Navigate the Unknowns of Cell & Gene Therapy Development with PPD
The unique demands of cell and gene therapy (CGT) clinical trials call for a uniquely prepared development partner. PPD’s extensive breadth and depth of therapeutic and functional expertise have 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 over two decades of experience – including more than 130 clinical trials in the past five years – to meet the distinct needs of patients, sites and sponsors.
The Promise of Cell and Gene Therapy Explained
Cell and Gene Therapies require unique operational and safety considerations. PPD’s Cell and Gene Therapy Institute focuses on the development of guidance documents, operational tools, education and thought leadership aimed at expediting your In-Vivo and Ex-Vivo clinical trials.
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.
Our experience by the numbers
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.
PPD’s record of partner success in cell and gene therapy clinical trials speaks for itself.
In the past five years alone, PPD’s cell and gene therapy experts have supported:
Over 130 CGT clinical trials across a wide range of therapeutic areas, including hematology, rare disease, oncology, immunology, cardiovascular and more
More than 55 cell therapy trials spanning both autologous and allogeneic platforms and technologies including CAR-NK cells, antigen-presenting cells, adoptive T-cells and primitive stem cells
Over 75 gene therapy trials across adenovirus/AAV, lentivirus and retrovirus delivery platforms
More than 300 peri- and post-approval projects in cell and gene therapy value demonstration, market access and commercial strategy
End-to-end services provide the ease of a single partner across the full development lifecycle
PPD offers 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 pathways
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 PPD supports customers across all phases
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 & 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 start and stay with CGT studies for their duration.
Ongoing development & 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.
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:
PPD’s 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 & 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.
PPD’s Cell and Gene Therapy Institute (CGTI) enables our customers to develop and execute successful trial strategies by leaning on PPD thought leaders and operations experts, a number of whom shaped the field of advanced therapy development from its origins. CGTI experts provide a wealth of experience that spans diverse technologies and delivery mechanisms, including cell, gene, gene-modified cell and advanced tissue technologies.
Benefits of CGTI for sponsors:
- Access to operational, functional, technical and therapeutic CGT expertise across all phases
- Better anticipation and mitigation of risks associated with these specialized trials
- Greater confidence and capabilities to advance your CGT asset through each stage in the process
How CGTI unites clinical & commercial perspectives to create compelling evidence narratives
PPD’S 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 and operational knowledge transfer, process and tool refinement and operational best practices by uniting our strategic and functional centers of excellence, including our:
By fostering collaboration between clinical and commercial leaders across the field, CGTI ensures each study is properly equipped to generate necessary 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 & complexity of CGT studies
CGT studies often require precise specialization, data and speed customized to the needs of each individual trial, 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 over 170 scientists and staff dedicated to cell and gene therapy. Our GMP lab has worked with over 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 & 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 time frames and other critical details.
Because the stakes are high, 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 live biological therapies are meticulously tracked from end to end.
Contact us to learn how we support your CGT development needs
CGT clinical trials call for a cell and gene therapy CRO with demonstrated success.
At PPD, we know how to meet the needs of patients, sites and sponsors to drive confidence in CGT trials – and safely and efficiently deliver your groundbreaking therapeutics.