Innovative Therapies and Personalised Medicine for Rare Diseases

Published by EJP RD

Cell Therapy Course Gene Therapy Regenerative Medicine Tissue Engineering

A course to learn how recent advances in medicine research, such as : gene therapy, protein-based treatments, regenerative medicine, cell therapy and tissue engineering and more in general, personalized approaches, are transforming the treatment of rare diseases.

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The target product profile

Published by CBER

Cell Cell Therapy Gene Therapy Research and Drug Development Target Product Profile Tissue

The objective of this presentation is to discuss the TPP and its role in the development of cellular and gene therapies

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Quality, preclinical and clinical aspects of Gene Therapy medicinal products

Published by EMA

Advanced Therapy Cell Therapy clinical Gene Therapy Genetically Modified Cell Non-Clinical Quality somatic cell

Guide for the development and evaluation of Gene Therapy Medicinal Products (GTMPs) intended for use in humans and presented for Marketing Authorisation Application (MAA). Its focus is on the quality, Safety and efficacy requirements of GTMPs.

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Genetherapy.net

Published by GeneTherapy.net

Advanced Therapy Cell Therapy Drug Discovery EMA Gene Editing Tool Gene Therapy Non-Viral Vector Regulation Affairs Research and Drug Development

Web resource for basic and clinical research in Gene Therapy, cell therapy, and genetic vaccines. In addition, it provides an overview of the different international regulations and guidelines associated with clinical Gene Therapy trials.

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The Innovation Management Toolbox was originally developed through funding from the EJP RD project, which received funding from the European Union’s Horizon 2020 research and innovation programme under grant agreement No. 825575. The continued development and maintenance of the Toolbox are now supported by the ERDERA partnership, funded under the European Union’s Horizon Europe Research & Innovation programme, grant agreement No. 101156595.

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