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Current Pipeline
AAV-GT

SKY-AS

Skylark Bio is exploring an investigational Adeno-Associated Virus Gene Therapy (AAV-GT) approach to treating Angelman syndrome.

Pre-clinical

Discovery & Dev

Pre-clinical

Phase 1

Phase 2

Phase 3

To patients

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Disclaimer

This website contains information for a broad audience and may include information on current and upcoming programs that are not yet approved or accessible The information provided is for general informational purposes only and is not intended as medical advice, diagnosis, or treatment. While FAST strives to provide accurate and up-to-date information, the content on this site may not always reflect the most current research or clinical guidelines. The inclusion of clinical trial information, treatments or specific healthcare providers does not imply endorsement, recommendation or guarantee of safety, efficacy, or availability. Reliance on any information provided by this website is solely at your own risk. FAST disclaims any liability for any errors or omissions in the information provided or for any decisions made based on this information. For personalized medical advice or specific health concerns including participation in any clinical trial, please consult a qualified healthcare professional.

Therapeutic Approach

A therapeutic approach where a healthy copy of a virus, Adeno-Associated Virus (AAV), is used to carry a healthy copy of a gene to a target organ. This is most commonly delivered in-vivo (inside the body). For Angelman syndrome (AS) the healthy copy of the missing or non-functional UBE3A gene is packaged inside the AAV, and is injected into the fluid that surrounds the brain, called the cerebrospinal fluid (CSF). Once in the fluid, the virus and gene can directly reach important cells of the brain, called neurons, and replace the non-functional or missing copy of the gene.

Investigational Drug Name

SKY-AS

Recent Updates

  • September 2026

    Skylark Bio Expands Pipeline with First CNS Development Program, SKY-AS, Targeting UBE3A Restoration in Angelman Syndrome

    Read More

Disclaimer

This website contains information for a broad audience and may include information on current and upcoming programs that are not yet approved or accessible The information provided is for general informational purposes only and is not intended as medical advice, diagnosis, or treatment. While FAST strives to provide accurate and up-to-date information, the content on this site may not always reflect the most current research or clinical guidelines. The inclusion of clinical trial information, treatments or specific healthcare providers does not imply endorsement, recommendation or guarantee of safety, efficacy, or availability. Reliance on any information provided by this website is solely at your own risk. FAST disclaims any liability for any errors or omissions in the information provided or for any decisions made based on this information. For personalized medical advice or specific health concerns including participation in any clinical trial, please consult a qualified healthcare professional.