Hemab Therapeutics recently announced positive results from a Phase 2 clinical study of their investigational subcutaneous therapy sutacimig. 

Sutacimig is a laboratory-engineered bispecific antibody being developed as the first-ever prophylactic treatment for individuals with Glanzmann thrombasthenia (GT), one of several diseases the company is looking to ultimately target with this investigational, sub-Q therapy. 

On December 4, 2025, Representatives Kean (R-NJ), Barragán (DCA), Miller-Meeks (R-IA), Auchincloss (D-MA), Fitzpatrick (R-PA), and Watson-Coleman (DNJ) reintroduced the Help Ensure Lower Patient (HELP) Copays Act to make necessary and life-saving prescription medications more affordable for patients with rare, serious, and chronic conditions such as hemophilia, von Willebrand disease, and other rare bleeding disorders.

CSL recently announced the publication of five years’ worth of data from their HOPE-B clinical trial program, which has been investigating the company’s hemophilia B gene therapy Hemgenix® (etranacogene dezaparvovec).

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Fiscal Year 2026 Funding Update 

Star Therapeutics recently announced interim data from an ongoing phase 1/2 clinical trial evaluating its investigational von Willebrand disease (VWD) therapy VGA039. 

NBDF is excited to announce that its Facts About Inhibitors booklet is now available in an Arabic language version. Using easy-to-understand language, this publication includes important information about how inhibitors develop in people with hemophilia, key risk factors, and tips on getting screened.

The holiday season is a great time to recognize the many dedicated caregivers in the bleeding disorders community. This edition of the Resource Roundup (RR) includes resources that address everyday challenges universal to all caregivers as well as those unique to individuals with bleeding disorders. 

NBDF’s inaugural Research Roundtable was held in Washington, D.C. on October 15-16, 2025. The meeting brought together lived experience experts (LEEs), researchers, healthcare providers, and industry leaders in a pre-competitive, product-agnostic setting to address barriers faced by women, girls, and people with the potential to menstruate in bleeding disorders research. Although the FDA could not attend due to the government shutdown, NBDF engaged with them on key agenda topics beforehand and plans to resume the dialog with them further once the government reopens.

The World Federation of Hemophilia (WFH) recently published an Arabic language version of their booklet Women and Girls with Hemophilia (2023) which is meant to aid outreach and educational efforts around the world, especially for such a classically underserved subgroup. Historically, women and girls affected by hemophilia have experienced protracted delays in securing an accurate diagnosis and care that takes bleeding disorders into full account.

Pathway to Cures, NBDF’s venture philanthropy fund, has added SeraGene to their portfolio of emerging biotech companies addressing unmet needs in the inheritable blood and bleeding disorders community. Meet the team!

Three new documents have been issued by NBDF’s Medical and Scientific Advisory Council (MASAC), which creates recommendations and advisories on current treatment, research matters, and other general health concerns for the inheritable bleeding disorders community.

Off-Label Use of Emicizumab in Acquired Hemophilia A

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Government Shutdown 

The commercialization of hemophilia AAV gene therapies in recent years has created a steep learning curve for clinicians, patients, and other stakeholders in the bleeding disorders community. As a novel treatment unlike any that has come before, gene therapy has compelled a series of questions and considerations relevant to its efficacy, safety, patient eligibility, implementation, cost/reimbursement, and more.

NBDF monitors emerging therapies and changes in the therapeutic landscape that may affect people living with bleeding disorders. One of those developments involves BioMarin’s decision to out-license (allowing another company to market and sell) ROCTAVIAN, their gene therapy for adults with severe hemophilia A. 

This statement from BioMarin explains their decision:

The latest Resource Roundup (RR) is dedicated to women, girls, and people with the propensity to menstruate (WGPPM), all historically underserved groups within the inherited bleeding disorders community. While recognition of these populations and their struggles to achieve healthy equity has increased recently, affected individuals continue to face challenges when it comes to timely diagnosis and management. 

Pathway to Cures, the venture philanthropy fund of the National Bleeding Disorders Foundation is focused on early-stage companies developing cures, therapies, or enabling technologies in support of the inheritable blood and bleeding disorders community.  We carry out our work with volunteer expert advisors on our scientific advisory group, investment committee and board of directors.  Today we spotlight our investment committee, our process for determining what is presented to this committee and the criteria used in making NBDF’s Pathway to Cures investments.

NBDF is pleased to inform U.S.-based clinicians and other interested individuals of a new series of virtual educational opportunities organized by our friends at the Irish Haemophilia Society (IHS). The IHS is hosting a series of clinical webinars on key topics developed for healthcare providers practicing in the global bleeding disorders community. The six webinars will be held on Zoom during the months of October, November, and December.

Star Therapeutics recently announced new financing (totaling $125 million) that will help the San Francisco-based biotechnology company continue to develop their investigational von Willebrand disease therapy VGA039. The U.S. Food and Drug Administration granted Fast Track designation to the experimental subcutaneous von Willebrand disease therapy (VWD) in January 2025.

Novo Nordisk has submitted a Biologics License Application (BLA) to the U.S. Food and Drug Administration for Mim8 (denecimig), an investigational bispecific antibody therapy. The antibody promotes coagulation by binding two key proteins (factor IXa and factor X), essentially mimicking the function of the missing factor VIII in the clotting cascade. 

While it is generally understood that the social determinants of health (SDOH) are significant factors contributing to an individual’s overall health, research specific to SDOH and its impact upon the bleeding disorders community has been rather limited. Authors of a newly published paper sought to address this knowledge gap by conducting a pilot study on SDOH in bleeding disorders, and gain insight on how these factors pose challenges among underserved groups in the U.S. 

With the fall scholastic season now in full swing, we thought it would be a great time to share a few back-to-school resources to support students, families, and school personnel. This edition of the Resource Roundup features articles, educational videos and fact sheets, all of which speak to both school-based topics and bleeding disorders.

Five people who are inspired by the bleeding disorders community are lacing up for an immense physical challenge: running the TCS New York City Marathon on November 2, 2025. They’ll be running across the Verrazano-Narrows Bridge, through the streets of Brooklyn and Queens, and down 5^th Avenue in Manhattan – and each step will help NBDF’s mission of helping people and families living with bleeding disorders to thrive.

Decades of advocacy spearheaded by the World Federation of Hemophilia (WFH) have resulted in a victory for the global bleeding disorders community. The World Health Organization (WHO) Model List of Essential Medicines (EML) has been updated to include safer and more effective medicines for the treatment of bleeding disorders. 

The U.S. Food and Drug Administration (FDA) has approved a supplemental Biologics License Application for VONVENDI®, which expands its indication to include prophylaxis use to reduce the frequency of bleeding episodes in adults with von Willebrand disease (VWD), including those with type 1 and 2 disease, and for on-demand and perioperative management of bleeding in pediatric patients with VWD.

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2025 Bleeding Disorders Conference 

NBDF wrapped up another successful Bleeding Disorders Conference. The policy team was busy with sessions on blood and product safety, access to insurance, and sharing updates on what’s happening in Washington, DC. This year there was also a session dedicated to NBDF’s advocacy efforts focused on women and girls. You can see the highlights from this year’s conference here.