Therapeutics
Details checkedStatus confirmedHistorical/discontinuedCheckedtrial

CGF166 by Novartis

Also known as: CGF166, Hath1 gene therapy, ATOH1 gene therapy

Novartis

Primary source
Company
Novartis
Program Type
Therapeutic
Modality
Gene Therapy
Stage
Phase 1/2
Status
Completed
Target Gene
ATOH1
Route / Mode
Intralabyrinthine infusion
Confidence
89%
Status Confidence
Status confirmed
Evidence Strength
Needs more support
Last Reviewed
2026-05-31
Geography
United States
Population
adult, bilateral, profound
Trial IDs
1
Mechanism

Adenoviral ATOH1 gene transfer was intended to induce supporting cells to become new sensory hair cells in severe hearing loss.

Indications
Notes

Historically important first-wave inner-ear gene therapy; clinical proof-of-concept was limited, but CGF166 established the feasibility of human hair-cell-regeneration gene transfer studies.

Evidence & Sources
3 structured sources
company
Supports: canonicalName, company, mechanism, targetGene, indications
IDs: CGF166
Open
clinicaltrials
Supports: trialIds, stage, route, populationTags, geography
IDs: NCT02132130
Open
company
Supports: route, targetGene, notes
IDs: CGF166
Open
Key Events
Clinical Trials (1)
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