Bleeding Disorders

Gene Transfer Therapy Updates

Yesterday, 10-28-25, we reported BioMarin’s press release announcing divestiture of their hemophilia A gene transfer therapy, valoctocogene roxaparvovec (Roctavian®). Previously, on February 25, 2025, Pfizer announced their intention to discontinue hemophilia B gene transfer therapy, fidanacogene elaparvovec-dzkt (Becvez®). Press releases from both indicate underperformance in sales. The remaining hemophilia B therapy, etranacogene dezaparvovec-drlb (HEMGENIX®), CSL […]

Bleeding Disorders

BioMarin Plans to Divest Struggling Gene Therapy

Thanks to Dean Willett of Precision BioLogic Inc. for the heads-up on BioMarin’s 10-27-25 post announcing their divestment of the hemophilia A gene transfer therapy, Roctavian. See BioMarin’s 10-27 press release.

Bleeding Disorders

The Blood Project: TTP Module

Click here for a comprehensive review of TTP. This is a segment within The Blood Project, a not-for-profit organization committed to advancing medical knowledge about the crucial role of blood in human health, disease, and therapeutics. TBP offers open-access, web-based, and expertly curated learning tools and resources with the following goals: Enhance Hematological Knowledge: TBP […]

Bleeding Disorders

Precision BioLogic Webinar: Advancing Hemophilia A Testing with a Bovine Chromogenic FVIII Assay

Listen as Precision BioLogic’s Chief Scientific Officer F. Jon Geske, PhD and Marketing Director Ian Burns chair the webinar, Advancing Hemophilia A Testing with a Bovine Chromogenic FVIII Assay. Joining them are… Tara Quinton, R&D Scientist and part of Precision BioLogic’s Chromogenic FVIII assay design team, discussing the assay’s expanded utility along with new data […]

Bleeding Disorders

Activated Protein Complex Concentrate in Emicizumab Prophylaxis

Here is a current open-access review describing APCC usage in emicizumab therapy: Sidonio RF Jr, Young G, Escuriola Ettingshausen C, et al. Activated prothrombin complex concentrate in patients receiving emicizumab prophylaxis: from evidence to clinical practice. Res Pract Thromb Haemost. 2025;9:102926. doi: 10.1016/j.rpth.2025.102926. PMID: 40808796; PMCID: PMC12346059. Abstract Nonfactor therapies (NFTs) such as emicizumab are increasingly […]

Bleeding Disorders

University of Kansas Article About TTP

Here is an editorial review article about TTP that appears in the Spring-Summer edition of the University of Kansas “Kansas Medicine” magazine, entitled “In Their Blood.” The article features X. Long Zheng, MD, PhD, KU pathology head, who provided our January 6, 2021 webinar, “von Willebrand Factor, Friend or Foe?“

Bleeding Disorders

An Experimental Oral FVIII Mimetic

Click here in Hemostasis Today for a brief discussion of a developing phase 1 oral FVIII mimetic: Wolfgang Miesbach: Jan Astermark at the BIC 2025 presented Potentially the First Oral FVIII Mimetic Treatment for Hemophilia A.

Bleeding Disorders

Incubation to Inactivate Efanesoctocog Alfa Prior to Inhibitor Bethesda Titer

Click here for an open-access letter that indicates the need to inactivate plasma EHL FVIII efanesoctocog alfa (Altuuviiio®; Altuvoct®) before performing a Bethesda titer to measure anti-FVIII inhibitor: Bowyer A. Is incubation for 30 minutes at 56°C sufficient to fully inactivate efanesoctocog alfa? Haemophilia. 2025. doi: 10.1111/hae.70137. PMID: 40985057. The findings provide a recommended incubation […]

Bleeding Disorders

WFH Fibrinogen Disorders Booklet

Click here for a recent WFH booklet, What Are Fibrinogen Disorders?

Bleeding Disorders

Effect of Rebalancing Therapy on Coagulation Assays

Click to read this open-access article that explores the effects of antithrombin reduction (rebalancing) hemophilia therapy on a series of coagulation tests: Chhabra, ES ; Liu, M. Evaluation of the interference of fitusiran and antithrombin lowering in plasma on routine coagulation assays. Research and practice in thrombosis and haemostasis, 2025;9:102989–9.

Bleeding Disorders

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George Fritsma is a medical laboratory scientist, educator, and author. George is a member of the International Society on Thrombosis and Hemostasis, the International Society for Laboratory Hematology, and the American Society for Clinical Laboratory Science.

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