European Regenerative Medicine Firms & Their Strategic Approaches. Michael Morrison University of York
|
|
- Cassandra Green
- 6 years ago
- Views:
Transcription
1 European Regenerative Medicine Firms & Their Strategic Approaches Michael Morrison University of York
2 OVERVIEW Creating the European RM Universe of firms Characterizing the European RM Universe Strategic Analysis
3 CREATING THE EUROPEAN REGENERATIVE MEDICINE UNIVERSE
4 DEFINING REGENERATIVE MEDICINE Regenerative medicine is a heterogeneous domain incorporating biological sciences, engineering, materials science, medicine, and surgery. Broadly, regenerative medicines act therapeutically by stimulating, directing or augmenting the body s capacity for self-repair and regeneration. Multiple technological avenues of investigation, united by this overarching goal, but forming identifiable sub-sectors of regenerative medicine such as stem cell therapy, gene therapy, tissueand-bioscaffolds etc
5 DEFINING REGENERATIVE MEDICINE Our approach concentrates on technologies using novel biomaterials living cells, genes and bioscaffolds. Excludes small molecules and biologicals (eg antibodies) that aim to stimulate in vivo regenerative action. Alongside therapeutic approaches are cell diagnostics for drug testing and platform technologies, mainly tissue and biobanks.
6 DEFINING REGENERATIVE MEDICINE Large pharmaceutical companies with an interest in aspects of regenerative medicine such as Pfizer, Sanofi-Aventis, Smith and Nephew etc not included on the list Company must have some publicly available details (usually in the form of a website). Basic cell culture tools/media suppliers not included unless have specialist focus.
7 CHARACTERIZING THE EUROPEAN REGENERATIVE MEDICINE FIRM UNIVERSE
8 FIRM TYPE BY PRIMARY ORIENTATION Services/ other 14 Bioscaffold 12 Gene Therapy 6 Cell Therapy 27 N = 59
9 GEOGRAPHIC DISTRIBUTION OF FIRMS Other European 11 Switz'land 3 UK 21 France 4 Sweden 5 Germany 15 N =59
10 FIRMS BY SIZE (EMPLOYEE NOS) LARGE 3 MEDIUM 4 No Data 11 SMALL 41 N = 59 SMALL = 0-99 MEDIUM = LARGE = 250+
11 FIRMS BY AGE (YEAR FOUNDED) >20 years 8 No Data years years years 17 N = 59
12 SUMMARY OF FIRM CHARACTERISTICS Cell therapy is the largest single sector Significant no. of bioscaffold and service firms Most firms are small (< 99 employees) UK and Germany are the main geographic hubs of commercial RM activity Wide range of company ages many remaining from 1990s and pre 2002 crash
13 STRATEGIC ANALYSIS OF EUROPEAN REGENERATIVE MEDICINE FIRMS
14 AUTOLOGOUS VS ALLOGENIC CELL THERAPY Autologous Allogenic Both Total No. of Companies
15 STEM CELL VS SOMATIC CELL THERAPY Stem cell Non-stem cell Total No. of Companies
16 THERAPEUTIC FOCUS OF CELL THERAPY FIRMS Cell Stem cells Stem cells Cell therapy
17 CELL THERAPY LANDSCAPE Strong preference for autologous cell therapy Stem cells a significant but not dominant part of cell therapy landscape Somatic cell therapies concentrated in traditional T.E. domains Stem cell therapies have much broader therapeutic focus, generating new applications
18 GENE THERAPY COMPANIES Name Amsterdam Molecular Therapeutics (AMT) Locati on Size Found d Disease focus Notes NL Small 1998 Metabolic disorder Lead product Glybera submitted for EMA MAA in 2009 Ark Therapeutics UK Medium 1997 Vascular occlusion. Cancer Arthrogen BV NL Small 2005 Rheumatoid Arthritis Cerepro submitted to EMA for MAA in 2008, received rejection Trinam in phase III trials Lead product still at preclinical stage Diamyd Medical AB Sweden Small 1976 Chronic pain, cancer Main therapeutic programme is therapeutic vaccines for diabetes. Oxford BioMedica UK Small 1996 Neurodegenerative, Occular Lead product in phase I/II Transgene France Small 1979 Cancer Several cancer vaccines in development Mol Med Italy Small 1996 Graft vs Host disease Lead product in Phase III trials Mologen Germany Small 1998 Cancer Developing non- RM anti-cancer therapeutics
19 GENE THERAPY LANDSCAPE Small sub-sector, with small firms No clear trends in firm age, location or therapeutic focus Engagement with Advanced Therapy Medicinal Products regulation: Ark s Cerepro rejected but Amsterdam Molecular Therapeutics Glybera submitted for MAA MolMed (Italy) have cell-based ex-vivo gene therapy in phase III trials
20 SALEABLE THERAPEUTIC PRODUCTS Cell therapy for wound healing 3 Other cell therapy 1 Cell therapy for cartilage repair 13 Bioscaffold for tissue repair 19
21 RM PRODUCTS IN DEVELOPMENT RM Products in Development Bioscaffolds Gene therapy Preclinical Phase I Phase II Phase III Cell therapy
22 THERAPEUTIC LANDSCAPE Current market is dominated by Tissue Engineering products bioscaffolds and autologous chondrocyte transplantation therapies. However, there are a much wider range of products in development, including stem cell and gene therapies, aimed at a broader range of therapeutic applications. Indicates an industry in transition.
23 FOCUS OF DEDICATED SERVICE PROVISION FIRMS Service Total Stem cell? Cell and tissue based drug discovery / screening services / toxicity testing 4 2 Cell and tissue supply including research and clinical grade material 4 2 Contract manufacture of cells / cell line expansion, cell culturing and scale up 1 - Specialist tools and reagents 3 - Distribution of bioscaffolds for tissue 1 - repair, wound healing etc TOTAL 13 4
24 ADDITIONAL SERVICE PROVISION 18 Cell therapy companies have additional sources of revenue beyond lead therapeutic product Most common option is laboratory services, including cell culturing, cell and tissue supply, and cgmp facilities. Bioscaffold products and regenerative medicine consulting services are also popular options.
25 CONCLUSIONS
26 CONCLUSIONS Companies pursuing newer regenerative medicine models appear to co-exist with older tissue engineering and gene therapy firms. Strong industry preference for less risky technologies autologous cell therapy with somatic cells or adult stem cells. Little to no commercial investment in ips and embryonic stem cell technologies.
27 CONCLUSIONS Commercial development concentrated in Western Europe, and concentrated in hubs. Importance of biotechnology infrastructure and financial support. Regulatory environment is also an important shaping factor as with hescs Can be illustrated by Germany UK RM firm breakdown
28 CONCLUSIONS RM Sub-sector Germany UK Cell Therapy 9 6 Gene Therapy 1 2 Bioscaffolds 2 4 Service/ other 3 9 TOTAL 15 21
29
EMA s role & responsibility for the development of modern/advanced therapies
EMA s role & responsibility for the development of modern/advanced therapies Agenda Current regulatory picture Overall Regulatory framework New Committee at EMA Current Activities & Challenges Egbert Flory
More informationGlobal Gene Therapy Market Report -2026
Global Gene Therapy Market Report -2026 July 2018 Table of Contents 1 Executive Summary 2 Gene Therapy: Overview Viral Vectors for Gene Therapy Viral Vectors Non-Viral Vectors Types of Gene Therapy Somatic
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 informationRealizing the Future that Regenerative Medicine Will Open
470 Hitachi Review Vol. 65 (2016), No. 9 Featured Articles Realizing the Future that Regenerative Medicine Will Open Shizu Takeda, Ph.D. OVERVIEW: Regenerative medicine is an innovative approach to medicine
More informationLongevity Leaders Conference. February 4, 2019
Longevity Leaders Conference February 4, 2019 Forward Looking Statements The matters discussed in this presentation include forward looking statements which are subject to various risks, uncertainties,
More informationTHE GLOBAL MARKET FOR STEM CELLS
THE GLOBAL MARKET FOR STEM CELLS BIO035F May 2016 Paul Evers Project Analyst ISBN: 1-62296-292-3 BCC Research 49 Walnut Park, Building 2 Wellesley, MA 02481 USA 866-285-7215 (toll-free within the USA),
More informationAn Investment in the most comprehensive regenerative medicine platform for orthopedics
An Investment in the most comprehensive regenerative medicine platform for orthopedics Francois Binette PhD Vice President Research and Business Development The OrthoCyte Vision Our initial focus: Spine
More informationPioneering the Development of Safe and Effective Non-Viral Vectors and Processes for Human Gene Therapy and DNA Vaccination.
Pioneering the Development of Safe and Effective Non-Viral Vectors and Processes for Human Gene Therapy and DNA Vaccination. Founded 1999, Clague Hodgson, Ph.D. Chief Scientific Officer, VP, R&D, Jim Williams,
More informationAdult Stem Cells for Chronic Pain. Dr. John Hughes, DO January 24 th, 2018
Adult Stem Cells for Chronic Pain Dr. John Hughes, DO January 24 th, 2018 Dr. John Hughes, DO Doctor of Osteopathy From Georgia Arizona College of Osteopathic Medicine - 2007 Aspen Integrative Medicine
More informationTISSUE ENGINEERING AND REGENERATION: TECHNOLOGIES AND GLOBAL MARKETS
TISSUE ENGINEERING AND REGENERATION: TECHNOLOGIES AND GLOBAL MARKETS HLC101B August 2014 Yojana Jeevane Project Analyst ISBN: 1-56965-894-3 BCC Research 49 Walnut Park, Building 2 Wellesley, MA 02481 USA
More informationNanotechnology and Advanced Materials for more effective Healthcare
Nanotechnology and Advanced Materials for more effective Healthcare This challenge taps into the potential of nanotechnologies and advanced materials to enable more effective therapies and diagnostics
More informationAbout the Healthcare Strategic Analysis Team Geographic specific reports: Global issue reports: 1. About This Report Chapter structure Executive
About the Healthcare Strategic Analysis Team Geographic specific reports: Global issue reports: 1. About This Report Chapter structure Executive summary Licensing trends overview Evolving trends in the
More informationChallenges during the development of ATMPs
Challenges during the development of ATMPs CAT-DGTI Workshop Dresden 11.9.2014 Paula Salmikangas CAT Chair An agency of the European Union Gene Therapy Medicinal Products Somatic Cell Therapy Medicinal
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 informationREMEDiE Project London 7-8 May 2009 European Policies and Priorities for Stem Cell Research
REMEDiE Project London 7-8 May 2009 European Policies and Priorities for Stem Cell Research Charles Kessler European Commission DG Research, Health Directorate Outline of presentation Main features of
More informationRegulatory Perspectives of Japan
International Alliance for Biological Standardization (IABS) Challenge Toward Sound Scientific Regulation of Cell Therapy Products at Kyoto International Conference Center, Kyoto, Japan Regulatory Perspectives
More informationChondroCelect First ATMP approval in Europe
ChondroCelect First ATMP approval in Europe Innovation in Healthcare Brussel 20 May 2010 Wilfried Dalemans, PhD CTO & VP Regulatory Affairs Overview Introduction European regulatory framework The ChondroCelect
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 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 informationChapter 8 Healthcare Biotechnology
Chapter 8 Healthcare Biotechnology Outline: 8.1 Introduction 8.2 Biopharming 8.3 Models of Human Disease 8.4 Detecting and Diagnosing Human Disease 8.5 Monoclonal Antibodies 8.6 Gene Therapy 8.7 Tissue
More informationHorizon 2020 Health Summit-IIS La Fe. Valencia, 12 de diciembre de 2017
Horizon 2020 Health Summit-IIS La Fe Valencia, 12 de diciembre de 2017 2 History 3 AsphaTeam +75 members Consultants with Life Science Background 20 team members with PhD High knowhow in drug development
More informationThe Role of Adult Stem Cells in Personalized and Regenerative Medicine
The Role of Adult Stem Cells in Personalized and Regenerative Medicine Christopher J. Neill, Director of Corporate Operations American CryoStem Corporation Objective To illustrate the potential benefits
More informationPioneering the Development of Safe and Effective Non-Viral Vectors and Processes for Human Gene Therapy and DNA Vaccination.
Pioneering the Development of Safe and Effective Non-Viral Vectors and Processes for Human Gene Therapy and DNA Vaccination. Founded 1999, Clague Hodgson, Ph.D. Chief Scientific Officer, VP, R&D, Jim Williams,
More informationNanosystems in regenerative medicine. Jöns Hilborn Materials Chemistry The Ångström Laboratory Uppsala University Sweden
Nanosystems in regenerative medicine Jöns Hilborn Materials Chemistry The Ångström Laboratory Uppsala University Sweden Outline Motivation for tissue regeneration Cell based approaches Material based
More informationThe NYSCF Research Institute
Regenerative Medicine Research Update Susan L. Solomon The NYSCF Research Institute New York Pharma Forum February 25, 2016 Regenerative Medicine Research Regenerative medicine uses laboratory grown human
More informationGlobal Human Embryonic Stem Cell (hesc) Market Professional Survey Report 2016
Published on Market Research Reports Inc. (https://www.marketresearchreports.com) Home > Global Human Embryonic Stem Cell (hesc) Market Professional Survey Report 2016 Global Human Embryonic Stem Cell
More informationStem Cells: Introduction and Prospects in Regenerative Medicine.
Stem Cells: Introduction and Prospects in Regenerative Medicine www.gothamgazette.com/.../stemcell/stem_cell.jpg Ode to a Stem Cell, Part II by VCW There once was stem cell stuck in the hood Dividing endlessly,
More informationThe era of biological medicines
Chapter 1 The era of biological medicines Since their first use in the 1980s, biological medicines (including biosimilar medicines) have grown to become an indispensable tool in modern medicine. Worldwide,
More informationInvestor Presentation
Investor Presentation December 2013 Safe Harbor Statement This presentation contains forward-looking statements within the meaning of the safe harbor provisions of the Private Securities Litigation Reform
More informationCommercializing Regenerative Medicine and the Importance of Collaboration September 7 th, 2012
Commercializing Regenerative Medicine and the Importance of Collaboration September 7 th, 2012 Scientia Advisors, LLC 55 Cambridge Parkway, Suit 300 Cambridge, MA 02142 www.scientiaadv.com 2012 Scientia
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 informationleading the way in research & development
leading the way in research & development people. passion. possibilities. ABBVIE 2 immunology AbbVie Immunology has a demonstrated record of success in identifying and developing both small molecule and
More informationThe Cell and Gene Therapy Catapult UK clinical trials database
The Cell and Gene Therapy Catapult UK clinical trials database The UK clinical trials database covers cell and gene therapy clinical trial activities that the Cell and Gene Therapy Catapult (CGT Catapult)
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 informationThe Cell and Gene Therapy Catapult UK clinical trials database
The Cell and Gene Therapy Catapult UK clinical trials database The UK clinical trials database covers cell and gene therapy clinical trial activities that the Cell and Gene Therapy Catapult (CGT Catapult)
More informationTranslational Research
Translational Research Paddy Johnston Chair, MRC Translational Research Overview Group October 2012 Translational Research Strategy and its delivery To accelerate patient benefit and increase economic
More informationCambrex Cell Therapy. Buckingham Research Conference Call
Cambrex Cell Therapy Buckingham Research Conference Call April 14, 2005 Safe Harbor Statement Today s presentation and discussion will contain forward-looking statements including statements regarding
More informationthe PLATFORM Pipeline in Lyon AREA
the PLATFORM Pipeline in Lyon AREA Do you have development or partnership projects in Life Sciences? Are you interested in developing your business in France? This overview is designed to give you an idea
More informationLeading the world in novel adult stem cell therapies Half-Year Financial Results
Leading the world in novel adult stem cell therapies 2013 Half-Year Financial Results CAUTIONARY NOTE REGARDING FORWARD-LOOKING STATEMENTS This presentation, including any comments made during or following
More informationProduction and commercialisation of vascularized and customized bone Clinical trials, market authorisation Pre-clinical trials
Production and commercialisation of vascularized and customized bone Clinical trials, market authorisation Pre-clinical trials Basic Research: scaffolds, cells/ media, in vivo imaging, modelling www.vascubone.eu
More informationEUROPEAN COMMISSION HEALTH AND CONSUMERS DIRECTORATE-GENERAL. PHARMACEUTICAL COMMITTEE 22 October 2012
EUROPEAN COMMISSION HEALTH AND CONSUMERS DIRECTORATE-GENERAL Health systems and products Medicinal products authorisations, EMA PHARM 608 PHARMACEUTICAL COMMITTEE 22 October 2012 Subject: Hospital exemption
More informationCAR-T Cells enter center stage!
CAR-T Cells enter center stage! COSTEM, Berlin, October 2017 Molmed sponsored symposium»approaches to potentially overcome CAR-T cell toxicity: anticytokine antibodies and suicide genes" Christian CHABANNION
More informationGene therapy. Findings by Alert
Gene therapy Published Mar 21, 2000 Version 1 Findings by Alert Research in gene therapy has increased dramatically during the past 15 years, particularly in the United States. The research has encompassed
More informationUNIT CELL PROCESSES UNDERLYING TISSUE ENGINEERING AND REGENERATIVE MEDICINE
Massachusetts Institute of Technology Harvard Medical School Brigham and Women s Hospital VA Boston Healthcare System 2.79J/3.96J/20.441/HST522J UNIT CELL PROCESSES UNDERLYING TISSUE ENGINEERING AND REGENERATIVE
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 informationANAT 2341 Embryology Lecture 18 Stem Cells
ANAT 2341 Embryology Lecture 18 Stem Cells 29 September 2010 Dr Antonio Lee Neuromuscular & Regenera
More informationJoint Technology Initiative: Innovative Medicine Initiative
Joint Technology Initiative: Innovative Medicine Initiative Dr John E Butler-Ransohoff Global External Innovation and Alliances Bayer HealthCare AG, Berlin/Wuppertal Bari, 30 April 2015 Largest Public
More informationINUED DISCONTINUED DISCONTINUED DISCON MAKING THE IMPOSSIBLE POSSIBLE CENTER FOR REGENERATIVE MEDICINE
INUED DISCONTINUED DISCONTINUED DISCON MAKING THE IMPOSSIBLE POSSIBLE CENTER FOR > SOLUTIONS AND HOPE Millions of people worldwide suffer from deadly diseases, chronic conditions and congenital disorders
More informationSpecial Issue. Mesoblast Limited
Overview is an Australian biotechnology company committed to the commercialization of novel treatments for orthopedic conditions by using its unique adult stem cell technology for the regeneration and
More informationIndividualised medicine: regulatory challenges
www.pei.de Individualised medicine: regulatory challenges Outline Revision IVD Directive Opportunities and challenges Possibilities for interaction http://upload.wikimedia.org/wikipedia/commons/thumb/6/66/gummy_bears.jpg
More informationWorld Stem Cell Summit 2012
World Stem Cell Summit 2012 NYSE MKT: BTX December 4, 2012 Forward Looking Statements The matters discussed in this presentation include forward looking statements which are subject to various risks, uncertainties,
More informationThe National Institutes of Health ICs: mission and funding strategies
The National Institutes of Health ICs: mission and funding strategies Perry Kirkham, Ph.D. Office of the Vice President for Research E-mail: pkirkham@purdue.edu Phone: 63645 NIH Workshop topics and dates
More informationFamily Cord Blood Banks ADAPT TO THE Changing Business Landscape
By Laura Fusco CONTRIBUTING WRITER Family Cord Blood Banks ADAPT TO THE Changing Business Landscape R&D, awareness, science and the bottom line intersect as the industry evolves. T he cord blood banking
More informationSupplementary Materials for
www.sciencetranslationalmedicine.org/cgi/content/full/6/242/242cm6/dc1 Supplementary Materials for Sandbox: Building and Sharing Resources This PDF file includes: Mark David Lim E-mail: mlim@fastercures.org
More informationStem Cell Principle -
Effective Date: 31.10.2017 Doc ID: 20290214 Version: 1.0 Status: Approved Planned Effective Date: 31-Oct-2017 00:00 CET (Server Date) Stem Cell Principle - Rationale Research on human stem cells and their
More informationBayer Pharma s High Tech Platform integrates technology experts worldwide establishing one of the leading drug discovery research platforms
Bayer Pharma s High Tech Platform integrates technology experts worldwide establishing one of the leading drug discovery research platforms Genomics Bioinformatics HTS Combinatorial chemistry Protein drugs
More informationASX Spotlight Conference
ASX Spotlight Conference Duncan Thomson Head of Animal Health Singapore & Hong Kong October 2013 Improving quality of life through innovative cellular therapies Important Notice No offer to sell, issue
More informationASX Spotlight Conference
ASX Spotlight Conference Duncan Thomson Head of Animal Health Singapore & Hong Kong October 2013 Improving quality of life through innovative cellular therapies Important Notice No offer to sell, issue
More informationThe era of biological medicines
Chapter 1 The era of biological medicines Since their first use in the 1980s, biological medicines (including biosimilar medicines) have grown to become an indispensable tool in modern medicine. Worldwide,
More informationMarket Research at the NCBC Library
Market Research at the NCBC Library North Carolina Biotechnology Center Library 15 T.W. Alexander Drive, Research Triangle Park Phone: 919-549-8880 E-mail: library@ncbiotech.org The reports in this list
More informationE&T objectives in evolving pharma business models. Magda Chlebus Director Science Policy Brussels, 2 March 2015
E&T objectives in evolving pharma business models Magda Chlebus Director Science Policy Brussels, 2 March 2015 Education adapted to demand? Declining number of students choosing science Education still
More informationInsight to Gene Techno Science Co.,Ltd
Unlimited drug discovery from the beginning Ticker symbol: 4584 Insight to Gene Techno Science Co.,Ltd November 2017 2 Corporate Overview Chief Executive Founded March 2001 Masaharu Tani, President Listed
More informationAdis Journals and Newsletters The premier collection of drug-focused medical journals
adis.com Adis Journals and Newsletters The premier collection of drug-focused medical journals An invaluable resource for all involved in medical research, practice or teaching, drug regulation or reimbursement,
More informationREIMAGINING DRUG DEVELOPMENT:
Biology Reconstructed REIMAGINING DRUG DEVELOPMENT: Accurate Disease Modeling To Drive Successful Therapies Julia Kirshner, CEO julia@zpredicta.com 1 SUCCESS RATES OF DRUG DEVELOPMENT ARE LOW, " PARTICULARLY
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 informationCurrent Issues in EU Cell & Gene Therapy Regulation. Christiane Niederlaender, UK CAT Delegate, MHRA
Current Issues in EU Cell & Gene Therapy Regulation Christiane Niederlaender, UK CAT Delegate, MHRA.CASSS C> June 2018 Disclaimer The views expressed in this presentation are my personal views and may
More informationAndrew Finnerty General Manager - CCMI
Andrew Finnerty General Manager - CCMI Manufacturing Human Mesenchymal Stem Cells for Clinical Trials Quality Considerations Biopharma and Pharma RQA Regional Forum Bioclin Laboratories, Athlone 13 May
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 informationInnovative Medicines Initiative Sadallah Fatiha IMI JU Office, Brussels, Belgium
Innovative Medicines Initiative 2 2009-2014 Sadallah Fatiha IMI JU Office, Brussels, Belgium The Evolution of IMI: From bottlenecks in industry to bottlenecks in Industry and Society Make Drug R&D processes
More informationHow CAT Works: Regulatory Process, Patient Contribution and Outcomes
www.eurordis.org How CAT Works: Regulatory Process, Patient Contribution and Outcomes Michele Lipucci Di Paola AVLT/EURORDIS CAT/EMA Patient Representative michele.lipucci@eurordis.org EURORDIS MEMBERSHIP
More informationDiscovery of a First-In-Class Topically Bioavailable Kit Inhibitor With Clinical Activity Using Computational Chemogenomics Technology
20131009-03 Discovery of a First-In-Class Topically Bioavailable Kit Inhibitor With Clinical Activity Using Computational Chemogenomics Technology James Hendrix, Ph.D. President - Technology Med Chem &
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 informationRegenerative Medicine and Stem Cell Therapies
Regenerative Medicine and Stem Cell Therapies Regenerative Medicine Major component of successful regenerated / tissue engineered organs Scaffolds A critical element is the binding of the repopulating
More informationSCM Lifescience COMPANY PROFILE BUSINESS AREAS. [Address] 310, 366 Seohae-daero, Jung-gu, Incheon, Korea [Website]
SCM Lifescience [Address] 310, 366 Seohae-daero, Jung-gu, Incheon, Korea 22332 [Website] http://www.scmlifescience.com COMPANY PROFILE [Industry] SCM Lifescience is a research-based biopharmaceutical company
More informationStem cell and cell therapeutics research in Lithuania. Bernardas Morkunas Head of Business Development
Stem cell and cell therapeutics research in Lithuania Bernardas Morkunas Head of Business Development Lithuanian biotech Biotechnology is considered as one of 2014-2020 Smart Specialisation priority fields
More informationGeographical Distribution of the Tissue Engineering Market Regulatory Differences TABLE 3 DIFFERENCES IN REGULATORY APPROVAL: U.S. VS. EUR
CHAPTER ONE: INTRODUCTION... 1 STUDY GOALS AND OBJECTIVES... 1 REASONS FOR DOING THIS STUDY... 1 SCOPE AND FORMAT... 2 METHODOLOGY AND INFORMATION SOURCES... 2 INTENDED AUDIENCE... 2 ANALYST CREDENTIALS...
More informationABPI response to European Commission consultation on advanced therapy medicinal products
ABPI response to European Commission consultation on advanced therapy medicinal products 28 March 2013 ABPI response to European Commission consultation on the regulation of advanced therapy medicinal
More informationRegenerative Medicine and the Changing Regulatory Landscape
Regenerative Medicine and the Changing Regulatory Landscape May 3, 2018 Anne Marie Polak Leavitt Partners Gene Therapy CRISPR-Cas9 Inspired by genetic defense mechanisms found in bacteria Cas9 is an enzyme
More informationHarvard Stem Cell Institute
Welcome to the Harvard Stem Cell Institute The 1000+ scientists in the HSCI network share one goal: finding cures for human diseases. We aim to: Stimulate healing in patients by harnessing the potential
More informationVoyager Therapeutics - A Spinout from UMass Gene Therapy and RNAi Technologies
University of Massachusetts Medical School escholarship@umms UMass Center for Clinical and Translational Science Research Retreat 2016 UMass Center for Clinical and Translational Science Research Retreat
More informationAgenzia Italiana del Farmaco
Agenzia Italiana del Farmaco European Regulation on Advanced Therapies Cristina Pintus Head of European Relations Unit and Coordinator of the Advanced Therapy Project Italian Medicines Agency Proposal
More informationAdvanced Therapy Medicinal Products: Role and Funtion of the CAT. Martina Schuessler-Lenz,M.D. Paul-Ehrlich Institut, Germany
Advanced Therapy Medicinal Products: Role and Funtion of the CAT Martina Schuessler-Lenz,M.D. Paul-Ehrlich Institut, Germany Paul-Ehrlich Institut, Langen, Germany -German Federal Agency for Sera and Vaccines
More informationAnika Therapeutics, Inc.
Anika Therapeutics, Inc. Jefferies Global Healthcare Conference June 4, 2015 Safe Harbor Statement The statements made in this presentation that are not statements of historical fact are forward looking
More informationRegulation of Cell and Gene Therapy Products in Canada
Regulation of Cell and Gene Therapy Products in Canada Canadian Blood and Bone Marrow Transplant Group June 8, 2018 Nadine Kolas, PhD Senior Policy Analyst Blood, Cells, Tissues and Organs Biologics and
More informationThe European Landscape for Regenerative Medicine
Tokyo, December 8-9, 2014 The European Landscape for Regenerative Medicine Margarida Menezes Ferreira Senior Assessor at INFARMED PT expert at BWP/CHMP - EMA member of the CAT - EMA (margarida.menezes@infarmed.pt)
More informationNew Ways to Source Innovation in the Healthcare Industry In-licensing needs Oncology of Janssen/Johnson & Johnson Innovation
New Ways to Source Innovation in the Healthcare Industry In-licensing needs Oncology of Janssen/Johnson & Johnson Innovation Dr. Klaus Suwelack Johnson & Johnson Innovation, Janssen-Cilag Germany, Neuss
More informationT H E R A P Y REGENERATIVE MEDICINE NATURAL EFFECTIVE LIFE-CHANGING
S T E M C E L L T H E R A P Y REGENERATIVE MEDICINE NATURAL EFFECTIVE LIFE-CHANGING STEM CELL THERAPY Early stem cell research has traditionally been associated with the controversial use of embryonic
More informationIs traditional life science venture capital a resource for cell therapy companies yet? Edmond de Rothschild Investment Partners
Is traditional life science venture capital a resource for cell therapy companies yet? Gilles NOBÉCOURT Partner, Life Sciences Edmond de Rothschild Investment Partners PRIVATE EQUITY Content 1) Venture
More informationRNA BASED THERAPEUTICS, A R&D CUTTING EDGE
PRESENTAZIONE AZIENDALE A Ferrara University Spin off RNA BASED THERAPEUTICS, A R&D CUTTING EDGE MISSION: to discover, patent and develop innovative biotechnological drugs (Gene Therapy Medicinal Products)
More informationVision, aims and strategies. Department of Immunology, Genetics and Pathology Uppsala University
Vision, aims and strategies Department of Immunology, Genetics and Pathology Uppsala University April19,2011 SCOPEOFACTIVITIESATIGP Research at the Department focuses on translational medicine through
More informationInformation Day Leeds, UK 27 October 2015 Treating and managing disease
The societal challenge 'Health, demographic change and well-being' Work programme 2016-2017 Information Day Leeds, UK 27 October 2015 Treating and managing disease Image credit: Adam Fagen Rallying for
More informationThe Innovate UK response to the Innovative Medicines Initiative consultation Facilitating the translation of advanced therapies to patients in Europe
The Innovate UK response to the Innovative Medicines Initiative consultation Facilitating the translation of advanced therapies to patients in Europe Innovate UK and ATMP background 1. Innovate UK is the
More informationIndia as an Outsourcing Frontier in Biotechnology
1 India as an Outsourcing Frontier in Biotechnology K.V. Subramaniam President & CEO 3 rd Annual Biotech Supply Chain Academy Conference San Francisco Oct 26, 2010 Biotechnology Industry Issues Long gestation
More informationDrug /Device Combinations: The Convergence of Pharmaceutical and Biomaterial Science
Drug /Device Combinations: The Convergence of Pharmaceutical and Biomaterial Science Are We Just Raising the Stakes for Industry, Regulators, and Decision Makers? David Ames Director, Health Economics
More informationRe: National Coverage Analysis (NCA) Tracking Sheet for Chimeric Antigen Receptor (CAR) T-cell Therapy for Cancers (CAG-00451N)
June 15, 2018 Katherine B. Szarama, PhD Lead Analyst Coverage and Analysis Group Centers for Medicare & Medicaid Services Department of Health and Human Services 7500 Security Blvd. Baltimore, MD 21244
More informationPACT. PACT Program. Production Assistance for Cellular Therapies
PACT Production Assistance for Cellular Therapies University of Wisconsin PACT-sponsored Workshop Developing Cellular Therapies: From Preclinical Safety To Clinical Evaluation Tuesday, April 09, 2013 Robert
More informationThe Regulatory Environment for Therapeutic Vaccines. Thomas Hinz Head, Section Therapeutic Vaccines Paul Ehrlich Institute, Germany
The Regulatory Environment for Therapeutic Vaccines Thomas Hinz Head, Section Therapeutic Vaccines Paul Ehrlich Institute, Germany hinth@pei.de 1 Topics Addressed Regulatory environment - EU - Germany
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 informationUniversity of Florida College of Engineering EDGE Course EMA 6590/6938: Advances in Biomaterials and Tissue Engineering for Healthcare
University of Florida College of Engineering EDGE Course EMA 6590/6938: Advances in Biomaterials and Tissue Engineering for Healthcare Catalog Description Use of new bioactive and bio-nano structures,
More informationWorld s 1 st Clinical Trial with Allogeneic ipsc-derived Therapeutic Product (Cynata Therapeutics) Physician Led Study Using ipscs for
Table of Contents SCOPE AND METHODOLOGY... 8 Market Definition... 8 Market Scope... 8 Market Stakeholders... 9 Research Methodology... 10 Secondary and Primary Research Methodology... 10 Market Size Estimation
More information