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WO2024175586A3 - Inhibitors of synaptogyrin-3 expression - Google Patents

Inhibitors of synaptogyrin-3 expression Download PDF

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Publication number
WO2024175586A3
WO2024175586A3 PCT/EP2024/054276 EP2024054276W WO2024175586A3 WO 2024175586 A3 WO2024175586 A3 WO 2024175586A3 EP 2024054276 W EP2024054276 W EP 2024054276W WO 2024175586 A3 WO2024175586 A3 WO 2024175586A3
Authority
WO
WIPO (PCT)
Prior art keywords
synaptogyrin
inhibitors
expression
tauopathies
targetable
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.)
Ceased
Application number
PCT/EP2024/054276
Other languages
French (fr)
Other versions
WO2024175586A2 (en
Inventor
Ana Rita SANTOS
Patrik Verstreken
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Jay Therapeutics
Katholieke Universiteit Leuven
Vlaams Instituut voor Biotechnologie VIB
Original Assignee
Jay Therapeutics
Katholieke Universiteit Leuven
Vlaams Instituut voor Biotechnologie VIB
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 Jay Therapeutics, Katholieke Universiteit Leuven, Vlaams Instituut voor Biotechnologie VIB filed Critical Jay Therapeutics
Priority to EP24706719.2A priority Critical patent/EP4669750A2/en
Publication of WO2024175586A2 publication Critical patent/WO2024175586A2/en
Publication of WO2024175586A3 publication Critical patent/WO2024175586A3/en
Anticipated expiration legal-status Critical
Ceased legal-status Critical Current

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
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
    • 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
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Molecular Biology (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • General Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biochemistry (AREA)
  • Epidemiology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Medicinal Chemistry (AREA)
  • Plant Pathology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Microbiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)

Abstract

The invention relates to identification of regions within the synaptogyrin-3 RNA sequence that are targetable by oligonucleotide inhibitors such as siRNA molecules. The synaptogyrin-3 inhibitors disclosed herein are provided for use as a medicament in general, and for treating or inhibiting progression of tauopathies or symptoms of tauopathies in particular.
PCT/EP2024/054276 2023-02-21 2024-02-20 Inhibitors of synaptogyrin-3 expression Ceased WO2024175586A2 (en)

Priority Applications (1)

Application Number Priority Date Filing Date Title
EP24706719.2A EP4669750A2 (en) 2023-02-21 2024-02-20 HIBITORS OF SYNAPTOGYRIN-3 EXPRESSION

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
EP23157699 2023-02-21
EP23157699.2 2023-02-21

Publications (2)

Publication Number Publication Date
WO2024175586A2 WO2024175586A2 (en) 2024-08-29
WO2024175586A3 true WO2024175586A3 (en) 2024-10-03

Family

ID=85321107

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/EP2024/054276 Ceased WO2024175586A2 (en) 2023-02-21 2024-02-20 Inhibitors of synaptogyrin-3 expression

Country Status (2)

Country Link
EP (1) EP4669750A2 (en)
WO (1) WO2024175586A2 (en)

Citations (10)

* Cited by examiner, † Cited by third party
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WO2004045543A2 (en) * 2002-11-14 2004-06-03 Dharmacon, Inc. Functional and hyperfunctional sirna
WO2007106407A2 (en) * 2006-03-10 2007-09-20 Wyeth Microarray for monitoring gene expression in multiple strains of streptococcus pneumoniae
WO2008142034A2 (en) * 2007-05-22 2008-11-27 Basf Plant Science Gmbh Plants with increased tolerance and/or resistance to environmental stress and increased biomass production
US20110166037A1 (en) * 2006-03-31 2011-07-07 Affymetrix, Inc. Analysis of methylation using nucleic acid arrays
WO2012065143A1 (en) * 2010-11-12 2012-05-18 The General Hospital Corporation Polycomb-associated non-coding rnas
WO2014058317A1 (en) * 2012-10-10 2014-04-17 Stichting Het Nederlands Kanker Instituut-Antoni van Leeuwenhoek Ziekenhuis Methods and means for predicting resistance to anti-cancer treatment
WO2019016123A1 (en) * 2017-07-17 2019-01-24 Vib Vzw Targeting synaptogyrin-3 in tauopathy treatment
US10961585B2 (en) * 2018-08-08 2021-03-30 Pml Screening, Llc Methods for assessing risk of developing a viral of disease using a genetic test
US20220266037A1 (en) * 2018-04-27 2022-08-25 Boston Scientific Neuromodulation Corporation Multi-mode electrical stimulation systems and methods of making and using
WO2022212208A1 (en) * 2021-03-29 2022-10-06 University Of Massachusetts Oligonucleotides for syngr-3 modulation

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JP3756313B2 (en) 1997-03-07 2006-03-15 武 今西 Novel bicyclonucleosides and oligonucleotide analogues
NZ503765A (en) 1997-09-12 2002-04-26 Exiqon As Bi-cyclic and tri-cyclic nucleotide analogues
EP1152009B2 (en) 1999-02-12 2017-09-06 Daiichi Sankyo Company, Limited Novel nucleosides and oligonucleotide analogues
JP2002543214A (en) 1999-05-04 2002-12-17 エクシコン エ／エス L-ribo-LNA analog
IL155991A0 (en) 2000-12-01 2003-12-23 Max Planck Gesellschaft Rna interference mediating small rna molecules
AU2003281969B2 (en) 2002-11-18 2011-01-27 Roche Innovation Center Copenhagen A/S Amino-LNA, thio-LNA and alpha-L-oxy-LN
US8110674B2 (en) 2003-03-07 2012-02-07 Alnylam Pharmaceuticals, Inc. Therapeutic compositions
US8653252B2 (en) 2003-03-21 2014-02-18 Santaris Pharma A/S Short interfering RNA (siRNA) analogues
US7341847B2 (en) 2003-04-02 2008-03-11 Agency For Science, Technology And Research Promoter construct for gene expression in neuronal cells
WO2007031091A2 (en) 2005-09-15 2007-03-22 Santaris Pharma A/S Rna antagonist compounds for the modulation of p21 ras expression
CN102908630B (en) 2006-01-27 2014-11-19 Isis制药公司 6-modified bicyclic nucleic acid analogs
CA2644347C (en) 2006-03-23 2017-05-30 Santaris Pharma A/S Small internally segmented interfering rna
ES2386578T3 (en) 2006-05-05 2012-08-23 Isis Pharmaceuticals, Inc. Compounds and procedures to modulate PCSK9 expression
US7666854B2 (en) 2006-05-11 2010-02-23 Isis Pharmaceuticals, Inc. Bis-modified bicyclic nucleic acid analogs
US7547684B2 (en) 2006-05-11 2009-06-16 Isis Pharmaceuticals, Inc. 5′-modified bicyclic nucleic acid analogs
WO2008150729A2 (en) 2007-05-30 2008-12-11 Isis Pharmaceuticals, Inc. N-substituted-aminomethylene bridged bicyclic nucleic acid analogs
ES2386492T3 (en) 2007-06-08 2012-08-21 Isis Pharmaceuticals, Inc. Carbocyclic bicyclic nucleic acid analogs
ATE538127T1 (en) 2007-07-05 2012-01-15 Isis Pharmaceuticals Inc 6-DISUBSTITUTED BICYCLIC NUCLEIC ACID ANALOGUES
US8546556B2 (en) 2007-11-21 2013-10-01 Isis Pharmaceuticals, Inc Carbocyclic alpha-L-bicyclic nucleic acid analogs
WO2010036698A1 (en) 2008-09-24 2010-04-01 Isis Pharmaceuticals, Inc. Substituted alpha-l-bicyclic nucleosides
WO2011156202A1 (en) 2010-06-08 2011-12-15 Isis Pharmaceuticals, Inc. Substituted 2 '-amino and 2 '-thio-bicyclic nucleosides and oligomeric compounds prepared therefrom
AU2011302152B2 (en) 2010-09-15 2015-06-11 Alnylam Pharmaceuticals, Inc. Modified iRNA agents
LT3366775T (en) 2011-11-18 2022-08-10 Alnylam Pharmaceuticals, Inc. Modified rnai agents
IL316808A (en) 2014-08-20 2025-01-01 Alnylam Pharmaceuticals Inc Modified double-stranded rna agents and uses thereof
US11504391B1 (en) 2016-11-23 2022-11-22 Alnylam Pharmaceuticals, Inc. Modified RNA agents with reduced off-target effect
EP4022059A4 (en) 2019-10-06 2023-11-01 Wave Life Sciences Ltd. OLIGONUCLEOTIDE COMPOSITIONS AND METHODS OF USE THEREOF
IL302558A (en) 2020-11-08 2023-07-01 Wave Life Sciences Ltd Oligonucleotide compositions and methods thereof

Patent Citations (15)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP2213738A2 (en) * 2002-11-14 2010-08-04 Dharmacon, Inc. siRNA molecules targeting Bcl-2
WO2004045543A2 (en) * 2002-11-14 2004-06-03 Dharmacon, Inc. Functional and hyperfunctional sirna
WO2007106407A2 (en) * 2006-03-10 2007-09-20 Wyeth Microarray for monitoring gene expression in multiple strains of streptococcus pneumoniae
US20150038352A1 (en) * 2006-03-31 2015-02-05 Affymetrix, Inc. Analysis of methylation using nucleic acid arrays
US20110166037A1 (en) * 2006-03-31 2011-07-07 Affymetrix, Inc. Analysis of methylation using nucleic acid arrays
US20180251841A1 (en) * 2006-03-31 2018-09-06 Affymetrix, Inc. Analysis of methylation using nucleic acid arrays
WO2008142034A2 (en) * 2007-05-22 2008-11-27 Basf Plant Science Gmbh Plants with increased tolerance and/or resistance to environmental stress and increased biomass production
US10119144B2 (en) * 2010-11-12 2018-11-06 The General Hospital Corporation Polycomb-associated non-coding RNAs
WO2012065143A1 (en) * 2010-11-12 2012-05-18 The General Hospital Corporation Polycomb-associated non-coding rnas
WO2014058317A1 (en) * 2012-10-10 2014-04-17 Stichting Het Nederlands Kanker Instituut-Antoni van Leeuwenhoek Ziekenhuis Methods and means for predicting resistance to anti-cancer treatment
WO2019016123A1 (en) * 2017-07-17 2019-01-24 Vib Vzw Targeting synaptogyrin-3 in tauopathy treatment
EP3655013A1 (en) * 2017-07-17 2020-05-27 VIB vzw Targeting synaptogyrin-3 in tauopathy treatment
US20220266037A1 (en) * 2018-04-27 2022-08-25 Boston Scientific Neuromodulation Corporation Multi-mode electrical stimulation systems and methods of making and using
US10961585B2 (en) * 2018-08-08 2021-03-30 Pml Screening, Llc Methods for assessing risk of developing a viral of disease using a genetic test
WO2022212208A1 (en) * 2021-03-29 2022-10-06 University Of Massachusetts Oligonucleotides for syngr-3 modulation

Also Published As

Publication number Publication date
EP4669750A2 (en) 2025-12-31
WO2024175586A2 (en) 2024-08-29

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