KR102640198B1 - Actrii 리간드 트랩을 사용한 베타-탈라세미아의 치료 - Google Patents

Actrii 리간드 트랩을 사용한 베타-탈라세미아의 치료 Download PDF

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KR102640198B1
KR102640198B1 KR1020177035838A KR20177035838A KR102640198B1 KR 102640198 B1 KR102640198 B1 KR 102640198B1 KR 1020177035838 A KR1020177035838 A KR 1020177035838A KR 20177035838 A KR20177035838 A KR 20177035838A KR 102640198 B1 KR102640198 B1 KR 102640198B1
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certain embodiments
actriib
delete delete
signaling inhibitor
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KR20180006437A (ko
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케네스 엠. 아티에
아브데라만 라아뎀
라제쉬 쵸프라
제이 백스트롬
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셀진 코포레이션
악셀레론 파마 인코포레이티드
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/177—Receptors; Cell surface antigens; Cell surface determinants
    • A61K38/179—Receptors; Cell surface antigens; Cell surface determinants for growth factors; for growth regulators
    • 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
    • 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
    • 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
    • A61P7/06—Antianaemics
    • 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
    • C07K14/475—Growth factors; Growth regulators
    • C07K14/495—Transforming growth factor [TGF]
    • 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
    • C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
    • C07K14/71—Receptors; Cell surface antigens; Cell surface determinants for growth factors; for growth regulators
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/30—Non-immunoglobulin-derived peptide or protein having an immunoglobulin constant or Fc region, or a fragment thereof, attached thereto

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Zoology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Immunology (AREA)
  • Cell Biology (AREA)
  • Biochemistry (AREA)
  • Molecular Biology (AREA)
  • Genetics & Genomics (AREA)
  • Toxicology (AREA)
  • Biophysics (AREA)
  • Epidemiology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Diabetes (AREA)
  • Hematology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Dermatology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Peptides Or Proteins (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
KR1020177035838A 2015-05-13 2016-05-12 Actrii 리간드 트랩을 사용한 베타-탈라세미아의 치료 Active KR102640198B1 (ko)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US201562161136P 2015-05-13 2015-05-13
US62/161,136 2015-05-13
US201562173836P 2015-06-10 2015-06-10
US62/173,836 2015-06-10
US201562243457P 2015-10-19 2015-10-19
US62/243,457 2015-10-19
PCT/US2016/031999 WO2016183280A1 (en) 2015-05-13 2016-05-12 Treatment of beta-thalassemia using actrii ligand traps

Publications (2)

Publication Number Publication Date
KR20180006437A KR20180006437A (ko) 2018-01-17
KR102640198B1 true KR102640198B1 (ko) 2024-02-23

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KR1020177035838A Active KR102640198B1 (ko) 2015-05-13 2016-05-12 Actrii 리간드 트랩을 사용한 베타-탈라세미아의 치료

Country Status (15)

Country Link
US (1) US20180125928A1 (https=)
EP (1) EP3294320A4 (https=)
JP (2) JP6976859B2 (https=)
KR (1) KR102640198B1 (https=)
CN (1) CN107847562A (https=)
AU (2) AU2016261913B2 (https=)
CA (1) CA2985777A1 (https=)
HK (1) HK1251157A1 (https=)
IL (2) IL284686B2 (https=)
JO (1) JOP20160092B1 (https=)
MY (1) MY189601A (https=)
PH (1) PH12017502079A1 (https=)
TN (1) TN2017000468A1 (https=)
TW (2) TWI814187B (https=)
WO (1) WO2016183280A1 (https=)

Families Citing this family (38)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR20190006086A (ko) 2005-11-23 2019-01-16 악셀레론 파마 인코포레이티드 액티빈-ａｃｔｒⅱａ 길항제 및 골 성장을 촉진하기 위한 이들의 용도
US8895016B2 (en) 2006-12-18 2014-11-25 Acceleron Pharma, Inc. Antagonists of activin-actriia and uses for increasing red blood cell levels
US8216997B2 (en) 2008-08-14 2012-07-10 Acceleron Pharma, Inc. Methods for increasing red blood cell levels and treating anemia using a combination of GDF traps and erythropoietin receptor activators
SMT202300222T1 (it) 2008-08-14 2023-09-06 Acceleron Pharma Inc Trappole di gdf
BRPI1010587A2 (pt) 2009-06-08 2019-04-09 Acceleron Pharma Inc. métodos para aumentar adipócitos termogênicos
EP2440577A4 (en) 2009-06-12 2013-01-23 Acceleron Pharma Inc SHORTEN ACTRIIB FC FUSION PROTEINS
JP6267425B2 (ja) 2009-11-17 2018-01-24 アクセルロン ファーマ， インコーポレイテッド 筋ジストロフィー治療のためのユートロフィン誘導に関するａｃｔｒｉｉｂタンパク質およびその改変体およびその使用
US9809636B2 (en) 2012-04-06 2017-11-07 Acceleron Pharma Inc. Methods for increasing red blood cell levels comprising administering BMP9
NZ707477A (en) 2012-11-02 2019-09-27 Celgene Corp Activin-actrii antagonists and uses for treating bone and other disorders
WO2015192111A1 (en) 2014-06-13 2015-12-17 Acceleron Pharma, Inc. Methods and compositions for treating ulcers
MA41052A (fr) 2014-10-09 2017-08-15 Celgene Corp Traitement d'une maladie cardiovasculaire à l'aide de pièges de ligands d'actrii
MA41119A (fr) 2014-12-03 2017-10-10 Acceleron Pharma Inc Méthodes de traitement de syndromes myélodysplasiques et d'anémie sidéroblastique
JP2018501307A (ja) 2014-12-03 2018-01-18 セルジーン コーポレイション アクチビン−ＡｃｔＲＩＩアンタゴニスト及び貧血を治療するための使用
MA42160A (fr) 2015-05-20 2018-03-28 Maria Cappellini Procédés de culture de cellules in vitro pour la beta-thalassemie a l'aide de pieges a ligands du recepteur de l'activine de type ii
US11123430B2 (en) 2015-11-04 2021-09-21 Acceleron Pharma Inc. Methods for increasing red blood cell levels and treating ineffective erythropoiesis
CA3005975A1 (en) 2015-11-23 2017-06-01 Acceleron Pharma Inc. Methods for treating eye disorders
HRP20210694T1 (hr) 2016-07-15 2021-09-17 Acceleron Pharma, Inc. Pripravci koji sadrže actriia polipeptide za uporabu za liječenje plućne hipertenzije
PL3490582T3 (pl) 2016-07-27 2024-09-23 Acceleron Pharma Inc. Kompozycje do zastosowania w leczeniu mielofibrozy
CN110430890B (zh) 2016-11-10 2024-09-10 科乐斯疗法公司 激活素受体iia型变体及其使用方法
HRP20241477T1 (hr) * 2017-06-14 2025-01-03 Celgene Corporation Postupci liječenja mijeloproliferativne neoplazme povezane s mijelofibrozom i anemijom
EP3706777B1 (en) 2017-11-09 2024-05-22 Keros Therapeutics, Inc. Activin receptor type iia variants and methods of use thereof
IL320014A (en) 2018-01-12 2025-06-01 Keros Therapeutics Inc Activin receptor type iib variants and methods of use thereof
US12263205B2 (en) 2018-04-30 2025-04-01 The Children's Hospital Of Philadelphia Methods of improving anemias by combining agents
JP7405772B2 (ja) 2018-05-09 2023-12-26 ケロス セラピューティクス インコーポレイテッド アクチビンｉｉａ型受容体変異体および同変異体を含む医薬組成物
US20210346464A1 (en) * 2018-10-31 2021-11-11 Celgene Corporation TREATMENT OF ANEMIA DUE TO VERY LOW, LOW, OR INTERMEDIATE RISK MYELODYSPLASTIC SYNDROMES IN SUBJECTS WITH RING SIDEROBLASTS USING ACTIVIN-ACTRll LIGAND TRAPS
EP3999059A1 (en) 2019-07-19 2022-05-25 Vifor (International) Ag Ferroportin-inhibitors for the use in the treatment of transfusion-dependent beta-thalassemia (tdt)
CN112924684B (zh) * 2019-12-05 2022-07-29 中国科学院大连化学物理研究所 用于区分抑郁症和非抑郁症的生物标志物和包括其的诊断试剂盒
IL296394A (en) 2020-03-20 2022-11-01 Keros Therapeutics Inc Type ii activin receptor chimeras and methods of using them
WO2021189010A1 (en) 2020-03-20 2021-09-23 Keros Therapeutics, Inc. Methods of using activin receptor type iib variants
WO2021211418A1 (en) * 2020-04-13 2021-10-21 Celgene Corporation Methods for treating anemia using an actriib ligand trap and fedratinib
EP4142769A4 (en) 2020-04-28 2024-05-15 Acceleron Pharma Inc. ACTRII PROTEINS AND USE IN THE TREATMENT OF POSTCAPILLARY PULMONARY HYPERTENSION
US12186370B1 (en) 2020-11-05 2025-01-07 Celgene Corporation ACTRIIB ligand trap compositions and uses thereof
EP4240399A4 (en) * 2020-11-06 2024-10-09 Acceleron Pharma Inc. FORMULATIONS WITH ACTRII POLYPEPTIDE VARIANTS
EP4274600A4 (en) * 2021-01-08 2024-11-20 Acceleron Pharma Inc. COMPOSITIONS AND METHODS FOR THE TREATMENT OF PULMONARY HYPERTENSION
US20250268878A1 (en) 2021-01-20 2025-08-28 Vifor (International) Ag Ferroportin-inhibitors for the use in the treatment of myelodysplastic syndromes (mds)
EP4731218A1 (en) 2023-06-26 2026-04-29 Vifor (International) AG Ferroportin-inhibitors for the use in the treatment of hereditary haemochromatosis (hh)
WO2025122830A1 (en) * 2023-12-08 2025-06-12 Keros Therapeutics, Inc. Methods of using activin receptor type ii signaling inhibitors
WO2026041650A1 (en) 2024-08-20 2026-02-26 Vifor (International) Ag Ferroportin-inhibitors for the use in the treatment of lupus nephritis

Family Cites Families (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4247A (en) * 1845-11-01 Jordan l
US6231880B1 (en) * 1997-05-30 2001-05-15 Susan P. Perrine Compositions and administration of compositions for the treatment of blood disorders
DK2124999T3 (da) * 2006-12-18 2013-01-14 Acceleron Pharma Inc Activin-actrii antagonister og anvendelser til behandling af anæmi
AU2009262970A1 (en) * 2008-06-26 2009-12-30 Acceleron Pharma Inc. Methods for dosing an activin-ActRIIa antagonist and monitoring of treated patients
SMT202300222T1 (it) * 2008-08-14 2023-09-06 Acceleron Pharma Inc Trappole di gdf
BR112012005225B8 (pt) * 2009-09-09 2023-01-10 Acceleron Pharma Inc Uso de uma proteína de fusão actriib-fc para o tratamento de um transtorno relacionado ao osso ou associado à perda de músculo por crescimento muscular insuficiente
BR112014009528B1 (pt) * 2011-10-17 2020-12-29 Acceleron Pharma, Inc. usos de polipeptídeos actriib para a fabricação de um medicamento para tratar eritropoiese ineficaz
EA201491578A1 (ru) * 2012-03-30 2015-04-30 Шир Хьюман Дженетик Терапис, Инк. Подкожное введение идуронат-2-сульфатазы
AU2013334660B2 (en) * 2012-10-24 2018-08-09 Celgene Corporation Methods for treating anemia
CN104968801B (zh) * 2012-10-24 2021-06-15 细胞基因公司 用于治疗贫血症的生物标志物
SG11201504038XA (en) * 2012-11-27 2015-06-29 Childrens Medical Center Targeting bcl11a distal regulatory elements for fetal hemoglobin reinduction

Also Published As

Publication number Publication date
TN2017000468A1 (en) 2019-04-12
CN107847562A (zh) 2018-03-27
EP3294320A4 (en) 2018-12-26
IL284686B2 (en) 2023-05-01
AU2021258087A1 (en) 2021-11-25
CA2985777A1 (en) 2016-11-17
TW201709927A (zh) 2017-03-16
TWI814187B (zh) 2023-09-01
IL284686A (en) 2021-08-31
NZ737043A (en) 2025-02-28
PH12017502079A1 (en) 2018-06-11
JOP20160092B1 (ar) 2023-03-28
AU2016261913A1 (en) 2017-11-30
JP6976859B2 (ja) 2021-12-08
AU2016261913B2 (en) 2021-08-12
JP2018520094A (ja) 2018-07-26
US20180125928A1 (en) 2018-05-10
AU2021258087B2 (en) 2023-04-27
WO2016183280A1 (en) 2016-11-17
IL284686B (en) 2023-01-01
HK1251157A1 (zh) 2019-01-25
MY189601A (en) 2022-02-18
IL255527B (en) 2021-07-29
IL255527A (en) 2018-01-31
TW202231294A (zh) 2022-08-16
KR20180006437A (ko) 2018-01-17
TWI762444B (zh) 2022-05-01
JP2021191755A (ja) 2021-12-16
EP3294320A1 (en) 2018-03-21

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