Government Relations Publications

Gene editing is a process that repairs or changes a gene. Medical researchers are exploring ways to use gene editing to treat or prevent human diseases. This report provides a foundation for understanding the science of gene editing, its potential therapeutic applications and its current U.S. regulatory framework.

Download the White Paper 

The National Center for Advancing Translational Sciences (NCATS) issued a Request for Information on opportunities to increase the efficiency of human gene therapy trials in rare diseases, with particular interest in input regarding the use of viral vectors as platforms for therapeutic gene delivery to specific organs or cell types, and how to most efficiently develop such platforms in clinical trials for the treatment of multiple diseases. The potential impact of a platform approach is great for rare genetic diseases, given the large number of rare diseases that may be impacted by gene therapy and the limited financial resources available for gene therapy trials for each rare disease. ASGCT submitted this response to NCATS regarding rare disease trial efficiencies.

Download the NCATS Response

Committees in Action

The Government Relations Committee and the Clinical Trials & Regulatory Affairs Committee have both been tracking legislation, working with regulatory bodies and advocating for the members of ASGCT.

Legislative Updates

Learn about recent legislative issues that ASGCT is monitoring or taking action on and read more about regularoty changes that affect the field of gene and cell therapy. 

Regulatory Updates

Read about regulatory changes that effect the field of gene and cell therapy. 

21st Annual Meeting
May 16 - 19 | Chicago