Celgene inks $60M deal with newcomer Skyhawk to develop RNA drugs for ALS, Huntington’s

Illustration of three DNA helices
Skyhawk aims to target specific binding pocket regions on RNA, which can be left out during key moments in the splicing process, and reverse that skipping to treat the underlying causes of certain diseases. (Darwin Laganzon)

Celgene is launching a $60 million collaboration with Skyhawk Therapeutics to develop and commercialize small molecules targeting RNA mis-splicing in neurological diseases.

Under the deal, Celgene received the exclusive option to license up to five drug candidates born out of Skyhawk’s development platform, to help correct RNA exon skipping in amyotrophic lateral sclerosis, Huntington’s disease and other disorders. The $60 million upfront payment could be paired with future license fees, milestones and royalties.

In addition, the Waltham, Massachusetts-based Skyhawk closed a new funding round of common stock, bringing in another $40 million. The biotech hopes the combined $100 million will help push its first oncology drug to the clinic, separately from the Celgene collaboration, in 2019.


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That money joins $8 million in seed funding raised earlier this year, and included the return of investors Alexandria Venture Investments and the Duke of Bedford. Other investors this round included Greatpoint Ventures, ShangPharma and Agent Capital, as well as Celgene, plus family investors including Tim Disney, the great-nephew of Walt Disney.

RELATED: Skyhawk, copying Dragonfly model, gets cash for RNA R&D

This isn’t the first connection between Skyhawk and Celgene: last year, the startup’s executives and investors reached a $33 million commercialization deal through their sister company, Dragonfly Therapeutics, to develop natural killer cell-based blood cancer therapies. Dragonfly’s CEO, Bill Haney, co-founded Skyhawk and serves as its executive chairman.

RELATED: Celgene bags option on NK cell-based blood cancer assets

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

“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.

Skyhawk’s tech aims to target specific binding pocket regions on RNA, which can be left out during key moments in the splicing process. Reversing that skipping could treat the underlying causes of a range of diseases, the company hopes.

“Through our new strategic alliance and investment round, the company is well positioned and capitalized to advance both our partnered programs in neurology and internal programs that currently focus on a set of RNA splicing-driven cancer indications,” Haney said.

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