The U.S. Food and Drug Administration (FDA) established the Office of Tissues and Advanced Therapies (OTAT) to oversee the development and regulation of advanced therapy medicinal products (ATMPs). (ATMPs are a class of medicinal products that include gene therapies, cell therapies, and tissue-engineered products.) OTAT works with other FDA offices and centers to ensure that ATMPs are developed and regulated in a way that is consistent with the FDA’s mission to protect public health.
The FDA has established several regulatory pathways for ATMPs, including:
Investigational New Drug (IND) pathway: This pathway is for ATMPs that are in the early stages of development and are not yet approved for marketing. Sponsors of INDs must submit an IND application to the FDA, which includes data on the safety and effectiveness of the ATMP.
Biologics License Application (BLA) pathway: This pathway is for ATMPs that have completed clinical testing and are ready for marketing. Sponsors of BLAs must submit a BLA to the FDA, which includes data on the safety, effectiveness, and manufacturing of the ATMP.
Humanitarian Device Exemption (HDE) pathway: This pathway is for ATMPs that are intended to treat or diagnose rare diseases or conditions. Sponsors of HDE applications must submit an HDE application to the FDA, which includes data on the safety and effectiveness of the ATMP.
The FDA has also established guidelines for the development and testing of ATMPs, including guidelines on good manufacturing practices (GMPs), good tissue practices (GTPs), and good clinical practices (GCPs). These guidelines provide recommendations on how to ensure the quality, safety, and effectiveness of ATMPs. In summary, sponsors of ATMPs must follow FDA pathways and guidelines to ensure that their products are developed and regulated in a way that is consistent with the FDA’s mission to protect public health.
Single cell cloning is a useful technique for producing a population of genetically identical cells for use in the production of advanced therapy medicinal products (ATMPs), such as gene therapies, cell therapies, and tissue-engineered products. This technique helps to ensure the purity and consistency of the ATMP, which is important for the safety and effectiveness of the product.
Single cell cloning is typically performed using the following steps:
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REFERENCES
A Risk-based Approach for Cell Line Development, Manufacturing and Characterization of Genetically Engineered, Induced Pluripotent Stem Cell–derived Allogeneic Cell Therapies; Jennifer L. Dashnau, Qiong Xue, Monica Nelson, Eric Law, Lan Cao, Derek Hei. Cytotherapy. Volume 25, Issue 1, January 2023, Pages 1-13. Available online at: https://www.sciencedirect.com/science/article/pii/S1465324922007460.
Transitioning from Preclinical Evidence to Advanced Therapy Medicinal Product: A Spanish Experience; Paloma Gastelurrutia, Cristina Prat-Vidal, Joaquim Vives, Ruth Coll, Antoni Bayes-Genis,Carolina Gálvez-Montón. Frontiers in Cardiovascular Medicine, Volume 8, Article 604434, February 2021. Available online at: www.frontiersin.org.
U.S. Food and Drug Administration: www.fda.gov.
Kap Kumar has over 25 years of experience in the life sciences tools and reagents industry. He started in R&D and product development, where he launched products for cell biology and imaging applications. For the last 15 years, Kap has led strategic marketing, market development, and product management for a variety of companies, including Thermo Fisher Scientific (Life Technologies), Danaher (Beckman Coulter Life Sciences), Cell Signaling Technologies, Nexcelom Biosciences, and Avantor-VWR. Kap has diverse experience managing complex portfolios, including instruments, consumables, and reagents, both in early-stage and mature companies. Kap has a Ph.D. in Cell and Molecular Biology from Kent State University, a post-doctorate from Harvard Medical School, and an MBA from Babson College.
As drug discovery and disease modeling become increasingly sophisticated, 3D cell culture has emerged as a powerful alternative to traditional 2D assays and animal models. In a recent webinar Dr. Allysa Stern showcased how researchers are using the CERO 3D Incubator and Bioreactor to streamline development of organoid models that are scalable, reproducible, and biologically relevant.
Despite decades of optimization in drug development, the failure rate for candidate therapeutics remains high, often due to poor translation from animal models to human biology. That’s why researchers are shifting toward more predictive, human-relevant systems like organoids derived from induced pluripotent stem cells (iPSCs). These models better capture tissue-specific physiology and patient diversity, offering an edge in everything from disease modeling to toxicity screening.
The CERO 3D is a benchtop bioreactor system that enables gentle, dynamic suspension culture of spheroids and organoids. Designed for ease of use, it supports up to four independently controlled tubes, allowing researchers to optimize protocols in parallel. Unlike traditional bioreactors, the CERO 3D requires no impellers or external fluidics. Instead, it uses integrated baffles to create a low-shear, homogenized environment ideal for 3D culture.
Cell culture is the backbone of modern biomedical research, drug development, and regenerative medicine. Traditionally, cells are grown in static environments—like petri dishes or flasks—where stationary media can lead to inconsistent results. Today, researchers are adopting shear flow systems that use dynamic media circulation to mimic the natural forces and nutrient dynamics cells experience in vivo. Here’s why shear flow outperforms static cultures, with a focus on its revolutionary impact on cell culture media:
Physiological Relevance: Media Flow Activates Mechanobiology
Cells like endothelial or bone cells evolved to respond to fluid shear stress. Shear flow systems replicate these forces via media movement, activating critical pathways.
Continuous Fresh Media Supply: Eliminating Feast-or-Famine Cycles
Static cultures rely on manual media changes, creating peaks and crashes in nutrient availability. Shear flow systems ensure steady, automated media replenishment, optimizing cell health and reproducibility.
Constant Waste Removal: Cleaner Media, Healthier Cells
Static media allows metabolic waste (e.g., lactate, ammonia) to accumulate, poisoning cells. Shear flow continuously flushes byproducts, mirroring in vivo clearance mechanisms.
In the ongoing battle against antibiotic-resistant bacteria, or “superbugs,” scientists are constantly seeking innovative tools to understand the mechanisms of resistance and develop effective treatments. One such groundbreaking technology is shear flow systems, which have emerged as game-changers in microbiological research. By simulating the dynamic conditions of bacteria’s natural environments, such as the human body or food processing, shear flow systems provide unique insights into bacterial behavior and resistance mechanisms that traditional static methods simply cannot achieve. In this blog post, we’ll explore the benefits of using shear flow systems to combat antibiotic resistance and develop next-generation therapies.
Antibiotic resistance is a complex phenomenon influenced by a variety of factors, including genetic mutations, horizontal gene transfer, and the formation of biofilms. Traditional laboratory methods often fail to capture the dynamic nature of these processes, leading to incomplete or misleading results. Shear flow systems address this limitation by providing a more realistic environment for bacterial growth and interaction. Here’s how:
1. Mimicking Real-World Conditions
In the human body, bacteria are rarely in a static state. They are constantly exposed to fluid flow, such as blood circulation or urine flow, which influences their behavior and resistance mechanisms. Shear flow systems can replicate these conditions, allowing researchers to study how bacteria respond to antibiotics under realistic physiological conditions. This is critical for understanding how resistance develops and persists in vivo. This benefit was recently highlighted in a webinar presented by Dr. Katharina Richter, a microbiology researcher from the University of Adelaide in Australia. Using a high-throughput BioFlux Shear Flow System, Dr. Richter and her team were able to test 3 different methods of superbug treatments under physiological conditions.
2. Studying Biofilm Formation and Resistance
Biofilms—structured communities of bacteria encased in a protective matrix—are a major contributor to antibiotic resistance. Biofilms are notoriously difficult to treat because they shield bacteria from antibiotics and the immune system. Shear flow systems enable researchers to study biofilm formation in real time, observing how bacteria adhere to surfaces, form microcolonies, and develop resistance under flow conditions. For example, using a BioFlux, Dr. Richter was able to leverage high-resolution imaging to obtain unprecedented insight into the biofilm killing and prevention efficacy of a copper and diethyldithiocarbamate (DDC) combination nanoparticles¹. The group is currently investigating the use of a Cu-DDC infused gel that can be injected into wounds that are at high risk for chronic infection, such as hernia.
