"Hard cell": Potency testing for cellular therapy products

BioProcess International

Volumn 10, Issue SUPPL. 3, 2012, Pages 36-48

  Pritchett, Tom ^a   Little, Laureen ^b  

^a BioPharmaceutical Emerging Best Practices Association (BEBPA)   (United States)

^b NONE

Author keywords

[No Author keywords available]

Indexed keywords

7 AMINODACTINOMYCIN; APLIGRAF; INTERCELLULAR ADHESION MOLECULE 1; REMESTEMCEL L; SIPULEUCEL T; TRYPAN BLUE; UNCLASSIFIED DRUG; VASCULOTROPIN;

BIOREACTOR; CASTRATION RESISTANT PROSTATE CANCER; CELL THERAPY; CROHN DISEASE; CRYOPRESERVATION; DIABETIC FOOT; DRUG POTENCY; DRUG SCREENING; ENZYME LINKED IMMUNOSORBENT ASSAY; FLOW CYTOMETRY; FOOD AND DRUG ADMINISTRATION; GENE THERAPY; GOOD MANUFACTURING PRACTICE; GRAFT VERSUS HOST REACTION; HAPLOTYPE; HUMAN; IMMUNOPHENOTYPING; KERATINOCYTE; LEG ULCER; MESENCHYMAL STEM CELL TRANSPLANTATION; NONHUMAN; ORGANOGENESIS; PROTEIN EXPRESSION; PUBLIC HEALTH SERVICE; REVIEW; UPREGULATION;

EID: 84863557402     PISSN: 15426319     EISSN: None     Source Type: Trade Journal    
DOI: None     Document Type: Review

References (23)

1. Title 21 of the United States Code of Federal Regulations, numerous parts including: 210, 211, 312, 600, and 1271.
2. CBER. Guidance for Industry, Potency Tests for Cellular and Gene Therapy Products, January 2011.
3. Stroncek DF. Potency Analysis of Cellular Therapies: The Emerging Role of Molecular Assays. J. Transl. Med. 5(24) 2007: 45.
4. CBER. Guidance for Reviewers: Instructions and Template for Chemistry, Manufacturing, and Control (CMC) Reviewers of Human Somatic Cell Therapy Investigational New Drug Applications (INDs), April 2008.
5. ICH Q5E. Comparability of Biotechnological/Biological Products Subject to Changes in Their Manufacturing Process. US Fed. Reg. 70(125) 2005: 37861-37862.
6. Gavin DK (CBER Office of Cellular, Tissue and Gene Therapies, Division of Cellular and Gene Therapies). Presentation at the Cellular, Tissue, and Gene Therapies Advisory Committee Meeting, 9 February 2006. Slides and transcript available at www.fda.gov/ohrms/dockets/ac/cber06. html#CellularTissueGeneTherapies.
7. CBER. Guidance for FDA Reviewers and Sponsors: Content and Review of Chemistry, Manufacturing, and Control (CMC) Information for Human Somatic Cell Therapy Investigational New Drug Application (INDs), 2008.
8. Ritter N, et al. What Is Test Method Qualification? BioProcess Int. 2(8) 2004: 32- 47; www.bioprocessintl.com/multimedia/archive/00078/0208ar05-78564a. pdf.
9. Provost N. Potency Testing for an Autologous Cellular Immunotherapy. Presentation at the 9 February 2006 meeting of the Cellular, Tissue, and Gene Therapies Advisory Committee.
10. Based on FDA remarks, titled Cellular Therapy Products. 2006 Advisory Committee meeting on stability testing (presenter not listed); www.fda.gov/downloads/BiologicsBloodVaccines/InternationalActivities/UCM273197. pdf.
11. Benton K (CBER Division of Cellular and Gene Therapies, Office of Cellular, Tissue, and Gene Therapies). Characterization of Cell/Scaffold Products. Presentation at the Workshop on In VitroAnalysis of Cell/Scaffold Products, 6 December 2007.
12. Advisory Committee (CTGTAC) Meeting. Potency Measurements for Cellular and Gene Therapy Products, Cellular, Tissue and Gene Therapies. Gaithersburg Hilton, 9 February 2006. Slides and transcript available at www.fda.gov/ohrms/ dockets/ac/cber06.html#CellularTissueGeneTherapies.
13. US Pharmacopeia 28. Chapter 〈111〉 Design and Analysis of Biological Assays. United States Pharmacopeial Convention, Inc., Rockville, MD.
14. Final Questions for Committee Discussion of BLA 125400. Cellular, Tissue and Gene Therapies Advisory Committee Meeting, 17 November 2011; www.fda.gov/AdvisoryCommittees/CommitteesMeetingMaterials/ BloodVaccinesandOtherBiologics.
15. Sponsor-Supplied Briefing Document for Cellular, Tissue and Gene Therapies Advisory Committee Meeting, 17 November 2011; www.fda.gov/ AdvisoryCommittees/CommitteesMeetingMaterials/BloodVaccinesandOtherBiologics/ cellularTissueandGeneTherapiesAdvisoryCommittee/ucm279851.htm.
16. FDA Briefing Document for Cellular, Tissue and Gene Therapies Advisory Committee Meeting, 17 November 2011; www.fda.gov/AdvisoryCommittees/ CommitteesMeetingMaterials/BloodVaccinesandOtherBiologics/ cellularTissueandGeneTherapiesAdvisoryCommittee/ucm279851.htm.
17. Finn T. Summary Basis for Regulatory Action, PROVENGE (sipuleucel-T), 1 May 2010.
18. Danilkovitch A. Prochymal, The Dynamics of a New Age in Medicine. Presentation at the February 9, 2006 meeting of the Cellular, Tissue, and Gene Therapies Advisory Committee. Slides and transcript available at www.fda.gov/ohrms/dockets/ac/cber06.html#CellularTissueGeneTherapies (or access the slides only from the fourth link on www.fda.gov/ohrms/dockets/ac/06/slides/ 2006-4205s1.htm.)
19. Statement of L Wolfe, Genzyme, at the 9 February 2006 meeting of the Cellular, Tissue, and Gene Therapies Advisory Committee; www.fda.gov/ohrms/ dockets/ac/06/transcripts/2006-4205T1.pdf.
20. ICH Q6B. Guidance on Specifications: Test Procedures and Acceptance Criteria for Biotechnological/Biological Products. US Fed. Reg. 18 August 1999: 44928.
21. CBER. Guidance for Industry: Minimally Manipulated, Unrelated Allogeneic Placental/Umbilical Cord Blood Intended for Hematopoietic Reconstitution for Specified Indications, 2009.
22. FDA Briefing Document for Cellular, Tissue and Gene Therapies Advisory Committee Meeting, 17 November 2011: www.fda.gov/AdvisoryCommittees/ CommitteesMeetingMaterials/BloodVaccinesandOtherBiologics/ cellularTissueandGeneTherapiesAdvisoryCommittee/ucm279851.htm.
23. Butman BT. Potency Assays for Adenovector-Based Therapies. Presentation at the 9 February 2006 meeting of the Cellular, Tissue, and Gene Therapies Advisory Committee; www.fda.gov/ohrms/dockets/ac/06/transcripts/2006-4205T1.pdf.