A real breakthrough in Biotech
Casgevy in short
Casgevy therapy uses the CRISPR gene editing tool to modify the blood-producing stem cells in the patient’s bone marrow. Notably, Casgevy is the first gene-editing treatment on the market using Nobel Prize-winning CRISPR/Cas9 technology.
Vertex’s Casgevy is the first gene-editing therapy ever to receive regulatory green light. The Nobel Prize-winning technology CRISPR is used to edit human genes to treat diseases. The therapy is branded Casgevy (also known as exa-cel) and was developed in collaboration with CRISPR Therapeutics (ticker: CRSP).
Has been approved by many countries
On November 16, 2023, the British Medicines and Healthcare products Regulatory Agency (MHRA) approved the autologous cell therapy (exa-cel) proposed by Vertex Pharmaceuticals and CRISPR Therapeutics, trade name Casgevy, for the treatment of Conditions 12 years and older with sickle cell anemia (SCD) and transfusion-dependent beta-thalassemia (TDT).
Subsequently, the US FDA approved Casgevy, the first gene editing therapy in the United States, on December 8, 2023, marking a major scientific progress. The FDA is expected to decide on the use of the drug for TDT in early 2024. Bahrain also approved it in January 2024. The European Union has also approved Casgevy therapy for the treatment of sickle cell anemia (SCD) in February 2024.
Approval approvals from regulatory agencies in more countries should be coming soon, including Saudi Arabia and Canada.
Casgevy in detail
Casgevy is being developed as a potential one-time functional therapy for two rare blood disorders: sickle cell disease and transfusion-dependent beta thalassemia (TDT). Under the terms of the deal, Vertex will bear 60 percent of the project’s costs and will receive 60 percent of the profits from Casgevy. Commercialization of Casgevy is expected to cost $2.2 million.
Vertex is emerging in the rare disease drug market, focusing on diseases that have historically been underserved by the entire medical community. Its business, which generates billions of dollars in revenue and profits annually, currently revolves around a lineup of four products that are the only treatments on the market for the rare genetic disease cystic fibrosis. The company is also working on treatments in other disease areas, including a non-opioid drug to treat acute pain and two stem cell therapies for diabetes.
Casgevy’s first wave of approvals was initially expected to open the therapy to nearly 20,000 patients. However, looking at the incidence of sickle cell disease alone, it is estimated that nearly 100,000 people in the United States alone have the disease, and 20 million people worldwide. In short, this could be the start of a new era of growth for Vertex’s business, which is already in a strong position.
Technology used
The therapy works by using gene-editing tools to snip a section of DNA in bone marrow stem cells. This frees up the blocked gene to produce a type of hemoglobin (HbF) that is normally produced only by fetuses. HbF directs the production of normal hemoglobin, thereby treating SCD and TDT disorders.
Advantage
What’s more, the therapy only requires a single infusion to take effect, solving the problem of lifelong blood transfusions in patients with severe sickle cell disease.
Security concerns
Although the efficacy of exa-cel seems to be good, the FDA has concerns about the safety of this therapy, especially in terms of “off-target” effects. You know, since the birth of CRISPR gene editing technology in 2012, off-target has become one of the main factors limiting its development. If CRISPR gene editing technology cuts DNA fragments other than the intended target, it may destroy the function or regulation of non-target genes, causing serious consequences.
The importance of Casgevy
Vertex and CRISPR are the world’s first gene editing therapies approved for marketing. Casgevy is an important milestone in the biotechnology industry and may change the world’s biotech pharmaceutical landscape in the future.
Revenue split between Vertex and CRISPR
A course of treatment costs US$2.2 million in the United States. The two partners, Vertex and CRISPR, will split the proceeds in a 60/40 ratio, with 40% of profits and costs going to CRISPR.
Note: For Vertex and CRISPR, I will write an article each and post it on my blog in a few days to introduce these two companies respectively.
Potential opportunities
How big is the market?
According to statistics from Allied Market Research and Global Market Research, the global gene editing industry market size will grow from US$4.811 billion to US$5.412 billion from 2021 to 2022, with an annual growth rate of 12.49% in 2022. It is expected that the global gene editing market will reach US$36.061 billion in 2030, with a compound annual growth rate of 22.3%.
Don’t have high expectations
However, pharmaceutical analytics company GlobalData said that given that 88% of CRISPR drugs are in the early stages of development, it is unlikely to see CRISPR approved again in the short term.
Expand applicable groups
Casgevy may receive a label expansion, first to treat patients ages 5 to 11 years old — the current approval covers those 12 years or older. Additionally, hundreds of thousands of SCD patients are not eligible for Casgevy so far, but CRISPR and Vertex are working on potential label extensions to target them.
Drugs in pipeline
Beyond Casgevy, CRISPR therapies should make solid clinical progress over the next five years. In 2024 alone, the company expects clinical updates on its two leading oncology candidates, CTX112 and CTX131, and new clinical trials for other products to begin, while the company also has ongoing Phase 1 studies on other projects. In other words, 2024 should be a busy year for the company.
The work it does over the next 12 months should help lay the foundation for future significant regulatory successes beyond Casgevy. Within the next five years, the company is expected to gain approval for at least one CRISPR-based gene editing treatment.
I am the author of the original text, the essence of this story was originally featured on Smart Magazine, Issue of October 2024
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