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SOURCE Arcturus Therapeutics
- Arcturus proprietary LUNAR™ delivery technology demonstrates exceptional potency in vivo, ED(50) of 0.020 mg/kg
- No adverse events observed in a multiple-dose rat toxicology study, up to 30 mg/kg
- Functional in vivo delivery of distinct therapeutic classes of RNA, including siRNAs and messenger RNAs
- Non-human primate proof-of-concept achieved for flagship program targeting transthyretin (TTR)
SAN DIEGO, Jan. 30, 2014 /PRNewswire/ -- Arcturus Therapeutics, Inc. today announced it will present a poster at the Keystone Symposia Conference on RNA Silencing held January 31- February 5, 2014 in Seattle, Washington. The Arcturus poster titled "Design of a Novel, Potent, and Safe RNA Therapeutic Platform Utilizing LUNARTM Delivery Technology" (poster number 1037) will be presented on Saturday, February 1. Preclinical data of its flagship RNA therapeutic candidate, an Unlocked Nucleic Acid (UNA)-modified small interfering RNA targeting transthyretin (TTR) for TTR-mediated amyloidosis, will be included in the poster presentation.
"We are pleased to be selected by the highly regarded Keystone Symposia Committee to present a poster highlighting our enabling technology for the safe and effective delivery of RNA medicines," said Pad Chivukula, Ph.D., Arcturus' CSO and COO. "We have successfully discovered and developed a proprietary RNA technology platform called LUNARTM and have validated it through preclinical testing in multiple animal species, including non-human primates, for our lead program targeting TTR."
"Arcturus is excited to present at the Keystone Symposia and share our technology and therapeutic pipeline with the RNA community," said Zachary Zimmerman, Ph.D., Arcturus' Chief Business Officer. "At Arcturus, we are building a significant company leading the next generation of RNA therapeutics."
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, Calif., 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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