Ascidian: correcting mutations via trans-splicing to edit RNA exons

Launched by ATP with $50 million, Ascidian believes its RNA trans-splicing technique will provide the capability to correct genetic mutations by mimicking a naturally occurring process, offering a safety advantage and greater therapeutic reach than other gene therapy and gene editing approaches.

Ascidian Therapeutics Inc. is developing RNA exon-editing molecules that can replace mutations with wild-type genetic material at the pre-mRNA stage. The approach is designed to produce durable results akin to most gene therapies, but the company believes it can address a wider variety of genes and mutations without using exogenous enzymes that can pose risks...