The Bow Foundation, will provide a 1-year grant to support research related to GNAO1 Neurodevelopmental Disorder. Up to 2 awards will be granted at $100,000 each.
Background
GNAO1 (G Protein Subunit Alpha O1) is a protein coding gene. Most patients with a GNAO1 neurodevelopmental disorder are diagnosed as infants or young children. Many of the patients begin experiencing seizures, abnormal movements and developmental delays in their infancy. The seizures are often refractory, and the patients seem to cycle through numerous medications as they try to find relief. The abnormal movements seem to worsen as the patients age. Several patients have benefited from deep brain stimulation surgery, including the youngest patient in the world to receive the procedure (a 2-year-old in the UK).
The Orphan Disease Center, in collaboration with the Bow Foundation, is seeking grant applications that aim 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.
Identification of short-term biomarkers that can monitor disease activity and treatment response.
Establishment of outcome measures for future clinical trials.
Development of therapeutic approaches in early symptomatic patients.
Supporting pilot clinical trials, preclinical trials, or animal model trials that promote drug repurposing strategies.
Development of a standardized evaluation criteria for clinical projects allowing uniformity of patients as well as the severity and progression of the disease.
Development of cellular models (i.e. oligodendrocytes) for evaluation of therapeutic options to translate for clinical use.
Eligibility
All individuals holding a faculty-level appointment at an academic institution or a senior position
at a non-profit institution or foundation are eligible to respond to this RFA.
Full Applications are Due Friday, April 14, 2023 by 8pm EST
To Apply:
Step 1: Please review the RFA Guidelines.
Step 2: Download and complete the below forms:
Step 3: Apply through Submittable
For any scientific inquiries regarding this grant please email Deborah Requesens.
For any administrative inquiries regarding this grant please email Leslie Silverman.