# US 12,668,795 B2 · Inhibitor of miR-129 and uses thereof

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- Number: US 12,668,795 B2
- Document ID: US12668795B2
- Status: Granted
- Kind: B2
- Application number: 17/598,627
- Filed: 2020-03-26
- Published: 2022-05-26
- Granted: 2026-06-30
- Earliest claimed: 2019-03-27
- Assignees: Fondazione Irccs “Ca' Granda—Ospedale Maggiore” Policlinico Di Milano
- Applicants: Università Degli Studi Di Milano Bicocca; Fondazione Irccs “Ca' Granda—Ospedale Maggiore” Policlinico
- Inventors: Stefania Corti; Silvia Maria Luisa Barabino; Monica Nizzardo; Alessia Loffreda
- Web page: https://patentgrep.com/patent/US12668795B2

## CPC

- C12N 15/113: Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; {Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing (when used in plants C12N15/8218)}
- C12Q 1/6883: for diseases caused by alterations of genetic material
- C12N 2310/113: targeting other non-coding nucleic acids, e.g. antagomirs
- C12N 2310/3233: Morpholino-type ring
- C12Q 2600/178: miRNA, siRNA or ncRNA
- C12N: MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA (microbiological testing media C12Q1/00)
- C12Q: MEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS (immunoassay G01N33/53); COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES

## Abstract

The present invention relates to an inhibitor of miR-129, relative compounds and pharmaceutical compositions for use in the treatment and/or prevention of amyotrophic lateral sclerosis and Alzheimer's disease. The invention also relates to a method for the diagnosis and/or prognosis of Alzheimer's disease in a subject or to identify a subject at risk to develop amyotrophic lateral sclerosis or Alzheimer's disease and to a method for the measuring the efficacy of a therapy for amyotrophic lateral sclerosis or for Alzheimer's disease and relative kits.

## Claims

### Claim 1

A method for treating amyotrophic lateral sclerosis comprising administering a morpholino antisense oligonucleotide inhibitor of miR-129 to a subject diagnosed with amyotrophic lateral sclerosis, wherein said morpholino antisense oligonucleotide inhibitor of miR-129 comprises the sequence of SEQ ID NO: 3.

### Claim 2 (depends on 1)

The method according to claim 1, wherein said morpholino antisense oligonucleotide inhibitor of miR-129 is:
a single-stranded or double-stranded nucleic acid molecule, an siRNA molecule, an antisense oligonucleotide, derivatives and mixtures thereof.

### Claim 3 (depends on 1)

The method of claim 1, wherein the morpholino antisense oligonucleotide inhibitor of miR-129 is administered to the subject intracerebroventricularly, orally, rectally, transmucosal, intestinally, enterally, through inhalation, intraventricularly, intraperitoneally, intranasally, or parenterally.

### Claim 4 (depends on 1)

The method of claim 1, wherein the subject has SOD1-associated ALS.

### Claim 5 (depends on 1)

The method of claim 1, wherein said morpholino antisense oligonucleotide inhibitor of miR-129 consists the sequence of SEQ ID NO: 3.