Homology Medicines, Inc.: Diferență între versiuni
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Admin (discuție | contribuții) (Pagină nouă: Pagina dedicata companiei Homology Medicines, Inc. listata cu simbolul US.FIXX ==Descriere companie== Homology Medicines, Inc. (https://www.homologymedicines.com/) is a clinical-stage genetic medicines company. The Company focuses on patients suffering from rare genetic diseases by curing the underlying cause of the disease. Its platform is designed to utilize its human hematopoietic stem cell derived adeno-associated virus vectors (AAVHSCs), to deliver single administration...) |
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==Descriere companie== | ==Descriere companie== | ||
Homology Medicines, Inc. (https://www.homologymedicines.com/) is a clinical-stage genetic medicines company. The Company focuses on patients suffering from rare genetic diseases by curing the underlying cause of the disease. Its platform is designed to utilize its human hematopoietic stem cell derived adeno-associated virus vectors (AAVHSCs), to deliver single administration genetic medicines in vivo either through gene therapy or nuclease-free gene editing across a range of genetic disorders. Its various set of AAVHSCs allows it to target, via a single injection, a range of disease-relevant tissues, including the liver, central nervous system (CNS), peripheral nervous system (PNS), bone marrow, muscle and eye. Its lead product candidate, HMI-102, is a gene therapy for the treatment of adults with phenylketonuria (PKU). Its other gene therapy product development candidates for the treatment of lysosomal storage diseases are HMI-203, for the treatment of mucopolysaccharidosis type II (MPS II) and HMI-202. | Homology Medicines, Inc. (https://www.homologymedicines.com/) is a clinical-stage genetic medicines company. The Company focuses on patients suffering from rare genetic diseases by curing the underlying cause of the disease. Its platform is designed to utilize its human hematopoietic stem cell derived adeno-associated virus vectors (AAVHSCs), to deliver single administration genetic medicines in vivo either through gene therapy or nuclease-free gene editing across a range of genetic disorders. Its various set of AAVHSCs allows it to target, via a single injection, a range of disease-relevant tissues, including the liver, [[CENTRAL|central]] nervous system (CNS), peripheral nervous system (PNS), bone marrow, muscle and eye. Its lead product candidate, HMI-102, is a gene therapy for the treatment of adults with phenylketonuria (PKU). Its other gene therapy product development candidates for the treatment of lysosomal storage diseases are HMI-203, for the treatment of mucopolysaccharidosis type II (MPS II) and HMI-202. | ||
==Grafic actiuni companie== | ==Grafic actiuni companie== | ||