CURRENT STATUS
Gene therapy (Hemgenix, Roctavian) already FDA-approved for hemophilia B and A. CRISPR-based fixes entering trials. The first bleeding disorders are being functionally cured today.
KEY BREAKTHROUGHS
Hemgenix (etranacogene dezaparvovec) — first gene therapy for hemophilia B, FDA-approved 2022
Roctavian (valoctocogene roxaparvovec) — gene therapy for hemophilia A, FDA-approved 2023
CRISPR-based in vivo factor VIII correction entering Phase I trials
AI-designed AAV capsids showing 100× improved liver tropism
AI-COMPRESSED PIPELINE
AI TOOLS ACCELERATING CURES
KEY ORGANIZATIONS
ACTIVELY RECRUITING TRIALS
VIVID-6 — Subcutaneous VGA039 for Von Willebrand Disease (Phase 3)
ViewVega Therapeutics (Star Therapeutics)
A Phase 3, open-label study of VGA039 — a first-in-class subcutaneous antibody that rebalances coagulation by targeting Protein S — 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.
Early Genomic Testing for Inherited Bleeding Disorders
ViewQueen'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."
Heavy Menstrual Bleeding Progestin Treatment in Bleeding Disorders (MWELL)
ViewOregon 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.
TIMELINE ESTIMATE
Hemophilia A & B: Functionally cured (gene therapy available). Rare bleeding disorders: 2–4 years to gene therapy options.