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:
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.