Genethon advances Duchenne gene therapy trial while licensing Pompe tech to Bayer’s AskBio
Genethon advances gene therapies for Duchenne and Pompe disease. Find out what its nonprofit model and AskBio licensing deal mean for rare disease care.
What AGC Biologics’ role in Waskyra tells us about the future of ethical cell therapy scale-up
Waskyra’s FDA and EMA approvals spotlight AGC Biologics’ rare disease manufacturing model. Find out what this means for future nonprofit-led therapies.
ENYO Pharma’s vonafexor reverses kidney decline in Alport syndrome with sustained benefit
Vonafexor reversed kidney decline in Alport syndrome patients. Find out what ENYO Pharma’s Phase 2 results could mean for FXR drugs and kidney care.
What Orphalan’s Orphelia Pharma acquisition reveals about the next phase of rare pediatric drug consolidation
Orphalan, the international pharmaceutical company known for developing orphan drugs, has announced the acquisition of Orphelia Pharma, a specialist in rare and severe pediatric diseases. The deal enhances Orphalan’s position in the European rare disease market, particularly within pediatric neurology and oncology, and signals a strategic pivot toward deeper vertical integration in pediatric formulations and […]