It develops genetic medicines by …
ARSA is responsible for the creation of the arylsulfatase A (ARSA) protein, which is required for the breakdown of cellular components. In MLD, these cellular components accumulate and destroy myelin-producing cells in the peripheral and central nervous system leading to progressive and serious neurological deterioration. It’s the iteration of over 100 years of work that has been done to define what a gene is and what it does, and now we can finally use that information to work to cure severe genetic disorders.” This approach can potentially be curative including in rapidly dividing cells (e.g., hematopoietic CD34+ cells and pediatric liver cells).HMI-102 Gene Therapy for Adult Patients with PKUHomology Medicines’ genetic medicines platform has the potential to treat and cure a wide range of genetic diseases through gene editing or gene therapy.MLD is a rare lysosomal storage disorder caused by a mutation in the ARSA gene. The late infantile form of the disorder is estimated to affect 1 in 40,000 people, and it is fatal within 5-10 years after onset. With gene editing, a person’s DNA is permanently corrected. Homology Medicines, Inc. operates as a technology platform to design and develop treatments to address rare diseases at the genetic level. This approach can be potentially curative in slow- or non-dividing cells (e.g., adult liver or central nervous system).Gene editing involves the insertion of a corrected gene directly into the genome. Homology Medicines Announces Peer-Reviewed Publication of HMI-102 Investigational Gene Therapy Demonstrating Restoration of Normal Metabolic Pathway in PKU Disease Model 03/12/2020 Homology Medicines Reports Fourth Quarter and Full Year 2019 Financial Results and Recent Accomplishments
We are prioritizing diseases with significant unmet medical need and validated regulatory pathways to bring new treatments forward as rapidly as possible.HMI-103 Gene Editing for Pediatric Patients with PKUWe are in IND-enabling studies with a gene therapy candidate (HMI-202) for the late infantile form of MLD.Gene therapy involves the transfer of corrected genes to specific cells in the body. In both approaches, we are using our proprietary human hematopoietic stem cell-derived adeno-associated virus vectors (AAVHSCs) to deliver the treatment to patients.We are initially working to cure monogenic diseases, or diseases that are caused by a defect in a single gene. From an enhanced free experience to professional stock picking - we have a great plan for you “We have the opportunity now not just to treat patients but to potentially cure them.”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.“This is powerful technology. Homology Medicines Announces Peer-Reviewed Publication Describing Molecular Characterization of Precise In Vivo Nuclease-Free Gene Editing with PKU Program 05/12/2020 Homology Medicines Announces Presentations on its In Vivo Gene Therapy and Gene Editing Programs and Commercial Manufacturing Platform at the American Society of Gene & Cell Therapy Annual Meeting With gene therapy, the genes do not integrate into the genome. For each target, we are deploying either a gene therapy or gene editing approach based on disease biology. Homology Medicines Announces Peer-Reviewed Publication Describing Molecular Characterization of Precise In Vivo Nuclease-Free Gene Editing with PKU Program May 12, 2020 Homology Medicines Announces Presentations on its In Vivo Gene Therapy and Gene Editing Programs and Commercial Manufacturing Platform at the American Society of Gene & Cell Therapy Annual Meeting We are initially working to cure monogenic diseases, or diseases that are caused by a defect in a single gene. Homology Medicines’ genetic medicines platform has the potential to treat and cure a wide range of genetic diseases through gene editing or gene therapy.