Biogen, Incorporated

Other

Synta Pharmaceuticals Corporation

New England Medical Center Hospitals. Inc.

Biogen Idec Ma Inc.

Translate Bio, Inc.

Madrigal Pharmaceuticals, Inc.

James G Barsoum

R Blake Pepinsky

Alan Frankel

Carl Pabo

Stephen E Fawell

Howard P Ng

Weiwen Ying

Dinesh U Chimmanamada

Shijie Zhang

Teresa Przewloka

Kevin Paul Foley

Zhenjian Du

David James

Elena Kostik

Elliot J Androphy

Junghyun Chae

Chi-Wan Lee

Frederick M Boyce

Kevin M Foley

Arthur Mertz Krieg

Yumiko Wada

Rongzhen Lu

Michael Parr

Albert X Qin

Xiao-Qiang Qin

Shije Zhang

Methods for modulating expression of SMN1 and/or SMN2 in cells obtained from subjects having ALS or in subjects having ALS using single stranded oligonucleotides are provided. Methods for treating ALS using single stranded oligonucleotides are also provided.

Compositions and methods for treating amyotrophic lateral sclerosis

The present invention is directed to compositions and methods for modulating c-Rel-dependent cytokine production without materially altering the level of expression of NF&#x3ba;B and/or the amount of I&#x3ba;B. The present invention is also directed to screening for modulators of c-Rel activity as determined by assaying for altered subcellular localization of c-Rel but where the level of expression of NF&#x3ba;B and/or the amount of I&#x3ba;B is materially unaltered.

Compositions and methods for modulating c-Rel-dependent cytokine production

The present invention relates to substituted triazole compounds and compositions comprising substituted triazole compounds. The invention further relates to methods of inhibiting the activity of Hsp90 in a subject in need thereof and methods for preventing or treating hyperproliferative disorders, such as cancer, in a subject in need thereof comprising administering to the subject a substituted triazole compound of the invention, or a composition comprising such a compound.

Triazone compounds that modulate HSP90 activity

Triazole compounds that modulate Hsp90 activity

Triazole compounds that modulate HSP90 activity

A method of treating a Hsp70-responsive disorder in a subject includes administering to the subject an effective amount of a compound represented by Structural Formula I, or a pharmaceutically acceptable salt or solvate thereof.  Y is a covalent bond or an optionally substituted straight chained hydrocarbyl group, or, Y, taken together with both &#x3e;C&#x2550;Z groups to which it is bonded, is an optionally substituted aromatic group. R-Rare independently &#x2014;H, an optionally substituted aliphatic group, an optionally substituted aryl group, or Rand Rtaken together with the carbon and nitrogen atoms to which they are bonded, and/or Rand Rtaken together with the carbon and nitrogen atoms to which they are bonded, form a non-aromatic heterocyclic ring optionally fused to an aromatic ring. R-Rare independently &#x2014;H, an optionally substituted aliphatic group, or an optionally substituted aryl group. Z is O or S.

Bis(thio-hydrazide amides) for increasing Hsp70 expression

The invention provides methods and compositions for increasing the delivery of nucleic acids into a host by administering a nucleic acid encoding a therapeutic nucleic acid along with an agent that modulates Kupffer cell function in the host.

Method of enhancing delivery of a therapeutic nucleic acid

Methods and pharmaceutical compositions for modifying cells of a mammalian recipient with DNA encoding a secreted protein such as human interferon in situ are provided. The methods include forming a secreted protein expression system in vivo or ex vivo and administering the expression system to the mammalian recipient. The expression system and methods are useful for the localized and systemic delivery of interferons in situ.

Methods and compositions for therapies using genes encoding secreted proteins such as interferon-beta

Disclosed are methods, nucleic acids, and cells for expressing an exogenous gene in a mammalian cell, involving introducing into the cell a non-mammalian DNA virus (e.g., a baculovirus) having an altered coat protein, the genome of which virus carries an exogenous gene, and growing the cell under conditions such that the gene is expressed. Also disclosed are methods for treating gene deficiency disorders, neurological disorders, or cancers in a mammal by (1) providing to a cell a therapeutically effective amount of a non-mammalian DNA virus having an altered coat protein, the genome of which virus carries an exogenous, therapeutic gene and (2) growing the cell under conditions such that the exogenous gene is expressed in the mammal.

Expression of an exogenous gene in a mammalian cell by use of a non-mammalian DNA virus having an altered coat protein

A protocol for the ion exchange concentration of baculovirus is presented.

Chromatographically concentrated baculovirus and methods

This invention relates to delivery of biologically active cargo molecules, such as polypeptides and nucleic acids, into the cytoplasm and nuclei of cells in vitro and in vivo. Intracellular delivery of cargo molecules according to this invention is accomplished by the use of novel transport polypeptides which include HIV tat protein or one or more portions thereof, and which are covalently attached to cargo molecules. The transport polypeptides in preferred embodiments of this invention are characterized by the presence of the tat basic region (amino acids 49-57), the absence of the tat cysteine-rich region (amino acids 22-36) and the absence of the tat exon 2-encoded carboxy-terminal domain (amino acids 73-86) of the naturally-occurring tat protein. By virtue of the absence of the cysteine-rich region, the preferred transport polypeptides of this invention solve the potential problems of spurious trans-activation and disulfide aggregation. The reduced size of the preferred transport polypeptides of this invention also minimizes interference with the biological activity of the cargo molecule.

Tat-derived transport polypeptides

Growing community of inventors

Lexington, MA, United States of America

James G Barsoum