Title:
PHARMACEUTICAL COMPOSITION FOR TREATMENT OF SPINOCEREBELLAR ATAXIA
Document Type and Number:
WIPO Patent Application WO/2023/080225
Kind Code:
A1
Abstract:
Provided is a nucleic acid molecule having an antisense effect against a transcript of a mutated gene that includes an abnormal repeat region. The nucleic acid molecule has an antisense effect against a transcript of a mutated gene that includes an abnormal repeat region. The abnormal repeat region comprises a 5' region, a core repeat region, and a 3' region. The core repeat region comprises a base sequence in which the base sequence TGGAA repeats multiple times, or a base sequence that is complimentary thereto. The nucleic acid molecule includes a base sequence that is complimentary to four or more continuous bases in the abnormal repeat region of the transcript and includes a target-binding region that can hybridize to the transcript.
Inventors:
YOKOTA TAKANORI (JP)
ISHIKAWA KINYA (JP)
NAGATA TETSUYA (JP)
HIGASHI MIWA (JP)
YOSHIOKA KOTARO (JP)
KOIZUMI MAKOTO (JP)
ONISHI YOSHIYUKI (JP)
SHOJI TAKAO (JP)
ISHIKAWA KINYA (JP)
NAGATA TETSUYA (JP)
HIGASHI MIWA (JP)
YOSHIOKA KOTARO (JP)
KOIZUMI MAKOTO (JP)
ONISHI YOSHIYUKI (JP)
SHOJI TAKAO (JP)
Application Number:
PCT/JP2022/041282
Publication Date:
May 11, 2023
Filing Date:
November 07, 2022
Export Citation:
Assignee:
UNIV NAT CORP TOKYO MEDICAL & DENTAL (JP)
DAIICHI SANKYO CO LTD (JP)
DAIICHI SANKYO CO LTD (JP)
International Classes:
C12N15/113; A61K31/7088; A61P25/28
Domestic Patent References:
| WO2018062510A1 | 2018-04-05 |
Other References:
SATO NOZOMU, TAKESHI AMINO, KAZUHIRO KOBAYASHI, SHUICHI ASAKAWA, TARO ISHIGURO, TAIJI TSUNEMI, MAKOTO TAKAHASHI, TOHRU MATSUURA, K: "Spinocerebellar Ataxia Type 31 Is Associated with "Inserted" Penta-Nucleotide Repeats Containing (TGGAA)n", THE AMERICAN JOURNAL OF HUMAN GENETICS, vol. 85, no. 5, 13 November 2009 (2009-11-13), pages 544 - 557, XP093063032
KOSUKE MATSUZONO, IMAMURA KEIKO, MURAKAMI NAGAHISA, TSUKITA KAYOKO, YAMAMOTO TAKUYA, IZUMI YUISHIN, KAJI RYUJI, OHTA YASUYUKI, YAM: "Antisense Oligonucleotides Reduce RNA Foci in Spinocerebellar Ataxia 36 Patient iPSCs", MOLECULAR THERAPY-NUCLEIC ACIDS, vol. 8, 1 September 2017 (2017-09-01), US , pages 211 - 219, XP055560207, ISSN: 2162-2531, DOI: 10.1016/j.omtn.2017.06.017
MCFARLAND KAREN N., LIU JILIN, LANDRIAN IVETTE, GODISKA RONALD, SHANKER SAVITA, YU FAHONG, FARMERIE WILLIAM G., ASHIZAWA TETSUO: "SMRT Sequencing of Long Tandem Nucleotide Repeats in SCA10 Reveals Unique Insight of Repeat Expansion Structure", PLOS ONE, vol. 10, no. 8, pages e0135906, XP093063037, DOI: 10.1371/journal.pone.0135906
KOSUKE MATSUZONO, IMAMURA KEIKO, MURAKAMI NAGAHISA, TSUKITA KAYOKO, YAMAMOTO TAKUYA, IZUMI YUISHIN, KAJI RYUJI, OHTA YASUYUKI, YAM: "Antisense Oligonucleotides Reduce RNA Foci in Spinocerebellar Ataxia 36 Patient iPSCs", MOLECULAR THERAPY-NUCLEIC ACIDS, vol. 8, 1 September 2017 (2017-09-01), US , pages 211 - 219, XP055560207, ISSN: 2162-2531, DOI: 10.1016/j.omtn.2017.06.017
MCFARLAND KAREN N., LIU JILIN, LANDRIAN IVETTE, GODISKA RONALD, SHANKER SAVITA, YU FAHONG, FARMERIE WILLIAM G., ASHIZAWA TETSUO: "SMRT Sequencing of Long Tandem Nucleotide Repeats in SCA10 Reveals Unique Insight of Repeat Expansion Structure", PLOS ONE, vol. 10, no. 8, pages e0135906, XP093063037, DOI: 10.1371/journal.pone.0135906
Attorney, Agent or Firm:
HIRAKI & ASSOCIATES (JP)
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