New Technologies in Early Stage 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 discussed using new technologies in early stage research.

Find out how emerging technologies may be used for drug discovery, design and repurposing, transforming an often complex, decades-long mission into a more efficient process, reducing the timeline and cost to bring therapies to patients.

Notable quote from this session:

Find the people that have already been on the path that you have, learn from their mistakes or take a path they chose not to – don’t be afraid to collaborate or ask questions. The rare disease community is good at sharing, and wants to help you get to a solution faster. – Bruce Bloom

Key Takeaways:

  • Understanding the biology of the disease will help you understand which technologies are appropriate
  • Find the people that have done what you want to do and learn from their mistakes. Ask questions. The rare community is good at sharing and will want to help you.
  • Partner with organizations like EveryCure, ODC and Global Genes to help you build your roadmap

David Fajgenbaum, MD, MBA, MSc
Assistant Professor, Perelman School of Medicine, University of Pennsylvania

Bruce Bloom
Chief Collaboration Officer, Helix
Chief Science Officer, Kabuki Syndrome Foundation

Daniel Fischer, MS, MBA
Co-Founder, President and CEO, Board Member, Tevard Biosciences

Maureen Hart, PhD
Director of Patient Advocacy, Policy, and External Engagement, Creyon Bio

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