Vertex Pharmaceuticals Incorporated and CRISPR Therapeutics have announced a significant milestone in medical science with the U.S. Food and Drug Administration’s (FDA) approval of CASGEVY (exagamglogene autotemcel [exa-cel]), a revolutionary CRISPR/Cas9 genome-edited cell therapy. This approval marks a significant advancement in the treatment of sickle cell disease (SCD) for patients aged 12 and older experiencing recurrent vaso-occlusive crises (VOCs).

The approval of CASGEVY signifies a historic moment as it is the first CRISPR-based gene-editing therapy to be sanctioned in the U.S. It represents a pioneering treatment offering a potential one-time transformative therapy for eligible SCD patients.

Reshma Kewalramani, CEO and President of Vertex, expressed gratitude to patients and investigators for their trust, which facilitated this landmark approval. Samarth Kulkarni, Ph.D., CEO of CRISPR Therapeutics, shared his excitement about realizing the vision of transforming CRISPR technology into breakthrough therapies.

Dr. Stephan Grupp, Section Chief at the Children’s Hospital of Philadelphia, underscored the potential of CASGEVY to be a transformative treatment for SCD patients. Vertex is working with hospitals experienced in stem cell transplantation to establish a network of authorized treatment centers across the U.S. to offer CASGEVY.

CASGEVY, a genome-edited cellular therapy, modifies patients’ own hematopoietic stem cells to increase fetal hemoglobin production, thereby reducing or eliminating VOCs in SCD patients. This therapy has shown promising results in clinical studies.

Additionally, CASGEVY has received conditional marketing authorization in Great Britain and Bahrain for SCD and transfusion-dependent beta thalassemia (TDT) patients. It is currently under review by the European Medicines Agency and the Saudi Food and Drug Agency.

While CASGEVY’s use for TDT treatment in the U.S. remains investigational, Vertex has submitted a Biologics License Application (BLA) to the FDA for its potential use in TDT patients, with a target action date set for March 30, 2024.

The FDA’s approval of CASGEVY heralds a new chapter in the treatment of SCD, showcasing the potential of CRISPR/Cas9 technology in providing groundbreaking solutions for challenging medical conditions.

  CASGEVY, CRISPR Therapeutics, Dr. Stephan Grupp, Reshma Kewalramani, Sickle Cell Disease, US FDA, USA, Vertex Pharmaceuticals