Our Platform
Skyhawk Small molecule Therapeutics for Alternative modulation of RNA
THE CHALLENGE
RNA mis-expression has been found to cause a growing list of diseases, from orphan and neurological conditions to major cancers. Very few treatments are available for these challenging conditions, many of which have been considered “undruggable.”
MEDULLOBLASTOMA
ALS
FRONTOTEMPORAL DEMENTIA
SPINAL MUSCULAR ATROPHY
PANCREATIC CANCER
PARKINSON'S
THE OPPORTUNITY
Skyhawk’s novel approach corrects the underlying genetics of disease at the RNA level. Our drugs can modify expression of target genes previously considered undruggable for a variety of diseases such as cancer, neurogenerative, autoimmune, inflammatory, and infectious diseases.
OUR PLATFORM
Our SkySTAR* (Skyhawk Small molecule Therapeutics for Alternative modulation of RNA)
platform integrates information from computational, kinetic, & structural models of RNA
COMPUTATIONAL MODELS
KINETIC MODELS
CONFORMATIONAL STRUCTURAL MODELS
Generating unique and selective families of chemistry for each target
SKYHAWK LIBRARY 1
SKYHAWK LIBRARY 2
SKYHAWK LIBRARY 3
SKYHAWK LIBRARY 4
Our focused libraries are then tested in patient cells using proprietary assays, to quickly yield potent drug candidates