LONDON – CRISPR Therapeutics (NASDAQ:CRSP) and Vertex Pharmaceuticals (NASDAQ:) have achieved a significant milestone with the UK’s Medicines and Healthcare products Regulatory Agency (MHRA) granting conditional approval for their gene-edited therapy, Casgevy, to treat sickle cell disease (SCD) and transfusion-dependent beta thalassemia in patients 12 years and older. This landmark decision marks the first-ever global authorization for a CRISPR-based therapy, propelling the companies into a new era of medical innovation.
The therapy, known as exa-cel during its development phase, utilizes the revolutionary CRISPR/Cas9 gene-editing technology to correct genetic defects at their source. The treatment involves extracting stem cells from the patient, editing them in the laboratory to rectify the genetic anomaly causing the disease, and then reintroducing them after conditioning the body to prevent rejection.
The news of the approval sent CRISPR’s shares up approximately 6% in premarket trading today. As markets opened, CRISPR’s stock continued its ascent, climbing 5%, reflecting investor optimism over this groundbreaking achievement. The stock was trading around $59.10 per share, indicating a market valuation of about $4.5 billion. Vertex Pharmaceuticals, CRISPR’s partner in this endeavor, also saw a 1% rise in its shares following the announcement.
This development is particularly noteworthy as it follows positive feedback earlier this year from independent experts to the U.S. Food and Drug Administration (FDA). A decision from the FDA on exa-cel is eagerly anticipated by December 8. The UK approval could pave the way for further regulatory endorsements worldwide.
While the immediate financial impact on CRISPR’s earnings may be limited, the approval of Casgevy is expected to set a precedent for future gene therapies. CRISPR Therapeutics has already gained recognition in the scientific community after Emmanuelle Charpentier and Jennifer Doudna won the Nobel Prize in Chemistry in 2020 for their contributions to developing the CRISPR/Cas9 gene-editing technique.
Beyond SCD and beta thalassemia, CRISPR Therapeutics is exploring treatments for various other conditions including cancer, diabetes, and heart disease. As the company awaits a decision by the FDA on Casgevy’s potential use as a sickle cell treatment expected in March, industry veterans like Dana Blankenhorn are considering how FDA actions might influence stock volatility in the near future.
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