Exagamglogene autotemcel is under clinical development by CRISPR Therapeutics and currently in Pre-Registration for Beta Thalassaemia. According to GlobalData, Pre-Registration drugs for Beta Thalassaemia have a 75% phase transition success rate (PTSR) indication benchmark for progressing into Marketed. GlobalData’s report assesses how Exagamglogene autotemcel’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.
Exagamglogene autotemcel overview
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 Exagamglogene autotemcel’s drug-specific PTSR and LoA scores, buy the report here.