Developing a novel gene therapy requires a level of comfort with change and unpredictability. The path to regulatory approval is rarely straightforward, and roadblocks can enter at any turn. Premier Research was approached by a sponsor of an adeno-associated virus (AAV)-based gene silencing technology to conduct a natural history study, with a goal to identify eligible patients for a first-in-human (FIH) dosing study. When regulatory challenges arose amidst opening the sponsor’s investigational new drug (IND) application with the FDA, the partnership with Premier expanded to encompass support from our regulatory experts. This multi-dimensional approach ensured tight communication and seamless coordination across the natural history study and the IND process, enabling an on-time launch of the FIH study with no delays in dosing the first patient.
