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SalioGen Therapeutics taps development candidate for ABCA4-mediated Stargardt disease


SGT-1001 uses its novel gene coding technology for gene integration via transposition. It plans to advance SGT-1001 into the clinic in the first half of 2025.

(Image Credit: AdobeStock/Rattanathip)

(Image Credit: AdobeStock/Rattanathip)

SalioGen Therapeutics announced the nomination of a development candidate, SGT-1001, to treat Stargardt disease, a rare and progressive inherited retinal disease.

According to a news release from the company, 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 company noted in its news release the SGT-1001 development candidate is the first to use SalioGen's novel Gene Coding technology, which integrates large or multiple whole genes into the genome at precise locations through transposition. The mechanism, the company added, 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.1

Moreover, the company also pointed out that SGT-1001 demonstrates many of the capabilities of its novel technology.

"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 integrate a gene as large as ABCA4 into the retina with a proprietary lipid nanoparticle," Jason Cole, CEO of SalioGen Therapeutics, said in the news release. "This milestone is an important step forward for SalioGen and for people living with Stargardt disease who currently have no treatment options.”

Cole said the company hopes to complete IND-enabling studies for SGT-1001 in the second half of 2024 with a goal to bring this potential one-time therapy to the clinic in the first half of 2025.

According to the company, 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 Saliogase enzyme,

SalioGen noted that SGT-1001 is a mammalian-derived bioengineered enzyme that utilizes a proprietary lipid nanoparticle that is delivered subretinally and will be dosed only once.

Preclinical studies show 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.1

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.1

Although ABCA4 has proven to be a difficult research target given its size (6.8kb), scientists are making important progress for patients.

The company announced it will present preclinical data supporting SGT-1001 at the May annual meetings of the Association for Research in Vision and Ophthalmology (ARVO) and the American Society of Gene & Cell Therapy (ASGCT).

  1. Therapeutics S. SalioGen Therapeutics Announces Selection of Development Candidate for ABCA4-mediated Stargardt Disease. www.prnewswire.com. Accessed April 18, 2024. https://www.prnewswire.com/news-releases/saliogen-therapeutics-announces-selection-of-development-candidate-for-abca4-mediated-stargardt-disease-302118455.html
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