Wiskott-Aldrich syndrome gene therapy (Genethon)

WAS gene therapy (lentiviral vector, Wiskott-Aldrich syndrome)

WAS gene-transduced autologous CD34+ stem cell therapy (AAV, Wiskott-Aldrich syndrome)

Trecadrine

Eberconazole

C₁₈H₁₄Cl₂N₂

WAS Protein Stabilizers (XL Therapeutics)

TTR gene editing therapy (Trucode Gene)

CYBB gene editing therapy (Trucode Gene)

PAH gene editing therapy (Trucode Gene)

GENE-202