Category Archives: Induced Pluripotent Stem Cells

Insights on the Cell Expansion Industry in North America to 2027 – by Product, Cell Type, Application, End-user and Country – Yahoo Finance

Dublin, April 24, 2020 (GLOBE NEWSWIRE) -- The "North America Cell Expansion Market to 2027 - Regional Analysis and Forecasts by Product; Cell Type; Application; End User, and Country" report has been added to ResearchAndMarkets.com's offering.

The cell expansion market in North America is anticipated to reach USD 14,697.41 million by 2027 from USD 4,522.07 4 million in 2019; it is projected to grow at a CAGR of 15.9% during 2020-2027. The growth of the market is attributed to the increasing prevalence of cancer, rising number of new product launches, and increasing inclination of patients toward regenerative and personalized medicines. Also, growing R&D expenditure on cancer research is likely to have a positive impact on the growth of the market in the coming years. In addition, technological advancements in the pharmaceuticals industry and extensive developments in drug discovery are likely to stimulate the growth of cell expansion market in North America during the forecast period.

Cell expansion is the large-scale artificial production of daughter cells from a single cell, and the process is carried out to support the medical research. It plays a critical role in exploring a wider range of benefits and applications of fully differentiated stem cell cultures for their use in therapeutics, drug screening, or advanced research.

R&D is a significant part of a majority of pharmaceutical and biotech companies; they focus on R&D to come up with new molecules with the most significant medical and commercial potential for various therapeutic applications. The companies invest big amounts in these activities to deliver innovative, high-quality products to the market. Moreover, as per the report of Pharmaceutical Research and Manufacturers of America (PhRMA), the R&D expense of biopharmaceutical companies surged from US$ 49.6 billion in 2012 to US$ 58.8 billion in 2015.

Several government organizations are working on enhancing the detection methods and treatment procedures of cancer in the region. The National Cancer Institute (NCI) spends on various categories of the treatment, including specific cancer sites, cancer types, and cancer-related diseases, as well as types of NCI research mechanisms. The NCI allocated the funds of ~US$ 208.4 million for cell expansion research in 2017 from their total budget of US$ 5,636.4 million in that year for cancer research studies. Therefore, the growing R&D expenditure on cancer research by these companies is expected to provide them with opportunities for business expansion.

The North American cell expansion market has been segmented on the basis of cell type into human cells and animal cells. The human cells segment held a larger share of the market in 2018, and it is also projected to register a higher CAGR in it during the forecast period. Rise in research activities for the treatment of cancer is expected to offer considerable growth opportunities for the human cell expansion market players.

A few of the important secondary sources referred to for preparing this report on the cell expansion market are World Health Organization (WHO), Food and Drug Administration (FDA), Canadian Cancer Society, Centers for Disease Control and Prevention (CDC), and American Cancer Society.

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Key Topics Covered:

1. Introduction1.1 Scope of the Study1.2 Report Guidance1.3 Market Segmentation1.3.1 North America Cell Expansion Market - By Product1.3.2 North America Cell Expansion Market - By Cell Type1.3.3 North America Cell Expansion Market - By Application1.3.4 North America Cell Expansion Market - By End User1.3.5 North America Cell Expansion Market - By Country

2. North America Cell Expansion Market- Key Takeaways

3. Research Methodology3.1 Coverage3.2 Secondary Research3.3 Primary Research

4. North America Cell Expansion Market - Market Landscape4.1 Overview4.2 PEST Analysis4.2.1 Cell Expansion Market - North America PEST Analysis4.3 Expert Opinion

5. North America Cell Expansion Market - Key Market Dynamics5.1 Key Market Drivers5.1.1 Patient shift towards regenerative medicines5.1.2 Increasing number of patients suffering with cancer5.2 Key Restraints5.2.1 Risk of contamination associated with the cell expansion process5.3 Key Market Opportunities5.3.1 Growing R&D Expenditure for Cancer Research5.4 Future Trend5.4.1 Extensive development in drug discovery5.5 Impact Analysis

6. Cell Expansion Market - North America Analysis6.1 North America Cell Expansion Market Revenue Forecasts and Analysis6.2 Positioning Of Key Players

7. North America Cell Expansion Market Analysis And Forecasts To 2027 - Product7.1 Overview7.2 North America Cell Expansion Market, By Product 2018 & 2027 (%)7.2.1 North America Cell Expansion Market Revenue and Forecasts to 2027, By Product (US$ Mn)7.2.1.1 North America Consumables Market Revenue and Forecasts to 2027, By Type (US$ Mn)7.2.1.1.1 North America Disposables Market Revenue and Forecasts to 2027, By Type (US$ Mn)7.2.1.2 North America Instruments Market Revenue and Forecasts to 2027, By Type (US$ Mn)7.3 Consumables7.3.1 Overview7.3.2 North America Consumables Market Revenue and Forecast to 2027 (US$ Mn)7.3.3 Reagents, Media & Serum7.3.3.1 Overview7.3.3.2 North America Reagents, Media & Serum Market Revenue and Forecast to 2027 (US$ Mn)7.3.4 Disposables7.3.4.1 Overview7.3.4.2 North America Disposables Market Revenue and Forecast to 2027 (US$ Mn)7.3.4.3 Culture Tissue Flasks7.3.4.3.1 Overview7.3.4.3.2 North America Culture Tissue Flasks Market Revenue and Forecast to 2027 (US$ Mn)7.3.4.4 Bioreactor Accessories7.3.4.4.1 Overview7.3.4.4.2 North America Bioreactor Accessories Market Revenue and Forecast to 2027 (US$ Mn)7.3.4.5 Other Disposables7.3.4.5.1 Overview7.3.4.5.2 North America Other Disposables Market Revenue and Forecast to 2027 (US$ Mn)7.4 Instruments7.4.1 Overview7.4.2 North America Instruments Market Revenue and Forecasts to 2027 (US$ Mn)7.4.3 Cell Expansion Supporting Equipment7.4.3.1 Overview7.4.3.2 North America Cell Expansion Supporting Equipment Market Revenue and Forecast to 2027 (US$ Mn)7.4.4 Bioreactors7.4.4.1 Overview7.4.4.2 North America Bioreactors Market Revenue and Forecast to 2027 (US$ Mn)7.4.5 Automated Cell Expansion Systems7.4.5.1 North America Automated Cell Expansion Systems Market Revenue and Forecast to 2027 (US$ Mn)

8. North America Cell Expansion Market Analysis And Forecasts To 2027 - Cell Type8.1 Overview8.2 North America Cell Expansion Market, By Cell Type 2018 & 2027 (%)8.2.1 North America Cell Expansion Market Revenue and Forecasts to 2027, By Cell Type (US$ Mn)8.3 Human Cells8.3.1 Overview8.3.2 North America Human Cells Market Revenue and Forecast to 2027 (US$ Mn)8.3.3 Adult Stem Cells8.3.3.1 Overview8.3.3.2 North America Adult Stem Cells Market Revenue and Forecast to 2027 (US$ Mn)8.3.4 Induced Pluripotent Stem Cells8.3.4.1 Overview8.3.4.2 North America Induced Pluripotent Stem Cells Market Revenue and Forecast to 2027 (US$ Mn)8.3.5 Embryonic Stem Cells8.3.5.1 Overview8.3.5.2 North America Embryonic Stem Cells Market Revenue and Forecast to 2027 (US$ Mn)8.3.6 Differentiated Cells8.3.6.1 Overview8.3.6.2 North America Differentiated Cells Market Revenue and Forecast to 2027 (US$ Mn)8.4 Animal Cells8.4.1 Overview8.4.2 North America Animal Cells Market Revenue and Forecast to 2027 (US$ Mn)

9. North America Cell Expansion Market Analysis- By Application9.1 Overview9.2 North America Cell Expansion Market, By Application 2018 & 2027 (%)9.3 Regenerative Medicine and Stem Cell Research9.4 Cancer and Cell-based Research9.5 Other Applications

10. North America Cell Expansion Market Analysis- By End User10.1 Overview10.2 North America Cell Expansion Market, By End User 2018 & 2027 (%)10.3 Biopharmaceutical And Biotechnology Companies10.4 Research Institutes10.5 Cell Banks10.6 Other End Users

11. Cell Expansion Market Revenue And Forecasts To 2027 - Geographical Analysis11.1 North America Cell Expansion Market, Revenue and Forecast to 202711.1.1 North America Cell Expansion Market, Revenue and Forecast to 2027 (US$ Mn)11.1.2 North America Cell Expansion Market, Revenue and Forecast to 2027, By Product (US$ Mn)11.1.2.1 North America Consumables Market, Revenue and Forecast to 2027, By Type (US$ Mn)11.1.2.1.1 North America Disposables Market, Revenue and Forecast to 2027, By Type (US$ Mn)11.1.2.2 North America Instruments Market, Revenue and Forecast to 2027, By Type (US$ Mn)11.1.3 North America Cell Expansion Market, Revenue and Forecast to 2027, By Cell Type (US$ Mn)11.1.3.1 North America Human Cell Market, Revenue and Forecast to 2027, By Type (US$ Mn)11.1.4 North America Cell Expansion Market, Revenue and Forecast to 2027, By Application (US$ Mn)11.1.5 North America Cell Expansion Market, Revenue and Forecast to 2027, By End User (US$ Mn)11.1.6 North America Cell Expansion Market, Revenue and Forecast to 2027, By Country (%)11.1.7 US11.1.8 Canada11.1.9 Mexico

12. North America Cell Expansion Market- Industry Landscape12.1 Overview12.2 Growth Strategies In The Cell Expansion Market, 2017-201912.3 Organic Growth Strategies12.3.1 Overview12.3.1.1 Recent Organic Developments By Players In The Cell Expansion Market12.4 Inorganic Growth Strategies12.4.1 Overview12.4.2 Recent Developments By Players In The Cell Expansion Market

13. Global Cell Expansion Market-Key Company Profiles13.1 BD13.1.1 Key Facts13.1.2 Business Description13.1.3 Financial Overview13.1.4 Product Portfolio13.1.5 SWOT Analysis13.1.6 Key Developments13.2 Merck KGaA13.3 Thermo Fisher Scientific, Inc.13.4 Terumo Corporation13.5 General Electric Company13.6 Corning Incorporated13.7 Miltenyi Biotec13.8 Danaher13.9 Lonza13.10 STEMCELL Technologies, Inc.

14. Appendix14.1 About the Publisher14.2 Glossary of Terms

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Insights on the Cell Expansion Industry in North America to 2027 - by Product, Cell Type, Application, End-user and Country - Yahoo Finance

Induced pluripotent stem cells and CRISPR reversed diabetes in mice – Drug Target Review

Induced pluripotent stem cells made to produce insulin and CRISPR, used to correct a genetic defect, cured Wolfram syndrome in mice.

Using induced pluripotent stem cells (iPSCs) produced from the skin of a patient with a rare, genetic form of insulin-dependent diabetes called Wolfram syndrome, researchers transformed the human stem cells into insulin-producing cells and used CRISPR-Cas9 to correct a genetic defect that had caused the syndrome. They then implanted the cells into lab mice and cured the unrelenting diabetes in those models.

The findings, from researchers at Washington University School of Medicine in St. Louis, US, suggest this CRISPR-Cas9 technique may hold promise as a treatment for diabetes, particularly the forms caused by a single gene mutation and it also may be useful one day in some patients with the more common forms of diabetes, such as type 1 and type 2.

This is the first time CRISPR has been used to fix a patients diabetes-causing genetic defect and successfully reverse diabetes, said co-senior investigator Dr Jeffrey Millman, an assistant professor of medicine and of biomedical engineering at Washington University. For this study, we used cells from a patient with Wolfram syndrome because, conceptually, we knew it would be easier to correct a defect caused by a single gene. But we see this as a stepping stone toward applying gene therapy to a broader population of patients with diabetes.

Wolfram syndrome is caused by mutations to a single gene, providing the researchers an opportunity to determine whether combining stem cell technology with CRISPR to correct the genetic error also might correct the diabetes caused by the mutation.

Researchers at Washington University School of Medicine in St. Louis have transformed stem cells into insulin-producing cells. They used the CRISPR gene-editing tool to correct a defect that caused a form of diabetes, and implanted the cells into mice to reverse diabetes in the animals. Shown is a microscopic image of insulin-secreting beta cells (insulin is green) that were made from stem cells produced from the skin of a patient with Wolfram syndrome [credit: Millman lab Washington University].

Millman and his colleagues had previously discovered how to convert human stem cells into pancreatic beta cells. When such cells encounter blood sugar, they secrete insulin. Recently, these researchers developed a new technique to more efficiently convert human stem cells into beta cells that are considerably better at controlling blood sugar.

In this study, they took the additional steps of deriving these cells from patients and using the CRISPR-Cas9 gene-editing tool on those cells to correct a mutation to the gene that causes Wolfram syndrome (WFS1). Then, the researchers compared the gene-edited cells to insulin-secreting beta cells from the same batch of stem cells that had not undergone editing with CRISPR.

In the test tube and in mice with a severe form of diabetes, the newly grown beta cells that were edited with CRISPR more efficiently secreted insulin in response to glucose. Diabetes disappeared in mice with the CRISPR-edited cells implanted beneath the skin and the animals blood sugar levels remained in normal range for the entire six months they were monitored. Animals receiving unedited beta cells remained diabetic. Although their newly implanted beta cells could produce insulin, it was not enough to reverse their diabetes.

We basically were able to use these cells to cure the problem, making normal beta cells by correcting this mutation, said co-senior investigator Dr Fumihiko Urano, the Samuel E. Schechter Professor of Medicine and a professor of pathology and immunology. Its a proof of concept demonstrating that correcting gene defects that cause or contribute to diabetes in this case, in the Wolfram syndrome gene we can make beta cells that more effectively control blood sugar. Its also possible that by correcting the genetic defects in these cells, we may correct other problems Wolfram syndrome patients experience, such as visual impairment and neurodegeneration.

Were excited about the fact that we were able to combine these two technologies growing beta cells from induced pluripotent stem cells and using CRISPR to correct genetic defects, Millman said. In fact, we found that corrected beta cells were indistinguishable from beta cells made from the stem cells of healthy people without diabetes.

Moving forward, the process of making beta cells from stem cells should get easier, the researchers said. For example, the scientists have developed less intrusive methods, making iPSCs from blood and they are working on developing stem cells from urine samples.

The study is published in Science Translational Medicine.

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Induced pluripotent stem cells and CRISPR reversed diabetes in mice - Drug Target Review

Reversing diabetes with CRISPR and patient-derived stem cells – FierceBiotech

Insulin injections cancontrol diabetes, but patients still experience serious complications such as kidney disease and skin infections. Transplanting pancreatic tissues containing functional insulin-producing beta cells is of limited use, because donors are scarce and patients must take immunosuppressant drugs afterward.

Now, scientists atWashington University in St. Louis havedeveloped a way to use gene editing system CRISPR-Cas9 to edit a mutation in human-induced pluripotent stem cells (iPSCs) and then turnthem into beta cells. When transplanted into mice, the cells reversed preexisting diabetes in a lasting way, according to results published in the journal Science Translational Medicine.

While the researchers used cells from patients with Wolfram syndromea rare childhood diabetes caused by mutations in the WFS1 genethey argue that the combination of a gene therapy with stem cells could potentially treat other forms of diabetes as well.

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This virtual event will bring together industry experts to discuss the increasing pace of pharmaceutical innovation, the need to maintain data quality and integrity as new technologies are implemented and understand regulatory challenges to ensure compliance.

One of the biggest challenges we faced was differentiating our patient cells into beta cells. Previous approaches do not allow for this robust differentiation. We use our new differentiation protocol targeting different development and signaling pathways to generate our cells, the studys lead author, Kristina Maxwell, explained in a video statement.

Making pancreatic beta cells from patient-derived stem cells requires precise activation and repression of specific pathways, and atthe right times, to guide the development process. In a recent Nature Biotechnology study, the team described a successful method that leverages the link between a complex known as actin cytoskeleton and the expression of transcription factors that drive pancreatic cell differentiation.

This time, the researchers applied the technology to iPSCs from two patients with Wolfram syndrome. They used CRISPR to correct the mutated WFS1 gene in the cells and differentiated the edited iPSCs into fully functional beta cells.

After transplanting the corrected beta cells into diabetic mice, the animals saw their blood glucose drop quickly, suggesting the disease had been reversed. The effect lasted for the entire six-month observation period, the scientists reported. By comparison, those receiving unedited cells from patients were unable to achieve glycemic control.

RELATED:CRISPR Therapeutics, ViaCyte team up on gene-edited diabetes treatment

The idea of editing stem cells with CRISPR has already attracted interest in the biopharma industry. Back in 2018, CRISPR Therapeutics penned a deal with ViaCyte to develop off-the-shelf, gene-editing stem cell therapies for diabetes. Rather than editing iPSCs from particular patients themselves to correct a faulty gene, the pairs lead project used CRISPR to edit healthy cells so that they lackedthe B2M gene and expressed PD-L1 to protect against immune attack. The two companies unveiled positive preclinical data inSeptember.

Other research groups working on gene therapy or stem cells for diabetes include a Harvard University scientist and his startup Semma Therapeutics, whichdeveloped a method for selecting beta cells out of a mixture of cells developed from PSCs. Scientists at the University of Wisconsin-Madison recently proposed that removing the IRE1-alpha gene in beta cells could prevent immune T cells from attacking them in mice with Type 1 diabetes.

The Washington University team hopes its technology may help Type 1 diabetes patients whose disease is caused by multiple genetic and environmental factors as well as the Type 2 form linked to obesity and insulin resistance.

We can generate a virtually unlimited number of beta cells from patients with diabetes to test and discover new drugs to hopefully stop or even reverse this disease, Jeffrey Millman, the studys co-senior author, said in the video statement. Perhaps most importantly, this technology now allows for the potential use of gene therapy in combination with the patients own cells to treat their own diabetes by transplantation of lab-grown beta cells.

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Reversing diabetes with CRISPR and patient-derived stem cells - FierceBiotech

The integrated stress response: From mechanism to disease – Science Magazine

Proteostasis dISRupted

Despite their importance, many crucial networks for protein quality control within cells diminish with age. The resulting loss of proteostasis, the process by which the health of a cell's proteins is monitored and maintained, is associated with a wide range of age-related human diseases. Costa-Mattioli and Walter review the integrated stress response (ISR), a central signaling network that responds to proteostasis defects by tuning protein synthesis. The ISR is activated in a wide range of pathological conditions, so a mechanistic understanding of its pathway may help in the development of therapeutic tools through which it can be modulated.

Science, this issue p. eaat5314

The integrated stress response (ISR) is an evolutionarily conserved intracellular signaling network that helps the cell, tissue, and organism to adapt to a variable environment and maintain health. In response to different environmental and pathological conditions, including protein homeostasis (proteostasis) defects, nutrient deprivation, viral infection, and oxidative stress, the ISR restores balance by reprogramming gene expression. The various stresses are sensed by four specialized kinases (PERK, GCN2, PKR and HRI) that converge on phosphorylation of a single serine on the eukaryotic translation initiation factor eIF2. eIF2 phosphorylation blocks the action of eIF2s guanine nucleotide exchange factor termed eIF2B, resulting in a general reduction in protein synthesis. Paradoxically, phosphorylation of eIF2 also triggers the translation of specific mRNAs, including key transcription factors, such as ATF4. These mRNAs contain short inhibitory upstream open reading frames in their 5-untranslated regions that prevent translation initiation at their canonical AUGs. By tuning down general mRNA translation and up-regulating the synthesis of a few proteins that drive a new transcriptional program, the ISR aims to maintain or reestablish physiological homeostasis. However, if the stress cannot be mitigated, the ISR triggers apoptosis to eliminate the damaged cell.

Our understanding of the central mechanisms that govern the ISR has advanced vastly. The ISRs central regulatory hub lies in the eIF2eIF2B complex, which controls the formation of the eIF2GTPmethionyl-intiator tRNA ternary complex (TC), a prerequisite for initiating new protein synthesis. Assembly of functional TC is inhibited by eIF2-P, which blocks eIF2B noncompetitively. In mammalian cells, the phosphorylation of eIF2 is a tightly regulated process. In addition to the four specialized eIF2 kinases that phosphorylate eIF2, two dedicated phosphatases antagonize this reaction. Both phosphatases contain a common catalytic core subunit, the protein phosphatase 1 (PP1), and a regulatory subunit (GADD34 or CReP), which render the phosphatase specific to eIF2. Structural and biophysical approaches have elucidated the mechanism of action of eIF2B and its modulation by ISR inhibitors and activators. Gene expression analyses have revealed complex ISR-driven reprogramming. Although it has been long recognized that, in the brain, long-term memory formation requires new protein synthesis, recent causal and convergent evidence across different species and model systems has shown that the ISR serves as a universal regulator of this process. Briefly, inhibition of the ISR enhances long-term memory formation, whereas activation of the ISR prevents it. Consistent with this notion, unbiased genome-wide association studies have identified mutations in key components of the ISR in humans with intellectual disability. Furthermore, age-related cognitive disorders are commonly associated with the activation of the ISR. Most notably, oxidative stress, misfolded proteins, and other stressors induce the ISR in several neurodegenerative disorders, including Alzheimers disease. Recent genetic and pharmacological evidence suggest that tuning the ISR reverses cognitive dysfunction as well as neurodegeneration in a wide range of memory disorders that result from protein homeostasis defects. Thus, long-term memory deficits may primarily result as a consequence of ISR activation rather than from the particular proteostasis defects that lead to its induction. Finally, the ISR is also implicated in the pathogenesis of a plethora of other complex diseases, including cancer, diabetes, and metabolic disorders.

The ISR is emerging as a central regulator of protein homeostasis at both the cellular and organismal level. Mechanistically, much remains to be understood regarding additional inputs into the eIF2BeIF2 regulatory hub controlling TC concentration, as well as the IRSs connectivity to other intracellular signaling networks. As yet, little is known about the role of the specific proteins whose synthesis is altered during acute and persistent ISR activation and how these effectors collaborate to compute the life or death decisions cells make upon ISR activation. ISR gene expression signatures and functional consequences will need to be mapped across different tissues, cell types, and developmental stages. In addition, it will be invaluable to generate additional genetic and molecular tools that permit the direct temporal and spatial manipulation of ISR pathway in specific cells and circuits to determine their function. From a medical perspective, the ISR is implicated in the etiology of several disorders, and manipulation of the ISR is emerging as a promising therapeutic avenue for the treatment of a variety of diseases. The use of innovative mouse models, patient-derived induced pluripotent stem cells, and human organoids will greatly enhance our ability to explore the ISRs clinical relevance further and help define therapeutic windows in which ISR modulation may prove beneficial. Identifying additional specific small-molecule inhibitors and activators of the ISR will offer valuable opportunities to dissect the role of the ISR pharmacologically in health and disease. Finally, discovery and mechanistic understanding of additional ISR modulators will increase the repertoire of therapeutic targets and may further enable clinical development in a wide range of age-related human diseases.

Diverse deviations from homeostasis activate the ISR. The resulting dysregulation of translation contributes to numerous diseases.

Protein quality control is essential for the proper function of cells and the organisms that they make up. The resulting loss of proteostasis, the processes by which the health of the cells proteins is monitored and maintained at homeostasis, is associated with a wide range of age-related human diseases. Here, we highlight how the integrated stress response (ISR), a central signaling network that responds to proteostasis defects by tuning protein synthesis rates, impedes the formation of long-term memory. In addition, we address how dysregulated ISR signaling contributes to the pathogenesis of complex diseases, including cognitive disorders, neurodegeneration, cancer, diabetes, and metabolic disorders. The development of tools through which the ISR can be modulated promises to uncover new avenues to diminish pathologies resulting from it for clinical benefit.

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The integrated stress response: From mechanism to disease - Science Magazine

Diabetes reversed in mice with genetically edited stem cells derived from patients – Washington University School of Medicine in St. Louis

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CRISPR corrects genetic defect so cells can normalize blood sugar

Researchers at Washington University School of Medicine in St. Louis have transformed stem cells into insulin-producing cells. They used the CRISPR gene-editing tool to correct a defect that caused a form of diabetes, and implanted the cells into mice to reverse diabetes in the animals. Shown is a microscopic image of insulin-secreting beta cells (insulin is green) that were made from stem cells produced from the skin of a patient with Wolfram syndrome.

Using induced pluripotent stem cells produced from the skin of a patient with a rare, genetic form of insulin-dependent diabetes called Wolfram syndrome, researchers transformed the human stem cells into insulin-producing cells and used the gene-editing tool CRISPR-Cas9 to correct a genetic defect that had caused the syndrome. They then implanted the cells into lab mice and cured the unrelenting diabetes in those mice.

The findings, from researchers at Washington University School of Medicine in St. Louis, suggest the CRISPR-Cas9 technique may hold promise as a treatment for diabetes, particularly the forms caused by a single gene mutation, and it also may be useful one day in some patients with the more common forms of diabetes, such as type 1 and type 2.

The study is published online April 22 in the journal Science Translational Medicine.

Patients with Wolfram syndrome develop diabetes during childhood or adolescence and quickly require insulin-replacement therapy, requiring insulin injections multiple times each day. Most go on to develop problems with vision and balance, as well as other issues, and in many patients, the syndrome contributes to an early death.

This is the first time CRISPR has been used to fix a patients diabetes-causing genetic defect and successfully reverse diabetes, said co-senior investigator Jeffrey R. Millman, PhD, an assistant professor of medicine and of biomedical engineering at Washington University. For this study, we used cells from a patient with Wolfram syndrome because, conceptually, we knew it would be easier to correct a defect caused by a single gene. But we see this as a stepping stone toward applying gene therapy to a broader population of patients with diabetes.

Wolfram syndrome is caused by mutations to a single gene, providing the researchers an opportunity to determine whether combining stem cell technology with CRISPR to correct the genetic error also might correct the diabetes caused by the mutation.

A few years ago, Millman and his colleagues discovered how to convert human stem cells into pancreatic beta cells. When such cells encounter blood sugar, they secrete insulin. Recently, those same researchers developed a new technique to more efficiently convert human stem cells into beta cells that are considerably better at controlling blood sugar.

In this study, they took the additional steps of deriving these cells from patients and using the CRISPR-Cas9 gene-editing tool on those cells to correct a mutation to the gene that causes Wolfram syndrome (WFS1). Then, the researchers compared the gene-edited cells to insulin-secreting beta cells from the same batch of stem cells that had not undergone editing with CRISPR.

In the test tube and in mice with a severe form of diabetes, the newly grown beta cells that were edited with CRISPR more efficiently secreted insulin in response to glucose. Diabetes disappeared quickly in mice with the CRISPR-edited cells implanted beneath the skin, and the animals blood sugar levels remained in normal range for the entire six months they were monitored. Animals receiving unedited beta cells remained diabetic. Their newly implanted beta cells could produce insulin, just not enough to reverse their diabetes.

We basically were able to use these cells to cure the problem, making normal beta cells by correcting this mutation, said co-senior investigator Fumihiko Urano, MD, PhD, the Samuel E. Schechter Professor of Medicine and a professor of pathology and immunology. Its a proof of concept demonstrating that correcting gene defects that cause or contribute to diabetes in this case, in the Wolfram syndrome gene we can make beta cells that more effectively control blood sugar. Its also possible that by correcting the genetic defects in these cells, we may correct other problems Wolfram syndrome patients experience, such as visual impairment and neurodegeneration.

In the future, using CRISPR to correct certain mutations in beta cells may help patients whose diabetes is the result of multiple genetic and environmental factors, such as type 1, caused by an autoimmune process that destroys beta cells, and type 2, which is closely linked to obesity and a systemic process called insulin resistance.

Were excited about the fact that we were able to combine these two technologies growing beta cells from induced pluripotent stem cells and using CRISPR to correct genetic defects, Millman said. In fact, we found that corrected beta cells were indistinguishable from beta cells made from the stem cells of healthy people without diabetes.

Moving forward, the process of making beta cells from stem cells should get easier, the researchers said. For example, the scientists have developed less intrusive methods, making induced pluripotent stem cells from blood and they are working on developing stem cells from urine samples.

In the future, Urano said, we may be able to take a few milliliters of urine from a patient, make stem cells that we then can grow into beta cells, correct mutations in those cells with CRISPR, transplant them back into the patient, and cure their diabetes in our clinic. Genetic testing in patients with diabetes will guide us to identify genes that should be corrected, which will lead to a personalized regenerative gene therapy.

Maxwell KG, Augsornworawat P, Velazco-Cruz L, Kim MH, Asada R, Hogrebe NJ, Morikawa S, Urano F, Millman JR. Gene-edited human stem cell-derived cells from a patient with monogenic diabetes reverse pre-existing diabetes in mice. Science Translational Medicine, published online April 22, 2020.

This work was supported by the National Institute of Diabetes and Digestive and Kidney Diseases, the National Institute of General Medical Sciences, the National Cancer Institute and the National Center for Advancing Translational Sciences of the National Institutes of Health (NIH). Grant numbers R01 DK114233, DK112921, TR002065, TR002345, T32 DK108742, R25 GM103757, T32 DK007120, P30 DK020579, P30 CA91842, UL1 TR000448 and UL1 TR002345. Additional assistance was provided by the Washington University Genome Engineering and iPSC Center, the Washington University Diabetes Center, and the Washington University Institute of Clnical and Translational Science, with additional funding from the JDRF, the Washington University Center of Regenerative Medicine, startup funds from the Washington University School of Medicine Department of Medicine, the Unravel Wolfram Syndrome Fund, Silberman Fund, Stowe Fund, Ellie White Foundation for Rare Genetic Disorders, Eye Hope Foundation, Snow Foundation, Feiock Fund, Childrens Discovery Institute, Manpei Suzuki Diabetes Foundation, and a JSPS Overseas Research Fellowship.

Washington University School of Medicines 1,500 faculty physicians also are the medical staff of Barnes-Jewish and St. Louis Childrens hospitals. The School of Medicine is a leader in medical research, teaching and patient care, ranking among the top 10 medical schools in the nation by U.S. News & World Report. Through its affiliations with Barnes-Jewish and St. Louis Childrens hospitals, the School of Medicine is linked to BJC HealthCare.

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Diabetes reversed in mice with genetically edited stem cells derived from patients - Washington University School of Medicine in St. Louis

Business News: Induced Pluripotent Stem Cells Market Growth, Analysis and Forecast 2020 to 2025 | BlueRock Therapeutics, Corning Life Sciences, EMD…

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The chapter on company profiles studies the various companies operating in the global Induced Pluripotent Stem Cells market. It evaluates the financial outlooks of these companies, their research and development statuses, and their expansion strategies for the coming years. Analysts have also provided a detailed list of the strategic initiatives taken by the Induced Pluripotent Stem Cells market participants in the past few years to remain ahead of the competition.

Global Induced Pluripotent Stem Cells Market: Regional Segments

The chapter on regional segmentation details the regional aspects of the global Induced Pluripotent Stem Cells market. This chapter explains the regulatory framework that is likely to impact the overall market. It highlights the political scenario in the market and the anticipates its influence on the global Induced Pluripotent Stem Cells market.

The Middle East and Africa(GCC Countries and Egypt)North America(the United States, Mexico, and Canada)South America(Brazil etc.)Europe(Turkey, Germany, Russia UK, Italy, France, etc.)Asia-Pacific(Vietnam, China, Malaysia, Japan, Philippines, Korea, Thailand, India, Indonesia, and Australia)

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Report Highlights

Comprehensive pricing analysis on the basis of product, application, and regional segments

The detailed assessment of the vendor landscape and leading companies to help understand the level of competition in the global Induced Pluripotent Stem Cells market

Deep insights about regulatory and investment scenarios of the global Induced Pluripotent Stem Cells market

Analysis of market effect factors and their impact on the forecast and outlook of the global Induced Pluripotent Stem Cells market

A roadmap of growth opportunities available in the global Induced Pluripotent Stem Cells market with the identification of key factors

The exhaustive analysis of various trends of the global Induced Pluripotent Stem Cells market to help identify market developments

Table of Contents

Report Overview:It includes six chapters, viz. research scope, major manufacturers covered, market segments by type, Induced Pluripotent Stem Cells market segments by application, study objectives, and years considered.

Global Growth Trends:There are three chapters included in this section, i.e. industry trends, the growth rate of key producers, and production analysis.

Induced Pluripotent Stem Cells Market Share by Manufacturer:Here, production, revenue, and price analysis by the manufacturer are included along with other chapters such as expansion plans and merger and acquisition, products offered by key manufacturers, and areas served and headquarters distribution.

Market Size by Type:It includes analysis of price, production value market share, and production market share by type.

Market Size by Application:This section includes Induced Pluripotent Stem Cells market consumption analysis by application.

Profiles of Manufacturers:Here, leading players of the global Induced Pluripotent Stem Cells market are studied based on sales area, key products, gross margin, revenue, price, and production.

Induced Pluripotent Stem Cells Market Value Chain and Sales Channel Analysis:It includes customer, distributor, Induced Pluripotent Stem Cells market value chain, and sales channel analysis.

Market Forecast Production Side: In this part of the report, the authors have focused on production and production value forecast, key producers forecast, and production and production value forecast by type.

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About Us:Report Hive Research delivers strategic market research reports, statistical survey, and Industry analysis and forecast data on products and services, markets and companies. Our clientele ranges mix of United States Business Leaders, Government Organizations, SMEs, Individual and Start-ups, Management Consulting Firms, and Universities etc. Our library of 600,000+ market reports covers industries like Chemical, Healthcare, IT, Telecom, Semiconductor, etc. in the USA, Europe Middle East, Africa, Asia Pacific. We help in business decision-making on aspects such as market entry strategies, market sizing, market share analysis, sales and revenue, technology trends, competitive analysis, product portfolio and application analysis etc.

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Business News: Induced Pluripotent Stem Cells Market Growth, Analysis and Forecast 2020 to 2025 | BlueRock Therapeutics, Corning Life Sciences, EMD...

Stem Cell Therapy Market: Industry Size, Market Status, Influencing Factors, Competition, Outlook & Forecasts to 2027 – Cole of Duty

According to The Insight Partners market research study of Stem Cell Therapy Market to 2027 Global Analysis and Forecasts by Type, Treatment, Application, and End User. The global stem cell therapy market is expected to reach US$ 5,129.66 Mn in 2027 from US$ 1,534.55 Mn in 2019. The market is estimated to grow with a CAGR of 16.7% from 2020-2027. The report provides trends prevailing in the global stem cell therapy market and the factors driving market along with those that act as hindrances.

The global stem cell therapy market, based on the type, is segmented into adult stem cell, induced pluripotent stem cells, embryonic stem cell, and other stem cells. Adult stem cell therapy is further segmented into hematopoietic stem cells, mesenchymal stem cells, neuronal stem cells, and umbilical cord stem cells. The adult stem cell segment held the largest share of the market in 2019. The same segment is estimated to register the highest CAGR in the market during the forecast period due to its effectiveness for the treatment of chronic conditions coupled with higher compatibility with immunity system. The end user segment is segmented into academic and research institutes and hospitals & specialty clinics.

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Major Key Players:

The report studies established names and emerging startups in the industry, to give the flavor of the entire business canvas. Different case studies from industry experts and policymakers have been mentioned for a clear understanding of the Global Stem Cell Therapy Market. It also offers comprehensive information on the product or service portfolio. All these factors which are studied in this research report are predicted to propel the Global Stem Cell Therapy Market.

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Finally, all aspects of the Global Stem Cell Therapy Market are quantitatively as well qualitatively assessed to study the Global as well as regional market comparatively. This market study presents critical information and factual data about the market providing an overall statistical study of this market on the basis of market drivers, limitations and its future prospects. The report supplies the international economic competition with the assistance of Porters Five Forces Analysis and SWOT Analysis.

Following are the List of Chapter Covers in the Global Stem Cell Therapy Market:

About Us:The Insight Partners is a one stop industry research provider of actionable intelligence. We help our clients in getting solutions to their research requirements through our syndicated and consulting research services. We are a specialist in Technology, Semiconductors, Healthcare, Manufacturing, Automotive and Defense.

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Stem Cell Therapy Market: Industry Size, Market Status, Influencing Factors, Competition, Outlook & Forecasts to 2027 - Cole of Duty

Induced Pluripotent Stem Cells Market Analysis With Key Players, Applications, Trends And Forecasts To 2026 – Surfacing Magazine

DataIntelo report titled Global Induced Pluripotent Stem Cells Market provides detailed information and overview about the key influential factors required to make well informed business decision. This is a latest report, covering the current COVID-19 impact on the market. The pandemic of Coronavirus (COVID-19) has affected every aspect of life globally. This has brought along several changes in market conditions. The rapidly changing market scenario and initial and future assessment of the impact is covered in the report. Our data has been culled out by our team of experts who have curated the report, considering market-relevant information. This report provides latest insights about the markets drivers, restraints, opportunities, and trends. It also discusses the growth and trends of various segments and the market in various regions.

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Induced Pluripotent Stem Cells Market Report Includes:

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By Product Types:HepatocytesFibroblastsKeratinocytesAmniotic CellsOthers

The report is further broken down into various segments such as product types, applications, and regions.

By Applications:Academic ResearchDrug Development And DiscoveryToxicity ScreeningRegenerative Medicine

Our analysts drafted the report by gathering information through primary (through surveys and interviews) and secondary (included industry body databases, reputable paid sources, and trade journals) methods of data collection. The report encompasses an exhaustive qualitative and quantitative evaluation.

The study includes growth trends, micro- and macro-economic indicators, and regulations and governmental policies.

By Regions:Asia Pacific (China, Japan, India, and Rest of Asia Pacific)Europe (Germany, the UK, France, and Rest of Europe)North America (the US, Mexico, and Canada)Latin America (Brazil and Rest of Latin America)Middle East & Africa (GCC Countries and Rest of Middle East & Africa)

The Induced Pluripotent Stem Cells Market Report Covers the Following Companies:Fujifilm Holding CorporationAstellas PharmaFate TherapeuticsBristol-Myers Squibb CompanyViaCyteCelgene CorporationAastrom BiosciencesAcelity HoldingsStemCellsJapan Tissue EngineeringOrganogenesis

The subject matter experts analyzed various companies to understand the products and/services relevant to the market. The report includes information such as gross revenue, production and consumption, average product price, and market shares of key players. Other factors such as competitive analysis and trends, mergers & acquisitions, and expansion strategies have been included in the report. This will enable the existing competitors and new entrants understand the competitive scenario to plan future strategies.

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The Report Provides:

The Induced Pluripotent Stem Cells Market Report Addresses the Following Queries:

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About DataIntelo:DATAINTELO has set its benchmark in the market research industry by providing syndicated and customized research report to the clients. The database of the company is updated on a daily basis to prompt the clients with the latest trends and in-depth analysis of the industry. Our pool of database contains various industry verticals that include: IT & Telecom, Food Beverage, Automotive, Healthcare, Chemicals and Energy, Consumer foods, Food and beverages, and many more. Each and every report goes through the proper research methodology, validated from the professionals and analysts to ensure the eminent quality reports.

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Induced Pluripotent Stem Cells Market Analysis With Key Players, Applications, Trends And Forecasts To 2026 - Surfacing Magazine

Induced Pluripotent Stem Cells Market 2019 analysis, size, top companies, share, strategies and forecast to 2026 – WhaTech Technology and Markets News

Induced Pluripotent Stem Cells Market research report provides the details about Industry Chain structure, Market Competition, Market Size & Share, SWOT Analysis, Technology, Cost, Raw Materials, Consumer Preference, Development & Trends, Regional Forecast, Company & Profile, and Product & Service.

This Induced Pluripotent Stem Cells Market research report is focused on providing its reader with all the necessary details that can help them make necessary business decisions. It provides wholesome information that is necessary to understand the market inside-out.

ReportsnReports has recently added a new research report to its expanding repository. The research report, titled Induced Pluripotent Stem Cells Market, mainly includes a detailed segmentation of this sector, which is expected to generate massive returns by the end of the forecast period, thus showing an appreciable rate of growth over the coming years on an annual basis.

The research study also looks specifically at the need for Induced Pluripotent Stem Cells Market.

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Report Scope:

This study is focused on the market side of iPSCs rather than its technical side. Different market segments for this emerging market are covered.

For example, application-based market segments include academic research, drug development and toxicity testing, and regenerative medicine; product function-based market segments include molecular and cellular engineering, cellular reprogramming, cell culture, cell differentiation and cell analysis; iPSC-derived cell-type-based market segments include cardiomyocytes, hepatocytes, neurons, endothelia cells and other cell types; geography-based market segments include the U.S., Europe, Asia-Pacific and Rest of the World.

Research and market trends are also analyzed by studying the funding, patent publications and research publications in the field.

Report Includes:

59 tables

An overview of the global market for induced pluripotent stem cells

Analyses of global market trends with data from 2018 and 2019, and projections of compound annual growth rates (CAGRs) through 2024

Information on induced pluripotent stem cell research products, including various assays and kits, culture media and medium components, such as serum, growth factors and inhibitors, antibodies, enzymes

Complete understanding of the key technologies adopted for induced pluripotent stem cell research

Discussion of important manufacturers, technologies, and factors influencing market demand, such as the driving forces and limiting factors of induced pluripotent stem cell market growth

Profiles of major players in the industry, including Applied StemCell Inc., BlueRock Therapeutics, Corning Life Sciences, EMD Millipore, Lonza Group Ltd., Promega Corp. and Thermo Fisher Scientific Inc.

Summary

It has been over 10 years since the discovery of induced pluripotent stem cell (iPSC) technology. The market has gradually become an important part of the life sciences industry during recent years.

Particularly for the past five years, the global market for iPSCs has experienced a rapid growth. The market was estimated at REDACTED in 2018 and over REDACTED in 2019, with an average REDACTED growth.

The overall iPSC market is forecast to continue its steady growth and reach REDACTED in 2024, with anestimated compound annual growth rate (CAGR) of REDACTED from 2019 through 2024.

Key Drivers for Market Growth

This report has identified several key drivers for the rapidly growing market

iPSCs hold promising hope for therapeutic solutions for diseases without ethical issues. A series of technical breakthroughs were made in recent years for improving cellular reprogramming, differentiation and large-scale production of GMP- grade iPSCs derived cells toward clinical usability.

Advances in genetics such as NGS technologies have promoted the progress on precision medicine, where the availability of iPSCs from a variety of genetic, lifestyle and environment backgrounds will help make the precision healthcare a clinical reality. iPSC banking together with related technologies is developing into a platform for precision and personalized medicine, which is experiencing rapid growth globally.

In recent years, several iPSCs clinical trials have been or are going to be launched for a variety of diseases. The first human iPSC clinical trial started in August 2014, and the recent report of the first macular degeneration patient treated with the sheets of retinal pigmented epithelial cells made from iPSCs was encouraging.

The progresses toward clinical practice will drive the growth of the clinical market and the research market as well.

The pharmaceutical industry needs better cell sources such as iPSC-derived functional cells for drug toxicity testing and drug screening.

The U.S. government has been encouraging the marketing of stem cells, including iPSCs.

The U.S. Food and Drug Administration (FDA) has been authorized to provide orphan drug designations for many of the therapies developed for rare diseases such as Parkinsons and Huntingtons using stem cells.

The provisions of grants from organizations, such as the National Institutes of Health (NIH) and the California Institute for Regenerative Medicine (CIRM) have been encouraging for the research institutes to venture into iPSC research.

Rapidly growing medical tourism and contract research outsourcing drives the Asia-Pacific stem cell market.

Cellular reprogramming, including iPSC technology, was awarded the 2012 Nobel Prize.

New biotechnologies such as CRISPR/Cas genome editing technology are advancing iPSCs into more and better uses. For example, the hypoimmunogenic derivatives of engineered iPSCs have shown lost immunogenicity which would become a potential novel therapy to treat various diseases.

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Recent Industry Trend:

The report contains the profiles of various prominent players in the Global Induced Pluripotent Stem Cells Market. Different strategies implemented by these vendors have been analyzed and studied in order to gain a competitive edge, create unique product portfolios and increase their market share.

The study also sheds light on major global industry vendors. Such essential vendors consist of both new and well-known players.

In addition, the business report contains important data relating to the launch of new products on the market, specific licenses, domestic scenarios and the strategies of the organization implemented on the market.

Scope of the Report:

Through following the Induced Pluripotent Stem Cells Market through depth, the readers should find this study very helpful. The aspects and details are depicted by charts, bar graphs, pie diagrams, and other visual representations in theInduced Pluripotent Stem Cells Market study.

This intensifies the representation of the pictures and also helps to improve the facts of the Induced Pluripotent Stem Cells Market industry. At a substantial CAGR, the Induced Pluripotent Stem Cells Market is likely to grow.

Induced Pluripotent Stem Cells Market reports main objective is to guide the user to understand the market in terms of its definition, classification, industry potential, the latest trends, and the challenges facing the Induced Pluripotent Stem Cells Market.

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Induced Pluripotent Stem Cells Market 2019 analysis, size, top companies, share, strategies and forecast to 2026 - WhaTech Technology and Markets News

Cell Therapies Can Revolutionize Treatment, Automation Needed to Scale Production – ENGINEERING.com

Parker Hannifin has sponsored this post.

Cell therapies promise treatments for serious illnesses, but require automation and manufacturing expertise to scale up production for research and products. (Image courtesy of Parker Hannifin.)

Cellular therapies and bio-fabrication are two of the most revolutionary treatments for serious illnesses to be developed in the early 21st century, offering the hope of cures where once only symptomatic treatments were available. The 2006 discovery of Induced Pluripotent Stem Cells (iPSCs) formed a catalyst for research and development into these new therapeutic approaches. Stem cell therapies offer promising avenues for the treatment of devastating illnesses such as diabetes, cancer, heart disease and even neurological diseases.

Tailored cell therapies using iPSCs are considered to be the new Third Pillar of the drug and treatment industry, standing alongside small molecules and biologics as tools for treatment. However, the widespread research and treatment using cell therapies requires mass-produced iPSCs to be available in quantitywhich means advanced manufacturing techniques.

Cells are tiny living, complex organisms; they must be handled with precision and accuracy. Automated handling equipment needs a heightened level of dexterity and control. (Image courtesy of Parker Hannifin.)

Scaling up the production of iPSCs requires investmentsome of which is already in place with two deals: $70 million to the New Institute for Innovation in Manufacturing Biopharmaceuticals (NIIMBL) to advance U.S. leadership in the biopharmaceutical industry, and an 87-member coalition funded by the Defense Department called ARMI-BioFab USA, which aims to develop the next-generation techniques needed to repair and replace cells, tissues and organs for wounded military service veterans.

The key to success in the scale-up of production is advanced automation, which will improve the manufacturing process used to fabricate cell colonies.

Currently, most research and cell fabrication involves a significant amount of manual work and decision-making, which can be error-prone and represents a bottleneck in attempts to scale these fabrication processes.

One way to improve the manufacturing processes related to cell therapies is by partnering with experienced automation and manufacturing industry leaders, who can share their expertise. An example of this is the partnership between Parker Hannifin and CellX Technologies. Together, these companies have developed a platform to help researchers and clinicians quantify key morphological stem cells, automate the handling process and perform cell maintenance.

Current cell therapy research is hampered by difficulties with a lack of large field-of-view and high-resolution optics when imaging live cell cultures. This makes it difficult to monitor and quantify changes to the cells. Available devices and equipment for sampling, transfer or deletion of specific cells or colonies also lack the rigorous accuracy that manufacturing-scale production would require. Instead, visual assessment and manual transfer by lab technicians is the usual methodsacrificing speed and production volume.

An automated, image-based system would enable accurate quantitative metrics of biological performance and will be applicable at a cell-by-cell or a colony-by-colony basis, among other benefits.

Automated cell-handling equipment needs to be precise and finely calibrated in order to handle delicate cells with the necessary dexterity and control. Three primary handling techniques are used for this very difficult automation task:

Combining capabilities for these three functions into a single platform will enable multiple benefits, including improved reproducibility and quality of cells for research and products, reduce variability and costs from manual processes, improved lot traceability and documentation, and define quantitative process quality attributes and metrics.

Parker Hannifins expertise in manufacturability, digital pathology and additive manufacturing lends itself directly to the development of the CellX platform. CellX enables automation of the scanning and identification processes, and pairs this with cell selection and precision placement.

The CellX Device, developed by Parker Hannifin and CellX Technologies, combines large field-of-view imaging with precision instrumentation, fluidics, and documentation and control capabilities. (Image courtesy of Parker Hannifin.)

CellX also needed customization of standard products. Parker Hannifin has decades of experience in close tolerance special purpose fluidics and actuator technology, and developed enabling technology for the CellX central core, which consists of a high-quality automated inverted microscope and CCD camera with brightfield and fluorescent imaging capabilities.

Some of the specialized equipment that Parker Hannifin helped develop for CellX includes a load and removal station for disposable cell-picking tips, and environmentally controlled workspace to maintain sterility and oxygen levels, and an integrated sensor to accurately locate each new tip.

The combined precision and imaging capabilities of the CellX platform enable rapid data collection and high repeatability, which means researchers can rely on accurate data, healthy cell colonies and quantitative, reproducible standards for cell therapy development. Parker Hannifin has a proven history of developing new tools and instruments for manufacturing processes with their partner OEMsand in the case of CellX, accelerating the development of the future of cell therapies.

To learn more about Parker Hannifins development of the CellX platform, including use cases and details on the full complement of customized equipment and enabling features, download the full whitepaper from Parker Hannifin.

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Cell Therapies Can Revolutionize Treatment, Automation Needed to Scale Production - ENGINEERING.com