Best Regions for Genetics Research

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.

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.