公开(公告)号：US20250163419A1

公开(公告)日：2025-05-22

申请号：US18958374

申请日：2024-11-25

Applicant: QUELLTX, INC.

Inventor： Graham T. Dempsey ,  Owen McManus ,  Hongkang Zhang ,  David Gerber ,  Pin Liu ,  Dawei Zhang ,  Duncan Brown ,  Sudhir Agrawal ,  Caitlin Lewarch

IPC: C12N15/113 ,  A61K31/7088

Abstract: The invention provides non-opioid pain therapeutic compositions that include an antisense oligonucleotide (ASO) complementary to an identified target on a NaV channel mRNA. The ASO hybridizes to its target RNA and forms a duplex that recruits RNase H to degrade the RNA, thereby downregulating NaV channel synthesis, which inhibits the neuron's ability to contribute to the perception of pain. The ASO targets one of the specific identified targets, and may be provided as a gapmer that includes a central DNA segment flanked by modified RNA wings. When the composition is delivered to dorsal root ganglion (DRG) neurons in vitro, the DRG neurons exhibit a dose-dependent knockdown of NaV1.7, NaV1.8, or NaV1.9.