The USPTO decided in February that the rightful intellectual property owner of CRISPR in eukaryotes, a time-saving tool that makes it cheaper and easier to edit gene sequences, should be Feng Zhang, Ph.D., and The Broad Institute of MIT and Harvard, not Jennifer Doudna, Ph.D., and the University of California, Berkley, who had conducted the earlier research.

However, Doudna and her team, which included Emmanuelle Charpentier, now with Max Planck Institute in Berlin, are on track to obtain a European patent for CRISPR. They recently filed an appeal against the USPTO’s decision, setting the stage for a showdown.

CRISPR will allow an organism’s DNA to become “almost as editable as a simple piece of text.” Using CRISPR, scientists will have the capacity to alter, insert and delete genes in plants, animals and, even in humans.

The implications are very big indeed, both in terms of science and profits, and, especially, ethics. Universities and businesses stand to generate potentially billions of dollars. Medical research will never be the same.

[For a good description of how CRISPR-Cas9 works, go here. ]

The battle lines are being drawn to determine the rightful owner of aspects of the development: Berkeley and Dr. Charpentier vs. Broad Institute/MIT and Harvard. It could mean an eventual pay-out of billions of dollars.

World-Changing

In 2012, Cal biochemistry and molecular biology professor Jennifer Doudna and microbiologist Emmanuelle Charpentier, now of the Max Planck Institute, changed the world. They invented CRISPR-Cas9 (as opposed to eukaryotes, which is any organism with a nucleus enclosed in membranes), a gene editing tool that uses a protein found in Streptococcus bacteria to chop up and rearrange viral DNA with precision.

“The implications of the technology were immediately apparent, astonishing, and perhaps just a wee bit scary.” 

“The implications of the technology were immediately apparent, astonishing, and perhaps just a wee bit scary,” reports California Magazine. “Ultimately, CRISPR applications might be developed to wipe out genetic diseases, produce bespoke bacteria that could pump out everything from hormones to biofuels, and engineer exotic animal chimeras.”

It is one thing to use an editor to eliminate genetic mutations, such as those found in sickle-cell anemia, writes the Wall Street Journal, however, “it is quite another thing to edit the germ line—that is, to make changes that would be passed on to future offspring.

“Would it be permissible, Ms. Doudna asks, to lower an unborn child’s risk of Alzheimer’s disease? If so, would it also be permissible to edit for greater intelligence or athleticism or even, say, for a particular hair color? While all such uses would ultimately require regulatory and institutional review, it is the notion of building a social consensus that is particularly fraught.”

The three main researchers involved in these patent cases have developed their own companies that focus on CRISPR: Doudna developed Intellia Therapeutics, Zhang developed Editas Medicine and Charpentier, now at a Director at Max Planck’s Infection Biology, developed CRISPR Therapeutics. So, both universities and businesses stand to benefit.

These university-based cases often result in sharing through cross-licensing. Remicade, for example, a highly successful biologic for treating auto-immune responses like Crohn’s disease which has generated over a $1 billion so far, has multiple university participants, but is primarily owned by NYU.

Who Benefits?

Yet another question that is raised: Is it right for highly endowed universities like Harvard to get richer as a result of government-funded research? Almost 70% of university research is provided by the U.S. government. Harvard’s 2016 endowment was $36.4 billion.

With the potential impact on society so great, patents may play much more than a financial role. They depending who controls them, they may turn out to be the lynch-pin for ethical application of advanced gene-editing.

In the most interesting chapters of her new book, “A Crack in Creation,” Ms. Doudna wrestles with her ambivalence about the tool she has helped create. She concludes that she no longer feels comfortable operating inside her “familiar scientific bubble”: She must take on a role as a public citizen and address not just the power of gene editing but the ethics of it. At stake, she believes, is “nothing less than the future of our world.”

Image source: bloomberg.com; rsb.org.uk