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Formulations of protein molecules comprising iduronate 2-sulfatase

A technology of iduronic acid and protein, applied in the field of protein molecular preparations containing iduronic acid 2-sulfatase

Pending Publication Date: 2021-12-10
DENALI THERAPEUTICS INC
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  • Summary
  • Abstract
  • Description
  • Claims
  • Application Information

AI Technical Summary

Problems solved by technology

Recombinant forms of the enzymes that are defective in LSD could be used to treat the condition, but because the recombinant enzymes are difficult to deliver across the blood-brain barrier (BBB), such therapies may have little effect on the brain

Method used

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  • Formulations of protein molecules comprising iduronate 2-sulfatase
  • Formulations of protein molecules comprising iduronate 2-sulfatase
  • Formulations of protein molecules comprising iduronate 2-sulfatase

Examples

Experimental program
Comparison scheme
Effect test

Embodiment approach 1

[0283] Embodiment 1. A pharmaceutical composition comprising:

[0284] a. A protein molecule comprising:

[0285] i. a first Fc polypeptide; and

[0286] ii. a second Fc polypeptide linked to an enzyme replacement therapy (ERT) enzyme, ERT enzyme variant, or a catalytically active fragment thereof;

[0287] b. Buffers; and

[0288] c. Salt;

[0289] Wherein the pH of the pharmaceutical composition is about 5.5 to 7.0.

Embodiment approach 2

[0290] Embodiment 2. The pharmaceutical composition of embodiment 1, wherein the buffer is selected from the group consisting of phosphate buffer, acetate buffer, arginine buffer, and histidine buffer.

Embodiment approach 3

[0291] Embodiment 3. The pharmaceutical composition of embodiment 2, wherein the phosphate buffer is sodium phosphate buffer or potassium phosphate buffer.

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Abstract

Certain embodiments provide a pharmaceutical composition comprising: a protein molecule comprising an ERT enzyme-Fc fusion polypeptide and a modified Fc polypeptide; a buffer; an isotonicity agent; a surfactant; and a stabilizer; wherein the pH of the pharmaceutical composition is about 5.5 to 7.0, as well as methods of use thereof.

Description

[0001] Cross References to Related Applications [0002] This application claims priority to U.S. Provisional Application Serial No. 62 / 828,859, filed April 3, 2019, and U.S. Provisional Application Serial No. 62 / 832,032, filed April 10, 2019. The entire contents of the applications cited above are hereby incorporated by reference. Background technique [0003] Lysosomal storage disorders (LSDs) are relatively rare inherited metabolic disorders caused by defects in lysosomal function. LSD is usually caused by the deficiency of a single enzyme involved in the breakdown of metabolites in lysosomes. Product accumulation due to lack of enzymatic activity affects various organ systems and can lead to severe symptoms and premature death. Most LSDs also have a significant neurological component, which ranges from progressive neurodegeneration and severe cognitive impairment to epilepsy, behavioral and psychiatric disturbances. Recombinant forms of the enzymes that are defective in...

Claims

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Application Information

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IPC IPC(8): C12N9/16A61K47/68C07K16/28A61P3/00
CPCC12N9/16A61K47/68A61P3/00C07K2319/30C07K2317/52C12Y301/06013C07K2319/00C07K2319/02C07K2319/21A61K38/465A61K47/6815A61K47/26A61K47/20A61K9/19A61K47/183
Inventor D·安德森A·凯瑟曼T·吉斯G·坎南M·科里奥利C·马霍A·帕特尔
Owner DENALI THERAPEUTICS INC
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