RARE Daily

Ascidian Raises $40 Million to Advance Pipeline RNA Editors

November 13, 2023

Rare Daily Staff

Ascidian Therapeutics, a company focused on treating human diseases by rewriting RNA, raised $40 million in series A extension funding committed by Apple Tree Partners.

The extension financing follows ATP’s initial $50 million series A investment in Ascidian announced in October 2022.

The funds will be used to advance Ascidian’s lead program, an RNA exon editor to halt the progression of Stargardt disease or other ABCA4 retinopathies, and additional programs targeting the central nervous system and neuromuscular diseases in the company’s pipeline.

The company also announced that its founding CEO Michael Ehlers will return as interim president and CEO, effective immediately. Ehlers, who also chairs Ascidian’s Board of Directors and serves as chief scientific officer at ATP as well as a venture partner at the firm, succeeds Romesh Subramanian, who is departing Ascidian.

“Ascidian’s approach to edit RNA at kilobase scales has demonstrated its potential in non-human primate studies, and today we are poised to submit our plans for human trials of what we believe will be a one-time treatment to preserve sight in people with Stargardt disease,” said Ehlers. “This is only the beginning. The potential reach of altering RNA at this scale is vast.”

Ascidian designs RNA editors to replace multiple mutated exons simultaneously. Using this platform, Ascidian says it can edit genes too large to package in viral vectors and genes with high mutational variance, thereby addressing underlying causes of complicated genetic diseases that are beyond the reach of current gene therapy and base editing approaches. Because Ascidian’s technique does not introduce exogenous enzymes, the risk of adverse immune reactions is reduced. And because it does not modify DNA, the risk of off-target effects is decreased.

Earlier this year, Ascidian presented at the American Society of Gene and Cell Therapy Annual Meeting (ASGCT) six-month data from its lead program establishing the production of therapeutically relevant levels of full-length ABCA4 protein following a one-time treatment with a single AAV-delivered RNA exon editing development candidate in the non-human primate retina. These data represent the most efficient and durable RNA exon editing via trans-splicing ever achieved in large animals.

Ascidian anticipates submitting an Investigational New Drug Application to the U.S. Food and Drug Administration for its lead program in ABCA4 retinopathies in early 2024. Beyond its lead program, Ascidian is advancing programs with first-in-class potential in neurological and neuromuscular disorders.

Photo: Ascidian’s CEO Michael Ehlers

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