Biopharmaceutical company Ipsen and Exicure have collaborated to research, develop, and commercialize novel Spherical Nucleic Acids (SNAs) as potential investigational therapies to treat Angelman syndrome and Huntington’s disease.
Exicure’s SNAs are nanoscale constructs comprising synthetic nucleic acid sequences and provide distinct chemical and biochemical properties to oligonucleotides.
Oligonucleotides are synthetic structures of nucleic acids used to modulate gene expression through a range of processes such as splice-modulation, inhibition, and gene activation.
Ipsen stated that SNAs have shown that they boost the cell penetration properties, biodistribution and organ persistence properties of oligonucleotides in preclinical models.
Under the terms of the exclusive collaboration agreement signed by the companies, Ipsen will secure exclusive options to license SNA-based therapeutics arising from two collaboration programmes, which are currently under discovery evaluation for Huntington’s disease and Angelman syndrome.
Meanwhile, Exicure will carry out discovery and preclinical development of SNA-based therapies for Huntington’s and Angelman.
Exicure CEO David Giljohann said: “We are thrilled to partner with Ipsen, a leading global company with significant expertise and commitment to developing treatments for patients with rare neurological diseases.
“In collaboration with Ipsen, we have the opportunity to apply our technology to Huntington’s disease and Angelman syndrome, both indications requiring deep brain penetration and technological advances to reach previously hard-to-drug targets.
“We believe our platform technology with its deep penetration and persistence of medicinal effect will allow Exicure and Ipsen to overcome challenges from first-generation oligonucleotides and bring new medicines to patients in need.”
As per the terms of the deal, Exicure will receive an upfront payment of $20m in cash from Ipsen upon closing.
If Ipsen exercises its option, the company will lead further development and commercialisation of the licensed products and pay Exicure up to $1bn in exercise fees and milestone payments.