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Top 11 CRISPR Stocks To Invest In

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In this article, we discuss the top 11 CRISPR stocks to invest in. If you want to skip our discussion of the gene editing industry, head directly to Top 5 CRISPR Stocks To Invest In

Genome editing refers to a set of technologies enabling scientists to modify an organism's DNA by adding, removing, or altering genetic material at specific locations in the genome, as defined by MedlinePlus. One prominent method is CRISPR-Cas9, derived from bacteria's natural immune defense system against viruses. In this system, bacteria create CRISPR arrays by incorporating small pieces of viral DNA. If the viruses return, the bacteria use RNA segments from the CRISPR arrays to recognize and bind to specific regions of the viral DNA. The enzyme Cas9 then cuts the DNA, disabling the virus. CRISPR-Cas9 stands out for its speed, cost-effectiveness, accuracy, and efficiency compared to other genome editing methods.

CRISPR has broad medical applications. For example, Swedish researchers utilized CRISPR-Cas9 to edit the rs10071329 genetic variant in human brown adipocytes, transforming it from A/A to G/G. This alteration increased PPARGC1B levels, resulting in elevated triglyceride accumulation, enhanced mitochondrial gene expression, improved mitochondrial respiration, and a heightened lipolysis response to norepinephrine. These findings suggest the potential of PPARGC1B genetic variants as therapeutic targets for precision medicine in obesity. Meanwhile, Chinese researchers demonstrated prime editing using CRISPR-Cas12a and circular RNAs in human cells. They developed circular RNA-mediated prime editor (CPE) systems targeting T-rich areas, enabling potential multiplex editing with efficiencies reaching up to 40.75% in human cells.

The valuation of the global CRISPR Cas9 market was $2480.95 million in 2022, and it is anticipated to increase at a compound annual growth rate (CAGR) of 34.05% through the forecast period, reaching $14395.61 million by 2028. Cell and gene therapies show potential for treating or curing certain cancers and rare diseases, though they are among the most expensive treatments globally, with some costing between $400,000 and $2 million per dose. Despite the high costs, the Cell and gene therapies market is anticipated to nearly quadruple over the next three years, increasing from $5.3 billion in 2022 to an estimated $19.9 billion by 2027.

2023 marked a significant breakthrough for cell and gene therapies, with seven FDA approvals in the US and one in the European Union. The global landscape includes over 2,000 ongoing clinical trials, with approximately 10% in Phase III, suggesting that around 75 therapies could be approved well before 2030. Projections for 2024 estimate approximately 17 approvals in the US and EU combined. Presently, there are 33 approved cell and gene therapy products listed on the FDA's website, with seven of them gaining approval in 2023. These include treatments for sickle cell disease, Duchenne muscular dystrophy, wounds in dystrophic epidermolysis bullosa (DEB) patients, severe hemophilia A, neutrophil recovery, and type 1 diabetes using innovative cellular and gene-based approaches. In 2024, experts expect the initial approval of adoptive cell therapy for solid tumors and the first approval of an allogeneic T-cell therapy in the United States.