ACDN-01 is under clinical development by Ascidian Therapeutics and currently in Phase II for Retinopathy. According to GlobalData, Phase II drugs for Retinopathy have a 51% phase transition success rate (PTSR) indication benchmark for progressing into Phase III. GlobalData’s report assesses how ACDN-01’s drug-specific PTSR and Likelihood of Approval (LoA) scores compare to the indication benchmarks. Buy the report here.

GlobalData tracks drug-specific phase transition and likelihood of approval scores, in addition to indication benchmarks based off 18 years of historical drug development data. Attributes of the drug, company and its clinical trials play a fundamental role in drug-specific PTSR and likelihood of approval.

ACDN-01 overview

ACDN-01 is under development for the treatment of retinopathy, including Stargardt disease. The therapeutic candidate is administered through sub-retinal route. It comprises of a DNA construct, which transcribes into mutation free exon only RNA with a highly specific binding domain for pre-mRNA. It is being developed based on AAV delivered RNA-an exon editing platform and acts by targeting retinal specific ATP binding cassette transporter (ABCA4) gene.

Ascidian Therapeutics overview

Ascidian Therapeutics is a biotechnology company developing exon-editing RNA therapeutics that target the underlying causes of disease in the therapeutic areas of ophthalmology, neurological and neuromuscular disorders. Ascidian Therapeutics is headquartered in Boston, Massachusetts, the US.

For a complete picture of ACDN-01’s drug-specific PTSR and LoA scores, buy the report here.

This content was updated on 12 April 2024

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GlobalData, the leading provider of industry intelligence, provided the underlying data, research, and analysis used to produce this article.

GlobalData’s Likelihood of Approval analytics tool dynamically assesses and predicts how likely a drug will move to the next stage in clinical development (PTSR), as well as how likely the drug will be approved (LoA). This is based on a combination of machine learning and a proprietary algorithm to process data points from various databases found on GlobalData’s Pharmaceutical Intelligence Center.