LinkedIn Icon
April 2018
Boston, MA


Day One
Tuesday, April 25, 2017

Day Two
Wednesday, April 26, 2017

Chair’s Opening Remarks

Clinical Development of Gene Therapies for Rare Disease: FDA Perspective

  • Lei Xu Chief, General Medicine Branch 2, Division of Clinical Evaluation , and Pharmacology/Toxicology, OTAT, CBER, FDA


  • Overview of OTAT, CBER, FDA
  • Assessing the statutory requirement for new drug/biologics approval
  • Exploring regulatory pathways leading to approval
  • Improving interactions with the FDA
  • Evaluating the lessons learned from Spinraza

Improving the Clinical Development of Gene Therapy Approaches

Overcoming Patient Recruitment & Trial Design Challenges to Accelerate Clinical Progression


  • Learn from innovative gene therapy trial designs in the rare disease space – structure, patient selection/recruitment/retention and endpoints
  • Optimizing multi-site travel and identifying centers of excellence to accelerate clinical development
  • Overcoming the logistical challenges involved in long term patient monitoring


The Selection of Endpoints for Ophthalmology Gene Therapy Trials in the Rare Disease Space


  • What are the key endpoints being explored at the moment and in the future?
  • What endpoints are realistically measurable in the timescale?
  • What is the regulators’ feedback relating to these endpoints?

Morning Refreshments & Networking

Bridging Gaps Through Early Integration of the Internal Medical Affairs Function


  • Illustrating how medical affairs can contribute to early stage clinical development of gene therapies
  • Investigating how medical affairs can integrate multiple stakeholders to address the scientific needs of the community
  • Building key external relationships to advance the program and prepare for future challenges

Panel Discussion: What Does it Take to Succeed in the Gene Therapy Space?


With an array of companies vying to establish themselves in the gene therapy space, this is your oportunity to learn from organizations large and small who have early success stories to tell and who will really pioneer the field over the next year.

  • How can a small company establish itself in this rapidly evolving field?
  • Taking on developmental and manufacturing challenges with limited resources – learn from the most innovative small biotechs and how they have achieved success in this field
  • Investigating clinical case studies for gene therapies in rare disease
  • Key clinical, regulatory and manufacturing considerations to pave the way to successful commercialization of gene therapies

Lunch & Networking

Patient Advocacy, Pricing & Reimbursement Considerations

Patient Advocacy in the Gene Therapy Space: A Case Study in Hemophilia


  • How early in gene therapy development should companies begin their patient advocacy efforts?
  • What are some key considerations for patient advocacy that are unique to gene therapy?
  • Gain transferrable insights from the hemophilia space that can enhance patient advocacy efforts in other disease areas

Analyzing how a Single Gene Therapy Intervention May Be Priced Effectively for Different Rare Diseases


  • In an environment where the payers and market are accustomed to corrective therapies, how can potentially transformative or curative therapeutics be priced?
  • Establishing a reimbursement framework for gene therapies
  • Investigating the global pricing and reimbursement environment for gene therapies – contrasting between US and European approaches

Afternoon Refreshments & Networking

Commercialization & Effective Route to Market Strategies

Funding & Investment to Further the Development of Gene Therapies

  • Beth White AVP, Rare Disease Commercial Development, Pfizerx


  • How can the industry encourage funding for this space?
  • What are Big Pharma’s expectations for this type of therapy? How can we learn from past failures in the field to ensure funding is maintained and enhanced?
  • What are the inhibitors for investment in this industry?

Panel Discussion: Analyzing the Evolution of Deal Structures Between Companies Pioneering the Gene Therapy Space


  • Investigating how the field has moved from acquisitions to a more collaborative ecosystem
  • State of the gene therapy lancscape: Assessing past successes to inform future commercial strategies for companies in the gene therapy field
  • How will interactions between big pharma and pioneering small biotechs adjust to this rapidly evolving space?

Chair’s Closing Remarks

Close of Conference