MX2020003608A - Composiciones y métodos para la edición del gen ttr y el tratamiento de la amiloidosis attr. - Google Patents

Composiciones y métodos para la edición del gen ttr y el tratamiento de la amiloidosis attr.

Info

Publication number
MX2020003608A
MX2020003608A MX2020003608A MX2020003608A MX2020003608A MX 2020003608 A MX2020003608 A MX 2020003608A MX 2020003608 A MX2020003608 A MX 2020003608A MX 2020003608 A MX2020003608 A MX 2020003608A MX 2020003608 A MX2020003608 A MX 2020003608A
Authority
MX
Mexico
Prior art keywords
compositions
methods
gene editing
ttr gene
attr amyloidosis
Prior art date
Application number
MX2020003608A
Other languages
English (en)
Inventor
Walter Strapps
Shobu Odate
Reynald Michael Lescarbeau
Arti Mahendra Prakash Kanjolia
Jessica Lynn Seitzer
Original Assignee
Intellia Therapeutics 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 Intellia Therapeutics Inc filed Critical Intellia Therapeutics Inc
Publication of MX2020003608A publication Critical patent/MX2020003608A/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
    • A61K48/0008—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
    • 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
    • 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
    • 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
    • C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
    • C12N15/102—Mutagenizing nucleic acids
    • 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
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • 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
    • 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
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
    • 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
    • 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
    • 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
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/111—General methods applicable to biologically active non-coding nucleic acids
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
    • 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
    • C12N2800/00—Nucleic acids vectors
    • C12N2800/80—Vectors containing sites for inducing double-stranded breaks, e.g. meganuclease restriction sites

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Molecular Biology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Medicinal Chemistry (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Epidemiology (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • General Chemical & Material Sciences (AREA)
  • Psychiatry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Hospice & Palliative Care (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Toxicology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Manufacturing & Machinery (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)

Abstract

Se proporcionan las composiciones y los métodos para editar, por ejemplo, introducir ruptura bicatenaria, dentro del gen TTR. Se proporcionan las composiciones y los métodos para tratar sujetos que tienen amiloidosis asociada con la transtiretina (ATTR).
MX2020003608A 2017-09-29 2018-09-28 Composiciones y métodos para la edición del gen ttr y el tratamiento de la amiloidosis attr. MX2020003608A (es)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201762566236P 2017-09-29 2017-09-29
US201862671902P 2018-05-15 2018-05-15
PCT/US2018/053382 WO2019067872A1 (en) 2017-09-29 2018-09-28 COMPOSITIONS AND METHODS FOR EDITING THE TTR GENE AND TREATING AMYLOID DOSE ATTR

Publications (1)

Publication Number Publication Date
MX2020003608A true MX2020003608A (es) 2020-09-25

Family

ID=63858214

Family Applications (2)

Application Number Title Priority Date Filing Date
MX2020003608A MX2020003608A (es) 2017-09-29 2018-09-28 Composiciones y métodos para la edición del gen ttr y el tratamiento de la amiloidosis attr.
MX2025014378A MX2025014378A (es) 2017-09-29 2020-07-13 Composiciones utiles para la edicion del gen ttr y el tratamiento de la amiloidosis attr

Family Applications After (1)

Application Number Title Priority Date Filing Date
MX2025014378A MX2025014378A (es) 2017-09-29 2020-07-13 Composiciones utiles para la edicion del gen ttr y el tratamiento de la amiloidosis attr

Country Status (16)

Country Link
US (4) US20200248180A1 (es)
EP (1) EP3688161A1 (es)
JP (3) JP2021500864A (es)
KR (1) KR20200058509A (es)
CN (2) CN118530993A (es)
AU (2) AU2018338787C1 (es)
BR (1) BR112020005287A2 (es)
CA (1) CA3077251A1 (es)
CO (1) CO2020005116A2 (es)
IL (3) IL273317A (es)
MX (2) MX2020003608A (es)
MY (1) MY203849A (es)
PH (1) PH12020550364A1 (es)
SG (1) SG11202002565YA (es)
TW (1) TWI904069B (es)
WO (1) WO2019067872A1 (es)

Families Citing this family (21)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US12491261B2 (en) 2016-10-26 2025-12-09 Acuitas Therapeutics, Inc. Lipid nanoparticle formulations
ES2962277T3 (es) 2017-09-29 2024-03-18 Regeneron Pharma Roedores que comprenden un locus Ttr humanizado y métodos de uso
CA3134271A1 (en) * 2019-03-28 2020-10-01 Intellia Therapeutics, Inc. Compositions and methods comprising a ttr guide rna and a polynucleotide encoding an rna-guided dna binding agent
MA55527A (fr) * 2019-03-28 2022-02-09 Intellia Therapeutics Inc Polynucléotides, compositions et procédés d'expression de polypeptides
MX2021011690A (es) * 2019-03-28 2022-01-06 Intellia Therapeutics Inc Composiciones y métodos para la edición del gen ttr y el tratamiento de la amiloidosis attr que comprende un corticosteroide o uso de este.
EP3801011A1 (en) * 2019-06-04 2021-04-14 Regeneron Pharmaceuticals, Inc. Non-human animals comprising a humanized ttr locus with a beta-slip mutation and methods of use
EP4073249A1 (en) * 2019-12-11 2022-10-19 Intellia Therapeutics, Inc. Modified guide rnas for gene editing
JP2023548005A (ja) * 2020-10-28 2023-11-15 ノヴォ ノルディスク アー／エス 抗トランスサイレチン抗体およびその使用方法
KR20230129996A (ko) 2020-12-11 2023-09-11 인텔리아 테라퓨틱스, 인크. 탈아미노화를 포함하는 게놈 편집을 위한 폴리뉴클레오타이드,조성물 및 방법
CN112795595A (zh) * 2020-12-24 2021-05-14 中山大学 一种遗传性转甲状腺素蛋白淀粉样变性疾病的基因治疗系统
KR20240038705A (ko) 2021-06-22 2024-03-25 인텔리아 테라퓨틱스, 인크. 간 유전자의 생체 내 편집 방법
US20250127927A1 (en) * 2021-08-23 2025-04-24 The Board Of Trustees Of The Leland Stanford Junior University Targeted integration at beta-globin locus in human hematopoietic stem and progenitor cells
JP2024535888A (ja) 2021-09-20 2024-10-02 アルナイラム ファーマシューティカルズ， インコーポレイテッド インヒビンサブユニットベータｅ（ｉｎｈｂｅ）モジュレーター組成物およびその使用方法
CN116064598B (zh) * 2021-10-08 2024-03-12 苏州艾博生物科技有限公司 冠状病毒的核酸疫苗
EP4499829A2 (en) * 2022-03-29 2025-02-05 Accuredit Therapeutics (Suzhou) Co., Ltd. Compositions and methods for treatment of transthyretin amyloidosis
WO2024003810A1 (en) * 2022-06-30 2024-01-04 Geneditbio Limited Guide rna with chemical modifications
WO2024003805A1 (en) * 2022-06-30 2024-01-04 Geneditbio Limited Methods and compositions for ttr gene editing and therapy using crispr system
CN118853659A (zh) * 2023-04-14 2024-10-29 深圳瑞吉生物科技有限公司 提高mRNA翻译蛋白的输出量的核酸及其应用
WO2024259135A1 (en) 2023-06-13 2024-12-19 Intellia Therapeutics, Inc. Assays for analysis of ribonucleic acid (rna) molecules
WO2025087417A1 (zh) * 2023-10-25 2025-05-01 石药集团中奇制药技术（石家庄）有限公司 一种高效低脱靶的基因编辑工具
WO2025128871A2 (en) 2023-12-13 2025-06-19 Renagade Therapeutics Management Inc. Lipid nanoparticles comprising coding rna molecules for use in gene editing and as vaccines and therapeutic agents

Family Cites Families (34)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5585481A (en) 1987-09-21 1996-12-17 Gen-Probe Incorporated Linking reagents for nucleotide probes
US5378825A (en) 1990-07-27 1995-01-03 Isis Pharmaceuticals, Inc. Backbone modified oligonucleotide analogs
EP1044987B1 (en) 1991-12-24 2006-02-15 Isis Pharmaceuticals, Inc. Gapped 2'-modified oligonucleotides
US6169169B1 (en) 1994-05-19 2001-01-02 Dako A/S PNA probes for detection of Neisseria gonorrhoeae and Chlamydia trachomatis
US6974667B2 (en) 2000-06-14 2005-12-13 Gene Logic, Inc. Gene expression profiles in liver cancer
US7244764B2 (en) * 2003-06-23 2007-07-17 Neurochem (International) Limited Methods and compositions for treating amyloid-related diseases
US20060051405A1 (en) 2004-07-19 2006-03-09 Protiva Biotherapeutics, Inc. Compositions for the delivery of therapeutic agents and uses thereof
US20110237646A1 (en) 2008-08-07 2011-09-29 Isis Pharmaceuticals, Inc. Modulation of transthyretin expression for the treatment of cns related disorders
SG10201809460SA (en) 2008-10-20 2018-11-29 Alnylam Pharmaceuticals Inc Compositions and methods for inhibiting expression of transthyretin
WO2011056883A1 (en) 2009-11-03 2011-05-12 Alnylam Pharmaceuticals, Inc. Lipid formulated compositions and methods for inhibiting expression of transthyretin (ttr)
ES2625689T3 (es) 2010-04-29 2017-07-20 Ionis Pharmaceuticals, Inc. Modulación de la expresión de transtiretina
WO2013075035A1 (en) 2011-11-18 2013-05-23 Alnylam Pharmaceuticals Rnai agents, compositions and methods of use thereof for treating transthyretin (ttr) associated diseases
WO2014093622A2 (en) * 2012-12-12 2014-06-19 The Broad Institute, Inc. Delivery, engineering and optimization of systems, methods and compositions for sequence manipulation and therapeutic applications
WO2014093694A1 (en) 2012-12-12 2014-06-19 The Broad Institute, Inc. Crispr-cas nickase systems, methods and compositions for sequence manipulation in eukaryotes
WO2014093655A2 (en) 2012-12-12 2014-06-19 The Broad Institute, Inc. Engineering and optimization of systems, methods and compositions for sequence manipulation with functional domains
EP4282970A3 (en) 2012-12-17 2024-01-17 President and Fellows of Harvard College Rna-guided human genome engineering
KR102255108B1 (ko) 2013-03-08 2021-05-24 노파르티스 아게 활성제의 전달을 위한 지질 및 지질 조성물
CA2908697C (en) * 2013-04-16 2023-12-12 Regeneron Pharmaceuticals, Inc. Targeted modification of rat genome
PE20152002A1 (es) 2013-05-01 2016-01-21 Isis Pharmaceuticals Inc Composiciones y metodos para modular la expresion de ttr y vhb
SG11201510327TA (en) * 2013-06-17 2016-01-28 Broad Inst Inc Delivery, engineering and optimization of systems, methods and compositions for targeting and modeling diseases and disorders of post mitotic cells
US20150165054A1 (en) 2013-12-12 2015-06-18 President And Fellows Of Harvard College Methods for correcting caspase-9 point mutations
EP3623361B1 (en) 2013-12-19 2021-08-18 Novartis AG Lipids and lipid compositions for the delivery of active agents
JP2017516500A (ja) * 2014-05-28 2017-06-22 ツールゲン インコーポレイテッドＴｏｏｌｇｅｎ Ｉｎｃｏｒｐｏｒａｔｅｄ 標的特異的ヌクレアーゼを用いた標的ｄｎａの敏感な検出方法
EP3161128B1 (en) * 2014-06-26 2018-09-26 Regeneron Pharmaceuticals, Inc. Methods and compositions for targeted genetic modifications and methods of use
WO2016010840A1 (en) 2014-07-16 2016-01-21 Novartis Ag Method of encapsulating a nucleic acid in a lipid nanoparticle host
WO2016141224A1 (en) 2015-03-03 2016-09-09 The General Hospital Corporation Engineered crispr-cas9 nucleases with altered pam specificity
FI4104687T3 (fi) 2015-09-21 2024-03-01 Trilink Biotechnologies Llc Koostumuksia ja menetelmiä 5-huputettujen RNA:iden syntetisoimiseksi
CN108350454B (zh) * 2015-09-21 2022-05-10 阿克丘勒斯治疗公司 等位基因选择性基因编辑及其用途
WO2017077386A1 (en) * 2015-11-06 2017-05-11 Crispr Therapeutics Ag Materials and methods for treatment of glycogen storage disease type 1a
JP6932698B2 (ja) * 2015-12-01 2021-09-08 クリスパー・セラピューティクス・アクチェンゲゼルシャフトＣＲＩＳＰＲ Ｔｈｅｒａｐｅｕｔｉｃｓ ＡＧ アルファ１アンチトリプシン欠乏症の治療のための材料および方法
US11083799B2 (en) * 2016-03-16 2021-08-10 Crispr Therapeutics Ag Materials and methods for treatment of hereditary haemochromatosis
SMT202500211T1 (it) * 2016-03-30 2025-07-22 Intellia Therapeutics Inc Formulazioni di nanoparticelle lipidiche per componenti crispr/cas
WO2018007871A1 (en) * 2016-07-08 2018-01-11 Crispr Therapeutics Ag Materials and methods for treatment of transthyretin amyloidosis
IL267024B2 (en) 2016-12-08 2023-12-01 Intellia Therapeutics Inc Adapted leader RNAs for genomic editing

Also Published As

Publication number Publication date
US20230118592A1 (en) 2023-04-20
IL273317A (en) 2020-04-30
US20250207130A1 (en) 2025-06-26
AU2018338787A1 (en) 2020-04-16
KR20200058509A (ko) 2020-05-27
BR112020005287A2 (pt) 2020-09-24
US11965165B2 (en) 2024-04-23
CO2020005116A2 (es) 2020-05-15
CA3077251A1 (en) 2019-04-04
AU2025202742A1 (en) 2025-05-15
CN111417728A (zh) 2020-07-14
US11795460B2 (en) 2023-10-24
PH12020550364A1 (en) 2021-02-15
JP7707140B2 (ja) 2025-07-14
WO2019067872A1 (en) 2019-04-04
IL325460A (en) 2026-02-01
EP3688161A1 (en) 2020-08-05
JP2023029966A (ja) 2023-03-07
JP2025163016A (ja) 2025-10-28
CN118530993A (zh) 2024-08-23
US20230257747A1 (en) 2023-08-17
TW201932479A (zh) 2019-08-16
AU2018338787B2 (en) 2025-05-08
NZ762942A (en) 2024-10-25
AU2018338787C1 (en) 2025-10-23
JP2021500864A (ja) 2021-01-14
SG11202002565YA (en) 2020-04-29
IL311170B1 (en) 2026-02-01
IL311170A (en) 2024-04-01
TWI904069B (zh) 2025-11-11
MX2025014378A (es) 2026-01-07
US20200248180A1 (en) 2020-08-06
MY203849A (en) 2024-07-21

Similar Documents

Publication Publication Date Title
MX2020003608A (es) Composiciones y métodos para la edición del gen ttr y el tratamiento de la amiloidosis attr.
CO2021005884A2 (es) Moduladores de la alfa-1 antitripsina
CL2018003316A1 (es) Compuestos para la inhibición de k-ras y para el tratamiento del cáncer. (divisional solicitud 201702786)
ECSP19044183A (es) (aza)indol-, benzotiofen, y benzofuran-3-sulfonamidas
CO2020015255A2 (es) Métodos y composiciones para tratar el cáncer
CO2019013010A2 (es) Inhibidores de kras g12c y métodos para su uso
MX2021001070A (es) Composiciones y métodos para editar el gen hidroxiácido oxidasa 1 (hao1) para tratar la hiperoxaluria primaria tipo 1 (ph1).
MX2018005745A (es) Control de niveles redox celulares.
CR20190318A (es) Compuestos heterocíclicos como inmunomoduladores
MX2015005536A (es) Tratamiento de canceres utilizando moduladores de las isoformas de pi3 cinasa.
MX2016013457A (es) Inhibidores de mdm2 y metodos terapeuticos que utilizan el mismo.
ECSP20046463A (es) Compuestos, composiciones y métodos para tratar enfermedades que involucren tejidos con enfermedades ácidas o hipóxicas
CO2017004525A2 (es) Dihidropirrolopiridinas inhibidoras de ror-gamma
MX2018005315A (es) Composiciones y metodos para el tratamiento del cancer.
MX2018006745A (es) Metodos de tratamiento de neoplasias malignas.
MX2018000511A (es) Derivados de fenoximetilo.
UY36654A (es) Azabenzimidazoles con propiedades moduladoras del receptor ampa y composiciones farmacéuticas que los contienen
BR112017025711A2 (pt) ?composições, processo para preparação de siloxanos modificados por poliéter e uso das composições inovadoras?
MX2017016681A (es) Uso de compuestos de cloroquina y clemizol para el tratamiento de afecciones inflamatorias y cancerosas.
UY37800A (es) AGENTES DE iARN PARA LA INHIBICIÓN DE LA EXPRESIÓN DE ALFA-ENaC Y MÉTODOS DE USO
CO2022000214A2 (es) Composiciones para el tratamiento de la pérdida del cabello
MX2017012553A (es) Compuestos espirociclicos.
BR112018010456A2 (pt) ?composição, método para limpeza ou desinfecção de uma superfície e uso de uma composição?
MX2019010060A (es) Composiciones y metodo para tratar cancer.
UY36732A (es) Piridin pirazinonas novedosas como inhibidores de bromodominios de la familia bet