A sea change in RNA therapeutics

Ascidian is redefining the treatment of disease by rewriting RNA.

A new way to treat disease, inspired by nature

Ascidians – also known as sea squirts – are ocean creatures and primordial ancestors of vertebrates. To grow from larvae to adults, ascidians re-engineer their transcriptome through RNA trans-splicing and alternative splicing. Ascidian Therapeutics was founded to rewrite RNA by editing and replacing human exons, inspired by this example. In Ascidian’s labs, RNA biology meets today’s cutting-edge genomics, computational biology, and deep-sequencing technologies to create a new class of medicines that address the underlying causes of disease.

RNA exon editing has the potential to revolutionize our approach to treating human disease.

Precision RNA Therapeutics

Ascidian’s first-of-its kind RNA exon editing platform is designed to expand the therapeutic possibilities of RNA medicine and treat diseases not addressed by today’s gene editing technologies. By rewriting RNA, Ascidian’s exon editing technology provides the durability of gene therapy without the risks of direct DNA editing or gene replacement, while maintaining endogenous gene expression patterns and levels. Disease-causing genetic mutations are often located at different sites on the gene in different patients. Ascidian’s technological platform can replace multiple contiguous exons — not just single bases — providing a more powerful and versatile RNA therapeutic approach.

Ascidian enables precise post-transcriptional editing of genes, resulting in full-length, functional proteins:

at the right levels

in the right cells

at the right time

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