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AGTC and BCM Families Foundation Announce Collaboration to Develop AAV-Based Gene Therapy for Blue Cone Monochromacy
Posted on: 22 January 2016, source: BCM Families Foundation
Applied Genetic Technologies Corporation (Nasdaq:AGTC), a biotechnology company conducting human clinical trials of adeno-associated virus (AAV)-based gene therapies for the treatment of rare eye diseases, and the BCM Families Foundation, a non-profit organization focused on eradicating Blue Cone Monochromacy (BCM), today announced a collaboration to develop an AAV-based gene therapy for the disease. Blue Cone Monochromacy, also known as X-linked achromatopsia, is a rare genetic disease of the retina that almost exclusively affects males. It is a hereditary condition linked to the X chromosome that manifests with a partial dysfunction of the cones of the retina. BCM can result in reduced visual acuity, impaired color vision, photosensitivity, myopia and infantile-onset nystagmus. These manifestations are similar to those in achromatopsia, caused by mutations in the CNGB3 or CNGA3 gene, for each of which AGTC has ongoing clinical development activities.
Applied Genetic Technologies Corporation (Nasdaq:AGTC), a biotechnology company conducting human clinical trials of adeno-associated virus (AAV)-based gene therapies for the treatment of rare eye diseases, and the BCM Families Foundation, a non-profit organization focused on eradicating Blue Cone Monochromacy (BCM), today announced a collaboration to develop an AAV-based gene therapy for the disease. Blue Cone Monochromacy, also known as X-linked achromatopsia, is a rare genetic disease of the retina that almost exclusively affects males. It is a hereditary condition linked to the X chromosome that manifests with a partial dysfunction of the cones of the retina. BCM can result in reduced visual acuity, impaired color vision, photosensitivity, myopia and infantile-onset nystagmus. These manifestations are similar to those in achromatopsia, caused by mutations in the CNGB3 or CNGA3 gene, for each of which AGTC has ongoing clinical development activities.