JPWO2020176776A5 - - Google Patents
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- Publication number
- JPWO2020176776A5 JPWO2020176776A5 JP2021550217A JP2021550217A JPWO2020176776A5 JP WO2020176776 A5 JPWO2020176776 A5 JP WO2020176776A5 JP 2021550217 A JP2021550217 A JP 2021550217A JP 2021550217 A JP2021550217 A JP 2021550217A JP WO2020176776 A5 JPWO2020176776 A5 JP WO2020176776A5
- Authority
- JP
- Japan
- Prior art keywords
- therapeutic agent
- protein
- nmd exon
- nucleotides
- pharmaceutical composition
- 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
- 239000002773 nucleotide Substances 0.000 claims description 92
- 125000003729 nucleotide group Chemical group 0.000 claims description 92
- 101000631760 Homo sapiens Sodium channel protein type 1 subunit alpha Proteins 0.000 claims description 45
- 102100028910 Sodium channel protein type 1 subunit alpha Human genes 0.000 claims description 43
- 239000003814 drug Substances 0.000 claims description 41
- 229940124597 therapeutic agent Drugs 0.000 claims description 41
- 238000000034 method Methods 0.000 claims description 38
- 108020004999 messenger RNA Proteins 0.000 claims description 35
- 230000008685 targeting Effects 0.000 claims description 17
- 238000011144 upstream manufacturing Methods 0.000 claims description 16
- 208000036572 Myoclonic epilepsy Diseases 0.000 claims description 11
- 201000010099 disease Diseases 0.000 claims description 11
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims description 11
- 230000001939 inductive effect Effects 0.000 claims description 11
- 201000007547 Dravet syndrome Diseases 0.000 claims description 9
- 206010073677 Severe myoclonic epilepsy of infancy Diseases 0.000 claims description 9
- 208000014644 Brain disease Diseases 0.000 claims description 8
- 208000032274 Encephalopathy Diseases 0.000 claims description 8
- 208000002091 Febrile Seizures Diseases 0.000 claims description 8
- 108700028369 Alleles Proteins 0.000 claims description 6
- 206010010904 Convulsion Diseases 0.000 claims description 6
- 208000003078 Generalized Epilepsy Diseases 0.000 claims description 5
- 230000001037 epileptic effect Effects 0.000 claims description 5
- 230000007717 exclusion Effects 0.000 claims description 5
- 201000001993 idiopathic generalized epilepsy Diseases 0.000 claims description 5
- 206010061334 Partial seizures Diseases 0.000 claims description 4
- 208000023944 Sudden Unexpected Death in Epilepsy Diseases 0.000 claims description 4
- 230000000692 anti-sense effect Effects 0.000 claims description 4
- 230000001404 mediated effect Effects 0.000 claims description 4
- 108091032973 (ribonucleotides)n+m Proteins 0.000 claims description 3
- 201000008186 generalized epilepsy with febrile seizures plus Diseases 0.000 claims description 3
- 208000011580 syndromic disease Diseases 0.000 claims description 3
- 208000005875 Alternating hemiplegia of childhood Diseases 0.000 claims description 2
- 206010003805 Autism Diseases 0.000 claims description 2
- 208000020706 Autistic disease Diseases 0.000 claims description 2
- 206010021750 Infantile Spasms Diseases 0.000 claims description 2
- 208000035899 Infantile spasms syndrome Diseases 0.000 claims description 2
- 208000037004 Myoclonic-astatic epilepsy Diseases 0.000 claims description 2
- 208000033063 Progressive myoclonic epilepsy Diseases 0.000 claims description 2
- 201000006791 West syndrome Diseases 0.000 claims description 2
- 230000036461 convulsion Effects 0.000 claims description 2
- 201000009028 early myoclonic encephalopathy Diseases 0.000 claims description 2
- 208000014205 familial febrile seizures Diseases 0.000 claims description 2
- 201000007186 focal epilepsy Diseases 0.000 claims description 2
- 230000004777 loss-of-function mutation Effects 0.000 claims description 2
- 230000003211 malignant effect Effects 0.000 claims description 2
- 230000001617 migratory effect Effects 0.000 claims description 2
- 201000001204 progressive myoclonus epilepsy Diseases 0.000 claims description 2
- 208000012216 sick sinus syndrome 1 Diseases 0.000 claims description 2
- 239000008194 pharmaceutical composition Substances 0.000 claims 16
- 239000013598 vector Substances 0.000 claims 8
- 108090000623 proteins and genes Proteins 0.000 claims 7
- 102000004169 proteins and genes Human genes 0.000 claims 7
- 239000013603 viral vector Substances 0.000 claims 1
- 230000001105 regulatory effect Effects 0.000 description 7
- 230000001419 dependent effect Effects 0.000 description 2
- 208000001654 Drug Resistant Epilepsy Diseases 0.000 description 1
- 208000014204 generalized epilepsy with febrile seizures plus 2 Diseases 0.000 description 1
- 238000012986 modification Methods 0.000 description 1
- 230000004048 modification Effects 0.000 description 1
- 238000006467 substitution reaction Methods 0.000 description 1
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2025060382A JP2025098252A (ja) | 2019-02-27 | 2025-04-01 | 病態及び疾患の治療用アンチセンスオリゴマー |
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201962811511P | 2019-02-27 | 2019-02-27 | |
| US62/811,511 | 2019-02-27 | ||
| PCT/US2020/020175 WO2020176776A1 (en) | 2019-02-27 | 2020-02-27 | Antisense oligomers for treatment of conditions and diseases |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2025060382A Division JP2025098252A (ja) | 2019-02-27 | 2025-04-01 | 病態及び疾患の治療用アンチセンスオリゴマー |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2022522205A JP2022522205A (ja) | 2022-04-14 |
| JPWO2020176776A5 true JPWO2020176776A5 (enExample) | 2023-03-07 |
| JP7660515B2 JP7660515B2 (ja) | 2025-04-11 |
Family
ID=72239261
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021550217A Active JP7660515B2 (ja) | 2019-02-27 | 2020-02-27 | 病態及び疾患の治療用アンチセンスオリゴマー |
| JP2025060382A Pending JP2025098252A (ja) | 2019-02-27 | 2025-04-01 | 病態及び疾患の治療用アンチセンスオリゴマー |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2025060382A Pending JP2025098252A (ja) | 2019-02-27 | 2025-04-01 | 病態及び疾患の治療用アンチセンスオリゴマー |
Country Status (10)
| Country | Link |
|---|---|
| US (1) | US20220162605A1 (enExample) |
| EP (1) | EP3931329A4 (enExample) |
| JP (2) | JP7660515B2 (enExample) |
| KR (1) | KR20210134003A (enExample) |
| CN (1) | CN113748209A (enExample) |
| AU (1) | AU2020227825A1 (enExample) |
| CA (1) | CA3131591A1 (enExample) |
| IL (1) | IL285867A (enExample) |
| SG (1) | SG11202109371VA (enExample) |
| WO (1) | WO2020176776A1 (enExample) |
Families Citing this family (4)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| KR102604132B1 (ko) | 2015-12-14 | 2023-11-17 | 콜드스프링하버러보러토리 | 상염색체 우성 정신 지체 5 및 드라베 증후군의 치료를 위한 안티센스 올리고머 |
| SG11202001590RA (en) | 2017-08-25 | 2020-03-30 | Stoke Therapeutics Inc | Antisense oligomers for treatment of conditions and diseases |
| CA3159162A1 (en) * | 2019-12-06 | 2021-06-10 | Isabel AZNAREZ | Antisense oligomers for treatment of conditions and diseases |
| MX2022014151A (es) | 2020-05-11 | 2022-11-30 | Stoke Therapeutics Inc | Oligomeros antisentido de opa1 para tratamiento de afecciones y enfermedades. |
Family Cites Families (4)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CA2612180A1 (en) * | 2005-06-16 | 2006-12-21 | Bionomics Limited | Methods of treatment, and diagnosis of epilepsy by detecting mutations in the scn1a gene |
| ES2863526T3 (es) * | 2010-06-23 | 2021-10-11 | Curna Inc | Tratamiento de enfermedades relacionadas con la subunidad alfa del canal de sodio (SCNA), controlado por voltaje, mediante inhibición de la transcripción antisentido natural a SCNA |
| KR102604132B1 (ko) * | 2015-12-14 | 2023-11-17 | 콜드스프링하버러보러토리 | 상염색체 우성 정신 지체 5 및 드라베 증후군의 치료를 위한 안티센스 올리고머 |
| SG11202001590RA (en) * | 2017-08-25 | 2020-03-30 | Stoke Therapeutics Inc | Antisense oligomers for treatment of conditions and diseases |
-
2020
- 2020-02-27 CN CN202080031771.0A patent/CN113748209A/zh active Pending
- 2020-02-27 SG SG11202109371VA patent/SG11202109371VA/en unknown
- 2020-02-27 KR KR1020217031019A patent/KR20210134003A/ko active Pending
- 2020-02-27 CA CA3131591A patent/CA3131591A1/en active Pending
- 2020-02-27 AU AU2020227825A patent/AU2020227825A1/en active Pending
- 2020-02-27 JP JP2021550217A patent/JP7660515B2/ja active Active
- 2020-02-27 EP EP20763143.3A patent/EP3931329A4/en active Pending
- 2020-02-27 WO PCT/US2020/020175 patent/WO2020176776A1/en not_active Ceased
-
2021
- 2021-08-25 IL IL285867A patent/IL285867A/en unknown
- 2021-08-26 US US17/412,664 patent/US20220162605A1/en active Pending
-
2025
- 2025-04-01 JP JP2025060382A patent/JP2025098252A/ja active Pending
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