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East Coast Biotech Roundup: Scangos, CRISPR, Yumanity, Synlogic & More

Xconomy New York — 

Biotech and pharma executives from across the country descended on frigid midtown Manhattan this week for the BIO CEO & Investor Conference, and we’ve got a few tidbits from the festivities—as well as the rest of your East Coast headlines—below. But first, a programming note. The East Coast roundup has moved to a weekly Thursday spot, switching places with Alex Lash’s West Coast counterpart, which will now run each Friday—so set your DVRs accordingly. With that, on to the news.

— Biogen (NASDAQ: BIIB) CEO George Scangos did a chat this week at the BIO CEO & Investor Conference and dished on a wide range of topics. He weighed in on Biogen’s cultural shift over his tenure, the strategic decisions he’s made on certain programs, and of course drug pricing—where he warned that a firmer policy change could lead to fewer drugs.

—Separately, Biogen also joined what’s known as the Centre for Therapeutic Target Validation, which was formed via a collaboration between GlaxoSmithKline, the Wellcome Trust Sanger Institute and the European Bioinformatics Institute in 2014. The CTTV is using genetics and computational biology to help find new drug targets.

—The executives of a group of gene-editing companies—Editas Medicine (NASDAQ: EDIT), Intellia Therapeutics, and Cellectis (NASDAQ: CLLS) also gathered at BIO’s New York meeting this week. The topic of discussion was CRISPR-Cas9, the gene-editing system that, incidentally, earlier in the day, America’s top intelligence chief had called a potential “weapon of mass destruction.” The executives gave a thoughtful review of the many scientific and regulatory challenges that lie ahead before CRISPR-Cas9 can effectively be used to treat human disease. (The discussion apparently didn’t please investors of Editas, whose shares sank 20 percent on the day.)

—A year after making its public debut and announcing plans to seek out a big financing round, Cambridge, MA-based Yumanity Therapeutics resurfaced with a $45 million Series A round led by Fidelity Management & Research. I’ve profiled Yumanity in the past; the company, led by industry veteran Tony Coles and the Whitehead Institute for Biomedical Research’s Sue Lindquist, aims to use a yeast-based system to discover drugs for crippling neurodegenerative diseases like Alzheimer’s and Parkinson’s. Alex Lash has more on the financing and Yumanity’s drug discovery methods here.

—The good news: Cambridge-based Proteostasis Therapeutics (NASDAQ: PTI) raised $50 million in an IPO on Thursday, despite a tough environment for biotech and even IPOs in general. The bad news: Proteostasis had to significantly discount its deal to raise that money. It sold 6.25 million shares at $8 apiece, well below its projections. Here’s more on Proteostasis and the drugs it’s developing for cystic fibrosis and other diseases.

—Cambridge-based Aegerion Pharmaceuticals (NASDAQ: AEGR) hired a new CEO, Mary Szela last month, and her first big move was a restructuring. Aegerion announced plans to lay off about 25 percent of its global workforce, which would leave it with 230 employees. Aegerion will complete the job cuts by the end of the second quarter.

—Cambridge-based Synlogic, a startup combining elements of microbiome research with synthetic biology, inked its first collaboration this week. The company will work with AbbVie (NYSE: ABBV) to develop new drugs for inflammatory bowel disease. Terms of the deal weren’t disclosed, but Forbes has more on the pact here. Xconomy also profiled Synlogic, and scientific founder and synthetic biology pioneer Jim Collins, last year.

—A blizzard last month postponed what was to be a dramatic FDA advisory panel  for Sarepta Therapeutics’ (NASDAQ: SRPT) Duchenne muscular dystrophy drug eteplirsen. As a result, it’ll now be at least a few more months before we know the fate of eteplirsen. The FDA pushed back its decision date on eteplirsen to May 26, from Feb. 26. The agency still hasn’t set a new date for the advisory panel.

—In other Duchenne-related news, Cambridge-based Akashi Therapeutics said that the patient in a clinical trial of its experimental drug HT-100 who had been experiencing serious health issues has died. Akashi had already suspended the study. It’s now working with the FDA to find out what happened, and whether the patient’s death was related to HT-100.

—Astellas Pharma finally completed its buyout of Marlborough, MA-based stem cell drugmaker Ocata Therapeutics after some initial resistance from shareholders over the price of the deal (FierceBiotech has more on that here.). Astellas paid $8.50 a share, or about $380 million, for Ocata.

—An FDA advisory panel voted in favor of a biosimilar form of Johnson & Johnson’s blockbuster arthritis drug infliximab (Remicade) developed by Celltrion and New York-based Pfizer (NYSE: PFE). Reuters has more on the vote, and J&J’s objections, here.

Photo of the Waldorf-Astoria in New York City, site of the BIO CEO & Investor Conference, courtesy of Viaggio Routard via Creative Commons.