CELL & GENE THERAPY INSIGHTS

Gene delivery platform evolution

July 2021

  • The dose makes the poison: next generation AAV vectors can save the day

    J Fakhiri,
    Julia Fakhiri
    Roche Pharma Research and Early Development, Therapeutic Modalities, Roche Innovation Center Munich, Roche Diagnostics GmbH, Nonnenwald 2, 82377 Penzberg, Germany
    Julia Fakhiri obtained her Ph.D. in Biology from the University of Heidelberg (Germany), where she worked on different aspects of gene therapy, from parvovirus capsid engineering to in vivo applications of AAV-CRISPR/Cas9. In her postdoctoral training at the Department of Infectious Diseases of the University Hospital Heidelberg, she also worked on in vivo screening of AAV libraries in collaboration with industry partners. Dr. Fakhiri is currently a postdoctoral fellow at Roche Pharma Research and Early Development (pRED), Roche Diagnostics in Penzberg, Germany. Her research mainly focuses on the development of processes for production of recombinant viral vectors.
    J El Andari
    Jihad El Andari
    Dept. of Infectious Diseases/Virology, Medical Faculty, University of Heidelberg, Im Neuenheimer Feld 267, 69120 Heidelberg, Germany
    Jihad (josef) El Andari is currently a senior postdoctoral fellow in the laboratory of Dirk Grimm at the medical faculty and BioQuant center at the University of Heidelberg (Germany). His research primarily emphasizes on developing novel synthetic vectors with enhanced properties to target the musculature and the central nervous system. Dr. El Andari has obtained his Ph.D. in Molecular Microbiology from the University of Freiburg (Germany) that was followed by a postdoctoral stint at the Center of Synthetic Microbiology (SYNMIKRO) at the University of Marburg (Germany).
    13 July 2021
    Viewpoint
  • Expanding gene therapy and gene editing strategies to large and complex indications

    C Unzu,
    Carmen Unzu
    Apic Bio
    F Borel
    Florie Borel
    Apic Bio
    30 June 2021
    Editorial
  • Coming of age: taking the next steps with non-viral gene delivery

    M Stanton
    Matt Stanton
    Chief Scientific Officer, Generation Bio
    29 June 2021
    Interview
  • A bright future for lipid nanoparticles in gene therapy

    H Zhang
    Hongwei Zhang
    Associate Professor and Director of Pharmaceutical Sciences Program, Massachusetts College of Pharmacy and Health Sciences University
    Hongwei Zhang is Associate Professor and Director of Pharmaceutical Sciences Program at Massachusetts College of Pharmacy and Health Sciences University (MCPHS University) in Boston. The research in Zhang lab is focused on nanomedicine and drug/gene delivery. Dr Zhang received his BS in Pharmacy and PhD in Pharmaceutics from the West China School of Pharmacy at Sichuan University and completed postdoctoral training in the Gene Therapy Center at the University of Massachusetts Medical School. Dr Zhang has published ~70 peer-reviewed journal articles and conference papers in the field of gene delivery and gene therapy. He is the recipient of the Biotechnology Innovation Award from the American Association of Pharmaceutical Scientists. He is the co-inventor of an AAV gene therapy patent.
    9 July 2021
    Viewpoint
  • Cell and gene manufacturing: a case study approach to overcoming challenges – expert perspectives and solutions

    S Thompson
    Steven Thompson
    Director, Sales & Product Management at Sexton Biotechnologies
    9 July 2021
    FastFacts
  • Scalable upstream process development for the suspension-based production of lentiviral vectors for CAR T cell therapies with multiparallel & benchtop bioreactor systems & DoE methodology

    D Riethmüller,
    Diana Riethmüller
    Sartorius Stedim Biotech GmbH, Göttingen, Germany
    A Nyamay’Antu,
    Dr Alengo Nyamay’antu
    Polyplus-transfection, Illkirch, France
    Alengo Nyamay’antu is a Scientific Communication Specialist at Polyplus-transfection® SA, the leading biotechnology company that supports Gene and Cell therapy, biologics manufacturing and life science research with innovative nucleic acid transfection solutions. Alengo completed her MSc in structural and functional biochemistry at the University of Lyon and went on to specialise in protein biochemistry at the University of Manchester. She then continued to develop and widen her scientific and communication skills by joining the Max Planck Institute for Biomedicine as a postdoctoral researcher.
    F Bollmann
    Dr Franziska Bollmann
    Author for correspondence: Sartorius Stedim Biotech GmbH, Göttingen, Germany franziska.bollmann@sartorius.com
    8 July 2021
    Innovator Insight
  • Next steps in AAV preclinical and translational R&D

    A Tretiakova,
    Anna Tretiakova PhD
    Senior Vice President of Product Development, Asklepios Biopharmaceutical (Ask Bio)
    Anna Tretiakova, PhD, is a Senior Vice President of Product Development, leading the company’s research and translational programs to deliver clinical candidate vectors. Anna is an industry veteran with more than 30 years of of research and development experience that spans basic research and non-clinical translational sciences, progressing from a bench scientist in academia to senior leadership roles with biotechnology and pharmaceutical organizations. She has spent over a decade exclusively focused on AAV gene therapy for monogenic and non-monogenic diseases in various therapeutic areas at the University of Pennsylvania Gene Therapy Program, Pfizer Rare Disease Research Unit, SwanBio Therapeutics, and, most recently, at AskBio. She completed her graduate education in molecular biology and biochemistry with a PhD from Thomas Jefferson University in Philadelphia and diploma from Novosibirsk State University in Russia.
    S Gordon
    Shari Gordon
    Senior Director of Immunology, Asklepios Biopharmaceutical (Ask Bio)
    1 July 2021
    Interview
  • Plasmid processing for mRNA, gene therapy and other vector applications

    H Ihre
    Henrik Ihre
    Director Strategic Techonlogies
    Henrik Ihre has his roots in biopharma, leadership and product development for the biopharma downstream industry in specific. He is motivated by bringing new solutions and manufacturing of new pharmaceuticals for patients developed by partners of Cytiva. He has been the Director of Strategic Technologies since March 2020 with specific knowledge and background in the downstream purification of biopharmaceuticals for over 20 years. 
    1 July 2021
    FastFacts
  • Progressing genetic medicines at the interface of gene therapy and gene editing

    A Seymour
    Dr Albert Seymour
    Chief Scientific Officer, Homology Medicines
    Albert Seymour, PhD, serves as the Chief Scientific Officer of Homology Medicines and has spent more than 20 years coupling the discipline of Human Genetics with Pharmaceutical R&D resulting in the delivery of multiple therapeutic programs into development. At Homology, Dr Seymour is responsible for translating the Company’s dual in vivo gene therapy and gene editing platform into treatments for rare genetic disorders. Through his leadership, Homology’s phenylketonuria (PKU) program advanced from scientific concept to the world’s first gene therapy clinical trial for PKU in just under 3 years, in addition to building a pipeline of genetic medicine programs in late stage preclinical development. Prior to Homology, he was the Senior Vice President and Global Head of Research and Nonclinical Development at Shire Pharmaceuticals where he led a team responsible for the delivery of a sustained flow of rare disease therapeutics from idea to IND and supported the full R&D portfolio in the areas of toxicology, bioanalytics, and Drug Metabolism and Pharmacokinetics. Prior to that role, Dr Seymour served as the VP and Head of Drug Discovery and Translational Research at Shire where he doubled the rare disease discovery portfolio in three years. Subsequent to joining Shire, he spent 14 years at Pfizer leading a team in the application of human genetics and computational biology to discover and develop therapeutics and pharmacogenomics strategies in diabetes, inflammatory diseases, and oncology. He is a member of the Board of Directors of Ensoma. Dr Seymour received his undergraduate degree in Biology from the University of Delaware and MS degree in Molecular Biology from Johns Hopkins University where he focused on the molecular genetics of cancer. He received his PhD and post-doctoral training in Human Genetics at the University of Pittsburgh.
    14 June 2021
    Interview