Bringing Targeted In Vivo Therapies to Patients

A de-risking approach

Understanding how a drug is working in humans, by assessing initial efficacy and endpoints in exploratory patient populations, is crucial to our de-risking approach in early stage trials. Once we have identified the optimal patient population and signal, we can scale up to later phase trials with confidence. By overseeing early phase trials of viral-vector therapies, we are able to optimize the type and quality of data gathered.

Insights spur future clinical development

As an added benefit, our experience in early phase trials provides information that allows us to strategically innovate during future rounds of research and development and manufacturing. Insights gained during preliminary phase 1 trials can be used to improve upon existing manufacturing processes, develop novel treatment approaches, and aid in the development of future drugs.