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Genome-editing field moving at rapid pace
Editas cofounder Feng Zhang is awaiting action on a patent claim. (Katherine Taylor for The Globe/File)
By Sharon Begley
STAT

The public is only now hearing about the stunning potential of genome editing to treat diseases, and scientists are barely three years into using one of the hottest editing techniques for basic research.

So the announcement on Monday that a Cambridge company, Editas Medicine, plans to go public was unusual. It underlined the lightning speed with which the field of genome-editing is moving, and the temptation biotech startups face to let early investors cash out as buzz reaches a crescendo — even if the treatments they hope to sell might never materialize. In its filing Editas said it hoped to raise up to $100 million.

“I’m not surprised by the timing,’’ said Ashwin Murali, an analyst at Waltham-based hedge fund Matrix Capital Management. “Editas likely did this in order to cash-up prior to [incurring] any clinical trial costs’’ and has “both monetary and psychological’’ reasons for going public.

The psychological value of an IPO is the signal it sends to current and future employees that any stock options they hold or are offered will be valuable. Editas has issued more than 4 million shares of unvested restricted stock as of Nov. 30, 2015, at an average exercise price of $2.50 per share, according to its filing with the US Securities and Exchange Commission. Anyone who holds those options can, therefore, make a profit once shares begin to trade on the NASDAQ (under the ticker symbol EDIT) of the stock price minus $2.50.

It’s “a time of great variability and uncertainty in the biotech markets as a whole, but it is a time when gene therapy is highly hyped and valued,’’ said Murali. “I think the logic on Editas’s part is to capitalize on the very, very good publicity around gene therapy and, specifically, CRISPR,’’ the newest of the precision genome-editing techniques.

That publicity reflects the pace of discovery. Three years ago this month scientists reported that CRISPR-Cas9, a pathogen-fighting system in bacteria, could be used to snip out genes in human and other animal cells and replace them with new ones. It has since become known as a biological “find and replace’’ technique with the potential to remove disease-causing genes, such as that for sickle cell, and insert healthy ones.

Last week alone, for example, three separate teams of scientists reported they had used CRISPR to treat Duchenne muscular dystrophy in mice. Researchers led by Editas cofounder George Church of Harvard University announced last year they had used CRISPR to edit 62 genes in pigs in a way that could make the animals’ organs suitable for transplant into humans.

Such prospects allowed Editas to raise $163.3 million from private investors such as Flagship Ventures, Polaris Venture Partners, and Third Rock Ventures.

Editas acknowledged the many risks would-be investors face: “It will be many years, if ever, before we have a product candidate ready for commercialization,’’ the company said in its filing.

The company’s first clinical trial, for a rare genetic form of blindness called Leber congenital amaurosis (LCA) 10, will not even begin until 2017, chief executive Katrine Bosley announced in November. It plans to use $15 million to $20 million from the IPO on clinical trials for LCA 10. It also plans to develop genome-editing treatments for Usher syndrome 2a, another eye disease, and blindness caused by herpes simplex 1 infection.

One risk involves patents. Among Editas patents are rights to one issued to the Broad Institute of Harvard and MIT in 2014 for discoveries made by one of its cofounders, Feng Zhang, a biologist at the Broad Institute. But that patent it has been challenged by the University of California Berkeley, where Jennifer Doudna led a key CRISPR study that was published months before Zhang’s.

Moreover, Editas revealed in its filing that a previously overlooked claimant, Rockefeller University, has also challenged the Broad patent.

Late last month, the Patent and Trademark Office examiner handling the CRISPR patents recommended that a USPTO board institute a proceeding to determine whether Doudna or Zhang can claim the patent.

Doudna was a cofounder of Editas, but she left and cofounded a rival genome-editing company, Caribou Biosciences. Meanwhile, CRISPR Therapeutics, another competitor, has an exclusive license to patent rights from Emmanuelle Charpentier, who collaborated with Doudna on the key 2012 CRISPR breakthrough.

Of course, there is still no proof that CRISPR can cure any diseases. Even its safety has not been demonstrated. CRISPR is known to snip out at least some bits of DNA other than the ones it’s supposed to, raising fears that such “off-target effects’’ could make the technique too risky to use in patients.

Whatever the risk to investors, a CRISPR company going public at what could be a billion-dollar valuation could also affect ordinary patients.

“As money and excitement enter a field, there is a risk the patient population will incorrectly believe that clinical treatments are — or at least ought to be — available,’’ said legal scholar and bioethicist R. Alta Charo of the University of Wisconsin Law School.

That happened with stem cells 15 years ago, and since then countless clinics offering sketchy stem-cell-based “treatments’’ have popped up across the world. Patients and their families, seeing reports of the muscular dystrophy mice study and other genome-editing advances, might think “they may begin searching for clinics’’ offering CRISPR therapy, Charo warned — and might find them in countries with minimal health care oversight.

“It does not take much imagination to conjure up scenarios in which patients are misled into [at best] useless and [at worst] harmful interventions using not-yet proven applications of gene editing,’’ Charo said.

Sharon Begley can be reached at sharon.begley@statnews.com. Follow her on Twitter @sxbegle. Follow Stat on Twitter @statnews.