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FDA Approves, First CRISPR Gene Therapy, Casgevy, for Sickle Cell Disease

In a historic decision, the FDA has approved Casgevy, the first-ever CRISPR-based gene editing therapy for sickle cell disease (SCD), marking a milestone in medical science. With the potential to offer a one-time cure by modifying patients' blood cells, Casgevy brings hope to SCD patients. This development not only signifies a leap in gene therapy but also offers renewed optimism for rare genetic disorders.

Vertex Pharmaceuticals and CRISPR Therapeutics jointly announced the FDA's approval of Casgevy™ (exagamglogene autotemcel), a CRISPR/Cas9 genome-edited cell therapy, for sickle cell disease (SCD). This groundbreaking decision opens the door for thousands of SCD patients to become eligible for a durable one-time therapy that offers a functional cure for their disease.

“Sickle cell disease is a rare, debilitating and life-threatening blood disorder with significant unmet need, and we are excited to advance the field especially for individuals whose lives have been severely disrupted by the disease by approving two cell-based gene therapies today,” stated Dr. Nicole Verdun, Director of the Office of Therapeutic Products within the FDA’s Center for Biologics Evaluation and Research. “Gene therapy holds the promise of delivering more targeted and effective treatments, especially for individuals with rare diseases where the current treatment options are limited.”

A Beacon of Hope for SCD

Sickle cell disease, often referred to as SCD, is a hereditary blood disorder affecting approximately 100,000 people in the United States. It results from a genetic mutation that causes red blood cells to assume a characteristic crescent or "sickle" shape. These misshapen cells can obstruct blood vessels, leading to agonizing pain crises and the risk of organ damage. Patients with SCD have long faced limited treatment options.

“CASGEVY’s approval by the FDA is momentous: it is the first CRISPR-based gene-editing therapy to be approved in the US. As importantly, CASGEVY is a first-in-class treatment that offers the potential of a one-time transformative therapy for eligible patients with sickle cell disease,” said Dr. Reshma Kewalramani, CEO of Vertex.

Casgevy therapy harnesses the revolutionary CRISPR/Cas9 genome editing technology to target and modify a patient's hematopoietic (blood) stem cells, ultimately leading to the production of fetal hemoglobin (HbF). Elevated levels of HbF prevent the characteristic sickling of red blood cells, potentially eliminating severe pain crises and hospitalizations.

The journey to Casgevy's approval spans a decade. CRISPR technology, enabling precise DNA editing, was discovered just over a decade ago. In this short span of time, it has evolved from a laboratory experiment into a groundbreaking gene therapy.

The FDA's approval comes shortly after the United Kingdom and Bahrain granted Casgevy approval, drawing praise from leading scientists in the CRISPR field, including Nobel laureates Jennifer Doudna and Emmanuelle Charpentier.

A Price Worth Saving Lives?

However, concerns linger regarding the accessibility and affordability of Casgevy. The therapy carries a price tag of $2.2 million per patient, a figure that has generated both support and criticism.

Data Supporting Casgevy

In a trial involving 44 patients with Sickle Cell Disease (SCD), Casgevy's safety and efficacy were assessed. Patients, both adults and adolescents, had a history of multiple severe Vaso-Occlusive Crises (VOCs). Over a 24-month follow-up, 93.5% of the 31 evaluable patients avoided severe VOCs for 12 consecutive months. All participants achieved successful engraftment, with no cases of graft failure or rejection reported. Common side effects included low levels of platelets and white blood cells, mouth sores, nausea, pain, vomiting, febrile neutropenia, headaches, and itching, indicating a significant but manageable side effect profile.


About Author: Hüseyin Kandemir

As a medical journalist with over 20 years of experience, I have served as an Editor and Reporter for various magazines, newspapers, and online broadcasting platforms, consistently demonstrating a strong commitment to excellence in journalism. My specialization includes medical science, digital health, AI for health, data science, and biotechnology.

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