MedImmune, the global biologics research and development arm of AstraZeneca, and 4D Molecular Therapeutics (4DMT), a world-leader in Therapeutic Vector Evolution for adeno-associated virus (AAV) gene therapy vector discovery and product development, announced a collaboration to develop and commercialize a gene therapeutic for patients with chronic lung disease, utilizing 4DMT’s novel discovery platform to generate optimized AAV vectors.
AAV vectors are a leading delivery vehicle for transporting genes to accessible tissues in the body for in-vivo expression and therapeutic application. They target both dividing and nondividing cells without integrating genetic material into the host genome. The genetic material transferred by AAV vectors into cells is the blueprint for the production of a protein whose function targets pathological processes contributing to diseases.
Therapeutic Vector Evolution platform deploys over 100 million unique AAV variants from over 35 unique and proprietary 4DMT AAV libraries with extensive diversity. After defining the Target Product Profile, and the associated Target Vector Profile, 4DMT then applies proprietary methods to identify lead vectors from within our AAV libraries. The result is a customized, novel, and proprietary pharmaceutical-grade product uniquely designed for targeted therapeutic gene delivery and efficacy in humans.
The collaboration will leverage 4DMT’s expertise in vector discovery and engineering, optimization and process development. MedImmune will conduct product development beginning from early clinical stages, drawing on the company’s extensive expertise in respiratory science.
MedImmune is the global biologics research and development arm of AstraZeneca, a global, innovation-driven biopharmaceutical business that focuses on the discovery, development and commercialization of small molecule and biologic prescription medicines. The MedImmune headquarters is located in Gaithersburg, USA, one of AstraZeneca’s three global R&D centres, with additional sites in Cambridge, UK and South San Francisco, USA.
4DMT (Emeryville, California, USA) is a gene therapy company with a transformative discovery platform—Therapeutic Vector Evolution— that empowers us to invent optimized and proprietary AAV vectors, each specifically tailored for the treatment of a rare disease with a high unmet medical need.