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Current Pipeline
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di-siRNA

MGH

Mass General Hospital (MGH) is exploring an investigational di-siRNA (divalent small interfering RNA) approach to paternal gene activation as a potential treatment for Angelman syndrome.

Discovery & Dev

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 using small pieces of RNA, called divalent small interfering-RNA (di-siRNA) that are aimed to bind to the UBE3A-ATS and prevent the silencing of the paternal copy of the UBE3A gene, enabling UBE3A protein to be made.

Recent Updates

  • April 2026

    Career Development Awards Expand the Next Wave of Angelman Syndrome Research

    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.