FY20 DMDRP_IDA_PA_GG.pdf

This Program Announcement must be read in conjunction with the General Application Instructions, version 501. The General Application Instructions document is available for downloading from the Grants.gov funding opportunity announcement by selecting the “Package” tab, clicking “Preview,” and then selecting “Download Instructions.”

DoD FY20 Duchenne Muscular Dystrophy Idea Development Award 1

I. OVERVIEW OF THE FUNDING OPPORTUNITY

Program Announcement for the Department of Defense

Defense Health Program

Congressionally Directed Medical Research Programs

Duchenne Muscular Dystrophy Research Program

Idea Development Award

Announcement Type: Initial

Funding Opportunity Number: W81XWH-20-DMDRP-IDA

Catalog of Federal Domestic Assistance Number: 12.420 Military Medical Research and Development

SUBMISSION AND REVIEW DATES AND TIMES

• Pre-Application Submission Deadline: 5:00 p.m. Eastern time (ET), August 12, 2020

• Invitation to Submit an Application: September, 2020

• Application Submission Deadline: 11:59 p.m. ET, December 7, 2020

• End of Application Verification Period: 5:00 p.m. ET, December 11, 2020

• Peer Review: February 2021

• Programmatic Review: April 2021

DoD FY20 Duchenne Muscular Dystrophy Idea Development Award 2

TABLE OF CONTENTS

I. OVERVIEW OF THE FUNDING OPPORTUNITY

II. DETAILED INFORMATION ABOUT THE FUNDING OPPORTUNITY

II.A. Program Description

II.A.1. FY20 Idea Development Award Focus Areas

II.A.2. Award History

II.B. Award Information

II.C. Eligibility Information

II.C.1. Eligible Applicants

II.C.2. Cost Sharing

II.C.3. Other

II.D. Application and Submission Information

II.D.1. Address to Request Application Package

II.D.2. Content and Form of the Application Submission

II.D.3. Dun and Bradstreet Data Universal Numbering System (DUNS) Number and System for Award Management (SAM)

II.D.4. Submission Dates and Times

II.D.5. Funding Restrictions

II.D.6. Other Submission Requirements

II.E. Application Review Information

II.E.1. Criteria

II.E.2. Application Review and Selection Process

II.E.3. Integrity and Performance Information

II.E.4. Anticipated Announcement and Federal Award Dates

II.F. Federal Award Administration Information

II.F.1. Federal Award Notices

II.F.2. Administrative and National Policy Requirements

II.F.3. Reporting

II.G. Federal Awarding Agency Contacts

II.G.1. CDMRP Help Desk

II.G.2. Grants.gov Contact Center

II.H. Other Information

II.H.1. Program Announcement and General Application Instructions Versions

II.H.2. Administrative Actions

II.H.3. Application Submission Checklist

APPENDIX 1: ACRONYM LIST

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II. DETAILED INFORMATION ABOUT THE FUNDING

OPPORTUNITY

II.A. Program Description

Applications to the Fiscal Year 2020 (FY20) Duchenne Muscular Dystrophy Research Program (DMDRP)are being solicited for the Defense Health Agency (DHA) J9, Research and Development Directorate, by the U.S. Army Medical Research Acquisition Activity (USAMRAA) using delegated authority provided by United States Code, Title 10, Section 2358 (10 USC 2358). As directed by the Office of the Assistant Secretary of Defense for Health Affairs (OASD [HA]), the DHA manages the Defense Health Program (DHP) Research, Development, Test, and Evaluation (RDT&E) appropriation. The execution management agent for this Program Announcement is the Congressionally Directed Medical Research Programs (CDMRP). The DMDRP was initiated in 2011 to provide support for research of exceptional scientific merit and to promote the understanding, diagnosis, and treatment of Duchenne muscular dystrophy (DMD). Appropriations for the DMDRP from FY11 through FY19 totaled $29.6 million (M). The FY20 appropriation is $10M.

The vision of the FY20 DMDRP is to preserve and improve the function and quality of life and to extend the life span of all individuals with Duchenne. As such, the DMDRP is seeking to better support discovery and development of therapeutics, devices, and other interventions and to promote their rigorous clinical testing. Additionally, DMDRP supports the efforts of the National Institutes of Health (NIH) Muscular Dystrophy Coordinating Committee (MDCC) and the 2015 MDCC Action Plan for the Muscular Dystrophies, which prioritizes the needs to improve treatments and reduce the disease burden for muscular dystrophy, including Duchenne.

The proposed research must be relevant to active duty Service members, Veterans, military beneficiaries, and/or the American public.

II.A.1. FY20 Idea Development Award Focus Areas

All applications for the FY20 DMDRP Idea Development Award (IDA) must address opportunities and challenges in the development of safe and effective macromolecular and cellular therapies that address primary pathology of DMD. Eligible therapeutic strategies include: gene therapy, genome editing, oligonucleotide therapies, exon skipping, protein therapeutics, and cell therapies. Therapies that will be efficacious across the life-span, particularly in adolescents and adults are encouraged.

Studies proposed under this award may include:

• Delivery to skeletal muscle and heart (e.g., ligand assisted, nanoparticles, identification of biological barriers to delivery, and alternative vectors)

• Immunosuppression, vector modification, and other strategies to facilitate repeat administration of biologic therapies https://mdcc.nih.gov/Action_Plan

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• Targeting muscle stem cells

• Cell-based therapies, including but not limited to: selection of novel cell types, expansion, cell delivery and homing, differentiation, and integration

• Research that will inform and improve therapy in older individuals, including dosing challenges, genetic based therapies, tissue environments, and other factors that may compromise delivery and efficacy in this patient group

• Therapies addressing secondary pathologies of DMD to the extent that they augment therapies directed at primary disease mechanisms

Studies in the following research areas will not be supported by this award mechanism:

• Development of novel small molecule drugs

• Studies testing the efficacy of candidate therapeutics that are not fully characterized or lack a mechanistic rational

• Evaluation of vectors or delivery technologies already prevalent in research studies (e.g., AAV9)

Preliminary data to support the feasibility of the research hypotheses and research approaches are required; however, these data do not necessarily need to be derived from studies of muscular dystrophy.

II.A.2. Award History

The DMDRP Idea Development Award mechanism was first offered in FY19. Since then, 23 Idea Development Award applications have been received, and 5 have been recommended for funding.

II.B. Award Information

The DMDRP Idea Development Award supports the development of innovative, high-risk/high-reward research that could lead to critical discoveries or major advancements that will accelerate progress in improving outcomes for individuals with DMD. This award mechanism is designed to support innovative ideas with the potential to yield impactful data and new avenues of investigation. Preliminary data to support the feasibility of the research hypotheses and research approaches are required.

The types of awards made under the Program Announcement will be assistance agreements. An assistance agreement is appropriate when the Federal Government transfers a “thing of value” to a “state, local government,” or “other recipient” to carry out a public purpose of support or stimulation authorized by a law of the United States instead of acquiring property or service for the direct benefit and use of the U.S. Government. An assistance agreement can take the form of a grant or cooperative agreement. The level of involvement on the part of the Department of Defense (DoD) during project performance is the key factor in determining whether to award a

DoD FY20 Duchenne Muscular Dystrophy Idea Development Award 5 grant or cooperative agreement. If “no substantial involvement” on the part of the funding agency is anticipated, a grant award will be made (31 USC 6304). Conversely, if substantial involvement on the part of the funding agency is anticipated, a cooperative agreement will be made (31 USC 6305), and the award will identify the specific substantial involvement.

Substantial involvement may include, but is not limited to, collaboration, participation, or intervention in the research to be performed under the award. The award type, along with the start date, will be determined during the negotiation process.

The anticipated direct costs budgeted for the entire period of performance for an FY20 DMDRP Idea Development Award will not exceed $350,000. Refer to Section II.D.5, Funding Restrictions, for detailed funding information.

Awards will be made no later than September 30, 2021. For additional information refer to Section II.F.1, Federal Award Notices.

The CDMRP expects to allot approximately $4.48M to fund approximately 8 Idea Development Award applications. Funding of applications received is contingent upon the availability of Federal funds for this program as well as the number of applications received, the quality and merit of the applications as evaluated by scientific and programmatic review, and the requirements of the Government. Funds to be obligated on any award resulting from this funding opportunity will be available for use for a limited time period based on the fiscal year of the funds. It is anticipated that awards made from this FY20 funding opportunity will be funded with FY20 funds, which will expire for use on September 30, 2026.

Research Involving Human Anatomical Substances, Human Subjects, or Human Cadavers: All DoD-funded research involving new and ongoing research with human anatomical substances, human subjects, or human cadavers must be reviewed and approved by the U.S. Army Medical Research and Development Command (USAMRDC) Office of Research Protections (ORP), Human Research Protection Office (HRPO), prior to research implementation. This administrative review requirement is in addition to the local Institutional Review Board (IRB) or Ethics Committee (EC) review. Local IRB/EC approval at the time of submission is not required. Allow a minimum of 2 to 3 months for HRPO regulatory review and approval processes. Refer to the General Application Instructions, Appendix 1, and the Human Subject Resource Document available on the electronic Biomedical Research Application Portal (eBRAP) “Funding Opportunities & Forms” web page (https://ebrap.org/eBRAP/public/Program.htm) for additional information. If the proposed research is cooperative (i.e., involving more than one institution), a written plan for single IRB review arrangements must be provided at the time of application submission or award negotiation. The lead institution responsible for developing the master protocol and master consent form should be identified and should be the single point of contact for regulatory submissions and requirements.

Clinical trials are not allowed. A clinical trial is defined as a research study in which one or more human subjects are prospectively assigned to one or more interventions (which may include placebo or other control) to evaluate the effects of the interventions on biomedical or behavioral health-related outcomes.

https://ebrap.org/eBRAP/public/Program.htm

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Guidelines for Animal Research: All projects should adhere to a core set of standards for rigorous study design and reporting to maximize the reproducibility and translational potential of preclinical research. The standards are described in Landis, S.C., et al. A call for transparent reporting to optimize the predictive value of preclinical research, Nature 2012, 490:187-191 (http://www.nature.com/nature/journal/v490/n7419/full/nature11556.html). Applicants should consult the ARRIVE (Animal Research: Reporting In Vivo Experiments) guidelines to ensure relevant aspects of rigorous animal research are adequately planned for and, ultimately, reported.

The ARRIVE guidelines can be found at https://journals.plos.org/plosone/article/ file?type=supplementary&id=info:doi/10.1371/journal.pone.0146533.s001.

Research Involving Animals: All DoD-funded research involving new and ongoing research with animals must be reviewed and approved by the USAMRDC ORP Animal Care and Use Review Office (ACURO), in addition to the local Institutional Animal Care and Use Committee (IACUC) of record. IACUC approval at the time of submission is not required. Allow at least 3 to 4 months for ACURO regulatory review and approval processes for animal studies. Refer to the General Application Instructions, Appendix 1, for additional information.

II.C. Eligibility Information

II.C.1. Eligible Applicants

II.C.1.a. Organization: All organizations, including international organizations, are eligible to apply.

Government Agencies Within the United States: Local, state, and Federal Government agencies are eligible to the extent that applications do not overlap with their fully funded internal programs. Such agencies are required to explain how their applications do not overlap with their internal programs.

As applications for this Program Announcement may be submitted by extramural and intramural organizations, these terms are defined below.

Extramural Organization: An eligible non-DoD organization. Examples of extramural organizations include academic institutions, biotechnology companies, foundations, Federal Government organization other than the DoD, and research institutes.

Intramural DoD Organization: A DoD laboratory, DoD military treatment facility, and/or DoD activity embedded within a civilian medical center. Intramural Submission: Application submitted by a DoD organization for an intramural investigator working within a DoD laboratory or military treatment facility or in a DoD activity embedded within a civilian medical center.

USAMRAA makes awards to eligible organizations, not to individuals.

http://www.nature.com/nature/journal/v490/n7419/full/nature11556.html https://journals.plos.org/plosone/article/file?type=supplementary&id=info:doi/10.1371/journal.pone.0146533.s001 https://journals.plos.org/plosone/article/file?type=supplementary&id=info:doi/10.1371/journal.pone.0146533.s001

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II.C.1.b. Principal Investigator

Independent investigators at all academic levels (or equivalent) are eligible to be named as a Principal Investigator (PI).

An eligible PI, regardless of ethnicity, nationality, or citizenship status, must be employed by, or affiliated with, an eligible organization.

The CDMRP encourages all PIs to participate in a digital identifier initiative through Open Researcher and Contributor ID, Inc. (ORCID). Registration for a unique ORCID identifier can be done online at https://orcid.org/.

II.C.2. Cost Sharing

Cost sharing/matching is not an eligibility requirement.

II.C.3. Other

Organizations must be able to access .gov and .mil websites in order to fulfill the financial and technical deliverable requirements of the award and submit invoices for payment.

For general information on required qualifications for award recipients, refer to the General Application Instructions, Appendix 3.

Refer to Section II.H.2, Administrative Actions, for a list of administrative actions that may be taken if a pre-application or application does not meet the administrative, eligibility, or ethical requirements defined in this Program Announcement.

II.D. Application and Submission Information

Submission of applications that are essentially identical or propose essentially the same research project to different funding opportunities within the same program and fiscal year is prohibited and will result in administrative withdrawal of the duplicative application(s).

Extramural Submission:

• Pre-application content and forms must be accessed and submitted at eBRAP.org.

• Full application packages must be accessed and submitted at Grants.gov.

Intramural DoD Submission:

• Pre-application content and forms must be accessed and submitted at eBRAP.org.

• Full application packages must be accessed and submitted at eBRAP.org.

Note: Applications from an intramural DoD organization or from an extramural Federal Government organization may be submitted to Grants.gov through a research foundation.

https://orcid.org/ https://ebrap.org/ https://ebrap.org/

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II.D.1. Address to Request Application Package eBRAP is a multifunctional web-based system that allows PIs to submit their pre-applications electronically through a secure connection, to view and edit the content of their pre-applications and full applications, to receive communications from the CDMRP, and to submit documentation during award negotiations and period of performance.

Contact information for the CDMRP Help Desk and the Grants.gov Contact Center can be found in Section II.G, Federal Awarding Agency Contacts.

II.D.2. Content and Form of the Application Submission

Submission is a two-step process requiring both pre-application (eBRAP.org) and full application (eBRAP.org or Grants.gov) as indicated below. The submission process should be started early to avoid missing deadlines. There are no grace periods. Full application submission guidelines differ for extramural (Grants.gov) and intramural (eBRAP.org) organizations (refer to Table 1. Full Application Guidelines).

The application title, eBRAP log number, and all information for the PI, Business Official(s), performing organization, and contracting organization must be consistent throughout the entire pre-application and full application submission process. Inconsistencies may delay application processing and limit or negate the ability to view, modify, and verify the application in eBRAP. If any changes need to be made, the applicant should contact the CDMRP Help Desk at help@eBRAP.org or 301-682-5507 prior to the application submission deadline.

II.D.2.a. Step 1: Pre-Application Submission Content

During the pre-application process, eBRAP assigns each submission a unique log number.

This unique eBRAP log number is required during the full application submission process.

To begin the pre-application process, first select whether the submitting organization is extramural or intramural, then confirm your selection or cancel. Incorrect selection of extramural or intramural submission type will delay processing.

If an error has been made in the selection of extramural versus intramural and the pre-application submission deadline has passed, the PI or Business Official must contact the CDMRP Help Desk at help@eBRAP.org or 301-682-5507 to request a change in designation.

All pre-application components must be submitted by the PI through eBRAP (https://eBRAP.org/). Because the invitation to submit an application is based on the contents of the pre-application, investigators should not change the title or research objectives after the pre-application is submitted.

The applicant organization and associated PI identified in the pre-application should be the same as those intended for the subsequent application submission. If any changes are necessary after submission of the pre-application, the applicant must contact the CDMRP Help Desk at help@eBRAP.org or 301-682-5507.

mailto:help@eBRAP.org mailto:help@eBRAP.org mailto:help@eBRAP.org

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PIs with an ORCID identifier should enter that information in the appropriate field in the “My Profile” tab in the “Account Information” section of eBRAP.

The pre-application consists of the following components, which are organized in eBRAP by separate tabs (refer to the General Application Instructions, Section II.B, for additional information on pre-application submission):

• Tab 1 – Application Information

Submission of application information includes assignment of primary and secondary research classification codes, which may be found at https://ebrap.org/eBRAP/public/ Program.htm. Applicants are strongly encouraged to review and confirm the codes prior to making their selection.

• Tab 2 – Application Contacts

Enter contact information for the PI. Enter the organization’s Business Official responsible for sponsored program administration (the “person to be contacted on matters involving this application” in Block 5 of the Grants.gov SF424 Research & Related Form). The Business Official must be either selected from the eBRAP list or invited in order for the pre-application to be submitted.

Select the performing organization (site at which the PI will perform the proposed work) and the contracting organization (organization submitting on behalf of the PI, which corresponds to Block 5 on the Grants.gov SF424 Research & Related Form), and click on “Add Organizations to this Pre-application.” The organization(s) must be either selected from the eBRAP drop-down list or invited in order for the pre-application to be submitted.

It is recommended that PIs identify an Alternate Submitter in the event that assistance with pre-application submission is needed.

• Tab 3 – Collaborators and Key Personnel

Enter the name, organization, and role of all collaborators and key personnel associated with the application.

FY20 DMDRP Programmatic Panel members should not be involved in any pre-application or application. For questions related to panel members and pre-applications or applications, refer to Section II.H.2.c, Withdrawal, or contact the CDMRP Help Desk at help@eBRAP.org or 301-682-5507.

• Tab 4 – Conflicts of Interest

List all individuals other than collaborators and key personnel who may have a conflict of interest in the review of the application (including those with whom the PI has a personal or professional relationship).

https://ebrap.org/eBRAP/public/Program.htm https://ebrap.org/eBRAP/public/Program.htm http://cdmrp.army.mil/dmdrp/panels/panels20

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• Tab 5 – Pre-Application Files

Note: Upload documents as individual PDF files unless otherwise noted. eBRAP will not allow a file to be uploaded if the number of pages exceeds the limit specified below.

○ Preproposal Narrative (two-page limit): The Preproposal Narrative page limit applies to text and non-text elements (e.g., figures, tables, graphs, photographs, diagrams, chemical structures, drawings) used to describe the project. Inclusion of URLs that provide additional information to expand the Preproposal Narrative and could confer an unfair competitive advantage is prohibited and may result in administrative withdrawal of the pre-application.

The Preproposal Narrative should include the following:

– Research Strategy and Feasibility: State the project’s hypothesis(es)/objective(s), rationale, specific aims, and study design. This award cannot be used to conduct clinical trials.

– Innovation: Describe how the project may introduce a new paradigm, challenge current paradigms, introduce novel concepts or agents, or exhibit other uniquely creative qualities.

– Impact: Describe how the proposed project is expected to make an important and original contribution to advancing the development of safe and effective macromolecular and cellular therapies that address primary pathology of DMD and that can ultimately lead to improved outcomes for individuals with DMD.

○ Pre-Application Supporting Documentation: The items to be included as supporting documentation for the pre-application must be uploaded as individual files and are limited to the following:

– References Cited (one-page limit): List the references cited (including URLs if available) in the Preproposal Narrative using a standard reference format that includes the full citation (i.e., author[s], year published, reference title, and reference source, including volume, chapter, page numbers, and publisher, as appropriate).

– List of Abbreviations, Acronyms, and Symbols: Provide a list of abbreviations, acronyms, and symbols used in the Preproposal Narrative.

– Key Personnel Biographical Sketches (five-page limit per individual). All biographical sketches should be uploaded as a single combined file. Biographical sketches should be used to demonstrate background and expertise through education, positions, publications, and previous work accomplished.

• Tab 6 – Submit Pre-Application

This tab must be completed for the pre-application to be accepted and processed.

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Pre-Application Screening

• Pre-Application Screening Criteria

To determine the technical merits of the pre-application and the relevance to the mission of the DHP and the DMDRP, pre-applications will be screened based on the following criteria:

• Research Strategy and Feasibility: The degree to which the study design for accomplishing the specific aims is feasible and addresses the hypothesis or objective.

• Innovation: How well the project introduces a new paradigm, challenges current paradigms, introduces novel concepts or agents, or exhibits other uniquely creative qualities.

• Impact: Whether the proposed project has the potential to develop safe and effective macromolecular and cellular therapies that address primary pathology of DMD and can ultimately lead to improved outcomes for individuals with DMD.

• Notification of Pre-Application Screening Results

Following the pre-application screening, PIs will be notified as to whether or not they are invited to submit applications; however, they will not receive feedback (e.g., a critique of strengths and weaknesses) on their pre-application. The estimated timeframe for notification of invitation to submit an application is indicated in Section I, Overview of the Funding Opportunity. Invitations to submit a full application are based on the Pre-Application Screening Criteria listed above.

II.D.2.b. Step 2: Full Application Submission Content

Applications will not be accepted unless notification of invitation has been received.

The CDMRP cannot make allowances/exceptions to its policies for submission problems encountered by the applicant organization using system-to-system interfaces with Grants.gov.

Each application submission must include the completed full application package for this Program Announcement. The full application package is submitted by the Authorized Organizational Representative through Grants.gov (https://www.grants.gov/) for extramural organizations or through eBRAP (https://ebrap.org/) for intramural organizations. See Table 1 below for more specific guidelines.

II.D.2.b.i. Full Application Guidelines

Extramural organizations must submit full applications through Grants.gov. Applicants must create a Grants.gov Workspace for submission, which allows the application components to be completed online and routed through the applicant organization for review prior to submission.

Applicants may choose to download and save individual PDF forms rather than filling out webforms in Workspace. A compatible version of Adobe Reader must be used to view, complete, and submit an application package consisting of PDF forms. If more than one person https://www.grants.gov/

DoD FY20 Duchenne Muscular Dystrophy Idea Development Award 12 is entering text into an application package, the same version of Adobe Reader software should be used by each person. Check the version number of the Adobe software on each user’s computer to make sure the versions match. Using different versions of Adobe Reader may cause submission and/or save errors – even if each version is individually compatible with Grants.gov.

Refer to the General Application Instructions, Section III, and the “Apply For Grants” page of Grants.gov (https://www.grants.gov/web/grants/applicants/apply-for-grants.html) for further information about the Grants.gov Workspace submission process. Submissions of extramural applications through eBRAP may be withdrawn.

Do not password protect any files of the application package, including the Project Narrative.

Table 1. Full Application Submission Guidelines

Extramural Submissions Intramural DoD Submissions

Application Package Location Download application package components for W81XWH-20-DMDRP-IDA from Grants.gov (https://www.grants.gov) and create a Grants.gov Workspace. Workspace allows online completion of the application components and routing of the application package through the applicant organization for review prior to submission.

Download application package components for W81XWH-20-DMDRP-IDAfrom eBRAP (https://ebrap.org).

Full Application Package Components SF424 Research & Related Application for Federal Assistance Form: Refer to the General Application Instructions, Section III.A.1, for detailed information.

Tab 1 – Summary: Provide a summary of the application information.

Tab 2 – Application Contacts: This tab will be pre-populated by eBRAP; add Authorized Organizational Representative.

Descriptions of each required file can be found under Full Application Submission Components:

• Attachments

• Research & Related Personal Data

• Research & Related Senior/Key Person

Profile (Expanded)

• Research & Related Budget

• Project/Performance Site Location(s) Form

• Research & Related Subaward Budget

Attachment(s) Form (if applicable)

Tab 3 – Full Application Files: Upload files under each Application Component in eBRAP.

Descriptions of each required file can be found under Full Application Submission Components:

• Attachments

• Key Personnel

• Budget

• Performance Sites

Tab 4 – Application and Budget Data: Review and edit proposed project start date, proposed end date, and budget data pre-populated from the Budget Form.

https://www.grants.gov/web/grants/applicants/apply-for-grants.html https://www.grants.gov/

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Extramural Submissions Intramural DoD Submissions

Application Package Submission Create a Grants.gov Workspace.

Add participants (investigators and Business Officials) to Workspace, complete all required forms, and check for errors before submission.

Submit a Grants.gov Workspace Package.

An application may be submitted through Workspace by clicking the “Sign and Submit” button on the “Manage Workspace” page, under the “Forms” tab. Grants.gov recommends submission of the application package at least 24-48 hours prior to the close date to allow time to correct any potential technical issues that may disrupt the application submission.

Note: If either the Project Narrative or the budget fails eBRAP validation or if the Project Narrative or the budget needs to be modified, an updated Grants.gov application package must be submitted via Grants.gov as a “Changed/Corrected Application” with the previous Grants.gov Tracking ID prior to the application submission deadline. Do not password protect any files of the application package, including the Project Narrative.

Submit package components to eBRAP (https://ebrap.org).

Tab 5 – Submit/Request Approval Full Application: After all components are uploaded and prior to the full application submission deadline, enter your password in the space provided next to “Enter Your Password Here” and press the “Submit Full Application” button.

eBRAP will notify your Resource Manager/ Comptroller/Task Area Manager or equivalent Business Official by email. Do not password protect any files of the application package, including the Project Narrative.

Application Verification Period The full application package submitted to Grants.gov may be viewed and modified in eBRAP until the end of the application verification period.

During the application verification period, the full application package may be modified with the exception of the Project Narrative and Research & Related Budget Form.

After eBRAP has processed the full application, the organizational Resource Manager/Comptroller/ Task Area Manager or equivalent Business Official and PI will receive email notification of this status and will be able to view and modify application components in eBRAP. During the application verification period, the full application package may be modified with the exception of the Project Narrative and Research & Related Budget Form. Your Resource Manager/ Comptroller/Task Area Manager or equivalent Business Official should log into eBRAP to review and to approve prior to the application verification deadline.

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Extramural Submissions Intramural DoD Submissions

Further Information Tracking a Grants.gov Workspace Package.

After successfully submitting a Workspace package, a Grants.gov Tracking Number is automatically assigned to the package. The number will be listed on the “Confirmation” page that is generated after submission.

Refer to the General Application Instructions, Section III, for further information regarding Grants.gov requirements.

Refer to the General Application Instructions, Section IV, for further information regarding eBRAP requirements.

The full application package must be submitted using the unique eBRAP log number to avoid delays in application processing.

II.D.2.b.ii. Full Application Submission Components

• Extramural Applications Only

SF424 Research & Related Application for Federal Assistance Form: Refer to the General Application Instructions, Section III.A.1, for detailed information.

• Extramural and Intramural Applications

Attachments:

Each attachment to the full application components must be uploaded as an individual file in the format specified and in accordance with the formatting guidelines listed in the General Application Instructions, Appendix 4.

For all attachments, ensure that the file names are consistent with the guidance. Attachments will be rejected if the file names are longer than 50 characters or have incorrect file names that contain characters other than the following: A-Z, a-z, 0-9, underscore, hyphen, space, and period. In addition, there are file size limits that may apply in some circumstances.

Individual attachments may not exceed 20 MB, and the file size for the entire full application package may not exceed 200 MB. It is important to include the attachment name as a header on each page of the attachment files.

○ Attachment 1: Project Narrative (10-page limit): Upload as “ProjectNarrative.pdf”. The page limit of the Project Narrative applies to text and non-text elements (e.g., figures, tables, graphs, photographs, diagrams, chemical structures, drawings) used to describe the project. Inclusion of URLs that provide additional information to expand the Project Narrative and could confer an unfair competitive advantage is prohibited and may result in administrative withdrawal of the application.

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– Background: Present the ideas and reasoning behind the proposed research and include relevant literature citations. Describe and show the preliminary data to justify the rationale for the proposed project and its feasibility.

– Hypothesis or Objective: State the hypothesis to be tested or the objective to be reached.

– Specific Aims: Concisely explain the project’s specific aims to be supported by this application. If the proposed research is correlated with a larger study, present only tasks that this award would fund.

– Research Strategy: Describe the experimental design, methods, and analyses including appropriate controls, in sufficient detail for assessment. Address potential limitations and present alternative methods and approaches. If animal studies are proposed, describe how they will be conducted in accordance with the ARRIVE guidelines (https://www.elsevier.com/__data/promis_misc/622936arrive_ guidelines.pdf) to achieve reproducible and rigorous results. If human subjects or human biological samples will be used, include a detailed plan for the recruitment of subjects or the acquisition of samples. This award cannot be used to conduct clinical trials.

– Data Collection and Statistical Analysis Plan: Describe how data will be collected and analyzed in a manner that is consistent with the study objectives. Detail a statistical analysis plan for the resulting outcomes. If applicable, include a complete power analysis to demonstrate the sample size is appropriate to meet the objectives of the study. Describe how data will be reported and how it will be assured that the documentation will support a regulatory filing with the U.S. Food and Drug Administration (FDA), if applicable.

○ Attachment 2: Supporting Documentation: Combine and upload as a single file named “Support.pdf”. Start each document on a new page. If documents are scanned to PDF, the lowest resolution (100 to 150 dpi) should be used. The Supporting Documentation attachment should not include additional information such as figures, tables, graphs, photographs, diagrams, chemical structures, or drawings. These items should be included in the Project Narrative.

There are no page limits for any of these components unless otherwise noted. Include only those components described below; inclusion of items not requested or viewed as an extension of the Project Narrative will result in the removal of those items or may result in administrative withdrawal of the application.

References Cited: List the references cited (including URLs, if available) in the Project Narrative using a standard reference format that includes the full citation (i.e., author[s], year published, title of reference, source of reference, volume, chapter, page numbers, and publisher, as appropriate).

List of Abbreviations, Acronyms, and Symbols: Provide a list of abbreviations, acronyms, and symbols.

https://www.elsevier.com/__data/promis_misc/622936arrive_guidelines.pdf https://www.elsevier.com/__data/promis_misc/622936arrive_guidelines.pdf

DoD FY20 Duchenne Muscular Dystrophy Idea Development Award 16

Facilities, Existing Equipment, and Other Resources: Describe the facilities and equipment available for performance of the proposed project and any additional facilities or equipment proposed for acquisition at no cost to the award. Indicate whether or not Government-furnished facilities or equipment are proposed for use.

If so, reference should be made to the original or present Government award under which the facilities or equipment items are now accountable. There is no form for this information.

Publications and/or Patents: Include a list of relevant publication URLs and/or patent abstracts. If articles are not publicly available, then copies of up to five published manuscripts may be included in Attachment 2. Extra items will not be reviewed.

Letters of Organizational Support: Provide a letter (or letters, if applicable) signed by the Department Chair or appropriate organization official, confirming the laboratory space, equipment, and other resources available for the project. Letters of support not requested in the Program Announcement, such as those from members of Congress, do not impact application review or funding decisions.

Letters of Collaboration (if applicable): Provide a signed letter from each collaborating individual or organization that will demonstrate that the PI has the support or resources necessary for the proposed work. If an investigator at an intramural organization is named as a collaborator on an application submitted through an extramural organization, the application must include a letter from the collaborator’s Commander or Commanding Officer at the intramural organization that authorizes the collaborator’s involvement.

Availability of and access to research resources (to include proprietary material for the purpose/duration of the proposed research), and/or

Availability of and access to appropriate populations (and/or access to available samples/data or databases), if applicable.

Intellectual Property: Information can be found in Code of Federal Regulations, Title 2, Part 200.315 (2 CFR 200.315), “Intangible Property.”

Intellectual and Material Property Plan (if applicable): Provide a plan for resolving intellectual and material property issues among participating organizations.

Commercialization Strategy (if applicable): Describe the commercialization plan.

The plan should include intellectual property, market size, financial analysis, strengths and weaknesses, barriers to the market, competitors, and management team. Discuss the significance of this development effort, when it can be anticipated, and the potential commercial use for the technology being developed.

○ Attachment 3: Technical Abstract (one-page limit): Upload as “TechAbs.pdf”. The technical abstract is used by all reviewers. Abstracts of all funded research projects will be posted publicly. Do not include proprietary or confidential information. Use only

DoD FY20 Duchenne Muscular Dystrophy Idea Development Award 17 characters available on a standard QWERTY keyboard. Spell out all Greek letters, other non-English letters, and symbols. Graphics are not allowed.

Of particular importance, programmatic reviewers typically do not have access to the full application and therefore rely on the technical abstract for appropriate description of the project’s key aspects. Therefore, clarity and completeness within the space limits of the technical abstract are highly important. Technical abstracts should be written using the outline below.

– Background: Present the ideas and reasoning behind the proposed work.

– Objective/Hypothesis: State the objective to be reached/hypothesis to be tested.

Provide evidence or rationale that supports the objective/hypothesis.

– Specific Aims: State the specific aims of the study.

– Study Design: Briefly describe the study design, including appropriate controls.

– Innovation: Briefly describe how the proposed project is innovative.

– Impact: Summarize the potential impact of the proposed project toward the development of safe and effective macromolecular and cellular therapies for DMD.

○ Attachment 4: Lay Abstract (one-page limit): Upload as “LayAbs.pdf”. The lay abstract is used by all reviewers. Abstracts of all funded research projects will be posted publicly. Do not include proprietary or confidential information. Use only characters available on a standard QWERTY keyboard. Spell out all Greek letters, other non- English letters, and symbols. Graphics are not allowed.

Do not duplicate the technical abstract. Minimize the use of acronyms and abbreviations, where appropriate. The lay abstract is an important component of the application review process, because it addresses issues of particular interest to the consumer community.

Lay abstracts should be written using the outline below.

– Describe the scientific objective and rationale for the proposed project in a manner that will be readily understood by readers without a background in science or medicine.

– Describe the ultimate applicability of the research.

What types of patients will it help, and how will it help them?

What are the potential clinical applications, benefits, and risks? If the research is too basic for clinical applicability, describe the interim outcomes expected and their applicability to the field.

What is the projected time anticipated to achieve a clinically relevant outcome?

DoD FY20 Duchenne Muscular Dystrophy Idea Development Award 18

What are the likely contributions of this study to advancing the field of DMD research?

○ Attachment 5: Statement of Work (three-page limit): Upload as “SOW.pdf”. The suggested SOW format and examples specific to different types of research projects are available on the eBRAP “Funding Opportunities & Forms” web page (https://ebrap.org/eBRAP/public/Program.htm). For the Idea Development Award mechanism, use the SOW format example titled, “SOW Generic Format.” The SOW must be in PDF format prior to attaching.

The SOW should include a list of major tasks that support the proposed specific aims, followed by a series of subtasks outlined related to the major tasks and milestones within the period of performance. The SOW should describe only the work for which funding is being requested by this application and, as applicable, should also:

– Include the name(s) of the key personnel and contact information for each study site/ subaward site.

– Indicate the number (and type, if applicable) of research subjects (animal or human) and/or human anatomical samples projected or required for each task and at each site.

Refer to the General Application Instructions, Appendix 1, for additional information regarding regulatory requirements.

– For studies with prospective accrual of human subjects, indicate quarterly enrollment targets.

– Identify cell line(s) and commercial or organizational source(s) to be used.

– If applicable, indicate timelines required for regulatory approvals relevant to human subjects research by the FDA or other Government agency.

○ Attachment 6: Impact Statement (one-page limit): Upload as “Impact.pdf”. The Impact Statement should be written in plain language for lay persons. Describe how the proposed project is expected to make an important and original contribution to addressing opportunities and challenges in the development of safe and effective macromolecular and cellular therapies that address primary pathology of DMD. Detail the anticipated outcome(s)/product(s) (intellectual and/or tangible) that will be directly attributed to the results of the proposed research project (short-term impact). Compare the anticipated outcomes from the proposed project to DMD information/products currently available, if applicable. Describe the short-term and long-term impact of the expected results of the study on the field and on the outcomes of individuals with DMD and the well-being of their families/caregivers.

○ Attachment 7: Innovation Statement (one-page limit): Upload as “Innovation.pdf”.

Describe how the proposed work is innovative. Research deemed innovative may introduce a new paradigm, challenge current paradigms, look at existing problems from new perspectives, or exhibit other uniquely creative qualities in one or more of the following ways: concept or question, research methods or technologies, adaptations of

DoD FY20 Duchenne Muscular Dystrophy Idea Development Award 19 existing methods or technologies, or other ways. Describe how the proposed research represents more than an incremental advance beyond ongoing research and published data.

○ Attachment 8: Data and Research Resources Sharing Plan (one-page limit):

Upload as “Sharing.pdf”. Describe how data and resources generated during the performance of the project will be shared with the research community. State whether the proposed plan for data sharing includes databases most relevant to DMD and whether the plan describes organizational and technical capabilities sufficient to share project data in a timely manner. Refer to the General Application Instructions, Appendix 2, Section K, for more information about the CDMRP expectations for making data and research resources publicly available. Include plans for making raw data publicly available in appropriate databases (e.g., dbGaP, GEO) at the time of publication or at the time of conclusion of the funding. The Government reserves the right to identify repositories for submission of data for archive. Any costs associated with submission will be addressed during award negotiations.

○ Attachment 9: Animal Research Plan (three-page limit), if applicable: Upload as “AnimalPlan.pdf”. When the proposed study involves animals, the applicant is required to submit a summary describing the animal research that will be conducted. Applicants should not submit a verbatim replica of the protocol(s) to be submitted to the IACUC as the Animal Research Plan. The Animal Research Plan should address the following points for each proposed animal study:

– Briefly describe the research objective(s) of the animal study. Explain how and why the animal species, strain, sex, and model(s) being used can address the scientific objectives and, where appropriate, the study’s relevance to human biology.

– Summarize the procedures to be conducted. Describe how the study will be controlled.

– Describe the randomization and blinding procedures for the study, and any other measures to be taken to minimize the effects of subjective bias during animal treatment and assessment of results. If randomization and/or blinding will not be utilized, provide justification.

– Provide a sample size estimate for each study arm and the method by which it was derived, including power analysis calculations.

– Describe how data will be handled, including rules for stopping data collection, criteria for inclusion and exclusion of data, how outliers will be defined and handled, statistical methods for data analysis, and identification of the primary endpoints/outcomes to be measured.

– Describe how data will be reported and how it will be assured that the documentation will support a regulatory filing with the FDA, if applicable.

DoD FY20 Duchenne Muscular Dystrophy Idea Development Award 20

○ Attachment 10: Representations, if applicable (extramural submissions only):

Upload as “RequiredReps.pdf”. All extramural applicants must complete and submit the Required Representations template available on eBRAP (https://ebrap.org/eBRAP/ public/Program.htm). For more information, see the General Application Instructions, Appendix 5, Section B, Representations.

○ Attachment 11: Suggested Collaborating DoD Military Facility Budget Format, if applicable: Upload as “MFBudget.pdf”. If a military facility (Military Health System facility, research laboratory, medical treatment facility, dental treatment facility, or a DoD activity embedded with a civilian medical center) will be a collaborator in performance of the project, complete a separate budget, using “Suggested Collaborating DoD Military Facility Budget Format,” available for download on the eBRAP “Funding Opportunities & Forms” web page https://ebrap.org/eBRAP/public/Program.htm), including a budget justification, for each military facility as…