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Guidance for FDA Reviewers and Sponsors: Content and Review of Chemistry, Manufacturing, and Control (CMC) Information for Human Gene Therapy Investigational New Drug Applications (INDs)

[PDF Printable Version - 251 KB]

Additional copies of this guidance are available from the Office of Communication, Training, and Manufacturers Assistance (HFM-40), 1401 Rockville Pike, Suite 200N, Rockville, MD 20852-1448, or by calling 1-800-835-4709 or 301-827-1800, or from the Internet at http://www.fda.gov/cber/guidelines.htm.

For questions on the content of this guidance, contact the Office of Cellular, Tissue, and Gene Therapies at 301-827-5102.

U.S. Department of Health and Human Services
Food and Drug Administration
Center for Biologics Evaluation and Research
April 2008

Contains Nonbinding Recommendations


Table of Contents
I.Introduction A.How will FDA Reviewers and Sponsors Use this Guidance?
B.How is this Guidance Organized?

II.Administrative Information to be Documented by FDA Reviewers
III.Product Manufacturing and Characterization Information to be submitted by sponsors and Documented by FDA reviewers A.Product Manufacturing - Components and Materials 1.Vector
2.Cells
3.Reagents
4.Excipients
5.Additional Considerations

B.Product Manufacturing - Procedures 1.Vector Production/Purification
2.Preparation of ex Vivo Gene-Modified Autologous or Allogeneic Cells
3.Process Timing and Intermediate Storage
4.Final Formulation


IV.Product Testing A.Microbiological Testing 1.Sterility Testing (Bacterial and Fungal Testing)
2.Mycoplasma
3.Adventitious Agent Testing

B.Identity
C.Purity 1.Residual Contaminants
2.Pyrogenicity/Endotoxin

D.Potency
E.Other 1.General Safety Testing
2.Viability
3.Cell Number/Dose


V.Final Product Release criteria Testing
VI.Product Stability
VII.Other Issues A.Product Tracking
B.Labeling
C.Container/Closure
D.Environmental Impact
E.Qualification of the Manufacturing Process
F.Biostatistics

VIII.Preclinical Studies To Be Documented By FDA Reviewers A.Summary of Concept Studies
B.Gonadal Distribution

IX.Clinical Studies To Be Documented By FDA Reviewers A.Protocol Title
B.Subject Population
C.Route of Administration
D.Dose
E.Frequency
F.Genetic, Biochemical, and Immunological Testing
G.Informed Consent
H.Recombinant DNA Advisory Committee (RAC) Review

X.Recommendation To Be Determined By FDA Reviewers
XI.Comments to Sponsor Generated By FDA Reviewers A.Clinical Hold
B.Non-Clinical Hold

XII.References

Appendix A - Product Review Template (Gene Therapy)

Appendix B - Considerations for Development of Final Product Release Criteria Specifications and Stability Protocols
A.Development of Release Acceptance Criteria
B.Development of Acceptance Criteria Analytical Procedures
C.Development of Stability Protocols

http://www.fda.gov/BiologicsBloodVaccines/GuidanceComplianceRegulatoryInformation/Guidances/CellularandGeneTherapy/ucm072587.htm#ProductManufacturinga