FDA Okays First CRISPR Gene Therapy for Sickle Cell

The U.S. Food and Drug Administration (FDA) has approved two pioneering gene therapies for sickle cell disease (SCD), including the first-ever treatment in the United States based on the Nobel Prize-winning CRISPR gene editing technology.

Casgevy, developed by Vertex Pharmaceuticals and CRISPR Therapeutics, employs CRISPR technology, which utilizes molecular “scissors” to edit genes.

This groundbreaking approach, discovered by Jennifer Doudna and Emmanuelle Charpentier, has the potential to revolutionize treatments for genetic disorders.

An Alternative Approach by Bluebird Bio

In contrast, bluebird bio’s Lyfgenia works by inserting modified genes via disabled viruses. This method represents a different but equally significant advance in gene therapy.

Both treatments, aimed at patients aged 12 and older, are expected to be available in early 2024.

The therapies come with hefty price tags: $2.2 million for Casgevy and $3.1 million for Lyfgenia. These costs reflect the complexity and novelty of the treatments, which are pitched as one-time solutions.

Impact on Sickle Cell Disease

SCD, a painful, inherited blood disorder, affects approximately 100,000 people in the United States, predominantly in the Black community.

These therapies offer hope for a condition that can lead to debilitating symptoms and premature death. Robert Brodsky, President of the American Society of Hematology, hailed the approvals as

“A tremendous step forward for the SCD community, which has been historically overlooked and underfunded.”

Clinical Trials and FDA Remarks

Clinical trials showed significant improvements in patients with both treatments reducing painful episodes. FDA official Peter Marks emphasized,

“Today’s actions follow rigorous evaluations of the scientific and clinical data needed to support approval, reflecting the FDA’s commitment to facilitating development of safe and effective treatments for conditions with severe impacts on human health.”

The FDA has issued a warning for Lyfgenia due to a risk of blood cancer, a concern not observed in Vertex patients. Both companies plan to conduct a 15-year follow-up study to assess long-term safety risks.

Treatment with these gene therapies involves high-dose chemotherapy and a month-long hospital stay, with procedures like stem cell harvesting and gene editing in manufacturing facilities. Vertex’s CRISPR therapy is also under review for transfusion-dependent beta thalassemia, with a decision expected soon.

With information from Reuters.

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