Amplyx Pharmaceuticals Inc., of San Diego, said it raised $67 million through a series C financing led by Sofinnova Venture Partners, which included other new investors Lundbeckfonden Ventures, London-based Arix Bioscience plc and Pappas Capital, as well as existing investors New Enterprise Associates, RiverVest Venture Partners, 3×5 RiverVest II and BioMed Ventures.
Antibe Therapeutics Inc., of Toronto, said it received approval from Health Canada to begin a double-blind phase II gastrointestinal endoscopy trial involving 240 healthy volunteers, randomized to one of two groups.
It's not just for cancer anymore. Imbruvica (ibrutinib), the blockbuster BTK inhibitor for hematological cancers, won FDA approval today as the first therapy for patients with chronic graft-vs.-host disease (cGVHD) who have failed first-line corticosteroid treatment.
LONDON – U.S. scientists are calling for a rethink of current restrictions on using CRISPR/Cas9 gene editing in human embryos after successfully correcting the MYBPC3 hypertrophic cardiomyopathy mutation, the commonest cause of heart failure and sudden death in apparently healthy young people, without causing any off-target effects or mosaicism.
DUBLIN – A certain DNA endonuclease from Streptococcus pyogenes, Cas9, has gained enormous prominence as part of the revolutionary CRISPR-Cas9 genome editing system, but Hansa Medical AB is making waves with a different enzyme from the same bug, an endopeptidase that chews up immunoglobulin G (IgG) antibodies and which could reduce the risk of kidney transplant rejection in high-risk patients.
Spark Therapeutics Inc. is having a very good summer. Its BLA for Luxturna (voretigene neparvovec) in RPE65-mediated inherited retinal disease was accepted for priority review, as well as receiving a rare pediatric disease designation, while its hemophilia B gene therapy wowed with interim data at the International Society on Thrombosis and Haemostasis (ISTH) meeting in Berlin.
Aridis Pharmaceuticals Inc. kicked off a global pivotal study with Aerucin, a fully human monoclonal antibody (MAb) to treat acute pneumonia caused by gram-negative bacteria Pseudomonas aeruginosa.
The phase III win in March testing oral CFTR corrector tezacaftor and approved CFTR potentiator Kalydeco (ivacaftor) in cystic fibrosis (CF) patients left investors and analysts predicting success in early stage testing for triple-combo regimens featuring ivacaftor, tezacaftor and one of four possible next-generation CF correctors. But the phase I and phase II data unveiled late Tuesday by Vertex Pharmaceuticals Inc. simply blew away expectations. (See BioWorld Today, March 30, 2017.)
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