Merck Decision Tree

Merck is a global research-driven pharmaceutical company dedicated to putting patients first. Merck's highest priority areas are Alzheimer’s disease, atherosclerosis, cardiovascular disease, diabetes, novel vaccines, obesity, oncology, pain and sleep disorders. In addition, and importantly for licensing, the following other areas remain of high interest for focused investment in new compounds and mechanisms: antibiotics, antifungals, antivirals (HCV and HIV), asthma, COPD, neurodegeneration, ophthalmology, osteoporosis, schizophrenia, and stroke.

In RNA Therapeutics they are interested in:

siRNA sequence, structure, and modification
Novel chemistries for improving resistance to enzymatic degradation
Novel chemistries for reducing immunostimulation
Enhanced RISC incorporations
Long-term chemical stability
Improved target specificity siRNA delivery vehicles for systemic administration
Novel polymers and lipids for encapsulation
Low toxicity and biodegradable
Amenable to use of process development and manufacturing platforms
Amenable to molecular targeting strategies
Long-term storage stability
Delivery vehicles that improve endosomal escape
Approaches to molecular targeting of siRNA
Targeting using antibodies, peptides, or small molecules
Specific for disease-relevant cell types
Suitable for nanoparticle or liposome delivery
Assays for siRNA intracellular functions
Assays for siRNA endosomal escape
Biochemical assays for strand selection, RISC incorporation, and catalytic efficiency
Assays for clinical biodistribution
Cell-based assays to measure toll receptor activation
Validated siRNA targets
Demonstrated reduction in mRNA and protein levels in animals
Reductions have expected effect on disease phenotype miRNA Novel chemistries to create antagomirs
Demonstrated reduction of miRNA levels in animals, with evidence that reductions have expected effects on mRNA levels and/or disease phenotype
Demonstrated