The Institute for Human Gene Therapy (IHGT) and Drs. Mark Wilson, Mark Batshaw, and Steven Raper

Follow this link to find news coverage, public statements from The Institute for Human Gene Therapy, FDA warning letters, Office for Human Research Protections determination letters, and an update on Paul.

This page has civil settlement agreements, information on NIH grants awarded to Drs. Wilson, Raper, and Batshaw, links to transcripts of meetings of the National Institutes of Health Recombinant DNA Advisory Committee, and related documents.

Clinical Trials

Phase I Pilot Study of Liver-Directed Gene Therapy for Partial Ornithine Transcarbamylase Deficiency. Study chairs or principal investigators: Mark Batshaw M.D. Sponsors: National Institute of Child Health and Human Development (NICHD) and Children's National Medical Center. Record first received on 1999-10-18. Available from http://www.clinicaltrials.gov/ct/show/NCT00004386?order=2

Phase I Study of Adenoviral Vector Mediated Gene Transfer for Ornithine Transcarbamylase in Adults With Partial Ornithine Transcarbamylase Deficiency. Sponsors: FDA Office of Orphan Products Development and the University of Pennsylvania. Study chairs or principal investigators: Steven Raper M.D. Record first received on 1999-10-18. Available from http://www.clinicaltrials.gov/ct/show/NCT00004498?order=1

U.S. Patents

• United States Patent 5,240,846: Gene therapy vector for cystic fibrosis
• United States Patent 5,856,152: Hybrid adenovirus-AAV vector and methods of use therefore
• United States Patent 6,475,769: Methods and cell line useful for production of recombinant adeno-associated viruses
• 32 additional United States Patents assigned to the the University of Pennsylvania by James Wilson M.D., Ph.D. and colleagues

National Institutes of Health Recombinant DNA Advisory (RAC) Committee

One consequence of Jesse's death – and his father's persistent dedication to research protection advocacy – was the creation of a public adverse event database for gene transfer studies. This isn't enough.

NIH Recombinant DNA Advisory (RAC) Committee. Meeting Minutes. 1995-06-08 and 09. Available from http://www4.od.nih.gov/oba/rac/minutes/6-8-9-95.htm

NIH Recombinant DNA Advisory (RAC) Committee. Meetings and Conferences. Available from http://www4.od.nih.gov/oba/rac/meeting.html

A Phase I Study of Adenoviral Vector Mediated Gene Transfer to Liver in Adults with Partial Ornithine Transcarbamylase Deficiency. Protocol 9512-139. Post-mortem. Minutes of the NIH Recombinant DNA Advisory (RAC) Committee. 1999-12-08 through 10. Available from http://www4.od.nih.gov/oba/rac/minutes/1299rac.pdf

NIH Recombinant DNA Advisory (RAC) Committee Working Group on Current Issues in Adverse Event Reporting. Post-mortem/Fallout. 2000-03-08 through 10. Available from http://www4.od.nih.gov/oba/rac/minutes/RACmin3-00.htm#XII

NIH Recombinant DNA Advisory (RAC) Committee Working Group on Adenovirus Safety and Toxicity. Fallout cont. 2000-03-08 through 10. Available from http://www4.od.nih.gov/oba/rac/minutes/RACmin3-00.htm#XVIII

Initial Review of A Phase I Study of Adenoviral Vector Mediated Gene Transfer to Liver in Adults with Partial Ornithine Transcarbamylase Deficiency. Protocol 9512-139. Minutes of the NIH Recombinant DNA Advisory (RAC) Committee. 1995-12-04 and 05. Available from http://www4.od.nih.gov/oba/rac/minutes/124-5-95.htm

Mark Batshaw M.D. Report of subject death in Protocol 9512-139. 1999-09-20. Safety Reports and Adverse Events for Human Gene Transfer Protocols. NIH Recombinant DNA Advisory Committee Meeting. 1999-12-08 through 10. Available from http://www4.od.nih.gov/oba/rac/SAE_rpts/mod1299s.htm

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General Information Resources

NIH Office of Biotechnology Activities

Frequently Asked Questions (FAQs) about the NIH Review Process for Human Gene Transfer Trials. NIH Recombinant DNA Advisory (RAC) Committee.

NIH Guidance on Informed Consent For Gene Transfer Research. NIH Recombinant DNA Advisory (RAC) Committee.

Additional Resources for Informed Consent & Gene Transfer Research. NIH Recombinant DNA Advisory (RAC) Committee. (Excellent bibliography.)

FDA/CBER Cellular and Gene Therapy Publications

Disclosure of Information OTC Gene Therapy. 1999-12-01.

Genetic Modification Clinical Research Information System (GeMCRIS)

NIH/FDA Genetic Modification Clinical Research Information System (GeMCRIS). (Information)

GeMCRIS Public Information Site. (Search)

Scientific Abstract. A Phase I Study of Adenoviral Vector Mediated Gene Transfer to Liver in Adults with Partial Ornithine Transcarbamylase Deficiency. Protocol 9512-139. Available from http://www.gemcris.od.nih.gov/Abstracts/139_s.pdf

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National Institutes of Health Grants to James Wilson M.D., Ph.D.

Notes

1. 1993 NIH awards accessed 2005-02-27 at http://grants1.nih.gov/grants/award/state/FY1993.pennsylv.txt
2. 1994 NIH awards accessed 2005-02-27 at http://grants1.nih.gov/grants/award/state/FY1994.pennsylv.txt
3. 1995 NIH awards accessed 2005-02-27 at http://grants1.nih.gov/grants/award/state/FY1995.pennsylv.txt
4. 1996 NIH awards accessed 2005-02-27 at http://grants1.nih.gov/grants/award/state/FY1996.pennsylv.txt
5. 1997 NIH awards accessed 2005-02-27 at http://grants1.nih.gov/grants/award/state/FY1997.pennsylv.txt
6. 1998 NIH awards accessed 2005-02-27 at http://grants1.nih.gov/grants/award/state/fy1998.pennsylv.txt
7. 1999 NIH awards accessed 2005-02-27 at http://grants1.nih.gov/grants/award/state/fy1999.pennsylv.txt
8. 2000 NIH awards accessed 2005-02-27 at http://grants1.nih.gov/grants/award/state/fy2000.pennsylv.txt

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Summaries of National Institutes of Health Grants Awarded to James Wilson M.D., Ph.D.

1. 5U42RR011151-03: National Gene Vector Laboratory
2. 3U42RR011151-02S1: National Gene Vector Laboratory
3. 5U42RR011151-02: National Gene Vector Laboratory
4. 3U42RR011151-01S1: National Gene Vector Laboratory
5. 1U42RR011151-01: National Gene Vector Laboratory
6. 1T32DK007748-01: Gene Therapy Training Cystic Fibrosis/Genetic Diseases
7. 2R01HL049040-07: Xenograft Model of Cystic Fibrosis Therapy
8. 5R01HL049040-06: Xenograft Model of Cystic Fibrosis Therapy
9. 5R01HL049040-05: Xenograft Model of Cystic Fibrosis Therapy
10. 3R01HL049040-04S1: Xenograft Model of Cystic Fibrosis Therapy
11. 5R01HL049040-04: Xenograft Model of Cystic Fibrosis Therapy
12. 5R01DK047966-05: Gene Therapy of Urea Cycle Disorders
13. 5R01DK042193-05: Liver-Directed Gene Therapies
14. 1P50DK049136-040001: Biology of Adenovirus Mediated Gene Transfer to the Airway
15. 5P50DK049136-04: Gene Therapy for CF Specialized Center of Research
16. 5P50DK049136-03: Gene Therapy for CF Specialized Center of Research
17. 5P50DK049136-02: Gene Therapy for CF Specialized Center of Research
18. 5P50DK049136-01: Gene Therapy for CF Specialized Center of Research
19. 1P30DK050306-01A19004: Core Vector Facility
20. 5P30DK047757-059001: Core Vector Facility
21. 5P30DK047757-059004: Core Human Applications Laboratory
22. 5P30DK047757-050011: Pilot Study Ligand Mediated Approaches to Gene Transfer Into Airway Epithelium
23. 5P30DK047757-05: Gene Therapy for Cystic Fibrosis and Other Genetic Dis
24. 5P30DK047757-04: Gene Therapy for Cystic Fibrosis and Other Genetic Dis
25. 5P30DK047757-03: Gene Therapy for Cystic Fibrosis and Other Genetic Dis
26. 5P30DK047757-02: Gene Therapy for Cystic Fibrosis and Other Genetic Dis
27. 5P01HD032649-039001: Core Recombinant Viruses
28. 5P01HD032649-029001: Core Recombinant Viruses
29. 1P01HD032649-019001: Core Recombinant Viruses
30. 5P01HD032649-030001: Viral Mediated Gene Therapy for Ornithine Transcarbamylase Deficiency
31. 5P01HD032649-020001: Viral Mediated Gene Therapy for Ornithine Transcarbamylase Deficiency
32. 1P01HD032649-010001: Viral Mediated Gene Therapy for Ornithine Transcarbamylase Deficiency
33. 1P01CA066726-039001: Core Human Application
34. 5P01CA066726-029001: Core Human Application
35. 1P01AR043648-030001: Vectors for Gene Therapy of Duchennes Muscular Dystrophy
36. 5P01AR043648-020001: Vectors for Gene Therapy of Duchennes Muscular Dystrophy
37. 5M01RR000240-330396: Ex Vivo Gene Therapy for Familial Hypercholesterolemia
38. 2M01RR000240-320396: Ex Vivo Gene Therapy for Familial Hypercholesterolemia
39. 5M01RR000240-310396: Ex Vivo Gene Therapy for Familial Hypercholesterolemia
40. 5M01RR000240-300396: Ex Vivo Gene Therapy for Familial Hypercholesterolemia
41. 5M01RR000040-370542: Ex Vivo Gene Therapy for Familial Hypercholesterolemia
42. 5M01RR000040-360542: Ex Vivo Gene Therapy for Familial Hypercholesterolemia

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Summaries of National Institutes of Health Grants Awarded to Mark Batshaw M.D.

1. 1P01HD032649-01: Gene Therapy for Ornithine Transcarbamylase Deficiency
2. 5P01HD032649-02: Gene Therapy for Ornithine Transcarbamylase Deficiency
3. 5P01HD032649-03: Gene Therapy for Ornithine Transcarbamylase Deficiency
4. 5P01HD032649-04: Gene Therapy for Ornithine Transcarbamylase Deficiency
5. 5P01HD032649-05: Gene Therapy for Ornithine Transcarbamylase Deficiency
6. 3P01HD032649-05S1: Gene Therapy for Ornithine Transcarbamylase Deficiency
7. 1P01HD032649-010003: Human Studies of Ornithine Transcarbamylase Deficiency
8. 5P01HD032649-020003: Human Studies of Ornithine Transcarbamylase Deficiency
9. 5P01HD032649-030003: Human Studies of Ornithine Transcarbamylase Deficiency
10. 5P01HD032649-040003: Human Studies of Ornithine Transcarbamylase Deficiency
11. 5P01HD032649-050003: Human Studies of Ornithine Transcarbamylase Deficiency
12. 3P01HD032649-05S10003: Human Studies of Ornithine Transcarbamylase Deficiency

Summaries of National Institutes of Health Grants Awarded to Steven Raper M.D.

1. 5M01RR000040-390673: Gene Therapy for Ornithine Transcarbamylase Deficiency
2. 5P01DK049814-029002: Core Vector Facility
3. 5P01DK049814-039002: Core Vector Facility
4. 5P01DK049814-049002: Core Vector Facility
5. 5P01DK049814-059002: Core Vector Facility
6. 1P01HD032649-019002: Core Animal Models
7. 5P01HD032649-029002: Core Animal Models
8. 5P01HD032649-039002: Core Animal Models
9. 5P01HD032649-049002: Core Animal Models
10. 5P01HD032649-059002: Core Animal Models
11. 3P01HD032649-05S19002: Core Animal Models

Accessed 2005-02-27 from Computer Retrieval of Information on Scientific Projects (CRISP)

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U.S. Department of Justice Settlement Agreements

U.S. Settles Case of Gene Therapy Study That Ended With Teen's Death. United States Attorney's Office Press Release. 2005-02-09. Available from http://www.usdoj.gov/usao/pae/News/Pr/2005/feb/UofPSettlement%20release.html

Mark Batshaw M.D., and Children's National Medical Center (CNMC). U.S. Department of Justice Settlement Agreement. 2005-02-09. Available from http://www.circare.org/foia3/batshawsettlement.pdf

Institute for Human Gene Therapy, University of Pennsylvania. U.S. Department of Justice Settlement Exhibit. Promoting Safety in Clinical Research at the University of Pennsylvania. 2005-02-09. Available from http://www.circare.org/foia3/ihgt_exhibittosettlement.pdf

Institute for Human Gene Therapy, University of Pennsylvania. U.S. Department of Justice Settlement Agreement. 2005-02-09. Available from http://www.circare.org/foia3/IHGT_settlement_5.pdf

Steven Raper M.D. U.S. Department of Justice Settlement Agreement. 2005-02-09. Available from http://www.circare.org/foia3/raper_settlement.pdf

Steven Raper M.D. U.S. Department of Justice Settlement Exhibit 1: Medical Monitor. 2005-02-09. Available from http://www.circare.org/foia3/RAPER_exhibit_2.pdf

James Wilson M.D, Ph.D. U.S. Department of Justice Settlement Agreement. 2005-02-09. Available from http://www.circare.org/foia3/wilson5_settlementagreement.pdf

James Wilson, M.D., Ph.D. U.S. Department of Justice Settlement Exhibit 1: Medical Monitor. 2005-02-09. Available from http://www.circare.org/foia3/wilson_exhibit2.pdf

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Last Updated: 2006-03-19

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