Cell & Gene Therapy With Focus On Viral Vectors - (KL-15)
Cell & Gene Therapy With Focus On Viral Vectors - (KL-15)
Cell & Gene Therapy With Focus On Viral Vectors - (KL-15)
Lars Brandén
LEARNING OUTCOMES:
www.glceurope.com
Cell & Gene Therapy MasterClass 16-18 October, 2023
- with focus on viral vectors
Online MasterClass
- EU edition
EVENT INTRODUCTION
This 3-day online course will detail the fundamental biological components for gene therapy
and gene-modified cell therapies and outline important consideration for selecting the most
appropriate vectors and specific safety aspects.
Critical biological information to enable understanding of the cell and gene therapy tools and
therapies both existing today and coming in the near future.
The course will also address key points in manufacturing, purification and different technologies
for each of the types of therapies addressed during the course.
The lack of standardization regarding modular single-use consumables will be discussed as well as
methods for cell separation and scaling-up issues.
Participants will also be given a set of case studies to analyse from the perspective of the course
and provide feedback to the course leader who will select a few for general forum viewing after
anonymizing the person providing the analysis.
• Professionals in biotech
• Pharmaceutical industry
• Medtech industry
• Science reporters
• VC advisors in the life sciences
• Think-tank employees focusing on life sciences
• Senior decision makers that needs a deeper understanding of the field of cell- and gene
therapies to guide their companies into the future with a better understanding of the
underlying technologies driving much of the life science industry
Dr. Lars J. Brandén, Linnaeus University, Sweden, Innovation Advisor and independent consultant
for pharma, biotech and medtech industry with global experience for the last 10 years and with
over 15 years experience in academic research.
Accomplished leader in Biotech R&D both in academia and industry with a solid background in
cell- and gene therapy as well as in Bioinformatics, HTS, phenotypic screening and cell-based assay
development. Successfully built and operated two cutting edge High Throughput Cell Biology
centres from ground and up with industry-scale capacity.
After receiving his PhD from the Karolinska Institute, Sweden Dr. Brandén was recruited to three
senior director positions at Columbia University and Yale university and also as post-doctoral research
fellow at Memorial Sloan-Kettering Cancer Center, MSKCC. Dr. Brandén has also worked as the senior
director for Bioinformatics for an UK clinical company as well for several global pharmaceutical,
biotech and medtech companies.
• Virology
• Immunology
• Cell signalling
• Gene and Cell Therapy
• Bioinformatics
• Systems Biology
• Drug discovery process
• Laboratory automation
DAY1 DAY2
08:55 Connecting to the Online MasterClass 08:55 Connecting to the Online MasterClass
09:00 Session 1: 09:00 Session 4:
Understanding underlying Choosing the most appropriate
virology and cell biology vector for your application
Explain the wide variety of virus types and how • Importance of deep knowledge of the
this can be utilized to choose the best virus for the disease to be able to design the best
application and also the ability to use the native therapy ie choose the right vector
targeting ability for some virus types. Describe the system and genetic control systems for
development of the different GT viruses that has the therapeutic genetic elements.
been and are used today. What virus is good for • Explain the difference in ex vivo and in
some and not for other applications with details on vivo GT and the design of the different
the genetics and infectious cycle of example virus approaches plus discussing the use of
types to illustrate the reasons. The following will naked nucleic acid therapies v/s vector
be considered: based therapies.
• The difference in how VV (Viral Vectors)
• The ability of viruses to integrate with are used in gene-modified cell therapy
the host genome and insert viral DNA and gene therapy – GMCT – vector is the
• Engineering of viruses so they become raw material whereas in gene therapy
integrative but non-replicative in order the vector is the final product.
to ensure patient safety
• What are the viruses we use for CGT
applications: some strand of the HIV
virus which has been modified over
10:30 Coffee Break
time, lentivirus, adenovirus etc.
• CAR-T cell therapy 10:45 Session 5:
Choosing the most appropriate
10:30 Coffee Break viral vector for your application
Problems with large genes and also how the
10:45 Session 2: distant enhancers can cause issues in vector
Viral Vector manufacturing and design. The target cells might have a low level
therapeutic cell manufacturing of available receptors for the infection and
principles how this can affect the design of the therapy
and overall design.
• Elimination of bacterial DNA sequences from
plasmids and how different DNA sequences Brainstorm common factors
can cause immune reactions and cell death
plus other considerations of plasmid design. which influence VV selection:
Considerations of scaling up from the lab
to the clinical scale plus media composition • Size of the gene which
and cell line considerations due to potential needs to be transduced
contaminations causing immune reactions • Transduction efficiency
when injected into patient. • Serotype efficiency
• Design of production facilities with
positive pressure and strict protocols for
manufacturing etc 12:15 Coffee Break
• Plasmid design
• Growth in HEK293 Cells and expansion 12:30 Session 6:
to reach desired titration, 50l, 200l Group Exercise and feedback
• Industrialization of cell therapies
• Understanding the different
technologies available and when Groups will consider important factors and
to use what. design a viral vector for their cell and gene
• Facility requirements – operating in therapy application and feedback their
closed systems saves time and money thoughts to the group.
as you can operate in a grade C/D
facility
13:30 END
End of Day2
12:15 Coffee Break
12:30 Session 3:
Group Exercise
This will be the start of a rolling case study.
Delegates in groups will be presented with a
scenario where they develop a CGT application
which needs viral vectors for delivery or
engineering. Delegates will consolidate the
previous two sessions and consider key steps for
engineering a viral vector for their application.
DAY3
10:45 Session 8:
Purification Techniques
Discuss what techniques to use at what stage of
production and why they should be used. Also
discuss what can be seen and what cannot be
seen and how this can affect the overall design
of a therapy. Discuss the different ways to titre
viruses and how this can be relating to the specific
characteristics of the virus itself. Discuss inducible
as well as SIV approach and the issues related to
each methodology.
• ELISA
• q PCR
• HPLC
• TCID50
• Replication competent
lentivirus detection
• QC of cell therapeutic products
12:30 Session 9:
Group Exercise Session
In groups, delegates will consolidate the previous
talks and brainstorm the purification requirements
and techniques they need to use for their particular
viral vector or gene therapy methodology rolling
on from the previous group sessions.
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Participation fee includes: Full access to the live virtual training | Course material | Digital certificate