SalioGen Therapeutics, a biotechnology company developing next-generation genetic medicines based on its novel Gene CodingTM technology, today announced the nomination of a development candidate, SGT-1001, for the treatment of Stargardt disease, a rare and progressive inherited retinal disease. SGT-1001 is being developed as a one-time, non-viral therapy to slow or stop the progressive loss of central vision in people with Stargardt disease, regardless of the type of mutation.
The SGT-1001 development candidate is the first to use SalioGen’s novel Gene Coding technology. Gene Coding integrates large or multiple whole genes into the genome at precise locations through transposition. This mechanism does not induce double-strand breaks or use guide RNA and has the potential to overcome the safety risks and limitations of other genetic therapeutic approaches.
“The nomination of SalioGen’s first development candidate, SGT-1001, demonstrates many of the unique capabilities of our novel Gene Coding technology, including the ability to seamlessly integrate a gene as large as ABCA4 into the retina with a proprietary lipid nanoparticle,” said Jason Cole, CEO of SalioGen Therapeutics. “This milestone is an important step forward for SalioGen and for people living with Stargardt disease who currently have no treatment options. We look forward to completing IND-enabling studies for SGT-1001 in the second half of 2024 and aim to bring this potential one-time therapy to the clinic in the first half of 2025.”
SGT-1001 is designed to address the underlying genetic cause of Stargardt disease. It consists of a full-length ABCA4 gene construct and mRNA coding for the SaliogaseTM enzyme, SalioGen’s proprietary mammalian-derived bioengineered enzyme. SGT-1001 utilizes a proprietary lipid nanoparticle that is delivered subretinally and will be dosed only once. Preclinical studies show promising gene integration in photoreceptors and retinal pigment epithelium (RPE) cells, as well as sufficient expression of ABCA4 to reduce levels of lipofuscin A2E associated with macular degeneration in a validated animal efficacy model. As measured by Optical Coherence Tomography (OCT), tolerability in non-human primates was consistent with a commercially available gene therapy administered through sub-retinal injection.
SalioGen will present preclinical data supporting SGT-1001 at the May 2024 annual meetings of the Association for Research in Vision and Ophthalmology (ARVO) and the American Society of Gene & Cell Therapy (ASGCT).
About Stargardt disease
Stargardt disease is an inherited eye disease that causes progressive central vision loss. It is the most common inherited disease of the macula, with an estimated 30,000 people affected in the United States. Mutations in both copies of a person’s ABCA4 gene are the most common cause of Stargardt disease, and more than 1,000 different mutations in ABCA4 can lead to the disease. There are no treatments for Stargardt disease, but genetic medicine has the potential to address the underlying genetic cause of the disease. Although ABCA4 has proven to be a difficult research target given its size (6.8kb), scientists are making important progress for patients.
About SalioGen Therapeutics
SalioGen Therapeutics is developing next-generation genetic medicines for patients using its novel Gene CodingTM technology. Gene Coding is a non-viral method of integrating large or multiple whole genes into the genome at precise locations without double-strand breaks or guide RNA. SalioGen’s lead programs focus on one-time treatments for Stargardt disease and cystic fibrosis. The company is also developing its Gene Coding technology for additional indications, such as other inherited retinal diseases, and to engineer CAR-T cells for cancer and autoimmune conditions. SalioGen is headquartered in Lexington, Massachusetts. For more information, visit SalioGen.com and follow SalioGen on LinkedIn.