Our Clinical Trials

Deep scientific and clinical understanding drive our approach to clinical development. Our strategy is to identify synergistic indications and to pursue treatments for conditions where there are a large unmet medical need and a high potential for a meaningful benefit with gene therapy.

Through our partnerships with leading institutions, we have gained significant clinical access, providing us with an understanding of disease impact and progress as well as providing an advantage in clinical trial enrollment. This access has allowed us to scale a clinical strategy across multiple programs while still maintaining our focus on therapies for individual patients.

Through the strength of our team, we strive each day to deliver on the promise of combination therapy, bringing potentially curative therapies to patients living with devastating diseases.

Clinical Trials


Cohort study on patients leading to severe cases of MS


A regional study of co-morbidities to characterize cognitive decline within early stages of Alzheimer’s diagnosis


Condition characterization study of patients diagnosed with Disease X.

Better Health Outcomes is Our Mission