Benign Blood Disorders - 2021 World Medical Innovation Forum
May 2–4, 2022

Benign Blood Disorders

Benign Blood Disorders

Thursday, May 20, 2021

9:00 AM – 9:25 AM
Hot Topics

Hemophilia has been and remains a hallmark indication for the CGT. Given its well-defined biology, larger market, and limited need for gene transfer to provide therapeutic benefit, it has been at the forefront of clinical development for years, however, product approval remains elusive. What are the main hurdles to this success? Contrary to many indications that CGT pursues no therapeutic options are available to patients, hemophiliacs have an increasing number of highly efficacious treatment options. How does the competitive landscape impact this field differently than other CGT fields? With many different players pursuing a gene therapy option for hemophilia, what are the main differentiators? Gene therapy for hemophilia seems compelling for low and middle-income countries, given the cost of currently available treatments; does your company see opportunities in this market?

Nancy Berliner, MD
  • Chief, Division of Hematology, BWH
  • H. Franklin Bunn Professor of Medicine, HMS
Theresa Heggie
  • CEO, Freeline Therapeutics
Gallia Levy, MD, PhD
  • Chief Medical Officer, Spark Therapeutics
Amir Nashat, PhD
  • Managing Partner, Polaris Ventures
Suneet Varma
  • Global President of Rare Disease, Pfizer
  • Q&A

    09:30 AM – 09:45 AM

the Future of Medicine and AI

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