Ascidian Therapeutics develops a proprietary RNA exon editing platform that enables precise post-transcriptional modifications to replace multiple contiguous exons, addressing genetic mutations that vary among patients. This technology allows for the production of full-length, functional proteins at appropriate levels and in the correct cells, providing a safer alternative to traditional gene editing methods.
Funding
$98.1M raised to dateRaised to date based on public sources. This may differ from the amount the company actually raised and is based only on what is publicly available on the internet.
Founders
Product
Problem
Many genetic diseases are caused by mutations that vary among patients, making them difficult to address with traditional gene therapies that target single base changes. Existing gene editing technologies may also carry risks associated with direct DNA modification or gene replacement.
Solution
Ascidian Therapeutics is developing an RNA exon editing platform that enables precise post-transcriptional modification of RNA. This technology allows for the replacement of multiple contiguous exons, addressing genetic mutations that vary among patients. By rewriting RNA, Ascidian's approach aims to produce full-length, functional proteins at appropriate levels and in the correct cells, while maintaining endogenous gene expression patterns. This provides a potentially safer and more versatile alternative to traditional gene editing methods, without the risks of direct DNA editing or gene replacement. The platform is designed to expand the therapeutic possibilities of RNA medicine and treat diseases not addressed by current gene editing technologies.
Target Audience
The primary target audience includes researchers and pharmaceutical companies focused on developing treatments for genetic diseases with variable mutations.
Features
- RNA exon editing platform for precise post-transcriptional modification
- Ability to replace multiple contiguous exons, addressing diverse mutations
- Production of full-length, functional proteins at appropriate levels
- Maintains endogenous gene expression patterns and levels
- Avoids the risks associated with direct DNA editing or gene replacement