JP2024510972A - Ｘ連鎖性ミオチュブラーミオパチーを処置するための組成物及び方法 - Google Patents

Ｘ連鎖性ミオチュブラーミオパチーを処置するための組成物及び方法 Download PDF

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JP2024510972A
JP2024510972A JP2023555441A JP2023555441A JP2024510972A JP 2024510972 A JP2024510972 A JP 2024510972A JP 2023555441 A JP2023555441 A JP 2023555441A JP 2023555441 A JP2023555441 A JP 2023555441A JP 2024510972 A JP2024510972 A JP 2024510972A
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mmhg
respiratory
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trial
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JP2024510972A5 (https=
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ペレス，ジョバニー
グラハム，ロバート
リコ，サルバドール
プラサド，スヤシュ
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アステラス ジーン セラピーズ，インコーポレーテッド
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    • 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
    • A61M—DEVICES FOR INTRODUCING MEDIA INTO, OR ONTO, THE BODY; DEVICES FOR TRANSDUCING BODY MEDIA OR FOR TAKING MEDIA FROM THE BODY; DEVICES FOR PRODUCING OR ENDING SLEEP OR STUPOR
    • A61M16/00—Devices for influencing the respiratory system of patients by gas treatment, e.g. ventilators; Tracheal tubes
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61M—DEVICES FOR INTRODUCING MEDIA INTO, OR ONTO, THE BODY; DEVICES FOR TRANSDUCING BODY MEDIA OR FOR TAKING MEDIA FROM THE BODY; DEVICES FOR PRODUCING OR ENDING SLEEP OR STUPOR
    • A61M16/00—Devices for influencing the respiratory system of patients by gas treatment, e.g. ventilators; Tracheal tubes
    • A61M16/021—Devices for influencing the respiratory system of patients by gas treatment, e.g. ventilators; Tracheal tubes operated by electrical means
    • A61M16/022—Control means therefor
    • A61M16/024—Control means therefor including calculation means, e.g. using a processor
    • 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
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
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    • 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
    • C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4716—Muscle proteins, e.g. myosin, actin
    • 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
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    • A—HUMAN NECESSITIES
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    • A61M16/00—Devices for influencing the respiratory system of patients by gas treatment, e.g. ventilators; Tracheal tubes
    • A61M16/0003—Accessories therefor, e.g. sensors, vibrators, negative pressure
    • A61M2016/0027—Accessories therefor, e.g. sensors, vibrators, negative pressure pressure meter
    • A—HUMAN NECESSITIES
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    • A61M2205/00—General characteristics of the apparatus
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    • A61M2205/00—General characteristics of the apparatus
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    • A61M2205/3327—Measuring
    • A—HUMAN NECESSITIES
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    • A61M2230/00—Measuring parameters of the user
    • A61M2230/20—Blood composition characteristics
    • A—HUMAN NECESSITIES
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    • A61M2230/00—Measuring parameters of the user
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    • A61M2230/202—Blood composition characteristics partial carbon oxide pressure, e.g. partial dioxide pressure (P-CO2)
    • A—HUMAN NECESSITIES
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    • A61M2230/20—Blood composition characteristics
    • A61M2230/205—Blood composition characteristics partial oxygen pressure (P-O2)
    • A—HUMAN NECESSITIES
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    • A61M2230/00—Measuring parameters of the user
    • A61M2230/40—Respiratory characteristics
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    • A61M—DEVICES FOR INTRODUCING MEDIA INTO, OR ONTO, THE BODY; DEVICES FOR TRANSDUCING BODY MEDIA OR FOR TAKING MEDIA FROM THE BODY; DEVICES FOR PRODUCING OR ENDING SLEEP OR STUPOR
    • A61M2230/00—Measuring parameters of the user
    • A61M2230/40—Respiratory characteristics
    • A61M2230/43—Composition of exhalation
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    • A61M2230/00—Measuring parameters of the user
    • A61M2230/40—Respiratory characteristics
    • A61M2230/43—Composition of exhalation
    • A61M2230/432—Composition of exhalation partial CO2 pressure (P-CO2)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61M—DEVICES FOR INTRODUCING MEDIA INTO, OR ONTO, THE BODY; DEVICES FOR TRANSDUCING BODY MEDIA OR FOR TAKING MEDIA FROM THE BODY; DEVICES FOR PRODUCING OR ENDING SLEEP OR STUPOR
    • A61M2230/00—Measuring parameters of the user
    • A61M2230/63—Motion, e.g. physical activity
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

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  • Animal Behavior & Ethology (AREA)
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  • Biotechnology (AREA)
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  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Orthopedic Medicine & Surgery (AREA)
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  • Chemical Kinetics & Catalysis (AREA)
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  • Proteomics, Peptides & Aminoacids (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
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  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
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JP2023555441A 2021-03-11 2022-03-11 Ｘ連鎖性ミオチュブラーミオパチーを処置するための組成物及び方法 Pending JP2024510972A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202163159898P 2021-03-11 2021-03-11
US63/159,898 2021-03-11
PCT/US2022/019876 WO2022192622A1 (en) 2021-03-11 2022-03-11 Compositions and methods for treating x-linked myotubular myopathy

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JP2024510972A true JP2024510972A (ja) 2024-03-12
JP2024510972A5 JP2024510972A5 (https=) 2025-03-19
JPWO2022192622A5 JPWO2022192622A5 (https=) 2025-03-19

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US (1) US20240148906A1 (https=)
EP (1) EP4304664A4 (https=)
JP (1) JP2024510972A (https=)
CN (1) CN117255698A (https=)
AR (1) AR125077A1 (https=)
TW (1) TW202302156A (https=)
WO (1) WO2022192622A1 (https=)

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2012531246A (ja) * 2009-06-24 2012-12-10 オリディオン メディカル １９８７ リミテッド 人工呼吸から離脱させる統合肺指標
US20140249211A1 (en) * 2013-03-01 2014-09-04 Wake Forest University Health Sciences Systemic Gene Replacement Therapy for Treatment of X-Linked MyoTubular Myopathy (XLMTM)

Family Cites Families (21)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
SE9502543D0 (sv) * 1995-07-10 1995-07-10 Lachmann Burkhardt Artificial ventilation system
US6148814A (en) * 1996-02-08 2000-11-21 Ihc Health Services, Inc Method and system for patient monitoring and respiratory assistance control through mechanical ventilation by the use of deterministic protocols
US6804656B1 (en) * 1999-06-23 2004-10-12 Visicu, Inc. System and method for providing continuous, expert network critical care services from a remote location(s)
US7246618B2 (en) * 2001-06-21 2007-07-24 Nader Maher Habashi Ventilation method and control of a ventilator based on same
US8695593B2 (en) * 2007-03-31 2014-04-15 Fleur T. Tehrani Weaning and decision support system for mechanical ventilation
US20080295839A1 (en) * 2007-06-01 2008-12-04 Habashi Nader M Ventilator Apparatus and System of Ventilation
US8789529B2 (en) * 2009-08-20 2014-07-29 Covidien Lp Method for ventilation
US20160199608A1 (en) * 2010-02-18 2016-07-14 Orlando Morejon System and method of expediting weaning from ventilator support including an artificial airway cleaning apparatus
US20110213215A1 (en) * 2010-02-26 2011-09-01 Nellcor Puritan Bennett Llc Spontaneous Breathing Trial Manager
US20180280646A1 (en) * 2010-08-13 2018-10-04 Respiratory Motion, Inc. Respiratory volume monitor and ventilator
US9327090B2 (en) * 2012-06-29 2016-05-03 Carefusion 303, Inc. Respiratory knowledge portal
US20140060540A1 (en) * 2012-09-06 2014-03-06 Nellcor Puritan Bennett Llc Method and system for automated spontaneous breathing trial
US20140150796A1 (en) * 2012-11-30 2014-06-05 Covidien Lp System and method for detecting minimal ventilation support with proportional assist ventilation plus software and remote monitoring
US20140235959A1 (en) * 2013-02-15 2014-08-21 Covidien Lp Methods and algorithms for supervisory closed-loop determination of optimized scheduling of ventilator weaning trials
WO2018035311A1 (en) * 2016-08-19 2018-02-22 Gray John T Compositions and methods for modulating gene expression using reading frame surveillance
FI128346B (en) * 2018-11-19 2020-03-31 Hapella Oy A device for taking care of respiratory well-being and for practicing and improving respiratory function
US11166862B2 (en) * 2018-12-06 2021-11-09 General Electric Company System and method for a thermoregulated environment
WO2021110576A1 (en) * 2019-12-02 2021-06-10 Koninklijke Philips N.V. Providing information for assisting in the treatment of a ventilated and sedated patient
CN115052515B (zh) * 2019-12-05 2026-01-30 迪萨蒂医疗公司 用于确定患者呼吸时所付出的呼吸努力程度的系统和方法
WO2021142336A1 (en) * 2020-01-08 2021-07-15 Puretech Lyt, Inc. Vesicle compositions for oral delivery
EP4351725A4 (en) * 2021-05-24 2025-04-23 Astellas Gene Therapies, Inc. Compositions and methods for improved treatment of cross-linked myotube myopathy

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2012531246A (ja) * 2009-06-24 2012-12-10 オリディオン メディカル １９８７ リミテッド 人工呼吸から離脱させる統合肺指標
US20140249211A1 (en) * 2013-03-01 2014-09-04 Wake Forest University Health Sciences Systemic Gene Replacement Therapy for Treatment of X-Linked MyoTubular Myopathy (XLMTM)

Non-Patent Citations (3)

* Cited by examiner, † Cited by third party
Title
ANNOUSSAMY, M.ら: "X-linked myotubular myopathy: A prospective international natural history study", NEUROLOGY, vol. 92, 16, JPN6026008666, 16 April 2019 (2019-04-16), pages 1852 - 1867, ISSN: 0005813117 *
DUONG, T.ら: "Use of the Children’s Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP INTEND)", JOURNAL OF NEUROMUSCULAR DISEASES, vol. 8, 1, JPN6026008667, 9 September 2020 (2020-09-09), pages 63 - 77, ISSN: 0005813118 *
JOHNSTON, C.ら: "Weaning and Extubation in Pediatrics", CURRENT RESPIRATORY MEDICINE REVIEWS, vol. 8, 1, JPN6026008664, 1 February 2012 (2012-02-01), pages 68 - 78, ISSN: 0005813116 *

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AR125077A1 (es) 2023-06-07
WO2022192622A1 (en) 2022-09-15
US20240148906A1 (en) 2024-05-09
EP4304664A4 (en) 2025-07-30
TW202302156A (zh) 2023-01-16
EP4304664A1 (en) 2024-01-17
CN117255698A (zh) 2023-12-19

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