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


  • 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

  • Seng Cheng SVP & CSO, Rare Disease Research Unit , Pfizer


  • 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


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


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

11:00 am 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 a 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

11:30 am TransIT-VirusGEN GMP: An Innovative Transfection Platform for Cell and Gene Therapy Development and Manufacture


  • TransIT-VirusGEN Transfection Reagent and Kits were formulated specifically for high titer Lentivirus and Adeno-associated Virus production. Our RUO, SELECT and (upcoming) GMP configurations and supporting documentation equip researchers with unparalleled products for Cell and Gene Therapy development and manufacturing
  • Assessing AAV titers via genome copies per milliliter (GC/ml) is common, however it is not a robust indicator of virus quality. We will show how TransITVirusGEN Transfection Reagent and Kits enable not only higher GC/ml titers  but also higher functional titers than competing reagents

12:00 pm Analytical Method Development & Product Characterizations to Support

  • Xiaohui Lu Director, Analytical Development , Ultragenyx


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

12:30 pm Speaking Position Reserved for Bio-Rad


Making Informed Strategic Manufacturing Decisions and Designing Effective Facilities

11:00 am Standardizing Gene Therapy Manufacturing Facilities


  • Establishing standards and implementing 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

11:30 am m Quality & Regulatory Requirements from Clinical to Commercial Manufacturing

  • Mindy Leland Gene Therapy Quality Operations Team Lead, Pfizer


  • Keeping pace with the evolving regulations and interacting effectively with regulatory colleagues and agencies
  •  Understanding the regulatory requirements for a gene therapy multiproduct manufacturing facility
  • Managing the unique quality challenges encountered in the manufacturing of gene therapy products and scale up

12:00 pm Industrializing Gene Therapies: The Role of Manufacturing Technologies


  • Successful commercialization of gene therapies is dependent on 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

12:30 pm 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

Exploring the Continually Evolving Regulatory Landscape

11:00 am A Strategic Playbook for Key Rare Disease Gene Therapy Regulatory Milestones

  • Angela Johnson Senior Director, Regulatory Affairs , Sigilon Therapeutics


  • 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

11:30 am 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

12:00 pm Embedding Advocacy and Engagement in Your Clinical Development Program

  • 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


  •  Patient organization engagement roadmap
  • Critical success factors for integrating patient perspective into the development plan
  • Real-world insights into applications

12:30 pm 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

COMMERCIAL Understanding
the Payer Perspective

11:00 am Shifting Payment Paradigms: How One-Time High Cost Treatments Challenge the Traditional U.S. Payer Model

  • Katherine Dallow VP, Clinical Programs & Strategy , Blue Cross Blue Shield of Massachusetts


  • 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

11:30 am 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

12:00 pm The Evaluation of Cell and Gene Therapies

  • Nick Crabb Programme Director, Scientific Affairs NICE


  • 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’sexperience

12:30 pm Speaking Position Reserved for CareMetx


1:00 pm Networking Lunch

CMC & ANALYTICALEnhancing Analytical Characterization & Developability Assessments

2:00 pm 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

2:30 pm Rapid CMC Development & PreCommercial 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

3:00 pm PANEL DISCUSSION: Understanding Manufacturing and CMC Parameters that Require Standardization

  • Thomas Page VP, Engineering & Asset Development, FUJIFILM Diosynth Biotechnologies
  • Karen Walker Sr Advisor, Cell & Gene Therapy Manufacturing , Genentech
  • Mauricio Umana Associate Director, Global Regulatory CMC Lead , Biogen


  • 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

Establishing Scalable Manufacturing Processes

2:00 pm Speaking Position Reserved for Thermo Fisher Scientific


2:30 pm Scalable Adeno Associated Virus Production from Cell Culture to Purified Bulk


Manufacturing safe and efficacious clinical-grade AAV virus relies on scalable and cost-effective production processes. Cytiva has developed an efficient process for AAV production in suspension cell
culture to purified bulk product. In this presentation, we will present:

  • How to optimize of each process step of AAV production in suspension cell culture
  • How to evaluate and optimize cell culture conditions and plasmid transfection
  • Purification methods based on filtration and chromatography, developed for AAV production

2:30 pm 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.

  • 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

Building Clinical Trials to Maximize Meaningful Output

2:00 pm 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

2:30 pm 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-bycase basis
  • Investing in vectors with the best longterm outcomes

3:00 pm PANEL DISCUSSION: Evaluating Challenges Encountered in Small Patient Number Trials

  • Daniel Chung Medical Affairs & Clinical Ophthalmic Lead , Spark Therapeutics
  • Julie Lin Global Project Head of Early Clinical & Business Development , Sanofi Genzyme
  • Adam Shaywitz CMO, BridgeBio Gene Therapy


  • 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

COMMERCIAL Evaluating Gene Therapy Pricing & Reimbursement Models/span>

2:00 pm Precision Financing Framework and Pilots: Latest Finding from the MIT FoCUS Consortium

  • Mark Trusheim Strategic Director, NEWDIGS & Visiting Scientist , MIT


  •  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?

2:30 pm Pricing & Reimbursement Strategies for a Hemophilia B Gene Therapy Launch

  • Frank Zhang VP, Pricing, Market Access, Value & Policy , uniQure


  • 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 acces

3:00 pm PANEL DISCUSSION: Collecting & Utilizing Data to Support Gene Therapy Market Access

  • Mark Trusheim Strategic Director, NEWDIGS & Visiting Scientist , MIT


  •  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

3:30 pm Virtual Scientific Poster Session


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


• 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

  • Melissa Kotterman VP, Discovery & Engineering, Co-Founder , 4D Molecular Therapeutics


  • 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 •

  • Peter de Haan Chief Scientific Officer & Co-Founder , Amarna Therapeutics


  • 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

6:00 pm Chair’s Closing Remarks