FAST-funded project will apply directed evolution to develop new nucleoside analogs for RNA targeted technology designed to build on the progress already underway in Angelman syndrome research
AUSTIN, Texas, July 20, 2026 /PRNewswire/ -- Frances Arnold, PhD, winner of the 2018 Nobel Prize in Chemistry, has been awarded a two-year grant by the Foundation for Angelman Syndrome Therapeutics (FAST) to develop new molecular tools that could help advance future RNA-targeted therapeutics 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.
Angelman syndrome (AS) is a rare neurogenetic disorder caused by the loss of function of the maternally inherited UBE3A gene. It can cause significant developmental delays, motor challenges, seizures, sleep disruption, and limited or absent speech. Most individuals living with AS require lifelong support.
"Our biocatalysts have continued to push Nature's boundaries," said Dr. Arnold. "With directed evolution, we have achieved chemistry that no organism or chemist has ever accomplished, producing novel nucleoside analogs. It's exciting to demonstrate the translational utility of these new building blocks within RNA therapeutics for Angelman syndrome."
Nucleoside analogs are synthetic molecules designed to resist breakdown in the body, a property that may be useful in developing RNA therapeutics with improved durability. However, nucleoside analogs can be difficult and expensive to manufacture, which has limited their broader use in therapeutics. Dr. Arnold's lab will use directed evolution, along with machine learning, to engineer enzymes that can help produce nucleoside analogs more efficiently. The project will then incorporate those nucleoside analogs within RNA therapeutics to target the UBE3A-ATS in AS, generating new therapeutic candidates with improved physical properties.
The grant supports Pillar 2 of FAST's Angelman syndrome drug development pipeline, which focuses on developing therapies designed to restore expression of the silent paternal UBE3A gene. If promising candidates are identified, the next planned step would be to test lead candidates with collaborators in our AS research network in AS neuronal cell lines and animal models.
"FAST's research strategy is built around moving promising science toward the next critical decision point," said Dr. Allyson Berent, Chief Science Officer of FAST. "This grant brings one of the world's leading experts to a specific opportunity in the Angelman syndrome drug development pipeline: building novel RNA-targeted therapeutics with a long half-life and strong brain distribution may strengthen and extend the progress already being made in the field."
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.
"This is exactly the kind of investment FAST was built to make," said Alana Newhouse, President of FAST. "We are bringing world-class scientific expertise to Angelman syndrome, connecting novel work to the next stage of testing, and helping ensure promising ideas have a path forward. For families, that matters because progress depends not only on therapies in development today, but also on the tools that can make future therapies better."
The grant reflects FAST's broader research model: funding the tools, platforms, and translational studies needed to move Angelman syndrome research from scientific opportunity to therapeutic possibility.
About the Foundation for Angelman Syndrome Therapeutics (FAST)
FAST is the leading patient advocacy organization and the largest non-governmental funder of Angelman syndrome research in the world. Our goal is to drive forward transformative research and development programs as quickly as possible for those living with Angelman syndrome—regardless of age or genotype. Learn more at www.cureangelman.org.
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SOURCE AS2Bio
