Skylark Bio, a clinical-stage biotechnology company developing precision genetic medicines for monogenic disorders, is expanding its pipeline with SKY-AS, a differentiated gene therapy candidate designed to restore UBE3A expression in individuals with Angelman syndrome (AS). SKY-AS represents the company’s first central nervous system (CNS) development program and expands Skylark’s growing portfolio of precision, locally delivered genetic medicines.

AS is a rare monogenic neurodevelopmental disorder caused by loss of UBE3A gene function in neurons. The disease is characterized by severe developmental and neurological impairments that impact cognition, motor function, communication, sleep and seizure control. Affecting approximately 1 in 15,000 people worldwide, including an estimated 20,000 individuals in the United States, AS 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.”

To support the development of SKY-AS and a broadening CNS pipeline, renowned neuroscientist and researcher Edwin J. Weeber, Ph.D., has joined Skylark Bio as Scientific Advisor. Dr. Weeber is a recognized leader in Angelman syndrome research and UBE3A biology. He previously served as Head of Research and Discovery at the Foundation for Angelman Syndrome Therapeutics (FAST) and held senior scientific and leadership positions, including Vice President of Neuroscience/Biology at PTC Therapeutics and Professor and Chief Scientific Officer of the USF Health Byrd Alzheimer’s Institute.

“Decades of research have established the central role of UBE3A in neuronal function and development,” said Edwin J. Weeber, Ph.D., Scientific Advisor to Skylark Bio. “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 Bio has also been selected to present during the Innovations Spotlight: Emerging Track at the 2026 Alliance for Regenerative Medicine (ARM) Meeting on the Mesa. The company plans to showcase its precision genetic medicine platform and pipeline, including the newly announced SKY-AS program.

Skylark Bio Company Presentation

Date:

  

Tuesday, October 6, 2026

Time:

  

2:45 PM MST

Location:

  

Arizona Biltmore, Phoenix, AZ – FLW Ballroom G

Presenter:

  

Jodi Cook, Skylark Bio CEO

About SKY-AS and Angelman Syndrome

Angelman syndrome is a rare monogenic neurodevelopmental disorder caused by loss of function of the UBE3A gene, leading to deficient UBE3A expression in neurons. UBE3A is essential for normal neuronal development, synaptic function, and plasticity, biological processes that play critical roles in learning, memory, motor control and communication.

Leveraging Skylark’s targeted delivery platform, SKY-AS is designed as a potential best-in-class, one-time therapy to restore UBE3A protein expression by precisely delivering a functional copy of the UBE3A gene to neurons. By addressing the underlying cause of Angelman syndrome, SKY-AS is intended to support neuronal function and key neurological processes affected by the disease. Preclinical data show SKY-AS restores synaptic communication machinery, driving complete and durable behavioral rescue across motor coordination and daily function in Angelman syndrome models. The program is advancing through investigational new drug (IND)-enabling studies.

About Skylark Bio

Skylark Bio is a clinical-stage genetic medicine company developing precision, locally delivered, single-administration gene therapies for monogenic diseases, with the potential to transform patient outcomes and quality of life. The company’s lead program, SKY-GJB2, is a potential first-in-class, one-time gene therapy being evaluated in SONIX, a Phase 1/2 clinical trial for GJB2-related hearing loss, the leading cause of inherited pediatric hearing loss worldwide. Skylark’s second program, SKY-AS, is designed to restore UBE3A expression for individuals with Angelman syndrome and is advancing through IND-enabling studies. Additional pipeline programs include SKY-PEN for SLC26A4-related hearing loss.

The company’s proprietary gene therapy platform combines an engineered capsid designed for broad transduction with targeted microdose delivery, cell-specific expression control, and the potential for durable therapeutic benefit. Led by a team with deep expertise in genetic medicine and supported by world-class advisors, Skylark is advancing a pipeline of innovative therapies for hearing loss, central nervous system (CNS) disorders and other genetic diseases that profoundly impact patients and families.

For more information, visit our newly launched website at Skylarkbio.com and follow us on LinkedIn.

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