Aro Biotherapeutics to Present Updates on ABX1100, a CD71 Centyrin – Gys1 siRNA Conjugate for the Treatment of Pompe Disease at the 20th Annual WORLD Symposium 2024 Meeting

PHILADELPHIA--(BUSINESS WIRE)-- Aro Biotherapeutics, a clinical stage biotechnology company pioneering the development of tissue-targeted genetic medicines, today announced upcoming data presentations highlighting the therapeutic potential of its proprietary CD71 Centyrin – Gys1 siRNA conjugate ABX1100, for the treatment of Pompe Disease. The presentations will take place at the WORLD Symposium 2024 in San Diego, CA, from February 4-6, 2024.

In the upcoming presentations, Bartholomew Pederson, PhD, Aro collaborator and Professor of Medical Education at Ball State University, will present new data in the Pompe Disease mouse model that demonstrates potent and specific reduction of Gys1 mRNA, glycogen synthase activity and glycogen in muscle tissues, and improved impaired treadmill performance in male mice.

“We are excited for the opportunity to share our promising data with the Lysosomal Storage Disease and Pompe communities,” said Steven Nadler, Ph.D., Senior Vice President of Translation Research of Aro Biotherapeutics. “Dr. Pederson is a leading expert in the field of glycogen biology, and working with him affords us the ability to better understand the mechanism of action and the potential disease-modifying impact of our CD71 Centyrin - Gys1 siRNA conjugate for treatment of Pompe disease.”

Dr. Nadler will also present data from non-human primate studies, detailing the preclinical development work supporting progression of ABX1100 into first in human studies, which are now ongoing.

Following are additional details about the conferences and presentations:

Title: A novel Centyrin - siRNA targeting and delivery platform inhibits glycogen synthesis and reduces glycogen levels in skeletal and cardiac muscle in a mouse model of Pompe Disease
Presentation Type: Oral
Presenter: Bartholomew Pederson, Ph.D., Professor of Medical Education at Ball State University
Date: February 6^th, 2024 at 10:30am PST

Title: A novel Centyrin - siRNA targeting and delivery platform inhibits glycogen synthesis and reduces glycogen levels in skeletal and cardiac muscle in a mouse model of Pompe Disease
Presentation Type: Poster
Presenter: Bartholomew Pederson, Ph.D., Professor of Medical Education at Ball State University
Date: February 6^th, 2024 at 3:00pm PST

Title: Nonclinical studies in non-human primates on ABX1100: A Centyrin – siRNA conjugate for the treatment of Pompe disease
Presentation Type: Poster
Presenter: Steven Nadler, PhD, SVP Translational Research at Aro Biotherapeutics
Date: February 6^th, 2024 at 3:00pm PST

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 for tissue-specific targeting of therapeutics for a diverse set of diseases. For more information, visit www.arobiotx.com.

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