Columbia University's Institutional Biosafety Committee

Original: June, 2000. Revised: October, 2006; December, 2010; April, 2011; August, 2012; September, 2012, December 2012Page 5 of 7

Addendum 2:Human Gene TransferMeetings For time sensitive proposals, an ad hoc meeting will be convened. HGT review meetings will require the agreement of the IBC’s designated HGT expert. If the designated HGT expert is not able toattend the meeting in person, the Biosafety Officer will obtain feedback regarding the HGT protocol from the designated HGTexpert in advance of the meeting, and will relay this information to the Committee at the meeting.NOTE: The Guidelines delineate different requirements for ‘sponsors’ and ‘additional clinical trial sites’. It is anticipated that theUniversity’s status will remain exclusively as an ‘additional clinical trial site’.Submittal RequirementsPrincipal Investigators (PIs) must submit to the IBC: A completed Columbia University Institutional Biosafety Committee Application for the use of Recombinant DNA (rDNA)Molecules in Human Gene Transfer, which will indicate whether the PI or the Sponsor shall be responsible for the reportingrequirements of the Guidelines. If the Sponsor (not the PI) will be responsible for the reporting requirements, PI must submit a copy of the letter sent to the OBAof this delegation. The scientific abstract. The abstract from the grant proposal may be used. A copy of the Clinical Investigator’s Brochure. The Informed Consent Document that is currently (or will be) under consideration by the University’s Institutional Review Board(IRB). The Sponsor’s NIH submittal as per Appendix M of the rDNA Guidelines, Considerations for Human Gene Therapy. A copy of the NIH’s response to the Sponsor’s Appendix M submittal. If the aforementioned response, ‘Outcome of the Initial Review by the Recombinant DNA Activities Committee’, indicatescomments by RAC members, the Sponsor’s reply to the comments must be provided to the IBC.Voting & Approval Procedures All the materials in the Submittal Requirements section shall be sent to the IBC at least five days prior to the meeting. At the meeting, a member of the IBC shall present the proposal to the Committee. The presentation will include:o the clinical trial phase of the projecto the condition being addressedo a description of the vector, the gene product(s) to be expressed and how this might have a positive impact on studysubjecto whether viral replication is expected if a viral vector is being usedo any relevant information on adverse events from prior clinical or pre-clinical descriptionso the considerations to ensure that recombinant DNA or its products are not spread to personal contacts of trial subjects orthe community If a proposal is approved, the Secretary will inform the PI and the IRB. If a proposal is not approved, the Secretary will contact the PI and request further information or clarification as deemednecessary by the Committee for approval. For proposals where further information or clarification will be required for approval, the Committee shall determine if theresponses are to be brought to the next Committee meeting for a re-vote or if the Secretary may provide approval on behalf of theCommittee. In the latter case, the Secretary is responsible for reintroducing the proposal to the Committee if the PI’s responses do not providesufficient clarification or modification. Minutes of HGT discussion shall include:o any questions or issues brought up during the discussion and how they were addressedo whether the proposal was approved during the meeting or if approval was made contingent on the provision of additionalinformationo whether the Secretary was allowed to grant approval on behalf of the Committee or if discussion was to be continued at asubsequent meetingo any minority viewpoints presented, if the approval is not unanimousReporting Requirements Upon approval as an “Additional Clinical Trial Site”, the PI shall be notified by the IBC of reporting requirements in thefollowing areas:o enrollment (the signing of an Informed Consent form), which must be preceded by the PI’s submission to OBA of• IBC approvalOriginal: June, 2000. Revised: October, 2006; December, 2010; April, 2011; August, 2012; September, 2012, December 2012Page 6 of 7

oo• IRB approval and the IRB-approved informed consent document• Curriculum Vitae (CV) of the PI• NIH grant numbers if applicableannual reporting, which is typically done by the Sponsor, from data supplied from multiple clinical trial sites, willinclude• clinical trial information• progress report and data analysis• a copy of the updated clinical protocol, if applicablesafety reporting as applicable to any serious adverse event that is unexpected and associated (reasonable possibility) withthe use of the gene transfer product.The PI remains responsible for fulfilling safety reporting requirements even if this has been formally delegated to the Sponsor.(This requirement may be met by having the PI copy OBA on safety reports sent to the Sponsor.)Original: June, 2000. Revised: October, 2006; December, 2010; April, 2011; August, 2012; September, 2012, December 2012Page 7 of 7