RARE Daily

Vertex Partners with Skyhawk to Modulate RNA Splicing for Serious Diseases

December 22, 2020

Rare Daily Staff

Vertex Pharmaceuticals and Skyhawk Therapeutics have entered into strategic research collaboration and licensing agreement aimed at the discovery and development of novel small molecules that modulate RNA splicing for the treatment of serious diseases.

“We believe that splice site modulation holds significant promise for the treatment of diseases which today have limited or no therapeutic options,” said Mark Bunnage, senior vice president and site head, Boston Research at Vertex. “This collaboration brings Skyhawk’s innovative technology together with Vertex’s research and development experience; and fits perfectly with our strategy of investing in new technologies that will help us transform multiple serious diseases.”

Under their agreement, Vertex will pay Skyhawk $40 million upfront. Skyhawk will grant Vertex options to exclusively license worldwide intellectual property rights to candidates discovered and developed under the collaboration that are directed to program targets. Following Vertex’s exercise of its options, Vertex will be responsible for further development and commercialization. Skyhawk is also eligible to receive up to $2.2 billion in potential milestone payments, as well as potential royalties on future sales.

Skyhawk develops and commercializes therapies using its novel SkySTAR platform, building small molecules that provide breakthrough treatments for patients. Skyhawk has productive collaborations across a broad range of disease areas ranging from neurodegenerative disease to oncology.

RNA mis-splicing that leads to loss of RNA expression has been found to cause a growing list of diseases, from orphan and neurological conditions to major cancers. Very few treatments are available for these challenging conditions, many of which have been considered undruggable. Skyhawk’s proprietary SkySTAR platform integrated information from computational, kinetic, & structural models of RNA to correct the underlying genetics of disease at the mRNA level, with a focus on neurodegenerative disease and oncology.

Although the companies did not specify the focus of the collaboration, Skyhawk chief Bill Haney told Endpoints News that general focus is neurological, and since Skyhawk’s technology aims to correct the underlying genetics of disease, it will likely include orphan indications.

Photo: Mark Bunnage, senior vice president and site head, Boston Research at Vertex

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