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CRISPR gene therapy could free hemophilia b patients from frequent shots

NCT ID NCT06379789

Recruiting now Disease control Sponsor: Regeneron Pharmaceuticals Source: ClinicalTrials.gov ↗

First seen Nov 01, 2025 · Last updated May 15, 2026 · Updated 34 times

Summary

This study tests a new gene therapy called REGV131-LNP1265 for people with severe or moderately severe hemophilia B. The therapy uses CRISPR to insert a working gene so the body can produce its own clotting factor, potentially reducing or eliminating the need for regular factor replacement. About 130 participants will be enrolled to check safety, side effects, and how well the therapy works over time.

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This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.

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Contacts and locations

Study contacts

  • Contact

    Phone: •••-•••-•••• Email: •••••@•••••

Locations

  • Addenbrooke's Hospital, Cambridge University Hospitals NHS FT

    RECRUITING

    Cambridge, CB2 0QQ, United Kingdom

  • Careggi University Hospital

    RECRUITING

    Florence, Firenze, 50134, Italy

  • Children's Hospital Los Angeles

    RECRUITING

    Los Angeles, California, 90027, United States

  • Children's Hospital of Philadelphia (CHOP)

    RECRUITING

    Philadelphia, Pennsylvania, 19104, United States

  • Complejo Hospitalario Universitario de A Coruña (Edificio Teresa Herrera-Materno Infantil)

    RECRUITING

    A Coruña, Galicia, 15006, Spain

  • David Geffen School of Medicine at UCLA

    RECRUITING

    Los Angeles, California, 90024, United States

  • Fondazione Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS) Ca Granda Ospedale Maggiore Policlinico

    RECRUITING

    Milan, Lombardy, 20122, Italy

  • Glasgow Royal Infirmary - Clinical Research Facility

    RECRUITING

    Glasgow, Scotland, G31 2ER, United Kingdom

  • Haemostasis and Thrombosis Unit, Hospital La Fe

    RECRUITING

    Valencia, 46026, Spain

  • Hammersmith Hospital Comprehensive Care Centre

    RECRUITING

    London, W12 0HS, United Kingdom

  • Hemostase Clinique, Institut Coeur Poumon

    RECRUITING

    Lille, Nord, 59037, France

  • Hopital Necker

    RECRUITING

    Paris, Île-de-France Region, 75015, France

  • Hospices Civils de Lyon

    RECRUITING

    Bron, Lyon, 69677, France

  • Hospital Clinico Universitario Virgen De La Arrixaca

    RECRUITING

    El Palmar, Murcia, 30120, Spain

  • Hospital Universitario Central de Asturias

    RECRUITING

    Oviedo, Principality of Asturias, 33011, Spain

  • Hospital Universitario La Paz

    RECRUITING

    Madrid, 28046, Spain

  • Hospital Universitario Miguel Servet

    RECRUITING

    Zaragoza, 50009, Spain

  • Hospital Universitario Vall d'Hebron

    RECRUITING

    Barcelona, 08035, Spain

  • Hospital Universitario Virgen del Rocio

    RECRUITING

    Seville, Andalusia, 41013, Spain

  • Indiana Hemophilia and Thrombosis Center

    RECRUITING

    Indianapolis, Indiana, 46260, United States

  • Irccs Humanitas Research Hospital

    RECRUITING

    Rozzano, Lombardy, 20089, Italy

  • McGill University Health Center (MUHC)

    RECRUITING

    Montreal, Quebec, H4J 3A1, Canada

  • McMaster University Medical Centre - Hamilton Health Sciences

    RECRUITING

    Hamilton, Ontario, L8N 3Z5, Canada

  • Nationwide Children's Hospital

    RECRUITING

    Columbus, Ohio, 43205, United States

  • Orthopaedic Hemophilia Treatment Center

    RECRUITING

    Los Angeles, California, 90007, United States

  • Ospedale san Bortolo

    RECRUITING

    Vicenza, 36100, Italy

  • Pathology and Pharmacy Building, The Royal London Hospital

    RECRUITING

    London, E1 2ES, United Kingdom

  • Queen Elizabeth Hospital Birmingham

    RECRUITING

    Birmingham, West Midlands, B15 2TH, United Kingdom

  • Royal Free London NHS Foundation Trust

    RECRUITING

    London, NW3 2QG, United Kingdom

  • Royal Prince Alfred Hospital, Haemophilia Treatment Centre

    RECRUITING

    Camperdown, New South Wales, 2050, Australia

  • St. Thomas' Hospital

    RECRUITING

    London, SE1 7EH, United Kingdom

  • Tulane University School of Medicine, Louisiana Center for Bleeding and Clotting Disorders

    RECRUITING

    New Orleans, Louisiana, 70112, United States

  • University California San Francisco

    RECRUITING

    San Francisco, California, 94143, United States

  • University Hospital Frankfurt

    RECRUITING

    Frankfurt am Main, Hesse, 60590, Germany

  • University Hospital Hamburg Eppendorf

    RECRUITING

    Hamburg, 20246, Germany

  • University of Alberta Hospital

    RECRUITING

    Edmonton, Alberta, T6G 2B7, Canada

  • University of California Davis

    RECRUITING

    Sacramento, California, 95817, United States

  • University of Colorado Hemophilia and Thrombosis Center

    RECRUITING

    Aurora, Colorado, 80045, United States

  • University of Florida

    RECRUITING

    Gainesville, Florida, 32610, United States

  • University of Michigan

    RECRUITING

    Ann Arbor, Michigan, 48109, United States

  • Yale HTC

    RECRUITING

    New Haven, Connecticut, 06510, United States

Conditions

Explore the condition pages connected to this study.

HEMOPHILIA B