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Cambridge Healthtech Institute 第1回

Advances in Vector Production and Scale-Up for Cell and Gene Therapy
( 細胞療法と遺伝子療法に対応するベクターの製造とスケールアップ技術の進歩 )

2019年1月15日~16日

 

細胞療法と遺伝子療法に対応するベクターの製造とスケールアップ技術の進歩をテーマにしたこのカンファレンスプログラムでは、生物医薬品関連の企業、研究機関、政府機関の主要な研究者が一堂に会し、ベクターの設計と開発、および細胞療法と遺伝子療法に対応する製品の製造面の課題克服に向けた戦略について議論し、新機軸を披露します。

Final Agenda

1月15日(火)

1:00 pm Registration

1:30 Refreshment Break in the Exhibit Hall with Poster Viewing

トレンド、課題、可能性

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

In this presentation, I will discuss the current status of gene therapy manufacturing to support all stages of clinical development and commercialization.

2:45 Strategies and Advances in Lentiviral Vector Manufacturing and Scale-Up

Bo Kara, Head, Process Development, Cell & Gene Therapy Platform CMC, 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 Sponsored Presentation (Opportunity Available)

3:45 Refreshment Break in the Exhibit Hall with Poster Viewing

生産工程での技術革新の可能性

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 Opportunities for Innovation

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


5:45 - 8:45 Recommended Dinner Short Courses*

SC5: Transient Protein Production in Mammalian Cells

Click here for more details.

*Separate registration required

1月16日(水)

7:45 am Registration and Morning Coffee

大規模な生産に向けたベクターの設計、開発、特性評価

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

9:20 Sponsored Presentation (Opportunity Available)

9:50 Coffee Break in the Exhibit Hall with Poster Viewing

10:35 Scaling Adherent Manufacturing Systems for the Production of AAV Vectors

John Huynh, PhD, Senior Director, Manufacturing Science & Technology, Gene Therapy Program, University of Pennsylvania

Adherent cell culture is traditionally thought of as non-scalable; however, recent advances in fixed-bed bioreactor technology may address current challenges with scaling adherent production systems. Here, we describe the development of an AAV production process using the iCELLis bioreactor.

11:05 Analytical Development and Challenges to Characterize AAV Vector

Christine LeBec, PhD, Head of Analytical Development, Genethon

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:

Bo Kara, Head Process Development, Cell & Gene Therapy Platform CMC, GSK

Palani Palaniappan, PhD, Head, Tech Ops & Andover Site, Sarepta Therapeutics

Jacek Lubelski, PhD, Vice President, Global Pharmaceutical Development, uniQure

John Huynh, PhD, Senior Director, Manufacturing Science & Technology, Gene Therapy Program, University of Pennsylvania

12:05 pm Session Break

12:15 Luncheon Presentation (Sponsorship Opportunity Available) or Enjoy Lunch on Your Own

1:15 Session Break


2:00 PLENARY KEYNOTE PANEL

Click here for more details.

PepTalk Perspectives: Point-Counterpoint Discussions

Moderator:
Howard Levine, PhD, President and CEO, BioProcess Technology Consultants






Panelists:
Zhimei Du, PhD, Director, Bioprocess & Clinical Manufacturing, Merck






Lorenz Mayr, PhD, CTO, GE Healthcare Life Sciences




3:05 Refreshment Break in the Exhibit Hall with Poster Viewing

送達と治療固有の課題

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 Scalable Production of rAAV Vector for Gene Therapy Applications

Pranav Joshi, M. Tech, PhD Candidate, Bioengineering, McGill University

Adeno-Associated Virus (AAV)-based recombinant vectors are conclusively the most successful class of vectors for in vivo somatic cell gene delivery. Despite numerous advancements in production protocols, production of AAV to meet exceptionally high demand (1016-1017 VGs) in late clinical stages and eventually systemic delivery poses critical challenges. The insect-cell baculovirus system, a well-established platform for scalable production of vaccines and recombinant protein, is emerging for scalable manufacturing of clinical grade rAAVs.

5:35 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.

6:05 - 7:00 Networking Reception in the Exhibit Hall with Poster Viewing

7:00 Close of Advances in Vector Production and Scale-Up for Cell and Gene Therapy Conference

* 不測の事態により、事前の予告なしにプログラムが変更される場合があります。

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