FDA Clears Ascidian for First-Ever RNA Editing Trial in US
30 Jan 2024
INDFast TrackClinical Study
Pictured: FDA signage outside its headquarters/iStock, JHVEPhoto
Ascidian Therapeutics has received IND clearance from the FDA and has been granted Fast Track Designation for its asset to treat Stargardt disease, a genetic eye disease that can cause blindness in children and young adults, the company announced Monday.
Ascidian’s asset, known as ACDN-01, had been given clearance from the agency and is expected to start enrollment in Phase I/II study of the drug in Stargardt and other ABCA4 retinopathies sometime in the first half of 2024. The asset itself is a clinical-stage RNA exon editor and, according to Ascidian, is the only therapeutic going after the “genetic cause” of Stargardt.
“This open IND for ACDN-01 by the FDA—the first regulator to have cleared ACDN-01 for clinical development—represents an important milestone for Ascidian and the broader field of RNA editing,” Ascidian Interim CEO Michael Ehlers said in a statement.
“We chose to go to the FDA first because we have conviction in the rigor of our data, and that by editing RNA and not DNA, the Ascidian approach brings unique advantages with potential to transform the lives of people living with Stargardt disease and, more broadly, to expand the reach of genetic medicine dramatically,” Ehlers added.
The open-label study, known as Stellar, will investigate the safety and efficacy of a single dose of the asset and will be administered by a subretinal injection.
“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. Stargardt patients deserve treatment options, and I look forward to this promising approach's clinical evaluation." Byron Lam, the Mark J. Daily Inherited Retinal Disease Research Center director at the Bascom Palmer Eye Institute, University of Miami Miller School of Medicine, said in a statement.
Ascidian launched in 2022 and was named to BioSpace’s NextGen Class of 2024’s top life science companies to watch. The company is differentiating itself from other gene therapy companies by using the exon swaps in RNA transcripts that encode proteins.
When the company launched, it had $50 million in funding total with backing from Apple Tree Partners.
Tyler Patchen is a staff writer at BioSpace. You can reach him at tyler.patchen@biospace.com. Follow him on LinkedIn.
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