Universities Are Arguing Over Genome Editing Technology, But What Does The Law Have to Say About It?

Three major universities are now engaged in a patent lawsuit to protect their rights to use genome editing technology called CRISPR-Cas9. The fallout will have far more impact than the simple settling of ownership and intellectual property rights, however; experts believe that CRISPR-Cas9 may be the most efficient route toward a ticking off items on a laundry list of amazing biotechnological discoveries.

In April of 2014, the US Patent and Trademark Office (USPTO) awarded patents to Dr. Feng Zhang of the Broad Institute to develop the technology for commercial use. Zhang provided the research notebooks from his team in support of the contention that they invented the genome editing tool independently. The Zhang team also added innovations that the UC Berkeley team headed by Jennifer Doudna did not, according to Zhang. This provided the Broad Institute team with a competitive advantage before the USPTO.

UC Berkeley, Harvard, and MIT are all now battling for the IP rights to CRISPR-Cas9. The legal team of the UC Board of Regents asked the USPTO to reconsider award of patents to Harvard, MIT, and the Broad Institute in April. Their position is that the rights belong to Jennifer Doudna of UC Berkeley, the technology's rightful inventor. The UC Regents' legal team disputes the Zhang team's evidence-not in its entirety, but as to whether it proves enough independent innovation to award patents.

The regents are asking for a patent interference. If it is granted, an argument about who actually originated the CRISPR-Cas9 process will take place before a panel. This particular legal battle could cost millions and stretch out over the course of years. Greg Aharonian, director of the Center for Global Patent Quality, says "This is far from over."

This legal thicket may be messy and difficult to navigate. The Doudna team first described the technique in a 2012 paper in the journal Science. Since that time many scientists all over the world have used the CRISPR-Cas9 process to experiment on animal, human, and plant cells. The technology has also been used to research possible cures for cancer and sickle-cell anemia. In fact, last year there were more than quadruple the number of papers published describing work using the CRISPR-Cas9 process than there were in 2012 according to the National Library of Medicine.

The patents themselves are potentially worth billions of dollars. This is in large part because of how useful the experts believe the technology will soon be to determining the causes of genetic diseases-not to mention how instrumental the technology will be in DNA sequencing and modification.

CRISPR-Cas9 is notable because this gene editing technology allows scientists to insert defective portions of DNA with healthy sequences. This kind of DNA technology would do much more than just cure individuals stricken with genetic diseases. It can completely eliminate the faulty genes by erasing them from germline cells, guaranteeing that diseases like Huntington's are never passed down from one generation to another.

The CRISPR-Cas9 technology is "game changing," and works like "precise and programmable scissors" for reliable gene editing according to UC Berkeley Doudna Lab postdoctoral associate Brett Staahl. "We have a technology that in one treatment can cure the underlying cause of a genetic disease," said Staahl.

Ryan Clarke, a biochemistry PhD candidate and published scientist, along with James Hyun, a PhD student in the life sciences, have described some of the potential uses of CRISPR-Cas9 technology. The specific application of the technology may be instrumental to several breakthroughs: finding a cure for HIV; the a cellular ion pump, CFTR, which causes cystic fibrosis; the modification of the SHANK3 genes suspected of causing autism; the dystrophin gene which is typically mutated in muscular dystrophy; the engineering of CAR T Cells used to find and attack specific kinds of cancer; and various drug studies and discoveries.

Another reason this particular case is so notable is that the CRISPR-Cas9 technology is giving even its creator pause. Doudna led a team of ethical, legal, and scientific experts in a January 2014 discussion on the implications of editing germline cells using the CRISPR-Cas9 technology, noting, "The issue is especially human germline editing and the appreciation that this is now a capability in everyone's hands."

This conference was in large part an attempt to keep the use of the technology transparent and allay fears about "designer babies" enough to ensure that the technology can continued to be used for important disease-related applications.

"Given the speed with which the genome engineering field is evolving, the Napa meeting concluded that there is an urgent need for open discussion of the merits and risks of human genome modification by a broad cohort of scientists, clinicians, social scientists, the general public, and relevant public entities and interest groups," said an article summarizing the conference written by participants.

The conference also produced several guidelines for the use of the CRISPR-Cas9 technology, including: the avoidance of clinical application of human germline genome engineering; the creation of fora for discussion of the technology by bioethical and scientific experts; and the convening of a group representing experts and developers from around the world for policy reasons.

This technology and its capabilities can be used to edit out even innocuous or aesthetic traits as well as diseases. Of course the specter of designer children has been present before now, but the CRISPR-Cas9 technology makes creating children to specifications a much more immediate possibility. In fact, MIT neurobiologist Guoping Feng believes that humans with edited genes are only "10 to 20 years away,"-but only for those of a certain socioeconomic class.