Sentynl Therapeutics Inc., the United States-based rare disease subsidiary of Zydus Lifesciences Limited, has entered into a licensing agreement with South Korean biotechnology company PRG S&T to develop the investigational molecule Progerinin (SLC-D011) for Hutchinson-Gilford Progeria Syndrome, an ultra-rare genetic disorder that causes accelerated aging in children. The therapy has received orphan drug designation from […]
FDA grants Breakthrough Therapy Designation to Hemab Therapeutics’ sutacimig for Glanzmann thrombasthenia. Discover what this means for bleeding disorder treatment.
Genethon advances gene therapies for Duchenne and Pompe disease. Find out what its nonprofit model and AskBio licensing deal mean for rare disease care.
Waskyra’s FDA and EMA approvals spotlight AGC Biologics’ rare disease manufacturing model. Find out what this means for future nonprofit-led therapies.
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 […]
