CRISPR gene therapy aims to fix hemophilia b at the DNA level

NCT ID NCT06379789

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

First seen Jun 27, 2026 · Last updated Aug 06, 2026 · Updated 4 times

Summary

This study tests an experimental gene insertion therapy called REGV131-LNP1265 for people with severe or moderately severe hemophilia B. The therapy uses CRISPR/Cas9 to add a working copy of the clotting factor IX gene into the body's DNA, aiming to enable long-term production of the clotting factor. The trial will enroll 130 participants and focus on safety, dosing, and whether the treatment reduces bleeding episodes and the need for factor replacement therapy.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance: REGV131-LNP1265 (a CRISPR/Cas9 gene insertion therapy)
What this could lead to: If successful, this could allow people with hemophilia B to produce their own clotting factor long-term, reducing or eliminating the need for regular factor replacement infusions.
What could go wrong: This is an early Phase 1/2 trial, so safety and effectiveness are not yet proven. The body might develop antibodies against the therapy, reducing its effect or causing side effects.

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Conditions

The condition(s) this trial relates to.

hemophilia B

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

HEMOPHILIA B

Contacts and locations

Locations

Addenbrooke's Hospital, Cambridge University Hospitals NHS FT
RECRUITING

Cambridge, CB2 0QQ, United Kingdom
Alfred Hospital
RECRUITING

Melbourne, Victoria, 3004, Australia
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 Adelaide Hospital
RECRUITING

Adelaide, South Australia, 5000, Australia
Royal Brisbane and Women's Hospital
RECRUITING

Herston, Queensland, 4029, Australia
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
University of Texas Health Science Center at Houston
RECRUITING

Houston, Texas, 77030, United States
Vanderbilt University Medical Center
RECRUITING

Nashville, Tennessee, 37232, United States
Yale HTC
RECRUITING

New Haven, Connecticut, 06510, United States

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