Our Advocacy Approach

We have relationships with the Cystic Fibrosis Foundation, Foundation Fighting Blindness, Choroideremia Research Foundation and CureDuchenne. We will continue to evaluate new opportunities to partner with leading pharmaceutical and biotechnology companies, and with patient disease foundations and advocacy groups, that we believe maximize value and benefits for us and patients.

Patient Advocacy

Our partnership with the Foundation Fighting Blindness strives to enable the development of potentially breakthrough treatments and cures for patients with inherited retinal diseases by providing access to our proprietary AAV vectors optimized for intravitreal administration. 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.

We have received funding and scientific support from the Cystic Fibrosis Foundation, whose mission is to cure cystic fibrosis and to provide all people with the disease the opportunity to lead full, productive lives. This partnership supports the discovery, engineering and development of a 4DMT AAV gene therapy product designed to be 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 designed 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 us to execute our natural history clinical study, our clinical development plans and our clinical trials.

The mission of the Cure Duchenne organization is to save this generation of children and young adults with Duchenne muscular dystrophy. 4DMT has received funding and scientific support.