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What is gene and cell therapy?
What are ATMPs, and where do they fit into gene and cell therapy?
Why are animal models used when testing the safety of a new therapy?
What role do preclinical and clinical trials play in ensuring patient safety?
Why are some conditions more suitable for gene and cell therapies than others?
There is an approved treatment available for my condition - why can't I receive it?
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How are medicines developed and approved?

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Research Pathways

Bringing gene and cell therapies from the lab bench to patients is a complex process and multiple pathways exists. The Research Pathways directory contains resources to help various stakeholders interested in therapy development navigate this process. Research Pathways is centred around the legal and regulatory aspects for cell and gene therapy development in Europe and further expanded to additional aspects. It is free to use and all resources in this database are categorised for improved searchability.

How to use Research Pathways  EuroGCT's Key Resources

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Showing 10 of 39
EuroGCT Resource

Expediting marketing authorisation pathways

  • EU and UK
  • 2023
  • EuroGCT
Within the European Union, three legal procedures have been developed to expedite access to new medicines, including ATMPs: conditional marketing authorisation, marketing authorisation under exceptional circumstances, and accelerated assessment.
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Theme

  • Show all 2
  • Commercialisation
  • Market access for ATMPs
EuroGCT Resource

Support for innovative medicines’ development at the EMA level

  • EU and UK
  • 2023
  • EuroGCT
EMA's support for innovative medicines’ development aims to clarify the applicable regulatory requirements for the medicinal products in development. Both procedures and services support the developers through establishing best strategies as early as possible for obtaining the required data (robust quality, pre-clinical, and clinical data) for commercialising innovative medicinal products (including ATMPs) in the European Union (EU) through the applicable and most adapted regulatory pathway.
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  • Show all 3
  • Research and Innovation
  • Early interaction with regulators
  • Market access for ATMPs
EuroGCT Resource

Case Study: Early therapy classification considerations in developing skin equivalents

  • EU and UK
  • 2024
  • EuroGCT
How a therapy is developed and produced can affect its classification and determines the legal mechanisms that govern its regulation. In the case of developing skin equivalents for healing skin wounds, choices of preparation steps results in different therapy classification.
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Theme

  • Show all 10
  • Fundamental Research
  • Clinical research
  • Incentives
  • Early interaction with regulators
  • Legal classification
  • ATMPs
  • Not ATMPs: Human body elements for therapeutic applications
  • Good Manufacturing Practice
  • Market access for ATMPs
  • Pharmacovigilance
EuroGCT Resource

[Poster] The marketing authorisation of advanced therapy medicinal products under the regulation of the European Union

  • EU and UK
  • 2023
  • EuroGCT
This poster presents the European regulatory requirements and pathways for ATMPs to obtain marketing authorisation, and discuss the challenges of their commercialisation.
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Theme

  • Show all 2
  • ATMPs
  • Market access for ATMPs

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: Strimvelis

  • EU and UK
  • 2023
  • EuroGCT
Case Study on Strimvelis: Strimvelis is the first ex vivo gene therapy to be licensed, and its success is the result of a joint effort among different stakeholders. Here we collated existing information about each stage of its development as a guide for the research community to learn about its development process.
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  • Show all 13
  • Fundamental Research
  • Clinical research
  • Translational Science
  • Funding
  • Market access for ATMPs
  • Pharmacovigilance
  • Pricing & reimbursement
  • Data
  • Data protection
  • Data storage
  • Data collection, processing, controlling
  • Data sharing / Open Data
  • Mission creep / data misuse
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

Taking ATMPs to Market

  • UK
  • 2018
  • CGT Catapult
A presentation by Cell and Gene Catapult (CGT Catapult) on topics including: ATMP regulatory framework in EU, therapy classification, early development support by EMA (EU) and MHRA (UK), and market access pathways.
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Theme

  • Show all 4
  • Early interaction with regulators
  • Legal classification
  • ATMPs
  • Market access for ATMPs

Creating EU market access roadmaps for cell and gene therapies

  • UK
  • 2016
  • CGT Catapult
Market access roadmaps for cell and gene therapies published by Cell and Gene Therapy Catapult (CGT Catapult). Contains individual reimbursement roadmaps for UK (England and Wales), and EU countries (Spain, Italy, France, and Germany).
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Theme

  • Show all 9
  • Funding
  • 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
  • Market access for ATMPs
  • Pricing & reimbursement

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
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