Ascidian Therapeutics is pioneering a novel approach to RNA therapeutics by focusing on RNA exon editing, which allows for the precise rewriting of RNA to address genetic diseases. Inspired by the natural processes of ascidians, these marine organisms that re-engineer their transcriptome during development, Ascidian Therapeutics has developed a platform that enables the editing and replacement of human exons. This innovative technology aims to provide a more versatile and powerful therapeutic option compared to traditional gene editing methods, as it can replace multiple contiguous exons rather than just single bases. This capability allows for the creation of full-length, functional proteins that are expressed at appropriate levels in the correct cells, thereby addressing the underlying causes of various diseases.

The company's RNA exon editing platform is designed to offer the durability of gene therapy while minimizing the risks associated with direct DNA editing or gene replacement. By maintaining endogenous gene expression patterns, Ascidian's technology seeks to revolutionize the treatment landscape for genetic disorders that are often characterized by mutations at different sites across patients. This approach not only enhances the therapeutic possibilities of RNA medicine but also positions Ascidian Therapeutics at the forefront of the biopharmaceutical industry, as it develops solutions for diseases that have been previously overlooked by existing gene editing technologies.

United States

Ascidian Therapeutics is redefining the treatment of disease by rewriting RNA. By editing exons at the RNA level, Ascidian therapies enable precise post-transcriptional editing of genes, resulting in full-length, functional proteins at the right levels, in the right cells, at the right time. With active discovery and preclinical programs in ophthalmology, neurological and neuromuscular disorders, and other rare diseases, Ascidian is opening new therapeutic possibilities for patients in need of breakthroughs.

Ascidian Therapeutics

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