Heavy! Organoids are included in CDE gene therapy and cell therapy technology guidelines for the first time

Recently, the Center for Drug Evaluation (CDE) of the State Drug Administration issued three guiding principles related to gene therapy and cell therapy, For the first time, organoids are included in the validation guidelines for gene therapy and genetically modified cell therapy products.

All three guiding principles will be effective from the date of issuance. in:

1. “Technical Guiding Principles for Non-clinical Research and Evaluation of Gene Therapy Products (Trial)” states: Non-clinical pharmacological research on gene therapy products“If there is no suitable animal model to meet the experimental needs, corresponding animal models should be developed based on scientific principles or more complete in vitro test systems or alternative models (such as organoids) should be used to conduct experiments ”

2. “Technical Guiding Principles for Non-clinical Research on Gene Modified Cell Therapy Products (Trial)” states: For gene modified cell therapy products “When relevant animal models are lacking, cell- and tissue-based models (such as two-dimensional or three-dimensional tissue models, organoids and microfluidic models, etc.) can be used to provide useful supplementary information for the evaluation of effectiveness and safety.”

 

What are organoids?

Organoids are micro-organs with a three-dimensional structure grown in vitro using adult stem cells. Genetic background and histological characteristics that are highly similar to those of organs in the body; have complex structures similar to real organs, and can partially simulate the physiological functions of source tissues and organs; conduct tissue biology, developmental regeneration, disease modeling, organ transplantation technology improvement, drug discovery/efficacy evaluation, and toxicology research by simulating the endogenous environment.

In layman’s terms, It is to build an organ environment outside the body similar to that in the body, and then use targeted administration to screen out which drug is most suitable for the treatment of this environment, thereby giving patients a certain reference for medication.

Tumor tissue and body fluid (cancerous pleural and ascites fluid) containing tumor cells are obtained from the patient, cultured and expanded in vitro to become a “patient substitute” that can restore the tumor genetics and tumor biological characteristics in the body, which isTumor organoids.

In 2017, organoids were named Technology of the Year in the life sciences by Nature Methods magazine.

In 2021, documents from the National Ministry of Science and Technology, “Stem cell-based models of major refractory human diseases” has also been listed as the first batch of key projects in the “14th Five-Year Plan”. The document clearly states that malignant tumors are one of the major refractory diseases that have a long-term serious impact on the health of the Chinese people, and organoids, as an important emerging cutting-edge technology, can help disease research and treatment.

Because organoids can simulate the three-dimensional structure and function of real organs in the body, they provide new research methods and treatments for precision medicine, and have broad application prospects in a range of biology and biomedicine. It is expected that organoids will play an increasing role in the field of biological medicine in the future.