We will review challenges in translation required to develop the ATMPs pharmaceutical product (cell and gene therapy products) from nonclinical to the clinical stage, as well as the challenges related to the determination of proof-of-concept, how to choose clinical dose from nonclinical studies and address the nonclinical questions required to demonstrate ATMP product safety before going into the first-in-human study. Presenters will also discuss how European Regulatory Agencies help the development of ATMP pharmaceutical products.

 Key learning points :

  • Understand in-depth the product in relation to the intended use
  • Justify the study designs, test model(s) (in vitro, ex vivo, and/or in vivo), and/or absence of specific studies
  • Use integrated approach covering CMC/NC/Clinical aspects critical for ATMPs pharmaceutical product
  • Adapt QbD principles such as the development of an adapted Control Strategy based on Risk Assessment: it is possible and encouraged
  • Engage early dialogue with EMA and National agencies: strongly encouraged for successful such development
Romaldas

Professor Romaldas Maciulaitis

Professor Romaldas Maciulaitis has worked at the Institute of Physiology and Pharmacology in the Department Nephrology at the Lithuanian University of Health Studies for more than 28 years. He has also been a CHMP and CAT member at the EMA for 14 years.

The Professor completed his MD and PharmD studies more than 20 years ago and after PhD studies in Clinical Pharmacology, was involved into Drug Regulatory and Scientific Appraisals at the national (Lithuania) and international (European) levels. In 2009, he initiated new experimental Pharmacology research direction in his University in Kaunas on Regenerative Pharmacology applying cell preparations with research program focusing on PK/PD of cell therapies in renal and cartilage injuries. He has published more than 20 papers in reputed journals.

Dr Narayanan

Dr Gopalan Narayanan, Vice President, Disruptive Biologics

Within VCLS, Dr Narayanan provides leadership in the area of complex and disruptive biologics such as Cell and Gene therapies, called Advanced Therapies Medicinal Products (ATMPs) in Europe, including guidance on product development and regulatory strategy. In addition, he participates in the global effort of rationalising the development process, by enabling innovative regulatory mechanisms through which Advanced Therapies and other disruptive biologics can be developed and brought to patients faster and more efficiently.

Cécile_Rousseau

Cecile Rousseau, Director

With 10+ years in nonclinical and translational research and histotechnology, Dr Cécile F. Rousseau has considerable expertise in medical devices, engineered cellular therapies and companion diagnostics. 

Upon completing a Ph.D. program in Medical and Biological Engineering at the University Claude Bernard – Lyon 1 in 2002, Cécile proceeded with postdoctoral research in tissue engineering (including but not limited to cartilage, soft organs), toxicology and pharmacology at the Laboratory of Cartilage Biology and Engineering and at the Laboratory of Molecular Assembly of Biological Interest.

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