WO2026073260A1 - Formulations de nanoparticules lipidiques de trems - Google Patents

Formulations de nanoparticules lipidiques de trems

Info

Publication number
WO2026073260A1
WO2026073260A1 PCT/US2025/048805 US2025048805W WO2026073260A1 WO 2026073260 A1 WO2026073260 A1 WO 2026073260A1 US 2025048805 W US2025048805 W US 2025048805W WO 2026073260 A1 WO2026073260 A1 WO 2026073260A1
Authority
WO
WIPO (PCT)
Prior art keywords
mol
lnp
trem
lipid
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
PCT/US2025/048805
Other languages
English (en)
Inventor
Paulo Jia Ching LIN
Ying K. Tam
Stephen William EICHHORN
William F. Kiesman
Caroline Kohrer
Harsh Vardhan CHAUHAN
Samantha Michele PREZIOSO
Isabelle Myriam SANSAL
Weidong George LAI
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.)
Acuitas Therapeutics Inc
Flagship Pioneering Innovations VI Inc
Original Assignee
Acuitas Therapeutics Inc
Flagship Pioneering Innovations VI 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 Acuitas Therapeutics Inc, Flagship Pioneering Innovations VI Inc filed Critical Acuitas Therapeutics Inc
Publication of WO2026073260A1 publication Critical patent/WO2026073260A1/fr
Pending legal-status Critical Current
Anticipated expiration legal-status Critical

Links

Classifications

    • 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
    • A61K9/48—Preparations in capsules, e.g. of gelatin, of chocolate
    • A61K9/50—Microcapsules having a gas, liquid or semi-solid filling; Solid microparticles or pellets surrounded by a distinct coating layer, e.g. coated microspheres, coated drug crystals
    • A61K9/51—Nanocapsules; Nanoparticles
    • A61K9/5107—Excipients; Inactive ingredients
    • A61K9/5123—Organic compounds, e.g. fats, sugars
    • 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
    • A61K48/0008—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • A61K48/0025—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid
    • A61K48/0041—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid the non-active part being polymeric
    • 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
    • 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
    • C12N15/88—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation using microencapsulation, e.g. using amphiphile liposome vesicle

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)
  • Biotechnology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Biomedical Technology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Molecular Biology (AREA)
  • Medicinal Chemistry (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Physics & Mathematics (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Organic Chemistry (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Nanotechnology (AREA)
  • Optics & Photonics (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)

Abstract

La divulgation concerne de manière générale des molécules effectrices à base d'ARNt (TREM) formulées dans des nanoparticules lipidiques présentant une modification non naturelle, ainsi que des compositions et des méthodes s'y rapportant.
PCT/US2025/048805 2024-09-30 2025-09-30 Formulations de nanoparticules lipidiques de trems Pending WO2026073260A1 (fr)

Applications Claiming Priority (20)

Application Number Priority Date Filing Date Title
US202463701485P 2024-09-30 2024-09-30
US202463701488P 2024-09-30 2024-09-30
US63/701,488 2024-09-30
US63/701,485 2024-09-30
US202463728031P 2024-12-04 2024-12-04
US202463728036P 2024-12-04 2024-12-04
US63/728,036 2024-12-04
US63/728,031 2024-12-04
US202563804391P 2025-05-12 2025-05-12
US202563804395P 2025-05-12 2025-05-12
US63/804,391 2025-05-12
US63/804,395 2025-05-12
US202563868160P 2025-08-21 2025-08-21
US202563868163P 2025-08-21 2025-08-21
US63/868,163 2025-08-21
US63/868,160 2025-08-21
US202563884859P 2025-09-19 2025-09-19
US202563884876P 2025-09-19 2025-09-19
US63/884,859 2025-09-19
US63/884,876 2025-09-19

Publications (1)

Publication Number Publication Date
WO2026073260A1 true WO2026073260A1 (fr) 2026-04-02

Family

ID=97522321

Family Applications (2)

Application Number Title Priority Date Filing Date
PCT/US2025/048805 Pending WO2026073260A1 (fr) 2024-09-30 2025-09-30 Formulations de nanoparticules lipidiques de trems
PCT/US2025/048807 Pending WO2026073261A1 (fr) 2024-09-30 2025-09-30 Formulations de nanoparticules lipidiques de trems

Family Applications After (1)

Application Number Title Priority Date Filing Date
PCT/US2025/048807 Pending WO2026073261A1 (fr) 2024-09-30 2025-09-30 Formulations de nanoparticules lipidiques de trems

Country Status (1)

Country Link
WO (2) WO2026073260A1 (fr)

Citations (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2015199952A1 (fr) 2014-06-25 2015-12-30 Acuitas Therapeutics Inc. Nouveaux lipides et formulations nanoparticulaires lipidiques pour l'administration d'acides nucléiques
WO2017075531A1 (fr) 2015-10-28 2017-05-04 Acuitas Therapeutics, Inc. Nouveaux lipides et nouvelles formulations de nanoparticules de lipides pour l'administration d'acides nucléiques
WO2018081480A1 (fr) * 2016-10-26 2018-05-03 Acuitas Therapeutics, Inc. Formulations de nanoparticules lipidiques
WO2021243290A1 (fr) * 2020-05-29 2021-12-02 Flagship Pioneering Innovations Vi, Llc Compositions de trem et procédés associés
WO2023064513A2 (fr) * 2021-10-13 2023-04-20 Flagship Pioneering Innovations Vi, Llc Compositions de trem et procédés d'utilisation
WO2023220083A1 (fr) * 2022-05-09 2023-11-16 Flagship Pioneering Innovations Vi, Llc Compositions de trem et procédés d'utilisation pour traiter des troubles prolifératifs
US20230372537A1 (en) * 2016-10-26 2023-11-23 Acuitas Therapeutics, Inc. Lipid nanoparticle formulations
EP4342880A1 (fr) * 2021-07-07 2024-03-27 Jenkem Technology Co., Ltd. (Tianjin) Composé lipidique ionisable pour l'administration d'acide nucléique et composition lnp de celui-ci
WO2024107702A1 (fr) * 2022-11-14 2024-05-23 Board Of Regents, The University Of Texas System Nanoparticules lipidiques en aérosol et leurs utilisations

Patent Citations (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2015199952A1 (fr) 2014-06-25 2015-12-30 Acuitas Therapeutics Inc. Nouveaux lipides et formulations nanoparticulaires lipidiques pour l'administration d'acides nucléiques
US9738593B2 (en) 2014-06-25 2017-08-22 Acuitas Therapeutics Inc. Lipids and lipid nanoparticle formulations for delivery of nucleic acids
WO2017075531A1 (fr) 2015-10-28 2017-05-04 Acuitas Therapeutics, Inc. Nouveaux lipides et nouvelles formulations de nanoparticules de lipides pour l'administration d'acides nucléiques
WO2018081480A1 (fr) * 2016-10-26 2018-05-03 Acuitas Therapeutics, Inc. Formulations de nanoparticules lipidiques
US20230372537A1 (en) * 2016-10-26 2023-11-23 Acuitas Therapeutics, Inc. Lipid nanoparticle formulations
WO2021243290A1 (fr) * 2020-05-29 2021-12-02 Flagship Pioneering Innovations Vi, Llc Compositions de trem et procédés associés
EP4342880A1 (fr) * 2021-07-07 2024-03-27 Jenkem Technology Co., Ltd. (Tianjin) Composé lipidique ionisable pour l'administration d'acide nucléique et composition lnp de celui-ci
WO2023064513A2 (fr) * 2021-10-13 2023-04-20 Flagship Pioneering Innovations Vi, Llc Compositions de trem et procédés d'utilisation
WO2023220083A1 (fr) * 2022-05-09 2023-11-16 Flagship Pioneering Innovations Vi, Llc Compositions de trem et procédés d'utilisation pour traiter des troubles prolifératifs
WO2024107702A1 (fr) * 2022-11-14 2024-05-23 Board Of Regents, The University Of Texas System Nanoparticules lipidiques en aérosol et leurs utilisations

Non-Patent Citations (4)

* Cited by examiner, † Cited by third party
Title
"Stedman's Medical Dictionary", 1990, WILLIAMS & WILKINS
BUCK ET AL., PLOS ONE, 2012
HARTSEL SA ET AL., OLIGONUCLEOTIDE SYNTHESIS, 2005, pages 033 - 050
LIN ET AL., NUCLEIC ACIDS RESEARCH, vol. 47, no. W1, 2 July 2019 (2019-07-02), pages 542 - 547

Also Published As

Publication number Publication date
WO2026073261A1 (fr) 2026-04-02

Similar Documents

Publication Publication Date Title
JP7453269B2 (ja) 核酸のデリバリーのための新規脂質および脂質ナノ粒子製剤
JP7596343B2 (ja) 治療剤の細胞内送達のための化合物及び組成物
JP7114465B2 (ja) 薬剤の細胞内送達のための化合物および組成物
JP6182457B2 (ja) カチオン性脂質を含有するドラックデリバリーシステムのための脂質ナノ粒子
JP6226476B2 (ja) カチオン性脂質
KR101773643B1 (ko) 양이온성 지질
TWI576113B (zh) 陽離子性脂質
EP3638678A1 (fr) Composés et compositions pour l'administration intracellulaire d'agents
WO2017010573A1 (fr) COMPOSITION MÉDICINALE À INTERFÉRENCE ARN POUR LE SILENÇAGE DE L'EXPRESSION DU GÈNE β2GPI
EP3153172A1 (fr) Composition pharmaceutique à base d'arni réprimant la transcription du gène ckap5
WO2024152512A1 (fr) Composé lipidique pour l'administration d'un agent thérapeutique, son procédé de préparation et son utilisation
WO2026073260A1 (fr) Formulations de nanoparticules lipidiques de trems
EP4581012A2 (fr) Nouveaux lipides ionisables et nanoparticules lipidiques, ainsi que procédés d'utilisation associés
JP2018065789A (ja) カチオン性脂質
TW201726614A (zh) 作為陽離子性脂質之化合物
RU2798342C2 (ru) Соединения и композиции для внутриклеточной доставки терапевтических агентов
WO2025102261A1 (fr) Composés lipidiques pour l'administration d'un agent thérapeutique, leur procédé de préparation et leur utilisation
HK40059901B (zh) 脂质化合物及包含其的脂质载体、核酸脂质纳米粒组合物和药物制剂
WO2025064475A2 (fr) Compositions lipidoïdes ionisables et leurs utilisations thérapeutiques
HK40064048A (en) Novel lipids and lipid nanoparticle formulations for delivery of nucleic acids
CN121698795A (zh) 脂质化合物和脂质纳米颗粒组合物
HK40082129B (zh) 纳米材料