‘Patent cloud’ hangs over CRISPR therapy

Dec 12, 2023

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Driving The Day

This electron microscope image shows a blood cell altered by sickle cell disease.

The CRISPR technology used to develop gene-editing therapy to treat sickle cell disease (above) is at the center of a patent dispute. | National Institutes of Health via AP

MORE CRISPR RIGHTS AHEAD? A long-running dispute over intellectual property rights for the CRISPR technology at the heart of the first-ever approved gene-editing therapy could complicate its rollout.

The FDA’s approval Friday of the therapy for sickle cell disease is a watershed moment for CRISPR, the 12-year-old technology that scientists believe could be used to treat many debilitating diseases.

The backstory: The Broad Institute — a genomic research partnership between the Massachusetts Institute of Technology and Harvard University — holds the U.S. patents on editing human cells with CRISPR. The University of California and the two scientists awarded the Nobel Prize for discovering the technology, Jennifer Doudna and Emmanuelle Charpentier, have challenged Broad in court over the past several years.

While Doudna and Charpentier are credited with creating CRISPR, Broad and scientist Feng Zhang claim they were the first to apply the innovation to living cells. Both parties filed for patents around the time U.S. intellectual property law underwent a major shift. In 2013, patent law went from a “first-to-invent” standard to a “first-to-apply” one.

An appeal is pending in the U.S. Court of Appeals for the Federal Circuit.

Why it matters: Broad and its affiliates can sue companies that don’t have licenses for their patents for infringement, said Jacob Sherkow, a law professor at the University of Illinois at Urbana-Champaign College of Law who’s closely followed the dispute. But they can’t sue until a new product hits the market, he said, which will happen following the FDA’s recent approval.

“I would be surprised, bluntly, if [a lawsuit] does not get filed by the end of this year,” Sherkow said.

CRISPR Therapeutics, one of the companies behind the approved therapy, known as Casgevy, doesn’t hold a CRISPR license, Sherkow said, adding he doesn’t believe its partner, Vertex, does either. Spokespersons for both companies didn’t respond to requests for comment.

Editas Medicine is the exclusive licensee for Broad’s patent portfolio covering CRISPR technology. Spokesperson Cristi Barnett said the company regularly considers and grants sublicenses to other companies but wouldn’t confirm any “specific conversations until there is a signed agreement.”

The upshot: Under U.S. patent law, companies don’t have to hold patents to put inventions — like new drugs — through the regulatory approval process, Sherkow said. But product developers also know this so-called safe harbor is time-limited, he said, raising questions about why the patent dispute wasn’t settled earlier.

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TODAY ON OUR PULSE CHECK PODCAST, host Ben Leonard talks with POLITICO health care reporter Megan Messerly about her report on a coming mining boom that brings with it industry jobs that have good health care benefits — and how that can counterintuitively damage health care access, boosting it for some while hampering it for others.

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Eye on the FDA

Maj. Peter Osterbauer injects Botox, which is highly effective in treating headaches, into Staff Sgt. Gabriel Fierros neck at the traumatic brain injury clinic on Elmendorf Air Force base in Anchorage, Alaska in 2009.

A consumer advocacy group is pushing for stronger risk warnings on Botox labels. | Al Grillo/AP

SEEKING A LOUDER BOTOX WARNING — Public Citizen filed a petition with the FDA today, asking regulators to strengthen the risk warning on the labels of Botox and other botulinum toxin products approved for both medical and cosmetic use.

The consumer advocacy nonprofit urges the FDA to emphasize the risk of systemic iatrogenic botulism — symptoms of which include muscle weakness, double vision, drooping eyelids and difficulty swallowing and breathing — in both new and repeat patients, including those receiving recommended doses.

Public Citizen found more than 5,400 reports in the FDA’s Adverse Event Reporting System of “serious outcomes” from Botox and related products, including death, hospitalization and disabilities. FAERS reports don’t establish causation, but the group warned that the system’s voluntary reporting nature could underestimate the problem.

The group also calls for the removal of statements claiming “no definitive serious adverse event reports of distant spread of toxin” in the products’ labeling that it said is misleading. Public Citizen petitioned for a black box warning — the FDA’s most serious — for Botox and similar products in 2008; the FDA granted the request the following year.

FDA DETAILS OMUFA INDUSTRY TALKS — Notes from initial negotiations over the first reauthorization of the over-the-counter monograph drug user fee program, which seeks to expedite new over-the-counter drugs, published Monday reveal the FDA’s and drug industry’s priorities.

The agency wants stable funding for the program, improved consumer trust in over-the-counter drugs, improved transparency in the OTC monograph order process and more reliable regulatory efficiency and predictability. Industry also wants better transparency, improved communication and resources to be focused on “promoting innovation,” according to the summary of the Nov. 14 meeting.

The program expires on Sept. 30, 2025, if it is not reauthorized for fiscal 2025 through 2030.

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In Congress

TRANSPARENCY PACKAGE SAILS THROUGH — The House on Monday advanced a bipartisan measure to increase reporting requirements for insurers, hospitals and pharmacy benefit managers. PBMs have been a bipartisan target in both chambers, and the bill’s passage is the most significant action lawmakers have made against them this year.

The bill was slated to receive a vote in September but was pulled from the calendar amid questions over its support and House chaos over government spending.

What’s in it: It would renew a number of federal health programs and change Medicare payment policy so that drugs administered in a hospital outpatient department are reimbursed at the same rate as a doctors’ office, known as site-neutral payments.

Hospitals and PBMs oppose the bill and are vowing to continue pushing against the policies.

VETO THREAT OF HOUSE RULEMAKING BILL — President Biden would veto a bill that would require federal rulemaking to be initiated by a senior appointee and issued and signed by a Senate-confirmed presidential appointee, the White House said in a statement of administrative policy Monday.

“This bill would result in unnecessary delays in the regulatory process when Senate-confirmed positions are temporarily filled by senior officials while nominees await confirmation,” the statement said. “[The Ensuring Accountability in Agency Rulemaking Act] would also limit the flexibility of Senate-confirmed officials to delegate signature authority to experienced subordinates who have the requisite authority and experience to oversee regulatory development.”

The legislation, if enacted, would have an outsized impact on agencies like the FDA, which has had a number of acting commissioners over the past few decades.

At the start of the Biden administration, Principal Deputy Commissioner Janet Woodcock served as acting commissioner for more than a year, and under then-President Donald Trump, Stephen Ostroff, Ned Sharpless and Brett Giroir served as acting commissioner for various periods.

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Around the Agencies

SANOFI ABANDONS MAZE DEAL — French multinational drugmaker Sanofi announced Monday it will terminate its pending acquisition of Maze Therapeutics’ drug in development for the treatment of a rare genetic disorder called Pompe disease after the Federal Trade Commission moved to block the deal.

The antitrust agency issued an administrative complaint on Monday and authorized a lawsuit to challenge the deal, which it argued “would eliminate a nascent competitor poised to challenge Sanofi’s monopoly in the Pompe disease therapy market.”

Sanofi disagreed with the FTC but said it “would not be in the best interests of patients to contest” the challenge.

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Pharma Moves

The American Clinical Laboratory Association has named Mary Lee Watts vice president of government affairs and policy and Elyse Oveson as chief of advocacy operations. Watts previously worked as director of federal affairs at the American Society for Microbiology, and Oveson was senior director of federal relations at the Healthcare Association of New York State.

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Clients receive clear savings statements, outlining drug costs and Express Scripts’ value. They pay one simple fee, and if goals aren’t met, clients pay less – guaranteed.

Simplicity, savings, and trust – that’s our pharmacy benefit.