Aro Biotherapeutics to Present Clinical Update from its Pompe Disease Program at the 22nd Annual WORLDSymposium
Oral presentation to highlight first-ever clinical evidence supporting use of a targeted GYS1 substrate reduction therapy, ABX1100, in patients with late-onset Pompe disease (LOPD)
PHILADELPHIA–(BUSINESS WIRE)–Aro Biotherapeutics, a clinical-stage biotechnology company developing potent and tissue-targeted short-interfering RNA (siRNA) medicines, today announced that it will present interim clinical data from its ongoing Phase 1b clinical trial of ABX1100, a novel investigational therapy for the treatment of late-onset Pompe disease (LOPD), at the 22nd annual WORLDSymposium in San Diego, Calif.
In an oral presentation at the WORLDSymposium, Ozlem Goker-Alpan, M.D., president of the Lysosomal and Rare Disorders Research and Treatment Center in Fairfax, Va., will present preliminary analyses of the safety and tolerability, as well as biomarker and pharmacodynamic activity, of ABX1100 in patients with LOPD who are currently receiving enzyme replacement therapy (ERT). The data provide the first clinical evidence of a GYS1-targeting substrate reduction therapy and its potential utility in patients with LOPD.
The presentation details are as follows:
Title: Safety, tolerability and biological activity of ABX1100, a CD71 Centyrin siRNA conjugate targeting GYS1 in Late-Onset Pompe Disease Patients
Presentation Type: Oral
Presenter: Ozlem Goker-Alpan, M.D., president, Lysosomal and Rare Disorders Research and Treatment Center
Date: Thursday, February 5, 2026 at 1:30pm PST
More information about the Phase 1b trial of ABX1100 is available at ClinicalTrials.gov, using the ID no. NCT06109948.
About ABX1100
ABX1100, an investigational treatment for Pompe disease, is comprised of a CD71 receptor-binding Centyrin conjugated to a short-interfering RNA (siRNA) that specifically interferes with expression of GYS1 messenger RNA (mRNA), thereby reducing levels and overall activity of GYS1, the enzyme responsible for glycogen production, in muscle tissues. ABX1100 has received Orphan Drug Designation and Rare Pediatric Disease status from the United States Food and Drug Administration.
About Pompe Disease
Pompe disease is a rare neuromuscular disorder caused by a genetic deficiency of the acid alpha-glucosidase (GAA), which leads to a toxic buildup of glycogen in the muscle. This buildup leads to progressive loss of muscle function, weakness, and disability that can eventually progress to death from respiratory failure. Late-onset Pompe disease (LOPD) is a subtype of Pompe that has onset after the first year of life. The current standard of care for LOPD is enzyme replacement therapy (ERT), which aims to restore genetically deficient enzymes. Despite the availability of ERT, significant unmet need remains due to limited ERT efficacy and burden of care. ERT requires intravenous infusions that can last as long as 6 hours, multiple times a month.
About Aro Biotherapeutics
Aro Biotherapeutics is a biotechnology company working to develop potent and versatile tissue-targeted short-interfering RNA (siRNA) medicines for the treatment of immune-mediated diseases and diseases with high unmet medical need. The company is developing a wholly owned pipeline of therapeutic candidates for a diverse set of diseases, employing a proprietary protein technology called Centyrins, which are small proteins engineered for exceptional efficiency, versatility, and safety. For more information, visit https://www.AroBiotx.com/.
Contacts
SmithSolve
Elan Schnitzer
973-886-9150
[email protected]
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