GNAO1 Neurodevelopmental Disorder Pilot Grant Program

This grant aims to further progress our understanding of the disease, the available therapeutic options, and investigating strategies to establish outcome measurements.

The RFA could focus on one, or several, of the following aims to further advance GNAO1 research and therapeutic approaches:

• Novel therapeutic approaches, including, but not limited to, techniques in genome editing, RNA-based mechanisms, biologics, novel cell-based therapeutics, and development of novel therapeutic compounds, including through small molecule repurposing or screening against validated phenotypes in human cellular systems.
• Proposals that include collaboration across organizations or other rare diseases.
• Establishment of outcome measures for future clinical trials.
• Supporting pilot clinical trials, preclinical trials, or animal model trials that promote drug repurposing strategies.
• Other aims are welcome and will be considered.

Funding: $100,000 total 

Duration: 1 year

Research Authority due date: 6.2.25

LOI (required) due date: 14.2.25

Full proposal due date: 28.2.25