Advanced Therapy Products in Brazil: Regulatory Aspects

Silva, João Batista; Parca, Renata Miranda; Carvalho, Adriana Bastos

doi:10.1007/978-3-031-34567-8_7

Cited by 3 publications

(1 citation statement)

References 11 publications

Supporting

Mentioning

Contrasting

Order By: Relevance

“…With the first Brazilian regulatory norms established in 2018 and their most recent update in 2021, 5 gene therapy products have already been approved for clinical use in the country, and there are ongoing clinical trials for the registration of new therapies. 27 Indeed, different strategies to improve the understanding of the regulatory aspects of ATPs are being carried out, 27 and the technology of iPSCs will accompany these advances. While the translation into clinical practice still involves a long road ahead, public and private health institutions across the country are preparing to ensure patients have access to these new products with high quality and safety.…”

Section: Discussionmentioning

confidence: 99%

Manufacturing Parameters for the Creation of Clinical-Grade Human-Induced Pluripotent Stem Cell Lines From Umbilical Cord Mesenchymal Stromal Cells

Beckenkamp,

da Silva,

Von Hohendorff

et al. 2024

Stem Cells Translational Medicine

View full text Add to dashboard Cite

Induced pluripotent stem cells (iPSCs) are reprogrammed cells with a remarkable capacity for unlimited expansion and differentiation into various cell types. Companies worldwide are actively engaged in developing clinical-grade iPSC lines to address the needs of regenerative medicine, immunotherapies, and precision medicine. However, ensuring the safety and quality of iPSCs is essential, with adherence to Good Manufacturing Practices (GMP) and ethical considerations being paramount. Perinatal cell and tissue banks, such as umbilical cord (UC) blood and tissue banks, are emerging as ideal sources for generating iPSCs due to their unique characteristics and GMP compliance. These banks provide access to immature cells with limited environmental exposure, known family and medical histories of donors, and readily available resources, thereby reducing the time and cost associated with personalized treatment strategies. This study describes the establishment of the first clinical-grade iPSC lines from umbilical cord mesenchymal stromal cells in Brazil. The process involved rigorous quality control measures, safety assessments, and adherence to regulatory standards, resulting in iPSCs with the necessary characteristics for clinical use, including sterility, genomic integrity, and stability. Importantly, the study contributes to the development of a Current Good Manufacturing Practice-compliant iPSC production pipeline in Brazil, using commercially available, chemically defined, and xeno-free products, along with validation by national outsourced laboratories, thereby facilitating the adoption of this technology within the country. The study emphasizes Brazil’s contribution to the progress of translational medicine and the promotion of scientific advancements within the field of regenerative and precision medicine.

show abstract

Section: Discussionmentioning

confidence: 99%

Manufacturing Parameters for the Creation of Clinical-Grade Human-Induced Pluripotent Stem Cell Lines From Umbilical Cord Mesenchymal Stromal Cells

Beckenkamp,

da Silva,

Von Hohendorff

et al. 2024

Stem Cells Translational Medicine

View full text Add to dashboard Cite

show abstract

The translational gap for gene therapies in low- and middle-income countries

Doxzen,

Adair,

Fonseca Bazzo

et al. 2024

Sci. Transl. Med.

View full text Add to dashboard Cite

Gene therapies are designed to address the root cause of disease. As scientific understanding of disease prevention, diagnosis, and treatment improves in tandem with technological innovation, gene therapies have the potential to become safe and effective treatment options for a wide range of genetic and nongenetic diseases. However, as the medical scope of gene therapies expands, consideration must be given to those who will benefit and what proactive steps must be taken to widen development and access potential, particularly in regions carrying a high disease burden.

show abstract

Bioengineering breakthroughs: The impact of stem cell models on advanced therapy medicinal product development

Granjeiro,

Borchio,

Ribeiro

et al. 2024

World J Stem Cells

View full text Add to dashboard Cite

The burgeoning field of bioengineering has witnessed significant strides due to the advent of stem cell models, particularly in their application in advanced therapy medicinal products (ATMPs). In this review, we examine the multifaceted impact of these developments, emphasizing the potential of stem cell models to enhance the sophistication of ATMPs and to offer alternatives to animal testing. Stem cell-derived tissues are particularly promising because they can reshape the preclinical landscape by providing more physiologically relevant and ethically sound platforms for drug screening and disease modelling. We also discuss the critical challenges of reproducibility and accuracy in measurements to ensure the integrity and utility of stem cell models in research and application. Moreover, this review highlights the imperative of stem cell models to align with regulatory standards, ensuring using stem cells in ATMPs translates into safe and effective clinical therapies. With regulatory approval serving as a gateway to clinical adoption, the collaborative efforts between scientists and regulators are vital for the progression of stem cell applications from bench to bedside. We advocate for a balanced approach that nurtures innovation within the framework of rigorous validation and regulatory compliance, ensuring that stem cell-base solutions are maximized to promote public trust and patient health in ATMPs.

show abstract

Advanced Therapy Products in Brazil: Regulatory Aspects

Cited by 3 publications

References 11 publications

Manufacturing Parameters for the Creation of Clinical-Grade Human-Induced Pluripotent Stem Cell Lines From Umbilical Cord Mesenchymal Stromal Cells

Manufacturing Parameters for the Creation of Clinical-Grade Human-Induced Pluripotent Stem Cell Lines From Umbilical Cord Mesenchymal Stromal Cells

The translational gap for gene therapies in low- and middle-income countries

Bioengineering breakthroughs: The impact of stem cell models on advanced therapy medicinal product development

Contact Info

Product

Resources

About