HMI-103 genome integration was confirmed in a humanized liver model, which showed no evidence of off-target mutations or unwanted on-target changes to the genome. These nonclinical data give us great confidence in initiating our Phase 1 pheEDIT trial, and we look forward to continuing to work with the PKU community on our clinical programs.” The HMI-103 pheEDIT trial is expected to enroll up to nine patients ages 18-55 years old who have been diagnosed with PKU due to PAH deficiency. Once positive safety and efficacy results are established in the adult population, Homology plans to enroll younger patients in clinical trials. The Phase 1 dose-escalation trial is designed to evaluate three doses of HMI-103 to determine the recommended dose(s) for a future trial. In addition to safety endpoints, the trial will measure serum phenylalanine (Phe) changes. HMI-103 is designed as a one-time administration to maximize the expression of functional phenylalanine hydroxylase (PAH) in liver cells and thus restore the natural biochemical pathway t...