Drug Delivery to the Brain: Emerging Modalities (Q4)
February 17-20, 2025
| Keystone Resort, Keystone, CO, United States
James Gorman, Stina Syvänen and Danica Stanimirovic (in memoriam)
Scholarship Deadline: Nov. 27, 2024 | Abstract Deadline: Jan. 28, 2025 | Early Registration Deadline: Dec. 24, 2024
Monday, February 17, 2025
| 4:00–8:00 PM |
Registration |
Longs Peak Foyer |
| 6:00–8:00 PM |
Welcome Mixer |
Longs Peak Foyer |
Tuesday, February 18, 2025
| 7:00–8:00 AM |
Breakfast |
Shavano/Torreys/Red Cloud |
| 8:00–8:15 AM |
Welcome Remarks |
Longs Peak |
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* Stina Syvänen, Uppsala University
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* James Gorman, Wyss Institute
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| 8:15–9:15 AM |
Keynote Address |
Longs Peak |
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Robert G Thorne, Denali Therapeutics / University of Minnesota
Delivery of Biologics to the CNS via Different Routes of Administration: An Overview of Advantages, Disadvantages, and Applications |
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| 9:15–11:30 AM |
Advances in RMT Delivery Targets and Modalities |
Longs Peak |
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* Meritxell Teixidó, Gate2Brain S.L.
Gate2Brain Shuttles, going Beyond the Transport of Small Molecules |
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James Gorman, Wyss Institute
A Platform of Distinct Brain Shuttles Binding Known and Novel Targets |
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Benjamin A Smith, Biogen
Building a Toolbox for Efficacious and Safe Delivery of a Variety of Biologics and Oligonucleotides to the CNS |
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Kylie Chew, Denali Therapeutics
Short Talk: Dual Targeting of Transferrin Receptor and Cd98hc for Enhanced Brain Exposure of Large Molecules |
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| 9:45–10:05 AM |
Coffee Break |
Longs Peak Foyer |
| 11:15–1:00 PM |
Poster Setup |
Shavano/Torreys/Red Cloud |
| 11:15–5:00 PM |
On Own for Lunch |
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| 1:00–10:00 PM |
Poster Viewing |
Shavano/Torreys/Red Cloud |
| 3:30–4:30 PM |
Career Roundtable (Joint) |
Grays Peak |
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Luka Kulic, F. Hoffmann-La Roche Ltd.
Therapeutic Area Leader Dementias, Expert Scientist and Medical Director |
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Kylie Chew, Denali Therapeutics
Principal Scientist |
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Russell K Pachynski, Washington University in St. Louis
Associate Professor |
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Roosmarijn E Vandenbroucke, VIB and Ghent University
Group Leader & Professor |
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| 4:30–5:00 PM |
Coffee Available |
Longs Peak Foyer |
| 5:00–7:00 PM |
Advances in Brain Delivery of Gene Therapies With Viral Vectors |
Longs Peak |
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* Mathieu E Nonnenmacher, Voyager Therapeutics
BBB Receptors for Enhanced Delivery of AAV to the CNS |
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Qin Huang, Broad Institute of MIT and Harvard
An AAV Capsid Reprogrammed to Bind Human Transferrin Receptor Mediates Brain-wide Gene Delivery |
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Beverly L. Davidson, Children’s Hospital of Philadelphia and University of Pennsylvania
Selection of Delivery Approach in Gene Therapy for AD, LSD, HD and other CNS Diseases |
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Mansuo Shannon, AskBio
Short Talk: Identifying Bbb-Crossing Capsids with a Multiplexed In Vivo and In Vitro Screening Approach Across Species |
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Maria Praggastis, Regeneron Pharmaceuticals
Short Talk: A Durable, Bbb-Crossing Gene Therapy Corrects Cns and Visceral Pathologies in Acid Sphingomyelinase Deficiency Model Mice |
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| 7:00–8:00 PM |
Cocktail Hour Sponsored by Nicoya |
Shavano/Torreys/Red Cloud |
| 7:05–8:05 PM |
Dinner |
Shavano/Torreys/Red Cloud |
| 7:30–10:00 PM |
Poster Session 1 |
Shavano/Torreys/Red Cloud |
Wednesday, February 19, 2025
| 7:00–8:00 AM |
Breakfast |
Shavano/Torreys/Red Cloud |
| 8:00–11:00 AM |
Modeling for Translation: In vitro to in vivo |
Longs Peak |
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* Anna Jezierski, National Research Council Canada & University of Ottawa
On-Chip and Bioprinted BBB Models for Evaluation of Protein and Cell-based Therapeutics |
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Stina Syvänen, Uppsala University
Linking Brain PK and PD Using in vivo Imaging |
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Fei Hua, Certara
QSP Model to Support the Development of Novel Modalities for Drug Delivery to the Brain |
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Alice Stanton, MIT
3D Multicellular Brain-on-Chip for Modeling Delivery to Brain in Neurological Disease |
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Elvis Cuevas, UTMB-Galveston Campus
Short Talk: 17β-Estradiol Prevents The Neurotoxicity Of Amyloid-Beta Oligomers By Improving Blood-Brain Barrier Function |
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| 9:00–9:20 AM |
Coffee Break |
Longs Peak Foyer |
| 11:00–5:00 PM |
On Own for Lunch |
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| 2:30–4:30 PM |
Symposia Spotlight 1: Emerging Principles of Anti-TfR Shuttle Engineering |
Longs Peak |
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* Joy Yu Zuchero, Denali Therapeutics Inc
Session Chair |
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* James Gorman, Wyss Institute
Session Chair |
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Gillian Bonvicini, BioArctic AB
The Effects Of Dose, Valency, And Affinity On Tfr-Mediated Brain Delivery In Vivo |
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Colin F Greineder, University of Michigan
Repeated Dosing And Parenchymal Target Engagement Differentially Impact Brain Pharmacokinetics Of Cd98hc And Transferrin Receptor Shuttles |
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Yuki Noguchi, Chugai Pharmaceutical CO., LTD.
Novel Antibody Technology Enabling Efficient Blood-Brain Barrier Penetration And Long-Term Retention In The Brain |
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Michelle Pizzo, Denali Therapeutics
Engineering Anti-Amyloid Antibodies With Transferrin Receptor Targeting Improves Brain Biodistribution And Mitigates Aria |
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Damian G Wheeler, Translucence Biosystems, Inc
Brain-Wide Parenchymal Quantification Of BBB-Crossing Antibody Therapeutics |
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Ayman Ismail, Sanofi
Conserved pH-dependent Epitope on TfR1 Drives Brain Delivery of High Affinity anti-TfR1 NANOBODIES® |
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Liqun Wang, Wyss Institute at Harvard University
Antibody Brain Shuttle Platform for Drug Delivery to Brain |
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| 4:30–5:00 PM |
Coffee Available |
Longs Peak Foyer |
| 5:00–7:00 PM |
Nanoparticle and Cell-Based Platforms for Cross-BBB Delivery |
Longs Peak |
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* Derrick Gibbings, University of Ottawa
Extracellular Vesicle Trafficking and Therapeutic Delivery into the CNS |
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Samir Mitragotri, Harvard University
A Back-Pack Based Myeloid Cell Therapy for Multiple Sclerosis |
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Michael Mitchell, University of Pennsylvania
Predictive High-Throughput Platform for Dual Screening of mRNA Lipid Nanoparticle Blood−Brain Barrier Transfection and Crossing |
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Oscar A Marcos-Contreras, University of Pennsylvania
Short Talk: Redefining The Blood-Brain Barrier: From Obstacle To Opportunity With Vcam-Targeted Nanocarriers In Neuroinflammatory Diseases |
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Neeraja Revi, University of Illinois Chicago
Short Talk: Bbb Targeting Nanocarrier For Anti-Depressant Effects |
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| 7:00–8:00 PM |
Social Hour with Lite Bites |
Shavano/Torreys/Red Cloud |
| 7:30–10:00 PM |
Poster Session 2 |
Shavano/Torreys/Red Cloud |
Thursday, February 20, 2025
| 7:00–8:00 AM |
Breakfast |
Shavano/Torreys/Red Cloud |
| 8:00–11:00 AM |
Translational Studies and Representative Clinical Trials |
Longs Peak |
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* Luka Kulic, F. Hoffmann-La Roche Ltd.
Clinical Updates from the Brainshuttle AD study of trontinemab in people with Alzheimer's disease |
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Mathias Schmidt, JCR Pharma
Clinical Advancement and Measuring Efficacy of Biologics Crossing the Blood-Brain-Barrier |
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Bryan Laffitte, Novartis Institutes of Biomedical Research
AAV9 Vectorized Gene Replacement Therapy for SMA |
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Haiyan Peng, Alnylam Pharmaceuticals
The Translational Path of the ALN-APP From Mouse to NHP To Human Clinical Trials |
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Pawel Stocki, Ossianix
Short Talk: Novel TXP1 Brain Shuttle: Transforming the Treatment of CNS Diseases |
|
| 9:00–9:20 AM |
Coffee Break |
Longs Peak Foyer |
| 11:00–5:00 PM |
On Own for Lunch |
|
| 2:30–4:30 PM |
Symposia Spotlight 2: Emerging Modalities and Future Opportunities in Targeted Brain Delivery |
Longs Peak |
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* Forest H Andrews, Eli Lilly & Company
Session Chair |
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* Stina Syvänen, Uppsala University
Session Chair |
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Padma Akkapeddi, Denali Therapeutics
Targeting Transferrin Receptor to Transport Antisense Oligonucleotides Across the Blood-Brain Barrier |
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Urs Langen, Roche Innovation Center Basel
Beyond Antibody Delivery: Expanding the Cargo Space of the Brainshuttle™ |
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Sungwon An, ABL Bio Inc
Grabody B, an IGF1R-mediated BBB Shuttle, Increases Brain Penetration of Therapeutic Antibody by Novel Transcytosis Pathways |
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Kimberly Scearce-Levie, Manifold Bio
Rapid Discovery and Development of Novel Brain Shuttles for Multiple “Portals” Using Multiplexed In Vivo Screening |
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Yunxuan Xie, University of Michigan
Neuron-targeted Bispecific Antibody Brain Shuttles |
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Shuqi Yun, Mass General Hospital
Targeted CNS Delivery of CRISPR-Base Editors as a Novel Treatment for Familial Dysautonomia |
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Julien Spatazza, coave therapeutics
The Advanced Ligand Conjugation (ALIGATER™) Platform Chemically Redirects AAV Vectors Towards TfR1 Receptor |
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| 4:30–5:00 PM |
Coffee Available |
Longs Peak Foyer |
| 5:00–7:00 PM |
‘Tailoring’ Delivery to Disease Pathophysiology |
Longs Peak |
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* Nazira El-Hage, Florida International University
Macrophage-derived Extracellular Vesicles as Nanocarriers for Drug Delivery to the Brain |
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Joy Yu Zuchero, Denali Therapeutics Inc
Transport Vehicle: Utilizing the Brain Vasculature to Delivery CNS Therapeutics |
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Praveen Raju, Rady Children’s Hospital-San Diego
Enhancing Drug Delivery Past the Blood-Brain Barrier Through Activated Endothelium |
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Wade Yun-Huai Kuo, University of Michigan
Short Talk: Efficient And Ultra-Long-Lived Delivery Of Cytokines To The Central Nervous System |
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Peisheng Xu, University of South Carolina
Short Talk: Brain-targeted Nano-ERASER downregulates PD-L1 for Alzheimer’s disease therapy |
|
| 7:00–7:15 PM |
Junior Investigator Awards |
Longs Peak |
| 7:15–7:20 PM |
Meeting Wrap-Up: Outcomes and Future Directions (Organizers) |
Longs Peak |
| 7:15–8:15 PM |
Social Hour with Lite Bites |
Shavano/Torreys/Red Cloud |
| 8:00–9:00 PM |
Entertainment |
Longs Peak |
| 8:00–9:00 PM |
Cash Bar |
Shavano/Torreys/Red Cloud |
Friday, February 21, 2025
