What's Happening?
AGC Biologics has announced the approval of Waskyra, a gene therapy for Wiskott-Aldrich syndrome, by both the U.S. Food and Drug Administration and the European Commission. Developed by Fondazione Telethon, Waskyra is an ex vivo gene therapy using autologous CD34+ hematopoietic stem and progenitor cells. This therapy addresses a rare immune disorder that affects approximately 1 in 250,000 live male births, leading to severe infections and other complications. AGC Biologics played a crucial role in developing and producing the clinical-grade lentiviral vectors and genetically engineered cells necessary for this therapy. The approval marks a significant milestone for AGC Biologics, further establishing its Milan Cell and Gene Center of Excellence
as a leader in the field of cell and gene therapies.
Why It's Important?
The approval of Waskyra represents a significant advancement in the treatment of rare diseases, offering a new therapeutic option for patients with Wiskott-Aldrich syndrome. This development highlights the potential of gene therapies to address complex genetic disorders and underscores the importance of collaborations between biopharmaceutical companies and research organizations. For AGC Biologics, this approval enhances its reputation as a leader in cell and gene therapy manufacturing, potentially attracting more partnerships and projects. The success of Waskyra also demonstrates the viability of developing treatments for rare diseases, which are often considered economically challenging due to small patient populations.
