AGTC is also pursuing pre-clinical development of treatments for wet AMD using the company's experience in ophthalmology to expand into disease indications with larger markets.ĤDMT is a global leader in gene therapy product research & development. AGTC's lead product candidates focus on X-linked retinoschisis, achromatopsia and X-linked retinitis pigmentosa, which are inherited orphan diseases of the eye, caused by mutations in single genes that significantly affect visual function and currently lack effective medical treatments. Several AAV gene therapy products are in late-stage clinical development, and one product is approved in the EU.ĪGTC is a clinical-stage biotechnology company that uses its proprietary gene therapy platform to develop products designed to transform the lives of patients with severe diseases in ophthalmology. Once the gene is delivered into the correct cell, a therapeutic protein is naturally made by the cell from the therapeutic gene.Ībout Adeno-Associated Virus (AAV) VectorsĪAV vectors have emerged as a favored approach for gene therapy since they can deliver the genes for therapeutic proteins to accessible tissues in the body and are generally considered safe. In gene therapy, the healthy copy of a defective gene is packaged within a vector, which is used to transport the genetic information into the diseased cells within the body. The most common form of gene therapy involves using DNA that encodes a functional, therapeutic gene to replace a defective gene. Genes control heredity and provide the basic biological code for determining a cell's specific functions. Gene therapy is a growing field of medicine in which genes are introduced into the body to treat diseases. Together we can develop highly optimized and customized gene therapeutics, and this licensing agreement further underscores our commitment to create a robust product pipeline with our partners." "We believe that great synergies will result from combining our AAV-based gene therapy platform discovery with AGTC's expertise in ocular gene therapy development. "We are thrilled that AGTC, a global leader in ocular gene therapy, has elected to license our optimized next-generation vectors to target specific cell populations within the human retina," said David Kirn, M.D., co-founder and CEO of 4DMT. We expect that our combined expertise and our complementary proprietary technology and discovery platforms, will enable us to advance new treatments to market for patients with serious inherited ocular conditions who have few currently available therapeutic options." Sue Washer, President and CEO of AGTC, noted, "We are excited to partner with 4DMT to accelerate development of novel AAV-based gene therapies to treat rare eye diseases. As part of the agreement, 4DMT will deploy its proprietary AAV vector discovery platform, Directed Vector Evolution, to identify and optimize novel vectors. and SAN FRANCISCO, Ap(GLOBE NEWSWIRE) - Applied Genetic Technologies Corporation (Nasdaq:AGTC) and 4D Molecular Therapeutics (4DMT), leaders in next-generation adeno-associated virus (AAV) gene therapy vector discovery and product development, today announced a collaboration and license agreement to discover and develop optimized AAV vectors to treat specific ophthalmic disease indications with high unmet medical need.
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