Frequently Asked Questions
Frequently Asked Questions
We appreciate your interest in the Somatic Cell Genome Editing funding opportunities and hope that you and your team will choose to submit an application. This list of frequently asked questions will be updated periodically as new questions from potential applicants are received.
Will the applicant webinar be posted online afterward?
No. However this list of common FAQs will be updated to reflect questions asked during the webinar. Applicants are encouraged to send in additional questions to: SCGEprogram@nih.gov.
Would you please verify when the NIH application deadline is?
Proposals in response to RFA-RM18-022 through -025 are due October 18, 2018 at 5 PM local time of applicant’s organization. Applications in response to RFA-RM-18-014 are due August 24, 2018.
Are multi-PI applications allowed?
Yes.
What would be the eligibility rules regarding foreign researchers and institutions?
Foreign institutions are not allowed to apply to RFA-RM-18-014, but all the other funding opportunities accept foreign applicants and allow for foreign components. For additional information please see Section III.1 Eligible Applicants in each FOA.
Can NIH intramural investigators apply to any of the funding announcements?
Yes, intramural PIs are eligible to apply for all funding opportunities.
What are “U” grants and how are they different from R01s?
The 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.
What is the maximum amount of direct costs available for each application?
- RFA-RM-18-014: $750,000 in FY19-20; $1.4M per year FY21-23
- RFA-RM-18-022: $415,000 per year FY19-22
- RFA-RM-18-023: $500,000 per year FY19-21 (UG3); $1M per year FY22 (UH3)
- RFA-RM-18-024: $250,000 per year FY19-22
- RFA-RM-18-025: $150,000 per year FY19-20 (UH2); $300,000 per year FY21-22 (UH3)
Do I have to abide by the timeline outlined in the FOA?
Milestones must be completed within the timeframe outlined in the FOA. However, milestones may be completed early.
These are separate FOAs. An investigator can be PI on one application and a PI or multi-PI on a second separate application.
Yes. The Bayh-Dole Act is applicable to this program and intellectual property rights will remain with awardee institutions. The SCGE Steering Committee of investigators funded by FOAs RM18-012 through 018 and RM18-022 through 025 will develop procedures to facilitate maximal sharing while protecting intellectual property rights.
Can my project focus on more than one type of cell/tissue?
Yes.
No. Applications will be reviewed by a Special Emphasis Panel for each funding opportunity, and reviewers may not have an application among those being reviewed.
Will the applications submitted to all the FOAs be reviewed in one study section?
No. These are separate FOAs. Separate Special Emphasis Panels will be convened for each FOA.
Can I submit just the UG3 portion to RFA-RM-18-023?
No, only applications for the combined mechanism (UG3/UH3) will be deemed responsive to this FOA.
The goal of FOA RFA-RM-18-023 is to develop technologies to delivery genome editing machinery to specific cells and tissues. For initial proof of concept studies during the UG3 phase, investigators can use a disease model, if the use of that model can demonstrate delivery into target cell types. However, for conversion to the UH3 phase, independent validation of genome editing in target tissues or reporter animals is required. To meet this requirement, investigators will incorporate a genome editor targeted to a reporter gene present in the tester animals generated by the Rodent Testing Centers (RM-18-012), and send this material to the testing center for independent validation.
The platform may incorporate multiple cell/tissue types if they are relevant to the organ/system of focus.
No, we strongly encourage applicants to utilize cutting edge computational and bioinformatics approaches in their study design, but this is not an absolute requirement.
No. The SCGE funding announcements (RFAs) will not require SCR. For more information please see NOT-OD-12-140.
Can NIH provide additional guidance on including milestones in the application?
Milestones should be scientifically justified and well defined for each year of the project and be based on the proposed specific aims. Whenever feasible, milestones should provide quantitative benchmarks for comprehensively assessing the annual progress of the project. Milestones must not be simply a restatement of the specific aims. The specific aims describe the research goals of the project. Rather, the milestones should provide the means for assessing the progress made towards each aim and offer a timeline and a “pathway” for the testing of a discovery concept or development of a technology.
I am a young investigator, will applying for these RFAs make me lose ESI?
Yes, you would lose your ESI status for any FOA except RM18-025. For more information please visit here.
The NIH expects researchers to study both male and female vertebrate animals, thereby improving our understanding of health and disease in men and women. Failure to account for sex as a biological variable may undermine the rigor, transparency and generalizability of research findings. Applicants must provide strong justification for applications proposing to study only one sex. The absence of evidence regarding sex differences in an area of research does not constitute strong justification to study only one sex. For more information and guidance see:
What resources are available to craft a competitive proposal?
Please see NIH resources. In addition it also helpful to look for guidance and mentoring from more experienced investigators. The Scientific/Research Contact on the FOA of interest can also serve as a good resource.
What are examples of other Consortia that have multiple components?
Examples of other complex Common Fund consortia are the Human Microbiome Project (HMP), the first phase of which had 7 components and the Extracellular RNA Communication Program, which has 5 components.
How will data and resource sharing be addressed for the program?
The Steering Committee along with input from the NIH will discuss and set the guidelines.
Will there be annual consortium meetings?
Yes, the Consortium will meet twice a year in-person for steering committee meetings.
Yes, for all of the FOAs.
Are applications that have collaborations preferred or are individual applications acceptable?
Many FOAs might require diverse types of expertise, which may require investigators to find collaborators.
What info should be included in Letters of Intent?
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. A potential budget would also be helpful to NIH.
Will I receive feedback on a Letter of Intent?
No. The Letter of Intent will help NIH staff plan the review.
Is RFA-RM-18-022 (Biological Systems) only looking for proposals which focus on human cells?
Yes, applications should only propose studies in human cells.
The UG3 phase will involve requires independent evaluation of editing in specific tissues, thus it is necessary to deliver a specific functional genome editor to the cell of choice. However, for the application, preliminary evidence is not required.
Cell types of interest include but are not limited to disease-relevant cells, especially endogenous stem cells and cell types of origin for cancer, in the following organs or systems:
Applications proposing delivery ex vivo will have a lower priority; in vivo is preferred. However, if the technology could do both, that is considered responsive.
- Nervous System
- Cardiovascular System, including hematopoietic and immune cells
- Sensory Organs
- Kidney
- Muscle
- Bone
- Endocrine system
- Lymphatic organs
- Gastrointestinal tract
Applications proposing delivery ex vivo will have a lower priority; in vivo is preferred. However, if the technology could do both, that is considered responsive.
For RFA-RM-18-023, how should the UG3/UH3 transition be discussed in the application?
Please see FOA under Research Strategy in Section IV. It is not necessary to describe the independent validation studies, as those will be carried out in SCGE Testing Centers funded under RM-18-012. The UH3 phase should be written based on the assumption that the technology will be validated and therefore make it to UH3 phase.
No. While it is possible to propose using a disease model as a proof-of-concept for the UG3 phase of the study, the overall proposal should focus on developing the delivery technologies into the cell/tissue of choice. The UH3 portion of the award will be focused on scalability of the delivery technology into a large animal model.
This phase of the award is primarily focused on optimization and scale-up of delivery technologies for testing in large animals.
This FOA is trying to capture new and innovative concepts. Preliminary data is not required for this award; however, justification is needed for your proposal. It is important to demonstrate that the applicant(s) has the ability to follow-through on innovative approaches.
Is the first phase (UG3) of RFA-RM-18-023 focused only on delivery to small animals?
Investigators can choose which type of animal to work on during the UG3 phase. However, independent validation is a requirement for transition to the UH3 phase. For independent validation, delivery vehicles containing genome editors will be sent to the SCGE testing center (see RFA-RM-18-012) for validation of genome editing in target cells in the reporter animals, which will be mice.
For RFA-RM-18-023, should the budget for the UH3 portion of the award be included?
Yes.
For RFA-RM-18-023, can the UH3 phase include GMP manufacturing and GLP studies?
No.
As stated in the FOA, slight modifications or incremental advances over existing genome editing or gene therapy delivery approaches will not be considered responsive to this FOA. Examples of qualitative improvements that would substantially impact clinical application include but are not limited to:
- Greater capacity and versatility regarding the size and type of genome editing machinery delivered
- Reduced immunogenicity
- Avoidance of pre-existing immunity
- Improved or expanded cell-type targeting
- Simplification or increased scalability of production
- Less invasive mode of administration
- Support of transient or regulatable expression of genome editors
Does RFA-RM-18-024 allow the use of a modular budget since the cap is $250K?
Applications from a domestic organization applying for $250,000 or less per budget period in direct costs can use a modular budget form. Applications from foreign (non-U.S.) institutions must include only detailed (non-modular) budgets. More information can be found here: https://grants.nih.gov/grants/funding/modular/modular_faq_pub.htm#165
For RFA-RM-18-025, is there a specific animal model that should be used to validate the technology?
Applicants should validate the technology in a small animal model.
Can I submit just the UH2 portion of RFA-RM-18-025?
No, only applications for the combined mechanism (UH2/UH3) will be deemed responsive to this FOA.
For RFA-RM-18-025, what does "clinically-relevant approaches" mean?
Approaches using techniques, reagents, or methodologies that are already approved for human use, or are based on minimal modifications that are likely to be safe in humans as well.
While important, a project specifically focused on detecting a single protein product is not within the overall goal of this Common Fund program, and would likely be a better fit for a relevant IC program. However, if this approach was used to demonstrate proof of concept for a platform-based approach that could be adapted to detect a broad array of genome edited products or components, then would be responsive to this FOA.
Will there be future re-release of SCGE RFAs?
No, this is currently the last planned issuance of SCGE RFAs.
Can you comment on how much preliminary data would be needed for these mechanisms in general?
U01 (RM18-022 & -024) – preliminary data is required for this mechanism; applications should be approached similarly to R01s in this respect.
UG3/UH3 & UH2/UH3 (RM18-023 & -025) – Preliminary data are not required for these mechanisms, but may be included if available. Appropriate justification for the proposed work can be provided through literature citations, logic, and data from other sources.
UG3/UH3 & UH2/UH3 (RM18-023 & -025) – Preliminary data are not required for these mechanisms, but may be included if available. Appropriate justification for the proposed work can be provided through literature citations, logic, and data from other sources.
Yes, AAV-based applications are allowed; however, applications proposing to simply incorporate different genome editors into currently known AAV serotypes would not be responsive.
Any reasonable screen proposed would be considered responsive.
Both discovery of novel genome editing systems as well as optimization of existing editing platforms are responsive areas of research.
Are disease models allowed for RM18-022?
No, applications should focus on normal biology for testing the effects of genome editing.
How will milestones be evaluated?
U01 Mechanism (RM18-022 & -024): Milestones are used to assess progress and make sure grantees are on track.
UG3/UH3 & UH2/UH3 Mechanism (RM18-023 & -025): Milestones will be used to determine advancement into the UH3 phase for both award mechanisms. A clear go-/no-go transition milestone will designate this transition to the UH3 phase. Milestones can be negotiated between NIH program staff and the applicant.
For RM18-023, validation of in vivo delivery of genome editing machinery is key. The delivery awardee will work within the Consortium agreement to develop a protocol with the Small Animal Testing Center (RM18-012) in order to validate their delivery tool.
UG3/UH3 & UH2/UH3 Mechanism (RM18-023 & -025): Milestones will be used to determine advancement into the UH3 phase for both award mechanisms. A clear go-/no-go transition milestone will designate this transition to the UH3 phase. Milestones can be negotiated between NIH program staff and the applicant.
For RM18-023, validation of in vivo delivery of genome editing machinery is key. The delivery awardee will work within the Consortium agreement to develop a protocol with the Small Animal Testing Center (RM18-012) in order to validate their delivery tool.
All applications in response to RFA-RM18-022 through RFA-RM18-024 will be considered NEW applications.
