CAMBRIDGE, MASSACHUSETTS — Skylark Bio announced that it will present new preclinical data for SKY-PEN, its Pendrin gene therapy program targeting SLC26A4-related hearing loss, at the American Society of Gene & Cell Therapy (ASGCT) 2026 Annual Meeting.
According to the company, the data demonstrate dose-dependent hearing rescue across multiple mouse models, along with preservation of key inner-ear structures and targeted expression in cochlear cells in both mice and non-human primates.
“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 developing genetic medicines focused on inherited hearing loss conditions where no approved therapies currently exist. The company’s lead program, SKY-GJB2, is designed to address GJB2-related pediatric deafness, one of the most common causes of inherited non-syndromic hearing loss.
The SKY-PEN program targets SLC26A4-related hearing loss, including Pendred syndrome and DFNB4, which represent the second most common genetic cause of hearing loss.
ASGCT 2026 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 8:30 a.m. 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. According to the company, the therapy is built on a proprietary capsid platform designed for targeted micro-dose delivery with minimal systemic exposure.
Preclinical studies have demonstrated dose-dependent hearing rescue across multiple mouse models, and the program is currently 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.
Source: Skylark Bio
