Persistence and perseverance were the themes of two of our top stories this week.
First there’s the story of Nationwide Children’s Hospital. The Columbus, Ohio, hospital chose to invest in gene therapy when the field was in its darkest days. After some ups and downs, the team it put in place helped advance some of the most promising gene therapies currently in human testing—and put Nationwide in the national spotlight as one of those treatments nears FDA approval.
Then there’s Suzanne Ildstad. A longtime transplant surgeon, Ildstad cobbled together enough money through grant funding to start a company and develop a cell therapy for organ transplant patients. After some stops and starts and nearly 20 years of waiting, she is finally on the verge of a test that could lead to a regulatory approval if all goes well.
You don’t have to persevere any longer to get to the roundup, though. This week’s mashup of biopharma deals, data, regulatory news, and more are just ahead.
—Over the past two decades, Nationwide Children’s Hospital has quietly gone from a small regional facility with barely any in-house research of its own to a leader in the booming field of gene therapy. Xconomy profiled Nationwide’s rise through the ranks, which could soon culminate with the US approval of the spinal muscular atrophy gene therapy Zolgensma.
—Human testing of CRISPR-based gene editing technology has officially come to the US. This week, the first two patients in a study at the University of Pennsylvania were given an experimental cancer treatment tweaked with CRISPR. Federal oversight groups cleared the study, funded by the Parker Institute for Cancer Immunotherapy, in 2016. Here’s more from NPR.
—Blackstone Life Sciences led a $100 million investment into a startup, Talaris Therapeutics, developing a cell therapy meant to help free kidney transplant patients from the need for chronic immunosuppressive drugs. The financing marks the latest turn in a nearly two decade long quest by Talaris’s scientific founder and transplant surgeon Suzanne Ildstad, who saw her work, once licensed to Novartis (NYSE: NVS), stall when the Swiss pharma dissolved its cell and gene therapy division.
—Alnylam Pharmaceuticals (NASDAQ: ALNY) set out at a big medical meeting to bolster its case for givosiran, which could soon become just the second-ever FDA approved RNA interference medicine. Xconomy spoke with clinicians about the prospects for the drug, which is being developed for a debilitating genetic disease known as acute hepatic porphyria.
GREEN LIGHTS & RED LIGHTS
—The US Supreme Court nixed a long-running effort by Allergan (NYSE: AGN) to protect the patents protecting its best-selling glaucoma drug cyclosporine (Restasis) by transferring their rights to a Native American tribe. Elsewhere, two proxy advisory firms recommended that Allergan shareholders reject a bid by activist shareholder Appaloosa Management to split the roles of CEO and chairman, titles both held by Brent Saunders.
—Adverum Biotechnologies (NASDAQ: ADVM) said that the FDA had temporarily suspended a Phase 1 study of its experimental gene therapy for age-related macular degeneration earlier this month due to questions about the company’s manufacturing process. Adverum responded to the FDA last week.
—The National Institute for Health and Care Excellence (NICE), the UK’s drug-pricing authority, reversed course and recommended the use of inotersen (Tegsedi), an Akcea Therapeutics (NASDAQ: AKCA) drug for the rare genetic disease hereditary transthyretin amyloidosis. NICE changed its tune after Akcea dropped the drug’s price.
NEW STARTS & CASH GRABS
—Contract drug manufacturer Calatent (NYSE: CTLT) paid $1.2 billion for Paragon Bio Services, which makes viral “vectors” companies use to deliver gene therapies. The deal comes less than a month after Thermo Fisher (NYSE: TMO) bought Brammer Bio, another vector developer, for $1.7 billion.