Hemophilia Insights

Bleeding Rates in Severe Hemophilia A Drop with Prophylaxis Use

Prophylaxis treatment has significantly reduced bleeding rates in people with severe hemophilia A, according to recent studies. Regular infusions of clotting factor help prevent spontaneous bleeds and improve overall health outcomes for those with the condition.

Careful Management Needed for Patients Undergoing PCI Treatment

People with hemophilia need careful management when undergoing treatment for other conditions, as certain medications and procedures can increase the risk of bleeding. Vigilant monitoring and adjusted treatment plans are crucial to prevent complications.

UC San Diego Health Offers New Gene Therapy

UC San Diego Health is the first to offer a new gene therapy for hemophilia B, a treatment that could potentially cure the condition. This innovative therapy involves injecting a virus to deliver a healthy copy of the faulty gene responsible for hemophilia B.

Saliva Could Help Coagulation in Hemophilia Patients

Researchers have discovered that saliva from patients with hemophilia contains coagulation factors that could potentially be used to develop new treatments. This finding opens up new avenues for managing and treating hemophilia through saliva-based therapies.

Pfizer has decided to discontinue its collaboration with Sangamo Therapeutics on a gene therapy for hemophilia A, despite the therapy being on the brink of regulatory submission. This move by Pfizer, reported by Vinluan (2025), leaves Sangamo in search of a new partner to advance the program. Mast (2025) notes that Sangamo, a pioneer in gene therapy, has lost a significant financial support through this termination.

Furthermore, despite a successful Phase 3 trial earlier in the year, Pfizer's withdrawal has cast doubt on the future of this promising treatment, as highlighted by LaHucik (2025). Sangamo now faces the challenge of securing a new partnership to continue the development of the gene therapy, which had the potential to significantly impact the treatment landscape for hemophilia A.