News: CRISPR Genome-Editing Grows Up: Advanced Therapies Head For The Clinic.
Published 11:00 AM EST, Thur December 19, 2024
Barely a year after the first government approval for a gene-editing therapy, researchers at the American Society of Hematology’s annual meeting in San Diego, California, presented data on gene-editing approaches for treating cancer and blood disorders, and for making stem-cell transplants safer.
Gene-editing therapies are making significant advances, highlighted by the progress of CRISPR-based treatments such as Casgevy, which recently received approvals in multiple countries. At the American Society of Hematology’s annual meeting, researchers presented promising data on new applications of gene-editing for treating cancers, blood disorders, and enhancing the safety of stem-cell transplants. Notably, Casgevy has shown durable benefits, with its ability to produce fetal hemoglobin offering a functional cure for sickle-cell disease and β-thalassaemia. This therapy exemplifies how gene-editing can transform medicine, offering long-term health and quality-of-life improvements for individuals with heritable blood disorders.
Despite these breakthroughs, the complexity and cost of treatments like Casgevy pose significant challenges to accessibility. The therapy, priced at $2.2 million, is produced using a patient’s own blood stem cells in a months-long process, making it difficult to scale. Countries like the United Kingdom and the United States are incorporating Casgevy into healthcare systems selectively, with debates about cost-effectiveness, especially for treating sickle-cell disease. Public outcry has underscored the urgency of making such therapies widely available, as many patients remain unable to access these potentially life-changing treatments.
New data presented at the hematology meeting addressed concerns about the therapy’s durability, showing that benefits persist for at least five years. People treated with Casgevy reported reduced hospitalizations and blood transfusions, and over 90% of sickle-cell patients experienced no pain crises for a year. This evidence bolsters the case for broader adoption of gene-editing therapies, although economic and logistical hurdles remain key barriers to their widespread implementation.
The development of gene-editing therapies resonates with the cannabis industry, particularly in the context of advancing biotechnology for plant-based products. CRISPR technology has been applied to optimize cannabis strains, improving cannabinoid profiles, enhancing disease resistance, and boosting yield. Furthermore, the use of cannabinoids for managing pain, such as in sickle-cell disease, highlights a complementary therapeutic avenue. Both fields face challenges of accessibility, regulation, and cost, prompting a broader discussion about equitable healthcare innovations and the integration of biotech solutions into public systems. Lessons from gene-editing’s journey could inspire strategies for expanding cannabis-based therapies to underserved populations. Source: Nature
