# US 2020/0399641 A1 · Antisense oligonucleotides for modulating htra1 expression

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- Number: US 2020/0399641 A1
- Document ID: US20200399641A1
- Status: Published application
- Kind: A1
- Application number: 16/730,779
- Filed: 2019-12-30
- Published: 2020-12-24
- Earliest claimed: 2016-07-01
- Applicants: Hoffmann-La Roche, Inc.
- Inventors: Roberto Iacone; Peter Hagedorn; Susanne Kammler; Soren Ottosen; Sindri Traustason; Heidi Hudlebusch; Lykke Pedersen
- Web page: https://patentgrep.com/patent/US20200399641A1

## CPC

- C12N 15/1137: {against enzymes (viral enzymes C12N15/1131; receptors C12N15/1138)}
- A61P 19/02: for joint disorders, e.g. arthritis, arthrosis
- A61P 25/00: Drugs for disorders of the nervous system
- A61P 27/02: Ophthalmic agents
- A61P 43/00: Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
- C12N 2310/11: Antisense
- C12N 2310/315: Phosphorothioates
- C12N 2310/3231: having an additional ring, e.g. LNA, ENA
- C12N 2310/3341: 5-Methylcytosine
- C12N 2310/341: Gapmers, i.e. of the type ===---===
- C12N 2310/344: Position-specific modifications, e.g. on every purine, at the 3'-end
- C12N 2310/346: having a combination of backbone and sugar modifications
- C12N 2310/351: Conjugate
- C12N: MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA (microbiological testing media C12Q1/00)
- A61P: SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS

## Abstract

The present invention relates to antisense oligonucleotides (oligomers) that are complementary to HTRA1, leading to modulation of the expression of HTRA1. Modulation of HTRA1 expression is beneficial for a range of medical disorders, such as macular degeneration, e.g. age-related macular degeneration.

## Claims

### Claim 1

An antisense oligonucleotide of 10-30 nucleotides in length, wherein said antisense oligonucleotide targets a HTRA1 nucleic acid and comprises a contiguous nucleotide region of 10-22 nucleotides which are at least 90% such as 100% complementarity to SEQ ID NO 147:
SEQ ID NO 147: 5′ CCAACAACCAGGTAAATATTTG 3′