生产企业：Opus Genetics, Inc.
OPGx-BEST1 (formerly named IC-200) is an investigational AAV-based gene therapy developed by Opus Genetics, Inc. designed to address the underlying genetic cause of BEST1-related inherited retinal diseases by using an AAV vector to deliver a functional copy of the BEST1 gene to retinal pigment epithelial cells. The vector construct includes the human BEST1 cDNA under control of the VMD2/BEST1 promoter and is packaged in an AAV2 capsid (AAV2/2-VMD2-BEST1), and is administered by subretinal injection.
OPGx-BEST1 (formerly named IC-200) is an investigational AAV-based gene therapy developed by Opus Genetics, Inc. designed to address the underlying genetic cause of BEST1-related inherited retinal diseases by using an AAV vector to deliver a functional copy of the BEST1 gene to retinal pigment epithelial cells. The vector construct includes the human BEST1 cDNA under control of the VMD2/BEST1 promoter and is packaged in an AAV2 capsid (AAV2/2-VMD2-BEST1), and is administered by subretinal injection.
BEST1 gene augmentation: an AAV vector delivers a functional copy of the BEST1 gene to retinal pigment epithelial cells, addressing the underlying genetic cause of BEST1-related inherited retinal diseases. The vector construct includes the human BEST1 cDNA under control of the VMD2/BEST1 promoter packaged in an AAV2 capsid, administered by subretinal injection.
共 3 项相关疾病
每项发明一条，涵盖美国、欧洲、PCT 与中国的同族专利。
