Ascidian’s lead programme is an RNA exon editor, which can potentially prevent the progression of ABCA4 retinopathies. Credit: fizkes / Shutterstock.com.

Ascidian Therapeutics has raised $40m in a Series A extension funding round led by Apple Tree Partners to progress the development of its lead and pipeline programmes.

The latest investment comes after the company raised $50m in an initial Series A financing round in October 2022.

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The company plans to utilise the latest funding capital to develop its lead programme and others in the central nervous system (CNS) and neuromuscular ailments.

Its lead programme is an RNA exon editor that can potentially prevent the progression of Stargardt disease or other ABCA4 retinopathies.

Ascidian also named Dr Michael Ehlers as interim president and CEO on an immediate basis.

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Ehlers is the founding CEO of the company and will take over the new role from current CEO Romesh Subramanian.

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Ehlers stated: “Ascidian’s approach to editing 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.

“This is only the beginning. The potential reach of altering RNA at this scale is vast.”

Ascidian recently reported six-month findings in which its lead programme induced therapeutically significant levels of full-length ABCA4 protein following a single dose in the retina of non-human primates.

The company plans to file an investigational new drug application with the US Food and Drug Administration to launch a trial of this programme in ABCA4 retinopathies in early 2024.

This content was updated on 25 January 2024