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USPTO Publishes Four RNAi-Related Patent Applications: Jan 25, 2007

Title: Double-Stranded Compositions Comprising a 3’-Endo-Modified Strand for Use in Gene Modulation
Number: 20070015722
Filed: June 3, 2004 PCT Filed: June 3, 2004
Lead Inventor: Brian Kraynack, Isis Pharmaceuticals
The invention, the patent application’s abstract states, “provides double stranded compositions that have a region that is complementary to a target nucleic acid. One of the targeting strand or the second strand comprises linked ribofuranosyl nucleosides and the other strand comprises linked modified nucleosides that have 3'-endo conformational geometry. The strands can be linked together or separate and may contain additional groups,” the abstract adds. The invention “also provides methods of using the compositions for modulating gene expression.”

Title: Modulation of Endogenous AICAR Levels for the Treatment of Diabetes and Obesity
Number: 20070015720
Filed: Feb. 3, 2006
Lead Inventor: Karen Bulock, CytRx
“The invention relates to methods for treating type 2 diabetes, obesity, metabolic syndrome, and conditions associated with these by administering an AICAR-monophosphate (AICAR-MP) enhancing agent that increases endogenous AICAR-MP levels in a cell,” the patent application’s abstract states. “Inhibition of AICAR-formyltransferase activity (AICARFT) in a cell that regulates metabolic activity (such as fat, liver, muscle, pancreatic beta, or certain brain cells) increases AICAR-monophosphate levels, which in turn results in activation of the AMP-kinase (AMPK) pathway, and all of the downstream functions mediated by AMPK including increased fatty acid oxidation, enhanced glucose transport, and decreased fatty acid synthesis.”

Title: Novel Prostate-Specific or Testis-Specific Nucleic Acid Molecules, Polypeptides, and Diagnostics and Therapeutic Methods
Number: 20070015246
Filed: June 15, 2006
Inventor: Fahri Saatcioglu, University of Oslo
“The invention provides novel STAMP2 nucleic acid molecules, polypeptides, antibodies, and modulatory compounds for use in methods of diagnosing, treating, and preventing diseases and conditions of the prostate and testis, such as cancer,” the patent application’s abstract states.

Title: Use of Eukaryotic Gene Affecting Cell Cycle Control or Cell Cycle Progression for Diagnosis and Treatment of Proliferative Diseases
Number: 20070015161
Filed: Sept. 15, 2004 PCT Filed: Sept. 15, 2004
Lead Inventor: Chris Echeverri, Cenix BioScience
The invention, the patent application’s abstract states, “relates to the significant functional role of several C. elegans genes and of their corresponding gene products in cell cycle progression during cell division that could be identified by means of RNA-mediated interference and to the identification and isolation of functional orthologs of said genes including all biologically functional derivatives thereof. The invention further relates to the use of said genes and gene products (including said orthologs) in the development or isolation of anti-proliferative agents, particularly their use in appropriate screening assays, and their use for diagnosis and treatment of proliferative and other diseases. In particular, the invention relates to the use of small interfering RNAs derived from said genes for the treatment of proliferative diseases.”

The Scan

Study Finds Few FDA Post-Market Regulatory Actions Backed by Research, Public Assessments

A Yale University-led team examines in The BMJ safety signals from the US FDA Adverse Event Reporting System and whether they led to regulatory action.

Duke University Team Develops Programmable RNA Tool for Cell Editing

Researchers have developed an RNA-based editing tool that can target specific cells, as they describe in Nature.

Novel Gene Editing Approach for Treating Cystic Fibrosis

Researchers in Science Advances report on their development of a non-nuclease-based gene editing approach they hope to apply to treat cystic fibrosis.

Study Tracks Responses in Patients Pursuing Polygenic Risk Score Profiling

Using interviews, researchers in the European Journal of Human Genetics qualitatively assess individuals' motivations for, and experiences with, direct-to-consumer polygenic risk score testing.