Cell and gene therapy clinical trials sit at the forefront of advanced medicine, with the potential to transform treatment pathways in oncology, rare disease, immunology and other complex therapeutic areas. PHARMExcel’s Cell and Gene Therapy CRO Services are focused on the clinical trial stage, helping sponsors navigate the operational, regulatory and governance challenges that arise once a therapy is ready to enter human studies.
Our cross-functional teams combine therapeutic understanding, regulatory strategy and tailored clinical operations to help sponsors plan and deliver Advanced Therapy Medicinal Product (ATMP) trials in a way that is compliant, proportionate and workable in practice. From first-in-human and early phase studies through to later-stage clinical trials, we provide the governance and oversight needed to support robust, high-quality delivery.
Expertise in advanced therapy medicinal products (ATMPs)
Cell and gene therapy studies bring a level of complexity that goes beyond many conventional trials. Investigational products may involve autologous or allogeneic cell therapies, ex vivo gene transfer approaches or other advanced therapy modalities, each with distinct handling requirements, safety considerations and clinical pathways. These studies often take place in highly specialised centres, involve small or vulnerable patient populations and require close coordination across sponsors, sites and service partners.
PHARMExcel helps sponsors navigate these challenges through practical governance and oversight at the clinical trial stage. Our role is to ensure that protocols, site plans and operational processes reflect the realities of ATMP delivery, while maintaining the scientific integrity and patient protections expected in these high-risk, high-value studies.
Regulatory strategy for cell and gene therapy
Advanced therapy medicinal products are subject to complex and evolving regulatory expectations. PHARMExcel provides regulatory strategy support to help sponsors prepare for MHRA and EMA interactions, shape submissions for clinical trial approval and ensure that documentation reflects the unique safety, handling and follow-up requirements of ATMP studies.
Our teams help sponsors navigate first-in-human and early phase regulatory considerations, risk-based approaches to trial oversight and the longer-term monitoring expectations that often apply in cell and gene therapy research. The focus is always on preparing clear, well-structured regulatory packages that support compliant and realistic trial conduct.
Clinical operations and ATMP trial delivery
The operational demands of cell and gene therapy studies require careful planning and disciplined execution. PHARMExcel provides governance and oversight across site start-up, study management, monitoring and quality processes, helping sponsors align their protocol with the right specialist centres and delivery pathways.
Where trials involve chain of identity, chain of custody, time-sensitive handling requirements or complex coordination between collection, treatment and administration sites, we work with sponsors and partners to ensure that these processes are built into study operations in a way that is robust and auditable. Our role is not to carry out laboratory or manufacturing functions, but to ensure that the clinical trial environment is ready to support the safe and compliant delivery of the investigational product.
Data, oversight and long-term follow-up
Cell and gene therapy trials often require intensive safety oversight, detailed data review and long-term follow-up arrangements that reflect the nature of the investigational product. PHARMExcel provides the monitoring, data oversight and governance needed to support reliable trial conduct, clear safety reporting and robust documentation throughout the study lifecycle.
Where protocols involve prolonged follow-up, delayed adverse event surveillance or complex endpoint tracking, we work with sponsors and sites to ensure that these requirements are operationally feasible and properly embedded in the study plan. Our focus is on helping sponsors maintain patient safety, data quality and regulatory compliance in trials where the consequences of poor oversight can be significant.
Specialist centres, patient pathways and trial feasibility
Cell and gene therapy trials often rely on highly specialised sites with the right infrastructure, clinical expertise and patient pathways. PHARMExcel brings governance and oversight to this process by helping sponsors identify the most appropriate centres of excellence, referral routes and operational partners for their study.
This is particularly important in rare disease and oncology ATMP trials, where patient populations may be small, treatment pathways highly specialised and study procedures intensive. We do not directly recruit patients, but we help sponsors plan realistic, site-led enrolment pathways and ensure that trial processes reflect the lived realities of patients, investigators and specialist treatment centres.
Partnering with PHARMExcel for cell and gene therapy CRO support
Sponsors partner with PHARMExcel because we bring practical experience in complex early phase research, a quality-driven approach to trial governance and the responsiveness of a smaller CRO. As an ISO 9001 certified organisation, we provide clear oversight, hands-on senior involvement and continuity of team across studies that demand close coordination and careful risk management.
Our services are structured to reduce operational burden, support regulatory readiness and help sponsors deliver high-quality ATMP clinical trials in specialist settings. For organisations looking for a CRO that understands how to translate advanced therapies into well-governed human studies, PHARMExcel offers a collaborative and pragmatic clinical trial partner.
LAUREL
Phase I, Cell Therapy
Myelodysplastic Syndrome Study
3 UK sites, 1 EU site
View case study
INKRoc
Phase I, Device-ATMP Combination
Ovarian Cancer Study
2 UK sites
Wharton's Jelly
Phase I, Tissue Engineered
Cardiac (Paediatric) Study
1 UK site
Gene Transfer
Phase I, Gene Therapy
Haemophilia B Study
1 UK and 1 USA site
TRALA
Phase I/IIa, Targeted Therapy & Stem Cell Transplant
5 UK sites
View case study
Advanced therapy CRO services FAQs
What types of cell and gene therapy trials can PHARMExcel support?
PHARMExcel can support clinical trials involving a range of advanced therapy medicinal products, including autologous and allogeneic cell therapies, gene therapy approaches and other ATMP modalities entering human studies. Our role is focused on the clinical trial stage, particularly where governance, regulatory oversight and specialist site coordination are central to successful delivery.
What makes cell and gene therapy trials different from traditional clinical trials?
Cell and gene therapy trials often involve complex handling requirements, specialist treatment pathways, small or vulnerable patient populations and longer-term safety considerations. Requirements such as chain of identity, chain of custody, specialist site readiness and extended follow-up can add significant operational and regulatory complexity.
How does PHARMExcel support regulatory requirements for ATMPs?
PHARMExcel supports regulatory strategy and submissions for Advanced Therapy Medicinal Products, managing local regulatory expectations around clinical trial applications, first-in-human studies, safety oversight and long-term follow-up. Our focus is on ensuring that study documentation and trial processes are compliant, clear and realistic.
Can PHARMExcel support early phase cell and gene therapy trials?
Yes. PHARMExcel is well placed to support first-in-human and early phase ATMP studies where close oversight, specialist site coordination and disciplined safety management are essential. It does not support pre-clinical work, but we do help sponsors translate advanced therapies into well-governed human clinical trials.
How are logistics managed in cell and gene therapy trials?
ATMP studies often depend on carefully controlled processes such as chain of identity, chain of custody and time-sensitive coordination between specialist sites and service providers. PHARMExcel does not perform manufacturing or laboratory functions directly; it manages the governance and oversight to help ensure these operational pathways are clearly defined, auditable and aligned with protocol requirements.
Why is long-term follow-up important in gene therapy trials?
Some gene therapy studies may involve durable biological effects or delayed safety considerations, which can require extended follow-up beyond the initial treatment period. Long-term follow-up helps sponsors and investigators monitor patient safety, capture meaningful outcome data and meet regulatory expectations for responsible trial oversight.