Title:
MUSCLE TARGETING COMPLEXES AND USES THEREOF FOR MODULATION OF GENES ASSOCIATED WITH MUSCLE HEALTH
Document Type and Number:
WIPO Patent Application WO/2023/283531
Kind Code:
A3
Abstract:
Aspects of the disclosure relate to molecular payloads that modulate the expression or activity of genes involved in muscle growth and maintenance (e.g., MSTN, INHBA, ACVR1B, MLCK1, ACVR1, FBXO32, TRIM63, MEF2D, KLF15, MED1, MED13, and/or PPP1R3A), and complexes comprising a muscle-targeting agent covalently linked to such molecular payloads. In some embodiments, the muscle-targeting agent specifically binds to an internalizing cell surface receptor on a muscle cell (e.g., a cardiac muscle cell, a smooth muscle cell, a skeletal muscle cell). In some embodiments, the molecular payload is an oligonucleotide, such as an antisense oligonucleotide or RNAi oligonucleotide.
Inventors:
SUBRAMANIAN ROMESH (US)
QATANANI MOHAMMED (US)
DESJARDINS CODY (US)
BROWN DUNCAN (US)
KOTELIANSKI VICTOR (US)
WEEDEN TIMOTHY (US)
QUINN BRENDAN (US)
NAJIM JOHN (US)
QATANANI MOHAMMED (US)
DESJARDINS CODY (US)
BROWN DUNCAN (US)
KOTELIANSKI VICTOR (US)
WEEDEN TIMOTHY (US)
QUINN BRENDAN (US)
NAJIM JOHN (US)
Application Number:
PCT/US2022/073362
Publication Date:
March 16, 2023
Filing Date:
July 01, 2022
Export Citation:
Assignee:
DYNE THERAPEUTICS INC (US)
International Classes:
A61K39/395; A61K47/68; A61P21/00; C07K14/435; C07K16/18; C07K16/28; C12N15/113
Foreign References:
| US20110191871A1 | 2011-08-04 | |||
| US20190225690A1 | 2019-07-25 | |||
| US20110014122A1 | 2011-01-20 | |||
| US20190153083A1 | 2019-05-23 | |||
| US20130323789A1 | 2013-12-05 |
Attorney, Agent or Firm:
GE, Zhiyun et al. (US)
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