Genentech, Inc.

Shire Human Genetic Therapies, Inc.

Takeda Pharmaceutical Company Limited

Kiniksa Pharmaceuticals, Ltd.

Zahra Shahrokh

Pericles Calias

Jing Pan

Teresa Leah Wright

Gaozhong Zhu

Iphigenia Koumenis

Steven R Leong

Vanessa Hsei

Gerardo A Zapata

Lawrence Charnas

Kris Lowe

Richard Pfeifer

Roger Pai

Leonard G Presta

Thomas Mccauley

Vinh Nguyen

Jan Powell

Heather L Flores

Tanya P Lin

Timothy C Matthews

Nazila Salamat-Miller

Katherine Taylor

Paul Campolieto

James Christian

Mark Cornell Manning

Ryan Erik Holcomb

Farah Natoli

Jennifer Terew

Yuan Jiang

Jamie Tsung

Brian Vernaglia

Dave Nichols

Phillip M Levesque

Avi J Ashkenazi

Robert F Kelley

Xanthe M Lam

Rita L Wong

James Q Oeswein

Woon-Lam Susan Leung

Laura C Simmons

Richard L Fahrner

Sarah G Hymowitz

Sujit Basu

Sharon X Wang

Mark O'Connell

Boonsri Ongpipattanakul

Robert P Weissburg

Richard Fahrner

Rick Fahrner

Richard Fahmer

Evan E Shave

Patricia A Rancatore

Gerarado A Zapata

Philip M Levesque

Keethkumar Jain

Iphegenia Koumenis

Susan Leung

The present invention provides an effective and less invasive approach for direct delivery of therapeutic agents to the central nervous system (CNS). In some embodiments, the present invention provides methods including a step of administering intrathecally to a subject suffering from or susceptible to a lysosomal storage disease associated with reduced level or activity of a lysosomal enzyme, a composition comprising a replacement enzyme for the lysosomal enzyme.

CNS delivery of therapeutic agents

The present invention provides, among other things, compositions and methods for CNS delivery of lysosomal enzymes for effective treatment of lysosomal storage diseases. In some embodiments, the present invention includes a stable formulation for direct CNS intrathecal administration comprising an arylsulfatase A (ASA) protein, salt, and a polysorbate surfactant for the treatment of Metachromatic Leukodystrophy Disease.

Methods and compositions for CNS delivery of arylsulfatase A

The present invention provides, among other things, compositions and methods for CNS delivery of lysosomal enzymes for effective treatment of lysosomal storage diseases. In some embodiments, the present invention includes a stable formulation for direct CNS intrathecal administration comprising a heparan N-sulfatase (HNS) protein, salt, and a polysorbate surfactant for the treatment of Sanfilippo Syndrome Type A.

Methods and compositions for CNS delivery of heparan N-sulfatase

The present invention provides, among other things, compositions and methods for CNS delivery of lysosomal enzymes for effective treatment of lysosomal storage diseases. In some embodiments, the present invention includes a stable formulation for direct CNS intrathecal administration comprising an iduronate-2-sulfatase (I2S) protein, salt, and a polysorbate surfactant for the treatment of Hunters Syndrome.

Methods and compositions for CNS delivery of iduronate-2-sulfatase

The present invention provides, among other things, stable formulations comprising an anti-oncostatin M receptor (OSMR) antibody and having a pH ranging from approximately 6.0-7.6, wherein less than approximately 5% of the anti-OSMR antibody exists as high molecular weight (HMW) species in the formulation.

Stable anti-OSMR antibody formulation

Methods and compositions for CNS delivery of heparan n-sulfatase

The present invention provides, among oilier things, compositions and methods for CNS delivery of lysosomal enzymes (e.g., recombinant human arylsulfatase A (rhASA)) for effective treatment of lysosomal storage diseases (e.g. Metachromatic Leukodystrophy Disease), In some embodiments, the present invention includes a stable formulation for intrathecal administration comprising an ASA protein and a poloxamer, wherein less than 3% of the ASA protein exists in aggregated form.

Stable formulations for CNS delivery of arylsulfatase A

Compositions of proteins having free thiols, and methods of making and using such compositions, are described.

Stabilized compositions of proteins having a free thiol moiety

Growing community of inventors

Weston, MA, United States of America

Zahra Shahrokh