Ultragenyx Pharmaceutical RARE announced that it has submitted a biologics license application (BLA) to the FDA for its UX111 (ABO-102) AAV gene therapy as a treatment for patients with Sanfilippo syndrome...
Roche RHHBY announced data from the phase IIb PADOVA study which evaluated its investigational monoclonal antibody, prasinezumab, for treating people with early-stage Parkinson’s disease.The double-blind...
Krystal Biotech KRYS announced encouraging initial clinical data from an early to mid-stage study of its investigational immunotherapy candidate, KB707, to treat patients with solid tumors of the lung.The...
Shares of Edgewise Therapeutics EWTX were up more than 18% on Monday after it announced positive results from the mid-stage CANYON study on its lead pipeline drug, sevasemten, in patients with a rare genetic...
Shares of Editas Medicine EDIT plunged 23.8% on Dec. 13 after it announced a strategic decision to end the development of its lead gene editing therapy, reni-cel, following the failure of an extensive...
Krystal Biotech, Inc. KRYS announced initial clinical data updates on its two inhaled genetic medicine pipeline candidates, KB407 and KB408, which are being developed in early-stage studies for treating...
Shares of Q32 Bio QTTB lost nearly 77% yesterday after it reported mixed results from two separate phase IIa studies evaluating its investigational drug bempikibart one in atopic dermatitis (AD or eczema)...
Corcept Therapeutics Incorporated CORT announced data from the phase II DAZALS study evaluating its selective cortisol modulator, dazucorilant, for treating patients with amyotrophic lateral sclerosis...
Shares of Beam Therapeutics Inc. BEAM were up 5.5% on Dec. 6 after the company announced that it has appointed Sravan K. Emany as its new chief financial officer (CFO).He will assume his new role on Dec....
Affimed N.V. AFMD announced that the FDA has granted the Regenerative Medicine Advanced Therapy (RMAT) designation to its pipeline candidate, acimtamig (AFM13).The regulatory body granted the RMAT designation...