ENTSTAR001 - Stargardt Disease

Stargardt disease is a rare genetic eye disease that causes blindness with vision loss starting in childhood with no treatment options. Stargardt disease occurs due to a mutation in the ABCA4 gene that leads to fatty build-up. Treatments for Stargardt disease have remained elusive because the gene that encodes for ABCA4 is large and the currently available genetic cargo delivery platforms are  unable to deliver the gene effectively.

Leveraging the optimized Fusogenix PLV ocular formulation, Entos has demonstrated effective delivery of large genetic cargoes including ABCA4 to the relevant cells that impact Stargardt disease in non-human primates.

Entos is developing ENTSTAR001 for the treatment of Stargardt disease. ENTSTAR001 is a gene therapy that utilizes the Fusogenix PLV platform to deliver a functional full length copy of ABCA4 to the retina.