CTX-310 is under clinical development by CRISPR Therapeutics and currently in Phase II for Homozygous Familial Hypercholesterolemia (HoFH). According to GlobalData, Phase II drugs for Homozygous Familial Hypercholesterolemia (HoFH) have a 74% phase transition success rate (PTSR) indication benchmark for progressing into Phase III. GlobalData’s report assesses how CTX-310’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.

CTX-310 overview

CTX-310 is under development for the treatment of mixed dyslipidemia, severe hypertriglyceridemia, homozygous familial hypercholesterolemia (hoFH) and heterozygous familial hypercholesterolemia (heFH). The therapeutic candidate comprises mRNA encoding Cas9 which is enclosed in a lipid nanoparticle (LNP). It acts by targeting angiopoietin like protein 3 (ANGPTL3) and is being developed based on CRISPR, Cas 9 gene-editing technology. It is administered through intravenous route.

The therapeutic candidate was under development for the treatment of atherosclerotic cardiovascular disease (ASCVD).

CRISPR Therapeutics overview

CRISPR Therapeutics (CRISPR) is a gene editing company. It focuses on the development of transformative medicines using its proprietary CRISPR/Cas9 gene-editing platform. CRISPR/Cas9 is a gene-editing technology that modifies, deletes, or corrects disease-causing abnormalities at its genetic sources. Its major development programs include ex vivo programs involving gene editing of hematopoietic cells; ex vivo programs in immuno-oncology; in vivo programs targeting the liver and additional in vivo programs targeting other organ systems including muscle and lung. It has research and development operations in Cambridge, Massachusetts, the US and business operations in London, the UK. CRISPR is headquartered in Zug, Switzerland.

For a complete picture of CTX-310’s drug-specific PTSR and LoA scores, buy the report here.

This content was updated on 10 June 2024

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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.