Main Conference - Day 2 (May 13)
- Julia Alterman, Ph.D. - Assistant Professor, RNA Therapeutics Institute, University of Massachusetts Chan Medical
- Guillaume Jacquot, PhD - Program Director, Brain and Tissue Delivery of Oligonucleotides, Vect-Horus
- Liqun Wang, PhD - Principal Scientist, Brain Targeting Program, Wyss Institute at Harvard University
- Hien Zhao, PhD - Vice President, Neuroscience Research, Ionis Pharmaceuticals
- Felix Schumacher - Program Leader Targeted Therapeutics, Roche
- Stefano Zanotti, PhD - SVP, Head of Neuromuscular Research, Dyne Therapeutics
- Joe Lewcock, PhD - Chief Scientific Officer, Denali Therapeutics
- Stephen Spagnol, PhD - Director, Enabling Technologies, Merck
- Sakya Mohapatra - Director, Formulation Development, Recode Therapeutics
Current oligonucleotide delivery technologies are not optimized for diverse tissues and therapeutic product profiles. Using multiplexed in vivo screening, we tested thousands of nanobody candidates against hundreds of novel receptor "portals" in mice and non-human primates. These data reveal diverse biodistribution profiles across brain and peripheral tissues, identifying new conjugate partners for improved oligonucleotide delivery.
- Pierce Ogden, PhD - Co-Founder, CTO and Board Director, Manifold Bio
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
- Kyler Lugo, Ph.D - Principal Scientist, Nosis Bio
- Kayal Madhivanan, PhD - Senior Scientist, Arrowhead Pharmaceuticals
- Sean Kevlahan, PhD - Co-founder and CEO, Nanite