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<\/a><\/a>\n\u201cThe SKY-PEN data we\u2019re sharing at ASGCT, together with data supporting the IND filing for our lead program SKY-GJB2, reflect important progress toward our goal of bringing life-changing treatments to patients with inherited hearing loss,\u201d said Jodi Cook, PhD, CEO of Skylark Bio. \u201cIn the Pendrin program, we\u2019re seeing robust, dose\u2011dependent improvements in hearing across multiple disease models, preservation of critical inner\u2011ear structures, and precise expression in the right cochlear cells in both mice and non\u2011human primates.\u201d<\/p>
\nSkylark Bio is advancing a differentiated genetic medicine platform built for monogenic diseases for which no approved therapies exist and where precise local delivery and cell-specific expression determine success. The company\u2019s lead program is designed to address GJB2\u2011related pediatric deafness, the most common cause of inherited non-syndromic hearing loss and a condition affecting tens of thousands of patients. The Pendrin program is designed to address the second most common genetic cause of hearing loss and represents a compelling first-in-class opportunity to improve hearing and alter the course of disease.<\/p>
\nASGCT Annual Meeting Presentation Details:<\/b><\/p>
\nOral Presentation\n
\nPoster Presentation\n
\nAbout SKY\u2011PEN<\/b><\/p>
\nSkylark\u2019s SKY\u2011PEN gene therapy program is designed to address SLC26A4\u2011related hearing loss, the second most common genetic cause of severe\u2011to\u2011profound pediatric and young\u2011adult deafness that affects thousands of patients worldwide and for which no approved therapies exist. SKY\u2011PEN offers a first\u2011in\u2011class opportunity to improve hearing and meaningfully change long\u2011term outcomes for individuals with Pendred syndrome and related conditions. Built on Skylark\u2019s proprietary capsid, SKY\u2011PEN is designed for precise, micro\u2011dose delivery with minimal systemic exposure and has demonstrated dose\u2011dependent hearing rescue across multiple mouse models in preclinical studies. The program is advancing toward IND\u2011enabling studies.<\/p>
\nAbout Skylark Bio<\/b><\/p>
\nSkylark Bio is a genetic\u2011medicine company developing precision, locally delivered gene therapies for monogenic diseases, with the potential to greatly improve patient outcomes and quality of life. The company\u2019s lead GJB2 program is IND\u2011ready with first\u2011in\u2011human studies planned for mid-2026, followed by the Pendrin program targeting SLC26A4\u2011related hearing loss now advancing toward IND\u2011enabling studies. Built on a proprietary capsid engineered for broad transduction, Skylark\u2019s platform enables targeted, micro\u2011dose delivery with cell\u2011specific expression control and the potential for durable benefit in non\u2011dividing cells of interest. The company is led by a team with deep expertise in gene therapy and supported by world\u2011class advisors as it expands its platform into additional hearing and CNS indications. For more information, visit Skylarkbio.com<\/a> and follow us on Linkedin<\/a>.<\/p>