JP2019500350A5 - - Google Patents
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- Publication number
- JP2019500350A5 JP2019500350A5 JP2018529258A JP2018529258A JP2019500350A5 JP 2019500350 A5 JP2019500350 A5 JP 2019500350A5 JP 2018529258 A JP2018529258 A JP 2018529258A JP 2018529258 A JP2018529258 A JP 2018529258A JP 2019500350 A5 JP2019500350 A5 JP 2019500350A5
- Authority
- JP
- Japan
- Prior art keywords
- mrna
- pharmaceutical composition
- composition according
- item
- tuberin
- Prior art date
- Legal status (The legal status 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 status listed.)
- Granted
Links
- 108020004999 messenger RNA Proteins 0.000 claims description 100
- 108090000623 proteins and genes Proteins 0.000 claims description 79
- 102000004169 proteins and genes Human genes 0.000 claims description 69
- 230000000717 retained effect Effects 0.000 claims description 68
- 230000000692 anti-sense effect Effects 0.000 claims description 57
- 239000008194 pharmaceutical composition Substances 0.000 claims description 37
- 108700028369 Alleles Proteins 0.000 claims description 31
- 102000002027 Tuberin Human genes 0.000 claims description 31
- 108050009309 Tuberin Proteins 0.000 claims description 31
- 108020005067 RNA Splice Sites Proteins 0.000 claims description 29
- SZCZSKMCTGEJKI-UHFFFAOYSA-N tuberin Natural products COC1=CC=C(C=CNC=O)C=C1 SZCZSKMCTGEJKI-UHFFFAOYSA-N 0.000 claims description 17
- 230000002950 deficient Effects 0.000 claims description 12
- 238000002347 injection Methods 0.000 claims description 10
- 239000007924 injection Substances 0.000 claims description 10
- 230000000694 effects Effects 0.000 claims description 8
- 108020004707 nucleic acids Proteins 0.000 claims description 7
- 102000039446 nucleic acids Human genes 0.000 claims description 7
- 230000000295 complement effect Effects 0.000 claims description 6
- 208000026911 Tuberous sclerosis complex Diseases 0.000 claims description 5
- 230000007812 deficiency Effects 0.000 claims description 5
- 238000010255 intramuscular injection Methods 0.000 claims description 5
- 239000007927 intramuscular injection Substances 0.000 claims description 5
- 239000007928 intraperitoneal injection Substances 0.000 claims description 5
- 238000007913 intrathecal administration Methods 0.000 claims description 5
- 238000010253 intravenous injection Methods 0.000 claims description 5
- 238000007914 intraventricular administration Methods 0.000 claims description 5
- 238000004519 manufacturing process Methods 0.000 claims description 5
- 239000002243 precursor Substances 0.000 claims description 5
- 238000010254 subcutaneous injection Methods 0.000 claims description 5
- 239000007929 subcutaneous injection Substances 0.000 claims description 5
- 208000009999 tuberous sclerosis Diseases 0.000 claims description 5
- 108091093037 Peptide nucleic acid Proteins 0.000 claims description 4
- 238000012986 modification Methods 0.000 claims description 4
- 230000004048 modification Effects 0.000 claims description 4
- 150000007523 nucleic acids Chemical class 0.000 claims description 4
- ANCLJVISBRWUTR-UHFFFAOYSA-N diaminophosphinic acid Chemical compound NP(N)(O)=O ANCLJVISBRWUTR-UHFFFAOYSA-N 0.000 claims 1
- 239000003814 drug Substances 0.000 claims 1
- RYYWUUFWQRZTIU-UHFFFAOYSA-K thiophosphate Chemical compound [O-]P([O-])([O-])=S RYYWUUFWQRZTIU-UHFFFAOYSA-K 0.000 claims 1
- 201000008827 tuberculosis Diseases 0.000 claims 1
- 238000000034 method Methods 0.000 description 68
- 239000000203 mixture Substances 0.000 description 36
- 108091032973 (ribonucleotides)n+m Proteins 0.000 description 34
- 101000795659 Homo sapiens Tuberin Proteins 0.000 description 22
- 102100031638 Tuberin Human genes 0.000 description 20
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 description 15
- 239000002773 nucleotide Substances 0.000 description 11
- 125000003729 nucleotide group Chemical group 0.000 description 11
- 201000010099 disease Diseases 0.000 description 10
- 108091092195 Intron Proteins 0.000 description 8
- 108091027974 Mature messenger RNA Proteins 0.000 description 4
- 208000035475 disorder Diseases 0.000 description 3
- 210000004072 lung Anatomy 0.000 description 3
- -1 nucleic acid salts Chemical class 0.000 description 3
- 239000000546 pharmaceutical excipient Substances 0.000 description 3
- 230000002685 pulmonary effect Effects 0.000 description 3
- 238000011200 topical administration Methods 0.000 description 3
- 108091034117 Oligonucleotide Proteins 0.000 description 2
- 230000001594 aberrant effect Effects 0.000 description 2
- 239000000074 antisense oligonucleotide Substances 0.000 description 2
- 238000012230 antisense oligonucleotides Methods 0.000 description 2
- 230000035772 mutation Effects 0.000 description 2
- GFABGVSRKCKLKA-ONBPZOJHSA-N 2-[(2s,5r)-5-[(1r)-2-[4-(2-benzamidoethyl)phenoxy]-1-hydroxyethyl]-5-methyloxolan-2-yl]propan-2-yl acetate Chemical compound O1[C@H](C(C)(C)OC(=O)C)CC[C@]1(C)[C@H](O)COC(C=C1)=CC=C1CCNC(=O)C1=CC=CC=C1 GFABGVSRKCKLKA-ONBPZOJHSA-N 0.000 description 1
- GFABGVSRKCKLKA-UHFFFAOYSA-N Tuberine Natural products O1C(C(C)(C)OC(=O)C)CCC1(C)C(O)COC(C=C1)=CC=C1CCNC(=O)C1=CC=CC=C1 GFABGVSRKCKLKA-UHFFFAOYSA-N 0.000 description 1
- 210000003754 fetus Anatomy 0.000 description 1
- 238000010348 incorporation Methods 0.000 description 1
- 230000001939 inductive effect Effects 0.000 description 1
- 210000001161 mammalian embryo Anatomy 0.000 description 1
- 239000000126 substance Substances 0.000 description 1
- 238000011144 upstream manufacturing Methods 0.000 description 1
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2022053948A JP2022088533A (ja) | 2015-12-14 | 2022-03-29 | 結節性硬化症の処置のためのアンチセンスオリゴマー |
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201562267212P | 2015-12-14 | 2015-12-14 | |
| US62/267,212 | 2015-12-14 | ||
| PCT/US2016/066705 WO2017106375A1 (en) | 2015-12-14 | 2016-12-14 | Antisense oligomers for treatment of tuberous sclerosis complex |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022053948A Division JP2022088533A (ja) | 2015-12-14 | 2022-03-29 | 結節性硬化症の処置のためのアンチセンスオリゴマー |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2019500350A JP2019500350A (ja) | 2019-01-10 |
| JP2019500350A5 true JP2019500350A5 (enExample) | 2020-01-30 |
| JP7051683B2 JP7051683B2 (ja) | 2022-04-11 |
Family
ID=59057534
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2018529258A Active JP7051683B2 (ja) | 2015-12-14 | 2016-12-14 | 結節性硬化症の処置のためのアンチセンスオリゴマー |
| JP2022053948A Pending JP2022088533A (ja) | 2015-12-14 | 2022-03-29 | 結節性硬化症の処置のためのアンチセンスオリゴマー |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022053948A Pending JP2022088533A (ja) | 2015-12-14 | 2022-03-29 | 結節性硬化症の処置のためのアンチセンスオリゴマー |
Country Status (4)
| Country | Link |
|---|---|
| EP (1) | EP3390635A4 (enExample) |
| JP (2) | JP7051683B2 (enExample) |
| CA (1) | CA3005131A1 (enExample) |
| WO (1) | WO2017106375A1 (enExample) |
Families Citing this family (15)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| GB201410693D0 (en) | 2014-06-16 | 2014-07-30 | Univ Southampton | Splicing modulation |
| EP3201339A4 (en) | 2014-10-03 | 2018-09-19 | Cold Spring Harbor Laboratory | Targeted augmentation of nuclear gene output |
| EP3359685A1 (en) | 2015-10-09 | 2018-08-15 | University Of Southampton | Modulation of gene expression and screening for deregulated protein expression |
| US11096956B2 (en) | 2015-12-14 | 2021-08-24 | Stoke Therapeutics, Inc. | Antisense oligomers and uses thereof |
| KR102604132B1 (ko) | 2015-12-14 | 2023-11-17 | 콜드스프링하버러보러토리 | 상염색체 우성 정신 지체 5 및 드라베 증후군의 치료를 위한 안티센스 올리고머 |
| KR102712656B1 (ko) | 2017-01-23 | 2024-10-04 | 리제너론 파마슈티칼스 인코포레이티드 | Hsd17b13 변종 및 이것의 용도 |
| US11479802B2 (en) | 2017-04-11 | 2022-10-25 | Regeneron Pharmaceuticals, Inc. | Assays for screening activity of modulators of members of the hydroxy steroid (17-beta) dehydrogenase (HSD17B) family |
| SI3673080T1 (sl) | 2017-08-25 | 2024-03-29 | Stoke Therapeutics, Inc. | Protismiselni oligomeri za zdravljenje bolezenskih stanj in bolezni |
| AU2018348195B2 (en) | 2017-10-11 | 2025-05-15 | Regeneron Pharmaceuticals, Inc. | Inhibition of HSD17B13 in the treatment of liver disease in patients expressing the PNPLA3 I148M variation |
| BR112020018758A2 (pt) | 2018-03-21 | 2021-01-26 | Regeneron Pharmaceuticals, Inc. | agente de ácido ribonucleico de fita dupla, célula, vetor, composição farmacêutica, e, métodos para inibição da expressão de 17¿-hidroxiesteroide desidrogenases tipo 13, para tratamento de um indivíduo, para prevenção de um sintoma em um indivíduo, para redução do risco de desenvolver doença hepática crônica, para inibição da progressão de esteatose, para inibição do acúmulo de gotículas de lipídios |
| BR112020022512A2 (pt) | 2018-05-04 | 2021-05-04 | Stoke Therapeutics, Inc. | métodos e composições para tratamento de doença de armazenamento de éster de colesteril |
| CN114746550A (zh) * | 2019-08-19 | 2022-07-12 | 斯托克制药公司 | 用于调节剪接和蛋白质表达的组合物和方法 |
| AU2021270720A1 (en) | 2020-05-11 | 2022-12-08 | Stoke Therapeutics, Inc. | OPA1 antisense oligomers for treatment of conditions and diseases |
| EP4560019A1 (en) * | 2023-11-22 | 2025-05-28 | Sylentis S.A.U. | Sirna and compositions for prophylactic and therapeutic treatment of ocular retinal conditions |
| EP4560020A1 (en) * | 2023-11-22 | 2025-05-28 | Sylentis S.A.U. | Sirna and compositions for prophylactic and therapeutic treatment of ocular retinal conditions |
Family Cites Families (7)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2008043561A2 (en) * | 2006-10-11 | 2008-04-17 | MAX-PLANCK-Gesellschaft zur Förderung der Wissenschaften e.V. | Influenza targets |
| WO2011020118A1 (en) | 2009-08-14 | 2011-02-17 | Theramind Research, Llc | Methods and compositions for treatment of tuberous sclerosis complex |
| CA2773049C (en) * | 2009-09-08 | 2019-08-20 | Laboratory Corporation Of America Holdings | Compositions and methods for diagnosing autism spectrum disorders |
| CA2930859C (en) * | 2013-09-04 | 2022-05-03 | Cold Spring Harbor Laboratory | Reducing nonsense-mediated mrna decay |
| GB201410693D0 (en) * | 2014-06-16 | 2014-07-30 | Univ Southampton | Splicing modulation |
| EP3201339A4 (en) * | 2014-10-03 | 2018-09-19 | Cold Spring Harbor Laboratory | Targeted augmentation of nuclear gene output |
| JP7049249B2 (ja) * | 2015-12-14 | 2022-04-06 | コールド スプリング ハーバー ラボラトリー | 中枢神経系疾患の処置のための組成物および方法 |
-
2016
- 2016-12-14 CA CA3005131A patent/CA3005131A1/en active Pending
- 2016-12-14 WO PCT/US2016/066705 patent/WO2017106375A1/en not_active Ceased
- 2016-12-14 EP EP16876615.2A patent/EP3390635A4/en not_active Withdrawn
- 2016-12-14 JP JP2018529258A patent/JP7051683B2/ja active Active
-
2022
- 2022-03-29 JP JP2022053948A patent/JP2022088533A/ja active Pending
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