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Oligonucleotide Compositions And Methods Thereof
Abstract: Among other things, the present disclosure provides oligonucleotides, compositions, and methods thereof. Among other things, the present disclosure encompasses the recognition that structural elements of oligonucleotides, such as base sequence, chemical modifications (e.g., modifications of sugar, base, and/or internucleotidic linkages) or patterns thereof, conjugation with additional chemical moieties, and/or stereochemistry [e.g., stereochemistry of backbone chiral centers (chiral internucleotidic linkages)], and/or patterns thereof, can have significant impact on oligonucleotide properties and activities, e.g., knockdown ability, stability, delivery, etc. In some embodiments, the oligonucleotides decrease the expression, activity and/or level of a C9orf72 gene, including but not limited to, one comprising a repeat expansion, or a gene product thereof. In some embodiments, the present disclosure provides methods for treatment of diseases using provided oligonucleotide compositions, for example, in treatment of C9orf72-related disorders.
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Notices, Deadlines & Correspondence
Notices, Deadlines & Correspondence
Patent Information
Application #
Filing Date
04 March 2020
Publication Number
35/2020
Publication Type
INA
Invention Field
BIOTECHNOLOGY
Status
Email
kankrishmefer@gmail.com
Parent Application
Applicants
WAVE LIFE SCIENCES LTD.
7 Straits View #12-00
Marina One East Tower
Singapore 018936
VARGEESE, Chandra
c/o WAVE LIFE SCIENCES LTD.
733 Concord Avenue
Cambridge, Massachusetts 02138
ZHONG, Zhong
c/o WAVE LIFE SCIENCES LTD.
733 Concord Avenue
Cambridge, Massachusetts 02138
IWAMOTO, Naoki
c/o WAVE LIFE SCIENCES LTD.
733 Concord Avenue
Cambridge, Massachusetts 02138
ZHANG, Jason Jingxin
c/o WAVE LIFE SCIENCES LTD.
733 Concord Avenue
Cambridge, Massachusetts 02138
DODART, Jean-Cosme
c/o WAVE LIFE SCIENCES LTD.
733 Concord Avenue
Cambridge, Massachusetts 02138
LIU, Yuanjing
c/o WAVE LIFE SCIENCES LTD.
733 Concord Avenue
Cambridge, Massachusetts 02138
KANDASAMY, Pachamuthu
c/o WAVE LIFE SCIENCES LTD.
733 Concord Avenue
Cambridge, Massachusetts 02138
DIVAKARAMENON, Sethumadhavan
c/o WAVE LIFE SCIENCES LTD.
733 Concord Avenue
Cambridge, Massachusetts 02138
LU, Genliang
c/o WAVE LIFE SCIENCES LTD.
733 Concord Avenue
Cambridge, Massachusetts 02138
MARAPPAN, Subramanian
c/o WAVE LIFE SCIENCES LTD.
733 Concord Avenue
Cambridge, Massachusetts 02138
Inventors
1. VARGEESE, Chandra
c/o WAVE LIFE SCIENCES LTD.
733 Concord Avenue
Cambridge, Massachusetts 02138
2. ZHONG, Zhong
c/o WAVE LIFE SCIENCES LTD.
733 Concord Avenue
Cambridge, Massachusetts 02138
3. IWAMOTO, Naoki
c/o WAVE LIFE SCIENCES LTD.
733 Concord Avenue
Cambridge, Massachusetts 02138
4. ZHANG, Jason Jingxin
c/o WAVE LIFE SCIENCES LTD.
733 Concord Avenue
Cambridge, Massachusetts 02138
5. DODART, Jean-Cosme
c/o WAVE LIFE SCIENCES LTD.
733 Concord Avenue
Cambridge, Massachusetts 02138
6. LIU, Yuanjing
c/o WAVE LIFE SCIENCES LTD.
733 Concord Avenue
Cambridge, Massachusetts 02138
7. KANDASAMY, Pachamuthu
c/o WAVE LIFE SCIENCES LTD.
733 Concord Avenue
Cambridge, Massachusetts 02138
8. DIVAKARAMENON, Sethumadhavan
c/o WAVE LIFE SCIENCES LTD.
733 Concord Avenue
Cambridge, Massachusetts 02138
9. LU, Genliang
c/o WAVE LIFE SCIENCES LTD.
733 Concord Avenue
Cambridge, Massachusetts 02138
10. MARAPPAN, Subramanian
c/o WAVE LIFE SCIENCES LTD.
733 Concord Avenue
Cambridge, Massachusetts 02138
Specification
Documents
Application Documents
| # | Name | Date |
|---|---|---|
| 1 | 202017009198-FORM 3 [30-07-2022(online)].pdf | 2022-07-30 |
| 1 | 202017009198-STATEMENT OF UNDERTAKING (FORM 3) [04-03-2020(online)].pdf | 2020-03-04 |
| 2 | 202017009198-SEQUENCE LISTING(PDF) [04-03-2020(online)].pdf | 2020-03-04 |
| 2 | 202017009198.pdf | 2021-10-19 |
| 3 | abstract.jpg | 2021-10-19 |
| 3 | 202017009198-SEQUENCE LISTING [04-03-2020(online)].txt | 2020-03-04 |
| 4 | 202017009198-PROOF OF RIGHT [04-03-2020(online)].pdf | 2020-03-04 |
| 4 | 202017009198-FORM 3 [24-08-2021(online)].pdf | 2021-08-24 |
| 5 | 202017009198-PRIORITY DOCUMENTS [04-03-2020(online)].pdf | 2020-03-04 |
| 5 | 202017009198-FORM 3 [23-08-2021(online)].pdf | 2021-08-23 |
| 6 | 202017009198-FORM 18 [06-08-2021(online)].pdf | 2021-08-06 |
| 6 | 202017009198-FORM 1 [04-03-2020(online)].pdf | 2020-03-04 |
| 7 | 202017009198-FORM 3 [25-02-2021(online)].pdf | 2021-02-25 |
| 7 | 202017009198-DRAWINGS [04-03-2020(online)].pdf | 2020-03-04 |
| 8 | 202017009198-FORM 3 [03-09-2020(online)].pdf | 2020-09-03 |
| 8 | 202017009198-DECLARATION OF INVENTORSHIP (FORM 5) [04-03-2020(online)].pdf | 2020-03-04 |
| 9 | 202017009198-COMPLETE SPECIFICATION [04-03-2020(online)].pdf | 2020-03-04 |
| 9 | 202017009198-FORM-26 [12-03-2020(online)].pdf | 2020-03-12 |
| 10 | 202017009198-COMPLETE SPECIFICATION [04-03-2020(online)].pdf | 2020-03-04 |
| 10 | 202017009198-FORM-26 [12-03-2020(online)].pdf | 2020-03-12 |
| 11 | 202017009198-DECLARATION OF INVENTORSHIP (FORM 5) [04-03-2020(online)].pdf | 2020-03-04 |
| 11 | 202017009198-FORM 3 [03-09-2020(online)].pdf | 2020-09-03 |
| 12 | 202017009198-DRAWINGS [04-03-2020(online)].pdf | 2020-03-04 |
| 12 | 202017009198-FORM 3 [25-02-2021(online)].pdf | 2021-02-25 |
| 13 | 202017009198-FORM 1 [04-03-2020(online)].pdf | 2020-03-04 |
| 13 | 202017009198-FORM 18 [06-08-2021(online)].pdf | 2021-08-06 |
| 14 | 202017009198-FORM 3 [23-08-2021(online)].pdf | 2021-08-23 |
| 14 | 202017009198-PRIORITY DOCUMENTS [04-03-2020(online)].pdf | 2020-03-04 |
| 15 | 202017009198-FORM 3 [24-08-2021(online)].pdf | 2021-08-24 |
| 15 | 202017009198-PROOF OF RIGHT [04-03-2020(online)].pdf | 2020-03-04 |
| 16 | 202017009198-SEQUENCE LISTING [04-03-2020(online)].txt | 2020-03-04 |
| 16 | abstract.jpg | 2021-10-19 |
| 17 | 202017009198-SEQUENCE LISTING(PDF) [04-03-2020(online)].pdf | 2020-03-04 |
| 17 | 202017009198.pdf | 2021-10-19 |
| 18 | 202017009198-STATEMENT OF UNDERTAKING (FORM 3) [04-03-2020(online)].pdf | 2020-03-04 |
| 18 | 202017009198-FORM 3 [30-07-2022(online)].pdf | 2022-07-30 |