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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 22

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

ABPI Bridging between pre- and clinical boundary

  • United Kingdom
  • 2018
  • The Association of the British Pharmaceutical Industry (ABPI)
A report for a joint workshop held by the Academy of Medical Sciences and the Association of the British Pharmaceutical Industry (ABPI). The meeting looked to identify challenges, gaps, and opportunities for greater integration of pre-clinical and clinical science, and the vision for the future of translational research in the UK.
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  • Fundamental Research
  • Clinical research
  • Translational Science
  • Funding

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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  • 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

ARM Project A-Cell

  • Global
  • 2022
  • ARM
The Alliance for Regenerative Medicine (ARM) and the National Institute for Innovation in Manufacturing Biopharmaceuticals (NIIMBL) produced A-Cell, a case study-based guide to integrating Quality by design (QbD) principles in cell therapy Chemistry Manufacturing & Control (CMC) programs. 
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  • 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

EJP RD Innovation Management Toolbox

  • EU
  • EJP RD
The European Joint Programme on Rare Diseases (EJP RD) Innovation Management Toolbox is a reference library of resources in rare disease translational medicine. The toolbox includes: a searchable resources library, a questions and answers section, and use cases.
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  • Research and Innovation
  • Fundamental Research
  • Clinical research
  • Translational Science
  • Funding
  • Incentives
  • Early interaction with regulators
  • Therapy classification
  • Legal classification
  • ATMPs
  • Good Manufacturing Practice
  • Market access for ATMPs
  • Pharmacovigilance
  • Pricing & reimbursement
  • Data protection
  • Data storage
  • Patient & Public Involvement
  • Ethics

IGI CRISPR Genome Engineering Tools & Reagents

  • USA
  • IGI
CRISPR Genome Engineering Tools & Reagents published by the Innovative Genomics Institute (IGI). The resources include protocols, reagents, computational tools for setting up CRISPR experiments, and analyzing data.
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  • Fundamental Research

Alcyomics and NA-ATTC Current Guidelines on Advanced Cell Therapy Pre-Clinical Procedures

  • UK
  • 2022
  • ATTC
Alcyomics and the Northern Alliance Advanced Therapies Treatment Centre (NA-ATTC) have shared a report on the existing guidelines and some of the current in vitro technologies used within academia and industry to assess the safety, efficacy and potency of advanced cell therapies.
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  • Fundamental Research
  • ATMPs

ATTC Manufacturing and Preparation Toolkit

  • UK
  • ATTC
The Manufacturing and Preparation Toolkit developed by The Advanced Therapy Treatment Centres (ATTC) network contains information on best practice and expert guidelines for manufacturing Advanced Therapy Medicinal Products (ATMPs). The guide is categorised into areas of Cell Analysis, Cell Manufacture, and Pharmacy & Regulatory, and accompanied by case studies.
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  • Fundamental Research
  • Clinical research
  • Translational Science
  • Legal classification
  • ATMPs
  • Manufacturing
  • Manufacturing Authorisation
  • Good Manufacturing Practice
  • Investigational medicinal products dossier
  • Capability
  • Scalability
  • Packaging and labelling
  • Distribution
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