Patient Advocacy

Our partnership with the Foundation Fighting Blindness is focused on developing gene therapy products optimized for intravitreal administration to treat inherited retinal diseases. The Foundation brings a wide range of strengths to our collaboration, including the following: clinical expertise, a network of physician-scientist leaders and key opinion leaders, trial investigators, and patient networks. Our partnership strives to enable the development of breakthrough treatments and cures for patients with inherited retinal diseases.

Our 2015 and 2017 grants totaling more than $5 million dollars from the Cystic Fibrosis Foundation are designed to support the discovery, engineering and development of a 4DMT AAV gene therapy product optimized for aerosolized delivery to treat cystic fibrosis, a hereditary disease involving the lung and other tissues. The Foundation brings research and clinical expertise, a network of doctors, trial investigators and patients together to inform product design, R&D, clinical trial design and execution.

Our partnership with the Choroideremia Research Foundation is focused on developing a transformative gene therapy product optimized for intravitreal administration to treat Choroideremia, a hereditary retinal degenerative disease (caused by a mutation in the Rep-1 gene). The Foundation brings together grant support, disease-specific clinical and regulatory knowledge, a network of doctors and trial investigators, and patients and their families. Their resources and expertise helps 4DMT to execute our natural history clinical study, our clinical development plans and our clinical trials.
View Partnership Press Releases