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April 30 – May 2, 2018

Boston, MA
Earlybird Rate Closes Friday – Save $400

Day One
Tuesday, May 1, 2018

Day Two
Wednesday May 2, 2018

07.00
Registration, Coffee & Networking

07.50
Chair’s Opening Remarks

08.00
Keynote: Delivering on the Promise of Gene Therapies: Navigating Key Decision Points in the Path to a Groundbreaking Approval

Synopsis

  • 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

08.30
Cross-Industry Perspectives on Establishing Gene Therapies as Commercially Viable Therapeutics

Synopsis

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?

09.30
Evolving a Strategy to Narrow Down the Potential Scope of Development in the Gene Therapy Space

Synopsis

  • 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

10.00
Speed Networking & Morning Refreshments

Enhancing Manufacturing Practises in Readiness for Commercialization

11.30
Defining and Maintaining Effective Manufacturing and Regulatory Standards

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

Synopsis

  • 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

11.45
Comparing Manufacturing Methods for their Suitability for Long-Term Commercial Manufacturing

Synopsis

  • 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

12.15
Strategic Decision Making For Scaling Gene Therapy Manufacturing

Synopsis

  • 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

12.45
Lunch & Networking

Establishing Robust Preclinical & Clinical Development Strategies

13.45
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

Synopsis

  • 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

14.15
Defining Robust Clinical Development Strategies to Deal With the Unique Considerations of Rare Disorders

Synopsis

  • 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

14.45
Investigating the Key Factors Influencing the Clinical Development of Gene Therapies

Synopsis

  • 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

15.15
Afternoon Refreshments & Networking

Navigating the Late Stage Clinical Development & Regulatory Landscape

16.15
Navigating the Challenges of Gene Therapy Drug Development for Ultra-Rare Conditions

Synopsis

  • 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

16.45
Assessing the Key Advantages and Disadvantages of Accelerated Approval Pathways

Synopsis

  • 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

17.15
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

Synopsis

  • 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

17.45
Chair’s Closing Remarks