Genzyme Corporation

Ionis Pharmaceuticals, Inc.

The Brigham and Women's Hospital, Inc.

Sanofi

Isis Pharmaceuticals, Inc.

Cold Spring Harbor Laboratory

University of Iowa Research Foundation

Biogen Ma Inc.

Genezyme Corporation

University of Rochester

Erytech Pharma

Seng H Cheng

Lamya S Shihabuddin

John Marshall

Ronald K Scheule

David J Harris

Edward R Lee

Nelson S Yew

Simon J Eastman

Craig S Siegel

Marco A Passini

Canwen Jiang

C Frank Bennett

Gene Hung

Susan M Freier

Yunxiang Zhu

Holly Kordasiewicz

Don W Cleveland

Lisa Stanek

James C Dodge

Hongmei Zhao

Mathieu B Lane

Richard J Gregory

Alan E Smith

Ronnie Rong Wei

Thomas A Natoli

Shirley C Hubbard

Chau-Dung Chang

Michael Schlossmacher

Valerie Cullen

Luis Z Avila

John P Leonard

Rebecca G Bagley

Jozsef Karman

Andrew Charles Good

Frank Rigo

Ercole Rao

Christian Lange

Bradford Hirth

Yibin Xiang

Catherine R O'Riordan

Cassandra Celatka

William Harold Brondyk

Timothy D Connors

Markus Metz

Adrian R Krainer

Christian Beil

Catherine Prades

Zhong Zhao

Katherine W Klinger

Elyse Bourque

Abizer I Harianawala

Hanlan Liu

Elina Makino

Oxana Ibraghimov-Beskrovnaya

Bruce M Wentworth

Yimin Hua

Diane P Copeland

Oxana Beskrovnaya

Eric A Rowe

Yangde Chen

Andrew Leger

Catherine Devaud

Katherine Jancsics

Cynthia Marie Arbeeny

Maureen Magnay

David Meeker

Tristan Magnay

Herve Husson

Renu Bhardwaj

Leila Sevigny

Jianmei Kochling

Judith Peterschmitt

S Catherine Hubbard

Henry Hoppe, Iv

Shaona Lee Fang

Dietmar Hoffmann

Mario A Cabrera-Salazar

H Michael Shepard

Michael J Welsh

Renato Skerlj

Harry M Meade

Robert A MacLeod

Yann Godfrin

Donna Armentano

Daniel C Maneval

Timothy E Weeden

Carol A Nelson

Sanjay K Pandey

Paul Ditullio

Renato Tony Skerlj

Karl M Ebert

Nicholas P Clayton

Charles A Thornton

Sergio Pablo Sardi

Thurman Wheeler

Kathleen M Hehir

Chris Willis

Allen J Fasbender

Sucharita Paul

Jennifer D Tousignant

Compositions and methods for treating disorders affecting motor function, such as motor function affected by disease or injury to the brain and/or spinal cord, are disclosed.

Gene therapy for neurodegenerative disorders

Provided herein are methods, compounds, and compositions for reducing expression of huntingtin mRNA and protein in an animal. Such methods, compounds, and compositions are useful to treat, prevent, delay, or ameliorate Huntington's disease, or a symptom thereof.

Modulation of huntingtin expression

The invention relates to inhibitors of glucosylceramide synthase (GCS) of the Formula I: represented by the following structural formula or a pharmaceutically acceptable salt thereof, wherein the variables are as defined herein, useful for the treatment of metabolic diseases, such as lysosomal storage diseases, either alone or in combination with enzyme replacement therapy, cystic disease and for the treatment of cancer.

Glucosylceramide synthase inhibitors

The present invention provides a bispecific biologic comprising a ligand specific for CTLA-4 and a ligand specific for a pMHC complex.

Inhibitors of T-cell activation

Modulation of Huntington expression

This disclosure provides methods and compositions for treating lysosomal storage diseases in a subject. In one aspect of the invention, a transgene product is delivered to a subject by administering a recombinant neurotrophic viral vector containing the transgene to the brain. The viral vector delivers the transgene to a region of the brain which is susceptible to infection by the virus and which expresses the encoded recombinant viral gene product. Also provided are compositions for delivery of a transgene product to a subject by administering a recombinant neurotrophic viral vector containing the transgene to the subject's brain. The transgene product may be any that is deficient in a lysosomal storage disease.

Gene therapy for lysosomal storage diseases

Provided herein multispecific (e.g., bispecific) binding molecules comprising a first binding domain that binds an extracellular portion of dystroglycan and a second binding domain that binds laminin-2. Further provided herein are methods for making such binding molecules and uses of such binding molecules for treating and/or preventing alpha-dystroglycanopathies.

Multispecific binding molecules having specificity to dystroglycan and laminin-2

Neurological diseases, including lysosomal storage diseases, can be successfully treated using intraventricular delivery of the therapeutic agents to bypass the blood-brain barrier. Similarly, diagnostic agents and anesthetic agents can be delivered to the brain in this manner. The administration can be performed slowly to achieve maximum effect. Such administration permits greater penetration of distal portions of the brain.

Slow intraventricular delivery

The invention relates to inhibitors of glucosylceramide synthase (GCS), such as Compound of Formula I, shown below, as defined herein, useful for the treatment of metabolic diseases, such as lysosomal storage diseases, either alone or in combination with enzyme replacement therapy, cystic disease and for the treatment of cancer. 

The invention provides methods for the synthesis of oligosaccharides comprising an aminooxy group. The invention further provides oligosaccharides comprising an aminooxy group, methods for coupling oligosaccharides comprising an aminooxy group to glycoproteins, and oligosaccharide-protein conjugates. Also provided are methods of treating a lysosomal storage disorder in a mammal by administration of an oligosaccharide-protein conjugate.

Oligosaccharides comprising an aminooxy group and conjugates thereof

The present provides methods for treating spinal muscular atrophy using a self-complementary recombinant adeno-associated virus (rAAV) viral particle comprising a transgene expressing SMN. In one aspect, the viral particles are administered the spinal column or cisterna magna in a human subject; for example, a pediatric human subject. Viral particles comprising AAV9 capsids are contemplated.

Compositions and methods for treating spinal muscular atrophy

Growing community of inventors

Natick, MA, United States of America

Seng H Cheng