Back to results
US 2024/0102017 A1Published application
Oligonucleotide compositions and methods thereof
AssigneeAmylyx Pharmaceuticals Inc.
InventorsJoshua Barry Cohen, Justin Bernard Klee, Duncan Brown
Life of this patent
Estimating the term…
Abstract
Among other things, the present disclosure provides oligonucleotides targeting calpain-2 and compositions thereof. In some embodiments, the present disclosure provides methods for preventing or treating various conditions, disorders or diseases.
Claims40
An oligonucleotide, wherein:
- the base sequence of the oligonucleotide comprises 10 or more contiguous nucleobases of ATCAGTTTCTGTAGGCTTCC (SEQ ID NO:
- 1), GGCATACTGGTTCAGTTGAT (SEQ ID NO 2), GCTCAGGTCAGGCAGTGGTT (SEQ ID NO:
- 3), GAGAGCCTTTTTGCAGAGCT (SEQ ID NO:
- 4), TCCAGCTCTGTGCCTCTAGT (SEQ ID NO:
- 5), GTTCCAGCTTGGGCAGTTGT (SEQ ID NO:
- 6), or GGAAGCTTAGTCCTTGGCTG (SEQ ID NO:
- 7), wherein each T is optionally and independently replaced with U;
- and
- the oligonucleotide comprises a modified nucleobase, a modified sugar or a modified internucleotidic linkage.
6 more elements
An oligonucleotide having the structure of:
- (SEQ ID NO:
- 116)
- /52MOErA/*/i2MOErT/*/i2MOErC/*/i2MOErA/*/i2MOErG/
- *T*T*T*/iMe-dC/*T*G*T*A*G*G*/i2MOErC/*/i2MOErT/*/
- i2MOErT/*/i2MOErC/*/32MOErC/,
- (SEQ ID NO:
- 141)
- /52MOErG/*/i2MOErG/*/i2MOErC/*/i2MOErA/*/i2MOErT/
- *A*/iMe-dC/*T*G*G*T*T*/iMe-dC/*A*G*/i2MOErT/*/
- i2MOErT/*/i2MOErG/*/i2MOErA/*/32MOErT/,
- (SEQ ID NO:
- 173)
- /52MOErG/*/i2MOErC/*/i2MOErT/*/i2MOErC/*/i2MOErA/
- *G*G*T*/iMe-dC/*A*G*G*/iMe-dC/*A*G*/i2MOErT/*/
- i2MOErG/*/i2MOErG/*/i2MOErT/*/32MOErT/,
- (SEQ ID NO:
- 114)
- /52MOErG/*/i2MOErA/*/i2MOErG/*/i2MOErA/*/i2MOErG/
- */iMe-dC/*/iMe-dC/*T*T*T*T*T*G*/iMe-dC/*A*/
- i2MOErG/*/i2MOErA/*/i2MOErG/*/i2MOErC/*/32MOErT/,
- (SEQ ID NO:
- 179)
- /52MOErT/*/i2MOErC/*/i2MOErC/*/i2MOErA/*/i2MOErG/
- */iMe-dC/*T*/iMe-dC/*T*G*T*G*/iMe-dC/*/iMe-dC/
- *T*/i2MOErC/*/i2MOErT/*/i2MOErA/*/i2MOErG/*/
- 32MOErT/,
- (SEQ ID NO:
- 117)
- /52MOErG/*/i2MOErT/*/i2MOErT/*/i2MOErC/*/i2MOErC/
- *A*G*/iMe-dC/*T*T*G*G*G*/iMe-dC/*A*/i2MOErG/*/
- i2MOErT/*/i2MOErT/*/i2MOErG/*/32MOErT/,
- or
- (SEQ ID NO:
- 138)
- /52MOErG/*/i2MOErG/*/i2MOErA/*/i2MOErA/*/i2MOErG/
- */iMe-dC/*T*T*A*G*T*/iMe-dC/*/iMe-dC/*T*T*/
- i2MOErG/*/i2MOErG/*/i2MOErC/*/i2MOErT/*/32MOErG/,
- or a salt thereof, wherein:
- each of A, T and G is independently deoxyadenosine, thymidine, and deoxyguanosine, respectively:
36 more elements
A composition comprising:
- an oligonucleotide or a salt thereof, and
- one or more diastereomers of the oligonucleotide with respect to chiral linkage phosphorus, or one or more salts of the diastereomers,
- wherein the oligonucleotide is
- (SEQ ID NO:
- 116)
- /52MOErA/*/i2MOErT/*/i2MOErC/*/i2MOErA/*/i2MOErG/
- *T*T*T*/iMe-dC/*T*G*T*A*G*G*/i2MOErC/*/i2MOErT/*/
- i2MOErT/*/i2MOErC/*/32MOErC/,
- (SEQ ID NO:
- 141)
- /52MOErG/*/i2MOErG/*/i2MOErC/*/i2MOErA/*/i2MOErT/
- *A*/iMe-dC/*T*G*G*T*T*/iMe-dC/*A*G*/i2MOErT/*/
- i2MOErT/*/i2MOErG/*/i2MOErA/*/32MOErT/,
- (SEQ ID NO:
- 173)
- /52MOErG/*/i2MOErC/*/i2MOErT/*/i2MOErC/*/i2MOErA/
- *G*G*T*/iMe-dC/*A*G*G*/iMe-dC/*A*G*/i2MOErT/*/
- i2MOErG/*/i2MOErG/*/i2MOErT/*/32MOErT/,
- (SEQ ID NO:
- 179)
- /52MOErT/*/i2MOErC/*/i2MOErC/*/i2MOErA/*/i2MOErG/
- */iMe-dC/*T*/iMe-dC/*T*G*T*G*/iMe-dC/*/iMe-dC/
- *T*/i2MOErC/*/i2MOErT/*/i2MOErA/*/i2MOErG/*/
- 32MOErT/,
- (SEQ ID NO:
- 117)
- /52MOErG/*/i2MOErT/*/i2MOErT/*/i2MOErC/*/i2MOErC/
- *A*G*/iMe-dC/*T*T*G*G*G*/iMe-dC/*A*/i2MOErG/*/
- i2MOErT/*/i2MOErT/*/i2MOErG/*/32MOErT/,
- (SEQ ID NO:
- 138)
- /52MOErG/*/i2MOErG/*/i2MOErA/*/i2MOErA/*/i2MOErG/
- */iMe-dC/*T*T*A*G*T*/iMe-dC/*/iMe-dC/*T*T*/
- i2MOErG/*/i2MOErG/*/i2MOErC/*/i2MOErT/*/32MOErG/,
- or
- (SEQ ID NO:
- 114)
- /52MOErG/*/i2MOErA/*/i2MOErG/*/i2MOErA/*/i2MOErG/
- */iMe-dC/*/iMe-dC/*T*T*T*T*T*G*/iMe-dC/*A*/
- i2MOErG/*/i2MOErA/*/i2MOErG/*/i2MOErC/*/32MOErT/,
- wherein:
- each of A, T and G is independently deoxyadenosine, thymidine, and deoxyguanosine, respectively:
39 more elements
-24. (canceled)
(canceled)
-31. (canceled)
-44. (canceled)
-49. (canceled)
(canceled)
-55. (canceled)
(canceled)
Dependencies are inferred from the claim text.