The Food and Drug Administration is trying to cut years off the development timeline for cell and gene therapies. In draft guidance released this week, the agency laid out how drugmakers can use existing knowledge, rather than starting from scratch, to speed regulatory submissions for therapies that edit human genes.

The proposed framework encourages sponsors to leverage what is already known about their platforms: chemistry, manufacturing and controls data, nonclinical study results, and clinical information from earlier products. The goal is to reduce redundant testing without cutting corners on safety.

Karim Mikhail, acting director of the FDA’s Center for Biologics Evaluation and Research, said the guidance is meant to get treatments to patients faster, “particularly those living with rare and life-threatening diseases who have few or no other treatment options.”

The guidance dovetails with the FDA’s Plausible Mechanism Framework and a separate draft guidance on safety assessment of genome editing using next-generation sequencing, which recommends methods for evaluating off-target editing risks.

Vijay Kumar, acting director of CBER’s Office of Therapeutic Products, said the approach can “meaningfully streamline development programs and lower the cost barriers that have historically slowed access to these potentially life-changing treatments.”

The FDA is encouraging developers to engage early, even before submitting an investigational new drug application, through INTERACT and pre-IND meetings. The draft guidance is open for public comment for 90 days following publication in the Federal Register.

Source: FDA Press Release