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Gene therapy infused directly into brain aims to halt rare dementia

NCT ID NCT06064890

Recruiting now Disease control Sponsor: AviadoBio Ltd Source: ClinicalTrials.gov ↗

First seen Jun 25, 2026 · Last updated Aug 14, 2026 · Updated 3 times

Summary

This early-phase trial tests a gene therapy called AVB-101 for people with frontotemporal dementia caused by GRN gene mutations. The therapy delivers a working copy of the GRN gene directly into the brain during a one-time procedure. The study will enroll 18 adults aged 30-75 to check safety and whether it can restore normal levels of a key protein, potentially slowing the disease.

What this could mean

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

Active substance
AVB-101 (a gene therapy that delivers a working copy of the GRN gene into brain cells)
What this could lead to
If it works, this could slow or stop the progression of a devastating early-onset dementia by restoring a missing protein in the brain.
What could go wrong
This is a very early, small trial (18 people) testing safety first. Gene therapy delivered directly to the brain carries risks like brain swelling or bleeding, and it may not improve symptoms.

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Conditions

The condition(s) this trial relates to.

dementia frontotemporal dementia GRN-related frontotemporal lobar degeneration with Tdp43 inclusions primary progressive aphasia semantic dementia

As listed by the trial registrant

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

DEMENTIA, FRONTOTEMPORAL FRONTOTEMPORAL DEMENTIA FTD FTD-GRN

Contacts and locations

Locations

  • Amsterdam UMC

    RECRUITING

    Amsterdam, Netherlands

  • Cambridge University Hospitals NHS Foundation Trust

    RECRUITING

    Cambridge, CB2 0QQ, United Kingdom

  • Centrum Medyczne NeuroProtect Sp z o.o.

    RECRUITING

    Warsaw, Poland

  • Euromedis Sp. z o.o.

    RECRUITING

    Szczecin, 70-111, Poland

  • Fondazione IRCCS Istituto Neurologico Carlo Besta

    RECRUITING

    Milan, Italy

  • Hospital Clinic Barcelona

    RECRUITING

    Barcelona, 08036, Spain

  • Hospital Universitari i Politecnic La Fe

    RECRUITING

    Valencia, 46026, Spain

  • Houston Methodist Hospital

    RECRUITING

    Houston, Texas, 77030, United States

  • Mazowiecki Szpital Brodnowski Sp. z o. o.

    RECRUITING

    Warsaw, Poland

  • NEURO-CARE Sp. z o.o. Sp. Komandytowa

    RECRUITING

    Katowice, Poland

  • Neurologia Slaska Centrum Medyczne

    RECRUITING

    Katowice, Poland

  • Skåne University Hospital

    RECRUITING

    Lund, Sweden

  • Sunnybrook Health Sciences Centre

    RECRUITING

    Toronto, Ontario, Canada

  • The Ohio State University (OSU) Wexner Medical Center

    RECRUITING

    Columbus, Ohio, 43210, United States

  • UZ Leuven

    RECRUITING

    Leuven, Belgium

  • University College London Hospitals

    RECRUITING

    London, United Kingdom

  • University Hospital of Wales

    RECRUITING

    Cardiff, CF14 4XW, United Kingdom

  • Uniwersyteckie Centrum Kliniczne, SUM w Katowicach

    RECRUITING

    Katowice, Poland

  • Vanderbilt University Medical Centre

    RECRUITING

    Nashville, Tennessee, 37232, United States

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