Hope in a Dose: Cell & Gene Therapy

In this episode, we focus on building the right CMC foundation for cell and gene therapy success. From manufacturing variability and potency assays to comparability and cold chain logistics, the CMC story is often the story regulators focus on.

In this episode, we talk through what sponsors really need to think about when navigating regulatory pathways for cell and gene therapies. We cover everything from early conversations with regulators and managing CMC risk, to using accelerated approval pathways and building a joined up regulatory and operational strategy to help get innovative therapies to patients faster.

In this episode, Fortrea experts discuss how external control arms can enable efficient and ethical cell and gene therapy trials, particularly in rare diseases where traditional control arms are challenging. The conversation highlights study design strategies, regulatory considerations, and how sponsors can use high quality external data to accelerate development, reduce patient burden, and suppor...

In this episode we focus on one of the most dynamic regions in the CGT space: China. Why is this important? China has roughly become a major hub for cell and gene therapy research, driven by regulatory innovation, technological progress, and significant investment with a large patient population and high unmet medical need.

In this episode we tackle one of the most critical aspects of these programs: long-term follow-up. Why is it so important? With CAR-T therapies, lentiviral vector-based treatments, and gene editing approaches requiring up to 15 years of follow-up, we’re looking at a rapidly growing population of patients who will need monitoring for years—sometimes decades. Featuring insights from Gordon Cummings,...

Expanding cell therapy beyond oncology opens new doors for patients—but it also introduces unique challenges in site identification and startup. From competing studies and resource limitations to regulatory nuances and cross-department collaboration, these hurdles can delay timelines if not addressed early. In this episode of Hope in a Dose, host Louise Kearney is joined by Erin Jackson and Rielle...

Cell and gene therapy (CGT) trials are rewriting the rules of early-phase research. Unlike traditional first-in-human studies, these programs involve patients—not healthy volunteers—and often target rare or ultra-rare genetic diseases. That means smaller populations, complex logistics, and long-term follow-up requirements that can span decades. In this episode of Early Phase Challenges in Advanced...

In this episode of podcast series “Hope in a Dose” we discuss the essential elements for successful cell and gene therapy trials: understanding patient eligibility and journey, selecting the right sites, and acting with strategic urgency to accelerate outcomes. Learn how data-driven site selection, operational planning, and collaboration help overcome the unique challenges of CGT studies. Featurin...

In this episode we will discuss topics that are critical to the success of CGT programs: seamless collaboration between CDMOs and CROs. In cell and gene therapy, complexity isn't just in science, it's in the execution. And that's where integrative thinking comes in, where manufacturing and clinical operations work as one, we can accelerate timelines, reduce risk, and optimize costs. In this episod...

Today we're diving into one of the most exciting frontiers in medicine: cell and gene therapy for pediatric diseases. Why is it so important? Part of the answer lies in the fact that cell and gene therapy offers something extraordinary: a cure, not just symptom management. In this episode hear from leading experts about the science, challenges, and human impact of pediatric CGT clinical trials. Jo...

Discover what sets cell and gene therapy (CGT) clinical trials apart. From “one-and-done” treatments to complex logistics and evolving regulations, our first episode of this podcast series explores the unique challenges and transformative potential of CGT studies. Join us as we dive into science, strategy, and stories behind Hope in a Dose.