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CRISPR Breakthrough: Regulatory Green Light for Sickle Cell Cure

Monday, June 3, 2024


A novel gene therapy for sickle cell disease, which uses the genome-editing technology known as CRISPR, has received regulatory approval in the United Kingdom, United States and the European Union. This marks the first time that researchers have successfully transitioned CRISPR from the laboratory into clinical practice for treatment in humans.

CRISPR stands for Clustered Regularly Interspaced Short Palindromic Repeats and was developed only 11 years ago. Controversy surrounding the technology peaked in 2018 when scientist He Jiankui edited embryos, allegedly conferring HIV resistance, which resulted in the birth of twins in China. The research sparked a fierce uproar over the ethics of gene-editing, including the risk of unintended mutations and concerns over “designer babies”. The response was a significant increase in regulation over human genome-editing, making the recent approvals for therapeutic use particularly noteworthy.

Vertex Pharmaceuticals and CRISPR Therapeutics treated 30 patients with sickle-cell anemia by editing their bone marrow. Nearly all of the patients who volunteered in the trial were pain free within just one year. Similarly, 39 out of 42 patients receiving treatment for beta thalassemia no longer required blood transfusions to counter severe anemia. These results offer new hope for those living with the disease, which causes debilitating symptoms due to a single genetic mutation.

Several caveats. The expected price tag of the gene-editing treatment is approximately $2.2 million USD per patient. Navigating CRISPR licensing agreements, which are currently held by a limited number of companies, may pose further challenges to market accessibility. Vertex Pharmaceuticals also has no immediate plans to offer the treatment in Africa – where sickle cell disease is most common. In countries that still struggle to cover basic health needs, the procedure remains too demanding. On the medical front, significant challenges persist such as immunogenicity, where the immune system targets and combats the engineered cells.

Despite ongoing obstacles, the transition from bacterial experimentation to human treatment in just 11 years is an outstanding medical achievement. It may only be a matter of time before the technology is applied to other genetic conditions.

Author: Emily Groper, 2023-2024 Articling Student


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