MX2020003351A - Metodos, composiciones y elementos implantables que comprenden celulas activas. - Google Patents

Metodos, composiciones y elementos implantables que comprenden celulas activas.

Info

Publication number
MX2020003351A
MX2020003351A MX2020003351A MX2020003351A MX2020003351A MX 2020003351 A MX2020003351 A MX 2020003351A MX 2020003351 A MX2020003351 A MX 2020003351A MX 2020003351 A MX2020003351 A MX 2020003351A MX 2020003351 A MX2020003351 A MX 2020003351A
Authority
MX
Mexico
Prior art keywords
compositions
methods
implantable elements
active cells
active cell
Prior art date
Application number
MX2020003351A
Other languages
English (en)
Inventor
Omid Veiseh
Richard Heidebrecht
Paul Kevin Wotton
Robert James Miller
Guillaume Carmona
Francisco Caballerro Gonzalez
Matthias Alexander Oberli
David Peritt
Devyn Mckinley Smith
Jered A Sewell
Original Assignee
Sigilon 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 Sigilon Therapeutics Inc filed Critical Sigilon Therapeutics Inc
Publication of MX2020003351A publication Critical patent/MX2020003351A/es

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    • 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
    • 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
    • A61K35/30—Nerves; Brain; Eyes; Corneal cells; Cerebrospinal fluid; Neuronal stem cells; Neuronal precursor cells; Glial cells; Oligodendrocytes; Schwann cells; Astroglia; Astrocytes; Choroid plexus; Spinal cord tissue
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/54—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound
    • A61K47/545—Heterocyclic compounds
    • 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
    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • A61K9/0024—Solid, semi-solid or solidifying implants, which are implanted or injected in body tissue
    • 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
    • A61K9/4816—Wall or shell material
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P19/00—Drugs for skeletal disorders
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • A61P7/04—Antihaemorrhagics; Procoagulants; Haemostatic agents; Antifibrinolytic agents
    • 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
    • C07K14/52—Cytokines; Lymphokines; Interferons
    • C07K14/54—Interleukins [IL]
    • C07K14/55—IL-2
    • 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
    • C07K14/575—Hormones
    • C07K14/635—Parathyroid hormone, i.e. parathormone; Parathyroid hormone-related peptides
    • 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
    • C07K14/745—Blood coagulation or fibrinolysis factors
    • C07K14/755—Factors VIII, e.g. factor VIII C (AHF), factor VIII Ag (VWF)
    • 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
    • C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/06—Animal cells or tissues; Human cells or tissues
    • C12N5/0602—Vertebrate cells
    • C12N5/0618—Cells of the nervous system
    • C12N5/0621—Eye cells, e.g. cornea, iris pigmented cells
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2800/00—Nucleic acids vectors
    • C12N2800/22—Vectors comprising a coding region that has been codon optimised for expression in a respective host
    • 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
    • C12N2800/90—Vectors containing a transposable element
    • 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
    • C12N2999/00—Further aspects of viruses or vectors not covered by groups C12N2710/00 - C12N2796/00 or C12N2800/00

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Biomedical Technology (AREA)
  • Genetics & Genomics (AREA)
  • Medicinal Chemistry (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • Wood Science & Technology (AREA)
  • Molecular Biology (AREA)
  • Biophysics (AREA)
  • General Engineering & Computer Science (AREA)
  • Neurosurgery (AREA)
  • Epidemiology (AREA)
  • Cell Biology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Neurology (AREA)
  • Developmental Biology & Embryology (AREA)
  • Toxicology (AREA)
  • Microbiology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Ophthalmology & Optometry (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Endocrinology (AREA)
  • Hematology (AREA)
  • Immunology (AREA)
  • Virology (AREA)

Abstract

En el presente documento se describen composiciones celulares que comprenden una célula activa (por ejemplo, una célula activa modificada por ingeniería genética, por ejemplo, una célula RPE modificada por ingeniería genética), o derivados de las mismas, así como composiciones, productos farmacéuticos y elementos implantables que comprenden una célula activa, y métodos para fabricar y usar la misma. Las células y las composiciones pueden expresar un agente terapéutico útil para el tratamiento de una enfermedad, trastorno o afección descrita en el presente documento.
MX2020003351A 2017-09-27 2018-09-27 Metodos, composiciones y elementos implantables que comprenden celulas activas. MX2020003351A (es)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US201762563877P 2017-09-27 2017-09-27
US201862652882P 2018-04-04 2018-04-04
US201862652881P 2018-04-04 2018-04-04
PCT/US2018/053191 WO2019067766A1 (en) 2017-09-27 2018-09-27 METHODS, COMPOSITIONS AND IMPLANTABLE ELEMENTS COMPRISING ACTIVE CELLS

Publications (1)

Publication Number Publication Date
MX2020003351A true MX2020003351A (es) 2020-10-12

Family

ID=63878821

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2020003351A MX2020003351A (es) 2017-09-27 2018-09-27 Metodos, composiciones y elementos implantables que comprenden celulas activas.

Country Status (11)

Country Link
US (1) US20200263196A1 (es)
EP (1) EP3687580A1 (es)
JP (2) JP2020534837A (es)
KR (1) KR20200057051A (es)
CN (1) CN111372612A (es)
AU (2) AU2018338608C1 (es)
BR (1) BR112020006149A2 (es)
CA (1) CA3077380A1 (es)
IL (1) IL274200B2 (es)
MX (1) MX2020003351A (es)
WO (1) WO2019067766A1 (es)

Families Citing this family (46)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN105792775B (zh) 2013-09-24 2019-02-19 吉纳生命科学公司 用于细胞植入物的气体处理的系统
JP7626588B2 (ja) 2016-10-03 2025-02-04 シギロン セラピューティクス， インコーポレイテッド 化合物、デバイスおよびその使用
RU2019114817A (ru) 2016-11-15 2020-11-16 Джинер Лайф Сайенс, Инк. Устройство чрезкожной диффузии газов, подходящее для применения с подкожным имплантатом
WO2018093940A1 (en) 2016-11-15 2018-05-24 Giner, Inc. Self-regulating electrolytic gas generator and implant system comprising the same
JP7165717B2 (ja) 2017-03-15 2022-11-04 パンディオン・オペレーションズ・インコーポレイテッド 標的免疫寛容
WO2018204867A1 (en) 2017-05-04 2018-11-08 Giner, Inc. Robust, implantable gas delivery device and methods, systems and devices including same
AU2018273914A1 (en) 2017-05-24 2019-11-14 Pandion Operations, Inc. Targeted immunotolerance
BR112020002394A2 (pt) 2017-08-09 2020-07-28 Bioverativ Therapeutics Inc. moléculas de ácidos nucleicos e usos das mesmas
US10946068B2 (en) 2017-12-06 2021-03-16 Pandion Operations, Inc. IL-2 muteins and uses thereof
US10174091B1 (en) 2017-12-06 2019-01-08 Pandion Therapeutics, Inc. IL-2 muteins
USRE50550E1 (en) 2017-12-06 2025-08-26 Pandion Operations, Inc. IL-2 muteins and uses thereof
EP3759154A1 (en) * 2018-03-02 2021-01-06 Sigilon Therapeutics, Inc. Biocompatible hydrogel capsules and process for preparing same
AU2019226587A1 (en) 2018-03-02 2020-09-03 Sigilon Therapeutics, Inc. Afibrotic compounds, devices, and uses thereof
US20210145889A1 (en) * 2018-04-04 2021-05-20 Sigilon Therapeutics, Inc. Methods, compositions, and implantable elements comprising stem cells
WO2019195055A1 (en) 2018-04-04 2019-10-10 Sigilon Therapeutics, Inc. Implantable particles and related methods
WO2019222704A1 (en) 2018-05-17 2019-11-21 Giner Life Sciences, Inc. Electrolytic gas generator with combined lead and gas port terminals
KR102925832B1 (ko) 2018-08-09 2026-02-11 바이오버라티브 테라퓨틱스 인크. 핵산 분자 및 비바이러스 유전자 치료를 위한 이의 용도
US10842885B2 (en) 2018-08-20 2020-11-24 Ucl Business Ltd Factor IX encoding nucleotides
GB201813528D0 (en) 2018-08-20 2018-10-03 Ucl Business Plc Factor IX encoding nucleotides
UY38389A (es) * 2018-09-27 2020-04-30 Sigilon Therapeutics Inc Dispositivos implantables para terapia celular y métodos relacionados
KR20220023962A (ko) * 2019-03-27 2022-03-03 시질론 테라퓨틱스, 인크. 파브리병을 치료하기 위한 조성물, 디바이스 및 방법
CA3141327A1 (en) 2019-05-20 2020-11-26 Pandion Operations, Inc. Madcam targeted immunotolerance
EP3999647A1 (en) * 2019-07-18 2022-05-25 Lysogene Compositions and methods for the treatment of sanfilippo disease and other disorders
CN114269390A (zh) * 2019-08-08 2022-04-01 威廉马歇莱思大学 可植入构建体及其用途
CN114786667A (zh) * 2019-09-27 2022-07-22 西吉隆医疗股份有限公司 评估组合物中经小分子修饰的聚合物的方法
CN112575034B (zh) * 2019-09-29 2023-04-25 济南赛尔生物科技股份有限公司 一种治疗a型血友病的产品及应用
WO2021102271A1 (en) 2019-11-22 2021-05-27 Sigilon Therapeutics, Inc. Monoclonal cell lines expressing an exogenous substance and uses thereof
BR112022011321A2 (pt) * 2019-12-13 2022-08-23 Sigilon Therapeutics Inc Compostos, polímeros, dispositivos e seus usos
US12595462B2 (en) 2020-01-14 2026-04-07 William Marsh Rice University High throughput genetic barcoding and analysis methods
WO2021163242A1 (en) * 2020-02-11 2021-08-19 William Marsh Rice University Methods for improved delivery of therapeutic agents
WO2021168079A1 (en) 2020-02-21 2021-08-26 Pandion Operations, Inc. Tissue targeted immunotolerance with a cd39 effector
WO2021222654A1 (en) * 2020-04-29 2021-11-04 Saliogen Therapeutics, Inc. Compositions and methods for treatment of inherited macular degeneration
EP4189101A4 (en) * 2020-07-28 2024-09-04 Sigilon Therapeutics, Inc. GENETICALLY MODIFIED CELL LINES FOR EXPRESSING AN EXOGENOUS SUBSTANCE AND USES THEREOF
EP4274855A1 (en) 2021-01-11 2023-11-15 Adocia Hydrogels for cell therapy
CN117042723A (zh) * 2021-01-26 2023-11-10 西吉隆医疗股份有限公司 用于诱导抗原特异性免疫耐受的组合物、装置和方法
US20240209336A1 (en) * 2021-03-31 2024-06-27 Sigilon Therapeutics, Inc. Genetically modified human cell lines and uses thereof
WO2022260764A2 (en) * 2021-04-21 2022-12-15 Northwestern University Engineered cells for producing of therapeutic agents to be delivered by a hybrid bioelectronic device
CN113336841B (zh) * 2021-06-02 2022-09-23 中国医学科学院血液病医院(中国医学科学院血液学研究所) F8蛋白变体及利用其制备的基因治疗载体
AR126845A1 (es) * 2021-08-23 2023-11-22 Bioverativ Therapeutics Inc Producción de adn de extremo cerrado con secuencias repetidas de terminal invertida
CN115772226B (zh) * 2021-09-06 2025-09-30 上海怀越生物科技有限公司 一种滋养细胞、制备方法及其用途
MX2024005230A (es) * 2021-10-29 2024-08-27 Sigilon Therapeutics Inc Composiciones para terapias basadas en celulas y metodos relacionados.
CN114624224B (zh) * 2022-03-24 2025-11-14 中国人民解放军军事科学院军事医学研究院 一种超快速真菌捕获剂及其制备方法和用途
AU2023298424A1 (en) * 2022-07-01 2025-02-13 Sigilon Therapeutics, Inc. Covalently crosslinked polysaccharides and methods of use thereof
WO2024030950A2 (en) * 2022-08-03 2024-02-08 Sightstream Biotherapeutics, Inc. Compositions for the treatment of disease
WO2025038967A1 (en) * 2023-08-17 2025-02-20 William Marsh Rice University Devices for wound healing made from flowable materials
WO2025143742A1 (ko) * 2023-12-26 2025-07-03 삼성바이오에피스 주식회사 혈액응고인자 IX를 암호화하는 코돈-최적화된 폴리뉴클레오티드 및 이를 포함하는 rAAV 생산용 플라스미드

Family Cites Families (54)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU5772886A (en) 1985-04-12 1986-11-05 Genetics Institute Inc. Novel procoagulant proteins
KR910006424B1 (ko) 1985-08-21 1991-08-24 인코텍스 비.브이 편성브리프(brief) 제조방법
JP2525022B2 (ja) 1986-01-03 1996-08-14 ジェネティックス・インスチチュ−ト・インコ−ポレ−テッド ▲ＶＩＩＩ▼：ｃ因子型タンパク質の改良生産方法
DE3720246A1 (de) 1987-06-19 1988-12-29 Behringwerke Ag Faktor viii:c-aehnliches molekuel mit koagulationsaktivitaet
SE465222C5 (sv) 1989-12-15 1998-02-10 Pharmacia & Upjohn Ab Ett rekombinant, humant faktor VIII-derivat och förfarande för dess framställning
US7410948B2 (en) 1995-07-13 2008-08-12 Societe De Conseils De Recherches Et D'applications Scientifiques, Sas Analogs of parathyroid hormone
US7125841B2 (en) 2000-11-14 2006-10-24 The University Of Texas Systems Mutant human factor IX with an increased resistance to inhibition by heparin
IL157842A0 (en) 2001-03-22 2004-03-28 Novo Nordisk Healthcare Ag Coagulation factor vii derivatives
JP2005501547A (ja) 2001-09-04 2005-01-20 メルク　パテント　ゲゼルシャフト　ミット　ベシュレンクテル　ハフトング 修飾された第ｉｘ因子
DK2085470T3 (da) 2003-03-20 2012-08-06 Bayer Healthcare Llc FVII- eller FVIIa-varianter
ES2333598T5 (es) 2003-05-06 2013-09-04 Biogen Idec Hemophilia Inc Proteinas quimericas del factor de coagulacion fc para tratar la hemofilia.
US20080058255A1 (en) 2004-06-21 2008-03-06 Novo Nordisk Healthcare A/G Glycosylation-Disrupted Factor VII Variants
DK3896090T3 (da) 2006-06-14 2022-03-21 Csl Behring Gmbh Proteolytisk spalteligt fusionsprotein omfattende en blodkoagutionsfaktor
WO2007149406A2 (en) 2006-06-19 2007-12-27 Nautilus Technology Llc Modified coagulation factor ix polypeptides and use thereof for treatment
US7700734B2 (en) 2007-01-09 2010-04-20 Shu-Wha Lin Recombinant human factor IX and use thereof
EP2209487A4 (en) 2007-10-15 2012-06-20 Univ North Carolina Human factor ix variants with an extended half life
TWI465247B (zh) 2008-04-11 2014-12-21 Catalyst Biosciences Inc 經修飾的因子ｖｉｉ多肽和其用途
CA2721362A1 (en) 2008-04-16 2009-11-19 Bayer Healthcare Llc Site-directed modification of factor ix
MX2010011345A (es) 2008-04-16 2011-02-23 Bayer Healthcare Llc Polipeptidos modificados del factor ix y usos de los mismos.
JP5665733B2 (ja) 2008-04-21 2015-02-04 ノボ ノルディスク ヘルス ケア アーゲー 高度にグリコシル化されたヒト凝固第ｉｘ因子
WO2010005533A2 (en) * 2008-06-30 2010-01-14 The Johns Hopkins University Compositions and methods for the treatment of ocular oxidative stress and retinitis pigmentosa
EP2411038B1 (en) 2009-03-27 2016-12-28 Van Andel Research Institute Parathyroid hormone peptides and parathyroid hormone-related protein peptides and methods of use
WO2011020866A2 (en) 2009-08-20 2011-02-24 Csl Behring Gmbh Albumin fused coagulation factors for non-intravenous administration in the therapy and prophylactic treatment of bleeding disorders
DK2482841T4 (da) 2009-10-02 2022-10-24 Childrens Hospital Philadelphia Sammensætninger og fremgangsmåder til at forbedre koagulationsfaktor-viii-funktion
US8383417B2 (en) 2009-12-22 2013-02-26 Thermo Finnigan, Llc Assay for monitoring parathyroid hormone (PTH) variants by tandem mass spectrometry
PT3552619T (pt) 2010-07-09 2025-10-23 Bioverativ Therapeutics Inc Polipéptidos de fator ix e métodos de uso dos mesmos
WO2012058292A2 (en) 2010-10-26 2012-05-03 Arizona Board Of Regents Parathyroid hormone variants and assays related to disease
US9867781B2 (en) 2011-02-18 2018-01-16 Massachusetts Institute Of Technology Hydrogel encapsulated cells and anti-inflammatory drugs
JP5605969B2 (ja) 2011-05-10 2014-10-15 株式会社フジキン 流量モニタ付圧力式流量制御装置と、これを用いた流体供給系の異常検出方法並びにモニタ流量異常時の処置方法
EP4083074A1 (en) 2011-06-02 2022-11-02 Massachusetts Institute Of Technology Modified alginates for cell encapsulation and cell therapy
CA2850579A1 (en) 2011-10-18 2013-04-25 Carsten Horn Method for improving the stability of purified factor viii after reconstitution
WO2013120939A1 (en) 2012-02-15 2013-08-22 Csl Behring Gmbh Von willebrand factor variants having improved factor viii binding affinity
EP3404105A1 (en) 2012-07-06 2018-11-21 Bioverativ Therapeutics Inc. Cell line expressing single chain factor viii polypeptides and uses thereof
US9555007B2 (en) 2013-03-14 2017-01-31 Massachusetts Institute Of Technology Multi-layer hydrogel capsules for encapsulation of cells and cell aggregates
US10172791B2 (en) 2013-03-14 2019-01-08 Massachusetts Institute Of Technology Multi-layer hydrogel capsules for encapsulation of cells and cell aggregates
EP2881463A1 (en) 2013-12-09 2015-06-10 DRK-Blutspendedienst Baden-Württemberg-Hessen gGmbH Factor IX variants with clotting activity in absence of their cofactor and/or with increased F.IX clotting activity and their use for treating bleeding disorders
BR112016012129A8 (pt) * 2013-12-11 2020-05-12 Pfizer Ltd métodos para produção, expansão e purificação de células epiteliais do pigmento da retina, composição farmacêutica e uso das células
DK3628334T5 (da) * 2014-03-21 2024-09-02 Genzyme Corp Genterapi til behandling af retinitis pigmentosa
JP6277034B2 (ja) * 2014-03-26 2018-02-07 株式会社日本マイクロニクス 積層型二次電池
CA2956075C (en) 2014-08-01 2020-03-10 Massachusetts Institute Of Technology Modified alginates for anti-fibrotic materials and applications
SI3482766T1 (sl) 2014-08-11 2020-09-30 Delinia, Inc. Spremenjene variante IL-2, ki selektivno aktivirajo regulatorne T celice za zdravljenje avtoimunskih bolezni
GB2547179A (en) * 2015-10-26 2017-08-16 Quethera Ltd Genetic construct
WO2017075630A1 (en) 2015-11-01 2017-05-04 Massachusetts Institute Of Technology Materials with improved properties
CN116479001A (zh) * 2015-11-13 2023-07-25 武田药品工业株式会社 用于血友病a的基因治疗的具有增加的表达的编码重组fviii变体的病毒载体
GB201705484D0 (en) * 2017-04-05 2017-05-17 Quethera Ltd Genetic construct
US11492644B2 (en) * 2017-05-24 2022-11-08 Murdoch Childrens Research Institute Genetically induced nephron progenitors
EP3759154A1 (en) * 2018-03-02 2021-01-06 Sigilon Therapeutics, Inc. Biocompatible hydrogel capsules and process for preparing same
WO2019195055A1 (en) * 2018-04-04 2019-10-10 Sigilon Therapeutics, Inc. Implantable particles and related methods
UY38389A (es) * 2018-09-27 2020-04-30 Sigilon Therapeutics Inc Dispositivos implantables para terapia celular y métodos relacionados
US20220349896A1 (en) * 2019-09-27 2022-11-03 Sigilon Therapeutics, Inc. Methods of evaluating polypeptide-modified polymers in compositions
CN114786667A (zh) * 2019-09-27 2022-07-22 西吉隆医疗股份有限公司 评估组合物中经小分子修饰的聚合物的方法
WO2021102271A1 (en) * 2019-11-22 2021-05-27 Sigilon Therapeutics, Inc. Monoclonal cell lines expressing an exogenous substance and uses thereof
BR112022011321A2 (pt) * 2019-12-13 2022-08-23 Sigilon Therapeutics Inc Compostos, polímeros, dispositivos e seus usos
EP4189101A4 (en) * 2020-07-28 2024-09-04 Sigilon Therapeutics, Inc. GENETICALLY MODIFIED CELL LINES FOR EXPRESSING AN EXOGENOUS SUBSTANCE AND USES THEREOF

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