Research Pathways

Bringing gene and cell therapies from the lab bench to patients is a complex process.The Research Pathways directory contains resources to help guide various stakeholders interested in therapy development through this process. 

The Research Pathways is centred around the legal and regulatory aspects that are required for cell and gene therapy development in Europe, from binding law, guidelines and recommendations, then further expanded to additional aspects including ethics, scientific developments, commercialisation, health economics and more. 

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Key Pathways Resources

Signposting to key resources on ATMP development

Case Studies

Learn from specific development cases and relevant resources

Patient and Public Involvement

ATMP Engage Patient and Public Involvement Directory

Actors & Networks

Mapping the stakeholders in Europe and beyond

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Showing 10 of 15

Manufacturing process development of ATMPs within a regulatory framework for EU clinical trial & marketing authorisation applications

  • 2016
  • Cell And Gene Therapy Insights
In the European Union, the regulatory approval of ATMPs for use in patients requires that data demonstrating their quality, safety and efficacy are submitted in dossiers to regulatory agencies for review. Indeed, such dossiers have a specific format that, in the case of quality data in particular, is informative for the manufacturing process development strategy. This manuscript describes how dossier requirements can be implemented into the design of industrialized ATMP manufacturing processes and fulfilled to enable effective regulatory submissions.
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Theme

  • Show all 10
  • Clinical research
  • ATMPs
  • Manufacturing
  • Manufacturing Authorisation
  • Good Manufacturing Practice
  • Investigational medicinal products dossier
  • Capability
  • Scalability
  • Packaging and labelling
  • Market access for ATMPs
EuroGCT Resource

Case Study: Holoclar

  • EU and UK
  • 2023
  • EuroGCT
Case Study on Holoclar: Holoclar is one of the few ATMPs that have been granted marketing authorisation in Europe. We collated existing information about each stage of development for Holoclar, from early-stage research to approval, for the research community to learn about its development process.
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Theme

  • Show all 18
  • Research and Innovation
  • Fundamental Research
  • Clinical research
  • Translational Science
  • Funding
  • Incentives
  • Early interaction with regulators
  • Legal classification
  • ATMPs
  • Manufacturing
  • Manufacturing Authorisation
  • Good Manufacturing Practice
  • Investigational medicinal products dossier
  • Capability
  • Scalability
  • Packaging and labelling
  • Commercialisation
  • Market access for ATMPs

New guidance on the development and marketing of ATMPs in the UK and EU in the post-BREXIT landscape

  • UK
  • 2022
  • CGT Catapult
A guidance by the Cell and Gene Therapy Catapult (CGT Catapult) on the development and marketing of ATMPs in the UK and EU in the post-BREXIT landscape.
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Theme

  • Show all 15
  • Clinical research
  • Translational Science
  • Early interaction with regulators
  • Therapy classification
  • Legal classification
  • ATMPs
  • Not ATMPs: Human body elements for therapeutic applications
  • Not ATMPs: medical devices containing tissues and cells
  • Manufacturing Authorisation
  • Good Manufacturing Practice
  • Packaging and labelling
  • Market access for ATMPs
  • Distribution
  • Pharmacovigilance
  • Pricing & reimbursement

EMA Guidelines relevant for advanced therapy medicinal products

  • EU
  • European Medicines Agency (EMA)
Scientific guidelines developed by The European Medicines Agency (EMA) relevant for Advanced Therapy Medicinal Products (ATMPs).
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Theme

  • Show all 13
  • Fundamental Research
  • Clinical research
  • Translational Science
  • ATMPs
  • Manufacturing
  • Manufacturing Authorisation
  • Good Manufacturing Practice
  • Investigational medicinal products dossier
  • Capability
  • Scalability
  • Packaging and labelling
  • Market access for ATMPs
  • Pharmacovigilance
EuroGCT Resource

[Poster] The challenges of Advanced Therapy Medicinal Products manufacturing in the European Union: Strengths and limits of current regulatory tools

  • EU and UK
  • 2023
  • EuroGCT
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Theme

  • Show all 2
  • Manufacturing Authorisation
  • Good Manufacturing Practice

The EBMT/EHA CAR-T Cell Handbook

  • EU
  • 2022
  • EBMT, EHA and Springer
This open access CAR-T Cell Handbook published by Springer and co-promoted by the European Society for Blood and Marrow Transplantation (EBMT) and the European Hematology Association (EHA), provides an unparalleled overview of the CAR-T cell technology and covers several aspects of CAR-T cell treatments, including the underlying biology, indications, management of side-effects, access and manufacturing issues.
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Theme

  • Show all 14
  • Fundamental Research
  • Clinical research
  • Manufacturing
  • Manufacturing Authorisation
  • Good Manufacturing Practice
  • Investigational medicinal products dossier
  • Capability
  • Scalability
  • Packaging and labelling
  • Market access for ATMPs
  • Distribution
  • Pharmacovigilance
  • Pricing & reimbursement
  • Ethics

ATMP Sweden Regulatory Guide

  • Sweden
  • ATMP Sweden
ATMP Sweden's regulatory guide for the development of the Advanced Therapy Medicinal Products (ATMPs)
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Theme

  • Show all 12
  • Fundamental Research
  • Clinical research
  • Early interaction with regulators
  • Legal classification
  • ATMPs
  • Manufacturing
  • Manufacturing Authorisation
  • Good Manufacturing Practice
  • Investigational medicinal products dossier
  • Capability
  • Scalability
  • Packaging and labelling

ABPI ATMP Roadmap

  • UK
  • 2021
  • ABPI
The Association of the British Pharmaceutical Industry (ABPI) has developed an ATMP roadmap which sets out the key steps in the end-to-end pathway for ATMPs in UK, from non-clinical research through patient treatment and monitoring.
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Theme

  • Show all 15
  • Fundamental Research
  • Clinical research
  • Translational Science
  • Early interaction with regulators
  • Legal classification
  • ATMPs
  • Manufacturing Authorisation
  • Good Manufacturing Practice
  • Investigational medicinal products dossier
  • Commercialisation
  • Market access for ATMPs
  • Distribution
  • Pharmacovigilance
  • Pricing & reimbursement
  • Data storage

NCATS PaVe-GT: Paving the Way for Rare Disease Gene Therapies

  • USA
  • NCATS
The Platform Vector Gene Therapy (PaVe-GT) pilot project lead by National Center for Advancing Translational Sciences (NCATS) at National Institutes of Health (NIH) seeks to increase the efficiency of clinical trial startup by using the same gene delivery system and manufacturing methods for multiple rare disease gene therapies.
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Theme

  • Show all 9
  • Fundamental Research
  • Clinical research
  • Translational Science
  • ATMPs
  • Manufacturing Authorisation
  • Good Manufacturing Practice
  • Investigational medicinal products dossier
  • Capability
  • Scalability

ARM Project A-Gene

  • Global
  • 2021
  • ARM
The Alliance for Regenerative Medicine (ARM) and the National Institute for Innovation in Manufacturing Biopharmaceuticals (NIIMBL) produced A-Gene, a case study-based guide to integrating Quality by design (QbD) principles in gene therapy Chemistry Manufacturing & Control (CMC) programs.
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Theme

  • Show all 12
  • Fundamental Research
  • Clinical research
  • Translational Science
  • Early interaction with regulators
  • Manufacturing
  • Manufacturing Authorisation
  • Good Manufacturing Practice
  • Investigational medicinal products dossier
  • Capability
  • Scalability
  • Packaging and labelling
  • Market access for ATMPs
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