Orphagenix

OrphageniX is a biotechnology company focused on developing gene-editing therapies for rare, hereditary "orphan" diseases. Founded by pioneers in the field of gene repair, the company utilizes its proprietary Targeted Gene Alteration (TGA) platform, which employs single-stranded oligonucleotides (ssODNs) to correct single-point mutations in the human genome. OrphageniX's primary focus areas include sickle cell anemia, spinal muscular atrophy (SMA), and alpha-1 antitrypsin deficiency. By leveraging the body's natural DNA repair mechanisms, the company aims to provide safer and more effective treatments that correct defective genes at their source without the need for viral vectors or foreign gene insertion.