Next Generation Strategies for Orphan Drug Development

Original Air Date:  Monday, October 19, 2020
Time:  10:00 AM EDT/4:00 PM CET

The past decade has witnessed a remarkable surge in rare disease research leading to an impressive pipeline of promising investigational products. Novel approaches, including genomics, data mining and computational repurposing of drugs will continue to fuel discovery and consequent clinical development of medicines.

In this webinar, a panel of three rare disease experts will discuss current trends in orphan drug research, the role of open-label extension studies in rare disease development and the unique recruitment challenges for rare disease trials.

The convergence of next generation genomics, advances in clinical trials operations, and the strong commitment of all stakeholders active in the field will bring us closer to the ultimate goal of improved health for people living with rare diseases.

What will you learn from this webinar:

  • What are the current trends in the orphan space
  • How to harvest the most information from small population trials
  • What motivates patients to participate in clinical trials
  • What are the imperatives and pitfalls of open-label extension trials



Shi Yin Foo, MD, Ph.D
Chief Medical Officer, Cydan II

Dr. Shi Yin Foo brings 20 years of experience as a practicing physician and in drug development. She has expertise in broad therapeutic areas, including rare diseases, and an interest in novel trial designs. She is the Chief Medical Officer at Cydan II, a rare disease drug accelerator; she previously held executive positions in Cardioxyl, Imara and Tiburio Therapeutics, and founded Orchard Biomedical Consulting. She received her MD and Ph. D in immunology from Stanford University, and an MMSc in clinical science from Harvard Medical School. She was clinical faculty at the Massachusetts General Hospital and Harvard Medical School, and a dual Diplomate of the American Board of Internal Medicine and in Cardiology.


Ari Zimran, MD
Gaucher Unit, Shaare Zedek Medical Center

Professor Ari Zimran was the Founder and the Director of the Gaucher Unit at Shaare Zedek Medical Center in Jerusalem, Israel from 1990 to 2018, and is currently a senior physician at the Unit. This is the world’s largest referral center for Gaucher disease (GD), where more than 850 patients have been followed, and about 350 patients are treated with enzyme replacement therapy. Professor Zimran developed his interest in GD during his fellowship at the Scripps Research Institute, Department of Molecular and Experimental Medicine, in La Jolla, California, USA under the mentorship of Professor Ernest Beutler. He has published more than 320 professional papers and reviews and has edited three books.


Heschi Rotmensch, MD
Chief Medical Officer, CATO SMS

Dr. Heschi Rotmensch has over 25 years of experience leading clinical development and medical affairs teams at pharmaceutical companies (MSDRL) and CROs (Parexel International and Cato Research). Currently he serves as the Chief Medical Officer of CATO SMS. Prior to that, he held the position of Associate Director of the Division of Clinical Pharmacology at Jefferson Medical College, Philadelphia, PA and Chief of Medicine at the Edith Wolfson Medical Center, Holon, Israel. Heschi has published more than 80 scientific papers and reviews and 9 book chapters. He is board certified in Internal Medicine and Clinical Pharmacology and has an MBA from Kellogg School of Management, Northwestern University, Evanston, IL.

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