Northern California Regional Forum

Transcription

1 Northern California Regional Forum Cell and Gene Therapies South San Francisco Conference Center South San Francisco, CA Thursday, November 8, 2018 Co-chairs: Nomalie Jaya, Seattle Genetics, Inc. David Passmore, RubrYc Therapeutics, Inc.

2 Acknowledgements A special thanks to the program committee who helped develop this program! Program Committee Kris Antonsen, BioMarin Pharmaceutical, Inc. Greg Cantin, Five Prime Therapeutics, Inc. Jenny Chen, Nektar Therapeutics Judy Chou, Bayer Kathy Francissen, Genentech, a Member of the Roche Group Michelle Frazier, Coherus Biosciences Malou Gemeniano, Audentes Therapeutics, Inc. Nomalie Jaya, Seattle Genetics, Inc. Guifeng Jiang, Boehringer Ingelheim Vinaya Kapoor, Tricida, Inc. Robert McCombie, Sangamo Therapeutics, Inc. David Passmore, RubrYc Therapeutics, Inc. Joanne Severs, Bayer Bryan Silvey, Kite, a Gilead company Shelley Suggett, Halozyme Therapeutics Trevor Swartz, Genentech, a Member of the Roche Group Lance Wong, Strand Bio Min Young Christopher Yu, Genentech, a Member of the Roche Group Eike Zimmermann, Boehringer Ingelheim CASSS Staff Noelle Atkins, Administrative Coordinator Karen A. Bertani, CMP, Director, Global Engagement and Knowledge Sharing Stephanie L. Flores, CAE, Executive Director Julie Fowle, Program Planning and Event Specialist Anna Lingel, CMP, Exhibitor Relations and Technology Specialist Renee Olson, Senior Program Manager Catherine Stewart, Finance Manager Audio Visual Michael Johnstone, MJ Audio-Visual Productions 2

3 Scientific Program Summary Thursday, November 8, :00 09:00 Registration and Continental Breakfast in the South Lobby 09:00 09:15 CASSS Welcome and Introductory Comments David Passmore, RubrYc Therapeutics, Inc., San Ramon, CA 09:15 09:45 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 09:45 10:15 Process Considerations for Cryogenic Shipping Craig Vermeyen, Kite, a Gilead company, Santa Monica, CA USA 10:15 10:45 Networking Break 10:45 11:15 Unique Considerations for AAV Gene Therapy Manufacturing Khandan Baradaran, Ultragenyx Pharmaceutical, Novato, CA USA 11:15 11:45 Challenges and Opportunities in AAV Manufacturing for Gene Therapy Nicole Paulk, University of California, San Francisco, San Francisco, CA USA 11:45 13:00 Lunch in Salon G-J 12:00 13:00 Career Development Roundtable in Salon G-J 13:00 14:30 Workshop: Opportunities and Challenges Encountered when Developing and Marketing Cell and Gene Therapy Products Facilitator: Bryan Silvey, Kite, a Gilead Company, Santa Monica, CA USA 14:30 14:45 Closing Remarks Bryan Silvey, Kite, a Gilead Company, Santa Monica, CA USA 14:45 15:15 Networking Reception 3

4 Program Abstract Cell and Gene Therapies 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. NOTES: 4

5 Speaker Abstracts Unique Considerations for AAV Gene Therapy Manufacturing Jeffrey Teoh, Juno Therapeutics, a Celgene Company, Seattle, WA USA 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 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. NOTES: 5

6 Speaker Abstracts (continued) Unique Considerations for AAV Gene Therapy Manufacturing Khandan Baradaran, Ultragenyx Pharmaceutical, Novato, CA USA 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 Different manufacturing and purification approaches have been used in the production of recombinant AAVs for gene therapy, namely baculovirus-infected insect and transientlytransfected 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. NOTES: 6

7 Workshop: Discussion of Opportunities and Challenges Encountered when Developing and Marketing Cell & Gene Therapy Products FACILITATOR: Bryan Silvey, Kite, a Gilead Company, Santa Monica, CA USA SCOPE: 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. DISCUSSION QUESTIONS: 1. What should emerging companies pay most attention to when they consider industrializing a (largely research) process for either modality (Cellular Therapy/Gene Therapy) 2. When considering a Global Launch for genetically modified cellular therapy, what efforts should a company take in an attempt to harmonize global regulatory assessments? 3. 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? 4. What are the hurdles for off the shelf CAR-T type therapies? 5. What should one consider when developing a control strategy? Where should dialogue with the regulators go? 6. How do we approach management/oversight of the cell collection facility (complexity, variability, patient privacy etc.)? 7. What are the challenges of working with multiple CMOs for small companies to secure adequate amount of gene therapy vectors 8. What are the special CMC considerations for gene therapy for orphan product development? NOTES: 7

8 Career Development Luncheon Roundtable A table will be set aside during the networking lunch to discuss career development. There will be a total of 10 seats available for this discussion. Seating will be on a first come, first serve basis. The discussion points are listed below. FACILITATOR: Rob McCombie, Sangamo Therapeutics, Inc. SCOPE: We will be discussing career development within the Biotech and Pharma Industries. We will explore myths regarding the number of years one must acquire in order to attain a desired position in a different area or discipline of the industry. We will also discuss transferable skills, management experience and strategic skills sets. DISCUSSION POINTS: 1. Is it important to have a degree in the field that you are pursuing? a. For example, if the goal is to be a Director of Analytics, does one need a degree in chemistry or biology? Does one require a PhD? b. Is this the case for all careers? For example, does this hold true for Regulatory affairs or project management? 2. How does one move from technical jobs to regulatory affairs, project management, quality assurance, and vice versa etc.? a. What are transferable skills in the industry? b. How can you start thinking more cross-functionally? c. How can you think more strategically? 3. Is it better to have a more diverse background or focus on one field for many years to be successful? a. How can one gain more experience within one discipline? 4. How to transfer roles in Analytical Discovery and Analytical Development departments? Skills in common, and skills unique to each function. 5. Transitioning from academic to industry tips on getting that 1 st job in Biopharma. 8

9 The program committee gratefully acknowledges the following program partners and exhibitors for their generous support of the CASSS Northern California Regional Forum Strategic Diamond Program Partner Genentech, a Member of the Roche Group Program Partners BioMarin Pharmaceutical Inc. Boehringer Ingelheim International GmbH Exhibitor Partners Thermo Fisher Scientific Wyatt Technology Corporation 9

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