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CASSS NorCal Regional Forum Fall 2018: Scientific Program
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 To view the final program, click here.


Cell and Gene Therapies
Co-Chairs:  Nomalie Jaya, Seattle Genetics, Inc. and David Passmore, RubrYc Therapeutics Inc.
The CASSS Northern California Regional Forum is committed to bringing job-relevant education and regulatory trends for biopharmaceuticals to CASSS members through meeting programs. The November forum will focus on cell and gene therapies. The discussion will include content from other regional, national or international forums organized by CASSS. The program will also provide a platform for networking with both peers and regulatory authorities.

Cell and gene therapy products are providing novel solutions to treating life threatening diseases. However, these therapies also pose unique challenges to manufacturing, analytical characterization and establishing appropriate controls while providing opportunities for drug developers to “think outside the box” at innovative solutions to traditional approaches. Further, given the relative novelty of cell and gene therapies the regulatory landscape is mostly undefined. This forum on cell and gene therapies will provide practical discussion on CMC considerations, challenges and opportunities as more cell & gene therapies gain regulatory approval.


Confirmed Speakers

Unique Considerations for AAV Gene Therapy Manufacturing
Khandan Baradaran, Ultragenyx Pharmaceutical, Novato, CA USA
Click here to view the slides from this presentation.
AAV Gene Therapy manufacturing technology platforms are in development and consist of a wide array of strategies, including transient transfection, producer cell lines and packaging cell lines. Each has its own unique challenges – selecting the appropriate CMO, procurement of the necessary critical starting materials, scale and design of the downstream process, and the appropriate analytical tools to ensure consistently high quality product. Technology is evolving to keep up with clinical demand and dynamic clinical development programs in real time. Analytical tools to characterize the product and to develop robust processes are being developed concurrently. We will discuss these challenges in light of the opportunities that this new technology brings to address unmet medical need.
Challenges and Opportunities in AAV Manufacturing for Gene Therapy
Nicole Paulk, University of California, San Francisco, San Francisco, CA USA
Presentation slides are unavailable.
Different manufacturing and purification approaches have been used in the production of recombinant AAVs for gene therapy, namely baculovirus-infected insect and transiently-transfected human cell systems. Work from my lab has shown that each method produces vector lots exhibiting chemical, structural and functional differences in vitro and in vivo. These differences have implications for capsid folding, viral replication, receptor binding, intracellular trafficking, expression kinetics, stability, half-life regulation, and immunogenicity. Given these differences, we’re working actively to engineer novel solutions to overcome existing design and manufacturing challenges and develop quality control metrics. I will discuss our work and that of the broader field, as well as highlight opportunities for innovation that address key unresolved areas in the field of AAV gene therapy.
Navigating Unique Analytical Challenges in the Development of Autologous Chimeric Antigen Receptor T-cell Therapies
Jeffrey Teoh, Juno Therapeutics, a Celgene Company, Seattle, WA USA
Presentation slides are unavailable.

Autologous chimeric antigen receptor (CAR) T-cell therapy has demonstrated significant clinical benefit in hematological cancers. Patient heterogeneity introduces unique manufacturing process and analytical challenges in the CAR T cell therapy development space. Development of robust analytical methods is essential to the identification of and monitoring critical quality attributes (CQA) and understanding of manufacturing process control and product consistency. Moreover, with multifactorial mechanisms of action, a variety of cell-based methods and technologies are used to examine the breadth of functional activity exhibited by CAR T cells. This presentation will focus on analytical strategies used to characterize CAR T-cell products and to identify critical product quality attributes to associated with safety and efficacy of the final drug product, as well as understand manufacturing process controls. Hypothesis driven and hypothesis generating approaches will be discussed in the context of refining analytical strategies.

Process Considerations for Cryogenic Shipping
Craig Vermeyen, Kite, a Gilead company, Santa Monica, CA USA
Click here to view the slides from this presentation.

The rapid growth of the cell and gene therapy industry has led to a recent and significant increase in the use of cryogenic shippers for both clinical and commercial pharmaceutical products. Keeping products below -150ᵒC during shipping has clear benefits to product efficacy, however, the use of cryogenic shippers poses many novel supply chain challenges. Shippers of cryogenic cell and gene therapies must safely manage the filling and refilling of LN2 in cryogenic shippers, keep primary container materials from breaking, and work closely with partners along each step of the supply chain (packaging providers, logistics providers, hospitals, etc.) to reliably maintain temperature below -150ᵒC.


Workshop Session

Discussion of Opportunities and Challenges Encountered when Developing and Marketing Cell & Gene Therapy Products

Facilitator: Karen Walker, Seattle Genetics, Inc., Bothell, WA USA

This panel based Q &A session will focus on a broad range of Cell and Gene Therapy topics including but not limited to the questions listed below. The intent of the afternoon session is to foster an environment of learning and sharing based on an informal discussion. Audience participation is expected and  encouraged with additional questions that individual companies are tackling on the topics of Cell and Gene Therapies. 

The following questions will be discussed as well as any questions from attendees:

• What should emerging companies pay most attention to when they consider “industrializing” a (largely research) process for either modality (Cellular Therapy/Gene Therapy)
• When considering a “Global Launch” for genetically modified cellular therapy, what efforts should a company take in an attempt to “harmonize” global regulatory assessments?
• What does the future of Cell and Gene therapies look like in 10 years? What are the biggest hurdles to overcome to realize this future?
• What are the hurdles for “off the shelf” CAR-T type therapies?
• What should one consider when developing a control strategy? Where should dialogue with the regulators go?
• How do we approach management/oversight of the cell collection facility (complexity, variability, patient privacy etc.)
• What are the challenges of working with multiple CMOs for small companies to secure adequate amount of gene therapy vectors
• What are the special CMC considerations for gene therapy for orphan product development?

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