FDA Clears First Clinical Trial of RNA Exon Editor...
CMN Clinical
Jan. 31, 2024
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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.
