cell-and-gene embryonic stem cells, cellular therapy

Cell and Gene Therapy Solutions

Navigate the complexities of cell and gene therapy development with us

The unique demands of cell and gene therapy (CGT) clinical trials call for a uniquely prepared development partner. The extensive therapeutic and functional expertise of the PPD™ clinical research business of Thermo Fisher Scientific has guided a diverse range of cell and gene therapy assets through the development pipeline.

Our comprehensive, end-to-end cell and gene therapy CRO solutions leverage more than two decades of experience – including more than 130 clinical trials in the past five years – to meet the distinct needs of patients, sites, caregivers and sponsors.

Moreover, we can seamlessly integrate manufacturing, laboratory services, ultra-cold chain logistical support and clinical trial management to better serve sponsors needing a streamlined process to reduce vendor management risks and burden.

Cell and Gene Therapy Institute (CGTI)

Our cross-functional team of experts helps enable faster delivery and operational efficiencies in clinical research.

The promise of cell and gene therapy explained

Cell and gene therapies require unique operational, regulatory and safety considerations. Our Cell and Gene Therapy Institute focuses on the development of guidance documents, operational tools, education and thought leadership aimed at expediting in vivo gene- and cell-based clinical trials.

Expertise is built into every component of our cell and gene therapy CRO capabilities so that our customers can be confident they are receiving high-quality services at every stage. Our record of partner success in cell and gene therapy clinical trials speaks for itself.

In the past five years alone, our cell and gene therapy experts have supported:

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More than 130 CGT clinical trials across a wide range of therapeutic areas, including hematology, rare disease, oncology, immunology, urology, neurology, cardiovascular and more

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More than 55 cell therapy trials spanning both autologous and allogeneic cell types, including T cell infiltrating lymphocytes (TILs), CAR-T, CAR-NK, B cells, and dendritic cells

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More than 75 gene therapy trials across adenoviral, AAV, lentiviral, and gamma retroviral vector delivery platforms

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More than 300 peri- and post-approval projects in cell and gene therapy value demonstration, market access and commercial strategy

Advancing cell therapy in China

While initial strides in CAR-T therapies were primarily made in US markets, the focus is now shifting to China – but can both foreign and domestic players win in China’s CGT market? Explore the challenges and opportunities to advance cell therapy clinical trials in China in this European Biopharmaceutical Review article.

Sample pages from the Advancement of Cell Therapy in China - Challenges and Opportunities in 2023 download

End-to-end services provide the ease of a single partner across the full development lifecycle

We offer specialized support for customers’ needs at every stage of the CGT development journey. Working with a single cell and gene therapy CRO partner across the development lifecycle means better integration of services and capabilities at each stage, continuous support and a better experience for every stakeholder in the clinical trial landscape.

Preclinical/Early Phase

  • First-in-human planning and protocol optimization
  • Feasibility and regulatory strategy
  • Development of patient pathway

Clinical Stage

  • Expert operational support for complex CGT studies across all phases
  • Deep, established relationships with investigators at FACT- and JACIE-accredited sites worldwide
  • Dedicated patient support services to facilitate participation in CGT clinical trials

Peri- and Post-Approval

  • Extensive experience in long-term follow-up of patients
  • Expertise in registry programs to track dosed patients for 15+ years

How we support CGT customers across all phases

CRO experience
Tailored patient support We recognize the complexities of participation in cell and gene therapy trials for patients. Our commitment and support of patients enables them and their caregivers to be well informed of the study protocol, risks, and potential benefits and to stay engaged through the duration of the study, from day one through long-term follow-up.
Extensive safety protocols Because of the inherent unknowns associated with many CGT studies, we put patient safety at the forefront of each step in our strategies to closely monitor and mitigate risks. Our capabilities include developing sound dose-finding and dose escalation strategies and using Preclarus™ dashboards that combine study and lab data to identify trends and preemptively identify toxicities.
Full-spectrum laboratory services Our specialized, state-of-the-art and customizable lab capabilities include cell-based, bioanalytical and genomic testing capabilities to serve clients from preclinical through Good Manufacturing Practice (GMP) product testing.
Expert logistics management Whether it’s shipping viral vectors or cryopreserved autologous cell therapy investigational medicinal products, our global logistics management team securely maintains chain of custody and chain of identity for every product and ancillary component. We skillfully navigate the sensitive timelines and storage demands of cell and gene therapy development.
Comprehensive training We facilitate extensive team training in cell and gene therapy through our employee development programs, which include ongoing education and development for sites and investigators.
Diligent regulatory expertise We are dedicated to keeping pace with the evolving regulatory landscape (globally and regionally) for advanced therapy medicinal products (ATMPs). We work with clients to develop and execute regulatory strategies based on their business desires for current and future market access.
Evidence generation strategies Our evidence, value, and positioning team of experts routinely develop advanced models to generate total evidence packages (e.g., real-world evidence) for stakeholders to identify novel pricing, such as outcome-based reimbursement plans, for curative therapies.

Accelerate your gene therapy trial

Gene therapies are changing outcomes for rare neurological diseases.
We create streamlined gene therapy study protocols and designs to help you meet your goals effectively.

Extensive education and support ensure patient retention in CGT trials

Participation in a cell and gene therapy trial can place significant burdens on patients, from identifying trials to contending with a complicated trial experience that often requires five to 15 years of study follow-up. To best support patients and strengthen our understanding of their needs, we engage with patient advocacy groups and other stakeholders to bring patient voices into the trial development process as early as possible. This enables us to better recognize, appreciate and understand patient and caregiver challenges at each step of the patient journey.

Because of the high level of patient commitment and compliance required in CGT studies, we provide ongoing education and support to patients and caregivers to ensure that patients start and stay with the trial to the very end, including:

  • Robust education and awareness campaigns to connect patients with trial opportunities
  • “White glove” patient concierge service, which assigns a “personal assistant” to support each patient and caregiver
  • Decentralized and digital study capabilities, including eConsent, home health and telemedicine

Together, these services ease the burden on patients to yield better engagement so that patients are retained for the entire duration of the study.

Optimizing the patient journey starts with a unified approach to clinical development. Read more

Ongoing development and training enable sites to execute complex CGT studies

CGT clinical trials are long, incredibly complex and full of uncertainties for even the most experienced sites. PPD’s solutions were built with these challenges in mind, to set up every trial site for success from beginning to end.

To ensure the successful execution of CGT studies, we have a growing global network of FACT1-, JACIE2– and EBMT3-accredited sites to identify the most appropriate location for your study and initiate startup. We also invest in guiding sites through necessary accreditations to participate in studies.

Once a site is initiated, we provide ongoing partnership to support the small details that make CGT trials successful, such as:

  • Facilitating patient consent mock dry-runs
  • Training staff to recognize immune responses
  • Overseeing investigational product handling and sample tracking
  • Providing comprehensive logistics support

By working together over many years, we have developed trusting relationships with the investigators and sites most engaged in this difficult, but rewarding, work. The quality of our relationships, in turn, translates into faster startup times, better collaboration and improved experiences for our customers.

Download our survey report to dig deeper into trends and solutions in cell therapy development

Comprehensive training equips teams with CGT trial expertise

Our mature CGT solutions – and the opportunities to work on cutting-edge CGT programs while learning from eminent thought leaders – allow us to attract hard-to-find CGT talent. We are also committed to training, upskilling and reskilling our employees for future needs with our award-winning employee development programs. We develop specialized CGT expertise through our training programs and forums, including:

ACT Academy 12-hour cell therapy training targets all staff levels with core modules (e.g., manufacturing, cell therapy trial considerations), study and role-specific training, and personalized training (e.g., pre-study visits or site initiation visit mock training).
Gene Therapy Academy Operational training provides guidance on a broad range of topics including scientific concepts and approaches, case studies, genetic testing, study design and operational considerations, regulatory insights, the gene therapy process and aftercare, and insights into the patient journey.
ACT Now and GeT Ahead forums Actively engaged internal communities of CGT study team members across cell therapy and gene therapy share information, knowledge and best practices to leverage lessons learned in real time.

An exceptional learning culture and formal professional development not only support employees in their current roles, but also provide opportunities to expand their knowledge and skill sets. This positive culture increases employee engagement and retention while enhancing continuity in our customers’ research programs, ensuring stable relationships with skilled teams.

Cell and Gene Therapy Institute (CGTI) helps sponsors conquer complexities

Cell and gene therapy development often involves the most intricate clinical value chain in modern medicine, demanding a CRO partner that appreciates both the technical and financial nuances of the field. As a newer therapeutic category, CGT clinical trials and approvals require diligence in collecting safety and efficacy data, and present unique logistical and scientific challenges.

Our Cell and Gene Therapy Institute (CGTI) enables our customers to develop and execute successful trial strategies by leaning on our thought leaders, regulators and operations experts, a number of whom shaped the field of advanced therapy development from its origins. CGTI brings together a cross-functional team of experts to provide diverse experience in drug design, technologies, clinical operations and regulatory.

Benefits of CGTI for sponsors:

  • Access to operational, functional, technical, regulatory and therapeutic CGT expertise across all phases
  • Better anticipation and mitigation of risks and proactive risk mitigation associated with these specialized trials
  • Greater confidence and capabilities to advance your CGT asset through each stage in the process

How CGTI unites clinical and commercial perspectives to create compelling evidence narratives

Our CGTI supports each CGT program with the people, processes and tools necessary to generate comprehensive data packages and identify novel pricing and reimbursement opportunities. CGTI drives medical, regulatory and operational knowledge transfer, process and tool refinement and operational best practices by uniting our strategic and functional centers of excellence, including our:

Gene Therapy Operational Center of Excellence Operational leaders who build better operationalization and training strategies for gene therapy studies
Cell Therapy Center of Excellence Operations, medical, regulatory, and logistics leaders who optimize processes and resources for cell therapy studies
Advanced Therapies Forum Regulatory experts, some with working experience at regulatory agencies, who provide guidance on regional and global regulations on CGTs
Market Access and Value Demonstration Leadership Team Experts in evidence, positioning and value for payer and health technology assessment
Rare Disease and Pediatrics Center of Excellence A cross-functional team focused on the execution of innovative medical and operational solutions

By fostering collaboration between clinical and commercial leaders across the field, CGTI ensures each study is properly equipped to generate necessary quality data. The result? CGT clinical trials that run smoothly – and a cell and gene therapy asset that’s more likely to be approved by regulatory agencies, prescribed by physicians, wanted by patients and covered by payers at a price in line with the value it provides.

Specialized lab capabilities support the speed and complexity of CGT studies

CGT studies often require customized assays using state-of-the-art methods for better sensitivity and specificity, making the right lab partner critical to the success of your study. PPD Laboratory services apply decades of experience and combine high-quality scientific expertise with industry-leading technologies. Our bioanalytical lab services boast more than 20 years of experience, millions of samples analyzed and more than 170 scientists and staff dedicated to cell and gene therapy. Our GMP lab has worked with more than 60 cell and gene therapies across 15+ years.

PPD Laboratory services also offer a lab concierge program that takes seriously the security of the critical samples associated with CGT studies. This innovative program features a specialized project management team focused on sample management and logistics to ensure sample receipt, testing, initiation and coordination of data transfers.

Expert logistics management ensures chain of custody and chain of identity

Transporting, preparing and administering cell and gene therapy products is not just about securing resources and manpower. This important process requires expert navigation of multi-country transport, storage conditions, sensitive timeframes and other critical details.

Because the stakes are high, particularly for autologous therapies where there are minimal to no backup products, our global clinical logistics efforts are supported by two dedicated roles. The logistics portfolio lead develops the logistics strategy for each CGT protocol and oversees our clinical logistics coordinators, who work collaboratively with sites, sponsors and shipment vendors to ensure timely and seamless transport at every stage.

With this approach, we know where the therapy is at all times, and the status of testing at multiple laboratories in different parts of the world – and we can facilitate the measuring and monitoring to ensure biological therapies are meticulously tracked from end to end.

Contact us to learn how we support your CGT development needs

With our demonstrated success, we know how to meet the needs of patients, caregivers, sites and sponsors to drive confidence in CGT trials – and safely and efficiently deliver your groundbreaking therapeutics.