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


PARKINSON'S

PANCREATIC CANCER

SPINAL MUSCULAR ATROPHY
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
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
