[EN] SKELETAL MUSCLE DELIVERY PLATFORMS AND METHODS OF USE<br/>[FR] PLATEFORMES D'ADMINISTRATION À DES MUSCLES SQUELETTIQUES ET MÉTHODES D'UTILISATION
申请人:ARROWHEAD PHARMACEUTICALS INC
公开号:WO2022056269A1
公开(公告)日:2022-03-17
The present disclosure relates to delivery platforms that specifically and efficiently direct payloads to skeletal muscle cells in a subject, in vivo. The delivery platforms diclosed herein include targeting ligands (such as compounds that have affinity for integrins, including alpha- v-beta-6) and pharmacokinetic/pharmacodynamic (PK/PD) modulators, to facilitate the delivery of payloads to cells, including to skeletal muscle cells. Suitable payloads for use in the delivery platforms disclosed herein include RNAi agents, which can be linked or conjugated to the delivery platofrms, and when delivered in vivo, provide for the inhibition of gene expression in skeletal muscle cells. Pharmaceutical compositions that include the skeletal muscle cell delivery platform are also described, as well as methods of use for the treatment of various diseases and disorders where delivery of a therapeutic payload to a skeletal muscle cell is desirable.
本公开涉及将有效载荷有针对性地且高效地引导至体内骨骼肌细胞的传递平台。所述传递平台包括靶向配体(例如具有亲和力的化合物,包括α-v-β-6整合素)和药代动力学/药效学(PK/PD)调节剂,以促进有效载荷传递到细胞,包括骨骼肌细胞。适用于本公开的传递平台的有效载荷包括RNAi剂,可以链接或结合到传递平台上,在体内传递时,可以抑制骨骼肌细胞的基因表达。还描述了包括骨骼肌细胞传递平台的药物组合物,以及用于治疗各种疾病和疾病的方法,其中传递治疗有效载荷至骨骼肌细胞是可取的。