Delivery Solutions, Improvements and Alternatives for Gene & Cell Therapy
Changing the Cell Therapy Landscape with In-Vivo Therapies: Is this a Viable Alternative for Point of Care Therapeutics?
Achieving Best Practice in Quality Control and Assurance
Implementing New and Enabling Technologies into Existing Processes for Improved Commercial Outcomes
Implementing New and Enabling Technologies into Existing Processes for Improved Commercial Outcomes
Analytics, Data and IT Tech Solutions: An Overview of Best Practices and Technology
Industry 4.0 Approaches Transforming Cell and Gene Therapy
Accelerating the Digitalisation Revolution in Cell and Gene Therapy Development
Viral Vector Development: Safe and Effective Viral Vectors for Therapeutic Success

Delivery Solutions, Improvements and Alternatives for Gene & Cell Therapy

27 Jan 2022
Mirus Bio

13:30 Chairperson’s Opening
Kevin Ramdas, Director, Medical Affairs, Longeveron

13:35 Mitigation of AAV Gene Therapy Immunogenicity

  • Immunogenicity of AAV vectors can affect efficacy and safety of gene therapy
  • We have developed ImmTOR technology to mitigate immunogenicity to enhance transgene expression, mitigate hepatic inflammation and enable vector re-dosing
  • We are also developing a proprietary IgG protease to enable patients with pre-existing anti-AAV antibodies to become eligible for gene therapy

Kei Kishimoto, Chief Scientific Officer, Selecta BioSciences

13:50 Improved Transfection Methodologies for AAV and LV Manufacture

  • Present optimized transfection and scaling strategies for viral vectors generation to increase physical and functional titers and enable seamless transitions from R&D through large-scale production
  • Review the complex interactions of reagent, vector design, culture media, and HEK 293 cell type and how these impact virus quality and yield
  • Summarize how TransIT-VirusGEN® GMP Reagent and Kits offer workflow flexibility, cost savings, and expand manufacturing capabilities for gene and cell therapies

Miguel Dominguez, Director of Sales and Support, Mirus Bio

14:05 RNA Cell Therapy in And Beyond Oncology

  • RNA based cell engineering offers predictable, controlled exposure and excellent safety profile taking engineered cell therapies into unique clinical conditions that would not tolerate the toxicity of conventional DNA-engineered cells
  • RNA-based approaches allow engineering multiple therapeutic proteins into a single product
  • Some examples of unique clinical applications utilizing RNA-based cell applications currently in Phase 1/2 development are frontline multiple myeloma Myasthenia Gravis and Adult Respiratory Distress Syndrome

Metin Kurtoglu, Chief GMP Manufacturing Officer, Cartesian Therapeutics

14:20 Driving Down Time and Cost of Cell R&D and Manufacturing with Next Generation Transfection Technology

  • A new version of modified, discrete carbon nanotubes called MGMR  available with minimal supply limits has been developed that has minimal toxicity to cells and tissues unlike other forms of carbon nanotubes
  • MGMR can easily penetrate cell membranes carrying  a large variety of biologically active entities such as DNA, RNA, peptides, proteins and small molecule drugs and can release the entities inside the cell
  • MGMR has minimal impact to cell vitality and growth and has the ability to allow measurement of cargo delivered into the cell

Kurt Swogger, Chief Executive Officer, Chief Technical Officer, BioPact

14:35 Speaker Q&A and Panel Discussion


Kevin Ramdas
Director, Medical Affairs
Kei Kishimoto
Chief Scientific Officer
Selecta Bio
Miguel Dominguez
Director of Sales and Support
Mirus Bio
Metin Kurtoglu
Chief GMP Manufacturing Officer
Cartesian Therapeutics