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🩸HEMATOLOGY

Bleeding Disorders

From lifelong factor infusions to one-time gene therapy β€” the first functional cures for hemophilia are here, and von Willebrand disease is next

Hemophilia affects roughly 1 in 5,000 male births (Hemophilia A) and 1 in 25,000 (Hemophilia B); von Willebrand disease is the most common inherited bleeding disorder, affecting up to ~1% of the population. Rare factor deficiencies and platelet-function disorders add hundreds of thousands more.
CURE AVAILABLE
92/ 100
TO BROAD CURE

MILESTONEHemgenix and Roctavian are FDA-approved gene therapies for hemophilia B and A.

LATESTCasgevy was approved for ages 2+ with sickle cell disease and TDT.

An approved cure exists for a major form of this disease Β· Not medical advice.

CURRENT STATUS

Hematology has crossed the line from lifelong management to functional cure for its flagship disease. Two one-time gene therapies are now approved β€” Hemgenix for Hemophilia B (2022) and Roctavian for Hemophilia A (2023) β€” delivering a working clotting-factor gene to the liver so patients make their own factor for years. A parallel β€œrebalancing” class (fitusiran, concizumab) lowers natural anticoagulants instead of replacing factor, working across inhibitor status and by subcutaneous injection. Von Willebrand disease, long the neglected majority, finally has a mechanism-specific therapy (anti–Protein S) in pivotal trials. The remaining frontiers are durability, the rare factor deficiencies, and platelet-function disorders.

KEY BREAKTHROUGHS

Hemgenix (etranacogene dezaparvovec) β€” first gene therapy for hemophilia B, FDA-approved 2022

Roctavian (valoctocogene roxaparvovec) β€” gene therapy for hemophilia A, FDA-approved 2023

CRISPR-based in vivo factor VIII correction entering Phase I trials

AI-designed AAV capsids showing 100Γ— improved liver tropism

AI-COMPRESSED PIPELINE

AI TOOLS ACCELERATING CURES

AI Coagulation ModelingCRISPR Factor CorrectionAAV Vector OptimizationDigital Twin Dosing

KEY ORGANIZATIONS

BioMarinCSL BehringSpark TherapeuticsPfizeruniQure

KEY CLINICAL TRIALS

VIVID-6 β€” Subcutaneous VGA039 for Von Willebrand Disease (Phase 3)

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Vega Therapeutics (Star Therapeutics)

A Phase 3, open-label study of VGA039 β€” a first-in-class subcutaneous antibody that rebalances coagulation by targeting Protein S β€” given as prophylaxis to reduce bleeding across every type of von Willebrand Disease. A 24-week observational run-in is followed by ~49 weeks of treatment, sidestepping the need for frequent VWF-concentrate infusions.

πŸ‘₯ ~60 participants aged 12–75 with VWD (ABR β‰₯12/year)πŸ“ US β€” AR, CA, GA, MN + virtual (Science 37); multicenter

Early Genomic Testing for Inherited Bleeding Disorders

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Queen's University

Tests whether DNA testing (300+ genes linked to bleeding and clotting) introduced earlier in the diagnostic process can shorten the journey for the up to half of patients labeled "bleeding disorder of unknown cause."

πŸ‘₯ Recruiting β€” standard vs. standard + early genomic testingπŸ“ Canada β€” Kingston & Ottawa, ON (Toronto site planned)

Heavy Menstrual Bleeding Progestin Treatment in Bleeding Disorders (MWELL)

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Oregon Health & Science University

Compares the levonorgestrel IUD (LNG-IUD) vs. oral norethindrone acetate (NETA) for heavy menstrual bleeding in adolescents and young adults with inherited bleeding disorders, tracking bleeding, quality of life, and iron restoration over six months.

πŸ‘₯ ~300 participants aged 10–24 (multicenter)πŸ“ US β€” OHSU Portland + CA, CO, GA, MI, MO, PA, WA (planned)

TIMELINE ESTIMATE

Hemophilia B: functional cure available now (Hemgenix). Hemophilia A: gene therapy approved, durability under study. Von Willebrand disease: pivotal rebalancing trials, 2–4 years. Rare factors & platelet disorders: 5–10+ years.

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