CA3238700A1 - New tale protein scaffolds with improved on-target/off-target activity ratios - Google Patents

New tale protein scaffolds with improved on-target/off-target activity ratios Download PDF

Info

Publication number
CA3238700A1
CA3238700A1 CA3238700A CA3238700A CA3238700A1 CA 3238700 A1 CA3238700 A1 CA 3238700A1 CA 3238700 A CA3238700 A CA 3238700A CA 3238700 A CA3238700 A CA 3238700A CA 3238700 A1 CA3238700 A1 CA 3238700A1
Authority
CA
Canada
Prior art keywords
tale
seq
sequence
nuclease
domain
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
CA3238700A
Other languages
English (en)
French (fr)
Inventor
Philippe Duchateau
Alexandre Juillerat
Alex BOYNE
Selena KAZANCIOGLU
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.)
Cellectis SA
Original Assignee
Cellectis SA
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 Cellectis SA filed Critical Cellectis SA
Publication of CA3238700A1 publication Critical patent/CA3238700A1/en
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
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • 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
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4702—Regulators; Modulating activity
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/195—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from bacteria
    • 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
    • C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
    • C12N15/102—Mutagenizing nucleic acids
    • 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
    • C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
    • C12N15/1034—Isolating an individual clone by screening libraries
    • 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
    • C12N15/90—Stable introduction of foreign DNA into chromosome
    • C12N15/902—Stable introduction of foreign DNA into chromosome using homologous recombination
    • C12N15/907—Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian cells
    • 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 [RNase]; Deoxyribonucleases [DNase]
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/09—Fusion polypeptide containing a localisation/targetting motif containing a nuclear localisation signal

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Molecular Biology (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Medicinal Chemistry (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Toxicology (AREA)
  • Cell Biology (AREA)
  • Mycology (AREA)
  • Peptides Or Proteins (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
CA3238700A 2021-11-23 2022-11-23 New tale protein scaffolds with improved on-target/off-target activity ratios Pending CA3238700A1 (en)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US202163282453P 2021-11-23 2021-11-23
US63/282,453 2021-11-23
DKPA202270104 2022-03-15
DKPA202270104 2022-03-15
PCT/EP2022/082950 WO2023094435A1 (en) 2021-11-23 2022-11-23 New tale protein scaffolds with improved on-target/off-target activity ratios

Publications (1)

Publication Number Publication Date
CA3238700A1 true CA3238700A1 (en) 2022-11-23

Family

ID=84487474

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3238700A Pending CA3238700A1 (en) 2021-11-23 2022-11-23 New tale protein scaffolds with improved on-target/off-target activity ratios

Country Status (9)

Country Link
US (1) US20250002945A1 (enExample)
EP (1) EP4437091A1 (enExample)
JP (1) JP2024540639A (enExample)
KR (1) KR20240110844A (enExample)
AU (1) AU2022395500A1 (enExample)
CA (1) CA3238700A1 (enExample)
IL (1) IL312721A (enExample)
MX (1) MX2024006051A (enExample)
WO (1) WO2023094435A1 (enExample)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP4583890A1 (en) * 2022-09-09 2025-07-16 Iovance Biotherapeutics, Inc. Processes for generating til products using pd-1/tigit talen double knockdown
WO2025181336A1 (en) 2024-03-01 2025-09-04 Cellectis Sa Compositions and methods for hbb-editing in hspc
KR20250148471A (ko) * 2024-04-02 2025-10-14 재단법인 아산사회복지재단 핵산 결합 단백질 및 SsdA를 포함하는 융합단백질 및 이의 용도
WO2026046724A1 (en) 2024-08-30 2026-03-05 Cellectis Sa Tale protein scaffolds involving fusions of monopartite and bipartite nls

Family Cites Families (13)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4880635B1 (en) 1984-08-08 1996-07-02 Liposome Company Dehydrated liposomes
US4683195A (en) 1986-01-30 1987-07-28 Cetus Corporation Process for amplifying, detecting, and/or-cloning nucleic acid sequences
US4921757A (en) 1985-04-26 1990-05-01 Massachusetts Institute Of Technology System for delayed and pulsed release of biologically active substances
US4920016A (en) 1986-12-24 1990-04-24 Linear Technology, Inc. Liposomes with enhanced circulation time
JPH0825869B2 (ja) 1987-02-09 1996-03-13 株式会社ビタミン研究所 抗腫瘍剤包埋リポソ−ム製剤
US4911928A (en) 1987-03-13 1990-03-27 Micro-Pak, Inc. Paucilamellar lipid vesicles
US4917951A (en) 1987-07-28 1990-04-17 Micro-Pak, Inc. Lipid vesicles formed of surfactants and steroids
PT2816112T (pt) 2009-12-10 2018-11-20 Univ Iowa State Res Found Inc Modificação do adn modificada pelo efector tal
US20140115726A1 (en) 2011-04-05 2014-04-24 Cellectis New tale-protein scaffolds and uses thereof
EP2737066B1 (en) 2011-07-29 2017-11-08 Cellectis High throughput method for assembly and cloning polynucleotides comprising highly similar polynucleotidic modules
SG10201806573TA (en) 2012-05-25 2018-09-27 Cellectis Methods for engineering allogeneic and immunosuppressive resistant t cell for immunotherapy
US10378007B2 (en) * 2012-09-03 2019-08-13 Cellectis Methods for modulating TAL specificity
CA2913872C (en) 2013-05-31 2022-01-18 Cellectis A laglidadg homing endonuclease cleaving the t-cell receptor alpha gene and uses thereof

Also Published As

Publication number Publication date
EP4437091A1 (en) 2024-10-02
WO2023094435A1 (en) 2023-06-01
MX2024006051A (es) 2024-06-26
IL312721A (en) 2024-07-01
US20250002945A1 (en) 2025-01-02
AU2022395500A1 (en) 2024-05-23
KR20240110844A (ko) 2024-07-16
JP2024540639A (ja) 2024-10-31

Similar Documents

Publication Publication Date Title
US20250002945A1 (en) New tale protein scaffolds with improved on-target/off-target activity ratios
US12378546B2 (en) Coupling endonucleases with end-processing enzymes drives high efficiency gene disruption
US11834686B2 (en) Engineered target specific base editors
EP3004149B1 (en) A laglidadg homing endonuclease cleaving the c-c chemokine receptor type-5 (ccr5) gene and uses thereof
US20130045539A1 (en) Meganuclease recombination system
AU2014273091A1 (en) A LAGLIDADG homing endonuclease cleaving the T cell receptor alpha gene and uses thereof
EP2828384A1 (en) Method to overcome dna chemical modifications sensitivity of engineered tale dna binding domains
JP2026510339A (ja) Pcsk9発現のエピジェネティック調節のための組成物及び方法
WO2026046724A1 (en) Tale protein scaffolds involving fusions of monopartite and bipartite nls
CN118382695A (zh) 具有改进的靶上/靶外活性比的新的tale蛋白支架
US20160138047A1 (en) Improved polynucleotide sequences encoding tale repeats
Sato Engineered Recombinases: Tools for Therapeutic Human Genome Editing
HK1223373B (en) A laglidadg homing endonuclease cleaving the c-c chemokine receptor type-5 (ccr5) gene and uses thereof

Legal Events

Date Code Title Description
MFA Maintenance fee for application paid

Free format text: FEE DESCRIPTION TEXT: MF (APPLICATION, 2ND ANNIV.) - STANDARD

Year of fee payment: 2

U00 Fee paid

Free format text: ST27 STATUS EVENT CODE: A-1-1-U10-U00-U101 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE REQUEST RECEIVED

Effective date: 20241024

U11 Full renewal or maintenance fee paid

Free format text: ST27 STATUS EVENT CODE: A-1-1-U10-U11-U102 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE FEE PAYMENT DETERMINED COMPLIANT

Effective date: 20241024

Free format text: ST27 STATUS EVENT CODE: A-1-1-U10-U11-U102 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE FEE PAYMENT PAID IN FULL

Effective date: 20241024

MFA Maintenance fee for application paid

Free format text: FEE DESCRIPTION TEXT: MF (APPLICATION, 3RD ANNIV.) - STANDARD

Year of fee payment: 3

U00 Fee paid

Free format text: ST27 STATUS EVENT CODE: A-1-1-U10-U00-U101 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE REQUEST RECEIVED

Effective date: 20251027

U11 Full renewal or maintenance fee paid

Free format text: ST27 STATUS EVENT CODE: A-1-1-U10-U11-U102 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE FEE PAYMENT PAID IN FULL

Effective date: 20251027

P13 Application amended

Free format text: ST27 STATUS EVENT CODE: A-1-1-P10-P13-P146 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: BSL VERIFIED - NO DEFECTS

Effective date: 20251124