Advanced therapy medicinal products (ATMPs) are currently undergoing a boom. Several products including viral, non-viral and cell-based products have forged a path to market. In their wake, academics and clinicians continue to lead innovation and unlock the clinical potential of new approaches and technologies, whereas increased industry interest is stimulating investment and industrialization of the processes that lead to commercialization.
This online training course is designed to give a broad scientific and regulatory overview of the current state of the art and challenges facing ATMP development. It will follow the life cycle of an ATMP from concept to clinic and commercialization. and it will take into consideration global regulatory requirements and the often-challenging regulatory problems that ATMPs face.
Overview of small molecule drug development vs complex biological drug development
EU regulatory framework for ATMPs
US regulatory framework for ATMPs
2.Getting on the right track: early stage regulatory procedures for ATMP development
Early stage regulatory procedures brief overview Orphan drug designation (ODD), Paediatric investigation plans (PIPs) and Pediatric study plans (PSPs)., Innovation meetings / scientific advice / protocol assistance / pre-IND meetings, Risk-based assessment and PRIME
3.Chemistry, manufacturing and controls (CMC) for ATMPs
Introduction to GMP and GMP guidelines
Gene therapy manufacture main concepts
Cell-based therapy manufacture main concepts
QP release and import/export
Overview of nonclinical studies and timing
Certification of CMC and/or nonclinical data for ATMPs and SMEs
CTAs and IMPDs/IBs and INDs
Clinical trial design considerations
6.Marketing authorization procedures
Centralised procedure (EU) and Biologics license application (BLA) (USA)
Accelerated assessments, expedited pathways and conditional approval
Hospital exemption and compassionate use
Post-marketing commitments including PSUR and Risk management plans (RMPs)
Regulatory Affairs Manager, Officer and Specialist
working for pharmaceutical company, Biotech, spin-offs and CROs.
It is also addressed to Researchers and academics in the ATMP field.
Participants need to have a basic understanding of regulatory procedures and/or a basic knowledge of ATMP biology.
This limited enrolment online training course is intended to be a virtual class where various expert will guide you into the topics through real world case studies and examples and answering your questions.
Christopher has a PhD in Biomedicine. Christopher had an academic research career in gene therapy including muscle regeneration and muscular dystrophy as well as using muscle as a platform for gene therapy applications including diabetes. He has over seven years’ experience as a regulatory expert in medical writing and scientific and regulatory affairs for chemical and biologic products, including advanced therapies, biomaterials and complex new technologies. He has experience in both EU and US regulatory affairs including scientific advice/pre-IND meetings, clinical trial documentation, paediatric assessments, and commercialization procedures. As Scientific and Regulatory Affairs Associate Director at Asphalion, Christopher is involved in regulatory strategy, regulatory roadmaps and support for startups and biotechs, qualification of novel methodologies for biologicals, biomaterials, and advanced therapies. He also manages and leads regulatory Work Packages for H2020 projects.
Marta has a Pharmacy degree and a Specialist postgraduate degree qualification in Industrial Pharmacy and Galenic Formulation. She has over seven years’ experience in drug development in the pharma Industry as R&D Project Manager on the development of drug candidates, and over five years’ experience in regulatory affairs. As Scientific and Regulatory Affairs Associate Director at Asphalion, Marta is responsible of scientific and medical writing of regulatory documents working in regulatory strategy, regulatory roadmaps, ATMP designation, scientific advice and qualification of novel methodologies for biologicals, biomaterials, medical devices, and advanced therapies. She also manages regulatory Work Packages of H2020 projects.
Scientific and Regulatory Affairs Director, Asphalion S.L.
Núria has a PhD in Pharmacy. She has an academic research career and a broad scientific background in the area of microbiology and pharmacy with over ten years’ experience as regulatory expert in charge of medical and scientific writing. As the director of the Scientific and Regulatory Affairs unit, Núria is a recognized expert in small molecules as well as innovative, biological and advanced therapy medicinal products (ATMPs), including gene and somatic cell therapies. She is also responsible for managing regulatory work packages of FP7 and H2020 projects and working in regulatory strategy and has vast experience in a range of global procedures including registration dossiers, IMPDs, IBs, CTAs, INDs, scientific advice, pre-INDs meetings and regulatory procedures during development.
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