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1. (WO2019067587) USE OF METFORMIN AND ANALOGS THEREOF TO REDUCE RAN PROTEIN LEVELS IN THE TREATMENT OF NEUROLOGICAL DISORDERS
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Pub. No.: WO/2019/067587 International Application No.: PCT/US2018/052913
Publication Date: 04.04.2019 International Filing Date: 26.09.2018
IPC:
C07K 14/47 (2006.01) ,C07K 16/18 (2006.01) ,C12Q 1/68 (2018.01) ,G01N 33/68 (2006.01)
C CHEMISTRY; METALLURGY
07
ORGANIC CHEMISTRY
K
PEPTIDES
14
Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
435
from animals; from humans
46
from vertebrates
47
from mammals
C CHEMISTRY; METALLURGY
07
ORGANIC CHEMISTRY
K
PEPTIDES
16
Immunoglobulins, e.g. monoclonal or polyclonal antibodies
18
against material from animals or humans
C CHEMISTRY; METALLURGY
12
BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
Q
MEASURING OR TESTING PROCESSES INVOLVING ENZYMES OR MICRO-ORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
1
Measuring or testing processes involving enzymes or micro-organisms; Compositions therefor; Processes of preparing such compositions
68
involving nucleic acids
G PHYSICS
01
MEASURING; TESTING
N
INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
33
Investigating or analysing materials by specific methods not covered by groups G01N1/-G01N31/131
48
Biological material, e.g. blood, urine; Haemocytometers
50
Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing
68
involving proteins, peptides or amino acids
Applicants:
UNIVERSITY OF FLORIDA RESEARCH FOUNDATION, INCORPORATED [US/US]; 223 Grinter Hall Gainesville, FL 32611, US
Inventors:
RANUM, Laura; US
ZU, Tao; US
Agent:
CHAO, Jessica, J.; US
Priority Data:
62/563,58826.09.2017US
Title (EN) USE OF METFORMIN AND ANALOGS THEREOF TO REDUCE RAN PROTEIN LEVELS IN THE TREATMENT OF NEUROLOGICAL DISORDERS
(FR) UTILISATION DE METFORMINE ET D'ANALOGUES DE CELLE-CI POUR RÉDUIRE LES TAUX DE PROTÉINE RAN LORS D'UN TRAITEMENT DE TROUBLES NEUROLOGIQUES
Abstract:
(EN) The present disclosure provides the use of compounds of Formulae (I), (II), (III), (III-A), and (III-B) (e.g., metformin) in treating a neurological disease associated with repeat expansions and/or RAN protein accumulation, reducing the level of one or more repeat associated non-ATG (RAN) proteins, and reducing the accumulation of RAN proteins in a subject and/or biological sample. Also provided is the use of compounds of Formulae (I), (II), (III), (III-A), and (III-B) (e.g., metformin) in inhibiting RAN protein translation in a subject and in a biological sample (e.g., cells, tissue). Also provided in the present disclosure are pharmaceutical compositions, kits, and uses of compounds of Formulae (I), (II), (III), (III-A), and (III-B) (e.g., metformin) for treating diseases associated with repeat expansions. Exemplary diseases associated with repeat expansions include, but are not limited to, C90RFf72 amyotrophic lateral sclerosis (ALS), or C90RFf72 frontotemporal dementia; myotonic dystrophy type 1 (DM1) and myotonic dystrophy type 2 (DM2); spinocerebellar ataxia; Huntington' s disease; Fragile X Tremor Ataxia Syndrome (FXTAS); and Fragile XE syndrome (FRAXE).
(FR) La présente invention concerne l'utilisation de composés de formules (I), (II), (III), (III-A), et (III-B) (par exemple, metformine) dans le traitement d'une maladie neurologique associée à des expansions de répétition et/ou à l'accumulation de protéines RAN, la réduction du niveau d'une ou de plusieurs protéines non ATG (RAN) associées à une répétition, et la réduction de l'accumulation de protéines RAN chez un sujet et/ou dans un échantillon biologique. L'invention concerne également l'utilisation de composés de formules (I), (II), (III), (III-A), et (III -B) (par exemple, metformine) dans l'inhibition de la traduction de protéines RAN chez un sujet et dans un échantillon biologique (par exemple, cellules, tissu). La présente invention concerne en outre des compositions pharmaceutiques, des kits et des utilisations de composés de formules (I), (II), (III), (III-A), et (III-B) (par exemple, metformine) pour traiter des maladies associées à des expansions de répétition. Selon l'invention, des exemples de maladies associées à des expansions de répétition comprennent, mais sans caractère limitatif, la sclérose latérale amyotrophique C90RFf72 (ALS), ou la démence frontotemporale C90RFf72; la dystrophie myotonique de type 1 (DM1) et la dystrophie myotonique de type 2 (DM2); l'ataxie spinocérébelleuse; la maladie de Huntington; le syndrome de l'ataxie de tremblement associé à l'X Fragile (FXTAS); et le syndrome associé à l'XE Fragile (FRAXE).
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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, JO, 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: English (EN)
Filing Language: English (EN)