New venture, Arnatar Therapeutics, debuts with a $52 million investment, aiming to progress RNA treatments.
Arnatar Therapeutics, a San Diego-based biotechnology company, has made significant strides in the development of a potential treatment for Alagille Syndrome. The U.S. Food and Drug Administration (FDA) has granted both Orphan Drug and Rare Pediatric Disease designations to Arnatar Therapeutics' flagship product, ART4.
ART4 is a therapeutic candidate that uses a unique RNA platform to upregulate a protein responsible for bile duct development. This innovative approach could offer a promising solution for those affected by Alagille Syndrome, a rare and often debilitating genetic disorder.
The company launched in 2024 with a $52 million Series A funding round, led by Eight Roads and 3E Bioventures. Arnatar Therapeutics has been committed to advancing ART4 into the clinic as rapidly as possible, with the expected start date for clinical trials set for the second quarter of 2024.
Xuehai Liang, CEO of Arnatar Therapeutics, expressed his optimism about ART4's potential, stating that it has the potential to significantly address an unmet need and meaningfully alter the course of Alagille Syndrome. He further added that the company's goal is to bring the potentially transformative therapy of ART4 to patients and families who currently have no curative treatment options for the disease.
Arnatar Therapeutics is not limiting its focus to Alagille Syndrome. The company is also working on RNA-based therapies for other underserved diseases in the areas of cardiometabolic, liver, CNS, and kidney diseases.
The Orphan Drug and Rare Pediatric Disease designations from the FDA are significant milestones for Arnatar Therapeutics, as they offer various benefits, including tax credits for clinical testing, exemption from FDA application fees, and eligibility for market exclusivity upon approval. These designations provide a strong foundation for the company's continued efforts to bring ART4 to market and make a meaningful impact on the lives of those affected by Alagille Syndrome.
