Aro Biotherapeutics

Aro Biotherapeutics, headquartered in Philadelphia, is a pioneering biotechnology company focused on the development of tissue-targeted genetic medicines. The company’s innovative platform is based on proprietary Centyrin protein technology, which enables effective targeting and delivery of therapeutic agents to diseased tissues. Aro Biotherapeutics is advancing a wholly-owned pipeline of Centyrin-based therapeutic candidates and collaborating with industry partners to leverage Centyrins for tissue-specific targeting across a diverse range of diseases.

In November 2023, Aro Biotherapeutics secured $41.5 million in Series B financing, led by Cowen Healthcare Investments with participation from existing investors such as Johnson & Johnson Innovation – JJDC, Inc., Northpond Ventures, Healthcap, and BVF Partners, L.P. The company’s lead program, ABX1100, is a novel potential treatment for Pompe Disease. ABX1100 combines a CD71 receptor binding Centyrin with a small interfering RNA (siRNA) to specifically interfere with the expression of Gys1 mRNA, reducing the levels and activity of the Gys1 enzyme in muscle tissues. ABX1100 has received Orphan Drug Designation and Rare Pediatric Disease status from the United States Food and Drug Administration.

Aro Biotherapeutics announced the commencement of a first-in-human study with ABX1100 in October 2023. The Phase 1 trial, conducted in Canada, aims to assess the safety, tolerability, pharmacokinetics, and various pharmacodynamic biomarkers of ABX1100. The company is developing a novel approach to Pompe Disease known as substrate reduction therapy (SRT) and has capabilities to design and optimize oligonucleotides for highly efficient and specific gene knockdown in extrahepatic tissues using both Centyrin-siRNA and Centyrin-ASO conjugates.

Aro Biotherapeutics’ Centyrin platform is designed for enhanced efficacy and improved safety, featuring small size for better tissue penetration, lower protein load, subcutaneous dosing, and more efficient cargo delivery. Centyrins are small, engineered proteins derived from Tenascin C, a human protein found in the extracellular matrix of various tissues. The company holds exclusive rights to the extensive Centyrin intellectual property portfolio and is building a product pipeline focused on orphan genetic and immune-mediated diseases. Additionally, Aro Biotherapeutics is forming collaborations with industry leaders to develop new therapies, leveraging its Centyrin platform for targeted delivery of oligonucleotides and potentially other drug payloads to various cell types.