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

Ornithine(Primene)

C₅H₁₂N₂O₂

Ornithine carbamoyltransferase deficiency disease therapeutics (iECURE)

Avalotcagene ontaparvovec

N-(Phosphonoacetyl)-L-Ornithine

C₇H₁₅N₂O₆P

L-Ornithine-L-Aspartate

Rx

Ndelta-(N'-Sulphodiaminophosphinyl)-L-Ornithine

C₅H₁₅N₄O₆PS

CDKL5 deficiency disorder therapeutic program (Vyant Bio Inc/Cyclica Inc)

WWOX gene therapy(Mahzi Therapeutics/ Hebrew University)

CBS-deficiency(Mitorx Therapeutics)