Recombinant DNA Advisory Committee - 3/3-4/94 
accepted for clinical investigation by the FDA? What was the date of IND submission? 
Is there a sufficient supply of the clinical grade material available to complete the 
proposed study? If clinical grade material is unavailable, on what date will such material 
become available?" These additional questions that will be incorporated into the 
Expedited Review cover sheet will be included as item number 1 1 under Section B, 
Vector, Target Cell, and Transduction Procedures. 
The RAC-approved version of the Accelerated Review cover sheet reads: 
PROPOSED COVER SHEET FOR ACCELERATED REVIEW 
OF A HUMAN GENE TRANa^ EXPERIMENT 
A. 
BACKGROUND 
1. 
Provide the following information about the proposed study, title, principal investigators, and participating 
institutions. 
a 
VECTOR, TARGET CELL, AND TRANSDUCDON PROCEDURES 
1. 
What are the proposed gene and vector for this protocol? 
2. 
Has the proposed vector previously been approved by the RAC? If so, provide the title and principal 
invest igator(s) of at least one RAC-approved protocol utilizing this vector. If not, attach the complete vector 
sequence (hard copy and a 3Vi inch diskette in ASCII format). 
3. 
Who is the vector supplier? 
4. 
What is the target cell to be transduced by the proposed vector? 
5. 
What is the rate of transduction of the proposed vector and target cells in the proposed setting (i.e., your 
laboratory)? 
6. 
What is the level of gene expression demonstrated in the target cell? 
7. 
How was gene expression determined? 
8. 
What assay was used to detect replication-competent virus (RCR)? 
9. 
Was RCR detected? 
10. 
What is the level of sensitivity of the RCR assay? (Attach documentation). 
c 
CLINICAL PROTOCOL 
1. 
What in vitro or in vivo svstemfsl were used to determine nreclinical efficacy? 
2. 
What is the end point of the protocol? 
3. 
Is the proixjsed study identical to another RAC-approved protocol except that the study will be performed at 
a satellite institution? If so, provide a letter of cross-reference from the sponsoring institution. 
4. 
Is the proposed study similar to another RAC-approved protocol? Identify the maior differences. 
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Recombinant DNA Research, Volume 19 
