Category Archives: Induced Pluripotent Stem Cells

Space travel may change the human heart – Inverse

Spending time in the microgravity environment aboard the International Space Station appears to alter gene expression in the human heart. The results shed light on what life in space might do to the human body and what happens when that body returns to Earth.

Humans have been venturing out into space for over 50 years now, but very little is known about the toll microgravity might take on the human body. With the age of commercial space travel fast approaching, it is increasingly critical to understand how our bodies adapt to space flight.

Space is our next frontier. In the next 100 years, humans will be traveling through space all the time, says Joseph Wu, Stanford University professor and senior author on the study.

To understand the effect of space travel on our most crucial, blood-pumping organ, in 2016 Wus team sent beating, human-induced pluripotent stem cell-derived cardiomyocytes, a kind of heart muscle cell, to the International Space Station.

The results, published in the journal Stem Cell Reports, show that time in a microgravity environment alters gene expression in the heart muscle cells, but most of these changes revert after the cells are back on Earth.

This is the first study to look at the effects of microgravity on a cellular level, the researchers say.

To create the stem-cell model, the researchers harvested blood cells from three people, none of whom had a history of heart disease. They then reprogrammed the cells to become cardiomyocytes. The cells were sent to the ISS and cultured aboard, staying in the microgravity environment for a total of five and a half weeks before being flown home.

By comparing the cells gene expression in-space, on return and to controls, the researchers found that time in space altered the expression patterns in 2,635 of the cells genes. Most of the altered genes are related to mitochondrial metabolism, the process by which nutrients are converted into energy and used to carry out different functions in cells. Expression patterns looked similar to controls after 10 days back on Earth a possible sign that the body can reverse adaptations to life in space.

We do know that heart muscle cells can adapt. Its hard to tell if these changes are necessarily negative or if they are natural adaptations, says Alexa Wnorowski, a graduate student at Stanford University who was involved in the study.

Its hard for us to come up with a conclusion of what that means. It gives us a future direction to look into, she says.

The team plans to use the data and compare it with both records of physiological changes in astronauts during missions and with symptoms of heart disease in order to get a better sense of these adaptations long-term effects.

The study could also have implications on heart health for those that dont even plan to travel beyond the stars, say the researchers, offering insight into how the environment may affect gene expression in heart cells here on Earth.

Thats one of the hopes for the directions that this type of research might go, Wnorowski tells Inverse. If we figure out that microgravity is able to replicate some of the gene expressions we see in diseases on Earth.

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Space travel may change the human heart - Inverse

Here’s Why Fate Therapeutics Dropped as Much as 19.9% Today – Motley Fool

What happened

Shares of Fate Therapeutics (NASDAQ:FATE) fell nearly 20% today after the company reported third-quarter 2019 operating results and announced it will make six presentations at the upcoming American Society of Hematology (ASH) annual meeting in December. The biopharma disclosed several notable updates and is clearly making progress, but there are certainly a lot of moving parts for investors to understand.

Fate Therapeutics became the first entity to dose a patient with an engineered stem cell-derived cellular medicine in October, began enrolling a higher-dose cohort for a separate drug candidate, announced a new manufacturing facility, and presented several more updates. Therefore, it appears that today's sell-off is an attempt to buy time to digest the sudden increase in complexity, especially considering management decided to hold off important details until the ASH presentations next month.

As of 2:22 p.m. EST, the small-cap stock had settled to a 9.4% loss.

Image source: Getty Images.

Fate Therapeutics was one of the first companies to jump into developing natural killer (NK) cells as therapeutic agents. NK cells offer several notable advantages compared to T cells, the first immune cells to be widely studied in immuno-oncology, including the ability to dose patients multiple times. But they've largely failed to live up to the hype in early clinical studies.

The company is hoping its unique approach can lead to success. Rather than engineering each individual patient's own NK cells, Fate Therapeutics is using a single master cell line -- an induced pluripotent stem cell (iPSC) line -- to engineer cellular medicines that can be given to any individual. That should smooth over manufacturing obstacles, lower costs, and potentially lead to more reproducible outcomes compared to the initial approach used in CAR-T cell therapies.

But there's a lot to digest as the pipeline matures. Fate Therapeutics announced clinical progress for three drug candidates, explained a highly complex phase 1 clinical trial for FT500 in advanced solid tumors, and opened a new manufacturing facility. To the dismay of Wall Street analysts, executives provided few specific details of drug candidates on the third-quarter 2019 earnings conference call and instead chose to wait until the updates at ASH next month.

Investors interested in Fate Therapeutics shouldn't necessarily be discouraged by any recent developments. The company's technology platform ultimately will be judged by clinical results. Given the lack of specific details and a sudden increase in complexity and competition -- including a partnership between Allogene and Notch Therapeutics yesterday and recent fundraising rounds by A2 Biotherapeutics, Nkarta Therapeutics, and Achilles Therapeutics -- Wall Street simply took the "show me" approach. Analysts may get their answers in December.

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Here's Why Fate Therapeutics Dropped as Much as 19.9% Today - Motley Fool

Allogene Therapeutics and Notch Therapeutics Announce Collaboration to Research and Develop Induced Pluripotent Stem Cell (iPSC)-Derived Allogeneic…

Collaboration Includes Exclusive Rights and Targets for Initial Applications in Non-Hodgkin Lymphoma, Leukemia and Multiple Myeloma

Notch to Receive Upfront Payment, Research Funding and an Equity Investment Plus Development and Commercial Milestones and Royalties on Net Sales

SOUTH SAN FRANCISCO, Calif. and TORONTO, Nov. 05, 2019 (GLOBE NEWSWIRE) -- Allogene Therapeutics, Inc. (Nasdaq: ALLO), a clinical-stage biotechnology company pioneering the development of allogeneic CAR T (AlloCAR T) therapies for cancer, and Notch Therapeutics Inc., an immune cell therapy company creating universally compatible, allogeneic T cell therapies for the treatment of diseases of high unmet need, today announced an exclusive worldwide collaboration and license agreement to research and develop induced pluripotent stem cell (iPSC) AlloCAR therapy products for initial application in non-Hodgkin lymphoma, leukemia and multiple myeloma. Under the partnership, Allogene and Notch will create allogeneic cell therapy candidates from T cells or natural killer (NK) cells using Notchs Engineered Thymic Niche (ETN) platform.

Notch was established in 2018 by Juan Carlos Ziga-Pflcker, Ph.D. and Peter Zandstra, Ph.D., recognized pioneers in iPSC and T cell differentiation technology. Notch is developing a next-generation approach to differentiating mature immune cells from iPSCs. The Notch ETN technology platform offers potential flexibility and scalability for the production of stem cell-derived immune cell therapies. iPSCs may provide renewable starting material for AlloCAR T therapies that could allow for improved efficiency of gene editing, greater scalability of supply, product homogeneity and more streamlined manufacturing.

This collaboration exemplifies Allogenes long-term commitment to advancing the field of cancer treatment as we continue to expand and progress our innovative pipeline of off-the-shelf AlloCAR candidates, said David Chang, M.D., Ph.D., President, CEO and Co-Founder of Allogene Therapeutics. The scientific founders of Notch Therapeutics are among the most respected experts in the field of stem cell biology and its applications to generating T cells and other functional immune cells. We are confident that their technology and expertise, combined with Allogenes leadership in AlloCAR therapies, has the potential to unlock future generations of cell therapy treatments for patients.

Renewable-source, off-the-shelf cell therapies that may produce cells with greater consistency and at industrial scale have long been the dream for people working in this field, said Ulrik Nielsen, Ph.D., Executive Chairman of Notch. We are delighted to spring into the research collaboration for iPSC-based AlloCAR therapies with Allogene, a leader in the allogeneic CAR T field, with the goal of expanding options for patients.

Under the terms of the agreement, Notch will be responsible for preclinical research of next-generation iPSC AlloCAR T cells. Allogene will clinically develop the product candidates and holds exclusive worldwide rights to commercialize resulting products. Allogene will provide to Notch an upfront payment of $10 million. Notch will be eligible to receive up to $7.25 million upon achieving certain agreed research milestones, up to $4.0 million per exclusive target upon achieving certain pre-clinical development milestones, and up to $283 million per exclusive target and cell type upon achieving certain clinical, regulatory and commercial milestones as well as tiered royalties on net sales in the mid to high single digits. In addition to this collaboration and license agreement, Allogene has acquired a 25 percent equity position in Notch and will assume a seat on Notchs Board of Directors.

Master cell banks of genetically modified, induced pluripotent stem cells could provide an inexhaustible source of cell therapies that may improve outcomes and expand applicability to new areas, said Notch Co-Founder Juan Carlos Ziga-Pflcker, Ph.D., a senior scientist at Sunnybrook Research Institute and a Professor and Chair of the Department of Immunology at the University of Toronto.

This work with Allogene may also pave the way for additional off-the-shelf cell therapeutics that are custom-designed to treat other immunity-related diseases such as infectious diseases, autoimmune diseases and aging, said Notch Co-Founder and Chief Scientific Officer Peter Zandstra, Ph.D., a Professor at the University of British Columbia and University of Toronto.

About Notch Therapeutics ( is an immune cell therapy company creating universally compatible, allogeneic (off-the-shelf) T cell therapies for the treatment of diseases of high unmet medical need. Notchs technology platform uses genetically tailored stem cells as a renewable source for creating allogeneic T cell therapies that expand treatment options and deliver safer, consistently manufactured and more cost-effective cell immunotherapies to patients. At the core of Notchs technology is the synthetic Engineered Thymic Niche (ETN) platform, which drives the expansion and differentiation of stem cells in scalable, fully defined, feeder-free and serum-free cultures into T cells that can be genetically tailored for any T cell-based immunotherapeutic application. This technology was invented in the laboratories of Juan-Carlos Ziga-Pflcker, Ph.D. at Sunnybrook Research Institute and Peter Zandstra, Ph.D., FRSC at the University of Toronto. Notch was founded by these two institutions, in conjunction with MaRS Innovation (now Toronto Innovation Acceleration Partners) and the Center for Commercialization of Regenerative Medicine (CCRM) in Toronto.

About Allogene TherapeuticsAllogene Therapeutics, with headquarters inSouth San Francisco, is a clinical-stage biotechnology company pioneering the development of allogeneic chimeric antigen receptor Tcell (AlloCAR T) therapies for cancer. Led by a world-class management team with significantexperience in cell therapy, Allogene is developing a pipeline of off-the-shelf CAR T cell therapycandidates with the goal of delivering readily available cell therapy on-demand, more reliably, and atgreater scale to more patients. For more information, please, and follow @AllogeneTx on Twitter and LinkedIn.

Cautionary Note on Forward-Looking Statements This press release contains forward-looking statements for purposes of the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. The press release may, in some cases, use terms such as "predicts," "believes," "potential," "proposed," "continue," "estimates," "anticipates," "expects," "plans," "intends," "may," "could," "might," "will," "should" or other words that convey uncertainty of future events or outcomes to identify these forward-looking statements. Forward-looking statements include statements regarding intentions, beliefs, projections, outlook, analyses or current expectations concerning, among other things: the ability to progress the research collaboration, Notchs ability to develop a next-generation approach to differentiating mature immune cells from iPSCs, the ability to develop and manufacture new therapies from Notch technology, and the potential benefits of Notch technology and AlloCAR T therapy. Various factors may cause differences between Allogenes expectations and actual results as discussed in greater detail in Allogenes filings with theSecurities and Exchange Commission(SEC), including without limitation in its Form 10-Q for the quarter endedJune 30, 2019. Any forward-looking statements that are made in this press release speak only as of the date of this press release. Allogene assumes no obligation to update the forward-looking statements whether as a result of new information, future events or otherwise, after the date of this press release.

Allogene Media/Investor Contact:Christine CassianoChief Communications Officer(714)

Notch Media Contact:Mary MoynihanM2Friend

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Allogene Therapeutics and Notch Therapeutics Announce Collaboration to Research and Develop Induced Pluripotent Stem Cell (iPSC)-Derived Allogeneic...

Goldfinch Bio to Present Oral and Poster Presentations at the American Society of Nephrology Kidney Week 2019 Annual Meeting – BioSpace

Nov. 5, 2019 12:00 UTC

CAMBRIDGE, Mass.--(BUSINESS WIRE)-- Goldfinch Bio, a U.S.-based, clinical stage biotechnology company focused on discovering and developing precision medicines for the treatment of kidney diseases, today announced it will present one oral and two poster presentations at the American Society of Nephrology (ASN) Kidney Week 2019 Annual Meeting, taking place November 5-10, 2019, in Washington, D.C.

The oral presentation will address the ways in which Goldfinch Bios platform generates induced pluripotent stem cell (iPSC)-derived human podocytes and kidney organoids to enable target validation and preclinical assessment of prospective therapies for kidney disease. The poster presentations will include data on how GFB-887, a sub-type selective, small molecule transient receptor potential canonical 5 (TRPC5) ion channel inhibitor, was effective in reducing proteinuria in animal models of focal segmental glomerulosclerosis (FSGS) and other kidney diseases, as well as data on how Goldfinch Bios proprietary Kidney Genome AtlasTM can be leveraged to unravel molecular mechanisms of kidney diseases.

Details are as follows:

Abstract Title: GFB-887, a Small Molecule Inhibitor of TRPC5, Attenuates Proteinuria in Animal Models of FSGS, Minimal Change Disease, and Diabetic Nephropathy (poster presentation)Session Title: Glomerular Diseases: Podocyte Biology IPresenter: John F. Reilly, Ph.D., Goldfinch BioDate/Time: Thursday, November 7, 2019, from 10:00 a.m. to 12:00 p.m. ETLocation: Exhibit Hall, Walter E. Washington Convention CenterAbstract Number: TH-PO1063

Abstract Title: An iPSC platform for Human Preclinical Evaluation of Kidney Disease Targeting Compounds (oral presentation)Session Title: Glomerular Diseases: Technologies, Mechanisms, and TherapeuticsPresenter: Amy Duyen Westerling-Bui, Ph.D., Goldfinch BioDate/Time: Saturday, November 9, 2019, 5:30 p.m. to 5:42 p.m. ETLocation: 201, Walter E. Washington Convention CenterAbstract Number: SA-OR053

Abstract Title: Unraveling the Genetic Contributions to Kidney Disease with the Kidney Genome Atlas (poster presentation)Session Title: Genetic Diseases of the Kidney - IIIPresenter: Thomas Soare, Ph.D., Goldfinch BioDate/Time: Saturday, November 9, 2019, 10:00 a.m. to 12:00 p.m. ETLocation: Exhibit Hall, Walter E. Washington Convention CenterAbstract Number: SA-PO408

About the Kidney Genome AtlasTM

Goldfinch Bios Kidney Genome Atlas (KGA) is the most comprehensive patient registry to investigate the underlying mechanisms of kidney disease. Through the combination of genomic, transcriptomic and proteomic data with thousands of anonymized clinical patient profiles, Goldfinch Bio is able to conduct unprecedented analyses to elucidate pathways and novel targets for kidney disease.

In May 2019, Goldfinch Bio entered into a strategic collaboration with Gilead Sciences, Inc. to sequence 80,000 diabetic kidney disease (DKD) patients and diabetic controls. Goldfinch Bio received a $55 million upfront payment, including a $5 million equity investment, and a commitment of an additional $54 million to support the development of the KGA platform for DKD. Goldfinch Bio will validate targets and support discovery and development of products to which Gilead will have exclusive option rights in exchange for additional milestone payments.

About GFB-887

GFB-887 is a sub-type selective, small molecule transient receptor potential canonical 5 (TRPC5) ion channel inhibitor in clinical development for the treatment of focal segmental glomerulosclerosis (FSGS), treatment-resistant minimal change disease (TR-MCD) and diabetic nephropathy (DN). The ongoing Phase 1 study is evaluating the safety, tolerability, and pharmacokinetic profile of GFB-887 in healthy volunteers.

TRPC5 is a calcium-permeable ion channel implicated in the pathogenesis of kidney disease. Recent evidence has demonstrated that TRPC5 and Rac1, a critical regulator of cellular motility, form a vicious cycle that drives pathogenic remodeling of the actin cytoskeleton in podocytes. This causes podocyte loss and breach of the filtration barrier, leading to proteinuria, the hallmark of progressive kidney diseases such as FSGS, TR-MCD and DN. Inhibition of TRPC5 offers a potential point of therapeutic intervention to restore podocyte integrity and halt progression of these diseases.

About FSGS, TR-MCD and DN

Focal segmental glomerulosclerosis (FSGS) is a rare kidney disorder and histopathologic diagnosis characterized by scarring of the kidney's filtering units, or glomeruli, leading to proteinuria, an excess of essential proteins spilling into the urine. FSGS is associated with loss of podocytes, terminally differentiated cells of the kidney glomeruli essential for filtration and proper kidney function. Recent research into the genetics of kidney disease has identified over 50 genes associated with FSGS and implicates the podocyte as a central player in the pathogenesis of FSGS. There are currently no FDA-approved treatments available for patients with FSGS.

Similar to FSGS, treatment-resistant minimal change disease (TR-MCD) is a rare kidney disorder characterized by podocyte injury and is an important cause of nephrotic syndrome in children as well as adults. Clinical hallmarks of MCD include rapid onset of edema and weight gain. While MCD may be managed with corticosteroids, a subset of patients fail to respond and are considered treatment-resistant. There are currently no FDA-approved treatments available for patients with TR-MCD.

Diabetic nephropathy (DN) develops in approximately 30 to 40 percent of patients who have diabetes and is a leading cause of end-stage kidney disease, cardiovascular disease and early mortality worldwide. Despite current therapies, the number of people with DN continues to increase, highlighting the need for additional treatments that preserve kidney function.

About Goldfinch Bio

Goldfinch Bio, Inc. is a clinical stage biotechnology company that leverages a genomics-based, precision medicine approach to discovering and developing kidney disease treatments. Its Kidney Genome AtlasTM is a proprietary biology platform that drives candidate discovery, patient selection and biomarker development. The Companys lead candidate, GFB-887, is a transient receptor potential canonical 5 (TRPC5) ion channel inhibitor being evaluated in a Phase 1 clinical trial for the treatment of kidney diseases. Goldfinch Bio is also developing GFB-024, a peripherally-restricted cannabinoid receptor 1 (CB1) monoclonal antibody, for the treatment of rare and metabolic kidney diseases, which it licensed from Takeda Pharmaceutical Company Limited in October 2019. The company expects to submit an IND for GFB-024 in 2H 2020. Goldfinch Bio, headquartered in Cambridge, Massachusetts, was launched in 2016 by Third Rock Ventures and has an established strategic collaboration with Gilead Sciences, Inc. For more information about Goldfinch Bio, visit

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Goldfinch Bio to Present Oral and Poster Presentations at the American Society of Nephrology Kidney Week 2019 Annual Meeting - BioSpace

MD Anderson and Takeda Team Up on Next-Generation Immuno-Oncology Therapeutics – BioSpace

The University of Texas MD Anderson Cancer Center has partnered with Takeda Pharmaceutical on immuno-oncology therapies. Specifically, they announced an exclusive license deal and research agreement to develop cord-blood derived chimeric antigen receptor-directed natural killer (CAR NK)-cell therapies. They say these CAR-NK therapies will be armored with IL-15 to treat B-cell and other cancers.

Under the deal, Takeda will access MD Andersons CAR-NK technology platform and pick up the exclusive rights to develop and commercialize up to four programs. Those programs include a CD19-targeted CAR-NK-cell therapy and a B-cell maturation antigen (BCMA)-targeted CAR-NK therapy. They will collaborate on research to advance the programs.

Our vision is to improve upon existing treatments by developing armored CAR NKs that could be administered off-the-shelf in an outpatient settingenabling more patients to be treated effectively, quickly and with minimal toxicities, said Katy Rezvani, professor of Stem Cell Transplantation and Cellular Therapy at MD Anderson. With their expertise in hematologic malignancies and commitment to developing next-generation cell therapies, Takeda is the ideal collaborator to help our team advance CAR NK-cell therapies to patients in need of treatments.

MD Andersons allogeneic CAR NK technology platform collects umbilical cord blood, isolates NK cells for it, and then engineers those NK cells to express CARs against specific cancer targets. They utilize a retroviral vector to deliver genes to the CAR NK cells, which both improves their effectiveness and fine-tunes them for specific cancer cells. The CD19 CAR makes the cells even more specific for B-cell malignancies, and the IL-15 improves the proliferation and survival of the CAR-NK cells in the body.

Currently approved CAR-T therapies, which essentially means Novartis Kymriah (tisagenlecleucel) and Gilead Sciences Yescarta (axicabtagene ciloleucel), isolate T-cells from the patients blood, which are then engineered to express CARs against the patients specific cancer. The downside to this is that it is time-consuming, taking several weeks. So this approach, which others are also working to develop, would be more of a one-size-fits-all therapy that could be used to treat the patient immediately rather than uniquely engineer the CARs.

MD Anderson and Takeda expect their CD19 CAR NK therapy could be administered in an outpatient setting. There is an ongoing Phase I/IIa trial in patients with relapsed and refractory B-cell cancers. In it, there has been little or no evidence of the severe cytokine release syndrome (CRS) or neurotoxicity associated with Kymriah and Yescartaalthough those companies and the affiliated healthcare practitioners have developed protocols for minimizing those effects.

As well as developing the CAR NK-cell therapies, Takeda and its partners are working to improve the safety, efficacy and accessibility of the first-generation CAR-Ts, including gamma delta CAR-Ts, induced pluripotent stem cell-derived CAR-Ts, CAR-Ts that target solid tumors, and other approaches.

Takeda reportedly hopes to advance five oncology cell therapies into the clinic by the end of fiscal year 2020.

Under the agreement, Takeda will handle development, manufacturing and commercialization of CAR-NK products that come out of the partnership. MD Anderson will receive an undisclosed upfront payment and be eligible for various milestones for each target in addition to tiered royalties on net sales of any products that come out of the deal.

MD Andersons CAR-NK platform is led by Rezvani and supported by the adoptive cell therapy platform, Chronic Lymphocytic Leukemia Moon Shot and B-Cell Lymphoma Moon Shot, which are all part of MD Andersons Moon Shots Program.

MD Andersons CAR-NK platform represents the curative potential of cell therapies, which is why we are establishing the CD19 CAR NK as our lead cell therapy candidate in oncology, said Andy Plump, president of Research and Development at Takeda. We need to work swiftly and with purpose, and as such, we intend to initiate a pivotal study of the CD19 CAR NK in 2021.

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MD Anderson and Takeda Team Up on Next-Generation Immuno-Oncology Therapeutics - BioSpace

Induced Pluripotent Stem Cell Market is expected to witness a strong CAGR of 7.0% from 2018 to 2026 – Zebvo

The healthcare industry has been focusing on excessive research and development in the last couple of decades to ensure that the need to address issues related to the availability of drugs and treatments for certain chronic diseases is effectively met.

Healthcare researchers and scientists at the Li Ka Shing Faculty of Medicine of the Hong Kong University have successfully demonstrated the utilization of human induced pluripotent stem cells or hiPSCs from the skin cells of the patient for testing therapeutic drugs.

The success of this research suggests that scientists have crossed one more hurdle towards using stem cells in precision medicine for the treatment of patients suffering from sporadic hereditary diseases. iPSCs are the new generation approach towards the prevention and treatment of diseases that takes into account patients on an individual basis considering their genetic makeup, lifestyle, and environment. Along with the capacity to transform into different body cell types and same genetic composition of the donors, hiPSCs have surfaced as a promising cell source to screen and test drugs.

In the present research, hiPSC was synthesized from patients suffering from a rare form of hereditary cardiomyopathy owing to the mutations in Lamin A/C related cardiomyopathy in their distinct families. The affected individuals suffer from sudden death, stroke, and heart failure at a very young age. As on date, there is no exact treatment available for this condition. This team in Hong Kong tested a drug named PTC124 to suppress specific genetic mutations in other genetic diseases into the iPSC transformed heart muscle cells. While this technology is being considered as a breakthrough in clinical stem cell research, the team at Hong Kong University is collaborating with drug companies regarding its clinical application.

The unique properties of iPS cells provides extensive potential to several biopharmaceutical applications. iPSCs are also used in toxicology testing, high throughput, disease modeling, and target identification.

This type of stem cell has the potential to transform drug discovery by offering physiologically relevant cells for tool discovery, compound identification, and target validation. A new report by Persistence Market Research (PMR) states that the globalinduced pluripotent stem or iPS cell marketis expected to witness a strong CAGR of 7.0% from 2018 to 2026. In 2017, the market was worth US$ 1,254.0 Mn and is expected to reach US$ 2,299.5 Mn by the end of the forecast period in 2026.

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Customization to be the Key Focus of Market Players

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Entry of Multiple Small Players to be Witnessed in the Coming Years

Several leading players have their presence in the global market; however, many specialized products and services are provided by small and regional vendors. By targeting their marketing strategies towards research institutes and small biotechnology companies, these new players have swiftly established their presence in the market.

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Induced Pluripotent Stem Cell Market is expected to witness a strong CAGR of 7.0% from 2018 to 2026 - Zebvo

Can organoids, derived from stem cells, be used in disease treatments? – The Hindu

The story so far: On Monday, October 21, at Neuroscience 2019, the Society for Neurosciences 49th annual meeting, held in Chicago, U.S., two neuroscientists warned the gathering that fellow scientists are perilously close to crossing the ethical red line of growing mini-brains or organoids in the laboratory that can perceive or feel things. In some cases, scientists have already transplanted such lab-grown brain organoid to adult animals. The transplanted organoid had integrated with the animal brain, grown new neuronal connections and responded to light. Similarly, lung organoid transplanted into mice was able to form branching airways and early alveolar structures. These are seen as a step towards potential humanisation of host animals.

Organoids are a group of cells grown in laboratories into three-dimensional, miniature structures that mimic the cell arrangement of a fully-grown organ. They are tiny (typically the size of a pea) organ-like structures that do not achieve all the functional maturity of human organs but often resemble the early stages of a developing tissue. Most organoids contain only a subset of all the cells seen in a real organ, but lack blood vessels to make them fully functional. In the case of brain organoids, scientists have been able to develop neurons and even make specific brain regions such as the cerebral cortex that closely resemble the human brain. The largest brain organoids that have been grown in the laboratory are about 4 mm in diameter.

Organoids are grown in the lab using stem cells that can become any of the specialised cells seen in the human body, or stem cells taken from the organ or adults cells that have been induced to behave like stem cells, scientifically called induced pluripotent stem cells (iPSC). Stem cells are provided with nutrients and other specific molecules to grow and become cells resembling a specific organ. The growing cells are capable of self-organising into cellular structures of a specific organ and can partly replicate complex functions of mature organs physiological processes to regeneration and being in a diseased state.

Organoids of the brain, small intestine, kidney, heart, stomach, eyes, liver, pancreas, prostate, salivary glands, and inner ear to name a few have already been developed in the laboratory.

Since the use of embryonic stem cells to grow organs of interest has been mired in controversy leading to a ban on such research, researchers have turned to generating organoids using stem cells. Researchers have been successful in generating organoids of increasing complexity and diversity. Since the organoids closely resemble mature tissues, it opens up new vistas. These include studying the complex arrangements of cells in three-dimension and their function in detail, and understanding how cells assemble into organs.

Organoids can be used to study the safety and efficacy of new drugs and also test the response of tissues to existing medicines. Organoids will bring precision medicine closer to reality by developing patient-specific treatment strategies by studying which drugs the patient is most sensitive to. Since the use of animals during drug development studies is becoming increasingly difficult, the focus has been on refining, reducing and replacing them. While scientists have been increasingly using human cell lines and other methods, such alternatives have some inherent limitations they cannot mimic the whole organ system. Organoids are a far superior alternative to cell lines.

Organoids offer new opportunities to studying proteins and genes that are critical for the development of an organ. This helps in knowing how a mutation in a specific gene causes a disease or disorder. In a study in Europe using intestinal organoids from six patients with an intestine disorder, it became possible to identify the mutation in a gene that prevented the formation of a healthy intestine. Researchers have used brain organoids to study how the Zika virus affects brain development in the embryo.

Scientists are already using stem cells taken from tumours to grow organoids that are poised to develop cancer. The ability to grow organoids using cancer stem cells allows researchers to study the genes, proteins and signalling pathways that cancer cells use to develop and grow. They are also using healthy organoids to identify and verify the gene mutations that cause cancer.

In an opinion piece in Nature, scientists argued that the largest brain that has been grown in the laboratory is only 4 mm in diameter and contains only 2-3 million cells. In comparison, an adult human brain measures 1,350 cubic centimetres, and has 86 billion neurons and another 86 billion non-neuronal cells and a similar number of non-neuronal cells. The authors argue that organoids do not have sensory inputs and sensory connections from the brain are limited. Isolated regions of the brain cannot communicate with other brain regions or generate motor signals. They wrote: Thus, the possibility of consciousness or other higher-order perceptive properties [such as the ability to feel distress] emerging seems extremely remote.

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Can organoids, derived from stem cells, be used in disease treatments? - The Hindu

University team to seek approval for iPS-based heart treatment trial – The Japan Times

OSAKA A university research team will seek government approval by the end of October to carry out a clinical trial using iPS cells to treat a serious heart condition, Osaka University officials said Wednesday.

The treatment involves transplanting sheets of heart muscle cells, generated from induced pluripotent stem cells that can develop into any type of tissue, to individuals suffering from ischemic heart disease.

The disease is caused by the buildup of plaque in the coronary arteries, which partially or totally blocks blood flow to the heart.

The team, led by Yoshiki Sawa, a professor at Osaka Universitys Department of Cardiovascular Surgery, received approval for a clinical study from the Ministry of Health, Labor and Welfare in May 2018.

But the study was delayed after a powerful earthquake hit western Japan a month later, damaging a research facility where the necessary cells would have been cultivated.

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University team to seek approval for iPS-based heart treatment trial - The Japan Times

Global Gemcitabine Hydrochloride Market: Segmented By Application And Geography Trends, Growth And Forecasts To 2024 – Health News Office

The Induced Pluripotent Stem Cells (IPSCS)market research report added by Report Ocean, is an in-depth analysis of the latest trends, market size, status, upcoming technologies, industry drivers, challenges, regulatory policies, with key company profiles and strategies of players. The research study provides market introduction, INDUCED PLURIPOTENT STEM CELLS (IPSCS) market definition, regional market scope, sales and revenue by region, manufacturing cost analysis, Industrial Chain, market effect factors analysis, INDUCED PLURIPOTENT STEM CELLS (IPSCS) market size forecast, 100+ market data, Tables, Pie Chart, Graphs and Figures, and many more for business intelligence.


INDUCED PLURIPOTENT STEM CELLS (IPSCS) Market: Competitive Analysis by Key Players:

The Global INDUCED PLURIPOTENT STEM CELLS (IPSCS) Market also explains the competitive landscape among the major key players of the market, based on various parameters, such as:


Market Segment by Manufacturers,> Fujifilm Holding Corporation (CDI)> ReproCELL> Astellas Pharma Inc> Ncardia> Cell Inspire Biotechnology> Sumitomo Dainippon Pharma> Pluricell Biotech> Fate Therapeutics, Inc

Market Segment by Type,> Human iPSCs> Mouse iPSCsMarket Segment by Applications,> Academic Research> Drug Development and Discovery> Toxicity Screening> Regenerative Medicine

Key Developments in the Induced Pluripotent Stem Cells (iPSCs) MarketTo describe Induced Pluripotent Stem Cells (iPSCs) Introduction, product type and application, market overview, market analysis by countries, market opportunities, market risk, market driving force;To analyze the manufacturers of Induced Pluripotent Stem Cells (iPSCs), with profile, main business, news, sales, price, revenue and market share in 2016 and 2018;To display the competitive situation among the top manufacturers in Global, with sales, revenue and market share in 2016 and 2018;To show the market by type and application, with sales, price, revenue, market share and growth rate by type and application, from 2013 to 2019;To analyze the key countries by manufacturers, Type and Application, covering North America, Europe, Asia Pacific, Middle-East and South America, with sales, revenue and market share by manufacturers, types and applications;Induced Pluripotent Stem Cells (iPSCs) market forecast, by countries, type and application, with sales, price, revenue and growth rate forecast, from 2019 to 2026;To analyze the manufacturing cost, key raw materials and manufacturing process etc.To analyze the industrial chain, sourcing strategy and downstream end users (buyers);To describe Induced Pluripotent Stem Cells (iPSCs) sales channel, distributors, traders, dealers etc.To describe Induced Pluripotent Stem Cells (iPSCs) Research Findings and Conclusion, Appendix, methodology and data source

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Regional Analysis:

The market research study offers in-depth regional analysis along with the current market scenarios. The major regions analyzed in the study are:

Key highlights and important features of the Report:

Overview and highlights of product and application segments of the global INDUCED PLURIPOTENT STEM CELLS (IPSCS) Market are provided. Highlights of the segmentation study include price, revenue, sales, sales growth rate, and market share by product.

Explore about Sales data of key players of the global INDUCED PLURIPOTENT STEM CELLS (IPSCS) Market as well as some useful information on their business. It talks about the gross margin, price, revenue, products, and their specifications, type, applications, competitors, manufacturing base, and the main business of key players operating in the INDUCED PLURIPOTENT STEM CELLS (IPSCS)Market.

Explore about gross margin, sales, revenue, production, market share, CAGR, and market size by region.

Describe INDUCED PLURIPOTENT STEM CELLS (IPSCS) Market Findings and Conclusion, Appendix, methodology and data source;

Research Methodology:

The market research was done by adopting various tools under the category of primary and secondary research. For primary research, experts and major sources of information have been interviewed from suppliers side and industries, to obtain and verify the data related to the study of the Global INDUCED PLURIPOTENT STEM CELLS (IPSCS)Market. In secondary research methodology, various secondary sources were referred to collect and identify extensive piece of information, such as paid databases, directories and annual reports and databases for commercial study of the GlobalINDUCED PLURIPOTENT STEM CELLS (IPSCS) Market. Moreover, other secondary sources include studying technical papers, news releases, government websites, product literatures, white papers, and other literatures to research the market in detail.

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Global Gemcitabine Hydrochloride Market: Segmented By Application And Geography Trends, Growth And Forecasts To 2024 - Health News Office

Global consortium formed to combat unproven cell banking services – Drug Target Review

The International Society for Cell and Gene Therapy has created a group to tackle the rising number of commercial cell bank services that are misleading patients.

The International Society for Cell and Gene Therapy (ISCT) has announced the formation of a global consortium to combat the growing number of unproven commercial cell banking services.

The group will be made up of leading professional and education societies, including, among others:

The partnership has been formed following the ISCTs publication of its patient advice and concern on unproven T cell preservation services. These facilities comprise the banking of T cells, dental cells and cells for the derivation of induced pluripotent stem cells for potential therapeutic uses.

these cell banking services can deceive patients using tokens of scientific legitimacy

A joint statement from the ISCT and the consortium partners commented on certain commercial cell banking services and their lack of support from current scientific evidence. Furthermore, the society says that these cell banking services are unable to declare that cells they preserve may ever be appropriate for clinical usage or for manufacturing purposes.

The ISCT emphasises that there is no clear pathway to legitimate clinical use. As such, any parties offering these services commercially to patients is premature, misleading and drives false hope.

Any patients using these services are therefore prevented from giving full and valid informed consent, according to the ISCT.

The society highlights that these cell banking services can deceive patients using tokens of scientific legitimacy that suggest a stronger scientific basis than currently exists. These include endorsements from individuals or scientific advisory boards that may not fully support the specific products, links to scientific articles and references to ongoing clinical trials.

ISCTs raison detre is to lead the industry in supporting scientifically validated cell and gene therapies. As a result, ISCT will continue to welcome all innovations, including cell banking approaches, that increase the number of patients who can benefit from these therapies, said Bruce Levine,President-Elect, ISCT.However, ISCT also leads industry action on unproven cell therapies and services in the cell and gene sector.

This is why ISCT has forged a consortium throughout the industry against the marketing of speculative cell banking services that do not have appropriate pre-clinical and clinical evidence and a plausible pathway to the clinical use of banked cells. We collectively believe these banks have the potential to be detrimental to the future development of cell and gene therapies.

See the article here:
Global consortium formed to combat unproven cell banking services - Drug Target Review