Aro Biotherapeutics
Aro Biotherapeutics, based in Philadelphia, is a biotechnology company developing tissue-targeted genetic medicines using its proprietary Centyrin protein technology.
Company Overview
Aro Biotherapeutics, based in Philadelphia, is a biotechnology company focused on developing tissue-targeted genetic medicines. The company leverages its proprietary Centyrins platform to create therapeutic candidates aimed at treating a variety of diseases. Aro Biotherapeutics is advancing a wholly-owned pipeline while also collaborating with industry partners to utilize Centyrins for tissue-specific targeting.
Centyrins Platform
Aro Biotherapeutics' proprietary Centyrins platform is designed for enhanced efficacy and improved safety in drug delivery. Derived from Tenascin C, a human protein in the extracellular matrix, Centyrins are engineered proteins that facilitate effective targeting and productive delivery of siRNA and other drug payloads to diseased tissues. Centyrins' small size allows for better tissue penetration, lower protein load, sub-cutaneous dosing, and more efficient cargo delivery.
Lead Program: ABX1100 for Pompe Disease
ABX1100 is Aro Biotherapeutics' lead program, designed as a novel treatment for Pompe Disease. It features a CD71 receptor-binding Centyrin conjugated to siRNA, aimed at interfering with Gys1 mRNA expression to reduce enzyme levels in muscle tissues. ABX1100 has received Orphan Drug Designation and Rare Pediatric Disease status from the FDA. In October 2023, Aro Biotherapeutics started a Phase 1 trial in Canada to evaluate its safety, tolerability, pharmacokinetics, and pharmacodynamic biomarkers.
Funding and Investment
Aro Biotherapeutics secured $41.5M in Series B financing in November 2023. The funding round was led by Cowen Healthcare Investments and included participation from existing investors such as Johnson & Johnson Innovation – JJDC, Inc., Northpond Ventures, Healthcap, and BVF Partners, L.P. This investment follows the company's Series A financing round in 2020.
Research and Development
Aro Biotherapeutics is focused on designing and optimizing oligonucleotides for efficient and specific gene knockdown in extrahepatic tissues. The company's research includes Centyrin-siRNA and Centyrin-ASO conjugates. The company's initial internal pipeline targets orphan genetic and immune-mediated diseases. Aro Biotherapeutics is also forming collaborations with industry leaders to build a partnered pipeline aimed at developing new therapies.