BIOCHEMISTRY, BIOMEDICINE & PHARMACEUTICS

    Homology Medicine Highlights Homology Medicine is a genetic medicine company  Utilizes proprietary gene editing and gene therapy technologies into novel treatments for patients with rare diseases.  Based on Positive Data, Advancing pheNIX Gene Therapy Clinical Trial for PKU to Dose Expansion Phase $60 Million Equity Investment from Pfizer Inc. - Unveiled In Vivo Gene Therapy Program for Hunter Syndrome Homology Medicine is a genetic medicine company translating proprietary gene editing and gene therapy technologies into novel treatments for patients with rare diseases. Homology Medicines has a broad, powerful, and proprietary gene editing and gene therapy platform, an experienced and dedicated team, and a commitment to bring new treatments and cures forward for the rare disease community.  Stock Symbol: FIXX Platform The proprietary human hematopoietic stem cell-derived adeno-associated virus vectors (AAVHSCs) to are used to deliver the treatment to patients...

Gene Therapy Clinical Study in Adult PKU (pheNIX, Phase 1/2) Detailed Description:   This study will evaluate the safety and efficacy of HMI-102 gene therapy in adult subjects with PKU due to PAH deficiency. Subjects will receive a single dose of HMI-102 administered intravenously. Up to 3 dose levels of HMI-102 may be investigated in this study. At a given dose level, a minimum of 2 subjects will be enrolled and dosed. The dosing of the first two subjects will be staggered. Following the evaluation of data from the first 2 subjects in a cohort, a decision can be made to either escalate to the next dose level or expand the cohort at the selected dose level. If the cohort is expanded, additional subjects will be randomized to receive HMI-102 or a concurrent delayed treatment control arm. Subjects in the delayed treatment control will be eligible to receive HMI-102 after 24 weeks. Sponsor: Homology Medicine  Experiment Design: HMI-102 is an AAVHSC15 vector containi...