Vertex Pharmaceuticals Inc. plans to up its gene editing game by expanding an existing collaboration with Crispr Therapeutics AG and acquiring privately held Exonics Therapeutics Inc. to develop therapies for Duchenne muscular dystrophy (DMD) and myotonic dystrophy type 1 (DM1).
Axovant Sciences Ltd. has terminated its $187.5 million license and collaboration agreement with Benitec Biopharma Ltd., a Sydney-based gene therapy company, regarding AXO-AAV-OPMD (formerly BB-301) for treating oculopharyngeal muscular dystrophy (OPMD).
The FDA has approved Indianapolis-based Eli Lilly and Co.'s Emgality (galcanezumab-gnlm) solution (300 mg) for injection for treating episodic cluster headache in adults, making it the first and only calcitonin gene-related peptide (CGRP) inhibitor that reduces the frequency of these attacks.
Arvinas Inc. and Bayer AG will leverage Arvinas' PROTAC (PROteolysis TArgeting Chimeras) protein degrader platform to develop therapeutics for patients with cardiovascular, oncological and gynecological diseases.
Ridgeback Biotherapeutics LP said the FDA has granted orphan drug status to mAb-114, an experimental treatment for Ebola. It’s a step forward for the 4-year-old biotech focused on pediatric orphan and infectious diseases with limited or no treatment options for patients in the developing world.
Struggling Mereo Biopharma Group plc disclosed six-month data from the open-label arm of its phase IIb dose-ranging study in adults with type I, III or IV osteogenesis imperfecta treated with BPS-804 (setrusumab). Denise Scots-Knight, Mereo's CEO, said results from the Asteroid study were in line with company expectations and she expects 12-month headline data from blinded arms of the study in the second half of this year.
Amicus Therapeutics Inc. has upped the ante on its seven-month old collaboration with the University of Pennsylvania to research and develop gene therapies for lysosomal disorders and 12 additional rare diseases, many of them affecting extremely young children.
Eli Lilly and Co. acquired worldwide rights for CNTX-0290, a somatostatin receptor type 4 agonist as a non-opioid treatment for chronic pain, from Centrexion Therapeutics Corp.
Stoke Therapeutics Inc., fresh from its $90 million series B last October, has filed a prospectus for an IPO to raise up to $86.25 million. It is one of several companies in the past week that have filed for IPOs or are prepping for one.
Immunovant Inc., one of 14 'Vants in the Roivant Sciences Ltd. family, is initiating dosing in its phase IIa trial evaluating IMVT-1401 in patients with moderate to severe active Graves' ophthalmopathy.
