JP2023536618A - Ｃｓｒｐ３（システイン及びグリシンリッチタンパク質３）遺伝子治療 - Google Patents

Ｃｓｒｐ３（システイン及びグリシンリッチタンパク質３）遺伝子治療 Download PDF

Info

Publication number
JP2023536618A
JP2023536618A JP2023507388A JP2023507388A JP2023536618A JP 2023536618 A JP2023536618 A JP 2023536618A JP 2023507388 A JP2023507388 A JP 2023507388A JP 2023507388 A JP2023507388 A JP 2023507388A JP 2023536618 A JP2023536618 A JP 2023536618A
Authority
JP
Japan
Prior art keywords
polynucleotide
vector
mlp
promoter
seq
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
JP2023507388A
Other languages
English (en)
Japanese (ja)
Other versions
JP2023536618A5 (enExample
Inventor
クリストファー ディーン ハーツォグ
チェスター ビッテンコート サクラメント
ラジ プラバカール
デイビッド リックス
Original Assignee
スペースクラフト セブン リミテッド ライアビリティ カンパニー
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 スペースクラフト セブン リミテッド ライアビリティ カンパニー filed Critical スペースクラフト セブン リミテッド ライアビリティ カンパニー
Publication of JP2023536618A publication Critical patent/JP2023536618A/ja
Publication of JP2023536618A5 publication Critical patent/JP2023536618A5/ja
Pending legal-status Critical Current

Links

Images

Classifications

    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A61P9/04—Inotropic agents, i.e. stimulants of cardiac contraction; Drugs for heart failure
    • 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
    • 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
    • C07K14/4705—Regulators; Modulating activity stimulating, promoting or activating activity
    • 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
    • C07K14/4716—Muscle proteins, e.g. myosin, actin
    • 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
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00—Genetically modified animals
    • A01K2217/07—Animals genetically altered by homologous recombination
    • A01K2217/072—Animals genetically altered by homologous recombination maintaining or altering function, i.e. knock in
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00—Genetically modified animals
    • A01K2217/07—Animals genetically altered by homologous recombination
    • A01K2217/075—Animals genetically altered by homologous recombination inducing loss of function, i.e. knock out
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2227/00—Animals characterised by species
    • A01K2227/10—Mammal
    • A01K2227/105—Murine
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2267/00—Animals characterised by purpose
    • A01K2267/03—Animal model, e.g. for test or diseases
    • A01K2267/0306—Animal model for genetic diseases
    • 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)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Zoology (AREA)
  • Molecular Biology (AREA)
  • Medicinal Chemistry (AREA)
  • Biotechnology (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Cardiology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Toxicology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Virology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Hospice & Palliative Care (AREA)
  • Epidemiology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Immunology (AREA)
JP2023507388A 2020-08-05 2021-08-03 Ｃｓｒｐ３（システイン及びグリシンリッチタンパク質３）遺伝子治療 Pending JP2023536618A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202063061727P 2020-08-05 2020-08-05
US63/061,727 2020-08-05
PCT/US2021/044412 WO2022031756A1 (en) 2020-08-05 2021-08-03 Csrp3 (cysteine and glycine rich protein 3) gene therapy

Publications (2)

Publication Number Publication Date
JP2023536618A true JP2023536618A (ja) 2023-08-28
JP2023536618A5 JP2023536618A5 (enExample) 2024-08-08

Family

ID=80118758

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2023507388A Pending JP2023536618A (ja) 2020-08-05 2021-08-03 Ｃｓｒｐ３（システイン及びグリシンリッチタンパク質３）遺伝子治療

Country Status (11)

Country Link
US (1) US20230257431A1 (enExample)
EP (1) EP4192962A4 (enExample)
JP (1) JP2023536618A (enExample)
KR (1) KR20230042468A (enExample)
CN (1) CN116234916A (enExample)
AU (1) AU2021321410A1 (enExample)
BR (1) BR112023001336A2 (enExample)
CA (1) CA3184983A1 (enExample)
IL (1) IL300187A (enExample)
MX (1) MX2023000994A (enExample)
WO (1) WO2022031756A1 (enExample)

Families Citing this family (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
HUE055862T2 (hu) 2016-04-20 2021-12-28 Centro De Investig Energeticas Készítmények és eljárások PKLR fokozott génexpressziójára
SG11202002263TA (en) 2017-10-16 2020-04-29 Centro De Investigaciones Energeticas Medioambientales Y Tecnologicas Lentiviral vectors for delivery of pklr to treat pyruvate kinase deficiency
WO2019200167A1 (en) 2018-04-11 2019-10-17 Rocket Pharmaceuticals, Ltd. Compositions and methods for stem cell transplant
MX2020011386A (es) 2018-04-27 2021-01-29 Spacecraft Seven Llc Terapia genica para la degeneracion del snc.
EP4103214B1 (en) 2020-02-13 2026-04-29 Tenaya Therapeutics, Inc. Gene therapy vectors for treating heart disease
KR20230043869A (ko) 2020-08-07 2023-03-31 스페이스크래프트 세븐, 엘엘씨 Aav 벡터를 사용한 플라코필린-2(pkp2) 유전자 요법
JP2025508743A (ja) * 2022-02-18 2025-04-10 ギンコ バイオワークス インコーポレイテッド 不整脈原性心筋症のための遺伝子治療
US20250205365A1 (en) * 2022-03-18 2025-06-26 University Of Florida Research Foundation, Incorporated Methods and compositions for treating rbm20 related cardiomyopathy with a viral vector

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2003523743A (ja) * 2000-02-03 2003-08-12 シエーリング アクチエンゲゼルシャフト Ｍｌｐ遺伝子、核酸、ポリペプチド、及びその使用
JP2016516423A (ja) * 2013-04-17 2016-06-09 ウニヴェルジテーツクリニクム ハンブルク−エッペンドルフＵｎｉｖｅｒｓｉｔａｅｔｓｋｌｉｎｉｋｕｍ Ｈａｍｂｕｒｇ−Ｅｐｐｅｎｄｏｒｆ 心筋症を処置するための遺伝子治療ベクター
JP2020513811A (ja) * 2017-03-17 2020-05-21 リサーチ インスティチュート アット ネイションワイド チルドレンズ ホスピタル 筋ジストロフィーを治療するための筋肉特異的マイクロジストロフィンのアデノ随伴ウイルスベクター送達

Family Cites Families (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP2176283B1 (en) 2007-07-14 2016-11-02 University of Iowa Research Foundation Methods and compositions for treating brain diseases
ES2739288T3 (es) 2013-09-13 2020-01-30 California Inst Of Techn Recuperación selectiva
LT3137497T (lt) 2014-05-02 2021-07-26 Genzyme Corporation Aav vektoriai, skirti tinklainės ir cns genų terapijai
KR102423442B1 (ko) 2015-12-11 2022-07-20 캘리포니아 인스티튜트 오브 테크놀로지 아데노-관련 바이러스를 지시하기 위한 타겟팅 펩타이드
RU2770922C2 (ru) * 2017-09-20 2022-04-25 4Д Молекьюлар Терапьютикс Инк. Капсиды вариантов аденоассоциированных вирусов и методы их применения
US20210363193A1 (en) * 2018-04-27 2021-11-25 Universität Heidelberg Modified aav capsid polypeptides for treatment of muscular diseases
CA3100280A1 (en) * 2018-06-08 2019-12-12 University Of Florida Research Foundation, Incorporated Aav cardiac gene therapy for cardiomyopathy
EP3947700A4 (en) * 2019-04-01 2023-01-04 Tenaya Therapeutics, Inc. Adeno-associated virus with engineered capsid

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2003523743A (ja) * 2000-02-03 2003-08-12 シエーリング アクチエンゲゼルシャフト Ｍｌｐ遺伝子、核酸、ポリペプチド、及びその使用
JP2016516423A (ja) * 2013-04-17 2016-06-09 ウニヴェルジテーツクリニクム ハンブルク−エッペンドルフＵｎｉｖｅｒｓｉｔａｅｔｓｋｌｉｎｉｋｕｍ Ｈａｍｂｕｒｇ−Ｅｐｐｅｎｄｏｒｆ 心筋症を処置するための遺伝子治療ベクター
JP2020513811A (ja) * 2017-03-17 2020-05-21 リサーチ インスティチュート アット ネイションワイド チルドレンズ ホスピタル 筋ジストロフィーを治療するための筋肉特異的マイクロジストロフィンのアデノ随伴ウイルスベクター送達

Non-Patent Citations (4)

* Cited by examiner, † Cited by third party
Title
BOATENG, S. Y. ET AL.: "Cardiac dysfunction and heart failure are associated with abnormalities in the subcellular distribut", ALP-HEART CIRC PHYSIOL, vol. 292, JPN6025027061, 2007, pages 259 - 269, ISSN: 0005831928 *
KUHN, C. ET AL.: "Cardiac remodeling is not modulated by overexpression of muscle LIM protein(MLP)", BASIC RES CARDIOL, vol. vol.107:262, JPN6025027060, 2012, pages 1 - 14, ISSN: 0005831927 *
LANGE, S. ET AL.: "MLP and CARP are linked to chronic PKCα signalling in dilated cardiomyopathy", NATURE COMMUNICATIONS, vol. vol.7:12120, JPN6025027062, 2016, pages 1 - 11, ISSN: 0005831929 *
MOLECULAR THERAPY, vol. 15, no. 2, JPN6026012783, 2007, pages 320 - 329, ISSN: 0005831930 *

Also Published As

Publication number Publication date
CA3184983A1 (en) 2022-02-10
EP4192962A1 (en) 2023-06-14
WO2022031756A1 (en) 2022-02-10
AU2021321410A1 (en) 2023-04-06
MX2023000994A (es) 2023-03-01
KR20230042468A (ko) 2023-03-28
CN116234916A (zh) 2023-06-06
EP4192962A4 (en) 2025-04-23
BR112023001336A2 (pt) 2023-02-14
US20230257431A1 (en) 2023-08-17
IL300187A (en) 2023-03-01

Similar Documents

Publication Publication Date Title
US11883506B2 (en) Plakophilin-2 (PKP2) gene therapy using AAV vector
US12257317B2 (en) Adeno-associated virus vector delivery of a fragment of micro-dystrophin to treat muscular dystrophy
CN110997923B (zh) 腺相关病毒载体递送肌肉特异性微肌营养不良蛋白以治疗肌营养不良症
TWI856016B (zh) 腺相關病毒載體遞送肌肉特異性微小肌縮蛋白以治療肌肉萎縮症
JP2023536618A (ja) Ｃｓｒｐ３（システイン及びグリシンリッチタンパク質３）遺伝子治療
JP2023536902A (ja) Ｇｌｕｔ１発現のためのアデノ随伴ウイルスベクターおよびその使用
JP2025512574A (ja) ＡＡＶベクターを使用するＢ細胞リンパ腫２関連Ａｎｔｈａｎｏｇｅｎｅ ３ （ＢＡＧ３）遺伝子治療
US20240335566A1 (en) Troponin c (tnnc1) gene therapy using aav vector
JP2024546103A (ja) Ａａｖベクターを用いたジャンクトフィリン－２（ｊｐｈ２）遺伝子治療
KR20240142487A (ko) Glut1 발현을 위한 아데노-관련 바이러스 벡터 및 이의 용도
KR20250133681A (ko) Aav 벡터를 이용한 플라코필린-2 (pkp2) 유전자 치료

Legal Events

Date Code Title Description
RD04 Notification of resignation of power of attorney

Free format text: JAPANESE INTERMEDIATE CODE: A7424

Effective date: 20230522

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20240227

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20240731

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20240731

A977 Report on retrieval

Free format text: JAPANESE INTERMEDIATE CODE: A971007

Effective date: 20250625

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20250707

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20251003

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20251204

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20260227

A02 Decision of refusal

Free format text: JAPANESE INTERMEDIATE CODE: A02

Effective date: 20260402