FDA Fast Tracks First Clinical Stage RNA Exon Editor for Stargardt Disease

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Ascidian Therapeutics has announced that the FDA has cleared its investigational new drug (IND) application and granted Fast Track designation for ACDN-01. According to a company press release, ACDN-01 is the only clinical stage therapeutic targeting the genetic cause of Stargardt disease and the first-ever clinical stage RNA exon editor.¹

According to the Foundation Fighting Blindness, Stargardt disease is a genetic eye disorder that causes retinal degeneration and vision loss. The inherited disease is often diagnosed in childhood or adolescence. The progressive vision loss associated with Stargardt is caused by the degeneration of photoreceptor cells in the central portion of the retina called the macula. Stargardt disease is currently affecting about 30,000 people in the United States.²

"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," said Michael Ehlers, MD, PhD, president and interim CEO of Ascidian Therapeutics, in the press release. "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 dramatically expand the reach of genetic medicine."

Ascidian expects to initiate enrollment in the Phase I/II STELLAR study of ACDN-01 in Stargardt disease in the first half of 2024. The open-label study will evaluate the safety and efficacy of a single dose of ACDN-01 via subretinal injection. In addition to patients with Stargardt, the therapeutic will also be evaluated in individuals with other ABCA4 retinopathies.

"The advancement of Ascidian's first-of-its-kind RNA exon editor from the lab to the clinic is a unique and novel therapeutic approach targeting the genetic cause of Stargardt disease," said Byron L. Lam, MD, director of the Mark J. Daily Inherited Retinal Disease Research Center at the Bascom Palmer Eye Institute, University of Miami Miller School of Medicine, in the press release. "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 am looking forward to the clinical evaluation of this promising approach."

References

1. Ascidian Therapeutics Announces First-Ever IND for an RNA Exon Editor as FDA Approves Trial Plan and Fast Tracks ACDN-01 in Stargardt Disease and Other ABCA4 Retinopathies. News release. January 29, 2024. Accessed January 30, 2024. https://www.prnewswire.com/news-releases/ascidian-therapeutics-announces-first-ever-ind-for-an-rna-exon-editor-as-fda-approves-trial-plan-and-fast-tracks-acdn-01-in-stargardt-disease-and-other-abca4-retinopathies-302046287.html.
2. What is Stargardt Disease? Foundation Fighting Blindness. Accessed January 30, 2024. https://www.fightingblindness.org/diseases/stargardt-disease#:~:text=Stargardt%20Disease%20is%20the%20most,the%20retina%20called%20the%20macula.