On 26 June 2018, the drug development company Skyhawk Therapeutics entered a global five-year partnership deal with the big pharma company Celgene. The strategic collaboration details discovery, co-development, and commercialisation of small innovative therapies for neurology patients, which would be based on Skyhawk ’s innovative STAR technology platform.

The deal allows Celgene the exclusive option to in-license worldwide intellectual property associated with therapeutics for up to five programmes, which would potentially be used for treating amyotrophic lateral sclerosis (ALS), Huntington’s Disease, and other neurological disorders. Skyhawk has also announced it closed a seed round of investment that included investors such as Alexandria Venture Investments and The Duke of Bedford , and new investments by GreatPoint Ventures and Celgene Corporation.

The mechanism of action for Skyhawk’s STAR platform involves ‘exon skipping’, the mis-splicing of RNA that removes specific regions of the RNA during its splicing process. Skyhawk’s aim is to develop small molecules that target specific binding regions on the RNA, using both sequence and structural specificity during particular moments of the RNA splicing process. This approach can potentially reverse the mis-splicing, thereby treating the disease.

Skyhawk’s investment round and collaboration with Celgene has left the company in a good position to capitalize on further development of small molecules that amend RNA expression. Likewise, this collaboration with Skyhawk allows Celgene to continue pursuing novel treatments for patients suffering from neurological diseases.