Ascidian headed to clinic with first-ever RNA exon editor

29 Jan 2024
firstwordpharma.com
Gene TherapysiRNA
The FDA has given the all-clear for Ascidian Therapeutics to begin evaluating its experimental RNA exon editor in clinical trials, a first for this treatment modality. The company expects to begin recruiting patients in the Phase I/II STELLAR study of its lead ACDN-01 programme in Stargardt disease and other ABCA4 retinopathies in the first half.
The move comes a little over a year after the ATP-incubated startup launched with $50 million in Series A funding and a platform that it says can sidestep drawbacks of conventional approaches. It took in another $40 million in an extended round late last year.
The company's name is a tip of the hat to a group of marine organisms, also known as sea squirts, that undergo RNA trans-splicing and alternative splicing to modify their transcriptome as they transition from larvae to adults. Ascidian says its approach of "rewriting" RNA by editing and replacing human exons is inspired by this process.
"We have conviction in the rigour of our data, and that by editing RNA and not DNA, the Ascidian approach brings unique advantages," said interim CEO Michael Ehlers. These advantages include durability of gene therapy while reducing risks associated with DNA editing and manipulation.
Last year, Ascidian presented six-month data from its lead programme demonstrating the "efficient and durable" production of full-length ABCA4 protein with a single AAV-delivery of ACDN-01 in the retina of non-human primates.
The company notes that diseases caused by ABCA4 loss of function are examples of genetic disorders that cannot be addressed by standard gene replacement or base editing, either due to the large size of the gene or the extensive mutational variability within the affected gene itself. Stargardt disease has more than 1000 mutations across the ABCA4 gene.
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