Challenges remain for Casgevy despite U.S., U.K. approval

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With authorities in the United States and the United Kingdom approving gene-editing treatment Casgevy, there is new hope for patients suffering from genetic diseases. But obstacles including excessive treatment costs and periods, as well as complex treatment processes, still need to be overcome, according to experts.

The U.S. Food and Drug Administration (FDA) estimates the number of patients eligible for Casgevy treatments within the United States to be around 20,000. But industry sources predict that far fewer patients will benefit for practical reasons.

The biggest obstacle is the treatment’s prohibitive cost.

Vertex Pharmaceuticals revealed that using Casgevy for genetic editing alone costs $2.2 million. Accessibility is also severely limited, with only nine U.S. medical institutions offering Casgevy-based treatments.

The number of patients each medical institution can accommodate is also limited.

A single institution can treat only about five to 10 patients annually, meaning that currently only around 100 patients in the United States can receive the treatment.

Vertex Pharmaceuticals stated that it plans to expand the number of treatment centers to 50 moving forward, but even with the expansion, only a maximum of 500 patients can be treated annually.

Even if patients receive treatment, a lengthy process lies ahead.

Casgevy treatments start with the collection of the patient’s bone marrow stem cells, and it takes several months for enough stem cells to be collected.

Editing the collected stem cells with Casgevy gene scissors takes an additional 17 weeks. To inject the new, edited stem cells into the patient’s bone marrow, a procedure that involves the complete removal of the patient’s bone marrow via chemotherapy is also necessary.

During this period, patients must be hospitalized for over a month as their immune system disappears completely.

An inevitable legal battle over Casgevy’s foundational technology is forming as gene-editing companies file their patents.

Broad Institute, founded by MIT and Harvard, UC Berkeley in the United States, and Korean gene correction technology company ToolGen Inc. are upholding their CRISPR technology patents, an indication of the legal disputes ahead.