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

NCT ID NCT06379789

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

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now This study
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Sep 11, 2026 · Updated 5 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.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 1/2

Runs two stages together: safety and dose first, then whether the treatment works.

Participants

About 130 people

The number the study aims to enrol. It can still change while the study runs.

Started

Sep 2024

Expected to finish

Aug 2047

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

2 years and older

Sex

Male participants only

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Key Inclusion Criteria: 1. Confirmed diagnosis of severe or moderately severe hemophilia B with medical history of FIX functional activity (≤2% or \<0.02 IU/mL) or documented genotype known to produce severe hemophilia B 2. Currently taking FIX prophylaxis and previous experience with FIX therapy, as defined in the protocol 3. Participation in the lead-in period of this interventional study OR a separate lead-in study (R0000-HEMB-2187 \[NCT05568459\]) for at least 6 months for ABR data while taking FIX prophylaxis, as defined in the protocol Key Exclusion Criteria: 1. History of FIX inhibitor (clinical or laboratory-based assessment) on 2 or more occasions 2. Bethesda inhibitor titer greater than the Upper Limit of Normal (ULN) at screening 3. Detectable pre-existing antibodies to the AAV8 capsid; as measured by Enzyme-Linked ImmunoSorbent Assay (ELISA) at prescreening (or final lead-in visit, if applicable) 4. Any significant underlying liver disease such as: cholestatic liver disease, liver cirrhosis, portal hypertension, splenomegaly, hepatic encephalopathy 5. Evidence of advanced liver fibrosis or significant fatty liver, as defined in the protocol 6. Evidence of cirrhosis and/or portal hypertension as assessed by abdominal ultrasound at screening or measured within 6 months prior to the screening visit 7. History of arterial or venous thrombo-embolic events, as defined in the protocol 8. History of hypersensitivity to corticosteroids or known medical condition that requires chronic administration of corticosteroids 9. Previously received any AAV gene-based therapy or intends to receive approved or investigational AAV-based gene therapy other than REGV131-LNP1265 during the study period NOTE: Other Inclusion/Exclusion Protocol Defined Criteria Apply

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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

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The places running it

    49 sites in 9 countries. The list below names each one and where it is.

  2. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  3. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

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

  • Centro Estadual de Hemoterapia e Hematologia Marcos Daniel Santos

    RECRUITING

    Vitória, Espírito Santo, 29047-105, Brazil

  • 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

  • Hemocentro Unicamp

    RECRUITING

    Campinas, São Paulo, 13084-878, Brazil

  • Hemorio

    RECRUITING

    Rio de Janeiro, 20211030, Brazil

  • 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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