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Applied Genetic Technologies Corporation (AGTC), a clinical-stage gene therapy development company, has raised $11.8 million in venture capital funding to advance treatments for the retinal degenerative diseases Leber congenital amaurosis (LCA) and achromatopsia. The investment will also be used to fund research for an early onset form of emphysema. AGTC is extending the Phase I clinical study of gene therapy for LCA underway at the University of Pennsylvania and the University of Florida. Young adults in the study who were virtually blind have had some vision restored. The company is preparing for a Phase I/II study of gene therapy for LCA, which will include younger participants and higher treatment doses. LCA is a severe form of retinitis pigmentosa that causes children to be born blind or with severe vision loss. The Foundation Fighting Blindness funded pivotal, preclinical studies of this LCA treatment that made these clinical studies possible. AGTC is also planning to launch a clinical trial for gene therapy to treat achromatopsia, a retinal disease that causes blindness in daylight and brightly lit settings. The National Neurovision Research Institute (NNRI), the clinical support arm of the Foundation Fighting Blindness, is currently funding a preclinical study of the achromatopisa treatment at the University of Pennsylvania. “NNRI is very pleased that AGTC has received substantial financial support for the continued development of gene therapy products for LCA and achromatopsia,” says Stephen Rose, Ph.D., chief research officer, Foundation Fighting Blindness. “This financial infusion extends support of the current LCA clinical trial at the Universities of Pennsylvania and Florida, and brings this potential treatment closer to FDA approval. In addition, the support for preclinical studies for achromatopsia provides even greater potential for gene therapy to treat and cure another retinal degenerative disease that often does not receive the attention or investment necessary to bring promising treatments to patients.” AGTC is also developing a gene therapy for X-linked retinoschisis, an inherited form of retinal degeneration affecting young men, as well as a gene therapy for the wet form of age-related macular degeneration, the leading cause of blindness in people 55 and older living in developed countries. AGTC develops gene therapy treatments that utilize an adeno-associated virus or AAV to deliver therapeutic genes to targeted tissues. AAVs have been shown to be safe and effective in a variety of retinal disease studies, including Phase I LCA clinical trials underway at the Children’s Hospital of Philadelphia and Moorfields Eye Hospital in London. |
