Two New CRISPR-Based Gene Therapies for Sickle Cell Anemia Approved by the FDA

The FDA recently approved two groundbreaking CRISPR-based gene therapies for curing Sickle Cell Disease (SCD):

• Casgevy - developed by Vertex Pharmaceuticals and CRISPR Therapeutics

• Lyfgenia - developed by Bluebird Bio

By targeting the BCL11A gene, these therapies successfully correct SCD mutations and increase fetal hemoglobin (HbF) levels in patients. This is exciting news for patients and families living with this painful disease, as it presents a potential cure rather than just a treatment for symptoms. Increasing HbF levels helps alleviate the severity of SCD, reducing the frequency of painful crises and other complications associated with the disease.

While celebrating these advancements, it is crucial to acknowledge the challenges that remain in providing access to these transformative treatments. The staggering prices of $2.2 million for Casgevy and $3.1 million for Lyfgenia pose significant barriers to access for many patients. Healthcare systems and organizations are actively working to address these challenges by exploring various initiatives aimed at improving affordability and accessibility, including patient assistance programs and potential policy changes.

Furthermore, the approval of these therapies highlights the growing potential of CRISPR technology in the field of gene therapy. As research progresses, there is hope that additional CRISPR-based treatments will emerge for other genetic disorders, expanding the possibilities for curing diseases previously thought untreatable. The success of Casgevy and Lyfgenia sets a precedent for future innovations in gene therapy, inspiring a new era of precision medicine.

Video describing two newly FDA-approved gene therapies for sickle cell anemia and how they work

(Media source: https://www.youtube.com/watch?v=YWEFBWrDXyQ&t=1s)

Created: March 25th, 2024

Citation:

Commissioner, O. of the. (2023, December 8). FDA approves first gene therapies to treat patients with sickle cell disease. U.S. Food and Drug Administration. https://www.fda.gov/news-events/press-announcements/fda-approves-first-gene-therapies-treat-patients-sickle-cell-disease