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

*

Our Platform

SkySTAR  Skyhawk Small molecule Therapeutics for Alternative splicing RNA

SPINAL MUSCULAR ATROPHY

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

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                                    

Skyhawk Therapeutics, Inc.

35 Gatehouse Drive

 

Waltham, MA 02451

United States of America

 

info@skyhawktx.com

 

Tel: 617.858.0041

Skyhawk Therapeutics Europe GmbH

 

Basel Technology Park

Hochbergerstrasse 60C

Basel 4057

Switzerland

 swiss-info@skyhawktx.com

Tel: +41 (0) 61 638 23 50

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SKYHAWK® and SKYSTAR® are registered trademarks of Skyhawk Therapeutics, Inc. in the US and other countries.