TAKING A BOLD & INNOVATIVE APPROACH TO TRANSFORMING PATIENTS’ LIVES
We are a clinical stage company, developing a compelling portfolio of innovative AAV-based genetic medicines for people with serious rare neuromuscular diseases.
We see the patient community as our partners, collaborators, and teachers. We believe that their perspectives should be integrated throughout the drug development process from initiation of a clinical program to its completion and beyond.
The science of making AAV-based genetic medicines is complex. Our robust research laboratory and state-of-the-art vector manufacturing facility puts us at the forefront of developing innovative products. Our bold approach is driven by our commitment to bringing transformative treatments to patients living with serious rare neuromuscular diseases as rapidly as possible.
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