Skylark Bio has dosed the first patient in its SONIX Phase I/II clinical trial assessing SKY-GJB2 as a potential gene therapy for children with hearing loss linked to GJB2 gene changes.
The trial is designed to assess the safety, tolerability, pharmacokinetics and pharmacodynamics.
It will also evaluate the preliminary efficacy of SKY-GJB2's single unilateral intracochlear injection in paediatric patients aged between nine months and seven years across multiple sites.
The study aims to gather initial study findings by the end of 2026, while further data is expected during 2027.
SKY-GJB2 is designed to deliver a functional copy of the GJB2 gene directly to supporting cells within the cochlea that are integral for the natural expression of connexin 26, a protein required for normal hearing function.
Mutations in the GJB2 gene disrupt this protein's function and impact the gap-junction network within the inner ear, which is crucial for hearing.
Skylark Bio CEO Jodi Cook said: "Dosing the first patient in SONIX brings us one step closer to a future where children born with genetic hearing loss have new treatment options that could meaningfully change the trajectory of their lives. SONIX is designed to generate preliminary data by year-end 2026 with additional readouts anticipated in 2027.
"The recent approval of a targeted inner-ear AAV-delivered therapy provides important validation for the field and further reinforces our confidence in this approach. We are honoured by the trust of participating families and grateful to the investigators and site teams supporting this study."
Skylark Bio focuses on genetic medicines for monogenic diseases and is also developing other gene therapies targeting SLC26A4-related hearing loss, as well as investigational programmes in the central nervous system.
"First patient in Skylark Bio's Phase I/II trial receives SKY-GJB2 gene therapy" was originally created and published by Clinical Trials Arena, a GlobalData owned brand.