GSK and Isis Pharmaceuticals sign RNA therapeutics collaboration
GlaxoSmithKline (GSK) and Isis Pharmaceuticals have formed an alliance to develop novel RNA-based therapeutics for rare and infectious diseases. Isis will handle early discovery and development, with GSK holding options for further development and commercialization.

GlaxoSmithKline (GSK) and Isis Pharmaceuticals announced a strategic alliance to develop new RNA-based therapeutics targeting rare and serious diseases, including infectious diseases. The agreement covers up to six programs, with Isis Pharmaceuticals responsible for identifying and developing compounds through to clinical proof-of-concept (PoC). Following PoC, GSK has the option to license the compounds and will manage all further development and commercialization.
Under the terms, Isis Pharmaceuticals will receive an upfront payment of $35 million and is eligible for up to $20 million per program in milestones through the PoC phase. If all six programs are successfully developed and commercialized, Isis could receive total license and milestone payments nearing $1.5 billion. Additionally, Isis is set to receive double-digit royalties on sales of any successfully commercialized products.
The collaboration leverages Isis' antisense drug discovery platform, which targets RNA molecules to inhibit the production of disease-causing proteins. GSK stated that this approach offers new opportunities to target certain severe diseases that have previously lacked effective treatments. Isis Pharmaceuticals is recognized as a leader in antisense technology.
This alliance provides GSK access to Isis' expertise in the discovery and development of RNA-targeted therapeutics. Isis will retain responsibility for discovery and development up to PoC, after which GSK will oversee global late-stage development and commercialization. The focus areas include rare diseases, infectious diseases, and certain conditions affecting vision.