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Current Pipeline
FAST Logo Funded
Nucleoside Analogs

Caltech

Caltech is exploring a new investigational approach to build nucleoside analogs as a potential highly durable RNA-targeted therapy 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

Nucleoside analogs are synthetic molecules with a chemically altered backbone that helps them to resist breakdown by enzymes that normally degrade RNA-like molecules. They could be used to target the UBE3A-ATS, unsilencing the paternal copy of the UBE3A gene and enabling UBE3A protein to be made.

Program Overview

Frances Arnold, PhD, winner of the 2018 Nobel Prize in Chemistry, has received FAST funding to develop new molecular tools that could help develop highly durable RNA-targeted therapies for Angelman syndrome.

The project will apply directed evolution, the Nobel Prize-winning approach pioneered by Dr. Arnold, to engineer enzymes capable of producing new nucleoside analogs with properties that may be useful to target the UBE3A antisense (UBE3A-ATS) transcript.

The grant is supported by Maddie’s Mission Foundation in partnership with the Shaw Family, an organization dedicated to transforming care for individuals living with Angelman syndrome and other rare neurological conditions.  

Recent Updates

  • July 2026

    Nobel Laureate Frances Arnold Brings Directed Evolution to 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.