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IP Update: Recent Patents, Patent Applications Awarded to Alnylam; Opko; Harvard; and More


Title: Gene Silencing

Patent Number: 8,158,414

Filed: Sept. 23, 2005

Lead Inventor: Caius Rommens, JR Simplot

The invention, the patent's abstract states, “relates to unique strategies and constructs for producing a nucleic acid product that down-regulates or prevents expression of a desired target polynucleotide. Such construct may include an expressing cassette having two convergently-oriented promoters each operably linked to a polynucleotide, but not to a terminator. The two polynucleotides are fully identical in sequence over at least 23 nucleotides.”

Title: Lipid Formulation

Patent Number: 8,158,601

Filed: June 10, 2010

Lead Inventor: Jianxin Chen, Alnylam Pharmaceuticals

The invention, the patent's abstract states, comprises “an improved lipid formulation comprising a cationic lipid of [a predefined formula], and corresponding methods of use. Also disclosed are targeting lipids, and specific lipid formulations comprising such targeting lipids.”

Title: RNAi Modulation of RSV and Therapeutics Uses Thereof

Patent Number: 8,158,773

Filed: Jan. 28, 2008

Lead Inventor: Rachel Meyers, Alnylam Pharmaceuticals

The invention, the patent's abstract states, “is based on the in vivo demonstration that RSV can be inhibited through intranasal administration of iRNA agents, as well as by parenteral administration of such agents. Further, it is shown that effective viral reduction can be achieved with more than one virus being treated concurrently. Based on these findings, the ... invention provides general and specific compositions and methods that are useful in reducing RSV mRNA levels, RSV protein levels, and viral titers in a subject. .... These findings can be applied to other respiratory viruses.”

Title: RNAi Microbicides for Preventing and Treating Diseases

Application Number: 20120087973

Filed: March 23, 2011

Lead Inventor: Judy Lieberman, Harvard University

The invention relates to a “microbicidal composition comprising at least one siRNA,” the patent application's abstract states. “The siRNA is an RNA duplex made of one or two molecules. A portion of the siRNA is identical to a target sequence in an essential gene of a virus. The virus may be a herpesvirus, for example, HSV-1 or HSV-2. Preferably, the herpesvirus is HSV-2. The microbicidal composition further comprises a pharmaceutically acceptable carrier. Also included in the invention are methods to prevent and treat viral infections by administration of the microbicidal composition. Preferably, the microbicidal composition is administered transmucosally.”

Title: miRNAs as Therapeutic Targets in Cancer

Application Number: 20120087992

Filed: March 22, 2010

Lead Inventor: Jingfang Ju, Stony Brook University Medical Center

The invention comprises “methods for modulating expression of a component of a cell [by] contacting the cell with a nucleic acid comprising an miR-140 nucleic acid sequence in an amount sufficient to modulate the cellular component,” the patent application's abstract states. “Over-expression of miR-140 inhibits cell proliferation in both U-2 OS and HCT 116 cell lines. Cells transfected with miR-140 are more resistant to [the] chemotherapeutic agent methotrexate, [and] miR-140 expression is related to HDAC4 protein expression. The claimed methods reduce the protein expression level of HDAC4 without degrading the target mRNA.”

Title: Modified Oligonucleotide and Its Preparation and Application

Application Number: 20120088815

Filed: March 30, 2010

Inventor: Zicai Liang, Biomics Biotechnologies

The invention, the patent application's abstract states, relates to a “modified oligonucleotide, its preparation, and application. The invention enables stabilizing the oligonucleotide by introducing a relatively small amount of modified nucleotide at specific UA/UA ... CA/UG, and/or UG/CA site of the oligonucleotide, therefore to decrease the modification-related cytotoxicity and compromising effects on the biological activity.”

Title: Treatment of Antiviral Gene-Related Diseases by Inhibition of Natural Antisense Transcript to an Antiviral Gene

Application Number: 20120088817

Filed: May 28, 2010

Lead Inventor: Joseph Collard, Curna (Opko Health)

The invention relates to “antisense oligonucleotides that modulate the expression of and/or function of an antiviral gene, in particular, by targeting natural antisense polynucleotides of an antiviral gene,” according to the patent application's abstract. “The invention also relates to the identification of these antisense oligonucleotides and their use in treating diseases and disorders associated with the expression of antiviral genes.”

The Scan

Expanded Genetic Testing Uncovers Hereditary Cancer Risk in Significant Subset of Cancer Patients

In Genome Medicine, researchers found pathogenic or likely pathogenic hereditary cancer risk variants in close to 17 percent of the 17,523 patients profiled with expanded germline genetic testing.

Mitochondrial Replacement Therapy Embryos Appear Largely Normal in Single-Cell 'Omics Analyses

Embryos produced with spindle transfer-based mitochondrial replacement had delayed demethylation, but typical aneuploidy and transcriptome features in a PLOS Biology study.

Cancer Patients Report Quality of Life Benefits for Immune Checkpoint Inhibitors

Immune checkpoint inhibitor immunotherapy was linked in JAMA Network Open to enhanced quality of life compared to other treatment types in cancer patients.

Researchers Compare WGS, Exome Sequencing-Based Mendelian Disease Diagnosis

Investigators find a diagnostic edge for whole-genome sequencing, while highlighting the cost advantages and improving diagnostic rate of exome sequencing in EJHG.