CA3250345A1 - GJB2 REGULATORY ELEMENTS AND THEIR USES - Google Patents

GJB2 REGULATORY ELEMENTS AND THEIR USES

Info

Publication number
CA3250345A1
CA3250345A1 CA3250345A CA3250345A CA3250345A1 CA 3250345 A1 CA3250345 A1 CA 3250345A1 CA 3250345 A CA3250345 A CA 3250345A CA 3250345 A CA3250345 A CA 3250345A CA 3250345 A1 CA3250345 A1 CA 3250345A1
Authority
CA
Canada
Prior art keywords
gjb2
regulatory elements
regulatory
elements
gjb2 regulatory
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
CA3250345A
Other languages
English (en)
French (fr)
Inventor
Joseph Burns
Kathryn ELLIS
Tyler Gibson
Kevin Lebo
Gabriela PREGERNIG
Meghan Drummond Samuelson
Sarah CANCELARICH
Leah Sabin
Daniela DI BATTISTA MIANI
Original Assignee
Regeneron Pharmaceuticals Inc
Decibel Therapeutics 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 Regeneron Pharmaceuticals Inc, Decibel Therapeutics Inc filed Critical Regeneron Pharmaceuticals Inc
Publication of CA3250345A1 publication Critical patent/CA3250345A1/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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/1703—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • A61K38/1709—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0075—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the delivery route, e.g. oral, subcutaneous
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0046—Ear
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00—Drugs for disorders of the senses
    • A61P27/16—Otologicals
    • 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
    • C12N15/86—Viral vectors
    • 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
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Biotechnology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Molecular Biology (AREA)
  • Epidemiology (AREA)
  • Organic Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biochemistry (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Biophysics (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Microbiology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Plant Pathology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Immunology (AREA)
  • Marine Sciences & Fisheries (AREA)
  • Toxicology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Peptides Or Proteins (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
CA3250345A 2022-02-04 2023-02-03 GJB2 REGULATORY ELEMENTS AND THEIR USES Pending CA3250345A1 (en)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US202263306928P 2022-02-04 2022-02-04
US202263306941P 2022-02-04 2022-02-04
US63/306,928 2022-02-04
US63/306,941 2022-02-04
PCT/US2023/061953 WO2023150689A2 (en) 2022-02-04 2023-02-03 Gjb2 regulatory elements and uses thereof

Publications (1)

Publication Number Publication Date
CA3250345A1 true CA3250345A1 (en) 2023-08-10

Family

ID=87552983

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3250345A Pending CA3250345A1 (en) 2022-02-04 2023-02-03 GJB2 REGULATORY ELEMENTS AND THEIR USES

Country Status (11)

Country Link
US (1) US20250127928A1 (https=)
EP (1) EP4473005A4 (https=)
JP (1) JP2025506419A (https=)
KR (1) KR20240156606A (https=)
AU (1) AU2023214481A1 (https=)
CA (1) CA3250345A1 (https=)
CL (1) CL2024002306A1 (https=)
CO (1) CO2024012057A2 (https=)
IL (1) IL314705A (https=)
MX (1) MX2024009520A (https=)
WO (1) WO2023150689A2 (https=)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2026025095A2 (en) 2024-07-26 2026-01-29 Regeneron Pharmaceuticals, Inc. Otic drug delivery system and methods

Family Cites Families (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA3159549A1 (en) * 2019-10-30 2021-05-06 Decibel Therapeutics, Inc. Compositions and methods for treating sensorineural hearing loss using otoferlin dual vector systems
EP4164695A4 (en) * 2020-05-13 2024-06-05 Akouos, Inc. COMPOSITIONS AND METHODS FOR TREATING GJB2-ASSOCIATED HEARING LOSS

Also Published As

Publication number Publication date
CL2024002306A1 (es) 2024-12-27
JP2025506419A (ja) 2025-03-11
IL314705A (en) 2024-10-01
MX2024009520A (es) 2024-09-11
WO2023150689A2 (en) 2023-08-10
WO2023150689A8 (en) 2023-10-12
KR20240156606A (ko) 2024-10-30
CO2024012057A2 (es) 2024-09-30
EP4473005A4 (en) 2026-02-18
WO2023150689A3 (en) 2023-11-16
US20250127928A1 (en) 2025-04-24
AU2023214481A1 (en) 2024-08-08
EP4473005A2 (en) 2024-12-11

Similar Documents

Publication Publication Date Title
AU2022298654A1 (en) Degrader compounds and uses thereof
EP4162025A4 (en) RECOMBINANT MICROORGANISMS
CA3250345A1 (en) GJB2 REGULATORY ELEMENTS AND THEIR USES
EP4330403A4 (en) PLANT REGULATORY ELEMENTS AND THEIR USES
HK40120604A (en) Gjb2 regulatory elements and uses thereof
CA3263967A1 (en) SLC26A4 REGULATOR ELEMENTS AND THEIR USES
CA3266689A1 (en) PLANT REGULATORS AND THEIR USES
CA3267072A1 (en) REGULATORY ELEMENTS IN PLANTS AND THEIR USES
CA3261197A1 (en) PLANT REGULATORS AND THEIR USES
CA3292383A1 (en) Plant plasmodiophora-responsive promoter and use thereof
CA3296886A1 (en) Regulatory elements
HK40130563A (en) Slc26a4 regulatory elements and uses thereof
HK40128591A (zh) Slc26a4调控元件及其用途
CA3276902A1 (en) Substituted trianglamine and uses thereof
HK40114381A (en) Macromolecule-supported 8-sulfonyl-benzazepine compounds and their uses
HK40120400A (en) Bicyclic-heterocycle derivatives and related uses
CA3306175A1 (en) Cooker and temperer
EP4232587A4 (en) PLANT REGULATORY ELEMENTS AND USES THEREOF FOR AUTOEXCITATION
CA3301202A1 (en) Compounds and uses
HK40116989A (en) Oxoisoindolinyl-substituted tetrahydropyrimidinedione derivative and use thereof
EM150392450006S (en) Armchairs
EM150392450007S (de) Sessel
EM150302850003S (da) Lænestole
CA3289315A1 (en) Novel gspt1 degrader and use thereof
EM90786520001S (de) Sessel

Legal Events

Date Code Title Description
A00 Application filed

Free format text: ST27 STATUS EVENT CODE: A-0-1-A10-A00-A101 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: APPLICATION RECEIVED - PCT

Effective date: 20240730

P11 Amendment of application requested

Free format text: ST27 STATUS EVENT CODE: A-1-1-P10-P11-P101 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: AMENDMENT RECEIVED - VOLUNTARY AMENDMENT

Effective date: 20240730

A15 Pct application entered into the national or regional phase

Free format text: ST27 STATUS EVENT CODE: A-1-1-A10-A15-X000 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: NATIONAL ENTRY REQUIREMENTS DETERMINED COMPLIANT

Effective date: 20250204

P18 Priority claim added or amended

Free format text: ST27 STATUS EVENT CODE: A-1-1-P10-P18-P105 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: PRIORITY CLAIM REQUIREMENTS DETERMINED COMPLIANT

Effective date: 20250204

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: 20250205

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: 20250205

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: 20250205

W00 Other event occurred

Free format text: ST27 STATUS EVENT CODE: A-1-1-W10-W00-W100 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: LETTER SENT

Effective date: 20250217

W00 Other event occurred

Free format text: ST27 STATUS EVENT CODE: A-1-1-W10-W00-W111 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: CORRESPONDENT DETERMINED COMPLIANT

Effective date: 20250220

Free format text: ST27 STATUS EVENT CODE: A-1-1-W10-W00-W101 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: REQUEST TO REGISTER A DOCUMENT RECEIVED

Effective date: 20250220

W00 Other event occurred

Free format text: ST27 STATUS EVENT CODE: A-1-1-W10-W00-W111 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: CORRESPONDENT DETERMINED COMPLIANT

Effective date: 20250314

W00 Other event occurred

Free format text: ST27 STATUS EVENT CODE: A-1-1-W10-W00-W101 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: REQUEST TO REGISTER A DOCUMENT RECEIVED

Effective date: 20250327

Free format text: ST27 STATUS EVENT CODE: A-1-1-W10-W00-W111 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: CORRESPONDENT DETERMINED COMPLIANT

Effective date: 20250327

W00 Other event occurred

Free format text: ST27 STATUS EVENT CODE: A-1-1-W10-W00-W100 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: LETTER SENT

Effective date: 20251027

A00 Application filed

Free format text: ST27 STATUS EVENT CODE: A-1-1-A10-A00-A102 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: COMPLIANCE REQUIREMENTS DETERMINED MET

Effective date: 20251114

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: 20251114

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: 20260121

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: 20260122