CO2022010079A2 - Suministro rectal de arn mensajero - Google Patents

Suministro rectal de arn mensajero

Info

Publication number
CO2022010079A2
CO2022010079A2 CONC2022/0010079A CO2022010079A CO2022010079A2 CO 2022010079 A2 CO2022010079 A2 CO 2022010079A2 CO 2022010079 A CO2022010079 A CO 2022010079A CO 2022010079 A2 CO2022010079 A2 CO 2022010079A2
Authority
CO
Colombia
Prior art keywords
rectal delivery
messenger rna
mrna
present
rnase
Prior art date
Application number
CONC2022/0010079A
Other languages
English (en)
Inventor
Shrirang Karve
Frank Derosa
Ashish Sarode
Original Assignee
Translate Bio 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 Translate Bio Inc filed Critical Translate Bio Inc
Publication of CO2022010079A2 publication Critical patent/CO2022010079A2/es

Links

Classifications

    • 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
    • A61K9/0031—Rectum, anus
    • 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
    • A61K9/02—Suppositories; Bougies; Bases therefor; Ovules
    • 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—Liposomes
    • A61K9/1271—Non-conventional liposomes, e.g. PEGylated liposomes, liposomes coated with polymers
    • 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
    • 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
    • A61K9/513—Organic macromolecular compounds; Dendrimers
    • A61K9/5146—Organic macromolecular compounds; Dendrimers obtained otherwise than by reactions only involving carbon-to-carbon unsaturated bonds, e.g. polyethylene glycol, polyamines, polyanhydrides
    • 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
    • A61K9/5192—Processes
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • 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
    • A61K48/0091—Purification or manufacturing processes for gene therapy compositions

Landscapes

  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Public Health (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • General Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Physics & Mathematics (AREA)
  • Biomedical Technology (AREA)
  • Nanotechnology (AREA)
  • Optics & Photonics (AREA)
  • Genetics & Genomics (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Dispersion Chemistry (AREA)
  • Biochemistry (AREA)
  • Manufacturing & Machinery (AREA)
  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Medicinal Preparation (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)

Abstract

La presente invención proporciona, entre otras cosas, métodos y composiciones eficaces para suministrar ARN mensajero (ARNm) mediante suministro rectal. La presente invención se basa, en parte, en la observación inesperada de que el ARNm puede suministrase de manera eficaz a la circulación, hígado, riñón, colon y/o recto mediante suministro rectal a pesar de barreras tales como la ARNasa y la capa mucosa.
CONC2022/0010079A 2019-12-20 2022-07-18 Suministro rectal de arn mensajero CO2022010079A2 (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201962951844P 2019-12-20 2019-12-20
PCT/US2020/065945 WO2021127394A2 (en) 2019-12-20 2020-12-18 Rectal delivery of messenger rna

Publications (1)

Publication Number Publication Date
CO2022010079A2 true CO2022010079A2 (es) 2022-10-21

Family

ID=74554205

Family Applications (1)

Application Number Title Priority Date Filing Date
CONC2022/0010079A CO2022010079A2 (es) 2019-12-20 2022-07-18 Suministro rectal de arn mensajero

Country Status (12)

Country Link
US (1) US20230051811A1 (es)
EP (1) EP4076393A2 (es)
JP (1) JP2023508882A (es)
KR (1) KR20220142432A (es)
CN (1) CN115515559A (es)
AU (1) AU2020408059A1 (es)
BR (1) BR112022012085A2 (es)
CA (1) CA3162368A1 (es)
CO (1) CO2022010079A2 (es)
IL (1) IL294073A (es)
MX (1) MX2022007756A (es)
WO (1) WO2021127394A2 (es)

Families Citing this family (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP4395748A1 (en) 2021-09-03 2024-07-10 CureVac SE Novel lipid nanoparticles for delivery of nucleic acids
EP4422698A1 (en) 2021-10-29 2024-09-04 CureVac SE Improved circular rna for expressing therapeutic proteins
WO2023144330A1 (en) 2022-01-28 2023-08-03 CureVac SE Nucleic acid encoded transcription factor inhibitors
WO2023227608A1 (en) 2022-05-25 2023-11-30 Glaxosmithkline Biologicals Sa Nucleic acid based vaccine encoding an escherichia coli fimh antigenic polypeptide
US20240156949A1 (en) 2022-10-28 2024-05-16 Glaxosmithkline Biologicals Sa Nucleic Acid Based Vaccine
WO2024184500A1 (en) 2023-03-08 2024-09-12 CureVac SE Novel lipid nanoparticle formulations for delivery of nucleic acids

Family Cites Families (54)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
US4500707A (en) 1980-02-29 1985-02-19 University Patents, Inc. Nucleosides useful in the preparation of polynucleotides
US4668777A (en) 1981-03-27 1987-05-26 University Patents, Inc. Phosphoramidite nucleoside compounds
US4415732A (en) 1981-03-27 1983-11-15 University Patents, Inc. Phosphoramidite compounds and processes
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
DD273980B5 (de) * 1988-07-12 1994-04-14 Berlin Chemie Ag Verfahren zur Herstellung von Insulin-Pr{paraten zur rektalen Anwendung
US5047524A (en) 1988-12-21 1991-09-10 Applied Biosystems, Inc. Automated system for polynucleotide synthesis and purification
US5262530A (en) 1988-12-21 1993-11-16 Applied Biosystems, Inc. Automated system for polynucleotide synthesis and purification
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
US5885613A (en) 1994-09-30 1999-03-23 The University Of British Columbia Bilayer stabilizing components and their use in forming programmable fusogenic liposomes
US5700642A (en) 1995-05-22 1997-12-23 Sri International Oligonucleotide sizing using immobilized cleavable primers
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
WO2005121348A1 (en) 2004-06-07 2005-12-22 Protiva Biotherapeutics, Inc. Lipid encapsulated interfering rna
LT2578685T (lt) 2005-08-23 2019-06-10 The Trustees Of The University Of Pennsylvania Rnr, apimančios modifikuotus nukleozidus ir jų panaudojimo būdai
PL2350043T3 (pl) 2008-10-09 2014-09-30 Tekmira Pharmaceuticals Corp Ulepszone aminolipidy i sposoby dostarczania kwasów nukleinowych
AU2009311667B2 (en) 2008-11-07 2016-04-14 Massachusetts Institute Of Technology Aminoalcohol lipidoids and uses thereof
KR20230098713A (ko) 2009-06-10 2023-07-04 알닐람 파마슈티칼스 인코포레이티드 향상된 지질 조성물
JP5785168B2 (ja) 2009-07-31 2015-09-24 エスリス ゲーエムベーハーｅｔｈｒｉｓ ＧｍｂＨ タンパク質発現用未修飾および修飾ヌクレオチドの組み合わせを有するｒｎａ
PT3338765T (pt) 2009-12-01 2019-03-18 Translate Bio Inc Derivado de esteróide adequado para a administração de arnm em doenças genéticas humanas
JP6022557B2 (ja) 2011-06-08 2016-11-09 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 切断可能な脂質
UA119028C2 (uk) 2011-10-27 2019-04-25 Массачусеттс Інстітьют Оф Текнолоджі Функціоналізовані на n-кінці амінокислотні похідні, здатні утворювати мікросфери, що інкапсулюють лікарський засіб
EP3620447B1 (en) 2012-03-29 2021-02-17 Translate Bio, Inc. Ionizable cationic lipids
JP2016514970A (ja) 2013-03-14 2016-05-26 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 メッセンジャーｒｎａのキャップ効率の定量的評価
JP6586075B2 (ja) 2013-03-14 2019-10-02 トランスレイト バイオ， インコーポレイテッド メッセンジャーｒｎａの精製方法
CN112656954A (zh) * 2013-10-22 2021-04-16 夏尔人类遗传性治疗公司 用于递送信使rna的脂质制剂
US10059655B2 (en) 2013-12-19 2018-08-28 Novartis Ag Lipids and lipid compositions for the delivery of active agents
KR20220158867A (ko) 2014-04-25 2022-12-01 샤이어 휴먼 지네틱 테라피즈 인크. 메신저 rna 의 정제 방법
AU2015266764B2 (en) 2014-05-30 2019-11-07 Translate Bio, Inc. Biodegradable lipids for delivery of nucleic acids
SI3766916T1 (sl) 2014-06-25 2023-01-31 Acuitas Therapeutics Inc. Formulacije novih lipidov in lipidnih nanodelcev za dostavo nukleinskih kislin
AU2015283954B2 (en) 2014-07-02 2020-11-12 Translate Bio, Inc. Encapsulation of messenger RNA
WO2016004202A1 (en) 2014-07-02 2016-01-07 Massachusetts Institute Of Technology Polyamine-fatty acid derived lipidoids and uses thereof
EP3247363A4 (en) 2015-01-21 2018-10-03 Moderna Therapeutics, Inc. Lipid nanoparticle compositions
US20180085474A1 (en) 2015-01-23 2018-03-29 Moderna Therapeutics, Inc. Lipid nanoparticle compositions
LT3310764T (lt) 2015-06-19 2023-06-12 Massachusetts Institute Of Technology Alkenilu pakeisti 2,5-piperazindionai ir jų panaudojimas kompozicijose, skirtose agento pristatymui į subjektą ar ląstelę
SI3313829T1 (sl) 2015-06-29 2024-09-30 Acuitas Therapeutics Inc. Lipidi in formulacije lipidnih nanodelcev za dostavo nukleinskih kislin
US20190119357A1 (en) * 2015-07-23 2019-04-25 Modernatx, Inc. Messenger ribonucleic acids for the production of intracellular binding polypeptides and methods of use thereof
RS63030B1 (sr) 2015-09-17 2022-04-29 Modernatx Inc Jedinjenja i kompozicije za intracelularno isporučivanje terapeutskih sredstava
CA3003055C (en) 2015-10-28 2023-08-01 Acuitas Therapeutics, Inc. Lipids and lipid nanoparticle formulations for delivery of nucleic acids
EP3397613A1 (en) 2015-12-30 2018-11-07 Acuitas Therapeutics Inc. Lipids and lipid nanoparticle formulations for delivery of nucleic acids
CN117731805A (zh) 2016-03-30 2024-03-22 因特利亚治疗公司 用于crispr/cas成分的脂质纳米颗粒制剂
US10471153B2 (en) 2016-11-10 2019-11-12 Translate Bio, Inc. Ice-based lipid nanoparticle formulation for delivery of mRNA
MA46761A (fr) * 2016-11-10 2019-09-18 Translate Bio Inc Administration sous-cutanée d'arn messager
AU2017357758B2 (en) 2016-11-10 2023-11-16 Translate Bio, Inc. Improved process of preparing mRNA-loaded lipid nanoparticles
HUE065140T2 (hu) 2017-02-27 2024-05-28 Translate Bio Inc Hírvivõ RNS nagy léptékû szintézise
ES2899323T3 (es) 2017-02-27 2022-03-10 Translate Bio Inc Métodos de purificación de ARN mensajero
US10975369B2 (en) 2017-02-27 2021-04-13 Translate Bio, Inc. Methods for purification of messenger RNA
WO2018213476A1 (en) 2017-05-16 2018-11-22 Translate Bio, Inc. Treatment of cystic fibrosis by delivery of codon-optimized mrna encoding cftr

Also Published As

Publication number Publication date
CN115515559A (zh) 2022-12-23
WO2021127394A3 (en) 2021-08-26
IL294073A (en) 2022-08-01
MX2022007756A (es) 2022-09-27
JP2023508882A (ja) 2023-03-06
KR20220142432A (ko) 2022-10-21
BR112022012085A2 (pt) 2022-08-30
EP4076393A2 (en) 2022-10-26
WO2021127394A2 (en) 2021-06-24
US20230051811A1 (en) 2023-02-16
AU2020408059A1 (en) 2022-08-11
CA3162368A1 (en) 2021-06-24

Similar Documents

Publication Publication Date Title
CO2022010079A2 (es) Suministro rectal de arn mensajero
CL2020001126A1 (es) Oligonucleótidos para reducir la expresión de pd-l1. (divisional solicitud 202000865)
CO2019003846A2 (es) Tratamiento aav de la enfermedad de huntington
CL2016003041A1 (es) Ácidos ribonucleicos bicatenarios aislados, composiciones farmacéuticas que los comprenden y método para inhibir la expresión del gen alas1 basado en la administración de dicha composición. divisional de solicitud 2725-2014.
SV2018005661A (es) Composiciones oligonucleotidicas y sus metodos
PE20171766A1 (es) Oligomeros antisentido de tau y usos de estos
CL2023002870A1 (es) Composiciones de arni contra el angiotensinógeno (agt) y métodos para su uso
PE20190443A1 (es) Moleculas de acido nucleico para la reduccion de arnm de papd5 o papd7 para tratar la infeccion por hepatitis b
DOP2022000293A (es) Análogos de nucleósido de 1’-ciano y usos de los mismos
CO2018000696A2 (es) Derivados de 3-fenoximetil-[1,2,4]triazol-2-ona, 3-fenoximetil-[1,2,3]triazol-2-ona, 3-fenoximetil-piridazin-3(2h)-ona, 3-fenoximetil-indazol, 3-fenoximetil-tetrahidroindazol y 3-fenoximetil-pirazol
CR20190444A (es) Arn terapéutico
CL2019000980A1 (es) Oligonucleotidos antisentido de al menos 10 nucleotidos que inhiben de la señalización del gen tgf-ii o una región del arnm que lo codifica. (divisional solicitud 201701233).
PE20210633A1 (es) CONSTRUCTOS DE ARNi PARA INHIBIR LA EXPRESION DE PNPLA3 Y METODOS DE USO DE LOS MISMOS
ES2689523T3 (es) Composiciones a base de ARNM del gen CFTR y métodos y usos relacionados
PE20241069A1 (es) Composiciones y metodos para disminuir la expresion de tau
CU24387B1 (es) Inhibidores de bmi-1 de pirimidina inversa sustituida
CL2018002793A1 (es) Terapia de cáncer con un virus oncolítico combinado con un inhibidor de puntos de control
PE20211225A1 (es) Constructos de arni para inhibir la expresion de pnpla3
CL2023001062A1 (es) Compuestos fosfolípidos y usos de estos
DOP2023000037A (es) Compuestos fosfolípidos y usos de los mismos
MX2020006012A (es) Composiciones de arni de la caja 1 del grupo de alta movilidad (hmgb1) y métodos de uso de las mismas.
CL2019001106A1 (es) Composición para aliviar o tratar el dolor.
CO2024011897A2 (es) Compuestos y métodos para el tratamiento de infecciones virales
CO2023005218A2 (es) Nanopartículas lipídicas para administrar vacunas de arnm
PE20220250A1 (es) Uso de docetaxel en el tratamiento de cancer mediante control de niveles maximos en plasma