4D Molecular Therapeutics (4DMT), a provider of adeno-associated virus (AAV) gene therapy vector discovery and product development, and the Foundation Fighting Blindness (FFB), the world’s largest non-governmental source of research funding for inherited retinal degenerations (IRD) and dry age-related macular degeneration (AMD), announced a partnership to develop intravitreal gene therapeutics for patients with these blinding conditions using 4DMT-proprietary AAV vectors. Under the terms of the agreement, 4DMT will provide access to its vector technology, development expertise and manufacturing capabilities while FFB will identify potential academic and business collaborators, provide drug development expertise and fund approved projects to develop transformative gene therapy products. 4DMT retains all patent and commercial rights to its 4DMT proprietary AAV vector variants. FFB and 4DMT will jointly review and approve all programs initiated within this collaboration.
New vector technologies are critical to the successful use of gene therapies for IRDs in order to improve targeting to affected cells within the retina, and to maximize efficacy and safety. Vectors that can be delivered intravitreally would simplify the procedures used for treatment and reduce the costs of administration.
Affecting approximately 200,000 patients in the US, inherited retinal diseases (IRDs) are a major cause of adult and childhood blindness. Mutations in more than 260 genes are known to cause these rare, orphan conditions for which there are currently no approved therapies. Gene therapy holds tremendous promise for the treatment of these conditions by introducing genes to the retina that may be able to replace the lost or dysfunctional genes, correct underlying mutations, or deliver therapeutically-active genes that can prevent cell loss and degeneration.
“We are very impressed with 4D’s vector evolution approach, the company’s product pipeline and manufacturing expertise. The potential is great for developing a number of gene therapeutics that could treat those affected by retinitis pigmentosa and allied conditions using a simple intravitreal injection approach,” Patricia Zilliox PhD, Chief Drug Development Officer at the FFB, said in a news release.
“We are extremely excited by this collaboration with FFB, a globally-recognized leader in the effort to cure blindness due to inherited retinal degenerations. FFB has tremendous expertise identifying the best retinal research as well as an outstanding network of funded investigators and companies with whom we hope to collaborate to develop a portfolio of products that will benefit those affected with retinal degenerative diseases,” said David Kirn, MD, co-Founder and CEO of 4DMT.