Halting Ornithine Transcarbamylase Deficiency With Recombinant AAV in ChildrEn

计划入组 12 人 · 首次发布于 2021 年 10 月 25 日

Phase I Pilot Study of Liver-Directed Gene Therapy for Partial Ornithine Transcarbamylase Deficiency

首次发布于 1999 年 10 月 19 日

Phase I Study of Adenoviral Vector Mediated Gene Transfer for Ornithine Transcarbamylase in Adults With Partial Ornithine Transcarbamylase Deficiency

计划入组 21 人 · 首次发布于 1999 年 10 月 19 日

OTC gene therapy (eRNA/LNP, ornithine transcarbamylase deficiency)

Ornithine(Primene)

C₅H₁₂N₂O₂

Ornithine carbamoyltransferase deficiency disease therapeutics (iECURE)

Rockwell Medical

United States

Ai-Media Technologies Ltd

United States

Arcturus Therapeutics Initiates Phase 2 Trials for mRNA Therapies Targeting Cystic Fibrosis and OTC Deficiency

Arcturus Therapeutics Poised for Growth with Cystic Fibrosis and OTCD Therapies

Precision BioSciences' ARCUS Platform Shows Promising Clinical Outcomes in Gene Editing Trial