Expanding Access and Capacity in Cell and Gene Therapy Clinical Trials: Addressing a Shifting Landscape

The rapid advancement of cell and gene therapies (CGT) continues to reshape the clinical development landscape. As these innovative modalities move beyond ultra-rare diseases and academic institutions, clinical trial stakeholders must contend with a new set of operational realities—chief among them, the need to expand site networks, alleviate investigator and study team burden, and prepare new clinical centers traditionally unengaged in this space to be CGT ready.

Successfully navigating these changes requires a proactive, multifaceted approach that balances innovation with practical execution—considering site infrastructure, institutional alignment, departmental coordination, and staff preparedness at every step.

A Transforming Development Environment

The CGT field has evolved substantially in recent years. While early development efforts focused primarily on rare genetic conditions and oncology—and relied heavily on specialized academic centers—today’s pipeline reflects broader ambition. Increasingly, CGT is being investigated for more prevalent conditions and new patient populations.

This expansion introduces fresh challenges in site identification and readiness. Many experienced centers are nearing capacity, constrained by staffing limitations, heavy protocol volume, and increasingly overstretched Key Opinion Leaders (KOLs). These pain points are particularly acute in gene and cell therapy trials, where study protocols often demand complex pharmacy operations, specialized storage conditions, and extended patient follow-up.

To meet these demands, there’s growing momentum to bring new research centers into the fold—including community-based institutions and regional health systems. Doing so not only broadens access but also supports more inclusive enrollment and greater geographic diversity.

Bridging the Gap for New-to-CGT Sites

While many non-traditional sites express interest in participating in CGT research, the complexity of requirements can be daunting. From biosafety preparation to investigational product storage and administration, the learning curve can appear steep.

Structured onboarding and support can ease this transition, helping sites navigate Institutional Biosafety Committee (IBC) registration, align on IRB governance, and ensure product storage and handling protocols are in place. In many cases, virtual IBC inspections have helped streamline these efforts, reducing delays while ensuring appropriate safety controls are in place.

Beyond infrastructure, cross-functional planning is key. Successful CGT execution requires coordination among departments such as pharmacy, surgery, imaging, and apheresis. Procedural mapping, particularly around IP transport and patient flow including post-dosing requirements, helps reduce operational risk and ensures alignment across clinical and administrative teams.

At the departmental level, best practices include establishing SOPs for gene-modified organisms, aligning informed consent processes, and equipping teams with patient-facing materials that support comprehension and compliance—such as safety cards, immunosuppressant diaries,  or gene therapy-specific education tools.

Readiness Is a Team Sport

Site readiness for CGT trials is not achieved through infrastructure alone. It’s a holistic effort that spans institutional leadership, departmental coordination, and frontline staff preparedness.

Early training—well in advance of site initiation—is a critical enabler. Pharmacy and nursing teams need a clear understanding of cold-chain management, IP preparation, and immunosuppressive therapy protocols. Clinical coordinators and other staff must be equipped to handle adverse events and understand escalation pathways, particularly for first-in-human or first-in-indication studies.

Structured, modular training programs are especially effective when paired with real-time mentorship, “dry run” exercises, and access to protocol experts during initial patient enrollments. These resources help build confidence and consistency across site teams—particularly for those new to CGT studies.

Additionally, ensuring that medically trained staff that are familiar with specific modalities (e.g, CAR-T, AAV, RNA) are available on-call, including after hours, is essential for managing potential product-related events and maintaining patient safety.

Redefining What Makes a High-Potential Site

Prior experience with CGT is no longer the sole marker of readiness. Sites that demonstrate strong institutional coordination, functional infrastructure, and a willingness to invest in training can be excellent candidates—even if they’re entering the space for the first time.

Broadening the site footprint also improves access. While CGT research remains heavily concentrated in biopharma hubs like California, Massachusetts, and the Mid-Atlantic, capable sites in other regions offer significant opportunity for more equitable patient reach and reduced caregiver burden.

Accelerating the Future of CGT Trials

The continued growth of CGT pipelines demands a more inclusive and intentional approach to site strategy. While academic institutions remain essential for early-stage research, scaling programs will increasingly rely on activating and supporting new sites.

By embedding CGT readiness into feasibility planning, investing in infrastructure and training, and reinforcing cross-functional coordination, it’s possible to reduce startup delays, ease operational burdens, and build long-term capability across a broader network of sites.

If you’re preparing for your next CGT program and would like to discuss site strategy, contact our experts today.

ABOUT PREMIER RESEARCH:

Premier Research, a global clinical research, product development, and consulting company, is dedicated to helping innovators transform life-changing ideas and breakthrough science into new medical treatments. We offer strategic solutions across the entire development lifecycle, from pre-clinical through commercialization, specializing in smart study design and full-service clinical trial management.   

Leveraging technology and therapeutic expertise, we deliver clean, conclusive data with a focus on reducing development timelines, securing access to the right patients, and effectively navigating global regulations to ensure submission-ready results.   

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