SalioGen developing novel gene insertion therapy for Stargardt Disease
Key Takeaways
- SalioGen Therapeutics has announced development of a novel gene therapy for Stargardt disease.
- The therapy uses a new platform that allows genes of any size to be seamlessly integrated into precise, defined genomic locations.
SalioGen Therapeutics has announced development of SGT-1001, an emerging gene therapy for Stargardt disease, an inherited form of macular degeneration that causes vision loss in children and young adults. The new therapy targets mutations in the ABCA4 gene, which are responsible for most cases of Stargardt disease.
Current viral delivery systems for gene-augmentation therapies, i.e., therapies that provide healthy copies of defective genes, are limited in the size of the genetic cargo they can deliver. Many retinal disease genes — including ABCA4 — are too big for the viral containers used in these systems.
SalioGen’s therapy avoids this limitation by using a new platform, called Gene Coding, that works by adding new genomic code to turn on, off, or modify gene function. Gene Coding is based on enzymes called SaliogaseTM, which seamlessly integrate new DNA of any size into precise, defined genomic locations. This technology is designed to be more efficient and reliable than other gene-editing and gene-modifying approaches (e.g., CRISPR/Cas9) because it does not involve the double-stranded break and subsequent repair of the targeted gene.
SGT-1001 is intended to be a one-time treatment delivered by a subretinal (under the retina) injection. The company plans to launch a clinical trial for SGT-1001 in the first half of 2025. The RD Fund, the venture philanthropy arm of the Foundation Fighting Blindness, is an investor in the company. Rusty Kelley, PhD, managing director of the RD Fund, said, “There are currently no therapies for Stargardt disease. We are pleased that our investment is helping the company move SGT-1001 toward a first in human study — a step closer to meeting a significant unmet need.”
Edited by Miriam Kaplan, PhD
Source: Foundation Fighting Blindness, Eye on the Cure Research News, April 19, 2024; see source article