MedImmune and 4D Molecular Therapeutics Collaborate on AAV Gene Therapy for Lung Disease
Published: Jul 11, 2018 By Alex Keown
The two companies will harness 4DMT’s novel discovery platform to generate optimized adeno-associated virus (AAV) gene therapy vectors. AAV vectors are a delivery vehicle for transporting genes to targets in the body for in-vivo expression and therapeutic application, the companies said in a joint statement. AAV vectors target 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, the companies added.
4DMT’s Therapeutic Vector Evolution platform deploys over 100 million unique AAV variants from over 35 unique and proprietary 4DMT AAV libraries with extensive diversity, the company said.
The companies could be breaking new ground if they’re successful in developing a gene therapy for lung disease. There has been research to develop a gene therapy targeting Alpha-1 antitrypsin, particularly by California-based Adverum Biotechnologies.
David Kirn, chief executive officer and founder of 4DMT, said the collaboration with MedImmune may open the door to “significant advancements” in the treatment of respiratory patients.
“Our progress in customized AAV vectors enables us to unlock the potential of gene therapy and, with MedImmune’s expertise in protein engineering, we will continue to push boundaries in proprietary gene delivery to tissues and cells,” Kirn said in a statement.
Under the collaboration, MedImmune will conduct product development beginning from early clinical stages, while leveraging 4DMT’s expertise in vector discovery and engineering, optimization. Other than that bit of information, both companies were fairly light on what they intended to do or which lung disease they intended to target in the joint collaboration. There was also no mention of any financial figures surrounding the collaboration.
“Rapid advances in AAV therapy make this a promising tool to advance innovation in chronic lung disease, particularly in areas of high unmet need. This collaboration strategically pairs 4DMT’s expertise in AAV with MedImmune’s leadership in respiratory science, focused on early intervention and disease modification,” Roland Kolbeck, head of research & development in Respiratory, Inflammation and Autoimmunity at MedImmune, said in a statement.
This is the second respiratory collaboration that AstraZeneca has entered into this week. On Tuesday the U.K. parent company of MedImmune entered into an agreement with Revon Systems to evaluate CompEx, a novel therapeutic endpoint for asthma. CompEx is an algorithm combining patient-recorded lung function deterioration, symptoms and use of reliever medication with the traditional measurement of severe exacerbations. AstraZeneca is looking to integrate its clinical trial infrastructure with Revon's Smart Symptom Trackers to help accelerate the development of novel respiratory therapies in clinical trials.