Challenges associated with supply to larger patients populations: Elements of the CTL019 experience
|
|
- Louise Carpenter
- 6 years ago
- Views:
Transcription
1 Cell and Gene Therapy Challenges associated with supply to larger patients populations: Elements of the CTL019 experience Daniel Stark Head External Supply Cell- and Gene Therapies Development and Manufacturing EBE conference on ATMPs London, December 5 th, 2017
2 Disclaimer This presentation is based on publicly available information. These slides are intended for educational purposes only and for the personal use of the audience. These slides are not intended for wider distribution outside the intended purpose without presenter approval. The content of this slide deck is accurate to the best of the presenter s knowledge at the time of production. The views and opinions expressed in this presentation are those of the presenter and do not necessarily reflect the official policy or position of Novartis or any of its officers. 2 D. Stark, EBE conference on ATMPs, London, 5 th December 2017
3 CTL019 is a living drug designed to target CD19+ B cells Patient s T cell CTL019 cell Anti-CD19 CAR construct CD19 Tumor cell Dead tumor cell Native TCR Lentiviral vector 3 Ex vivo D. Stark, EBE conference on ATMPs, London, 5 th December 2017 Cytokine release CTL019 proliferation In vivo
4 CTL019 is an autologous immunocellular therapy 1 LEUKAPHERESIS 6 ADMINISTRATION Clinical Site Clinical Site Manufacturing Facility 2 ENRICHMENT & ACTIVATION 4 EXPANSION 3 TRANSDUCTION 5 FORMULATION & QUALITY ASSESSMENT 4 D. Stark, EBE conference on ATMPs, London, 5 th December 2017
5 Preparation for Commercial Manufacturing Donor Collection Site Moving from one academic facility to many collection sites, multiple manufacturing sites, and many patient treatment sites Manufacturing Administration Site Site Acad emic to Com merci al Donor Collection Sites Shipping Administration Sites Shipping Manufacturing Sites 5 D. Stark, EBE conference on ATMPs, London, 5 th December 2017
6 Chain of identity (COI) Clinical Site Clinical Site 6 Well-established standards for labelling leukapheresis and end-product, use of the Single European Code (Directive 2015/565/EC amending Directive 2006/86/EC on technical requirements for the coding of human tissues and cells) 6 D. Stark, EBE conference on ATMPs, London, 5 th December 2017
7 Leukapheresis site qualification and collection Clinical Site Clinical Site 6 Leukapheresis sites are typically accredited via FACT (US) or JACIE (EU) and in line with local regulations, using or implementing the ISBT-128 labelling standards Novartis performs on-site quality assessments, maintains quality agreements with each site, defines collection and processing requirements, and trains the sites accordingly. The leukapheresis material is cryopreserved prior to shipping Leukapheresis Reference Manual describing minimal requirements to equipment, anticoagulants, procedures, timing, cryopreservation FACT = Foundation for the Accreditation of Cellular Therapy JACIE = Joint Accreditation Committee ISCT-EBMT ISBT=International Society of Blood Transfusion. 7 D. Stark, EBE conference on ATMPs, London, 5 th December 2017
8 Consistent CTL019 T-cell product from individual patient material Clinical Site Clinical Site 6 T cells NK cells Monocytes B cells Incoming leukapheresis material Patient 1 Patient 2 Patient 3 Patient 4 Consistent final product within specifications Patient 1 Patient 2 Patient 3 Patient 4 8 D. Stark, EBE conference on ATMPs, London, 5 th December 2017
9 Final Product control: release tests Clinical Site Clinical Site 6 Identity ü Appearance ü Vector integration Purity ü %T Cells ü Cell viability ü Transduction efficiency Impurities ü Residual beads ü Residual B cells / MRD ü Vector residuals Potency ü Cytokine production ü CAR expression Safety ü Sterility ü Endotoxin ü Mycoplasma ü RCL / vector residuals 9 D. Stark, EBE conference on ATMPs, London, 5 th December 2017
10 Overall control strategy is based on understanding of CQAs Clinical Site Clinical Site 6 Starting Material In Process Controls Materials Controls Control Strategy Final Product Testing Change Control Process Validation CQA: Cri(cal Quality A/ribute CQAs 10 D. Stark, EBE conference on ATMPs, London, 5 th December 2017
11 Change Control: Managing life cycle of manufacturing Change Driver Process Improvements Automation implementation Etc. Development Summary Report Change Control Initiation Risk Assessment COMPLIANCE RISK Product RA Safety assessment Viral clearance Impurities Process RA Validation CPP/CQA Unit Operations Environment Patient RA Dose Immune response Business RA Sustainability Scalability OVERALL RISK Risk Assessment Report Minor Moderate Major/Uncertain Change Notification to Health Authorities Change Strategy Execute Strategy Study Report Partner Notification Change Implementation Change Control Closure 11 D. Stark, EBE conference on ATMPs, London, 5 th December 2017
12 Comparability: Use of Split Leukapheresis as starting material Heterogeneity of incoming material T Cells B Cells Monocytes NK Cells Leukapheresis materials Multiple process steps for T cell Isolation Evenly split Process A Process B Harvest Harvest Split healthy donor apheresis (or patient apheresis, if deemed necessary) is used to minimize unrelated variability which might be caused by different starting material in order to better assess the impact of the change. 12 D. Stark, EBE conference on ATMPs, London, 5 th December 2017
13 Reference manufacturing site model to manage complexity Receive: process changes, analytical changes, specs Gives: process data, trends, investigation, patient response data Technology Transfer Comparability Receive: Process data, trends, investigations, patient response data Future regions or expansion Receive: process changes, analytical changes, specs Gives: process data, trends, investigation, patient response data Gives: process optimization, product enhancements, knowledge Benefit: Preserves consistency of process and product in all regions Risk if not maintained: Process drift over time changes performance and quality of product 13 D. Stark, EBE conference on ATMPs, London, 5th December 2017
14 Conclusions Manufacturing and supply of autologous products needs to address the following intrinsic challenges: Assurance of Chain of Identity through all supply and manufacturing steps Scale-out of manufacturing and logistics by automation elements Compliant change control management and sound comparability exercises are key tools to perform development and life cycle activities.. Understanding of CQA s is key to successful and efficient development and subsequent commercialization of ATMPs Autologous cell therapies require novel integrated approach to end-toend data management and analysis (from patient starting material to clinical response of patient) to manage development and life cycle of commercial products 14 D. Stark, EBE conference on ATMPs, London, 5 th December 2017
15 Thank you
Quality development considerations - Regulatory perspective
Quality development considerations - Regulatory perspective CAT workshop on cell-based immunotherapies, London 15. 11.2016 Christiane Niederlaender CAT Member, MHRA An agency of the European Union Genetically
More informationCQAs for C> Products to Enable Comparability Assessment. Ben Thompson Snr Director, Biopharmaceutical CMC RA GlaxoSmithKline
CQAs for C> Products to Enable Comparability Assessment Ben Thompson Snr Director, Biopharmaceutical CMC RA GlaxoSmithKline Overview Demonstrate the value of defining CQAs early in product development
More informationCMC Considerations for Manufacturing of CAR T-Cell Product
CMC Considerations for Manufacturing of CAR T-Cell Product November 14, 2017 Joann M. Parker, R.Ph, M.S. Regulatory Global CMC; Pfizer Inc Session: New Modalities for Cancer Moonshot: Unique Regulatory,
More informationOpportunities and Challenges for Manufacturing Scale Up of CAR T Cells. Mark Dudley Institute of Medicine March 1, 2016
Opportunities and Challenges for Manufacturing Scale Up of CAR T Cells Mark Dudley Institute of Medicine March 1, 2016 Disclaimer This document represents proposals for discussion by Management. Strategies/Concepts/Projects
More informationRegulatory considerations for manufacturing and testing of investigational chimeric antigen receptor (CAR) T-cell products
Regulatory considerations for manufacturing and testing of investigational chimeric antigen receptor (CAR) T-cell products Xiaobin Victor Lu Product Reviewer Gene Therapies Branch DCGT/OCTGT/CBER/FDA MEASUREMENT
More informationAssessing and Controlling Potency of Vector and Drug Product for Chimeric Antigen Receptor T Cells
Assessing and Controlling Potency of Vector and Drug Product for Chimeric Antigen Receptor T Cells David M, Hambly, Ph.D. Director, Analytical Development April 4th, 2016 Forward Looking Statements/Safe
More informationComparability Studies for Autologous Cell Therapy Products. Chris Shen July, 2017
Comparability Studies for Autologous Cell Therapy Products Chris Shen July, 2017 Forward Looking Statements/Safe Harbor To the extent statements contained in this presentation are not descriptions of historical
More informationCurrent Considerations on Chemistry, Manufacturing and Control of Cell Therapy Products (CTPs)
Current Considerations on Chemistry, Manufacturing and Control of Cell Therapy Products (CTPs) Wei Wei Ph.D Center for Drug Evaluation, National Medical Products Administration (CDE,NMPA) 2018/12/04 Tokyo
More informationCOGS by Design: a systems approach to achieving commercially viable cellular therapy products. Tim Oldham 27 May 2016 ISCT 16 - Singapore
COGS by Design: a systems approach to achieving commercially viable cellular therapy products Tim Oldham 27 May 2016 ISCT 16 - Singapore Summary Identifying the total needle-to-needle cost of therapy to
More informationRegulatory Approval of Modern Gene-Based Cancer Immunotherapies CAR T Cells A product perspective
Regulatory Approval of Modern Gene-Based Cancer Immunotherapies CAR T Cells A product perspective ASQ509 Biomed/Biotech SIG 2/1/18 Xiaobin Victor Lu Division of Cellular and Gene Therapies Office of Tissues
More informationManufacturing Challenges with Cell and Gene Therapy Products: A Health Canada Perspective
Manufacturing Challenges with Cell and Gene Therapy Products: A Health Canada Perspective CASSS CMC Summer Forum 2017 Chris Storbeck, PhD Health Canada Outline Health Canada organization Regulation of
More informationContact details: Anna Silvani
Consultation Document Good Manufacturing Practice for Advanced Therapy Medicinal Products MolMed comments to the DG SANTE consultation on GMPs for ATMPs pursuant to Article 5 of Regulation 1394/2007. MolMed
More informationCase Study: Examples Relating to the Quality Control of Cell-based Products
Case Study: Examples Relating to the Quality Control of Cell-based Products CMC Strategy Forum Japan 2014 Yuuki Miyatake1), 2) 1) TEIJIN PHARMA LIMITED 2) Japan Pharmaceutical Manufacturers Association
More informationABOUT GLYCOSTEM. Company Overview
ABOUT GLYCOSTEM The company is a clinical stage biotech company established in the Netherlands in 2007. The company s headquarters and new state-of-the-art lab and production facilities are based at Pivot
More informationIntegration of work flows for the generation of gene-modified cell products
Integration of work flows for the generation of gene-modified cell products Boro Dropulić, PhD, MBA Chief Science Officer and General Manager Lentigen Technology Inc., A Miltenyi Biotec Company Miltenyi
More informationProduct Testing & Release. PACT Workshop: Design & Operation of GMP Cell Therapy Facilities April 4 th /5 th, 2006
Product Testing & Release PACT Workshop: Design & Operation of GMP Cell Therapy Facilities April 4 th /5 th, 2006 Product Testing Used to determine Safety, Purity, Identity, Potency, etc. Suitability of
More informationOutcomes in Mesenchymal Stem Cell Manufacturing. Athena Russell, MT(AAB) Human Cellular Therapy Laboratory Mayo Clinic Jacksonville, FL
Outcomes in Mesenchymal Stem Cell Manufacturing Athena Russell, MT(AAB) Human Cellular Therapy Laboratory Mayo Clinic Jacksonville, FL Background HCTL established in 1992 to support BMT programs of Mayo
More informationImmuno-Oncology Program
Immuno-Oncology Program 2018 Forward Looking Statements This presentation contains forward-looking statements within the meaning of the "safe harbor" provisions of the Private Securities Litigation Reform
More informationDevelopment of non-substantially manipulated cell-based ATMPs 1 : flexibility introduced via the application of the risk-based approach
3 July 2017 EMA/CAT/216556/2017 Inspections, Human Medicines, Pharmacovigilance and Committees Division Development of non-substantially manipulated cell-based ATMPs 1 : flexibility introduced via the
More informationRegulatory requirements for early stage clinical trials with cell-based medicinal products. Christopher A Bravery
Regulatory requirements for early stage clinical trials with cell-based medicinal products Christopher A Bravery cbravery@advbiols.com Opening Remarks Cell-based medicinal products are in themselves diverse,
More informationCell Characterization: Developing Assays to Address the Biological Parameters of Identity and Potency
Cell Characterization: Developing Assays to Address the Biological Parameters of Identity and Potency Jessica Carmen Ph.D and Jon Rowley Ph.D., LBS-TCS, May 2011 Disclaimer Certain matters discussed in
More informationRaw Materials: Sourcing and Qualification Testing
Raw Materials: Sourcing and Qualification Testing Scott R. Burger, MD Advanced Cell & Gene Therapy Technical Applications Track 2 ISCT 2009 Sourcing Raw Materials Raw materials for cell/gene therapy manufacturing
More informationCell Processing at CAGT. Overview. Adrian P. Gee Center for Cell & Gene Therapy. CAGT Introduction
Cell Processing at CAGT Adrian P. Center for Cell & Gene Therapy CAGT Introduction Overview Facility Description Products prepared Flow cytometry Quality Control Quality Assurance 1 Center for Cell & Gene
More informationCell Therapy Services Your Product. Our Passion.
Pharma&Biotech Cell Therapy Services Your Product. Our Passion. Complete Portfolio of Services for cgmp Manufacturing of Cell-based Products Pharma&Biotech Cell Therapy Services for cgmp Manufacturing
More informationTechnology of cell therapies
Technology of cell therapies what are the challengess and how can they be met? Professor Mark Lowdell Vice President, International Society for Cell Therapy Professor of Cell & Tissue Therapy, UCL plan
More informationForm 4006 R1.0: Cellular Therapy Infusion
Sequence Number: Date Received: - - CIBMTR Center Number: CIBMTR Research ID: Event date: - - If more than one type of cell therapy product is infused, each product type must be reported separately. Key
More informationProduct Testing & Release. PACT Workshop: Design & Operation of GMP Cell Therapy Facilities April 10 th /11 th, 2007
Product Testing & Release PACT Workshop: Design & Operation of GMP Cell Therapy Facilities April 10 th /11 th, 2007 Product Testing Used to determine Safety, Purity, Identity, Potency, Quality Suitability
More informationConsiderations in Product Development with Advanced Therapies and Cancer Vaccines
Considerations in Product Development with Advanced Therapies and Cancer Vaccines Thomas Hinz Head of Section Therapeutic Vaccines Paul-Ehrlich-Institut hinth@pei.de Thomas Hinz, October 29, 2008, San
More informationSpecifications. February 19, 2015, TOKYO, JAPAN. Takao Hayakawa, Ph.D. Director, Pharmaceutical Research and Technology Institute, Kindai University
Specifications February 19, 2015, TOKYO, JAPAN Takao Hayakawa, Ph.D. Director, Pharmaceutical Research and Technology Institute, Kindai University Norihisa Sakamoto, M.D., Ph.D. Deputy Review Director
More informationWhitepaper. Small Molecule Vs Cell Therapy Clinical Trial Supply Chain. Martin Lamb, Executive Vice President for Sales and Marketing
Small Molecule Vs Cell Therapy Clinical Trial Supply Chain Page 1 Whitepaper Small Molecule Vs Cell Therapy Clinical Trial Supply Chain Martin Lamb, Executive Vice President for Sales and Marketing Small
More informationRegulatory Implications for Global Manufacturing Development of Regenerative Medicines
Regulatory Implications for Global Manufacturing Development of Regenerative Medicines Katherine Tsokas, JD June 2017 Global Regulatory Affairs Janssen Research & Development, LLC Jessica Riley, Shells
More informationISBT 128 Labeling Overview. Fran Rabe University of Minnesota Molecular and Cellular Therapeutics
ISBT 128 Labeling Overview Fran Rabe University of Minnesota Molecular and Cellular Therapeutics ICCBBA u Not for profit organization u Formally: International Council for Commonality in Blood Banking
More informationAdoptive cellular therapies are based on the administration of live cells into a patient in order for them to serve a therapeutic purpose.
Adoptive cellular therapies are based on the administration of live cells into a patient in order for them to serve a therapeutic purpose. Pharmaceutical products have been historically made of purified
More informationComparability Is Not a Nightmare, Just Think Ahead!
Comparability Is Not a Nightmare, Just Think Ahead! Margarida Menezes Ferreira Senior Assessor at INFARMED PT expert at BWP/CHMP - EMA member of the CAT - EMA (margarida.menezes@infarmed.pt) I attend this
More informationAncillary Materials for Cell & Tissue Therapies Definitions, US Regulatory Approach, and USP s Risk-Tiered Approach
USP/ISCT Workshop 2012 Seattle, WA, USA Ancillary Materials for Cell & Tissue Therapies Definitions, US Regulatory Approach, and USP s Risk-Tiered Approach Elizabeth Read, MD Head of Product Development
More informationAdvancing Manufacturing for Advanced Therapies
Advancing Manufacturing for Advanced Therapies Peter Marks, MD, PhD Center For Biologics Evaluation and Research, FDA CASSS Cell & Gene Therapy Symposium July 10, 2018 Overview Cell and gene therapy products
More informationAdvanced Therapy Medicinal Products and GMP. Ashley Isbel
Advanced Therapy Medicinal Products and GMP Ashley Isbel Session Overview What are ATMPs? The State of Regulations GMP and Other Challenges for ATMP production Some Solutions The Future What are ATMPs?
More informationThe Early Chimeric Antigen Receptor (CAR) T-cell Experience From An Academic Perspective. CARs: LEARNING TO DRIVE
The Early Chimeric Antigen Receptor (CAR) T-cell Experience From An Academic Perspective CARs: LEARNING TO DRIVE Jakub Svoboda, MD University of Pennsylvania Philadelphia, PA Cancer Drug Development Forum
More informationRegulation of advanced blood cell therapies
Regulation of advanced blood cell therapies www.pei.de Clinical trials using cell-based products Substantially manipulated cells and cells for non-homologous use Quality, safety and non-clinical aspects
More informationOrchard Therapeutics. Overcoming the complex challenges associated with ex vivo gene therapies. Adrien Lemoine VP Business Development & Operations
Orchard Therapeutics Overcoming the complex challenges associated with ex vivo gene therapies Adrien Lemoine VP Business Development & Operations Orchard at a glance Who we are Our mission Team Academic
More informationDevices and Manufacturing Equipment Used for Production of Immune Cell Therapy and Cell Expansion: Scientific/User Issues
Devices and Manufacturing Equipment Used for Production of Immune Cell Therapy and Cell Expansion: Scientific/User Issues Bruce Levine, Ph.D. Department of Pathology and Laboratory Medicine University
More informationCellular Therapy Product
Cellular Therapy Product Registry Use Only Sequence Number: Date Received: CIBMTR Center Number: Event date: / / CIBMTR Form 4003 revision 1 (page 1 of 7). Last Updated January, 2018. Cellular Therapy
More informationQ4 and Full Year 2017 Conference Call. February 22, 2018
Q4 and Full Year 2017 Conference Call February 22, 2018 Agenda Welcome McDavid Stilwell VP, Corporate Communications and Investor Relations Q4 2017 and Full Year Review and Recent Highlights Dr. Sandy
More informationRe: Public Consultation Paper on the Regulation of Advanced Therapy Medicinal Products
ISCT HEAD OFFICE Suite 201 375 West 5 th Avenue Vancouver, BC Canada V5Y 1J6 Tel: 604-874-4366 Fax: 604-874-4378 isct@celltherapysociety.org www.celltherapysociety.org March 30, 2013 European Commission,
More informationJustification of Specifications (JOS): What Product and Process Development was all About. Christopher A Bravery
Justification of Specifications (JOS): What Product and Process Development was all About Christopher A Bravery cbravery@advbiols.com 1 The Importance of Characterisation 2 http://www.advbiols.com/documents/importance
More informationGENERATION OF OFF-THE-SHELF TCR-LESS CAR T CELLS FROM RENEWABLE PLURIPOTENT CELLS
GENERATION OF OFF-THE-SHELF TCR-LESS CAR T CELLS FROM RENEWABLE PLURIPOTENT CELLS Bob Valamehr, PhD Vice President, Fate Therapeutics AACR Annual Meeting 2018 Press Program McCormick Place North/South
More informationCMC Writing for IND Applications. David H. McKenna, Jr., M.D. University of Minnesota PACT Web Seminar II July 14, 2005
CMC Writing for IND Applications David H. McKenna, Jr., M.D. University of Minnesota PACT Web Seminar II July 14, 2005 CMC Writing for IND Applications ~Outline~ What is the CMC section? Who writes the
More informationSharon Tindle, MS, CQA (ASQ) QA Manager, BMT Tissue Services Mount Sinai Hospital, New York, NY. June 7, 2016
Sharon Tindle, MS, CQA (ASQ) QA Manager, BMT Tissue Services Mount Sinai Hospital, New York, NY June 7, 2016 1 Brief description of the Mount Sinai Cellular Therapy Laboratory Overview of BM transplant
More informationLifecycle Management: Challenges and Lessons Learned from Kymriah (CD19 CAR T)
Lifecycle Management: Challenges and Lessons Learned from Kymriah (CD19 CAR T) Anthony Thatcher, Site Head MS&T Novartis Pharmaceuticals Cell and Gene Technical Development and Manufacturing July, 2018
More informationMANUFACTURING CONTROL STRATEGY FOR CELL, GENE AND TISSUE PRODUCTS Christopher A Bravery
MANUFACTURING CONTROL STRATEGY FOR CELL, GENE AND TISSUE PRODUCTS Christopher A Bravery CBRAVERY@ADVBIOLS.COM 1 INTRODUCTION What is a manufacturing control strategy? Why is it important? Common issues
More informationSpecialty Lab Services. Deep science at scale
Specialty Lab Services Deep science at scale Advancing biomarker research Our broad expertise and global laboratory footprint deliver deep science at scale Specialty assays drive insight into preclinical
More informationAddressing Variability in Cell and Gene Therapy Manufacturing Carl Burke
Addressing Variability in Cell and Gene Therapy Manufacturing Carl Burke Exploring Sources of Variability Related to the Clinical Translation of Regenerative Engineering Products A Workshop National Academies
More information4006: Cellular Therapy Infusion
4006: Cellular Therapy Infusion Registry Use Only Sequence Number: Date Received: Key Fields CIBMTR Center Number: Event date: / / CIBMTR Form 4006 revision 1 (page 1 of 9). Last Updated July, 2016. If
More informationPrecision Biospecimen Solutions: Paving the Way to Personalized Medicine
Precision Biospecimen Solutions: Paving the Way to Personalized Medicine ACCELERATING RESEARCH. IMPROVING OUTCOMES. The companies under the Precision for Medicine banner deliver specialized strategic and
More informationCell and gene therapy: scaling up and moving to mass production
EDITORIAL Cell and gene therapy: scaling up and moving to mass production Nigel Whittle In order to fulfil the promise of cell therapy, it is important that manufacture of these therapies can be industrialized
More informationCOMMITTEE FOR PROPRIETARY MEDICINAL PRODUCTS (CPMP)
The European Agency for the Evaluation of Medicinal Products Evaluation of Medicines for Human Use London, 31 May 2001 CPMP/BWP/41450/98 COMMITTEE FOR PROPRIETARY MEDICINAL PRODUCTS (CPMP) POINTS TO CONSIDER
More informationדרישות איכות ורגולציה בשלבים הראשונים של תרפיה תאית ד"ר עפרה אקסלרוד המכון לביקורת ותקנים של חומרי רפואה יוני 2013
דרישות איכות ורגולציה בשלבים הראשונים של תרפיה תאית ד"ר עפרה אקסלרוד המכון לביקורת ותקנים של חומרי רפואה יוני 2013 Advanced Therapy Medicinal Products ATMPs - European Term Gene Therapy Somatic Cell Therapy
More informationClinical qualification of specifications - a Regulator s view
Clinical qualification of specifications - a Regulator s view Mats Welin Medical Products Agency, Uppsala, Sweden Disclaimer: The opinions expressed are my own and do not necessarily represent those of
More informationForm 4006 R2.0: Cellular Therapy Infusion
Key Fields Sequence Number: Date Received: - - CIBMTR Center Number: CIBMTR Research ID: Event date: - - Cellular Therapy Product Identification Questions: 1-27 If more than one type of cell therapy product
More informationCurrent situation on nonclinical safety evaluation of regenerative medical products in Japan
Current situation on nonclinical safety evaluation of regenerative medical products in Japan Takuya Nishimura Office of Cellular and Tissue based Products PMDA Disclaimers The views expressed in this presentation
More informationTrue Confessions: Ancillary Materials in a Cell Processing Lab. Cell Processing Section, DTM, CC, NIH David Stroncek, MD Chief, CPS, DTM, CC
True Confessions: Ancillary Materials in a Cell Processing Lab Cell Processing Section, DTM, CC, NIH David Stroncek, MD Chief, CPS, DTM, CC Mission Provide cellular and gene therapy products and services
More informationCMC Consideration for the Development of Regenerative Medical Products
CMC Strategy Forum Japan 2018 December 4, 2018, Tokyo Marriot Hotel, Tokyo, Japan CMC Consideration for the Development of Regenerative Medical Products Kazunobu Oyama, PhD Deputy Review Director, Office
More informationRegulatory requirements for cell based medicinal products
Regulatory requirements for cell based medicinal products 資料 3-2 Committee 25 August 2010 Dr. Bettina Klug, MSc Paul-Ehrlich-Institut, Langen klube@pei.de Paul-Ehrlich-Institut Federal Institute for Vaccines
More informationGood Manufacturing Practice for ATMPs Rocio Salvador Roldan DG SANTE, Unit B5
Good Manufacturing Practice for ATMPs Rocio Salvador Roldan DG SANTE, Unit B5 This presentation only reflects the views of its author and does not necessarily reflect the opinion of the Commission Overview
More informationRaw materials for cell & gene therapy: exploring regulatory and supply issues
CELL & GENE THERAPY RAW MATERIALS: GETTING IT RIGHT FROM THE START INNOVATOR INSIGHT Raw materials for cell & gene therapy: exploring regulatory and supply issues As Vice President of Development & Production
More informationUpdate from the Center for Biologics Evaluation and Research (CBER) Peter Marks, M.D., Ph.D. GMP By The Sea 2017
Update from the Center for Biologics Evaluation and Research (CBER) Peter Marks, M.D., Ph.D. GMP By The Sea 2017 Outline Products regulated Significance of complex biologics Product and process Cutting
More informationA Life Cycle Approach to Raw Material Qualification for Cell and Gene Therapy Products
A Life Cycle Approach to Raw Material Qualification for Cell and Gene Therapy Products Angela Whatley, Ph.D. Office of Tissues and Advanced Therapies CBER/FDA CMC Strategy Forum on Cell & Gene Therapies
More informationHematopoietic Progenitor Cell Product Characterization
Hematopoietic Progenitor Cell Product Characterization Carolyn A. Taylor, Ph.D. Professor of Medicine Director of BMT Program Cell Processing Laboratory Product Testing and Characterization Goals Required
More informationJustifying Potency and other Specifications: Justification of Specifications: What Product and Process Development was all About
Justifying Potency and other Specifications: Justification of Specifications: What Product and Process Development was all About Christopher A Bravery cbravery@advbiols.com 1 Introduction What does justification
More informationRisk Assessments for Host Cell Protein Control Strategies: CDER Experiences
Risk Assessments for Host Cell Protein Control Strategies: CDER Experiences Laurie Graham FDA/CDER Office of Pharmaceutical Quality (OPQ) Office of Policy for Pharmaceutical Quality (OPPQ) 1 Disclaimer
More informationManufacturing development and comparability assessments of Cell and Gene Therapy products for marketing in Europe
Manufacturing development and comparability assessments of Cell and Gene Therapy products for marketing in Europe Margarida Menezes Ferreira Senior Assessor at INFARMED PT expert at BWP/CHMP - EMA member
More informationPrioritizing and Managing Key CMC Elements
Prioritizing and Managing Key CMC Elements Using 21 st Century Principles during Early Development Laurie Graham Product Quality/CMC Reviewer FDA/CDER/OPS/OBP Division of Monoclonal Antibodies 1 Disclaimer
More informationStem Cell Services. Driving Innovation for Stem Cell Researchers
Driving Innovation for Stem Cell Researchers Stem Cell Services Partner with us and have access to the most advanced and comprehensive stem cell services available today. 675 W. Kendall St. Cambridge,
More informationCopyright. Jeremiah J. Kelly (2015). All rights reserved. Further dissemination without express written consent strictly prohibited.
Statutory Framework for Biologics Drugs Investigational Use Application IND Pre-Market Approval Applications 505(b)(1) NDA 505(b)(2) NDA 505(j) ANDA Over-the-Counter (OTC) Non- Rx Drugs Monograph Biologics
More informationQuality of raw materials and manufacturing of advanced therapies. Fouad Atouf, Ph.D. Head, Global Biologics July 12, 2018
Quality of raw materials and manufacturing of advanced therapies Fouad Atouf, Ph.D. Head, Global Biologics July 12, 2018 Evolution of the compendia 1820: a single recipe book 2018: Procedures and acceptance
More informationTraceability of Autologous Drug Product from Cell Procurement through Infusion
Traceability of Autologous Drug Product from Cell Procurement through Infusion Ian O Reilly February 22, 2017 Nasdaq : BLUE Contents Highlighting and exploring the intricate autologous supply chain and
More informationINTERNATIONAL STANDARDS FOR HEMATOPOIETIC CELLULAR THERAPY PRODUCT COLLECTION, PROCESSING, AND ADMINISTRATION
INTERNATIONAL STANDARDS FOR HEMATOPOIETIC CELLULAR THERAPY PRODUCT COLLECTION, PROCESSING, AND ADMINISTRATION Seventh Edition DRAFT May 2017 NOTICE These Standards are designed to provide minimum guidelines
More informationMaking Hope A Reality bluebird style. November, 2017 Nasdaq : BLUE
Making Hope A Reality bluebird style November, 2017 Nasdaq : BLUE 1 Forward Looking Statements These slides and the accompanying oral presentation contain forward-looking statements and information. The
More informationGMP for ATMP should be a document annexed to standard GMP (a new Annex) and not a stand-alone document.
Leem comments on Commission proposal for Good Manufacturing Practice for Advanced Therapy Medicinal Products 1. General comments Line number(s) of the relevant text General comment
More informationINTERNATIONAL STANDARDS FOR HEMATOPOIETIC CELLULAR THERAPY PRODUCT COLLECTION, PROCESSING, AND ADMINISTRATION
INTERNATIONAL STANDARDS FOR HEMATOPOIETIC CELLULAR THERAPY PRODUCT COLLECTION, PROCESSING, AND ADMINISTRATION Sixth Edition Version 6.1 NOTICE These Standards are designed to provide minimum guidelines
More informationPrecigen Company Update
Precigen Company Update Helen Sabzevari, PhD President, Precigen 9 January 2019 JP Morgan 37 th Annual Healthcare Conference Forward-looking statements Precigen, Inc. is a subsidiary of Intrexon Corporation
More informationINTERNATIONAL STANDARDS FOR CELLULAR THERAPY PRODUCT COLLECTION, PROCESSING, AND ADMINISTRATION
INTERNATIONAL STANDARDS FOR CELLULAR THERAPY PRODUCT COLLECTION, PROCESSING, AND ADMINISTRATION Fifth Edition Version 5.1 March 2012 NOTICE These Standards are designed to provide minimum guidelines for
More informationComments from: 1. General comments. International Plasma Fractionation Association (IPFA) Our ref. IP
Submission of comments on Commission proposal for Good Manufacturing Practice for Advanced Therapy Medicinal Products Comments from: Name of organisation or individual International
More informationAdvanced-therapy medicinal products: new competencies in hospital pharmacy Seminar PH4. Relevant Financial Relationships - None
EAHP March 2016 Advanced-therapy medicinal products: new competencies in hospital pharmacy Seminar PH4 Dr. Lenka Taylor Pharmacy University Hospital Heidelberg Disclosure Relevant Financial Relationships
More informationQP Essentials: Your EU Gateway to Clinical and Commercial Distribution of Cell-Based Therapeutics
QP Essentials: Your EU Gateway to Clinical and Commercial Distribution of Cell-Based Therapeutics By Colin Grant, Quality and Regulatory Manager, Fisher BioServices 1 Share th is eb o o k! www.fis herbios
More informationISCT Telegraft Column: Mesenchymal Stromal Cell (MSC) Product Characterization and Potency Assay Development
ISCT Telegraft Column: Mesenchymal Stromal Cell (MSC) Product Characterization and Potency Assay Development University of Wisconsin-Madison, Production Assistance for Cellular Therapies (PACT) Over the
More informationQuality Program: Supplies and Reagents
Quality Program: Supplies and Reagents J. Wade Atkins, MS, MT(ASCP) SBB, CQA (ASQ) Supervisor, Quality Assurance and Regulatory Affairs Department of Transfusion Medicine, Clinical Center, NIH Disclosures:
More informationINTRODUCTION. How we regulate Cell and Gene Therapy Product? Presented by Azizah Ab Ghani National Pharmaceutical Control Bureau
INTRODUCTION How we regulate Cell and Gene Therapy Product? Presented by Azizah Ab Ghani National Pharmaceutical Control Bureau 1 CGTP Multidisciplinary approach MOH Medical Development Division Medical
More informationThis presentation contains forward-looking statements within the meaning of the "safe harbor" provisions of the Private Securities Litigation Reform
This presentation contains forward-looking statements within the meaning of the "safe harbor" provisions of the Private Securities Litigation Reform Act of 1995, as amended. These forward-looking statements
More informationDevelopment Stage of Therapeutic Vaccines: The Regulator s View
Development Stage of Therapeutic Vaccines: The Regulator s View Thomas Hinz Head, Section Therapeutic Vaccines Paul Ehrlich Institute, Germany thomas.hinz@pei.de 1 DISCLAIMER This is the personal views
More informationCell Therapy Product Manufacturing Considerations. July 17, 2017 CMC Strategy Forum Mo Heidaran, Ph.D.
Cell Therapy Product Manufacturing Considerations July 17, 2017 CMC Strategy Forum Mo Heidaran, Ph.D. Office of Tissues and Advanced Therapies FDA/CBER Overview Establishing Manufacturing Control Applying
More informationMANUFACTURING CONTROL STRATEGY FOR CELL, GENE AND TISSUE PRODUCTS Christopher A Bravery
MANUFACTURING CONTROL STRATEGY FOR CELL, GENE AND TISSUE PRODUCTS Christopher A Bravery CBRAVERY@ADVBIOLS.COM 1 INTRODUCTION What is a manufacturing control strategy? Why is it important? Common issues
More informationCHANGES TO A GENE THERAPY MEDICINAL PRODUCT: REGULATORY VIEWS
CHANGES TO A GENE THERAPY MEDICINAL PRODUCT: REGULATORY VIEWS Maria Cristina Galli, Ph.D. Istituto Superiore di Sanità Roma, Italy EATRIS ATMP platform chair ISCT 2016 Global Regulatory Perspective Workshop
More informationComparability for Advanced Therapy Medicinal Products. Christopher A Bravery
Comparability for Advanced Therapy Medicinal Products Christopher A Bravery cbravery@advbiols.com 1 Introduction Taster for the preconference workshop Hypothetical case study to illustrate the principles
More informationISBT 128 coding system and the Single European Code: Introduction. Presented by: Jessy Lardon
ISBT 128 coding system and the Single European Code: Introduction Presented by: Jessy Lardon Single European Code (= SEC) The SEC is the unique identifier applied to tissues and cells distributed in the
More informationEXAMPLE OF VENDOR QUALIFICATION FOR IMMUNE EFFECTOR CELL PRODUCT MANUFACTURER
EXAMPLE OF VENDOR QUALIFICATION FOR IMMUNE EFFECTOR CELL PRODUCT MANUFACTURER Disclaimer: This example is just one potential example of a vendor qualification form to verify the adequacy of the internal
More informationStem Cells, Regenerative Medicine and cgmp (GTP)
Stem Cells, Regenerative Medicine and cgmp (GTP) Encompass Stem cell based therapies activities Collection source Purification Isolation from other cell types if needed Manipulation Minimal vs Moderate
More informationBiotechnology: Quality Assurance and Validation
r Biotechnology: Quality Assurance and Validation Drug Manufacturing Technology Series Volume 4 Kenneth E. Avis Carmen M. Wagner Vincent L. Wu Editors Interpharm Press, Inc. Buffalo Grove, Illinois CONTENTS
More informationAcademic and Industry Partnerships
Academic and Industry Partnerships Technology Transfer and Scale-Up Stewart Abbot 8 th June 2017 Disclosure Stewart Abbot is an employee and share holder of Fate Therapeutics Inc. Fate Therapeutics Inc.
More informationFDA Perspectives On Viral & Vector Shedding Studies. Daniel Takefman, Ph.D. Chief, Gene Therapy Branch FDA/CBER ICH Workshop: October 31, 2007
FDA Perspectives On Viral & Vector Shedding Studies Daniel Takefman, Ph.D. Chief, Gene Therapy Branch FDA/CBER ICH Workshop: October 31, 2007 Overview Define shedding studies Outline basis for need of
More information