IP Library › Granted Patent US 12,409,210
Granted Patent B2
US 12,409,210 · App. 17/820,155 · Granted Sep 9, 2025

CNS delivery of therapeutic agents

Inventors: Pericles Calias (Melrose, MA); Jing Pan (Boxborough, MA); Jan Powell (Concord, MA); Lawrence Charnas (Natick, MA); Thomas McCauley (Cambridge, MA); Teresa Leah Wright (Lexington, MA); Richard Pfeifer (North Granby, CT); Zahra Shahrokh (Weston, MA)
Assignee: Takeda Pharmaceutical Company Limited
A61K38/47A61K9/0019A61K9/0085A61K9/08A61K9/19A61K35/76A61K35/761A61K38/46A61K38/465A61K47/02A61K47/26C07K14/65C12N9/2402C12N9/2437C12Y301/06008C12Y301/06013C12Y302/01045C12Y302/01046C12Y302/0105C12Y310/01001
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Quick Facts
Patent No.
US 12,409,210
App. No.
17/820,155
Granted
Sep 9, 2025
Kind
B2
Abstract

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.

Claims (5)

1. A formulation comprising a lysosomal enzyme at a concentration of at least 5 mg/ml and a surfactant, wherein the formulation comprises no greater than 5 mM phosphate and has a pH of 5.5-7.0, wherein the surfactant is polysorbate 20 or polysorbate 80, and wherein the formulation is suitable for CNS delivery.

2. The formulation of claim 1 , wherein the lysosomal enzyme is present at a concentration ranging from 10-20 mg/ml.

3. The formulation of claim 1 , wherein the lysosomal enzyme is present at a concentration of 10 mg/ml.

4. The formulation of claim 1 , further comprising sodium chloride present at a concentration up to 175 mM.

5. The formulation of claim 1 , wherein the formulation is a liquid formulation.

Assignments (2)
ASSIGNMENT OF ASSIGNOR'S INTEREST Recorded Nov 21, 2023
From: SHIRE HUMAN GENETIC THERAPIES, INC.
To: TAKEDA PHARMACEUTICAL COMPANY LIMITED
Reel/Frame 065658/0287 →
ASSIGNMENT OF ASSIGNOR'S INTEREST Recorded Aug 17, 2022
From: CALIAS, PERICLES; PAN, JING; POWELL, JAN; CHARNAS, LAWRENCE; MCCAULEY, THOMAS; WRIGHT, TERESA LEAH; PFEIFER, RICHARD; SHAHROKH, ZAHRA
To: SHIRE HUMAN GENETIC THERAPIES, INC.
Reel/Frame 060831/0833 →
Continuity (11)
Continuation 16573557 · Sep 17, 2019
Continuation 15016141 · Feb 4, 2016
Division 13168961 · Jun 25, 2011
Provisional Application 61358857 · Jun 25, 2010
Provisional Application 61360786 · Jul 1, 2010
Provisional Application 61387862 · Sep 29, 2010
Provisional Application 61435710 · Jan 24, 2011
Provisional Application 61442115 · Feb 11, 2011
Provisional Application 61476210 · Apr 15, 2011
Provisional Application 61495268 · Jun 9, 2011
Related Publication 20230165942A1 · Jun 1, 2023
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