Gene editing was in the news again this week, thanks to Intellia Therapeutics, which looks poised to become the second company developing drugs with CRISPR-Cas9 technology to go public. It wouldn’t be a surprise to see a third, CRISPR Therapeutics, follow suit. That means investors will have a chance to place and hedge their bets among the CRISPR-Cas9 players, which have each quickly grown amassed high-powered executive teams, and executed a string of industry partnerships. The first human data from CRISPR studies is coming soon, however, likely bringing all the twists, turns, and surprises that a new technology typically faces. That story and more below.
—Just a few months after Editas Medicine (NASDAQ: [[ticker:EDIT]]) became the first publicly traded company using CRISPR-Cas9 to develop drugs, Intellia Therapeutics has made its pitch to follow suit. Cambridge, MA-based Intellia filed papers to go public this week, and simultaneously cut a deal with Tarrytown, NY-based Regeneron Pharmaceuticals (NASDAQ: [[ticker:REGN]]) to develop a group of CRISPR-Cas9 drugs—the first being for a rare liver disease called transthyretin amyloidosis. Alex Lash has more on Intellia’s proposed IPO, and the state of CRISPR-Cas9’s race to the clinic.
—Last week, we held our annual “What’s Hot in Boston Biotech” event, in which we brought together a broad array of local life sciences leaders to give folks a snapshot of some of the issues and innovations going on in Cambridge. On Monday, I posted some takeaways from the event, which include some thoughts on drug pricing, neuroscience, and immunotherapy. You can check out some of the photos here.
—Speaking of What’s Hot, Harvard University chemical biologist and serial biotech entrepreneur Greg Verdine dished some details at the event about a new startup he has in the works, Fog Pharmaceuticals. And as with a number of other startups he’s co-founded over the years—among them Aileron Therapeutics and Warp Drive Bio—he’s trying to come up with a creative way to get to drug targets that are out of the reach of existing technologies. Here’s more on Fog Pharma, and the other efforts Verdine has undertaken to get to tough-to-reach drug targets.
—Cambridge-based orphan drug startup accelerator Cydan launched its second company this week, Imara, with a $31 million Series A round and a drug candidate aimed at sickle cell disease. I spoke with Cydan CEO Chris Adams and chief scientific officer James McArthur about Imara, and Cydan’s progress in the three years since it was formed by venture firm New Enterprise Associates.
—Also on the fundraising front, Waltham, MA-based Kala Pharmaceuticals bagged a $68 million Series C round from a broad group of investors of the sort that could eventually support an IPO. Kala is using the cash to fund late-stage trials of what’s known as KP-121, a new formulation of loteprednol etabonate (Lotemax), a potential treatment for dry eye and other eye diseases.
—Summit, NJ-based Celgene (NASDAQ: [[ticker:CELG]]) exercised an option to license a group of cancer immunotherapy products being developed by Seattle’s Juno Therapeutics (NASDAQ: [[ticker:JUNO]]). Celgene paid Juno $50 million in exchange for rights outside of the U.S. and China to the programs. Celgene got the option as part of a deal the two companies signed in 2015.
—Lexington, MA-based Synta Pharmaceuticals (NASDASQ: [[ticker:SNTA]]) executed a reverse merger with privately held Fort Washington, PA-based Madrigal Pharmaceuticals. The deal reshapes struggling Synta—whose lead drug failed a late-stage trial in lung cancer last year—into a developer of treatments for cardiovascular-metabolic diseases and nonalcoholic steatohepatitis. The new company will be 64 percent owned by Madrigal shareholders and based in the Philadelphia area.
—Medford, MA-based Seventh Sense Biosystems began a pivotal trial for its touch activated phlebotomy, or “TAP” device, a supposedly painless method to quickly draw blood for diagnostic tests. The study involves 120 patients at three hospitals in the Northeast, and should be completed in April. Seventh Sense aims to file for approval of the TAP device in the U.S. and Europe during the second quarter. Here’s more on Seventh Sense and TAP.
—New York’s Memorial Sloan-Kettering Cancer Center is one of six founding member institutions of a new venture called the Parker Institute for Cancer Immunotherapy, which was started this week with the help of a $250 million gift from Napster founder Sean Parker. Sloan-Kettering’s efforts will be led by melanoma expert Jedd Wolchok. Here’s more on Sloan-Kettering’s participation, and MIT Technology Review has more here on Parker’s initiative.
