by GlobeNewsWire | Sep 6, 2016 | Globe Newswire
CHICAGO, Sept. 06, 2016 (GLOBE NEWSWIRE) — AveXis, Inc. (NASDAQ:AVXS) today announced that it intends to offer and sell, subject to market conditions, 3,566,474 shares of its common stock in an underwritten public offering. Additionally, PBM Capital Investments,...
by GlobeNewsWire | Aug 11, 2016 | Globe Newswire
— Interim data through July 1 showed no “events” — — Continued motor function improvement with 25% of patients in the proposed therapeutic dose group in the normal range — — FDA requested Type B Meeting to discuss SMA Type 1 clinical...
by GlobeNewsWire | Aug 11, 2016 | Globe Newswire
Ongoing Phase 1/2 trial DTX101 for hemophilia B clinical data expected later this year Lead IMD program, DTX301 for OTC deficiency, progressing toward IND filing Advancing broad inherited metabolic disease (IMD) portfolio with programs addressing citrullinemia type 1,...
by GlobeNewsWire | Aug 11, 2016 | Globe Newswire
VY-AADC01 Phase 1b Study for Advanced Parkinson’s Disease On Track to Report Six-Month Safety, Motor Function, and Biomarker Data from Cohorts 1 and 2 by Year-End 2016 Pipeline Advances with Selection of Lead Clinical Candidates for Multiple Pipeline Programs by Late...
by GlobeNewsWire | Aug 10, 2016 | Globe Newswire
Updated timing of the completed BLA submission for voretigene neparvovecEarlier today, released additional positive data from the pivotal Phase 3 trial of voretigene neparvovecReported positive preliminary safety and efficacy from the Phase 1/2 clinical trial of...