Cell and Gene Therapy

Integrated End-to-end Cell and Gene Therapy Capabilities

Emerging technologies have driven cell and gene therapies to the forefront of health care. These therapeutics now address a growing number of disease conditions, including rare diseases with unmet medical needs. As cell and gene therapies continue to evolve, regulatory agencies are embracing these therapies with marked interest and cautious optimism.

PPD partners with clients of all sizes to advance development of their cell and gene therapy trials across numerous indications and all development phases.

We provide:

Early development, regulatory and market access strategy
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Comprehensive laboratory services
End-to-end product development and clinical execution
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Patient and sample concierge services

broad therapeutic expertise

Over the past five years, we have conducted more than 50 cell and gene therapy studies across multiple types, including:

  • Adenovirus/AAV
  • Lentivirus
  • Virus-based immunotherapies
  • Non-viral vectors
  • CAR-T cells
  • Cell-based medicinal products

We have a wide range of therapeutic experience in cell and gene therapy, including key areas such as oncology, neuroscience and rare diseases.

CAR-T STUDIES

Our teams have experience across 500 oncology studies, 120 immuno-oncology studies and seven adoptive T-cell therapy trials.

This experience informs how we manage the unique intricacies of CAR-T studies and cutting-edge vectors to: ensure patient safety, provide efficient and rigorous oversight of vein-to-vein time, and carefully handle and track samples.

COMPREHENSIVE LABORATORY SERVICES

PPD® Laboratories has accumulated a wealth of experience across a wide variety of drug substances and delivery mechanisms. Specifically, our teams have worked with cell and gene therapies for more than 15 years. Our expertise spans both GMP and bioanalytical  and genomic applications, and specimen concierge services are provided for precious samples.

ANALYTES AND DELIVERY MECHANISMS

  • Oligonucleotides
  • mRNA
  • CAR-T therapy
  • CRISPR/Cas9
  • Enzyme replacement therapy
  • Lipid nanoparticles
  • Polymers
  • Adeno-associated virus (AAV)
  • Targeted delivery systems

REGULATORY EXPERTISE THROUGHOUT THE DEVELOPMENT LIFE CYCLE

The regulatory environment for cell and gene therapies is complex and rapidly advancing. Regulatory intelligence combined with frequent and early interactions with regulatory agencies is critical to pave the most efficient path to approval and market access. Our global regulatory teams provide full, end-to-end regulatory support from program design through
execution.

We deliver regulatory expertise to our clients through:

  • Early engagement activities: Scientific advice, RMAT and ATMP classifications, orphan drug designation, pediatric plans
  • Genetically modified organisms (GMO) expertise: Authoring of environmental submission dossiers, interaction with country specific agencies
  • Clinical trial applications: IND and IMPD authoring and gap analysis, protocol review, global submissions
  • Consultancy: Strategic regulatory consultancy, clinical/non-clinical consult

MARKET ACCESS AND VALUE DEMONSTRATION

A record number of cell and gene therapy products are maturing toward approval. Many global systems have not anticipated these types of interventions. Out-of-the-box thinking is required to successfully navigate cell and gene therapies through these processes.

In addition, there is limited precedent that challenges reimbursement “fit” for authorities. Our evidence and peri-/post-approval group, Evidera, has significant expertise in value demonstration and reimbursement strategy for these new therapies that can accelerate the development process for cell and gene therapies.

With experience across more than 200 cell and gene therapy projects, we can offer extensive expertise in:

  • Market access
  • Reimbursement and pricing strategy
  • Outcomes research

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