BR112021012240A2 - Terapia de gene-célula-tronco hematopoiética para síndrome wiskott-aldrich - Google Patents
Terapia de gene-célula-tronco hematopoiética para síndrome wiskott-aldrichInfo
- Publication number
- BR112021012240A2 BR112021012240A2 BR112021012240A BR112021012240A BR112021012240A2 BR 112021012240 A2 BR112021012240 A2 BR 112021012240A2 BR 112021012240 A BR112021012240 A BR 112021012240A BR 112021012240 A BR112021012240 A BR 112021012240A BR 112021012240 A2 BR112021012240 A2 BR 112021012240A2
- Authority
- BR
- Brazil
- Prior art keywords
- wiskott
- aldrich syndrome
- stem cell
- gene therapy
- hematopoietic stem
- Prior art date
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
- A61K48/005—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
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
- A61K35/12—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
- A61K35/28—Bone marrow; Haematopoietic stem cells; Mesenchymal stem cells of any origin, e.g. adipose-derived stem cells
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P37/00—Drugs for immunological or allergic disorders
-
- 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
- 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
- C12N15/11—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
- C12N15/1137—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 against enzymes
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
- C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
- C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
- C12N15/86—Viral vectors
-
- 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
- C12N2310/11—Antisense
-
- 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
- C12N2310/50—Physical structure
- C12N2310/53—Physical structure partially self-complementary or closed
- C12N2310/531—Stem-loop; Hairpin
-
- 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
- C12N2330/00—Production
- C12N2330/50—Biochemical production, i.e. in a transformed host cell
- C12N2330/51—Specially adapted vectors
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2740/00—Reverse transcribing RNA viruses
- C12N2740/00011—Details
- C12N2740/10011—Retroviridae
- C12N2740/15011—Lentivirus, not HIV, e.g. FIV, SIV
- C12N2740/15041—Use of virus, viral particle or viral elements as a vector
- C12N2740/15043—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2740/00—Reverse transcribing RNA viruses
- C12N2740/00011—Details
- C12N2740/10011—Retroviridae
- C12N2740/16011—Human Immunodeficiency Virus, HIV
- C12N2740/16041—Use of virus, viral particle or viral elements as a vector
- C12N2740/16043—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y204/00—Glycosyltransferases (2.4)
- C12Y204/02—Pentosyltransferases (2.4.2)
- C12Y204/02008—Hypoxanthine phosphoribosyltransferase (2.4.2.8)
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Genetics & Genomics (AREA)
- Engineering & Computer Science (AREA)
- Chemical & Material Sciences (AREA)
- Organic Chemistry (AREA)
- Biotechnology (AREA)
- Biomedical Technology (AREA)
- Zoology (AREA)
- General Health & Medical Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Molecular Biology (AREA)
- Wood Science & Technology (AREA)
- General Engineering & Computer Science (AREA)
- Medicinal Chemistry (AREA)
- Biophysics (AREA)
- Biochemistry (AREA)
- Virology (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Physics & Mathematics (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Immunology (AREA)
- Epidemiology (AREA)
- Cell Biology (AREA)
- Developmental Biology & Embryology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Hematology (AREA)
- Gastroenterology & Hepatology (AREA)
- Toxicology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201862784508P | 2018-12-23 | 2018-12-23 | |
| PCT/US2019/068233 WO2020139796A1 (en) | 2018-12-23 | 2019-12-23 | Haematopoietic stem cell-gene therapy for wiskott-aldrich syndrome |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| BR112021012240A2 true BR112021012240A2 (pt) | 2022-01-18 |
Family
ID=69326699
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| BR112021012240A BR112021012240A2 (pt) | 2018-12-23 | 2019-12-23 | Terapia de gene-célula-tronco hematopoiética para síndrome wiskott-aldrich |
Country Status (8)
| Country | Link |
|---|---|
| US (1) | US20210316013A1 (https=) |
| EP (1) | EP3897745A1 (https=) |
| JP (1) | JP2022514955A (https=) |
| KR (1) | KR102942447B1 (https=) |
| CN (1) | CN113518825A (https=) |
| AU (1) | AU2019417697A1 (https=) |
| BR (1) | BR112021012240A2 (https=) |
| WO (1) | WO2020139796A1 (https=) |
Families Citing this family (11)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN111093715A (zh) * | 2017-09-18 | 2020-05-01 | 儿童医院医疗中心 | 强绝缘子和其在基因递送中的用途 |
| EP4329822A1 (en) | 2021-04-26 | 2024-03-06 | CSL Behring L.L.C. | Lentiviral vectors useful for the treatment of disease |
| EP4402264A4 (en) * | 2021-09-16 | 2025-10-15 | Agency Science Tech & Res | MODULATION OF TJP1 EXPRESSION TO TREAT LIVER DISEASES |
| CN115838765A (zh) * | 2021-09-18 | 2023-03-24 | 济南赛尔生物科技股份有限公司 | 一种治疗异染性脑白质营养不良的慢病毒载体 |
| CN114990163A (zh) * | 2022-03-31 | 2022-09-02 | 中海峡(福建)细胞生物科技有限公司 | 用于干细胞基因修饰的慢病毒载体及其构建方法和应用 |
| WO2024171034A2 (en) * | 2023-02-13 | 2024-08-22 | Csl Behring Llc | Optimised polynucleotides |
| EP4720100A1 (en) | 2023-05-25 | 2026-04-08 | CSL Behring LLC | Modified vectors |
| WO2025077575A1 (zh) * | 2023-10-08 | 2025-04-17 | 上海齐鲁制药研究中心有限公司 | 截短的绝缘子及其用于重组蛋白的瞬时表达的用途 |
| WO2025215591A1 (en) | 2024-04-12 | 2025-10-16 | Csl Behring L.L.C. | Modified vectors for xla gene therapy |
| CN120536433A (zh) * | 2024-05-20 | 2025-08-26 | 上海本导基因技术有限公司 | 一种能够调节基因表达的调控元件及应用 |
| WO2026003694A1 (en) * | 2024-06-24 | 2026-01-02 | Csl Behring Llc | Methods of enveloped virus transduction |
Family Cites Families (27)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5958928A (en) | 1995-03-27 | 1999-09-28 | Chugai Seiyaku Kabushiki Kaisha | Pharmaceutical agents containing methotrexate derivative |
| US6013516A (en) | 1995-10-06 | 2000-01-11 | The Salk Institute For Biological Studies | Vector and method of use for nucleic acid delivery to non-dividing cells |
| EP1191097A1 (en) | 2000-09-21 | 2002-03-27 | Leids Universitair Medisch Centrum | Induction of exon skipping in eukaryotic cells |
| WO2005080581A2 (en) | 2004-02-17 | 2005-09-01 | University Of Florida Research Foundation, Inc. | Insulated herpesvirus-derived gene expression cassettes for sustained and regulatable gene expression |
| EP2933332A1 (en) | 2004-06-28 | 2015-10-21 | The University Of Western Australia | Antisense oligonucleotides for inducing exon skipping and methods of use thereof |
| MX2007010008A (es) * | 2005-02-16 | 2008-01-18 | Lentigen Corp | Vectores lentivirales y su uso. |
| WO2007098089A2 (en) | 2006-02-17 | 2007-08-30 | Novacea, Inc. | Treatment of hyperproliferative diseases with methotrexate n-oxide and analogs |
| CN103849629B (zh) | 2006-06-21 | 2017-06-09 | 尤尼克尔Ip股份有限公司 | 具有经修饰的用于在昆虫细胞中产生aav的aav‑rep78翻译起始密码子的载体 |
| BR122020021379B1 (pt) | 2008-10-24 | 2021-05-11 | Sarepta Therapeutics, Inc. | oligômero morfolino fosforodiamidato, composição que compreende o mesmo e uso do dito oligômero para tratar distrofia muscular |
| TR201816523T4 (tr) | 2009-11-12 | 2018-11-21 | Univ Western Australia | Patolojilerin tedavisine yönelik antisens moleküller ve yöntemler. |
| US20110294114A1 (en) | 2009-12-04 | 2011-12-01 | Cincinnati Children's Hospital Medical Center | Optimization of determinants for successful genetic correction of diseases, mediated by hematopoietic stem cells |
| DK2699247T3 (en) * | 2011-04-20 | 2018-06-14 | Univ California | PROCEDURE FOR COMBINED CONDITIONING AND CHEMOS SELECTION IN A SIMPLE CYCLE |
| US20130085139A1 (en) | 2011-10-04 | 2013-04-04 | Royal Holloway And Bedford New College | Oligomers |
| HUE054260T2 (hu) | 2012-09-06 | 2021-08-30 | Univ Chicago | Antiszensz polinukleotidok exon-ugrás indukálására és eljárások disztrófiák kezelésére |
| US20140329762A1 (en) | 2013-03-15 | 2014-11-06 | Sarepta Therapeutics, Inc. | Compositions for treating muscular dystrophy |
| US20140363454A1 (en) | 2013-06-06 | 2014-12-11 | Igenica Biotherapeutics, Inc. | Antibody-Drug Conjugates, Compositions and Methods of Use |
| MX374532B (es) * | 2013-06-17 | 2025-03-06 | Broad Inst Inc | Suministro, uso y aplicaciones terapéuticas de los sistemas y composiciones crispr-cas, para actuar sobre trastornos y enfermedades utilizando componentes víricos. |
| JP7103750B2 (ja) * | 2013-12-12 | 2022-07-20 | ザ・ブロード・インスティテュート・インコーポレイテッド | ゲノム編集のためのＣＲＩＳＰＲ－Ｃａｓ系及び組成物の送達、使用及び治療適用 |
| CN104805120A (zh) * | 2014-01-27 | 2015-07-29 | 苟德明 | 一种shRNA-Ago2共表达慢病毒RNAi载体、重组质粒及其构建方法 |
| PL3134432T3 (pl) | 2014-04-25 | 2020-10-19 | Bluebird Bio, Inc. | Promotor mnd chimeryczne receptory antygenowe |
| PL3294893T3 (pl) | 2015-05-13 | 2024-04-29 | Csl Behring Gene Therapy, Inc. | Bioprodukcja wektorów lentiwirusowych |
| HK1247963A1 (zh) | 2015-05-18 | 2018-10-05 | Csl贝林基因治疗股份有限公司 | 用於hiv的治疗的基因疗法及其用途 |
| CN108495640B (zh) * | 2016-02-19 | 2022-11-04 | 加利福尼亚大学董事会 | 短发夹rna(shrna734)及其用于阳性选择和消除遗传修饰的细胞的用途 |
| CN109641051A (zh) | 2016-06-17 | 2019-04-16 | 美真达治疗公司 | 用于耗尽细胞的组合物和方法 |
| IL263744B2 (en) | 2016-06-17 | 2023-11-01 | Magenta Therapeutics Inc | Compositions and methods for the depletion of cd117 plus cells |
| CN111164211B (zh) * | 2017-07-18 | 2024-08-02 | 杰特贝林基因治疗股份有限公司 | 用于治疗β-血红蛋白病的组合物和方法 |
| JP7470119B2 (ja) * | 2018-08-24 | 2024-04-17 | シーエスエル ベーリング ジーン セラピー インコーポレイテッド | 無血清培地におけるベクター産生 |
-
2019
- 2019-12-23 EP EP19842992.0A patent/EP3897745A1/en active Pending
- 2019-12-23 JP JP2021536371A patent/JP2022514955A/ja active Pending
- 2019-12-23 KR KR1020217023216A patent/KR102942447B1/ko active Active
- 2019-12-23 BR BR112021012240A patent/BR112021012240A2/pt unknown
- 2019-12-23 WO PCT/US2019/068233 patent/WO2020139796A1/en not_active Ceased
- 2019-12-23 AU AU2019417697A patent/AU2019417697A1/en active Pending
- 2019-12-23 CN CN201980092125.2A patent/CN113518825A/zh active Pending
-
2021
- 2021-06-21 US US17/353,586 patent/US20210316013A1/en active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| WO2020139796A1 (en) | 2020-07-02 |
| KR102942447B1 (ko) | 2026-03-24 |
| AU2019417697A1 (en) | 2021-07-08 |
| US20210316013A1 (en) | 2021-10-14 |
| JP2022514955A (ja) | 2022-02-16 |
| CN113518825A (zh) | 2021-10-19 |
| EP3897745A1 (en) | 2021-10-27 |
| KR20210118402A (ko) | 2021-09-30 |
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| BR112021012240A2 (pt) | Terapia de gene-célula-tronco hematopoiética para síndrome wiskott-aldrich | |
| MX2022006427A (es) | "construcciones para terapia génica con microdistrofina y uso de las mismas. | |
| MX2020000676A (es) | Composiciones y metodos para tratar beta-hemoglobinopatias. | |
| CY1125385T1 (el) | Συστημα πολλαπλων φορεων και χρησεις αυτου | |
| BR122021025194A8 (pt) | Molécula de ácido nucleico, método para obter rna, rna, método para obter um peptídeo ou proteína e usos in vitro ou ex vivo do rna | |
| MX2022007858A (es) | Polipeptidos efectores crispr-cas y metodos de uso de estos. | |
| BR112016018598A2 (pt) | vetor de vírus adeno-associados | |
| MX2020004149A (es) | Variantes de desoxirribonucleasa (dnasa). | |
| BR112017028205A2 (pt) | molécula de rna, vetor, célula hospedeira, composição farmacêutica, kit, e, uso de uma ou mais utrs. | |
| BR112018074325A2 (pt) | anticorpo para ligação a receptor de interleucina 4 | |
| PH12018502645A1 (en) | Optimized mini-dystrophin genes and expression cassettes and their use | |
| SA517390590B1 (ar) | Ix عامل معدل، وتركيبات وطرق واستخدامات لنقل الجين إلى الخلايا، والأعضاء والأنسجة | |
| BR112019001887A2 (pt) | composições e métodos para o tratamento de doença associada a cep290 | |
| CY1119928T1 (el) | Διμερης λειτουργικη συμπληρωματικοτητα επαγομενη απο διττο αντιγονο | |
| EA201990861A1 (ru) | Рнк-направляемые модифицирующие нуклеиновые кислоты ферменты и способы их применения | |
| BR112018010639A2 (pt) | método de transfecção transitória para produção retroviral. | |
| MX2017007105A (es) | Variantes de lipasa y polinucleotidos que las codifican. | |
| MX2018007485A (es) | Variantes de enzimas y polinucleotidos que las codifican. | |
| BR112018011059A2 (pt) | synp161, um promotor para a expressão específica de genes em fotoreceptores de haste | |
| AR114540A1 (es) | VARIANTE DE ARNi CONTRA a-SINUCLEÍNA | |
| EA202290001A1 (ru) | Векторы aav с промотором миелинового белка ноль и их применение для лечения заболеваний, связанных со шванновскими клетками, таких как болезнь шарко-мари-тута | |
| BR112021016783A2 (pt) | Composições e métodos para tratar laminopatias | |
| CL2018000164A1 (es) | Vector recombinante del virus orf. | |
| MX2021005435A (es) | Transgen abcb11 con codones optimizados para el tratamiento de colestasis intrahepatica familiar progresiva tipo 2 (pfic2). | |
| CO2020000231A2 (es) | Composiciones inmunogénicas que comprenden cea muc1 y tert |