WO2026072709A1 - Récepteurs antigéniques chimériques ciblant bcma - Google Patents

Récepteurs antigéniques chimériques ciblant bcma

Info

Publication number
WO2026072709A1
WO2026072709A1 PCT/US2025/047769 US2025047769W WO2026072709A1 WO 2026072709 A1 WO2026072709 A1 WO 2026072709A1 US 2025047769 W US2025047769 W US 2025047769W WO 2026072709 A1 WO2026072709 A1 WO 2026072709A1
Authority
WO
WIPO (PCT)
Prior art keywords
mol
pharmaceutical composition
sequence
lipid
rna
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
Application number
PCT/US2025/047769
Other languages
English (en)
Inventor
Robert Mabry
Isin DALKILIC-LIDDLE
Jui DUTTA-SIMMONS
Rebecca SILVER
Muthusamy Jayaraman
Ganapathy Subramanian SANKARAN
Karolina Anna KOSAKOWSKA
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.)
Orna Therapeutics Inc
Renagade Therapeutics Management Inc
Original Assignee
Orna Therapeutics Inc
Renagade Therapeutics Management Inc
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 Orna Therapeutics Inc, Renagade Therapeutics Management Inc filed Critical Orna Therapeutics Inc
Publication of WO2026072709A1 publication Critical patent/WO2026072709A1/fr
Pending legal-status Critical Current
Anticipated expiration legal-status Critical

Links

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    • 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
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K40/00—Cellular immunotherapy
    • A61K40/10—Cellular immunotherapy characterised by the cell type used
    • A61K40/11—T-cells, e.g. tumour infiltrating lymphocytes [TIL] or regulatory T [Treg] cells; Lymphokine-activated killer [LAK] cells
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K40/00—Cellular immunotherapy
    • A61K40/30—Cellular immunotherapy characterised by the recombinant expression of specific molecules in the cells of the immune system
    • A61K40/31—Chimeric antigen receptors [CAR]
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K40/00—Cellular immunotherapy
    • A61K40/40—Cellular immunotherapy characterised by antigens that are targeted or presented by cells of the immune system
    • A61K40/41—Vertebrate antigens
    • A61K40/42—Cancer antigens
    • A61K40/4202—Receptors, cell surface antigens or cell surface determinants
    • A61K40/4214—Receptors for cytokines
    • A61K40/4215—Receptors for tumor necrosis factors [TNF], e.g. lymphotoxin receptor [LTR], CD30
    • 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
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/88—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation using microencapsulation, e.g. using amphiphile liposome vesicle
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K16/00—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies
    • C07K16/18—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material from animals or humans
    • C07K16/28—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants
    • C07K16/2878—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants against the NGF-receptor/TNF-receptor superfamily, e.g. CD27, CD30, CD40, CD95
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K16/00—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies
    • C07K16/18—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material from animals or humans
    • C07K16/32—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material from animals or humans against translation products of oncogenes
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2317/00—Immunoglobulins specific features
    • C07K2317/50—Immunoglobulins specific features characterized by immunoglobulin fragments
    • C07K2317/56—Immunoglobulins specific features characterized by immunoglobulin fragments variable (Fv) region, i.e. VH and/or VL
    • C07K2317/569—Single domain, e.g. dAb, sdAb, VHH, VNAR or nanobody®
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2317/00—Immunoglobulins specific features
    • C07K2317/60—Immunoglobulins specific features characterized by non-natural combinations of immunoglobulin fragments
    • C07K2317/62—Immunoglobulins specific features characterized by non-natural combinations of immunoglobulin fragments comprising only variable region components
    • C07K2317/622—Single chain antibody (scFv)
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2317/00—Immunoglobulins specific features
    • C07K2317/70—Immunoglobulins specific features characterized by effect upon binding to a cell or to an antigen
    • C07K2317/77—Internalization into the cell
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/03—Fusion polypeptide containing a localisation/targetting motif containing a transmembrane segment
    • 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
    • C12N2840/00—Vectors comprising a special translation-regulating system
    • C12N2840/20—Vectors comprising a special translation-regulating system translation of more than one cistron
    • C12N2840/203—Vectors comprising a special translation-regulating system translation of more than one cistron having an IRES

Landscapes

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

Abstract

L'invention concerne des séquences d'ARN codant pour un récepteur antigénique chimérique (CAR) comprenant une molécule de liaison à l'antigène de maturation des lymphocytes B (BCMA), ainsi que des compositions et des méthodes associées. Dans certains modes de réalisation, l'invention décrit des ARN circulaires codant pour la molécule de liaison à BCMA ou le CAR BCMA, ainsi que des compositions, des méthodes et des précurseurs associés. Dans certains modes de réalisation, ces compositions comprennent un véhicule de transfert, par exemple, comprenant un lipide ionisable décrit ici.
PCT/US2025/047769 2024-09-25 2025-09-24 Récepteurs antigéniques chimériques ciblant bcma Pending WO2026072709A1 (fr)

Applications Claiming Priority (6)

Application Number Priority Date Filing Date Title
US202463699026P 2024-09-25 2024-09-25
US63/699,026 2024-09-25
US202563743962P 2025-01-10 2025-01-10
US63/743,962 2025-01-10
US202563825337P 2025-06-17 2025-06-17
US63/825,337 2025-06-17

Publications (1)

Publication Number Publication Date
WO2026072709A1 true WO2026072709A1 (fr) 2026-04-02

Family

ID=97598916

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2025/047769 Pending WO2026072709A1 (fr) 2024-09-25 2025-09-24 Récepteurs antigéniques chimériques ciblant bcma

Country Status (1)

Country Link
WO (1) WO2026072709A1 (fr)

Citations (41)

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Publication number Priority date Publication date Assignee Title
WO1993013121A1 (fr) 1991-12-24 1993-07-08 Isis Pharmaceuticals, Inc. Oligonucleotides modifies en 2', a ouverture
US5378825A (en) 1990-07-27 1995-01-03 Isis Pharmaceuticals, Inc. Backbone modified oligonucleotide analogs
WO1995032305A1 (fr) 1994-05-19 1995-11-30 Dako A/S Sondes d'acide nucleique peptidique de detection de neisseria gonorrhoeae et de chlamydia trachomatis
US5585481A (en) 1987-09-21 1996-12-17 Gen-Probe Incorporated Linking reagents for nucleotide probes
US5948902A (en) 1997-11-20 1999-09-07 South Alabama Medical Science Foundation Antisense oligonucleotides to human serine/threonine protein phosphatase genes
US6319494B1 (en) 1990-12-14 2001-11-20 Cell Genesys, Inc. Chimeric chains for receptor-associated signal transduction pathways
US20040014194A1 (en) 2002-03-27 2004-01-22 Schering Corporation Beta-secretase crystals and methods for preparing and using the same
US7741465B1 (en) 1992-03-18 2010-06-22 Zelig Eshhar Chimeric receptor genes and cells transformed therewith
WO2014068079A1 (fr) 2012-11-01 2014-05-08 Max-Delbrück-Centrum für Molekulare Medizin Anticorps qui se lie à cd269 (bcma) pouvant être utilisé dans le traitement de maladies des cellules plasmatiques comme le myélome multiple et les maladies auto-immunes
WO2015158671A1 (fr) 2014-04-14 2015-10-22 Cellectis Récepteurs antigéniques chimériques spécifiques de bcma (cd269), utiles dans l'immunothérapie du cancer
WO2016014565A2 (fr) 2014-07-21 2016-01-28 Novartis Ag Traitement du cancer au moyen d'un récepteur d'antigène chimérique anti-bcma humanisé
WO2016090320A1 (fr) 2014-12-05 2016-06-09 Memorial Sloan-Kettering Cancer Center Récepteurs antigéniques chimériques ciblant l'antigène de maturation des cellules b et leurs utilisations
WO2017025038A1 (fr) 2015-08-11 2017-02-16 Nanjing Legend Biotech Co., Ltd. Récepteurs d'antigènes chimériques basés sur des anticorps à domaine unique et leurs méthodes d'utilisation
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WO2018028647A1 (fr) 2016-08-10 2018-02-15 Legend Biotech Usa Inc. Récepteurs d'antigène chimériques ciblant bcma et leurs procédés d'utilisation
WO2019006072A1 (fr) 2017-06-30 2019-01-03 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Récepteurs antigéniques chimériques vers l'antigène de maturation des lymphocytes b présentant des domaines humains
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WO2020150339A1 (fr) 2019-01-16 2020-07-23 Caribou Biosciences, Inc. Anticorps bcma humanisé et cellules bcma-car-t
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WO2020237227A1 (fr) 2019-05-22 2020-11-26 Massachusetts Institute Of Technology Compositions et procédés d'arn circulaire
WO2020243546A1 (fr) 2019-05-30 2020-12-03 Lentigen Technology, Inc. Compositions et méthodes de traitement du cancer par immunothérapie anti-bcma
WO2021113777A2 (fr) 2019-12-04 2021-06-10 Orna Therapeutics, Inc. Méthodes et compositions d'arn circulaire
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WO2022061490A1 (fr) 2020-09-22 2022-03-31 华为数字能源技术有限公司 Système de génération d'énergie photovoltaïque, et procédé et dispositif de détection de défaut de masse dans des chaînes photovoltaïques
US20220251578A1 (en) 2020-06-25 2022-08-11 The Board Of Trustees Of The Leland Stanford Junior University Genetic elements driving circular rna translation and methods of use
US20220288176A1 (en) 2019-08-28 2022-09-15 The Board Of Trustees Of The Leland Stanford Junior University Circular rna modification and methods of use
WO2022261490A2 (fr) 2021-06-10 2022-12-15 Orna Therapeutics, Inc. Méthodes et compositions d'arn circulaire
WO2022271965A2 (fr) 2021-06-25 2022-12-29 The Board Of Trustees Of The Leland Stanford Junior University Compositions et procédés pour une traduction de protéines améliorée à partir d'arn circulaires recombinants
WO2023056033A1 (fr) 2021-09-30 2023-04-06 Orna Therapeutics, Inc. Compositions de nanoparticules lipidiques pour l'administration de polynucléotides circulaires
WO2023081526A1 (fr) 2021-11-08 2023-05-11 Orna Therapeutics, Inc. Compositions de nanoparticules lipidiques pour l'administration de polynucléotides circulaires
WO2023098846A1 (fr) 2021-12-03 2023-06-08 江苏先声药业有限公司 Nanocorps anti-bcma et son utilisation
WO2023141586A1 (fr) 2022-01-21 2023-07-27 Orna Therapeutics, Inc. Administration systémique de polynucléotides d'arn circulaire codant pour des protéines musculaires ou des complexes protéiques
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WO2023250375A1 (fr) 2022-06-24 2023-12-28 Orna Therapeutics, Inc. Arn circulaire codant pour des récepteurs antigéniques chimériques ciblant bcma
WO2024102677A1 (fr) 2022-11-08 2024-05-16 Orna Therapeutics, Inc. Compositions d'arn circulaire

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* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5585481A (en) 1987-09-21 1996-12-17 Gen-Probe Incorporated Linking reagents for nucleotide probes
US5378825A (en) 1990-07-27 1995-01-03 Isis Pharmaceuticals, Inc. Backbone modified oligonucleotide analogs
US6319494B1 (en) 1990-12-14 2001-11-20 Cell Genesys, Inc. Chimeric chains for receptor-associated signal transduction pathways
WO1993013121A1 (fr) 1991-12-24 1993-07-08 Isis Pharmaceuticals, Inc. Oligonucleotides modifies en 2', a ouverture
US7741465B1 (en) 1992-03-18 2010-06-22 Zelig Eshhar Chimeric receptor genes and cells transformed therewith
WO1995032305A1 (fr) 1994-05-19 1995-11-30 Dako A/S Sondes d'acide nucleique peptidique de detection de neisseria gonorrhoeae et de chlamydia trachomatis
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US20040014194A1 (en) 2002-03-27 2004-01-22 Schering Corporation Beta-secretase crystals and methods for preparing and using the same
WO2014068079A1 (fr) 2012-11-01 2014-05-08 Max-Delbrück-Centrum für Molekulare Medizin Anticorps qui se lie à cd269 (bcma) pouvant être utilisé dans le traitement de maladies des cellules plasmatiques comme le myélome multiple et les maladies auto-immunes
WO2015158671A1 (fr) 2014-04-14 2015-10-22 Cellectis Récepteurs antigéniques chimériques spécifiques de bcma (cd269), utiles dans l'immunothérapie du cancer
WO2016014565A2 (fr) 2014-07-21 2016-01-28 Novartis Ag Traitement du cancer au moyen d'un récepteur d'antigène chimérique anti-bcma humanisé
WO2016090320A1 (fr) 2014-12-05 2016-06-09 Memorial Sloan-Kettering Cancer Center Récepteurs antigéniques chimériques ciblant l'antigène de maturation des cellules b et leurs utilisations
WO2017025038A1 (fr) 2015-08-11 2017-02-16 Nanjing Legend Biotech Co., Ltd. Récepteurs d'antigènes chimériques basés sur des anticorps à domaine unique et leurs méthodes d'utilisation
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WO2019006072A1 (fr) 2017-06-30 2019-01-03 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Récepteurs antigéniques chimériques vers l'antigène de maturation des lymphocytes b présentant des domaines humains
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WO2020237227A1 (fr) 2019-05-22 2020-11-26 Massachusetts Institute Of Technology Compositions et procédés d'arn circulaire
WO2020243546A1 (fr) 2019-05-30 2020-12-03 Lentigen Technology, Inc. Compositions et méthodes de traitement du cancer par immunothérapie anti-bcma
US20220288176A1 (en) 2019-08-28 2022-09-15 The Board Of Trustees Of The Leland Stanford Junior University Circular rna modification and methods of use
WO2021113777A2 (fr) 2019-12-04 2021-06-10 Orna Therapeutics, Inc. Méthodes et compositions d'arn circulaire
WO2021189059A2 (fr) 2020-03-20 2021-09-23 Orna Therapeutics, Inc. Méthodes et compositions d'arn circulaire
WO2021226597A2 (fr) 2020-05-08 2021-11-11 Orna Therapeutics, Inc. Compositions d'arn circulaire et méthodes
WO2021236855A1 (fr) 2020-05-19 2021-11-25 Orna Therapeutics, Inc. Méthodes et compositions d'arn circulaire
US20220251578A1 (en) 2020-06-25 2022-08-11 The Board Of Trustees Of The Leland Stanford Junior University Genetic elements driving circular rna translation and methods of use
WO2022061490A1 (fr) 2020-09-22 2022-03-31 华为数字能源技术有限公司 Système de génération d'énergie photovoltaïque, et procédé et dispositif de détection de défaut de masse dans des chaînes photovoltaïques
WO2022261490A2 (fr) 2021-06-10 2022-12-15 Orna Therapeutics, Inc. Méthodes et compositions d'arn circulaire
WO2022271965A2 (fr) 2021-06-25 2022-12-29 The Board Of Trustees Of The Leland Stanford Junior University Compositions et procédés pour une traduction de protéines améliorée à partir d'arn circulaires recombinants
WO2023056033A1 (fr) 2021-09-30 2023-04-06 Orna Therapeutics, Inc. Compositions de nanoparticules lipidiques pour l'administration de polynucléotides circulaires
WO2023081526A1 (fr) 2021-11-08 2023-05-11 Orna Therapeutics, Inc. Compositions de nanoparticules lipidiques pour l'administration de polynucléotides circulaires
WO2023098846A1 (fr) 2021-12-03 2023-06-08 江苏先声药业有限公司 Nanocorps anti-bcma et son utilisation
WO2023138666A1 (fr) * 2022-01-19 2023-07-27 Utc Therapeutics (Shanghai) Co., Ltd. Arn circulaire et son utilisation
WO2023141586A1 (fr) 2022-01-21 2023-07-27 Orna Therapeutics, Inc. Administration systémique de polynucléotides d'arn circulaire codant pour des protéines musculaires ou des complexes protéiques
WO2023250375A1 (fr) 2022-06-24 2023-12-28 Orna Therapeutics, Inc. Arn circulaire codant pour des récepteurs antigéniques chimériques ciblant bcma
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WO2024102677A1 (fr) 2022-11-08 2024-05-16 Orna Therapeutics, Inc. Compositions d'arn circulaire

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