Over the course of several months, with a great deal of care and the most advanced techniques available in a cutting edge laboratory, a researcher can produce a small protein. In contrast, an E. coli bacterium can produce a protein several orders of magnitudes longer in a matter of seconds. As a result, over the last fifteen years, biotechnology has focused not on creating new techniques out of whole cloth, but rather have attempted to adapt proteins and molecular machinery that already exist in cells to do tasks far more efficiently than otherwise possible. One company launching itself into the crowded biotechnology space is Editas, which plans to use the new technique of CRISPR gene editing to cure all manner of human diseases. While excitement for the company is high, it is important for investors not to be overly optimistic about the company or the technique it champions.
CRISPR may be one of the most consequential scientific discoveries of the 21st century. The Streptococcus pyogenes bacteria, familiar to most people as the microbe that causes strep throat, has a surprisingly complex immune system for a simple organism. A protein, called Cas9, uses a long string of RNA to guide it to a place in an intruder’s genome, where it can cut DNA, rendering viral invaders inactive. This protein, introduced into human cells, has enormous potential for treating human diseases. If research develops along the course it is currently expected to, the use of CRISPR can make extremely accurate changes to the genetic code, potentially curing inherited, otherwise untreatable diseases like Huntington’s Chorea.
While CRISPR may seem like a miracle to those who see the possibility of a cure for diseases that now represent a death sentence, there are many challenges to the widespread use of CRISPR for genetic editing in human beings. However, some intrepid entrepreneurs have jumped into the business anyway. Editas (EDIT), a biotechnology company explicitly focusing on the use of CRISPR, began trading on the public market on February 3rd. After opening at $16, Editas shares are now up by about 15%, but as of press time, was trading at about its starting level ($15.99). Selling 5.9 million shares, the firm raised $97.8 million, just short of its $100 million goal. However, all the buzz about CRISPR deserves a second look by investors before they choose to put their money in it.
First of all, the intellectual property relating to CRISPR is currently in great contention. Two laboratories, Jennifer Doudna’s at UC Berkeley and Feng Zhang’s at MIT, each claim the intellectual property that undergirds the technology. While both have a hand in Editas as a corporation, and have made their research available to the public for the purpose of further experimentation, the universities for which they worked at the time of discovery retain rights to patents issued. As of now, that means that MIT has the right to license CRISPR, but legal changes to the patent filing system mean that the lawsuit is unlikely to be resolved in the next two years. Meanwhile, all of Editas’ prospective profits rely on its ability to license the technology from the ultimate winner. That is, of course, assuming it is able to use CRISPR as an effective therapy. Numerous other biological systems, including engineering of Zinc-finger nucleases and transcription-activator-like effector nucleases, or TALENs, were received as revolutionary changes in human gene therapy, both have now been supplanted by CRISPR, as their initial promise never translated into the kind of easy gene editing their proponents hoped.
Of course, should CRISPR eventually be usable to edit genomes easily, it would not only represent a revolution in medicine, but would also present great challenges to the way society functions. Germ-line editing, or making gene edits that could be passed on, would not only raise the prospect of “designer babies,” but also might have unforeseen side-effects that could cause serious harm. The New Yorker reported in November that Doudna has dreamed of Hitler enquiring about the use of her technology. The first major study of CRISPR in non-viable human embryos took place last year at Sun Yat-Sen University in China. The research was considered so controversial that scientific journals of record including Science and Nature declined to publish it. While a study on viable human embryos was recently approved in Great Britain, fears about gene editing, especially as CRISPR develops as a technology may lead to legal intervention that could limit its use, and also limit profits from any company which spends a large amount on research and development, only to find regulatory barriers too high to bring its technology to market.
Though its founders are illustrious, and getting in on the technology of the century is certainly an attractive proposition for investors, caution is necessary. CRISPR has the potential to revolutionize the treatment of diseases of all kinds, but despite its widespread adoption in laboratories, its discovery is so recent that no Nobel Prize has been awarded for it yet, a sign the scientific community is still waiting to see if the many kinks in its practical use can be worked out.