Ascidian Therapeutics
Boston, United States· Est.
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Claim profilePrivate Company
Total funding raised: $50M
Overview
Developing RNA exon editing therapies to correct disease-causing mutations without altering DNA.
Genetics & GenomicsRare DiseaseNeuroscience
Technology Platform
An RNA exon editing platform that uses engineered trans-splicing molecules to correct disease-causing mutations at the RNA transcript level.
Funding History
1Total raised:$50M
Venture$50M
Opportunities
Potential to treat a broad range of genetic disorders, including those with large genes or dominant mutations, where DNA editing carries higher risks.
Risk Factors
Unproven delivery and durability of RNA-editing effect in humans, alongside competition from more mature genetic medicine platforms.
Competitive Landscape
Competes in the advanced genetic medicine space against DNA editors (CRISPR) and antisense oligonucleotides, requiring proof of key advantages in safety and applicability.