Some content of this application is unavailable at the moment.
If this situation persist, please contact us atFeedback&Contact
1. (WO2018201516) COMPOUND AND APPLICATION THEREOF FOR TREATMENT OF CATARACT
Latest bibliographic data on file with the International Bureau    Submit observation

Pub. No.: WO/2018/201516 International Application No.: PCT/CN2017/084437
Publication Date: 08.11.2018 International Filing Date: 16.05.2017
IPC:
C07J 9/00 (2006.01) ,C07J 73/00 (2006.01) ,C07J 17/00 (2006.01) ,C07J 41/00 (2006.01) ,C07J 71/00 (2006.01) ,A61K 31/575 (2006.01) ,A61K 31/58 (2006.01) ,A61K 31/585 (2006.01) ,A61P 27/12 (2006.01)
C CHEMISTRY; METALLURGY
07
ORGANIC CHEMISTRY
J
STEROIDS
9
Normal steroids containing carbon, hydrogen, halogen, or oxygen, substituted in position 17 beta by a chain of more than two carbon atoms, e.g. cholane, cholestane, coprostane
C CHEMISTRY; METALLURGY
07
ORGANIC CHEMISTRY
J
STEROIDS
73
Steroids in which the cyclopenta[a]hydrophenanthrene skeleton has been modified by substitution of one or two carbon atoms by hetero atoms
C CHEMISTRY; METALLURGY
07
ORGANIC CHEMISTRY
J
STEROIDS
17
Normal steroids containing carbon, hydrogen, halogen, or oxygen, having an oxygen-containing hetero ring not condensed with the cyclopenta[a]hydrophenanthrene skeleton
C CHEMISTRY; METALLURGY
07
ORGANIC CHEMISTRY
J
STEROIDS
41
Normal steroids containing one or more nitrogen atoms not belonging to a hetero ring
C CHEMISTRY; METALLURGY
07
ORGANIC CHEMISTRY
J
STEROIDS
71
Steroids in which the cyclopenta[a]hydrophenanthrene skeleton is condensed with a heterocyclic ring
A HUMAN NECESSITIES
61
MEDICAL OR VETERINARY SCIENCE; HYGIENE
K
PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
31
Medicinal preparations containing organic active ingredients
56
Compounds containing cyclopenta[a]hydrophenanthrene ring systems; Derivatives thereof, e.g. steroids
575
substituted in position 17 beta by a chain of three or more carbon atoms, e.g. cholane, cholestane, ergosterol, sitosterol
A HUMAN NECESSITIES
61
MEDICAL OR VETERINARY SCIENCE; HYGIENE
K
PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
31
Medicinal preparations containing organic active ingredients
56
Compounds containing cyclopenta[a]hydrophenanthrene ring systems; Derivatives thereof, e.g. steroids
58
containing heterocyclic rings, e.g. aldosterone, danazol, stanozolol, pancuronium, digitogenin
A HUMAN NECESSITIES
61
MEDICAL OR VETERINARY SCIENCE; HYGIENE
K
PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
31
Medicinal preparations containing organic active ingredients
56
Compounds containing cyclopenta[a]hydrophenanthrene ring systems; Derivatives thereof, e.g. steroids
58
containing heterocyclic rings, e.g. aldosterone, danazol, stanozolol, pancuronium, digitogenin
585
containing lactone rings, e.g. oxandrolone, bufalin
A HUMAN NECESSITIES
61
MEDICAL OR VETERINARY SCIENCE; HYGIENE
P
SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
27
Drugs for disorders of the senses
02
Ophthalmic agents
12
for cataracts
Applicants:
清华大学 TSINGHUA UNIVERSITY [CN/CN]; 中国北京市 100084信箱82分箱清华大学专利办公室 Tsinghua University Patent Office Mailbox 82 Box Beijing 100084, CN
Inventors:
饶燏 RAO, Yu; CN
闫永彬 YAN, Yongbin; CN
杨兴林 YANG, Xinglin; CN
杨子默 YANG, Zimo; CN
陈祥军 CHEN, Xiangjun; CN
Agent:
北京纪凯知识产权代理有限公司 JEEKAI & PARTNERS; 中国北京市 丰台区广安路9号5号楼15A层 Floor 15A, Building No.5 9 Guang'an Road, Fengtai District Beijing 100055, CN
Priority Data:
201710311108.605.05.2017CN
Title (EN) COMPOUND AND APPLICATION THEREOF FOR TREATMENT OF CATARACT
(FR) COMPOSÉ ET SON APPLICATION DANS LE TRAITEMENT DE LA CATARACTE
(ZH) 化合物及其在治疗白内障中的应用
Abstract:
(EN) A compound and an application thereof for treatment of cataract. The structural formula of the compound is as shown in formula I. The compound of formula I, a prodrug thereof, or a pharmaceutically acceptable salt thereof, can be used to prevent, alleviate, or reverse aggregation of lens proteins in cells. In lens cells, more than 90% of protein components are crystallins (CRYs), comprising the three families of α, β, and γ-CRY, and mutation of the lens proteins causes aggregation of proteins in the cells, leading to cataract diseases. α-CRY family mutants αA-Y118D and αB-R120G, an β-CRY family mutant βB2-V187E, and γ-CRY family mutants γC-G129C and γD-W43R were selected for a study model of cataract diseases to detect effects of the compound. Compared with existing micromolecules (such as C29, Science 350, 674), the micromolecule with a novel structure has better activity in inhibiting protein aggregation caused by intracellular lens protein mutation, improves absorbability of drugs by the body and has no toxic side effects on normal lens cells.
(FR) L'invention concerne un composé et une application de celui-ci dans le traitement de la cataracte. La formule structurale du composé est telle que représentée dans la formule I, le composé de formule I, un promédicament de celui-ci, ou un sel pharmaceutiquement acceptable de celui-ci, peuvent être utilisés pour prévenir, atténuer ou neutraliser l'agrégation des protéines de cristallin dans des cellules. Dans les cellules de cristallin, plus de 90 % des composants protéiques sont des cristallins (CRY), comprenant les trois familles α, β et γ-CRY, et la mutation des protéines de cristallin provoque l'agrégation de protéines dans les cellules, conduisant à des maladies de la cataracte. Les mutants de la famille α-CRY, αA-Y118D et αB-R120G, un mutant de la famille β-CRY, βB2-V187E, et les mutants de la famille γ-CRY, γC-G129C et γD-W43R ont été sélectionnés pour un modèle d'étude des maladies de la cataracte afin de détecter des effets du composé. Par comparaison avec des micromolécules existantes (telles que C29, Science 350, 674), la micromolécule ayant une nouvelle structure présente une meilleure activité dans l'inhibition de l'agrégation de protéines provoquée par une mutation de protéine de cristallin intracellulaire, améliore la capacité d'absorption de médicaments par le corps et n'a pas d'effets secondaires toxiques sur les cellules normales de cristallin.
(ZH) 一种化合物及其在治疗白内障中的应用。所述化合物的结构式如式I所示。式I所示化合物、其前药或其药学上可接受的盐可用于阻止、缓解或者逆转晶状体蛋白在细胞内的聚集;在晶状体细胞中,90%以上的蛋白组分是晶状体蛋白(crystallin,CRY),包括α、β、γ-CRY三个家族,而晶状体蛋白发生突变后,会引发细胞内的蛋白聚集,导致白内障疾病。选取α-CRY家族突变体αA-Y118D,αB-R120G,β-CRY家族突变体βB2-V187E, γ-CRY家族突变体γC-G129C和γD-W43R为白内障疾病的研究模型检测了化合物的效果。提供的具有新颖结构的小分子,与现有的小分子(如C29,Science,350,674)相比,在抑制细胞内晶状体蛋白突变导致的蛋白聚集具有更好的活性,且提高药物的可被机体吸收性,并且对正常晶状体细胞没有毒副作用。
Designated States: AE, AG, AL, AM, AO, AT, AU, AZ, BA, BB, BG, BH, BN, BR, BW, BY, BZ, CA, CH, CL, CN, CO, CR, CU, CZ, DE, DJ, DK, DM, DO, DZ, EC, EE, EG, ES, FI, GB, GD, GE, GH, GM, GT, HN, HR, HU, ID, IL, IN, IR, IS, JP, KE, KG, KH, KN, KP, KR, KW, KZ, LA, LC, LK, LR, LS, LU, LY, MA, MD, ME, MG, MK, MN, MW, MX, MY, MZ, NA, NG, NI, NO, NZ, OM, PA, PE, PG, PH, PL, PT, QA, RO, RS, RU, RW, SA, SC, SD, SE, SG, SK, SL, SM, ST, SV, SY, TH, TJ, TM, TN, TR, TT, TZ, UA, UG, US, UZ, VC, VN, ZA, ZM, ZW
African Regional Intellectual Property Organization (ARIPO) (BW, GH, GM, KE, LR, LS, MW, MZ, NA, RW, SD, SL, ST, SZ, TZ, UG, ZM, ZW)
Eurasian Patent Office (AM, AZ, BY, KG, KZ, RU, TJ, TM)
European Patent Office (EPO) (AL, AT, BE, BG, CH, CY, CZ, DE, DK, EE, ES, FI, FR, GB, GR, HR, HU, IE, IS, IT, LT, LU, LV, MC, MK, MT, NL, NO, PL, PT, RO, RS, SE, SI, SK, SM, TR)
African Intellectual Property Organization (BF, BJ, CF, CG, CI, CM, GA, GN, GQ, GW, KM, ML, MR, NE, SN, TD, TG)
Publication Language: Chinese (ZH)
Filing Language: Chinese (ZH)