March 27, 2014
– LUNARTM technology for the delivery of RNA medicines to multiple target organs and tumors will be presented
– UNA oligomers for allele-specific silencing of the TTR V30M mutant will be highlighted
San Diego, Calif., March 27, 2014 – Arcturus Therapeutics, Inc., a next wave RNA medicines company pursuing orphan diseases, today announced that its president & CEO, Joseph E. Payne, has been invited to present at the 21st Annual BioCentury Future Leaders in the Biotech Industry Conference, being held on March 28, 2014. In addition to providing a corporate summary, Payne will present data on selective silencing of transthyretin (TTR) V30M mutant allele which is the causative agent of familial amyloidosis polyneuropathy (FAP), a rare disease with limited treatment options. Included in the presentation will be highlights of RNA medicine delivery to multiple organs including the liver and kidney utilizing Arcturus’ proprietary LUNARTM technology as well as broad biodistribution including tumor uptake. The company presentation will be on Friday, March 28, 2:50 PM, taking place at the Millennium Broadway Hotel in New York City.
“Data generated by Arcturus scientists demonstrate that UNA oligomers provide reduced off-target effects and improved potency. Furthermore, we have shown UNA oligomers can be designed to selectively target mutant alleles that can be the causative agents of many significant diseases like V30M for FAP and CAG-repeat disorders including Huntington’s disease,” said Joseph E. Payne, president and CEO, Arcturus. “The core technologies of Arcturus — LUNARTM delivery and UNA chemistry — enable us to develop the next wave of premier RNA medicines.”
About Transthyretin (TTR)-Mediated Amyloidosis
Transthyretin (TTR)-mediated amyloidosis is a genetically mediated fatal disease caused by mutations in the TTR gene. Mutated TTR, which is mainly synthesized in the liver, causes errant amyloid proteins to aggregate and deposit, destroying body organs and tissue, such as the peripheral nerves and heart. TTR-mediated polyneuropathy (FAP) affects approximately 10,000 people and TTR-mediated cardiomyopathy (FAC) affects at least 40,000 people with the mean survival of ~2.5 years. With limited therapeutic options, there is a substantial unmet need for effective medicines for patients suffering from TTR-mediated diseases.
About Arcturus Therapeutics, Inc.
Founded in 2013 and based in San Diego, Arcturus Therapeutics is focused on RNA medicines for the treatment of rare diseases. Arcturus has developed a novel, potent and safe RNA Therapeutics platform called LUNARTM, a proprietary lipid-enabled delivery system for RNA medicines including small interfering RNA, messenger RNA, antisense, and microRNA oligotherapeutics. The company owns Unlocked Nucleic Acid (UNA) chemistry technology and patent portfolio (32 patents, USPTO granted) enabling the targeting of any gene in the human genome. For more information, visit www.ArcturusRx.com.
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