CELL & GENE THERAPY INSIGHTS

CRISPR gene editing for sickle cell disease: one disease,...

L Michaels,
Lisa A Michaels, MD
Chief Medical Officer, Editas Medicine
B Eaton
Bruce E Eaton, PhD
Chief Business Officer, Editas Medicine
5 August 2021
Editorial

Strengthening the clinical supply chain for individualized...

A Case
James Andrew Case
Head of Clinical Supply Chain – Individualized Therapies, Genentech
5 August 2021
Interview

Driving clinical progress of gene therapy in cystic fibrosis

U Griesenbach
Uta Griesenbach
NHLI, Imperial College, Manresa Rd london SW3 6LR
28 July 2021
Interview

Challenges in the gene therapy of bone marrow failure syndromes

J Bueren
Juan Bueren
Director of the Biomedical Innovative Unit at the CIEMAT, IIS Fundación Jiménez Díaz and Biomedical Network Centre on Rare Diseases, Madrid, Spain and Vice-President, European Society for Cell and Gene Therapy
Juan Bueren is the Head of the Division of Hematopoietic Innovative Therapies of CIEMAT and CIBER of Rare Diseases. Dr Bueren is also Coordinator of the Mixed Unit of Advanced Therapies CIEMAT/IIS-Jiménez Díaz Foundation and consultant of Rocket Pharmaceuticals Inc, and currently serves as Vice-President of the European Society for Cell and Gene Therapy. Dr Bueren is Scientific Director of gene therapy trials for patients with Fanconi anemia and also for patients with the primary immunodeficiency, leukocyte adhesion deficiency type I, and has participated in the development of therapeutic lentiviral vectors designed as Orphan Drugs by the European Medicines Agency and by the FDA for these two diseases. Both ODs have been licensed to Rocket Pharmaceuticals which is currently developing different global gene therapy programs in Europe and in the USA for Fanconi anemia and LAD-I.
16 July 2021
Interview

Learning lessons from the long, troubled history of stem...

J Rasko
John EJ Rasko
Director, Department of Cell and Molecular Therapies at Royal Prince Alfred Hospital and the Gene and Stem Cell Therapy Program at the Centenary Institute, University of Sydney
John EJ Rasko is an Australian pioneer in the application of adult stem cells and genetic therapy. Since 1999 he has directed the Department of Cell and Molecular Therapies at Royal Prince Alfred Hospital and the Gene and Stem Cell Therapy Program at the Centenary Institute, University of Sydney. John Rasko is a clinical hematologist, pathologist and scientist with an international reputation in gene and stem cell therapy, experimental hematology and molecular biology. In over 200 publications he has contributed to the understanding of stem cells and blood cell development, gene therapy technologies, cancer causation and treatment, human genetic diseases and molecular biology. He serves on Hospital, state and national bodies including Chair of GTTAC, Office of the Gene Technology Regulator – responsible for regulating all genetically-modified organisms in Australia – and immediate past Chair of the Advisory Committee on Biologicals, Therapeutic Goods Administration. Contributions to scientific organizations include co-founding (2000) and past-President (2003–5) of the Australasian Gene & Cell Therapy Society; President (2018–20), President-Elect (2016–18) and Vice President (2008–12) of the International Society for Cell & Gene Therapy; Scientific Advisory Committees and Board member for philanthropic foundations; and several Human Research Ethics Committees. He is a founding Fellow of the Australian Academy of Health and Medical Sciences. In 2018, the Board of the ABC honored him as the sixtieth Boyer Lecturer. He is the recipient of national (RCPA, RACP, ASBMB) and international awards in recognition of his commitment to excellence in medical research, including appointment as an Officer of the Order of Australia.
15 July 2021
Interview

Addressing issues in clinical development of AAV-driven...

S Sallah
Sabah Sallah
Senior Vice President Gene Therapy, Translational Medicine and Hematology at Freeline Therapeutics, Boston, MA, USA
14 July 2021
Interview
  • Gain deeper insights into the mechanisms of immune cell killing of tumor...

    C Souders,
    T Dale,
    N Bevan et al.
    Colby Souders
    Colby Souders
    Chief Scientific Officer at Abveris
    Tim  Dale
    Tim Dale
    Head of BioAnalytics Applications at Sartorius
    Nicola Bevan
    Nicola Bevan
    Manager of Cell Imaging Applications at Sartorius
    Jeffrey Skolnik
    Jeffrey Skolnik
    Senior Vice President, Clinical Development at Inovio Pharmaceuticals, Inc
    6 Oct 2021
    7
    Days
    23
    Hrs
    53
    Min
    Register
  • Considerations for developing scalable and efficient collection network...

    L Anderson,
    S Butler
    Lacey Anderson
    Lacey Anderson
    Senior Manager, Collection Network Management at Be The Match BioTherapies
    Sara Butler
    Sara Butler
    at Nebraska Medicine
    28 Oct 2021
    30
    Days
    18
    Hrs
    20
    Min
    Register
  • Platform optimization for efficient AAV purification – insights from...

    N Laroudie,
    V Ravault
    Nicolas Laroudie
    Nicolas Laroudie
    Senior Field Application Specialist, Purification at Thermo Fisher Scientific
    Vincent Ravault
    Vincent Ravault
    Technician, Process Development and Industrialization Department at Yposkesi
    26 Oct 2021
    27
    Days
    23
    Hrs
    53
    Min
    Register
  • Optimization of AAV production for high-yielding and scalable GMP processes

    C Wartel-Weill,
    M Porte,
    B Kondragunta
    Claire Wartel-Weill
    Claire Wartel-Weill
    Director, Quality & Compliance at Polyplus-transfection
    Mathieu Porte
    Mathieu Porte
    Project Leader at Polyplus-transfection
    Bhargavi Kondragunta
    Bhargavi Kondragunta
    Director, Internal R&D and Product Development at Catalent Cell and Gene therapy
    20 Oct 2021
    21
    Days
    23
    Hrs
    53
    Min
    Register
  • cGMP manufacturing made easy – a case study by ElevateBio

    M Paglia,
    S Eaker
    Michael Paglia
    Michael Paglia
    Senior Vice President - CMC Operations at Elevatebio
    Shannon Eaker
    Shannon Eaker
    Cell Therapy Enterprise Technical Leader
    14 Oct 2021
    15
    Days
    23
    Hrs
    53
    Min
    Register
  • The therapeutic gene editing solution – cell therapy for solid tumors

    B Sasu,
    P Sivakumar,
    J Trager et al.
    Barbra Sasu
    Barbra Sasu
    Chief Scientific Officer at Allogene Therapeutics, Inc
    Pallavur Sivakumar
    Pallavur Sivakumar
    Scientific Vice President and Head of Discovery Immuno-Oncology and Cell Therapy Thematic Research Center at Bristol Myers Squibb
    James Trager
    James Trager
    Chief Scientific Officer at Nkarta Therapeutics, Inc
    Jennifer Harbottle
    Jennifer Harbottle
    Senior Scientist, R&D, Cell and Gene Therapy at Horizon Discovery
    30 Sep 2021
    1
    Days
    23
    Hrs
    53
    Min
    Register
  • Scalable, flexible manufacture: simplifying cell & gene therapy production

    J Bornholdt,
    N Kulkarni,
    C Houts
    James Bornholdt
    James Bornholdt
    Director, SlateXpace Technical Operations at CRB
    Niranjan Kulkarni
    Niranjan Kulkarni
    Director, Operations Improvement at CRB
    Carol Houts
    Carol Houts
    Director, Regulatory & Quality at Germfree Laboratories
    8 September 2021
    Watch now
  • Are you finishing strong in cell therapy manufacturing? Tackling your final...

    D Sethi,
    A Cunningham
    Dalip Sethi
    Dalip Sethi
    Manager, Senior Scientist at Terumo BCT
    Annie  Cunningham
    Annie Cunningham
    Cell Therapy Laboratory Scientist
    31 August 2021
    Watch now
  • GMP manufacturing for exosome-based therapies: the critical role of analytics...

    M Langtry,
    D Zocco
    Marcos Langtry
    Marcos Langtry
    Head of Allogeneic Cell Therapy - Commercial Development at Lonza
    Davide Zocco
    Davide Zocco
    Head of Allogeneic Cell Therapy Commercial Development at Lonza
    28 September 2021
    Watch now
  • Get your non-viral T cell engineering process ready for clinical manufacturing

    M Rietenbach,
    S Wild,
    K Patel
    Melanie Rietenbach
    Melanie Rietenbach
    Global Product Manager at Miltenyi Biotec
    Stefan Wild
    Stefan Wild
    Senior Manager Research and Development at Miltenyi Biotec
    Kunal Patel
    Kunal Patel
    Process engineering manager at Miltenyi Biotec
    21 September 2021
    Watch now
  • AAV manufacturing with stable producer cells for industrial scale vector production...

    B Hudjetz
    Ben Hudjetz
    Ben Hudjetz
    Senior Scientist, Cell Line Development at CEVEC Pharmaceuticals GmbH
    16 September 2021
    Watch now
  • The Setup and Payoff: Entering the clinic and the market efficiently by leveraging...

    K Beck
    Kevin Beck
    Kevin Beck
    Associate Director of Account Management. at Miltenyi Biotec
    1 September 2021
    Watch now
  • Channel content

    Reports

    FEATURING

    Evolving technology trends and regulatory pathways in the tissue engineered product space

    W Fodor
    William Fodor
    Chief Scientific Officer, Biostage
    William Fodor received his B.S. degree in Genetics and his Ph.D. degree in Molecular Genetics from The Ohio State University. He then completed his post-doctoral training in Immunobiology at the Yale School of Medicine (Mentor, R.A. Flavell). Dr. Fodor started his industrial career as a founding scientist of Alexion Pharmaceuticals, Inc. During his tenure at Alexion he directed the xenotransplantation, cell and tissue and organ engineering programs and advanced the program to include a pilot manufacturing facility for cell production. Dr. Fodor then moved to academia as an Associate Professor in the Center for Regenerative Biology at the University of Connecticut where he spent 3 years renovating his research to focus on stem cell biology for tissue repair. Dr. Fodor then returned to industry as Senior Director of Product Development at Viacell Inc., where he was responsible for various stem cell product development projects for hematologic indications as well as islet stem cell applications for diabetes. Following his tenure at Viacell, he continued his career in biotechnology as an independent consultant, serving many clients within the regenerative medicine field. Dr. Fodor’s relationship with Biostage was first as a consultant then as the Chief Scientific Officer, his current role.

    Broadening horizons for extracellular vesicles as both therapeutic modality and delivery tool

    J Correia
    Joana Correia
    CEO, Exogenus Therapeutics
    Joana Correia co-founded Exogenus Therapeutics in 2015, after a successful participation in COHiTEC program for technology commercialization. Having been awarded several honors, including three entrepreneurship prizes (Everis Foundation Award, Young Entrepreneur ANJE, and Prémio Empreendedor XXI BPI/CaixaBank), Joana has raised €3.5M in funds for R&D, and is currently a leader in the field of EV–based therapeutics recognized internationally. With a PhD in Human Biology, she dedicated 20 years of her career to R&D in the area of human diseases and healthcare, is inventor of two patents, has several publications in high impact journals, and is invited researcher of the Center for Neuroscience and Cell Biology (CNC, Coimbra, Portugal). Joana is an enthusiastic entrepreneur and activist, with a creative mindset and a strong drive to solve healthcare challenges.

    FEATURING

    Solving the problem of financing one-time treatments with evidence uncertainty: which types of outcomes-based payment models could work best for novel CAR-T therapies in multiple myeloma? A systematic review of the published literature

    C Dietrich,
    Cassidy-Candice Dietrich
    MSc Student, Department of Health Sciences and Medicine, University of Lucerne, Switzerland
    C Hague,
    Clare Hague
    Author for correspondence Honorary Lecturer & Academic Supervisor, Department of Health Sciences and Medicine, University of Lucerne, Switzerland and Therapy Area Market Access Leader, Hematology, Janssen EMEA Region
    S Boes
    Stefan Boes
    Professor of Health Economics, Department of Health Sciences and Medicine, University of Lucerne, Switzerland

    Critical need for establishing value that justifies the current rising costs of cell and gene therapy

    R Maziarz
    Richard T Maziarz, MD
    Professor of Medicine, Knight Cancer Institute, Oregon Health & Science University, Portland, OR 97232, USA maziarzr@ohsu.edu

    FEATURING

    Regulatory CMC in cell and gene therapy: navigating an evolving space

    L Starke
    Lawrence C. Starke
    Global RA-CMC Policy and Intelligence, Cell & Gene Therapy, Novartis
    Lawrence C. Starke is currently head of Regulatory CMC Policy and Intelligence at Novartis Pharmaceuticals Corporation in East Hanover, NJ. Prior to assuming his currently role, Dr. Starke was RA-CMC Unit Head for Cell and Gene Therapy Products at Novartis and held positions of increasing responsibility within the biologic RA-CMC roles at Eli Lilly and Company and at Merck. Dr. Starke received his Ph.D. in Cell and Molecular Biology at Duke University in Durham, North Carolina and was a research associate at Baylor College of Medicine in Houston, Texas prior to his career in the pharmaceutical industry.

    FEATURING

    Accelerating the development of novel gene therapies for central nervous system diseases

    S Prasad
    Suyash Prasad, MBBS, MSc, MRCP, MRCPCH, FFPM
    Chief Medical Officer and Head of Research and Development, Taysha Gene Therapies
    Suyash Prasad is a Pediatrician, Clinical Development Physician, Translational Scientist and Executive Leader with 20 years’ experience in the biopharmaceutical industry. He currently serves as the Chief Medical Officer and Head of Research and Development at Taysha Gene Therapies, an organization dedicated to developing AAV gene therapy approaches for treating children and adults with severe neurological disease. Suyash graduated in medicine at the University of Newcastle upon Tyne, UK where he received commendations for Pediatrics, Obstetrics and Gynecology, and Medical Ethics. His pediatric training was completed at recognized centers of excellence in the UK and Australia before he moved to industry. His industry career progressed at Eli Lilly, Genzyme, and BioMarin, and more recently he was Chief Medical Officer at the gene therapy company, Audentes Therapeutics (now Astellas Gene Therapies), before moving to Taysha. He is a UK board-certified physician and is a member of the Royal College of Physicians (MRCP) and the Royal College of Paediatrics and Child Health (MRCPCH). Suyash received his Diploma in Pharmaceutical Medicine from the Royal College of Physicians of the UK, and his Masters in Translation Science with distinction from Kings College, London. He is a Fellow of the Faculty of Pharmaceutical Medicine and is a past recipient of the Outstanding Contribution award from the Faculty. Suyash has dedicated his career to the well-being and advocacy of children who are afflicted by rare and severe disease.

    FEATURING

    Boldly de-risking development of impactful cell and gene therapies: the California Stem Cell Agency’s $3B funding model

    S Patel,
    Shyam Patel
    Associate Director, Portfolio Development & Review at California Institute For Regenerative Medicine
    S Talib,
    Sohel Talib
    Director, Therapeutics at California Institute For Regenerative Medicine
    M Millan
    Maria Millan
    Vice President, Therapeutics at California Institute For Regenerative Medicine

    Driving disruptive innovation in the ATMP field

    K Papenfuss
    Kerstin Papenfuss
    Associate Director, Therapeutics at Deep Science Ventures
    Kerstin Papenfuss started her PhD at German Cancer Research Centre and then moved to a lab at Imperial College London to work on novel treatment options within the field of tumor immunology. After a Post Doc in academic drug discovery, she since has spent almost ten years in leadership roles at impact-driven organizations advancing medicine and therapeutics, while also securing an executive MBA and a Women in Business award. Before joining DSV to develop science companies designing more effective therapies, Kerstin was transforming ideas for cutting edge cell and gene therapies into investable propositions at UK’s Cell and Gene Therapy Catapult.

    Latest content

    Gain deeper insights into the mechanisms of immune cell killing of tumor...

    C Souders,
    T Dale,
    N Bevan et al.
    Dr Colby Souders
    Dr Colby Souders
    Chief Scientific Officer, Abveris
    Tim Dale, PhD
    Tim Dale, PhD
    Head of BioAnalytics Applications, Sartorius
    Nicola Bevan
    Nicola Bevan
    Manager of Cell Imaging Applications, Sartorius
     Jeffrey Skolnik, M.D.
    Jeffrey Skolnik, M.D.
    Senior Vice President, Clinical Development, Inovio
    6 Oct 2021
    7
    Days
    23
    Hrs
    53
    Min
    Register

    Considerations for developing scalable and efficient collection network...

    L Anderson,
    S Butler
    Lacey Anderson
    Lacey Anderson
    Senior Manager, Collection Network Management, Be The Match BioTherapies
    Sara Butler
    Sara Butler
    Network Liaison, Be The Match BioTherapies
    28 Oct 2021
    30
    Days
    18
    Hrs
    20
    Min
    Register

    Scalable, flexible manufacture: simplifying cell & gene therapy production

    J Bornholdt,
    N Kulkarni,
    C Houts
    JP Bornholdt AIA, PE, LEED AP
    JP Bornholdt AIA, PE, LEED AP
    Director, SlateXpace Technical Operations, CRB
    Niranjan Kulkarni, PhD
    Niranjan Kulkarni, PhD
    Director, Operations Improvement, CRB
    Carol Houts
    Carol Houts
    Vice President of Quality and Business Strategy, Germfree
    8 September 2021
    Watch now

    Optimization of AAV production for high-yielding and scalable GMP processes

    C Wartel-Weill,
    M Porte,
    B Kondragunta
    Claire Wartel-Weill
    Claire Wartel-Weill
    Director, Quality & Compliance at Polyplus-transfection
    Mathieu Porte
    Mathieu Porte
    BioProduction R&D Manager, Polyplus-transfection
     Bhargavi Kondragunta
    Bhargavi Kondragunta
    Director of Internal R&D & Product Development, Catalent
    20 Oct 2021
    21
    Days
    23
    Hrs
    53
    Min
    Register

    Get your non-viral T cell engineering process ready for clinical manufacturing

    M Rietenbach,
    S Wild,
    K Patel
    Melanie Rietenbach
    Melanie Rietenbach
    Global Product Manager Miltenyi Biotec
    Stefan Wild
    Stefan Wild
    Senior Manager, Research and Development, Miltenyi Biotec
    Kunal Patel
    Kunal Patel
    Process Engineering Manager, Miltenyi Biotec
    21 September 2021
    Watch now

    Navigating regulations - novel cell therapy platforms and their path to clinical...

    N Bauer,
    N Calhoun,
    A Davies et al.
    Nina Bauer
    Nina Bauer
    Head of Commercial, Gene Editing and Novel Modalities, Merck KGaA
    Natika Calhoun
    Natika Calhoun
    Regulatory Consultant, Merck KGaA
    Anthony Davies
    Anthony Davies
    Founder and CEO, Dark Horse Consulting
    Matt Muldoon
    Matt Muldoon
    Senior Director, Supplier Management, Allogene Therapeutics
    27 May 2021
    Watch now

    Addressing the challenges of purification & quality control in gene therapy

    A Bhattacharya,
    L Kopp,
    K Richter et al.
    Dr. Akash Bhattacharya
    Dr. Akash Bhattacharya
    Senior Application Engineer, Beckman Coulter
    Leisha Kopp
    Leisha Kopp
    Applications Scientist, Mirus Bio
    Klaus Richter, PhD
    Klaus Richter, PhD
    Group Leader AUC, Coriolis Pharma
    Dr. Audrey Chang
    Dr. Audrey Chang
    Vice President of Quality Control and Analytical Service, Vigene Biosciences
    Shawn Sternisha
    Shawn Sternisha
    Senior Field Applications Scientist, Beckman Coulter Life Sciences
    24 June 2021
    Watch now