7:45 am Registration, Coffee & Networking

8:35 am Chair’s Opening Remarks

  • Catalina Driscoll Global Head, New Product Planning, Rare Blood Disorders, Sanofi Genzyme

EVALUATING COMMERCIAL STRATEGIES TO SUCCESSFULLY LAUNCH GENE THERAPIES FOR BLOOD DISORDERS

9:00 am Putting Theory into Practice: A Case Study from Freeline Therapeutics

Synopsis

  • Discussing the AAV vector platform Freeline has developed to treat Hemophilia B
  • Presenting recent clinical data showing progress so far
  • Overcoming manufacturing challenges to deliver therapies to patients effectively

9:30 am Panel Discussion – Assessing the Commercial Realities of Launching Gene Therapies in the Blood Disorder Space

  • Eileen Sawyer Vice President, Global Medical Affairs, uniQure
  • Catalina Driscoll Global Head, New Product Planning, Rare Blood Disorders, Sanofi Genzyme
  • Matt Trudeau Vice President, Commercial Operations, bluebird bio
  • Brendan Hayes Director, External Affairs, National Hemophilia Foundation

Synopsis

  • Determining the most effective ways of demonstrating product value
  • Ensuring accessibility of products through co-operation with payers and patients
  • Discussing creative solutions to increase accessibility to patients by overcoming cost and geographic barriers

10:15 am 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.

10:45 am Morning Refreshments

OVERCOMING BARRIERS TO PATIENT RECRUITMENT IN CLINICAL TRIALS FOR NON-MALIGNANT BLOOD DISORDERS

11:30 am Exploring Hemophilia-Specific Recruitment Challenges

  • Paul Monahan Clinical and Translational Lead, Hematology, Spark Therapeutics

Synopsis

  • Discussing eligibility of patients with neutralizing antibodies to the proposed AAV vector
  • Evaluating how the number of hemophilia clinical trials impacts recruitment
  • Determining whether current treatment is adequate for the hemophilia community

12:00 pm Exploring Patient Recruitment Challenges in Sickle Cell Disease and β-Thalassemia

Synopsis

  • Discussing community-specific patient recruitment challenges of sickle cell disease
  • Highlighting β-thalassemia-specific patient dynamics and how they influence patient recruitment
  • Prioritizing education of eligible patient groups to encourage trial participation
  • Understanding how to optimize patient experience considering the nature of the treatment

12:30 pm Lunch & Networking

1:30 pm Durability Mastermind: Does Durability Really Matter? Assessing Relative Impacts of Reduced Efficacy Over Time on a Gene Therapy Program

Synopsis

In this Mastermind Session, different groups will be given the same set of questions to answer around the following topics:

  • How will durability impact payer decisions?
  • Re-dosing considerations
  • How to build value dependent on efficacy results
  • What happens if expression starts to drop off?
  • How can we utilize pre-clinical studies to better predict long-term effects?
  • Why might factor expression drop? Is there a difference between Factor VIII and Factor IX expression?

 

Following this, insights from different groups will be collated into a single document which will then be circulated to the entire group following the session.

2:15 pm Afternoon Break & Refreshments

INVESTIGATING THE NEXT GENERATION OF HEMOPHILIA GENE THERAPIES

2:45 pm Hemophilia B Gene Therapy in Mice Using a Novel Chimeric AAV Capsid Combined with a Potency Enhanced FIX Variant

  • Grant Blouse Vice President Translational Research, Catalyst Biosciences

Synopsis

  • Advantages of next generation AAV capsids combined with transgenes having increased functionality to lower vector doses
  • Developing a novel AAV vector technology to reduce systemic payload of hemophilia B gene therapies
  • Discussing pre-clinical data on the performance of the novel technology

3:15 pm Evaluating Immunogenic Responses in Systemic Administration: Determining the ‘Right’ Dose

Synopsis

  • Discussing immunogenicity concerns in systemic administration of AAV vectors
  • Determining relative impacts of capsid type and payload on immune response
  • Optimizing dose levels to minimize immune response to the gene therapy

3:45 pm Chair’s Closing Remarks

  • Catalina Driscoll Global Head, New Product Planning, Rare Blood Disorders, Sanofi Genzyme

4:00 pm End of Day One