BR112013005673A2 - uso de lipase de ácido lisossomal para tratamento de deficiência de lipase de ácido lisossomal em pacientes - Google Patents
uso de lipase de ácido lisossomal para tratamento de deficiência de lipase de ácido lisossomal em pacientesInfo
- Publication number
- BR112013005673A2 BR112013005673A2 BR112013005673A BR112013005673A BR112013005673A2 BR 112013005673 A2 BR112013005673 A2 BR 112013005673A2 BR 112013005673 A BR112013005673 A BR 112013005673A BR 112013005673 A BR112013005673 A BR 112013005673A BR 112013005673 A2 BR112013005673 A2 BR 112013005673A2
- Authority
- BR
- Brazil
- Prior art keywords
- acid lipase
- lysosomal acid
- deficiency
- treatment
- lal
- Prior art date
Links
Classifications
-
- 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
- A61K38/43—Enzymes; Proenzymes; Derivatives thereof
- A61K38/46—Hydrolases (3)
- A61K38/465—Hydrolases (3) acting on ester bonds (3.1), e.g. lipases, ribonucleases
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/13—Amines
- A61K31/135—Amines having aromatic rings, e.g. ketamine, nortriptyline
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/13—Amines
- A61K31/135—Amines having aromatic rings, e.g. ketamine, nortriptyline
- A61K31/138—Aryloxyalkylamines, e.g. propranolol, tamoxifen, phenoxybenzamine
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/33—Heterocyclic compounds
- A61K31/335—Heterocyclic compounds having oxygen as the only ring hetero atom, e.g. fungichromin
- A61K31/365—Lactones
- A61K31/366—Lactones having six-membered rings, e.g. delta-lactones
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/33—Heterocyclic compounds
- A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
- A61K31/397—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having four-membered rings, e.g. azetidine
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
- A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
- A61P1/16—Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P3/00—Drugs for disorders of the metabolism
- A61P3/06—Antihyperlipidemics
-
- 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)
- C12N9/18—Carboxylic ester hydrolases (3.1.1)
-
- 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)
- C12N9/18—Carboxylic ester hydrolases (3.1.1)
- C12N9/20—Triglyceride splitting, e.g. by means of lipase
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y301/00—Hydrolases acting on ester bonds (3.1)
- C12Y301/01—Carboxylic ester hydrolases (3.1.1)
- C12Y301/01013—Sterol esterase (3.1.1.13)
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K2300/00—Mixtures or combinations of active ingredients, wherein at least one active ingredient is fully defined in groups A61K31/00 - A61K41/00
Abstract
uso de lipase de ácido lisossomal para tratamento de deficiência de lipase de ácido lissomalem pacientes. a presente invenção provê tratamento de deficiência de lal (por exemplo, doença de wolman, cesd) compreendendo administrar a um mamífero uma quantidade terapeuticamente eficaz de lipase de ácido lisossomal com uma frequência de dosagem eficaz. métodos de aperfeiçoamento de crescimento e função hepática, aumento da concentração de lal no tecido e aumento da atividade de lal em um paciente humano sofrendo de deficiênte de lal são também providos.
Priority Applications (1)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
BR122019021757-2A BR122019021757B1 (pt) | 2010-09-09 | 2011-09-09 | composição farmacêutica para tratamento de um paciente humano sofrendo de uma deficiência de lipase ácida lisossomal (lal) |
Applications Claiming Priority (9)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US40301110P | 2010-09-09 | 2010-09-09 | |
US61/403,011 | 2010-09-09 | ||
US45601410P | 2010-10-29 | 2010-10-29 | |
US61/456,014 | 2010-10-29 | ||
US201161432372P | 2011-01-13 | 2011-01-13 | |
US61/432,372 | 2011-01-13 | ||
PCT/US2011/033699 WO2011133960A2 (en) | 2010-04-23 | 2011-04-23 | Lysosomal storage disease enzyme |
USPCT/US2011/033699 | 2011-04-23 | ||
PCT/US2011/051096 WO2012050695A1 (en) | 2010-09-09 | 2011-09-09 | Use of lysosomal acid lipase for treating lysosomal acid lipase deficiency in patients |
Publications (2)
Publication Number | Publication Date |
---|---|
BR112013005673A2 true BR112013005673A2 (pt) | 2017-09-19 |
BR112013005673B1 BR112013005673B1 (pt) | 2020-12-15 |
Family
ID=45806910
Family Applications (2)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
BR112013005673-8A BR112013005673B1 (pt) | 2010-09-09 | 2011-09-09 | Uso de lipase ácida lisossomal (lal) humana recombinante no tratamento da deficiência de lipase ácida lisossomal |
BR122019021757-2A BR122019021757B1 (pt) | 2010-09-09 | 2011-09-09 | composição farmacêutica para tratamento de um paciente humano sofrendo de uma deficiência de lipase ácida lisossomal (lal) |
Family Applications After (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
BR122019021757-2A BR122019021757B1 (pt) | 2010-09-09 | 2011-09-09 | composição farmacêutica para tratamento de um paciente humano sofrendo de uma deficiência de lipase ácida lisossomal (lal) |
Country Status (30)
Country | Link |
---|---|
US (5) | US8663631B2 (pt) |
EP (3) | EP2977057B1 (pt) |
JP (3) | JP5693728B2 (pt) |
KR (1) | KR20150038636A (pt) |
CN (2) | CN103200958A (pt) |
AR (1) | AR082953A1 (pt) |
AU (1) | AU2011314293B2 (pt) |
BR (2) | BR112013005673B1 (pt) |
CA (2) | CA2810999C (pt) |
CL (1) | CL2013000664A1 (pt) |
CO (1) | CO6710913A2 (pt) |
CY (1) | CY1116544T1 (pt) |
DK (2) | DK2613798T4 (pt) |
ES (2) | ES2535605T5 (pt) |
HK (2) | HK1220907A1 (pt) |
HR (1) | HRP20150438T1 (pt) |
HU (1) | HUE048688T2 (pt) |
IL (2) | IL225095A (pt) |
ME (1) | ME02062B (pt) |
MX (2) | MX365007B (pt) |
NZ (3) | NZ715014A (pt) |
PL (2) | PL2613798T5 (pt) |
PT (2) | PT2977057T (pt) |
RS (1) | RS53947B1 (pt) |
RU (1) | RU2550961C2 (pt) |
SG (2) | SG10201507199UA (pt) |
SI (2) | SI2977057T1 (pt) |
SM (1) | SMT201500108B (pt) |
TW (1) | TWI482625B (pt) |
WO (1) | WO2012050695A1 (pt) |
Families Citing this family (13)
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ES2328446T5 (es) * | 2000-02-04 | 2014-02-27 | Children's Hospital Research Foundation | Uso de lipasa ácida lisosomal para tratar la aterosclerosis y enfermedades asociadas |
US20090297496A1 (en) * | 2005-09-08 | 2009-12-03 | Childrens Hospital Medical Center | Lysosomal Acid Lipase Therapy for NAFLD and Related Diseases |
EP3205351B1 (en) | 2010-04-23 | 2023-04-12 | Alexion Pharmaceuticals, Inc. | Lysosomal storage disease enzyme |
MX365007B (es) | 2010-09-09 | 2019-05-20 | Alexion Pharmaceuticals Inc Star | Uso de acido lipasa lisosomal para tratar la dificiencia de acido lipasa lisosomal en pacientes. |
KR20140006037A (ko) | 2011-02-15 | 2014-01-15 | 시나게바 바이오파르마, 코포레이션 | 리소좀의 산 리파제 결핍을 치료하는 방법들 |
WO2013055888A2 (en) | 2011-10-12 | 2013-04-18 | Synageva Biopharma Corp. | Recombinant human naglu protein and uses thereof |
KR20140130443A (ko) * | 2012-03-02 | 2014-11-10 | 시나게바 바이오파르마, 코포레이션 | 절두된 리소좀 산 리파제 |
CN106885910B (zh) * | 2015-12-15 | 2018-12-21 | 上海吉涛生物科技有限公司 | 肿瘤易感微环境检测装置及试剂盒 |
US11235036B2 (en) | 2016-06-17 | 2022-02-01 | Alexion Pharmaceuticals, Inc. | Lysosomal acid lipase deficiency compositions and methods |
WO2018039163A1 (en) | 2016-08-23 | 2018-03-01 | Alexion Pharmaceuticals, Inc. | Method of purifying a heterologous protein from an egg white |
US10633705B2 (en) | 2016-10-14 | 2020-04-28 | Alexion Pharmaceuticals, Inc. | N-acetyl-alpha-D-glucosaminidase deficiency compositions and methods |
US11396499B2 (en) | 2018-12-12 | 2022-07-26 | University Of Washington | Lysosomal acid lipase assay |
WO2021117122A1 (ja) * | 2019-12-10 | 2021-06-17 | 株式会社リボルナバイオサイエンス | ライソゾーム酸性リパーゼ欠損症の予防または治療剤 |
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2011
- 2011-09-09 MX MX2015002597A patent/MX365007B/es unknown
- 2011-09-09 JP JP2013528353A patent/JP5693728B2/ja active Active
- 2011-09-09 EP EP14200334.2A patent/EP2977057B1/en active Active
- 2011-09-09 DK DK11758644.6T patent/DK2613798T4/en active
- 2011-09-09 RS RS20150239A patent/RS53947B1/en unknown
- 2011-09-09 BR BR112013005673-8A patent/BR112013005673B1/pt active IP Right Grant
- 2011-09-09 CN CN201180053951XA patent/CN103200958A/zh active Pending
- 2011-09-09 DK DK14200334.2T patent/DK2977057T3/da active
- 2011-09-09 BR BR122019021757-2A patent/BR122019021757B1/pt active IP Right Grant
- 2011-09-09 CA CA2810999A patent/CA2810999C/en active Active
- 2011-09-09 AU AU2011314293A patent/AU2011314293B2/en active Active
- 2011-09-09 HU HUE14200334A patent/HUE048688T2/hu unknown
- 2011-09-09 PT PT142003342T patent/PT2977057T/pt unknown
- 2011-09-09 CA CA3209456A patent/CA3209456A1/en active Pending
- 2011-09-09 AR ARP110103302A patent/AR082953A1/es unknown
- 2011-09-09 SG SG10201507199UA patent/SG10201507199UA/en unknown
- 2011-09-09 ES ES11758644.6T patent/ES2535605T5/es active Active
- 2011-09-09 PT PT117586446T patent/PT2613798E/pt unknown
- 2011-09-09 SI SI201131841T patent/SI2977057T1/sl unknown
- 2011-09-09 SG SG2013017181A patent/SG188456A1/en unknown
- 2011-09-09 NZ NZ715014A patent/NZ715014A/en unknown
- 2011-09-09 KR KR20157006132A patent/KR20150038636A/ko not_active Application Discontinuation
- 2011-09-09 PL PL11758644T patent/PL2613798T5/pl unknown
- 2011-09-09 NZ NZ608292A patent/NZ608292A/en not_active IP Right Cessation
- 2011-09-09 WO PCT/US2011/051096 patent/WO2012050695A1/en active Application Filing
- 2011-09-09 ES ES14200334T patent/ES2769836T3/es active Active
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