SGT-1001

SGT-1001 is an investigational gene therapy developed by SalioGen Therapeutics for the treatment of Stargardt disease, a rare inherited retinal disorder that leads to progressive central vision loss. The therapy is designed as a one-time, non-viral intervention and utilizes SalioGen’s proprietary Gene Coding technology to integrate a full-length ABCA4 gene construct and mRNA coding for the Saliogase enzyme into the genome via transposition. This approach allows precise insertion of large or multiple genes without inducing double-strand breaks or using guide RNA, potentially reducing safety risks associated with other genetic therapies. Delivery is achieved through subretinal injection using proprietary lipid nanoparticles capable of carrying large genetic payloads such as ABCA4. Preclinical studies have demonstrated effective gene integration in photoreceptors and retinal pigment epithelium cells, sufficient expression of ABCA4 protein to reduce toxic lipofuscin A2E levels linked to macular degeneration, and favorable tolerability in non-human primates[2][3][5][7].