Robin, Who Wears a Hood

16 October 2014 | Genes^{PATENT POWER} | № 7

Meet CRISPR, acronym for the slightly less wieldy Clustered Regular Interspaced Short Palindromic Repeats. You may have come across the name on your socially mandated internet news update wanderings. CRISPR is a new genetic tool; a technique for manipulating genes that allows scientists to easily and efficiently modify genes in living cells. CRISPR is a process (originally discovered in bacteria) that generates Cas, enzymes matched with guide RNA sequences (think, an RNA-based recognition system) that lead them to specific DNA targets and allows them to make cuts at precise locations (i.e., RNA-guided nucleases with customizable specificities).

The basic idea: CRISPR is the newest in cut-and-paste gene editing technique. And it’s fast. And cheaper than your run in the mill targeted genome editing tool.

The realm of biology (and medical biology) has mapped the human genome. The next summit is personalized medicine, here: tailor-made therapies for patients using their own cells to correct genetic diseases. The field of genetic engineering is in rapid flux, but suffers from the theme of producing expensive and time-consuming techniques. CRISPR is powerful because it can modify genes in biomedically important cells -that have been traditionally challenging to manipulate genetically- in a matter of hours, not weeks, not months. The molecular medicine applications are enormous.

Now stop. Think of the inevitable questions that start popping up. Think about ownership.

April 2014, the USPTO granted the Broad Institute and MIT US Patent No. 8697359, “CRISPR-Cas systems and Methods for Altering Expression of Gene Products”. This is a patent on the ability to edit a genome to treat human genetic diseases. This is an exclusive right to make, use, and sell CRISPR-Cas technology. Have sickle cell? Want to give the CRISPR-Cas a run? Take it up with Broad Institute and MIT.

I am not against patents. In fact, patents are a powerful policy tool.

The justification behind patents is to offer a quid pro quo to the inventor: the government gives the inventor a limited monopoly on their invention (i.e., all the profits for a set number of years) in exchange for detailed public disclosure of the invention. Patents foster and encourage innovation by incentivizing disclosure. Disclosure is the beating heart of progress: the global research community builds on each other only through detailed and precise communication. Without an incentive, innovators would be hesitant to share their inventions; what if someone steals it and claims it as their own, leaving the inventor without any reward for their toil?

There are parameters. Patents are not given out for scientific discovery – the laws of nature, physical phenomena, and abstract ideas are not patentable. Policy-wise, the law tries to sort out human-made inventions from products of nature, living or not. This is the crux of the broadly-labeled ‘gene patent dispute’(case law, for the eager out there: Mayo (SCOTUS, 2012), Myriad (SCOTUS, 2013)). DNA, RNA, proteins, cells, fluids – these are naturally occurring. But what about this engineered RNA-guided nuclease? Does it belong to the public domain because it could (conceivably) occur naturally? Remember: you cannot get a patent on mere scientific discovery; discovering RNA-guided nucleases and their use in targeted genetic modification is not patentable subject matter. The USPTO granted the patent to the Broad Institute and MIT for their engineered nucleases. Now, the CRISPR-Cas technology belongs to the Broad Institute and MIT. And they have the exclusive right to make, use and sell CRISPR-Cas technology.

We have a duty to think about these questions, because they concern us and our bodies. Think about the way in which you own the software on your computer. Short and sweet: you don’t own it. You sign a license (an End User License Agreement – EULA) whereby you are allowed to use it as permitted by the software developer license terms. Now, translate this ownership scheme to genetic engineering techniques. If a surgeon uses CRISPR technology to play around and rewrite your DNA, does your DNA (or at least the inputted segments) still belong to you? Naturally-occurring DNA, RNA, cells, proteins…can these be patentable subject material even if they are a laboratory product?

It’s a brave new world out there, and the currency is knowledge. Our technology transcends magic. And whether or not we chose to acknowledge them, our laws are powerful instruments. Yes, it does matter what the Patent Act says. What is patentable subject matter is not a question reserved for scholars; it is a question of ownership. So, who owns your DNA?

For those in awe at the sheer magnitude of human progress, caveat: CRISPR is still in its experimental stages and just starting to undergo first clinical trials.

keywords: targeted genome editing, personalized medicine, CRISPR, patents, quid pro quo, ownership, EULA, molecular therapeutics