Why Choose Medpace for Complex Cell and Gene Therapy Clinical Trials?
Despite market growth, cell and gene therapy (CGT) clinical development continues to drive innovation through its evolving scientific and operational landscape.
At Medpace, our multi-disciplinary teams take an active approach to surmounting complex clinical and regulatory barriers common to the development of CGT clinical studies. Maximize your CGT trial budget and expedite trial timelines by partnering with our global, integrated team to unlock efficiencies across medical, regulatory, and operational considerations and requirements.
The Medpace cell and gene therapy team offers cross-functional therapeutic expertise to accelerate the development of cell and gene therapies. Our extensive CGT experience spans:
CNS / Neuroscience
Cardiovascular
Endocrine & Metabolic
Hematology & Oncology
Infectious Diseases (ID)
Ophthalmology
Pediatrics
Rare Disease
Rheumatology
This extensive experience in managing CGT trials has led to market authorization approvals and demonstrates Medpace’s ability to successfully manage the most complex and innovative trials.
Medpace Cell and Gene Experience:
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Countries
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Making the Complex Seamless® in Global Cell and Gene Clinical Development
Medpace supports cell and gene therapy clinical trials globally through training, specialized processes, and extensive experience:
Integrated clinical operations, including a logistics team
Regulatory submissions and affairs
Laboratory services with assays specific for CGT trials
Patient recruitment
Site management
We provide Sponsors with comprehensive strategies that address the rapidly evolving clinical, operational, and regulatory challenges, as well as the unique set of hurdles presented in crossover therapies. Additionally, our patient-centric approach enables our team to keep patients engaged throughout the entirety of the study.
“Implementing a strategy of integrated, cross-functional activities, which leverages best practices early in clinical development, can enrich the likelihood of success in executing cell and gene therapy trials.”
Jeff Vassallo, PhD Executive Director, Clinical Trial Management
Cell and Gene CRO Capabilities
Medpace, a full-service clinical research organization (CRO), has conducted over 160 cell and gene therapy trials, and applies this experience to help Sponsors advance their CGT therapies. Leverage Medpace’s cross-functional cell and gene therapy clinical development services from preclinical to post-approval.
Extensive experience across various products (cell, gene, tissue-based)
Network of globally trained investigators with the capabilities and experience to store, prepare and administer these unique products
Investigational product experience, such as first-in-human (FIH), allogeneic and autologous cellular products, ex vivo and in vivo gene transfer, and gene editing
Patient-Centricity in Cell and Gene Therapy Trials
Medpace recognizes the significant burdens involved in patient participation in CGT trials. We place patients at the center of a trial to increase awareness, reach, and enrollment while helping yield successful outcomes in terms of study design, patient education and engagement, and study results.
Patient education is one of the most critical contributions to a successful patient recruitment and retention strategy. Educating patients on cell and/or gene therapy trials (potential benefits or risks, side effects, and adverse events) can help ease any hesitation or discomfort a patient may have about participating in a trial. Our dedicated clinical trial management teams follow a multifactorial approach towards awareness, which includes developing educational materials to ensure patients are informed throughout the study.
WHITEPAPER – Lessons Learned in Emerging Cell and Gene Therapies
Medpace seamlessly supports the advanced needs of cell and gene therapy development through our comprehensive array of lab capabilities.
Our specialized teams can collaborate and consult internally to provide a comprehensive approach to your study, leading to efficient, organized, and standardized trial management.
Medpace Core Labs (MCL) provides a breadth of expertise across a broad spectrum of CGT imaging. Notably, we use a web-based image management system with integrated quantitative tools to analyze a wide range of medical imaging modalities such as MRI, CT, PET, x-ray, etc. for confirmation of eligibility, safety, and efficacy evaluations. Our imaging and clinical trial experience ensures that imaging components are seamlessly integrated into the complex structure of CGT trials.
Cell and Gene Therapy Imaging Expertise:
CGT Specific Medical Imaging Protocols
Qualitative Visualization
Serial Quantitative Measurements
Our wholly-owned central laboratories—with locations in the US, Belgium, China, and Singapore— offers a menu of validated biomarkers associated with Cell and Gene Therapy with the ability to rapidly establish and validate novel assays as needed.
Cell and Gene Therapy Testing Services:
Genomic and Molecular Testing
Flow Cytometry
Anatomical Pathology
Cell-based and Soluble Biomarker Assays
Specialized Product Storage and Distribution Capabilities
Medpace’s central laboratories and bioanalytical laboratory, with their teams of scientific experts, collaborate closely with Sponsors to develop unique solutions for various cell and gene therapy programs. Our globally harmonized laboratories utilize standardized platforms, validated workflows, and state-of-the-art instrumentation to deliver consistent high-quality results across global studies.
Cell and Gene Testing Offerings:
Vector and Transgene Detection/Quantitation (qPCR and digital PCR)
CAR-T Biodistribution Testing
Sanger Sequencing and Next Generation Sequencing (NGS)
Drug Product Characterization
Immune Reconstitution Analysis
Cytokine Profiling
Anti-Drug Antibody Assay
Neutralizing Anti-Transgene Antibodies Assay
Comprehensive Regulatory & Operational Expertise in CGT
Cell and gene therapy clinical studies are often delayed due to lack of therapeutic experts. Medpace has a team of therapeutically-aligned operations, regulatory and technical experts with relevant experience in novel therapies, who work collaboratively with our specialized medical team in order to leverage expertise for the benefit of trial programs. The Medpace Technical Advisor team provides in-depth regulatory support regarding country and regional guidelines, assesses regulatory readiness of the study documentation, develops environmental risk assessments and other technical documents required for Biosafety submissions and assist sites with regulatory activities such as obtaining site level certificates and accreditations.
An effective regulatory strategy is critical to navigate cell and gene therapies through clinical development and achieve expedited approval. From early engagement to marketing approval, Medpace Regulatory Affairs provides strategic guidance and support for regulatory interactions throughout development including agency meetings and scientific advice procedures, special designations (RMAT, ATMP classification, Orphan Drud Designation, Breakthrough, Fast Track) and expedited marketing authorization pathways (PRIME, accelerated approval, conditional marketing authorization).
Study start-up for CGT trials is highly complex, with many factors to consider, such as limitations at sites or with vendors or lack of connections between therapeutic experts. Medpace holds the experience in planning and managing cell and gene therapy clinical trials and can provide Sponsors with proactive solutions to operational complexities inherent in these therapies, such as start up requirements, site selection, patient recruitment and retention.
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Achieving Successful Cell Therapy Outcomes through Integrated Expertise
Proactively identify and address issues before the study is impacted by leveraging Medpace’s broad therapeutic experience and expertise. The Medpace Cell and Gene Therapy team is comprised of Medical Directors, Advanced Clinical Practitioners, and Clinical Trial Managers who meet routinely to build upon lessons learned in CGT development.
Success Stories in Cell and Gene Therapy Research
Our strong relationships with investigators, key sites, regulators, institutions, specialized laboratories, and KOLs in CGT help facilitate strategic site selection, start-up, and recruitment efforts. The experience and expertise combined with long-standing relationships will readily translate into the successful and timely recruitment, conduct, and delivery of a study.
Cell Tracking and Logistical Support
Medpace has developed a proven approach to manage complex cell therapy trials, including cell tracking. Through our customizable process, we provide Sponsors with seamless logistics and cell tracking to ensure the Chain of Custody and Identity is maintained.
Through our customizable process, we provide Sponsors with expertise on the logistics process, a study-specific portal, site-facing guidance, and rigorous site training to ensure that patients get the right product at the right time and under the appropriate storage conditions
An integrated and intuitive cell therapy manual is developed for each trial; the manual is reviewed by medical monitors and advanced clinical practitioners who have advanced training and experience in cell therapy to ensure it is conducive with institutional standards
Successful Approaches from Rare Disease & Cellular and Gene Therapy Product Approvals
Understanding and proactively addressing these common challenges and considerations ensures that a trial is best positioned for success and will support the future goal of marketing approval.
