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Recent Advances in Extended Half-Life Products, Nonfactor Replacement Therapies, and Gene Therpy for the Treatment of

Prakash Vishnu1, David M Aboulafia2

  • 1Fred Hutch Cancer Center, 825 Eastlake Avenue E (LG7-710), Seattle, WA 98109, USA; University of Washington, Seattle, WA, USA.

Clinics in Laboratory Medicine
|October 29, 2025
PubMed

Hemophilia, a rare X-linked hereditary bleeding disorder, is characterized by deficiency of coagulation factors. Symptoms range from spontaneous joint and muscle bleeds to life-threatening hemorrhage, for which patients require frequent infusion of coagulation factors, profoundly impacting their quality of life. Novel therapeutics and advances in gene therapy now offer patients long-term disease control and improved quality of life. Here, we focus on important developments in the use of extended half-life factor products, non-factor replacement therapies, and gene therapy. We also highlight the mechanism of action, clinical efficacy, and safety of these newer approaches.

Keywords:
Adeno-associated virus vectorsCoagulation cascadeExtended half-life factor productsGene therapyHemophiliaInhibitorsNonfactor replacement therapiesNovel therapies

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