Personalized Antisense Oligonucleotide Therapy for Rare Pediatric Genetic Disease: SCN2A

计划入组 1 人 · 首次发布于 2024 年 3 月 18 日

Antisense Oligonucleotide for Spinal Muscular Atrophy

计划入组 1,000 人 · 首次发布于 2022 年 1 月 11 日

Safety and Efficacy of Mutation-targeted Precision Genetic Therapy for Ataxia-Telangiectasia (A-T)

计划入组 10 人 · 首次发布于 2025 年 10 月 10 日

nL-CHCHD-001

GFAP antisense oligonucleotide therapy

USH2A stereopure antisense oligonucleotide therapy

Antisense Therapeutics Ltd

METABOLIC THERAPY, INC.

United States

Everads Therapy Ltd.

Israel

Antisense Oligonucleotide Therapy Reverses Developmental Defects in Spinal Muscular Atrophy Organoids

Aptar and Aceso Therapeutics Partner to Advance Inhaled Antisense Oligonucleotide Therapy for Cystic Fibrosis

Vanda Pharmaceuticals Secures FDA Rare Pediatric Disease Designation for Antisense Oligonucleotide Therapy VCA-894A in CMT2S