Public Release: 

UMMS announces European RNAi patent issues

University of Massachusetts Medical School

WORCESTER, Mass.--The University of Massachusetts Medical School (UMMS) announced today that the European Patent Office (EPO) has granted European Patent EP 1,309,726 in the Tuschl I patent series. The newly granted patent is based on the one of the earliest applications in a UMMS portfolio covering the seminal research of Phillip D. Zamore, PhD, a Howard Hughes Medical Institute Investigator and the Gretchen Stone Cook Chair of Biomedical Sciences and Professor of Biochemistry & Molecular Pharmacology at UMMS.

This new patent grant extends the scope and breadth of UMMS's fundamental intellectual property estate that comprises numerous issued or granted patents and a large number of pending patent applications that together broadly cover RNAi therapeutics, including small interfering RNAs, or siRNAs, the molecules that mediate RNAi. The patent, "RNA sequence-specific mediators of RNA interference," is based on a discovery by Zamore; Thomas Tuschl, PhD, now of Rockefeller University; Nobel Laureate Phillip A. Sharp, PhD, of the Massachusetts Institute of Technology; and David P. Bartel, PhD, of the Whitehead Institute for Biomedical Research. It is considered to be the fundamental patent on siRNAs, describing the design and use of duplex RNAs of a length too short to induce an interferon response, thereby allowing RNA interference in mammalian cells.

"We are very pleased with the EPO's decision to grant this first European patent from our Tuschl I portfolio, a seminal patent portfolio directed to methods of mediating RNA interference in cells or organisms, such as mice or humans, for a variety of uses ranging from research and development to treating diseases or conditions associated with the presence of a particular target protein," said James P. McNamara, PhD, Executive Director of the Office of Technology Management at UMMS. "This first patent claims a variety of methods of using the generic class of double-stranded RNA (dsRNA) mediators of RNAi of 21-23 nucleotides in length including both naturally produced dsRNA or chemically synthesized analogs. We believe Tuschl I is an essential part of the patent landscape required for the development and commercialization of all RNAi therapeutics."

The Tuschl I patent consists of 19 claims broadly covering RNAi methods, including methods of reducing the expression of a gene, including those of mammalian or viral origin, with double-stranded RNA (dsRNAs) between 21 and 23 nucleotides in length. The patent also includes claims covering methods of examining the function of a gene, as well as the use of both unmodified and chemically modified dsRNAs.

"It is wonderful to have the European Patent Office recognize our discovery that small RNA duplexes direct RNAi in animals," Zamore said. "siRNAs are just now showing clinical promise, and I look forward to the day when the fruits of my collaborative work with Tom, Dave and Phil make a real difference in the lives of patients."

"The issuance of this patent is an important step in securing the pathway to the clinic for siRNA-based therapeutics," said Terence R. Flotte, MD, Dean, Provost, and Executive Deputy Chancellor of UMMS. "Appropriate patent protection will encourage future investment in innovative siRNA therapies for neurodegenerative diseases, cancer, infectious diseases and other conditions for which current therapies are inadequate."

In September 2009, UMMS broke ground on the $400 million Albert Sherman Center, a 480,000 square-foot, 9-story state-of-the-art research and education facility designed to maximize collaboration among researchers, educators and students to encourage innovation and learning across disciplines. To be completed in 2012, the Albert Sherman Center (ACS) will be home to the Advanced Therapeutics Cluster, comprising the RNA Therapeutics Institute, the Center for Stem Cell Biology and Regenerative Medicine and the Gene Therapy Center.

The RNA Therapeutics Institute (RTI), led by Zamore; Nobel Laureate Craig C. Mello, PhD, Howard Hughes Medical Institute Investigator, Blais University Chair in Molecular Medicine and professor of molecular medicine and cell biology; Melissa J. Moore, PhD, Howard Hughes Medical Institute Investigator and professor of biochemistry & molecular pharmacology; and Victor Ambros, PhD, Silverman Chair in Natural Sciences and professor of molecular medicine, is building a community of scientists passionate about RNA.

By uniting researchers studying the fundamental biology and mechanisms of cellular RNAs with those working to devise human therapies using or targeting nucleic acids, the RTI represents a new model for scientific exploration. The co-location of basic and applied nucleic acid scientists with clinicians dedicated to finding new cures creates a new paradigm for organizing molecular research that enables the rapid application of new biological discoveries to solutions for unmet challenges in human health.


About RNA Interference (RNAi)

RNAi (RNA interference) is a revolution in biology, representing a breakthrough in understanding how genes are turned on and off in cells, and a completely new approach to drug discovery and development. Its discovery, which was awarded the 2006 Nobel Prize in Medicine, has been heralded as "a major scientific breakthrough that happens once every decade or so," and represents one of the most promising and rapidly advancing frontiers in biology and drug discovery today. RNAi is a natural process of gene silencing that occurs in organisms ranging from plants to mammals.

By harnessing the natural biological process of RNAi occurring in our cells, scientists hope to create a major new class of medicines, known as RNA therapeutics. RNA therapeutics target the cause of diseases by potently silencing specific messenger RNAs (mRNAs), thereby preventing disease-causing proteins from being made. RNA therapeutics have the potential to treat disease and help patients in a fundamentally new way.

About the University of Massachusetts Medical School

The University of Massachusetts Medical School attracts more than $240 million in research funding annually, and its innovative programs are the centerpiece of the Massachusetts Life Sciences Initiative. Consistently ranked by U.S.News & World Report as one of the leading medical schools in the nation for primary care education, UMMS comprises a medical school, graduate school of nursing, graduate school of biomedical sciences and an active research enterprise, and is a leader in health sciences education, research and public service. UMMS is the academic partner of UMass Memorial Health Care. For more information, visit

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