Date: 2015-10-29
Type of information: R&D agreement
Compound: messenger RNA (mRNA) therapeutics using UNA Oligomer™ chemistry and LUNAR™ nanoparticle delivery platform
Company: Ultragenyx Pharmaceutical (USA - CA) Arcturus Therapeutics (USA - CA)
Therapeutic area: Rare diseases
Type agreement: R&D licensing
Action mechanism: RNA. The LUNAR™ platform is a proprietary lipid-enabled delivery system for RNA medicines including small interfering RNA, messenger RNA, antisense and microRNA oligotherapeutics. These novel particles have shown excellent safety and potency in multiple animal species. LUNAR utilizes a GMP-ready microfluidic formulation process that yielded small particles (< 80 nm) with a low polydispersity index (< 0.05). This formulation process is scalable and the resulting particles have been shown to be stable. Unlocked nucleomonomer agent (UNA) oligomer is a flexible monomer that enable modulation of affinity and specificity. UNA-modified duplexes have been shown to increase double-stranded RNA stability in serum and decrease off-target effects observed with conventional siRNAs. Specifically, unlocked nucleic acid (UNA) modification of the 5′ end of canonical (i.e., 19+2) siRNAs abrogates gene silencing of the modified strand. One or two modifications near the 3’ end of the oligonucleotide increases stability to 3’ exonuclease degradation. UNA nucleotides can be incorporated into either DNA or RNA oligonucleotides using standard automated synthesis procedures and UNA phosphoramidites. UNA Oligomers™, which are covered by its patent portfolio (34 patents and patent applications, issued in the US, Japan, and other countries). The patented UNA technology can be used to target any gene in the human genome, as well as viral genes, and other species for therapeutic purposes.
Disease:
Details: * On October 29, 2015, Ultragenyx Pharmaceutical, a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, and Arcturus Therapeutics, a RNA medicines company, announced that they have entered into a research collaboration and license agreement to discover and develop messenger RNA (mRNA) therapeutics to certain rare disease targets using Arcturus\' UNA Oligomer™ chemistry and LUNAR™ nanoparticle delivery platform.
Financial terms: Under the terms of the agreement, Ultragenyx will make an upfront payment of $10 million to Arcturus. During the initial phase of the collaboration, Arcturus will design and optimize mRNA therapeutics for two selected rare disease targets. Ultragenyx has the option to add up to eight additional rare disease targets during the collaborative research period. Ultragenyx will be responsible for the development and commercialization of all products under the collaboration. Arcturus will be entitled to preclinical, clinical, regulatory, and sales milestone payments of up to $156 million for each target, as well as reimbursement of all research expenses and mid-single to low double-digit royalties on commercial sales.
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