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.
In a modern twist on this age-old biological process, Ascidian Therapeutics is developing ways to edit and replace human exons through precision splicing of pre-mRNA.
RNA exon editing has potential to bring about a revolutionary new approach to treating human disease.
In Ascidian’s labs, ancient biology meets today’s cutting-edge therapeutic delivery systems, large-scale DNA and RNA synthesis, and deep sequencing technologies, to advance exciting new therapeutic possibilities.
By harnessing designer RNA to replace mutated exons that cause disease, Ascidian’s RNA-based exon editing technology provides the durability of gene therapy without the risks of direct DNA editing or gene replacement, while maintaining endogenous gene expression circuitry.
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, versatile RNA therapeutic approach.
Ascidian therapies enable precise post-transcriptional editing of genes, resulting in full length, functional proteins:
at the right levels
in the right cells
at the right time
Ascidian exon editors could help many more patients, with many more diseases, than is possible today.
Come aboard! We’re looking for scientists who are passionate about translating cutting-edge science into urgently needed new treatments for patients.
