Case Study

DNA Therapy RAC Submission And IND Application

Source: SynteractHCR
DNA Therapy RAC Submission And IND Application

In January 2013, SynteractHCR began working with Tacere Therapeutics (A Benitec Biopharma company) to prepare for an NIH Recombinant DNA Advisory Committee (RAC) meeting prior to submitting an Investigational New Drug (IND) application to the FDA for its TT-034 Hepatitis C therapy.

TT-034 is a transformative therapeutic intended to provide a “one dose cure” with a single injection. In preclinical studies it has shown that it specifically targets and transfects liver cells without causing toxic effects. However, because DNA therapies are relatively new and complex, the FDA seeks additional guidance with regard to human safety, which is the purpose of the RAC. The RAC committee consists of multiple independent reviewers, all of whom are experts in their respective fields and all of whom can ask questions during the presentation.