Our programs, projects and other research activities tackle ongoing challenges in translational research so that new treatments and other health solutions can reach people faster.
Bespoke Gene Therapy Consortium (BGTC)
We help lead this public-private partnership program that focuses on developing platforms and standards to speed the development and delivery of gene therapies for rare diseases.
Platform Vector Gene Therapy (PaVe-GT)
Our pilot project is testing the feasibility of increasing the efficiency of gene therapy clinical trials by using the same gene delivery system and manufacturing methods for multiple gene therapies.
Somatic Cell Genome Editing
We help lead this NIH Common Fund program that aims to create and make available high-quality tools for safe and effective genome editing to decrease the time and cost of creating new therapies.