KR20240129627A9 - Unc13a 유전자 전사체의 조정제를 사용한 신경계 질환의 치료 - Google Patents
Unc13a 유전자 전사체의 조정제를 사용한 신경계 질환의 치료Info
- Publication number
- KR20240129627A9 KR20240129627A9 KR1020247021710A KR20247021710A KR20240129627A9 KR 20240129627 A9 KR20240129627 A9 KR 20240129627A9 KR 1020247021710 A KR1020247021710 A KR 1020247021710A KR 20247021710 A KR20247021710 A KR 20247021710A KR 20240129627 A9 KR20240129627 A9 KR 20240129627A9
- Authority
- KR
- South Korea
- Prior art keywords
- unc13a
- modulators
- treatment
- neurological disorders
- gene transcript
- 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
- A61K31/712—Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
- A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
-
- 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
-
- 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
-
- 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
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/33—Chemical structure of the base
- C12N2310/332—Abasic residue
-
- 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
- C12N2310/33—Chemical structure of the base
- C12N2310/334—Modified C
- C12N2310/3341—5-Methylcytosine
-
- 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
- C12N2310/34—Spatial arrangement of the modifications
- C12N2310/344—Position-specific modifications, e.g. on every purine, at the 3'-end
-
- 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
- C12N2310/35—Nature of the modification
- C12N2310/351—Conjugate
-
- 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
- C12N2310/35—Nature of the modification
- C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
- C12N2310/3525—MOE, methoxyethoxy
-
- 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
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)
- Organic Chemistry (AREA)
- General Health & Medical Sciences (AREA)
- Molecular Biology (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- General Engineering & Computer Science (AREA)
- Biotechnology (AREA)
- Medicinal Chemistry (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Biochemistry (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Biophysics (AREA)
- Microbiology (AREA)
- Neurosurgery (AREA)
- Epidemiology (AREA)
- Neurology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Psychiatry (AREA)
- Hospice & Palliative Care (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Saccharide Compounds (AREA)
- Peptides Or Proteins (AREA)
- Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicinal Preparation (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
Applications Claiming Priority (7)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202163285786P | 2021-12-03 | 2021-12-03 | |
| US63/285,786 | 2021-12-03 | ||
| US202263350206P | 2022-06-08 | 2022-06-08 | |
| US63/350,206 | 2022-06-08 | ||
| US202263398987P | 2022-08-18 | 2022-08-18 | |
| US63/398,987 | 2022-08-18 | ||
| PCT/US2022/051713 WO2023102225A2 (en) | 2021-12-03 | 2022-12-02 | Treatment of neurological diseases using modulators of unc13a gene transcripts |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| KR20240129627A KR20240129627A (ko) | 2024-08-27 |
| KR20240129627A9 true KR20240129627A9 (ko) | 2025-12-10 |
Family
ID=86613080
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| KR1020247021710A Pending KR20240129627A9 (ko) | 2021-12-03 | 2022-12-02 | Unc13a 유전자 전사체의 조정제를 사용한 신경계 질환의 치료 |
Country Status (16)
| Country | Link |
|---|---|
| US (1) | US20250051766A1 (https=) |
| EP (1) | EP4441225A4 (https=) |
| JP (1) | JP2024543214A (https=) |
| KR (1) | KR20240129627A9 (https=) |
| CN (1) | CN119790152A (https=) |
| AU (1) | AU2022400851A1 (https=) |
| CA (1) | CA3239482A1 (https=) |
| CL (1) | CL2024001657A1 (https=) |
| CO (1) | CO2024008076A2 (https=) |
| CR (1) | CR20240269A (https=) |
| DO (1) | DOP2024000107A (https=) |
| IL (1) | IL313203A (https=) |
| JO (1) | JOP20240125A1 (https=) |
| MX (1) | MX2024006763A (https=) |
| PE (1) | PE20250677A1 (https=) |
| WO (1) | WO2023102225A2 (https=) |
Families Citing this family (6)
| 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 |
| AU2023356317A1 (en) * | 2022-10-05 | 2025-05-15 | Trace Neuroscience, Inc. | Unc13a antisense oligonucleotides and uses thereof |
| WO2024178223A1 (en) * | 2023-02-24 | 2024-08-29 | Northwestern University | Antisense oligonucleotides for preventing unc13a misplicing |
| WO2025186619A1 (en) * | 2024-03-05 | 2025-09-12 | Takeda Pharmaceutical Company Limited | Compositions and methods for splicing modulation of unc13a |
| WO2025255388A1 (en) * | 2024-06-05 | 2025-12-11 | Camp4 Therapeutics Corporation | Modulation of syngap1 gene transcription using antisense oligonucleotides targeting regulatory rnas |
| WO2026080323A1 (en) | 2024-10-09 | 2026-04-16 | Quralis Corporation | Treatment of neurological diseases using modulators of unc13a gene transcripts |
Family Cites Families (5)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US20060008800A1 (en) * | 2001-11-05 | 2006-01-12 | Christian Rosenmund | Unc-13 in the modulation of neurotransmission and secretion events |
| AU2013203395A1 (en) * | 2012-03-30 | 2013-10-17 | Isis Pharmaceuticals, Inc. | Compositions and methods for modulating TAU expression for reducing seizure and modifying a neurodegenerative syndrome |
| DK3155101T3 (da) * | 2014-06-16 | 2020-05-04 | Univ Johns Hopkins | Sammensætninger og fremgangsmåder til ekspression af CRISPR-leder-RNA´er under anvendelse af H1-promotoren |
| WO2020102472A1 (en) * | 2018-11-15 | 2020-05-22 | President And Fellows Of Harvard College | Methods and compositions related to targeting ffar2 and ilc3 populations for the treatment of a gastrointestinal disease |
| AU2022255175A1 (en) * | 2021-04-06 | 2023-11-23 | Trace Neuroscience, Inc. | Compositions and methods for treating tdp-43 proteinopathy |
-
2022
- 2022-12-02 CN CN202280090919.7A patent/CN119790152A/zh active Pending
- 2022-12-02 AU AU2022400851A patent/AU2022400851A1/en active Pending
- 2022-12-02 MX MX2024006763A patent/MX2024006763A/es unknown
- 2022-12-02 EP EP22902264.5A patent/EP4441225A4/en active Pending
- 2022-12-02 JP JP2024532831A patent/JP2024543214A/ja active Pending
- 2022-12-02 PE PE2024001294A patent/PE20250677A1/es unknown
- 2022-12-02 CA CA3239482A patent/CA3239482A1/en active Pending
- 2022-12-02 IL IL313203A patent/IL313203A/en unknown
- 2022-12-02 US US18/715,644 patent/US20250051766A1/en active Pending
- 2022-12-02 WO PCT/US2022/051713 patent/WO2023102225A2/en not_active Ceased
- 2022-12-02 KR KR1020247021710A patent/KR20240129627A9/ko active Pending
- 2022-12-02 CR CR20240269A patent/CR20240269A/es unknown
-
2024
- 2024-06-02 JO JOJO/P/2024/0125A patent/JOP20240125A1/ar unknown
- 2024-06-03 DO DO2024000107A patent/DOP2024000107A/es unknown
- 2024-06-03 CL CL2024001657A patent/CL2024001657A1/es unknown
- 2024-06-21 CO CONC2024/0008076A patent/CO2024008076A2/es unknown
Also Published As
| Publication number | Publication date |
|---|---|
| EP4441225A2 (en) | 2024-10-09 |
| AU2022400851A1 (en) | 2024-06-20 |
| IL313203A (en) | 2024-07-01 |
| EP4441225A4 (en) | 2026-03-11 |
| WO2023102225A2 (en) | 2023-06-08 |
| JOP20240125A1 (ar) | 2024-06-02 |
| CR20240269A (es) | 2024-09-13 |
| CN119790152A (zh) | 2025-04-08 |
| CO2024008076A2 (es) | 2024-11-18 |
| WO2023102225A3 (en) | 2024-03-28 |
| US20250051766A1 (en) | 2025-02-13 |
| KR20240129627A (ko) | 2024-08-27 |
| CA3239482A1 (en) | 2023-06-08 |
| MX2024006763A (es) | 2024-08-15 |
| CL2024001657A1 (es) | 2024-12-20 |
| PE20250677A1 (es) | 2025-03-04 |
| JP2024543214A (ja) | 2024-11-19 |
| DOP2024000107A (es) | 2024-09-30 |
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| KR20240129627A9 (ko) | Unc13a 유전자 전사체의 조정제를 사용한 신경계 질환의 치료 | |
| EP4010072A4 (en) | TREATMENT OF CENTRAL NERVOUS SYSTEM DISEASES | |
| EP3957357C0 (en) | MULTI-CHANNEL STIMULATION SYSTEM FOR REGENERATION OF INJURED CORNEAL NERVES | |
| EP3813704B8 (en) | Systems for treatment of nasal disorders | |
| GB2605422B (en) | Treatment of the central nervous system | |
| HRP20251579T1 (hr) | Pegilirana cistationin beta sintaza za enzimsku terapiju za liječenje homocistinurije | |
| IL313920A (en) | Treatment of heart and lung disorders | |
| DK3956310T3 (da) | Modificerede aminosyrederivater til behandling af neurologiske sygdomme og udvalgte psykiatriske lidelser | |
| EP4028123A4 (en) | Delivery of therapeutic neuromodulation | |
| GB201914034D0 (en) | Treatment of neurological disorders | |
| IL282360A (en) | Treatment for neurological diseases | |
| IL263080B (en) | Treatment of neurological disorders | |
| EP3844294A4 (en) | GENE THERAPY FOR THE TREATMENT OF GALACTOSEMIA | |
| EP4096680A4 (en) | Antisense oligonucleotides for treatment of neurological disorders | |
| EP4514357A4 (en) | Methods for the treatment of neurological disorders | |
| IL313539A (en) | Methods for treating neurological disorders | |
| KR20240099184A9 (ko) | 재생불량성 질환 치료를 위한 mrna 레귤론 요법 | |
| EP3630986A4 (en) | GENE THERAPY FOR TREATMENT OF PEROXISOMAL DISEASES | |
| EP4514341A4 (en) | Treatment of neurological disorders | |
| IL289012A (en) | Treatment of cns disorders with sleep disturbances | |
| HUE071387T2 (hu) | 4-fenil-tetrahidropiridin-származékok hallásbetegségek kezelésére | |
| EP4419671A4 (en) | GENE THERAPY FOR THE TREATMENT OF WILSON'S DISEASE | |
| HK40130763A (zh) | 梭菌神经毒素变体用於治疗神经障碍的用途 | |
| IL315909A (en) | Gene therapy for the treatment of cognitive disorders | |
| HK40105908A (en) | Sirna targeting tmprss6 for the treatment of myeloproliferative disorders |
Legal Events
| Date | Code | Title | Description |
|---|---|---|---|
| PA0105 | International application |
St.27 status event code: A-0-1-A10-A15-nap-PA0105 |
|
| PG1501 | Laying open of application |
St.27 status event code: A-1-1-Q10-Q12-nap-PG1501 |
|
| E13 | Pre-grant limitation requested |
Free format text: ST27 STATUS EVENT CODE: A-2-3-E10-E13-LIM-X000 (AS PROVIDED BY THE NATIONAL OFFICE) |
|
| E13-X000 | Pre-grant limitation requested |
St.27 status event code: A-2-3-E10-E13-lim-X000 |
|
| P11 | Amendment of application requested |
Free format text: ST27 STATUS EVENT CODE: A-2-2-P10-P11-NAP-X000 (AS PROVIDED BY THE NATIONAL OFFICE) |
|
| P11-X000 | Amendment of application requested |
St.27 status event code: A-2-2-P10-P11-nap-X000 |
|
| P19 | Errors in documents containing ipogçös decisions corrected |
Free format text: ST27 STATUS EVENT CODE: A-3-3-P10-P19-OTH-PG1701 (AS PROVIDED BY THE NATIONAL OFFICE) |
|
| PG1701 | Publication of correction |
St.27 status event code: A-3-3-P10-P19-oth-PG1701 Patent document republication publication date: 20251210 Republication note text: Request for Correction Notice (Document Request) Gazette number: 1020240129627 Gazette reference publication date: 20240827 |