Stuart K. Hayashi
The following is a section of the longer essay, “How Billionaires and Capitalism Save Billions of Lives — Including Yours.” That essay includes an index listing various case studies of a for-profit initiative saving lives. The blog post below is of at least one such case study. You can return to that index here.
Since then, the exciting opportunities in biotechnology have expanded. The gene-splicing method of CRISPR takes advantage of a protein from bacteria called Cas9. Viruses such as bacteriophages prey upon bacteria, and bacteria have hence evolved their own immune systems to protect them. The chemical Cas9 is a weapon whereby the bacteria slice viruses into separate pieces, neutralizing the threat they pose.
A team at U.C. Berkeley led by Jennifer Doudna demonstrated that Cas9 can be employed to slice strands of human DNA in specific places. The precision of Cas9 renders the 1971 method of Herbert Boyer and Stanley Cohen crude by comparison.
Various life-threatening diseases are caused by inherited genes. Such illnesses include sickle-cell anemia, cystic fibrosis, and Huntington’s disease. CRISPR can be employed to enter someone’s DNA and edit out the sequences that cause these illnesses to manifest. Although Dr. Doudna has stated that she refuses to go so far, it is even theoretically possible to edit such inheritable diseases out of an embryo. Once the embryo grows into an adult human being and has children of her own, there would be a substantially reduced risk of her own children and descendants inheriting that same genetic disorder. One generation inheriting the CRISPR-edited improvements of previous generations has been dubbed “germline genetic engineering.”
To commercialize medical treatments developed from CRISPR, Jennifer Doudna co-founded Caribou Biosciences with a former graduate student who worked under her, Rachel Haurwitz. Anticipating the profits to be made from the lifesaving treatments to be fostered, investors have driven up demand for shares in this publicly traded corporation. As of this writing, Caribou’s market capitalization — the total dollar value of all the company’s shares — happens to be $600 million. As Dr. Doudna
owns 4.19 percent of the company, her fortune comes to $24 million. The single largest shareholder is Dr. Haurwitz at 6.09 percent, which is over $36 million.
Other good work continues at a company with which Dr. Doudna has sometimes collaborated, Regeneron. Recall that to make trastuzumab — originally an antibody in mice — into something that the human body would accept, Axel Ullrich at Genentech had to employ genetic engineering to alter the antibody. This technique became one traditionally performed by biotech companies. They would first have the antibody inside of mice. Then, one antibody at a time, they would amend the chemical into one suitable for humans. That is a laborious process. Starting in 1985 at Columbia University, immunologist George Yancopoulos resolved there had to be another procedure that would save on time and resources.
Yancopoulos’s idea was to alter the mice themselves so that their immune systems would be more similar to those of human beings. That had an added benefit of addressing a recurring issue with testing the effect of particular pathogens or antibodies on mice. When medical scientists conduct tests on mice by exposing them to any particular substance, the mice are usually a model for how that same substance will affect human beings. Yet the immune system of a mouse, as a default, has significant differences from that of a human.
On numerous occasions, a particular treatment that worked well in mice turned out not to have the same effect in human patients. By genetically engineering mice to have immune systems more like those of humans, Yancopoulos has improved laboratory experiments. The effects on mice
have become more representative of what will happen to a human being.
Yancopoulos has earned over 100 U.S. patents, and his work as the chief of research and development at Regeneron Corporation has won the confidence of investors. Similar to what Jennifer Doudna has done for Caribou, the potential of the applications of Yancopoulos’s insights has driven demand for ownership in the company. The price of Yancopoulos’s stake has soared,
making him the first corporate R-and-D head to attain the status of billionaire.
The collaboration of Regeneron with a company in which Caribou Biosciences holds a 21.9 percent stake, Intellia, has already saved lives. Patrick Doherty inherited a rare degenerative nerve disease called transthyretin amyloidosis. Under this condition, a protein in the body destroys the nerves in the hands and feet of sufferers, crippling and ultimately killing them. In an experiment funded by both Regeneron and Intellia, Doherty and five other patients had their genomes altered by means of a CRISPR technique that Regeneron and Intellia have refined.
This procedure was one of doctors inserting billions of nanoparticles into the patients, each nanoparticle designed to trigger the CRISPR gene-editor. Once the nanoparticles entered the patient’s liver, the nanoparticles activated the gene-editor. Cas9 sliced apart the DNA inside the liver that was producing the destructive protein. The patients have
thus shown significant improvement.
Yancopoulos and Doudna take this as empirical evidence that CRISPR can indeed be employed to mitigate much-more-common genetic disorders.
Return to index of case studies of lifesaving for-profit ventures.
