The Bow Foundation will provide one-year grants to support research related to GNAO1 Neurodevelopmental Disorder. At least 2 awards will be granted at $100,000 each.

 Background

The Bow Foundation, in collaboration with the Orphan Disease Center, is seeking proposals to advance research related to GNAO1 Neurodevelopmental Disorder. We are looking for 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.

• 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.

At least 2 one-year grants for up to $100,000 each (total cost) will be awarded.

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, March 28, 2025 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.

JACK BEAR FOUNDATION 2025 GRANT PROGRAM

The Jack Bear Foundation, in collaboration with the Orphan Disease Center, will provide a 1-year grant to support several critical components of basic, clinical and translational research on disorders associated with genetic mutations in the RUBCN gene with a particular emphasis on the topics outlined below:

Scientific Topics

• Basic understanding of canonical and noncanonical functions of SCAR-15 disease associated with the RUBCN gene

• Identification of biomarkers for RUBCN gene/SCAR-15 disease using patient samples

• Development and characterization of patient-derived cell models of SCAR-15 disease (ex. iPSCs, neurons, reporter lines)

• Development and phenotypic characterization of animal models with the RUBCN gene/SCAR-15 disease

• Development of a standardized evaluation criteria for clinical projects allowing uniformity of patients as well as the severity and progression of the disease.

• Support small animal studies or assays that investigate drug repurposing strategies.

Please take special consideration to describe the proposed work and its potential impact on SCAR-15 disease in a language appropriate for non-scientific audiences.

Duration and Budget

• Expected duration is 12 months

• Up to 2 awards of up to $50,000 per grant / At least 1 award of up to $100,000

• Renewals could be considered at the end of the 1 year grant period

• IDCs of up to 10% should be included in the total award amount

Background

Spinocerebellar Ataxia Recessive Type 15 (SCAR-15) is a rare degenerative genetic disorder impacting the cerebellum, which is characterized by early-onset progressive loss of coordination of hands, gait, speech, eye movement, dysarthria, and developmental delay. Epilepsy and abnormal reflexes exist in a subset of affected individuals. SCAR-15 is inherited in an autosomal recessive manner and can be caused by homozygous or compound heterozygous mutations in the KIAA0226 (or RUBCN gene). Today there are only a handful cases of SCAR-15 reported in literature, and around a dozen cases in the world are known to the Foundation. However, given that the RUBCN gene was only recently upgraded to a causative disease gene in 2021, it is likely that this condition is underdiagnosed in the general population.

The Orphan Disease Center, in collaboration with the Jack Bear Foundation, is seeking grant applications for multidisciplinary teams of scientists 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 scientific topics above to further advance SCAR-15 research and therapeutic approaches.

Requirements and Eligibility

Data generated as a result of Jack Bear Foundation seed funding must be made accessible to the Foundation. Data can be embargoed for a period of time to be defined in a Data Use Agreement and/or Material Transfer Agreement to provide time to publish, protect intellectual property, etc. Resources generated as a result of Jack Bear Foundation funding (development of cell or animal models) must be made available to other interested researchers and the Jack Bear Foundation Biorepository.

We welcome applications from tenure-track faculty, non-tenure-track faculty and postdoctoral (PhD or MD) fellows; applications submitted by postdoctoral fellows must include a statement of feasibility and support from their faculty mentor. This opportunity is open to investigators at established academic and research institutions worldwide. We also welcome applications from individuals in a senior position at a non-profit institution or foundation. Collaboration with existing SCAR-15 researchers is encouraged but not required.

Letters of Interest (LOI) are due Friday, April 18, 2025 by 8pm ET.

To Apply:

1. Please review the JACK BEAR FOUNDATION 2025 GRANT PROGRAM RFA Guidelines.

2. Complete the Application Form on Submittable.

For any scientific inquiries regarding this grant please email Debbie Requesens

For any administrative inquiries regarding this grant please email psom-odcadmin@pobox.upenn.edu