RYR-1 Foundation: Individual Investigator Research Grant

Overview

Individual Investigator Research Grant Awards are designed to concentrate research in areas that will have the greatest potential to move towards treatments and cures for RYR-1-RD.

As appropriate, applicants are encouraged to use one or more of RYR-1-RD murine models currently available from commercial, corporate, academic, and other sources, including recently developed animal models. Recently developed murine models of RYR-1-RD demonstrating moderate-severe myopathy phenotypes, including mice with mutations on exons 34 and 96. Please see “Entry #1” and “Entry #2” on the spreadsheet entitled “Mouse Models of RYR-1-Related Diseases” at www.ryr1.org/mice.

The RYR-1 Foundation has identified Research Priority Areas (RPAs) that align with its mission; and the purpose of this targeted open call for applications is to address specific gaps in current research related to RYR-1-RD. While applications addressing the areas of particular interest (below) will be given priority consideration, The RYR-1 Foundation will also consider proposals for research that do not fit easily within these goals.

Eligibility

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Application Details

Individual Investigator Research Grant Awards
Request for Letter of Intent (LOI) for Research Grant
Application
Deadline: May 18, 2024
The RYR-1 Foundation expects to fund a limited number of Individual Investigator
Research Grants, to be awarded in early-2025. Preference will be given to LOI
grant applications that address the “Research Priority Areas.” Additional and detailed
information can be found below on pages two through four (2 - 4).
You are invited to submit a Letter of Intent (LOI) for our next round of research grant
applications to The RYR-1 Foundation by May 18, 2024. Please make sure your LOI is no
more than two (2) pages in length. You will be notified of your status by June 1, 2024. For
any questions please contact Patricia Raffaele at patricia@ryr1.org. The final deadline for
applications is October 19, 2024.
The following is required to be addressed in your LOI:
1. Title of the RYR-1-Related Diseases (RYR-1-RD) Project
2. Applicant/PI
3. Institution and Contacts
4. Key Personnel
5. Lay Abstract including: Research Priority Area(s) & Specific Aims
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DESCRIPTION:
Individual Investigator Research Grant Awards are designed to concentrate research in
areas that will have the greatest potential to move towards treatments and cures for
RYR-1-RD.
As appropriate, applicants are encouraged to use one or more of RYR-1-RD murine
models currently available from commercial, corporate, academic, and other sources,
including recently developed animal models. Recently developed murine models of
RYR-1-RD demonstrating moderate-severe myopathy phenotypes, including mice
with mutations on exons 34 and 96. Please see “Entry #1” and “Entry #2” on the
spreadsheet entitled “Mouse Models of RYR-1-Related Diseases” at
www.ryr1.org/mice. For information on obtaining these mice, please contact Patricia
Raffaele at patricia@ryr1.org.
The RYR-1 Foundation has identified Research Priority Areas (RPAs) that align with its
mission; and the purpose of this targeted open call for applications is to address specific
gaps in current research related to RYR-1-RD. While applications addressing the areas of
particular interest (below) will be given priority consideration, The RYR-1 Foundation will
also consider proposals for research that do not fit easily within these goals.
RESEARCH PRIORITY AREAS (RPAs):
Priority will be given to research that has direct application to clinical therapies
for RYR-1-RD. Specific areas of research interest may include the following:
A. Novel therapeutic approaches to RYR-1-RD. All approaches will be considered,
but potential interventions include:
i. Novel mechanisms to efficiently deliver therapeutic technologies (e.g.
gene editing, gene therapy, etc) to skeletal muscle throughout the entire
body. Given the size of the RYR1 gene, an emphasis should be placed on
novel vectors with increased cargo capacities (e.g. non-AAV viral vectors,
lipid nanoparticles, protein nanoparticles, antibodies, etc).
ii. Gene editing (e.g., CRISPR/Cas9, Base editing, Prime editing, etc.).
iii. Novel gene therapy strategies (e.g. split intein protein transplicing, etc.).
iv. Epigenetic modifying therapies.
v. mRNA-based protein replacement therapies
vi. t-RNA-based therapies.
vii. Development of therapeutic interventions that modify RyR1 receptor
activity, reduce muscle degradation, and/or increase regenerative capacity
of skeletal muscle.
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B. Cell and molecular mechanisms of RYR-1-RD and other muscle disease: Basic
research designed to define the underlying pathogenic mechanisms of RYR-1-RD and
possible common, “downstream” pathways that are also present in other muscle
diseases (e.g. calcium leak via the RyR1 receptor, ER stress, mitochondrial dysfunction,
post-translational modifications of myosin and other proteins associated with muscle
structure and function).
C. Regenerative Medicine (RM): Development of strategies that provide functional
rescue or replacement of degenerating and/or dead muscle cells that can lead to
improvement of muscle function.
LOI INSTRUCTIONS
KEY PERSONNEL
Each individual Investigator Research Grant Application must be directed by a single
Principal Investigator who is responsible for the conduct and management of the project.
Co-Investigators are allowed, and must be identified.
LAY ABSTRACT: RESEARCH PRIORITY AREA(S) & SPECIFIC AIMS
Provide an abstract of the proposed research project, written in lay terms for a non-
scientific audience.
RPAs: Identify specific RYR-1-RD research priority area(s) (see above for details).
SPECIFIC AIMS: 1) Describe the overall goal(s) for the proposed project. Numerically list
the specific aims. 2) For clinical research projects, identify the specific disease(s) and
patient population(s) to be studied. Include genomic diagnosis whenever possible.
ELIGIBILITY:
Applicants must hold a Ph.D., M.D., D.V.M., D.O., or equivalent degree and have a
faculty position/equivalent at a domestic/foreign nonprofit organization, or
public/private institution (such as a university, college, medical school, hospital,
research institute, or laboratory).
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AWARD:
The total award will be up to $120,000 USD over a period of two years (up to $60,000
USD per year). Exceptions may be made on a case-by-case basis. Research projects that
are expected to exceed two years in duration will also be considered for funding.
Note: The RYR-1 Foundation and its Directors do not provide funds for indirect,
administrative, or overhead costs. The RYR-1 Foundation does not provide funds for
fringe benefits and annual “cost of living” increases. In addition, The RYR-1 Foundation
does not pay for costs related to construction or renovation.
Purchase of capital equipment is generally not supported by The RYR-1 Foundation.
Capital equipment is defined as a permanent or semi-permanent apparatus, device, or
system costing more than $5,000 per device or system. Applicants must obtain prior
approval from the Chair/Co-Chair(s) of Research to submit an application to purchase
capital equipment.
If any of the investigators have either an active or pending grant on RYR-1-RD matters,
the list will need to be provided in the full application.
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