Rewriting the Book of Life: A New Era in Precision Gene Editing

By Elsy Boglioli

What if scientists could correct the mutations that cause diseases like cancer, cystic fibrosis, muscular dystrophy, and sickle-cell anemia? Or engineer cells to fight cancer? Or restore the genes that were bred out of plants long ago, increasing yield and pathogen resistance or improving nutritional properties?

We have the technology to do so. It’s called gene editing. Scientists can now modify DNA sequences by inserting or deleting genes in living cells and organisms. And the newest gene-editing techniques have become safe, effective, and cost-efficient enough to gain traction in a wide range of industries. In particular, two nuclease-based gene-editing technologies, TALE and CRISPR, allow genetic scientists to edit genes with extraordinary precision in just weeks, as opposed to months or years.

Not surprisingly, this revolution has sparked intense patent activity (with an annual growth rate exceeding 40 percent), led to new big-pharma partnerships, and triggered a series of high-profile IPOs. It has also captured the enthusiasm of investors. Over the last two years, more than $1 billion in venture-capital funding has been invested in emerging gene-editing technologies.

As the pace of development accelerates, we see several areas where gene-editing technologies will have a tremendous impact:

  • Curing Disease. Companies are already making strides toward modifying immune system cells to prevent them from being infected with HIV, developing therapies to treat hemophilia, and engineering immune cells to specifically target and kill cancer cells.
  • Improving Agriculture. The twenty-first century agricultural business will be transformed by nuclease-based gene editing. This technology offers much more precise and controlled ways to modify critical traits in crops and animals. The CRISPR and TALE systems make it possible to introduce mutations that are genetically indistinguishable from those resulting from natural breeding. Scientists have therefore proposed that such traits not be classified as GMOs, which could dramatically increase their public acceptance. (See Crop Farming 2030: The Reinvention of the Sector, BCG Focus, April 2015.)

This is just the beginning. More disruptive changes are sure to follow, including bioproduction (the use of modified organisms as living factories to create materials with biological origins, such as wool, leather, wood, and rubber) and even gene editing in human embryonic stem cells. The National Academy of Sciences and the National Academy of Medicine have launched a new initiative to inform decision making related to the controversial area of human gene-editing research. In December 2015, the initiative will convene global experts to discuss scientific, ethical, and governance issues.

In short, gene editing is here to stay. Big pharma, biotechs, and investors have already made big bets and the value will continue to grow as the industry develops new uses and new generations of technologies. The key challenge for all stakeholders will be to pursue the right opportunities. The winners will be those that invest based on a deep understanding of the technologies and their performance in terms of several criteria: efficiency, specificity (minimizing off-target activity, which directly affects safety), ease of delivery with relevant vectors, and ease of design. Now is the time for the pharma industry to build the capabilities needed to deliver these benefits.

Click here to learn more about how to assess gene-editing technologies and gauge their performance.