Pipeline

Transforming treatment
for GJB2-related hearing loss

At Skylark, we are dedicated to developing gene therapies that have the potential to greatly improve patient outcomes and quality of life.

Our gene therapy candidate uses an optimized AAV vector to deliver therapeutic genes directly to the cells affected by the GJB2 mutation.

By targeting cells with limited turnover within the contained environment of the inner ear (cochlea), the therapy minimizes systemic exposure and potentially reduces adverse effects while preserving benefits over time.

GJB2 and 
Hearing Loss

There are many different causes of hearing loss. Mutations in the GJB2 gene are the most common cause of inherited, non-syndromic hearing loss worldwide. These genetic changes disrupt the function of connexin 26 protein in the inner ear. The result is hearing loss that is often present at birth and can significantly impair communication and quality of life.

Unlike syndromic hearing loss, which is accompanied by other medical conditions, GJB2-related hearing loss occurs in isolation, making the gene a precise target for therapeutic intervention.

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