ACDN-01

ACDN-01 is a first-in-class, in vivo RNA exon editor developed for the treatment of Stargardt disease and other ABCA4-related retinopathies. Unlike conventional gene therapies that deliver or edit DNA, ACDN-01 targets RNA, specifically re-writing exons within the ABCA4 transcript to restore production of full-length, functional protein. This approach allows for correction of a wide range of mutations across multiple exons simultaneously and circumvents challenges associated with delivering large genes like ABCA4 using viral vectors. Delivered as a single subretinal injection via a single vector system, ACDN-01 has demonstrated efficient and durable RNA exon editing in preclinical models including non-human primate retina and human retinal explants. The therapy aims to provide persistent therapeutic benefit with one dose by enabling post-transcriptional correction without permanent changes to DNA[1][2][4][5][8].

Other names

ABCA4 RNA exon editorABCA-4 RNA exon editorABCA 4 RNA exon editor