Beam Therapeutics Inc. (NASDAQ:BEAM) has secured U.S. FDA orphan drug designation for BEAM-302, its pioneering genetic ...

The Cambridge, Mass.-based biotechnology company's drug, BEAM-302, is a liver-targeting lipid nanoparticle formulation that is designed to correct for a mutation in patients with alpha-1 antitrypsin ...

Beam Therapeutics Inc. (Nasdaq: BEAM), a biotechnology company developing precision genetic medicines through base editing, today announced that the United States (U.S.) Food and Drug Administration ...

Eton Pharmaceuticals is able to buy orphan drugs from large pharma companies, where they are too small to matter. Read why ...

POLB 001 has been granted FDA orphan drug designation as a preventative therapy for cytokine release syndrome caused by ...

The FDA has granted Orphan Drug designation to NS-229 for the treatment of eosinophilic granulomatosis with polyangiitis.

(Alliance News) - Nuformix PLC on Thursday said its lead programme, NXP002, has been granted orphan drug designation by the European Medicines Agency for the treatment of idiopathic pulmonary fibrosis ...

The European Medicines Agency (EMA) has designated allopurinol as the first orphan drug for the treatment of Marfan syndrome, ...

The European Medicines Agency (EMA) has designated allopurinol as the first orphan drug for the treatment of Marfan syndrome, a rare connective tissue disease which has no cure to date. This disease ...

Stocks slipped in Asia on Friday and the U.S. dollar drooped with Treasury yields as investors digested an appeals court kept ...

Poolbeg Pharma (AIM: POLB, 'Poolbeg' or the 'Company'), a clinical-stage biopharmaceutical company focussed on the development of innovative medicines to address unmet medical needs, announces that ...