[go: up one dir, main page]

MX2016005236A - Administracion de acido ribonucleico mensajero al sistema nervioso central y sus usos. - Google Patents

Administracion de acido ribonucleico mensajero al sistema nervioso central y sus usos.

Info

Publication number
MX2016005236A
MX2016005236A MX2016005236A MX2016005236A MX2016005236A MX 2016005236 A MX2016005236 A MX 2016005236A MX 2016005236 A MX2016005236 A MX 2016005236A MX 2016005236 A MX2016005236 A MX 2016005236A MX 2016005236 A MX2016005236 A MX 2016005236A
Authority
MX
Mexico
Prior art keywords
mrna
present
delivery
compositions
cns
Prior art date
Application number
MX2016005236A
Other languages
English (en)
Inventor
Michael Heartlein
Frank Derosa
Karve Shrirang
Original Assignee
Shire Human Genetic Therapies
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 Shire Human Genetic Therapies filed Critical Shire Human Genetic Therapies
Publication of MX2016005236A publication Critical patent/MX2016005236A/es

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
    • A61K9/10—Dispersions; Emulsions
    • A61K9/127—Synthetic bilayered vehicles, e.g. liposomes or liposomes with cholesterol as the only non-phosphatidyl surfactant
    • A61K9/1271—Non-conventional liposomes, e.g. PEGylated liposomes or liposomes coated or grafted with polymers
    • A61K9/1272—Non-conventional liposomes, e.g. PEGylated liposomes or liposomes coated or grafted with polymers comprising non-phosphatidyl surfactants as bilayer-forming substances, e.g. cationic lipids or non-phosphatidyl liposomes coated or grafted with polymers
    • 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
    • A61K48/0008—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
    • A61K48/0033—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 non-polymeric
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • 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
    • 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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • C07H21/02—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with ribosyl as saccharide radical

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Organic Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • Epidemiology (AREA)
  • Molecular Biology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Neurology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • Neurosurgery (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Immunology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Marine Sciences & Fisheries (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Dispersion Chemistry (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Psychiatry (AREA)
  • Hospice & Palliative Care (AREA)
  • Medicinal Preparation (AREA)

Abstract

La presente invención proporciona, entre otras cosas, métodos y composiciones para la administración eficaz de ARN mensajero (ARNm) al sistema nervioso central (SNC); en particular, la presente invención proporciona métodos y composiciones para administrarle por vía intratecal a un sujeto que necesita la administración de una composición que comprende un ARNm codificante de una proteína encapsulado dentro de un liposoma de modo que la administración de la composición produzca la administración intracelular de ARNm a las neuronas en el cerebro y/o la médula espinal; la presente invención es particularmente útil para el tratamiento de enfermedades, trastornos y afecciones del SNC, como la atrofia muscular espinal.
MX2016005236A 2013-10-22 2014-10-22 Administracion de acido ribonucleico mensajero al sistema nervioso central y sus usos. MX2016005236A (es)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201361894246P 2013-10-22 2013-10-22
US201462020161P 2014-07-02 2014-07-02
PCT/US2014/061786 WO2015061461A1 (en) 2013-10-22 2014-10-22 Cns delivery of mrna and uses thereof

Publications (1)

Publication Number Publication Date
MX2016005236A true MX2016005236A (es) 2016-08-12

Family

ID=51844900

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2016005236A MX2016005236A (es) 2013-10-22 2014-10-22 Administracion de acido ribonucleico mensajero al sistema nervioso central y sus usos.

Country Status (10)

Country Link
US (4) US20150110857A1 (es)
EP (2) EP4036241A1 (es)
JP (6) JP6893785B2 (es)
CN (1) CN105658800A (es)
AU (3) AU2014340149B2 (es)
BR (1) BR112016008832A2 (es)
CA (1) CA2928040A1 (es)
EA (1) EA201690588A1 (es)
MX (1) MX2016005236A (es)
WO (1) WO2015061461A1 (es)

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US12458604B2 (en) 2020-10-14 2025-11-04 The Trustees Of The University Of Pennsylvania Methods of lipid nanoparticle manufacture and compositions derived therefrom

Families Citing this family (66)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
MX353900B (es) 2008-11-07 2018-02-01 Massachusetts Inst Technology Lipidoides de aminoalcohol y usos de los mismos.
ES2666559T3 (es) 2009-12-01 2018-05-07 Translate Bio, Inc. Entrega del mrna para la aumentación de proteínas y enzimas en enfermedades genéticas humanas
DK2691443T3 (da) 2011-03-28 2021-05-03 Massachusetts Inst Technology Konjugerede lipomerer og anvendelser af disse
ES2663360T3 (es) 2011-06-08 2018-04-12 Translate Bio, Inc. Lípidos escindibles
BR112013031553A2 (pt) 2011-06-08 2020-11-10 Shire Human Genetic Therapies, Inc. composições, mrna que codifica para uma hgla e seu uso, uso de pelo menos uma molécula de mrna e um veículo de transferência e uso de um mrna que codifica para proteína exógena
NZ747501A (en) 2011-10-27 2020-05-29 Massachusetts Inst Technology Amino acid derivatives functionalized on the n-terminal capable of forming drug encapsulating microspheres
EP2859102A4 (en) 2012-06-08 2016-05-11 Shire Human Genetic Therapies NUCLEASE RESISTANT POLYNUCLEOTIDES AND USES THEREOF
WO2014153052A2 (en) 2013-03-14 2014-09-25 Shire Human Genetic Therapies, Inc. Cftr mrna compositions and related methods and uses
EP4446413A3 (en) 2013-03-14 2024-12-18 Translate Bio, Inc. Methods for purification of messenger rna
HUE055044T2 (hu) 2013-03-14 2021-10-28 Translate Bio Inc MRNS-kódolt ellenanyagok bejuttatására szolgáló eljárások és készítmények
ES2707966T3 (es) 2013-10-22 2019-04-08 Translate Bio Inc Terapia de ARNm para la deficiencia en síntesis de argininosuccinato
CN105658242A (zh) 2013-10-22 2016-06-08 夏尔人类遗传性治疗公司 用于苯丙酮尿症的mrna疗法
CN106164248B (zh) 2014-04-25 2019-10-15 川斯勒佰尔公司 信使rna的纯化方法
JP6384863B2 (ja) * 2014-10-24 2018-09-05 国立大学法人北海道大学 脂質膜構造体
HRP20230494T1 (hr) * 2015-06-19 2023-08-04 Massachusetts Institute Of Technology Alkenil supstituirani 2,5-piperazindioni i njihova upotreba u pripravcima za isporuku sredstva subjektu ili stanici
KR20180086266A (ko) * 2015-12-14 2018-07-30 더 트러스티스 오브 더 유니버시티 오브 펜실바니아 척수성 근위축증의 치료에 유용한 아데노-관련 바이러스 벡터
US10835583B2 (en) * 2016-06-13 2020-11-17 Translate Bio, Inc. Messenger RNA therapy for the treatment of ornithine transcarbamylase deficiency
AU2017357758B2 (en) * 2016-11-10 2023-11-16 Translate Bio, Inc. Improved process of preparing mRNA-loaded lipid nanoparticles
CN110225980B (zh) 2016-11-21 2023-01-06 纳米线科技公司 化学组合物及其使用方法
CN115845040A (zh) 2017-01-27 2023-03-28 卫理公会医院 用于免疫疗法的核/壳结构平台
MX2019010155A (es) 2017-02-27 2020-12-10 Translate Bio Inc Arnm de cftr optimizado por codón novedoso.
EP3971291A1 (en) 2017-02-27 2022-03-23 Translate Bio, Inc. Methods for purification of messenger rna
HUE059594T2 (hu) 2017-02-27 2022-11-28 Translate Bio Inc Hírvivõ RNS tisztítására szolgáló eljárások
JOP20190200A1 (ar) * 2017-02-28 2019-08-27 Univ Pennsylvania تركيبات نافعة في معالجة ضمور العضل النخاعي
WO2018213476A1 (en) 2017-05-16 2018-11-22 Translate Bio, Inc. Treatment of cystic fibrosis by delivery of codon-optimized mrna encoding cftr
MA49421A (fr) 2017-06-15 2020-04-22 Modernatx Inc Formulations d'arn
EP3641834B1 (en) * 2017-06-19 2023-10-04 Translate Bio, Inc. Messenger rna therapy for the treatment of friedreich's ataxia
BR102017016440A2 (pt) * 2017-07-31 2019-03-19 Universidade Federal Do Rio Grande Do Sul Composição para terapia gênica do sistema nervoso central, processo de obtenção e uso da mesma
WO2019046809A1 (en) 2017-08-31 2019-03-07 Modernatx, Inc. METHODS OF MANUFACTURING LIPID NANOPARTICLES
EP3775202A4 (en) * 2018-03-27 2022-04-06 Factor Bioscience Inc. NUCLEIC ACID BASED THERAPEUTICS
EP3794146B1 (en) 2018-05-14 2025-12-10 Bruker Spatial Biology, Inc. Method for identifying a predetermined nucleotide sequence
AU2019271132B2 (en) * 2018-05-15 2026-02-05 Translate Bio, Inc. Subcutaneous delivery of messenger RNA
US20200016274A1 (en) * 2018-05-30 2020-01-16 Translate Bio, Inc. Messenger rna vaccines and uses thereof
WO2020041500A1 (en) * 2018-08-21 2020-02-27 Georgia State University Research Foundation, Inc. Treatment of flavivirus infections in humans using mus musculus resistant 2'-5' oligoadenylate synthetase 1b
KR20210060480A (ko) 2018-08-24 2021-05-26 트랜슬레이트 바이오 인코포레이티드 전령 rna의 정제 방법
KR20210091120A (ko) 2018-08-29 2021-07-21 트랜슬레이트 바이오 인코포레이티드 Mrna-로딩된 지질 나노입자를 제조하는 개선된 공정
CA3113651A1 (en) 2018-09-20 2020-03-26 Modernatx, Inc. Preparation of lipid nanoparticles and methods of administration thereof
KR20210093232A (ko) * 2018-10-09 2021-07-27 더 유니버시티 오브 브리티시 콜롬비아 유기용매와 세제가 없는 형질감염 적격 소포를 포함하는 조성물과 시스템 및 관련 방법
WO2020097509A1 (en) 2018-11-08 2020-05-14 Translate Bio, Inc. Methods and compositions for messenger rna purification
CA3120647A1 (en) 2018-11-21 2020-05-28 Translate Bio, Inc. Treatment of cystic fibrosis by delivery of nebulized mrna encoding cftr
CA3116630A1 (en) * 2018-11-30 2020-06-04 Novartis Ag Aav viral vectors and uses thereof
EP3911302B1 (en) 2019-01-17 2025-09-10 Georgia Tech Research Corporation Drug delivery systems containing oxidized cholesterols
CA3165388A1 (en) 2019-12-20 2021-06-24 Translate Bio, Inc. Improved process of preparing mrna-loaded lipid nanoparticles
BR112022013837A2 (pt) * 2020-01-21 2022-09-13 Etherna Immunotherapies Nv Nanopartículas lipídicas
CA3173035A1 (en) 2020-02-25 2021-09-02 Translate Bio, Inc. Improved processes of preparing mrna-loaded lipid nanoparticles
US20240342269A1 (en) * 2020-05-07 2024-10-17 Translate Bio, Inc. Optimized nucleotide sequences encoding sars-cov-2 antigens
RU2742837C1 (ru) * 2020-06-02 2021-02-11 Общество С Ограниченной Ответственностью "Анабион" Кодон-оптимизированная нуклеиновая кислота, которая кодирует белок SMN1, и ее применение
CA3186776A1 (en) * 2020-06-11 2021-12-16 Etherna Immunotherapies Nv Lipid nanoparticles
WO2022099003A1 (en) 2020-11-06 2022-05-12 Sanofi Lipid nanoparticles for delivering mrna vaccines
WO2022169329A1 (ko) * 2021-02-08 2022-08-11 (주)아이엠지티 초음파 감응성 리포좀을 유효성분으로 포함하는 혈액-뇌 장벽 투과용 조성물
US20240374754A1 (en) * 2021-06-01 2024-11-14 The University Of British Columbia Mrna delivery using lipid nanoparticles
CA3230031A1 (en) 2021-09-03 2023-03-09 Patrick Baumhof Novel lipid nanoparticles for delivery of nucleic acids
US20240316216A1 (en) * 2021-10-06 2024-09-26 Massachusetts Institute Of Technology Lipid nanoparticles for drug delivery to microglia in the brain
US20250027108A1 (en) 2021-10-29 2025-01-23 CureVac SE Improved circular rna for expressing therapeutic proteins
CN114306369B (zh) * 2021-12-23 2023-12-26 北京悦康科创医药科技股份有限公司 一种硫代寡核苷酸注射液及其制备方法
EP4469091A1 (en) 2022-01-28 2024-12-04 CureVac SE Nucleic acid encoded transcription factor inhibitors
CN119300865A (zh) * 2022-05-04 2025-01-10 卡耐基梅隆大学 用于中枢神经系统递送的脂质纳米颗粒制剂
JP2025517508A (ja) 2022-05-25 2025-06-05 キュアバック エスイー 核酸ベースのワクチン
DE202023106198U1 (de) 2022-10-28 2024-03-21 CureVac SE Impfstoff auf Nukleinsäurebasis
JP2025540306A (ja) * 2022-12-07 2025-12-11 ザ・ユニバーシティ・オブ・ブリティッシュ・コロンビア 中枢神経系へのペプチドまたはタンパク質の供給のための組成物および方法
CN118105357B (zh) * 2023-01-27 2025-04-01 北京剂泰医药科技有限公司 一种鞘内递送制剂及其用途
WO2024156083A1 (en) * 2023-01-27 2024-08-02 Beijing Jitai Pharmaceutical Technology Co., Ltd. Ai-assisted design of lnps for effective cns delivery of rna
DE112024001143T5 (de) 2023-03-08 2025-12-18 CureVac SE Neue lipid-nanopartikel-formeln für die abgabe von nukleinsäuren
WO2024230934A1 (en) 2023-05-11 2024-11-14 CureVac SE Therapeutic nucleic acid for the treatment of ophthalmic diseases
WO2024235159A1 (zh) * 2023-05-12 2024-11-21 上海金珂博生物技术有限公司 一种携带smn基因表达框的病毒载体及其用途
WO2025143118A1 (ja) * 2023-12-27 2025-07-03 アステラス製薬株式会社 脊髄損傷の治療用医薬組成物及び脊髄損傷の非ヒト霊長類動物モデルの作製方法

Family Cites Families (123)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4500707A (en) 1980-02-29 1985-02-19 University Patents, Inc. Nucleosides useful in the preparation of polynucleotides
US5132418A (en) 1980-02-29 1992-07-21 University Patents, Inc. Process for preparing polynucleotides
US4458066A (en) 1980-02-29 1984-07-03 University Patents, Inc. Process for preparing polynucleotides
US4415732A (en) 1981-03-27 1983-11-15 University Patents, Inc. Phosphoramidite compounds and processes
US4668777A (en) 1981-03-27 1987-05-26 University Patents, Inc. Phosphoramidite nucleoside compounds
US4973679A (en) 1981-03-27 1990-11-27 University Patents, Inc. Process for oligonucleo tide synthesis using phosphormidite intermediates
US4401796A (en) 1981-04-30 1983-08-30 City Of Hope Research Institute Solid-phase synthesis of polynucleotides
US4373071A (en) 1981-04-30 1983-02-08 City Of Hope Research Institute Solid-phase synthesis of polynucleotides
US4897355A (en) 1985-01-07 1990-01-30 Syntex (U.S.A.) Inc. N[ω,(ω-1)-dialkyloxy]- and N-[ω,(ω-1)-dialkenyloxy]-alk-1-yl-N,N,N-tetrasubstituted ammonium lipids and uses therefor
US4737323A (en) 1986-02-13 1988-04-12 Liposome Technology, Inc. Liposome extrusion method
US5153319A (en) 1986-03-31 1992-10-06 University Patents, Inc. Process for preparing polynucleotides
US5262530A (en) 1988-12-21 1993-11-16 Applied Biosystems, Inc. Automated system for polynucleotide synthesis and purification
US5047524A (en) 1988-12-21 1991-09-10 Applied Biosystems, Inc. Automated system for polynucleotide synthesis and purification
US6214804B1 (en) 1989-03-21 2001-04-10 Vical Incorporated Induction of a protective immune response in a mammal by injecting a DNA sequence
FR2645866B1 (fr) 1989-04-17 1991-07-05 Centre Nat Rech Scient Nouvelles lipopolyamines, leur preparation et leur emploi
US5334761A (en) 1992-08-28 1994-08-02 Life Technologies, Inc. Cationic lipids
US5820873A (en) * 1994-09-30 1998-10-13 The University Of British Columbia Polyethylene glycol modified ceramide lipids and liposome uses thereof
EP0708178A1 (en) * 1994-10-19 1996-04-24 Institut National De La Sante Et De La Recherche Medicale (Inserm) Survival motor neuron (SMN) gene: a gene for spinal muscular atrophy
US5965434A (en) 1994-12-29 1999-10-12 Wolff; Jon A. Amphipathic PH sensitive compounds and delivery systems for delivering biologically active compounds
US5700642A (en) 1995-05-22 1997-12-23 Sri International Oligonucleotide sizing using immobilized cleavable primers
JP4335310B2 (ja) 1995-06-07 2009-09-30 ザ ユニバーシティ オブ ブリティッシュ コロンビア 疎水性脂質−核酸複合中間体を通して調製される脂質−核酸粒子、及び遺伝子移送のための使用
US7422902B1 (en) 1995-06-07 2008-09-09 The University Of British Columbia Lipid-nucleic acid particles prepared via a hydrophobic lipid-nucleic acid complex intermediate and use for gene transfer
US5981501A (en) 1995-06-07 1999-11-09 Inex Pharmaceuticals Corp. Methods for encapsulating plasmids in lipid bilayers
US5705385A (en) 1995-06-07 1998-01-06 Inex Pharmaceuticals Corporation Lipid-nucleic acid particles prepared via a hydrophobic lipid-nucleic acid complex intermediate and use for gene transfer
US5744335A (en) 1995-09-19 1998-04-28 Mirus Corporation Process of transfecting a cell with a polynucleotide mixed with an amphipathic compound and a DNA-binding protein
EP2338478B1 (en) 2002-06-28 2014-07-23 Protiva Biotherapeutics Inc. Method for producing liposomes
JP4709545B2 (ja) 2002-07-26 2011-06-22 ノバルティス バクシンズ アンド ダイアグノスティックス，インコーポレーテッド 修飾された低分子干渉ｒｎａ分子および使用方法
US7994149B2 (en) 2003-02-03 2011-08-09 Medtronic, Inc. Method for treatment of Huntington's disease through intracranial delivery of sirna
US7803397B2 (en) 2003-09-15 2010-09-28 Protiva Biotherapeutics, Inc. Polyethyleneglycol-modified lipid compounds and uses thereof
US7928217B2 (en) 2004-05-27 2011-04-19 Alnylam Pharmaceuticals, Inc. Nuclease resistant double-stranded ribonucleic acid
WO2005120152A2 (en) 2004-06-07 2005-12-22 Protiva Biotherapeutics, Inc. Cationic lipids and methods of use
EP1766035B1 (en) 2004-06-07 2011-12-07 Protiva Biotherapeutics Inc. Lipid encapsulated interfering rna
CN101346393B (zh) 2005-11-02 2015-07-22 普洛体维生物治疗公司 修饰的siRNA分子及其应用
WO2007070705A2 (en) * 2005-12-15 2007-06-21 The Trustees Of The University Of Pennsylvania Cationic lipid-mediated vectors
EP2049665A2 (en) 2006-07-28 2009-04-22 Applera Corporation Dinucleotide mrna cap analogs
WO2009058911A2 (en) 2007-10-31 2009-05-07 Applied Biosystems Inc. Preparation and isolation of 5' capped mrna
JP5635412B2 (ja) 2007-12-04 2014-12-03 アルニラム ファーマスーティカルズ インコーポレイテッドＡｌｎｙｌａｍ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ， Ｉｎｃ． 標的化脂質
EP2238251B1 (en) 2007-12-27 2015-02-11 Protiva Biotherapeutics Inc. Silencing of polo-like kinase expression using interfering rna
CA2711236A1 (en) 2008-01-02 2009-07-16 Alnylam Pharmaceuticals, Inc. Screening method for selected amino lipid-containing compositions
EP2274425A2 (en) 2008-04-11 2011-01-19 Alnylam Pharmaceuticals Inc. Site-specific delivery of nucleic acids by combining targeting ligands with endosomolytic components
AU2009238175C1 (en) 2008-04-15 2023-11-30 Arbutus Biopharma Corporation Novel lipid formulations for nucleic acid delivery
EP2276727A1 (en) 2008-05-19 2011-01-26 The University of North Carolina At Chapel Hill Methods and compositions comprising novel cationic lipids
EP2350043B9 (en) 2008-10-09 2014-08-20 TEKMIRA Pharmaceuticals Corporation Improved amino lipids and methods for the delivery of nucleic acids
MX353900B (es) 2008-11-07 2018-02-01 Massachusetts Inst Technology Lipidoides de aminoalcohol y usos de los mismos.
CN111909020A (zh) 2008-11-10 2020-11-10 阿布特斯生物制药公司 用于递送治疗剂的脂质和组合物
CA2750561C (en) 2009-01-26 2017-10-10 Protiva Biotherapeutics, Inc. Compositions and methods for silencing apolipoprotein c-iii expression
KR20240015723A (ko) 2009-05-05 2024-02-05 알닐람 파마슈티칼스 인코포레이티드 지질 조성물
CA2764609C (en) 2009-06-10 2018-10-02 Alnylam Pharmaceuticals, Inc. Improved cationic lipid of formula i
WO2010147992A1 (en) 2009-06-15 2010-12-23 Alnylam Pharmaceuticals, Inc. Methods for increasing efficacy of lipid formulated sirna
US9018187B2 (en) 2009-07-01 2015-04-28 Protiva Biotherapeutics, Inc. Cationic lipids and methods for the delivery of therapeutic agents
EP2449106B1 (en) 2009-07-01 2015-04-08 Protiva Biotherapeutics Inc. Compositions and methods for silencing apolipoprotein b
WO2011000106A1 (en) 2009-07-01 2011-01-06 Protiva Biotherapeutics, Inc. Improved cationic lipids and methods for the delivery of therapeutic agents
US8716464B2 (en) 2009-07-20 2014-05-06 Thomas W. Geisbert Compositions and methods for silencing Ebola virus gene expression
ES2666559T3 (es) 2009-12-01 2018-05-07 Translate Bio, Inc. Entrega del mrna para la aumentación de proteínas y enzimas en enfermedades genéticas humanas
WO2011075656A1 (en) 2009-12-18 2011-06-23 The University Of British Columbia Methods and compositions for delivery of nucleic acids
WO2011141705A1 (en) 2010-05-12 2011-11-17 Protiva Biotherapeutics, Inc. Novel cationic lipids and methods of use thereof
US9006417B2 (en) 2010-06-30 2015-04-14 Protiva Biotherapeutics, Inc. Non-liposomal systems for nucleic acid delivery
WO2012016184A2 (en) 2010-07-30 2012-02-02 Alnylam Pharmaceuticals, Inc. Methods and compositions for delivery of active agents
EP2600901B1 (en) 2010-08-06 2019-03-27 ModernaTX, Inc. A pharmaceutical formulation comprising engineered nucleic acids and medical use thereof
CN104531812A (zh) 2010-10-01 2015-04-22 现代治疗公司 设计核酸及其使用方法
WO2012075040A2 (en) 2010-11-30 2012-06-07 Shire Human Genetic Therapies, Inc. mRNA FOR USE IN TREATMENT OF HUMAN GENETIC DISEASES
US10849975B2 (en) * 2011-02-03 2020-12-01 Thomas Jefferson University Multivalent vaccines for rabies virus and filoviruses
US8710200B2 (en) 2011-03-31 2014-04-29 Moderna Therapeutics, Inc. Engineered nucleic acids encoding a modified erythropoietin and their expression
WO2012158736A1 (en) 2011-05-17 2012-11-22 modeRNA Therapeutics Engineered nucleic acids and methods of use thereof for non-human vertebrates
ES2663360T3 (es) 2011-06-08 2018-04-12 Translate Bio, Inc. Lípidos escindibles
BR112013031553A2 (pt) 2011-06-08 2020-11-10 Shire Human Genetic Therapies, Inc. composições, mrna que codifica para uma hgla e seu uso, uso de pelo menos uma molécula de mrna e um veículo de transferência e uso de um mrna que codifica para proteína exógena
US9464124B2 (en) 2011-09-12 2016-10-11 Moderna Therapeutics, Inc. Engineered nucleic acids and methods of use thereof
WO2013039861A2 (en) 2011-09-12 2013-03-21 modeRNA Therapeutics Engineered nucleic acids and methods of use thereof
EP2755986A4 (en) 2011-09-12 2015-05-20 Moderna Therapeutics Inc MANIPULATED NUCLEIC ACIDS AND METHOD OF APPLICATION THEREFOR
CN104114571A (zh) 2011-10-05 2014-10-22 普洛体维生物治疗公司 用于沉默醛脱氢酶的组合物和方法
NZ747501A (en) * 2011-10-27 2020-05-29 Massachusetts Inst Technology Amino acid derivatives functionalized on the n-terminal capable of forming drug encapsulating microspheres
EP2791364A4 (en) 2011-12-14 2015-11-11 Moderna Therapeutics Inc PROCESS FOR RESPONSE TO A BIOLOGICAL THREAT
US20140343129A1 (en) 2011-12-14 2014-11-20 Moderna Therapeutics, Inc. Modified nucleic acids, and acute care uses thereof
PL2791160T3 (pl) 2011-12-16 2022-06-20 Modernatx, Inc. Kompozycje zmodyfikowanego mrna
JP2015510495A (ja) 2011-12-21 2015-04-09 モデルナ セラピューティクス インコーポレイテッドＭｏｄｅｒｎａ Ｔｈｅｒａｐｅｕｔｉｃｓ，Ｉｎｃ． 器官または器官移植片の生存可能性または寿命を延長する方法
EP2797634A4 (en) 2011-12-29 2015-08-05 Moderna Therapeutics Inc MODIFIED MRNAS FOR THE CODING OF CELL-PENETRATING POLYPEPTIDES
RS58077B1 (sr) 2012-02-24 2019-02-28 Arbutus Biopharma Corp Trialkil katjonski lipidi i postupci njihove primene
BR112014024131A2 (pt) * 2012-03-29 2017-07-25 Shire Human Genetic Therapies lipídios catiônicos ionizáveis
JP6211054B2 (ja) * 2012-03-29 2017-10-11 シャイアー ヒューマン ジェネティック セラピーズ インコーポレイテッド 脂質誘導される中性ナノ粒子
AU2013243949A1 (en) 2012-04-02 2014-10-30 Moderna Therapeutics, Inc. Modified polynucleotides for the production of biologics and proteins associated with human disease
US20150050354A1 (en) 2012-04-02 2015-02-19 Moderna Therapeutics, Inc. Modified polynucleotides for the treatment of otic diseases and conditions
US9572897B2 (en) 2012-04-02 2017-02-21 Modernatx, Inc. Modified polynucleotides for the production of cytoplasmic and cytoskeletal proteins
US9283287B2 (en) 2012-04-02 2016-03-15 Moderna Therapeutics, Inc. Modified polynucleotides for the production of nuclear proteins
US20140275229A1 (en) 2012-04-02 2014-09-18 Moderna Therapeutics, Inc. Modified polynucleotides encoding udp glucuronosyltransferase 1 family, polypeptide a1
US10501512B2 (en) 2012-04-02 2019-12-10 Modernatx, Inc. Modified polynucleotides
HK1206779A1 (en) 2012-04-02 2016-01-15 Modernatx, Inc. Modified polynucleotides for the production of nuclear proteins
DK2922554T3 (en) 2012-11-26 2022-05-23 Modernatx Inc Terminalt modificeret rna
EP2929035A1 (en) 2012-12-07 2015-10-14 Shire Human Genetic Therapies, Inc. Lipidic nanoparticles for mrna delivering
US20150315541A1 (en) 2012-12-13 2015-11-05 Moderna Therapeutics, Inc. Modified polynucleotides for altering cell phenotype
AU2013374345A1 (en) 2013-01-17 2015-08-06 Moderna Therapeutics, Inc. Signal-sensor polynucleotides for the alteration of cellular phenotypes
EP2968397A4 (en) 2013-03-12 2016-12-28 Moderna Therapeutics Inc DIAGNOSIS AND TREATMENT OF FIBROSIS
EP2968391A1 (en) 2013-03-13 2016-01-20 Moderna Therapeutics, Inc. Long-lived polynucleotide molecules
EP2971010B1 (en) 2013-03-14 2020-06-10 ModernaTX, Inc. Formulation and delivery of modified nucleoside, nucleotide, and nucleic acid compositions
WO2014153052A2 (en) * 2013-03-14 2014-09-25 Shire Human Genetic Therapies, Inc. Cftr mrna compositions and related methods and uses
US10077439B2 (en) 2013-03-15 2018-09-18 Modernatx, Inc. Removal of DNA fragments in mRNA production process
WO2014144711A1 (en) 2013-03-15 2014-09-18 Moderna Therapeutics, Inc. Analysis of mrna heterogeneity and stability
US11377470B2 (en) 2013-03-15 2022-07-05 Modernatx, Inc. Ribonucleic acid purification
ES2795249T3 (es) * 2013-03-15 2020-11-23 Translate Bio Inc Mejora sinérgica de la administración de ácidos nucleicos a través de formulaciones mezcladas
US20160032273A1 (en) 2013-03-15 2016-02-04 Moderna Therapeutics, Inc. Characterization of mrna molecules
WO2014144767A1 (en) 2013-03-15 2014-09-18 Moderna Therapeutics, Inc. Ion exchange purification of mrna
US10138507B2 (en) 2013-03-15 2018-11-27 Modernatx, Inc. Manufacturing methods for production of RNA transcripts
US8980864B2 (en) 2013-03-15 2015-03-17 Moderna Therapeutics, Inc. Compositions and methods of altering cholesterol levels
PT3019619T (pt) 2013-07-11 2021-11-11 Modernatx Inc Composições que compreendem polinucleótidos sintéticos que codificam proteínas relacionadas com crispr e sgarn sintéticos e métodos de utilização
CN105555757A (zh) 2013-07-23 2016-05-04 普洛体维生物治疗公司 用于递送信使rna的组合物和方法
US20160194625A1 (en) 2013-09-03 2016-07-07 Moderna Therapeutics, Inc. Chimeric polynucleotides
EP3041938A1 (en) 2013-09-03 2016-07-13 Moderna Therapeutics, Inc. Circular polynucleotides
US10023626B2 (en) 2013-09-30 2018-07-17 Modernatx, Inc. Polynucleotides encoding immune modulating polypeptides
EP3052511A4 (en) 2013-10-02 2017-05-31 Moderna Therapeutics, Inc. Polynucleotide molecules and uses thereof
EP3052479A4 (en) 2013-10-02 2017-10-25 Moderna Therapeutics, Inc. Polynucleotide molecules and uses thereof
CA2927393A1 (en) 2013-10-18 2015-04-23 Moderna Therapeutics, Inc. Compositions and methods for tolerizing cellular systems
WO2015085318A2 (en) 2013-12-06 2015-06-11 Moderna Therapeutics, Inc. Targeted adaptive vaccines
EP3053585A1 (en) 2013-12-13 2016-08-10 Moderna Therapeutics, Inc. Alternative nucleic acid molecules and uses thereof
PL3981437T3 (pl) 2014-04-23 2025-02-24 Modernatx, Inc. Szczepionki z kwasem nukleinowym
JP2017536092A (ja) 2014-10-02 2017-12-07 プロティバ バイオセラピューティクス インコーポレイテッド Ｂ型肝炎ウイルスの遺伝子発現をサイレンシングするための組成物及び方法
WO2016071857A1 (en) 2014-11-07 2016-05-12 Protiva Biotherapeutics, Inc. Compositions and methods for silencing ebola virus expression
EP3461904A1 (en) 2014-11-10 2019-04-03 ModernaTX, Inc. Alternative nucleic acid molecules containing reduced uracil content and uses thereof
WO2016077123A1 (en) 2014-11-10 2016-05-19 Moderna Therapeutics, Inc. Multiparametric nucleic acid optimization
WO2016100812A1 (en) 2014-12-19 2016-06-23 Moderna Therapeutics, Inc. Terminal modifications of polynucleotides
EP3247363A4 (en) 2015-01-21 2018-10-03 Moderna Therapeutics, Inc. Lipid nanoparticle compositions
WO2016118725A1 (en) 2015-01-23 2016-07-28 Moderna Therapeutics, Inc. Lipid nanoparticle compositions
WO2016154127A2 (en) 2015-03-20 2016-09-29 Protiva Biotherapeutics, Inc. Compositions and methods for treating hypertriglyceridemia
WO2016164762A1 (en) 2015-04-08 2016-10-13 Moderna Therapeutics, Inc. Polynucleotides encoding low density lipoprotein receptor egf-a and intracellular domain mutants and methods of using the same
KR20210021943A (ko) 2018-04-25 2021-03-02 에트리스 게엠베하 미립자 제형용 동결 방지제

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US12458604B2 (en) 2020-10-14 2025-11-04 The Trustees Of The University Of Pennsylvania Methods of lipid nanoparticle manufacture and compositions derived therefrom

Also Published As

Publication number Publication date
EP4036241A1 (en) 2022-08-03
CA2928040A1 (en) 2015-04-30
EP3060671A1 (en) 2016-08-31
US20150110857A1 (en) 2015-04-23
US10780052B2 (en) 2020-09-22
JP2025013512A (ja) 2025-01-24
AU2021201220A1 (en) 2021-03-11
AU2021201220B2 (en) 2024-05-02
BR112016008832A2 (pt) 2017-10-03
JP2023041951A (ja) 2023-03-24
US20240408013A1 (en) 2024-12-12
US20210196633A1 (en) 2021-07-01
US20180028445A1 (en) 2018-02-01
EA201690588A1 (ru) 2016-09-30
JP2016539916A (ja) 2016-12-22
US12016954B2 (en) 2024-06-25
EP4036241A9 (en) 2023-03-22
AU2024205165A1 (en) 2024-08-15
JP2021073313A (ja) 2021-05-13
EP3060671B1 (en) 2021-12-29
AU2014340149B2 (en) 2020-12-24
JP7586858B2 (ja) 2024-11-19
CN105658800A (zh) 2016-06-08
AU2014340149A1 (en) 2016-05-12
JP2022121548A (ja) 2022-08-19
WO2015061461A1 (en) 2015-04-30
JP2019065047A (ja) 2019-04-25
JP6893785B2 (ja) 2021-06-23

Similar Documents

Publication Publication Date Title
MX2016005236A (es) Administracion de acido ribonucleico mensajero al sistema nervioso central y sus usos.
NZ718817A (en) Lipid formulations for delivery of messenger rna
MX2024000327A (es) Composiciones y metodos para la administracion de arn mensajero.
PH12019501339A1 (en) Amino acid compositions and methods for the treatment of muscle diseases and disorders
WO2014145159A3 (en) Charge-engineered antibodies or compositions of penetration-enhanced targeting proteins and methods of use
EP4537906A3 (en) Compositions and methods for delivery of biomacromolecule agents
TW201613954A (en) Interleukin-10 fusion proteins and uses thereof
IN2014DN10386A (es)
EP4406597A3 (en) Immune-modifying particles for the treatment of inflammation
MX2019000586A (es) Virus adenoasociados (aav) sflt-1 para usarse en el tratamiento de degeneración macular relacionada con la edad (amd).
PH12013502240B1 (en) Novel compounds as modulators of protein kinase
EP4331590A3 (en) Nuclear factor erythroid 2-like 2 (nrf2) for use in treatment of age-related macular degeneration
MX2014004359A (es) Proteina naglu humana recombinante y usos de la misma.
GB2496337A (en) Combination pharmaceutical composition and methods of treating diseases or conditions associated with neurodegenerative diseases
MX2013011908A (es) Composiciones y usos terapeuticos de inhibidores de cinasa epsilon relacionados con cinasa i-kappa b (ikk) y cinasa 1 de union tank.
NZ616989A (en) Therapeutic nuclease compositions and methods
EP4414376A3 (en) Novel depsipeptide and uses thereof
WO2014184576A3 (en) Delivery of a therapeutic agent to the cerebral cortex by administering a viral vector by convection enhanced diffusion into the white matter of the brain
IN2014DN06920A (es)
WO2016149659A3 (en) Antisense-induced exon exclusion in myostatin
PH12015501271A1 (en) Stabilized insulin-like growth factor polypeptides
EA202091020A1 (ru) Замещенные имидазопиридинамиды и их применение
MX2013011421A (es) Derivados de pirazolo-pirimidina.
MX2013011996A (es) Metodos para regular la expresion de genes de sirtuina.
MX358598B (es) Partículas inmuno-moduladoras modificadas.