From Discovery through Commercialization

Cell and gene therapies aim to treat, prevent, or potentially cure diseases. Both approaches have the potential to alleviate the underlying cause of genetic and acquired diseases. Although critically important, the cell and gene therapy landscape is especially challenging to navigate from R&D to regulatory approval and market access.

  • Cell and gene therapies can have exaggerated and unexpected behavior, and animal models may fail to predict the potential for human toxicity or beneficial immune modulation
  • Efficacy and safety issues have contributed to unsuccessful regulatory outcomes, creating uncertainty in regulatory pathways
  • Cell and gene therapies pose pricing and market access challenges as systems wrestle with paying upfront for a lifetime of benefits

Certara’s Cell and Gene Therapy Practice de-risks and accelerates the entire process, from discovery to market access with an integrated approach and experienced team. We are uniquely positioned to anticipate risks early, provide concrete insights to make informed and critical decisions, and set your cell and gene programs up for success.

With extensive experience successfully supporting sponsors in more than two dozen cell and gene therapy programs, Certara’s Cell and Gene Therapy Practice offers broad and integrated experience and knowledge.

Integrated, commercial, best-in-class approach

From discovery through commercialization, our integrated team of experts leverages extensive experience and knowledge of development, regulatory pathways, commercialization, and lifecycle management to provide our partners with the support you need to advance your program and help to increase probability of success.

  • With the most experienced team of experts in pharmacometrics, mechanistic modeling, regulatory science, and market access, we are dedicated to achieving and accelerating your success
  • We use modeling and simulation across the entire drug discovery and development life cycle to increase confidence in critical decisions and help you reach your goals, smarter and faster

Agility, communication, quality, and trust are critical to our partnerships. Together with you, we are one integrated team that creates a robust strategy and delivers with seamless implementation

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Getting the Dose Right - Certara Virtual™ Twin-QSP

Considered personalized medicine, each ‘therapy’ is based on individual patient data detail to repair or enhance faulty genes. Too low a dose might exclude that patient from any future benefit, while too high a dose can be very dangerous, since any immunogenic side effects may be irreversible and could last for years. Threading this needle requires a novel development approach.

Certara’s Virtual Twin-QSP platform has demonstrated success in multiple rare disease gene therapies in predicting and informing Phase I/II and III studies. We create a computer-simulated model of each patient (virtual twin), replicating the patient’s various attributes, including morphology that affect a drug’s fate in their body and hence its effects. Hundreds of virtual twins are simulated in virtual trials to evaluate the impact of different drug doses, schedules, and combinations so that we can predict an optimal dosing regimen for each patient.

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Extensive experience in complex biologics

Certara provides substantive support in the development of Complex Biologic programs, including cell and gene therapies. We combine our deep knowledge of drug development with in silico modeling and simulation approaches to maximize program efficiency and increase the likelihood of success. We create and help clients execute on a Model-Informed Drug Development strategy, including:

  • IND-enabling pre-clinical activities
  • Early clinical development and regulatory strategy and program support, including Phase 1 and Proof-of-Concept trial design, and human dose projections for first-in-human trials.
  • Opportunities to leverage in silico and quantitative methods for optimizing Phase II/III studies and streamlining post-marketing requirements.
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Innovative pricing to expand market access

Cell and gene therapies pose pricing and market access challenges to manufacturers and payers as systems wrestle with paying upfront for a lifetime of health benefits.

Optimize value, positioning, and evidence synthesis across all stages of the reimbursement journey with Certara’s Evidence and Access team. Our combination of science and understanding of public health decision-makers’ challenges is the key to effective market access and pricing. Using an array of methods, we assess the value of health interventions.

Anticipating challenges ensures success with:

  • Strategy aligned with health authority and payer/HTA expectations to eliminate unnecessary data collection activities and deliver development programs efficiently
  • Adaptive response to changing market dynamics
  • Understanding of sources of data and evidence for historical comparators that improve value communication and capture
  • Support for both investor and investee needs as gene therapy manufacturers move across the funding trajectory
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De-risking the regulatory process

Clinical efficacy and safety issues have played a major role in unsuccessful outcomes from a regulatory standpoint. As this is an emerging arena, there is much uncertainty in regulatory pathways, evidenced by recent FDA decisions.

Preparing a successful regulatory submission for this challenging therapeutic area requires significant expertise, effort, and attention to detail. 庞大而多样化的团队、众多工具、计划和流程必须协调一致才能取得成功。Complexity, risk, time, and costs typify every cell and gene therapy submission. Without careful execution and the ability to navigate changing regulatory requirements, your submission may face costly delays.

Certara Regulatory Science, also known as Synchrogenix, assures and accelerates regulatory success through a unique combination of customized:

  • regulatory strategy
  • document authoring
  • medical communications and publications
  • operations solutions

Synchrogenix’s proven record of success is built on its expertise, and an unrivaled commitment to quality and compliance combined with the use of advanced technologies to enhance speed and efficiency.

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Piet van der Graaf,药学博士,博士 定量系统药理学部高级副总裁

Piet 曾任职于赛诺菲和辉瑞,在制药行业拥有 20 多年经验,为 QSP 项目带来了丰富的技能和经验,并为 Certara 的战略发展做出了贡献。他还担任《临床药理学与药物治疗学 (CPT)》主编。

Maximilian Vargas Sr. Director, US Access Strategy

Maximilian Vargas, PhD MBA, is a Senior Director, US Access Strategy and Account Management within Certara’s Evidence and Access Group since 2012. He brings both scientific training and experience in healthcare product development that are important for evidence-based value and access strategies for pipeline and marketed products.

Emily Jean Woodward, PhD Director, Customer Engagement

Emily has 15 years of experience in biotech, pharmaceuticals, and diagnostic devices spanning R&D, clinical development, and regulatory affairs. As the Director of the Customer Engagement group, she pairs her diverse industry experience with her scientific background to build teams of scientists, writers, and consultants who are tailored to meet clients’ evolving needs. Her passion lies in accelerating clinical programs for rare and orphan disease therapeutics by identifying efficiencies leveraging a lean, but compliant, approach.

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