Welcome!

News Feed Item

Arcturus Therapeutics, Inc. Announces an RNAi Therapeutic Targeting Transthyretin-mediated Amyloidosis as its Flagship Program

New Preclinical Results in Non-Human Primates Demonstrate Significant and Sustained Reduction of Transthyretin Using its Proprietary LUNARTM Delivery Technology

SAN DIEGO, Jan. 13, 2014 /PRNewswire/ -- Arcturus Therapeutics, Inc., an industry leader in RNA technologies, today announced its flagship program – an RNAi therapeutic targeting transthyretin (TTR)-mediated amyloidosis.  Preclinical non-human primate data will be presented at the Biotech Showcase being held January 13, 2014 in San Francisco, California.

(Logo: http://photos.prnewswire.com/prnh/20130531/MM24393LOGO)

Preclinical results from a non-human primate (NHP) study demonstrated > 75% knockdown of serum TTR protein levels after 10 days, with the effect sustained over 3 weeks (0.3 mg/kg, i.v. dosing).

In addition to NHP knockdown data, no adverse events were observed using Arcturus LUNARTM delivery technology, in particular, no injection site reactions occurred. Furthermore, a substantial safety window was demonstrated in a multiple dose rat toxicology study – no adverse events observed at the highest doses (30 mg/kg total; administered three times at 10 mg/kg over 15 days). Serum levels of the alanine aminotransferase (ALT) and aspartate aminotransferase (AST) were determined after intravenous administration and no changes in these liver enzymes were observed.

"We are pleased to announce our flagship program targeting TTR including our successful achievement of non-human primate data with durable biological effect and having an impressive safety profile," said Pad Chivukula, Ph.D., Arcturus' CSO and COO. "With this data in hand, we are laser-focused on selecting a wholly-owned clinical drug candidate in 2014 and entering the clinic soon after."

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, cause 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 worldwide and TTR-mediated cardiomyopathy (FAC) affects at least 40,000 people worldwide with the mean survival of ~2.5 years.  With no approved therapies, there is a substantial medical need for effective therapeutics 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 technologies for the treatment of rare diseases and high unmet medical needs. 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, 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.

SOURCE Arcturus Therapeutics, Inc.

More Stories By PR Newswire

Copyright © 2007 PR Newswire. All rights reserved. Republication or redistribution of PRNewswire content is expressly prohibited without the prior written consent of PRNewswire. PRNewswire shall not be liable for any errors or delays in the content, or for any actions taken in reliance thereon.

Latest Stories
Between the mockups and specs produced by analysts, and resulting applications built by developers, there exists a gulf where projects fail, costs spiral, and applications disappoint. Methodologies like Agile attempt to address this with intensified communication, with partial success but many limitations. In his session at 18th Cloud Expo, Charles Kendrick, CTO & Chief Architect at Isomorphic Software, will present a revolutionary model enabled by new technologies. Learn how business and devel...
You think you know what’s in your data. But do you? Most organizations are now aware of the business intelligence represented by their data. Data science stands to take this to a level you never thought of – literally. The techniques of data science, when used with the capabilities of Big Data technologies, can make connections you had not yet imagined, helping you discover new insights and ask new questions of your data. In his session at @ThingsExpo, Sarbjit Sarkaria, data science team lead ...
The IoT has the potential to create a renaissance of manufacturing in the US and elsewhere. In his session at 18th Cloud Expo, Florent Solt, CTO and chief architect of Netvibes, will discuss how the expected exponential increase in the amount of data that will be processed, transported, stored, and accessed means there will be a huge demand for smart technologies to deliver it. Florent Solt is the CTO and chief architect of Netvibes. Prior to joining Netvibes in 2007, he co-founded Rift Technol...
If there is anything we have learned by now, is that every business paves their own unique path for releasing software- every pipeline, implementation and practices are a bit different, and DevOps comes in all shapes and sizes. Software delivery practices are often comprised of set of several complementing (or even competing) methodologies – such as leveraging Agile, DevOps and even a mix of ITIL, to create the combination that’s most suitable for your organization and that maximize your busines...
Struggling to keep up with increasing application demand? Learn how Platform as a Service (PaaS) can streamline application development processes and make resource management easy.
New Relic, Inc. has announced a set of new features across the New Relic Software Analytics Cloud that offer IT operations teams increased visibility, and the ability to diagnose and resolve performance problems quickly. The new features further IT operations teams’ ability to leverage data and analytics, as well as drive collaboration and a common, shared understanding between teams. Software teams are under pressure to resolve performance issues quickly and improve availability, as the comple...
The proper isolation of resources is essential for multi-tenant environments. The traditional approach to isolate resources is, however, rather heavyweight. In his session at 18th Cloud Expo, Igor Drobiazko, co-founder of elastic.io, will draw upon their own experience with operating a Docker container-based infrastructure on a large scale and present a lightweight solution for resource isolation using microservices. He will also discuss the implementation of microservices in data and applicat...
Join IBM June 8 at 18th Cloud Expo at the Javits Center in New York City, NY, and learn how to innovate like a startup and scale for the enterprise. You need to deliver quality applications faster and cheaper, attract and retain customers with an engaging experience across devices, and seamlessly integrate your enterprise systems. And you can't take 12 months to do it.
See storage differently! Storage performance problems have only gotten worse and harder to solve as applications have become largely virtualized and moved to a cloud-based infrastructure. Storage performance in a virtualized environment is not just about IOPS, it is about how well that potential performance is guaranteed to individual VMs for these apps as the number of VMs keep going up real time. In his session at 18th Cloud Expo, Dhiraj Sehgal, in product and marketing at Tintri, will discu...
Machine Learning helps make complex systems more efficient. By applying advanced Machine Learning techniques such as Cognitive Fingerprinting, wind project operators can utilize these tools to learn from collected data, detect regular patterns, and optimize their own operations. In his session at 18th Cloud Expo, Stuart Gillen, Director of Business Development at SparkCognition, will discuss how research has demonstrated the value of Machine Learning in delivering next generation analytics to im...
This is not a small hotel event. It is also not a big vendor party where politicians and entertainers are more important than real content. This is Cloud Expo, the world's longest-running conference and exhibition focused on Cloud Computing and all that it entails. If you want serious presentations and valuable insight about Cloud Computing for three straight days, then register now for Cloud Expo.
As you respond to increasing requests for new analytics, you need fast and flexible technology in your arsenal so that you can deploy the right workload to the right platform for the need at hand. Do you need self-service and fast time to value? Do you have data and application control and privacy needs, along with strict SLAs to meet? IBM dashDB™ is data warehouse technology powered by in-memory computing and in-database analytics that are designed for fast results, scalability and more.
SYS-CON Events announced today that SoftLayer, an IBM Company, has been named “Gold Sponsor” of SYS-CON's 18th Cloud Expo, which will take place on June 7-9, 2016, at the Javits Center in New York, New York. SoftLayer, an IBM Company, provides cloud infrastructure as a service from a growing number of data centers and network points of presence around the world. SoftLayer’s customers range from Web startups to global enterprises.
So, you bought into the current machine learning craze and went on to collect millions/billions of records from this promising new data source. Now, what do you do with them? Too often, the abundance of data quickly turns into an abundance of problems. How do you extract that "magic essence" from your data without falling into the common pitfalls? In her session at @ThingsExpo, Natalia Ponomareva, Software Engineer at Google, will provide tips on how to be successful in large scale machine lear...
Up until last year, enterprises that were looking into cloud services usually undertook a long-term pilot with one of the large cloud providers, running test and dev workloads in the cloud. With cloud’s transition to mainstream adoption in 2015, and with enterprises migrating more and more workloads into the cloud and in between public and private environments, the single-provider approach must be revisited. In his session at 18th Cloud Expo, Yoav Mor, multi-cloud solution evangelist at Cloudy...