top of page


Aro Biotherapeutics Announces Start of First in Human Study with ABX1100, a Novel First in Class Centyrin-siRNA Conjugate in Development for Pompe Disease

Philadelphia, PA, October 25, 2023 ---  Aro Biotherapeutics, a clinical stage biotechnology company pioneering the development of tissue-targeted genetic medicines, today announced the enrollment of the first subject in its Phase 1 trial of ABX1100, Aro’s lead Centyrin-siRNA program for the treatment of Pompe Disease.  ABX1100 is a first-in-class molecule representing a new approach to reducing toxic levels of glycogen that build up in muscles of patients with Pompe Disease.

“We are thrilled to advance ABX1100 into the clinic,” said Mittie Doyle, MD, FACR, Chief Medical Officer of Aro Biotherapeutics. “Initiating our Phase 1 study represents a critical step toward our goal of transforming the lives of patients with Pompe Disease.”

The Phase 1 trial, which has initiated in Canada, will commence with a placebo-controlled, double-blinded, single ascending dose portion in normal, healthy volunteers.  The study is designed to assess safety, tolerability, pharmacokinetics along with various pharmacodynamic biomarkers that will be evaluated to demonstrate target engagement.

“Our Phase 1 study of ABX1100 provides an excellent opportunity to demonstrate the potential of our Centyrin-siRNA platform, for targeting siRNAs to tissues beyond the liver,” said Susan Dillon, PhD, President, and Chief Executive Officer of Aro Biotherapeutics.  “We are proud of the work our team has accomplished from discovery through initial clinical testing of ABX1100 and are motivated by our shared goal of bringing a new therapy with unsurpassed efficacy, safety and durability to patients with Pompe Disease.” 

Pompe Disease is a rare, genetic lysosomal storage disease characterized by toxic buildup of glycogen that drives debilitating, progressive muscle weakness and ultimately may result in loss of ambulation and ability to respire independently. Aro has previously presented preclinical data in the Pompe mouse model underscoring the potential of ABX1100 to reduce extra-hepatic glycogen levels via muscle-specific delivery of an siRNA targeting Glycogen Synthase 1 (Gys1), the major enzyme responsible for glycogen synthesis in muscle.  The company anticipates presenting data from the Phase 1 study in 2024.

About ABX1100

ABX1100, a novel potential treatment for Pompe Disease, is comprised of a CD71 receptor binding Centyrin conjugated to a small interfering RNA (siRNA) which specifically interferes with expression of Gys1 mRNA, thereby reducing levels and overall activity of the Gys1 enzyme in muscle tissues.   ABX1100 demonstrated a favorable safety profile in GLP toxicology studies, with durable reductions in Gys1 mRNA in muscles supporting the potential for monthly or quarterly dosing.   ABX1100 has received Orphan Drug Designation and Rare Pediatric Disease status from the United States Food and Drug Administration.


About Aro Biotherapeutics

Based in Philadelphia, Aro Biotherapeutics is a biotechnology company pioneering the development of tissue-targeted genetic medicines with a platform based on a proprietary protein technology called Centyrins. The company is developing a wholly-owned pipeline of Centyrin-based therapeutic candidates and is working with industry partners to leverage Centyrins for tissue-specific targeting of therapeutics for a diverse set of diseases.


Media Contact

Mike Beyer

Sam Brown Inc.


bottom of page