The Expanding Role of Pharmacists in Gene Therapy and Pharmacogenomics

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June 3, 2025

Pharmacists are playing a transformative role in the evolving landscape of biotechnology, particularly in the fields of gene therapy and pharmacogenomics. These innovations enable personalised medicine, allowing treatments to be tailored to an individual’s genetic makeup—improving efficacy, reducing adverse effects, and optimizing drug safety.

Gene Therapy and CRISPR-Cas9

  • Gene therapy modifies or replaces defective genes to treat diseases, with CRISPR-Cas9 being a breakthrough tool that allows for precise DNA editing.
  • A major milestone was reached in 2023 when the FDA approved exa-cel (Casgevy), the first CRISPR-based medicine for sickle cell disease and β-thalassemia.
  • Despite its promise, gene therapy faces challenges such as high costs, limited accessibility, and insurance restrictions, making it financially burdensome.

Pharmacists’ Involvement in Gene Therapy

Pharmacists are responsible for:

  • Managing storage, preparation, and administration of gene therapy products.
  • Educating patients and health care providers.
  • Monitoring for side effects.
  • Advocating for equitable access and helping health systems evaluate cost-benefit aspects.

Pharmacogenomics : Tailoring Medicine to Genetics

  • Pharmacogenomics studies how genetic differences affect a person’s response to drugs, enabling more personalized treatments—especially in areas like oncology and mental health.
  • It supports better outcomes and reduces adverse drug reactions.
  • However, it raises ethical issues, such as:
    • Privacy of genetic information.
    • Potential discrimination by insurers or employers.
    • Inequitable access, especially in under-resourced regions.

Ethical and Financial Challenges

Pharmacists are key to addressing:

  • Cost barriers in accessing advanced therapies.
  • Ensuring informed use of genetic data with strong privacy protections.
  • Advocating for inclusive research and fair distribution of pharmacogenomic benefits across communities.

Key Takeaways

  • Pharmacists are vital to integrating gene therapy and pharmacogenomics into mainstream healthcare.
  • CRISPR-Cas9 technologies offer potential cures but are expensive and not yet broadly accessible.
  • Pharmacogenomics enhances treatment personalization but must be implemented ethically and equitably.
  • Ongoing pharmacist education and involvement are essential to ensure responsible and effective use of these cutting-edge therapies.

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