Monday, September 12, 2016

Soon There Will Be Three Publicly Traded Gene Editing CRISPR Co's.

In yesterday's Technology Review's "50 Smartest Companies 2016" I mentioned my initial surprise when reviewing the second half of the list. What I neglected to point out was the reason for that surprise.
There are a lot of companies doing work in the biological sciences that were new to me, and I consider myself pretty darn fashion-forward, thank you very much. Here's another one.

From Xconomy Boston:

Seeking to Join Editas, Intellia, CRISPR Therapeutics Makes Long Awaited IPO Push
Investors hoping to buy into the vast, yet still unproven drugmaking potential of the gene editing system CRISPR-Cas9 will soon have three different companies to choose from. CRISPR Therapeutics filed for an IPO on Friday, meaning it could soon join Boston-area rivals Editas Medicine and Intellia Therapeutics in the expanding club of publicly traded CRISPR-Cas9 drug developers.

Basel, Switzerland- and Cambridge, MA-based CRISPR has been slowly amassing rounds of venture funding, adding partners, and filling out an executive team over the past few years. Editas (NASDAQ: EDIT) and Intellia (NASDAQ: NTLA) have each gone public in 2016, and CRISPR has long been expected to join them. Should CRISPR complete the IPO, it’ll trade on the Nasdaq under the ticker symbol “CRSP.”

Over the past several years, CRISPR-Cas9 has captured the imaginations of scientists and drugmakers alike. It’s a tool currently used in research labs across the globe that one day might help perform genetic surgery to correct devastating diseases. There’s a long way to go first, of course. CRISPR-based therapies have only just begun their first clinical tests in human patients, and many questions about their safety and effectiveness will have to be answered. A high-profile patent fight between various of the technology’s scientific founders is also playing out as Editas, Intellia, and CRISPR push their way forward.

But CRISPR companies are also taking cues from the developers of gene therapies and RNA interference, two other complicated drugmaking methods that have gone through a series of ups and downs over the past few decades. Methods that gene therapy and RNAi drugmakers use to deliver their therapies into patients are a starting point for CRISPR drugs, before developers go on to potentially tackle more complicated biological problems.

Editas, for instance, is starting out developing a CRISPR drug for a form of genetic blindness—a different variation of the disease Spark Therapeutics (NASDAQ: ONCE) is going after with a gene therapy that could soon be approved in the U.S. Intellia has an experimental CRISPR drug for the liver disease transthyretin amyloidosis, the same disease targeted by Alnylam Pharmaceuticals’s (NASDAQ: ALNY) lead drug patisiran. And CRISPR is going after the blood diseases beta-thalassemia and sickle cell disease, each of which is the target of a gene therapy from Bluebird Bio (NASDAQ: BLUE)....