Phase I
The FDA has approved Alnylam Pharmaceuticals’ Amvuttra (vutrisiran) for the treatment of hereditary transthyretin-mediated amyloidosis (hATTR) with polyneuropathy in adults.
Shares of CRISPR Therapeutics fell more than 11% on Monday as investors react negatively to the endorsement of a rival beta-thalassemia gene therapy developed by bluebird bio.
BioSpace looks at several companies developing new gene therapies in the wake of the FDA recommending two of bluebird bio’s lentiviral vector gene therapies.
CRISPR Therapeutics shared positive initial data from its Phase I COBALT-LYM trial of CTX130 for cancer which demonstrated safety and efficacy when used to target CD70.
The European Hematology Association’s 2022 Hybrid Congress is in full swing in Vienna, Austria and will feature the newest and most promising developments in blood cancer research.
It was an extraordinarily busy week for clinical trial news and updates due to several prominent international conferences. Here’s a look at some of the highlights.
Owkin and BMS signed a deal to utilize Owkin’s artificial intelligence capabilities to carry out more targeted and efficient clinical trials on a wide range of therapeutic candidates.
Cemdisiran, an RNAi mono-therapeutic intended to treat IgAN is poised to enter Phase III clinical studies after positive Phase II topline results were shared Thursday morning.
Precision BioSciences’ clinical program exploring allogeneic CAR T therapy may well produce a market pioneer after initial data showed its ability to deliver a 100% overall response rate.
At ENDO 2022, Ascendis will share Phase II and III data showing clinical efficacy for its investigational product candidate for hypoparathyroidism. For this and more, see inside.
PRESS RELEASES