Exagamglogene autotemcel is under clinical development by CRISPR Therapeutics and currently in Phase III for Sickle Cell Disease. According to GlobalData, Phase III drugs for Sickle Cell Disease have a 47% phase transition success rate (PTSR) indication benchmark for progressing into Pre-Registration. 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.

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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

Exagamglogene autotemcel (Casgevy) is a genetically modified autologous CD34+
cell enriched population that contains human hematopoietic stem and progenitor
cells edited ex vivo by CRISPR/Cas9 at the erythroid-specific enhancer region of the BCL11A gene. It is formulated as suspension for intravenous route of administration. Casgevy is indicated for the treatment of transfusion-dependent β-thalassemia and also for the treatment of sickle cell disease in patients 12 years of age and older with recurrent vaso occlusive crises who have the βS/βS, βS/β+ or βS/β0 genotype in patients 12 years of age and older for whom hematopoietic stem cell transplantation is appropriate and a human leukocyte antigen matched related hematopoietic stem cell donor is not available.
Exagamglogene autotemcel (CTX-001) is under development for the treatment of sickle cell disease and beta-thalassemia in adults and pediatrics. The therapeutic candidate constitutes of ex vivo corrected CD34 positive hematopoietic stem and progenitor cells (HSPCs). It is administered through intravenous route and is being developed based on CRISPR/Cas9 gene-editing technology.

CRISPR Therapeutics overview

CRISPR Therapeutics is a gene editing company. The company’s portfolio consists of multiple therapeutic programs, including its proprietary technology, CRISPR (Clustered, Regularly Interspaced Short Palindromic Repeats)/Cas9 (CRISPR-associated protein-9), to precisely alter specific sequences of genomic deoxyribonucleic acid (DNA). The company’s products are primarily used in the healthcare industry, specifically in the treatment and prevention of both rare and common diseases. Its portfolio includes multiple therapeutic programs in areas such as hemoglobinopathies, immuno-oncology, autoimmune diseases, and type 1 diabetes. The company’s advanced program, Casgevy, is approved for treating severe sickle cell disease (SCD) and transfusion-dependent beta-thalassemia (TDT). CRISPR Therapeutics is headquartered in Zug, Switzerland.

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

This content was updated on 12 April 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.