Regeneron’s Yancopoulos on CRISPR, Gene-Editing Challenges

  • Gene-editing biotechs overvalue CRISPR technology, Regeneron’s top scientist George Yancopoulos said.
  • “It’s not just about the simple, initial CRISPR technology and IP,” Yancopoulos told Insider.
  • The future of gene editing depends on solving delivery challenges for these treatments, he said.

Gene editing is one of the hottest spaces in biotech, but the companies specializing in that area need to think beyond the core technology, one of the drug industry’s top scientific leaders said.

George Yancopoulos, Regeneron’s chief scientific officer, said in an interview that gene-editing biotechs are too narrowly focused on CRISPR, the tool used to edit DNA. Instead, the 62-year-old billionaire said the future of gene-editing companies will be in finding better ways to deliver these medicines inside the body. 

“I think a lot of people, outsiders as well as people in these companies, overvalue the contribution of CRISPR itself,” Yancopoulos said in an interview on the sidelines of the Hlth conference in Boston. “It’s really all the other components that are truly limiting, from delivery to expanding the capabilities.”

“It’s not just about the simple, initial CRISPR technology and IP,” he added. “It’s about taking it to other levels.”

The future of gene editing is delivering CRISPR treatments into the body

A scientist wearing a white coat and pouring liquid into a beaker at Intellia Therapeutics' lab in Cambridge, Massachusetts.

Intellia Therapeutics is developing CRISPR-based gene-editing medicines that could potentially cure diseases.


Yancopoulos is a big believer in genetic medicine, saying there isn’t a more exciting area in biotech. And his $63 billion biotech is hoping to work on these challenges to move CRISPR forward. 

In 2012, scientists found that a component of the bacterial immune system, which they called CRISPR, was a simple and powerful tool to precisely edit genetic code. Since this Nobel Prize-winning discovery, CRISPR has excited scientists, patients, and investors alike with its potential to treat a wide array of genetic diseases.

In the past decade, many companies have launched to try using CRISPR to treat diseases. Today, seven gene-editing biotechs have gone public, commanding a combined market value of $30 billion, led by Intellia Therapeutics, CRISPR Therapeutics, and Beam Therapeutics.

Regeneron partnered with Intellia in 2016, and the two presented the first human data this summer showing encouraging early results for a CRISPR drug delivered inside the body. The one-time treatment lowered levels of a disease-causing protein for a rare liver disease.

That drug was constructed in a similar way as the Pfizer and Moderna coronavirus vaccines. The CRISPR treatment was delivered by messenger RNA, genetic code that tells the cells to build certain proteins. That mRNA molecule is wrapped in a lipid nanoparticle, which slips inside of cells to deliver the mRNA.

In 2020, Regeneron and Intellia doubled down on their work to develop CRISPR drugs delivered inside the body, expanding their collaboration to go into at least 2024. The two plan to codevelop up to 15 such medicines.

There are still plenty of CRISPR delivery challenges to overcome

CRISPR-Cas9 is a customizable tool that lets scientists cut and insert small pieces of DNA at precise areas along a DNA strand. This lets scientists study our genes in a specific, targeted way.

CRISPR-Cas9 is a customizable tool that lets scientists cut and insert small pieces of DNA at precise areas along a DNA strand.

National Human Genome Research Institute, NIH

The vast majority of the pipeline of CRISPR-based drugs relies on editing cells outside of the body. That requires extracting immature cells called hematopoietic stem cells from a patient’s bone marrow — a lengthy and sometimes painful process.  Scientists then edit those cells with CRISPR in a lab and reinfuse them back in the body.

Yancopoulos said this approach, called “ex vivo” editing, doesn’t realize the full potential of CRISPR. 

But despite Yancopoulos’ excitement, the mRNA delivery approach also has a key limitation. Lipid nanoparticles are most effective in targeting the liver. To treat other diseases, scientists are researching delivery systems that go beyond the liver. 

For instance, the base-editing specialist Beam Therapeutics bought Guide Therapeutics earlier this year, a biotech researching how to design lipid nanoparticles that reach areas in the body other than the liver.

Yancopoulos is confident that delivering CRISPR treatments into the body will be the most important advancement in genetic editing.

“If all you can do is modify hematopoietic stem cells, that’s not the dream,” he said. “For that purpose, it doesn’t deserve a Nobel Prize. By itself, it’s not that powerful.”

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