C9ORF72

Clinical trials for C9ORF72 explained in plain language.

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Trials to join now! 1

Brain injection aims to halt devastating dementia

Disease control Recruiting now

This early-stage study is testing a one-time gene therapy called PBFT02 for adults with specific genetic forms of frontotemporal dementia (FTD). The therapy is delivered via a single injection into the spinal fluid, aiming to deliver a working copy of a missing or faulty gene to …

Matched conditions: C9ORF72

Phase: PHASE1, PHASE2 • Sponsor: Passage Bio, Inc. • Aim: Disease control

Last updated Mar 31, 2026 12:12 UTC

C9ORF72

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Brain injection aims to halt devastating dementia