Clinical Progression & Natural History Research
Partnering with academic centres, clinicians, and patient advocates to advance therapeutic readiness and natural-history understanding....
Muscle Biology & Pathology
Scalable, modular gene therapy constructs engineered to accelerate programs across neuromuscular and metabolic diseases....
Gene Replacement Technology
A first-in-class therapeutic designed to restore functional CHKB expression using rAAV9 for targeted muscle, heart, and CNS delivery....
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