Category Archives: Induced Pluripotent Stem Cells

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

Due to the evolving needs of the research community, the demand for specialized cell lines have increased to a certain point where most vendors offering these products cannot depend solely on sales from catalog products.

The quality of the products and lead time can determine the choices while requesting custom solutions at the same time. Companies usually focus on establishing a strong distribution network for enabling products to reach customers from the manufacturing units in a short time period.

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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.

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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

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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.

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

Bloomberg Philanthropies, Johns Hopkins University School of Medicine, and The New York Stem Cell Foundation Research Institute Announce an…

NEW YORK, Oct. 22, 2019 /PRNewswire/ -- Bloomberg Philanthropies, Johns Hopkins University School of Medicine (JHUSOM), and The New York Stem Cell Foundation (NYSCF) Research Institute today announced an initiative to fundamentally advance and expand the science of precision medicine, in which diagnostic disease markers are defined with pinpoint accuracy to help researchers understand disease pathways and customize therapeutic approaches. The collaboration will combine the renowned clinical and medical expertise of Johns Hopkins with the unique stem cell technologies and research capabilities of the NYSCF Research Institute to accelerate Hopkins' pioneering Precision Medicine Initiatives.

"Johns Hopkins is working intensively to realize the great promise of precision medicine for all those in our care, locally and globally," said Johns Hopkins President Ronald J. Daniels. "This significant new collaboration with Bloomberg Philanthropies and NYSCF moves us ever closer to that aim as we join together our far-reaching research capacities to advance knowledge and deliver better health outcomes for populations and people around the world."

This collaboration will also establish an unprecedented cache of human disease models available to researchers worldwide thus promoting the real world application of precision medicine and driving a new paradigm for understanding and improving the approach to human disease.

"Bloomberg Philanthropies' mission is to ensure better, longer lives for the greatest number of people," said Michael R. Bloomberg, founder of Bloomberg LP and Bloomberg Philanthropies. "For years, Johns Hopkins University and the New York Stem Cell Foundation have shared that mission and we're honored to deepen our partnerships with them as they explore new, innovative ways to save lives through the application of precision medicine."

Diseases manifest themselves differently in different patients. To understand the basis of these differences and to tailor treatments for specific patients, researchers need more accurate biological tools. Stem cell models provide a "biological avatar" of the patient from which they were created, allowing scientists and clinicians to better understand, define, and account for differences in individual patients and groups of patients.

The new initiative will use induced pluripotent stem cells to study disease characteristics in subgroups of patients, identifying markers that lead to varying disease manifestations. For example, by examining stem cells from seemingly similar patients with different forms of multiple sclerosis, we may be able to better understand the full range of disease mechanisms and pathways.

The Johns Hopkins Precision Medicine Initiative already includes 16 Precision Medicine Centers of Excellence (PMCOE), each focusing on a specific disease, and is now working to develop 50 Precision Medicine Centers in the next five years. Johns Hopkins believes that this advancement in the study and application of precision medicine has the potential to transform the diagnosis and management of many diseases.Often, what is now categorized as a single disease is actually made up ofmultiple diseases that display similar symptoms, but require quite different therapies. Using a wide range of data sources, precision medicine seeks to better elucidate these differences, so that doctors can treat patients with precisely targeted therapies. At Johns Hopkins, dozens of researchers are bringing this idea to reality across a spectrum of debilitating and life-altering diseases.

In this collaboration, the process will begin with the full consent of patients in JHUSOM PMCOEs who wish to participate. Biological samples from the JHUSOM PMCOEs will be collected by the NYSCF Research Institute where scientists will create stem cell models of disease using the NYSCF Global Stem Cell Array, the world's first end-to-end automated system for generating human stem cells in a parallel, highly controlled process.Integrating robotics and machine learning, NYSCF's technology reprograms skin or blood cells into stem cells, differentiates them into disease-relevant cell types, and performs genome editing to unravel the genetic basis of disease.

"The NYSCF Research Institute has invented and scaled the most advanced methods of human cell manipulation, which is critical for studying disease at the level of the individual patient," explained NYSCF CEO Susan L. Solomon. "By combining our capabilities with Johns Hopkins' extensive clinical data and expertise, we will be able to develop effective, personalized therapies for patients suffering from diseases with a high unmet need."

The stem cells generated by NYSCF will be used to research and drive effective therapeutic and diagnostic development in a wide range of diseases that include, but are not limited to, Multiple Sclerosis, Alzheimer's, chronic renal failure, and cancers of the lung, breast, prostate, pancreas, and bladder. These stem cell lines will reside in the NYSCF Repository and serve as an extraordinary resource in perpetuity for the disease research community. This vast collection will allow scientists unprecedented insights into the biochemical and genetic mechanisms underlying different diseases and subtypes thereof, thereby illuminating avenues for effective, tailored interventions.

"Stem cell science holds enormous potential for the treatment of a wide range of diseases," said Paul B. Rothman, dean of the School of Medicine and CEO of Johns Hopkins Medicine. "By combining this approach with Johns Hopkins' groundbreaking work on precision medicine, we are creating a scientific powerhouse that will help us advance medicine and science at an even faster pace. I am excited to see the discoveries and innovations that will be produced by this collaboration."

About Bloomberg PhilanthropiesBloomberg Philanthropies invests in 510 cities and 129 countries around the world to ensure better, longer lives for the greatest number of people. The organization focuses on five key areas for creating lasting change: Arts, Education, Environment, Government Innovation, and Public Health. Bloomberg Philanthropies encompasses all of Michael R. Bloomberg's giving, including his foundation and personal philanthropy as well as Bloomberg Associates, a pro bono consultancy that works in cities around the world. In 2018, Bloomberg Philanthropies distributed $767 million. For more information, please visitbloomberg.orgor follow us on Facebook, Instagram, YouTube, and Twitter.

About The New York Stem Cell Foundation Research Institute The New York Stem Cell Foundation (NYSCF) Research Institute is an independent non-profit organization accelerating cures and better treatments for patients through stem cell research. The NYSCF global community includes over 180 researchers at leading institutions worldwide, including the NYSCF Druckenmiller Fellows, the NYSCF Robertson Investigators, the NYSCF Robertson Stem Cell Prize Recipients, and NYSCF Research Institute scientists and engineers. The NYSCF Research Institute is an acknowledged world leader in stem cell research and in developing pioneering stem cell technologies, including the NYSCF Global Stem Cell Array and in manufacturing stem cells for scientists around the globe. NYSCF focuses on translational research in an accelerator model designed to overcome barriers that slow discovery and replace silos with collaboration. For more information, visit http://www.nyscf.org or follow us on Twitter, Facebook, and Instagram.

Press Contacts:

The New York Stem Cell Foundation Research Institute David McKeon dmckeon@nyscf.org 212-365-7440

Johns Hopkins University School of Medicine Vanessa Wasta wasta@jhmi.edu

SOURCE The New York Stem Cell Foundation

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Bloomberg Philanthropies, Johns Hopkins University School of Medicine, and The New York Stem Cell Foundation Research Institute Announce an...

ISCT forms cell and gene therapy sector-wide coalition to combat the rise of unproven commercial cell banking services – PharmiWeb.com

Vancouver, Canada, October 21, 2019 ISCT, the International Society for Cell and Gene Therapy, the global professional society of clinicians, researchers, regulatory specialists, technologists and industry partners in the cell and gene therapy sector, today announces it has formed a global consortium of a wide range of leading professional and education societies to combat the rise in the number of unproven commercial cell banking services. Full details of the statement can be foundhere.

The consortium partners include the International Society for Stem Cell Research (ISSCR), Society for Immunotherapy of Cancer (SITC), American Society for Transplantation and Cellular Therapy (ASTCT),American Society of Gene & Cell Therapy (ASGCT), European Society for Blood and Marrow Transplantation (EBMT), Foundation for the Accreditation of Cellular Therapy (FACT), Joint Accreditation Committee ISCT-EBMT (JACIE) and the Forum for Innovative Regenerative Medicine (FIRM).

The consortium has been formed following ISCT issuingpatient advice and concern on unproven T-cell preservation services on August 7, 2019. These services include the banking of T-cells, dental cells and cells for the derivation of induced pluripotent stem cells for potential therapeutic uses.

The joint statement from ISCT and the consortium partners includes an agreement on a number of key points. Commercial cell banking services are not supported by current scientific evidence, as opposed to the range of cell therapies such as CAR-T therapies, that follow established approval processes. Additionally, cell banking services cannot claim to know that the cells they preserve today could ever be appropriate for clinical use, could be used by manufacturers, or meet the requirements of many national and international regulatory agencies. As a result, there is no clear pathway to legitimate clinical use. All parties agree offering these services commercially to patients is thus premature, misleading, and drives false hope.

In addition, the ISCT joint statement makes clear that patients, being misled by these services, are thus prevented from giving a full and valid informed consent. Cell banking companies mislead patients in a number of ways, including using tokens of scientific legitimacy that suggest a stronger scientific basis than currently exists. These tokens include endorsements from individuals or scientific advisory boards that might not fully endorse 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 and one of the inventors of CAR-T therapies.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.

About ISCT

Established in 1992, ISCT, the International Society for Cell and Gene Therapy is a global society of clinicians, regulators, researchers, technologists and industry partners with a shared vision to translate cellular therapy into safe and effective therapies to improve patients lives worldwide.

ISCT is the global leader focused on pre-clinical and translational aspects of developing cell-based therapeutics, thereby advancing scientific research into innovative treatments for patients. ISCT offers a unique collaborative environment that addresses three key areas of translation: Academia, Regulatory and Commercialization. Through strong relationships with global regulatory agencies, academic institutions and industry partners, ISCT drives the advancement of research into standard of care.

Comprised of over 1,500 cell therapy experts across five geographic regions and representation from over 50 countries, ISCT members are part of a global community of peers, thought leaders and organizations invested in cell therapy translation. For more information about the society, key initiatives and upcoming meetings, please visit:

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ISCT forms cell and gene therapy sector-wide coalition to combat the rise of unproven commercial cell banking services - PharmiWeb.com

Mutations Linked to Huntington’s Increase Cells’ Resistance to Manganese, Study Finds – Huntington’s Disease News

Mutations associated with Huntingtons disease increase nerve cells resistance to high levels of manganese, according to a recent study.

The results of the study, Huntingtons disease associated resistance to Mn neurotoxicity is neurodevelopmental stage and neuronal lineage dependent, were published in NeuroToxicology.

Manganese (Mn) is a trace metal that plays a key role in many cellular processes. It is essential in the production of neurotransmitters chemical substances that allow communication between nerve cells and in the regulation of nerve cells metabolism. However, high levels of Mn in the body are associated with neurotoxicity.

Levels of Mn change substantially in different regions of the brain throughout its development. However, it is still unclear if these regional differences could be linked to the fact that certain types of nerve cells may be more sensitive to higher levels of Mn than others at specific time-points during brain development.

Certain neurological disorders have been associated with alterations in brain Mn levels. It has been shown that human and mouse nerve cell precursors containing a genetic mutation associated with Huntingtons disease have limited access to Mn and are more resilient to its neurotoxic effects.

Investigators from Vanderbilt University and their collaborators now set out to explore the sensitivity of different types of neurons at different developmental stages, from patients with Huntingtons disease and healthy individuals (controls), to Mn neurotoxicity.

We hypothesized that there would be differences in Mn sensitivity between lineages and developmental stages, the researchers said.

The team used several lines of human-induced pluripotent stem cells (hiPSCs) fully matured cells that can be reprogrammed back to a stem cell state, where they are able to grow into almost any type of cell from patients and controls to generate neuroprogenitor cells (NPCs).

The NPCs then were cultured in a lab dish with different cocktails of growth factors to differentiate them into distinct types of neurons. Specifically, there were three different types: striatal neurons, which can be found in the striatum, a brain region involved in motor control; cortical neurons, which can be found in the cortex, or the outer layer of the brain; and midbrain dopaminergic neurons, which can be found in the substantia nigra, a brain region involved in the control of voluntary muscle movements.

The researchers then compared sensitivity to Mn neurotoxicity during each developmental time-point for each cell type between the two groups those with and without Huntingtons.

Their findings revealed that striatal and cortical NPCs derived from Huntingtons patients were more resistant to high levels of Mn compared with those that had been obtained from individuals who did not have the disease. These results were similar to those seen in other studies.

Moreover, the investigators found that patient-derived hiPSCs were themselves more resistant to Mn neurotoxicity than their counterparts.

However, at intermediate stages of development, midbrain neurons that had been derived from patients became more sensitive to the toxic effects of Mn.

The researchers said the sensitivity of midbrain NPCs and mature cortical neurons to Mn neurotoxicity was similar in both groups.

Altogether, these findings suggest that the harmful effects of Mn can be influenced by the presence of genetic mutations associated with Huntingtons disease. That, in turn, depends on the particular developmental stages and neuronal cell types.

In conclusion, our findings may provide insight into therapeutic strategies for diseases in which Mn has been shown to play a role such as HD [Huntingtons disease], especially through specific lineage-targeted interventions, the researchers said.

Joana is currently completing her PhD in Biomedicine and Clinical Research at Universidade de Lisboa. She also holds a BSc in Biology and an MSc in Evolutionary and Developmental Biology from Universidade de Lisboa. Her work has been focused on the impact of non-canonical Wnt signaling in the collective behavior of endothelial cells cells that make up the lining of blood vessels found in the umbilical cord of newborns.

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Ana holds a PhD in Immunology from the University of Lisbon and worked as a postdoctoral researcher at Instituto de Medicina Molecular (iMM) in Lisbon, Portugal. She graduated with a BSc in Genetics from the University of Newcastle and received a Masters in Biomolecular Archaeology from the University of Manchester, England. After leaving the lab to pursue a career in Science Communication, she served as the Director of Science Communication at iMM.

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Mutations Linked to Huntington's Increase Cells' Resistance to Manganese, Study Finds - Huntington's Disease News

Stem Cells Market : Insights Into the Competitive Scenario of the Market – Online News Guru

In theglobal stem cells marketa sizeable proportion of companies are trying to garner investments from organizations based overseas. This is one of the strategies leveraged by them to grow their market share. Further, they are also forging partnerships with pharmaceutical organizations to up revenues.

In addition, companies in the global stem cells market are pouring money into expansion through multidisciplinary and multi-sector collaboration for large scale production of high quality pluripotent and differentiated cells. The market, at present, is characterized by a diverse product portfolio, which is expected to up competition, and eventually growth in the market.

Some of the key players operating in the global stem cells market are STEMCELL Technologies Inc., Astellas Pharma Inc., Cellular Engineering Technologies Inc., BioTime Inc., Takara Bio Inc., U.S. Stem Cell, Inc., BrainStorm Cell Therapeutics Inc., Cytori Therapeutics, Inc., Osiris Therapeutics, Inc., and Caladrius Biosciences, Inc.

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As per a report by Transparency Market Research, the global market for stem cells is expected to register a healthy CAGR of 13.8% during the period from 2017 to 2025 to become worth US$270.5 bn by 2025.

Depending upon the type of products, the global stem cell market can be divided into adult stem cells, human embryonic stem cells, induced pluripotent stem cells, etc. Of them, the segment of adult stem cells accounts for a leading share in the market. This is because of their ability to generate trillions of specialized cells which may lower the risks of rejection and repair tissue damage.

Depending upon geography, the key segments of the global stem cells market are North America, Latin America, Europe, Asia Pacific, and the Middle East and Africa. At present, North America dominates the market because of the substantial investments in the field, impressive economic growth, rising instances of target chronic diseases, and technological progress. As per the TMR report, the market in North America will likely retain its dominant share in the near future to become worth US$167.33 bn by 2025.

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Investments in Research Drives Market

Constant thrust on research to broaden the utility scope of associated products is at the forefront of driving growth in the global stem cells market. Such research projects have generated various possibilities of different clinical applications of these cells, to usher in new treatments for diseases.Since cellular therapies are considered the next major step in transforming healthcare, companies are expanding their cellular therapy portfolio to include a range of ailments such as Parkinsons disease, type 1 diabetes, spinal cord injury, Alzheimers disease, etc.

The growing prevalence of chronic diseases and increasing investments of pharmaceutical and biopharmaceutical companies in stem cell research are the key driving factors for the stem cells therapeutics market. The growing number of stem cell donors, improved stem cell banking facilities, and increasing research and development are other crucial factors serving to propel the market, explains the lead analyst of the report.

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Stem Cells Market : Insights Into the Competitive Scenario of the Market - Online News Guru