Text ::   Skip Nav ::

CIRCARE logo


Brochure Contact Home Privacy Site Map Terms of Use Tools Prev

Link to CIRCARE Link To ::

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: 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: http://www.clinicaltrials.gov/ct/show/NCT00004498?order=1

U.S. Patents

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: http://oba.od.nih.gov/oba/rac/minutes/6-8-9-95.pdf

NIH Recombinant DNA Advisory (RAC) Committee. Meetings and Conferences: http://oba.od.nih.gov/rdna_rac/rac_meetings.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: http://oba.od.nih.gov/oba/rac/minutes/1299rac.pdf
Data Management Report: http://oba.od.nih.gov/oba/rac/SAE_rpts/mod1299s.pdf
Protocol Amendments, Updates: http://oba.od.nih.gov/oba/rac/amendments/Mod399a.pdf

NIH Recombinant DNA Advisory (RAC) Committee Working Group on Current Issues in Adverse Event Reporting. Post-mortem/Fallout, Minutes. 2000-03-08 through 10: http://oba.od.nih.gov/oba/rac/minutes/RACmin3-00.pdf
Data Management Report: http://oba.od.nih.gov/oba/rac/SAE_rpts/mgtrpt3-00.pdf

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: http://oba.od.nih.gov/oba/rac/minutes/124-5-95.pdf

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 Minutes. 1999-12-08 through 10: http://oba.od.nih.gov/oba/rac/minutes/1299rac.pdf
Data Management Report: http://oba.od.nih.gov/oba/rac/SAE_rpts/mod1299s.pdf
Protocol Amendments, Updates: http://oba.od.nih.gov/oba/rac/amendments/Mod1299a.pdf

top

General Information Resources

NIH Office of Biotechnology Activities

Recombinant DNA Program

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.

Educational Materials. NIH Recombinant DNA Advisory (RAC) Committee. (Selection of articles and other resources)

FDA Cellular and Gene Therapy Guidances

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), Index of 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

top

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

NIH Funds Awarded 1993-2000: $29,187,243 (not a complete list)

NIH 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

top

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

2010-04-09: these links no longer work; we appreciate your patience while we update them.

  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

top

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

2010-04-09: these links no longer work; we appreciate your patience while we update them.

  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.

2010-04-09: these links no longer work; we appreciate your patience while we update them.

  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)

top

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: http://web.archive.org/web/20050214120445/http://www.justice.gov/usao/pae/News/Pr/2005/feb/UofPSettlement+release.html

Mark Batshaw M.D., and Children's National Medical Center (CNMC). U.S. Department of Justice Settlement Agreement. 2005-02-09: 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: 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: http://www.circare.org/foia3/IHGT_settlement_5.pdf

Steven Raper M.D. U.S. Department of Justice Settlement Agreement. 2005-02-09: 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: 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: 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: http://www.circare.org/foia3/wilson_exhibit2.pdf


top

Last Updated: 2010-04-09

If you find the information on this page helpful please support CIRCARE with a tax-deductible contribution today. Because CIRCARE doesn't accept funds from pharmaceutical or medical device manufacturers, we depend on contributions from individuals like you to help us advocate for meaningful protection of human subjects in research. Find out more on our Support page


All material on this site © CIRCARE Incorporated (2002- ) or as indicated. Single copies can be downloaded for personal education.   Adobe Reader™ ::   ::

Valid HTML 4.01! Valid CSS!