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Symposium
[Free Online Symposium] Gene Therapy: Discovery, Development and Regulatory Perspectives
Speakers:
Mark Milton (ex Novartis), Dhaval Shah (SUNY Buffalo), Brian Long (Biomarin), Abigail Johnson (FDA), Emmanuel Gyamfi (FDA)
Organizers:
Shradda Sadekar (Genentech), Nagendra Chemuturi (Moderna), Dale Miles (Genentech), Snow Ge (BridgeBio)
Date:
2022-12-07- 12/7/2022
Time:
8:30-12:30 Pacific Time
Registration fee:
0
Location:
Online via Zoom
Major Sponsor:
Vendor show vendors registered to date:
Registration: http://www.PBSS.org
Registration deadline:2022-12-04
(it will close sooner if the seating cap is reached)
About the Topic
Objectives of the Symposium:
- Gain an understanding of the different MOAs and durable pharmacology enabled by gene therapy, state of the field, and future outlook
- Appreciate unique challenges to gene delivery, PKPD translation, immunogenicity management, safety, and manufacturing of gene therapies
- Gain regulatory and preclinical and clinical development perspectives for effective bench-bedside development of gene therapies
Time (PST)
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Time (EST)
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Topic
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Presenter
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Moderator
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8:30 - 8:35 am
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11:30 - 11:35 am
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PBSS Introduction/Welcome Remarks
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Snow Ge, BridgeBio
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Enas Mahmoud (PBSS)
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8:35 - 8:40 am
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11:35 - 11:40 am
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Symposium Introduction
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Shradda Sadekar, Genentech
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Snow Ge (BridgeBio)
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8:40 - 9:20 am
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11:40 - 12:20 pm
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In vivo AAV-based gene therapy- a brief primer
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Mark Milton, PhD, Principal, Lake Boon Pharmaceutical Consulting, LLC
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Shradda Sadekar (Genentech)
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9:20 - 10:00 am
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12:20 - 1:00 pm
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Towards Development of a Platform PBPK Model for AAV Mediated Delivery of MAb Genes
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Dhaval Shah, Associate Professor, PhD, MS, B.Pharm., SUNY Buffalo
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Shradda Sadekar (Genentech)
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10:00 - 10:40 am
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1:00 - 1:40 pm
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Immunogenicity Monitoring for AAV-Based Gene Therapies
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Brian Long, PhD, Principal Scientist, Clinical Immunology at BioMarin Pharmaceutical Inc.
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Shradda Sadekar (Genentech)
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10:40 - 10:50 am
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1:40 - 1:50 pm
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QA Round 1 (Mark Milton, Dhaval Shah, Brian Long)
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Shradda Sadekar (Genentech)
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10:50 - 11:00 am
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1:50 - 2:00 pm
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Break
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Dale Miles (Genentech)
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11:00 - 11:40 am
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2:00 - 2:40 pm
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Gene Therapy Products in Oncology: Clinical and Regulatory Perspective
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Abigail Johnson, RN, BSN, Clinical Reviewer/Analyst, FDA
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Dale Miles (Genentech)
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11:40 -12:20 pm
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2:40 - 3:20 pm
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Gene Therapy Product Chemistry, Manufacturing and Controls: Regulatory Perspective
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Emmanuel Adu-Gyamfi, PhD, CMC Reviewer (Gene and Cell Therapy), FDA
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Dale Miles (Genentech)
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12:20 -12:30 pm
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3:20 - 3:30 pm
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QA Round 2 (Abigail Johnson, Emmanuel Adu-Gyamfi)
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Dale Miles (Genentech)
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Note: This is an online meeting, The Zoom link will be sent to the registrants 1+ days before the event.
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