Conference Agenda (Updated 2/20/2024)
| SESSION | TOPIC | SPEAKER | COMPANY | TIME |
| Registration and Breakfast | 7:30-8:30 | |||
| Conference Opening | Dr. Akintunde Bello, Senior Vice President Clinical Pharmacology | BMS | 8:30-8:40 | |
| Opening Plenary Lecture | Past, Present, and Future of Gene Therapy | Mark Milton | Retired (Novartis) | 8:40-9:20 |
Session I: Current State-of-the-Art Gene Delivery
Moderators: Vibha Jawa, BMS & Steven Louie, Moderna
| SESSION | TOPIC | SPEAKER | COMPANY | TIME |
| Session Introduction | 9:20-9:25 | |||
| Development of Viral Delivery for Precision Medicine | R. Jude Samulski | UNC School of Medicine | 9:25-9:50 | |
| ADME for In Vivo Genome Editing: Assessing Effectiveness of Delivery for Editing Cargo | Everett Perkins | Eli Lilly | 9:50-10:15 | |
| AM Break | 10:15-10:35 | |||
| Role of DMPK in non-viral GT | Nagendra Chemuturi | Eli Lilly | 10:35-11:00 | |
| Assessment of ADME and DDI for LNP-mRNA Therapeutics | Lei Ci | Moderna | 11:00-11:25 | |
Session II: Translational PK/PD and Clinical Pharmacology Aspects
Session Chairs: Hardik Mody, Genentech, Dale Miles, Genentech & Stephanie Pasas-Farmer, BioData Solutions
| SESSION | TOPIC | SPEAKER | COMPANY | TIME |
| Session Introduction | 11:25-11:30 | |||
| Gene Therapy and Translational Sciences - How are we doing? | Nicholas Buss | Eli Lilly | 11:30-11:55 | |
| Preclinical considerations for development of GTx | Glen Banks | BMS | 11:55-12:20 | |
| Lunch | 12:20-1:35 | |||
| Construction of an Integrated Translational PBPK-QSP Modeling Platform to Investigate Tissue Distribution and Protein Expression Dynamics of LNP-mRNA Based Therapeutics | Kenji Miyazawa | Moderna | 1:35-2:00 | |
| Development of AAV-Based Retina Gene Therapy through the Eyes of the Clinical Pharmacologist | Jennifer Ford | Janssen | 2:00-2:25 | |
| Vendor Talk - Troubleshooting in Flow Cytometry Method Development: Ions, Titers, and Bears (Oh My!) | Brian Wile |  |
2:25-2:35 | |
| PM Break | 2:35-2:55 |
Session III: Biomarkers and Clinical Translation Aspects of Gene Therapy
Moderators: Yan Ni, Passage Bio & Anshul Gupta, Generation Bio
| SESSION | TOPIC | SPEAKER | COMPANY | TIME |
| Session Introduction | 2:55-3:00 | |||
| Leveraging Clinical Diagnostic Assays for Rare Disease Drug Development | Yan Ni | Passage Bio | 3:00-3:25 | |
| Learnings from Duchenne Muscular Dystrophy: A Biomarker Perspective | Jyoti Malhotra | Sarepta Therapeutics | 3:25-3:50 | |
| Pre-existing antibody assays for patient enrollment and Cdx - Title TBA | Jennifer Jones | Spark Therapeutics | 3:50-4:15 | |
| Can the Immunogenicity of AAV Vectors be Mitigated? | Ronit Mazor | FDA | 4:15-4:40 | |
| Closing Remarks | 4:40-4:45 | |||
| Reception | 4:45-5:45 |