A Look at CRISPR, 'Biotech's Biggest Discovery of the Century,' and the 3 Companies Knee Deep in It

A Look at CRISPR, 'Biotech's Biggest Discovery of the Century,' and the 3 Companies Knee Deep in It October 17, 2016
By Alex Keown, BioSpace.com Breaking News Staff

BOSTON – It’s no secret that CRISPR, a gene-editing technology, is one of the most intriguing things to hit medical research circles. There are high hopes the technology can provide the key to numerous diseases and three companies, Editas Medicine, Intellia Therapeutics and CRISPR Therapeutics, are leading the way with the technology.

"CRISPR" refers to Clustered Regularly Interspaced Short Palindromic Repeats that occur in the genome of certain bacteria, from which the system was discovered. Cas9 is a CRISPR-associated endonuclease (an enzyme) known to act as the "molecular scissors" that cut and edit, or correct, disease-associated DNA in a cell. Gene therapy essentially transforms cells inside a patient to harness their immune system to fight an invading disease on its own. CRISPR-Cas9 is considered revolutionary technology, and as such is likely, at some level, to be used by many companies and institutions.

The technology was discovered by UC Berkeley professor Jennifer Doudna and Emmanuelle Charpentier. Doudna went on to found Caribou Biosciences, which later formed Intellia Therapeutics. Charpentier sold her part of the rights of the CRISPR-Cas9 platform to CRISPR Therapeutics, a company making big moves in the gene therapy field.

With the promise of CRISPR technology, analysts at The Motley Fool dive into the numbers to see if investors should buy stock in these companies. Editas and Intellia are already publicly traded and CRISPR Therapeutics is close to its IPO, the Fool said. Earlier this summer Caribou netted $30 million in Series B financing to expand its CRISPR gene editing technology platform and “accelerate our efforts in highly promising application areas in agriculture, therapeutics, biological research, and industrial biotechnology.

Maxx Chatsko, writing for the Fool, said not only is CRISPR technology revolutionary, it’s also inexpensive to use and delivers results quickly. Of the three companies highlighted, Chatsko said they each remain “in the earliest stages of development, as reflected by financial statements and the fact that no company has entered clinical trials to date.” He does note that Editas is likely to move its first drug candidate into clinical trials next year.

If investors are looking at these companies, Chatsko said investments would likely be for long-term holdings, which means there are risks.

“At the moment, CRISPR stocks are all about potential. They do not generate large amounts of revenue, and aside from potential milestone payments from partners, they will not do so for years,” Chatsko said. “There are certainly big hopes for CRISPR stocks. However, investors should be cautioned that early enthusiasm for RNAi after it won the Nobel Prize in 2006 and gene therapy in the last several decades has failed to deliver big wins to this day. That may be changing with our improving understanding of biology, but many challenges remain for gene editing.”

That being said, Chatsko was quick to point out that these companies have forged deals with much larger pharmaceutical companies, such as Novartis , Juno Therapeutics , Vertex , Bayer and Regeneron , which means there is keen interest in making the technology work. In 2015, Juno Therapeutics struck a $727 million deal with Editas Medicine , the gene editing company, for a partnership that will forge three research programs marrying Editas’ technologies, including CRISPR-Cas9, to Juno’s CAR-T and TCR technologies.

Shares of Editas are currently trading at $13.13. Since July the stock has slid from a high of $28.20. Intellia is trading at $12.51 per share this morning. Since August that company has seen its share prices plunge from a high of $24.50.

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