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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 Implementing Effective Analytical Comparability Strategies For Gene Therapy Products

  • Mark Galbraith Head of Quality Control & Analytical Sciences , Spark Therapeutics

Synopsis

  • Establishing comparability and bridging approaches to link early and late-stage development
  • Anticipating and understanding the challenges encountered in in Gene Therapy product development
  • Designing analytical comparability approaches to satisfy regulatory agencies
  • Understanding how analytical methods need to evolve as development reaches the later stages

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

Speed networking is the ideal opportunity to get face-to-face time with many of the brightest minds working in gene therapy. Benchmark against the industry leaders and establish meaningful business relationships to pursue for the rest of the conference and beyond.

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 Developing Phase-Appropriate and Clinically-Applicable Potency Assays
• Understanding the potential requirements for multiple potency assays measuring both function and expression
• In cases where potency assays are impossible to produce, what kinds of surrogates can be utilized? How are these surrogate approaches viewed by the regulators?
• Understanding potency assays that are able to show correlation between vector potency and drug product potency or between drug product potency and clinical outcomes
Fraser Wright, Professor, Pediatrics, Stanford University & CTO, Axovant

11:00am Understanding the Key Challenges Involved in Gene Therapy Commercial Facility Design
• Investigating the unique challenges posed by gene therapy facilities and contrasting with manufacturing facilities for other therapeutics
• Utilizing appropriate technologies to ensure efficiency and costeffectiveness
• Delivering on the promise of gene therapies in multiple indications by designing and maintaining facilities capable of commercial scale manufacturing
Keith Life, Senior Director, Gene Therapy Manufacturing & Viral Vector Production, BioMarin

11:00am Understanding Regulatory Expectations Along the Gene Therapy Timeline
• Clarifying type and standard of data required by regulatory authorities
• Highlighting the minimum requirements for approval
• Discussing appropriate trial designs for rare disorders
Robert Pietrusko, SVP, Regulatory Affairs & Quality Assurance, Voyager 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 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

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 Speaking Position Reserved for Polyplus Transfection

12:00pm Speaking Position Reserved for Project Farma

12:00pm 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

12:00pm Speaking Position Reserved forCareMetx

12:30 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 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

2:00pm Speaking Position Reserved for Brammer Bio

2:00pm Emphasizing the Role of the Patient in Trial Design
• Understanding what a meaningful outcome is for the patient
• Designing trials to minimize impact on patient everyday life
• Establishing meaningful endpoints using patient input
Jill Dolgin, Head of Patient Advocacy, AGTC

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 CMC Developability Assessments of Gene Therapy Viral Vector Products Prior to the Transition from Research into Development
• Transition of new gene therapy molecules from research into development space can result in unexpected challenges putting CMC on the critical path
• Develop-ability assessments of new molecules can help predict where the CMC challenges may lie
• Companies can then decide where to invest their resources in CMC, including an outsource v. internalization strategy
Kathleen Hehir, Head of Gene Therapy Manufacturing & Development, Sanofi

2:30pm Challenges of Manufacturing Gene Therapies into Commercial Realities
• Overview of bluebird bio’s efforts to advance gene therapies for severe genetic disorders and cancer immunotherapy
• Challenges faced in manufacturing cell therapies as they move toward commercial phase – with a particular focus on managing a network of CDMOs
• Approaches bluebird bio is implementing to overcome some of these challenges
Steven Goodman, Head, Drug Product Manufacturing, bluebird bio

2:30pm Extracting Meaningful Data from Clinical Trials to Present to Regulatory Authorities
• Discussing acceptable risk levels when designing a clinical trial
• Understanding regulatory standpoints on surrogate endpoints and natural history comparators
• Collaborating with regulators to build a successful clinical trial with meaningful outcomes
Alexander Kuta, SVP Regulatory Affairs, uniQure

2:30pm Strategies to Place Scientific Evidence into a Value Context
• Approaches to establish value early in product development
• Communicating value to multiple decision makers, including payers and regulators
• Updates to the ICER framework
Pamela Bradt, CSO, ICER

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
Yuan Zhao, Leader, Gene Therapy Section, NIBSC, MHRA
Mark Galbraith, Head of Quality Control & Analytical Sciences, Spark
Therapeutics
Fraser Wright, Professor, Pediatrics, Stanford University & CTO, Axovant

3:00pm Panel Discussion: Selecting & Implementing the Most Appropriate Methodologies to Ensure Robust, Cost-Effective & Scalable Manufacturing
• 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 costeffectiveness of manufacturing platforms
Bryan Piras, Lead Scientist, Process Development, St. Jude Children’s
Research Hospital
Kathleen Hehir, Head of Gene Therapy Manufacturing & Development, Sanofi

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

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
Sunil Karnawat, VP, Market Access & Government Affairs, Ultragenyx
Mark Trusheim, Strategic Director, NEWDIGS & Visiting Scientist, MIT
Lisa Feng, Senior Director, Policy & Advocacy, Cystic Fibrosis Foundation

3:30 pm Afternoon Refreshments & Scientific Poster Session

Synopsis

The scientific poster session is an ideal opportunity to communicate your new results and expertise to a niche audience of industry experts, get feedback from peers and colleagues in the industry and learn how others in the field have been tackling similar challenges.

Analyzing & Enhancing AAV Vectors & Pioneering Novel Vector Approaches

4:30 pm Speaking Position Reserved for BIA Separations

5:00 pm Rapid, Non-Invasive Whole-Body Imaging of AAV Gene Transfer Vectors

Synopsis

  • I-124, cyclotron generated positron emitter, can be covalently linked to AAV capsids, enabling noninvasive, time-dependent imaging of vector biodistribution following administration.
  • Images from 1 hour to several days permits tracking of vector capsid localization as a function of time.
  • Studies in non-immune and anti-vector immune nonhuman primates help to define the initial biodistribution of AAV vectors.
  • This technology is readily adaptable to humans

5:30 pm Understanding the Criteria Essential in Assessing the Potential of Novel Vectors

Synopsis

  • Evaluating the importance of the following characteristics in new vectors:
    – Size of the transgene
    – Immunogenicity issues
    – Cost of manufacturing and cost of goods
    – Accuracy of targeting
    – Investigating the requirements for vectors in different indications and matching vector technology to the specific requirements of different indications

6:00 pm Improving AAV Specificity using Directed Evolution

  • Casey Case SVP, Research & Development , 4D Molecular Therapeutics

Synopsis

  • Current AAV serotypes have relatively modest tissue selectivity. Not all tissue types can be effectively transduced
  • Directed Evolution can dramatically enhance AAV specificity 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

6:30 pm A Platform for the Rational Discovery of Novel AAV Serotypes

Synopsis

  • AAV remains a black box of a virus, despite its widespread use in the clinic
  • AAV has many attributes that we’d like to optimize
  • Fundamental understanding arising from rational design is a valid path to improved manufacturing and clinical outcomes

7:00 pm Chair’s Closing Remarks