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Targeting the Immune System to Treat Rare Disease

May 2, 2020




Paula Ragan, the CEO of X4 Pharmaceuticals, a late-stage clinical biopharmaceutical company and a leader in the discovery and development of novel therapies with a focus on rare disease joins eHealth Radio and the Health News and Pharmaceutical Channels.

Listen to interview with host Eric Michaels & guest Paula Ragan discuss the following:

  1. Can you tell us about your background? Where you're from, how you came to be interested in rare disease?
  2. What is the science behind CXCR4 and what role does it play in the functioning of the immune system?
  3. What is mavorixafor, what is its origin and how does it work? What clinical studies are your currently in?
  4. What is WHIM syndrome? How is someone generally diagnosed with the condition and how does your treatment help?
  5. You are also looking at mavorixafor as a treatment for certain cancers. What happens to the immune systems in these cancers and what is mavorixafor expected to do?
  6. Can you tell me about your current partnerships and collaborations?

Dr. Ragan has been X4’s President and Chief Executive Officer and a member of the X4 Board of Directors since July 2014. She has more than 18 years of experience building companies in the biotechnology industry.

Prior to founding X4, Dr. Ragan consulted as Chief Business Officer at Lysosomal Therapeutics Inc., a private biopharmaceutical company, where she led the company’s business development activities.

Prior to that, Dr. Ragan held leadership roles in corporate development and operations at Genzyme Corporation, a Sanofi company, where she led strategic partnering efforts for Genzyme’s Rare Disease business and headed the supply chain planning for Genzyme’s flagship commercial products.

Other professional roles include business roles at Hydra Biosciences, Oscient Corporation and Celera Corporation. Dr. Ragan received her B.S. from Tufts University and her Ph.D. from Massachusetts Institute of Technology and completed post-doctoral studies at Harvard Medical School.





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