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Method For Treating Muscular Dystrophy By Targeting Utrophin Gene
Abstract: Polynucleotides comprising the following base sequences: (a) a base sequence encoding a fusion protein of a nuclease-deficient CRISPR effector protein and a transcription activator, and (b) a base sequence encoding a guide RNA targeting a continuous region of 18 to 24 nucleotides in length in a region set forth in SEQ ID NO: 104, 105, 135, 141, 153, 167, or 172 in the expression regulatory region of human Utrophin gene are expected to be useful for treating or preventing DUCHENNE muscular dystrophy or BECKER muscular dystrophy.
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Notices, Deadlines & Correspondence
Notices, Deadlines & Correspondence
Patent Information
Application #
Filing Date
01 June 2021
Publication Number
26/2021
Publication Type
INA
Invention Field
BIO-CHEMISTRY
Status
Email
patent@depenning.com
Parent Application
Applicants
ASTELLAS PHARMA INC.
5-1, Nihonbashi-Honcho 2-chome, Chuo-ku, Tokyo
1038411
MODALIS THERAPEUTICS CORPORATION
3-11-5, Nihonbashi-Honcho, Chuo-ku, Tokyo 1030023
Inventors
1. YOSHIMI, Eiji
c/o Astellas Pharma Inc., 5-1, Nihonbashi-Honcho 2-chome, Chuo-ku, Tokyo
1038411
2. YOSHIOKA, Katsuro
c/o Astellas Pharma Inc., 5-1, Nihonbashi-Honcho 2-chome, Chuo-ku, Tokyo
1038411
3. YAMAGATA, Tetsuya
c/o Modalis Therapeutics Inc.
51 Moulton Street
Cambridge, Massachusetts 02138
4. QIN, Yuanbo
c/o Modalis Therapeutics Inc.
51 Moulton Street
Cambridge, Massachusetts 02138
5. THOMPSON, Iain Robert
c/o Modalis Therapeutics Inc.
51 Moulton Street
Cambridge, Massachusetts 02138
6. KHANNA, Nidhi
c/o Modalis Therapeutics Inc.
51 Moulton Street
Cambridge, Massachusetts 02138
Specification
Documents
Application Documents
| # | Name | Date |
|---|---|---|
| 1 | 202147024347-STATEMENT OF UNDERTAKING (FORM 3) [01-06-2021(online)].pdf | 2021-06-01 |
| 2 | 202147024347-SEQUENCE LISTING(PDF) [01-06-2021(online)].pdf | 2021-06-01 |
| 3 | 202147024347-SEQUENCE LISTING [01-06-2021(online)].txt | 2021-06-01 |
| 4 | 202147024347-PROOF OF RIGHT [01-06-2021(online)].pdf | 2021-06-01 |
| 5 | 202147024347-PRIORITY DOCUMENTS [01-06-2021(online)].pdf | 2021-06-01 |
| 6 | 202147024347-POWER OF AUTHORITY [01-06-2021(online)].pdf | 2021-06-01 |
| 7 | 202147024347-FORM 1 [01-06-2021(online)].pdf | 2021-06-01 |
| 8 | 202147024347-DRAWINGS [01-06-2021(online)].pdf | 2021-06-01 |
| 9 | 202147024347-DECLARATION OF INVENTORSHIP (FORM 5) [01-06-2021(online)].pdf | 2021-06-01 |
| 10 | 202147024347-COMPLETE SPECIFICATION [01-06-2021(online)].pdf | 2021-06-01 |
| 11 | 202147024347-CLAIMS UNDER RULE 1 (PROVISIO) OF RULE 20 [01-06-2021(online)].pdf | 2021-06-01 |
| 12 | 202147024347-RELEVANT DOCUMENTS [08-07-2021(online)].pdf | 2021-07-08 |
| 13 | 202147024347-MARKED COPIES OF AMENDEMENTS [08-07-2021(online)].pdf | 2021-07-08 |
| 14 | 202147024347-FORM 13 [08-07-2021(online)].pdf | 2021-07-08 |
| 15 | 202147024347-Annexure [08-07-2021(online)].pdf | 2021-07-08 |
| 16 | 202147024347-AMMENDED DOCUMENTS [08-07-2021(online)].pdf | 2021-07-08 |
| 17 | 202147024347-FORM-26 [24-08-2021(online)].pdf | 2021-08-24 |
| 18 | 202147024347.pdf | 2021-10-18 |
| 19 | 202147024347-FORM 3 [25-11-2021(online)].pdf | 2021-11-25 |
| 20 | 202147024347-FORM 3 [01-06-2022(online)].pdf | 2022-06-01 |
| 21 | 202147024347-Correspondence_Form1, Power of Attorney_23-09-2022.pdf | 2022-09-23 |
| 22 | 202147024347-RELEVANT DOCUMENTS [15-11-2022(online)].pdf | 2022-11-15 |
| 23 | 202147024347-MARKED COPIES OF AMENDEMENTS [15-11-2022(online)].pdf | 2022-11-15 |
| 24 | 202147024347-FORM 18 [15-11-2022(online)].pdf | 2022-11-15 |
| 25 | 202147024347-FORM 13 [15-11-2022(online)].pdf | 2022-11-15 |
| 26 | 202147024347-AMMENDED DOCUMENTS [15-11-2022(online)].pdf | 2022-11-15 |
| 27 | 202147024347-FORM 3 [27-02-2023(online)].pdf | 2023-02-27 |