Key Pathway Resources

European Medicines Agency

Key European Medicines Agency (EMA) resources for cell and gene therapy development.

ATMP Sweden Regulatory Guide

ATMP Sweden has compiled supporting resources and reports regarding ATMP development. Learn more about ATMP Sweden's Regulatory Guide and additional resources:

EJP RD Innovation Management Toolbox

EJP RD's searchable reference library of resources in rare disease translational medicine. Learn more about this toolbox and other EJP RD resources:

ARM Project A-Gene and Project A-Cell

The Alliance for Regenerative Medicine (ARM) and partners have produced A-Gene and A-Cell, case study-based guides to integrating Quality by design (QbD) principles in gene therapy and cell therapy Chemistry Manufacturing & Control (CMC) programs. Read about these projects and addiitonal ARM resources:

ABPI ATMP Roadmap

The Association of the British Pharmaceutical Industry (ABPI) has developed an ATMP roadmap, setting out the key steps in the end-to-end pathway for ATMPs in England from non-clinical research through to patient treatment & monitoring. Learn more about this roadmap and other relevant resources developed by ABPI:

EATRIS Transmed Academy and ADVANCE Project

See resource developed by EATRIS, including education and training initiatives for the next generation of ATMP professionals, patient engagement and more:

IRDiRC Orphan Drug Development Guide

A patient-focused guidebook by the International Rare Diseases Research Consortium (IRDiRC) on developing drugs or therapies for rare disease indications.

ATTC Manufacturing and Preparation Toolkit

The Advanced Therapy Treatment Centres (ATTC) developed the Manufacturing and Preparation Toolkit to provide expert guidelines for manufacturing ATMPs. Learn more about this toolkit and more from ATTC:

NCATS Platform Vector Gene Therapy (PaVe-GT) pilot project

The National Center for Advancing Translational Sciences (NCATS) PaVe-GT pilot project aims to increase the efficiency of clinical trial startup by using the same gene delivery system and manufacturing methods for multiple rare disease gene therapies. Learn more about this project and additional resources developed by NCATS:

Restore Project

The Restore project develops technologies that will enable the reproducible and economically feasible manufacturing of Advanced Therapies.

Read about the roadmaps, reports, factsheets produced by this project:

Orphanet

The Orphanet knowledgebase gathers and provides high-quality information on rare diseases for improving the diagnosis, care and treatment of patients with rare diseases.

Key UK Resources

A collection of key resources for gene and cell therapy developers in the UK context