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April 30 – May 2, 2018

Boston, MA
The Countdown Is On – 1 Week To Go!

Day One
Tuesday, May 1, 2018

Day Two
Wednesday May 2, 2018

Registration, Coffee & Networking

Chair’s Opening Remarks

Keynote: Delivering on the Promise of Gene Therapies: Navigating Key Decision Points in the Path to a Groundbreaking Approval


  • Investigating key takeaways from the development of Luxterna: How can the field use this pioneering therapeutic to guide the way for future progress
  • Establishing how to bridge the gap between early-stage academic research and commercially viable gene therapy products
  • Sharing the factors involved in the decision to fully integrate manufacturing capabilities and understanding how this will impact future development
  • Pioneering gene therapy development in the haemophilia space

Cross-Industry Perspectives on Establishing Gene Therapies as Commercially Viable Therapeutics


Gene therapies are set to redefine the treatment of rare diseases but these conditions present many distinct development challenges. This panel discussion will bring together experts from the companies pioneering this field to analyse how we can overcome these hurdles and realize the commercial potential of gene therapies

  • Ensuring that consistent standards are implemented throughout every stage of the development of gene therapy products
  • Evaluating the unique considerations that potentially curative therapeutics demand – how will current systems need to adapt to this paradigm shift?
  • Is the field beginning to transition to more common indications? To what extent are rare diseases ‘testing grounds’ to prepare for indications with much higher patient populations?

Evolving a Strategy to Narrow Down the Potential Scope of Development in the Gene Therapy Space


  • Identifying and prioritizing therapeutic areas in which to focus development
  • Understanding the key factors in deciding whether to base a development strategy on specific organs, systems or diseases
  • Learning from the development of gene therapies in the past and integrating these lessons into current thinking

Speed Networking & Morning Refreshments

Enhancing Manufacturing Practises in Readiness for Commercialization

Establishing Effective Manufacturing & Regulatory Standards

  • Sven Kili VP, Head of Cell & Gene Therapy Development, GSK


  • Defining the elements of gene therapy development that need to be standardized
  • Investigating strategies to overcome analytical limitations to make comparability easier
  • Establishing effective comparability practices

Comparing Manufacturing Methods for their Suitability for Long-Term Commercial Manufacturing


  • Delivering insights into the quality and purity of material generated by various manufacturing methods
  • Investigating new developments to optimize the productivity of the platforms currently available
  • Highlighting the approaches most amenable to scale-up and late-stage development

Strategic Decision Making For Scaling Gene Therapy Manufacturing


  • Establishing robust processes to ensure consistency through development and commercialisation
  • Investigating the key aspects of the manufacturing processes capable of producing sufficient vector for more prevalent rare diseases like DMD
  • Ramping up manufacturing – investigating the key aspects of the scale-up process

Assembling the Range of Resources Required for Rare Disease R&D Organizations

  • Manuel Duval Director, Data Science & Bioinformatics,


  • Investigating how the identification of the causative variants requires the deployment of specifically geared DNA-Seq workflows due to their inherently very low frequency in the human population.
  • Understanding hurdles in the clinical space, including developing highly specific and sensitive models using clinical numerical and categorical values that can be deployed to health care providers for detection and diagnostic applications, and how running these programs involves provisioning highly specialized trades at different stages of the pipeline
  • Reviewing how the platform provides the means to significantly improve the efficiency of these operations

Lunch & Networking

Establishing Robust Preclinical & Clinical Development Strategies

Developing Robust Preclinical Development Strategies to Deal With the Unique Considerations of Gene Therapies

  • Alison McVie-Wylie Senior Director, Gene Therapy and Biologics Pharmacology, Rare Diseases, Sanofi


  • Taking gene therapies to the clinic – understanding the key factors that constitute a robust preclinical development plan
  • Identifying and utilizing animal models to build robust preclinical data to support clinical development
  • Building a robust preclinical plan regardless of indication

Defining Robust Clinical Development Strategies to Deal With the Unique Considerations of Rare Disorders


  • Approaching development challenges and common clinical trial design issues faced when developing gene therapies in the rare disease space
  • Contrasting clinical development in the rare disease space with more common disease areas – what are the unique features?
  • Understanding the promise of AAV gene therapies in addressing significant unmet need

Investigating the Key Factors Influencing the Clinical Development of Gene Therapies


  • Understanding the unique factors influencing gene therapy clinical trials
  • Approaches to recruit sufficient numbers of patients to deliver meaningful clinical results
  • Case study: key takeaways from the clinical development of a stem-cell gene therapy for cerebral adrenoleukodystrophy

Talk Details to be Confirmed

Afternoon Refreshments & Networking

Navigating the Late Stage Clinical Development & Regulatory Landscape

Navigating the Challenges of Gene Therapy Drug Development for Ultra-Rare Conditions


  • Investigating clinical and clinical-regulatory strategy, including study design, endpoints and the identification of patient populations
  • Gaining insights into managing the availability of clinical trial supply and other CMC challenges
  • Forging key partnerships with academic centers and patient advocacy groups

Assessing the Key Advantages and Disadvantages of Accelerated Approval Pathways


  • Understanding the Regenerative Medicine Advanced Therapy (RMAT) designation: where is it applicable and why?
  • Investigating the role of real world evidence and novel endpoints in the gene therapy space
  • Utilizing natural history data to support clinical development

FDA Office of Orphan Products Development: Core Programs to Facilitate Gene Therapy Development for Rare Diseases

  • Gumei Liu Reviewer & Project Officer, Office of Orphan Products Development, FDA


  • Understanding orphan drug designation and incentives
  • Navigating the orphan drug designation process, specifically in relation to gene therapy products
  • Orphan products grant programs for rare diseases

Chair’s Closing Remarks