Inotek Pharmaceuticals Corporation

Shire Human Genetic Therapies, Inc.

Takeda Pharmaceutical Company Limited

Archemix Corporation

Thomas Mccauley

William K McVicar

Zahra Shahrokh

John Louis Diener

Norman N Kim

Pericles Calias

Teresa Leah Wright

Richard Pfeifer

Prakash Jagtap

Martin Stanton

Jing Pan

Charles Wilson

David Epstein

Lawrence Charnas

Jan Powell

Ann Barbier

Dilara Grate

Charles W Richard, Iii

Rudolf A Baumgartner

Brian Felice

Hongsheng Xie

Dilârâ McCauley

Errol Desouza

Charles W Iii Richard

William K Mcvicar

Jeffrey Preiss

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 a method of treating cognitive impairment of Hunter syndrome. Among other things, the present invention provides a method comprising a step of administering intrathecally to a subject in need of treatment a recombinant iduronate-2-sulfatase (I2S) enzyme at a therapeutically effective dose and an administration interval for a treatment period sufficient to improve, stabilize or reduce declining of one or more cognitive, adaptive, motor, and/or executive functions relative to a control.

Treatment of cognitive impairment of hunter syndrome by intrathecal delivery of iduronate-2-sulfatase

Treatment of cognitive impairment of Hunter syndrome by intrathecal delivery of iduronate-2-sulfatase

The present invention provides a method of treating cognitive impairment of Hunter syndrome. Among other things, the present invention provides a method comprising a step of administering intrathecally to a subject in need of treatment a recombinant iduronate-2-sulfatase (I2S) enzyme at a therapeutically effective dose and an administration interval for a treatment period sufficient to improve, stabilize or reduce declining of one or more cognitive, adaptive, motor, and/or executive functions relative to a control.

The present invention provides, among other things, compositions, kits and methods for subcutaneous delivery of lysosomal enzymes for effective treatment of lysosomal storage diseases. In some embodiments, the present invention provides methods for treating Hunter syndrome by subcutaneous administration of a replacement iduronate-2-sulfatase (I2S) protein. In some embodiments, the present invention provides a kit comprising an arrangement of components for subcutaneously administering iduronate-2-sulfatase (I2S) protein.

Subcutaneous administration of iduronate-2-sulfatase

The present invention is directed to a composition or a kit comprising a combination of a prostaglandin analog and an adenosine receptor Aagonist and to a method of reducing intraocular pressure (IOP) in a subject using such combination or kit. In some embodiments, the adenosine receptor Aagonist is Compound A, ((2R,3S,4R,5R)-5-(6 -(cyclopentylamino)-9H-purin-9-yl)-3,4-dihydroxytetrahydrofuran-2-yl)methyl nitrate, having the structure, 

Combination, kit and method of reducing intraocular pressure

Provided herein is a method of reducing intraocular pressure (IOP) in humans using N6-cyclopentyladenosine (CPA), CPA derivatives or prodrugs or enhanced cornea permeability formulations of CPA. In one embodiment, the invention is directed to CPA derivatives or prodrugs that are permeable to the cornea. In another embodiment, the invention is directed to uses of certain compounds in human subjects for reducing and/or controlling elevated or abnormally fluctuating IOPs in the treatment of glaucoma or ocular hypertension (OHT).

Method of reducing intraocular pressure in humans

The present invention is directed to a combination or a kit comprising a prostaglandin analog and an adenosine receptor Aagonist and to a method of reducing intraocular pressure (IOP) in a subject using such combination or kit. In one embodiment, the prostaglandin analog is latanoprost and the adenosine receptor Aagonist is Compound A, ((2R,3S,4R,5R)-5-(6-(cyclopentylamino)-9H-purin-9-yl)-3,4-dihydroxytetrahydrofuran-2-yl)methyl nitrate, having the structure, 

Provided herein is a pharmaceutical composition or a kit comprising a combination of a non-selective beta-adrenergic receptor blocker and an adenosine Areceptor agonist. Also provided herein is a method of reducing intraocular pressure (IOP) in a subject using such a combination or kit. In a particular embodiment, provided herein is a combination of timolol marketed under the brand Timoptic&#x2122; and Compound A.

Combination compositions for reducing intraocular pressure

Provided herein is a pharmaceutical composition or a kit comprising a combination of a carbonic anhydrase inhibitor analog and an adenosine Areceptor agonist. Also provided herein is a method of reducing intraocular pressure (IOP) in a subject using such a combination or kit. In a particular embodiment, provided herein is a combination of dorzolamide marketed under the brand Trusopt&#x2122; and Compound A.

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Cambridge, MA, United States of America

Thomas Mccauley