Despite strong favorable signs from highly engaged regulators, accelerated regulatory pathways and special designations for cell and gene therapies, only acfew have been approved so far. For the ones that were approved, the number of patients enrolled in pivotal studies were significantly lower than for traditional drug approvals.
Making the best out of these opportunities and achieving a successful fast-track approval requires specialized and dedicated regulatory knowledge and experience, specific to the emerging field of cell and gene therapy.

Regulatory milestones during the traditional drug lifecycle


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The traditional drug development lifecycle is the same for cell and gene therapy programs, but is often very compressed, creating both opportunities for a faster pathway to commercialization, as well as challenges to meet key milestones and avoid bottlenecks towards cGMP readiness.


Regulatory needs in the cell and gene therapy space


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Our regulatory cell and gene therapy offering is customized to support you at each critical time and milestone as your therapy progresses through the regulatory pathway towards its commercialization.


Our approach to de-risk your regulatory pathway, with continuous support throughout the product lifecycle


The regulatory services we provide serve one main purpose: to de-risk your regulatory pathway. In the pioneering field of cell and gene therapy, companies are trailblazing and achieving many 'firsts' in the field. They need the ability to rely on a dedicated partner that will provide the expertise and experience they need to focus on the science and the efficacy of their drug. So how can we de-risk your therapy's pathway?

In the evolving field of cell and gene therapy, best-in-class regulatory support starts with monitoring and staying fully up-to-date with the latest regulatory trends and landscape. We do this through our active involvement in regulatory conversations in industry, through our alliances and partnerships during dedicated regulatory workshops. We are proud sponsors of the Alliance for Regenerative Medicine (ARM) and its legislative fly-in meeting, which we join each year and leverage our practical experience to advocate for favorable regulation in the field of cell and gene therapy.

We'll partner with you to identify the appropriate number of runs prior to IND/IMPD filings and PPQ to support the success of your filings. We will also work to anticipate and be prepared for facility and equipment questions and audits.

As you progress through the regulatory pathway, your regulatory needs evolve. As a true partner, we will support you each step of the way, with the right, scaled service you need.


cGMP support Strategic and technical support Regulatory submission support
  • Process Development, Technology Transfer and cGMP Manufacturing (clinical and commercial)
  • Import of critical raw material (e.g. MCB / MWCB)
  • Animal-origin materials
  • Export of drug products to clinical sites globally
  • Permits and Licenses (e.g. USDA, CDC, US F&W)
  • Additional advice, reports and documentation as requested
  • Response to questions
  • Scientific Advice - EU & FDA (briefing book and meeting preparation)
  • Meetings with regulatory authorities
  • Regulatory strategy
  • Post approval work (APR, Variations and DMF updates)
  • Write or review e-CTD ready regulatory submissions:
    • Draft or review IMPD / IND CMC sections for clinical trial approval
    • Summary of chemistry, manufacturing and control data
  • Draft or review MAA / BLA Module 3 and Appendices:
    • Detailed chemistry, manufacturing and control (CMC) data
    • Facility and equipment information
    • Adventitious agents safety evaluation
  • Write, submit and maintain DMF (as required)

Our raw/starting materials specifications include:

  • Qualification Program
  • Single source
  • Research Use Only
  • Additional testing (e.g. FBS)

Why choose us for regulatory support?

  • Access to distinguished company with deep heritage in biologics manufacturing, 20+ years of cell and gene therapy GMP expertise
  • In the past 6 years alone: 60+ biologicals IND/IMPDs to support Global Clinical Trial Applications, 12+ biologicals MAA/NDA/BLA-3.2.S and Appendices and tailored support for module sections
  • Global, dedicated CGT regulatory team of 6 with an average of 10+ years of industry experience
  • Supporting completed and ongoing filings of 3 BLAs and 5 INDs/IMPDs CGT customers
  • Flexibility to address needs of small or large companies
  • Regulatory support from pre-IND all the way to commercialization