Ascidian Therapeutics has received FDA clearance for its investigational new drug (IND) application and Fast Track designation for ACDN-01. ACDN-01 is marked as the first-ever clinical-stage RNA exon editor, representing a novel therapeutic approach to target the genetic cause of Stargardt disease.
In a press release, Ascidian Therapeutics expressed its anticipation to commence enrollment in the Phase 1/2 STELLAR study for ACDN-01 in the first half of 2024. Stargardt disease and other ABCA4 retinopathies will be the focus of this clinical trial.
ACDN-01, an in vivo RNA exon editor delivered through a single vector, has exhibited efficient and durable RNA exon editing in non-human primate retina and human retinal explants during ex vivo testing.
Michael Ehlers, MD, PhD, president and Interim CEO of Ascidian Therapeutics, emphasized the significance of FDA's clearance as a pivotal milestone for both Ascidian and the broader RNA editing field. He highlighted the unique advantages of Ascidian's approach, particularly its focus on RNA editing rather than DNA, bringing transformative potential for individuals affected by Stargardt disease and expanding the horizons of genetic medicine.
Byron L Lam, director of the Mark J Daily Inherited Retinal Disease Research Center at the Bascom Palmer Eye Institute, University of Miami Miller School of Medicine, underscored the innovative nature of Ascidian's RNA exon editor. Lam emphasized its potential to overcome challenges that have traditionally hindered conventional gene therapies from addressing Stargardt disease.
The Phase 1/2 STELLAR study, an open-label trial, will assess the safety and efficacy of a single subretinal injection of ACDN-01 in individuals with Stargardt disease and other ABCA4 retinopathies. The Fast Track designation granted to ACDN-01 by the FDA aims to expedite the development process, allowing Ascidian to receive regular feedback from the regulatory authority.
Stargardt disease, affecting around 30,000 individuals in the U.S., is the most common form of inherited macular degeneration with no FDA-approved treatments. It is caused by mutations in the ABCA4 gene, leading to progressive retinal degeneration and vision loss, typically starting in childhood or young adulthood. Ascidian's ACDN-01, with its innovative RNA exon editing approach, offers hope for addressing the challenges posed by the large size and mutational variance of the ABCA4 gene in treating Stargardt disease and related conditions.
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