PM360 2022 Innovative Startup Ascidian Therapeutics

Ascidian Therapeutics

Contact Person:
Dan Rosan, MBA, Chief Financial & Business Officer

2022 has been a defining year for Ascidian Therapeutics. Founded, built, and funded by life sciences venture capital leader Apple Tree Partners (ATP), Ascidian made its debut in October as a next-generation RNA therapeutics company, with the potential to revolutionize the field and expand the therapeutic possibilities of RNA medicine for patients awaiting breakthroughs.

By editing exons—long sequences of RNA which encode proteins in our cells—at the pre-mRNA level, Ascidian Therapeutics is creating medicines with the potential to treat diseases beyond the reach of current gene editing or RNA therapeutic approaches. If you imagine a lengthy RNA sequence as a “book,” where gene therapies edit single letters, Ascidian seeks to edit entire chapters. This approach allows Ascidian to address more mutations for more patients in more diseases than current gene editing or base editing approaches.

With active preclinical and discovery programs in ophthalmology, neurological and neuromuscular disorders, and rare diseases, Ascidian is developing a broad pipeline with the potential to impact some of the world’s most devastating diseases. Currently, IND-enabling activities are under way for Ascidian’s lead program targeting ABCA4 retinopathy, including Stargardt disease, the most common form of inherited blindness. There is no approved treatment for ABCA4 retinopathy, and diseases caused by ABCA4 loss of function are examples of genetic disorders that cannot be addressed by standard gene replacement, given the large size of the gene, or by base editing, due to the high mutational variance of the affected gene. Ascidian’s RNA exon editing approach has the potential to treat most patients with disease-causing ABCA4 mutations using a “one and done” dose of a single exon-editing RNA therapeutic.


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