Ipsen and Exicure 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.

Philippe Lopes-Fernandes, chief business officer at Ipsen, said “Neuroscience is deeply rooted within Ipsen as a key strategic driver for our business. We are pleased to partner with Exicure to progress development of investigational treatment options for Huntington’s disease and Angelman syndrome, two areas of significant unmet need. This collaboration marks an important step in maximizing the potential of this novel technology, bringing together the expertise of Exicure and the robust heritage of Ipsen in neuroscience. With this new collaboration we will deepen our commitment to people living with neurological conditions around the world.”