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IP Update: Recent Patents, Patent Applications Awarded to Alnylam, Rosetta, Dicerna, and More

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Title: Pharmaceutical Composition of Nanoparticles

Patent Number: 8,449,915

Filed: Dec. 8, 2010

Lead Inventor: Hsing-Wen Sung, GP Medical

The invention relates to “a pharmaceutical composition of multifunctional core-shell nanoparticles, each nanoparticle comprising a core portion comprised of hydrophobic poly(D,L-lactic-co-glycolic acid) (PLGA) and a detection-enhancing substance; and a shell portion comprised of positively charged chitosan adapted to cause no aggregation of nanoparticles due to electrostatic repulsion between the positively charged nanoparticles,” the patent’s abstract states.


Title: Compositions and Methods for Therapy of Macular Degeneration

Patent Number: 8,450,473

Filed: April 30, 2010

Inventor: Jack Sullivan, State University of New York

The patent, its abstract states, claims “compositions and methods for therapy of macular degeneration. … The method entails administering … [a] polynucleotide that can facilitate a reduction in the amount of rod opsin mRNA … a second polynucleotide that can facilitate a reduction in the amount of RPE65 mRNA … or a combination thereof. The polynucleotides of the invention are hammerhead ribozymes or shRNAs. The polynucleotides target a sequence in [rod opsin] mRNA or RPE65 mRNA and facilitate reduction in the target mRNA via ribozymatic cleavage of the target, or by hybridization to the target, which leads to RNAi mediated degradation of the target mRNA.”


Title: Methods and Compositions for Delivery of Active Agents

Application Number: 20130129785

Filed: May 10, 2011

Inventor: Muthiah Manoharan, Alnylam Pharmaceuticals

The invention comprises a lipid particle, including a cationic lipid, the patent application’s abstract states. “Synthesis of the cationic lipid can include a ylide-based reaction, such as a Wittig reaction or sulfur ylide reaction. In some cases, the synthesis can also include a Michael addition or a related addition reaction.”


Title: Bioinformatically Detectable Group of Novel Viral Regulatory Genes and Uses Thereof

Application Number: 20130130231

Filed: Nov. 26, 2002

Inventor: Isaac Bentwich, Rosetta Genomics

The invention relates to a group of “novel viral RNA regulatory gene … [that] selectively inhibit translation of known host target genes, and are believed to represent a novel pervasive viral attack mechanism,” according to the patent application’s abstract. “Several nucleic acid molecules are provided … encoding several [of the] genes, as are vectors and probes both comprising the nucleic acid molecules, and methods and systems for detecting [the] genes, and for counteracting their activity.”


Title: Programmable Oligonucleotide Synthesis

Application Number: 20130130321

Filed: Jan. 14, 2013

Lead Inventor: Peer Staehler, Synthetic Genomics

The invention relates to “methods and devices for preparing synthetic nucleic acids,” according to the patent application’s abstract. Included in these are ones that modulate gene expression via RNAi.


Title: Novel siRNA Structure for Minimizing Off-Target Effects Caused by Antisense Strands and Use Thereof

Application Number: 20130130377

Filed: Nov. 4, 2010

Lead Inventor: Dong-ki Lee, Sungkyunkwan University (Biomolecular Therapeutics)

The invention relates to a “double-stranded siRNA molecule comprising an antisense strand and a sense strand, wherein the siRNA molecule has at least one single nucleotide bulge formed by introducing a single nucleotide into the antisense strand, particularly at position 2 from the 5' end, and to a method of using the same to silence a target gene,” the patent application’s abstract states. “The siRNA molecule of the invention shows high target gene silencing efficiency while minimizing off-target effects caused by the antisense strand, and thus has improved target selectivity. Accordingly, the siRNA molecule of the invention can be substituted for conventional siRNA molecules and can be widely be used in siRNA-based gene-silencing techniques, including gene therapy.”


Title: Oligonucleotides Comprising Acyclic and Abasic Nucleosides and Analogs

Application Number: 20130130378

Filed: April 22, 2011

Lead Inventor: Muthiah Manoharan, Alnylam Pharmaceuticals

The invention relates to “acyclic and abasic nucleosides and oligonucleotides prepared therefrom,” the patent application’s abstract states.


Title: Method for Inhibiting HIV Replication in Mammalian and Human Cells

Application Number: 20130130971

Filed: April 1, 2011

Lead Inventor: Celia Berta Fernandez Ortega, Center for Genetic Engineering and Biotechnology of Cuba

The invention comprises a “method to inhibit replication of the human immunodeficiency virus by negatively modulating or altering the cytoskeleton, more precisely the proteins forming the intermediate cytoskeletal filaments … vimentin and/or keratin-10,” the patent application’s abstract states. “The … invention is also related to the use of … peptides and/or interfering RNA and/or lipidic compounds … [to produce] a negative modulation or alteration of the cytoskeleton to prevent or to treat the HIV infection.“


Title: Reduced Size Self-delivering RNAi Compounds

Application Number: 20130131141

Filed: March 24, 2011

Lead Inventor: Anastasia Khvorova, RXi Pharmaceuticals

The invention relates to “methods for in vivo administration of sd-rxRNA molecules,” the patent application’s abstract states.


Title: RNA Interference in Ocular Indications

Application Number: 20130131142

Filed: March 24, 2011

Lead Inventor: Lyn Libertine, RXi Pharmaceuticals

The invention relates to “ocular administration of sd-rxRNA and rxRNAori molecules,” the patent application’s abstract states.


Title: RTP801 siRNA Compounds and Methods of Uses Thereof

Application Number: 20130131143

Filed: Oct. 12, 2012

Lead Inventor: Elena Feinstein, Quark Pharmaceuticals

“The invention provides chemically modified siRNA oligonucleotides that target RTP801L, compositions comprising same, and to the use of such molecules to treat … respiratory diseases including acute and chronic pulmonary disorders, eye diseases including glaucoma and ION, microvascular disorders, angiogenesis- and apoptosis-related conditions, and hearing impairments,” the patent application’s abstract states.


Title: Compositions and Methods for Altering RNAi

Application Number: 20130131145

Filed: Nov. 5, 2012

Lead Inventor: Richard Carthew, Northwestern University

The invention, the patent application’s abstract states, “relates to compositions and methods for altering RNAi. In particular, the … invention provides systems and methods for identifying regulators of RNAi … [as well as] methods of identifying inhibitors of RNAi regulators and methods of using the same.”


Title: Methods and Compositions for the Specific Inhibition of Androgen Receptor by Double-stranded RNA

Application Number: 20130131149

Filed: Dec. 21, 2012

Lead Inventor: Bob Brown, Dicerna Pharmaceuticals

“This invention relates to compounds, compositions, and methods useful for reducing [androgen receptor] target RNA and protein levels via use of dsRNAs,” the patent application’s abstract states.

The Scan

Shape of Them All

According to BBC News, researchers have developed a protein structure database that includes much of the human proteome.

For Flu and More

The Wall Street Journal reports that several vaccine developers are working on mRNA-based vaccines for influenza.

To Boost Women

China's Ministry of Science and Technology aims to boost the number of female researchers through a new policy, reports the South China Morning Post.

Science Papers Describe Approach to Predict Chemotherapeutic Response, Role of Transcriptional Noise

In Science this week: neural network to predict chemotherapeutic response in cancer patients, and more.