Sarepta Therapeutics Inc. CEO Doug Ingram said the U.S. FDA has promised to schedule “expeditiously” an advisory committee meeting on the BLA related to SRP-9001 (delandistrogene moxeparvovec) for Duchenne muscular dystrophy (DMD). Read More
As it struggles to launch a recently approved drug, Acer Therapeutics Inc. hit pause in its ACER-801 (osanetant) program after top-line data in the phase IIa proof-of-concept study failed to hit statistical significance. Read More
By adapting computational methods for dealing with large volumes of data, and slimming down that data, researchers at the Icahn School of Medicine at Mount Sinai have discovered previously unknown genetic associations with 19 rare diseases, and validated three of those associations. Read More
Artificial intelligence (AI)-powered drug discovery startup Protai Bio Ltd. raised $12 million in additional seed funding, bringing the total amount to $20 million that will see the company build out an oncology drug discovery pipeline derived from its AI proteomics platform. Read More
In Alzheimer’s, the amyloid beta hypothesis has proved most persistent in terms of drug development efforts to date, but aggregation of other pathogenic factors – phosphorylated tau (p-tau), APOE4, TREM2 and alpha-synuclein, for example – have also emerged as hallmarks of the disease. It’s that aggregation that seven-year-old Truebinding Inc. aims to target with its lead program, TB-006, a monoclonal antibody against galectin-3. Read More
Clinical updates, including trial initiations, enrollment status and data readouts and publications: Aldeyra, Alphamab, Alterity, Anaveon, Arbutus, Journey, Lipella, Oculis, Olipass, Purple, Pyxis, Tonix, Trethera. Read More
Biopharma happenings, including deals and partnerships, grants, preclinical data and other news in brief: Aeterna Zentaris, Aitia, Biogen, BMS, Evotec, ICER, Pharmanovia, Sanofi, Saol, UCB. Read More
Regulatory snapshots, including global drug submissions and approvals, clinical trial approvals and other regulatory decisions and designations: Abbisko, Clearmind, Everest, Glenmark. Read More
