The US, China and the UK are the top regions for genetics research.
Genetics research is notable for its cross-border, collaborative projects, with a worldwide network of scientists pulling together to make the latest breakthroughs. Perhaps most notably, the Human Genome Project, spearheaded by the US government, was undertaken at universities throughout the US, UK, France, Germany, Japan and China.
However, despite the international nature of genetics research, some regions are stronger than others for genetics investing. Notably, China, the US and the UK are pioneering research in this field.
CRISPR/Cas9 is changing the genetics landscape
An article from Wired describes it as “the genesis engine” because CRISPR/Cas9 has the power to easily alter DNA. For Quartz, the easiest analogy is “a biological find-and-replace tool” — the idea is that CRISPR/Cas9 can seek out a specific genetic sequence in a cell and replace it with another one.
The revolutionary technique has the potential to transform how we think about genetics; it can reverse the mutations that cause blindness, make wheat invulnerable to killer fungi and make cells impervious to the virus that causes AIDS.
American heavyweights battling for patent
On June 28, 2012, UC Berkeley biochemist Jennifer Doudna and her team published an article in Science, outlining the discovery that CRISPR/Cas9 can find specific strands of DNA, slice them with a precise molecular scalpel and change them into something new. In a corresponding patent application, Doudna suggests that this technology could be an elegant solution for genome engineering.
Meanwhile, Feng Zhang, a molecular biologist at the Broad Institute of MIT and Harvard, was similarly interested in the potential of CRISPR/Cas9. In January 2013, Zhang’s team published a paper in Science illustrating the ways in which CRISPR/Cas9 edits genes in human and mouse cells. It was at that time that Zhang asked the Broad Institute to file for an accelerated patent on his behalf (seven months after Doudna filed for hers). A year later, Doudna received news that the patent had been awarded to Zhang. Not willing to go down without a fight, UC Berkeley is actively fighting to get patent rights to this technology.
In April 2016, Doudna’s team filed a notice informing the board that a settlement between Berkeley and the Broad Institute could not be reached. That means the interference proceedings, initiated in 2015, will run their full course. Investors will want to watch how it plays out in 2017, but keep this in mind: the patent dispute could take several years to resolve.
This story of battling scientists at two of America’s most prestigious institutions is important for genetics investing because a huge amount of money is on the line. Any company that wants to work with anything other than microbes will have to licence this patent, bringing in billions of dollars in royalties (the products created with this technology have the potential to bring in billions more). Whoever ultimately controls the patent will have a huge influence over the future of genetics research and investing.
Geneticist George Church of Harvard Medical School presented some disruptive research associated with CRISPR/Cas9 in October 2015. The geneticist and his colleagues used the CRISPR/Cas9 gene-editing technology to inactivate 62 porcine endrogenous retroviruses in pig embryos.
This virus, as Nature notes, is embedded in all pigs, and, until now, could not be neutralized. That led to complications with pig-to-human organ transplantation, as there is an increased risk of rejection because of the virus. Church’s research has taken a significant step towards creating a steady supply of non-human organs for transplantation.
China pushing forward with experimental genetics research
Across the ocean, researchers at Sun Yat-sen University in Guangzho, China have caused international controversy over their experimentation with CRISPR/Cas9 technology. In April 2015, a research team led by Junjiu Huang published the world’s first scientific paper on the editing of human embryos.
The team was attempting to correct mutations in the gene causing beta thalassemia, a disorder that inhibits a person’s ability to make healthy red blood cells. According to another article from Quartz, the team injected 86 embryos with engineered CRISPR/Cas9 sequences that target the genes responsible for beta thalassemia.
Only 71 of the embryos survived the 48-hour period necessary for CRISPR/Cas9 to work; ultimately only 28 had the defective sequence removed, and even fewer successfully received the healthy genetic sequence in its place.
This experiment drew outcry for its experimentation with embryos. The team justified the ethics of this experiment by clarifying that it was carried out on abnormal, non-viable embryos. However, that didn’t reassure the scientific community, which by and large views this experimentation as the beginning of a slippery slope towards more invasive and selective types of genetic engineering.
“This news emphasizes the need for an immediate global ban on the creation of GM designer babies,” said Human Genetics Alert Director Dr. David King in an article in The Telegraph. “It is critical that we avoid a eugenic future in which the rich can buy themselves a baby with built-in genetic advantages.”
But in April 2016, another group of Chinese scientists published research which involved editing human embryos—this time to make them more resistant to HIV infection. Again, the experiment used non-viable embryos—and again, it led to criticism.
Tetsuya Ishii, a Japanese bioethicist, believes the experiment did not cross any ethical boundaries since informed consent was received from all egg donors. Nevertheless, he deems the study unnecessary. “Introducing CCR5Δ32 and trying repair, even in non-viable embryos, is just playing with human embryos,” he told Nature.
Other scientists agree. George Daley, a stem cell biologist, noted that the study makes no notable advancement to the field: “The paper doesn’t look like it offers much more than anecdotal evidence that it works in human embryos,” he told Nature, “which we already knew.”
UK rising to the challenge
While there’s certainly a lot of genetics research going on in the US and China, when it comes to cancer and rare diseases, the UK is looking to take the top spot. In 2014, Prime Minister David Cameron announced a series of investments totaling GBP$300 million and aimed at “unlock[ing] the power of DNA.” The program, which is expected to span four years, is being run by Genomics England and is expected to look at 100,000 genomes. The project “will see the UK lead the world in genetic research within years,” Cameron said.
The country has partnered with Illumina (NASDAQ:ILMN) for genome sequencing. On the partnership, Life Sciences Minister George Freeman commented, “Genomics England’s ground breaking partnership with Illumina confirms Britain’s position as a world leader in the field of genetic medicine. This project will help us map genomes on an unprecedented scale and bring better treatments to people with cancers and rare diseases for generations to come.”
And in 2016, the UK Human Fertilisation and Embryology Authority (HFEA) became the world’s first regulatory group to approve genetic modification in viable human embryos.
A team of London scientists, based out of the Francis Crick Institute, will alter genes that are active soon after fertilization, then cease experiments after seven days. The goal? To glean information that may one day help researchers develop infertility treatments.
Genetics investing frontiers
The high-profile genetics controversies in the US and China make it clear that these countries are at the forefront of genetics investing and research. And with the UK plowing forward with its own genetics research, it will be interesting to watch the sector for new breakthroughs and developments.
This is an updated version of an article originally published on Life Science Investing News on July 28, 2015.