FT-004

FT-004 is an investigational dual-vector adeno-associated virus (AAV) gene therapy developed by Frontera Therapeutics for the treatment of Stargardt disease, an inherited retinal dystrophy caused by mutations in the ABCA4 gene. Because the ABCA4 coding sequence exceeds the standard AAV packaging capacity of approximately 4.7 kb, FT-004 utilizes a dual-vector approach where the gene expression cassette is split into two halves—represented by the components F2C22C and DL001Z—and packaged into separate AAV capsids. Upon co-infection of the target retinal cells, the two fragments undergo recombination or trans-splicing to form a full-length, functional ABCA4 protein. FT-004 is designed for intravitreal administration, leveraging Frontera's proprietary AAV capsid platform to achieve efficient retinal transduction without the need for invasive subretinal surgery.

Other names

ABCA4 dual-vector gene therapyABCA-4 dual-vector gene therapyABCA 4 dual-vector gene therapyFrontera Stargardt gene therapy