Please join for an Open Scientific Discussion (OSD) hosted by the AAPS Biomarkers and Precision Medicine (BPM) and Gene and Cell Therapeutic Products (GCTPs) Communities on Oct 21st, 12 – 1 pm ET on the following topic:
Navigating the Accelerated Approval Pathway: Strategy and Success
- What is required for drug approval under the accelerated pathway vs. the traditional pathway?
- Can we qualify Biomarkers to support Rare Disease Regulatory Pathways: Case Study of Heparan Sulfate in MPSIII
- Tofersen case study: NfL as a surrogate biomarker for predicting clinical outcome in SOD1-ALS
Discussion Leaders:
- Linda Jeng (CDER, FDA)
- Binodh DeSilva (Ultragenyx)
- Carrie Rubel (Biogen)
Organized by: Lakshmi Amaravadi (Astra Zeneca), Yan Ni (Passage Bio), Karen Quadrini (Prothena Biosciences) and Carmen Fernández-Metzler (PharmaCadence)
We start at 12:00 ET sharp. To avoid interruptions, please dial in before noon and mute yourself until Q&A starts.
(Please remain on mute unless asking questions)
When: Tuesday, Oct 21, 2025, 12 pm – 1 pm ET
Where: Microsoft Teams
Join the meeting now
Meeting ID: 222 924 429 383 2
Passcode: Gy7Fr7o9
Please feel free to share with colleagues who may be interested in this topic.
Thanks,
On Behalf of Leadership Teams of the AAPS BPM and GCTPs Communities
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Karen Quadrini, PhD
Director, Clinical Biomarkers
Prothena Biosciences
Brisbane, CA
[email protected]Disclaimer: Opinions expressed are solely my own and do not express the views or opinions of my employer.
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