We’re reimagining the potential of human enzymes to change the course of rare metabolic diseases.
Focused on progressive and debilitating disorders with limited treatment options, we leverage our unique protein engineering expertise to modify human enzymes – creating therapeutic catalysts that modulate the chemical imbalances that drive disease. By addressing metabolic deficiencies, we believe our therapies can help restore an element of balance to the lives of the patients and families we serve.
Our wholly-owned pipeline is purposely built to deliver transformative therapeutics for multiple diseases where enzyme approaches have not previously been considered.
Our medicines are investigational. Safety and efficacy have not been established by any agency.
- Research Research
- Ph 1/2
- Ph 3
- PH 1/2