By WallStreetDaily.com

Here at Wall Street Daily, we’ve covered topics such as “neural dust,” an Earth-saving membrane, and human colonization of the cosmos.

These are some phenomenal topics to tackle.

And yet gene editing and the act of altering the species may top them all.

Indeed, the MIT Technology Review described a recent breakthrough in the technology as, “the biggest biotech discovery of the century.”

Imagine being able to choose your baby’s eye color, height, or nose shape. Maybe you want a child with a 210 IQ.

Well, we just might have the technology to do it — a “potentially revolutionary tool for editing DNA,” as Carl Zimmer writes in Quanta magazine.

Of course, there are more prosaic matters to be dealt with before we contemplate such “Super Babies,” such as sorting out competing intellectual property claims.

And the U.S. Defense Advanced Research Projects Agency (DARPA) is preparing protocols that seem to be bracing for gene warfare, due to concern in some quarters that the tech can be used to create biological weapons.

There are bioethical concerns as well, including the possibility that those of means will be availed of technology that’s unaffordable for most, plus the very basic question of whether we should make such alterations just because we can.

But the technology is incredible: It holds the very real possibility of quickly and easily identifying and eliminating genetic anomalies so we can treat and cure diseases.

It’s called CRISPR (an acronym for “clustered regularly interspaced short palindromic repeats”), and it’s all over the news because of recent advances in its use to alter genomes.

If you’ve read about it, you know the name of Jennifer Doudna, executive director of the Innovative Genomics Initiative at the University of California, Berkeley and the University of California, San Francisco, who published the seminal research on the technology four years ago.

But the technology is incredible: It holds the very real possibility of quickly and easily identifying and eliminating genetic anomalies so we can treat and cure diseases.

In a paper published by Nature on Aug. 17, 2012, Doudna and colleagues concluded: “Our study reveals a family of endonucleases that use dual-RNAs for site-specific DNA cleavage and highlights the potential to exploit the system for RNA-programmable genome editing.”

On January 29, 2013, a Doudna-led research team published the finding that “Type II CRISPR immune systems in bacteria use a dual RNA-guided DNA endonuclease, Cas9, to cleave foreign DNA at specific sites,” thus setting the stage for “RNA-programmed genome editing… in human cells.”

CRISPR is actually the extension of a naturally occurring immune response system in bacteria that was discovered way back in 1987, though at that time, the Japanese researchers who made the discovery, led by Yoshizumi Ishino, admitted that the “biological significance of these sequences is not known.”

Genome engineering had previously relied on highly complex processes to create new “nuclease pairs,” or binding proteins, for every genomic target.

CRISPR’s two components — a guide RNA and a nonspecific CRISPR-associated enzyme known as Cas9 — simplify the work of altering genes. And the technology is highly adaptable across species and sequences.

During the past four years, Doudna told the BBC, “laboratories around the world” have applied CRISPR “in animals, plants, humans, fungi, other bacteria: essentially any kind of organism they are studying.”

But it’s the human element that’s created the most excitement — for researchers, bioethicists and patent attorneys.

Genome engineering had previously relied on highly complex processes to create new “nuclease pairs,” or binding proteins, for every genomic target.

There are multiple entities lined up to profit from CRISPR’s potential, including Editas Medicine Inc., which holds the rights to the only patent yet issued for CRISPR technology.

Editas went public in February, pricing 5.9 million shares at $16. It currently trades around $15, with a market cap of $536 million.

Doudna, along with researchers from the MIT-Harvard Broad Institute, are co-founders. Feng Zhang of Broad and MIT is named as holder of the critical patent.

When the patent was issued to Feng, Doudna left Editas to form Caribou Biosciences. Doudna’s patent pending before the U.S. Patent and Trademark Office is Caribou’s primary asset.

Caribou and its financial backer, Atlas Venture, founded Intellia Therapeutics Inc. in 2014.

Novartis AG became the first “super pharma” to enter the world of CRISPR technology by licensing Intellia’s IP for all human therapeutic applications — including cancer treatments.

Intellia debuted on the Nasdaq in May, selling 6 million shares at $18. It currently trades around $19.

Privately held Crispr Therapeutics was founded in 2014 by Emmanuelle Charpentier, who worked with Doudna on the research that led to the discovery of CRISPR.

Crispr Therapeutics recently filed a registration statement with the Securities and Exchange Commission signaling its intent to raise up to $90 million in an IPO. It will trade under the symbol CRSP on the Nasdaq.

Cellectis SA, meanwhile, claims worldwide rights to use the technology covered by the patent rights of the family labeled “Engineering Plant Genomes Using CRISPR/Cas Systems.”

Cellectis has already used other gene editing systems to develop agriculture crops, and will use CRISPR to augment its food offerings.

NBNBC

Novartis AG, like IBM, is a big company that knows how to take big risks.

That’s the lesson from its decision to fund Intellia Therapeutics and Caribou Biosciences via collaboration and licensing agreements for CRISPR genome editing technology and the development of drug discovery tools, respectively.

Novartis is already a leader in chimeric antigen receptor T-cell, or CAR-T, therapy research to fight cancer, specifically leukemia, using a patient’s own cells. Its work on immunotherapy should get a boost from its association with Intellia and Caribou.

Its first-mover status among large pharmaceutical companies should pay dividends in coming years.

Smart Investing,

David Dittman
Editorial Director, Wall Street Daily

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