Skyhawk Therapeutics, Inc. has entered a global, five-year strategic collaboration with Celgene Corp. to discover, develop and commercialize innovative small molecule treatments for patients with neurological diseases based on Skyhawk’s STAR technology platform.
 
Celgene has 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 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.  
 
“Targeting RNA with therapeutics is becoming an increasingly important approach in neurological research,” said Richard Hargreaves, Ph.D., corporate vice president, Neuroscience and Imaging Research and Early Development for Celgene. “This collaboration to discover and develop small molecule splicing modifiers extends our commitment to the neurodegenerative disease area consistent with our leadership and focus on protein homeostasis, and strategy to collaborate with innovators who are focused on disruptive approaches to treat disease. We are looking forward to working with Skyhawk’s team of world-leading experts and hope this collaboration will assist each company to realize its mission to bring innovative therapies to patients.”
 
“Through execution of this strategic alliance with Celgene and our new investment round, Skyhawk is well positioned and capitalized to continue advancing our mission of revolutionizing disease treatment with small molecules that correct RNA expression,” said Bill Haney, co-founder and chief executive officer of Skyhawk Therapeutics. “Celgene is a preeminent biopharmaceutical company with a demonstrated history of recognizing disruptive science that may lead to new treatment options for patients with neurological disease. We look forward to a successful collaboration with Celgene, and to continued progress with our internal programs that target a set of exon-skipping-driven cancer indications.”