Novartis Reveals Sustained Efficacy and Safety of Fabhalta in Patients Switching from Anti-C5 Therapies for Paroxysmal Nocturnal Hemoglobinuria (PNH)

Fabhalta (iptacopan), Paroxysmal Nocturnal Hemoglobinuria (PNH), Anti-C5 therapies, Efficacy and safety, Switching therapies, Hemoglobin improvement, Transfusion avoidance

Protara’s TARA-002 Cell Therapy Shows Exceptional Promise in Treating Non-Muscle Invasive Bladder Cancer

Protara Therapeutics, TARA-002, Non-Muscle Invasive Bladder Cancer (NMIBC), Phase 2 ADVANCED-2 trial, Cell therapy, BCG-Unresponsive, BCG-Naïve

Sarepta Therapeutics and Arrowhead Pharmaceuticals Enter $825M Global Licensing and Collaboration Agreement for Multiple siRNA Programs

Sarepta Therapeutics, Arrowhead Pharmaceuticals, siRNA programs, Global Licensing and Collaboration Agreement, Rare genetic diseases, Gene therapy, RNA interference