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 ...

(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 ...

MALVERN, PA — Ocugen, Inc. (NASDAQ: OCGN) has announced that the U.S. Food and Drug Administration (FDA) has granted Rare ...

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

Regional Trends Shaping the Idiopathic Pulmonary Fibrosis Management Industry: North America leads with innovation, while Asia-Pacific shows rapid diagnostic adoption and rising treatment demand.” — ...

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

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

The FDA granted rare pediatric disease designation for OCU410ST, a gene therapy designed to treat ABCA4-associated ...

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

Ocugen (NASDAQ:OCGN) disclosed on Tuesday that its gene therapy candidate, OCU410ST, has been granted rare pediatric disease ...