Aerska Launches with $21M to Use Brain ‘Shuttles’ for RNAi Delivery in Neurological Diseases

Aerska; Irish biotech; RNA interference (RNAi); brain shuttles; blood-brain barrier; neurological diseases; Alzheimer’s; Parkinson’s; gene silencing; precision medicine; antibody-oligonucleotide conjugates

Enanta Presses Ahead with RSV Drug Zelicapavir Despite Missing Primary Endpoint in Phase IIb Trial

Enanta Pharmaceuticals; RSV; zelicapavir; Phase IIb trial; primary endpoint miss; secondary endpoints; clinical trials; FDA Fast Track; elderly; COPD; congestive heart failure; hospitalization rates; future development

FDA restricts bluebird bio’s Skysona to CALD patients without suitable stem cell donors after higher blood cancer risk emerges

FDA; bluebird bio; Skysona; elivaldogene autotemcel; eli-cel; cerebral adrenoleukodystrophy; CALD; hematologic malignancies; myelodysplastic syndrome (MDS); acute myeloid leukemia (AML); boxed warning; label update; postmarketing safety; HLA-matched donor; gene therapy safety