Why This Massachusetts Biotech Could be the Tesla of Healthcare

August 21, 2017
By Mark Terry, BioSpace.com Breaking News Staff

Investors who put their money down on Tesla Motors (TSLA) when the electric car company launched, have watched their investment grow by almost 1,400 percent since 2010. Is there a current equivalent in biotech? It’s probably anybody’s guess, but Keith Speights, writing for The Motley Fool, thinks Editas Medicine (EDIT) might be the equivalent in biotech.

Based in Cambridge, Mass., Editas focuses on using CRISPR-Cas9 gene editing technology to treat rare diseases. As Speights points out, Tesla wasn’t the first company to work on electric cars—the first electric car was developed around 1890—and Editas isn’t the first or only company focused on gene editing or CRISPR-Cas9. Most notable is CRISPR Therapeutics (CRSP), cofounded by Jennifer Doudna and Emmanuelle Charpentier, the researchers generally credited with discovering the technology. However, Feng Zhang, a researcher at the MIT-Harvard Broad Institute, is also a competing discoverer, and the groups have ongoing patent lawsuits over it. To make that backstory a little more complicated, originally Doudna founded Caribou Biosciences, but then cofounded Editas with Zhang and George Church.

Editas’ approach appears to be both better and faster, as well as less expensive, than other gene editing technologies. And the company isn’t focused on just a single disease. Speights writes, “The biotech’s pipeline currently includes seven pre-clinical programs. One of those programs is a collaboration with Juno Therapeutics (JUNO) to use gene editing in engineering T cells to fight cancer. Editas’ lead program EDIT-101 targets treatment of rare genetic eye disease Leber Congenital Amaurosis type 10. The company plans to submit an Investigational New Drug (IND) application for EDIT-101 next year.”

On August 9, Editas announced it had hit its second milestone under its Juno collaboration. As a result, Editas will receive $2.5 million. The milestone was related to its technical progress in overcoming the tumor microenvironment in its program to develop engineered T cells with chimeric antigen receptors (CAR) and T cell receptors (TCR) in cancer.

Also, in March 2017, Editas and Allergan plc (AGN) inked a strategic research and development deal that could exceed $1 billion in milestone payments. That deal gave Allergan exclusive access and the option to license up to five of Editas’ genome-editing ocular programs.

In addition to the original CRISPR-Cas9 technology, Editas has developed another type, CRISPR-Cpf1, which may be better suited for some mutations than Cas9.

Speights writes, “What does this mean for Editas’ potential? There are around 6,000 diseases caused by genetic mutations. Over 95 percent of them don’t have an approved therapeutic alternative. Even where there are approved therapies, they often only treat the symptoms of the disease. CRISPR-Cas9 and CRISPR-Cpf1 hold the potential to be used to treat many of these genetic diseases, particularly those affecting bone marrow, eyes, liver, lung, and muscles. Editas will be a go-to partner for many biopharmaceutical companies wanting to target those diseases.”

So far none of Editas’ programs have reached clinical trials. And the technology isn’t without its concerns. In June 2017, a paper was published in Nature Methods indicating that CRISPR-Cas9 created unexpected mutations throughout the genome. Another paper published in bioRxiv, re-evaluated the data and contradicted it (welcome to science).

So, what does that controversy mean for CRISPR? Steven Salzberg, the Bloomberg Distinguished Professor of Biomedical Engineering, Computer Science, and Biostatistics at Johns Hopkins University wrote in Forbes, “Even though CRISPR is probably being overhyped right now, it is nonetheless genuinely exciting technology.”

Speights notes that the likelihood of all of Editas’ clinical programs being approved is very low. It’s also possible that someone will find a better technology or some sort of refinement on CRISPR-Cas9 or CRISPR-Cpf1 that will undercut Editas’ efforts. It’s also entirely possible that there are as-yet-unseen problems with the technology once it moves into the clinic. But Editas appears to be leading the way at the moment.

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