Merck KGaA strikes $2B RNA splicing deal with Skyhawk to tackle tough brain diseases

Merck KGaA is partnering with Skyhawk Therapeutics on a drug discovery pact worth up to $2 billion plus royalties to develop RNA-targeted therapies for neurological disorders.

Why it matters: The deal targets diseases with limited treatment options, using RNA modulation to potentially create first-in-class therapies for neurological conditions that have resisted traditional drug approaches.

Backstory:

• Skyhawk’s SkySTAR platform uses small molecules to modulate RNA splicing, aiming to reduce disease-driving proteins before they’re produced.

• The biotech has attracted a string of pharma partnerships, including Merck & Co. (the other Merck from the U.S.), Ipsen, Takeda and Vertex.

• Neuroscience is a key focus, with its lead candidate, a splicing modulator for Huntington’s disease, already in phase 2/3 testing.

Big picture: Pharma companies are betting big on RNA targeting technologies as the next frontier in drug development. These approaches could extend beyond neurology to cancer, fibrosis and autoimmune conditions, redefining how “undruggable” diseases are tackled.

What´s next:

• Skyhawk will run discovery and preclinical work; Merck KGaA can license candidates for clinical development.

• The company will face competition from Arrakis and Remix Therapeutics, both pursuing RNA modulation to drug hard targets.