A revolutionary new technology called gene editing recently had a watershed moment. For the first time, gene editing successfully altered a defective genetic mutation in affected patients, presenting the possibility of a cure for a deadly genetic disease. Gene editing is one example of accelerating innovation in healthcare.
The view that gene editing is not just a technology but an innovation that will create an entire ecosystem is critical to our investment strategy. There are highly complex and specialized processes in every step–from research and technology needed to identify the mutations and deliver the drug throughout the human body to diagnostic testing equipment and specialized manufacturers for scaling commercial treatments.
When the first human genome was sequenced 20 years ago, the process cost $2.7 billion and took 13 years.1 Today it costs around $750 and can be done in a few days. These advancements may make it possible to build out data sets with critical mass that can offer further insights toward more personalized and effective treatments and cures.
Thanks to a technology called CRISPR-Cas9, gene editing is well on the way to these cures. Essentially, CRISPR-Cas9 enables geneticists and medical researchers to edit parts of the genome by removing, adding or altering sections of the DNA sequence. The ability to make highly specific changes in a DNA sequence, essentially customizing its genetic makeup, provides a path to remove or nullify bad mutations in genes as a way to cure diseases.
Source: Illumina, NHGRI, US Census Bureau as of 2019
The continued growth in genome data sets serves as a foundation for several key biotechnologies to grow, creating potential opportunity for investors. A recent clinical trial that deployed CRISPR-Cas9 gene editing directly into the body yielded highly promising results for those with a rare and fatal condition transthyretin amyloidosis. After a single treatment of the gene-editing therapy, within four weeks, the levels of bad protein causing the deadly condition plummeted over 80%. This data is the first good evidence that CRISPR-Cas9 can succeed when administered systematically. The possibility of now being able to edit genes directly in the body opens the door to treating a wider range of diseases and may influence a broader set of companies.
As we look to invest across this ecosystem, we believe small and mid-sized growth companies with exposure to gene editing technologies and manufacturing processes will become more interesting. Among the gene editing technologies we are investing in is a next-generation technology called base editing, which builds on the CRISPR-Cas9 construct but seeks to make less invasive edits. Another company is working to develop off-the-shelf cancer therapies enhanced by gene editing. We also own a company that pioneered a critical technology for RNAi medicines. Similar to the gene editing constructs, the company’s technology uses lipid nanoparticles to deliver the drug through the human body and to the liver – these are also used in today’s mRNA Covid vaccines, which many of us have recently received.
Within the supply chain, many companies are well positioned as “enablers” – or companies involved in all aspects of the gene editing ecosystem, from research and development to identify mutations and deliver the drug, to diagnostic testing, and to manufacturing and production for scaling commercial treatments. The world will have to prepare for commercial volumes of drugs based on genetic material. One of the companies we own is focused on improving manufacturing efficiency for the gene therapy reagents to meet a demand curve that will increase exponentially. Academic research in the field of genetic medicine is exploding and another company we invest in provides tools to simplify and accelerate CRISPR-Cas9 experiments and analysis.
As the technology continues to advance, we believe the opportunity to positively impact portfolios will as well.
1. Source: Forbes, “How Human Genome Sequencing Went From $1 Billion A Pop To Under $1,000” as of October 2020.