EP4724461A1 - Particules de type viral modifiées (evlp) pour la transduction sélective de cellules cibles - Google Patents

Particules de type viral modifiées (evlp) pour la transduction sélective de cellules cibles

Info

Publication number
EP4724461A1
EP4724461A1 EP24737622.1A EP24737622A EP4724461A1 EP 4724461 A1 EP4724461 A1 EP 4724461A1 EP 24737622 A EP24737622 A EP 24737622A EP 4724461 A1 EP4724461 A1 EP 4724461A1
Authority
EP
European Patent Office
Prior art keywords
acid sequence
virus
particle
seq
amino acid
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
EP24737622.1A
Other languages
German (de)
English (en)
Inventor
David R. Liu
Jack QUEENAN
Nouraiz AHMED
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.)
Broad Institute Inc
Harvard University
Original Assignee
Broad Institute Inc
Harvard University
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 Broad Institute Inc, Harvard University filed Critical Broad Institute Inc
Publication of EP4724461A1 publication Critical patent/EP4724461A1/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
    • C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
    • 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
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00—Medicinal preparations containing antigens or antibodies
    • A61K2039/51—Medicinal preparations containing antigens or antibodies comprising whole cells, viruses or DNA/RNA
    • A61K2039/525—Virus
    • A61K2039/5258—Virus-like particles
    • 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
    • C07K2319/00—Fusion polypeptide
    • C07K2319/33—Fusion polypeptide fusions for targeting to specific cell types, e.g. tissue specific targeting, targeting of a bacterial subspecies
    • 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
    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/16011—Human Immunodeficiency Virus, HIV
    • C12N2740/16023—Virus like particles [VLP]
    • 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
    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/16011—Human Immunodeficiency Virus, HIV
    • C12N2740/16041—Use of virus, viral particle or viral elements as a vector
    • C12N2740/16043—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • 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
    • C12N2760/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssRNA viruses negative-sense
    • C12N2760/00011—Details
    • C12N2760/20011—Rhabdoviridae
    • C12N2760/20211—Vesiculovirus, e.g. vesicular stomatitis Indiana virus
    • C12N2760/20222—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes

Landscapes

  • Life Sciences & Earth Sciences (AREA)
  • Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Virology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Biophysics (AREA)
  • Biomedical Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Biochemistry (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Medicinal Chemistry (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Peptides Or Proteins (AREA)

Abstract

La présente divulgation concerne des compositions et des procédés pour la transduction sélective et l'édition génomique de cellules humaines (par exemple, des cellules souches et progénitrices hématopoïétiques, HSPC) à l'aide de particules de type viral modifiées (eVLP). Des aspects de la divulgation concernent des compositions eVLP comprenant des protéines de fusion comprenant une fraction de ciblage. Dans certains modes de réalisation, les protéines de fusion comprennent une cytokine conjuguée à une protéine transmembranaire et/ou à une glycoprotéine d'enveloppe. Dans d'autres modes de réalisation, les protéines de fusion comprennent un domaine de fraction de ciblage, un domaine de protéine tige, un domaine de glycoprotéine transmembranaire et/ou d'enveloppe. La divulgation concerne également des architectures d'eVLP ciblées comprenant divers domaines de ciblage, des domaines de tige, des domaines transmembranaires et des glycoprotéines d'enveloppe. D'autres aspects de la divulgation concernent des compositions eVLP comprenant des glycoprotéines d'enveloppe comprenant des sucres non naturels et des procédés de conjugaison desdites eVLP à diverses fractions de ciblage à l'aide d'une chimie click bio-orthogonale. La divulgation concerne également des polynucléotides, des vecteurs, des cellules et des kits utiles pour produire les articles, et la mise en œuvre des procédés divulgués.
EP24737622.1A 2023-06-07 2024-06-06 Particules de type viral modifiées (evlp) pour la transduction sélective de cellules cibles Pending EP4724461A1 (fr)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US202363506823P 2023-06-07 2023-06-07
PCT/US2024/032867 WO2024254346A1 (fr) 2023-06-07 2024-06-06 Particules de type viral modifiées (evlp) pour la transduction sélective de cellules cibles

Publications (1)

Publication Number Publication Date
EP4724461A1 true EP4724461A1 (fr) 2026-04-15

Family

ID=91758903

Family Applications (1)

Application Number Title Priority Date Filing Date
EP24737622.1A Pending EP4724461A1 (fr) 2023-06-07 2024-06-06 Particules de type viral modifiées (evlp) pour la transduction sélective de cellules cibles

Country Status (2)

Country Link
EP (1) EP4724461A1 (fr)
WO (1) WO2024254346A1 (fr)

Families Citing this family (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US9322037B2 (en) 2013-09-06 2016-04-26 President And Fellows Of Harvard College Cas9-FokI fusion proteins and uses thereof
EP3592853A1 (fr) 2017-03-09 2020-01-15 President and Fellows of Harvard College Suppression de la douleur par édition de gène
US12522807B2 (en) 2018-07-09 2026-01-13 The Broad Institute, Inc. RNA programmable epigenetic RNA modifiers and uses thereof
WO2020191233A1 (fr) 2019-03-19 2020-09-24 The Broad Institute, Inc. Procédés et compositions pour l'édition de séquences nucléotidiques
US12473543B2 (en) 2019-04-17 2025-11-18 The Broad Institute, Inc. Adenine base editors with reduced off-target effects

Family Cites Families (15)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP1235914A2 (fr) 1999-11-24 2002-09-04 Joseph Rosenecker Polypeptides comprenant des multimeres de signaux de localisation nucleaire ou de domaines de transduction de proteine et utilisations de ces derniers pour transferer des molecules dans des cellules
US20150165054A1 (en) 2013-12-12 2015-06-18 President And Fellows Of Harvard College Methods for correcting caspase-9 point mutations
US10077453B2 (en) 2014-07-30 2018-09-18 President And Fellows Of Harvard College CAS9 proteins including ligand-dependent inteins
RS61907B1 (sr) * 2015-04-06 2021-06-30 Subdomain Llc Polipeptidi koji sadrže de novo vezujući domen i njihova primena
IL310721B2 (en) 2015-10-23 2025-11-01 Harvard College Nucleobase editors and uses thereof
EP3235908A1 (fr) * 2016-04-21 2017-10-25 Ecole Normale Superieure De Lyon Procédés pour moduler de manière sélective l'activité des sous-types distinctes de cellules
CN110214183A (zh) 2016-08-03 2019-09-06 哈佛大学的校长及成员们 腺苷核碱基编辑器及其用途
KR20240116572A (ko) 2017-03-23 2024-07-29 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 핵산 프로그램가능한 dna 결합 단백질을 포함하는 핵염기 편집제
CN111801345A (zh) 2017-07-28 2020-10-20 哈佛大学的校长及成员们 使用噬菌体辅助连续进化(pace)的进化碱基编辑器的方法和组合物
WO2019084057A2 (fr) 2017-10-24 2019-05-02 Magenta Therapeutics, Inc. Compositions et procédés pour la déplétion des cellules cd117+
US12157760B2 (en) 2018-05-23 2024-12-03 The Broad Institute, Inc. Base editors and uses thereof
WO2020191233A1 (fr) 2019-03-19 2020-09-24 The Broad Institute, Inc. Procédés et compositions pour l'édition de séquences nucléotidiques
KR20230010231A (ko) * 2020-05-11 2023-01-18 아빈투스 바이오, 인코포레이티드 생체내 형질도입을 위한 벡터 및 방법
EP4182447A4 (fr) * 2020-07-14 2024-10-23 Ichilov Tech Ltd. Virus pseudotypés conçus pour exprimer un car dans des lymphocytes t
JP2024521811A (ja) * 2021-05-28 2024-06-04 サナ バイオテクノロジー，インコーポレイテッド 短縮型ヒヒ内在性レトロウイルス（ＢａＥＶ）エンベロープ糖タンパク質を含む脂質粒子、ならびに関連する方法及び使用

Also Published As

Publication number Publication date
WO2024254346A1 (fr) 2024-12-12
WO2024254346A8 (fr) 2025-10-02

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