CA3213590A1 - Compositions and methods for treating tdp-43 proteinopathy - Google Patents
Compositions and methods for treating tdp-43 proteinopathy Download PDFInfo
- Publication number
- CA3213590A1 CA3213590A1 CA3213590A CA3213590A CA3213590A1 CA 3213590 A1 CA3213590 A1 CA 3213590A1 CA 3213590 A CA3213590 A CA 3213590A CA 3213590 A CA3213590 A CA 3213590A CA 3213590 A1 CA3213590 A1 CA 3213590A1
- Authority
- CA
- Canada
- Prior art keywords
- seq
- antisense oligonucleotide
- bases
- unc13a
- exon
- Prior art date
- Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
- Pending
Links
Classifications
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/113—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
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/70—Carbohydrates; Sugars; Derivatives thereof
- A61K31/7088—Compounds having three or more nucleosides or nucleotides
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
- A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/11—Antisense
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/31—Chemical structure of the backbone
- C12N2310/315—Phosphorothioates
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/32—Chemical structure of the sugar
- C12N2310/321—2'-O-R Modification
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/32—Chemical structure of the sugar
- C12N2310/323—Chemical structure of the sugar modified ring structure
- C12N2310/3231—Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
- C12N2320/33—Alteration of splicing
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
- C12N2320/34—Allele or polymorphism specific uses
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Biomedical Technology (AREA)
- Chemical & Material Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Molecular Biology (AREA)
- Organic Chemistry (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- General Health & Medical Sciences (AREA)
- Plant Pathology (AREA)
- Microbiology (AREA)
- Biophysics (AREA)
- Biochemistry (AREA)
- Physics & Mathematics (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Medicinal Chemistry (AREA)
- Veterinary Medicine (AREA)
- Pharmacology & Pharmacy (AREA)
- Neurosurgery (AREA)
- Epidemiology (AREA)
- Neurology (AREA)
- Psychiatry (AREA)
- Hospice & Palliative Care (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Peptides Or Proteins (AREA)
- Enzymes And Modification Thereof (AREA)
- Preparation Of Compounds By Using Micro-Organisms (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202163171522P | 2021-04-06 | 2021-04-06 | |
| US63/171,522 | 2021-04-06 | ||
| US202263312808P | 2022-02-22 | 2022-02-22 | |
| US63/312,808 | 2022-02-22 | ||
| PCT/US2022/023559 WO2022216759A1 (en) | 2021-04-06 | 2022-04-05 | Compositions and methods for treating tdp-43 proteinopathy |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| CA3213590A1 true CA3213590A1 (en) | 2022-10-13 |
Family
ID=81384958
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CA3213590A Pending CA3213590A1 (en) | 2021-04-06 | 2022-04-05 | Compositions and methods for treating tdp-43 proteinopathy |
Country Status (9)
| Country | Link |
|---|---|
| US (1) | US20250011773A1 (https=) |
| EP (1) | EP4320236A1 (https=) |
| JP (1) | JP2024513237A (https=) |
| KR (1) | KR20240004467A (https=) |
| AU (1) | AU2022255175A1 (https=) |
| CA (1) | CA3213590A1 (https=) |
| IL (1) | IL307305A (https=) |
| MX (1) | MX2023011794A (https=) |
| WO (1) | WO2022216759A1 (https=) |
Families Citing this family (13)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| GB2603454A (en) * | 2020-12-09 | 2022-08-10 | Ucl Business Ltd | Novel therapeutics for the treatment of neurodegenerative disorders |
| CA3227115A1 (en) * | 2021-07-21 | 2023-01-26 | Wen-Hsuan Chang | Unc13a antisense oligonucleotides |
| CN119790152A (zh) * | 2021-12-03 | 2025-04-08 | 奎里斯公司 | 使用unc13a基因转录本的调节剂治疗神经系统疾病 |
| GB202117758D0 (en) * | 2021-12-09 | 2022-01-26 | Ucl Business Ltd | Therapeutics for the treatment of neurodegenerative disorders |
| CN118974254A (zh) * | 2021-12-21 | 2024-11-15 | 豪夫迈·罗氏有限公司 | 靶向unc13a的反义寡核苷酸 |
| AU2023356317A1 (en) * | 2022-10-05 | 2025-05-15 | Trace Neuroscience, Inc. | Unc13a antisense oligonucleotides and uses thereof |
| WO2024155986A2 (en) * | 2023-01-20 | 2024-07-25 | AcuraStem Incorporated | Unc13a antisense oligonucleotides |
| WO2024178223A1 (en) * | 2023-02-24 | 2024-08-29 | Northwestern University | Antisense oligonucleotides for preventing unc13a misplicing |
| AU2024283557A1 (en) * | 2023-06-02 | 2026-01-08 | Quralis Corporation | Modified unc13a oligonucleotides |
| GB202309922D0 (en) * | 2023-06-29 | 2023-08-16 | Univ Edinburgh | Antisense treatment |
| WO2025007194A1 (en) * | 2023-07-05 | 2025-01-09 | Macquarie University | Modulation of gene expression |
| GB202315883D0 (en) * | 2023-10-17 | 2023-11-29 | Univ Oxford Innovation Ltd | Method |
| WO2025186619A1 (en) * | 2024-03-05 | 2025-09-12 | Takeda Pharmaceutical Company Limited | Compositions and methods for splicing modulation of unc13a |
Family Cites Families (11)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP2016528873A (ja) * | 2012-05-16 | 2016-09-23 | ラナ セラピューティクス インコーポレイテッド | 遺伝子発現を調節するための組成物及び方法 |
| ES2762326T5 (es) | 2012-10-15 | 2023-04-27 | Ionis Pharmaceuticals Inc | Métodos para modular la expresión de C9ORF72 |
| EP4166667A3 (en) | 2013-10-11 | 2023-08-02 | Ionis Pharmaceuticals, Inc. | Compositions for modulating c9orf72 expression |
| EP4410805A3 (en) | 2014-03-18 | 2024-11-27 | University of Massachusetts | Raav-based compositions and methods for treating amyotrophic lateral sclerosis |
| WO2016167780A1 (en) | 2015-04-16 | 2016-10-20 | Ionis Pharmaceuticals, Inc. | Compositions for modulating expression of c9orf72 antisense transcript |
| BR112018012894A2 (en) | 2015-12-23 | 2018-12-04 | Crispr Therapeutics Ag | Materials and Methods for Treatment of Amyotrophic Lateral Sclerosis and / or Frontotemporal Lobular Degeneration |
| TW201920672A (zh) | 2017-08-08 | 2019-06-01 | 新加坡商波濤生命科學有限公司 | 寡核苷酸組合物及其使用方法 |
| CA3079727A1 (en) | 2017-10-24 | 2019-05-02 | Sangamo Therapeutics, Inc. | Methods and compositions for the treatment of rare diseases |
| JP7795914B2 (ja) * | 2019-06-03 | 2026-01-08 | クラリス コーポレーション | 神経学的疾患を処置するためのオリゴヌクレオチドおよびその使用方法 |
| WO2022018187A1 (en) * | 2020-07-23 | 2022-01-27 | F. Hoffmann-La Roche Ag | Oligonucleotides targeting rna binding protein sites |
| GB2603454A (en) * | 2020-12-09 | 2022-08-10 | Ucl Business Ltd | Novel therapeutics for the treatment of neurodegenerative disorders |
-
2022
- 2022-04-05 AU AU2022255175A patent/AU2022255175A1/en active Pending
- 2022-04-05 US US18/285,802 patent/US20250011773A1/en active Pending
- 2022-04-05 EP EP22718532.9A patent/EP4320236A1/en active Pending
- 2022-04-05 WO PCT/US2022/023559 patent/WO2022216759A1/en not_active Ceased
- 2022-04-05 MX MX2023011794A patent/MX2023011794A/es unknown
- 2022-04-05 IL IL307305A patent/IL307305A/en unknown
- 2022-04-05 CA CA3213590A patent/CA3213590A1/en active Pending
- 2022-04-05 KR KR1020237038205A patent/KR20240004467A/ko active Pending
- 2022-04-05 JP JP2023561290A patent/JP2024513237A/ja active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| IL307305A (en) | 2023-11-01 |
| US20250011773A1 (en) | 2025-01-09 |
| MX2023011794A (es) | 2024-01-08 |
| EP4320236A1 (en) | 2024-02-14 |
| JP2024513237A (ja) | 2024-03-22 |
| KR20240004467A (ko) | 2024-01-11 |
| WO2022216759A1 (en) | 2022-10-13 |
| AU2022255175A1 (en) | 2023-11-23 |
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| US20250011773A1 (en) | Compositions and methods for treating tdp-43 proteinopathy | |
| JP7049249B2 (ja) | 中枢神経系疾患の処置のための組成物および方法 | |
| US20230116704A1 (en) | Antisense oligomers for treatment of conditions and diseases | |
| AU2019396450B2 (en) | Methods of treating schizophrenia and other neuropsychiatric disorders | |
| JP2024075673A (ja) | 統合失調症及び他の神経精神障害の治療方法 | |
| WO2021076883A2 (en) | Muscle regeneration and growth | |
| CA3136949A1 (en) | Neurogenesis | |
| US20240409890A1 (en) | Ufmylation inhibition to target tauopathy in human neurons | |
| US12319928B2 (en) | Methods of treating Danon disease | |
| CN117580950A (zh) | 用于治疗tdp-43蛋白病的组合物和方法 | |
| CN118679257A (zh) | 用于脆性x相关病症的治疗性治疗 | |
| JP2025535055A (ja) | Ｕｎｃ１３ａアンチセンスオリゴヌクレオチド及びその使用 | |
| US20260022377A1 (en) | Polynucleotide compositions and methods for treatment of neurodegenerative diseases | |
| HK40129245A (zh) | Unc13a反义寡核苷酸和其用途 | |
| JP2024514424A (ja) | Ｍｕｓｋとｂｍｐ受容体との間の結合相互作用の特性評価 | |
| KR20240130136A (ko) | 근육 세포 표면에서 발현된 단백질을 표적화하는 조직-특이적 영상화제 및 치료제 | |
| Halim | The Exocyst Subunit EXOC2 Regulates GGGGCC Repeats Toxicity in C9ORF72-ALS/FTD | |
| WO2025111653A1 (en) | Improved compositions and methods for treatment of monogenic neurodevelopmental disorders | |
| HK1262959B (en) | Compositions and methods for treatment of central nervous system diseases | |
| HK1262959A1 (en) | Compositions and methods for treatment of central nervous system diseases |
Legal Events
| Date | Code | Title | Description |
|---|---|---|---|
| R11 | Change to the name of applicant or owner or transfer of ownership requested |
Free format text: ST27 STATUS EVENT CODE: A-1-1-R10-R11-R127 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: TRANSFER RECORDAL REQUEST OR RESPONSE Effective date: 20241101 Free format text: ST27 STATUS EVENT CODE: A-1-1-R10-R11-R103 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: CHANGE OF NAME REQUEST RECEIVED Effective date: 20241101 |
|
| W00 | Other event occurred |
Free format text: ST27 STATUS EVENT CODE: A-1-1-W10-W00-W111 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: CORRESPONDENT DETERMINED COMPLIANT Effective date: 20250318 |
|
| R14 | Transfer of ownership recorded |
Free format text: ST27 STATUS EVENT CODE: A-1-1-R10-R14-R129 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: TRANSFER REQUIREMENTS DETERMINED COMPLIANT Effective date: 20250530 |
|
| MFA | Maintenance fee for application paid |
Free format text: FEE DESCRIPTION TEXT: MF (APPLICATION, 3RD ANNIV.) - STANDARD Year of fee payment: 3 |
|
| U00 | Fee paid |
Free format text: ST27 STATUS EVENT CODE: A-1-1-U10-U00-U101 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE REQUEST RECEIVED Effective date: 20250617 |
|
| U11 | Full renewal or maintenance fee paid |
Free format text: ST27 STATUS EVENT CODE: A-1-1-U10-U11-U102 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE FEE PAYMENT PAID IN FULL Effective date: 20250617 |
|
| R13 | Change to the name of applicant or owner recorded |
Free format text: ST27 STATUS EVENT CODE: A-1-1-R10-R13-R104 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: CHANGE OF NAME REQUIREMENTS DETERMINED COMPLIANT Effective date: 20250619 |
|
| W00 | Other event occurred |
Free format text: ST27 STATUS EVENT CODE: A-1-1-W10-W00-W100 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: LETTER SENT Effective date: 20251127 |
|
| MFA | Maintenance fee for application paid |
Free format text: FEE DESCRIPTION TEXT: MF (APPLICATION, 4TH ANNIV.) - STANDARD Year of fee payment: 4 |
|
| U00 | Fee paid |
Free format text: ST27 STATUS EVENT CODE: A-1-1-U10-U00-U101 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE REQUEST RECEIVED Effective date: 20260310 |
|
| U11 | Full renewal or maintenance fee paid |
Free format text: ST27 STATUS EVENT CODE: A-1-1-U10-U11-U102 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE FEE PAYMENT PAID IN FULL Effective date: 20260310 |
|
| D11 | Substantive examination requested |
Free format text: ST27 STATUS EVENT CODE: A-1-1-D10-D11-D117 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: REQUEST FOR EXAMINATION RECEIVED Effective date: 20260407 |
|
| P11 | Amendment of application requested |
Free format text: ST27 STATUS EVENT CODE: A-1-1-P10-P11-P101 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: AMENDMENT RECEIVED - VOLUNTARY AMENDMENT Effective date: 20260407 |
|
| W00 | Other event occurred |
Free format text: ST27 STATUS EVENT CODE: A-1-1-W10-W00-W111 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: CORRESPONDENT DETERMINED COMPLIANT Effective date: 20260407 |
|
| P11 | Amendment of application requested |
Free format text: ST27 STATUS EVENT CODE: A-1-1-P10-P11-P102 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: AMENDMENT DETERMINED COMPLIANT Effective date: 20260414 |
|
| P13 | Application amended |
Free format text: ST27 STATUS EVENT CODE: A-1-1-P10-P13-X000 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: APPLICATION AMENDED Effective date: 20260414 |
|
| W00 | Other event occurred |
Free format text: ST27 STATUS EVENT CODE: A-1-1-W10-W00-W111 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: CORRESPONDENT DETERMINED COMPLIANT Effective date: 20260414 |
|
| D00 | Search and/or examination requested or commenced |
Free format text: ST27 STATUS EVENT CODE: A-1-1-D10-D00-D118 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: REQUEST FOR EXAMINATION REQUIREMENTS DETERMINED COMPLIANT Effective date: 20260415 |
|
| D11 | Substantive examination requested |
Free format text: ST27 STATUS EVENT CODE: A-1-2-D10-D11-D155 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: ALL REQUIREMENTS FOR EXAMINATION DETERMINED COMPLIANT Effective date: 20260415 |
|
| W00 | Other event occurred |
Free format text: ST27 STATUS EVENT CODE: A-2-2-W10-W00-W100 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: LETTER SENT Effective date: 20260415 |