IND-enabling Studies for Platform Clinical Trials of Genome Editing in Multiple Diseases (U01 Clinical Trial Not Allowed)

IND-enabling Studies for Platform Clinical Trials of Genome Editing in Multiple Diseases (U01 Clinical Trial Not Allowed) is sponsored by NIH Common Fund. Funding for IND-enabling studies to support platform clinical trials of genome editing in multiple diseases.

Expired RFA-RM-24-001: IND-enabling Studies for Platform Clinical Trials of Genome Editing in Multiple Diseases (U01 Clinical Trial Not Allowed) This notice has expired. Check the NIH Guide for active opportunities and notices. Department of Health and Human Services Part 1.

Overview Information Participating Organization(s) National Institutes of Health ( NIH ) Components of Participating Organizations Office of Strategic Coordination ( Common Fund ) This Notice of Funding Opportunity (NOFO) is developed as a Common Fund initiative (http://commonfund. nih. gov/) through the NIH Office of the NIH Director, Office of Strategic Coordination (https://dpcpsi.

nih. gov/). All NIH Institutes and Centers participate in Common Fund initiatives.

The NOFO will be administered by the National Center for Advancing Translational Science, (NCATS/NIH) ( https://ncats. nih. gov/ ) on behalf of the NIH.

Funding Opportunity Title IND-enabling Studies for Platform Clinical Trials of Genome Editing in Multiple Diseases (U01 Clinical Trial Not Allowed) U01 Research Project Cooperative Agreements August 31, 2022 - Implementation Changes for Genomic Data Sharing Plans Included with Applications Due on or after January 25, 2023. See Notice NOT-OD-22-198 . August 5, 2022 - Implementation Details for the NIH Data Management and Sharing Policy.

See Notice NOT-OD-22-189 . Funding Opportunity Number (FON) Companion Funding Opportunity See Section III. 3.

Additional Information on Eligibility. Assistance Listing Number(s) Funding Opportunity Purpose The purpose of this NOFO is to provide support for applications for IND-enabling studies for the development of a novel in vivo genome editing therapeutic platform (genome editor plus delivery system) for two or more disease indications, using the same genome editor, route of administration, and delivery system.

Through this initiative, NIH aims to explore the extent to which the use of a therapeutic genome editing platform can streamline the regulatory path for multiple disease indications and to disseminate this regulatory information (and supporting documentation) to the scientific community.

Open Date (Earliest Submission Date) Letter of Intent Due Date(s) Renewal / Resubmission / Revision (as allowed) AIDS - New/Renewal/Resubmission/Revision, as allowed All applications are due by 5:00 PM local time of applicant organization. Applicants are encouraged to apply early to allow adequate time to make any corrections to errors found in the application during the submission process by the due date.

Required Application Instructions It is critical that applicants follow the instructions in the Research (R) Instructions in the How to Apply - Application Guide , except where instructed to do otherwise (in this NOFO or in a Notice from NIH Guide for Grants and Contracts ). Conformance to all requirements (both in the Application Guide and the NOFO) is required and strictly enforced.

Applicants must read and follow all application instructions in the Application Guide as well as any program-specific instructions noted in Section IV. When the program-specific instructions deviate from those in the Application Guide, follow the program-specific instructions. Applications that do not comply with these instructions may be delayed or not accepted for review.

Part 1. Overview Information Part 2. Full Text of Announcement Section I.

Notice of Funding Opportunity Description Section II. Award Information Section III. Eligibility Information Section IV.

Application and Submission Information Section V. Application Review Information Section VI. Award Administration Information Section VII.

Agency Contacts Section VIII. Other Information Part 2. Full Text of Announcement Section I.

Notice of Funding Opportunity Description The NIH Somatic Cell Genome Editing (SCGE) program is funded through the NIH Common Fund, which supports cross-cutting programs that are expected to have exceptionally high impact.

All Common Fund initiatives invite investigators to develop bold and innovative approaches to address problems that may seem intractable or to seize new opportunities that offer the potential for transformation of research processes.

The simplicity and broad applicability of targeted and programmable genome editing approaches, including, but not limited to those based on CRISPR-Cas9, raise the possibility of a fundamentally new way to treat a variety of genetic diseases. However, many challenges need to be overcome before such techniques could be widely used in the clinic.

To maximize the potential of genome editing technology, the SGCE program was developed to accelerate the translation of genome editing technology into clinical applications.

Based on input received from stakeholders from academia, industry, and regulatory agencies, as well as the substantial progress in the field of genome editing since the launch of the first five-year phase of the SCGE program, the second five-year phase of SCGE will focus on translating and accelerating safe and effective genome editing therapeutics into the clinic.

Specifically, SCGE Phase 2 will support the following initiatives: 1) Technologies and Assays for Therapeutic Genome Editing INDs; 2) IND-enabling Studies of Somatic Genome Editing Therapeutic Leads; 3) IND-enabling Studies and Platform Clinical Trials of Somatic Genome Editing for Multiple Diseases and 4) Somatic Cell Genome Editing Translational Coordination and Dissemination Center (TCDC).

The SCGE program will involve collaborative research by a consortium of award recipients with differing expertise to develop, optimize and demonstrate improved candidate genome editing therapeutic as treatments for human disease.

Recipients from all four SCGE program components will form a consortium, governed by a steering committee of investigators and NIH staff that will develop consensus policies and procedures for Consortium-wide activities such as data and resource sharing.

Collectively, these initiatives are intended to substantially expand the number of genetic diseases treated by in vivo genome editing, ultimately allowing this technology to achieve its potential as a therapeutic platform to treat genetic disease . The traditional path to drug development is to develop a single drug to treat a single disease.

In contrast, technologies such as genome editing are fundamentally different, in that they are therapeutic platforms that are in principle applicable to a large number of diseases. Therefore, clinical trial design and the associated regulatory pathway should reflect the therapeutic platform capacity of genome editing.

The purpose of this Notice of Funding Opportunity (NOFO) is to provide support for applications that propose Investigational New Drug (IND)-enabling studies for the development of a novel in vivo genome editing therapeutic platform (genome editor plus delivery system) for two or more disease indications.

Furthermore, it is expected that projects supported under this NOFO will explore strategies to increase efficiencies in the regulatory path for a therapeutic platform leading to genome editing clinical trial(s) for two or more disease indications.

We anticipate that dissemination of the data and knowledge gained by projects supported by this NOFO will allow genome editing technology to achieve its promise as a therapeutic platform for the treatment of genetic diseases. Program Formation and Governance The awards funded under this NOFO will be cooperative agreements (see Section VI. 2.

Cooperative Agreement Terms and Conditions of Award). Close interactions among the recipients and NIH will be required to maintain this complex program.

The whole SCGE program governance will rest with the SCGE Program Steering Committee in collaboration with NIH program officials, with input and feedback from Program Consultants (PCs) providing critical scientific and managerial insights, and subject to oversight by the NIH SCGE Working Group.

The NIH SCGE Working Group consists of NIH programmatic staff from multiple Institutes and Centers of the NIH as well as the Office of the Director. This group will be primarily responsible for the stewardship of the SCGE program.

The SCGE Working Group is co-chaired by the Director of the National Center for Advancing Translational Sciences (NCATS) and the Director of the National Institute for Neurological Disorders and Stroke (NINDS). It reports to the Directors of the Office of Strategic Coordination/Common Fund and the Division of Program Coordination, Planning, and Strategic Initiatives for final funding decisions.

Research Scope and Objectives The primary objectives of this NOFO are to: 1) explore the extent to which the use of a therapeutic platform consisting of a single genome editor and delivery system, can streamline the regulatory path to in vivo genome editing clinical trial(s) for two or more disease indications, and 2) to disseminate this regulatory information (and supporting documentation) to the scientific community.

Examples of streamlining in this context include, but are not limited to, reducing the number of biodistribution; toxicology and chemistry, manufacturing, and controls (CMC) studies necessary to support the regulatory approval process for clinical trial(s) two or more disease indications.

The extent of streamlining should be considered in comparison to the traditional regulatory path for two entirely independent submissions, one for each disease indication, in which every step of the regulatory path is required for each disease indication, without consideration of the therapeutic platform. Importantly, it is up to the FDA to decide how much, if any, streamlining is acceptable.

Therefore, applications submitted in response to this NOFO will be evaluated based on the extent to which the approach proposed in the application is technically feasible and could streamline the regulatory approval process if it were to be accepted by the FDA.

To achieve the objectives, this NOFO is intended to support IND-enabling studies of a therapeutic platform (genome editor and delivery system) that will be used in a subsequent platform clinical trial of more than one disease indication. However, this NOFO will only support the IND-enabling studies necessary to prepare and submit a complete IND package, not the actual trial.

IND-enabling studies proposed in the application may include, but are not limited to, in vitro studies, proof of concept (POC) studies in one or more animal models, toxicology, biodistribution, CMC studies, and preparation of an investigator’s brochure and clinical protocol.

Given the time frame of the Award, we anticipate that applicants will at a minimum have in vivo POC data from at least one animal model of disease using the therapeutic platform prior to submission. The IND-enabling studies and resulting IND for the clinical trial(s) must utilize the same genome editor, route of administration, and in vivo delivery system for the target disease indications.

The editor may be delivered in the form of DNA, mRNA, peptide nucleic acids, or protein, using either viral or non-viral based systems. Any genome editor, including but not limited to those that create a double-strand break, base editors, prime editors, RNA editors, or epigenome editors, can be chosen for use.

Examples of potential regulatory approaches include but are not limited to a single IND for two or more disease indications, or two or more related INDs in which each substantially cross-references the other(s) based upon the use of the same editor and same delivery vehicle for both disease indications.

Formal and informal meetings with the FDA (i.e., INTERACT and pre-IND meetings) are essential components of projects supported by this NOFO, and will be required as project milestones. By the end of the project, award recipients must (1) successfully prepare and submit a complete IND regulatory package to initiate the FDA review process; and (2) provide documentation of this to NIH.

Please note the documentation submitted to the NIH must also include but is not limited to the full IND package, all FDA-Sponsor interactive review and responses during the 30-day IND review, and FDA communication concerning meeting feedback and IND status.

Additionally, award recipients must provide lessons learned and information to the TCDC about their approach to obtaining the IND(s) for more than one disease indication, as well as responses from regulatory authorities, so that this information can be disseminated within and outside the SCGE consortium.

Genome editing therapy development is a complex and time-consuming undertaking, and applicants should consider forming multidisciplinary teams that may consist of preclinical and clinical scientists, pharmacokinetic (PK) experts, CMC experts, regulatory experts, statisticians, project manager, and other academic/industry experts relevant to the therapeutic modality and disorder.

Applicants are strongly encouraged to employ team and project management principles as appropriate. Collaborations with commercial entities that are developing genome editing therapeutics are encouraged. Applications containing risk mitigation strategies that proactively address potential delays or risks in meeting the milestones are strongly encouraged.

The following are examples of projects that would be considered non-responsive to this NOFO: IND-enabling studies for a genome editing for a single disease, even if targeting different causative genes Projects involving editing cells ex vivo , followed by in vivo injection of edited cells.

Clinical trials of genome editing in humans In preparing applications to this NOFO, investigators should be aware of requirements for gene editing clinical trials as identified in the relevant FDA Guidance documents (see https://www. fda. gov/vaccines-blood-biologics/biologics-guidances/cellular-gene-therapy-guidances ).

NCATS, as part of NIH, strives for rigor and transparency in all research it funds. For this reason, NCATS explicitly emphasizes the NIH application instructions related to rigor and transparency ( https://grants. nih.

gov/policy/reproducibility/guidance. htm ). For example, the biological rationale for the proposed experiments must be based on rigorous and robust supporting data, which means that data should be collected via methods that minimize the risk of bias and be reported in a transparent manner.

If previously published or preliminary studies do not meet these standards, applicants should address how the current study design addresses the deficiencies in rigor and transparency. Proposed experiments should likewise be designed in a manner that minimizes the risk of bias and ensures validity of experimental results.

Pre-application Consultation As an U01 cooperative agreement, implementation will include substantial involvement of NCATS Program staff in the planning and execution of the therapy-directed projects. Applicants and their multidisciplinary team are strongly encouraged to consult with NCATS Scientific/Research staff when planning an application.

Early contact provides an opportunity for NCATS Scientific/Research staff to provide further guidance on program scope, goals, developing appropriate milestones, and budget. Staff will not evaluate the technical and scientific merit of the proposed project; technical and scientific merit will be determined during peer review using the review criteria indicated in this NOFO. See Section VIII.

Other Information for award authorities and regulations. Section II. Award Information Cooperative Agreement: A financial assistance mechanism used when there will be substantial Federal scientific or programmatic involvement.

Substantial involvement means that, after award, NIH scientific or program staff will assist, guide, coordinate, or participate in project activities. See Section VI. 2 for additional information about the substantial involvement for this NOFO.

Application Types Allowed The OER Glossary and the SF424 (R&R) Application Guide provide details on these application types. Only those application types listed here are allowed for this NOFO. Not Allowed: Only accepting applications that do not propose clinical trials.

Need help determining whether you are doing a clinical trial? Funds Available and Anticipated Number of Awards The NIH Common Fund intends to commit a total budget of up to $6,0 00,000 per year in FY24-25 and $9,000,000 in FY26-27 to fund up to two awards.

Application budgets are limited to no more than $2M direct costs per year in FY24-25 and $3M direct costs per year in FY26-27, and should reflect the actual needs of the proposed project. The scope of the proposed project should determine the project period. The maximum project period is 4 years.

NIH grants policies as described in the NIH Grants Policy Statement will apply to the applications submitted and awards made from this NOFO. Section III. Eligibility Information All organizations administering an eligible parent award may apply for a supplement under this NOFO.

Higher Education Institutions Public/State Controlled Institutions of Higher Education Private Institutions of Higher Education The following types of Higher Education Institutions are always encouraged to apply for NIH support as Public or Private Institutions of Higher Education: Hispanic-serving Institutions Historically Black Colleges and Universities (HBCUs) Tribally Controlled Colleges and Universities (TCCUs) Alaska Native and Native Hawaiian Serving Institutions Asian American Native American Pacific Islander Serving Institutions (AANAPISIs) Nonprofits Other Than Institutions of Higher Education Nonprofits with 501(c)(3) IRS Status (Other than Institutions of Higher Education) Nonprofits without 501(c)(3) IRS Status (Other than Institutions of Higher Education) For-Profit Organizations (Other than Small Businesses) City or Township Governments Special District Governments Indian/Native American Tribal Governments (Federally Recognized) Indian/Native American Tribal Governments (Other than Federally Recognized) Eligible Agencies of the Federal Government-Including the NIH Intramural Program U.S. Territory or Possession Independent School Districts Public Housing Authorities/Indian Housing Authorities Native American Tribal Organizations (other than Federally recognized tribal governments) Faith-based or Community-based Organizations Non-domestic (non-U.S.) Entities (Foreign Organizations) are not eligible to apply.

Non-domestic (non-U.S.) components of U.S. Organizations are not eligible to apply. Foreign components, as defined in the NIH Grants Policy Statement , are allowed. Applicant organizations must complete and maintain the following registrations as described in the SF 424 (R&R) Application Guide to be eligible to apply for or receive an award.

All registrations must be completed prior to the application being submitted. Registration can take 6 weeks or more, so applicants should begin the registration process as soon as possible. Failure to complete registrations in advance of a due date is not a valid reason for a late submission, please reference NIH Grants Policy Statement Section 2.

3. 9. 2 Electronically Submitted Applications for additional information.

System for Award Management (SAM) Applicants must complete and maintain an active registration, which requires renewal at least annually . The renewal process may require as much time as the initial registration. SAM registration includes the assignment of a Commercial and Government Entity (CAGE) Code for domestic organizations which have not already been assigned a CAGE Code.

NATO Commercial and Government Entity (NCAGE) Code Foreign organizations must obtain an NCAGE code (in lieu of a CAGE code) in order to register in SAM. Unique Entity Identifier (UEI) - A UEI is issued as part of the SAM. gov registration process.

The same UEI must be used for all registrations, as well as on the grant application. eRA Commons - Once the unique organization identifier is established, organizations can register with eRA Commons in tandem with completing their Grants. gov registration; all registrations must be in place by time of submission.

eRA Commons requires organizations to identify at least one Signing Official (SO) and at least one Program Director/Principal Investigator (PD/PI) account in order to submit an application. Grants. gov Applicants must have an active SAM registration in order to complete the Grants.

gov registration. Program Directors/Principal Investigators (PD(s)/PI(s)) All PD(s)/PI(s) must have an eRA Commons account. PD(s)/PI(s) should work with their organizational officials to either create a new account or to affiliate their existing account with the applicant organization in eRA Commons.

If the PD/PI is also the organizational Signing Official, they must have two distinct eRA Commons accounts, one for each role. Obtaining an eRA Commons account can take up to 2 weeks.

Eligible Individuals (Program Director/Principal Investigator) Any individual(s) with the skills, knowledge, and resources necessary to carry out the proposed research as the Program Director(s)/Principal Investigator(s) (PD(s)/PI(s)) is invited to work with their organization to develop an application for support.

Individuals from diverse backgrounds, including underrepresented racial and ethnic groups, individuals with disabilities, and women are always encouraged to apply for NIH support. See, Reminder: Notice of NIH's Encouragement of Applications Supporting Individuals from Underrepresented Ethnic and Racial Groups as well as Individuals with Disabilities, NOT-OD-22-019 .

For institutions/organizations proposing multiple PDs/PIs, visit the Multiple Program Director/Principal Investigator Policy and submission details in the Senior/Key Person Profile (Expanded) Component of the SF424 (R&R) Application Guide. This NOFO does not require cost sharing as defined in the NIH Grants Policy Statement Section 1. 2 Definition of Terms .

3. Additional Information on Eligibility Applicant organizations may submit more than one application, provided that each application is scientifically distinct. The NIH will not accept duplicate or highly overlapping applications under review at the same time, per NIH Grants Policy Statement Section 2.

3. 7. 4 Submission of Resubmission Application .

This means that the NIH will not accept: A new (A0) application that is submitted before issuance of the summary statement from the review of an overlapping new (A0) or resubmission (A1) application. A resubmission (A1) application that is submitted before issuance of the summary statement from the review of the previous new (A0) application.

An application that has substantial overlap with another application pending appeal of initial peer review (see NIH Grants Policy Statement 2. 3. 9.

4 Similar, Essentially Identical, or Identical Applications ). Section IV. Application and Submission Information 1.

Requesting an Application Package The application forms package specific to this opportunity must be accessed through ASSIST, Grants. gov Workspace or an institutional system-to-system solution. Links to apply using ASSIST or Grants.

gov Workspace are available in Part 1 of this NOFO. See your administrative office for instructions if you plan to use an institutional system-to-system solution. 2.

Content and Form of Application Submission It is critical that applicants follow the instructions in the Research (R) Instructions in the How to Apply - Application Guide except where instructed in this notice of funding opportunity to do otherwise. Conformance to the requirements in the Application Guide is required and strictly enforced.

Applications that are out of compliance with these instructions may be delayed or not accepted for review. Although a letter of intent is not required, is not binding, and does not enter into the review of a subsequent application, the information that it contains allows IC staff to estimate the potential review workload and plan the review. By the date listed in Part 1.

Overview Information , prospective applicants are asked to submit a letter of intent that includes the following information: Descriptive title of proposed activity Name(s), address(es), and telephone number(s) of the PD(s)/PI(s) Names of other key personnel Participating institution(s) Number and title of this funding opportunity The letter of intent should be sent to: All page limitations described in the How to Apply Application Guide and the Table of Page Limits must be followed.

Instructions for Application Submission The following section supplements the instructions found in the How to Apply Application Guide and should be used for preparing an application to this NOFO. All instructions in the SF424 (R&R) Application Guide must be followed. SF424(R&R) Project/Performance Site Locations All instructions in the SF424 (R&R) Application Guide must be followed.

SF424(R&R) Other Project Information All instructions in the SF424 (R&R) Application Guide must be followed. SF424(R&R) Senior/Key Person Profile All instructions in the SF424 (R&R) Application Guide must be followed. All instructions in the SF424 (R&R) Application Guide must be followed.

Also, applicants must provide a detailed budget and budget justification for each budget period of the U01. All applicants must budget for travel for personnel to attend two SCGE program PI meetings per year in the Washington, DC metropolitan area. NIH will generally not provide F&A costs unless the recipient has established an F&A cost rate covering the applicable activities and period of time.

Applicant organizations that do not have an established F&A cost rate should review the NIH Grants Policy Statement reimbursement of facilities and administrative (F&A) costs policies prior to application submission. All instructions in the SF424 (R&R) Application Guide must be followed. PHS 398 Cover Page Supplement All instructions in the SF424 (R&R) Application Guide must be followed.

All instructions in the SF424 (R&R) Application Guide must be followed, with the following additional instructions: This section should provide a concise description of the aims for the project, including a description of the proposed regulatory approach for studying a genome editing therapeutic platform for more than one disease.

Describe the genome editor, delivery system, route of administration, target tissue, and disease indication that will be studied in the future clinical trial. Provide biological rationale for each. Provide a description of any completed IND-enabling studies for the in vivo genome editing therapeutic platform (genome editor plus delivery system) as preliminary data.

These data may be obtained from in vitro studies, as well as one or more in vivo animal models representative of the intended patient population. Given the aggressive timeline for the work to be carried out under this NOFO, investigators are expected to have substantial preclinical data on hand regarding the proposed editor and delivery system prior to submitting the application.

At a minimum, this would include POC data in an animal model of at least one of the proposed disease indications, using the intended editor and delivery system. Provide a Target Product Profile (TPP) based on the FDA guidance [ https://www. fda.

gov/vaccines-blood-biologics/biologics-guidances/cellular-gene-therapy-guidances ] that summarizes the minimal/ideal profile of the final product and the ultimate goals of the proposed drug development effort, such as patient population, delivery mode, treatment duration, treatment regimen, and standards for clinical efficacy for each disease indication.

Describe the disease indications to be studied, the clinical characteristics, and current and projected prevalence of the number of patients with each of the proposed disease indications that will be studied in the clinical trial. Explain how the project offers an approach to treating the patient population as proposed in the TPP.

Describe collaborations with commercial entities that are developing genome editing technologies, if applicable.

Describe all non-clinical testing necessary to support the filing of an IND, including the standards to which the testing will comply (e.g., Good Laboratory Practices (GLP), Good Manufacturing Practices (GMP)) Describe the development of in vitro assays to identify and test guide RNAs to be used for the two disease indications in the clinical trial.

Describe plans for contact with and submissions to the FDA (e.g., pre-submission meetings, IND submissions, other interactions with FDA personnel. Include plans for how FDA feedback will be addressed (e.g., if additional IND-enabling studies are suggested).

Address how the approach for clinical trial design of more than one disease indication results in increased efficiency compared to the standard approach of two or more independent clinical trials. Applications must include a Milestone Plan. The Milestone plan should include : A project timeline in the form of a Gantt chart, with all milestones included, to obtain regulatory approval to conduct the clinical trial.

Required milestones include conducting and completing IND-enabling studies; INTERACT meeting; pre-IND meeting; compilation and submission of an IND package to the FDA; and providing documentation of the IND and FDA communications, submissions, and lessons learned to the NIH and the SCGE Translational Coordination and Dissemination Center (TCDC).

Milestones should be well described (specific, quantitative) with clear indicators of a success. Provide any risk mitigation strategies that proactively address potential delays or risks in meeting the milestones. The filename "Milestone Plan-PI-NAME.

pdf" should be used, must not exceed 50 characters, and will be reflected in the final image bookmarking for easy access by reviewers. The Milestone Plan is limited to 3 pages. Applications that do not include the Milestone Plan will be considered incomplete and will not be reviewed.

Provide a team management plan that describes how multidisciplinary expertise is incorporated in the overall project plan. Include if and how members have previous experience with the IND process. Describe how the team, including consultants, will work over the course of the project (e.g., recurring team meetings, review and report of data across disciplines, decision-making, participate in meetings with NIH, communication, etc.).

Letters of support: Statements of individual and institutional commitment, as appropriate to the Research Project, should be included in this section. Letters of support should not be generic, but instead indicate what the collaborator has contributed so far and what they expect to provide during the project to allow an evaluation of team engagement.

Indicate the willingness of the PD/PI(s) and key personnel to operate under the cooperative agreement terms and conditions outlined in section VI. 2. Of the NOFO.

Note: Multi-PI plans should not duplicate information from the multi-PI plan. Individuals are required to comply with the instructions for the Resource Sharing Plans as provided in the SF424 (R&R) Application Guide. The following modifications also apply: Applicants should indicate their willingness to make some applicant-generated animal and/or human samples from genome-editing therapeutic studies to other Consortium members.

Recipients are encouraged to collaborate with other SCGE Consortium members to help evaluate the utility and performance of the assay(s) to further advance the field.

Specific Plan for Protocol, Tool, and Reagent Sharing: As one of the primary goals of this program is to advance research through development, establishment, broad dissemination and use of community resources across the research community, NIH intends that protocols, tools, and reagents generated by the SCGE program be broadly available and distributed at no to minimal cost, and without undue intellectual property constraints, so that they can be as widely used as possible for research purposes by the larger scientific community, while encouraging rapid adoption and commercialization of the technologies for the development of genome editing therapies.

For all applications and where otherwise applicable, the applicant should discuss plans for sharing and distribution of non-data resources that will be generated by the proposed project, including animal strains, protocols, biomaterials, and reagents.

The SCGE TCDC will work with all SCGE program investigators to collect, curate, and disseminate information regarding tools and reagents being developed by the program and to disseminate this information through the SCGE Toolkit and other sources as appropriate and consistent with achieving the goals of the program.

Specific Plan for Sharing Software: A software dissemination plan, with appropriate timelines, is expected in applications that are developing software. There is no prescribed single license for software produced in this project; however, reviewers will be asked to evaluate the software sharing and dissemination plan based on its likely impact.

A dissemination plan guided by the following principles is thought to promote the largest impact: The software should be freely available to biomedical researchers and educators in the non-profit sector, such as institutions of education, research institutions, and government laboratories.

The terms should also permit the dissemination and commercialization of enhanced or customized versions of the software, or incorporation of the software or pieces of it into other software packages. To preserve utility to the community, the software should be transferable such that another individual or team can continue development in the event that the original investigators are unwilling or unable to do so.

The terms of software availability should include the ability of researchers outside the project and its collaborating projects to modify the source code and to share modifications with other colleagues. An applicant should take responsibility for creating the original and subsequent official versions of a piece of software.

Applicants are asked to propose a plan to manage and disseminate the improvements or customizations of their tools and resources by others. This proposal may include a plan to incorporate the enhancements into the official