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

*

Our Platform

SkySTAR  Skyhawk Small molecule Therapeutics for Alternative splicing RNA

THE OPPORTUNITY

Skyhawk’s novel approach corrects the underlying genetics of disease at the mRNA level, in the nucleus of the cell.  Our drugs can target major neurological conditions, targets previously considered undruggable, such as master transcriptional factors in cancer and a host of targets in immunology, inflammation and infectious diseases.

Skyhawk small molecules

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

OUR PIPELINE

Our current pipeline includes oncology and neurology targets.

Preclinical                       Lead Selection                           File IND

Skyhawk Oncology Target 1  

Skyhawk Neuro Target 1                                  

Skyhawk Neuro Target 2                                    

Skyhawk Neuro Target 3