Skylark Bio, a biotechnology company developing targeted genetic medicines for hearing loss, today announced that it will present new preclinical data for SKY-PEN, its Pendrin gene therapy program in development for SLC26A4-related hearing loss, at the American Society of Gene & Cell Therapy (ASGCT) 2026 Annual Meeting.
“The SKY-PEN data we’re 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,” said Jodi Cook, PhD, CEO of Skylark Bio. “In the Pendrin program, we’re seeing robust, dose‑dependent improvements in hearing across multiple disease models, preservation of critical inner‑ear structures, and precise expression in the right cochlear cells in both mice and non‑human primates.”
Skylark 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’s lead program is designed to address GJB2‑related 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.
ASGCT Annual Meeting Presentation Details:
Oral Presentation
Title: Targeted AAV Gene Therapy for Genetic Hearing Loss in Pendred Syndrome and DFNB4
Presentation ID: 300
Presenter: Domenica Karavitaki, PhD
Date and Time: Thursday, May 14, 2026, at 8:30am ET
Location: MCEC Room 205ABC (Level 2)
Poster Presentation
Title: Improvement of Hearing and Prevention of Inner Ear Pathology in a Severe SLC26A4 Mouse Model of Pendred Syndrome/DFNB4
Presentation ID: 1443
Presenter: André Landin-Malt, PhD
Location: MCEC Exhibit & Poster Hall (Halls B2-C, Exhibit Level)
About SKY‑PEN
Skylark’s SKY‑PEN gene therapy program is designed to address SLC26A4‑related hearing loss, the second most common genetic cause of severe‑to‑profound pediatric and young‑adult deafness that affects thousands of patients worldwide and for which no approved therapies exist. SKY‑PEN offers a first‑in‑class opportunity to improve hearing and meaningfully change long‑term outcomes for individuals with Pendred syndrome and related conditions. Built on Skylark’s proprietary capsid, SKY‑PEN is designed for precise, micro‑dose delivery with minimal systemic exposure and has demonstrated dose‑dependent hearing rescue across multiple mouse models in preclinical studies. The program is advancing toward IND‑enabling studies.
About Skylark Bio
Skylark Bio is a genetic‑medicine company developing precision, locally delivered gene therapies for monogenic diseases, with the potential to greatly improve patient outcomes and quality of life. The company’s lead GJB2 program is IND‑ready with first‑in‑human studies planned for mid-2026, followed by the Pendrin program targeting SLC26A4‑related hearing loss now advancing toward IND‑enabling studies. Built on a proprietary capsid engineered for broad transduction, Skylark’s platform enables targeted, micro‑dose delivery with cell‑specific expression control and the potential for durable benefit in non‑dividing cells of interest. The company is led by a team with deep expertise in gene therapy and supported by world‑class advisors as it expands its platform into additional hearing and CNS indications. For more information, visit Skylarkbio.com and follow us on Linkedin.
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