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Glossary

Adeno-associated virus (AAV)
Adenovirus
Adult stem cells
Antibody
Antigen
Base editors
Cas9 nuclease
Chimeric Antigen Receptor (CAR)
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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 20
EuroGCT Resource

Translational Science

  • EU and UK
  • 2023
  • EuroGCT
Translational science translates basic science discoveries into therapeutic applications.
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  • Show all 2
  • Research and Innovation
  • Translational Science
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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Theme

  • 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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  • 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

Viral vectors-what are the solutions to current scale up challenges?

  • UK
  • 2019
  • CGT Catapult
A presentation by Cell and Gene Therapy Catapult (CGT Catapult) on the current state, barriers, and solutions to challenges in viral vector manufacturing
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  • Fundamental Research
  • Clinical research
  • Translational Science
  • Capability
  • Scalability

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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  • 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

EU Regulatory Pathways for ATMPs: Standard, Accelerated and Adaptive Pathways to Marketing Authorisation

  • 2019
  • Molecular Therapy - Methods & Clinical Development
Advanced therapy medicinal products (ATMPs) require evaluation by the European Medicines Agency’s Committee for Advanced Therapies prior to being placed on the European market, subject to a Marketing Authorisation granted by the European Commission. This review discusses how the regulatory pathway for any particular ATMP, with or without PRIME designation, is determined and navigated.
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  • Clinical research
  • Translational Science
  • Legal classification
  • ATMPs
  • Good Manufacturing Practice
  • Market access for ATMPs
  • Pharmacovigilance

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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  • 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

EATRIS Transmed Academy

  • EU
  • EATRIS
EATRIS Transmed Academy is the EATRIS online learning environment for translational scientists. Find here self-paced online courses, live courses, workshops, recorded webinars, guidance documents, resources and more.
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  • Research and Innovation
  • Translational Science
  • Funding
  • Legal classification
  • Manufacturing
  • Commercialisation

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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  • 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
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