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April 2018
Boston, MA


Day One
Tuesday, April 25, 2017

Day Two
Wednesday, April 26, 2017

Registration, Coffee & Networking

Chair’s Opening Remarks

Keynote: From Academic to Commercial Scale – Managing the Rapid Progression of Gene Therapies


  • Bridging the gap between academic proof-of-concept studies to commercialization of gene therapy products
  • Investigating aspects of the drug development path that differentiate gene therapies as therapeutics
  • Understanding the need for coherent, broadly applicable, industry-wide standards to measure the determinants of reactivity of gene therapies

Keynote Panel Discussion: The Future of Gene Therapies for Rare Disease

  • Sam Wadsworth CSO, Dimension Therapeutics
  • Seng Cheng Global Head of Research, Rare Diseases, Sanofi
  • Sven Kili VP & Head of Cell & Gene Therapy Development, GSK


Gene therapy is gaining momentum, but how do we ensure this momentum is sustained and avoid the mistakes that have cost the field in the past? Pioneering biotech companies at the forefront of the field will open the conference by sharing their insights into the key developments that will affect change over the next year:

  • Assess the relative progress of gene therapies in rare ocular, CNS, hepatic and muscular disorders
  • Understand the key challenges standing in the way of pioneering gene therapy approvals
  • How can lessons from the past be used to guide future success in this field?
  • Learn about the critical nature of characterization and standardization – of manufacturing processes, clinical development, regulatory frameworks and pricing and reimbursement
  • Investigate the factors you need to consider as gene therapies gear up for commercialization in the coming years – what steps can you take now to stay ahead of the curve?

Improve Decision Making Early in the Gene Therapy Drug Development Process to Enable Successful Progression Through the Clinic

  • Vivian Choi Associate Director, Head of Gene Therapy Research, Shire Pharmaceuticals


  • Delivering the right gene to the right place at the right time with the right control: What novel approaches can improve our ability to do this?
  • Understanding the need of the disease and how this should influence the design and route of administration of the molecule
  • Analyzing key decision points in the early development of gene therapies for ophthalmic, CNS and hematological rare disorders

Speed Networking & Morning Refreshments

Enhancing Manufacturing Practises in Readiness for Commercialization

Overcoming the Capacity Bottleneck: Managing In-House & Outsourced Value Chains Effectively


  • In-house vs. contact manufacturing: Key considerations when evaluating future commercial-scale manufacturing
  • How to identify and partner with key contract manufacturing organizations throughout the supply chain
  • Managing relationships with contract providers: The importance of quality agreements
  • Identifying strategies to deal with the Catch-22 manufacturing situation: What is the most effective way to manage scale-up in the interim period before commercial scale manufacturing facilities are more readily available?

Investigating CMC Considerations for the Production of a Gene Therapy Product

  • Victoria Sluzky SVP, Quality & Process Development , BioMarin Pharmaceuticals


  • Identifying unique manufacturing considerations for gene therapy products
  • Understanding unique validation considerations for gene therapy products
  • Learning about the unique quality and analytical consideration for gene therapy products

Lunch & Networking

Optimizing the Scalability of Gene Therapy Manufacturing


  • Moving from academic scale to GMP commercial scale manufacturing – how can the key challenges be overcome?
  • What requirements are regulators placing on commercial scale manufacturing and how can these be met?
  • Improving the yield of vector produced by manufacturing processes: Case study examining higher yield required for gene therapies in the haemophilia space

The Patient Pathway: Examining the Complex Patient Logistics Required to Progress Ex Vivo Gene Therapies


  • Investigating the safety and efficacy of ex vivo approaches
  • What are the patient requirements for effective ex vivo gene therapy at a commercial scale?
  • Overcoming the challenge of rejection – pre-conditioning patients prior to ex vivo gene therapy

Afternoon Refreshments & Networking

Gene Therapy for Rare Diseases: EMA Perspective

  • Rune Kjeken Scientific Director, Advanced Therapies , CAT, EMA


  • Through the maze – Navigating the EMA regulatory landscape
  • What regulators want to see –  Lessons from EMA scientific advices on gene therapy products
  • Looking into the future – Identifying gaps in information regarding vector technologies and how the field can work to address these issues

Panel Discussion: Cross-Industry Perspectives on Defining the Regulatory Course for Gene Therapies in Rare Disease


  • Learning about regulatory differences across diseases
  • Manufacturing elements that impact on the regulatory environment
  • Evaluating preclinical models and how the regulators view these
  • Exploring clinical development and endpoints
  • International perspective – examining differences between the European and US regulatory landscapes

Chair’s Closing Remarks