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 ...

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 ...

Biopharmaceutical company Poolbeg Pharma said the US Food and Drug Administration has granted Orphan Drug Designation (ODD) ...

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 ...

Those with ALS have an average survival of around two to five years from diagnosis, with most ALS patients dying from ...

The author has a $2.75 Price Target on CytoDyn (CYDY) in the Next 6 to 12 months, representing significant upside from its ...

LUND, SE / ACCESS Newswire / May 26, 2025 / Alligator Bioscience (STO:ATORX) - Lund, Sweden - 26 May 2025 - Alligator Bioscience (Nasdaq Stockholm: ATORX) today announces that the European Commission ...

Qure divested for $25M, advanced AMT-130 with FDA approvals, and building infrastructure. A strong balance sheet backs ...