NOTICE OF LIMITED COMPETITION REQUEST FOR COMPETING APPLICATIONS: NHLBI PROGRAMS OF EXCELLENCE IN GENE THERAPY FOR HEART, LUNG AND BLOOD DISEASES RELEASE DATE: April 23, 2004 NOTICE: NOT-HL-04-110 National Heart, Lung, and Blood Institute (NHLBI) (http://www.nhlbi.nih.gov/) APPLICATION RECEIPT DATE: October 21, 2004 The NHLBI is requesting competing renewal applications from the four NHLBI Programs of Excellence in Gene Therapy (PEGTs) (http://www.med.cornell.edu/pegt/). These programs were awarded as a result of Request for Applications (RFA) HL-00-008, Programs of Excellence in Gene Therapy (http://grants.nih.gov/grants/guide/rfa-files/RFA-HL-00-008.html). This re- competition will continue to facilitate clinical gene therapy studies and provide unique ongoing resources and education programs to the NHLBI investigators for a one time renewal cycle. The following four activities in the PEGTs will be emphasized in the renewal period: o Preclinical projects to facilitate the translation of gene therapy into clinical studies o Clinical Phase I/II studies to test safety and efficacy of gene therapy studies in cardiovascular, pulmonary and hematological disorders o National Cores to provide resources and services to NHLBI-supported investigators at no cost o Training positions in clinical projects to train MD,MD/PhD and PhD scientists in conducting gene therapy clinical trials Each PEGT must provide a multidisciplinary structure that will coordinate activities between basic science investigators and clinicians in order to enable the translation of basic research to clinical application. Preclinical Projects: Each PEGT must consist of preclinical projects that will generate sufficient data to support the movement of the project to the next clinical phase. For example: 1) experiments addressing the safety and evaluation of the vector and the toxicity of the expressed protein(s); 2) experiments verifying that the administration route successfully transports the gene of interest to the target site; 3) experiments evaluating the immune response against the gene therapy product; and 4) experiments evaluating the biodistribution of the gene of interest. Clinical Projects: Each PEGT must have two or more clinical projects (Phase I and/or Phase II studies). Clinical projects that were initiated and supported as preclinical projects during the previous award cycle and not completed are expected to be included in the competing renewal application. If previous clinical studies were completed or terminated, new clinical studies may be proposed. Clinical projects can be proposed as preclinical studies in years 06 and/or 07 in order to meet the requirements for an Investigational New Drug (IND) application prior to initiating the clinical studies. However, by the beginning of year 08, these clinical projects must plan to initiate Phase I and/or Phase II clinical studies. Phase III studies will not be considered. Foreign collaborations for clinical studies may be included if they have fulfilled all the regulatory requirements as required in the United States ie. IRB, IBC, DSMB, RAC and OHRP. National Cores: All National Cores should continue in this new cycle. However, if investigators do not wish to continue a specific National Core due to the lack of interest from the community, they may propose a new National Core with prior approval from NHLBI. Each National Core must be submitted as a separate section with its own budget. The progress report section should include a description of all the services performed in the previous cycle with a detailed budget for these services. If the PEGT has a similar core for the PEGT itself, it is not considered a National Core and should only be included in the PEGT local Core section. Training Program: Each PEGT must have two clinical training positions associated with the proposed clinical trials. Trainees will be supported by the PEGT for up to two years at which time the trainees will be expected to apply for other support. The National Cores may propose courses and training in state of the art gene transfer technology. All PEGTs may compete for the Administrative Coordinating Center (ACC) that replaces the Data Coordinating Center . This must be submitted as a separate application from the PEGT application. The ACC will maintain the PEGT website, manage and administer the service requests for the National Cores and arrange meetings such as Steering Committee meetings, Grantees meetings and NHLBI Data and Safety Monitoring Committee meetings. MECHANISM OF SUPPORT The PEGTs use the cooperative agreement (U01) administrative and funding mechanism of support. Under the cooperative agreement, the NIH assists, supports, and/or stimulates, and is substantially involved with recipients in conducting a study by facilitating performance of the effort in a "partner" role. Details of the responsibilities, relationships, and governance of a study funded under this cooperative agreement will be the same as in the current terms and conditions of the award. Organization, Operation, and Oversight, as well as Terms and Condition of award remain unchanged from the original RFA (http://grants.nih.gov/grants/guide/rfa-files/RFA-HL-00-008.html). The total project period for an application submitted in response to this Notice may not exceed five (5) years. This program will end as of August 31, 2010. No renewal of the program will occur. The anticipated award date is September 1, 2005. FUNDS AVAILABLE The NHLBI intends to commit approximately $14 million total costs per year to support 4 Programs of Excellence in Gene Therapy (PEGT), 6 National Cores, and 1 Administrative Coordinating Core. The Programs of Excellence in Gene Therapy (PEGT) applicants may request a project period of 5 years. The requested budget for each PEGT should include direct costs and associated facilities and administrative (F&A) costs and may not exceed 10% over the total costs awarded in the year 05 budget. The National Core applicants may request a project period of 5 years. The requested budget for each National Core should include direct costs and associated facilities and administrative (F&A) costs and may not exceed 10% over the total costs awarded in the year 04 budget. The Administrative Coordinating Core (ACC) must request a project period of up to 5 years. The requested budget for the ACC should include direct costs and associated facilities and administrative (F&A) costs, and the cost for the biannual NHLBI Gene Therapy DSMB meetings. The total costs for this budget may not exceed $500 thousand per year. A composite budget representing all components for the entire project period is required. Budget Form pages 4 and 5 of PHS Form 398 should be completed for each component listed in the composite budget. A separate budget is required for each pre-clinical, clinical, local core, national core and any subcontracts. The budget pages must be clearly labeled as to which component activity they address. Applicants should provide adequate budget justification, and all applicable direct costs, and facilities and administrative (F&A) costs. In addition, the utilization relationships between research projects and any cores that serve those projects should be presented. SUBMITTING AN APPLICATION Applications must be prepared using the PHS 398 research grant application instructions and forms (rev. 5/2001). Applications must have a DUN and Bradstreet (D&B) Data Universal Numbering System (DUNS) number as the Universal Identifier when applying for Federal grants or cooperative agreements. The DUNS number can be obtained by calling (866) 705-5711 or through the web site at http://www.dunandbradstreet.com/. The DUNS number should be entered on line 11 of the face page of the PHS 398 form. The PHS 398 document is available at http://grants.nih.gov/grants/funding/phs398/phs398.html in an interactive format. For further assistance contact Grants Info, Telephone (301) 710-0267, Email: GrantsInfo@nih.gov. SUPPLEMENTAL INSTRUCTIONS: Only competing renewal applications which were funded as part of RFA HL-00-008, Programs of Excellence in Gene Therapy for Heart, Lung and Blood Diseases will be considered. For each clinical study, there must be a timeline, a detailed monitoring plan and a copy of all RAC communications, IRB and IBC communications or approvals and pre-IND or IND number. For every application, Item 2 on the Face Page should be checked "Yes"; after "Number:" enter "NOT-HL-04-104" and after "Title:" enter "Competing Applications for PEGTs". SENDING AN APPLICATION TO THE NIH: Submit a signed, typewritten original of the application, including the Checklist, and three signed, photocopies, in one package to: Center For Scientific Review National Institutes of Health 6701 Rockledge Drive, Room 1040, MSC 7710 Bethesda, MD 20892-7710 Bethesda, MD 20817 (for express/courier service) At the time of submission, two additional copies of the application plus all five collated sets of appendix material must be sent to: Chief, Review Branch National Heart, Lung, and Blood Institute National Institutes of Health 6701 Rockledge Drive, Room 7214 Bethesda, MD 20892-7924 Bethesda, MD 20817 (for express/ courier service) Telephone: 301-435-0270 FAX: 301-480-0730 APPLICATION PROCESSING: Applications must be received on or before the application receipt date listed in the heading of this Notice. If an application is received after that date, it will be returned to the applicant without review. Although there is no immediate acknowledgment of the receipt of an application, applicants are generally notified of the review date within 8 weeks. PEER REVIEW PROCESS Applications that are complete will be evaluated for scientific and technical merit by an appropriate peer review group convened by the NHLBI in accordance with the process stated below. PROGRAMS OF EXCELLENCE IN GENE THERAPY APPLICATIONS Each project will receive a priority score. The scientific merit of each preclinical and clinical project will be evaluated using the standard review criteria (Significance, Approach, Innovation, Investigator, and Environment). Each core (except the National Cores) will be Recommended or Not Recommended based on whether the core is essential to the program and has the capability to fulfill the proposed function. Reviewers will evaluate the number of projects serviced by the core; strengths and weaknesses of the proposed approaches, resources, and interactions; and whether the investigators are qualified for their role(s). Each National Core will be assigned a priority score using the review criteria listed below; this score will have no effect on the overall score of the application. REVIEW CRITERIA FOR A PEGT APPLICATION 1) Scientific merit of the preclinical and clinical projects, including the progress and proposed plans. 2) Multidisciplinary nature of the proposed studies, the coordination and synergy between basic and clinical researchers, and the likelihood that the application will enable the translation of basic research to clinical application. 3) Quality of the training program, including whether the trainees have been adequately trained, and whether there are plans for assessing the quality and benefit of the educational opportunities offered. 4) Access to patients for collaborative clinical research, and demonstrated willingness of the investigators to work as part of the collaborative clinical research component with the NHLBI Project Scientist. REVIEW CRITERIA FOR NATIONAL CORES National Cores will be evaluated as to the services provided and their accessibility to NHLBI-supported investigators. The evaluation of each National Core includes the following: 1) Significance: Does the National Core address an important area, and have the potential to provide an impact? 2) Progress: Have the services and/or resources produced by this Core been beneficial to the scientific community? 3) Plans: Are the proposed plans adequately developed and feasible? Are the plans for assessing the quality and benefit of these services and/or resources reasonable? REVIEW CRITERIA FOR THE ADMINISTRATIVE COORDINATING CENTER 1) Quality of the plans for coordination and collaboration of the activities with NHLBI Gene Therapy by setting up meetings for the Programs of Excellence in Gene Therapy (including activities of the Steering Committee, NHLBI Gene Therapy Data Safety and Monitoring Board, and Grantees Meeting); the adequacy of the proposed plan to organize communications within the program via websites, e-mail listserves, and conference calls. 2) Quality of the administrative, supervisory, and collaborative arrangements for achieving the goals of the program, including willingness to cooperate with the participating Centers and the NHLBI; quality of the proposed organizational structure and management plan. 3) Availability of the facilities and equipment to effectively coordinate the activities of the Programs in Excellence in Gene Therapy, and assist customers in providing National Core services. ADDITIONAL REVIEW CRITERIA FOR ALL APPLICATIONS: In addition to the above criteria, the following items will be considered in the determination of scientific merit and the priority score: PROTECTION OF HUMAN SUBJECTS FROM RESEARCH RISK: The involvement of human subjects and protections from research risk relating to their participation in the proposed research will be assessed. INCLUSION OF WOMEN, MINORITIES AND CHILDREN IN RESEARCH: The adequacy of plans to include subjects from both genders, all racial and ethnic groups (and subgroups), and children as appropriate for the scientific goals of the research. Plans for the recruitment and retention of subjects will also be evaluated. CARE AND USE OF VERTEBRATE ANIMALS IN RESEARCH: If vertebrate animals are to be used in the project, the five items described under Section f of the PHS 398 research grant application instructions (rev. 5/2001; updated 09/09/2003) will be assessed. BUDGET: The reasonableness of the proposed budget and the requested period of support in relation to the proposed research. AWARD CRITERIA Factors that will be considered in making awards include: o Scientific merit (as determined by peer review) o Availability of funds o Programmatic priorities. SCHEDULE Application Receipt Date: October 21, 2004 Peer Review: February - March 2005 Council Review: June 16, 2005 Start Date: September 1, 2005 INQUIRIES o Direct your questions about scientific/programmatic issues to: Sonia I. Skarlatos, Ph.D. Deputy Director Division of Heart and Vascular Diseases National Heart, Lung, and Blood Institute Rockledge II, Room 9158 6701 Rockledge Drive Bethesda, MD 20892-7940 Telephone: (301) 435-0477 FAX: (301) 480-7971 email: skarlats@nhlbi.nih.gov o Direct your questions about peer review issues to: Chief, Review Branch National Heart, Lung, and Blood Institute National Institutes of Health 6701 Rockledge Drive, Room 7214 Bethesda, MD 20892-7924 Bethesda, MD 20817 (for express/ courier service) Telephone: 301-435-0270 FAX: 301-480-0730 o Direct your questions about financial or grants management issues to: Ms. Vicki Maurer Grants Operations Branch Division of Extramural Affairs National Heart, Lung, and Blood Institute Rockledge II, Room 7164, MSC 7926 6701 Rockledge Drive Bethesda, MD 20892-7926 Telephone: (301) 435-0185 FAX: (301) 480-0422 email: maurerv@nhlbi.nih.gov


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