Yes, the Hot Topic Session on Biomarkers and Accelerated Approval for Advanced Therapy, participated by Dr. Kakkis (CEO or Ultragenyx) and Dr. Celia Witten (FDA CBER, Deputy Director) was standing room only! GCT development for rare diseases faces many challenges, but also presents a great opportunity for changing the current 'feels, functions, survivals' based drug approval paradigm and leveraging disease-cause biomarkers towards an accelerated process. Is your company embarking on this journey? What challenges have you faced? Shall we discuss it in our #OpenScientificDiscussions forum?
The GCTPs community also had a very productive membership meeting at NBC. Many participants have made good suggestions for future efforts. You can find information on our sub-teams and current efforts on the attached slide deck. Please reach out to either Yan Ni ([email protected]), @Hardik Mody ([email protected]) if you are interested in joining one of our sub-teams.
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Yan Ni Ph.D.
Executive Director
Passage Bio
Philadelphia PA
[email protected]Disclaimer: Opinions expressed are solely my own and do not express the views or opinions of my employer.
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Original Message:
Sent: 05-30-2024 13:10
From: Amanda Hays
Subject: Post NBC Discussion
We're just a few weeks from concluding the AAPS NBC meeting in San Francisco. It was great to catch up and see everyone in person. We had a great discussion at our Biomarkers and Precision Medicine Community meeting there and heard some great feedback on potential topics we can showcase this year. If you were not able to attend, I attached the slide deck that our Vice Chair @Karen Quadrini presented at the meeting. It was great to see all the accomplishments and efforts underway from this group.
Another thing to keep on the lookout for is the NBC recordings, once those are released. It is a great way of catching up with any sessions that were missed. One that I will be re-watching is the keynote presentation from Emil Kakkis, CEO and President at Ultragenyx. Dr. Kakkis presented on using primary disease activity biomarkers as a driving force in accelerating rare and ultra rare disease drug development. There was a Hot Topic later that day hosted by @Yan Ni where Dr. Kakkis and Dr. Celia Witten, FDA Deputy Director, discussed biomarkers and accelerated approval for advanced therapy. Both were great sessions and definitely worth the listen!
If you are interested in joining our monthly meetings and getting more involved, just drop us a message here!
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Amanda Hays Ph.D.
Scientific Officer
BioAgilytix
Overland Park KS
[email protected]
Disclaimer: Opinions expressed are solely my own and do not express the views or opinions of my employer.
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