Biotech is heralding the future of medicine
Gene-editing tools may transform approaches to human disease
The sequencing of the human genome is often considered the grand crossing of the Rubicon that pivoted biotech companies to the forefront of drug development. But from our perspective it is the tools used to analyze and test genomic data that have led us to this new golden age--the most notable being gene-editing tools such as CRISPR-Cas9. CRISPR stands for Clustered Regularly Interspaced Short Palindromic Repeats. It works like a pair of molecular scissors, cutting DNA at specific locations and either can delete disease-causing regions and/or replace them with sequences restoring gene function.
The impact of CRISPR so far has been hard to quantify given its enormous therapeutic potential to correct the underlying pathology of rare and common diseases. Since the first publication in demonstrating the utility of CRISPR in human cells in 2012, more than a billion dollars in venture funding has been invested in gene editing companies. The three original CRISPR companies have generated enormously outsized returns since their initial public offers in 2016. In some industries, this may signal a peak but for biotechs this is just scratching the surface.
Nonetheless, with more than 10,000 known monogenic genetic diseases and around two million people diagnosed with cancer in the U.S. alone, according to the National Cancer Institute, three companies are unlikely to solve all the problems at hand. Given the need and the promise already seen with initial gene-editing-based therapeutics, more than 50 public and private companies are utilizing gene editing to develop next-generation medicines for a wide range of diseases.
We have a potentially curative therapy, but who is going to pay for it?
The investments needed to prevent a child from going blind or a person dying from a rare form of cancer are unlikely to be impacted by the macro-economic environment—especially considering how past and current drug-pricing debates have largely focused on diabetes, heart disease and other chronic diseases affecting the masses. Instead, companies bringing curative and life-saving therapies to the market are pivoting toward value-based pricing or demonstrating the value of such therapies to help alleviate human suffering and, in the process, benefit humanity. Rather than being a burden on the health-care system because of its initial expense, a one-time therapy to eliminate a cause of childhood blindness, for example, or a debilitating, life-threatening disease is much more likely to generate a greater return over the long term based on quality of life and productivity.
Multiple gene-edited-based products are now in the clinic, with next-generation companies launching at a dizzying pace. We believe new companies present investment opportunities, but the challenge is to pick the winners that will move the field forward and change the way we approach human disease.
Francis Collins, Director of the National Institutes of Health, sums up the great potential that lies ahead for CRISPR-Cas9: “Once in a long time, an advance comes along that utterly transforms an entire field and does so very rapidly.” There’s good reason for all of us to be excited about the future of medicine.