What is an Investigational New Drug (IND) application?

An Investigational New Drug (IND) application is a formal submission made to the United States Food and Drug Administration (FDA) that requests permission to begin clinical trials of a new therapeutic in human participants. It is a critical regulatory milestone in the development of any new drug or biologic, including cell-based therapies like RMS’s iPSC-derived ILC therapeutic.

An IND application contains detailed information about the therapy’s composition, preclinical safety and efficacy data, proposed clinical trial design, and the qualifications of the investigators who will conduct the trial. The FDA reviews the submission to determine whether it is safe to proceed to human testing. If the IND is approved, or if the FDA does not place a clinical hold within 30 days, the sponsor may begin the proposed Phase I clinical trial.

For RMS, submitting an IND application represents the culmination of its preclinical development program and the formal beginning of its path to clinical evaluation. RMS is currently preparing its IND submission, a process that reflects the maturity of the company’s preclinical data package and the readiness of its manufacturing and safety documentation. This is a significant milestone in the translation of RMS’s iPSC technology from laboratory research to potential clinical use.

Learn more about the diabetes research that has led us toward clinical trials.