Report on Webinar Series Cell and Gene Therapy: From Concept to Clinical Use

With the launch of the UK Academy of Pharmaceutical Sciences Advanced Therapy Medicinal Products Focus Group in late 2020, a webinar series reviewing the current and emerging trends in cell and gene therapy was held virtually in May 2021. This webinar series was timely given the recent withdrawal of the United Kingdom from the European Union and the global COVID-19 pandemic impacting all sectors of the pharmaceutical sciences research landscape globally and in the UK. Delegates from the academic, industry, regulatory and NHS sectors attended the session where challenges and opportunities in the development and clinical implementation of cell and gene therapies were discussed. Globally, the cell and gene therapy market has reached a value of 4.3 billion dollars in 2020, having increased at a compound annual growth rate of 25.5% since 2015. This webinar series captured all the major developments in this rapidly evolving area and highlighted emerging concepts warranting cross-sector efforts from across the community in the future.

[1]  C. Dufès,et al.  Regression of Melanoma Following Intravenous Injection of Plumbagin Entrapped in Transferrin-Conjugated, Lipid–Polymer Hybrid Nanoparticles , 2021, International journal of nanomedicine.

[2]  T. Darwish,et al.  Apolipoprotein E Binding Drives Structural and Compositional Rearrangement of mRNA-Containing Lipid Nanoparticles , 2021, ACS nano.

[3]  H. Leung,et al.  Octadecyl chain-bearing PEGylated poly(propyleneimine)-based dendrimersomes: physicochemical studies, redox-responsiveness, DNA condensation, cytotoxicity and gene delivery to cancer cells. , 2020, Biomaterials science.

[4]  S. Boye,et al.  Current Clinical Applications of In Vivo Gene Therapy with AAVs , 2020, Molecular therapy : the journal of the American Society of Gene Therapy.

[5]  S. Nierkens,et al.  Gene Augmentation and Editing to Improve TCR Engineered T Cell Therapy against Solid Tumors , 2020, Vaccines.

[6]  J. Gauthier,et al.  Taming the beast: CRS and ICANS after CAR T-cell therapy for ALL , 2020, Bone Marrow Transplantation.

[7]  J Fraser Wright,et al.  Quality Control Testing, Characterization and Critical Quality Attributes of Adeno‐Associated Virus Vectors Used for Human Gene Therapy , 2020, Biotechnology journal.

[8]  P. Alves,et al.  Advances in Lentivirus Purification , 2020, Biotechnology journal.

[9]  L. Rittié,et al.  Short time to market and forward planning will enable cell therapies to deliver R&D pipeline value. , 2020, Human gene therapy.

[10]  Michael L. Wang,et al.  Lisocabtagene maraleucel for patients with relapsed or refractory large B-cell lymphomas (TRANSCEND NHL 001): a multicentre seamless design study , 2020, The Lancet.

[11]  Elizabeth L Siegler,et al.  Neurotoxicity and Cytokine Release Syndrome After Chimeric Antigen Receptor T Cell Therapy: Insights Into Mechanisms and Novel Therapies , 2020, Frontiers in Immunology.

[12]  C. Vink,et al.  Rapid Lentiviral Vector Producer Cell Line Generation Using a Single DNA Construct , 2020, Molecular therapy. Methods & clinical development.

[13]  W. Jiskoot,et al.  Particulate impurities in cell-based medicinal products traced by flow imaging microscopy combined with deep learning for image analysis. , 2020, Cytotherapy.

[14]  A. Bornot,et al.  Arrayed CRISPR Screening Identifies Novel Targets That Enhance the Productive Delivery of mRNA by MC3-Based Lipid Nanoparticles , 2020, SLAS discovery : advancing life sciences R & D.

[15]  S. Rives,et al.  Point-Of-Care CAR T-Cell Production (ARI-0001) Using a Closed Semi-automatic Bioreactor: Experience From an Academic Phase I Clinical Trial , 2020, Frontiers in Immunology.

[16]  A. Jones,et al.  Endocytic Profiling of Cancer Cell Models Reveals Critical Factors Influencing LNP-Mediated mRNA Delivery and Protein Expression. , 2019, Molecular therapy : the journal of the American Society of Gene Therapy.

[17]  H. Leung,et al.  Camptothecin-based dendrimersomes for gene delivery and redox-responsive drug delivery to cancer cells. , 2019, Nanoscale.

[18]  S. Grupp,et al.  ASTCT Consensus Grading for Cytokine Release Syndrome and Neurologic Toxicity Associated with Immune Effector Cells. , 2019, Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation.

[19]  Arpan S. Desai,et al.  Using macropinocytosis for intracellular delivery of therapeutic nucleic acids to tumour cells , 2018, Philosophical Transactions of the Royal Society B.

[20]  C. Dufès,et al.  Targeted nonviral gene therapy in prostate cancer , 2018, International journal of nanomedicine.

[21]  Elizabeth Csaszar,et al.  Commercial Scale Manufacturing of Allogeneic Cell Therapy , 2018, Front. Med..

[22]  B. Pickard,et al.  PEGylation of polypropylenimine dendrimers: effects on cytotoxicity, DNA condensation, gene delivery and expression in cancer cells , 2018, Scientific Reports.

[23]  C. Dufès,et al.  Tumor regression following intravenous administration of lactoferrin- and lactoferricin-bearing dendriplexes , 2015, Nanomedicine : nanotechnology, biology, and medicine.

[24]  Guideline on the quality, non-clinical and clinical aspects of gene therapy medicinal products , 2015 .

[25]  L. Couture,et al.  Production of CGMP-Grade Lentiviral Vectors. , 2012, BioProcess international.

[26]  Laurence Tetley,et al.  Tumor regression after systemic administration of a novel tumor-targeted gene delivery system carrying a therapeutic plasmid DNA. , 2010, Journal of controlled release : official journal of the Controlled Release Society.