Our strategic approach to gene therapy has positioned us to launch a sustainable multi-platform pipeline of treatments that directly target the genetic mutation in the affected cells for rare, life-threatening childhood disorders, including Fanconi Anemia (FA), Leukocyte Adhesion Deficiency-I (LAD-I), Danon Disease, Pyruvate Kinase Deficiency (PKD), PKP2 arrhythmogenic cardiomyopathy (ACM), BAG3-associated dilated cardiomyopathy (DCM) and multiple undisclosed diseases.