                         SEQUENCE LISTING

<110>  Arrowhead Pharmaceuticals Inc.
 
<120>  Skeletal Muscle Delivery Platforms and Methods of Use
<130>  30689-WO

<150>  63/077,284
<151>  2020-09-11

<160>  8    

<170>  PatentIn version 3.5

<210>  1
<211>  21
<212>  RNA
<213>  Artificial Sequence

<220>
<223>  AD06326 Antisense Strand


<220>
<221>  modified_base
<222>  (1)..(4)
<223>  phosphorothioate linked nucleoside

<220>
<221>  modified_base
<222>  (1)..(1)
<223>  2'-O-methyl corresponding nucleoside

<220>
<221>  modified_base
<222>  (2)..(2)
<223>  2'-fluoro corresponding nucleoside

<220>
<221>  modified_base
<222>  (3)..(3)
<223>  2'-O-methyl corresponding nucleoside

<220>
<221>  modified_base
<222>  (4)..(4)
<223>  2'-fluoro corresponding nucleoside

<220>
<221>  modified_base
<222>  (5)..(5)
<223>  2'-O-methyl corresponding nucleoside

<220>
<221>  modified_base
<222>  (6)..(6)
<223>  2'-fluoro corresponding nucleoside

<220>
<221>  modified_base
<222>  (7)..(11)
<223>  2'-O-methyl corresponding nucleoside

<220>
<221>  modified_base
<222>  (12)..(12)
<223>  2'-fluoro corresponding nucleoside

<220>
<221>  modified_base
<222>  (13)..(13)
<223>  2'-O-methyl corresponding nucleoside

<220>
<221>  modified_base
<222>  (14)..(14)
<223>  2'-fluoro corresponding nucleoside

<220>
<221>  modified_base
<222>  (15)..(15)
<223>  2'-O-methyl corresponding nucleoside

<220>
<221>  modified_base
<222>  (16)..(16)
<223>  2'-fluoro corresponding nucleoside

<220>
<221>  modified_base
<222>  (17)..(17)
<223>  2'-O-methyl corresponding nucleoside

<220>
<221>  modified_base
<222>  (18)..(18)
<223>  2'-fluoro corresponding nucleoside

<220>
<221>  modified_base
<222>  (19)..(19)
<223>  2'-O-methyl corresponding nucleoside

<220>
<221>  modified_base
<222>  (20)..(20)
<223>  phosphorothioate linked nucleoside

<220>
<221>  modified_base
<222>  (20)..(20)
<223>  2'-fluoro corresponding nucleoside

<220>
<221>  modified_base
<222>  (21)..(21)
<223>  2'-O-methyl corresponding nucleoside

<400>  1
uguuacagca agaucauggc c                                                 21

<210>  2
<211>  24
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  AD06326 Sense Strand


<220>
<221>  modified_base
<222>  (1)..(1)
<223>  inverted abasic deoxyribose residue

<220>
<221>  modified_base
<222>  (1)..(1)
<223>  5' end (NH2-C6) modification with phosphorothioate linkage

<220>
<221>  modified_base
<222>  (1)..(2)
<223>  phosphorothioate linked nucleoside

<220>
<221>  misc_feature
<222>  (1)..(1)
<223>  n is a, c, g, t or u

<220>
<221>  modified_base
<222>  (2)..(9)
<223>  2'-O-methyl corresponding nucleoside

<220>
<221>  modified_base
<222>  (10)..(12)
<223>  2'-fluoro corresponding nucleoside

<220>
<221>  modified_base
<222>  (13)..(21)
<223>  2'-O-methyl corresponding nucleoside

<220>
<221>  modified_base
<222>  (22)..(23)
<223>  phosphorothioate linked nucleoside

<220>
<221>  modified_base
<222>  (23)..(23)
<223>  inverted abasic deoxyribose residue

<220>
<221>  modified_base
<222>  (23)..(24)
<223>  C6-SS-C6 linked nucleoside

<220>
<221>  misc_feature
<222>  (23)..(23)
<223>  n is a, c, g, t or u

<220>
<221>  modified_base
<222>  (24)..(24)
<223>  2'-deoxythymidine-3'-phosphate

<400>  2
nggccaugau cuugcuguaa cant                                              24

<210>  3
<211>  21
<212>  RNA
<213>  Artificial Sequence

<220>
<223>  AD06912 Antisense Strand


<220>
<221>  modified_base
<222>  (1)..(4)
<223>  phosphorothioate linked nucleoside

<220>
<221>  modified_base
<222>  (1)..(1)
<223>  2'-O-methyl corresponding nucleoside

<220>
<221>  modified_base
<222>  (2)..(2)
<223>  2'-fluoro corresponding nucleoside

<220>
<221>  modified_base
<222>  (3)..(3)
<223>  2'-O-methyl corresponding nucleoside

<220>
<221>  modified_base
<222>  (4)..(4)
<223>  2'-fluoro corresponding nucleoside

<220>
<221>  modified_base
<222>  (5)..(5)
<223>  2'-O-methyl corresponding nucleoside

<220>
<221>  modified_base
<222>  (6)..(6)
<223>  2'-fluoro corresponding nucleoside

<220>
<221>  modified_base
<222>  (7)..(11)
<223>  2'-O-methyl corresponding nucleoside

<220>
<221>  modified_base
<222>  (12)..(12)
<223>  2'-fluoro corresponding nucleoside

<220>
<221>  modified_base
<222>  (13)..(13)
<223>  2'-O-methyl corresponding nucleoside

<220>
<221>  modified_base
<222>  (14)..(14)
<223>  2'-fluoro corresponding nucleoside

<220>
<221>  modified_base
<222>  (15)..(15)
<223>  2'-O-methyl corresponding nucleoside

<220>
<221>  modified_base
<222>  (16)..(16)
<223>  2'-fluoro corresponding nucleoside

<220>
<221>  modified_base
<222>  (17)..(17)
<223>  2'-O-methyl corresponding nucleoside

<220>
<221>  modified_base
<222>  (18)..(18)
<223>  2'-fluoro corresponding nucleoside

<220>
<221>  modified_base
<222>  (19)..(19)
<223>  2'-O-methyl corresponding nucleoside

<220>
<221>  modified_base
<222>  (20)..(20)
<223>  phosphorothioate linked nucleoside

<220>
<221>  modified_base
<222>  (20)..(20)
<223>  2'-fluoro corresponding nucleoside

<220>
<221>  modified_base
<222>  (21)..(21)
<223>  2'-O-methyl corresponding nucleoside

<400>  3
uguuacagca agaucauggc c                                                 21

<210>  4
<211>  24
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  AD06912 Sense Strand


<220>
<221>  modified_base
<222>  (1)..(1)
<223>  inverted abasic deoxyribose residue

<220>
<221>  modified_base
<222>  (1)..(1)
<223>  5' end (Alk-cyHex) modification with phosphorothioate linkage

<220>
<221>  modified_base
<222>  (1)..(2)
<223>  phosphorothioate linked nucleoside

<220>
<221>  misc_feature
<222>  (1)..(1)
<223>  n is a, c, g, t or u

<220>
<221>  modified_base
<222>  (2)..(9)
<223>  2'-O-methyl corresponding nucleoside

<220>
<221>  modified_base
<222>  (10)..(12)
<223>  2'-fluoro corresponding nucleoside

<220>
<221>  modified_base
<222>  (13)..(21)
<223>  2'-O-methyl corresponding nucleoside

<220>
<221>  modified_base
<222>  (22)..(23)
<223>  phosphorothioate linked nucleoside

<220>
<221>  modified_base
<222>  (23)..(23)
<223>  inverted abasic deoxyribose residue

<220>
<221>  modified_base
<222>  (23)..(24)
<223>  C6-SS-C6 linked nucleoside

<220>
<221>  misc_feature
<222>  (23)..(23)
<223>  n is a, c, g, t or u

<220>
<221>  modified_base
<222>  (24)..(24)
<223>  2'-deoxythymidine-3'-phosphate

<400>  4
nggccaugau cuugcuguaa cant                                              24

<210>  5
<211>  21
<212>  RNA
<213>  Artificial Sequence

<220>
<223>  AD06916 Antisense Strand


<220>
<221>  modified_base
<222>  (1)..(4)
<223>  phosphorothioate linked nucleoside

<220>
<221>  modified_base
<222>  (1)..(1)
<223>  2'-O-methyl corresponding nucleoside

<220>
<221>  modified_base
<222>  (2)..(2)
<223>  2'-fluoro corresponding nucleoside

<220>
<221>  modified_base
<222>  (3)..(3)
<223>  2'-O-methyl corresponding nucleoside

<220>
<221>  modified_base
<222>  (4)..(4)
<223>  2'-fluoro corresponding nucleoside

<220>
<221>  modified_base
<222>  (5)..(5)
<223>  2'-O-methyl corresponding nucleoside

<220>
<221>  modified_base
<222>  (6)..(6)
<223>  2'-fluoro corresponding nucleoside

<220>
<221>  modified_base
<222>  (7)..(11)
<223>  2'-O-methyl corresponding nucleoside

<220>
<221>  modified_base
<222>  (12)..(12)
<223>  2'-fluoro corresponding nucleoside

<220>
<221>  modified_base
<222>  (13)..(13)
<223>  2'-O-methyl corresponding nucleoside

<220>
<221>  modified_base
<222>  (14)..(14)
<223>  2'-fluoro corresponding nucleoside

<220>
<221>  modified_base
<222>  (15)..(15)
<223>  2'-O-methyl corresponding nucleoside

<220>
<221>  modified_base
<222>  (16)..(16)
<223>  2'-fluoro corresponding nucleoside

<220>
<221>  modified_base
<222>  (17)..(17)
<223>  2'-O-methyl corresponding nucleoside

<220>
<221>  modified_base
<222>  (18)..(18)
<223>  2'-fluoro corresponding nucleoside

<220>
<221>  modified_base
<222>  (19)..(19)
<223>  2'-O-methyl corresponding nucleoside

<220>
<221>  modified_base
<222>  (20)..(20)
<223>  phosphorothioate linked nucleoside

<220>
<221>  modified_base
<222>  (20)..(20)
<223>  2'-fluoro corresponding nucleoside

<220>
<221>  modified_base
<222>  (21)..(21)
<223>  2'-O-methyl corresponding nucleoside

<400>  5
uguuacagca agaucauggc c                                                 21

<210>  6
<211>  24
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  AD06326 Sense Strand


<220>
<221>  modified_base
<222>  (1)..(1)
<223>  inverted abasic deoxyribose residue

<220>
<221>  modified_base
<222>  (1)..(1)
<223>  5' end (NH2-C6) modification with phosphorothioate linkage

<220>
<221>  modified_base
<222>  (1)..(2)
<223>  phosphorothioate linked nucleoside

<220>
<221>  misc_feature
<222>  (1)..(1)
<223>  n is a, c, g, t or u

<220>
<221>  modified_base
<222>  (2)..(9)
<223>  2'-O-methyl corresponding nucleoside

<220>
<221>  modified_base
<222>  (10)..(12)
<223>  2'-fluoro corresponding nucleoside

<220>
<221>  modified_base
<222>  (13)..(21)
<223>  2'-O-methyl corresponding nucleoside

<220>
<221>  modified_base
<222>  (22)..(23)
<223>  phosphorothioate linked nucleoside

<220>
<221>  modified_base
<222>  (23)..(23)
<223>  inverted abasic deoxyribose residue

<220>
<221>  modified_base
<222>  (23)..(24)
<223>  C6-SS-C6 linked nucleoside

<220>
<221>  misc_feature
<222>  (23)..(23)
<223>  n is a, c, g, t or u

<220>
<221>  modified_base
<222>  (24)..(24)
<223>  2'-deoxythymidine-3'-phosphate

<400>  6
nggccaugau cuugcuguaa cant                                              24

<210>  7
<211>  21
<212>  RNA
<213>  Artificial Sequence

<220>
<223>  AD06569 Antisense Strand


<220>
<221>  modified_base
<222>  (1)..(4)
<223>  phosphorothioate linked nucleoside

<220>
<221>  modified_base
<222>  (1)..(1)
<223>  2'-O-methyl corresponding nucleoside

<220>
<221>  modified_base
<222>  (1)..(1)
<223>  5' end cyclopropyl phosphonate

<220>
<221>  modified_base
<222>  (2)..(2)
<223>  2'-fluoro corresponding nucleoside

<220>
<221>  modified_base
<222>  (3)..(3)
<223>  2'-O-methyl corresponding nucleoside

<220>
<221>  modified_base
<222>  (4)..(4)
<223>  2'-fluoro corresponding nucleoside

<220>
<221>  modified_base
<222>  (5)..(5)
<223>  2'-O-methyl corresponding nucleoside

<220>
<221>  modified_base
<222>  (6)..(6)
<223>  2'-fluoro corresponding nucleoside

<220>
<221>  modified_base
<222>  (7)..(11)
<223>  2'-O-methyl corresponding nucleoside

<220>
<221>  modified_base
<222>  (12)..(12)
<223>  2'-fluoro corresponding nucleoside

<220>
<221>  modified_base
<222>  (13)..(13)
<223>  2'-O-methyl corresponding nucleoside

<220>
<221>  modified_base
<222>  (14)..(14)
<223>  2'-fluoro corresponding nucleoside

<220>
<221>  modified_base
<222>  (15)..(15)
<223>  2'-O-methyl corresponding nucleoside

<220>
<221>  modified_base
<222>  (16)..(16)
<223>  2'-fluoro corresponding nucleoside

<220>
<221>  modified_base
<222>  (17)..(17)
<223>  2'-O-methyl corresponding nucleoside

<220>
<221>  modified_base
<222>  (18)..(18)
<223>  2'-fluoro corresponding nucleoside

<220>
<221>  modified_base
<222>  (19)..(19)
<223>  2'-O-methyl corresponding nucleoside

<220>
<221>  modified_base
<222>  (20)..(20)
<223>  phosphorothioate linked nucleoside

<220>
<221>  modified_base
<222>  (20)..(20)
<223>  2'-fluoro corresponding nucleoside

<220>
<221>  modified_base
<222>  (21)..(21)
<223>  2'-O-methyl corresponding nucleoside

<400>  7
uguuacagca agaucaugac c                                                 21


<210>  8
<211>  24
<212>  DNA
<213>  Artificial Sequence

<220>
<223>  AD06569 Sense Strand


<220>
<221>  modified_base
<222>  (1)..(1)
<223>  inverted abasic deoxyribose residue

<220>
<221>  modified_base
<222>  (1)..(1)
<223>  5' end (NH2-C6) modification with phosphorothioate linkage

<220>
<221>  modified_base
<222>  (1)..(2)
<223>  phosphorothioate linked nucleoside

<220>
<221>  misc_feature
<222>  (1)..(1)
<223>  n is a, c, g, t or u

<220>
<221>  modified_base
<222>  (2)..(9)
<223>  2'-O-methyl corresponding nucleoside

<220>
<221>  modified_base
<222>  (10)..(12)
<223>  2'-fluoro corresponding nucleoside

<220>
<221>  modified_base
<222>  (13)..(21)
<223>  2'-O-methyl corresponding nucleoside

<220>
<221>  modified_base
<222>  (22)..(23)
<223>  phosphorothioate linked nucleoside

<220>
<221>  modified_base
<222>  (23)..(23)
<223>  inverted abasic deoxyribose residue

<220>
<221>  modified_base
<222>  (23)..(24)
<223>  C6-SS-C6 linked nucleoside

<220>
<221>  misc_feature
<222>  (23)..(23)
<223>  n is a, c, g, t or u

<220>
<221>  modified_base
<222>  (24)..(24)
<223>  2'-deoxythymidine-3'-phosphate

<400>  8
nggucaugau cuugcuguaa cant                                              24


