Skyhawk and Celgene Partner to Develop Innovative Treatments for ALS, Other Diseases

Skyhawk Therapeutics and Celgene have agreed to collaborate for five years to discover, develop, and commercialize innovative small molecule treatment options for neurological diseases with high unmet medical needs, including amyotrophic lateral sclerosis (ALS) and Huntington’s disease.

Several genetic disorders are caused by “exon skipping,” which means there’s an error in the early process of gene activity, leaving crucial pieces of ribonucleic acid (RNA) out during RNA splicing, which eventually leads to the production of a mutated or defective protein.

RNA is the in-between molecule in the process that produces a protein from a gene (the RNA Society website says, “DNA makes RNA makes protein.”) Before RNA molecules give rise to proteins, they go through a process called splicing, which consists of gluing together all the protein-coding sequences, known as exons, while removing small parts that are not required, called introns.

The new collaboration will make use of Skyhawk’s innovative STAR* technology platform, which takes small engineered molecules that target precise RNA regions at “time-specific points” during the RNA splicing process, correcting the genetic error and hopefully treating the disease.

Under the agreement, Celgene has the exclusive option to license up to five treatment candidates created using Skyhawk’s technology and aimed at helping correct RNA exon-skipping in ALS, Huntington’s, and other diseases.

Celgene will pay Skyhawk an up-front payment of $60 million, along with possible future license fees, milestone payments, and royalties.

Skyhawk has also completed a new equity investment round. Participants included Alexandria Venture Investments, the Duke of Bedford, GreatPoint Ventures, and Celgene Corporation.

“Targeting RNA with therapeutics is becoming an increasingly important approach in neurological research,” Richard Hargreaves, PhD, corporate vice president, neuroscience and imaging research and early development for Celgene, said in a news release.

“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,” Hargreaves said.

“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,” he added.

Bill Haney, co-founder and CEO of Skyhawk Therapeutics, said that through the collaboration with Celgene and the 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.”