Cambridge, Mass. — Skylark Bio, a clinical-stage biotechnology company developing genetic medicines for hearing loss and other monogenic disorders, has dosed the first patient in its SONIX Phase 1/2 clinical trial evaluating SKY-GJB2 for GJB2-related hearing loss.
SKY-GJB2 is an investigational inner-ear-targeted gene therapy designed to treat hearing loss caused by mutations in the GJB2 gene, one of the most common genetic causes of pediatric deafness.
“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,” said Jodi Cook, Ph.D., CEO of Skylark Bio.
Cook said the company expects preliminary data from the study by the end of 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,” Cook said. “We are honored by the trust of participating families and grateful to the investigators and site teams supporting this study.”
Skylark Bio is developing a genetic medicine platform for monogenic diseases that lack approved therapies and require precise local delivery and cell-specific expression.
“Launching the SONIX trial marks an exciting milestone for children and families affected by GJB2-related hearing loss,” said Daniel Lee, M.D., FACS, Ansin Foundation Chair in Otolaryngology at Massachusetts Eye and Ear and Scientific Advisor to Skylark Bio. “Although current interventions provide meaningful benefits, they do not address the underlying genetic cause. We look forward to evaluating SKY-GJB2 and its potential to support more natural hearing and spoken-language development.”
SONIX is a multicenter, open-label Phase 1/2 trial evaluating the safety, tolerability, pharmacokinetics, pharmacodynamics and preliminary efficacy of SKY-GJB2.
The study will assess a single unilateral intracochlear injection of the gene therapy in children between 9 months and 7 years of age.
