Gene pioneers share Philly science award for CRISPR, with patent fight in background

Three researchers will share a venerable Philadelphia science award for their pioneering work on a powerful new gene-editing technique that is expected to enable dramatic advances in the field of medicine.

Not listed on Friday's program? The lawyers.

They might as well be. The universities where two of the three scientists did their research are engaged in a messy patent fight over the technique, known as CRISPR-Cas9, with untold millions of dollars at stake.

The three are sharing the John Scott Medal, an annual award that has been given since 1822, named for the chemist and pharmacist who endowed it.

A second Scott Medal is going to University of Pennsylvania scientist Carl H. June, who has used a different type of genetic engineering to drive certain kinds of leukemia into remission. And in a sign of CRISPR's popularity, June is starting to use it too.

"Previous techniques are being updated all the time," June said in a news release, alluding to his fellow honorees. "It's an honor to be recognized with them."

The three who worked on CRISPR are Emmanuelle Charpentier, Jennifer Doudna, and Feng Zhang. In October, a separate group was the first to inject a human patient with cells modified using the technique in China.

Doudna, of the University of California, Berkeley, collaborated with Charpentier, of the Max Planck Institute for Infection Biology in Germany. Zhang, a researcher at Massachusetts Institute of Technology and the Broad Institute, leads a rival team.

Doudna and Charpentier published their results first, in 2012, but Zhang was the first to secure a patent, in 2014. Berkeley has challenged that patent, and the matter has yet to be resolved.

Their gene-editing method relies on an immune-like response that was first identified years earlier in bacteria. The scientists have harnessed this response to create a kind of molecular scalpel, precisely engineered to seek out and "edit" a specific region in an organism's DNA.

CRISPR-Cas9 stands for clustered regularly interspaced short palindromic repeats, with the name of an associated enzyme tacked onto the end.

The fact that the three are sharing the award is a reminder that successes in modern science are rarely achieved by the lone scientist of popular imagination, rather by multiple teams that contribute incremental advances.

But the underlying patent dispute is likewise a reminder that the free-market system is in the business of crowning winners, with universities eager to cash in on that rare research project that can electrify a balance sheet.

In 2008, for example, Children's Hospital of Philadelphia sold its share of the rights to a rotavirus vaccine for $182 million. And Northwestern University has earned $1.4 billion in royalties from its research that led to the development of the drug Lyrica.

The eventual royalties from CRISPR are widely expected to exceed that realm, as the method potentially has applications in any field that involves living things, from agriculture to medicine. Even before the patent issue is resolved, companies seeking to implement the technique have been rewarded handsomely by venture capitalists.

The ceremony for the Scott Award, which is dedicated to the memory of Benjamin Franklin, is at the American Philosophical Society.

Candidates are recommended each year by an advisory committee of Philadelphia-area scientists and physicians. The prize — $12,000 for June, and $6,000 for each of the others — is bestowed by the Board of City Trusts, a group that administers 118 charitable trusts for which the city is named as trustee.

June, Zhang, and Charpentier are expected to attend the ceremony, with Charpentier also giving a lecture at Penn's Perelman School of Medicine. Doudna is not able to make it.

But in the news release, she expressed gratitude. "I am delighted to accept the John Scott Award, an award honoring the memory of Benjamin Franklin – a Founding Father, influential scientist and inventor, and strong advocate for science, technology, and medicine," she said.