Where Biomarkers And Gene Therapy Trials Intersect: Benefits Abound

By Kenneth Ndugga-Kabuye

We are currently witnessing a pivotal era in clinical research within the field of gene therapies. These intricate treatments offer a beacon of hope to more than 30 million individuals in the United States who grapple with over 1000 rare diseases, many of which are life-threatening and, unfortunately, lack viable treatment options¹.

To attain regulatory approval, gene therapy products must adhere to the same rigorous standards as other biologics or pharmaceuticals, necessitating substantial evidence of safety and efficacy garnered through clinical investigations. Given the enduring impact of gene therapies, regulators place a premium on observing a measurable reduction in irreversible morbidity or mortality during clinical studies. The careful selection of a biomarker for a trial can serve as a valuable indicator of a product's efficacy.

In a bid to offer regulatory guidance to sponsors navigating the intricate path to approval, the U.S. FDA's Office of Tissues and Advanced Therapies (OTAT) within the Center for Biologics Evaluation Research initiated a virtual Town Hall series dedicated to the discussion of OTAT-regulated products. The town hall session held on February 7, 2023, was centered around gene therapy clinical development for rare diseases and featured insights from OTAT on the optimal utilization of biomarker endpoints in early-phase studies.

In this article, the author sheds light on several pivotal insights regarding this topic, encompassing the selection of the appropriate biomarker, the pursuit of Accelerated Approval, leveraging biomarkers for dose determination, and the potential for streamlining the duration of clinical trials.