OVERVIEW, CURRENT LANDSCAPE & FUTURE TRENDS
- Setting the scene: comparison of rare disease/CGT and traditional pharma
- The importance of stakeholder engagement: perspectives across different functions
- Creating a CGT platform: stakeholder engagement as a tool to support not only the
development/commercialization of specific assets, but to build a sustainable platform
- Complex challenges and key considerations for cell & gene therapy development
- Pre-clinical solutions to address scientific, regulatory, and commercial risks to accelerate development
- How to maximize the potential and simplify the transition from basic cell therapy research to clinical manufacturing and beyond
- Current trends and innovations to advance cell & gene therapies
Wenliang Dong, Chief Operating Officer, ORCA Therapeutics B.V.
Alejandro Fernandez-Martell, Former Principal Bioprocess Engineer, Achilles Therapeutics
- Challenges and obstacles in bringing this highly complex therapies to market
- Stakeholder collaboration on the road towards a solution
- Lessons learned and applications for new entrants
- Strategies that can be taken to improve timely market access and access for patients
Doug Danison, Head of Commercial, Cell and Gene Therapy Unit, Bayer
- Standardized approaches being developed to address complexity of trial design and lack of
experience with CGT trials
- Considerations to overcome constraints in manufacturing capacity
- Strategies and best practices in facilitating patient recruitment and retention in CGT clinical trials
- T-Cell Receptors modified T-cell therapies (TCR-T) unlock immense tumour-selective target space
- Identifying the best targets and TCRs requires a systematic Precision Biology approach.
- Precision Biology platform that uses programmable human cells to systematically map the target space and engineer TCRs.
Reagan Jarvis, CEO and Co-founder, Anocca
Zhimei Du, VP, Process Sciences, Atara Biotherapeutics
- PDC*line is a new potent and scalable therapeutic cancer vaccines based on a proprietary allogeneic
cell line of Plasmacytoid Dendritic Cells
- PDC*line is much more potent to prime and boost antitumor antigen, including neoantigens, specific
cytotoxic T-cells than conventional vaccines and improves the response to checkpoint inhibitors
- The technology can be applied for any cancer
Eric Halioua, President & Chief Executive Officer, PDC*line Pharma
- Introduce the development of novel gene therapies
- Introduce pivotal non-clinical studies
- Discuss the relevant non-clinical considerations within the context of a case study of a novel gene
Mark Singh, Non-Clinical Manager, Cell and Gene Therapy Catapult
- Muscular dystrophies as paradigm for orphan drug development
- Most promising gene therapy strategies for the treatment of muscular dystrophy
- Future perspectives; orphan drugs in the pipeline
Alberto Malerba, Lecturer in Gene Therapy, Royal Holloway University of London
- Present innovative approaches for flexible Cell Therapy facility design solutions.
- Provide an overview of the latest Cell Therapy manufacturing technologies.
- Outline the benefits of implementing robust methods of process closure in the manufacturing
- Discuss sustainability factors associated with the design of modern manufacturing facilities.
- Outline how all these factors can increase speed to market while reducing the environmental footprint
of a facility.
Stephen Judd, Process SME – Biologics / Cell & Gene Therapy, DPS Group
- Why we need to consider the long-term when developing processes, even at phase I
- When is it appropriate to consider automation?
- Why automation early in the product development lifecycle matters.
Stuart M Curbishley, Chief Manufacturing and Development Officer, adthera
- Current GMP and general process validation concepts and practices that applies to CGT manufacturing
processes and analytical methods
- Key challenges in analytical method validation and proposed solutions.
- Extensive regulatory requirements for a controlled validation
- Robust CMC package that creates a path toward commercialization
- Vigorous process validation strategy through the life cycle of these novel therapies