Lentiviral vector bioprocess economics for cell and gene therapy commercialization

Lentiviral Vectors for the Treatment and Prevention of Cystic Fibrosis Lung Disease

(2019) Laura Marquez Loza et al.   Genes

Lentiviral Gene Therapy Combined with Low-Dose Busulfan in Infants with SCID-X1

(2019) Ewelina Mamcarz et al.   NEW ENGLAND JOURNAL OF MEDICINE

Lentiviral haemopoietic stem/progenitor cell gene therapy for treatment of Wiskott-Aldrich syndrome: interim results of a non-randomised, open-label, phase 1/2 clinical study

(2019) Francesca Ferrua et al.   Lancet Haematology

Gene Therapy Leaves a Vicious Cycle

(2019) Reena Goswami et al.   Frontiers in Oncology

Towards a scalable purification protocol of GaLV-TR pseudotyped lentiviral vectors.

(2019) Driss Boudeffa et al.   Human Gene Therapy Methods

Estimating Capital Investment and Facility Footprint in Cell Therapy Facilities

(2019) Tania D Pereira Chilima et al.   BIOCHEMICAL ENGINEERING JOURNAL

Development of a forward-oriented therapeutic lentiviral vector for hemoglobin disorders

(2019) Naoya Uchida et al.   Nature Communications

Preclinical Proof-of-Concept, Analytical Development, and Commercial Scale Production of Lentiviral Vector in Adherent Cells

(2019) Hanna M. Leinonen et al.   Molecular Therapy-Methods & Clinical Development

Lentiviral Vector Purification Using Nanofiber Ion-Exchange Chromatography

(2019) Jelena Ruscic et al.   Molecular Therapy-Methods & Clinical Development

Production of Lentiviral Vectors Using Suspension Cells Grown in Serum-free Media

(2019) Matthew Bauler et al.   Molecular Therapy-Methods & Clinical Development

Cost-effective bioprocess design for the manufacture of allogeneic CAR-T cell therapies using a decisional tool with multi-attribute decision-making analysis

(2018) Michael J. Jenkins et al.   BIOCHEMICAL ENGINEERING JOURNAL

Impact of allogeneic stem cell manufacturing decisions on cost of goods, process robustness and reimbursement

(2018) Tania D. Pereira Chilima et al.   BIOCHEMICAL ENGINEERING JOURNAL

Induction of a central memory and stem cell memory phenotype in functionally active CD4+ and CD8+ CAR T cells produced in an automated good manufacturing practice system for the treatment of CD19+ acute lymphoblastic leukemia

(2018) Franziska Blaeschke et al.   CANCER IMMUNOLOGY IMMUNOTHERAPY

Gene therapy clinical trials worldwide to 2017: An update

(2018) Samantha L. Ginn et al.   JOURNAL OF GENE MEDICINE

Long-Term Follow-Up of a Phase I/II Study of ProSavin, a Lentiviral Vector Gene Therapy for Parkinson's Disease

(2018) Stéphane Palfi et al.   Human Gene Therapy Clinical Development

Resveratrol improves ex vivo expansion of CB-CD34+ cells via downregulating intracellular reactive oxygen species level

(2018) Chaochun Tang et al.   JOURNAL OF CELLULAR BIOCHEMISTRY

Progressing from transient to stable packaging cell lines for continuous production of lentiviral and gammaretroviral vectors

(2018) Jaeyoung Park et al.   Current Opinion in Chemical Engineering

Optimization of lentiviral vector production for scale-up in fixed-bed bioreactor

(2017) A J Valkama et al.   GENE THERAPY

Lentiviral Vector Gene Transfer of Endostatin/Angiostatin for Macular Degeneration (GEM) Study

(2017) Peter A. Campochiaro et al.   HUMAN GENE THERAPY

Gene Therapy for β-Hemoglobinopathies

(2017) Marina Cavazzana et al.   MOLECULAR THERAPY

Gene Therapy in a Patient with Sickle Cell Disease

(2017) Jean-Antoine Ribeil et al.   NEW ENGLAND JOURNAL OF MEDICINE

Autologous lymphapheresis for the production of chimeric antigen receptor T cells

(2017) Elizabeth S. Allen et al.   TRANSFUSION

Considerations in T Cell Therapy Product Development for B Cell Leukemia and Lymphoma Immunotherapy

(2017) Andrew D. Fesnak et al.   Current Hematologic Malignancy Reports

Development of the First World Health Organization Lentiviral Vector Standard: Toward the Production Control and Standardization of Lentivirus-Based Gene Therapy Products

(2017) Yuan Zhao et al.   Human Gene Therapy Methods

Scalable Lentiviral Vector Production Using Stable HEK293SF Producer Cell Lines

(2017) Aziza P. Manceur et al.   Human Gene Therapy Methods

Efficient Ex Vivo Engineering and Expansion of Highly Purified Human Hematopoietic Stem and Progenitor Cell Populations for Gene Therapy

(2017) Erika Zonari et al.   Stem Cell Reports

Genetic Engineering and Manufacturing of Hematopoietic Stem Cells

(2017) Xiuyan Wang et al.   Molecular Therapy-Methods & Clinical Development

Viral vectors for gene therapy and gene modification approaches

(2016) Otto-Wilhelm Merten et al.   BIOCHEMICAL ENGINEERING JOURNAL

Patient-specific hiPSC bioprocessing for drug screening: Bioprocess economics and optimisation

(2016) Michael Jenkins et al.   BIOCHEMICAL ENGINEERING JOURNAL

The growing world of CAR T cell trials: a systematic review

(2016) Astrid Holzinger et al.   CANCER IMMUNOLOGY IMMUNOTHERAPY

Challenges of up-scaling lentivirus production and processing

(2016) Alexandra McCarron et al.   JOURNAL OF BIOTECHNOLOGY

Process change evaluation framework for allogeneic cell therapies: impact on drug development and commercialization

(2016) Sally Hassan et al.   Regenerative Medicine

Lentiviral hematopoietic stem cell gene therapy for X-linked severe combined immunodeficiency

(2016) Suk See De Ravin et al.   Science Translational Medicine

Preparation for a first-in-man lentivirus trial in patients with cystic fibrosis

(2016) Eric W F W Alton et al.   THORAX

CAR-T Cell Therapies From the Transfusion Medicine Perspective

(2016) Andrew Fesnak et al.   TRANSFUSION MEDICINE REVIEWS

Production of lentiviral vectors

(2016) Otto-Wilhelm Merten et al.   Molecular Therapy-Methods & Clinical Development

Process Development of Adenoviral Vector Production in Fixed Bed Bioreactor: From Bench to Commercial Scale

(2015) Hanna P. Lesch et al.   HUMAN GENE THERAPY

Outcomes Following Gene Therapy in Patients With Severe Wiskott-Aldrich Syndrome

(2015) Salima Hacein-Bey Abina et al.   JAMA-JOURNAL OF THE AMERICAN MEDICAL ASSOCIATION

474. A New cGMP Facility To Produce Clinical Grade Batches of Lentiviral Vectors and CAR-T Cell in Europe

(2015) Abdel Zemmar et al.   MOLECULAR THERAPY

NY-ESO-1–specific TCR–engineered T cells mediate sustained antigen-specific antitumor effects in myeloma

(2015) Aaron P Rapoport et al.   NATURE MEDICINE

Chimeric Antigen Receptor T Cells against CD19 for Multiple Myeloma

(2015) Alfred L. Garfall et al.   NEW ENGLAND JOURNAL OF MEDICINE

Allogeneic cell therapy bioprocess economics and optimization: downstream processing decisions

(2015) Sally Hassan et al.   Regenerative Medicine

Large-scale production of lentiviral vector in a closed system hollow fiber bioreactor

(2015) Jonathan Sheu et al.   Molecular Therapy-Methods & Clinical Development

Going viral: chimeric antigen receptor T-cell therapy for hematological malignancies

(2014) Saar Gill et al.   IMMUNOLOGICAL REVIEWS

Universal Real-Time PCR-Based Assay for Lentiviral Titration

(2014) Wojciech Barczak et al.   MOLECULAR BIOTECHNOLOGY

EIAV-Based Retinal Gene Therapy in the shaker1 Mouse Model for Usher Syndrome Type 1B: Development of UshStat

(2014) Marisa Zallocchi et al.   PLoS One

Preclinical safety and efficacy of an anti–HIV-1 lentiviral vector containing a short hairpin RNA to CCR5 and the C46 fusion inhibitor

(2014) Orit Wolstein et al.   Molecular Therapy-Methods & Clinical Development

Allogeneic cell therapy bioprocess economics and optimization: Single-use cell expansion technologies

(2013) Ana S. Simaria et al.   BIOTECHNOLOGY AND BIOENGINEERING

Good Manufacturing Practices (GMP) manufacturing of advanced therapy medicinal products: a novel tailored model for optimizing performance and estimating costs

(2013) Mohamed Abou-El-Enein et al.   CYTOTHERAPY

Lentiviral Hematopoietic Stem Cell Gene Therapy in Patients with Wiskott-Aldrich Syndrome

(2013) A. Aiuti et al.   SCIENCE

Lentiviral Hematopoietic Stem Cell Gene Therapy Benefits Metachromatic Leukodystrophy

(2013) A. Biffi et al.   SCIENCE

Development of Hematopoietic Stem Cell Based Gene Therapy for HIV-1 Infection: Considerations for Proof of Concept Studies and Translation to Standard Medical Practice

(2013) David DiGiusto et al.   Viruses-Basel

Fed-batch and perfusion culture processes: Economic, environmental, and operational feasibility under uncertainty

(2012) James Pollock et al.   BIOTECHNOLOGY AND BIOENGINEERING

Integration of stochastic simulation with multivariate analysis: Short-term facility fit prediction

(2012) Adam Stonier et al.   BIOTECHNOLOGY PROGRESS

Raising the Bar: The Curative Potential of Human Cancer Immunotherapy

(2012) S. A. Rosenberg   Science Translational Medicine

Downstream Processing of Lentiviral Vectors: Releasing Bottlenecks

(2012) Vanessa Bandeira et al.   Human Gene Therapy Methods

Transduction of Human CD34+ Repopulating Cells with a Self-Inactivating Lentiviral Vector for SCID-X1 Produced at Clinical Scale by a Stable Cell Line

(2012) Michael R. Greene et al.   Human Gene Therapy Methods

Suicide Gene Therapy to Increase the Safety of Chimeric Antigen Receptor-Redirected T Lymphocytes

(2012) Monica Casucci et al.   Journal of Cancer

Gene therapy finds its niche

(2011) Cormac Sheridan   NATURE BIOTECHNOLOGY

Large-Scale Production Means for the Manufacturing of Lentiviral Vectors

(2010) M. Schweizer et al.   CURRENT GENE THERAPY

Large-Scale Manufacture and Characterization of a Lentiviral Vector Produced for Clinical Ex Vivo Gene Therapy Application

(2010) Otto-Wilhelm Merten et al.   HUMAN GENE THERAPY

A Stable Producer Cell Line for the Manufacture of a Lentiviral Vector for Gene Therapy of Parkinson's Disease

(2010) Hannah J. Stewart et al.   HUMAN GENE THERAPY

Transfusion independence and HMGA2 activation after gene therapy of human β-thalassaemia

(2010) Marina Cavazzana-Calvo et al.   NATURE

Hypothermic storage of isolated human hepatocytes: a comparison between University of Wisconsin solution and a hypothermosol platform

(2009) Alina Ostrowska et al.   ARCHIVES OF TOXICOLOGY

Simplified production and concentration of HIV-1-based lentiviral vectors using HYPERFlask vessels and anion exchange membrane chromatography

(2009) Robert H Kutner et al.   BMC BIOTECHNOLOGY

Enrichment of cell subpopulations applying automated MACS technique: purity, recovery and applicability for PCR-based chimerism analysis

(2009) A Willasch et al.   BONE MARROW TRANSPLANTATION

Hematopoietic stem cell gene therapy for adenosine deaminase deficient-SCID

(2009) Alessandro Aiuti et al.   IMMUNOLOGIC RESEARCH

The genotoxic potential of retroviral vectors is strongly modulated by vector design and integration site selection in a mouse model of HSC gene therapy

(2009) Eugenio Montini et al.   JOURNAL OF CLINICAL INVESTIGATION

Chimeric Receptors Containing CD137 Signal Transduction Domains Mediate Enhanced Survival of T Cells and Increased Antileukemic Efficacy In Vivo

(2009) Michael C. Milone et al.   MOLECULAR THERAPY

Hematopoietic Stem Cell Gene Therapy with a Lentiviral Vector in X-Linked Adrenoleukodystrophy

(2009) N. Cartier et al.   SCIENCE

Correction of the disease phenotype in the mouse model of Stargardt disease by lentiviral gene therapy

(2008) J Kong et al.   GENE THERAPY

From retroviral vector production to gene transfer: spontaneous inactivation is caused by loss of reverse transcription capacity

(2008) M. Carmo et al.   JOURNAL OF GENE MEDICINE

Decision-Support Tool for Assessing Biomanufacturing Strategies under Uncertainty: Stainless Steel versus Disposable Equipment for Clinical Trial Material Preparation

(2005) Suzanne S. Farid et al.   BIOTECHNOLOGY PROGRESS

Application of a Decision-Support Tool to Assess Pooling Strategies in Perfusion Culture Processes under Uncertainty

(2005) Ai Chye Lim et al.   BIOTECHNOLOGY PROGRESS