Ultragenyx and Kyowa Kirin Announce Intent to Submit Supplemental Biologics License Application to U.S. FDA for Crysvita® (burosumab) in Tumor-Induced Osteomalacia (TIO) September 10, 2019
United States Orphan Drug Designation for Treatment of Soft Tissue Sarcomas Granted to SPEAR T-cells Targeting MAGE-A4 September 9, 2019
Insilico’s AI networks generate custom lead compounds for fibrosis in less than 50 days September 4, 2019
Ultragenyx Announces Positive Data from Phase 1/2 Study of DTX401 Gene Therapy in Glycogen Storage Disease Type Ia September 4, 2019
Denali Therapeutics Announces First Patient Dosed in Phase 1b Study of DNL151 for Parkinson’s Disease and Launch of Its Engage Parkinson’s Website September 4, 2019
Orchard Therapeutics Announces Encouraging Update from Proof-of-Concept Study of OTL-203 for the Treatment of Mucopolysaccharidosis Type I (MPS-I) September 4, 2019
Beam Therapeutics Appoints Financial Industry Veteran, Terry-Ann Burrell, as Chief Financial Officer September 4, 2019