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Polynucleotides encoding low density lipoprotein receptor

A low-density lipoprotein and polynucleotide technology, applied in the field of polynucleotides encoding low-density lipoprotein receptors, can solve problems such as adverse events of heart disease

Inactive Publication Date: 2016-09-28
MODERNA THERAPEUTICS INC
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  • Summary
  • Abstract
  • Description
  • Claims
  • Application Information

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Problems solved by technology

In addition, serious adverse events in cardiac patients occurred in the mipomersen arm of the Phase III trial

Method used

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  • Polynucleotides encoding low density lipoprotein receptor
  • Polynucleotides encoding low density lipoprotein receptor
  • Polynucleotides encoding low density lipoprotein receptor

Examples

Experimental program
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preparation example Construction

[0491] Formulations of the pharmaceutical compositions described herein may be prepared by any method known or hereafter developed in the art of pharmacology. In general, such preparation methods comprise the steps of combining the active ingredient with excipients and / or one or more other auxiliary ingredients, and subsequently, if necessary and / or required, dividing, shaping and / or packaging the product into the desired single-dose or multiple-dose units.

[0492] The relative amounts of the active ingredients, pharmaceutically acceptable excipients and / or any additional ingredients in the pharmaceutical compositions of the present invention will vary depending on the identity, size and / or condition of the subject to be treated, and further depending on the composition to be administered path changes. By way of example, the composition may comprise between 0.1% and 100%, such as between 0.5% and 50%, between 1-30%, between 5-80%, at least 80% (w / w) active ingredient .

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Embodiment 1

[1205] Example 1. Production of polynucleotides

[1206] Manufacture of polynucleotides and / or portions or regions thereof in accordance with the present invention may be made using U.S. 61 / 800,049, filed March 15, 2013, entitled "Manufacturing Methods for Producing RNA Transcripts" (Attorney Docket No. M500 ), the contents of which are incorporated herein by reference in their entirety.

[1207] Purification methods may include USSN 61 / 799,872, filed March 15, 2013, entitled "Methods for Removing DNA Fragments in mRNA Production" (Attorney Docket No. M501); Those taught in USSN 61 / 794,842 (Attorney Docket No. M502) for "Purification of Ribonucleic Acids," the contents of each of which are hereby incorporated by reference in their entirety.

[1208] Methods of detection and characterization of polynucleotides may be performed as taught in U.S. 61 / 798,945 (Attorney Docket No. M505), entitled "Characterizing mRNA Molecules," filed March 15, 2013, the contents of which are inc...

Embodiment 2

[1210] Example 2. Chimeric polynucleotide synthesis: the triphosphate pathway

[1211] introduction

[1212] According to the present invention, two regions or portions of a chimeric polynucleotide can be joined or linked using triphosphate chemistry.

[1213] According to this method, a first region or portion of 100 nucleotides or less is chemically synthesized, a 5' monophosphate and a terminal 3' desOH or blocked OH. If the region is longer than 80 nucleotides, it can be synthesized as two strands for ligation.

[1214] If the first region or part is synthesized as a region or part modified in a non-positional manner using in vitro transcription (IVT), then a 5' monophosphate followed by capping of the 3' end can be followed.

[1215] The monophosphate protecting group may be selected from any of those known in the art.

[1216] The second region or portion of the chimeric polynucleotide can be synthesized using chemical synthesis or IVT methods. IVT methods can in...

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Abstract

The invention relates to polynucleotides encoding a low density lipoprotein receptor. The invention relates to compositions and methods for the preparation, manufacture and therapeutic use of polynucleotide molecules encoding low density lipoprotein receptor comprising at least one mutation (e.g., an LDLR signally enhancing mutation).

Description

[0001] Cross References to Related Applications [0002] This application claims U.S. Provisional Patent Application No. 62 / 052,139, filed September 18, 2014, entitled "Polynucleotide Encoding Low Density Lipoprotein Receptor," U.S. Provisional Patent Application No. 61 / 952,906, entitled "Polynucleotides Encoding Low Density Lipoprotein Receptors," filed October 3, 2013, U.S. Provisional Patent Application No. 61 / 886,137 and priority of U.S. Provisional Patent Application No. 61 / 903,485, entitled "Polynucleotide Encoding Low Density Lipoprotein Receptor," filed November 13, 2013, the contents of each of which are incorporated by reference in their entirety and into this article. [0003] References to Sequence Listings [0004] This application is filed with a sequence listing in electronic format. The Sequence Listing is provided as a file named M070PCT.txt, created October 3, 2014, 2,607,599 bits in size. The information in the sequence listing in electronic format is inc...

Claims

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Application Information

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IPC IPC(8): C07K14/705
CPCC07K14/705A61K38/00A61P3/06A61P43/00C07K14/485C12N2310/317C12N2310/334C12N2310/335
Inventor 杰夫·L·埃尔斯沃思J·B·博伦F·M·格雷瓜尔贾斯汀·基尔德
Owner MODERNA THERAPEUTICS INC
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