All SCGE Phase 2 FOAs allow applications from for-profit institutions. RFA- RM-22-016 encourages collaborations with commercial entities developing genome editing therapeutics. Please contact the Grants Management Officer listed on the FOA for additional information.

Yes.

These are separate FOAs. An investigator can be a PI on one application and a PI or multi-PI on a second separate application.

Foreign institutions are allowed to apply to RFA-RM-22-014. RFA-RM-22-015, RFA-RM-22-016, and RFA-RM-22-017 do not accept foreign applicants but allow for foreign components. For additional information please see Section III.1 Eligible Applicants in each FOA.

Intramural investigators are eligible to apply to RFA-RM-22-014, RFA-RM-22-015, and RFA-RM-22-016, but not to RFA-RM-22-017.

SCGE Phase I eligibility is not a requirement for any of the SCGE Phase II FOAs.

Yes.

Please learn more about ESI status at the link below: https://grants.nih.gov/grants/esi-status.pdf

Yes.

Applications to RFA-RM-22-014, RFA-RM-22-015, RFA-RM-22-016, and RFA-RM-22-017 are due July 19, 2022.

Letters of intent for RFA-RM-22-014, RFA-RM-22-015, RFA-RM-22-016, and RFA-RM-22-017 are due June 17, 2022.

No, letters of intent are recommended but not required.

Milestones must be completed within the timeframe outlined in the FOA. However, milestones may be completed early.

The SCGE FOAs use the cooperative agreement funding mechanism. A cooperative agreement supports discrete, specified, circumscribed projects to be performed by investigators in an area representing their specific interest and competencies, and is used when substantial NIH programmatic involvement is anticipated.

See section IV of FOA; Provide PI name(s) and telephone number(s), names of other key personnel, title of project, participating institution, and name and number of FOA.

No. The Letter of Intent will help NIH staff plan the review.

Subcontract, if they’re not eligible as primary awardees.

No. The NIH does not have a preference.

The U19 resource cores should be part of the U19 application and the resulting research project. The U19 mechanism is one that is self-contained, where the research cores are in support of the research projects of the same U19 application.

Not within the U19, but would best fit under the umbrella of RFA-RM-22-017.

Both resource and administrative cores will receive only descriptive ratings. Descriptors are “exceptional,” “adequate,” or “inadequate.” However, “lack of enthusiasm” or “high enthusiasm” for either or both cores will contribute to the overall impact assessment score of the U19 project. For more information, please see Section V of RFA-RM-22-015.

Epigenome editors count as editing.

Yes.

Yes.

Each of the RFAs have a section on NIH data sharing requirements. If you have any particular IP concerns, you can seek advice from the Scientific Contacts on the RFA you want to apply to.

Data sharing between projects within a U19 award, different U19 awards, and the broader SCGE consortium is expected.

Existing teams or prior work between collaborators is not an eligibility requirement for RFA-RM-22-015.

Yes.

Cores provide a consistent type of support to at least two or more projects. E.g., If a lab is providing service (e.g., regulatory, animal testing, or assay dev) to projects A and B, it could be conceptualized as a core. But if a lab is only providing support or services to a single project or is providing different kinds of support to projects A and B (e.g., animal testing to project A but provision of delivery reagents to project B), then it’s considered a collaborator.

Yes. The U19 can take “multiple shots on goal” and have multiple projects targeting the same disease with different approaches, e.g., it’d be perfectly acceptable if there were one project targeting disease X using nanoparticles and base editors, and one project targeting disease X using dual AAV and prime editors. There need to be at least 3 projects, and it’s important to remember that there are distinct and defined entry criteria for both the trailblazer and non-trailblazer projects. It’s expected that the projects will focus on a related tissue or cell type because projects are required to demonstrate that progress in the lead candidate will and can be leveraged for the other projects.

Yes.

There are no specific requirements. Either delivery strategy can be utilized.

The $25M budget is the total cost for RFA-RM-22-015, which includes both direct and indirect costs.

Yes.

Yes.

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 Administrative Core of each U19 facilitates and participates with the shared learning and dissemination programs of the Translational Coordination and Dissemination Center (TCDC) to help create a shared learning and training education hub. As part of this goal, investigators will be expected to provide information to the TCDC about the general approach across research projects to lead identification, optimization, and obtaining the IND to the TCDC, so that information can be disseminated within and outside the consortium.

Yes.

One page of preliminary data will be accepted for single component applications or for each component of a multi-component application up to 30 days before the review meeting.

No, only applications for the combined mechanism (UG3/UH3) will be deemed responsive to this FOA.

Epigenome editors count as editing

Yes.

Yes.

Each of the RFAs have a section on NIH data sharing requirements. If you have any particular IP concerns, you can seek advice from the Scientific Contacts on the RFA you want to apply to.

Existing teams or prior work between collaborators is not an eligibility requirement for RFA-RM-22-016.

The IP rights for private sector applicants/collaborators are the same as for academic applicants/collaborators. Any pre-existing IP is retained by patentee, and any new IP is subject to US Patent law and inventorship criteria.

Cas9 and dCas9 would be different editors.

To be responsive to RFA RM 22-016, applicants must target different diseases. A clinical genome editing trial in a single disease, even if targeting different causative genes, would be non-responsive.

Sufficient details should be included to allow effective peer review.

No.

The TCDC intends to have an educational component for the consortium, which could include materials to help investigators in areas they are not familiar with such as safety and cGMP manufacturing.

NIH programmatic support will be provided. The data from the SCGE Phase 1 toolkit would need to be maintained for the Phase 2 platforms. Our vision is that there would be continuity in bringing in data from Phase 1; however, the program is moving into an IND regulatory space, so the type of data that is already in that platform is not new data, which is why the NIH is hoping that the new TCDC will have a vision for developing platforms with a wide variety of data. The target is the public – who will use this resource, those that would be continuing regulatory pipelines and conducting genome editing clinical trials. The TCDC applicant would propose a strategy for that. We have an expectation of a timeline with milestones, but the applicant’s expertise would drive the feasibility of that. The anticipation of the types of data we’d be expecting is written in the RFA.

Yes. This is a cooperative agreement, which comes with the expectation that other grantees within the consortium will participate in the data curation and submission to the TCDC. All applicants are encouraged to read the TCDC FOA.