Two separate groups have recently shown that in mouse models, inactivation of a single gene was enough to directly convert other cell types in the brain into neurons.
According to an analysis conducted by BioWorld of the fourth-quarter and year-end 2019 financial reports filed by the top 100 public biopharmaceutical companies ranked by market cap, and excluding big pharma companies, the amount that was invested in research and development (R&D) during the year increased 35% compared to the same period in 2018.
Everything’s good for something. Including, it turns out, 5’ untranslated trinucleotide repeats. In the Feb. 17, 2020, issue of Nature Neuroscience, researchers have demonstrated a role for such repeats in controlling protein levels of fragile X mental retardation protein (FMRP).
Philadelphia-based Aro Biotherapeutics Co. CEO Sue Dillon told BioWorld her firm has “incoming interest from other companies to apply Centyrins to other kinds of drug conjugates,” which could mean more deals with the platform like the one sealed with Ionis Pharmaceuticals Inc.
It’s a big day for privately held Empirico Inc. as it embarks on a massive combo pharma deal with Ionis Pharmaceuticals Inc. while racking up a $17 million series A-2 financing.
Akcea Therapeutics Inc., a majority owned affiliate of Ionis Pharmaceuticals Inc., and Pfizer Inc. signed an exclusive, worldwide licensing deal for AKCEA-ANGPTL-3-LRx, which is being developed to treat cardiovascular and metabolic diseases. The deal follows a period during which Akcea's stock (NASDAQ:AKCA) has sagged prior to rising Monday and big changes in the company's C-suite.
