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The field of gene editing technology is rapidly expanding in the global healthcare industry. The groundbreaking gene editing technology that garnered Emmanuelle Charpentier and Jennifer Doudna the Nobel Prize in 2020. With its potential to treat even the most daunting diseases like cancer, gene editing has the power to revolutionize healthcare worldwide. The forecast for the global gene editing market highlights the rapid progress of CRISPR gene editing technology, which has made its way from laboratories to clinical applications in just a decade.
BioIntel360 predicts that the global gene editing technology market will experience a compound annual growth rate (CAGR) of 28.2% from 2023 to 2027, reaching a value of US$8.4 billion in 2027, up from US$3.0 billion in 2023.
CRISPR gene editing has not only become the foundation for numerous innovative biotech startups but has also generated substantial value in the billions of dollars. Major pharmaceutical players like Bayer, Pfizer, and Regeneron have recognized the potential of gene editing technology and have shown interest in this field. These companies have formed strategic partnerships with pioneers in gene editing, aiming to find cures for life-threatening diseases, including cancer.
The recent FDA review of exa-cel marks a significant milestone for CRISPR. As the first of its kind to undergo scrutiny by U.S. regulators, exa-cel will serve as a test case for the agency's stance on gene editing. Additionally, this innovative therapy has the potential to become the first gene-based treatment for sickle cell disease, a condition with limited available treatments.
Sickle cell disease is distinguished by the existence of abnormally shaped red blood cells, leading to the occurrence of painful and potentially fatal obstructions. Preliminary results indicate that exa-cel can effectively alleviate these symptoms in a sustainable manner for most participants in clinical trials, although the long-term durability of these effects remains uncertain.
The investment community closely monitored the FDA review, as exa-cel will compete with Bluebird Bio's gene therapy for sickle cell, lovo-cel. Bluebird Bio submitted its application three weeks after exa-cel, and the FDA has until late June to decide whether to accept it. The agency's decision to split the review into two parts, with priority given to sickle cell, came as a surprise to some analysts. Despite the therapy having received other regulatory designations aimed at expediting evaluation, the faster review for sickle cell holds greater significance due to the larger market opportunity presented by the more prevalent disease.
The scarcity of treatment options for sickle cell likely influenced the FDA's decision, given that patients already have access to Bluebird's Zynteglo, another approved genetic medicine. Meanwhile, Vertex and CRISPR Therapeutics have unveiled promising new results for exa-cel in Phase 3 studies for sickle cell and beta thalassemia. These findings, to be further detailed in an upcoming medical meeting, demonstrate that the therapies have achieved their pre-specified goals.
In the beta thalassemia study, a significant proportion of patients did not require blood transfusions for at least a year, while others saw substantial reductions in transfusion volumes. In the sickle cell study, most participants experienced a significant reduction in pain crises for an extended period, with one patient having a complex medical history that included chronic pain.
Vertex and CRISPR Therapeutics have also submitted applications for exa-cel in Europe and the U.K., further underscoring the potential global impact of this innovative CRISPR-based therapy. As further developments unfold, the medical community eagerly awaits the FDA's decision and the opportunity to transform the lives of individuals affected by sickle cell disease and beta thalassemia.
BioIntel360 suggests that the field of gene editing technology is experiencing rapid growth and holds immense potential for revolutionizing healthcare globally. With the forecast predicting a significant compound annual growth rate (CAGR) in the global gene editing market, major pharmaceutical players are recognizing the value and forming strategic partnerships to harness the potential of gene editing in treating life-threatening diseases.
