5th Annual RNAi-Based Therapeutics Summit
Join WuXi AppTec at the 5th Annual RNAi-Based Therapeutics Summit in Boston. This conference will showcase novel platforms and strategies for targeting beyond the liver to enable the development of RNAi therapeutics to target the undruggable.
We provide a wide range of RNA-related services, comprised of in vitro screening assays, in vivo animal models, and custom synthesis and conjugation.
Our capabilities include:
- Knockdown effect in cell lines and primary cells (mRNA & protein assessment)
- Assessment of cell-based delivery using GalNAc/ASGPR binding & uptake assays
- In vitro toxicity: immune response profiling and cytotoxicity assessment in primary human hepatocytes
- Off-target effects (transcriptome analysis): off-target profiling by RNAseq
- Quantification of siRNA in the RNA-induced silencing complex (RISC)
- In vivo PD studies in HDI, transgenic, and AAV mouse models; confirm cellular location of knockdown
- In vivo PD studies in NHPs, determine duration of knockdown effect
- Synthesis of siRNAs and ASOs
- GalNAc-siRNA conjugation
- Lipid and peptide conjugation