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Fusogenic lipid nanoparticles and methods for the manufacture and use thereof for the target cell-specific production of a therapeutic protein and for the treatment of a disease, condition, or disorder associated with a target cell

A technology of lipid nanoparticles and therapeutic proteins, applied in the field of medicine, can solve problems such as obstacles

Pending Publication Date: 2021-02-02
OISIN BIOTECHNOLOGIES INC
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  • Summary
  • Abstract
  • Description
  • Claims
  • Application Information

AI Technical Summary

Problems solved by technology

These shortcomings in the art have hampered the development of safe and effective therapies to treat certain cancers and slow the effects of aging

Method used

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  • Fusogenic lipid nanoparticles and methods for the manufacture and use thereof for the target cell-specific production of a therapeutic protein and for the treatment of a disease, condition, or disorder associated with a target cell
  • Fusogenic lipid nanoparticles and methods for the manufacture and use thereof for the target cell-specific production of a therapeutic protein and for the treatment of a disease, condition, or disorder associated with a target cell
  • Fusogenic lipid nanoparticles and methods for the manufacture and use thereof for the target cell-specific production of a therapeutic protein and for the treatment of a disease, condition, or disorder associated with a target cell

Examples

Experimental program
Comparison scheme
Effect test

Embodiment 1

[0258] P14 FAST fusogenic lipid nanoparticles (LNPs) enhance gene delivery to tumors

[0259] This example shows that Fusogenix using p14 FAST fusion of reptilian reovirus TM (Innovascreen, Halifax, Nova Scotia, Canada) Lipid nanoparticles enable efficient delivery of plasmid DNA constructs into target tumors.

[0260] Will use 64 Cu( 64 Cu NOTA-liposome)-labeled fusogenic lipid nanoparticles with or without p14 FAST fusion protein (described in PCT patents with publication numbers WO2002044206A2 and WO2012040825A1) were administered intravenously to the M16 mouse prostate cancer model system. Seo, Bioconjug Chem 19(12) :2577-2585 (2009) and Reeves, Cancer Therapy 136(7) : 1731-1740 (2014). Twenty-four hours after immunization, PC3 tumors were visualized by positron emission tomography (PET). Figure 7A and Figure 7B

[0261] Figure 8 The data shown show that, with no p14 FAST fusion protein 64 Cu NOTA-liposomes compared to PC3 prostate tumors with p14 FAST fusion...

Embodiment 2

[0263] P14 administered in vivo FAST fusogenic lipid nanoparticles are nontoxic and well tolerated

[0264] This example shows that Fusogenix using p14 FAST fusion of reptilian reovirus TM (Innovascreen, Halifax, Nova Scotia, Canada) Lipid nanoparticles exhibited no adverse side effects in major mammalian organ systems when administered in vivo to Sprague-Dawley rats and were able to efficiently deliver plasmid DNA constructs to target in the tumor.

[0265] Presented here is a comparative study of 20 male rats treated with (i) LNP-free (PBS), (ii) LNP-free p14, or (iii) Fusogenix lipid nanoparticles with p14 (LNP) processing. Each animal received three injections of 15 mg / kg in the tail over a 4 day period. Treatment of animals with p14-containing LNP did not result in acute changes in animal behavior, nor did treatment with p14-containing LNP affect animal growth. Animals treated with p14-containing LNP had similar organ weights compared to all other animal groups st...

Embodiment 3

[0272] In vivo suppression of p16-positive senescent cell burden in aged mice

[0273] This example demonstrates target cell-specific inhibition of pl6-positive senescent cell burden following in vivo administration of exemplary pl6-targeting constructs in a mouse model system.

[0274] Mouse models of aging exhibit a senescent cell burden (defined by the presence of p16+ cells) and secretion of factors associated with senescence-associated secretory phenotypes. (SASP; van Deursen, Nature 509(7501) :439-446(2014)).

[0275]In vivo administration of a formulation comprising a vector and an expression construct, such as a lipid nanoparticle (LNP) vector, such as a fusogenic LNP comprising a fusogenic protein such as p14FAST, comprising a p16-iCasp9 expression construct (pVAX1 -16s-iCasp9; SEQ ID NO:06; Figure 16 ), which includes exemplary p16-targeting constructs for target cell-specific expression of inducible caspase 9 (iCasp9) protein in p16-expressing target cells, su...

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Abstract

Provided nucleic acid- based expression construct for the target cell-specific production of a therapeutic protein, such as a pro-apoptotic protein, within a target cell, including a target cell thatis associated with aging, disease, or other condition, in particular a target cell that is a senescent cell or a cancer cell. Also provided are formulations and systems, including fusogenic lipid nanoparticle (LNP) formulations and systems, for the delivery of nucleic acid-based expression constructs as well as methods for making and using such nucleic acid-based expression constructs, formulations, and systems for reducing, preventing, and / or eliminating the growth and / or survival of a cell, such as a senescent cell and / or a cancer cell, which is associated with aging, disease, or other condition as well as methods for the treatment of aging, disease, or other conditions by the in vivo administration of a formulation, such as a fusogenic LPN formulation, comprising an expression constructfor the target cell-specific, production of a therapeutic protein, such as a pro-apoptotic protein, in a target cell that is associated with aging, disease, or other condition, in particular a targetcell that is a senescent cell or a cancer cell.

Description

[0001] Cross-references to related fields [0002] This PCT application was filed April 18, 2019 and claims the benefit of U.S. Provisional Patent Application 62 / 659,676, filed April 18, 2018, and U.S. Provisional Patent Application 62 / 821,084, filed March 20, 2019. The entire contents of Provisional Patent Applications 62 / 659,676 and 62 / 821,084 are hereby incorporated by reference in their entirety. [0003] sequence listing [0004] This application includes a sequence listing in electronic format, the txt file name is "OSIN-01-0304WO01_2019-04-18_SEQLIST_ST25", which was created on April 18, 2019, and the size is 68,831 bytes. This application also includes the Sequence Listing as a PDF file entitled "OSIN-01-0304WO01_2019-04-18_SEQLIST_ST25", created on April 18, 2019. The txt and PDF files titled "OSIN-01-0304WO01_2019-04-18_SEQLIST_ST25" are identical in content and are incorporated herein by reference. technical field [0005] The present disclosure relates genera...

Claims

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Application Information

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Patent Type & Authority Applications(China)
IPC IPC(8): A61K31/7088A61K31/7105A61K31/713A61K47/18A61K47/24
CPCA61K31/713A61K9/5123A61K9/0019C12N9/50C12N9/78C12Y305/04001C12Y304/22056C12Y304/22061C12Y304/22062A61K47/6911A61K47/62A61K48/0041A61K48/005A01K2227/105A01K2267/0331A01K2267/035A01K2207/12C12N2720/12222A61K9/1271A61K31/7105C12N15/88C12N2320/32C12N9/16C12N2830/002C12N2310/20A61K31/711
Inventor 马修·赖因·斯科尔茨
Owner OISIN BIOTECHNOLOGIES INC
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