Inspired by ocean “sea squirts,” Ascidian Therapeutics is floating to the surface with $50 million in series A treasure to fund its ambitions to rewrite RNA for therapeutic benefit.
The company, launched by venture capital firm Apple Tree Partners, is led by former Dyne Therapeutics CEO Romesh Subramanian, Ph.D., according to Wednesday’s unveiling. The primary focus for the company will be further developing its lead asset targeting ABCA4 retinopathy, including Stargardt disease, which the biotech said is moving toward an application to enter the clinic. The company did not include a more detailed timeline on when it expects to start phase 1 trials.
“I am excited to join Ascidian because the company’s technology truly has the potential to rewrite RNA,” Subramanian said in a release. “This will enable us to expand the therapeutic potential of RNA medicines for patients who are awaiting breakthroughs.”
According to the company’s website, the biotech found inspiration from its namesake. Ascidians, or ‘sea squirts’, are ocean creatures that are able to re-engineer their transcriptome through RNA trans-splicing and alternative splicing.
Ascidian is focusing squarely on RNA exon editing as the foundation of its platform and with Stargardt as the initial focus, the company is looking to remedy some 900 disease-causing mutations in the ABCA4 gene. Ascidian says it presented data earlier this year at the American Society of Gene and Cell Therapy annual meeting that the administration of one of its exon editors resulted in the expression of a full-length ABC4 protein in non-human primates.
Prior to joining Ascidian, Subramanian led muscular disease-focused Dyne as co-founder, chief scientific officer and CEO. He also co-founded mRNA-focused Translate Bio, which was bought by Sanofi in 2021.
The R&D work at Ascidian will be led by two men: Robert Bell, Ph.D., a former associate research fellow for gene therapy in the rare divisions unit at Pfizer, will lead the research team while former Harvard associate professor Jesse Gray, Ph.D., will head up the discovery unit.