Investors Fuel a Crispr Rush

"Increasing venture-capital investment in gene-editing technology is fueling a rush to secure intellectual property in a sector that promises to spur advances across several areas of biotechnology.

Technology known as Crispr enables researchers to make precise changes, or edits, to the genetic code, which could lead to therapies for genetic diseases, cancer and other conditions. Because of its broad commercial potential, the gene-editing technology has been surrounded by intellectual-property disputes for several years.

In one such dispute, U.S. patent authorities ruled that the Broad Institute, a partnership including the Massachusetts Institute of Technology and Harvard University, was first to invent a method of editing plant and animal cells using the enzyme Cas9, a type of molecular scissors that can cut DNA. The dispute pitted Broad against the University of California, Berkeley, the University of Vienna and Emmanuelle Charpentier, who shared the 2020 Nobel Prize in Chemistry with Berkeley professor Jennifer Doudna for their Crispr-Cas9 research.

The U.S. Patent and Trademark Office decision affects companies that use Cas9, favoring those with ties to Broad, including Editas Medicine Inc., and going against drugmakers such as Intellia Therapeutics Inc. that have licenses from the University of California and its partners. Intellia last week said it had filed patent applications for its own Crispr-Cas9 innovations and that it doesn't expect the ruling to affect its ability to develop future Crispr-based medicines.

Several Crispr-based startups raising venture capital recently are using enzymes other than Cas9 to enable new applications for gene editing, avoid existing patents and generate new intellectual property. Last week's ruling will spur even more Crispr innovation, said Katherine Ann Rubino, chair of the life-sciences practice group for law firm Caldwell Intellectual Property Law.

Globally, venture capitalists invested $1.08 billion in 31 Crispr startup financings last year, compared with $172.9 million in 18 deals in 2020, according to market tracker PitchBook Data Inc. Part of the appeal is the efficiency, specificity and simplicity of Crispr gene editing, said Ursheet Parikh, a partner with venture firm Mayfield.

"It will become one of these very fundamental, pervasive technologies," Mr. Parikh said.

Crispr-based biotech startups raising venture capital recently include Brisbane, Calif.-based Mammoth Biosciences Inc., which Dr. Doudna co-founded in 2017 to deploy a new set of Cas enzymes. Mammoth, whose investors include Mayfield, is developing its own therapeutics and collaborating with drugmakers including Bayer AG.

Enzymes in Mammoth's tool kit, such as Cas14, that are smaller than Cas9 could enable new types of therapies, said co-founder and CEO Trevor Martin. Crispr-delivery tools such as adeno-associated virus, for example, can only hold so much freight. With a smaller protein, the treatment could include more machinery, he said.

Mammoth has exclusive licenses from UC Berkeley and its own separate IP portfolio stemming from its research, according to General Counsel Gary Loeb.

Similarly, Scribe Therapeutics Inc., an Alameda, Calif.-based biotech startup also co-founded by Dr. Doudna, is developing custom-engineered enzymes for Crispr-based treatments for neurodegenerative and other diseases. Scribe's technologies are distinct from earlier approaches, co-founder and CEO Benjamin Oakes said.

Other new companies, including Hayward, Calif.-based Spotlight Therapeutics Inc., focus on delivering gene-editing drugs more effectively to sites of disease in the body. Using cell-targeting proteins, for example, Spotlight potentially could deliver gene-editing enzymes into immune cells surrounding a tumor in an effort to treat cancer, said CEO Mary Haak-Frendscho.

Because gene-editing technology is advancing so quickly, patenting for the sake of patenting could lose its value because companies are rapidly devising new ways to edit genetic code, said K. Lance Anderson, a patent lawyer with law firm Dickinson Wright PLLC. He said he expects companies will start to take more targeted approaches to intellectual property as a result, seeking patents on core aspects of their technology." [1]

 So what, passing gas, Lithuanian brothers? It's okay to pass gas. The human body produces a lot of gas. It’s a bad thing we don’t do anything else. After all, we were hoping to win a Nobel Prize for Crispr. We have not received, but we do not invest, we do not use knowledge. What a shame.

1.  Finance News: Investors Fuel a Crispr Rush
Gormley, Brian.
Wall Street Journal, Eastern edition; New York, N.Y. [New York, N.Y]. 11 Mar 2022: B.10.