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USPTO Publishes Five New RNAi-Related Patent Applications

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Title: Recombinant Gene Containing Inverted Repeat Sequence and Utilization Thereof. Number: 20040010131. Filed: June 16, 2003. (PCT Filed: Feb. 21, 2002.) Lead Inventor: Motoya Katsuki, National Institute for Basic Biology.

According to the patent application’s abstract, the invention is designed to “improve a method for introducing dsRNA in such a way that RNAi effect is sustained in mammalian (mainly mouse) cells for a long period of time.” It adds that the invention “provides a recombinant gene, which contains inverted repeats of a target gene [that] can be expressed in mammalian cells.”


Title: Modulation of PTP1B Expression and Signal Transduction by RNA Interference. Number: 20040009946. Filed: May 23, 2003. Lead Inventor: Stephen Patrick Lewis, Ceptyr.

The patent application’s abstract states that the invention covers “compositions and methods relating to small interfering RNA (siRNA) polynucleotides … as pertains to modulation of biological signal transduction.” Specifically, the abstract notes, the application relates to “siRNA polynucleotides that interfere with expression of PTP1B, a member of the protein tyrosine phosphatase (PTP) class of enzymes that mediate signal transduction.”

The abstract adds that “in certain preferred embodiments [of the invention], siRNA modulate[s] signal transduction pathways comprising human or murine PTP1B and, in certain further embodiments, insulin receptor and/of Jak2.”

Modulation of PTP1B-mediated signal transduction, the abstract notes, “has uses in diseases associated with defects in cell proliferation, cell differentiation, and/or cell survival, such as metabolic disorders (including diabetes and obesity), cancer, autoimmune disease, infections and inflammatory disorders, and other conditions.”


Title: Method for Treating Heart Failure by Inhibiting the Sarcolemmal Sodium/Calcium Exchange. Number: 20040009894. Filed: June 30, 2003. Lead Inventor: Ion Hobai, Johns Hopkins University.

“The present invention discloses a method of enhancing cardiac contactility in a subject with heart failure,” the patent application’s abstract states.

“The method consists of administering a compound that inhibits the sarcolemmal sodium/calcium exchanger, whose activity is elevated in heart failure,” it notes. “This method results in correction of cellular calcium handling and enhancement of cardiac contractility to healthy levels. This methods can be used for treatment of acute heart failure, cardiogenic shock, and congestive heart failure.”

The patent application specifically claims a method of inhibiting a sarcolemmal sodium/calcium exchanger “using RNA interference techniques.”


Title: Identification and Tissue Distribution of Two Novel Spliced Variants of the Mouse LATS2 Gene. Number: 20040009502. Filed: March 4, 2003. Lead Inventor: JH David Wu, University of Rochester Medical Center.

The patent application’s abstract states that the “invention relates to isolated nucleic acid molecules encoding splice variants LATS2b and LATS2c; isolated LATS2b and LATS2c protein or polypeptides; and antibodies to the LATS2b and LATS2c proteins and polypeptides.”

The invention also relates to methods of using the nucleic acid molecules, as well as the proteins or peptides, for purposes including: detecting expression of LATS2b or LATS2c in biological samples; regulating LATS2b or LATS2c expression; screening drugs that regulate LATS2b or LATS2c activity and expression; regualting cell growth and differentiation; and treating disease conditions.

“Also disclosed are expression vectors, host cells, and transgenic animals transformed with LATS2b and LATS2c nucleic acid molecules,” the abstract adds.

The patent application specifically claims an RNAi nucleic acid molecule that contains a region “the same as and a region complementary to the nucleic acid molecule” described above. Also claimed are “an expression vector comprising a transcriptional and translational regulatory DNA operably linked to the RNAi nucleic acid molecule … [and] a host cell transformed with the RNAi nucleic acid molecule.”


Title: Selections of Genes and Methods of Using the Same for Diagnosis and for Targeting the Therapy of Select Cancers. Number: 20040009154. Filed: May 31, 2002. Lead Inventor: Javed Khan, NCI.

According to the patent application’s abstract, the invention is “a method of diagnosing a disease that includes obtaining experimental data on gene selections, [which function] to characterize a cancer when expression of that gene selection is compared to the identical selection from a noncancerous cell or a different type of cancer cell.”

The invention also includes “a method of targeting at least one product of a gene that includes administration of a therapeutic agent,” the abstract notes. “The invention also includes the use of a gene selection for diagnosing a cancer.”

The patent application specifically claims a method of targeting a gene product with a therapeutic agent “chosen from the group consisting of: vaccines, antibodies, oligonucleotide DNA antisense, RNAi, chemical molecules, proteins, inhibitors, antagonists, and combinations thereof.”


Alnylam Pharmaceuticals announced this week that it has taken a non-exclusive license to RNAi intellectual property from Cold Spring Harbor Laboratory. Additional details can be found on p. 8.

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