C9ORF72

Clinical trials for C9ORF72 explained in plain language.

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Not yet finished but already full! 1

Experimental gene therapy targets genetic form of dementia

Disease control Ongoing

This study tests a one-time gene therapy called PBFT02 for frontotemporal dementia (FTD) in adults who have a mutation in the GRN or C9ORF72 gene. The therapy aims to deliver a working copy of the GRN gene to the brain to help control the disease. About 30 participants will recei…

Matched conditions: C9ORF72

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

Last updated May 17, 2026 04:25 UTC

C9ORF72

Get alerted when new C9ORF72 trials appear

Experimental gene therapy targets genetic form of dementia