• UGI USER
  • Dec 09, 2023
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SICKLE CELL DISEASE

Why in the News?

  • The U.S. Food and Drug Administration (FDA) granted approval for two gene therapies targeting sickle cell disease.
  • Lyfgenia, developed by bluebird bio, and Casgevy, a collaboration between Pharmaceuticals Vertex and CRISPR Therapeutics, received regulatory clearance.
Source: GBD

Utilization of CRISPR Gene Editing Technology

  • Casgevy marks a significant milestone as the first-ever treatment for sickle cell disease utilizing the groundbreaking CRISPR gene editing technology.
  • Both therapies are specifically sanctioned for individuals aged 12 years and older.

About CRISPR technology:

  • Overview: CRISPR, or Clustered Regularly Interspaced Short Palindromic Repeats, is a gene editing technology inspired by bacteria’s natural defence mechanism against viruses, utilizing the Cas9 protein.
  • Genetic Engineering Process: CRISPR involves manipulating genes, either adding new ones or suppressing existing ones, constituting a form of genetic engineering.
  • No External Gene Introduction: Unlike some genetic engineering methods, CRISPR doesn’t introduce external genes but works with the organism’s existing genetic material through the CRISPR-Cas9 system.
  • CRISPR-Cas9 as ‘Genetic Scissors’: CRISPR-Cas9 is often referred to as ‘Genetic Scissors’ for its precision in cutting and modifying genetic material, making it a powerful tool in genetic manipulation.

About Sickle Cell Disease 

  • It is an inherited blood disorder causing pain and a heightened risk of premature death.
  • In sickle cell disease, red blood cells produce flawed, sickle-shaped haemoglobin, impeding the proper transport of oxygen and leading to complications such as intense pain, strokes, and organ failure.

Impact :

  • Sickle cell disease, affecting around 100,000 individuals in the U.S., predominantly among the Black population.

Challenges and Considerations

  • Despite being positioned as potential one-time treatments, uncertainties persist regarding the long-term efficacy of both gene therapies.
  • Experts express caution, preferring to label these interventions as “transformative therapy” rather than outright cures due to continued challenges patients may face post-gene therapy.