WebMar 18, 2024 · A single dose of HMI-102 — a candidate gene therapy for phenylketonuria (PKU) by Homology Medicines — lowered blood levels of phenylalanine and kept this … WebApr 16, 2024 · Subject was previously administered HMI 102. Subject is able to understand the purpose and risks of the study and is willing to provide informed consent. Subject is …
Homology Medicines Announces Presentations on HMI-203
WebApr 10, 2024 · HMI-102 is an investigational gene therapy in clinical development for the treatment of phenylketonuria (PKU) in adults. HMI-102 is designed to encode the PAH gene, which is mutated in people with PKU, and delivered via the liver-tropic AAVHSC15 vector. Homology has received Fast Track Designation and orphan drug designation for HMI … WebHMI-102 - Ph 1/2 Trial * Phase 2: HMI-102 was granted Orphan Drug Designation and Fast Track Designation by the U.S. Food and Drug Administration and Orphan Drug … hsa with a ppo
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WebAbout HMI-102 Gene Therapy and HMI-103 Gene Editing Product Candidates HMI-102 is an investigational gene therapy in clinical development for the treatment of phenylketonuria (PKU) in adults. HMI-102 is designed to encode the PAH gene, which is mutated in people with PKU, and delivered via the liver-tropic AAVHSC15 vector. WebMar 13, 2024 · Single administration of AAVHSC15-PAH (HMI-102) produced a sustained reduction in phenylalanine (Phe), the key biomarker in management of PKU, and an increase in tyrosine, a Phe metabolite and precursor to neurotransmitters, over the lifespan of the murine model. Brain Phe, 5-HIAA, and coat color normalized, further indicating … WebMar 13, 2024 · Single administration of AAVHSC15-PAH (HMI-102) produced a sustained reduction in phenylalanine (Phe), the key biomarker in management of PKU, and an … hobby art gallery singapore