CRISPR Medicine

Ascidian Therapeutics

Company Type: Therapeutic development

Main focus: Developing RNA therapeutics to treat diseases

Company stage: Clinical

Diseases: Stargardt disease and and other ABCA4 retinopathies, as well as neuro- and neuromuscular diseases

Genome-editing tool: RNA exon editing

Funding stage: Series A

Location: Boston, Massachusetts, USA

Website: https://ascidian-tx.com/

Pipeline: https://ascidian-tx.com/programs/

Partners:


Ascidian Therapeutics is a clinical-stage company that aims to develop RNA medicines to treat various genetic diseases. The company's RNA exon-editing platform provides precise post-transcriptional editing of mRNA transcripts to restore full-length, functional proteins. Ascidian is currently initiating a clinical trial, called STELLAR, to evaluate the exon-editing candidate ACDN-01 in adults with Stargardt disease and other ABCA4 retinopathies.

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