MA42695A - Oligomères antisens modifiés pour l'inclusion d'exons dans l'atrophie musculaire spinale - Google Patents
Oligomères antisens modifiés pour l'inclusion d'exons dans l'atrophie musculaire spinaleInfo
- Publication number
- MA42695A MA42695A MA042695A MA42695A MA42695A MA 42695 A MA42695 A MA 42695A MA 042695 A MA042695 A MA 042695A MA 42695 A MA42695 A MA 42695A MA 42695 A MA42695 A MA 42695A
- Authority
- MA
- Morocco
- Prior art keywords
- exons
- inclusion
- muscle atrophy
- antisense oligomers
- modified antisense
- Prior art date
Links
- 108700024394 Exon Proteins 0.000 title 1
- 206010028289 Muscle atrophy Diseases 0.000 title 1
- 230000000692 anti-sense effect Effects 0.000 title 1
- 230000020763 muscle atrophy Effects 0.000 title 1
- 201000000585 muscular atrophy Diseases 0.000 title 1
Classifications
-
- 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
- A61K31/70—Carbohydrates; Sugars; Derivatives thereof
- A61K31/7088—Compounds having three or more nucleosides or nucleotides
- A61K31/712—Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
-
- 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
- A61K31/70—Carbohydrates; Sugars; Derivatives thereof
- A61K31/7088—Compounds having three or more nucleosides or nucleotides
- A61K31/7125—Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P21/00—Drugs for disorders of the muscular or neuromuscular system
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
- A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
-
- 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
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
-
- 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
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/31—Chemical structure of the backbone
- C12N2310/314—Phosphoramidates
- C12N2310/3145—Phosphoramidates with the nitrogen in 3' or 5'-position
-
- 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
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/32—Chemical structure of the sugar
- C12N2310/323—Chemical structure of the sugar modified ring structure
- C12N2310/3233—Morpholino-type ring
-
- 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
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/33—Chemical structure of the base
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- General Health & Medical Sciences (AREA)
- Molecular Biology (AREA)
- Pharmacology & Pharmacy (AREA)
- Organic Chemistry (AREA)
- Animal Behavior & Ethology (AREA)
- Medicinal Chemistry (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Biomedical Technology (AREA)
- Genetics & Genomics (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biochemistry (AREA)
- General Engineering & Computer Science (AREA)
- Neurology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Biotechnology (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Epidemiology (AREA)
- Neurosurgery (AREA)
- Psychology (AREA)
- Microbiology (AREA)
- Physics & Mathematics (AREA)
- Plant Pathology (AREA)
- Biophysics (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Physical Education & Sports Medicine (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicinal Preparation (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Peptides Or Proteins (AREA)
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201562211678P | 2015-08-28 | 2015-08-28 | |
| US201662379696P | 2016-08-25 | 2016-08-25 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| MA42695A true MA42695A (fr) | 2018-07-04 |
Family
ID=56883867
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| MA042695A MA42695A (fr) | 2015-08-28 | 2016-08-26 | Oligomères antisens modifiés pour l'inclusion d'exons dans l'atrophie musculaire spinale |
Country Status (8)
| Country | Link |
|---|---|
| US (3) | US10905709B2 (enExample) |
| EP (1) | EP3341480A1 (enExample) |
| JP (2) | JP6987041B2 (enExample) |
| AU (1) | AU2016317667A1 (enExample) |
| CA (1) | CA2996164A1 (enExample) |
| HK (1) | HK1257498A1 (enExample) |
| MA (1) | MA42695A (enExample) |
| WO (1) | WO2017040271A1 (enExample) |
Families Citing this family (6)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US10436802B2 (en) | 2014-09-12 | 2019-10-08 | Biogen Ma Inc. | Methods for treating spinal muscular atrophy |
| MA42695A (fr) | 2015-08-28 | 2018-07-04 | Sarepta Therapeutics Inc | Oligomères antisens modifiés pour l'inclusion d'exons dans l'atrophie musculaire spinale |
| EP3697910A4 (en) * | 2017-10-18 | 2021-07-14 | Sarepta Therapeutics, Inc. | ANTISENSE OLIGOMER COMPOUNDS |
| US12215382B2 (en) | 2019-03-01 | 2025-02-04 | The General Hospital Corporation | Liver protective MARC variants and uses thereof |
| AU2020368539A1 (en) | 2019-10-16 | 2022-04-28 | Massachusetts Institute Of Technology | Engineered muscle targeting compositions |
| CN120505310A (zh) | 2020-02-28 | 2025-08-19 | Ionis制药公司 | 用于调节smn2的化合物和方法 |
Family Cites Families (29)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| FR2802206B1 (fr) | 1999-12-14 | 2005-04-22 | Sod Conseils Rech Applic | Derives de 4-aminopiperidine et leur utilisation en tant que medicament |
| US6784291B2 (en) | 2000-05-04 | 2004-08-31 | Avi Biopharma, Inc. | Splice-region antisense composition and method |
| US8361977B2 (en) * | 2005-06-23 | 2013-01-29 | Isis Pharmaceuticals, Inc. | Compositions and methods for modulation of SMN2 splicing |
| US8067571B2 (en) | 2005-07-13 | 2011-11-29 | Avi Biopharma, Inc. | Antibacterial antisense oligonucleotide and method |
| ES2657400T3 (es) | 2006-05-10 | 2018-03-05 | Sarepta Therapeutics, Inc. | Análogos de oligonucleótidos que tienen enlaces intersubunitarios catiónicos |
| JP6047270B2 (ja) | 2006-08-11 | 2016-12-21 | バイオマリン テクノロジーズ ベー．フェー． | Ｄｎａリピートの不安定性に関連した遺伝的障害を治療するための方法及び手段 |
| US20100016215A1 (en) | 2007-06-29 | 2010-01-21 | Avi Biopharma, Inc. | Compound and method for treating myotonic dystrophy |
| WO2010120820A1 (en) * | 2009-04-13 | 2010-10-21 | Isis Pharmaceuticals, Inc. | Compositions and methods for modulation of smn2 splicing |
| PT3449926T (pt) * | 2009-06-17 | 2019-11-12 | Cold Spring Harbor Laboratory | Composições e métodos de modulação de excisões de smn2 em um sujeito |
| US8802642B2 (en) | 2010-04-28 | 2014-08-12 | Iowa State University Research Foundation, Inc. | Spinal muscular atrophy treatment via targeting SMN2 catalytic core |
| JP5585822B2 (ja) | 2010-05-11 | 2014-09-10 | 東レ・ファインケミカル株式会社 | 光学活性ニペコチン酸誘導体の製造方法 |
| JP2013530154A (ja) | 2010-05-28 | 2013-07-25 | サレプタ セラピューティクス， インコーポレイテッド | 修飾されたサブユニット間結合および／または末端基を有するオリゴヌクレオチドアナログ |
| EP2582397A4 (en) * | 2010-06-15 | 2014-10-29 | Isis Pharmaceuticals Inc | COMPOUNDS AND METHOD FOR MODULATING INTERACTION BETWEEN PROTEINS AND TARGET NUCLEIC ACIDS |
| US9161948B2 (en) | 2011-05-05 | 2015-10-20 | Sarepta Therapeutics, Inc. | Peptide oligonucleotide conjugates |
| WO2012150960A1 (en) | 2011-05-05 | 2012-11-08 | Avi Biopharma, Inc. | Peptide oligonucleotide conjugates |
| AU2012272656A1 (en) * | 2011-06-23 | 2014-02-06 | Cold Spring Harbor Laboratory | Phenocopy model of disease |
| EP2785729B1 (en) | 2011-11-30 | 2020-11-04 | Sarepta Therapeutics, Inc. | Oligonucleotides for treating expanded repeat diseases |
| AU2012345638C1 (en) * | 2011-11-30 | 2018-10-18 | Sarepta Therapeutics, Inc. | Induced exon inclusion in spinal muscle atrophy |
| US9725716B2 (en) * | 2011-12-06 | 2017-08-08 | Ohio State Innovation Foundation and Research Institute at Nationwide Children's Hospital | Non-ionic, low osmolar contrast agents for delivery of antisense oligonucleotides and treatment of disease |
| CA2868174A1 (en) | 2012-03-20 | 2013-09-26 | Sarepta Therapeutics, Inc. | Boronic acid conjugates of oligonucleotide analogues |
| SG11201407483YA (en) * | 2012-05-16 | 2014-12-30 | Rana Therapeutics Inc | Compositions and methods for modulating smn gene family expression |
| WO2013173789A2 (en) * | 2012-05-17 | 2013-11-21 | Isis Pharmaceuticals, Inc. | Antisense oligonucleotide compositions |
| WO2014110291A1 (en) * | 2013-01-09 | 2014-07-17 | Isis Pharmaceuticals, Inc. | Compositions and methods for modulation of smn2 splicing in a subject |
| US9856474B2 (en) * | 2013-01-16 | 2018-01-02 | Iowa State University Research Foundation, Inc. | Deep intronic target for splicing correction on spinal muscular atrophy gene |
| EP3560502B1 (en) * | 2013-04-12 | 2025-09-24 | The Curators of the University of Missouri | Smn2 element 1 antisense compositions and methods and uses thereof |
| US9988626B2 (en) * | 2013-07-29 | 2018-06-05 | Universität Zu Köln | Neurocalcin delta inhibitors and therapeutic and non-therapeutic uses thereof |
| EP3044317A1 (en) * | 2013-09-13 | 2016-07-20 | The University Of Western Australia | Antisense oligomers and methods for treating smn-related pathologies |
| WO2015120450A1 (en) * | 2014-02-10 | 2015-08-13 | Ohio State Innovation Foundation | Antisense oligonucleotides for treatment of spinal muscular atrophy |
| MA42695A (fr) | 2015-08-28 | 2018-07-04 | Sarepta Therapeutics Inc | Oligomères antisens modifiés pour l'inclusion d'exons dans l'atrophie musculaire spinale |
-
2016
- 2016-08-26 MA MA042695A patent/MA42695A/fr unknown
- 2016-08-26 WO PCT/US2016/048965 patent/WO2017040271A1/en not_active Ceased
- 2016-08-26 CA CA2996164A patent/CA2996164A1/en not_active Abandoned
- 2016-08-26 JP JP2018510718A patent/JP6987041B2/ja active Active
- 2016-08-26 EP EP16762935.1A patent/EP3341480A1/en not_active Withdrawn
- 2016-08-26 US US15/754,782 patent/US10905709B2/en active Active
- 2016-08-26 HK HK18116288.7A patent/HK1257498A1/zh unknown
- 2016-08-26 AU AU2016317667A patent/AU2016317667A1/en not_active Abandoned
-
2020
- 2020-11-23 US US16/949,980 patent/US12121532B2/en active Active
- 2020-12-25 JP JP2020216610A patent/JP2021048877A/ja not_active Withdrawn
-
2024
- 2024-08-23 US US18/813,696 patent/US20250241940A1/en active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| US20210169918A1 (en) | 2021-06-10 |
| US12121532B2 (en) | 2024-10-22 |
| US20250241940A1 (en) | 2025-07-31 |
| JP6987041B2 (ja) | 2021-12-22 |
| AU2016317667A1 (en) | 2018-03-22 |
| US10905709B2 (en) | 2021-02-02 |
| CA2996164A1 (en) | 2017-03-09 |
| US20190015440A1 (en) | 2019-01-17 |
| JP2021048877A (ja) | 2021-04-01 |
| EP3341480A1 (en) | 2018-07-04 |
| JP2018525015A (ja) | 2018-09-06 |
| HK1257498A1 (zh) | 2019-10-25 |
| WO2017040271A1 (en) | 2017-03-09 |
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| IL247663B (en) | Antisense oligomers for skipping exon 51 in the human dystrophin gene | |
| MA42695A (fr) | Oligomères antisens modifiés pour l'inclusion d'exons dans l'atrophie musculaire spinale | |
| EP3389671A4 (en) | ANTISENSE OLIGOMERS FOR THE TREATMENT OF ALAGILLE SYNDROME | |
| PL3159409T3 (pl) | Antysensowne kwasy nukleinowe do zastosowania do leczenia dystrofii mięśniowej duchenne'a | |
| EP3325520A4 (en) | POLYMER FORMULATIONS FOR USE WITH ELECTROOPTIC MEDIA | |
| EP3390635A4 (en) | Antisense oligomers for the treatment of a tuberous sclerosis complex | |
| EP3313238A4 (en) | FASTENING STRUCTURE FOR SUSPENSION SEAT | |
| MA44119A (fr) | Vecteurs viraux adéno-associés à utiliser dans le traitement de l'amyotrophie spinale | |
| EP3389782A4 (en) | ANTISENSE OLIGOMERS FOR THE TREATMENT OF A POLYCYSTIC KIDNEY DISEASE | |
| HUE069888T2 (hu) | Vegyületek az S1P1 receptorral összefüggõ állapotok kezelésében történõ alkalmazásra | |
| DK3481846T3 (da) | 11-substituerede 24-hydroxysteroler til anvendelse til behandling af nmda-relaterede tilstande | |
| LT2945642T (lt) | Faktoriaus 1 baltymas, skirtas naudoti ligų gydymui arba prevencijai | |
| AU2022271491B2 (en) | Antisense oligonucleotides for use in treating alzheimer's disease | |
| FR3030564B1 (fr) | Procede et dispositif pour la reduction des composes aromatiques polycycliques lourds dans les unites d'hydrocraquage | |
| EP3394719A4 (en) | HARDWARE SUPPRESSION MONITOR FOR SLIDING COMMA OPERATIONS | |
| EP3337485A4 (en) | CRYSTALLINE FORMS OF IBRUTINIB | |
| EP3479835A4 (en) | COMPOSITION FOR USE IN IMPROVING THE NUTRITIONAL STATE | |
| EP3350155A4 (en) | TRIAMINE STARCH USING DISUURES | |
| PL3377909T3 (pl) | Laktoferyna do zastosowania w diagnozowaniu lub prognozowaniu choroby Alzheimera | |
| EP3204028A4 (en) | SYNERGISTIC COMPOSITION FOR OSTEOARTHRITIS | |
| EP3394184A4 (en) | COMPOSITION FOR IMMOBILIZING AN INK JET INK | |
| FR3020758B1 (fr) | Composition pour ameliorer l'hygiene buco-dentaire | |
| EP3374979A4 (en) | COMMUNICATION HUB FOR EMERGENCY RESPONDER | |
| PL3285755T3 (pl) | Chlorowodorek kreatyny do leczenia choroby Huntingtona | |
| DK3283064T3 (da) | Derivater til anvendelse i behandling af muskelatrofi |