- Country: The Netherlands
- Official Title: Medical Specialist
- Institute: Amsterdam University Medical Centers
- E-Mail: m.coppens@amsterdamumc.nl
Speech Title
Current Progress in Gene Therapy for Hemophilia: Advances and Potential
Hemophilia, a genetic bleeding disorder, imposes a significant disease burden on patients, characterized by frequent bleeding episodes, joint damage, and reduced quality of life. Despite recent advances in factor and non-factor treatments, including extended half-life clotting factors and non-factor agents like monoclonal antibodies, the unmet needs remain substantial. Current therapies often require lifelong management and fail to address the underlying genetic cause, highlighting the necessity for more transformative treatment options.
Gene therapy has emerged as a promising advancement, particularly for hemophilia B, which is caused by factor IX deficiency. This innovative approach aims to deliver a functional copy of the faulty gene, offering the potential for a one-time, durable treatment. Clinical trials have shown encouraging results, with patients achieving significant and sustained increases in factor IX activity levels, reduced bleeding rates, and a decreased need for prophylactic treatment. This presentation explores the clinical significance of gene therapy in hemophilia, emphasizing its potential to alter the treatment paradigm by providing long-term benefits that current therapies cannot offer. As gene therapy moves closer to real-world application, it represents a critical step toward addressing the ongoing unmet needs of the hemophilia community.