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Buyer beware of this $1 million gene therapy for aging – MIT Technology Review

By daniellenierenberg

Its said that nothing is certain except death and taxes. But doubt has been cast over the former since the 1970s, when scientists picked at the seams of one of the fundamental mysteries of biology: the molecular reasons we get old and die.

The loose thread they pulled had to do with telomeresmolecular timepieces on the ends of chromosomes that shorten each time a cell divides, in effect giving it a fixed life span. Some tissues (such as the gut lining) renew almost constantly, and it was found that these have high levels of an enzyme called telomerase, which works to rebuild and extend the telomeres so cells can keep dividing.

That was enough to win Elizabeth Blackburn, Carol Greider, and Jack Szostak a Nobel Prize in 2009. The obvious question, then, was whether telomerase could protect any cell from agingand maybe extend the life of entire organisms, too.

While telomere-extending treatments in mice have yielded intriguing results, nobody has demonstrated that tweaking the molecular clocks has benefits for humans. That isnt stopping one US startup from advertising a telomere-boosting genetic therapyat a price.

Libella Gene Therapeutics, based in Manhattan, Kansas, claims it is now offering a gene therapy to repair telomeres at a clinic in Colombia for $1 million a dose. The company announced on November 21 that it was recruiting patients into what it termed a pay-to-play clinical trial.

Buyer beware, though: this trial is for an unproven, untested treatment that might even be harmful to your health.

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The company proposes to inject patients with viruses carrying the genetic instructions cells need to manufacture telomerase reverse transcriptase, a molecule involved in extending the length of telomeres.

The dangers are enormous, says Jerry Shay, a world expert on aging and cancer at the University of Texas Southwestern Medical Center. Theres a risk of activating a pre-cancerous cell thats got all the alterations except telomerase, especially in people 65 and over.

For years now, people involved in the company have made shifting claims about the study, raising uncertainty about who is involved, when it might start, and even where it would occur. Trial listings posted in October to currently show plans for three linked experiments, each with five patients, targeting critical limb ischemia, Alzheimers, and aging, respectively.

Jeff Mathis, president of Libella, told MIT Technology Review that two patients have already paid the enormous fee to take part in the study: a 90-year-old-woman and a 79-year-old man, both US citizens. He said they could receive the gene therapy by the second week of January 2020.

The decision to charge patients a fortune to participate in the study of an experimental treatment is a red flag, say ethics experts. Whats the moral justification for charging individuals with Alzheimers? asks Leigh Turner, at the University of Minnesotas Center for Bioethics. Why charge those bearing all the risk?

The telomere study is occurring outside the US because it has not been approved by the Food and Drug Administration. Details posted to indicate that the injections would be carried out at the IPS Arcasalud SAS medical clinic in Zipaquir, Colombia, 40 kilometers (25 miles) north of Bogot.

It takes a lot longer, is a lot more expensive, to get anything done in the US in a timely fashion, Mathis says of Libellas choice to go offshore.

To some promoters of anti-aging cures, urgency is justified. Heres the ethical dilemma: Do you run fast and run the risk of low credibility, or move slowly and have more credibility and global acceptancebut meanwhile people have died? says Mike Fossel, the president of Telocyte, a company planning to run a study of telomerase gene therapy in the US if it can win FDA signoff.

Our reporting revealed a number of unanswered questions about the trial. According to the listings, the principal investigatorwhich is to say the doctor in charge--is Jorge Ulloa, a vascular surgeon rather than an expert in gene transfer. I dont see someone with relevant scientific expertise, says Turner.

Furthermore, Bill Andrews, who is listed as Libellas chief scientific officer, says he does not know who Ulloa is, even though on Libellas website, the mens photos appear together on the list of team members. He said he believed that different doctors were leading the trial.

Turner also expressed concerns about the proposed 10-day observation period described in the posting for the overseas study: If someone pays, shows up, has treatment, and doesnt stick around very long, how are follow-up questions taking place? Where are they taking place?

Companies seeking to try the telomere approach often point to the work of Maria Blasco, a Spanish scientist who reported that telomere-lengthening gene therapy benefited mice and did not cause cancer. Blasco, director of the Spanish National Centre for Cancer Research, says she believes many more studies should be done before trying such a gene experiment on a person.

This isnt the first time Libella has announced that its trial would begin imminently. It claimed in late 2017 that human trials of the telomerase therapy would begin in the next few weeks. In 2016, Andrews (then partnered with biotech startup BioViva) claimed that construction of an age reversal clinic on the island nation of Fiji would be complete before the end of the year. Neither came to pass.

Similar questions surround Libellas most recent claims that it has two paying clients. Pedro Fabian Davalos Berdugo, manager of Arcasalud, said three patients were awaiting treatment in December. But Bioaccess, a Colombian contract research organization facilitating the Libella trial, said that no patients had yet been enrolled.

Also unclear is where Libella is obtaining the viruses needed for the treatment. Virovek, a California biotech company identified by several sources as Libellas manufacturer, did not answer questions about whether any treatment had been produced.

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Global Cell Therapy Processing Market Growth, Demand, Industry Verticals, and Forecast upto 2022 – News Description

By daniellenierenberg

TheCell Therapy Processing marketreport [6 Year Forecast 2016-2022] focuses on Major Leading Industry Players, providing info likeCell Therapy Processing product scope, market overview, market opportunities, market driving force and market risks.Profile the top manufacturers of Cell Therapy Processing, with sales, revenue and globalmarket share ofCell Therapy Processingare analyzed emphatically bylandscape contrastandspeak to info.Upstream raw materials and instrumentation and downstream demand analysis is additionally administrated. The Cell Therapy Processing marketbusiness development trends and selling channelssquare measure analyzed. From a global perspective, It also represents overall Cell Therapy Processing industry size by analyzingqualitative insights and historical data.



There are numerous indications that can be cured using cell therapies, and with increased R&D activities for cell therapies, the number of therapeutic uses is anticipated to increase in the near future. Some of the indications under investigation for the treatment using cell therapy are cerebral disorders such as Parkinsons disease and Alzheimers disease, and also cardiovascular disease. Cardiovascular disease could be treated using cell therapies with the aim to restore normal heart functions. Moreover, many studies are undergoing in the attempt to improve the safety and efficacy in treatment of different malignancies. Cell therapy could also be used to cure metabolic disorder such as diabetes mellitus type 1 where there is lack of insulin production in the patient. Researchers are also trying to restore normal liver and kidney function by introducing modified cells of respective origins. Presently, cell therapy could be a promising technique for the treatment of numerous conditions such as orthopedic, oncology, neurological and variety of autoimmune diseases. The increase in the potential of cell therapies in the treatment of diseases associated with lungs using stem cell therapies is anticipated to drive the markets growth in the near future. In addition, improved understanding of the role of stem cells in inducing development of functional lung cells from both embryonic stem cells (ESCs) and induced pluripotent stem (iPS) cells offers lucrative opportunities for the cell therapy processing markets growth. The rising significance of stem cell therapies provides further understanding of lung biology and repair after lung injury, and further a sound scientific basis for therapeutic use of cell therapies and bioengineering approaches in the treatment of lung diseases.

Report Scope:

This research report presents an in-depth analysis of the global cell therapy processing market by offering type, application and geographic regional markets. The report includes key inhibitors that affect various factors that help in growth of cell therapy processing. The report discusses the role of supply chain members from manufacturers to researchers. The report analyzes key companies operating in the global cell therapy processing market. In-depth patent analysis in the report will provide extensive technological trends across years and regions such as North America, Europe, Asia-Pacific and ROW.

The cell therapy processing market is mainly segmented into three major components: offering type, application and region. Based on offering type, the market is segmented into products (cell lines, instruments, among others), services (product design, process design, among others) and software (enabling software). Based on application, the market is categorized into cardiovascular diseases, bone repair, neurological disorders, skeletal muscle repair, cancer and others. The market is segmented by region into North America, Europe, Asia-Pacific and the ROW.

The cell therapy processing market is mainly segmented into three major components: offering type, application and region. Based on offering type, the market is segmented into products (cell lines, instruments, among others), services (product design, process design, among others) and software (enabling software). Based on application, the market is categorized into cardiovascular diseases, bone repair, neurological disorders, skeletal muscle repair, cancer and others. The market is segmented by region into North America, Europe, Asia-Pacific and the ROW.

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Report Includes:

40 data tables and 25 additional tables

An overview of the global market for cell therapy processing technologies

Analyses of global market trends, with data from 2016 and 2017, and projections of compound annual growth rates (CAGRs) through 2022

Analysis of the market by technology, application, and region

An outline of the present state of applications of rainwater harvesting

Descriptions of trends in price and price-performance and other factors, including demand in the market

Profiles of key companies in the market, including Biotime Inc., Cell Design Labs., Flodesign Sonics, Lonza Group Ltd. and Sanbio Co. Ltd.

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ResearchMozMr. Nachiket Ghumare,Tel: +1-518-621-2074Toll Free: 866-997-4948 (US-CANADA)Email:[emailprotected]Follow us on LinkedIn @

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Risk of taking expired viagra – Viagra and red bull safe – Buy viagra online reddit – Laughlin Entertainer

By daniellenierenberg

Risk of taking expired viagra - Viagra and red bull safe - Buy viagra online reddit  Laughlin Entertainer

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Stem Cells Market 2019 Global Growth Analysis and Forecast Report by 2025 – Markets Gazette 24

By daniellenierenberg

New York, November 26, 2019: The global stem cells market is expected to grow at an incredible CAGR of 25.5% from 2018to 2024and reach a market value of US$ 467 billion by 2024. The emergence of Induced Pluripotent Stem (iPS) cells as an alternative to ESCs (embryonic stem cells), growth of developing markets, and evolution of new stem cell therapies represent promising growth opportunities for leading players in this sector.

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Due to the increased funding from Government and Private sector and rising global awareness about stem cell therapies and research are the main factors which are driving this market. A surge in therapeutic research activities funded by governments across the world has immensely propelled the global stem cells market. However, the high cost of stem cell treatment and stringent government regulations against the harvesting of stem cells are expected to restrain the growth of the global stem cells market.

This report will definitely help you make well informed decisions related to the stem cell market. The stem cell therapy market includes large number of players that are involved in development of stem cell therapies of the treatment of various diseases. Mesoblast Ltd. (Australia), Aastrom Biosciences, Inc. (U.S.), Celgene Corporation (U.S.), and StemCells, Inc. (U.S.) are the key players involved in the development of stem cell therapies across the globe.

This market research report categorizes the stem cell therapy market into the following segments and sub-segments:

The Global Stem Cell Market this market is segmented on the basis of Mode of Therapy, Therapeutic Applications and Geography.

By Mode of Therapy this market is segmented on the basis of Allogeneic Stem Cell Therapy Market and Autologous Stem Cell Therapy Market. Allogeneic Stem Cell Therapy Market this market is segmented on the basis of CVS Diseases, CNS Diseases, GIT diseases, Eye Diseases, Musculoskeletal Disorders, Metabolic Diseases, Immune System Diseases, Wounds and Injuries and Others. Autologous Stem Cell Therapy Market this market is segmented on the basis of GIT Diseases, Musculoskeletal Disorders, CVS Diseases, CNS Diseases, Wounds and Injuries and Others. By Therapeutic Applications this market is segmented on the basis of Musculoskeletal Disorders, Metabolic Diseases, Immune System Diseases, GIT Diseases, Eye Diseases, CVS Diseases, CNS Diseases, Wounds and Injuries and Others.

By Regional Analysis this market is segmented on the basis of North America, Europe, Asia-Pacific and Rest of the World.

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Table of Contents


2 Research Methodology

2.1 Research Data2.1.1 Secondary Data2.1.1.1 Key Data From Secondary Sources2.1.2 Primary Data2.1.2.1 Key Data From Primary Sources2.1.2.2 Breakdown of Primaries2.2 Market Size Estimation2.2.1 Bottom-Up Approach2.2.2 Top-Down Approach2.3 Market Breakdown and Data Triangulation2.4 Research Assumptions

3 Executive Summary

4 Premium Insights

5 Market Overview

6 Industry Insights

7 Global Stem Cell Therapy Market, By Type

8 Global Stem Cell Therapy Market, By Therapeutic Application

9 Global Stem Cell Therapy Market, By Cell Source

10 Stem Cell Therapy Market, By Region

11 Competitive Landscape

12 Company Profiles

12.1 Introduction

12.1.1 Geographic Benchmarking

12.2 Osiris Therapeutics, Inc.

12.3 Medipost Co., Ltd.

12.4 Anterogen Co., Ltd.

12.5 Pharmicell Co., Ltd.

12.6 Holostem Terapie Avanzate Srl

12.7 JCR Pharmaceuticals Co., Ltd.

12.8 Nuvasive, Inc.

12.9 RTI Surgical, Inc.

12.10 Allosource

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Takeda sees cell, gene therapy in its future. Is it too late? – BioPharma Dive

By daniellenierenberg

Thanks to a $62 billion acquisition of Shire, Takeda is one of the world's largest developers of rare disease drugs.

Despite that, the 238-year-old Japanese pharmaceutical company lacks any mid- or late-stage cell or gene therapies, two technologies that figure to play a large role in how many rare cancers and inherited diseases will eventually be treated.

It's a mismatch Takedais putting substantial effort into addressing. Last week, executives made cell and gene therapy a notable focus of the company's first R&D day since closing its Shire deal.

"We have a world-class gene therapy platform," Dan Curran, head of Takeda's rare disease therapeutic area unit, told investors and Wall Street analysts gathered in New York city.

"We intend to build on that over the next five years. Because as we look to lead in the second half of [next]decade, we believe patients will demand and we can deliver transformative and curative therapies to patients globally."

But right now that's just an ambition. While Takedahas begun to explore how it can improve on current gene therapies, its candidates are early stage and lag their would-be competitors.

"Our heme A program we're behind. Our heme B program we're behind," admitted Curran in an interview. "But we're behind the first generation and when has there only been one generation of anything?"

Takeda's hemophilia A program is currently in Phase 1, with the hemophilia B candidate about to join it in human testing well back from leaders BioMarin Pharmaceutical, Spark Therapeutics and SangamoTherapeutics in hemophilia A and UniQure in hemophilia B.

Curran laid out three priorities for Takeda'spush: exploring whether gene therapy, typically pitched as a one-time treatment, can be re-dosed; lowering the doses currently used for first-generation therapies; and developing alternative gene delivery vehicles than the adeno-associatedand lentiviralvectors that are predominant today.

"We need to figure out how to re-dose AAVvectors if we want to provide functional cures for patients for the rest of their lives."

How long a gene therapy's benefit lasts is a critical question. In theory, it could last decades or potentially for life, depending on the treatment's target.

But clinical evidence presented to date suggests that benefit for some therapies could wane over time. BioMarin, for example, presented data this year that it argued is proof its gene therapy could raise Factor VIII expression levels in patients with hemophilia A above the threshold for mild disease for at least eight years a long time, to be sure, but not life-long.

Still, it's an unusual objective. Much of gene therapy's promise lies in the potential for it to be given just once and still deliver lasting benefits. And the therapies that have reached market most notably Spark Therapeutics' Luxturna, Novartis' Zolgensma and Bluebird bio's Zynteglo are among the most expensive drugs to ever reach market. Were a gene therapy to be re-dosed, the current value proposition those drugmakers describe would need to be re-evaluated.

Curran recognizes that bringing down costs substantially will be essential to any attempt to advance a multi-use gene therapy. But Takeda might have an advantage. In buying Shire, the pharma inherited a viral vector manufacturing plant, originally built by Baxalta, that Curran calls the company's "best kept secret."

"It's an enormous competitive advantage," he said, adding that Takeda believes it's among the industry's top three facilities by production capacity. "Roche trying to acquire Spark, Novartis and AveXis a significant component of value of those transactions was that these companies had actually invested in manufacturing capabilities."

Curran emphasized that Takeda's ambitions in gene therapy will require it to partner with academic leaders in the field, a playbook that it's followed over the past three years as it's worked to expand into cell therapy.

"In the cell space, there's more innovation you can bring up into proof of principle milestones in academia," said Andy Plump,Takeda'shead of R&D, in an interview.

"An academic can manipulate a cell, but it's very hard in an academic setting to optimize a small molecule," he added. "This is a space where Novartis, and now we, have been quite successful in creating those relationships."

Takeda has put partnerships in place with Japan's Center for iPS Cell Research and Application, GammaDelta, Noile-Immune Biotech, Memorial Sloan Kettering Cancer Center and, just this month, The University of Texas MD Anderson Cancer Center.

That last collaboration gives Takeda access to a chimeric antigen receptor-directed natural killer, or NK, cell therapy.The drugmaker believes NK cells could offer advantages over the T cells modified to create the currently available cell therapies Kymriah and Yescarta.

Most notably, MD Anderson's approach uses NK cells isolated from umbilical cord blood, rather than extracting T cells from each individual patient a time-consuming and expensive process that has complicated the market launch of Kymriah and Yescarta. Cord blood-derived NK cells are designed to be allogeneic, or administered "off the shelf."

Additionally, CAR NK cells haven't been associated (yet) with cytokine release syndrome or neurotoxicity, two significant side effects often associated with CAR-T cell therapies. That could help Takeda position its cell therapies as an outpatient option.

"Even if we were a company that entered a little bit later into the immuno-oncology space, we've very much tried to turn this into an advantage," said Chris Arendt, head of Takeda's oncology drug discovery unit, at the company's event.

"We believe we have a chance to establish a leadership position rather than jumping on the bandwagon and being a follower."

While Takeda's choice to pursue NK cell therapy stands out, its choice of target does not. TAK-007, a drug candidate from MD Anderson that is now Takeda's lead cell therapy program, is aimed at a cell surface protein called CD19 that's found in leukemias and lymphomas.

Both Yescarta and Kymriah target CD19, and a recent count by the Cancer Research Institute tracked 181 cell therapy projects aimed at the antigen.

Takeda is planning to advance TAK-007 into pivotal studies in two types of lymphoma and chronic lymphocytic leukemia by 2021, with a potential filing for approval in 2023.

By then, Kymriah and Yescarta will have been on the market for six years and current bottlenecks in cell therapy treatment could be solved, helping both Takeda's potential entry as well as the host of competitors it will likely face.

Next year will be a test of how productive Takeda'scell therapy unit can be. In addition to TAK-007, the pharmaexpects to have four other CAR-T and gamma delta cell therapies in the clinic, two of which will target solid tumors.

Cell and gene therapy are part of what Takeda calls its "second wave" of R&D projects, a group of early-stage drugs and programs that it sees as progressing to regulatory stages by 2025 or later.

In the nearer term, the drugmakeris advancing a "first wave" of clinical candidates that it told investors will deliver 14 new molecular entities by 2024. Five of those will come in rare disease, with the others spread across oncology, neuroscience, gastro-enterology and vaccines.

"We think the cascade of news coming forward on these programs will transform how people view Takeda," Curran said.

More importantly to the investors gathered in New York, Takeda expects these experimental drugs will eventually earn $10 billion in peak annual sales, which would represent a sizable addition to a business that generated $30 billion in sales last year.

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Fujifilm adding $120M facility to its gene therapy operations in U.S. – FiercePharma

By daniellenierenberg

Continuing its expansion efforts, Japans Fujifilmwill make a major investment in its U.S. gene therapy operation with plans for a $120 million addition to its facilities in Texas.

The company said its CDMO Fujifilm Diosynth Biotechnologies will add a building to its Dallas campus which will include6,000 square meters of new laboratories as well as eight new 500 / 2,000L single use bioreactors. A Fujifilm spokeswoman said the company intends to add 75 to 100 more scientists when the first phase of the new project is ready in 2021.

Fujifilm is aggressively pursuing growth strategies with both capital investment and in-house development of high-efficiency and high-productivity new technologies,the company said in a statement.

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RELATED:Fujifilm pays Biogen nearly $1B for Denmark biologics site

The announcement comes shortly after the CDMO completed a deal to buy a biologics plant in Denmark from Biogen, taking on 800 employees. Fujifilm and Biogen also agreed the CDMO would provide Biogen with the drugs the Cambridge, Massachusetts-based biotech had been manufacturing at the site. That includes its multiple sclerosis drug Tysabri.

The Japanese company has been on a spending spree in the last couple of years. In addition the the recent deals, it paid about $800 million to buy a pair of cell culture media units from Japans JXTG Holdings. In January, Fujifilm said it would invest about $90 million to expand its biologics plant in Morrisville, North Carolina, and the company is ramping up its induced pluripotent stem cell technologies for its own pipeline of regenerative drugs and intendsto manufacture iPS cells for others.

The company says it expects the CDMO business to generate nearly a $1 billion by the end of fiscal year 2021, ending March 2022.

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Pricing Of Approved Cell Therapy Products – BioInformant

By Dr. Matthew Watson

Swiss pharmaceutical giant Novartis made history as the first company to win FDA approval for a CAR-T therapy in the United States. Novartis announced that its genetically modified autologous (self-derived) immunocellular therapy, Kymriah, will cost $475,000 per treatment course. Shortly thereafter, Kite Pharma announced the approval of its CAR-T therapy, Yescarta, in the U.S. with a list price of $373,000. While these prices are expensive, they are far from trendsetting.

In this article:

Pricing of cell therapies is controversialbecause most cell therapy products are priced exponentially higher than traditional drugs. Unfortunately, most drugs can be manufactured and stockpiled in large quantities for off-the-shelf use, while cell therapies involve living cells that require a different approach to commercial-scale manufacturing, transit, stockpiling, and patient use.

To date, the highest priced treatment has not been a cell therapy, but a gene therapy (Glybera). At the time of its launch, Glybera was the first gene therapy approved in the Western world, launching for sale in Germany at a cost close to $1 million per treatment.[1] The record-breaking price tag got revealed in November 2014, when Uniqure and its marketing partner Chiesi, filed a pricing dossier with German authorities to launch Glybera. Unfortunately, Glybera was later withdrawn from the European market due to lack of sales.

Following the approval of Glybera, Kymriah, Yescarta, and more than a dozen other cell therapies, conversations surrounding pricing and reimbursement have become a focal point within the cell therapy industry.

In contrast to pharmaceutical drugs, cell therapies require a different pricing analysis. Below, price tags are shown for approved cell therapy products that have reached the market (prices in US$) and for which there is standardized market pricing.

Pricing of Approved Cell Therapy Products:

Apligrafby Organogenesis & Novartis AG in USA = $1,500-2,500 per use [2]Carticelby Genzyme in USA = $15,000 to $35,000 [3]Cartistemby MEDIPOST in S. Korea = $19,000-21,000 [4],[5]Cupistemby Anterogen in South Korea = $3,000-5,000 per treatment [6]ChondroCelectby Tigenix in EU = ~ $24,000 (20,000) [7]Dermagraftby Advanced Tissue Science in USA = $1,700 per application [8],[9]Epicelby Vericel in theUnited States = $6,000-10,000 per 1% of total body surface area [10]Hearticellgramby FCB-Pharmicell in South Korea = $19,000 [11]HeartSheetby Terumo in Japan = $56,000 (6,360,000) for HeartSheet A Kit; $15,000 (1,680,000) for HeartSheet B Kit (*Each administration uses one A Kit and 5 B Kits)[12]Holoclarby Chiesi Framaceutici in EU = Unknown (very small patient population)Kymriahby Novartis in USA = $425,000 per treatment[13]Osteocelby NuVasive in USA = $600 per cc [14],[15]Prochymalby Osiris Therapeutics and Mesoblast in Canada = ~ $200,000 [16]Provengeby Dendreon and Valeant Pharma in USA = $93,000 [17], [18]SpheroxbyCO.DON AG in EU = $9,500 $12,000 (8,000 10,000) per treatment[19]Strimvelisby GSK in EU = $665,000 (One of worlds most expensive therapies) [20],[21]Temcellby JCR Pharmaceuticals Co. Ltd. in Japan = $115,000-170,000 [22]*Pricing of TEMCELL is $7,600 (868,680 per bag), with one bag of 72m cells administered twice weekly and 2m cells/kg of body weight required per administration[23]Yescartaby Kite Pharma in USA =$373,000[24]

As shown in the list above, wound care products tend to have the lowest cell therapy pricing, typically costing $1,500 to $2,500 per use. For example, Apligrafis created from cells found in healthy human skin and is used to heal ulcers that do not heal after 3-4 weeks ($1,500-2,500 per use), and Dermagraftis a skin substitute that is placed on your ulcer to cover it and to help it heal ($1,700 per application).

Interestingly, Epicel is a treatment for deep dermal or full thickness burns comprising a total body surface area of greater than or equal to 30%. It has higher pricing of $6,000-10,000 per 1% of total body surface area, because it is not used to treat a single wound site, but rather used to treat a large surface area of the patients body.

Next, cartilage-based cell therapy products tend to have mid-range pricing of $10,000 to $35,000. For example, Carticelis a product that consists of autologous cartilage cells (pricing of $15,000 to $35,000), CARTISTEM is a regenerative treatment for knee cartilage (pricing of $19,000 to $21,000), and ChondroCelectis a suspension for implantation that contains cartilage cells (pricing of $24,000).In July 2017,the EMA in Europe also approved Spheroxas a product for articular cartilage defects of the knee with a pricing of$9,500 $12,000 (8,000 10,000) per treatment.

The next most expensive cell therapy products are the ones that are administered intravenously, which range in price from approximately $90,000 to $200,000. For example, Prochymal is an intravenously administered allogenic MSC therapy derived from the bone marrow of adult donors (pricing of $200,000), Provenge is an intravenously administered cancer immunotherapy for prostate cancer ($93,000), and Temcell is an intravenously administered autologous MSC product for the treatment of acute GVHD after an allogeneic bone marrow transplant (pricing of $115,000-170,000).

Finally, many of the worlds most expensive cell therapies are gene therapies, ranging in price from $500,000 to $1,000,000. For example, Kymriah is the first CAR-T cell therapy to be FDA approved in the United States (pricing of $475,00 per treatment course).Strimvelis isan ex-vivo stem cell gene therapy to treat patients with a very rare disease called ADA-SCID (pricing of $665,000).

Although these generalizations do not hold true for every cell therapy product, they explain the majority of cell therapy pricing and provide a valuable model for estimating cell therapy pricing and reimbursement. This information is summarized in the following table.

TABLE. Pricing Scale for Approved Cell Therapies

Another point of reference is also valuable. The RIKEN Institute launched the worlds first clinical trial involving an iPSC-derived product when it transplanted autologous iPSC-derived RPE cells into a human patient in 2014.While the trial was later suspended due to safety concerns, it resumed in 2016, this time using an allogeneic iPSC-derived cell product.

The research team indicated that by using stockpiled iPS cells, the time needed to prepare for a graft can be reduced from 11 months to as little as one month, and the cost, currently around 100 million ($889,100), can be cut to one-fifth or less.[25]

While many factors contribute to cell therapy pricing, key variables that can be used to predict market pricing include:

Another compounding factor is market size, because wound healing and cartilage replacement therapies have significant patient populations, while several of the more expensive therapies address smaller patient populations.[26]

To learn more about this rapidly expanding industry, view the Global Regenerative Medicine Industry Database Featuring 700+ Companies Worldwide.

What variable do you think influence the cost of cell therapies? Share your thoughts in the comments below.

BioInformant is the first and only market research firm to specialize in the stem cell industry. Our research has been cited by major news outlets that include the Wall Street Journal, Nature Biotechnology, Xconomy, and Vogue Magazine. Serving industry leaders that include GE Healthcare, Pfizer, Goldman Sachs, and Becton Dickinson. BioInformant is your global leader in stem cell industry data.

Footnotes[1] $1-Million Price Tag For Glybera Gene Therapy: Trade Secrets. Available at Web. 21 Aug. 2017.[2] 2017 Apligraf Medicare Product and Related Procedure Payment, Organogenesis. Available at: Web. 3 Mar. 2017.[3] CARTICEL (Autologous Chondrocyte Implantation, Or ACI). Available at: Web. 3 Aug. 2017.[4] Cartistem?, What. What Is The Cost Of Cartistem? Available at: N.p., 2017. Web. 3 Mar. 2017.[5] Cartistem. Available at: Web. 3 Aug. 2017.[6] Stem Art, Stem Cell Therapy Pricing. Available at: Web. 3 Mar. 2017.[7]Are Biosimilar Cell Therapy Products Possible? Presentation by Christopher A Bravery [PDF]. Available at: Web. 3 Aug. 2017.[8] Artificial Skin, Presentation by Nouaying Kue (BME 281). Available at: Web. 3 Mar. 2017.[9] Allenet, et al. Cost-effectiveness modeling of Dermagraft for the treatment of diabetic foot ulcers in the french context. Diabetic Metab. 2000 Apr;26(2):125-32.[10] Epicel Skin Grafts, Sarah Schlatter, Biomedical Engineering, University of Rhode Island. Available at: Web. 31 July. 2017.[11] Nature. (2011). South Koreas stem cell approval. [online] Available at: Web. 3 Sept. 2017.[12] Novick, Coline Lee. Translated version of the first two pages of Terumos Conditionally Approved HeartSheet NHI Reimbursement Price. [Twitter Post] Available Web. 21 Sep. 2017.[13] (2017). Is $475,000 Too High a Price for Novartiss Historic Cancer Gene Therapy? [online] Available at: Web. 8 Sept. 2017.[14] Skovrlj, Branko et al. Cellular Bone Matrices: Viable Stem Cell-Containing Bone Graft Substitutes. The Spine Journal 14.11 (2014): 2763-2772. Available at: Web. April 12, 2017.[15] Hiltzik, Michael. Sky-High Price Of New Stem Cell Therapies Is A Growing Concern. Available at: Web. 1 Sept. 2017.[16] Counting Coup: Is Osiris Losing Faith In Prochymal?, Busa Consulting LLC. Available at: Web. 3 Aug. 2017.[17] Dendreon Sets Provenge Price At $93,000, Says Only 2,000 People Will Get It In First Year | Xconomy. Available at: Web. 3 Mar. 2017.[18] Dendreon: Provenge To Cost $93K For Full Course Of Treatment | Fiercebiotech. Available at: Web. 3 Mar. 2017.[19]Warberg Research.CO.DON (CDAX, Health Care). Available at: Web. 21 Sept. 2017.[20] GSK Inks Money-Back Guarantee On $665K Strimvelis, Blazing A Trail For Gene-Therapy Pricing | Fiercepharma. Available at: Web. 3 Mar. 2017.[21] Strimvelis. Available at: Web. 13 Aug. 2017.[22] MesoblastS Japan Licensee Receives Pricing For TEMCELL HS Inj. For Treatment Of Acute Graft Versus Host Disease. Mesoblast Limited, GlobeNewswire News Room. Available at: Web. 3 Mar. 2017.[23]TEMCELL HS Inj. Receives NHI Reimbursement Price Listing, JCR Pharmaceuticals Co., Ltd. News Release, November 26, 2015. Available at: Web. 3 Mar. 2017.[24]Kites Yescarta (Axicabtagene Ciloleucel) Becomes First CAR T Therapy Approved by the FDA for the Treatment of Adult Patients With Relapsed or Refractory Large B-Cell Lymphoma After Two or More Lines of Systemic Therapy. Business Wire.Web. 19 Oct. 2017.[25]Riken-Linked Team Set To Test Transplanting Eye Cells Using Ips From Donor | The Japan Times. The Japan Times. N.p., 2017. Web. 23 July. 2017.[26]LinkedIn Comment, by David Caron. Available at: Web. 21 Sept. 2017.

Pricing Of Approved Cell Therapy Products Stem Cells, CAR-T, And More

Pricing Of Approved Cell Therapy Products - BioInformant

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Gene & Cell Therapy FAQs | ASGCT – American Society of …

By Dr. Matthew Watson

The challenges of gene and cell therapists can be divided into three broad categories based on disease, development of therapy, and funding.

Challenges based on the disease characteristics: Disease symptoms of most genetic diseases, such as Fabrys, hemophilia, cystic fibrosis, muscular dystrophy, Huntingtons, and lysosomal storage diseases are caused by distinct mutations in single genes. Other diseases with a hereditary predisposition, such as Parkinsons disease, Alzheimers disease, cancer, and dystonia may be caused by variations/mutations in several different genes combined with environmental causes. Note that there are many susceptible genes and additional mutations yet to be discovered. Gene replacement therapy for single gene defects is the most conceptually straightforward. However, even then the gene therapy agent may not equally reduce symptoms in patients with the same disease caused by different mutations, and even the samemutationcan be associated with different degrees of disease severity. Gene therapists often screen their patients to determine the type of mutation causing the disease before enrollment into a clinical trial.

The mutated gene may cause symptoms in more than one cell type. Cystic fibrosis, for example, affects lung cells and the digestive tract, so the gene therapy agent may need to replace the defective gene or compensate for its consequences in more than one tissue for maximum benefit. Alternatively, cell therapy can utilizestem cellswith the potential to mature into the multiple cell types to replace defective cells in different tissues.

In diseases like muscular dystrophy, for example, the high number of cells in muscles throughout the body that need to be corrected in order to substantially improve the symptoms makes delivery of genes and cells a challenging problem.

Some diseases, like cancer, are caused by mutations in multiple genes. Although different types of cancers have some common mutations, every tumor from a single type of cancer does not contain the same mutations. This phenomenon complicates the choice of a single gene therapy tactic and has led to the use of combination therapies and cell elimination strategies. For more information on gene and cell therapy strategies to treat cancer, please refer to the Cancer and Immunotherapy summary in the Disease Treatment section.

Disease models in animals do not completely mimic the human diseases and viralvectorsmay infect various species differently. The testing of vectors in animal models often resemble the responses obtained in humans, but the larger size of humans in comparison to rodents presents additional challenges in the efficiency of delivery and penetration of tissue.Gene therapy, cell therapy, and oligonucleotide-based therapy agents are often tested in larger animal models, including rabbit, dog, pig and nonhuman primate models. Testing human cell therapy in animal models is complicated by immune rejections. Furthermore, humans are a very heterogeneous population. Their immune responses to the vectors, altered cells, or cell therapy products may differ or be similar to results obtained in animal models.

Challenges in the development of gene and cell therapy agents: Scientific challenges include the development of gene therapy agents that express the gene in the relevant tissue at the appropriate level for the desired duration of time. There are a lot of issues in that once sentence, and while these issues are easy to state, each one requires extensive research to identify the best means of delivery, how to control sufficient levels or numbers of cells, and factors that influence duration of gene expression or cell survival. After the delivery modalities are determined, identification and engineering of a promoter and control elements (on/off switch and dimmer switch) that will produce the appropriate amount of protein in the target cell can be combined with the relevant gene. This gene cassette is engineered into a vector or introduced into thegenomeof a cell and the properties of the delivery vehicle are tested in different types of cells in tissue culture. Sometimes things go as planned and then studies can be moved onto examination in animal models. In most cases, the gene/cell therapy agent may need to be improved further by adding new control elements to obtain the desired responses in cells and animal models.

Furthermore, the response of the immune system needs to be considered based on the type of gene or cell therapy being undertaken. For example, in gene or cell therapy for cancer, one aim is to selectively boost the existing immune response to cancer cells. In contrast, to treat genetic diseases like hemophilia and cystic fibrosis the goal is for the therapeutic protein to be accepted as an addition to the patients immune system.

If the new gene is inserted into the patients cellularDNA, the intrinsic sequences surrounding the new gene can affect its expression and vice versa. Scientists are now examining short DNA segments that may insulate the new gene from surrounding control elements. Theoretically, these insulator sequences would also reduce the effect of vector control signals in the gene cassette on adjacent cellular genes. Studies are also focusing on means to target insertion of the new gene into safe areas of the genome, to avoid influence on surrounding genes and to reduce the risk of insertional mutagenesis.

Challenges of cell therapy include the harvesting of the appropriate cell populations and expansion or isolation of sufficient cells for one or multiple patients. Cell harvesting may require specific media to maintain the stem cells ability toself-renew and mature into the appropriate cells. Ideally extra cells are taken from the individual receiving therapy. Those additional cells can expand in culture and can be induced to becomepluripotent stem cells(iPS), thus allowing them to assume a wide variety of cell types and avoiding immune rejection by the patient. The long term benefit of stem cell administration requires that the cells be introduced into the correct target tissue and become established functioning cells within the tissue. Several approaches are being investigated to increase the number of stem cells that become established in the relevant tissue.

Another challenge is developing methods that allow manipulation of the stem cells outside the body while maintaining the ability of those cells to produce more cells that mature into the desired specialized cell type. They need to provide the correct number of specialized cells and maintain their normal control of growth and cell division, otherwise there is the risk that these new cells may grow into tumors.

Challenges in funding: In most fields, funding for basic or applied research for gene and cell therapy is available through the National Institutes of Health (NIH) and private foundations. These are usually sufficient to cover the preclinical studies that suggest a potential benefit from a particular gene and cell therapy. Moving into clinical trials remains a huge challenge as it requires additional funding for manufacturing of clinical grade reagents, formal toxicology studies in animals, preparation of extensive regulatory documents, and costs of clinical trials.Biotechnology companies and the NIH are trying to meet the demand for this large expenditure, but many promising therapies are slowed down by lack of funding for this critical next phase.

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Cell Therapy World Asia – IMAPAC – Imagine your Impact

By Dr. Matthew Watson

Globally, the stem cell therapy market is expected to be worth $40 billion by 2020 and $180 billion by 2030. The largest number of marketed cell therapy products is used for the treatment of notably non-healing wounds/skin (46%) and muscular-skeletal injuries (35%). This trend will change as more and stem cell therapy products for cancer and heart disease complete their clinical trials and are approved for market release.

Adult stem cell leads the market due to low contamination during sub-culture and expansion, relatively low labour production and compatibility with the human body.Just the Induced pluripotent stem cells (IPScs) are expected to report revenue of over USD 4.5 billion by 2020, on account of the analogous nature of its origin.With the continued growth of medical tourism hubs like India, Singapore, and Thailand, Asia is expected to maintain its place as the epicentre of stem cell research and therapy. These opportunities include contract research outsourcing and rising patient population with neurological and other chronic conditions in the region. Japan, Singapore and South Korea are the frontrunners and are set to dominate the APAC stem cell market in the coming years.

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iPS Cells for Disease Modeling and Drug Discovery

By Dr. Matthew Watson

Cambridge Healthtech Institutes 4th AnnualJune 19-20, 2019

With advances in reprogramming and differentiation technologies, as well as with the recent availability of gene editing approaches, we are finally able to create more complex and phenotypically accurate cellular models based on pluripotent cell technology. This opens new and exciting opportunities for pluripotent stem cell utilization in early discovery, preclinical and translational research. CNS diseases and disorders are currently the main therapeutic area of application with some impressive success stories resulted in clinical trials. Cambridge Healthtech Institutes 4th Annual iPS Cells for Disease Modeling and Drug Discovery conference is designed to bring together experts and bench scientists working with pluripotent cells and end users of their services, researchers working on finding cures for specific diseases and disorders.

Day 1 | Day 2 | Download Brochure | Speaker Biographies

Wednesday, June 19

12:00 pm Registration Open

12:00 Bridging Luncheon Presentation:Structural Maturation in the Development of hiPSC-Cardiomyocyte Models for Pre-clinical Safety, Efficacy, and Discovery

Nicholas Geissse, PhD, CSO, NanoSurface Biomedical

Alec S.T. Smith, PhD, Acting Instructor, Bioengineering, University of Washington

hiPSC-CM maturation is sensitive to structural cues from the extracellular matrix (ECM). Failure to reproduce these signals in vitro can hamper experimental reproducibility and fidelity. Engineering approaches that address this gap typically trade off complexity with throughput, making them difficult to deploy in the modern drug development paradigm. The NanoSurface Car(ina) platform leverages ECM engineering approaches that are fully compatible with industry-standard instrumentation including HCI- and MEA-based assays, thereby improving their predictive power.

12:30 Transition to Plenary


2:20Booth Crawl and Dessert Break in the Exhibit Hall with Poster Viewing

2:25 Meet the Plenary Keynotes

3:05 Chairpersons Remarks

Gabriele Proetzel, PhD, Director, Neuroscience Drug Discovery, Takeda Pharmaceuticals, Inc.

3:10 KEYNOTE PRESENTATION: iPSC-Based Drug Discovery Platform for Targeting Innate Immune Cell Responses

Christoph Patsch, PhD, Team Lead Stem Cell Assays, Disease Relevant Cell Models and Assays, Chemical Biology, Therapeutic Modalities, Roche Pharma Research and Early Development

The role of innate immune cells in health and disease, respectively their function in maintaining immune homeostasis and triggering inflammation makes them a prime target for therapeutic approaches. In order to explore novel therapeutic strategies to enhance immunoregulatory functions, we developed an iPSC-based cellular drug discovery platform. Here we will highlight the unique opportunities provided by an iPSC-based drug discovery platform for targeting innate immune cells.

3:40 Phenotypic Screening of Induced Pluripotent Stem Cell Derived Cardiomyocytes for Drug Discovery and Toxicity Screening

Arne Bruyneel, PhD, Postdoctoral Fellow, Mark Mercola Lab, Cardiovascular Institute, Stanford University School of Medicine

Cardiac arrhythmia and myopathy is a major problem with cancer therapeutics, including newer small molecule kinase inhibitors, and frequently causes heart failure, morbidity and death. However, currentin vitromodels are unable to predict cardiotoxicity, or are not scalable to aid drug development. However, with recent progress in human stem cell biology, cardiac differentiation protocols, and high throughput screening, new tools are available to overcome this barrier to progress.

4:10 Disease Modeling Using Human iPSC-Derived Telencephalic Inhibitory Interneurons - A Couple of Case Studies

Yishan Sun, PhD, Investigator, Novartis Institutes for BioMedical Research (NIBR)

Human iPSC-derived neurons provide the foundation for phenotypic assays assessing genetic or pharmacological effects in a human neurobiological context. The onus is on assay developers to generate application-relevant neuronal cell types from iPSCs, which is not always straightforward, given the diversity of neuronal classes in the human brain and their developmental trajectories. Here we present two case studies to illustrate the use of iPSC-derived telencephalic GABAergic interneurons in neuropsychiatric research.

4:40 Rethinking the Translational The Use of Highly Predictive hiPSC-Derived Models in Pre-Clinical Drug Development

Stefan Braam, CEO, Ncardia

Current drug development strategies are failing to increase the number of drugs reaching the market. One reason for low success rates is the lack of predictive models. Join our talk to learn how to implement a predictive and translational in vitro disease model, and assays for efficacy screening at any throughput.

5:10 4th of July Celebration in the Exhibit Hall with Poster Viewing

5:30 - 5:45 Speed Networking: Oncology

6:05 Close of Day

5:45 Dinner Short Course Registration

6:15 Dinner Short Course*

*Separate registration required.

Day 1 | Day 2 | Download Brochure | Speaker Biographies

Thursday, June 20

7:15 am Registration

7:15 Breakout Discussion Groups with Continental Breakfast

8:10 Chairpersons Remarks

Jeff Willy, PhD, Research Fellow, Discovery and Investigative Toxicology, Vertex

8:15 Levering iPSC to Understand Mechanism of Toxicity

Jeff Willy, PhD, Research Fellow, Discovery and Investigative Toxicology, Vertex

The discovery of mammalian cardiac progenitor cells suggests that the heart consists of not only terminally differentiated beating cardiomyocytes, but also a population of self-renewing stem cells. We recently showed that iPSC cardiomyocytes can be utilized not only to de-risk compounds with potential for adverse cardiac events, but also to understand underlying mechanisms of cell-specific toxicities following xenobiotic stress, thus preventing differentiation and self-renewal of damaged cells.

8:45Pluripotent Stem Cell-Derived Cardiac and Vascular Progenitor Cells for Tissue Regeneration

Nutan Prasain, PhD, Associate Director, Cardiovascular Programs, Astellas Institute for Regenerative Medicine (AIRM)

This presentation will provide the review on recent discoveries in the derivation and characterization of cardiac and vascular progenitor cells from pluripotent stem cells, and discuss the therapeutic potential of these cells in cardiac and vascular tissue repair and regeneration.

9:15 Use of iPSCDerived Hepatocytes to Identify Treatments for Liver Disease

Stephen A. Duncan, PhD, Smartstate Chair in Regenerative Medicine, Professor and Chairman, Department of Regenerative Medicine and Cell Biology, Medical University of South Carolina

MTDPS3 is a rare disease caused by mutations in the DGUOK gene, which is needed for mitochondrial DNA (mtDNA) replication and repair. Patients commonly die as children from liver failure primarily caused by unmet energy requirements. We modeled the disease using DGOUK deficient iPSC derived hepatocytes and performed a screen to identify drugs that can restore mitochondrial ATP production.

9:45Industrial-Scale Generation of Human iPSC-Derived Hepatocytes for Liver-Disease and Drug Development Studies

Liz Quinn, PhD, Associate Director, Stem Cell Marketing, Marketing, Takara Bio USA

Our optimized hepatocyte differentiation protocol and standardized workflow mimics embryonic development and allows for highly efficient differentiation of hPSCs through definitive endoderm into hepatocytes. We will describe the creation of large panels of industrial-scale hPSC-derived hepatocytes with specific genotypes and phenotypes and their utility for drug metabolism and disease modeling.

10:00 Sponsored Presentation (Opportunity Available)

10:15 Coffee Break in the Exhibit Hall with Poster Viewing

10:45 Poster Winner Announced

11:00 KEYNOTE PRESENTATION: Modeling Human Disease Using Pluripotent Stem Cells

Lorenz Studer, MD, Director, Center for Stem Cell Biology, Memorial Sloan Kettering Cancer Center

One of the most intriguing applications of human pluripotent stem cells is the possibility of recreating a disease in a dish and to use such cell-based models for drug discovery. Our lab uses human iPS and ES cells for modeling both neurodevelopmental and neurodegenerative disorders. I will present new data on our efforts of modeling complex genetic disease using pluripotent stem cells and the development of multiplex culture systems.

11:30 Preclinical Challenges for Gene Therapy Approaches in Neuroscience

Gabriele Proetzel, PhD, Director, Neuroscience Drug Discovery, Takeda Pharmaceuticals, Inc.

Gene therapy has delivered encouraging results in the clinic, and with the first FDA approval for an AAV product is now becoming a reality. This presentation will provide an overview of the most recent advances of gene therapy for the treatment of neurological diseases. The discussion will focus on preclinical considerations for gene therapy including delivery, efficacy, biodistribution, animal models and safety.

12:00 pm Open Science Meets Stem Cells: A New Drug Discovery Approach for Neurodegenerative Disorders

Thomas Durcan, PhD, Assistant Professor, Neurology and Neurosurgery, McGill University

Advances in stem cell technology have provided researchers with tools to generate human neurons and develop first-of-their-kind disease-relevant assays. However, it is imperative that we accelerate discoveries from the bench to the clinic and the Montreal Neurological Institute (MNI) and its partners are piloting an Open Science model. By removing the obstacles in distributing patient samples and assay results, our goal is to accelerate translational medical research.

12:30 Elevating Drug Discovery with Advanced Physiologically Relevant Human iPSC-Based Screening Platforms

Fabian Zanella, PhD, Director, Research and Development, StemoniX

Structurally engineered human induced pluripotent stem cell (hiPSC)-based platforms enable greater physiological relevance, elevating performance in toxicity and discovery studies. StemoniXs hiPSC-derived platforms comprise neural (microBrain) or cardiac (microHeart) cells constructed with appropriate inter- and intracellular organization promoting robust activity and expected responses to known cellular modulators.

1:00Overcoming Challenges in CNS Drug Discovery through Developing Translatable iPSC-derived Cell-Based Assays

Jonathan Davila, PhD, CEO, Co-Founder, NeuCyte Inc.

Using direct reprogramming of iPSCs to generate defined human neural tissue, NeuCyte developed cell-based assays with complex neuronal structure and function readouts for versatile pre-clinical applications. Focusing on electrophysiological measurements, we demonstrate the capability of this approach to identify adverse neuroactive effects, evaluate compound efficacy, and serve phenotypic drug discovery.

1:15Enjoy Lunch on Your Own

1:35 Dessert and Coffee Break in the Exhibit Hall with Poster Viewing

1:45 - 2:00 Speed Networking: Last Chance to Meet with Potential Partners and Collaborators!

2:20 Chairpersons Remarks

Gary Gintant, PhD, Senior Research Fellow, AbbVie

2:25 The Evolving Roles of Evolving Human Stem Cell-Derived Cardiomyocyte Preparations in Cardiac Safety Evaluations

Gary Gintant, PhD, Senior Research Fellow, AbbVie

Human induced pluripotent stem cell-derived cardiomyocytes (hiPSC-CMs) hold great promise for preclinical cardiac safety testing. Recent applications focus on drug effects on cardiac electrophysiology, contractility, and structural toxicities, with further complexity provided by the growing number of hiPSC-CM preparations being developed that may also promote myocyte maturity. The evolving roles (both non-regulatory and regulatory) of these preparations will be reviewed, along with general considerations for their use in cardiac safety evaluations.

2:55 Pharmacogenomic Prediction of Drug-Induced Cardiotoxicity Using hiPSC-Derived Cardiomyocytes

Paul W. Burridge, PhD, Assistant Professor, Department of Pharmacology, Center for Pharmacogenomics, Northwestern University Feinberg School of Medicine

We have demonstrated that human induced pluripotent stem cell-derived cardiomyocytes successfully recapitulate a patients predisposition to chemotherapy-induced cardiotoxicity, confirming that there is a genomic basis for this phenomenon. Here we will discuss our recent work deciphering the pharmacogenomics behind this relationship, allowing the genomic prediction of which patients are likely to experience this side effect. Our efforts to discover new drugs to prevent doxorubicin-induced cardiotoxicity will also be reviewed.

3:25 Exploring the Utility of iPSC-Derived 3D Cortical Spheroids in the Detection of CNS Toxicity

Qin Wang, PhD, Scientist, Drug Safety Research and Evaluation, Takeda

Drug-induced Central Nervous System (CNS) toxicity is a common safety attrition for project failure during discovery and development phases due low concordance rates between animal models and human, absence of clear biomarkers, and a lack of predictive assays. To address the challenge, we validated a high throughput human iPSC-derived 3D microBrain model with a diverse set of pharmaceuticals. We measured drug-induced changes in neuronal viability and Ca channel function. MicroBrain exposure and analyses were rooted in therapeutic exposure to predict clinical drug-induced seizures and/or neurodegeneration. We found that this high throughput model has very low false positive rate in the prediction of drug-induced neurotoxicity.

3:55 Linking Liver-on-a-Chip and Blood-Brain-Barrier-on-a-Chip for Toxicity Assessment

Sophie Lelievre, DVM, PhD, LLM, Professor, Cancer Pharmacology, Purdue University College of Veterinary Medicine

One of the challenges to reproduce the function of tissues in vitro is the maintenance of differentiation. Essential aspects necessary for such endeavor involve good mechanical and chemical mimicry of the microenvironment. I will present examples of the management of the cellular microenvironment for liver and blood-brain-barrier tissue chips and discuss how on-a-chip devices may be linked for the integrated study of the toxicity of drugs and other molecules.

4:25 Close of Conference

Day 1 | Day 2 | Download Brochure | Speaker Biographies

Arrive early to attend Tuesday, June 18 - Wednesday, June 19

Chemical Biology and Target Validation

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iPS Cells for Disease Modeling and Drug Discovery

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CloneR hPSC Cloning Supplement – Stemcell Technologies

By Dr. Matthew Watson

'); jQuery('.cart-remove-box a').on('click', function(){ link = jQuery(this).attr('href'); jQuery.ajax({ url: link, cache: false }); jQuery('.cart-remove-box').remove(); setTimeout(function(){window.location.reload();}, 800); }); }); //jQuery('#ajax_loader').hide(); // clear being added addToCartButton.text(defaultText).removeAttr('disabled').removeClass('disabled'); addToCartButton.parent().find('.disabled-blocker').remove(); loadingDots.remove(); clearInterval(loadingDotId); jQuery('body').append(""); setTimeout(function () {jQuery('.add-to-cart-success').slideUp(500)}, 5000); }); } try { jQuery.ajax( { url : url, dataType : 'json', type : 'post', data : data, complete: function(){ if(jQuery('body').hasClass('product-edit') || jQuery('body').hasClass('wishlist-index-configure')){ jQuery.ajax({ url: "", cache: false }).done(function(html){ jQuery('header#header .top-cart').replaceWith(html); }); jQuery('#ajax_loader').hide(); jQuery('body').append(""); setTimeout(function () {jQuery('.add-to-cart-success').slideUp(500)}, 5000); } }, success : function(data) { if(data.status == 'ERROR'){ jQuery('body').append(''); }else{ ajaxComplete(); } } }); } catch (e) { } // End of our new ajax code this.form.action = oldUrl; if (e) { throw e; } } }.bind(productAddToCartForm); productAddToCartForm.submitLight = function(button, url){ if(this.validator) { var nv = Validation.methods; delete Validation.methods['required-entry']; delete Validation.methods['validate-one-required']; delete Validation.methods['validate-one-required-by-name']; if (this.validator.validate()) { if (url) { this.form.action = url; } this.form.submit(); } Object.extend(Validation.methods, nv); } }.bind(productAddToCartForm); function setAjaxData(data,iframe,name,image){ if(data.status == 'ERROR'){ jQuery('body').append(''); }else{ if(data.sidebar && !iframe){ if(jQuery('.top-cart').length){ jQuery('.top-cart').replaceWith(data.sidebar); } if(jQuery('.sidebar .block.block-cart').length){ if(jQuery('#cart-sidebar').length){ jQuery('#cart-sidebar').html(jQuery(data.sidebar).find('#mini-cart')); jQuery('.sidebar .block.block-cart .subtotal').html(jQuery(data.sidebar).find('.subtotal')); }else{ jQuery('.sidebar .block.block-cart p.empty').remove(); content = jQuery('.sidebar .block.block-cart .block-content'); jQuery('').appendTo(content); jQuery('').appendTo(content); content.find('#cart-sidebar').html(jQuery(data.sidebar).find('#mini-cart').html()); content.find('.actions').append(jQuery(data.sidebar).find('.subtotal')); content.find('.actions').append(jQuery(data.sidebar).find('.actions button.button')); } cartProductRemove('#cart-sidebar li.item a.btn-remove', { confirm: 'Are you sure you would like to remove this item from the shopping cart?', submit: 'Ok', calcel: 'Cancel' }); } jQuery.fancybox.close(); jQuery('body').append(''); }else{ jQuery.ajax({ url: "", cache: false }).done(function(html){ jQuery('header#header .top-cart').replaceWith(html); jQuery('.top-cart #mini-cart li.item a.btn-remove').on('click', function(event){ event.preventDefault(); jQuery('body').append('Are you sure you would like to remove this item from the shopping cart?OkCancel'); jQuery('.cart-remove-box a').on('click', function(){ link = jQuery(this).attr('href'); jQuery.ajax({ url: link, cache: false }); jQuery('.cart-remove-box').remove(); setTimeout(function(){window.location.reload();}, 800); }); }); jQuery.fancybox.close(); jQuery('body').append(''); }); } } setTimeout(function () {jQuery('.add-to-cart-success').slideUp(500)}, 5000); } //]]> CloneR is a defined, serum-free supplement designed to increase the cloning efficiency and single-cell survival of human embryonic stem cells (ES cells) and induced pluripotent stem cells (iPS cells). CloneR enables the robust generation of clonal cell lines without single-cell adaptation, thus minimizing the risk of acquiring genetic abnormalities.

CloneR is compatible with the TeSR family of media for human ES and iPS cell maintenance as well as your choice of cell culture matrix.


Greatly facilitates the process of genome editing of human ES and iPS cells Compatible with any TeSR maintenance medium and your choice of cell culture matrix Does not require adaptation to single-cell passaging Increases single-cell survival at clonal density across multiple human ES and iPS cell lines

Cell Type:

Pluripotent Stem Cells


Cell Culture

Area of Interest:

Cell Line Development; Stem Cell Biology; Disease Modeling


Defined; Serum-Free

Document Type

Product Name

Catalog #

Lot #


This product is designed for use in the following research area(s) as part of the highlighted workflow stage(s). Explore these workflows to learn more about the other products we offer to support each research area.

Research Area Workflow Stages for

Workflow Stages

Figure 1. hPSC Single-Cell Cloning Workflow with CloneR

On day 0, human pluripotent stem cells (hPSCs) are seeded as single cells at clonal density (e.g. 25 cells/cm2) or sorted at 1 cell per well in 96-well plates in TeSR (mTeSR1 or TeSR-E8) medium supplemented with CloneR. On day 2, the cells are fed with TeSR medium containing CloneR supplement. From day 4, cells are maintained in TeSR medium without CloneR. Colonies are ready to be picked between days 10 - 14. Clonal cell lines can be maintained long-term in TeSR medium.

Figure 2. CloneR Increases the Cloning Efficiency of hPSCs and is Compatible with Multiple hPSC Lines and Seeding Protocols

TeSR medium supplemented with CloneR increases hPSC cloning efficiency compared with cells plated in TeSR containing ROCK inhibitor. Cells were seeded (A) at clonal density (25 cells/cm2) in mTeSR1 and TeSR-E8 and (B) by single-cell deposition using FACS (seeded at 1 cell/well) in mTeSR1.

Figure 3. CloneR Increases the Cloning Efficiency of hPSCs at Low Seeding Densities

hPSCs plated in mTeSR1 supplemented with CloneR demonstrated significantly increased cloning efficiencies compared to cells plated in mTeSR1 containing ROCK inhibitor (10M Y-27632). Shown are representative images of alkaline phosphatase-stained colonies at day 7 in individual wells of a 12-well plate. H1 human embryonic stem (hES) cells were seeded at clonal density (100 cells/well, 25 cells/cm2) in mTeSR1 supplemented with (A) ROCK inhibitor or (B) CloneR on Vitronectin XF cell culture matrix.

Figure 4. CloneR Yields Larger Single-Cell Derived Colonies

hPSCs seeded in mTeSR1 supplemented with CloneR result in larger colonies than cells seeded in mTeSR1 containing ROCK inhibitor (10M Y-27632). Shown are representative images of hPSC clones established after 7 days of culture in mTeSR1 supplemented with (A) ROCK inhibitor or (B) CloneR.

Figure 5. Clonal Cell Lines Established Using CloneR Display Characteristic hPSC Morphology

Clonal cell lines established using mTeSR1 or TeSR-E8 medium supplemented with CloneR retain the prominent nucleoli and high nuclear-to-cytoplasmic ratio characteristic of hPSCs. Representative images at passage 7 after cloning are shown for clones derived from the parental (A) H1 hES cell and (B) WLS-1C human induced pluripotent stem (iPS) cell lines.

Figure 6. Clonal Cell Lines Established with CloneR Express High Levels of Undifferentiated Cell Markers

hPSC clonal cell lines established using mTeSR1 supplemented with CloneR express comparable levels of undifferentiated cell markers, OCT4 (Catalog #60093) and TRA-1-60 (Catalog #60064), as the parental cell lines. (A) Clonal cell lines established from parental H1 hES cell line. (B) Clonal cell lines established from parental WLS-1C hiPS cell line. Data is presented between passages 5 - 7 after cloning and is shown as mean SEM; n = 2.

Figure 7. Clonal Cell Lines Established Using CloneR Display a Normal Karyotype

Representative karyograms of clones derived from parental (A) H1 hES cell and (B) WLS-1C hiPS cell lines demonstrate that the clonal lines established with CloneR have a normal karyotype. Cells were karyotyped 5 passages after cloning, with an overall passage number of 45 and 39, respectively.

Figure 8. Clonal Cell Lines Established Using CloneR Display Normal Growth Rates

Fold expansion of clonal cell lines display similar growth rates to parental cell lines. Shown are clones (red) and parental cell lines (gray) for (A) H1 hES cell and (B) WLS-1C hiPS cell lines.


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The addition of human iPS cell-derived neural progenitors …

By Dr. Matthew Watson

JavaScript is disabled on your browser. Please enable JavaScript to use all the features on this page.Highlights

Human iPS cell-derived neural progenitors influence the contractile property of cardiac spheroid.

The contractile function of spheroids depends on the ratio of neural progenitors to cardiac cells.

Neural factors may influence the contractile function of the spheroids.

We havebeen attempting to use cardiac spheroids to construct three-dimensional contractilestructures for failed hearts. Recent studies have reported that neuralprogenitors (NPs) play significant roles in heart regeneration. However, theeffect of NPs on the cardiac spheroid has not yet been elucidated.

This studyaims to demonstrate the influence of NPs on the function of cardiac spheroids.

Thespheroids were constructed on a low-attachment-well plate by mixing humaninduced pluripotent stem (hiPS) cell-derived cardiomyocytes and hiPScell-derived NPs (hiPS-NPs). The ratio of hiPS-NPs was set at 0%, 10%, 20%,30%, and 40% of the total cell number of spheroids, which was 2500. The motionwas recorded, and the fractional shortening and the contraction velocity weremeasured.

Spheroidswere formed within 48 h after mixing the cells, except for the spheroidscontaining 0% hiPS-NPs. Observation at day 7 revealed significant differencesin the fractional shortening (analysis of variance; p=0.01). The bestfractional shortening was observed with the spheroids containing 30% hiPS-NPs.Neuronal cells were detected morphologically within the spheroids under aconfocal microscope.

Theaddition of hiPS-NPs influenced the contractile function of the cardiacspheroids. Further studies are warranted to elucidate the underlying mechanism.

Human iPS cell


Neural progenitor


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What Are Induced Pluripotent Stem Cells? – Stem Cell: The …

By Dr. Matthew Watson

Today, induced pluripotent stem cells are mostly used to understand how certain diseases occur and how they work. By using IPS cells, one can actually study the cells and tissues affected by the disease without causing unnecessary harm to the patient.For example, its extremely difficult to obtain actual brain cells from a living patient with Parkinsons Disease. This process is even more complicated if you want to study the disease in its early stages before symptoms begin presenting themselves.

Fortunately, with genetic reprogramming, researchers can now achieve this. Scientists can do a skin biopsy of a patient with Parkinsons disease and create IPS cells. These IPS cells can then be converted into neurons, which will have the same genetic make-up as the patients own cells.

Because of IPS cells, researchers can now study conditions like Parkinsons disease to determine what went wrong and why. They can also test out new treatment methods in hopes of protecting the patient against the disease or curing it after diagnosis.

In addition, IPS cells have also been looked to as a way to replace cells that are often destroyed by certain diseases. However, there is still research to be done here.

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Stem Cell Key Terms | California’s Stem Cell Agency

By Dr. Matthew Watson

En Espaol

The term stem cell by itself can be misleading. In fact, there are many different types of stem cells, each with very different potential to treat disease.

Stem CellPluripotentEmbryonic Stem CellAdult Stem CelliPS CellCancer Stem Cell

By definition, all stem cells:

Pluripotent means many "potentials". In other words, these cells have the potential of taking on many fates in the body, including all of the more than 200 different cell types. Embryonic stem cells are pluripotent, as are induced pluripotent stem (iPS) cells that are reprogrammed from adult tissues. When scientists talk about pluripotent stem cells, they mostly mean either embryonic or iPS cells.

Embryonic stem cells come from pluripotent cells, which exist only at the earliest stages of embryonic development. In humans, these cells no longer exist after about five days of development.

When isolated from the embryo and grown in a lab dish, pluripotent cells can continue dividing indefinitely. These cells are known as embryonic stem cells.

James Thomson, a professor in the Department of Cell and Regenerative Biology at the University of Wisconsin, derived the first human embryonic stem cell lines in 1998. He now shares a joint appointment at the University of California, Santa Barbara, a CIRM-funded institution.

Adult stem cells are found in the various tissues and organs of the human body. They are thought to exist in most tissues and organs where they are the source of new cells throughout the life of the organism, replacing cells lost to natural turnover or to damage or disease.

Adult stem cells are committed to becoming a cell from their tissue of origin, and cant form other cell types. They are therefore also called tissue-specific stem cells. They have the broad ability to become many of the cell types present in the organ they reside in. For example:

Unlike embryonic stem cells, researchers have not been able to grow adult stem cells indefinitely in the lab, but this is an area of active research.

Scientists have also found stem cells in the placenta and in the umbilical cord of newborn infants, and they can isolate stem cells from different fetal tissues. Although these cells come from an umbilical cord or a fetus, they more closely resemble adult stem cells than embryonic stem cells because they are tissue-specific. The cord blood cells that some people bank after the birth of a child are a form of adult blood-forming stem cells.

CIRM-grantee IrvWeissman of the Stanford University School of Medicine isolated the first blood-forming adult stem cell from bone marrow in 1988 in mice and later in humans.

Irv Weissman explains the difference between an adult stem cell and an embryonic stem cell (video)

An induced pluripotent stem cell, or iPS cell, is a cell taken from any tissue (usually skin or blood) from a child or adult and is genetically modified to behave like an embryonic stem cell. As the name implies, these cells are pluripotent, which means that they have the ability to form all adult cell types.

Shinya Yamanaka, an investigator with joint appointments at Kyoto University in Japan and the Gladstone Institutes in San Francisco, created the first iPS cells from mouse skin cells in 2006. In 2007, several groups of researchers including Yamanaka and James Thomson from the University of Wisconsin and University of California, Santa Barbara generated iPS cells from human skin cells.

Cancer stem cells are a subpopulation of cancer cells that, like stem cells, can self-renew. However, these cellsrather than growing into tissues and organspropagate the cancer, maturing into the many types of cells that are found in a tumor.

Cancer stem cells are a relatively new concept, but they have generated a lot of interest among cancer researchers because they could lead to more effective cancer therapies that can treat tumors resistant to common cancer treatments.

However, there is still debate on which types of cancer are propelled by cancer stem cells. For those that do, cancer stem cells are thought to be the source of all cells that make up the cancer.

Conventional cancer treatments, such as chemotherapy, may only destroy cells that form the bulk of the tumor, leaving the cancer stem cells intact. Once treatment is complete, cancer stem cells that still reside within the patient can give rise to a recurring tumor. Based on this hypothesis, researchers are trying to find therapies that destroy the cancer stem cells in the hopes that it truly eradicates a patients cancer.

John Dick from the University of Toronto first identified cancer stem cells in 1997. Michael Clarke, then at the University of Michigan, later found the first cancer stem cell in a solid tumor, in this case, breast cancer. Now at Stanford University School of Medicine, Clarke and his group have found cancer stem cells in colon cancer and head and neck cancers.

Find out More:

Catriona Jamieson talks about therapies based on cancer stem cells (4:32)

Stanford Publication: The true seeds of cancer

UCSD Publication: From Bench to Bedside in One Year: Stem Cell Research Leads to Potential New Therapy for Rare Blood Disorder

Updated 2/16

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Advance Stem Cell Therapy in India | Stem Cell Treatment …

By Dr. Matthew Watson

Plan your Stem Cell Therapy in India with Tour2India4Health Consultants

Stem cell therapy in India is performed by highly skilled and qualified doctors and surgeons in India. Our hospitals have state-of-art equipment that increase success rate of stem cell treatment in India. Tour2India4Health is a medical value provider that offers access to the stem cell therapy best hospitals in India for patients from any corner of the world. We offer low cost stem cell therapy at the best hospitals in India.

Stem cells have the ability to differentiate into specific cell types. The two defining characteristics of a stem cell are perpetual self-renewal and the ability to differentiate into a specialized adult cell type.

Serving as a sort of repair system, they can theoretically divide without limit to replenish other cells for as long as the person or animal is still alive. When a stem cell divides, each "daughter" cell has the potential to either remain a stem cell or become another type of cell with a more specialized function, such as a muscle cell, a red blood cell, or a brain cell.

There are three classes of stem cells i.e totipotent, pluripotent and multipotent (also known as unipotent).

Many different terms are used to describe various types of stem cells, often based on where in the body or what stage in development they come from. You may have heard the following terms:

Adult Stem Cells or Tissue-specific Stem Cells: Adult stem cells are tissue-specific, meaning they are found in a given tissue in our bodies and generate the mature cell types within that particular tissue or organ. It is not clear whether all organs, such as the heart, contain stem cells. The term adult stem cells is often used very broadly and may include fetal and cord blood stem cells.

Fetal Stem Cells: As their name suggests, fetal stem cells are taken from the fetus. The developing baby is referred to as a fetus from approximately 10 weeks of gestation. Most tissues in a fetus contain stem cells that drive the rapid growth and development of the organs. Like adult stem cells, fetal stem cells are generally tissue-specific, and generate the mature cell types within the particular tissue or organ in which they are found.

Cord Blood Stem Cells: At birth the blood in the umbilical cord is rich in blood-forming stem cells. The applications of cord blood are similar to those of adult bone marrow and are currently used to treat diseases and conditions of the blood or to restore the blood system after treatment for specific cancers. Like the stem cells in adult bone marrow, cord blood stem cells are tissue-specific.

Embryonic Stem Cells: Embryonic stem cells are derived from very early embryos and can in theory give rise to all cell types in the body. While these cells are already helping us better understand diseases and hold enormous promise for future therapies, there are currently no treatments using embryonic stem cells accepted by the medical community.

Induced Pluripotent Stem Cells (IPS cells): In 2006, scientists discovered how to reprogram cells with a specialized function (for example, skin cells) in the laboratory, so that they behave like an embryonic stem cell. These cells, called induced pluripotent cells or IPS cells, are created by inducing the specialized cells to express genes that are normally made in embryonic stem cells and that control how the cell functions.

Embryonic stem cells are derived from the inner cell mass of a blastocyst: the fertilized egg, called the zygote, divides and forms two cells; each of these cells divides again, and so on. Soon there is a hollow ball of about 150 cells called the blastocyst that contains two types of cells, the trophoblast and the inner cell mass. Embryonic stem cells are obtained from the inner cell mass.

Stem cells can also be found in small numbers in various tissues in the fetal and adult body. For example, blood stem cells are found in the bone marrow that give rise to all specialized blood cell types. Such tissue-specific stem cells have not yet been identified in all vital organs, and in some tissues like the brain, although stem cells exist, they are not very active, and thus do not readily respond to cell injury or damage.

Stem cells can also be obtained from other sources, for example, the umbilical cord of a newborn baby is a source of blood stem cells. Recently, scientists have also discovered the existence of cells in baby teeth and in amniotic fluid that may also have the potential to form multiple cell types. Research on these cells is at a very early stage.

Stem cell therapy is the use of stem cells to treat certain diseases. Stem cells are obtained from the patients own blood bone marrow, fat and umbilical cord tissue or blood. They are progenitor cells that lead to creation of new cells and are thus called as generative cells as well.

The biological task of stem cells is to repair and regenerate damaged cells. Stem cell therapy exploits this function by administering these cells systematically and in high concentrations directly into the damaged tissue, where they advance its self-healing. The process that lies behind this mechanism is largely unknown, but it is assumed that stem cells discharge certain substances which activate the diseased tissue. It is also conceivable that single damaged somatic cells, e.g. single neurocytes in the spinal cord or endothelium cells in vessels, are replaced by stem cells. Most scientists agree that stem cell research has great life-saving potential and could revolutionize the study and treatment of diseases and injuries.

Stem cell therapy is useful in certain degenerative diseases like

If stem cell therapy is an option, a detailed treatment plan is prepared depending on the type of treatment necessary. Once the patient has consented to the treatment plan, an appointment is scheduled for bone marrow extraction. Please note that this is a minimally invasive surgical procedure, so it is important that patients do not take any blood-thinning medication in the ten days prior to the appointment. It is necessary for each patient to consult their own doctor before discontinuing this type of medication.

The treatment procedure include:

Bone Marrow Extraction: Bone marrow is extracted from the hip bone by the physicians. This procedure normally takes around 30 minutes. First, local anesthetic is administered to the area of skin where the puncture will be made. Then, a thin needle is used to extract around 150-200 ml of bone marrow. The injection of local anesthetic can be slightly painful, but the patient usually does not feel the extraction of bone marrow.

Isolation, Analysis and Concentration of the Stem Cells in the Laboratory: The quality and quantity of the stem cells contained in the collected bone marrow are tested at the laboratory. First, the stem cells are isolated. Then a chromatographical procedure is used to separate them from the red and white blood corpuscles and plasma. The sample is tested under sterile conditions so that the stem cells, which will be administered to the patient, are not contaminated with viruses, bacteria or fungi. Each sample is also tested for the presence of viral markers such as HIV, hepatitis B and C and cytomegalia. The cleaned stem cells are counted and viability checks are made. If there are enough viable stem cells, i.e. more than two million CD34+ cells with over 80 percent viability, the stem cell concentrate is approved for patient administration.

Stem Cell Implantation: The method of stem cell implantation depends on the patient's condition. There are four different ways of administering stem cells:

Intravenous administration:

It is important to understand that while stem cell therapy can help alleviate symptoms in many patients and slow or even reverse degenerative processes, it does not work in all cases. Based on additional information, patient's current health situation and/or unforeseen health risks, the medical staff can always, in the interest of the individual patient, propose another kind of stem cell transplantation or in exceptional situations cancel the treatment.

Allogeneic Stem Cell Transplantation: Allogeneic stem cell transplantation involves transferring the stem cells from a healthy person (the donor) to your body after high-intensity chemotherapy or radiation. It is helpful in treating patients with high risk of relapse or who didnt respond to the prior treatment. Allogeneic stem cell transplant cost in India is comparatively less when contrasted with alternate nations.

Autologous Stem Cell Transplant: Patients own blood-forming stem cells are collected and then it is treated with high doses of chemotherapy. The high-dose treatment kills the cancer cells. They are used to replace stem cells that have been damaged by high doses of chemotherapy, used to treat the patient's underlying disease.

The side effects of stem cell therapy differ from person to person. Listed below are the side effects of stem cell therapy :

According to the Indian Council of Medical Research, all is considered to be experimental, with the exception of bone marrow transplants. However, the guidelines that were put into place in 2007 are largely non-enforceable. Regardless, stem cell therapy is legalized in India. Umbilical cord and adult stem cell treatment are considered permissible. Embryonic stem cell therapy and research is restricted.

There is about a 60% to 80% overall success rate in the use of stem cell therapy in both India and around the world. However, success rates vary depending on the disease being treated, the institute conducting the procedures, and the condition of the patient. In order to receive complete information you will have to contact the medical institutes and ask specific questions concerning the patient's condition.

Mrs. Selina Naidoo with her Son from Malaysia

Tour2India4Health has proved to be a blessing in disguise for me. A medical tourism company with everything at par with our expectations has given me the most satisfactory and relieving experience of my life. I went to them for my sons surgery who was suffering from a serious illness and stem cell therapy was the only choice I had. Trust it was heart wrenching to leave my son under any hands on the operation table. Nevertheless, courageously I had to because thats what I was here for and thats what could get my son a new and healthy life. Sitting at a corner outside the operation theatre was taking my heartbeats away with every second. Finally, the surgery was over and I was there in front of the doctor with closed eyes. He declared that the surgery was successful and my son is fine but needs some extra care and some cautious post operative measures for recovery. All through our stay in the hospital, everything went on brilliantly and after my son recovered completely, I came back to my home country. Even after that for many months, I received regular calls to verify and virtually monitor the health of my child. Now, its been 5 years and when I see my child today it feels as if no surgery was ever done on him. Thanks to the doctor who treated him and to the entire team of nurses and travel professionals who displayed extra warmth and care. Thanks is just a small word to say as a mother of a child.

India is the most preferable destination for patients who are looking for low cost stem cell therapy. Indian doctors and healthcare professionals are renowned world over for their skills with many of them holding high positions in leading hospitals in US, UK and other countries around the world. There are significant numbers of highly skilled experts in India, including many who have relocated to India after having worked in the top hospitals across the world.

The Cost of stem cell treatment in India are generally about a tenth of the costs in US and are significantly cheaper compared with even other medical travel destinations like Thailand

*The price for the Stem Cell Therapy is an average collected from the 15 best corporate hospitals and 10 Top Stem Cell Experts of India.

*The final prices offered to the patients is based on their medical reports and is dependent on the current medical condition of the patient, type of room, type of therapy, hospital brand and the surgeon's expertise.

We have worked out special packages of the Stem Cell Therapy for our Indian and International patients. You can send us your medical reports to avail the benefits of these special packages.


There are many reasons for India becoming a popular medical tourism spot is the low cost stem cell treatment in the area. When in contrast to the first world countries like, US and UK, medical care in India costs as much as 60-90% lesser, that makes it a great option for the citizens of those countries to opt for stem cell treatment in India because of availability of quality healthcare in India, affordable prices strategic connectivity, food, zero language barrier and many other reasons.

The maximum number of patients for stem cell therapy comes from Nigeria, Kenya, Ethiopia, USA, UK, Australia, Saudi Arabia, UAE, Uzbekistan, Bangladesh.

Cities where top and world renowned Stem Cell Therapy hospitals and clinics situated are :

We have PAN-India level tie ups with TOP Hospitals for Stem Cell Therapy across 15+ major cities in India. We can provide you with multiple top hospitals & best surgeons recommendations for Stem Cell Therapy in India.

India has now been recognized as one of the leaders in medical field of research and treatment. Tour2India4Health Group was established with an aim of providing best medical services to its patients and since then has been working hard in maintaining itself as one of the most professional healthcare tourism providers in India. With a number of world-renowned medical facilities affiliated, we have the resources to offer you the finest medical treatment in India, and help your speedy recovery. Tour2India4Health Group has always believed and practiced providing its patients best surgery and treatment procedure giving a second chance to live a more better and normal life. Our team serves the clientele most comfortable and convenient measures of healthcare services thus, making your medical tour to India very fruitful experience.

Our facilitation:

We has been operating patients from all major countries like USA, United Kingdom, Italy, Australia, Canada, Spain, New Zealand, and Kuwait etc. We have network of selected medical centers, surgeons and physicians around various cities in India, who qualify our assessment criteria to ensure that our core values of Safety, Excellence and Trust are maintained in all our services.

Below are the downloadable links that will help you to plan your medical trip to India in a more organized and better way. Attached word and pdf files gives information that will help you to know India more and make your trip to India easy and memorable one.

Best Stem Cell Therapy in India, Cost of Stem Cell Therapy in India, Stem Cell Therapy Best Hospitals in India, Success Rate of Stem Cell Treatment in India, Stem Cell Therapy Treatment Cost in India, Allogeneic Stem cell Transplant Cost in India, autologous Stem Cell Transplant Cost in India, Stem Cell Therapy in India, Low Cost Stem Cell Therapy India, Stem Cell Benefits in India, Top Stem Cell Centers in India, Best Doctors for Stem Cell Therapy in India, List of Best Stem Cell Treatment Clinics in India, Allogeneic stem cell transplantation, Allogeneic Stem Cell Transplant Cost in India, Autologous Stem Cell Transplant, Autologous Stem Cell Transplant Cost in India

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Autologous iPS cell therapy for Macular Degeneration: From bench-to-bedside

By Dr. Matthew Watson

Presented At:Gibco - 24 Hours of Stem Cells Virtual Event

Presented By:Kapil Bharti - Stadtman Investigator, NIH, Unit on Ocular Stem Cell & Translational Research

Speaker Biography:Dr. Kapil Bharti holds a bachelor's degree in Biophysics from the Panjab University, Chandigarh, India, a master's degree in biotechnology from the M.S. Rao University, Baroda, India, and a diploma in molecular cell biology from Johann Wolfgang Goethe University, Frankfurt, Germany. He obtained his Ph.D. from the same institution, graduating summa cum laude. His Ph.D. work involved research in the areas of heat stress, chaperones, and epigenetics.

Webinar:Autologous iPS cell therapy for Macular Degeneration: From bench-to-bedside

Webinar Abstract:Induced pluripotent stem (iPS) cells are a promising source of personalized therapy. These cells can provide immune-compatible autologous replacement tissue for the treatment of potentially all degenerative diseases. We are preparing a phase I clinical trial using iPS cell derived ocular tissue to treat age-related macular degeneration (AMD), one of the leading blinding diseases in the US. AMD is caused by the progressive degeneration of retinal pigment epithelium (RPE), a monolayer tissue that maintains vision by maintaining photoreceptor function and survival. Combining developmental biology with tissue engineering we have developed clinical-grade iPS cell derived RPE-patch on a biodegradable scaffold. This patch performs key RPE functions like phagocytosis of photoreceptor outer segments, ability to transport water from apical to basal side, and the ability to secrete cytokines in a polarized fashion. We confirmed the safety and efficacy of this replacement patch in animal models as part of a Phase I Investigational New Drug (IND)-application. Approval of this IND application will lead to transplantation of autologous iPS cell derived RPE-patch in patients with the advanced stage of AMD. Success of NEI autologous cell therapy project will help leverage other iPS cell-based trials making personalized cell therapy a common medical practice.

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Human iPS cell-derived dopaminergic neurons function in a …

By Dr. Matthew Watson

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Human iPS cell-derived dopaminergic neurons function in a ...

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Stem Cell Therapy for Neuropathy: What Can We Expect …

By Dr. Matthew Watson

As the body ages, its only natural that some of its processes should break down. Humans become clumsier, stiffer, their reaction times slower, their senses duller. This is often due to the fact that nerves in the extremities grow less sensitive over time, transmitting messages to the brain more slowly and feeling less acutely a condition known as peripheral neuropathy or simply neuropathy.

While some of that is normal, especially in the golden years, neuropathy often manifests in people much too young in their 30s, 40s, or 50s as a result of a disease such as diabetes or autoimmune issues. Unfortunately, the condition can significantly hamper a persons quality of life, making mobility difficult and limiting everyday activities.

The good news? Neuropathy may have a cure, or at least a solid treatment, on the horizon. Stem cells show great promise for a wide variety of conditions, and nerve damage is the latest of these. To see how it can help, its important to understand what stem cell treatment is, what neuropathy is and what causes it, and how the former can address the latter.

In this article:

The body is made of trillions of tissue-specific cells, making up organs, skin, muscle, bone, nerves, and all other tissue. Some of these can renew indefinitely, such as blood cells. Others, however, cannot replace themselves: Once they have divided a certain number of times or become damaged, theyre dead for good. That goes for nerves and brain tissue, for example.

There is, however, an answer. The developing embryo uses stem cells, or master cells capable of differentiating into any kind of tissue in the human body, to transform one fertilized egg into a fully functional baby human. While adult humans lack these pluripotent stem cells that can transform into anything, they do have multipotent stem cells, which are tissue-specific master cells (such as blood cells).

By harvesting these multipotent stem cells from blood or fat tissue, scientists can induce the cells to become pluripotent, meaning theyre now capable of becomingany tissue in the human body. Essentially, researchers have figured out how to reverse-engineer adult stem cells to become all-powerful embryonic cells. This meansstem cells have a huge range of possible uses.

In other cases, multipotent stem cells alone are enough to heal some parts of the human bodysuch as nerves.

Peripheral neuropathymanifests in a number of ways. It causes pain, weakness, and tingling in affected areas, making it hard to lift objects, grasp items, walk competently, and more. Typically it affects the hands and feet most strongly, though it can also cause symptoms in the arms, legs, and face. Not only does it affect motor coordination,but it also makes it hard for the body to sense the environment, including temperature, pain, vibration, and touch.

A more serious manifestation of the disease is autonomic neuropathy, which influences more than the periphery of the body. It also messes with blood pressure, bladder and bowel function, digestion, sweating, and heart rate. Polyneuropathy is when the condition starts at the periphery of the body but gradually spreads inward.

Diabetic neuropathy is the most well-known incarnation of this disease. It is a result of high glucose and fat levels in the blood, which can damage nerves.Other causes include:

If the bad news is there are so many potential causes of neuropathy, the good news is stem cell treatments have the potential to address all of them.

In the case of neuropathy, stem cell treatment is simpler than in other conditions. Mesenchymal stem cells (certain types of multipotent stem cells) releaseneuroprotective and neuroregenerative factors, so when they are injected into the bloodstream they can begin to rebuild nerves and undo the damage caused by the disease. Also, because these stem cells replicate indefinitely, they will offer these benefits for the rest of the patients life.

The basic process is that scientists harvest these cells from the patient (autologous transplant) or from a donor (allogeneic transplant), then cultivate them until they reach certain levels before reinjecting them back into the patient. The stem cells, with the help of hormones and growth factors, seek out and repair the damage done by neuropathy.

The main risks to stem cell treatment include reaction to the injection. In an autologous transplant, the patient may react to the preservatives and other chemicals used by way of necessity. In an allogeneic transplant, the patient may exhibit an immune response to donor cells, or vice versa with the donor cells seeing the patients body as an invader and attacking it. All of the above reactions can prove minor or, on the other end of the spectrum, fatal.

The severity of the problem will, therefore, dictate whether or not it is worth moving forward. Note that those whodochoose to pursue the treatment often have extremely good results.

Unlike some other stem cell treatments, which remain in preliminary stages, stem cell therapy for neuropathy has thus far received serious attention. However, thesmall sample size and difficult conditions of clinical trialsmake it hard to say yet whether this treatment will become widespread or receive FDA approval.Other studies have demonstrated more significant resultsin the treatment of facial pain and may pave the way for future neuropathy treatments using stem cells.

For now, those suffering from neuropathy should seek the advice of a physician. If there are clinical trials available nearby, thats the place to start. Its possible to seek stem cell therapy through a clinic as well as through a clinical study or research institution, but make sure to research the provider thoroughly. With stem cells becoming such a relevantapproach to medical conditions of all kinds, its not safe to conclude that all providers are equally experienced or effective.

If you found this blog valuable, subscribe to BioInformants stem cell industry updates.

As the first and only market research firm to specialize in the stem cell industry, BioInformant research is cited by The Wall Street Journal, Xconomy, AABB, and Vogue Magazine. Bringing you breaking news on an ongoing basis, we encourage you to join more than half a million loyal readers, including physicians, scientists, executives, and investors.

Did this article address your concerns about neuropathy? Let us know in the comments section below.

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Stem Cell Therapy for Neuropathy: What Can We Expect

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Stem Cell Therapy for Neuropathy: What Can We Expect ...

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What is CAR-T Cell Therapy | CAR-T Definition | Bioinformant

By Dr. Matthew Watson

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Japan’s Laws Supporting Accelerated Pathways for Cell …

By Dr. Matthew Watson

In late 2014, Japan passed two new laws that revolutionizedthe commercialization ofcell therapies within the country by providing an accelerated pathway for product approvals. While there has been much discussion about these laws, few people have a clear understanding of the implications of these regulations on a global scale.

Below, we summarize the laws, identify their importance, and most importantly, speak to howJapan has become a gateway country for regenerative medicines.

New regulations accelerating the approval of regenerative therapeutics in Japan took effect November 25, 2014. The significance of these regulations is that they allow companies to receive conditional marketing approval and commercialize regenerative medicine products while clinical trials continue through the later stages.

The accelerated commercialization of cell therapies is part of the economic revitalization plan initiated by Prime Minister Shinz Abe. Under Shinz Abe, Japan has been pursuing regenerative medicine and cellular therapy as key strategies to the Japans economic growth. Japans Education Ministry also indicated that it is planning to spend 110 billion yen ($1.13 billion) on iPS cellresearch during the next 10 years, and the Japanese parliament has been discussing bills that would speed the approval process and ensure the safety of such treatments.[1]

In late 2014, Japan exercised the following acts:

The aim of the first act was to accelerate the clinical application and commercialization of innovative regenerative medicine therapies. It covers clinical research and medical practice using processed cells and specifies the procedure required for clearance to administer cell procedures to humans. These guidelines are very important to the use the cells within clinical stages.

The PMD Acts definition of regenerative medicine includes tissue-engineered products, cell therapy products, and gene therapy products.

The intent of the laws is to accelerate the commercialization of cell therapeutics within Japan by allowing companies to benefit from conditional marketing authorization.

Therefore, cell therapies that show safety and probable efficacy during Phase I and Phase II trials can get conditional approval for up to seven years, during which time:

1) Larger-scale, later-stage clinical trials are performed2) Revenue from the cell therapy is pursued within the Japanese market

During the seven-year conditional approval period, companies must continue to submit clinical trial data to Japans Pharmaceuticals and Medical Devices Agency (PMDA), and subsequentlyapply for final marketing approval or withdraw the product within seven years.

This safety data can then be used by non-Japanese participants, which is a massive benefit to foreign companies, such as those located in the United States. The regulatory environment in Japan provides companies with the unique opportunity to fast track a clinical trial and seek approval of a new cell therapy product within the Japanese market.

As Kaz Hirao, CEO of Cellular Dynamics International (CDI), shared with BioInformant:This has made Japan a gate country for developing innovative cell therapies with the potential to address major unmet medical needs. It has has provided a strategic opportunity to American companies, because they can benefit from fast track applications through doing clinical testing within Japan and subsequently developing its cell therapy across the rest of the world. Numerous American and Australian companies are pursuing this strategy, as well as other companies from other countries worldwide.[2]

Footnotes[1] Dvorak, K. (2014).Japan Makes Advance on Stem-Cell Therapy[Online]. Available at: Web. 8 Apr. 2015.[2]Interview with Kaz Hirao, CEO of Cellular Dynamics International (CDI), a FUJIFILM Company. Conducted by BioInformants President/CEO, Cade Hildreth [January 29, 2017]. Available at:

Japans Laws Supporting Accelerated Pathways for Cell Therapies

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