It’s not a stretch to say that the progress of a gene editing technology known as CRISPR-Cas9 will be one of the most closely watched biotech stories of this decade. And two important developments along that path are taking place over the next two years. 2016 is the year that the first crop of CRISPR drug developers, led by Editas Medicine and Intellia Therapeutics, are going public. And 2017 will likely be the year the world begins to find out how CRISPR therapies fare in human patients, as the first clinical trials get underway. Yet as this week’s East Coast headlines show, before those trials begin, other technologies are already emerging that could one-up CRISPR. That and much more below.
—Lexington, MA-based Homology Medicines launched this week with a $43.5 million financing round and a bold claim that its underlying science might be more precise, and thus safer, than CRISPR and other gene editing methods. The idea behind Homology—to recreate homologous recombination, a natural process cells use to repair their DNA—isn’t new. The “big leap,” CEO Arthur Tzianabos said, is that Homology may have found a more effective way of doing it.
—Speaking of gene editing, later today Intellia Therapeutics is expected to become the second CRISPR-Cas9 drug developer to go public, following Editas Medicine (NASDAQ: EDIT, which completed an IPO in February. Alex Lash has more here on Intellia, and the questions surrounding CRISPR-Cas9 as the technology heads towards its first human trials.
—Cambridge, MA-based Biogen (NASDAQ: BIIB) announced plans to spin out its hemophilia business into a new publicly traded, standalone company. The deal, which Biogen expects to complete in either late 2016 or early 2017, isn’t necessarily a surprise. Biogen’s hemophilia drugs, which include two FDA approved replacement clotting factors and an experimental gene therapy, don’t fit strategically with the rest of its business, something CEO George Scangos spoke about at a conference in February. Still, Biogen is giving up a growing revenue stream to focus on high-risk, high-reward drugs, something RBC Capital Markets analyst Michael Yee wrote in a research note might “lead to some head-scratching amongst investors.”
—Cambridge-based Bind Therapeutics (NASDAQ: BIND) filed for bankruptcy this week, pushed into court protection when a lender demanded payment for a roughly $15 million loan. Bind filed for Chapter 11, meaning it’ll try to reorganize or sell its assets—a different process from an outright Chapter 7 liquidation. Stockholders are typically wiped out in these proceedings, which is why Bind’s stock is now worth just under $0.40.
—Reuters, citing unnamed sources, reported that New York-based Pfizer (NYSE: PFE) is considering making an offer for Medivation (NASDAQ: MDVN) that would top Sanofi’s $9.3 billion, $52.50 per share hostile bid.
—Tarrytown, NY-based Regeneron Pharmaceuticals (NASDAQ: REGN) announced positive data from a mid-stage trial of a pain drug called fasinumab it’s developing for people with osteoarthritis. But while the study hit its primary goal—offering more pain relief than a placebo—analysts questioned the drug’s safety profile. In a research note, Leerink analyst Geoffrey Porges pointed out some “neuro-musculoskeletal based” side effects that have been problematic for similar-type drugs in the past. While those side effects weren’t necessarily a surprise, Porges noted that they “will likely be a major focus moving forward” as patients in Regeneron’s study are followed up in the coming months.
—Boston Children’s Hospital formed a partnership with a new gene therapy startup that launched this week in the U.K. called Orchard Therapeutics. David Williams, the president of Dana-Farber-Boston Children’s Cancer and Blood Disorders Center, and Alessandra Biffi, who runs the center’s gene therapy work, are on Orchard’s scientific advisory board. The two are part of a team that have been conducting clinical trials on gene therapies for rare disorders like “bubble boy” disease, Wiskott-Aldrich syndrome, and chronic granulomatous disease. Orchard, like Cambridge-based Bluebird Bio (NASDAQ: BLUE), is using modified lentiviruses—the group of viruses that includes HIV—to deliver genetic fixes into patients with rare diseases.
—Cambridge-based Foundation Medicine (NASDAQ: FMI) launched a blood-based test for cancer, what’s known as a “liquid biopsy,” called FoundationACT. The test is meant to be administered either when tissue biopsies either aren’t feasible or when a tumor sample can’t be obtained, and to identify a variety of genetic alterations present in a tumor.
—Shares of Cambridge-based Alnylam Pharmaceuticals (NASDAQ: ALNY) fell more than 11 percent after the company disclosed a development path for a drug for the ultra rare disease paroxysmal nocturnal hemoglobinuria that disappointed investors. Alnylam said that early data on the drug, known as ALN-CC5, show that it might be best suited as a therapy for people that don’t respond to Alexion Pharmaceuticals’ (NASDAQ: ALXN) eculizumab (Soliris), rather than competing directly with Alexion’s drug. Alnylam will disclose data from an early-stage trial of ALN-CC5 at a medical meeting next month.