FDA prior knowledge guide seeks to speed cell, gene therapy

In the latest part of its plan to cut barriers to cell and gene therapy (CGT) development, the FDA has published draft guidance to help developers draw on "existing science" to reduce the need for testing and bring them to patients more quickly.

The new document sets out the regulator's thinking on how CGT sponsors can use publicly available information and established knowledge – such as chemistry, manufacturing and controls (CMC) data, nonclinical studies, and clinical information – to "streamline" regulatory submissions.

The move comes after a series of other initiatives announced by the FDA to lighten the regulatory load on sponsors of new therapies for diseases with no or limited treatment options, including a plausible mechanism framework for ultra-rare diseases with too few patients to make conventional clinical trials feasible, and measures to make it easier to manufacture CGTs.

It also sits alongside draft guidance published in April on methods for evaluating off-target editing risks for genome-editing therapies, also aimed at accelerating the development of individualised therapies.

Karim Mikhail, who is acting director of the FDA's Center for Biologics Evaluation and Research (CBER) following the departure of Vinay Prasad in March, said the aim of the prior knowledge guidance is to "get safe and effective cell and gene therapies to patients faster, particularly those living with rare and life-threatening diseases who have few or no other treatment options."

The initial version of the guidance also focuses on genome-editing products, but the principles may also be applicable to other CGTs, including adeno-associated viral (AAV) vectors, as well as nanoparticle-based and ex vivo-modified cell-based gene therapies that do not involve gene editing, according to the FDA.

Sources of prior knowledge could include internal company platforms, third-party platform data submitted to the FDA via master files, publicly available resources, or materials generated by collaborative efforts between academic institutions, industry, and other stakeholders, according to the guidance.

"Leveraging prior knowledge does not mean lowering the bar; it means raising our collective efficiency while maintaining the highest standards of safety and efficacy," said Vijay Kumar, acting director of the CBER's Office of Therapeutic Products.

"For patients living with serious or rare diseases, time matters," he added. "We encourage developers to engage with this guidance because their perspectives are essential to shaping a regulatory framework that works for everyone, and most importantly, for the patients who are counting on us."

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