Cambridge Healthtech Institute’s Inaugural
Advances in Vector Production and Scale-Up for Cell and Gene Therapy
Enabling Vector Design, Expression, Development To Meet Commercial Scale Production Demands
January 15-16, 2019
Two autologous CAR T therapies are now on the market, but how will companies manufacture these product at the commercial scale? What technologies and production processes are needed to meet the commercial scale demand? Cambridge Healthtech Institute’s
Inaugural Advances in Vector Production and Scale-Up for Cell and Gene Therapy conference will bring together leading scientists from biopharmaceutical industry, academia and government to discuss and showcase innovation in design and engineering
of vectors and strategies to overcome production challenges for cell and gene therapy products. Through new presentations, informative panel discussions, high-level poster presentations, and interactive discussions, top scientists will share new insights
into various expression systems and production aspects for AAA, Lentivirus, Adenovirus as well as non-viral vector carriers such as nanoparticles, electroporation systems and novel biomaterials.
Final Agenda
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TUESDAY, JANUARY 15
1:00 pm Registration (Sapphire West Foyer)
1:30 Refreshment Break in the Exhibit Hall with Poster Viewing (Sapphire Ballroom)
2:00 Chairperson’s Opening Remarks
Sandro Matosevic, PhD, Assistant Professor, Department of Industrial and Physical Pharmacy, Purdue University
KEYNOTE PRESENTATION
2:05 Current Trends and Challenges of Gene Therapy Manufacturing
Palani Palaniappan, PhD, Head, Tech Ops & Andover Site, Sarepta Therapeutics
The keynote will review current status of gene therapy CMC and manufacturing with a view towards future and share Sarepta’s vision in this area. Progress in manufacturing area to align with clinical progression of the pipeline will be highlighted.
2:45 Strategies and Advances in Lentiviral Vector Manufacturing and Scale-Up
Cindy Jung, PhD, Scientific Leader, Vector Process Development, Cell & Gene Therapy Platform CMC, Platform Technology & Science, GSK
In this presentation, we will discuss strategies and advances in lentiviral vector manufacturing and scale-up such as transient vs. stable cell line approaches, development and optimization of upstream and downstream scalable unit operations, improving
process robustness and cost of goods.
3:15 NEW: Addressing Large-Manufacturing of Clinical Grade Viral Vectors Using an Optimized PEI-Based Transfection Process
Géraldine Guérin-Peyrou, Director of Scientific & Technical Support, Polyplus-transfection, France
We describe an optimized PEI-based virus production process for high-yielding viral vector production, compatible with different cell culture adherent and suspension systems. We further demonstrate the robust viral vector production yields, as well
as the adaptability and reliability of the PEI-based transient gene expression approach to efficiently manufacture GMP-grade viral vectors at a sufficiently large scale for more advanced clinical trials, and in fine to drive commercialization
of therapeutic vectors.
3:45 Refreshment Break in the Exhibit Hall with Poster Viewing (Sapphire Ballroom)
4:30 Optimizing Sf9-Based Stable Cell Lines for the Production of Highly Infectious rAAV Vectors
Sergei Zolotukhin, PhD, Professor, Department of Pediatrics,
College of Medicine, University of Florida
We describe a new insect cell-based production platform utilizing attenuated Kozak sequence and a leaky ribosome scanning to achieve a serotype-specific modulation of AAV capsid proteins stoichiometry. By way of example, rAAV5 and rAAV9 were produced
and comprehensively characterized side by side with HEK293-derived vectors. The data will be presented demonstrating a superior infectivity and higher genetic identity of OneBac-derived rAAV vectors providing a scalable platform for good manufacturing
practice (GMP)-grade vector production.
5:00 LVV Production Process: Recent Advances and Key Challenges
Yogesh Waghmare, PhD, Associate Director, Vector Downstream Process
Development, Bluebird Bio
LentiViral Vector (LVV)-based Cell and Gene Therapy products are steadily increasing in number. Industrial production of LVV poses significant challenges compared to AAV due to the large size, complexity, and labile nature of LVV. An overview of industrial
LVV production process evolution, recent technological advances, and LVV specific challenges will be presented.
5:30 Close of Day
5:30 - 5:45 Short Course Registration (Sapphire Ballroom)
5:45 - 8:45 Recommended Dinner Short Courses*
SC5: Transient Protein Production in Mammalian Cells - Detailed Agenda
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*Separate registration required
Day 1 | Day 2 | Download Brochure
WEDNESDAY, JANUARY 16
7:45 am Registration and Morning Coffee (Sapphire West Foyer)
8:15 Chairperson’s Remarks
Junghae Suh, PhD, Associate Professor, Bioengineering, Rice University
8:20 Synthetic Virology Approaches to Designing AAV Vectors
Junghae Suh, PhD, Associate Professor, Bioengineering, Rice University
Adeno-associated virus (AAV)-based gene delivery vectors are some of the most promising in the gene therapy field today. To make viral gene delivery a more predictable process, we must obtain control over the naturally encoded biomolecular programs
already embedded in the AAV capsids. I will discuss my lab’s work on rewriting the details of what cues can be accepted as input and what functional outputs can be produced by AAV.
8:50 Vector Development and Large-Scale Manufacturing
Jacek Lubelski, PhD, Vice President, Global Pharmaceutical Development, uniQure
Scaling up of rAAV manufacturing process displays various challenges, one of which is the variability introduced by starting and raw materials. I will discuss our effort to limit the sensitivity of uniQure’s rAAV production system
to fluctuation in input materials. Furthermore, to exploit the BEVS potential to support large scale rAAV manufacturing I will present our experience with baculoviruses/insect cell system in stirred tank bioreactor and our efforts to make
stronger and more specific promoters. Finally, I will discuss the vector quality and potency generated by insect and mammalian cell production systems.
9:20 Bioprocessing of Adenovirus – Technical and Economic Considerations
Mats Lundgren, Customer Applications Director, GE Healthcare Life Sciences
Vaccines based on viruses and viral vectors are becoming increasingly important for prevention and the treatment of many diseases. This presentation is focused on manufacturing of Adenovirus (AdV) and the development of an efficient and scalable
process for AdV production by evaluation of each process step. Based on analytical data different downstream process alternatives were compared regarding load capacity, recovery and purity and we propose a robust and scalable process with
a favorable process economy.
9:50 Coffee Break in the Exhibit Hall with Poster Viewing (Sapphire Ballroom)
10:35 Considerations for the Use of Analytical Ultracentrifugation for Characterization and QC Testing of AAV Gene Delivery Vectors
Christopher Sucato, Senior Scientist, Biophysical Characterization, Charles River Laboratories
Analytical Ultracentrifugation (AUC) in the biologics has traditionally been employed in the analysis of aggregation and higher order structure, where monomeric protein is commonly the analyte. More recently, the rise of gene delivery vectors
to treat pathologies has opened avenues for AUC-based methodologies. Here we explore the parameters of an AUC method which conform to ICH/cGMP validation, and which may remain challenging due to issues inherent with current AUC instrumentation.
11:05 Challenges in Viral Vector Manufacture and Analytics
Tristan Thwaites, PhD, Lead Technical Scientist, Industrialization, Cell & Gene Therapy Catapult
The number of viral gene products entering early and late phase clinical trials is significantly on the rise. To meet demand, there is a need to move into scalable and controllable production and purification systems. This presentation will
focus on the work at Cell & Gene Therapy Catapult to develop rapid, high-throughput analytical systems to accelerate understanding of the critical process parameters.
11:35 PANEL DISCUSSION: Challenges and Opportunities in Viral and Non- Viral Vector Development and Production
- New vectors
- Closing the production gap
- New production technologies
- Vector characterization
Moderator:
Sandro Matosevic, PhD, Assistant Professor, Department of Industrial and Physical Pharmacy, Purdue University
Panelists:
Cindy Jung, PhD, Scientific Leader, Vector Process Development, Cell & Gene Therapy Platform CMC, Platform Technology & Science, GSK
Palani Palaniappan, PhD, Head, Tech Ops & Andover Site, Sarepta Therapeutics
Jacek Lubelski, PhD, Vice President, Global Pharmaceutical Development, uniQure
Christopher Sucato, Senior Scientist, Biophysical Characterization, Charles River Laboratories
12:05 pm Session Break
12:15 Luncheon Presentation (Sponsorship Opportunity Available) or Enjoy Lunch on Your Own
1:15 Session Break
PLENARY KEYNOTE PANEL (Aqua Salon)
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PepTalk Perspectives: Point-Counterpoint Discussions
2:00 Plenary Keynote Introduction
Norman Packard, PhD, CEO, Daptics
2:10 Plenary Keynote Panel
Moderator:
Howard Levine, PhD, President and CEO, BioProcess Technology Consultants
Panelists:
George Badescu, PhD, Vice President, Scientific Affairs, Heidelberg Pharma AG
Manon Cox, PhD, Co-Founder & CEO, NextWaveBio
Zhimei Du, PhD, Director, Bioprocess & Clinical Manufacturing, Merck
Paul Jorjorian, Vice President, BioProcess Sciences, Thermo Fisher Scientific
Marina Kirkitadze, PhD, Deputy Director, Head of Biophysics and Conformation Unit, Analytical R&D Biochemistry, Sanofi Pasteur, Canada
Stefan R. Schmidt, PhD, MBA, Head, Operations (COO), BioAtrium AG
3:05 Refreshment Break in the Exhibit Hall with Poster Viewing (Sapphire Ballroom)
4:00 Chairperson’s Remarks
Yogesh Waghmare, PhD, Associate Director, Vector Downstream Process Development, Bluebird Bio
4:05 Non-Viral Immunometabolic Reprogramming of Natural Killer Cells for Immunotherapies of Solid Tumors
Sandro Matosevic, PhD, Assistant Professor, Department of Industrial and Physical Pharmacy,
Purdue University
The anti-tumor immunity of natural killer (NK) cells is highly impaired due to immunometabolic suppression in the microenvironment of solid tumors. For that reason, reprogramming these cells is a therapeutic necessity to enhance their effector
function. Here, we discuss the genetic reprogramming of NK cells using non-viral approaches, including recent work focused on imparting new functionality upon NK cells targeting immunometabolism and immune evasion by cancer cells.
4:35 Strategies to Optimize Lentiviral and Retroviral Transduction of NK and T Cells for Adoptive Immunotherapy
Evren Alici, MD, PhD, Assistant Professor of Hematology, Karolinska Institutet, Department of Medicine,
Stockholm, Sweden
In order to manufacture more efficient NK cell therapy products, it is essential to develop novel strategies such as genetic modification of NK cells. The introduction of either activating or chimeric antigen receptors customized for NK
cells presents an attractive prospect for further clinical applications. Although NK cells are inherently resistant to retroviral and lentiviral transductions, recently, our group has significantly enhanced retroviral and lentiviral
gene delivery to NK cells through enhanced proliferation and targeting intracellular viral defense mechanism by small molecule inhibitors.
5:05 Breakout Discussions
Join the moderated discussions to share ideas, gain insights, establish collaborations, or commiserate about persistent challenges. Then continue the discussion as you head into the lively Exhibit Hall.
Topic: Different Flavors Of Vectors And What Does It Mean?
Moderator: Yogesh Waghmare, PhD, Associate Director, Vector Downstream Process Development, Bluebird Bio
- For Gene Therapy product vector is the product and for Cell therapy products, vector is starting material. How does this difference practically translate into:
- Regulatory strategy
- Target quality profile
- Process development goals etc.
Topic: Cell-Based Immunotherapies for Solid Tumors: Are We There Yet?
Moderator: Sandro Matosevic, PhD, Assistant Professor, Department of Industrial and Physical Pharmacy, Purdue University
- Tumor microenvironment immunosuppression: not like hematological malignancies
- Cell engineering for tumor homing and persistence
- Vector design considerations to enhance immune cell cytotoxicity
Topic: Gene Therapy Validation and Commercialization
Moderator: Tristan Thwaites, PhD, Lead Technical Scientist, Industrialization, Cell & Gene Therapy Catapult
- Quality by Design
- Gene therapy analytics and characterization
- Process qualification and validation
6:05 - 7:00 Networking Reception in the Exhibit Hall with Poster Viewing (Sapphire Ballroom)
7:00 Close of Advances in Vector Production and Scale-Up for Cell and Gene Therapy Conference
Day 1 | Day 2 | Download Brochure