Aro’s protein-siRNA conjugate for Pompe disease; plus Rheos, Mozart and more

Aro Biotherapeutics Co. showed its muscle-targeted Centyrin-GYS1 siRNA conjugate, ABX1100, reduced toxic accumulation of muscle glycogen in a mouse model of Pompe disease. Aro revealed at the TIDES USA 2022 meeting, which covers therapeutic oligos, peptides, mRNA and genome editing products, that ABX1100 reduced GYS1 mRNA and protein in skeletal muscle. Enzyme replacement therapies, the only FDA approved method to treat Pompe disease, are limited in safety and efficacy because they often don’t reach skeletal muscle.

Aro, a spinout of Johnson & Johnson (NYSE:JNJ), is developing a pipeline of Centyrins: small, stable, soluble proteins that allow for tissue-specific delivery of conjugated drug payloads via the addition of antigen-specific binding sites. The foundational Centyrin technology was developed through Janssen Pharmaceuticals’ Centyrex unit...