De-coding the future of healthcare

How gene-editing breakthroughs are revolutionizing disease treatments


Jeff Spiegel Aug 26, 2021

Key Takeaways

  • Recent breakthroughs in genomics are helping drive next-generation breakthroughs in medicine.
  • Gene therapies could transform the healthcare industry and could become a $36 billion market by 2027.1
  • Megatrend ETFs can help capture potential medical breakthroughs and keep investors diversified across numerous stocks.

    Once considered science fiction, the ability to edit malfunctioning DNA inside the body  became reality this summer with clinical data supporting the technique.2 A first-of-its-kind trial for a gene-editing treatment showed promising results for patients with an inherited liver disease — and no apparent safety issues.3

    The development, which draws on Nobel Prize-winning research, represents one of many in the field of genomic therapies.4 Such therapies work by inserting genetic materials into cells, an approach that researchers hope will prove to be more precise than many existing types of drugs.5 Genomics and related biological innovations have the potential to alleviate 1% to 3% of global diseases in the next 10-20 years.6

    The pandemic has demonstrated how drugmakers can quickly and effectively harness medical innovations. The mRNA technology used in vaccines to fight COVID-19 is being studied for potential deployment against rabies, influenza, Zika, HIV and cancer.7

    For investors, modern medicine breakthroughs could represent potential long-term growth opportunities. Consider that the market for gene therapies is expected to grow 34% annually, to $36 billion, by 2027.8 The U.S. Food and Drug Administration estimates that by 2025 it will be approving anywhere from 10-20 cell and gene therapy products per year.9

    Genetic sequencing at scale

    Advances in genetic sequencing underpin many advancements in life sciences, and the sequencing of millions of DNA codes has become cheaper and easier. Researchers can use sequenced codes to determine the genetic makeup of thousands of rare diseases, such as cystic fibrosis, hemophilia and sickle-cell disease, that afflict millions of people globally. Over the past two decades, the cost of genetic sequencing has come down more than 100-fold — from $100,000 in 2001 to less than $1,000 in 2020 — which allows for scalable scientific use and paves the way for a new generation of personalized therapies.10

    Sequencing costs ($) per Human Genome

    Genetic sequencing at scale

    Source: National Human Genome Research Institute, “DNA Sequencing Costs: Data,” August 2020.


    One revolutionary gene-editing technology is the CRISPR-Cas9 system. This technology allows researchers to isolate and manipulate parts of the human DNA that have “defects.” CRISPR-Cas9 consists of two components: a guide RNA, which can locate and recognize the sequence of DNA to be edited, and the Cas9 protein, which can cut out the part of DNA that is faulty. A guide RNA and Cas9 protein work together like Clippy, the iconic digital assistant in the office program Microsoft Word, locating and removing the faulty parts of the gene and replacing them with the correct ones.

    CRISPR-Cas9: A Cut and Paste Job

    CRISPR-Cas9: A Cut and Paste Job

    Source: BlackRock and Mayo Clinic. For illustrative purposes only.


    Falling costs of genetic sequencing technology, combined with the successful application of gene-editing, is bringing potential cures to these diseases much faster and cheaper than the last generation of drug discovery.

    Conclusion

    Given the remarkable groundbreaking results in genomics research, investors may want to consider a megatrends approach to investing in targeted themes like genomics. Index-tracking megatrend ETFs can help capture the potential of medical breakthroughs, while still diversifying across the many companies that are poised to potentially benefit from growth in the theme.

    Advances in biological sciences with the use of computing, data and artificial intelligence are opening new doors in healthcare that can provide more precise therapies for a broad array of diseases.

    Jeff Spiegel

    Jeff Spiegel

    Head of U.S. iShares Megatrend and International ETFs

    Tanya Chanda

    Vice President

    Contributor

    Mark Orans

    Associate

    Contributor