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Cell and Gene Therapy

Cell and Gene Therapy

REMOVE UNCERTAINTY AND BRING GROUNDBREAKING TREATMENTS SAFELY TO PATIENTS

Saying “each trial is unique” may be cliché for some areas of development – not for cell and gene therapy.

PPD has the experience and end-to-end expertise to help you advance your development program, informed by over a decade of work in these highly specialized clinical trials. In this pioneering development space, PPD can help you better predict how patients, caregivers, sites, regulators and payers will react to your therapy.

INTEGRATED CELL AND GENE THERAPY DEVELOPMENT SERVICES FROM PRE-CLINICAL TO POST-APPROVAL

Operational execution for complex studies involving rare and at-risk patients
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Teams experienced in close chain of custody management patient samples and investigational products
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Comprehensive labs services spanning GMP, bioanalytical and genomic applications
Comprehensive regulatory strategy, gap analysis and first-in-human planning
Strategies to generate compelling evidence and identify novel pricing and reimbursement for curative therapies
Experience in long-term follow up and registry programs to track dosed patients for up to 15 years

Experience with a Diverse set of a range of delivery mechanisms across multiple therapeutic areas

Over the past five years, PPD has conducted more than 50 cell and gene therapy studies across multiple delivery platforms. Our experience in cell and gene therapy crosses all therapeutic areas, with key strengths in oncology, neuroscience and rare diseases.

EXPERIENCE ACROSS DELIVERY PLATFORMS

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

From recognizing uncertain immune response risks, to monitoring vein-to-vein time and leveraging detailed knowledge of necessary investigational product handling and sample tracking, PPD delivers the operational and medical know-how required for these complex studies.

Additionally, we understand how to design studies that account for the safety, efficacy and durability measures required for cell and gene therapy assets. PPD has a long history of success with lengthy studies requiring extensive data collection, recruitment of at-risk patients, and sustained patient engagement through many years of follow-up.

CLINICAL CELL AND GENE THERAPY EXPERIENCE – PAST FIVE YEARS

REGULATORY EXPERTISE THROUGHOUT THE DEVELOPMENT LIFE CYCLE

We deliver regulatory expertise to our clients through:

  • Early engagement activities: Scientific advice, designation strategies including orphan, Regenerative Medicine and Advanced Therapy (RMAT), Advanced Therapy Medicinal Products (ATMP), pediatric studies and priority review planning
  • 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

“At PPD, our focus on a fully integrated approach to cell and gene therapy, which ties together regulatory, marketing access and value demonstration strategy; clinical planning and execution; and highly customized labs services, helps our clients better anticipate acceptability of regulators and payors and get these innovative treatments to patients faster.”

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OUR GLOBAL TEAMS ARE IMPLEMENTING INNOVATIVE STRATEGIES TO ADDRESS THE CHALLENGES OF COVID-19

See how PPD can keep your trial on track.

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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

MARKET ACCESS AND VALUE DEMONSTRATION

With experience across more than 300 cell and gene therapy projects, we can offer extensive expertise in:

  • Market access
  • Reimbursement and pricing strategy
  • Outcomes research – registries and natural history studies