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. The therapeutic candidate constitutes of ex vivo corrected CD34 positive hematopoietic stem and progenitor cells (HSPCs). It is administered through the intravenous route and is developed based on CRISPR/Cas9 gene-editing technology.

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.

This content was updated on 18 March 2024

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