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Main Conference - Day 2 (May 13)
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Main Conference - Day 2 (May 13)
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Delivery of Macromolecules
8:25am - 8:30am
Chairwoman’s Remarks
- Julia Alterman, Ph.D. - Assistant Professor, RNA Therapeutics Institute, University of Massachusetts Chan Medical
8:30am - 9:00am
TfR-mediated Delivery of Oligonucleotides to the Deep Brain Using the VECTrans® Platform: Initial Learnings and Perspectives
- Guillaume Jacquot, PhD - Program Director, Brain and Tissue Delivery of Oligonucleotides, Vect-Horus
9:00am - 9:30am
Brain Delivery of Antibody-oligonucleotide Conjugates for CNS Diseases
- Liqun Wang, PhD - Principal Scientist, Brain Targeting Program, Wyss Institute at Harvard University
9:30am - 10:00am
Leveraging IGF1R Receptor for Antisense Delivery
- Hien Zhao, PhD - Vice President, Neuroscience Research, Ionis Pharmaceuticals
Showing 1 of 1 Streams
Delivery of Macromolecules
10:45am - 11:15am
Beyond the Standard: Advancing TfR-Mediated Delivery of Oligonucleotide Therapeutics to the CNS
- Felix Schumacher - Program Leader Targeted Therapeutics, Roche
11:15am - 11:45am
Unlocking the Therapeutic Potential of Oligonucleotides for CNS Disorders Through Targeted Delivery
- Stefano Zanotti, PhD - SVP, Head of Neuromuscular Research, Dyne Therapeutics
11:45am - 12:15pm
Bridging the Divide: Leveraging the Transport Vehicle Platform to Deliver Enzyme, Antibody, and Oligonucleotide-based Therapeutics across the BBB
- Joe Lewcock, PhD - Chief Scientific Officer, Denali Therapeutics
12:15pm - 12:20pm
Transition to Spotlight Presentation Rooms
Concurrent Tracks
Showing 1 of 1 Streams
Delivery of Macromolecules
1:55pm - 2:00pm
Chairman’s Remarks
- Stephen Spagnol, PhD - Director, Enabling Technologies, Merck
2:00pm - 2:30pm
Selective Organ-Targeting (SORT) LNP Delivery Platform
- Sakya Mohapatra - Director, Formulation Development, Recode Therapeutics
2:30pm - 3:00pm
Targeted Delivery Platform: Leveraging a Barcoded Platform
- Pierce Ogden, PhD - Co-Founder, CTO and Board Director, Manifold Bio
3:00pm - 3:30pm
In Vivo Generation of Functional CAR T Cells via Targeted Non-Viral Lipid Nanoparticles Enables Potent Immune Cell Depletion in Non-Human Primates
We describe a targeted lipid nanoparticle platform enabling in vivo generation of transient CAR T cells to achieve rapid, B cell depletion without lymphodepletion. In non-human primates, delivery of anti-CD20 CAR mRNA resulted in CAR expression on CD8+ T cells and induced profound B cell depletion in blood and lymphoid tissues including spleen, bone marrow and lymph nodes. Demonstrating an excellent safety and tolerability in NHPs, this off-the-shelf candidate allows B cells recovery to occur with predominantly naïve phenotypes, supporting immune reset and translational potential for autoimmune diseases.
- Ali Nahvi, Ph.D - Chief Technology Officer, Nava Therapeutics
Showing 1 of 1 Streams
Delivery of Macromolecules
4:15pm - 4:45pm
Deep Learning-based Design of Cell-targeting Ligands Enables Functional siRNA Delivery in Multiple Extrahepatic Tissues
- Kyler Lugo, Ph.D - Principal Scientist, Nosis Bio
4:45pm - 5:15pm
Systemic RNAi Targeting MAPT: Advancing Tau Suppression Across the CNS with TRiM™ SC
- Kayal Madhivanan, PhD - Senior Scientist, Arrowhead Pharmaceuticals
5:15pm - 5:45pm
Late Breaking Presentation
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