Investigators may submit an RSA after:

• Completing the online Investigator Registration application form on the NHLBI Gene Therapy Resource Program (GTRP) main webpage (http://www.gtrp.org) and
• Receiving registration approval by the NHLBI Gene Therapy Group (GTG) allowing him/her to submit an RSA to the GTRP.  Receipt of approval is predicated on the Investigator conducting scientific research
• In gene therapy for heart, lung, or blood diseases consistent with the NHLBI Mission (http://www.nhlbi.nih.gov/about/org/mission.htm) and
• With his/her primary gene therapy research site in the U.S.

What are the requirements for the various types of RSAs?

For all types of RSAs

• The scientific research in gene therapy that is the subject of the RSA must be consistent with the NHLBI Mission (http://www.nhlbi.nih.gov/about/org/mission.htm).
• The submitting investigator must be registered and approved as described under
  “Who Can Submit an RSA?”
• RSA approval and implementation is contingent upon availability of funds in the GTRP. 
• Services provided through an approved RSA are at no cost to the investigator.
• The various types of RSAs are independent of one another.  An applicant Investigator may submit any type of RSA for a qualified gene therapy research project regardless of past or future utilization of GTRP resources for the project.

In Addition

For Preclinical Vector Production

• The applicant must be working in basic or translational research in heart, lung, or blood disease(s).
• Funding must already be secured, and demonstrated in writing, for the project for which the vector is requested.
• View University of Pennsylvania, NHLBI GTRP Core Vector Laboratory webpage for further information. (http://www.gtrp.org/lab_penn.aspx).

For Pharmacology/Toxicology Studies

• The applicant must provide documentation of a pre-IND FDA meeting/review and FDA-specified pharmacology/toxicology testing recommendations.
• A brief description of the anticipated gene therapy clinical trial must be provided by the applicant.
• View Lovelace Biomedical and Environmental Research Institute, the NHLBI GTRP Core Pharmacology/ Toxicology Laboratory webpage for further information (http://www.gtrp.org/lab_lovelace.aspx).

For Clinical-Grade AAV or Lentiviral Vector Production

• The applicant must have adequate preclinical data and the requisite regulatory and other oversight reviews for the proposed clinical protocol in which the clinical-grade vector will be used.
• Funding must already be secured and documented for the proposed clinical trial.
• View the Children’s Hospital of Philadelphia, NHLBI GTRP Core AAV Clinical-grade Vector Laboratory webpage (http://www.gtrp.org/lab_aav.aspx) or the Indiana University, NHLBI GTRP Core Lentiviral Clinical-grade Vector Laboratory webpage (http://www.gtrp.org/lab_ind.aspx) for further information.

For Regulatory Support

• The applicant must be, or have been, an NHLBI grantee.  However, the gene therapy project for which the regulatory assistance is requested does not have to be part of an NHLBI grant.
• View the NHLBI GTRP Clinical Coordinating Center webpage for further information (http://www.gtrp.org/ccc.aspx).

 
For Phase I or II Clinical Trial Funding Assistance

• The applicant must:
  • Be an NHLBI investigator
  • Provide the clinical protocol for which funding is sought
  • Provide an implementation plan for recruitment and enrollment of research participants
  • Provide documentation of any secured financial support for the clinical trial
• View the NHLBI GTRP Clinical Coordinating Center webpage for further information (http://www.gtrp.org/ccc.aspx)

• Once approved as an Applicant Investigator by the NHLBI Gene Therapy Group (GTG) you will be notified by the Clinical Coordinating Center (CCC). You will use the User Name (email address) and password which you created during the Registration Application process to access the secure area of
  the website.
  
  
• To access the RSA forms and other information available to Approved Investigators, click on the “Request for Service Applications” menu item located near the top of the GTRP Home page. The initial information displayed is available to the public. Near the bottom of the page, click on Member Login. Use your User Name and password to Login and view the Investigator’s Main Page.

• Select the type of GTRP service you are interested in.  You will be asked to submit information specific to the service being requested.   From the Main GTRP webpage, you can access the webpage(s) for each of the service areas to determine the type of information that is required on the RSA form.  Go to the FAQ section of the specific service area webpage and click on “What type of information will an approved applicant investigator need to provide to the laboratory as part of the Request for Service Application?”

• You will be allowed to enter information and save your RSA in stages; you will initially submit the RSA as a “preliminary” submission. 

• Each RSA will be assigned a unique identifier at the time of the initial “save”.  Refer to this number in all correspondence or submission of supplemental forms and materials.

• You will be able to edit “preliminary” submissions. 

• Following your “preliminary” submission you must discuss your request with the respective laboratory or the clinical coordinating center (if applicable) before submitting it as a “final” RSA.  Only you, as the RSA author, will have edit rights to your preliminary RSA.  If needed, you may submit multiple “preliminary” versions of the RSA before submitting a “final” version.

• Once submitted as a “final” the RSA can not be edited.

• Once the RSA is submitted as “final” it enters a queue for review and subsequent actions.  The review process is described in the document “The Request for Service Application Review Process”. 

• Before your RSA can receive program review, you must submit the following additional forms to the clinical coordinating center via email.  These forms can be downloaded from the RSA page. 
   
  • Investigator and Institution Certifications and Agreement (for all vector production RSAs and the Pharmacology/Toxicology RSA)
    
    
  • Conflict of Interest Statement for Investigators (for all investigators submitting an RSA)