It sounds like magic: Molecules that can seek out a misfiring sequence of DNA and snip it off, potentially curing a patient’s genetic disease.
The technology behind this groundbreaking advancement is called CRISPR/Cas9. Although it has been recognized with a Nobel Prize in Chemistry in 2020, it has remained largely inaccessible to patients who could greatly benefit from it – until now.
The Arrival of “Genetic Scissors”
The long-awaited moment has finally arrived. On Thursday morning, Vertex Pharmaceuticals (ticker: VRTX) and its partner CRISPR Therapeutics (CRSP) proudly announced that their CRISPR/Cas9 treatment Casgevy has received conditional approval from U.K. regulators. This marks the first ever approval for any CRISPR/Cas9 therapy worldwide.
Casgevy, previously known as exa-cel, offers hope for patients battling sickle cell disease and transfusion-dependent beta thalassemia – two serious genetic blood disorders. The Food and Drug Administration is also expected to make decisions on its approval for sickle cell treatment in early December and for beta thalassemia treatment next year.
Steady Response In the Stock Market
With the anticipation of U.K. approval already in mind, the announcement did not cause significant movement in the stock market. Vertex shares witnessed a minor decline of 0.5% on Thursday, while CRISPR shares experienced a modest increase of 0.9%. The S&P 500 as a whole saw a slight 0.2% drop.
Empowering Genetic Medicine with Challenges Ahead
The approval of Casgevy represents a significant leap forward for CRISPR/Cas9 technology and gene editing as a whole. However, it also heralds the challenges faced by these companies when it comes to commercializing the product, especially considering the astronomical prices they are expected to demand.
Drugmakers are notorious for setting eye-popping price tags on potentially curative therapies like gene editing drugs. Although the specific price for Casgevy treatment has not been announced yet, William Blair analyst Tim Lugo predicts that beta thalassemia patients in Europe may face a staggering cost of $1.6 million per patient.
Innovation Meets Complexity: The Treatment Process
The Casgevy treatment is an intricate process involving multiple steps. It begins with the extraction of blood stem cells from a patient’s bone marrow, followed by the editing of those cells. Once edited, the cells are then reintroduced into the patient’s body through infusion. Throughout this process, the patient undergoes chemotherapy and is required to stay hospitalized for approximately one month.
The conditional approval of the CRISPR/Cas9 therapy Casgevy in the U.K. sets the stage for a new era in genetic medicine. While challenges may lie ahead in terms of commercialization and affordability, the potential for life-changing treatments using gene editing technology cannot be underestimated.
Advancements in Gene Therapy: A Step Forward for CRISPR/Cas9
Gene therapy has long been a promising field in the medical community, offering the potential to revolutionize the treatment of genetic disorders. However, the high price tags associated with these therapies have often posed challenges, particularly in the European market. In fact, just two years ago, biotech company bluebird bio (BLUE) decided to halt the sale of its beta thalassemia gene therapy, Zynteglo, in Europe due to difficulties in securing payment from European payers.
Bluebird had initially proposed a price of $1.8 million per Zynteglo treatment in Europe, but faced significant obstacles in gaining support from payers. Andrew Obenshain, the company’s president, expressed his frustrations, stating that “European payers have not yet evolved their approach to gene therapy in a way that can recognize the innovation and the expected life-long benefit of these products.”
Despite setbacks in Europe, Zynteglo remains available in the U.S., where it was launched at a list price of $2.8 million per patient. Bluebird announced in November that 16 patients had commenced the process of receiving Zynteglo since its launch in the third quarter of 2022.
Analysts anticipate challenges in persuading insurers to cover the costs of gene therapies. William Blair’s Lugo emphasized that “securing broad access and reimbursement for gene therapies is not trivial and is more challenging than the same process for small molecules.” Lugo further predicted that both Vertex and CRISPR will secure approval for their respective drugs, which target sickle cell disease and beta thalassemia.
Following a positive assessment by an FDA advisory committee, Vertex shares experienced a slight decline of 2.4% on Tuesday and 5.6% on Wednesday. These declines were attributed to concerns surrounding ongoing trials for the company’s non-opioid pain medicine, VX-548, as discussed in a recent feature. Unfortunately, the approval of Casgevy was unable to reverse the stock’s downward trajectory on Thursday. Analysts estimate that Vertex’s risk-adjusted profits from the drug could reach $1.2 billion by 2028.
Nonetheless, this approval serves as a significant milestone for CRISPR/Cas9 and its parent company, CRISPR, both of which have an array of other drugs utilizing the same technology in their pipeline. CRISPR’s CEO, Samarth Kulkarni, expressed optimism for the future, stating, “I hope this represents the first of many applications of this Nobel Prize-winning technology to benefit eligible patients with serious diseases.”
In conclusion, gene therapy continues to advance, offering hope for patients with genetic disorders. While challenges remain, particularly in securing coverage for these therapies, the recent approval of Casgevy represents a notable step forward for CRISPR/Cas9 and the field of gene therapy as a whole.