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AR108916A1 - OLIGONUCLEÓTIDOS ANTISENTIDO TO MODULATE THE EXPRESSION OF HTRA1 - Google Patents

OLIGONUCLEÓTIDOS ANTISENTIDO TO MODULATE THE EXPRESSION OF HTRA1

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Publication number
AR108916A1
AR108916A1 ARP170101802A ARP170101802A AR108916A1 AR 108916 A1 AR108916 A1 AR 108916A1 AR P170101802 A ARP170101802 A AR P170101802A AR P170101802 A ARP170101802 A AR P170101802A AR 108916 A1 AR108916 A1 AR 108916A1
Authority
AR
Argentina
Prior art keywords
seq
disease
antisense oligonucleotide
amd
nucleotides
Prior art date
Application number
ARP170101802A
Other languages
Spanish (es)
Inventor
Lykke Pedersen
Heidi Hudlebusch
Sindri Traustason
Sren Ottosen
Susanne Kammler
Peter Hagedorn
Roberto Iacone
Original Assignee
Hoffmann La Roche
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Hoffmann La Roche filed Critical Hoffmann La Roche
Publication of AR108916A1 publication Critical patent/AR108916A1/en

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Abstract

Reivindicación 1: Oligonucleótido antisentido de entre 10 y 30 nucleótidos de longitud, en el que dicho oligonucleótido antisentido presenta como diana un ácido nucleico de HTRA1 y comprende una región de nucleótidos contigua de entre 10 y 22 nucleótidos, que es por lo menos 90%, tal como 100%, complementaria respecto a la SEC ID nº 147: SEC ID nº 147: 5’ CCAACAACCAGGTAAATATTTG 3’. Reivindicación 2: Oligonucleótido antisentido según la reivindicación 1 ó 2, en el que la región de nucleótidos contigua es idéntica a una secuencia presente en una secuencia seleccionada de entre el grupo que consiste de SEC ID nº 138, 139, 140, 141, 142, 143, 144 y 145: SEC ID nº 138: CAAATATTTACCTGGTTG SEC ID nº 139: TTTACCTGGTTGTTGG SEC ID nº 140: CCAAATATTTACCTGGTT SEC ID nº 141: CCAAATATTTACCTGGTTGT SEC ID nº 142: ATATTTACCTGGTTGTTG SEC ID nº 143: TATTTACCTGGTTGTT SEC ID nº 144: ATATTTACCTGGTTGT SEC ID nº 145: ATATTTACCTGGTTGTT. Reivindicación 12: Composición farmacéutica que comprende el oligonucleótido según las reivindicaciones 1 a 10 o el conjugado según la reivindicación 11 y un diluyente, solvente, portador, sal y/o adyuvante farmacéuticamente aceptable. Reivindicación 17: Utilización del oligonucleótido según cualquiera de las reivindicaciones 1 a 10 o el conjugado según la reivindicación 11 o la composición farmacéutica según la reivindicación 12, para la preparación de un medicamento destinado al tratamiento o la prevención de una enfermedad seleccionada de entre el grupo que consiste de degeneración macular (tal como DMAE húmeda, DMAE seca, atrofia geográfica, DMAEd intermedia, retinopatía diabética), enfermedad de Parkinson, enfermedad de Alzheimer, distrofia muscular de Duchenne, artritis, tal como osteoartritis y enfermedad isquémica hereditaria de vasos pequeños cerebrales.Claim 1: Antisense oligonucleotide between 10 and 30 nucleotides in length, wherein said antisense oligonucleotide targets an HTRA1 nucleic acid and comprises a contiguous nucleotide region of between 10 and 22 nucleotides, which is at least 90%, such as 100%, complementary to SEQ ID No. 147: SEQ ID No. 147: 5 'CCAACAACCAGGTAAATATTTG 3'. Claim 2: Antisense oligonucleotide according to claim 1 or 2, wherein the contiguous nucleotide region is identical to a sequence present in a sequence selected from the group consisting of SEQ ID NO: 138, 139, 140, 141, 142, 143, 144 and 145: SEQ ID 138: CAAATATTTACCTGGTTG SEQ ID 139: TTTACCTGGTTGTTGG SEQ ID 140: CCAAATATTTACCTGGTT SEQ ID 141: CCAAATATTTACCTGGTTGT SEQ ID 142: ATATTTACCTGGTTGTTG SEQ ID 143: TATTTACCTGGTTGTT SEQ ID 144: ATATTTACCTGGTTGT SEQ ID No. 145: ATATTTACCTGGTTGTT. Claim 12: Pharmaceutical composition comprising the oligonucleotide according to claims 1 to 10 or the conjugate according to claim 11 and a pharmaceutically acceptable diluent, solvent, carrier, salt and / or adjuvant. Claim 17: Use of the oligonucleotide according to any one of claims 1 to 10 or the conjugate according to claim 11 or the pharmaceutical composition according to claim 12, for the preparation of a medicament for the treatment or prevention of a disease selected from the group consisting of macular degeneration (such as wet AMD, dry AMD, geographic atrophy, intermediate AMD, diabetic retinopathy), Parkinson's disease, Alzheimer's disease, Duchenne muscular dystrophy, arthritis, such as osteoarthritis and hereditary ischemic disease of small cerebral vessels .

ARP170101802A 2016-07-01 2017-06-29 OLIGONUCLEÓTIDOS ANTISENTIDO TO MODULATE THE EXPRESSION OF HTRA1 AR108916A1 (en)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
EP16177508 2016-07-01

Publications (1)

Publication Number Publication Date
AR108916A1 true AR108916A1 (en) 2018-10-10

Family

ID=56292595

Family Applications (1)

Application Number Title Priority Date Filing Date
ARP170101802A AR108916A1 (en) 2016-07-01 2017-06-29 OLIGONUCLEÓTIDOS ANTISENTIDO TO MODULATE THE EXPRESSION OF HTRA1

Country Status (1)

Country Link
AR (1) AR108916A1 (en)

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