Ascidian Therapeutics offers RNA Exon Editing Platform
ProductOperating Status: Currently Offered
Ascidian Therapeutics' platform replaces mutated exons at the pre-mRNA level via trans-splicing, without DNA edits or foreign enzymes.
Ascidian Therapeutics' platform replaces mutated exons at the pre-mRNA level via trans-splicing, without DNA edits or foreign enzymes.
Ascidian Therapeutics' platform designs RNA exon editor molecules that use pre-mRNA trans-splicing to replace multiple mutated exons simultaneously with wild-type exons. The approach works at the RNA level, preserving native gene expression control and avoiding the packaging limits and immunological risks of DNA editing.
The lead program targets ABCA4 retinopathy, including Stargardt disease, and the platform supports both viral and non-viral delivery vehicles.
Korro Bio's OPERA platform uses engineered oligonucleotides to make precise RNA edits, co-opting endogenous ADAR to correct disease-causing transcripts.
A programmable RNA editing platform developing therapies for genetic disease.
Wave Life Sciences' PRISM platform combines stereopure nucleic acid chemistry with ADAR-recruiting AIMers for therapeutic RNA editing in humans.
An engineered transfer RNA platform that suppresses premature stop codons to restore protein translation.
Korro Bio's OPERA platform uses engineered oligonucleotides to make precise RNA edits, co-opting endogenous ADAR to correct disease-causing transcripts.
A programmable RNA editing platform developing therapies for genetic disease.
Wave Life Sciences' PRISM platform combines stereopure nucleic acid chemistry with ADAR-recruiting AIMers for therapeutic RNA editing in humans.
An engineered transfer RNA platform that suppresses premature stop codons to restore protein translation.