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Cell and Gene Therapy

Cell and Gene Therapy – Center of Excellence

Cell and Gene Therapy (CGT), also known as Advanced Therapy Medicinal Products or ATMPs, holds the promise to deliver transformative outcomes in a wide array of hard-to-treat diseases. Unfortunately, so far, most have failed in development and few have been successful in the marketplace.

Our Cell and Gene Therapy Center of Excellence helps clients around the world accelerate their novel, advanced therapeutic products to market. ProPharma Group’s team of experts has supported more than 100 different cell and gene therapy development programs. These programs contain a diverse set of CGT/ATMP technologies, including various viruses and vector types, oncolytic viruses, and an increasing number of gene modified cell therapy modalities.

Our team has a combined 25 years of experience delivering unique lifesaving treatments to market in both EU, US, and Japanese regulatory environments. We have a diverse talent pool comprised of technical, scientific, quality assurance (QA), and regulatory affairs professionals to support you across the full development lifecycle to effectively and efficiently bring products to patients. We accomplish this by assisting our cell and gene therapy clients through early-stage and nonclinical product development, clinical trials, marketing authorization and all associated regulatory aspects, and GMP manufacturing.

At ProPharma Group, our approach is to begin with the end in mind. We identify potential challenges and help craft strategies and solutions before you are confronted with them during a regulatory assessment.

Guidance and Services for Cell and Gene Therapy

There are many regulatory, technical, and scientific challenges that can significantly impact the successful outcomes in the product development and commercialization of cell and gene therapies. Our Center of Excellence services, specific to CGT, include but are not limited to:

  • Regulatory strategies and gap analysis development plans, to include a forecast of key milestones, necessary documentation, overall effort, cost, and plan of action
  • Formal Meetings with regulatory agencies, including formal meetings with FDA, EMA, and national competent authorities (NCAs)
  • Expedited approvals with FDA and EMA, including PRIME applications, orphan designation applications, Emergency Use Authorizations (EUAs), Priority Review, Fast Track, Breakthrough Therapy, and Accelerated Approval designations
  • Consulting on appropriate development strategies (eg, biodistribution, tumorigenicity, and orphan designations)
  • Clinical Trial Application (CTA) and Investigational New Drug (IND) support, working with you to develop and submit a complete and successful application including genetically modified organism (GMO) applications in the EU
  • SME Status Application
  • Pediatric Investigation Plans (PIP) that meet the needs of both the FDA and EMA, allowing your product to advance to the next regulatory milestone
  • Clinical trial support
  • EU Marketing Authorization Application (MAA) and FDA Biologics License Application (BLA) support, including development and submission of the application to the regulators
  • Good Manufacturing Practice (GMP) compliance assistance, including the design of GMP manufacturing facilities, including commission, qualification, and validation
  • Quality Management System (QMS) design, including the development and implementation of effective and customized QMS programs or the optimization of existing programs
  • Technical Transfer(s)
  • Classification advice – is your product a CGT/ATMP, a medical device, or a combination product?
  • Consultation on comparability studies for your product before and after changes are made to the manufacturing process
  • Regulatory submission and deliverable support, including data summary and document writing (eg, IMPD, IB)
  • FDA and EMA classification and certification procedures for CGTs/ATMPs
  • Regulatory aspects of grant applications
  • Clinical investigations for biomaterials
  • Risk assessment and remediation of data packages, existing processes and systems, etc.
  • Process Optimization
  • Vendor selection and auditing

While the science, quality demands, regulations, and clinical use of cell and gene therapy evolves, companies need a partner that can define appropriate development strategies to manage the ever-changing regulatory environment and anticipate what will be required in years to come. ProPharma Group is that partner.

Our vast breadth of service offerings paired with our diverse panel of cell and gene therapy/ATMP specialists is unlike anything you have ever experienced. With a full suite of global services that spans the entire product lifecycle, our team of cell and gene therapy experts is comprised of the best regulatory and compliance consultants across the globe. Whether you are in the early stages of development looking for assistance with your entire development program, you need to ensure your approved product remains compliant with applicable regulations and requirements, or anywhere in between, we have the expertise to help you obtain successful interactions with the regulators.

Let Our Work Speak for Itself

Our name truly does say it all: Cell and Gene Therapy – Center of Excellence. Delivering excellent work is not just our goal, it is all we will accept. But rather than tell you about it, here are some case studies that prove it.

US to EU Transition (Cell Therapy):
  • Company in clinical development in the US and planning to open trial sites in the EU
  • Regulatory gap analysis and development plan provided to define the EU regulatory strategy
  • EU scientific advice taken
  • Successful orphan application
  • IMPD written, liaising with EU manufacturer to provide the detail expected by national agencies in the EU
  • Regulatory support provided throughout the CTA
Full EU Regulatory Provision (Gene Therapy):
  • Company in clinical development in US
  • Regulatory gap analysis and development plan provided to define the EU regulatory strategy, support in scientific advice, and Orphan and PRIME submissions
  • Carried out the role of EU regulatory support to Sponsor, providing full CTA management services for over 20 CTA submissions and GMO application support
  • MAA leadership through to approval

A Global Team Bringing Broad and Deep Expertise in Cell and Gene Therapy

Our Cell and Gene Therapy – Center of Excellence nurtures a think tank of cell and gene therapy experts. We provide a comprehensive suite of integrated services and solutions to advance your product to the next milestone while maintaining the highest compliance standards. Our team’s expertise supports you throughout the full lifecycle of cell and gene therapy product development, from early-stage concept development through post-commercialization.

To learn more about our full suite of cell and gene therapy consulting services and how we can help you accomplish your goals, contact us today.

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