A CRISPR milestone
In keeping with the lightning-fast pace of CRISPR R&D, gene editing in human embryos has zoomed stateside: Researchers at Oregon Health and Science University have successfully CRISPR'd viable human embryos, MIT Technology Review reports. Of course, none of the embryos has been allowed to mature past a few days — and they’ve certainly never been implanted in a womb. Up until this point, such work has taken place only in China.
The embryos were created using sperm from men with a genetic mutation that the researchers were planning to repair with CRISPR. Unlike previous efforts by scientists in China, the OHSU work produced very few “off-target” effects — and avoided “mosaicism,” in which the CRISPRing only occurs in a few cells.
The dream that such germline editing might help parents avoid passing on dangerous DNA has propelled much of the current CRISPR research. “But we anticipated that there would need to be a lot of research to see if you could make these changes without any unintentional effects,” bioethicist Alta Charo of the University of Wisconsin told STAT's Sharon Begley.
The work from OHSU is one big step down that path.
Crystal ball, do your thing
We had a great time at last night's inaugural STAT Plus subscriber event. Lively crowd, good beer, great conversation. Our favorite question from the audience: What's your most controversial prediction for the industry in the coming year?
Adam Feuerstein fired back a headline: “Combination cancer immunotherapy disappoints.”
Damian Garde's forecast was a bit more dramatic. His prediction: Axovant's Alzheimer's drug, intepirdine, will hit one of its primary endpoints but fail the other, improving either cognition or function but not both. The company will file for approval anyway. An FDA panel will recommend rejection on a close vote.
“Then it leads up to whatever the PDUFA date is... and at 6:01 p.m., big Scott Gottlieb comes in and approves the drug,” he said. “It’s like a season-ending cliffhanger.”
Join us at the next Plus event and share your predictions. Or hear more of ours.
Dear Biogen: Buy Sage
Yesterday, after Biogen pointed excitedly to the money incensing its pockets, we asked you what the big biotech should buy. And about 39 percent of respondents said Sage Therapeutics, the neuroscience-focused biotech making headlines with interesting data and an uncommonly forthcoming CEO.
Coming in second was Axovant Sciences, whose superlatively binary Alzheimer's disease trial is slated to read out in September; followed by the migraine-targeting Alder Biopharmaceuticals; and Sarepta Therapeutics, which needs no introduction.
Gilead on that $36.6 billion question
Gilead has hit some bumps in the past year, but now it's sitting pretty: Its earnings were unexpectedly bullish this quarter, thanks in part to “better than expected” hepatitis C and HIV drug sales — leading the company to boost its guidance to a range of $24 to $25 billion in net sales. And its coffers are still stuffed with cash —$36.6 billion, to be exact.
As for the perennial question of when, oh when, Gilead will finally buy something, CEO John Milligan gave this (thoroughly unsatisfying) answer:
“We’re working very hard behind the scenes on a number of things,” he said. “I can’t direct you to anything specific, but we’re very, very active and when things are right for us, we’ll announce them.”
100 pharmas prune, 100 biotechs portend
There’s an oft-stated and perhaps over-simplified meme in the drug industry that what pharma sees as trash can make for biotech treasure. Anyway, this week, the barges are full.
GlaxoSmithKline has more than 30 would-be drugs that will need new homes, the company said. It's part of a sweeping reorganization that will see it prioritize skin cream, toothpaste, and other things that don’t cost $1 billion to bring to market. (Also on the way out: The much-discussed gene therapy for so-called “bubble boy” disease, which costs $665,000 and has been taken by literally two people.)
All of which is to say that if your favorite business development professional didn’t return your email yesterday, it’s probably because she was tied up in a pan-industry diligence-off that has likely spawned at least six new 'Vants since you began this sentence.
- Testimony is over in the Martin Shkreli trial, with closing arguments scheduled for today and jury deliberations tomorrow. (Bloomberg)
- The startling history behind Merck's cancer blockbuster. (Forbes)
- Study: An all-male team is more likely to get VC funding than one with any women. (The Outline)
- The CRISPR patent fight enters next round. (Science)