Navigation Links
Arcturus Therapeutics, Inc. Announces an RNAi Therapeutic Targeting Transthyretin-mediated Amyloidosis as its Flagship Program
Date:1/13/2014

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.
Copyright©2012 PR Newswire.
All rights reserved

Related medicine technology :

1. Arcturus Therapeutics Acquires Cutting-Edge UNA Patent Estate from Marina Biotech
2. Cell Therapeutics, Inc. (CTI) to Report First Quarter Financial Results on April 20
3. Zenobia Therapeutics, Inc. And BioBlocks, Inc. Announce Sale Of A Joint Fragment Library Ideal For Screening By All Popular Methods
4. Cell Therapeutics, Inc. Reports Results of Annual Meeting of Shareholders
5. Cell Therapeutics, Inc. (CTI) Appoints Former Managing Director, Head of West Coast Biotechnology for Oppenheimer/CIBC Matthew J. Plunkett Executive Vice President, Corporate Development
6. Cell Therapeutics, Inc. (CTI) Reduces Net Loss for Third Quarter and Nine Months Ended September 30, 2012 Compared to Same Periods in 2011
7. Caliber and ImmunGene Launch Valor Biotherapeutics, a Joint Venture to Develop Antibody-Interferon Fusion Therapeutics to Treat Cancers
8. Cadence Pharmaceuticals Announces Termination of Option Agreement to Acquire Incline Therapeutics, Inc.
9. Northwest Biotherapeutics, Inc. Announces Full Exercise of Over- Allotment Option
10. Epigenetics Technology Market (Epigenomics, DNA Methylation, Histone Modifications, RNA Interference, Cancer Therapeutics, Personalized Medicine) (2012 - 2017)
11. Arno Therapeutics, Inc. Appoints Randy Thurman to the Board of Directors
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:2/12/2016)... Feb. 12, 2016   National Community Pharmacists Association ... MBA issued the following statement today in response to ... the Centers for Medicare & Medicaid Services (CMS) ... beneficiary advocates and others: patient advocacy organizations ... still reviewing the full CMS analysis. Our initial reaction ...
(Date:2/12/2016)... ALEXANDRIA, Va. , Feb. 12, 2016  This ... & Specialty Pharmacy (JMCP) takes an in-depth look ... the recent spike in prescription drug spending, which has ... JMCP Editor-in-Chief Laura E. Happe ... Laura E. Happe , PharmD, MPH. --> ...
(Date:2/12/2016)... , Feb. 12, 2016 Hypermarcas S.A. ... cash tender offer (the "Tender Offer") to purchase ... aggregate principal amount of its 6.500% Senior Notes ... Nos. US44915JAA88/ USP5246AAF05) (the "Notes"). In connection with ... "Consent Solicitation") consents (the "Consents") of the holders ...
Breaking Medicine Technology:
(Date:2/12/2016)... ... February 12, 2016 , ... ... a new initiative—the Siemens Foundation-PATH Ingenuity Fellowships—to develop the advanced skills needed ... top students from U.S. universities who will draw from Siemens’ deep knowledge ...
(Date:2/12/2016)... ... February 12, 2016 , ... ... to funding innovative lymphoma research and serving the lymphoma community through a comprehensive ... members of South Florida’s philanthropic community at its 10th anniversary Fashion Luncheon on ...
(Date:2/12/2016)... Church, VA (PRWEB) , ... February 12, 2016 , ... CDRH Enforcement Trends: , Back ... http://www.fdanews.com/cdrhenforcementtrends , As Winston Churchill said, “Those who don’t learn from ... will show what to expect when they come knocking this year. But that takes time. ...
(Date:2/12/2016)... ... February 12, 2016 , ... ... 1:30 p.m. – 3:00 p.m. EST, http://www.fdanews.com/fixeddosecombination , ... of pharmaceutical products, garnering increased attention from all stakeholders in the development of ...
(Date:2/12/2016)... ... 12, 2016 , ... Each year, the American Physical Therapy Association (APTA) offers ... the Anaheim Convention Center. Almost 10,000 physical therapists across the country are expected to ... more about their chosen field and network with their colleagues. As in years ...
Breaking Medicine News(10 mins):