SKY-GJB2 (SONIX)
Study sponsor: Skylark Bio Inc.
A first-in-human pediatric study of an AAV GJB2 gene-replacement therapy delivered once to one cochlea using the SKY-CAT investigational delivery system.
Plain-language summary
Study overview
- Current study status
- Actively Recruiting
- Who is being studied
- 9 months–7 years
- How treatment is given
- Single unilateral intracochlear infusion via transcanal endoscopic tympanotomy using SKY-CAT
- Study start date
- May 28, 2026
Study start date is reported by ClinicalTrials.gov and may differ from the date the first participant received treatment.
Official study record
NCT07627971 · Status shown: Actively Recruiting · Registry information last checked Jul 15, 2026
Terminology Dictionary
- Target gene
- The gene the treatment is designed to address.
- Gene replacement
- Delivery of a working gene copy intended to restore a missing or impaired function.
- Vector / payload
- The delivery vehicle and the genetic material it carries.
- Promoter
- A DNA control sequence that helps determine where and how strongly the delivered gene is active.
- Cochlear access
- The route used to reach the inner ear and administer the treatment.
- Laterality
- Whether treatment is given to one ear or both ears.
- Phase 1 / 2
- An early human study designed mainly to examine safety, dosing, and initial signs of biological or clinical effect.
- Early clinical
- An early human study for which a formal clinical phase is not assigned or publicly stated.
- Biallelic
- A disease-causing change is present in both copies of a gene.
- Dual vector
- Two delivery vehicles carry separate parts of a gene that is too large to fit into one vehicle.
- Intracochlear
- Delivered into the fluid-filled inner-ear structure involved in hearing.
Who is being studied
The population described by the public study record.
- Study population
- Children with biallelic GJB2-mediated hearing loss
- Age range
- 9 months–7 years
- Planned enrollment
- 10
- Study regions
- United States
This summary describes the study population; it cannot determine whether an individual qualifies.
Therapy design
The genetic treatment and how its activity is controlled.
- Target gene
- GJB2
- Treatment approach
- Gene replacement
- Delivery vector and gene cargo
- AAV gene-replacement vector delivering functional GJB2 via a proprietary Skylark capsid; the capsid identity is not publicly disclosed
- Promoter
- Not publicly disclosed
Delivery to the inner ear
How the therapy is administered and whether one or both ears are treated.
- How it is delivered
- Single unilateral intracochlear infusion via transcanal endoscopic tympanotomy using SKY-CAT
- How the inner ear is reached
- Round-window access via transcanal endoscopic tympanotomy
- Fenestration
- No additional fenestration reported
- One or both ears
- Unilateral
- Study start date
- May 28, 2026
Public evidence reviewed
Evidence and limitations
The registry and official SONIX materials describe a single unilateral intracochlear infusion using SKY-CAT. The participating Lehigh Valley trial page specifies a transcanal endoscopic tympanotomy to access the round window. These program-specific sources do not report an additional LSCC, stapes-footplate, or other auxiliary fenestration.
Details not publicly disclosed
- Capsid identity
- Promoter identity