3. Class III-C experiments

Class III-C experiments require IBC and Institutional Review Board (IRB) approvals and RAC review before research participant enrollment. These experiments involve:

  1. The deliberate transfer of recombinant or synthetic nucleic acids, or DNA or RNA derived from recombinant or synthetic nucleic acid molecules, into one or more human research participants.

Human gene transfer is the deliberate transfer into human research participants of either:

  • Recombinant nucleic acid molecules, or DNA or RNA derived from recombinant nucleic acid molecules, or
  • Synthetic nucleic acid molecules, or DNA or RNA derived from synthetic nucleic acid molecules, that meet any one of the following criteria:
    • Contain more than 100 nucleotides; or
    • Possess biological properties that enable integration into the genome (e.g., cis elements involved in integration);
      or
    • Have the potential to replicate in a cell; or
    • Can be translated or transcribed.

PIs shall submit relevant information on the proposed human gene transfer experiments for RAC review and discussion; see Appendix M-I-B-2, Public RAC Review and Discussion. After a human gene transfer experiment is reviewed by the RAC at a regularly scheduled meeting, NIH OBA will send a letter within 10 working days to the NIH Director, the PI, the sponsoring institution, and other DHHS components, as appropriate, summarizing RAC comments and recommendations (if any).

For a clinical trial site that is added after the RAC review process, no research participant shall be enrolled at the clinical trial site until the following documentation has been submitted to the NIH OBA:

  • IBC approval (from the clinical trial site);
  • IRB approval;
  • IRB-approved informed consent document;
  • Curriculum vitae of the PI’s (no more than 2 pages in biographical sketch format); and
  • NIH grant number(s), if applicable.

In order to maintain public access to information regarding human gene transfer protocols, NIH OBA will maintain the documentation described in Appendices M-I through M-V. The information provided in response to Appendix M should not contain any confidential commercial information or trade secrets, enabling all aspects of RAC review to be open to the public.