US2022170042A1PendingUtilityA1

Compositions and methods for regulating production of a precursor protein

Assignee: WYVERN PHARMACEUTICALS INCPriority: Nov 27, 2020Filed: Nov 27, 2020Published: Jun 2, 2022
Est. expiryNov 27, 2040(~14.3 yrs left)· nominal 20-yr term from priority
A61K 35/763C07K 14/705C12N 15/86C12N 2710/16643C12N 2750/14141
63
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Claims

Abstract

The present disclosure relates to one or more agents, therapies, treatments, and methods of use of the agents and/or therapies and/or treatments for increasing production of a TLR3 precursor protein. Embodiments of the present disclosure can be used as a therapy or a treatment for a subject that has a condition whereby the subject's immune system is, or is likely to become, dysregulated and where the production of the TLR3 precursor protein may result in an increased production of a functional and bioavailable TLR3 protein product, which may be of therapeutic benefit.

Claims

exact text as granted — not AI-modified
The invention claimed is: 
     
         1 . A recombinant virus vector (RVV), the RVV comprising:
 a. a nucleotide sequence encoding a toll-like receptor 3 (TLR3) precursor protein; and   b. an inverted terminal repeat.   
     
     
         2 . The RVV of  claim 1 , wherein the nucleotide sequence encoding the TLR3 precursor protein is SEQ ID No. 1. 
     
     
         3 . The RVV of  claim 1 , wherein the precursor protein has an amino acid sequence of SEQ ID No. 2. 
     
     
         4 . The RVV of  claim 1 , wherein the inverted terminal repeat is SEQ ID No. 4 or SEQ ID No. 5. 
     
     
         5 . The RVV of  claim 1 , wherein the inverted terminal repeat is a first inverted terminal repeat of SEQ ID No. 4 and a second inverted terminal repeat of SEQ ID No. 5 and, wherein the nucleotide sequence encoding a toll-like receptor 3 (TLR3) precursor protein is positioned between the first inverted terminal repeat and the second inverted terminal repeat. 
     
     
         6 . The RVV of  claim 1 , wherein the RVV is an adeno-associated virus. 
     
     
         7 . A composition that comprises a nucleotide sequence according to SEQ ID No. 1 that can be expressed in a target cell. 
     
     
         8 . A pharmaceutical composition comprising the composition of  claim 7  and one or more pharmaceutically acceptable carriers and/or one or more excipients. 
     
     
         9 . An insert for use with a recombinant virus vector (RVV), wherein the insert has a nucleotide sequence according to SEQ ID No. 1. 
     
     
         10 . A method of making an agent/target cell complex, the method comprising a step of administering a recombinant virus vector (RVV) to a target cell for forming the agent/target cell complex, wherein the agent/target cell complex causes the target cell to increase production of a TLR3 precursor protein. 
     
     
         11 . The method of  claim 10 , wherein the RVV comprises a nucleotide sequence according to SEQ ID No. 3 for increasing the target cell's production of the TLR3 precursor protein. 
     
     
         12 . The method of  claim 10 , wherein the RVV comprises a nucleotide sequence according to SEQ ID No. 1 for increasing the target cell's production of the TLR3 precursor protein. 
     
     
         13 . The method of  claim 10 , wherein the TLR3 precursor protein has an amino acid sequence of SEQ ID No. 2. 
     
     
         14 . The method of  claim 10 , wherein the TLR3 precursor protein is subjected to one or more post-translation modification processes within the target cell to produce a TLR3 protein product. 
     
     
         15 . The method of  claim 10 , wherein the target cell is one or more of an innate immune cell, an acquired immune cell, an adrenal gland cell; a bile duct cell; a chondrocyte; a cochlear cell; a corneal cell; an endocardium cell; an endometrial cell; an endothelial cell; an epithelial cell; a fibroblast; a hair follicle cell; a hepatocyte; a lymph node cell; a mucosal cell; a myocyte; a neuron; a glomeruli cell; an optic nerve cell; an osteoblast; an ovarian tissue cell; a pancreatic islet beta cell; a pericardium cell; a platelet; a red blood cell (RBC); a retinal cell; a scleral cell; a Schwann cell; a T cell; a testicular tissue cell; a thyroid gland cell; a uveal cell; a tumor cell, and combinations thereof.

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