Ipsen and Exicure Inc. have signed an exclusive collaboration agreement to research, develop, and commercialize novel Spherical Nucleic Acids (SNAs) as potential investigational treatments for Huntington's disease and Angelman syndrome. Oligonucleotides are synthetic structures of nucleic acids that can be used to modulate gene expression via a range of processes, including gene activation, inhibition, and splice-modulation. These molecules have demonstrated potential in many different therapeutic areas.

Achieving efficient oligonucleotide delivery to target organs and tissues, including the brain, remains a major limitation to their use. Exicure's SNAs provide distinct chemical and biochemical properties to oligonucleotides. In preclinical models, SNAs have been shown to enhance the cell penetration, biodistribution and organ persistence properties of oligonucleotides, which may potentially enhance drug delivery to previously inaccessible target tissues, including deep brain regions.

Under the agreement, Ipsen will receive exclusive options to license SNA-based therapeutics arising from two collaboration programs for Huntington's disease and Angelman syndrome. Ipsen will pay Exicure a cash upfront payment of $20 million upon closing and Exicure will be responsible for discovery and certain pre-clinical development activities. In the event Ipsen exercises its option, Ipsen will be responsible for further development and commercialization of the licensed products.

Exicure will receive a $20 million upfront payment and is eligible to receive up to $1 Billion in option exercise fees and milestone payments should Ipsen opt into both programs, as well as tiered royalties.