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IP Update: Recent Patents, Patent Applications Awarded to Alnylam; USDA; Johns Hopkins; and More

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Title: Compositions and Methods for Inhibiting Expression of Huntingtin Gene

Patent Number: 8,080,532

Filed: April 2, 2009

Lead Inventor: Dinah Sah, Alnylam Pharmaceuticals

“The invention relates to a double-stranded ribonucleic acid for inhibiting the expression of the huntingtin gene comprising an antisense strand having a nucleotide sequence which is less than 25 nucleotides in length and which is substantially complementary to at least a part of the ... gene,” the patent's abstract states. “The invention also relates to a pharmaceutical composition comprising the dsRNA together with a pharmaceutically acceptable carrier; methods for treating diseases caused by the expression of the [huntingtin] gene, or a mutant form thereof, using the pharmaceutical composition; and methods for inhibiting the expression of the huntingtin gene in a cell.”


Title: Targeting PAX2 for the Treatment of Breast Cancer

Patent Number: 8,080,534

Filed: Feb. 18, 2010

Inventor: Carlton Donald, Phigenix

The invention comprises “methods of prevention and/or treatment of breast cancer in a subject by inhibiting expression of PAX2,” according to the patent's abstract. “In the certain embodiments, the method of inhibiting expression of PAX2 is to administrate the subject a nucleic acid encoding an siRNA for PAX2.”


Title: Pesticidal Double-Stranded RNA Composition and Method of Use Thereof

Patent Number: 8,080,648

Filed: March 9, 2007

Lead Inventor: Julia Pridgeon, US Department of Agriculture

The invention, the patent's abstract states, comprises a “pesticidal compound that regulates programmed cell death pathways via the topical application of double-strand RNA. ... The compound is constructed as a specific-target pesticide that is a biosafe nucleic acid pesticide for pest control.”


Title: Methods for Expansion of Hematopoietic Stem and Progenitor Cells

Application Number: 20110305675

Filed: Jan. 21, 2010

Lead Inventor: David Scadden, Massachusetts General Hospital

The patent application claims “methods and compositions for expanding hematopoietic stem and progenitor cells ex vivo and in vivo, and methods and compositions for transplanting HSPCs and treatment of anemia in a human subject,” its abstract states. “HSPCs are expanded in the presence of an inhibitor of serine threonine kinase 38, wherein said inhibitor is preferably an RNAi agent.”


Title: Compositions and Methods for Ex Vivo Hepatic Nucleic Acid Delivery

Application Number: 20110305772

Filed: Feb. 26, 2010

Inventor: Andrew Cameron, Johns Hopkins University

“The invention provides compositions and methods for delivery of nucleic acids to the liver ex vivo,” according to the patent application's abstract. “The method includes slow, low pressure infusion of the nucleic acid into the liver to efficiently transduce cells with minimal cell damage.”


Title: RNA Interference-Mediating Small RNA Molecules

Application Number: 20110306651

Filed: July 12, 2010

Lead Inventor: Thomas Tuschl, Max Planck Institute

“Double-stranded RNA induces sequence-specific post-transcriptional gene silencing in many organisms by a process known as RNA interference,” the patent application's abstract states. “Using a Drosophila in vitro system, we demonstrate that [19 to 23 nucleotide-long] short RNA fragments are the sequence-specific mediators of RNAi. The short interfering RNAs are generated by an RNase III-like processing reaction from long dsRNA. Chemically synthesized siRNA duplexes with overhanging 3' ends mediate efficient target RNA cleavage in the lysate, and the cleavage site is located near the center of the region spanned by the guiding siRNA. Furthermore, we provide evidence that the direction of dsRNA processing determines whether sense or antisense target RNA can be cleaved by the produced siRNP complex.”

The Scan

Drug Response Variants May Be Distinct in Somatic, Germline Samples

Based on variants from across 21 drug response genes, researchers in The Pharmacogenomics Journal suspect that tumor-only DNA sequences may miss drug response clues found in the germline.

Breast Cancer Risk Gene Candidates Found by Multi-Ancestry Low-Frequency Variant Analysis

Researchers narrowed in on new and known risk gene candidates with variant profiles for almost 83,500 individuals with breast cancer and 59,199 unaffected controls in Genome Medicine.

Health-Related Quality of Life Gets Boost After Microbiome-Based Treatment for Recurrent C. Diff

A secondary analysis of Phase 3 clinical trial data in JAMA Network Open suggests an investigational oral microbiome-based drug may lead to enhanced quality of life measures.

Study Follows Consequences of Early Confirmatory Trials for Accelerated Approval Indications

Time to traditional approval or withdrawal was shorter when confirmatory trials started prior to accelerated approval, though overall regulatory outcomes remained similar, a JAMA study finds.