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
Extreme Computing is the ability to leverage highly performant infrastructure and software to accelerate Big Data, machine learning, HPC, and Enterprise applications. High IOPS Storage, low-latency networks, in-memory databases, GPUs and other parallel accelerators are being used to achieve faster results and help businesses make better decisions. In his session at 18th Cloud Expo, Michael O'Neill, Strategic Business Development at NVIDIA, focused on some of the unique ways extreme computing is...
I wanted to gather all of my Internet of Things (IOT) blogs into a single blog (that I could later use with my University of San Francisco (USF) Big Data “MBA” course). However as I started to pull these blogs together, I realized that my IOT discussion lacked a vision; it lacked an end point towards which an organization could drive their IOT envisioning, proof of value, app dev, data engineering and data science efforts. And I think that the IOT end point is really quite simple…
"We view the cloud not really as a specific technology but as a way of doing business and that way of doing business is transforming the way software, infrastructure and services are being delivered to business," explained Matthew Rosen, CEO and Director at Fusion, in this SYS-CON.tv interview at 18th Cloud Expo, held June 7-9, 2016, at the Javits Center in New York City, NY.
Redis is not only the fastest database, but it is the most popular among the new wave of databases running in containers. Redis speeds up just about every data interaction between your users or operational systems. In his session at 19th Cloud Expo, Dave Nielsen, Developer Advocate, Redis Labs, will share the functions and data structures used to solve everyday use cases that are driving Redis' popularity.
Aspose.Total for .NET is the most complete package of all file format APIs for .NET as offered by Aspose. It empowers developers to create, edit, render, print and convert between a wide range of popular document formats within any .NET, C#, ASP.NET and VB.NET applications. Aspose compiles all .NET APIs on a daily basis to ensure that it contains the most up to date versions of each of Aspose .NET APIs. If a new .NET API or a new version of existing APIs is released during the subscription peri...
Organizations planning enterprise data center consolidation and modernization projects are faced with a challenging, costly reality. Requirements to deploy modern, cloud-native applications simultaneously with traditional client/server applications are almost impossible to achieve with hardware-centric enterprise infrastructure. Compute and network infrastructure are fast moving down a software-defined path, but storage has been a laggard. Until now.
"My role is working with customers, helping them go through this digital transformation. I spend a lot of time talking to banks, big industries, manufacturers working through how they are integrating and transforming their IT platforms and moving them forward," explained William Morrish, General Manager Product Sales at Interoute, in this SYS-CON.tv interview at 18th Cloud Expo, held June 7-9, 2016, at the Javits Center in New York City, NY.
To leverage Continuous Delivery, enterprises must consider impacts that span functional silos, as well as applications that touch older, slower moving components. Managing the many dependencies can cause slowdowns. See how to achieve continuous delivery in the enterprise.
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 ...
SYS-CON Events announced today the Kubernetes and Google Container Engine Workshop, being held November 3, 2016, in conjunction with @DevOpsSummit at 19th Cloud Expo at the Santa Clara Convention Center in Santa Clara, CA. This workshop led by Sebastian Scheele introduces participants to Kubernetes and Google Container Engine (GKE). Through a combination of instructor-led presentations, demonstrations, and hands-on labs, students learn the key concepts and practices for deploying and maintainin...
Security, data privacy, reliability, and regulatory compliance are critical factors when evaluating whether to move business applications from in-house, client-hosted environments to a cloud platform. Quality assurance plays a vital role in ensuring that the appropriate level of risk assessment, verification, and validation takes place to ensure business continuity during the migration to a new cloud platform.
Extracting business value from Internet of Things (IoT) data doesn’t happen overnight. There are several requirements that must be satisfied, including IoT device enablement, data analysis, real-time detection of complex events and automated orchestration of actions. Unfortunately, too many companies fall short in achieving their business goals by implementing incomplete solutions or not focusing on tangible use cases. In his general session at @ThingsExpo, Dave McCarthy, Director of Products...
WebRTC is bringing significant change to the communications landscape that will bridge the worlds of web and telephony, making the Internet the new standard for communications. Cloud9 took the road less traveled and used WebRTC to create a downloadable enterprise-grade communications platform that is changing the communication dynamic in the financial sector. In his session at @ThingsExpo, Leo Papadopoulos, CTO of Cloud9, discussed the importance of WebRTC and how it enables companies to focus...
Security, data privacy, reliability and regulatory compliance are critical factors when evaluating whether to move business applications from in-house client hosted environments to a cloud platform. In her session at 18th Cloud Expo, Vandana Viswanathan, Associate Director at Cognizant, In this session, will provide an orientation to the five stages required to implement a cloud hosted solution validation strategy.
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...