US2024016889A1PendingUtilityA1

Compositions and methods for treating motor neuron diseases

59
Assignee: UNIV COLUMBIAPriority: Dec 13, 2017Filed: Sep 22, 2023Published: Jan 18, 2024
Est. expiryDec 13, 2037(~11.4 yrs left)· nominal 20-yr term from priority
Inventors:Umrao MonaniJeong Ki-Kim
A61K 38/1709A61P 21/00A61K 9/0019A61K 9/0085A61K 35/761A61K 38/465C12N 15/113A61K 48/00A61K 45/06A61K 31/7088A61K 31/713C07K 14/47A01K 67/0275A01K 2227/105A01K 2267/0306A01K 2217/15C12N 2750/14143C12N 2310/11C12N 2310/14
59
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Claims

Abstract

The present disclosure provides compositions and methods of treating motor neuron diseases, including spinal muscular atrophy (SMA) and amyotrophic lateral sclerosis (ALS). A modulator of a heat shock protein, such as an Hsp70 family member protein, may be used in the present method. Alternatively, a mutant heat shock protein may be used.

Claims

exact text as granted — not AI-modified
What is claimed is: 
     
         1 . A method of treating a neurodegenerative disorder in a subject, the method comprising administering an effective amount of a modulator of Hsa8 to the subject. 
     
     
         2 . The method of  claim 1 , wherein the modulator is an inhibitor of Hsa8. 
     
     
         3 . The method of  claim 1 , wherein the modulator is a small molecule, a polynucleotide, or an antibody or antigen-binding portion thereof. 
     
     
         4 . The method of  claim 3 , wherein the polynucleotide is a small interfering RNA (siRNA) or an antisense molecule. 
     
     
         5 . The method of  claim 1 , wherein the modulator comprises a CRISPR/Cas system. 
     
     
         6 . The method of  claim 1 , wherein the modulator binds to a substrate binding domain of Hspa8. 
     
     
         7 . The method of  claim 1 , wherein the modulator binds to an ATPase domain of Hspa8. 
     
     
         8 . The method of  claim 1 , wherein the modulator decreases a chaperone activity of Hspa8. 
     
     
         9 . The method of  claim 1 , wherein the modulator increases a microautophagy activity of Hspa8. 
     
     
         10 . The method of  claim 1 , wherein the neurodegenerative disorder is a motor neuron disease. 
     
     
         11 . The method of  claim 10 , wherein the motor neuron disease is spinal muscular atrophy (SMA) or amyotrophic lateral sclerosis (ALS). 
     
     
         12 . The method of  claim 10 , wherein the motor neuron disease is hereditary spastic paraplegia (HSP), primary lateral sclerosis (PLS), progressive muscular atrophy (PMA), progressive bulbar palsy (PBP), or pseudobulbar palsy. 
     
     
         13 . The method of  claim 1 , wherein the modulator is administered to the central nervous system (CNS) of the subject. 
     
     
         14 . The method of  claim 1 , wherein the modulator is administered to the spinal cord of the subject. 
     
     
         15 . The method of  claim 1 , wherein the modulator is administered by intrathecal injection. 
     
     
         16 . The method of  claim 1 , wherein the modulator is administered orally, intravenously, intramuscularly, topically, arterially, or subcutaneously. 
     
     
         17 . The method of  claim 1 , further comprising administering a SMN2 splicing modifier to the subject. 
     
     
         18 . The method of  claim 1 , wherein the subject is a mammal. 
     
     
         19 . The method of  claim 18 , wherein the mammal is a human, a rodent, or a simian. 
     
     
         20 . The method of  claim 18 , wherein the mammal is a human.

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