Rare disease-focused Ovid Therapeutics Inc. has paused development of its investigational Angelman syndrome therapy, OV-101, after initial phase III results showed no difference between the drug and a placebo.
Scientists working at the University of North Carolina, Chapel Hill reported in the Oct. 21, 2020, issue of Nature on the successful development of a one-time specific sequence-directed gene therapy approach using the combination of AAV with CRISPR technology that successfully prevented the presentation of Angelman syndrome throughout the lifetime of a mouse model.
Wall Street dinged Reata Pharmaceuticals Inc. earlier this week after mixed regulatory news on the nuclear factor erythroid-2 related factor 2 (NRF2) activators omaveloxolone for Friedreich’s ataxia (FA) and bardoxolone for Alport syndrome (AS), but the Plano, Texas-based firm stayed resolute, and its recent deal with Blackstone Life Sciences (BXLS) provides cause for optimism. Increased clarity on paths forward “debunks the bear-case view” of Reata, in the opinion of Jefferies analyst Maury Raycroft, who went as far as to say in a report that the stock “overreaction create[d] a buying opportunity.”
PERTH, Australia – Melbourne-based Neuren Pharmaceuticals Ltd. will be able to progress NNZ-2591 to the clinic for three orphan indications after raising AU$20 million (US$13.75 million) in a placement to institutional and sophisticated investors in Australia, New Zealand, Hong Kong and the U.K.
PERTH, Australia – Melbourne-based Neuren Pharmaceuticals Ltd. will be able to progress NNZ-2591 to the clinic for three orphan indications after raising AU$20 million (US$13.75 million) in a placement to institutional and sophisticated investors in Australia, New Zealand, Hong Kong and the U.K.
