Abstract: Among other things, the present disclosure provides various oligonucleotide technologies including chirally controlled oligonucleotide compositions and technologies for manufacturing such oligonucleotide compositions. In some embodiments, a method is a method of treatment or prevention of Huntington's Disease in a subject in need thereof; a method of allele-specific knockdown of a mutant Huntingtin transcript in a subject; a method for delaying the onset of and/or reducing the severity of at least one symptom of Huntington's Disease in a subject with Huntington's Disease; a method of reducing the expression, level, amount and/or activity of a mutant Huntingtin gene or a gene product thereof; and/or a method of preparation of a medicament for treatment of Huntington's Disease, wherein the method pertains to the use of an oligonucleotide described herein, administered at a dose described herein. In some embodiments, the present disclosure provides doses, dosages, and formulations of an oligonucleotide described herein.
| # | Name | Date |
|---|---|---|
| 1 | 202517015214-STATEMENT OF UNDERTAKING (FORM 3) [21-02-2025(online)].pdf | 2025-02-21 |
| 3 | 202517015214-Sequence Listing in PDF [21-02-2025(online)].pdf | 2025-02-21 |
| 4 | 202517015214-REQUEST FOR EXAMINATION (FORM-18) [21-02-2025(online)].pdf | 2025-02-21 |
| 5 | 202517015214-POWER OF AUTHORITY [21-02-2025(online)].pdf | 2025-02-21 |
| 6 | 202517015214-NOTIFICATION OF INT. APPLN. NO. & FILING DATE (PCT-RO-105-PCT Pamphlet) [21-02-2025(online)].pdf | 2025-02-21 |
| 7 | 202517015214-FORM 18 [21-02-2025(online)].pdf | 2025-02-21 |
| 8 | 202517015214-FORM 1 [21-02-2025(online)].pdf | 2025-02-21 |
| 9 | 202517015214-DRAWINGS [21-02-2025(online)].pdf | 2025-02-21 |
| 10 | 202517015214-DECLARATION OF INVENTORSHIP (FORM 5) [21-02-2025(online)].pdf | 2025-02-21 |
| 11 | 202517015214-COMPLETE SPECIFICATION [21-02-2025(online)].pdf | 2025-02-21 |
| 12 | 202517015214-Proof of Right [06-03-2025(online)].pdf | 2025-03-06 |
| 13 | 202517015214-FORM 3 [14-08-2025(online)].pdf | 2025-08-14 |