Ascidian Therapeutics develops RNA exon editors that can replace multiple mutated exons simultaneously to address diseases beyond the reach of viral-vector gene therapy and base editing. Its platform can target genes too large for viral packaging and those with high mutational variance. The approach does not introduce exogenous enzymes, reducing the risk of adverse immune reactions, and it does not modify DNA, lowering off-target risk. The company’s lead program is an RNA exon editor intended to halt progression of Stargardt disease and other ABCA4 retinopathies. Ascidian plans to submit an IND for its lead ABCA4 program to the FDA in early 2024. Beyond ABCA4, it is advancing programs with first-in-class potential in neurological and neuromuscular disorders. The company is based in Boston, MA. Ascidian Therapeutics is a Boston, MA-based biotechnology company whose platform enables therapeutic targeting of large genes and genes with high mutational variance while maintaining native gene expression patterns and levels. Its approach aims to deliver the durability of gene therapy while reducing risks associated with DNA editing by deploying RNA editors that can replace multiple mutated exons without modifying DNA. The platform combines high-throughput molecular biology with computational biology and does not require the introduction of exogenous enzymes, which the company says lowers the risk of immunogenicity. Ascidian's lead program for ABCA4 retinopathy is in IND-enabling studies, and the company is advancing a pipeline across ophthalmology, neurological, neuromuscular, and rare diseases. The company plans to use proceeds from the Series A to expand development efforts to extend the therapeutic potential of RNA medicines. Romesh Subramanian serves as CEO.

Total raised

$90M

Funding rounds

2

Latest round

Series A

Latest activity

Nov 2023