Armatus Bio, a late-preclinical stage biotech innovator developing vectorized RNAi medicines in neuromuscular disorders, today announced a new publication in Molecular Therapy Advances summarizing the development of a novel, robust in vitro potency assay platform to accelerate the advancement of gene silencing therapies for serious neuromuscular diseases including facioscapulohumeral muscular dystrophy (FSHD) and Charcot-Marie-Tooth disease type 1A (CMT1A).

Precision RNA interference (RNAi) therapies delivered with AAV vectors have emerged as a powerful approach for addressing genetically-driven diseases. Yet a major bottleneck in advancing development has been the lack of standardized, reproducible and scalable potency assays to ensure product consistency, quality, and regulatory compliance.

“With a deep body of knowledge on the mechanisms of engineered microRNAs, we aimed to design a simple, reliable, and broadly applicable assay system that can be implemented early in development and scaled through clinical and commercial stages,” said Scott Harper, PhD, Principal Investigator at the Nationwide Children’s Hospital Center for Gene Therapy and Chief Scientific Advisor to Armatus Bio.

In this study, researchers engineered human cell lines to overcome traditional limitations in in vitro AAV testing. By incorporating the universal AAV receptor (AAVR) and a luciferase-based reporter system linked to disease-relevant gene sequences, the team created a potency assay platform for assessing product strength and stability of gene silencing products. Based on the study, this assay platform delivers:

• Quantifiable, dose-dependent readouts of gene silencing products
• Streamlined assay development or product release methods for gene silencing strategies
• Versatility across multiple gene targets, demonstrating applicability beyond FSHD and CMT1A

“By providing reliable, quantifiable assessments of critical attributes such as lot release, stability and manufacturing comparability, our potency assay technology will contribute to reduced development timelines and improved regulatory readiness,” said Rachel Salzman, DVM, CEO of Armatus Bio. “Importantly, this solves a commonly encountered bottleneck in the early development of precision gene therapies, thus opening accelerated paths to clinical translation to support a wide variety of urgent unmet needs, particularly in rare diseases.”

About Armatus

Armatus Bio is a late-preclinical stage, privately held biotech innovator developing advanced medicines that leverage vectorized RNAi (RNA interference). Armatus’ uniquely specific, engineered microRNAs are noncoding RNAs responsible for regulating gene expression by mirroring innate cellular biogenesis processes without altering the underlying genetic make-up. The company's two lead assets are designed to target neuromuscular disorders: TVR110 for Charcot-Marie-Tooth disease type 1A (CMT1A), and ARM-201 for Facioscapulohumeral Muscular Dystrophy (FSHD), which together affect more than 225,000 people in the U.S. and European Union. In preclinical studies, these investigational drugs demonstrated robust signals of target engagement and biomarker improvement, and both are advancing toward the clinic. For more information, visit www.ArmatusBio.com.

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