New treatments based on CRISPR have been in the works for years. In the final weeks of 2023, one from Vertex, Casgevy, became the first to earn regulatory approval in both the UK and the US for its ability to cure sickle-cell disease, a life-threatening condition.

Just six weeks later, the FDA widened the approval of Vertex’s CRISPR treatment to treat beta thalassemia (TDT), an inherited blood disorder in which the body doesn’t make as much beta globin as it should.

The relevance of CRISPR-based treatments like Vertex’s Casgevy for mental illnesses lies in the potential of CRISPR technology in treating genetic components of various disorders, including some mental illnesses.

Genetic Basis of Some Mental Illnesses: Certain mental illnesses have a genetic component. For example, disorders like schizophrenia, bipolar disorder, and major depressive disorder have been linked to specific genetic variations. The success of CRISPR in treating diseases with a clear genetic cause, like sickle-cell disease and TDT, opens the door to the possibility of using similar techniques to target and modify genes associated with mental illnesses.

Expanding Understanding of Mental Illnesses: The research and development of CRISPR-based treatments also contribute to a deeper understanding of the genetic underpinnings of mental illnesses. This enhanced understanding can lead to better diagnostic tools and more effective therapeutic strategies beyond gene editing.

The regulatory approval of CRISPR treatments for diseases like sickle-cell disease and TDT indicates a growing acceptance and understanding of gene-editing technologies. This sets a precedent for future approvals and may accelerate the development and testing of CRISPR-based treatments for mental illnesses.

In October 2023, NIH gave Yale a $40 million grant to research CRISPR-based treatments for brain diseases.

It’s coming.

References:

  • https://www.biopharmadive.com/news/fda-casgevy-approval-beta-thalassemia-vertex-crispr/704663/
  • https://www.forbes.com/sites/roberthart/2023/12/08/fda-approves-first-crispr-treatment-that-might-cure-sickle-cell/?sh=53b614463045