Aro Biotherapeutics

Aro Biotherapeutics is a clinical-stage biotechnology company based in Philadelphia that specializes in developing tissue-targeted genetic medicines. The company's core technology is a proprietary protein platform called Centyrins, which enables efficient and tissue-specific delivery of siRNA and other genetic drugs. Aro's lead candidate, ABX1100, is being developed for the treatment of Pompe disease, a rare genetic disorder characterized by toxic glycogen accumulation in muscles. ABX1100 comprises a CD71 receptor-binding Centyrin conjugated to a small interfering RNA (siRNA) that targets the Gys1 gene, reducing levels and activity of the Gys1 enzyme in muscle tissues. The therapy has shown promising results in preclinical studies, demonstrating potent and muscle-specific reductions in Gys1 mRNA and protein in the Pompe mouse model. In October 2023, Aro initiated a Phase 1 clinical trial of ABX1100 in Canada, with initial data expected in 2024. The US Food and Drug Administration granted ABX1100 Orphan Drug Designation and Rare Pediatric Disease status. Beyond Pompe disease, Aro is exploring the potential of its Centyrin-siRNA conjugate programs for developing new treatments for autoimmune disorders.