Workshop A

Tuesday, March 03

8:00 am - 11:00 am

Enhancing Gene Editing Techniques to Improve Gene Therapy Programs for Blood Disorders


Gene editing is a revolutionary technique which is starting to make its way into clinical trials for a variety of indications. It is evident that more and more companies are realizing the potential of gene editing techniques in the blood disorders space. With real promise to deliver durable, curative and transformative therapies to patients with disorders such as sickle cell disease and β-thalassemia, improving these gene editing technologies and techniques is vital to ensure these gene therapies reach the patients who need it.  

Attend this workshop to learn about:

  •  Improving the precision of gene editing
  • Minimizing off-target effects
  • Requiring more than one genome edit
  • How difficult is it to insert larger genes?
  • Which animal models are most appropriate?
  • Discussing transferrable insights across gene editing platforms
  • Exploring applications of CRISPR in hematopoietic stem cells


Workshop Leader: John Woolley, Principal Investigator, University of Liverpool

Workshop B

Tuesday, March 03

11:30 am - 2:30 pm

Establishing a Presence in the Blood Disease Space: Understanding Key Clinical & Commercial Considerations at the Beginning of Your Gene Therapy Program


There continues to be great interest in creating gene therapies for blood disorders, with one indication approved in this space, and several others in late-stage clinical trials. As more and more companies begin investing in the field, it is crucial that they have an excellent understanding of clinical, commercial and regulatory aspects of the program from the very beginning. Entering the blood disorders space with an excellent grounding will set up for success later in the pipeline.

Attend this workshop to learn about:

  • Beginning a program for blood disorders in the gene therapy space
  • Exploring key regulatory requirements
  • Challenges in translating from pre-clinical studies into the clinic
  • Discussing the importance of a commercial outlook from early stages of the program
  • How to transition into a more industrialized setting, and understanding the possible limitations


Workshop leaders:

  • Colleen Dansereau, Director of Clinical Operations, Boston Children’s Hospital
  • Matt Trudeau, VP Commercial Operations, bluebird bio

Workshop C

Tuesday, March 03

3:00 pm - 6:00 pm

Strategic Gene Therapy Product Platforms and Partnerships: From Codevelopment to Companion Products


With more investments in gene therapies to treat blood diseases than ever before, it is imperative that your product stands out in what promises to be a competitive market. By building an effective codevelopment program, understanding new FDA guidances surrounding codevelopment, and identifying which companion and diagnostics products would best enhance your platform, you can ensure your therapy will have a competitive advantage that enables you to stand out from the crowd.


Attend this workshop to learn about:

  • How companies collaborate to form an effective business partnership
  • Regulatory strategy in codevelopment
  • Using companion products in program development
  • Trial design and clinical strategy for codevelopment
  • Challenges of having multiple sponsors and concurrent programs
  • Companion product marketing and commercialization


Workshop leader: Angela Johnson, Senior Director of Regulatory Affairs, Sigilon Therapeutics