Regulatory & Development Solutions for Cell & Gene Therapies
Why PrimeRA
PrimeRA is a partnership of regulatory and biotech leaders across the UK, US, and EU, united by a shared mission: advancing cell and gene therapies from lab to patient. We have individually honed our technical expertise within world-leading cell and gene therapy organisations - navigating EMA, FDA, and MHRA pathways at the most critical stages. As partners, we leverage our collective strategic and operational experience to solve the challenges that matter most to our clients.
Our approach is simple
Deploy experienced leaders who understand your technology.
Create tailored solutions that fit your programme, not a template.
Unlock innovation by de-risking regulatory complexity.
How We Can Help
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To help clients effectively plan the interaction between the product development process and regulatory interactions/milestones, the PrimeRA team will prepare a bespoke regulatory roadmap outlining the regulatory processes and steps to be undertaken placed into the context of data generation and timelines.
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Whether you are thinking about starting a clinical trial in a single country or seeking confirmation of late-stage changes in the development of a product, engagement with the regulatory authorities is critical for de-risking your activities.
At PrimeRA, we consider the bigger picture, the impact on other regions and the way in which you will be aiming to bring your product to patients.
We have experience engaging with national regulatory authorities and the European Medicines Agency (EMA) at all stages of development.
You can rely on our latest experiences, broad strategic view and working relationship with the agencies to navigate your interactions with ease.
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Achieving regulatory designations is both a mark of the importance of the project and also strategically provides key benefits that can be leveraged by the organisation aimed at making development more efficient.
Our experience in leveraging these designations allows us to work with you on the most appropriate timing for applying for any relevant designations as well as utilising the benefits accordingly.
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We have significant experience working on rare diseases that impact children. Critical to the development of medicines for children is the agreement on the paediatric investigational plan (PIP) with the European Medicines Agency.
A PIP is mandatory for both products being developed for children as well as those intended initially for adults, so getting the paediatric strategy right should be a key concern for all medicinal product developers.
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The PrimeRA Pharma Partners teams have extensive experience working in rare and ultra-rare diseases and especially those impacting children.
The first step on the regulatory pathway for potential products that might being developed in the rare disease space is often to apply for orphan designation with EMA and/or FDA to validate this. PrimeRA Pharma Partners are pleased to be able to prepare, negotiate and maintain orphan designations on behalf of our clients.
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Investors generally ask us, “What do the FDA think about this?”. Based on our experience, engagement with US FDA is critical in the development of most products and hence we have built a significant experience in engaging with FDA. Whether it is a pre-IND meeting or a meeting to resolve a specific issue, we can guide you through the process strategically and prepare the relevant documents to communicate your positions appropriately.
In addition to our working knowledge of the processes and precedent, we are able to take care of the interaction with FDA directly through our US Agent services.
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PrimeRA Pharma Partners are able to support access to opening an IND or starting an Investigator Initiated Trial (IIT) in China through our collaborations with local Chinese biotechs, clinical sites, consultants, CROs and manufacturing sites.
The China IIT pathway may offer cell and gene therapy companies an ability to generate de-risking proof-of-concept clinical data for a fraction of the time and cost compared to running the same study in the EU or US and are an attractive opportunity for obtaining initial clinical experience. Without local connections, many companies will struggle to enter this paradigm. This is where PrimeRA can help.
China should be considered in combination with regulatory interactions and opportunities in the UK, Europe, the US and other regions globally in order to ensure an appropriately aligned strategic and operational plan for the most efficient path to reaching patients.
To find out how PrimeRA Pharma Partners can help accelerate your development through China’s emerging regulatory pathways, please reach out to our team directly for a confidential discussion.
Regulatory Strategy
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The PrimeRA Pharma Partners team have been fortunate to work on a number of already authorised ATMPs in both the EU and US.
In particular, our team members have contributed to the development, commercialisation and/or maintenance of:
- The first gene therapy ever to be approved in Europe (AAV)
- The first CAR-T for CD19 directed malignancies to be submitted for approval in Europe
- The first CAR-T cell therapy product to be submitted for approval in the US
- The first CRISPR gene editing product to be approved globally
Our team are able to effectively navigate the Marketing Authorisation / Biologics License Application process, from strategic planning, to Rapporteur/Agency engagement, use of accelerated pathways, writing/reviewing MAA/BLA sections through to procedure management, team coordination, Agency management and negotiation and operational delivery of the review process including preparing responses to questions across all disciplines (regulatory, CMC, non-clinical, clinical).
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A local US point of contact is mandatory for all non-US companies wishing to interact with the US Food and Drug Administration (FDA).
Utilising our presence in the US, PrimeRA are able to directly represent clients in their interactions with the US FDA.
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A local EU point of contact is mandatory for all non-EU companies wishing to interact with the European Medicines Agency (EMA) or other EU competent authorities.
Utilising our presence in Dublin, PrimeRA are able to directly represent clients in their interactions with the EMA and relevant national competent authorities across EU Member States.
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We take a tailored approach to navigating the end-to-end regulatory paradigm with depth of experience in:
Preparing and leading regulatory meetings including pre-CTA/IND, INTERACT and Innovation Office
Securing designations and accelerated development pathways including Fast Track, Breakthrough, Orphan, RMAT and PRIME
Clinical Trial Strategy including initial CTA and IND applications and compilation of IMPD/Module 3
Marketing Authorisation and Biologics License Applications
Investment due diligence and ‘Technology to Spin-Out’ input
The PrimeRA team are able to write, review and strategically lead all regulatory procedures, with a depth of experience in delivery for cell and gene therapy products. The approach can be tailored to involve the scientists and other available client team members as needed, with PrimeRA filling in the resource gaps, however big or small, to ensure the key deliverables are achieved in line with expectations.
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Leverage our expert team and global network to pressure test your regulatory strategies and challenges.
We can contribute to your own Regulatory Advisory Board or lead it’s formation, hand-selecting the relevant consultants, delivering pre-read materials and coordinating relevant feedback.
Both solutions aim to ensure your strategy is robust and designed to meet regulatory expectations.
Submission & Compliance
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From building teams to delivering key milestones, our partners bring many years of hands-on global regulatory strategy and CMC leadership experience, providing a highly functional partner to our clients and enabling us to meet your organisation’s goals.
Our Partners will integrate with your team and become a pivotal department in your development program.
The benefits of interim and fractional leadership are clear - all the experience, ability to integrate and ability to scale as needed drawing on the wider PrimeRA team when projects get busy whilst maintaining a cost effective and highly efficient lean structure.
This approach is invaluable for early biotech companies and academic projects who require leadership without the full team headcount.
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PrimeRA Pharma Partners routinely work with cutting edge products that require significant innovation in the regulatory approaches and frameworks. In collaboration with our clients, we will evaluate all possible existing and new regulatory pathways to be able to bring the most efficient and effective development pathway to life.
Building on our early experiences in AAV gene therapy and CAR-T cell therapies, our team have contributed to programs designed around both genetically modified and un-modified cell processes, different vector types including (but not limited to) AAV, Adenovirus, Lentivirus, different cell types (including but not limited to: T-cell, HSPC, iPSC) and using different editing techniques (Including but not limited to: CRISPR, Base Editing).
We have spoken at length about the potential benefits and opportunities of Platform approaches in Europe (e.g. EMA Quality Innovation Group (QIG), November 2024; MEB Science Day, 2025) and have used emerging opportunities to the clear benefit of our clients (PRIME Pilot (EU); ATMP Pilot for Academics (EU); CATT Meeting (US)).
PrimeRA will always evaluate new and emerging opportunities to bring life changing medicines to patients globally in the most efficient manner possible. We look forward to discussing your development program and to finding the most effective regulatory strategies to facilitate your ambitions.
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PrimeRA Pharma Partners are please to be able to support investors with their due diligence on ATMP projects, drawing on the decades of experience translating these scientific research activities into commercially available medicinal products.
Leadership & Due Diligence
Approval Timeline
