Pre-Conference Workshop Day - Tuesday, March 9

*All times are shown in EST

Workshop A

8.30am - 11.00am

Gene Therapy for Blood Disorders 101

An introduction into the basic science of gene therapies work and the specific challenges for the developers targeting blood disorders.

As you seek to learn more about the gene therapy field, join this workshop to:

  • Get up to speed with the basics of the science of gene therapy
  • Improve your understanding of the specific challenges for blood disorder patient populations specific to gene therapy
  • Discuss the therapeutic specific implications for the clinical and commercial aspects of the field compared to other therapeutics

Workshop B

11.30am - 2.30pm

Exploring Advances in Preconditioning for Ex Vivo Therapeutics

Moving beyond oncology applications, discussing the latest preclinical research to optimize the patient conditioning process for ex vivo gene therapies.

Attend this workshop to:

  • Hear the latest advances in preclinical conditioning regimes using antibody-based techniques
  • Discuss the suitability of the technique to sickle cell disease and beta thalassemia
  • Understand the risks this technique would mitigate for future gene therapy patients

Workshop C

3pm - 6pm

Navigating Current Regulatory Guidelines for Gene Therapies for Blood Disorders

From ultra-rare blood disorders to more common blood disorders, this workshop will cover what is currently known about regulatory guidance and experiences of gene therapy developers in the space.

At this workshop you will:

  • Learn about the feedback given to approved products in gene therapy
  • Navigate the vague guidelines issued by the FDA
  • Discuss common pitfalls and regulatory strategies for future success in the blood disorders landscape