PBFT02
Asset
AAV1 gene therapy delivering functional human progranulin
- Stage
- Phase 1
- Modality
- Gene therapy
- Target
- Autophagy
- 5
- Indications
- 1
- Trial
- 1
- Developer
Indications
5C9orf72
Phase 1, Active not recruiting, NCT04747431, clinicaltrials.gov, 7 Oct 2026
Dementia frontotemporal
Phase 1, Active not recruiting, NCT04747431, clinicaltrials.gov, 7 Oct 2026
Frontotemporal dementia
Phase 1, Active not recruiting, NCT04747431, clinicaltrials.gov, 7 Oct 2026
2 more
In the full profile
- Targets and what it contains4
- Owner and partners1
- Trials1
- Evidence for its targets4
- Competitors3
- Aging relevance2
Every asset’s record: target, stage, trials and the evidence behind it.