Skyhawk Therapeutics announced a global strategic collaboration with a Celgene Corporation affiliate to discover, develop and commercialize innovative small molecule treatment options for patients with neurological diseases based on Skyhawk’s innovative STAR technology platform.
The collaboration grants Celgene the exclusive option to in-license worldwide intellectual property rights associated with therapeutic candidates for up to five programs with potential utility in the treatment of amyotrophic lateral sclerosis (ALS), Huntington’s Disease, and additional neurological disorders. The collaboration includes an upfront payment of $60 million, and potential future license fees, milestone payments, and royalties.
Skyhawk is targeting diseases driven by a type of RNA mis-splicing called “exon skipping,” where key regions on the RNA are left out during the RNA splicing process. Skyhawk’s proprietary technology enables the rational design of small molecules that target specific binding pocket regions on RNA, using both sequence and structural specificity, at particular moments in the RNA splicing process. This approach has the potential to reverse the mis-splicing and treat the disease.
Skyhawk Therapeutics, based in Massachusets, USA, develops molecule therapeutics and drugs for the treatment of cancer and neurological diseases.