Bio Roundup: CRISPR Advances, Obamacare Lives, FDA Nods & More

Xconomy National — 

Gene editing took an important step this week when a group of U.S. researchers used CRISPR-Cas9 technology to correct a genetic error in dozens of human embryos without complications. It’s a significant achievement, but amidst the hype, it’s worth cautioning just how much work has to be done before the technology leads to a safe and effective human therapeutic. Years of clinical testing, and likely unforeseen twists and turns, lay ahead, with many more biological mysteries to solve along the way. For a dose of reality, look no further than a separate research paper also submitted this week by one of the CRISPR field’s pioneers, Feng Zhang, who warned drugmakers about potential problems that could arise in human testing.

Beyond CRISPR, we’ve got the latest on the ongoing healthcare soap opera, details on new drug approvals, and much more. Read on below.


—In a paper published in Nature Medicine, Feng Zhang, one of the pioneers of CRISPR-Cas9 gene editing at the Broad Institute of MIT and Harvard, urged companies to analyze the DNA of patients before giving them experimental medicines that alter their genes with the breakthrough technology.

—Just two days later, Nature published the details of the paper showing a team of researchers used CRISPR to correct, in dozens of human embryos, a genetic mutation known to cause a specific type of heart disease.

—In related news, Bedford, MA-based Homology Medicines, a startup trying to advance a gene editing alternative to CRISPR, raised an $83.5 million Series B round, giving it a whopping $127 million in raised cash since its inception last year.


—The GOP’s efforts to repeal and replace the Affordable Care Act fell short last week, but while the law lives, for now, its future remains unclear. The Trump administration again threatened to withhold critical subsidies that fund part of the ACA, rattling insurers, and tried to browbeat Congress into moving forward with a new repeal and replace effort. Yet Congress appeared to have other ideas. Some indicated it was time to move on to tax reform, while others scheduled hearings to begin working toward a bipartisan fix to the current system.

—-By a 94-1 vote, the U.S. Senate passed a bill that reauthorizes the fees collected from drug and medical device developers in order to fund the FDA’s regulatory review of new medical products. Before that vote, the Senate passed a “right to try” bill that would expand dying patients’ access to experimental drugs.


—The 2010 alliance between Celgene (NASDAQ: CELG) and Agios Pharmaceuticals (NASDAQ: CELG) paid dividends this week with FDA approval of acute myeloid leukemia (AML) drug enasidenib (Idhifa). The drug is the first marketed product for Agios, and one it was able to advance from discovery to finish line far quicker than the norm.

—Jazz Pharmaceuticals (NASDAQ: JAZZ) was cleared to begin selling its own AML drug, branded as Vyxeos, which the company acquired when it bought Celator Pharmaceuticals last year.

—The FDA approved ibrutinib (Imbruvica) for adults with chronic graft-versus-host disease. The drug, sold by Johnson & Johnson (NYSE: JNJ) and AbbVie (NYSE: ABBV), is already on the market for a variety of blood cancers.

—The agency also gave the green light to a new hepatitis C medicine, glecaprevir/pibrentasvir (Mavyret), from Enanta Pharmaceuticals (NASDAQ: ENTA) and partner AbbVie.

—AstraZeneca’s 2015 investment in Netherlands and Redwood City, CA-based Acerta Pharma may soon pay off. The FDA will decide by early next year whether to approve Acerta lymphoma drug alacabrutinib; if it does, Acerta’s backers would get $1.5 billion from AstraZeneca.

—Eli Lilly (NYSE: LLY) plans to seek FDA approval of migraine drug lasmitidan next year now that a second Phase 3 trial has succeeded. Lilly got the drug when it bought CoLucid Pharmaceuticals in January.

—An FDA advisory committee voted to recommend approval of a hepatitis B vaccine, Heplisav-B, from Dynavax Technologies (NASDAQ: [[ticker: [[NASDAQ:DVAX[]]), and shares shot up 70 percent. But on Thursday, the Berkeley, CA, company announced that the FDA wants more information about post-marketing study plans, which will delay an FDA decision until November.

—FDA advisors didn’t, however, recommend approval of sirukumab (Plivensia), a rheumatoid arthritis drug developed by J&J, citing safety concerns. The agency will decide the drug’s fate by Sept. 23.

—New details emerged showing the internal rift between FDA staffers over the agency’s controversial 2016 approval of Duchenne muscular dystrophy drug eteplirsen (Exondys 51). Here’s more from Undark.


—Bristol-Myers Squibb (NYSE: BMY) paid $300 million to buy Cambridge, MA, startup IFM Therapeutics and get ahold of some cancer drugs the startup has ben developing. IFM, however, kept some assets in-house and is spinning them into a new company called IFM Therapeutics LLC.

—Takeda formed a new startup in Cambridge, MA, Cardurion Pharmaceuticals, which will develop drugs for heart failure and other cardiovascular diseases.

—Amplyx Pharmaceuticals of San Diego raised a $67 million Series C round of fund testing of an anti-fungal drug.

—San Francisco-based Invitae (NASDAQ: NVTA) reached deals to acquire Good Start Genetics and Combimatrix (NASDAQ: CBMX), moves to expand its gene testing menu to include carrier and newborn screening.

—Vertitas Genetics, a Boston company that offers whole genome sequencing for less than $1,000, acquired Curoverse, also based in Boston, to help bring artificial intelligence and machine learning tools to genetic analysis.


—Shares of Spark Therapeutics (NASDAQ: ONCE) climbed 16 percent after it provided the first small, early look at a trial testing an experimental gene therapy for hemophilia A; the company quickly raised $350 million in a stock offering.  BioMarin Pharmaceutical (NASDAQ: BMRN) is ahead of Spark with its own hemophilia A gene therapy.

—Meanwhile, Lexington, MA, and Amsterdam-based UniQure (NASDAQ: QURE) reacquired European rights to its experimental hemophilia B gene therapy from partner Chiesi.


—Pain drug developer PixarBio (OTC: PXRB) slashed its headcount by 17 and relocated its headquarters from Massachusetts to New Hampshire, according to an SEC filing. But in an e-mail to Endpoints, CEO Frank Reynolds insisted that the company is in Cambridge, MA, and remains on target for an FDA decision on its drug in 2019.

—Ocular Therapeutix (NASDAQ: OCUL) laid off 19 percent of its staff, a cost-saving move that follows the FDA’s rejection of the Bedford, MA, company’s drug delivery device for the eye.

Frank Vinluan contributed to this report.