News

FDA Grants Orphan Drug Status to Beam Therapeutics Inc. (BEAM)’ AATD Gene Therapy
Preliminary data from ongoing Phase 1/2 trials show BEAM-302 is well tolerated and achieves dose-dependent, durable ...
Beam Therapeutics Says FDA Grants Orphan Drug Designation to Lung, Liver Treatment
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 ...
Targeted Oncology3d
Novel CRS Prevention Therapy Granted FDA Orphan Drug Designation
POLB 001 has been granted FDA orphan drug designation as a preventative therapy for cytokine release syndrome caused by ...
Ocugen wins FDA rare pediatric disease status for genetic eye disorder treatment
Ocugen (NASDAQ:OCGN) disclosed on Tuesday that its gene therapy candidate, OCU410ST, has been granted rare pediatric disease ...