Drug Delivery to the Brain: Emerging Modalities
Feb 17–20, 2025 | Keystone Resort, Keystone, CO, United States
Scientific Organizers:
James Gorman, Stina Syvänen, and Danica Stanimirovic (in memoriam)
In Person
On Demand
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Feb 17–20, 2025 | Keystone Resort, Keystone, CO, United States
Scientific Organizers: James Gorman, Stina Syvänen, and Danica Stanimirovic (in memoriam)
James Gorman
Stina Syvänen
Danica Stanimirovic (in memoriam)
Available Formats:
Supported by:
In Person
On Demand
***Meeting program subject to change.
Available Formats: = In Person = On DemandMonday, February 17, 2025
Fundraising
Booking Function
Merchandise Options
Registration Options
Registration
4:00–8:00 PM
Longs Peak Foyer
Welcome Mixer
6:00–8:00 PM
Longs Peak Foyer
Tuesday, February 18, 2025
Breakfast
7:00–8:00 AM
Shavano/Torreys/Red Cloud
Welcome Remarks
8:00–8:15 AM
Longs Peak
* Stina Syvänen, Uppsala University
* James Gorman, Wyss Institute
Keynote Address
8:15–9:15 AM
Longs Peak
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
Delivery of Biologics to the CNS via Different Routes of Administration: An Overview of Advantages, Disadvantages, and Applications
Advances in RMT Delivery Targets and Modalities
9:15–11:30 AM
Longs Peak
* Meritxell Teixidó, Gate2Brain S.L.
Gate2Brain Shuttles, going Beyond the Transport of Small Molecules
Gate2Brain Shuttles, going Beyond the Transport of Small Molecules
James Gorman, Wyss Institute
A Platform of Distinct Brain Shuttles Binding Known and Novel Targets
A Platform of Distinct Brain Shuttles Binding Known and Novel Targets
Benjamin A Smith, Biogen
Building a Toolbox for Efficacious and Safe Delivery of a Variety of Biologics and Oligonucleotides to the CNS
Building a Toolbox for Efficacious and Safe Delivery of a Variety of Biologics and Oligonucleotides to the CNS
Kylie Chew, Denali Therapeutics
Short Talk: Dual Targeting of Transferrin Receptor and Cd98hc for Enhanced Brain Exposure of Large Molecules
Short Talk: Dual Targeting of Transferrin Receptor and Cd98hc for Enhanced Brain Exposure of Large Molecules
Coffee Break
9:45–10:05 AM
Longs Peak Foyer
Poster Setup
11:15–1:00 PM
Shavano/Torreys/Red Cloud
On Own for Lunch
11:15–5:00 PM
Poster Viewing
1:00–10:00 PM
Shavano/Torreys/Red Cloud
Career Roundtable (Joint)
3:30–4:30 PM
Grays Peak
Luka Kulic, F. Hoffmann-La Roche Ltd.
Therapeutic Area Leader Dementias, Expert Scientist and Medical Director
Therapeutic Area Leader Dementias, Expert Scientist and Medical Director
Kylie Chew, Denali Therapeutics
Principal Scientist
Principal Scientist
Russell K Pachynski, Washington University in St. Louis
Associate Professor
Associate Professor
Roosmarijn E Vandenbroucke, VIB and Ghent University
Group Leader & Professor
Group Leader & Professor
Coffee Available
4:30–5:00 PM
Longs Peak Foyer
Advances in Brain Delivery of Gene Therapies With Viral Vectors
5:00–7:00 PM
Longs Peak
* Mathieu E Nonnenmacher, Voyager Therapeutics
BBB Receptors for Enhanced Delivery of AAV to the CNS
BBB Receptors for Enhanced Delivery of AAV to the CNS
Qin Huang, Broad Institute of MIT and Harvard
An AAV Capsid Reprogrammed to Bind Human Transferrin Receptor Mediates Brain-wide Gene Delivery
An AAV Capsid Reprogrammed to Bind Human Transferrin Receptor Mediates Brain-wide Gene Delivery
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
Selection of Delivery Approach in Gene Therapy for AD, LSD, HD and other CNS Diseases
Mansuo Shannon, AskBio
Short Talk: Identifying Bbb-Crossing Capsids with a Multiplexed In Vivo and In Vitro Screening Approach Across Species
Short Talk: Identifying Bbb-Crossing Capsids with a Multiplexed In Vivo and In Vitro Screening Approach Across Species
Maria Praggastis, Regeneron Pharmaceuticals
Short Talk: A Durable, Bbb-Crossing Gene Therapy Corrects Cns and Visceral Pathologies in Acid Sphingomyelinase Deficiency Model Mice
Short Talk: A Durable, Bbb-Crossing Gene Therapy Corrects Cns and Visceral Pathologies in Acid Sphingomyelinase Deficiency Model Mice
Cocktail Hour Sponsored by Nicoya
7:00–8:00 PM
Shavano/Torreys/Red Cloud
Dinner
7:05–8:05 PM
Shavano/Torreys/Red Cloud
Poster Session 1
7:30–10:00 PM
Shavano/Torreys/Red Cloud
Wednesday, February 19, 2025
Breakfast
7:00–8:00 AM
Shavano/Torreys/Red Cloud
Modeling for Translation: In vitro to in vivo
8:00–11:00 AM
Longs Peak
* Anna Jezierski, National Research Council Canada & University of Ottawa
On-Chip and Bioprinted BBB Models for Evaluation of Protein and Cell-based Therapeutics
On-Chip and Bioprinted BBB Models for Evaluation of Protein and Cell-based Therapeutics
Stina Syvänen, Uppsala University
Linking Brain PK and PD Using in vivo Imaging
Linking Brain PK and PD Using in vivo Imaging
Fei Hua, Certara
QSP Model to Support the Development of Novel Modalities for Drug Delivery to the Brain
QSP Model to Support the Development of Novel Modalities for Drug Delivery to the Brain
Alice Stanton, MIT
3D Multicellular Brain-on-Chip for Modeling Delivery to Brain in Neurological Disease
3D Multicellular Brain-on-Chip for Modeling Delivery to Brain in Neurological Disease
Elvis Cuevas, UTMB-Galveston Campus
Short Talk: 17β-Estradiol Prevents The Neurotoxicity Of Amyloid-Beta Oligomers By Improving Blood-Brain Barrier Function
Short Talk: 17β-Estradiol Prevents The Neurotoxicity Of Amyloid-Beta Oligomers By Improving Blood-Brain Barrier Function
Coffee Break
9:00–9:20 AM
Longs Peak Foyer
On Own for Lunch
11:00–5:00 PM
Symposia Spotlight 1: Emerging Principles of Anti-TfR Shuttle Engineering
2:30–4:30 PM
Longs Peak
* Joy Yu Zuchero, Denali Therapeutics Inc
Session Chair
Session Chair
* James Gorman, Wyss Institute
Session Chair
Session Chair
Gillian Bonvicini, BioArctic AB
The Effects Of Dose, Valency, And Affinity On Tfr-Mediated Brain Delivery In Vivo
The Effects Of Dose, Valency, And Affinity On Tfr-Mediated Brain Delivery In Vivo
Colin F Greineder, University of Michigan
Repeated Dosing And Parenchymal Target Engagement Differentially Impact Brain Pharmacokinetics Of Cd98hc And Transferrin Receptor Shuttles
Repeated Dosing And Parenchymal Target Engagement Differentially Impact Brain Pharmacokinetics Of Cd98hc And Transferrin Receptor Shuttles
Yuki Noguchi, Chugai Pharmaceutical CO., LTD.
Novel Antibody Technology Enabling Efficient Blood-Brain Barrier Penetration And Long-Term Retention In The Brain
Novel Antibody Technology Enabling Efficient Blood-Brain Barrier Penetration And Long-Term Retention In The Brain
Michelle Pizzo, Denali Therapeutics
Engineering Anti-Amyloid Antibodies With Transferrin Receptor Targeting Improves Brain Biodistribution And Mitigates Aria
Engineering Anti-Amyloid Antibodies With Transferrin Receptor Targeting Improves Brain Biodistribution And Mitigates Aria
Damian G Wheeler, Translucence Biosystems, Inc
Brain-Wide Parenchymal Quantification Of BBB-Crossing Antibody Therapeutics
Brain-Wide Parenchymal Quantification Of BBB-Crossing Antibody Therapeutics
Ayman Ismail, Sanofi
Conserved pH-dependent Epitope on TfR1 Drives Brain Delivery of High Affinity anti-TfR1 NANOBODIES®
Conserved pH-dependent Epitope on TfR1 Drives Brain Delivery of High Affinity anti-TfR1 NANOBODIES®
Liqun Wang, Wyss Institute at Harvard University
Antibody Brain Shuttle Platform for Drug Delivery to Brain
Antibody Brain Shuttle Platform for Drug Delivery to Brain
Coffee Available
4:30–5:00 PM
Longs Peak Foyer
Nanoparticle and Cell-Based Platforms for Cross-BBB Delivery
5:00–7:00 PM
Longs Peak
* Derrick Gibbings, University of Ottawa
Extracellular Vesicle Trafficking and Therapeutic Delivery into the CNS
Extracellular Vesicle Trafficking and Therapeutic Delivery into the CNS
Samir Mitragotri, Harvard University
A Back-Pack Based Myeloid Cell Therapy for Multiple Sclerosis
A Back-Pack Based Myeloid Cell Therapy for Multiple Sclerosis
Michael Mitchell, University of Pennsylvania
Predictive High-Throughput Platform for Dual Screening of mRNA Lipid Nanoparticle Blood−Brain Barrier Transfection and Crossing
Predictive High-Throughput Platform for Dual Screening of mRNA Lipid Nanoparticle Blood−Brain Barrier Transfection and Crossing
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
Short Talk: Redefining The Blood-Brain Barrier: From Obstacle To Opportunity With Vcam-Targeted Nanocarriers In Neuroinflammatory Diseases
Neeraja Revi, University of Illinois Chicago
Short Talk: Bbb Targeting Nanocarrier For Anti-Depressant Effects
Short Talk: Bbb Targeting Nanocarrier For Anti-Depressant Effects
Social Hour with Lite Bites
7:00–8:00 PM
Shavano/Torreys/Red Cloud
Poster Session 2
7:30–10:00 PM
Shavano/Torreys/Red Cloud
Thursday, February 20, 2025
Breakfast
7:00–8:00 AM
Shavano/Torreys/Red Cloud
Translational Studies and Representative Clinical Trials
8:00–11:00 AM
Longs Peak
* Luka Kulic, F. Hoffmann-La Roche Ltd.
Clinical Updates from the Brainshuttle AD study of trontinemab in people with Alzheimer's disease
Clinical Updates from the Brainshuttle AD study of trontinemab in people with Alzheimer's disease
Mathias Schmidt, JCR Pharma
Clinical Advancement and Measuring Efficacy of Biologics Crossing the Blood-Brain-Barrier
Clinical Advancement and Measuring Efficacy of Biologics Crossing the Blood-Brain-Barrier
Bryan Laffitte, Novartis Institutes of Biomedical Research
AAV9 Vectorized Gene Replacement Therapy for SMA
AAV9 Vectorized Gene Replacement Therapy for SMA
Haiyan Peng, Alnylam Pharmaceuticals
The Translational Path of the ALN-APP From Mouse to NHP To Human Clinical Trials
The Translational Path of the ALN-APP From Mouse to NHP To Human Clinical Trials
Pawel Stocki, Ossianix
Short Talk: Novel TXP1 Brain Shuttle: Transforming the Treatment of CNS Diseases
Short Talk: Novel TXP1 Brain Shuttle: Transforming the Treatment of CNS Diseases
Coffee Break
9:00–9:20 AM
Longs Peak Foyer
On Own for Lunch
11:00–5:00 PM
Symposia Spotlight 2: Emerging Modalities and Future Opportunities in Targeted Brain Delivery
2:30–4:30 PM
Longs Peak
* Forest H Andrews, Eli Lilly & Company
Session Chair
Session Chair
* Stina Syvänen, Uppsala University
Session Chair
Session Chair
Padma Akkapeddi, Denali Therapeutics
Targeting Transferrin Receptor to Transport Antisense Oligonucleotides Across the Blood-Brain Barrier
Targeting Transferrin Receptor to Transport Antisense Oligonucleotides Across the Blood-Brain Barrier
Urs Langen, Roche Innovation Center Basel
Beyond Antibody Delivery: Expanding the Cargo Space of the Brainshuttle™
Beyond Antibody Delivery: Expanding the Cargo Space of the Brainshuttle™
Sungwon An, ABL Bio Inc
Grabody B, an IGF1R-mediated BBB Shuttle, Increases Brain Penetration of Therapeutic Antibody by Novel Transcytosis Pathways
Grabody B, an IGF1R-mediated BBB Shuttle, Increases Brain Penetration of Therapeutic Antibody by Novel Transcytosis Pathways
Kimberly Scearce-Levie, Manifold Bio
Rapid Discovery and Development of Novel Brain Shuttles for Multiple “Portals” Using Multiplexed In Vivo Screening
Rapid Discovery and Development of Novel Brain Shuttles for Multiple “Portals” Using Multiplexed In Vivo Screening
Yunxuan Xie, University of Michigan
Neuron-targeted Bispecific Antibody Brain Shuttles
Neuron-targeted Bispecific Antibody Brain Shuttles
Shuqi Yun, Mass General Hospital
Targeted CNS Delivery of CRISPR-Base Editors as a Novel Treatment for Familial Dysautonomia
Targeted CNS Delivery of CRISPR-Base Editors as a Novel Treatment for Familial Dysautonomia
Julien Spatazza, coave therapeutics
The Advanced Ligand Conjugation (ALIGATER™) Platform Chemically Redirects AAV Vectors Towards TfR1 Receptor
The Advanced Ligand Conjugation (ALIGATER™) Platform Chemically Redirects AAV Vectors Towards TfR1 Receptor
Coffee Available
4:30–5:00 PM
Longs Peak Foyer
‘Tailoring’ Delivery to Disease Pathophysiology
5:00–7:00 PM
Longs Peak
* Nazira El-Hage, Florida International University
Macrophage-derived Extracellular Vesicles as Nanocarriers for Drug Delivery to the Brain
Macrophage-derived Extracellular Vesicles as Nanocarriers for Drug Delivery to the Brain
Joy Yu Zuchero, Denali Therapeutics Inc
Transport Vehicle: Utilizing the Brain Vasculature to Delivery CNS Therapeutics
Transport Vehicle: Utilizing the Brain Vasculature to Delivery CNS Therapeutics
Praveen Raju, Rady Children’s Hospital-San Diego
Enhancing Drug Delivery Past the Blood-Brain Barrier Through Activated Endothelium
Enhancing Drug Delivery Past the Blood-Brain Barrier Through Activated Endothelium
Wade Yun-Huai Kuo, University of Michigan
Short Talk: Efficient And Ultra-Long-Lived Delivery Of Cytokines To The Central Nervous System
Short Talk: Efficient And Ultra-Long-Lived Delivery Of Cytokines To The Central Nervous System
Peisheng Xu, University of South Carolina
Short Talk: Brain-targeted Nano-ERASER downregulates PD-L1 for Alzheimer’s disease therapy
Short Talk: Brain-targeted Nano-ERASER downregulates PD-L1 for Alzheimer’s disease therapy
Junior Investigator Awards
7:00–7:15 PM
Longs Peak
Meeting Wrap-Up: Outcomes and Future Directions (Organizers)
7:15–7:20 PM
Longs Peak
Social Hour with Lite Bites
7:15–8:15 PM
Shavano/Torreys/Red Cloud
Entertainment
8:00–9:00 PM
Longs Peak
Cash Bar
8:00–9:00 PM
Shavano/Torreys/Red Cloud
Friday, February 21, 2025
Departure
12:00–11:59 PM
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