Cambridge, Mass. — Skylark Bio, a clinical-stage biotechnology company developing precision genetic medicines for monogenic disorders, has expanded its pipeline with SKY-AS, a gene therapy candidate designed to restore UBE3A expression in people with Angelman syndrome.
SKY-AS is the company’s first central nervous system development program and broadens its portfolio of locally delivered genetic medicines beyond hearing loss.
Angelman syndrome is a rare neurodevelopmental disorder caused by loss of UBE3A gene function in neurons. It can cause severe impairments affecting cognition, motor function, communication, sleep and seizure control. The condition affects about 1 in 15,000 people worldwide, including an estimated 20,000 people in the United States, and currently has no approved disease-modifying therapies.
“The addition of SKY-AS marks an important evolution for Skylark as we expand beyond hearing loss into serious neurological disorders with substantial unmet need,” said Jodi Cook, Ph.D., Chief Executive Officer of Skylark Bio. “With a growing body of data, we believe SKY-AS represents a promising opportunity to address the underlying cause and change the treatment paradigm in Angelman syndrome, and we look forward to advancing the program toward an IND submission.”
Skylark also named Edwin J. Weeber, Ph.D., as Scientific Advisor to support development of SKY-AS and the company’s broader CNS pipeline.
Weeber is an Angelman syndrome and UBE3A biology researcher who previously served as Head of Research and Discovery at the Foundation for Angelman Syndrome Therapeutics. He has also held senior positions at PTC Therapeutics and the USF Health Byrd Alzheimer’s Institute.
“Decades of research have established the central role of UBE3A in neuronal function and development,” Weeber said. “SKY-AS is designed to deliver a functional copy of the UBE3A gene directly to neurons, increasing expression of the deficient protein, with the goal of supporting pathways involved in learning, memory, motor skills and communication. I am excited to join Skylark to advance the development of this innovative gene therapy for those living with Angelman syndrome.”
Skylark has also been selected to present during the Innovations Spotlight: Emerging Track at the 2026 Alliance for Regenerative Medicine Meeting on the Mesa. The company plans to discuss its precision genetic medicine platform and pipeline, including SKY-AS.
Cook is scheduled to present Oct. 6 at 2:45 p.m. MST at the Arizona Biltmore in Phoenix.
