Ascidian Therapeutics announced that the FDA has approved testing their new drug, ACDN-01, in humans and given it Fast Track status. ACDN-01 is a treatment targeting the genetic cause of Stargardt disease, a type of inherited eye disease. The company plans to start enrolling patients for a Phase 1/2 clinical trial called the STELLAR study in the first half of 2024. This trial will test the safety and effectiveness of ACDN-01.
What this means for Usher syndrome: While this announcement is specific to Stargardt disease, the successful development and FDA clearance of a clinical trial for ACDN-01 as an RNA exon editor highlight a significant advancement in genetic medicine. For conditions like Usher syndrome, which involve complex genetic mutations similar to those in Stargardt disease, this progress suggests potential future applications of RNA editing technologies. These therapies could provide new avenues for treating Usher syndrome by addressing the underlying genetic causes of retinitis pigmentosa.
