New White Paper on Advanced Therapies Sets up Developers for Success, Published by Xtalks

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A new white paper on development of advanced therapies for rare diseases is available for free download. This authoritative 20-page publication sets up innovators for success with an up-to-the-minute review of current challenges (including COVID-19), best practices and hands-on wisdom from industry leaders.

Xtalks Life Science Webinars

Gene-altering and other advanced medicines are radically rewriting the future for people with some of the rarest, and most devastating, diseases on earth. However, these complex treatments present challenges over and above the well-documented hurdles of rare-disease research.

This new white paper, published by Xtalks in collaboration with experts at Medpace, Inc., skillfully weaves industry hands-on knowledge with the academic literature. It presents hard-won lessons learned and best practices surrounding the unique realities of advanced-therapy development. Industry experts and a patient advocate share insiders’ gems with readers, backed up by the latest data, case studies and published commentary from regulators including the U.S. Food and Drug Administration (FDA).

The White Paper boosts developers’ knowledge of:

  • The current industry landscape
  • The added challenges of advanced therapies in rare-disease clinical trials
  • Setting up for success through
  •     o Collaboration
  •     o Relevant and simple endpoints
  •     o Integrating patients into all trial functions
  •     o Partnering with regulators early
  •     o Planning for site variability and long follow up
  •     o Adjusting for COVID-19

“These trials are inspiring and – if this is the right word – intimate. The community is so small and the number of patients in the world is so tiny, that there’s a sense of unity for everyone involved; they’re exciting for the science behind them and the potential benefits for these patients and the sites are enthused to participate.”

For more information on the Xtalks White Paper, visit The Intersection of Rare Disease and Advanced Therapies: What it Means for Clinical Development.


Xtalks, powered by Honeycomb Worldwide Inc., is a leading provider of educational webinars to the global life science, food and medical device community. Every year thousands of industry practitioners (from life science, food and medical device companies, private & academic research institutions, healthcare centers, etc.) turn to Xtalks for access to quality content. Xtalks helps Life Science professionals stay current with industry developments, trends and regulations. Xtalks webinars also provide perspectives on key issues from top industry thought leaders and service providers.

To learn more about Xtalks visit
For information about hosting a webinar visit

For information about publishing an Xtalks white paper, contact John Hughes at +1 (416) 977-6555 x 228 or

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Sydney Perelmutter
+1 (416) 977-6555 x 352
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