Understanding Data for Basic Research – 2023 RARE Drug Development Symposium

May 10, 2023

In May 2023, Global Genes and the Orphan Disease Center of the University of Pennsylvania hosted the RARE Drug Development Symposium and talked about understanding data for basic research. Yssa DeWoody, Cofounder and Director of Research, Ring14 USA, led a discussion about the basic research needed to understand data for drug development research programs.

Building a firm foundation for your research program helps avoid wasted time, money, and effort. Why is the data collected and produced by basic research important? What role does it play in driving research strategies? How can you evaluate the reliability and validity of basic research data? How can you identify the remaining research to be done and support this part of the drug development process?

“GENE TARGET framework falls into four domains (genetic, preclinical, clinical, ethical.” – Maya Chopra

Source: GENE TARGET: A framework for evaluating Mendelian neurodevelopmental disorders for gene therapy

“Try to make sure every agreement you have has that data sharing provision in it.  This is a long standing problem. Bring your groups together and confront them about it.” – Cara Weismann

Key Takeaways:

  • You’re never done with basic science. As research progresses, organizations often find themselves going back to find answers that only basic science can provide.
  • Balance the needs of patients and families who need the process to move quickly with the researchers’ desire to have the best results before moving forward.
  • Don’t depend on a few scientists. If they are older, they will eventually leave. Cultivate relationships with younger people by offering seed grants for post-docs.

Yssa DeWoody, PhD
Cofounder, Director of Research, and Treasurer, Ring14 USA


Maya Chopra, MBBS, FRACP
Director, Translational Genomic Medicine, Boston Children’s Hospital

Eric Marsh, M.D., Ph.D.
Clinical Director, Orphan Disease Center, University of Pennsylvania

Rodney Samaco, Ph.D.
Assistant Professor, Baylor College of Medicine

Cara Weismann, Ph.D.
Director of the MPS Program of Excellence, Orphan Disease Center, University of Pennsylvania

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