Division of Cellular and Gene Therapies DCGT Overview of Activities .


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Division of Cellular and Gene Therapies (DCGT) Overview of Activities. Raj K. Puri, M.D., Ph.D. Division Director, Division of Cellular and Gene Therapies FDA, Center for Biologics Evaluation and Research. Outline. Mission and organizational structure Regulatory scope Approach to research
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Division of Cellular and Gene Therapies (DCGT) Overview of Activities Raj K. Puri, M.D., Ph.D. Division Director, Division of Cellular and Gene Therapies FDA, Center for Biologics Evaluation and Research

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Outline Mission and authoritative structure Regulatory degree Approach to research Tumor Vaccines Biotechnology Branch (TVBB) Site Visit September, 2006

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OCTGT Structure

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DCGT Structure Division of Cellular and Gene Therapies Raj Puri, Ph.D., M.D., Division Director Kimberly Benton, Ph.D., Deputy Director Gene Therapies Branch Daniel Takefman, Ph.D., Chief Gene Transfer and Immunogenicity Branch Eda Bloom, Ph.D., Chief Cell Therapies Branch Keith Wonnacott, Ph.D., Chief Tumor Vaccines and Biotechnology Branch Raj Puri, Ph.D., M.D., Chief* Cellular and Tissue Therapy Branch Steven Bauer, Ph.D., Chief* *Acting

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Products Evaluated by DCGT Cellular treatments Gene treatments Tumor immunizations and immunotherapy Tissue designed items Xenotransplantation items Combination items Devices utilized with cells/tissue

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DCGT administrative portfolio and exercises Over 1100 dynamic INDs, IDEs, and ace documents, a few thousand alterations for each year, counsel surveys One authorized item, a developing number of items in phase 3 Devices: 510ks, PMAs, HDEs Pre-INDs, pre-pre-IND exhortation Advisory board of trustees gatherings Inspections Enforcement activities

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Additional DCGT administrative portfolio and exercises Partnerships: National Toxicology Program, NIH, CDC, NCI/IOTF program, NIH foundational microorganism team, NIST, MATES Participation in global gathering on harmonization (ICH), WHO Outreach talks at meetings, scholastic organizations, customer and patient promotion assemble gatherings Liaison exercises with expert gatherings Publication of papers on administrative points

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Roles and work of specialist commentators Product application audit, arrangement and direction improvement, administrative effort, administrative coaching, consultative council planning, requirement, worldwide exercises Research, preparing/tutoring Administrative: branch boss obligations, CBER peer survey and planning panels, explore administration Grant composing Participation in established researchers: logical talks, leading of meeting sessions, altering, peer investigating original copies and concede applications

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Approach to research: Stay on the ball as items and advances advance For this wide range, can\'t have inquire about identified with every item . On the off chance that exploration tended to just the items and tests of today, we would as of now be out of date. We should manage pre-IND work, set up the route for expected items.

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Research Strategy in DCGT items are different and quickly developing. They require new administrative standards which are growing instead of built up Critical Path: fill holes, manage logical difficulties, make sense of what is vital Sponsors examine singular items, comes about frequently restrictive CBER researchers perform considers important to whole item classes, make comes about open, and in this manner available to all backers, to propel the whole field

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DCGT Research Priorities: Led by Critical Path Challenges Virology Retroviruses, adeno, herpes, filo Immunology Anti-viral invulnerability, immunobiology of cell treatment and xenotransplantation Cell science Control of separation in creature models, immature microorganism science Cancer science Molecular biomarkers, creature models Biotechnology Genomics, stream cytometry, proteomics

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PIs Reviewed in TVBB Raj K. Puri, M.D., Ph.D. Disease Biology and Genomics Program Michail Alterman, Ph.D. Proteomics Program

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Public Health Issue and Regulatory Challenges Cancer, a most troublesome general wellbeing problem* Biology of growth – suitable targets Appropriate tests and biomarkers for virtue, personality and intensity Animal models – security and viability Biomarkers for malady observing and reaction * 1,372,910 New Cases of Cancer in 2005 570,280 Cancer Deaths in 2005 Source: Cancer Facts and Figures, 2005 (American Cancer Society)

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Critical Path Scientific Research Addressing Regulatory Challenges Specific Aims: Characterization of tumor related cell surface proteins (antigens or receptors) to build up personality of tumor antibodies and focus for disease treatment Establish creature models of human malignancy to evaluate security and adequacy of tumor focused on operators and quality treatment items Characterization of tumor immunizations and foundational microorganisms by genomics innovation to recognize biomarkers of immaculateness, personality and power and disease undifferentiated organisms

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Characterization of tumor related cell surface receptors and antigens Discovery of Th-2 inferred cytokine receptors Interleukin-4 receptors Renal cell carcinoma, head and neck growth, harmful glioma, AIDS-Kaposi\'s tumors, colon growth, bosom carcinoma, NSCLC, prostate malignancy, pancreatic tumor, ovarian disease, mesothelioma, and hematological malignancies (CLL-B) Interleukin-13 receptors Renal cell carcinoma, threatening glioma, ovarian carcinoma, AIDS-related Kaposi\'s sarcoma, pediatric glioma, and head and neck tumors

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Animal models of human tumor to survey security, danger and viability of tumor focused on specialists Ovarian Cancer Pancreatic tumor Glioblastoma multiforme SCCHN Mesothelioma Lung cancer AIDS-related Kaposi\'s tumors Breast disease Hodgkin\'s lymphoma Serous adeno Clear cell CA

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Analytical proteomics for the portrayal of biologic items and disclosure of biomarkers Michail Alterman, Ph.D.

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Specific Aims Development of mass spectrometry-based expository devices for testing of natural item quality and personality; Identification of proteomics-based cell sub-atomic marks to be tried as indicators of helpful achievement; Characterization of cell substrates used to create quality treatment items and preventive and restorative immunizations. Proteomic portrayal (profiling) of various cell lines with accentuation on undeveloped cell lines. Proteomic profiling of cytochrome P450 isozymes expression in tumors as potential biomarkers of malignancy.

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Expected Outcome and Deliverables Development of a basic non specific example pre-fractionation method empowering dependable investigation of an agent part of the phone proteome Proteomic profiling of MDCK and MRC-5 cells and other cell substrates Identification of a one of a kind protein signature (biomarkers) of hESC and CD34+ cells and examination of quantitative and subjective changes amid the separation of hESC to CD34+ cells. The disclosure of new CYP isozymes in tumors may prompt new biomarkers and advancement of new hostile to growth medications and treatments.

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Regulatory result Identification of new antigens for malignancy immunizations portrayal and focus for disease treatment Developing creature models for an assortment of human tumor to test wellbeing and viability of focused operators Promote advancement of novel advances (e.g., genomics, proteomics) for item portrayal (e.g., biomarker for personality, immaculateness, and power) and security Unique chance to recognize atomic markers with in vivo results

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