Top 5 hemophilia stories of 2025
Most-read stories covered treatment advances, sexual health, Beqvez halt
In 2025, Hemophilia News Today delivered readers timely reporting on research, therapeutic options, and clinical trials for hemophilia.
Below is a list of the top five most-read stories we published this year. As we enter 2026, we look forward to continuing to support and serve as a source of news and information for the hemophilia community.
No. 5 – Patients move beyond replacement therapy with Hemgenix
Early in the year, two patients in the U.S. received CSL Behring’s Hemgenix (etranacogene dezaparvovec-drlb), making them among the first to receive the gene therapy following its approval for hemophilia B. Hemgenix delivers a functional version of the F9 gene to liver cells, the body’s source of clotting proteins. Liver cells then use the gene’s information to produce factor IX, the clotting protein that is faulty or missing in hemophilia B. Both patients had experienced years of frequent bleeding and joint damage. After their Hemgenix infusions, they did not need replacement therapy, at least for the period covered in the reports.
Read more here.
No. 4 – Sexual health overlooked in men with hemophilia
Hemophilia can affect a man’s sexual function and well-being in a substantial way, according to a study from the Netherlands. While many patients experience difficulties, they rarely discuss these concerns with their doctors. This lack of communication can result in important health needs going unmet. Educational tools may help normalize the topic and encourage open conversations throughout different stages of life, researchers said.
Read more here.
No. 3 – Metagenomi’s gene-editing therapy moves closer to human testing
In 2025, Metagenomi advanced its preclinical work on MGX-001, an experimental gene-editing therapy for hemophilia A. The company plans to file regulatory applications in the U.S. and other countries to initiate clinical trials in 2026. MGX-001 delivers a functional version of the F8 gene, along with the tools to insert it into a highly active part of the DNA. This enables liver cells to produce sufficient factor VIII, preventing spontaneous bleeds. Recent data from a non-human primate study show that a single dose of MGX-001 can restore that clotting protein, which is missing or faulty in hemophilia A, bringing the therapy one step closer to human testing.
Read more here.
No. 2 – Hemlibra for hemophilia A gets high marks from patients
Roche’s Hemlibra (emicizumab-kxwh), an antibody that mimics the action of factor VIII, continued to show strong benefits for patients with hemophilia A without inhibitors, with 28 published studies indicating it can control bleeding and is well received by patients. Inhibitors are neutralizing antibodies that may cause standard replacement therapy to fail. In one study, patients gave Hemlibra an average score of 9 out of 10, and none reported a desire to return to their previous replacement therapy.
Read more here.
No. 1 – Pfizer stops marketing Beqvez for hemophilia B
Pfizer stopped developing and marketing Beqvez (fidanacogene elaparvovec-dzkt) for adults with moderate to severe hemophilia B, citing the limited interest patients and doctors have shown in gene therapy so far. Beqvez, which delivers a functional copy of the F9 gene to liver cells, which they use to produce factor IX, was the second gene therapy to be approved in the U.S. for the treatment of hemophilia B.
Read more here.
We hope that these stories, along with all our reporting at Hemophilia News Today, have provided valuable information to the hemophilia community throughout 2025. We look forward to continuing to serve as a trusted source for news in 2026, and we wish all our readers a happy new year!
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