7:50 am Chair’s Opening Remarks
Pioneering the Next Generation of Gene Therapies
8:00 am Keynote: FDA’s Efforts to Enhance Gene Therapy Regulatory Interactions
Synopsis
- Sharing FDA considerations regarding product manufacturing, including process validation
- Understanding how regulatory oversight will need to adapt to keep pace with technical innovations in gene therapy manufacturing and development
- Introducing the CATT meeting – an additional early opportunity to engage with regulators around key issues encountered in the broader development of gene therapies
- Distinguishing how CATT meetings are different from INTERACT meetings
8:30 am Building an Industry-Leading Therapy Portfolio in Rare Disease
Synopsis
- Working strategy and future commitment to gene therapy of rare diseases in Pfizer
- Role of external partnerships and collaborations for a sustainable gene therapy pipeline
- Infrastructure and supporting elements to support timely and broad access to emerging gene medicines
9:00 am Industry Leaders Panel: Delivering on the Promise of Gene Therapies
Synopsis
Gene therapies continue to redefine the treatment of rare diseases, but these conditions present many distinct development challenges. This panel discussion will bring together experts from leading organizations in this field to analyse how we can overcome these hurdles to realize the potential of gene therapies
- Navigating uncharted territory: sharing the decisions industry pioneers have made to pioneer progress without a defined pathway
- Changing the mind-set: understanding how the unique characteristics of gene therapies are in the process of changing the healthcare paradigm and how companies have to adapt to this new landscape
- Gearing up for commercialization: sharing how manufacturers, regulators and the wider field are preparing for a new wave of gene therapies in 2020 and beyond
9:30 am Speaking Position Reserved for Lonza
10:00 am Morning Refreshments & Speed Networking
Synopsis
Grab a quick cup of tea or coffee from the comfort of your own kitchen and jump straight into your opportunity to connect with new contacts from active companies in the field and exchange digital business cards. Network and form lasting connections through this exclusive virtual speed networking!
CMC & ANALYTICAL
Establishing Analytical Assays to Assess Critical Quality Attributes
MANUFACTURING SCALE-UP
Making Informed Strategic Manufacturing Decisions and Designing Effective Facilities
CLINICAL
Exploring the Continually Evolving Regulatory Landscape
COMMERCIAL
Understanding the Payer Perspective
11:00am Considerations Towards Technical Development of Viral Vector-Based Gene Therapies
• Technical Development for viral vector-based gene therapies: which approaches and elements could be and which ones shouldn’t be transferred from MABs
• Development of stable cell lines toolkit to put industrialization of viral vector-based gene therapies to another level
• Tackling the Bioassay challenge: applying a phase appropriate approach, surrogate strategies, and the matrix concept
Markus Haindl, Lead Gene Therapy Technical Development, Roche
11:00am Standardizing Gene Therapy Manufacturing Facilities
• Establishing standards andimplementing these in internal and external facilities
• Prioritizing standards and communicating this effectively to partners
• Understanding the knock-on effects of differing quality standard on overall program development
Speaker to be confirmed
11:00am A Strategic Playbook for Key Rare Disease Gene Therapy Regulatory Milestones
• Examine evolution of type and standard of data required by regulatory authorities and key precedent products
• Highlighting the success criteria at key milestones throughout development
• Discussing strategic trial designs for rare and ultra-rare disorders in the context of outcomes and endpoints
Angela Johnson, Senior Director, Regulatory Affairs, Sigilon Therapeutics
11:00am Shifting Payment Paradigms: How One-Time High Cost Treatments Challenge the Traditional U.S. Payer Model
• Gaining insights into the unique challenges gene therapies pose to the classical payer model
• Providing a framework for how payers need to consider the balance between access and affordability in the context of innovative but expensive therapeutics
• Understanding the traditional stop loss financing model for high cost cases and how that model is not sustainable for gene therapies
• Case Study: Self-funded account and cumulative gene therapy cost impacts on stop loss
Katherine Dallow, VP, Clinical Programs & Strategy, Blue Cross Blue Shield of Massachusetts
11:30am Next-Generation Transfection Reagent for Large Scale AAV Manufacturing
• Transient transfection of
suspension cells is the most
commonly used method for AAV
manufacturing
• However, this method shows some
limitations when upscaling the process
• To address this concern, we
developed a novel transfection
reagent showing increased AAV
titers and improved transfection
protocol for large scale bioreactors
Géraldine Guérin-Peyrou, Director of Marketing & Communication, Polyplus Transfection
11:30am Quality & Regulatory Requirements from Clinical to Commercial Manufacturing
• Keeping pace with the evolving regulations and interacting effectively with regulatory colleagues and agencies
• Understanding the regulatory requirements for a gene therapy multi-product manufacturing facility
• Managing the unique quality challenges encountered in the manufacturing of gene therapy products and scale up
Mindy Leland, Gene Therapy Quality Operations Team Lead, Pfizer
11:30am Harmonizing a Global Program for Gene Therapies
• Gaining insights into creating a global program
• Understanding regulatory requirements for gene therapy development in Europe and the USA
• Leveraging lessons learned from previous approvals in both geographies
• Identifying opportunities/ hurdles for accelerated global development
Anne Dupraz-Poiseau, Chief Regulatory Officer, Orchard Therapeutics
11:30am Payer Perspective on How Innovative Financing for Gene Therapies is Evolving
• Examining financing strategies that are working, those that are not and how payers are course-correcting
• Understanding the nuances of pricing strategies and exploring emerging models
• How might government action help or hinder pricing and value based agreements?
• Case study: Using Zolgensma as a demonstration of the consequences of uncertainty around the FDA label
Michael Sherman, CMO & SVP, Harvard Pilgrim Healthcare Institute
12:00pm Analytical Method Development & Product Characterizations to Support the Late Stage Development of Gene Therapy Products
• New analytical methods to ensure product quality and process consistency
• Gaining valuable biophysical characterization from technologies such as AUC and SEC
• Advanced product characterization to ensure comparability
Xiaohui Lu, Director, Analytical
Development, Ultragenyx
12:00pm Industrializing Gene Therapies: The Role of Manufacturing Technologies
• Successful commercialization of gene therapies is dependent robust, consistent manufacturing methods that are appropriate for the scale required for the targeted indication
• Current manufacturing is often dependent on technologies that cannot be scaled in a series of disconnected unit operations reducing consistency and robustness
• This talk will demonstrate the creation of a full end-to-end, scalable platform solution for viral vector manufacturing
• This solution can be deployed rapidly allowing therapies to get to market rapidly
Clive Glover, Director, Cell & Gene Therapy, Pall Biotech
12:00pm Embedding Advocacy and Engagement in Your Clinical Development Program
• Patient organization engagement roadmap
• Critical success factors for integrating patient perspective into the development plan
• Real-world insights
Kendall Davis, Manager, Patient Advocacy & Engagement Strategy, Center for Rare Diseases, PRA Health Sciences
Amy Raymond, Director of Therapeutic Expertise, Center for Rare Diseases, PRA Health Sciences
12:00pm The Evaluation of Cell and Gene Therapies
• To outline the challenges of applying health technology assessment to cell and gene therapies
• To consider the need for managed access arrangements
• To provide case histories from NICE’s experience
Nick Crabb, Programme Director - Scientific Affairs, NICE
12:30pm Speaking Position Reserved for Bio-Rad
12:30pm Make vs. Buy Considerations
in the Gene and Cell Therapy
Space
Capital vs operational expenditures
associated with the existing
strategies
• Implications to intellectual property
and quality control
• Remaining solution-driven in the
face of tight timelines
Jesse McCool, CTO, Cytovance
Biologic
Tony Khoury, VP, Technical Services,
Project Farma
12:30pm Panel Discussion: Investigating the Current Global Regulatory Landscape in Light of Recent Approvals
• Learning from previous approvals to shape standards for future approvals
• Emphasizing safe and responsible development of gene therapies
• Exploring impacts on regulation of increased numbers of gene therapies looking for approval
Anne Dupraz-Poiseau, Chief Regulatory Officer, Orchard Therapeutics
Peter Marks, CBER, FDA
Alex Xu, Chief Scientist, CFDA/CDE
Aron Stein, VP Global Regulatory Affairs, Sangamo Therapeutics
12:30pm Speaking Position Reserved forCareMetx
1:00 pm Networking Lunch
CMC & ANALYTICAL
Enhancing Analytical Characterization & Developability Assessments
MANUFACTURING SCALE-UP
Establishing Scalable Manufacturing Processes
CLINICAL
Building Clinical Trials to Maximize Meaningful Output
COMMERCIAL
Evaluating Gene Therapy Pricing & Reimbursement Models
2:00pm Keeping CMC Activities Off the
Critical Path for Gene Therapy Development
• Accelerating AAV-based gene
therapy requires a strategic
development plan
• Advantages of early investment in
CMC activities
• Improving AAV Manufacturability
• Balancing speed-to-market and product quality
Rajiv Gangurde, Senior Director, CMC Strategy & Execution, Voyager Therapeutics
2:00pm Speaking Position Reserved for Brammer Bio
2:00pm Extracting Meaningful Data from Clinical Trials to Present to Regulatory Authorities
• Developing a data extraction strategy and tool for data collection, taking regulators and patients into account
• Enabling successful medical record collection to support robust collection of data and managing extraction during a pandemic
• Setting up the team to analyze the extracted data and to make sense of it
Kathleen Kirby, Senior Vice President, Development Operations, BridgeBio Gene Therapy
2:00pm Precision Financing Framework and Pilots: Latest Finding from the MIT FoCUS Consortium
• US federal policy limitations on reimbursement innovation
• US precision financing in action
• Administrative and execution challenges of reimbursement – discussing patient mobility and tracking issues
• Is Europe leading the US in financing orphan therapeutics?
Mark Trusheim, Strategic Director, NEWDIGS & Visiting Scientist, MIT
2:30pm Rapid CMC Development & Pre-Commercial Considerations for rAAV Gene Therapy Products for Rare Diseases
• Establishing a fast-to- clinic CMC development strategy
• Increasing volumetric productivity and improving yields across chromatography and filtration steps
• Demonstrating scalability to 250L within 3 months post-receipt of new expression cassette
• Developing analytical characterization methods to enable product quality assessment to maintain comparability and enhance product understanding
James Warren, VP, Pharmaceutical Development, Ultragenyx
2:30pm PANEL DISCUSSION: Selecting & Implementing the Most Appropriate
Methodologies to Ensure Robust, Cost-Effective & Scalable Manufacturing
It’s evident that to scale-up any manufacturing within gene therapy, two key considerations are cost-effectiveness of the process, as well as how robust the process is itself. This panel will discuss how to identify the most appropriate methodologies for certain indications and talk about how to go on to implement these effectively to save both time and money in the manufacturing process.
2:30pm Recognizing the Best Clinical Candidates for a Gene Therapy
• Identifying the best clinical candidate for specific indications
• Establishing approaches that could robustly select vectors on a case-by-case basis
• Investing in vectors with the best longterm outcomes
Vivian Choi, Head of Global Gene Therapy Research, Takeda
2:30pm Pricing & Reimbursement Strategies for a Hemophilia B Gene Therapy Launch
• How do we show value of the product to different stakeholders?
• Exploring potential pricing strategies and how to implement this in practice
• Understanding different payment model dynamics and how this links to patient access
Frank Zhang, VP, Pricing, Market Access, Value & Policy, uniQure
3:00pm Panel Discussion: Understanding Manufacturing and CMC Parameters that Require Standardization
• Reducing variability of assays – what approaches are involved
• Updating historically variable assays such as the TCID50 infectivity assay – what alternatives are there?
• Investigating automation as an approach to reduce variability
Thomas Page, VP, Engineering & Asset Development, Fujifilm Diosynth
Karen Walker, Sr Advisor, Cell and Gene Therapy Manufacturing, Genentech
Mauricio Umana, Associate Director, Global Regulatory CMC Lead, Biogen
• Insights into process development efforts to achieve greater transfection efficiency
• Upstream optimizations involved in transitioning between manufacturing processes
• Cutting through outdated information to give a realistic impression of the cost-effectiveness of manufacturing platforms
Luca Alberici, CBO, MolMed
Additional speakers to be confirmed
3:00pm Panel Discussion: Evaluating Challenges Encountered in Small Patient Number Trials
• Discussing how to choose the most suitable comparator
• Emphasizing the importance of understanding the natural history of the disease
• Examining likelihood of randomised placebo-controlled studies in rare disorder trials
Mohammad Asmal, VP, Head of Clinical Research and Development,
bluebird bio
Daniel Chung, Global Medical Strategy Lead, Ophthalmology, Spark
Therapeutics
Julie Lin, Global Project Head of Early Clinical & Business Development, Rare Diseases, Sanofi
Adam Shaywitz, CMO, BridgeBio Gene Therapy
Speaker to be Confirmed Invitae
3:00pm Panel Discussion: Collecting & Utilizing Data to Support Gene Therapy Market Access
• Understanding approaches ensure appropriate data is collected at an appropriate time to support market access decision making
• Investigating approaches to measure the performance of data gathered
• Reality check: discussing how to demonstrate efficacy in real life and incorporating the patient perspective into market access decision making
Mark Trusheim, Strategic Director, NEWDIGS & Visiting Scientist, MIT
Additional speakers to be confirmed
3:30 pm Virtual Scientific Poster Session
Synopsis
The Poster Session is an informal part of the conference agenda, allowing you to connect with your peers using our online platform and continue to forge new and existing relationships. During this session over 25 scientific posters will be presented, showcasing the latest breakthroughs in the field and giving you the opportunity to gain an insight into the future of the field.
Investigating Novel Vector Technologies With the Promise of Enhancing Gene Delivery
4:30 pm 3rd Generation AAV Manufacturing Platform & In-Process Controls
Synopsis
• A non-affinity AAV manufacturing process to manage formation of complexes and result in extra low hcDNA contaminants will be presented
• New column to better separate empty and full AAV particles will be introduced
• Rapid and sensitive analytics for in-process control of AAV manufacturing using HPLC and different in-line detectors will be shown
• New ultra-sensitive DNA assay showing major AAV-associated DNA contamination will be presented
5:00 pm Improving AAV Capsids Using Directed Evolution
Synopsis
- Current AAV serotypes have relatively modest tissue selectivity. Not all tissue types can be effectively transduced
- Directed Evolution can dramatically enhance AAV preference and lead to effective transduction of otherwise unavailable tissue types
- Promoter engineering can further enhance specificity
- These “Next Generation” vectors have the potential to broaden the effective and safe use of gene therapy
5:30 pm SV40-Based Gene Delivery Vectors as an Alternative to AAV Vectors for In Vivo Gene Therapy •
Synopsis
- Replication-defective Simian Virus 40 (SV40)-based gene delivery vectors are safe
- SV40 vectors are non-immunogenic and unique in their capacity to induce immune tolerance to the transgene products in humans
- SV40 vectors are an attractive alternative to AAV vectors for clinical in vivo gene therapy