Jove
Visualize
Contact Us
JoVE
x logofacebook logolinkedin logoyoutube logo
ABOUT JoVE
OverviewLeadershipBlogJoVE Help Center
AUTHORS
Publishing ProcessEditorial BoardScope & PoliciesPeer ReviewFAQSubmit
LIBRARIANS
TestimonialsSubscriptionsAccessResourcesLibrary Advisory BoardFAQ
RESEARCH
JoVE JournalMethods CollectionsJoVE Encyclopedia of ExperimentsArchive
EDUCATION
JoVE CoreJoVE BusinessJoVE Science EducationJoVE Lab ManualFaculty Resource CenterFaculty Site
Terms & Conditions of Use
Privacy Policy
Policies

Related Concept Videos

Preclinical Development: Overview01:28

Preclinical Development: Overview

5.6K
Preclinical development consists of a series of tests that ensure the safety and efficacy of a new therapeutic compound before it is tested in humans. There are four main phases to this process. First, safety pharmacology tests are conducted to ensure the drug does not produce any acutely harmful effects. These tests examine parameters such as bronchoconstriction, cardiac dysrhythmias, blood pressure changes, and ataxia. Next, preliminary toxicological testing is performed to determine the...
5.6K
Cell Lines01:16

Cell Lines

9.7K
A cell line is a population of cells grown in vitro that can be subcultured over several generations. Normal cells cease to divide after a certain number of cell divisions, a process known as replicative senescence. This number, called the Hayflick limit, was conceptualized by Leonard Hayflick in 1961 when he observed that fetal cells grown in culture could only divide 40-60 times. This limit is due to the shortening of the telomeres during each round of cell division, preventing cell division...
9.7K

You might also read

Related Articles

Articles linked to this work by shared authors, journal, and citation graph.

Sort by
Same author

Artificial Intelligence for Regulatory Evidence: A Systematic Document Analysis of European Medicines Agency Regulatory Advice and Public Reports.

Clinical pharmacology and therapeutics·2026
Same author

Reduced Genotoxicity Testing Is Possible for Noncoding Oligonucleotide-Based Therapeutics Containing Well-Characterized Modifications: A European Regulatory Perspective.

Nucleic acid therapeutics·2026
Same author

Core outcome domain sets for clinical trials in epidermolysis bullosa - a COSEB protocol to achieve consensus on "what" to measure.

Trials·2025
Same author

The European Medicines Agency's review of elexacaftor/tezacaftor/ivacaftor: extending its use to all people with cystic fibrosis aged 2 years and older who do not have two class I <i>CFTR</i> variants.

The European respiratory journal·2025
Same author

Addressing global regulatory challenges in rare disease drug development.

Drug discovery today·2025
Same author

From roadmap to a sustainable end-to-end individualized therapy pathway.

Therapeutic advances in rare disease·2025

Related Experiment Video

Updated: Dec 12, 2025

High Content Screening in Neurodegenerative Diseases
13:32

High Content Screening in Neurodegenerative Diseases

Published on: January 6, 2012

18.0K

Mining scientific advice reports on cell-based products: Insight into the nonclinical development program.

Tineke van den Hoorn1, Tahira Nakchedi1, A Charlotte M T de Wolf1

  • 1Medicines Evaluation Board, Graadt van Roggenweg 500, Utrecht, AH, 3531, The Netherlands.

British Journal of Clinical Pharmacology
|August 14, 2020
PubMed
Summary

Nonclinical development for cell-based therapies is challenging due to species-specificity. Studies show a tailored approach focusing on proof-of-concept, with in vitro methods potentially sufficing for tumorigenicity assessment.

Keywords:
advanced therapy medicinal productbiodistributioncell-based productsnonclinicalscientific advicetumourigenicity

More Related Videos

Preclinical Drug Testing in Scalable 3D Engineered Muscle Tissues
08:07

Preclinical Drug Testing in Scalable 3D Engineered Muscle Tissues

Published on: April 7, 2023

4.1K
Autonomously Bioluminescent Mammalian Cells for Continuous and Real-time Monitoring of Cytotoxicity
04:47

Autonomously Bioluminescent Mammalian Cells for Continuous and Real-time Monitoring of Cytotoxicity

Published on: October 28, 2013

10.4K

Related Experiment Videos

Last Updated: Dec 12, 2025

High Content Screening in Neurodegenerative Diseases
13:32

High Content Screening in Neurodegenerative Diseases

Published on: January 6, 2012

18.0K
Preclinical Drug Testing in Scalable 3D Engineered Muscle Tissues
08:07

Preclinical Drug Testing in Scalable 3D Engineered Muscle Tissues

Published on: April 7, 2023

4.1K
Autonomously Bioluminescent Mammalian Cells for Continuous and Real-time Monitoring of Cytotoxicity
04:47

Autonomously Bioluminescent Mammalian Cells for Continuous and Real-time Monitoring of Cytotoxicity

Published on: October 28, 2013

10.4K

Area of Science:

  • Pharmacology and Toxicology
  • Regenerative Medicine
  • Biotechnology

Background:

  • Cell-based therapies are transitioning from experimental to established treatments for human diseases.
  • Designing nonclinical development programs for these products is complex due to species-specific limitations.
  • Evaluating regulatory advice reports is crucial for understanding nonclinical development strategies.

Purpose of the Study:

  • To analyze the nonclinical development programs for cell-based products.
  • To identify common approaches and challenges in regulatory submissions.
  • To assess the necessity and design of in vivo and in vitro studies, including biodistribution and tumorigenicity.

Main Methods:

  • Review of European Medicines Agency (EMA) advice reports from 2013-2018.
  • Analysis of the number, purpose, and design of in vivo and in vitro studies.
  • Evaluation of consistency in nonclinical development programs across different cell-based products.

Main Results:

  • In vivo studies predominantly focused on proof-of-concept (87%), followed by safety (74%), biodistribution (57%), and tumorigenicity (54%).
  • For 7% of products, no animal studies were proposed, particularly for those with extensive human clinical data.
  • In vitro methods were deemed sufficient for tumorigenicity assessment in some cases, and in vivo biodistribution/tumorigenicity studies were omitted for one-third of products.

Conclusions:

  • Nonclinical development for cell-based products is more tailored and proof-of-concept driven than for conventional medicines.
  • In vitro approaches may be adequate for evaluating tumorigenicity.
  • Omitting in vivo studies is feasible for products with substantial clinical experience.