EP4010004A4 - Compositions et méthodes de modulation d'expression génique - Google Patents

Compositions et méthodes de modulation d'expression génique Download PDF

Info

Publication number
EP4010004A4
EP4010004A4 EP20851042.0A EP20851042A EP4010004A4 EP 4010004 A4 EP4010004 A4 EP 4010004A4 EP 20851042 A EP20851042 A EP 20851042A EP 4010004 A4 EP4010004 A4 EP 4010004A4
Authority
EP
European Patent Office
Prior art keywords
modulation
compositions
methods
gene expression
gene
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
EP20851042.0A
Other languages
German (de)
English (en)
Other versions
EP4010004A2 (fr
Inventor
Fyodor Urnov
John A. Stamatoyannopoulos
Joycelynn PEARL
Matthew WILKEN
Christie CIARLO
Shon GREEN
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.)
Altius Institute for Biomedical Sciences
Original Assignee
Altius Institute for Biomedical Sciences
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 Altius Institute for Biomedical Sciences filed Critical Altius Institute for Biomedical Sciences
Publication of EP4010004A2 publication Critical patent/EP4010004A2/fr
Publication of EP4010004A4 publication Critical patent/EP4010004A4/fr
Pending legal-status Critical Current

Links

Classifications

    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
    • C07K14/70503—Immunoglobulin superfamily
    • C07K14/7051—T-cell receptor (TcR)-CD3 complex
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
    • C07K14/70503—Immunoglobulin superfamily
    • C07K14/70521—CD28, CD152
    • 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
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
    • C07K14/70503—Immunoglobulin superfamily
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
    • C07K14/70596—Molecules with a "CD"-designation not provided for elsewhere
    • 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
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/70—Fusion polypeptide containing domain for protein-protein interaction
    • C07K2319/71—Fusion polypeptide containing domain for protein-protein interaction containing domain for transcriptional activaation, e.g. VP16
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/80—Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/80—Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor
    • C07K2319/81—Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor containing a Zn-finger domain for DNA binding
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/001—Vector systems having a special element relevant for transcription controllable enhancer/promoter combination
    • C12N2830/005—Vector systems having a special element relevant for transcription controllable enhancer/promoter combination repressible enhancer/promoter combination, e.g. KRAB
    • 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
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases RNAses, DNAses

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Immunology (AREA)
  • Zoology (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • Molecular Biology (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Cell Biology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • Wood Science & Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Medicinal Chemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Toxicology (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Peptides Or Proteins (AREA)
EP20851042.0A 2019-08-07 2020-08-06 Compositions et méthodes de modulation d'expression génique Pending EP4010004A4 (fr)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US201962884028P 2019-08-07 2019-08-07
US201962898434P 2019-09-10 2019-09-10
US201962937011P 2019-11-18 2019-11-18
PCT/US2020/045174 WO2021026336A2 (fr) 2019-08-07 2020-08-06 Compositions et méthodes de modulation d'expression génique

Publications (2)

Publication Number Publication Date
EP4010004A2 EP4010004A2 (fr) 2022-06-15
EP4010004A4 true EP4010004A4 (fr) 2023-09-13

Family

ID=74502663

Family Applications (1)

Application Number Title Priority Date Filing Date
EP20851042.0A Pending EP4010004A4 (fr) 2019-08-07 2020-08-06 Compositions et méthodes de modulation d'expression génique

Country Status (2)

Country Link
EP (1) EP4010004A4 (fr)
WO (1) WO2021026336A2 (fr)

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20230287391A1 (en) * 2020-08-14 2023-09-14 The Governing Council Of The University Of Toronto Krab fusion repressors and methods and compositions for repressing gene expression
CN114591441B (zh) * 2021-10-18 2023-06-20 翌圣生物科技(上海)股份有限公司 重组TET酶MBD3-NgTET1及其在提高TET酶氧化产物中5caC占比的应用
CN114600837B (zh) * 2022-04-15 2023-05-02 润康生物医药(苏州)有限公司 一种粒细胞缺乏症动物模型及其构建方法以及ikzf1和cmyb在构建模型中的应用

Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2012104729A1 (fr) * 2011-02-02 2012-08-09 Ulla Bonas Domaines moléculaires de liaison à l'adn et leurs procédés d'utilisation
WO2014059173A2 (fr) * 2012-10-10 2014-04-17 Sangamo Biosciences, Inc. Composés modifiant les lymphocytes t et leurs utilisations
WO2017069958A2 (fr) * 2015-10-09 2017-04-27 The Brigham And Women's Hospital, Inc. Modulation de nouvelles cibles de points de contrôle immunitaires
WO2017093969A1 (fr) * 2015-12-04 2017-06-08 Novartis Ag Compositions et procédés d'immuno-oncologie

Family Cites Families (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6919204B2 (en) * 2000-09-29 2005-07-19 Sangamo Biosciences, Inc. Modulation of gene expression using localization domains
JP6144691B2 (ja) * 2011-11-16 2017-06-07 サンガモ セラピューティクス， インコーポレイテッド 修飾されたｄｎａ結合タンパク質およびその使用
KR102218562B1 (ko) * 2012-08-29 2021-02-19 상가모 테라퓨틱스, 인코포레이티드 유전적 병태를 치료하기 위한 방법 및 조성물
SI3068881T1 (sl) * 2013-11-13 2019-05-31 Children's Medical Center Corporation Z nukleazo posredovano uravnavanje izražanja genov
US9616090B2 (en) * 2014-07-30 2017-04-11 Sangamo Biosciences, Inc. Gene correction of SCID-related genes in hematopoietic stem and progenitor cells
EP3781193A4 (fr) * 2018-04-18 2022-01-26 Altius Institute For Biomedical Sciences Polypeptides dérivés de pathogènes animaux et leurs utilisations pour l'ingénierie génétique

Patent Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2012104729A1 (fr) * 2011-02-02 2012-08-09 Ulla Bonas Domaines moléculaires de liaison à l'adn et leurs procédés d'utilisation
WO2014059173A2 (fr) * 2012-10-10 2014-04-17 Sangamo Biosciences, Inc. Composés modifiant les lymphocytes t et leurs utilisations
WO2017069958A2 (fr) * 2015-10-09 2017-04-27 The Brigham And Women's Hospital, Inc. Modulation de nouvelles cibles de points de contrôle immunitaires
WO2017093969A1 (fr) * 2015-12-04 2017-06-08 Novartis Ag Compositions et procédés d'immuno-oncologie

Also Published As

Publication number Publication date
WO2021026336A3 (fr) 2021-03-11
WO2021026336A2 (fr) 2021-02-11
EP4010004A2 (fr) 2022-06-15

Similar Documents

Publication Publication Date Title
EP3510152A4 (fr) Méthodes et compositions pour moduler l'expression génique
EP3576737A4 (fr) Compositions et procédés de modulation de l'activité de la déshydrogénase à chaîne courte
EP3962296A4 (fr) Compositions de cannabinoïdes et procédés d'utilisation
EP3573623A4 (fr) Compositions et méthodes d'inhibition de l'expression génique du facteur xii
EP3781705A4 (fr) Compositions et méthodes pour l'édition génique
EP4054621A4 (fr) Compositions et procédés de remplacement d'allèles d'adn codé par l'arn
EP3883577A4 (fr) Compositions et procédés de modulation de l'activité de la déshydrogénase à chaîne courte
EP4010004A4 (fr) Compositions et méthodes de modulation d'expression génique
EP3768854A4 (fr) Modulation de l'expression de hsd17b13
EP3761972A4 (fr) Compositions bioréactives et leurs procédés d'utilisation
EP3906240A4 (fr) Compositions et procédés de modulation de la pousse des cheveux
EP3765058A4 (fr) Procédés et compositions pour expression inductible de facteurs neurotrophiques
EP3946374A4 (fr) Modulation, à base d'oligonucléotides, de c9orf72
EP3976798A4 (fr) Compositions et procédés de régulation sélective de gènes
EP3941927A4 (fr) Compositions et procédés de modification de molécules cibles
EP3982949A4 (fr) Inhibiteurs de sarm1
EP3980011A4 (fr) Inhibiteurs de sarm1
EP3980004A4 (fr) Compositions de tributyrine et procédés associés
EP3931305A4 (fr) Compositions et méthodes pour la culture à long terme d'hépatocytes
EP4003246A4 (fr) Compositions et procédés de traitement de la presbytie
EP3841114A4 (fr) Méthodes et compositions pour l'expression de l'arn d'inhibiteurs de myc
EP3976811A4 (fr) Caractérisation métabolomique de microorganismes
EP3950961A4 (fr) Application d'un gène kdm5a et d'un gène atrx
EP3445406A4 (fr) Compositions et méthodes permettant d'obtenir une expression améliorée du gène de pklr
EP3987024A4 (fr) Compositions et méthodes pour l'édition génomique améliorée

Legal Events

Date Code Title Description
STAA Information on the status of an ep patent application or granted ep patent

Free format text: STATUS: THE INTERNATIONAL PUBLICATION HAS BEEN MADE

PUAI Public reference made under article 153(3) epc to a published international application that has entered the european phase

Free format text: ORIGINAL CODE: 0009012

STAA Information on the status of an ep patent application or granted ep patent

Free format text: STATUS: REQUEST FOR EXAMINATION WAS MADE

17P Request for examination filed

Effective date: 20220210

AK Designated contracting states

Kind code of ref document: A2

Designated state(s): AL AT BE BG CH CY CZ DE DK EE ES FI FR GB GR HR HU IE IS IT LI LT LU LV MC MK MT NL NO PL PT RO RS SE SI SK SM TR

RAP3 Party data changed (applicant data changed or rights of an application transferred)

Owner name: ALTIUS INSTITUTE FOR BIOMEDICAL SCIENCES

DAV Request for validation of the european patent (deleted)
DAX Request for extension of the european patent (deleted)
A4 Supplementary search report drawn up and despatched

Effective date: 20230814

RIC1 Information provided on ipc code assigned before grant

Ipc: C12N 9/22 20060101ALI20230808BHEP

Ipc: C07K 14/705 20060101ALI20230808BHEP

Ipc: C07K 14/725 20060101ALI20230808BHEP

Ipc: C12N 15/85 20060101ALI20230808BHEP

Ipc: C12N 15/00 20060101ALI20230808BHEP

Ipc: C07K 19/00 20060101ALI20230808BHEP

Ipc: C07K 14/195 20060101ALI20230808BHEP

Ipc: A61K 38/00 20060101AFI20230808BHEP