DRAFT DRAFT DRAFT
BIOLOGICAL RESPONSE MODIFIERS ADVISORY COMMITTEE
Meeting #29
April 5-6, 2001
Holiday Inn, Bethesda, MD
Thursday, April 5
9:00 a.m. Call to Order
Dr. Daniel Salomon, Chair
9:10 Conflict of Interest Statement
Ms. Gail Dapolito, Executive Secretary
Session I
9:15 FDA Introduction
Overview of March 6, 2000 FDA Gene Therapy Letter
Product-Related Issues
Dr. Joyce Frey-Vasconcells
Division of Cellular and Gene Therapies, CBER
9:30 Responses to Gene Therapy Letter:
Multi-Use Facility, QA/QC Issues
Ms. Mary Malarkey
Division of Case Management, CBER
9:50 Questions from the Committee
10:00 Responses to FDA Gene Therapy Letter:
RCR and Different Packaging Cell Lines for Retroviral Vector Manufacture
Dr. Carolyn Wilson
Divison of Cellular and Gene Therapies, CBER
10:10 Committee Discussion
10:30 Break
10:45 Responses to FDA Gene Therapy Letter:
Testing of Plasmids as Manufacturing Intermediates in Gene Therapy Products
Dr. Suzanne Epstein
Division of Cellular and Gene Therapies, CBER
10:55 Committee Discussion
11:15 Responses to FDA Gene Therapy Letter :
Adenovirus Vector Titer Measurements and RCA Levels
Dr. Steven Bauer
Division of Cellular and Gene Therapies, CBER
Biological Response Modifiers Advisory Committee
Meeting #29
Thursday, April 5 (Cont'd)
11:35 Clinical Issues of Adenovirus Infection in Marrow Transplant Recipients
Dr. Stephen Chanock
Pediatric Oncology Branch, NCI, NIH
12:05 p.m. Committee Discussion
12:30 Lunch
Session II
1:30 p.m. Open Public Hearing
2:00 FDA Introduction
March 6, 2000 FDA Gene Therapy Letter
Preclinical and Clinical Issues
Dr. Karen Weiss, CBER
Division of Clinical Trial Design and Analysis, CBER
2:05 Responses to FDA Gene Therapy Letter:
Preclinical and Clinical Issues
Dr. Patricia Keegan
Division of Clinical Trial Design and Analysis, CBER
Results of Gene Therapy Clinical Site Inspections
Ms. Elaine Cole Mr. Joseph Salewski
Division of Inspections and Surveillance, CBER
3:05 Break
3:15 Committee Discussion
Update: CBER Intramural Research Programs
Division of Monoclonal Antibodies
4:15 Dr. Jay Siegel, Director
Office of Therapeutics Research and Review
4:20 Dr. Marjorie Shapiro
Laboratory of Molecular and Developmental Immunology
Biological Response Modifiers Advisory Committee
Meeting #29
Thursday, April 5 (Cont'd)
Division of Cellular and Gene Therapies
4:45 Dr. Philip Noguchi, Director
4:50 Dr. Thomas Eggerman
Laboratory of Molecular and Tumor Biology
5:15 Adjourn Open Session
5:30 Closed Session
6:00 Adjourn Closed Session
Friday, April 6
8:30 a.m. Call to Order
Dr. Daniel Salomon, Chair
8:45 Presentation of Certificate of Appreciation for Committee Service Dr. Kathryn Zoon, Director, CBER
Dr. Jay Siegel, Director, OTRR, CBER
Session III
9:00 FDA Introduction
Long-Term Follow-Up of Gene Therapy Patients
Dr. Philippe Bishop
Division of Clinical Trial Design and Analysis, CBER
9:05 UNOS Presentation
Dr. Mary Ellison
Mr. Berkeley Keck
United Network for Organ Sharing
9:20 a.m. FDA Presentation
Long-Term Follow-Up of Gene Therapy Patients Dr. Carolyn Wilson
Dr. Philippe Bishop
9:50 Break
10:00 Committee Discussion
11:00 Open Public Hearing
Biological Response Modifiers Advisory Committee
Meeting #29
Friday, April 6 (Cont'd)
Session IV
11:30 Introduction - FDA Proposed Rule
Availability for Public Disclosure and Submission to FDA for
Public Disclosure of Certain Data and Information Related
to Human Gene Therapy or Xenotransplantation
Dr. Philip Noguchi
12:00 noon Break
12:10 p.m. Committee Discussion
1:15 Adjourn