Genzyme Corporation

Sanofi

Modernatx, Inc.

Patrick Finn

Clark Qun Pan

Luis Z Avila

Robert J Miller

James E Stefano

Yunxiang Zhu

Qun Zhou

Michael Reardon

Lauren Young

Paul A Konowicz

Xiaoyang Zheng

Joseph P Kutzko

Andreas Peer

Duncan E Paterson

John Harrahy

Huawei Qiu

Kevin Brower

Christopher Winter

Marcella Yu

Joachim Theilhaber

Rao Koduri

Richard C Gregory

Parminder Mankoo

Natalia Malkova

Gary Shapiro

Jack R Pollard

Feng Song Liu

Paolo G V Martini

Vladimir Presnyak

Athanasios Dousis

Kanchana Ravichandran

Ding An

Kristina An Haack

Feng Liu

Raj Rajendran

Catherine Wilson

Zhijian Zhuo

Andrea Lea Frassetto

Michael R Reardon

Duncan Patterson

Paolo GV Martini

Duncan E Patterson

The present application provides methods of treating Pompe disease such as infantile-onset Pompe disease (IOPD) using a pharmaceutical composition comprising an oligosaccharide-acid α-glucosidase (GAA) conjugate, such as avalglucosidase alfa. Also provided are formulations of the oligosaccharide-GAA conjugates.

Compositions and methods for treating Pompe disease

Provided herein are conjugates comprising a protein and an oligosaccharide of one of Formulae I-VI. Also provided herein are pharmaceutical compositions comprising such conjugates. Further provided herein are methods of treating a lysosomal storage disorder in a mammal by administration of an oligosaccharide-glycoprotein conjugate.

Oligosaccharide-protein conjugates

The invention provides an improved pan-TGF-β antibody for treatment of conditions that are mediated by TGF-β, including autoimmune diseases, fibrotic conditions, and cancers. Also provided are methods and uses of the antibody in conjunction with other immunomodulatory agents such as an anti-PD-1 antibody.

Anti-TGF-beta antibodies and their use

This disclosure relates to mRNA therapy for the treatment of hyperphenylalaninemias such as phenylketonuria (PKU). mRNAs for use in the invention, when administered in vivo, encode human phenylalanine hydroxylase (PAH), functional fragments thereof (e.g., those comprising the catalytic domain or the catalytic domain and the tetramerization domains), and fusion proteins comprising PAH. mRNAs of the invention are preferably encapsulated in lipid nanoparticles (LNPs) to effect efficient delivery to cells and/or tissues in subjects, when administered thereto. mRNA therapies of the invention increase and/or restore deficient levels of PAH expression and/or activity in subjects. mRNA therapies of the invention further decrease abnormal accumulation of phenylalanine associated with deficient PAH activity in subjects.

Polynucleotides encoding phenylalanine hydroxylase for the treatment of phenylketonuria

This disclosure relates mRNA therapy for the treatment of ornithine transcarbamylase deficiency (OTCD). mRNAs for use in the invention, when administered in vivo, encode human ornithine transcarbamylase (OTC), isoforms thereof, functional fragments thereof, and fusion proteins comprising OTC. mRNAs of the invention are preferably encapsulated in lipid nanoparticles (LNPs) to effect efficient delivery to cells and/or tissues in subjects, when administered thereto. mRNA therapies of the invention increase and/or restore deficient levels of OTC expression and/or activity in subjects. mRNA therapies of the invention further decrease levels of toxic ammonia associated with deficient OTC activity in subjects.

Polynucleotides encoding ornithine transcarbamylase for the treatment of urea cycle disorders

The invention provides an improved pan-TGF-&#x3b2; antibody for treatment of conditions that are mediated by TGF-&#x3b2;, including autoimmune diseases, fibrotic conditions, and cancers. Also provided are methods and uses of the antibody in conjunction with other immunomodulatory agents such as an anti-PD-1 antibody.

Anti-TGF-&#x3b2; antibodies and their use

Growing community of inventors

Franklin, MA, United States of America

Patrick Finn