CL2024001657A1 - Tratamiento de enfermedades neurológicas mediante el uso de moduladores de transcripciones génicas de unc13a - Google Patents

Tratamiento de enfermedades neurológicas mediante el uso de moduladores de transcripciones génicas de unc13a

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Publication number
CL2024001657A1
CL2024001657A1 CL2024001657A CL2024001657A CL2024001657A1 CL 2024001657 A1 CL2024001657 A1 CL 2024001657A1 CL 2024001657 A CL2024001657 A CL 2024001657A CL 2024001657 A CL2024001657 A CL 2024001657A CL 2024001657 A1 CL2024001657 A1 CL 2024001657A1
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CL
Chile
Prior art keywords
unc13a
neurological diseases
modulators
treatment
gene transcription
Prior art date
Application number
CL2024001657A
Other languages
English (en)
Spanish (es)
Inventor
Hinckley Sandra
Brown Duncan
Elbaum Daniel
Elizabeth Kamelgarn Marisa
Original Assignee
Quralis Corp
Priority date (The priority date 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 date listed.)
Filing date
Publication date
Application filed by Quralis Corp filed Critical Quralis Corp
Publication of CL2024001657A1 publication Critical patent/CL2024001657A1/es

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    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
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    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • A61K31/712Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
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    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61P25/00Drugs for disorders of the nervous system
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    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
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    • C12N2310/32Chemical structure of the sugar
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    • C12N2310/30Chemical structure
    • C12N2310/35Nature of the modification
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    • C12N2310/3525MOE, methoxyethoxy
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    • C12N2320/00Applications; Uses
    • C12N2320/30Special therapeutic applications
    • C12N2320/33Alteration of splicing

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  • Wood Science & Technology (AREA)
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  • Biotechnology (AREA)
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  • Animal Behavior & Ethology (AREA)
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  • 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)
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  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
CL2024001657A 2021-12-03 2024-06-03 Tratamiento de enfermedades neurológicas mediante el uso de moduladores de transcripciones génicas de unc13a CL2024001657A1 (es)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202163285786P 2021-12-03 2021-12-03
US202263350206P 2022-06-08 2022-06-08
US202263398987P 2022-08-18 2022-08-18

Publications (1)

Publication Number Publication Date
CL2024001657A1 true CL2024001657A1 (es) 2024-12-20

Family

ID=86613080

Family Applications (1)

Application Number Title Priority Date Filing Date
CL2024001657A CL2024001657A1 (es) 2021-12-03 2024-06-03 Tratamiento de enfermedades neurológicas mediante el uso de moduladores de transcripciones génicas de unc13a

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)

* Cited by examiner, † Cited by third party
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)

* Cited by examiner, † Cited by third party
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

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
PE20250677A1 (es) 2025-03-04
KR20240129627A9 (ko) 2025-12-10
JP2024543214A (ja) 2024-11-19
DOP2024000107A (es) 2024-09-30

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