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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This NCATS-led pilot project will test whether it is possible to increase the efficiency of Gene Therapy clinical trial startup by using the same Gene Delivery System and manufacturing methods for multiple gene therapies.
View this resource Bookmark this resourceIt is a premier source of important information, knowledge, and insights in translational and clinical research.
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Quality, preclinical and clinical aspects of Gene Therapy medicinal products
Published by EMA
Regulatory AffairsAdvanced TherapyGene TherapyQuality GuidelineATMPNon-ClinicalGuide 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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Guideline On The Non-Clinical Studies Required Before First Clinical Use Of Gene Therapy Medicinal Products
Published by EMA
Early Access SupportRegulatory AffairsAdvanced TherapyGene TherapyNon-ClinicalFirst Clinical UseThis document defines scientific principles and provides guidance to applicants developing Gene Therapy medicinal products (GTMPs). It focuses on the non-clinical studies required before the first use of a GTMPs in human subjects.
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Guideline On Development And Manufacture Of Lentiviral Vectors
Published by EMA
Regulatory AffairsEarly Access SupportAdvanced TherapyGene TherapyATMPLentiviral VectorThis guideline describes quality aspects that are relevant for lentiviral vectors (LV). It applies to LV intended for ex vivo or in vivo application.
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Certification for advanced therapy medicinal products
Published by IRDiRC
Regulatory AffairsEarly Access SupportAdvanced TherapyCertificationCommittee for Advanced TherapiesATMP CertificationThe European Medicines Agency’s Committee for Advanced Therapies (CAT) provides a certification procedure for advanced therapy medicinal products (ATMPs) under development by micro-, small- and medium-sized enterprises (SMEs) only
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Magisterial hospital preparations – hospital exemptions
Published by IRDiRC
Regulatory AffairsEarly Access SupportHospital ExemptionATMPsAdvanced TherapyArticle 28 of the ATMP Regulation also empowers Member States to permit the manufacturing and use of certain non- routine produced advanced therapies that have not been authorised by the Commission under certain conditions (so-called “Hospital Exemption”) outside the scope of the Medicinal Product Directive 2001/23.
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Regenerative Medicine Advanced Therapy (RMAT) Designation
Published by IRDiRC
Regulatory AffairsEarly Access SupportAdvanced TherapyRegenerative Medicine Advanced Therapy DesignationThe RMAT Designation is analogous to the Breakthrough Designation designed for traditional drug candidates but applies to regenerative medicine treatments, and allows companies to interact with the FDA more frequently during the clinical development of the therapy. An RMAT-designated therapy is eligible for priority review and accelerated approval.
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