Homology’s proprietary platform is designed to utilize its human hematopoietic stem cell-derived adeno-associated virus vectors (AAVHSCs) to precisely and efficiently deliver genetic medicines in vivo either through a gene therapy or nuclease-free gene editing modality across a broad range of genetic disorders. Homology’s proprietary platform is designed to utilize its human hematopoietic stem cell-derived adeno-associated virus vectors (AAVHSCs) to precisely and efficiently deliver genetic medicines in vivo either through a gene therapy or nuclease-free gene editing modality across a broad range of genetic disorders. While the trial will track these patients for one year, this first glimpse included data on only the first few months following treatment of the three patients. Presents New Data Characterizing AAVHSCs as Potential Gene Therapies for Nervous System Disorders BEDFORD, Mass., July 21, 2020 (GLOBE NEWSWIRE) -- Homology Medicines, Inc. (Nasdaq: FIXX), a genetic medicines company, announced today that Jeff Poulton has been appointed to the Board of Directors. He also serves as a member of the Board of Directors at EIP Pharmaceuticals. Homology's leadership in gene therapy and gene editing manufacturing will be the subject of presentations highlighting our efficient commercial process and scalable internal GMP manufacturing platform, which currently serves the needs of our clinical and preclinical programs. In addition, we have established home-based care and monitoring services for patients in our Phase 1/2 pheNIX gene therapy trial for PKU, making their safety and continuity of the study priorities. Our gene therapy construct includes a functional copy of the gene and a promoter sequence that is designed to enable the gene to be turned on in the cell and ultimately transcribed to express a therapeutic protein without integrating into the genome.Homology Medicines’ gene therapy approach utilizes our proprietary AAVHSC vectors to deliver a functional gene to a cell where there is a missing or mutated gene. For the two patients on the low dose, the company measured vector copy numbers in the blood, which is evidence the therapy is present, he said.Topics covered: Pharma, biotech, FDA, gene therapy, clinical trials, drug pricing and much more.By comparison, BioMarin's Palynziq (pegvaliase) showed a roughly 62% reduction in Phe levels among patients treated with a 20 mg once daily dose of the enzyme substitution therapy.Tuesday's update includes data from the first three adults with PKU treated with Homology's gene therapy, known as HMI-102. ""The Homology team's experience, agility, and forward-thinking capabilities are a tremendous asset to the Company as we operate within this global health crisis, and as a result, our programs have continued…"We are looking forward to sharing data that demonstrate the potential of our…"I had the pleasure of working with Jeff before, and I know first-hand how his strategic financial and operational leadership contributed to the…In "AAVHSCs Transduction Does Not Significantly Elicit p53-Mediated Apoptosis or Alter Cell Cycle in Human iPSCs and Primary Cells When Compared to Non-Clade F AAV Vectors," the studies demonstrate that AAVHSCs:"We are looking forward to sharing data that demonstrate the potential of our AAVHSC genetic medicines platform to treat rare genetic diseases such as PKU and MLD," stated Albert Seymour, Ph.D., Chief Scientific Officer of Homology Medicines. Homology Medicines’ gene therapy approach utilizes our proprietary AAVHSC vectors to deliver a functional gene to a cell where there is a missing or mutated gene. Clade F AAVHSCs cross the blood brain barrier and transduce the central nervous system in addition to peripheral tissues following intravenous administration in nonhuman primates. Homology Medicines, Inc. (Nasdaq: FIXX), a genetic medicines company, announced today that Jeff Poulton has been appointed to the Board of Directors. Our approach with PKU exemplifies the duality and flexibility of our technology platform where we choose the best genetic medicines approach based on human biology and the underlying cause of disease. Homology Medicines, Inc. (FIXX), a genetic medicines company, announced today that it has commenced enrollment of the Phase 1/2 pheNIX trial … "Jeff has also displayed a long-term commitment to help patients and their families, which aligns with Homology's mission to develop our gene therapy and nuclease-free gene editing platform into new treatments and potential cures. Dr. Tzianabos continued, “We begin this year on track to start and report initial data from our Phase 1/2 gene therapy trial for adults with PKU, and we have begun IND-enabling studies with our first gene editing program for children with PKU. Mr. Poulton brings 25 years of experience to Homology’s Board of Directors, including leading finance, business development and commercial ope
A fourth patient received the higher dose after Dec. 2, the cut-off for compiling results, Seymour said.