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Thursday, July 18, 2024

Breakthrough Sickle-Cell Therapy Casgevy Offers Hope to Patients

By John Adiasi

Breakthrough Sickle-Cell Therapy Casgevy Offers Hope to Patients

In an extraordinary development for medical science, Casgevy, the first Crispr-based therapy, has emerged as a potential game-changer in the treatment of sickle-cell disease. Marie Tornyenu, a patient who has long endured pain in her hips and legs due to this debilitating condition, represents the countless individuals who could benefit from this groundbreaking treatment. Sickle-cell disease, predominantly affecting Black individuals, inflicts severe pain, organ damage, and premature death. While existing therapies have addressed complications, they have not targeted the root cause of the disease. With approximately 100,000 affected individuals in the United States and over 20 million worldwide, the urgency for a transformative solution is undeniable.

Vertex Pharmaceuticals, the manufacturer and marketer of Casgevy, is expected to unveil its pricing strategy imminently. According to the Institute for Clinical and Economic Review, a nonprofit organization that advises insurers and drugmakers on medication value, a fair price range for this therapy, considering its immense health benefits, would be between $1.35 million and $2.05 million per patient.

The rapid transformation of Crispr, a revolutionary gene-editing technology, from a scientific discovery to a commercial product is nothing short of remarkable. Just over a decade has passed since the description of Crispr’s gene-editing capabilities in a 2012 scientific paper by Jennifer Doudna and Emmanuelle Charpentier, which earned them the 2020 Nobel Prize in Chemistry. Building upon this groundbreaking work, biotech companies swiftly emerged to capitalize on the potential of Crispr. Among the pioneers was Crispr Therapeutics, co-founded by Charpentier and a key player in the development of Casgevy.

Boston Children’s Hospital played a pivotal role in this journey. Scientists at the hospital, inspired by the possibilities presented by Crispr, harnessed the technology in their pursuit of sickle-cell research. Their earlier identification of a gene that suppresses the production of fetal hemoglobin led to a breakthrough. By leveraging Crispr to deactivate this gene, the researchers successfully reignited fetal hemoglobin production, providing a viable substitute for the dysfunctional adult hemoglobin seen in sickle-cell patients. The patented research from Boston Children’s Hospital was licensed to Crispr Therapeutics, further expediting the development of Casgevy.

Dr. Daniel Bauer, one of the scientists involved in this transformative discovery, expressed awe at the speed with which the technology progressed from the laboratory to a therapy that holds the potential to improve the lives of numerous individuals. The realization of Casgevy as a viable treatment option marks a significant milestone in the fight against sickle-cell disease, offering hope and relief to those affected.


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