Our Platform

Skyhawk Small molecule Therapeutics for Alternative splicing RNA

THE CHALLENGE

RNA mis-splicing leading to loss of RNA 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 mRNA 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 splicing in 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

PROPRIETARY ASSAYS IN PATIENT CELL LINES

PROPRIETARY SPLICING MODELS

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