University of Massachusetts

University of Medicine and Dentistry of New Jersey

Sanford-burnham Medical Research Institute

University of California

Other

Ptc Therapeutics, Inc.

Sanford-burnham Medical Research Inslilute

Tariq M Rana

Natarajan Tamilarasu

Ikramul Huq

Mario Stevenson

Seongwoo Hwang

John E Hearst

Zuoshang Xu

Akbar Ali

Hong Cao

Matt Ban

Robin Scott Nathans

Disclosed herein, inter alia, are agents having antiviral activity and methods of use thereof.

Broad spectrum antiviral compounds and uses thereof

The present invention provides compositions for RNA interference and methods of use thereof. In particular, the invention provides small interfering RNAs (siRNAs) having modification that enhance the stability of the siRNA without a concomitant loss in the ability of the siRNA to participate in RNA interference (RNAi). The invention also provides siRNAs having modification that increase targeting efficiency. Modifications include chemical crosslinking between the two complementary strands of an siRNA and chemical modification of a 3' terminus of a strand of an siRNA. Preferred modifications are internal modifications, for example, sugar modification, nucleobase modification and/or backbone modifications. Such modifications are also useful, e.g., to improve uptake of the siRNA by a cell. Functional and genomic and proteomic methods are featured. Therapeutic methods are also featured.

In vivo gene silencing by chemically modified and stable siRNA

The present invention is based on the seminal discovery that several kinases play important roles in barrier pathways in somatic cell reprogramming. The present invention provides that modulating expression or activity of these kinases can significantly promote or enhance cell reprogramming efficiency. Key kinases are identified and key regulation networks involving such kinases are also identified that may be advantageously targeted to significantly increase reprogramming efficiency as well as direct differentiation of induced pluripotent stem (iPS) cells.

Methods for promoting cell reprogramming

Provided are compositions and methods for delivery of therapeutic agents, such as chemically stabilized antisense oligonucleotides useful in RNA silencing. The compositions include interfering nanoparticles (iNOPs) associated with one or more agents. Several functional iNOP derivatives are provided which allow for targeted delivery of agents to specific cell types as well as exhibiting reduced cellular toxicity.

Delivery of agents using interfering nanoparticles

The present invention is based on the seminal concept of combining genomics and chemical biology to identify new agents useful for induced pluripotent stem cell (iPSC) generation. The invention provides a method of generating an iPSC utilizing agents that antagonize a cell specific gene or upregulate expression or activity of a nuclear reprogramming gene, as well as a method of screening for such agents.

Method and compounds for generation of iPSCs

The present invention provides methods of carrying out the safe and reliable preparation of lipids comprising quaternary amines. Such lipids are especially suited for introducing therapeutic agents into cells or organisms. In particular, the lipids of the invention are suitable for the efficient transfer of gene therapy agents into mammalian cells or organisms in a cell type specific or tissue specific manner.

Methods and compositions for the efficient delivery of therapeutic agents to cells and animals

The invention features chemically modified small interfering RNAs (siRNAs) that are stable in vivo and retain the ability to form an A-form helix when in association with a target RNA. The features siRNA are effective therapeutics, particularly for targeting SOD1.

RNA interference agents for therapeutic use

The present invention provides compounds and compositions for inhibiting Vif and methods for treating viral infection, e.g., HIV infection.

Composition and synthesis of new reagents for inhibition of HIV replication

This invention relates to the use of oligourea molecules to specifically inhibit protein-nucleic acid interactions. In particular, it provides an oligourea molecule that competes with the Tat molecule for the TAR RNA of HIV-1. Also provided is a method specifically inhibiting protein-nucleic and interactions, and kits.

Tat-derived oligourea and its method of production and use in high affinity and specific binding HIV-1 TAR RNA

Site-specific modified proteins and method for producing site-specific modified proteins using amino acid analogs are disclosed. Methods for labeling proteins at a desired site in the presence of nucleophilic side chains, including lysine and cysteine side chains, are also disclosed. Methods for labeling the site-specific modified proteins are also disclosed.

Site-specific protein modification

The present invention relates to methods of screening for compounds that bind RNA molecules. In particular, the methods of the invention comprise screening a library of test compounds, each of which is attached to a solid support, with a dye-labeled RNA molecule to form a dye-labeled target RNA:support-attached test compound complex. By virtue of the dye label on the target RNA, the support becomes labeled and can be separated from unlabeled solid supports. The present invention further relates to methods of inhibiting an RNA-protein interaction, to methods of screening for compounds that increase or decrease the production of a protein, and to methods of screening for a compound that is capable of treating or preventing a disease whose progression is associated with an in vivo binding of a test compound to a target RNA.

Methods for identifying RNA binding compounds

The present invention relates to novel carbamates, ureas, and pharmaceutically acceptable salts thereof, compositions comprising the carbamate, urea, or a pharmaceutically acceptable salt thereof, and methods for treating or preventing cancer, inflammation, or a viral infection comprising administering to a patient in need of such treatment or prevention a therapeutically effective amount of the carbamate, urea, or pharmaceutically acceptable salt thereof.

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San Diego, CA, United States of America

Tariq M Rana