September Summary

I’m trying something new with HemaBlog: a quick rundown on what’s new this past month in the bleeding disorders community. New therapies are moving through clinical development, gene therapy remains an important—and increasingly complicated—part of the conversation, and major policy changes could affect access to treatment for some families. At the same time, greater attention is being paid to von Willebrand disease, rare bleeding disorders, and the long-overlooked needs of women and girls.

Here are several developments worth watching this month.

Gene Therapy Is Still Very Much in the Conversation

The past year has been challenging for hemophilia gene therapy, but hematologists have not lost interest.

A recent survey of European hematologists found that roughly 90% believe gene therapies will play a role in the future of hemophilia care. That is striking considering the commercial difficulties the field has experienced, including the discontinuation of some gene therapy programs and supply issues affecting HEMGENIX earlier this year.

The bigger question may no longer be simply whether gene therapy works. We now have to ask: Who is the best candidate? How durable will the effect be? What risks are acceptable? And will patients choose gene therapy when other highly effective treatments are already available?

Those questions will become increasingly important as gene therapy research continues.

Patients Like Their Treatments—but They Still Want Better

Another recent survey offers an important perspective from people actually living with hemophilia.

A Pfizer/Harris Poll survey of 1,000 people with hemophilia worldwide found that 84% were somewhat or very satisfied with their current treatment. But satisfaction did not mean that treatment burden had disappeared.

Participants still reported challenges such as declining venous access, difficulty being spontaneous, and treatment interfering with social life. When asked what might persuade them to change therapies, patients emphasized better effectiveness, easier administration and strong real-world evidence.

This reflects an important evolution in hemophilia treatment.

We once measured success primarily by survival and the prevention of serious bleeding. Today, patients are understandably asking for more: fewer bleeds, healthier joints, easier administration, freedom to travel and exercise, and less disruption of everyday life.

New Approaches to von Willebrand Disease

One of the most encouraging developments in bleeding disorders research is the growing attention being paid to von Willebrand disease (VWD). Hemab Therapeutics recently reported early clinical data on HMB-002, an experimental antibody being developed as a non-replacement treatment for VWD. In an early Phase 1/2 study, HMB-002 produced at least a 2.4-fold peak increase in von Willebrand factor and factor VIII. The treatment is being designed as a potentially monthly subcutaneous therapy rather than traditional replacement therapy.

These are early results and the study was not designed to prove clinical effectiveness. Still, the findings are interesting because they represent a fundamentally different way of approaching VWD treatment. Additional clinical data are expected later in 2026 or early 2027.

Medicaid Changes Deserve Attention Now

For U.S. families, one of the most consequential stories may have nothing to do with a new drug. New federal Medicaid work-reporting requirements are scheduled to take effect no later than January 1, 2027 for certain adults ages 19–64. Bleeding disorder organizations are warning that patients should begin preparing now.

CMS guidance recognizes that people with serious or complex medical conditions may qualify for exemptions, and bleeding-disorder advocates have specifically pushed for hemophilia and related conditions to be appropriately recognized. However, having a bleeding disorder may not automatically guarantee an exemption.

Patients who rely on Medicaid should keep their contact information current, carefully read notices from their state Medicaid program and discuss possible medical exemptions with their hemophilia treatment center. For a community dependent on extremely expensive, life-sustaining therapies, even a temporary loss of insurance coverage can be serious.

Women and Girls With Bleeding Disorders Take Center Stage

Recognition of bleeding disorders in women continues to grow internationally. This month the World Federation of Hemophilia highlighted Project Elevate Her, an unusual global awareness campaign led by mountaineer Maia Meier, who lives with Type 2A von Willebrand disease.

Meier is attempting the Seven Summits and a Last Degree ski expedition to the South Pole while using each expedition to highlight issues facing women and girls with bleeding disorders. Her September expedition to Manaslu focuses on a critical question: once a woman finally receives a bleeding disorder diagnosis, can she actually obtain appropriate care?

That question remains relevant around the world, where heavy menstrual bleeding and other bleeding symptoms in women are still too often normalized or overlooked.

And Globally, Access Still Matters Most

While much of the news in wealthier countries concerns gene therapy and sophisticated new drugs, the World Federation of Hemophilia offered an important reminder this month from Morocco. Long-term cooperation among the WFH Humanitarian Aid Program, the Moroccan Hemophilia Association, treatment centers, healthcare professionals and national authorities has significantly expanded access to treatment and comprehensive bleeding-disorder care there. It is worth remembering that for many people around the world, the breakthrough they need most is not gene editing or a new monoclonal antibody.

It is simply reliable diagnosis, access to treatment and knowledgeable medical care. And ultimately, that remains one of the central challenges facing the global bleeding disorders community.

Sources

Hemophilia Federation of America: Gene Therapy Remains Top of Mind for Hematologists

Hemophilia Federation of America: Latest bleeding disorders news and policy updates

Hemab Therapeutics: August 2026 clinical and corporate update

World Federation of Hemophilia: Project Elevate Her

World Federation of Hemophilia: Hemophilia care improvements in Morocco