Ascidian grabs first-ever IND for RNA editing 

Jan. 29, 2024

Boston-based biotech startup Ascidian Therapeutics announced that the FDA has cleared its IND application and granted Fast Track designation for its RNA exon editor in retinal diseases, marking the first time an RNA editor has been cleared in the U.S. to enter the clinic.

Ascidian's lead treatment, ACDN-01, is now the only clinical-stage therapeutic targeting the genetic cause of Stargardt disease, the most common form of inherited macular degeneration. Affecting approximately 30,000 individuals in the U.S. alone, Stargardt is caused by mutations in the ABCA4 gene which lead to progressive retinal degeneration and vision loss.

Ascidian's RNA exon editing platform is designed to expand the therapeutic possibilities of RNA medicine by replacing multiple contiguous exons — not just single bases — providing a more versatile RNA therapeutic approach.

“This is a critical step toward overcoming the challenges of Stargardt disease, such as the size of the ABCA4 gene and large number of mutations within the patient population, that have long kept Stargardt out of reach for conventional gene therapies," said Byron L. Lam, M.D., director of the Mark J. Daily Inherited Retinal Disease Research Center at the Bascom Palmer Eye Institute, University of Miami Miller School of Medicine.

The biotech expects to initiate enrollment in the phase 1/2 STELLAR trial, which will evaluate the safety and efficacy of a single dose of ACDN-01, administered via subretinal injection in individuals with Stargardt disease and other ABCA4 retinopathies, in the first half of 2024. 

Ascidian is fresh off a $40 million Series A funding round led by life science VC Apple Tree Partners. During the same November announcement, the company revealed that its board of directors had reinstated its founding CEO, Michael Ehlers.