Navigation Links
Study Published in Nature Biotechnology Demonstrates Ability of Moderna's Novel Therapeutic Paradigm to Repair and Regenerate Damaged Heart Tissue
Date:9/8/2013

CAMBRIDGE, Mass., Sept. 8, 2013 /PRNewswire/ -- Moderna Therapeutics, the pioneering company developing messenger RNA therapeutics™, a revolutionary new treatment modality to enable the in vivo production of therapeutic proteins, announced today that two of its academic co-founders published a study in Nature Biotechnology demonstrating that Moderna's messenger RNA therapeutics™ platform induced in vivo production of proteins to stimulate blood vessel growth, repair damaged heart tissue, and improve outcomes in a mouse model of myocardial infarction (MI).

The research, led by Moderna's academic co-founder Kenneth Chien, M.D., Ph.D., with the help of academic co-founder Derrick Rossi, Ph.D., provides proof-of-concept that messenger RNA therapeutics™ provide an effective and robust platform for gene transfer that could be applicable across a number of diseases. The Nature Biotechnology paper is the first peer-reviewed scientific publication describing the ability of messenger RNA therapeutics™ to stimulate in vivo production of human proteins and to achieve a long-term therapeutic effect.

Researchers injected mice with a single dose of VEGF-A-coded messenger RNA in the heart shortly following myocardial infarction to trigger production of the VEGF-A protein. VEGF-A (vascular endothelial growth factor-A) is an important angiogenic factor protein that mediates the growth of blood vessels. Mice injected with messenger RNA went on to generate new cardiovascular cells and show improvements on measures of cardiac function and survival when compared with controls.

"Regeneration of heart tissue through the mobilization of heart progenitor cells holds tremendous potential for the treatment of myocardial infarction and other forms of heart injury but has remained an elusive goal for over a decade," said Dr. Chien. "Over the past 20 years, many labs have attempted to repair damaged heart muscle in animal models by delivering VEGF-A and other factors to the heart using recombinant proteins, DNA plasmids, and engineered viruses—with little if any clinical success. The results we have seen using messenger RNA therapeutics™ mark the start of a new and exciting phase of drug development with potentially profound implications for patients."

In the study, the mice injected with VEGF-A-coded messenger RNA produced a "pulse-like" expression of a significant quantity of VEGF-A near the injection site. The production of VEGF-A in turn mobilized heart progenitor cells to produce functional, intact blood vessels in the heart and led to marked improvements in heart function and improved long-term survival of the mice for at least one year post-MI. Researchers attribute these effects to a reduction in the size of infarct region and an increase in capillary density in the affected region. In this way, the high-level, transient production of VEGF-A led to a sustained therapeutic improvement by altering the behavior of heart progenitor cells. 

Introduction of modRNA was not associated with an innate immune response, further supporting the safety and effectiveness of this novel approach.

"We are extremely proud that our messenger RNA therapeutics™ platform enabled Ken to realize his vision and clinical intuition of the last 20 years," said Stephane Bancel, president and founding CEO of Moderna. "Beyond the considerable implications for the treatment of heart injury, this publication provides an important validation of the potential of messenger RNA therapeutics™ across a wide range of diseases. The ability to stimulate protein production within cells will enable the treatment of many diseases that cannot be addressed today. What is also exciting is the fact that the Moderna team has considerably improved our technology since its performance for this groundbreaking study. The current generation of our technology is capable of producing 50 times more protein per dose, and our R&D team has many new improvements in the making, further increasing the potential of messenger RNA therapeutics™ to transform drug development and improve patients' lives."

Moderna in March announced an exclusive strategic option agreement with AstraZeneca to discover, develop, and commercialize messenger RNA therapeutics™ for the treatment of serious cardiovascular, metabolic, and renal diseases as well as selected targets in oncology. Targets covered under the agreement included the use of VEGF in the treatment of cardiovascular disease.

"Cardio-metabolic medicine is a core therapy area for AstraZeneca, and part of our research focuses on identifying novel therapies that will activate progenitor cells within the heart to turn into healthy heart cells. Recent findings by Dr. Chien and his team will play an important role in our work with Karolinska Institutet to advance cardiac regenerative therapy and our collaboration with Moderna Therapeutics to discover and develop messenger RNA therapeutics™," said Dr. Marcus Schindler, Head of the AstraZeneca Cardiovascular and Metabolic Disease Innovative Medicines Unit.

About Moderna Therapeutics

Moderna is pioneering messenger RNA therapeutics™, an entirely new in vivo drug modality that produces human proteins or antibodies inside patient cells, which are in turn active intracellularly or secreted. This breakthrough platform addresses currently undruggable targets and offers a superior alternative to existing drug modalities for a wide range of disease conditions. Moderna has developed a broad intellectual property estate, including 144 patent applications with 6,910 claims ranging from novel nucleotide chemistries to specific drug compositions. The company plans to develop and commercialize its innovative messenger RNA drugs—initially for rare diseases and oncology—while partnering drug candidates in other therapeutic areas in order to rapidly deliver this innovation to patients. Moderna is a privately held company based in Cambridge, Mass. Visit www.modernatx.com to learn more.

For information contact:
Dan Quinn
617-761-6732
Dan.Quinn@fkhealth.com


'/>"/>
SOURCE Moderna Therapeutics
Copyright©2012 PR Newswire.
All rights reserved

Related biology technology :

1. iLab Solutions Announces the Results of the 2013 Core Facility Benchmarking Study
2. ResearchMoz.us: Private Healthcare Market - Investigation, Study, Size, Share in Central Europe and Poland and Poland 2013
3. Orexigen Therapeutics Prepares for the Light Study Interim Analysis Based on Direction from Data Monitoring Committee
4. Personalized Medicine Makes Its Way to Pets With Cancer: Morphogenesis and SEVO-Med Team up for a Canine Cancer Field Study, First of Its Kind, Side-Effect-Free Process to Treat Companion Animals
5. Oncopeptides Initiates Phase II Clinical Study with Melflufen in the Treatment of Multiple Myeloma Patients
6. MiMedx Plantar Fasciitis Study Published In The Foot & Ankle International Journal
7. Technical Study: Vertex Pharma Inc., Orexigen Therapeutics Inc., MAKO Surgical Corp., and Alere Inc.
8. BioStorage Technologies Launches INTEGRITY Study to Measure Effect of Time, Temperature and Preparation Methods on DNA and RNA Samples
9. Study Scavenger and CenterWatch Partner to Improve Patient Recruitment, Enrollment and Mobile Access to Clinical Trials with New Study Scavenger Smartphone App
10. VG Energy Chief Scientist Awarded $200,000 AgriLife Grant to Study Lipid Secretion
11. ImmusanT Initiates Study at Joslin Diabetes Center to Investigate Relationship Between Celiac Disease and Type 1 Diabetes
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:6/24/2016)... ... June 24, 2016 , ... Researchers at the Universita ... miRNAs in people with peritoneal or pleural mesothelioma. Their findings are the subject of ... now. , Diagnostic biomarkers are signposts in the blood, lung fluid or tissue ...
(Date:6/23/2016)... 23, 2016   Boston Biomedical , an ... designed to target cancer stemness pathways, announced that ... Orphan Drug Designation from the U.S. Food and ... cancer, including gastroesophageal junction (GEJ) cancer. Napabucasin is ... inhibit cancer stemness pathways by targeting STAT3, and ...
(Date:6/23/2016)... 23, 2016 Houston Methodist Willowbrook Hospital ... Sports Association to serve as their official health ... Methodist Willowbrook will provide sponsorship support, athletic training ... association coaches, volunteers, athletes and families. ... Sports Association and to bring Houston Methodist quality ...
(Date:6/23/2016)... -- The Biodesign Challenge (BDC), a university competition that asks ... systems and biotechnology, announced its winning teams at the ... York City . The teams, chosen ... MoMA,s Celeste Bartos Theater during the daylong summit. Keynote ... of architecture and design, and Suzanne Lee , ...
Breaking Biology Technology:
(Date:4/15/2016)... , April 15, 2016  A new partnership ... more accurate underwriting decisions in a fraction of ... competitively priced and high-value life insurance policies to ... With Force Diagnostics, rapid testing (A1C, ... data readings (blood pressure, weight, pulse, BMI, and ...
(Date:4/13/2016)... CHICAGO , April 13, 2016  IMPOWER physicians ... are setting a new clinical standard in telehealth ... By leveraging the higi platform, IMPOWER patients can ... weight, pulse and body mass index, and, when they ... quick and convenient visit to a local retail location ...
(Date:3/29/2016)... , March 29, 2016 ... "Company") LegacyXChange "LEGX" and SelectaDNA/CSI Protect are pleased to ... ink used in a variety of writing instruments, ensuring ... of originally created collectibles from athletes on LegacyXChange will ... analysis of the DNA. Bill Bollander ...
Breaking Biology News(10 mins):