Oryzon’s Iadademstat Approved for Phase 1b Sickle Cell Disease Study by EMA
Oryzon Genomics has received EMA approval for a Phase 1b trial of iadademstat in sickle cellsickle cell disease (SCD)....
Artelo Biosciences: New Data on Non-Opioid Pain Treatment
Artelo Biosciences announced positive safety and pharmacokinetic data from a Phase 1 single-ascending-dose (SAD) trial, including a preliminary food...
Cytokinetics Announces Five Aficamten Presentations at ESC Congress 2025
Cytokinetics will present data from several aficamten trials at the upcoming European Society of Cardiology (ESC) Congress 2025, including...
Sionna Therapeutics Doses First Subjects in Phase 1 Cystic Fibrosis Trial
Sionna Therapeutics has initiated dosing in a Phase 1 trial evaluating dual combinations of its lead candidate, the NBD1...
Why FDA’s Draft 510(k) Guidance is Forcing Sponsors to Rethink Device Submissions
The device team thought they were following the playbook. They chose a predicate that had been cleared two decades...
New Gene Therapy Guidelines for Duchenne Muscular Dystrophy Released
The Muscular Dystrophy Association (MDA) and Parent Project Muscular Dystrophy (PPMD) have jointly released consensus guidelines for the delivery...
Argenx’s Vyvgart Shows Promise in AChR-Ab Seronegative gMG Patients
Argenx’s efgartigimod met its primary endpoint in the ADAPT SERON trial, demonstrating a statistically significant improvement in MG-ADL scores...
Blossomhill Doses First Patient in Expanded Phase 1/2 Solara Trial Cohorts
BlossomHill Therapeutics has initiated dosing in the expansion cohorts of its Phase 1/2 SOLARA trial evaluating BH-30643, a novel...
Scilex CEO to Focus on Semnur’s SP-102 for Sciatica Treatment
Semnur Pharmaceuticals, a majority-owned subsidiary of Scilex Holding Company, has secured a $20 million private placement to fund the...
Krystal Biotech Focuses on Inhaled KB707 for Non-Small Cell Lung Cancer
Krystal Biotech has prioritized the inhaled formulation of KB707, its redosable gene therapy for solid tumors, based on early...