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Oxford-Harrington Rare Disease Scholar Award
October 25, 2023
PHILADELPHIA--(BUSINESS WIRE)--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.