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Technology Platform – Drug Delivery
Novel polymers and lipids for encapsulation that exhibit
Low toxicity
Biodegradability
Efficient cellular uptake
Efficient endosomal escape
Molecular Targeting
Targeting agents (antibodies, peptides, or small molecules) that are suitable for direct siRNA conjugation or for nanoparticle/liposome delivery
Local Delivery
Systems exhibiting direct siRNA delivery to eye, lung, CNS, and muscle tissue
Assays for siRNA and Delivery Vehicles
Techniques for tethered polymer complex evaluation (quantitative and qualitative)
Biochemical assays for strand selection, RISC incorporation, and catalytic efficiency
Cell-based assays to measure Toll Like receptor binding and activation
Medium and high throughput screening techniques for siRNA quantitation
Techniques for evaluation of siRNA trafficking, endosomal escape and distribution (high and medium resolution approaches)
Assays for clinical biodistribution
Assays for pharmacodynamic evaluation of miRNA activity
Molecular modeling tools for oligonucleotides and for lipid/polymer delivery systems
Delivery Vehicle Screening Strategies
Microassembly techniques
High-throughput in vivo screening technologies for determining mRNA silencing
Oligonucleotide Optimization
Novel chemistries for siRNA Sequence, Structure, and Modification
Improving resistance to enzymatic degradation
Reducing immunostimulation
Enhancing Ago2/RISC incorporation and potency
Increasing chemical stability>
Improving target specificity
miRNA
Novel chemistries to create miRNA mimics and/or antagonists
Therapeutic agents that
Demonstrate reduction of miRNA levels in animals, with evidence that reductions have expected effects on mRNA levels and/or disease phenotype
Demonstrate delivery of miRNA in animals, with evidence that increases in miRNA have expected effects on mRNA levels and/or disease phenotype
RNA Manufacturing
Advancements in large-scale production of modified siRNA
Improved processes for increased quality, efficiency, and reduced COG
Novel chemistries
Universal linker-based solid support for production of siRNAs
Robust LC-MS method for determination of impurity profile of phosphoramidite raw materials and oligonucleotides (siRNAs)
Alternative methods to produce siRNA clinical supplies
Not interested in
Plasmid DNA-based methods for RNA therapies
Viral delivery methods for RNA therapies
Aptamers as therapeutics
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