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Surge in the Adoption of Stem Cell-Derived Cells to Fuel the Growth of the Stem Cell-Derived Cells Market Through the Assessment Period 2019 2029 -…

The comprehensive report published by Persistence Market Research offers an in-depth intelligence related to the various factors that are likely to impact the demand, revenue generation, and sales of the Stem Cell-Derived Cells Market. In addition, the report singles out the different parameters that are expected to influence the overall dynamics of the Stem Cell-Derived Cells Market during the forecast period 2019 2029.

As per the findings of the presented study, the Stem Cell-Derived Cells Market is poised to surpass the value of ~US$ XX by the end of 2029 growing at a CAGR of ~XX% over the assessment period. The report includes a thorough analysis of the upstream raw materials, supply-demand ratio of the Stem Cell-Derived Cells in different regions, import-export trends and more to provide readers a fair understanding of the global market scenario.

ThisPress Release will help you to understand the Volume, growth with Impacting Trends. Click HERE To get SAMPLE PDF (Including Full TOC, Table & Figures) athttps://www.persistencemarketresearch.co/samples/28780

The report segregates the Stem Cell-Derived Cells Market into different segments to provide a detailed understanding of the various aspects of the market. The competitive analysis of the Stem Cell-Derived Cells Market includes valuable insights based on which, market players can formulate impactful growth strategies to enhance their presence in the Stem Cell-Derived Cells Market.

Key findings of the report:

The report aims to eliminate the following doubts related to the Stem Cell-Derived Cells Market:

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key players in stem cell-derived cells market are focused on generating high-end quality cardiomyocytes as well as hepatocytes that enables end use facilities to easily obtain ready-made iPSC-derived cells. As the stem cell-derived cells market registers a robust growth due to rapid adoption in stem cellderived cells therapy products, there is a relative need for regulatory guidelines that need to be maintained to assist designing of scientifically comprehensive preclinical studies. The stem cell-derived cells obtained from human induced pluripotent stem cells (iPS) are initially dissociated into a single-cell suspension and later frozen in vials. The commercially available stem cell-derived cell kits contain a vial of stem cell-derived cells, a bottle of thawing base and culture base.

The increasing approval for new stem cell-derived cells by the FDA across the globe is projected to propel stem cell-derived cells market revenue growth over the forecast years. With low entry barriers, a rise in number of companies has been registered that specializes in offering high end quality human tissue for research purpose to obtain human induced pluripotent stem cells (iPS) derived cells. The increase in product commercialization activities for stem cell-derived cells by leading manufacturers such as Takara Bio Inc. With the increasing rise in development of stem cell based therapies, the number of stem cell-derived cells under development or due for FDA approval is anticipated to increase, thereby estimating to be the most prominent factor driving the growth of stem cell-derived cells market. However, high costs associated with the development of stem cell-derived cells using complete culture systems is restraining the revenue growth in stem cell-derived cells market.

The global Stem cell-derived cells market is segmented on basis of product type, material type, application type, end user and geographic region:

Segmentation by Product Type

Segmentation by End User

The stem cell-derived cells market is categorized based on product type and end user. Based on product type, the stem cell-derived cells are classified into two major types stem cell-derived cell kits and accessories. Among these stem cell-derived cell kits, stem cell-derived hepatocytes kits are the most preferred stem cell-derived cells product type. On the basis of product type, stem cell-derived cardiomyocytes kits segment is projected to expand its growth at a significant CAGR over the forecast years on the account of more demand from the end use segments. However, the stem cell-derived definitive endoderm cell kits segment is projected to remain the second most lucrative revenue share segment in stem cell-derived cells market. Biotechnology and pharmaceutical companies followed by research and academic institutions is expected to register substantial revenue growth rate during the forecast period.

North America and Europe cumulatively are projected to remain most lucrative regions and register significant market revenue share in global stem cell-derived cells market due to the increased patient pool in the regions with increasing adoption for stem cell based therapies. The launch of new stem cell-derived cells kits and accessories on FDA approval for the U.S. market allows North America to capture significant revenue share in stem cell-derived cells market. Asian countries due to strong funding in research and development are entirely focused on production of stem cell-derived cells thereby aiding South Asian and East Asian countries to grow at a robust CAGR over the forecast period.

Some of the major key manufacturers involved in global stem cell-derived cells market are Takara Bio Inc., Viacyte, Inc. and others.

The report covers exhaustive analysis on:

Regional analysis includes

Report Highlights:

In order to get a strategic overview of the market,Access Research Methodology Prepared By Experts athttps://www.persistencemarketresearch.co/methodology/28780

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Tags: Stem CStem Cell-Derived Cells MarketStem Cell-Derived Cells Market DynamicsStem Cell-Derived Cells Market GrowthStem Cell-Derived Cells Market KeyplayersStem Cell-Derived Cells Market Trends

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Surge in the Adoption of Stem Cell-Derived Cells to Fuel the Growth of the Stem Cell-Derived Cells Market Through the Assessment Period 2019 2029 -...

Osaka University transplants iPS cell-based heart cells in world’s first clinical trial – The Japan Times

OSAKA An Osaka University team said it has carried out the worlds first transplant of cardiac muscle cells created from iPS cells in a physician-initiated clinical trial.

In the clinical project to verify the safety and efficacy of the therapy using induced pluripotent stem cells, Yoshiki Sawa, a professor in the universitys cardiovascular surgery unit, and colleagues aim to transplant heart muscle cell sheets over the course of three years into 10 patients suffering from serious heart malfunction caused by ischemic cardiomyopathy.

As part of its first step in the project, the team conducted an operation on a patient this month, which was a success. The patient has since moved to the general ward at a hospital.

The cells on the degradable sheets attached to the surface of the patients hearts are expected to grow and secrete a protein that can regenerate blood vessels and improve cardiac function. The iPS cells have already been derived from healthy donors blood cells and stored.

Each sheet is around 4 to 5 centimeters wide and 0.1 millimeter thick.

The team will continue to monitor the patient over the next year.

I hope that (the transplant) will become a medical technology that will save as many people as possible, as Ive seen many lives that I couldnt save, Sawa said at a news conference.

The researchers said Monday they decided to conduct a clinical trial instead of a clinical study in hopes of obtaining approval from the health ministry for clinical applications as soon as possible.

The trial involves stringently evaluating risks, particularly cancer probabilities, and the efficacy of transplanting some 100 million cells per patient that may include tumor cells.

This is the second iPS cell-based clinical trial in Japan. The first was conducted on eye disease patients by the Riken research institute.

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Osaka University transplants iPS cell-based heart cells in world's first clinical trial - The Japan Times

Osaka University-based team successfully conducts first transplantation of cardiac muscle cells around the globe – Medical Herald

A team based at Osaka University stated how it had succeeded in carrying out the first transplant of cardiac muscle cells, around the globe, developed from iPS cells in a clinical trial which as physician-initiated.

A professor in Osaka Universitys cardiovascular surgery unit, Yoshiki Sawa, along with his colleagues at the university, intend to transplant heart muscle cell sheets into 10 individuals experiencing severe heart malfunction as a result of ischemic cardiomyopathy, in a clinical trial, to validate the safety and the effectiveness of the therapy with the use of induced pluripotent stem cells.

On the surface of the hearts of the partaking individuals, the cells on the degradable sheets are attached. It is predicted that these cells will develop to release a protein that can allow for the regeneration of blood vessels as well as the improvement of the cardiac function.

Already, the iPS cells have been taken, and then stored, from the blood cells donated by healthy individuals

On Monday, the researchers stated how they chose to carry out a clinical trial in a clinical researchs stead as they had hoped to attain, as early as possible, authorization from the health ministry for clinical applications.

There are severe evaluating risks involved in the clinical trial. These may include the possibility of cancer as well as the efficacy of transplanting many million cells per patient, which may consist of tumor cells.

In Japan, this will be marked as the second clinical trial based on iPS. The first clinical trial of such kind was carried out on patients suffering from eye-linked ailments. This was done so by the Riken research institute.

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Osaka University-based team successfully conducts first transplantation of cardiac muscle cells around the globe - Medical Herald

Kyoto University team gets OK from ministry for plan to transplant iPS-derived cartilage into knee joints – The Japan Times

KYOTO An expert panel of the health ministry on Friday approved a clinical research program proposed by a Kyoto University team to transplant cartilage made from induced pluripotent stem (iPS) cells to damaged knee joints.

Professor Noriyuki Tsumaki and other members of the team are planning to create cartilage with a diameter of 2 to 3 millimeters using iPS cells stored at the universitys Center for iPS Cell Research and Application (CiRA).

The team aims to carry out the first transplant this year. After a clinical trial by Asahi Kasei Corp., which supports the project, it hopes to put the technology into practical use in 2029.

Four people between the ages of 20 and 70 will undergo transplant operations using iPS cell-derived cartilage for their damaged knee joints, with the area of damage ranging from 1 centimeter to 5 centimeters. The team does not plan to seek additional patients for the program.

The team will monitor the four patients for one year after the operations to keep an eye out for possible development of tumors. If the operations succeed, the transplanted material will fuse with existing cartilage.

There are many patients experiencing inconvenience due to damaged cartilage, Tsumaki told a news conference at the Kyoto University Hospital on Friday. Well work hard so that we can offer therapy methods.

The team will also aim to apply the therapy to patients with osteoarthritis.

In 2014, Riken, a Japanese government-affiliated research institute, transplanted retina cells made from iPS cells as a treatment for an incurable eye disease, in the worlds first transplant of iPS-derived cells.

Later, similar transplant operations were conducted by Kyoto University for Parkinsons disease and by Osaka University for corneal disease.

Excerpt from:
Kyoto University team gets OK from ministry for plan to transplant iPS-derived cartilage into knee joints - The Japan Times

The Kyoto University team’s plan to transplant iPS cartilage into knee joints is OK – gotech daily

KYOTO A panel of experts from the Ministry of Health approved a clinical research program proposed by a team from the University of Kyoto on Friday for the transplantation of cartilage from induced pluripotent stem cells [iPS] into damaged knee joints.

Professor Noriyuki Tsumaki and other members of the team are planning to produce 2 to 3 millimeter diameter cartilage using iPS cells, which will be stored at the Universitys Center for iPS Cell Research and Application [CiRA].

The team plans to perform the first transplant this year. According to a clinical study by Asahi Kasei Corp., which supports the project, the technology should be put into practice in 2029.

Four people between the ages of 20 and 70 are transplanted with iPS cell cartilage for their damaged knee joints, with the damage range between 1 cm and 5 cm. The team does not plan to seek additional patients for the program.

Immunosuppressors are not used in the transplant because cartilage usually does not show an immune response.

The team will monitor the four patients for possible tumor development for a year after the operation. If the operations are successful, the transplanted material melts into the existing cartilage.

There are many patients who experience discomfort from cartilage damage, said Tsumaki at a press conference at Kyoto University Hospital on Friday. We will work hard to offer therapy methods.

The team will also try to apply the therapy to patients with osteoarthritis.

In 2014, Riken, a research institute affiliated with the Japanese government, transplanted retina cells made from iPS cells to treat an incurable eye disease in the worlds first transplant of iPS-derived cells.

Similar transplants were later performed by Kyoto University for Parkinsons and Osaka University for corneal diseases.

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The Kyoto University team's plan to transplant iPS cartilage into knee joints is OK - gotech daily

Update on stem cell treatment cost for 2018 from ongoing …

I get asked many questions about stem cell therapies, but one of the most common over the years has been about the stem cell treatment cost. For instance, a reporter might ask, How much does a stem cell treatment for MS cost? and a patient might ask me, How much is a fair cost for a stem cell therapy for arthritis? Or, patients will voluntarily tell me what they paid or mention it in the comments. We hear various numbers thrown around about costs so I decided to do a poll on this. I even did an early update on the results of this poll, voicing my skepticism that the costs paid were worth it.

But the poll has gotten well over 500 responses now so I thought I would revisit it and what it might mean.

You can see a screenshot of the images. Its fair to say, as much as Internet polls arent considered particularly accurate, that this one largely fits with what is reported out in the field.

(On a side note, I wish there was such a thing as going out into the field for stem cell scientists as Ive always been a bit jealous of scientists who really do go out in the field. What do we do, go out in the wild and catch wild or feral stem cells in the bush?)

Patients self-reported most often paying between $2,500 and $7,500 for their stem cell therapy so if we take the average of those we get that $5,000 figure that is what I hear most often from others. Yes, not necessarily very rigorous, but the result makes good sense. Not far behind though were responses in the $7,500-20,000 range.

About 1 in 10 respondents reported paying $20,000 or more, including some beyond $100,000. Thats a whopping stem cell treatment cost, especially for something most often unproven and unapproved by the FDA.

If we consider these responses, the average cost may be more like $7,500-$10,000.

Notably, about 1/16 respondents indicated their stem cells were free. Im not sure what that means in terms of how that came to be.

Interestingly, most respondents who also went on to answer a 2nd poll in that post about where they got the treatment indicate it was at a stem cell clinic (scroll down in that Oct. 2017 post and youll see the 2nd poll). This 2nd poll has about 200 responses.

So today buying a simple stem cell treatment, most often unproven and non-FDA approved, is often not so different in cost than buying a 10-year old used car, while less often it is similar to buy various new cars including at the high end of stem cell therapy cost, some very expensive new cars. This cost and the risks involved are why I have suggested to patients in the past to be assertive when considering a stem cell treatment, ask questions, dont just accept too good to be true kinds of answers, etc. In short, be at least (or ideally much more) rigorous about unproven stem cell treatments as you are about buying a car.

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Update on stem cell treatment cost for 2018 from ongoing ...

Psychiatric body condemns use of stem cell therapies to treat psychiatric disorders – Moneycontrol.com

The Indian Psychiatric Society (IPS) the professional body that represents psychiatrists in India, strongly condemned the use of stem cell therapy in psychiatric disorders, particularly autism, until such a time that research evidence substantiated its effectiveness.

IPS, in its position statement on stem cell therapy on January 17, said that till now, there is no scientifically validated and scrutinized research evidence that proves that stem cells are helpful in any psychiatric disorders including autism.

Autism is a complex neurodevelopmental disorder with no known single cause.

The advisory from the IPS comes at a time when stem cell therapy clinics that claim to have developed stem cell therapies to treat complex psychiatric problems such as autism, cerebral palsy (movement disorder), muscular dystrophy (weakness of muscles), mental retardation, spinal cord injury and brain stroke have mushroomed across the country.

These stem cell therapy centres extract stem cells from the bone marrow of each child and then inject it into the childs spinal canal. The whole procedure takes place under general anaesthesia.

These clinics use aggressive marketing techniques and false claims to lure parents of children who are suffering from disease like autism.

The Indian Council of Medical Research (ICMR) has already published guidelines that cover the various diseases that are applicable for stem cell treatment. No psychiatric disorders, including autism, are listed there under this advisory.

Stem cells are special human cells that have the ability to develop into many different cell types, from muscle cells to brain cells. In some cases, they also have the potential to repair damaged tissues, and provide a cure for various diseases. But the clinical evidence at this point is low.

Psychiatric disorders including autism are combined derangements of both neurodevelopmental and neurodegenerative trajectories of brain and are polygenetic in origin. So they actually are symptomatic manifestations of a variety of different pathogenetic processes about which scientific evidence is as yet inconclusive, IPS said.

Originally posted here:
Psychiatric body condemns use of stem cell therapies to treat psychiatric disorders - Moneycontrol.com

Allele and Astellas Enter into an Expanded License for the Development of iPSC Lines – BioSpace

SAN DIEGO--(BUSINESS WIRE)-- Allele Biotechnology and Pharmaceuticals, Inc. (President and CEO: Jiwu Wang, Ph.D., Allele), a San Diego-based private company, and Astellas Pharma Inc. (TSE: 4503, President and CEO: Kenji Yasukawa, Ph.D., Astellas), through its Massachusetts-based subsidiary Astellas Institute for Regenerative Medicine (AIRM), entered into a licensing agreement to expand Astellas access to Alleles induced pluripotent stem cell (iPSC) technologies for various cell therapy programs.

Astellas, one of the largest pharmaceutical companies in Japan and already a leader in the development of cell-based therapeutics, has further dedicated to development of the field through its commitment to state-of-the-art iPS cell generation, modification, and manufacturing. iPSC lines can differentiate into all somatic tissue types, enabling a wide variety of therapeutic applications. The field of iPSC-derived cells has seen dramatic growth in clinical trials recently--the majority of the ~12 clinical trials around the world were initiated within the last 18 months and many more are upcoming.

Allele has been developing its core strength in reprogramming somatic cells into iPSCs with granted patents and the first commercial cGMP system it developed over the past 10 years. Allele also engages in more than a dozen different human tissue derivation activities through its own R&D efforts for internal programs and partnerships. To realize the unparalleled potential of iPSC, Alleles researchers and cGMP team are committed to setting up and validating cell assays for product quality control, genome analysis pipelines, closed-system automation for reprogramming, and machine learning in iPSC-related fields.

Under the terms of the new license agreement, Astellas will pay Allele upfront and milestones, product-based royalties, and potentially manufacture fees.

About Allele Allele Biotechnology and Pharmaceuticals was founded in 1999. In 2015, the company completed an 18,000 square foot state-of-the-art facility in San Diego for the production of GMP-grade human iPSC lines. The facility also supports the production of tissue-specific cells differentiated from these iPSCs, including pancreatic beta cells, neural progenitor cells, and cardiomyocytes.

View source version on businesswire.com: https://www.businesswire.com/news/home/20200113005668/en/

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Allele and Astellas Enter into an Expanded License for the Development of iPSC Lines - BioSpace

Why stem cells could be the medical innovation of the century – World Economic Forum

Right now, your bodys stem cells are working hard replacing your skin every two weeks, creating new red and white blood cells and completing thousands of other tasks essential to life. They are your own personalized fountain of youth.

Scientists generally agree that a stem cell should be able to do both of the following:

One theory of ageing suggests that between the ages of 30 and 50, our stem cells reach a turning point and start to decline in number and function. This results in the typical features associated with ageing.

There does not seem to be a single discoverer of stem cells. Accounts date back to the 1800s and even further, but the first successful medical procedure was a bone marrow transfusion in 1939. Advances in immunology led to donor matching, initially via siblings and close relatives. Unrelated donor matching flourished in the 1970s, alongside donor registries.

In the 1980s, scientists identified embryonic stem cells in mice, leading to the 1997 cloning of Dolly the Sheep. This created immense interest for human and medical applications and a backlash in the US as federal R&D funding was essentially halted in 2001.

In 2012, a Nobel Prize was awarded for the earlier discovery of induced pluripotent stem cells (iPS). Essentially, they return potency and self-renewal properties to mature non-stem cells, essentially making them act like stem cells again.

In the decade between 2010 and 2019, the first wave of stem cell start-ups emerged, alongside R&D programmes at many large pharmaceutical companies, leading to innovation and the first human clinical trials for iPS and other related therapies.

According to Q3 2019 data from the Alliance for Regenerative Medicine, there are 959 regenerative medicine companies worldwide sponsoring 1,052 active clinical trials; 525 of these companies are in North America, 233 in Europe and Israel, and 166 in Asia. In aggregate, $7.4 billion has been invested in regenerative medicine companies in 2019; $5.6 billion of which has been dedicated to gene and gene-modified cell therapy, $3.3 billion in cell therapy, and $114 million in tissue engineering.

Overview of the cancer stem cells market

Perhaps most excitingly, curative therapies are hitting the market and the results are astonishing: 60% of Acute Lymphoblastic Leukemia patients taking Novartis Kymirah showed a complete response (no traces of cancer) and were declared in full remission. Meanwhile, 75% of patients with Transfusion-Dependent -Thalassaemia treated with bluebird bios Zynteglo achieved independence from transfusions. Perhaps most astonishingly, 93% of spinal muscular atrophy patients treated with Novartis Zolgensma were alive without permanent ventilation 24 months after treatment. We should expect more medical breakthroughs in the coming years.

New science, new start-ups: several companies in the sector have gone public or been acquired. These exits led to the recycling of talent and capital into new companies. Because the science and commercial systems have also advanced, the companies in the next wave are pursuing bigger challenges, driving innovation, with even greater resources.

Patients are eager: the current market for stem cell therapies is growing at 36% per year, though it will rapidly expand when a breakthrough occurs toward the treatment of a non-communicable disease (such as cancer, diabetes, heart disease) or a lifestyle factor (for example, growing hair in the correct places, expanding cognitive abilities or increasing healthy lifespan).

New R&D models: funding is flowing into the sector from large companies, VC funds, and institutions such as the California Institute for Regenerative Medicine (CIRM) and New York State Stem Cell Science programme (NYSTEM). Some of the leading university R&D platforms include the Center for the Commercialization of Regenerative Medicine in Toronto, the Stanford Institute for Stem Cell Biology and Regenerative Medicine, the Oxford Stem Cell Institute, and most notably, the Harvard Stem Cell Institute (HSCI).

Founded in 2004, HSCI has established a phenomenal track record. It provided the first $200,000 in funding to Derrick Rossis lab, which inspired the largest biotech IPO to date. HSCI scientists were also co-founders or principals in the three most prominent gene-editing companies (CRISPR Tx, Intellia and Editas), the combined $1.55-billion True North/iPierian acquisitions and the recent $950-million acquisition of Semma Tx, Frequency Tx, Fate Tx, Epizyme Inc., and Magenta Tx.

For the casual investor, Evercore ISI is building a Regenerative Medicine Index, which may be the simplest way to build a portfolio. For institutions and those with deeper pockets, regenerative medicine funds are forming, including the Boston-centric Hexagon Regenerative Medicine Fund, which aims to create companies out of the Harvard Stem Cell Institute.

Caveat emptor. Though patients needs are immediate, those seeking treatments should think very carefully about the risks. There are many dubious clinics touting expensive stem cell treatments and some patients have experienced horrifying complications. Dr. Paul Knoepfler of UC-Davis has written a practical and scientifically accurate guide, a strongly recommended read if you or a family member are considering treatment or a clinical trial.

The leading causes of death in 1900 were mostly infectious/communicable diseases. While the prevalence of most causes has diminished, the largest increases include heart disease (+40%) and cancer (+300%). Granted, this is partly due to doubling life expectancy and a lack of death from other causes. However, given time and resources, scientists and physicians may cure these challenging diseases.

Total disease burden by disease or injury

Today, six of the seven leading causes of death are non-communicable diseases (heart disease, stroke, lung diseases, cancer, Alzheimers disease and diabetes). Based on the early promise mentioned above, regenerative medicine may be our best hope to solve the great non-communicable diseases of our time, and perhaps the single most transformative medical innovation in a century.

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World Economic Forum articles may be republished in accordance with our Terms of Use.

The views expressed in this article are those of the author alone and not the World Economic Forum.

Excerpt from:
Why stem cells could be the medical innovation of the century - World Economic Forum

Allele and Astellas Enter into an Expanded License for the Development of iPSC Lines – Business Wire

SAN DIEGO--(BUSINESS WIRE)--Allele Biotechnology and Pharmaceuticals, Inc. (President and CEO: Jiwu Wang, Ph.D., Allele), a San Diego-based private company, and Astellas Pharma Inc. (TSE: 4503, President and CEO: Kenji Yasukawa, Ph.D., Astellas), through its Massachusetts-based subsidiary Astellas Institute for Regenerative Medicine (AIRM), entered into a licensing agreement to expand Astellas access to Alleles induced pluripotent stem cell (iPSC) technologies for various cell therapy programs.

Astellas, one of the largest pharmaceutical companies in Japan and already a leader in the development of cell-based therapeutics, has further dedicated to development of the field through its commitment to state-of-the-art iPS cell generation, modification, and manufacturing. iPSC lines can differentiate into all somatic tissue types, enabling a wide variety of therapeutic applications. The field of iPSC-derived cells has seen dramatic growth in clinical trials recently--the majority of the ~12 clinical trials around the world were initiated within the last 18 months and many more are upcoming.

Allele has been developing its core strength in reprogramming somatic cells into iPSCs with granted patents and the first commercial cGMP system it developed over the past 10 years. Allele also engages in more than a dozen different human tissue derivation activities through its own R&D efforts for internal programs and partnerships. To realize the unparalleled potential of iPSC, Alleles researchers and cGMP team are committed to setting up and validating cell assays for product quality control, genome analysis pipelines, closed-system automation for reprogramming, and machine learning in iPSC-related fields.

Under the terms of the new license agreement, Astellas will pay Allele upfront and milestones, product-based royalties, and potentially manufacture fees.

About AlleleAllele Biotechnology and Pharmaceuticals was founded in 1999. In 2015, the company completed an 18,000 square foot state-of-the-art facility in San Diego for the production of GMP-grade human iPSC lines. The facility also supports the production of tissue-specific cells differentiated from these iPSCs, including pancreatic beta cells, neural progenitor cells, and cardiomyocytes.

Excerpt from:
Allele and Astellas Enter into an Expanded License for the Development of iPSC Lines - Business Wire

Where Are They Now? Top 3 Biotech Startups From NextGen Bio Class of 2018 – BioSpace

Every year, BioSpace analyzes the biotech industry, looking for the hot new biotech startups to watch. We then produce the NextGen Bio Class of, twenty companies ranked based on several categories, including Finance, Collaborations, Pipeline, and Innovation. The companies were typically launched no more than 18 months before the list was created.

We thought it would be insightful to look back at our previous lists to see where some of those companies are today. Heres a look at the top three companies from the Top 20 Life Science Startups to Watch in 2018.

#1. BlueRock Therapeutics. Founded in 2016, BlueRock was #1 on our list of companies to watch in 2018. With facilities in Ontario, Canada; Cambridge, Massachusetts; and New York, New York, BlueRock launched in December 2016 with a $225 million Series A financing led by Bayer AG and Versant Ventures. The company focuses on cell therapies to regenerate heart muscle in patients who have had a heart attack or chronic heart failure, as well as therapies for patients with Parkinsons disease.

In October 2017, BlueRock and Seattle-based Universal Cells entered into a collaboration and license deal to create induced pluripotent stem (iPS) cell lines that can be used in the manufacture of allogeneic cellular therapies. Shortly afterwards, the company established its corporate headquarters in Cambridge, and in April 2018, established a research-and-development hub in New York City, as well as formalizing a sponsored research collaboration with the Center for Stem Cell Biology at Memorial Sloan Kettering (MSK) Cancer Center. The collaboration focuses on translating Ketterings expertise in creating multiple types of authentic neural cells from stem cells to address diseases of the central and peripheral nervous system. BlueRock also received $1 million from the State of New York and Empire State Development under its economic development initiatives program.

In April 2019, BlueRock partnered with Editas Medicine (which was on BioSpaces NextGen Bio Class of 2015 list) to combine their genome editing and cell therapy technologies to focus on novel engineered cell medicines. Part of the deal was to collaborate on creating novel, allogeneic pluripotent cell lines using a combination of Editas CRISPR genome editing technology and BlueRocks iPSC platform.

And finally, in August 2019, Bayer AG acquired BlueRock for the remaining stake in the company for about $240 million in cash and an additional $360 million in pre-defined development milestones.

#2. Prelude Fertility. Prelude Fertility is a bit of an outlier from the typical BioSpace NextGen company, because it isnt quite a biopharma company. It is a life sciences company whose business model is aimed at in vitro fertilization and egg freezing. It was founded with a $200 million investment by entrepreneur Martin Varsavsky. The investment was in the largest in vitro fertilization clinic in the Southeast, Reproductive Biology Associates of Atlanta, and its affiliate, My Egg Bank, the largest frozen donor egg bank in the U.S.

Since then it has expanded in various parts of the country, including adding San Francisco-based Pacific Fertility Center (PFC) to its network in September 25, 2017; partnering with Houston Fertility Institute and acquiring Vivere Health; partnering with the Advanced Fertility Center of Chicago; and in October 2018, partnered with NYU Langone Health.

In March 2019, Prelude merged with Inception Fertility to establish the Prelude Network as the fastest-growing network of fertility clinics and largest provider of comprehensive fertility services in the U.S. Inception is acting as the parent company, with the Prelude Network, both having board representatives from the previous organizations.

#3. Relay Therapeutics. Ranking #3 on our list for 2018, Relay Therapeutics launched in September 2016 with a $57 million Series A financing led by Third Rock Ventures with participation form D.E. Shaw Research. On December 14, 2017, it closed on a Series B round worth $63 million, led by BVF Partners, with new investors GV (formerly Google Ventures), Casdin Capital, EcoR1 Capital and Section 32.

The company focuses on the relationship between protein motion and function. It merges computational power with structural biology, biophysics, chemistry and biology. In December 2018, the company completed a $400 million Series C financing. It was led by the SoftBank Vision fund and included additional new investors, Foresite Capital, Perceptive Advisors and Tavistock Group. Existing investors also participated.

The company announced at the time it planned to use the funds to accelerate the implementation of its long-term strategy, expanding its discovery efforts, advancing existing programs into the clinic and improving its platform.

Read more:
Where Are They Now? Top 3 Biotech Startups From NextGen Bio Class of 2018 - BioSpace

Treating a tricky skin disease | Interviews – The Naked Scientists

Imagine if your skin was so fragile that even the slightest knock caused it to blister and tear. This is the reality for people with a condition called epidermolysis bullosa. It occurs when a person inherits faulty copies of the genes that make the crucial skin protein collagen. But help may now be at hand,because Columbia University researcher Joanna Jackow has found a way to make stem cells, called iPS cells, from patients skin cells; edit the faulty genes in the stem cells, and use the now-repaired cells to grow new, healthy skin. It's the first step towards skin replacements for patients with these sorts of genetic skin diseases...

Joanna - Patients have an extensive blistering of the skin because they were born with this mutation. The skin starts to blister right after birth. These blisters are chronic wounds that are not healed, and these chronic ones convert to extensive scarring and, finally, with increasing age, the patients get a skin cancer called squamous cell carcinoma.

Chris - What's the approach you've taken to try to put this right?

Joanna - Using this magic genetic scissors called CRISPR, we can fix this mutation in cells called induced pluripotent stem cells, which are cells that we can generate from the patient's own cells. Because the cells have a potential of differentiation to any cell type we want, in our case skin cells, we can develop skin equivalents, which we called grafts, and these skin equivalents can be grafted onto the wounded areas of the skin.

Chris - So you're saying 'make some stem cells, fix the gene problem in those stem cells, and then grow new rafts of skin from the fixed stem cells so that you've got new skin to put on to the individuals with the condition?

Joanna - That's correct.

Chris - How do you go, though, from those "fixed" skin cells into actually making skin?

Joanna - Yes, we take the right cells now and put them together in a matrix called collagen, and the cells will grow into a normal skin that we called a skin-equivalent; and the skin equivalent can be grafted on the patients.

Chris - Have you tested this though, in the sense that: you've got these patches of skin-equivalents, do they survive in the long term and for instance, if you put them onto an animal in place of its own skin, do they work?

Joanna - Yes. We used for this immune deficient mouse model, which is a model which doesn't have immune system and will not reject this graft. And we've been testing the survival of this graft two months post grafting and we could demonstrate that the grafts survived and produced this protein that was missing in previously in the patient's skin.

Chris - In other words, the implication is, were you to do this in a patient, because it would be their own cells, there wouldn't be an immune problem. So you could just put these skin patches on in place of the individual's injured skin, and it should take over the function of their injured skin and give them a healthy working skin?

Joanna - Exactly. That's exactly what is the concept of our strategy.

Chris - Big problem though, when you consider how big a person is, I mean the surface area of a human that's, you know, metres squared of skin, isn't it? So is it feasible to actually do this on the scale of the entire body? Because you'd have to replace all their skin, wouldn't you?

Joanna - Yes, this is an excellent question and we've been already thinking of this. So, we would like to first cover the large wounds of the patient's body and we hope that, because we are deriving the skin equivalents from keratinocytes, that - hopefully - have also a population of stem cells. Eventually, these grafts can take over and cover the whole body of the patient.

Chris - Thing is, skin isn't just skin-producing cells, is it? There's hair follicles in there; there are more complicated structures, like sweat glands, as well. Those aren't going to be present in the grafts you make, are they?

Joanna - That's what we are thinking as a next step, to make more complex skin including all these very important components. As you mentioned, hair follicle and sweat glands. This is what we keep in mind in the future...

Continued here:
Treating a tricky skin disease | Interviews - The Naked Scientists

Global Stem Cell Therapy Market to Surpass US$ 40.3 Billion by 2027 Coherent Market Insights – Business Wire

SEATTLE--(BUSINESS WIRE)--According to Coherent Market Insights, the global stem cell therapy market was valued at US$ 7,313.6 million in 2018, and is expected to exhibit a CAGR of 21.1% over the forecast period (2019-2027).

Key Trends and Analysis of the Stem cell therapy Market:

Key trends in market are increasing incidence of cancer and osteoporosis, rising number of research and development activities for product development, and adoption of growth strategies such as acquisitions, collaborations, product launches by the market players.

Key players are focused on launches of production facility for offering better stem cell therapy in the potential market. For instance, in January 2019, FUJIFILM Cellular Dynamics, Inc., a subsidiary of FUJIFILM Corporation, announced to invest around US$ 21 Mn for building new cGMP-compliant production facility, in order to enhance production capacity of induced pluripotent stem (iPS) cell for the development of cell therapy and regenerative medicine products. The new facility is expected to begin its operations by March 2020.

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Market players are adopting inorganic growth strategies such as acquisitions and collaborations, in order to enhance their offerings in the potential market. For instance, in August 2019, Bayer AG acquired BlueRock Therapeutics, a company developing cell therapies based on induced pluripotent stem cell (iPSC) platform. This acquisition is expected to strengthen Bayers market position in the stem cell therapy market.

Furthermore, increasing research and development activities of stem cells by research organizations to provide efficient treatment options to patients suffering from various chronic diseases is expected to drive growth of the stem cell therapy market over the forecast period. For instance, in January, 2019, the Center for Beta Cell Therapy in Diabetes and ViaCyte, Inc. initiated a trial of human stem cell-derived product candidates in type 1 diabetes patients in Europe.

However, high cost of preservation of stem cells and other factors is expected to hamper growth of stem cell therapy market over the forecast period. High cost of stem cell storage is a factor that is expected to hinder growth of the market. For instance, according to the Meredith Corporation, a private bank generally charges US$ 1,200 to US$ 2,300 to collect cord blood at the time of delivery, with annual storage fees of US$ 100 to US$ 300 each year. Thus, high cost associated with stem cell storage combined with high production cost are expected to hinder growth of the market, especially in emerging economies.

Key Market Takeaways:

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Market Segmentations:

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Global Stem Cell Therapy Market to Surpass US$ 40.3 Billion by 2027 Coherent Market Insights - Business Wire

Lineage Cell Therapeutics and AgeX Therapeutics Announce Issuance of US Patent for Method of Generating Induced Pluripotent Stem Cells – BioSpace

The issuance of this patent highlights Lineages dominant position in the field of cell therapy, stated Brian M. Culley, CEO of Lineage. Our efforts to develop new treatments rely on well-characterized and NIH-approved human cell lines. These lines are not genetically manipulated, which avoids the safety concerns associated with genetic aberrations arising from the creation of iPS cells. We believe the Lineage cell lines provide the safest option for our current clinical-stage programs, particularly in immune-privileged anatomical sites such as the eye (OpRegen for the treatment of dry AMD) and spinal cord (OPC1, for the treatment of spinal cord injury). However, the vast intellectual property estate which underlies our cell therapy platform has never been limited to these particular cell lines. As one example, this newly-issued patent provides us with proprietary methods for producing induced pluripotent stem cells, or, as it was practiced by us prior to Yamanaka, Analytical Reprogramming Technology (ART). In certain settings, an ART/iPS approach might offer important advantages, such as for an autologous treatment or when the selection of preferential attributes from a series of iPS lines is desirable. Questions as to which stem cell technology is preferred ultimately will be answered by clinical safety and efficacy and likely will be indication-specific, so we believe it is in the best interest of our shareholders to generate patented technology which enables us to pursue programs in either or both formats which we believe will ensure the highest probability of success.

This patent broadly describes multiple techniques for reprogramming cells of the body back to the all-powerful stem cell state, said Dr. Michael D. West, CEO of AgeX and first inventor on the patent. Perhaps more significantly, it includes certain factors that address some of the difficulties currently encountered with iPS cells. It also reflects the foundational work our scientists have undertaken to apply reprogramming technology to age-reversal, specifically, induced Tissue Regeneration (iTR) which is currently a focus of AgeX product development.

Induced Pluripotent Stem Cells (iPS) are typically derived from adult skin or blood cells which have been reprogrammed or induced to retrace their developmental age and regain the potential to form all of the young cell and tissue types of the body. In 2010 inventors of the -723 patent issued today demonstrated that this reversal of developmental aging even extended to the telomere clock of cell aging. This reprogramming technology provides an alternate source of starting material for the manufacture of potentially any type of human cell needed for therapeutic purposes. Because iPSCs can be derived directly from adult tissues, they can be used to generate pluripotent cells from patients with known genetic abnormalities for drug discovery or as an alternative source of cell types for regenerative therapies.

U.S. Patent No. 10,501,723, entitled Methods of Reprogramming Animal Somatic Cells was assigned to Advanced Cell Technology of Marlborough, Massachusetts (now Astellas Institute for Regenerative Medicine) and licensed to Lineage and sublicensed to AgeX Therapeutics for defined fields of use. Inventors of the patent include Michael D. West, CEO of AgeX and previous CEO of Advanced Cell Technology, Karen B. Chapman, Ph.D., and Roy Geoffrey Sargent, Ph.D.

About Lineage Cell Therapeutics, Inc.

Lineage Cell Therapeutics is a clinical-stage biotechnology company developing novel cell therapies for unmet medical needs. Lineages programs are based on its proprietary cell-based therapy platform and associated development and manufacturing capabilities. With this platform Lineage develops and manufactures specialized, terminally-differentiated human cells from its pluripotent and progenitor cell starting materials. These differentiated cells are developed either to replace or support cells that are dysfunctional or absent due to degenerative disease or traumatic injury or administered as a means of helping the body mount an effective immune response to cancer. Lineages clinical assets include (i) OpRegen, a retinal pigment epithelium transplant therapy in Phase I/IIa development for the treatment of dry age-related macular degeneration, a leading cause of blindness in the developed world; (ii) OPC1, an oligodendrocyte progenitor cell therapy in Phase I/IIa development for the treatment of acute spinal cord injuries; and (iii) VAC2, an allogeneic cancer immunotherapy of antigen-presenting dendritic cells currently in Phase I development for the treatment of non-small cell lung cancer. Lineage is also evaluating potential partnership opportunities for Renevia, a facial aesthetics product that was recently granted a Conformit Europenne (CE) Mark. For more information, please visit http://www.lineagecell.com or follow the Company on Twitter @LineageCell.

About AgeX Therapeutics

AgeX Therapeutics, Inc. (NYSE American: AGE) is focused on developing and commercializing innovative therapeutics for human aging. Its PureStem and UniverCyte manufacturing and immunotolerance technologies are designed to work together to generate highly-defined, universal, allogeneic, off-the-shelf pluripotent stem cell-derived young cells of any type for application in a variety of diseases with a high unmet medical need. AgeX has two preclinical cell therapy programs: AGEX-VASC1 (vascular progenitor cells) for tissue ischemia and AGEX-BAT1 (brown fat cells) for Type II diabetes. AgeXs revolutionary longevity platform induced Tissue Regeneration (iTR) aims to unlock cellular immortality and regenerative capacity to reverse age-related changes within tissues. AGEX-iTR1547 is an iTR-based formulation in preclinical development. HyStem is AgeXs delivery technology to stably engraft PureStem cell therapies in the body. AgeX is developing its core product pipeline for use in the clinic to extend human healthspan and is seeking opportunities to establish licensing and collaboration agreements around its broad IP estate and proprietary technology platforms. For more information, please visit http://www.agexinc.com or connect with the company on Twitter, LinkedIn, Facebook, and YouTube.

Forward-Looking Statements

Lineage cautions you that all statements, other than statements of historical facts, contained in this press release, are forward-looking statements. Forward-looking statements, in some cases, can be identified by terms such as believe, may, will, estimate, continue, anticipate, design, intend, expect, could, plan, potential, predict, seek, should, would, contemplate, project, target, tend to, or the negative version of these words and similar expressions. Such statements include, but are not limited to, Lineages exploration of alternative cell therapy platforms. Forward-looking statements involve known and unknown risks, uncertainties and other factors that may cause Lineages actual results, performance or achievements to be materially different from future results, performance or achievements expressed or implied by the forward-looking statements in this press release, including risks and uncertainties inherent in Lineages business and other risks in Lineages filings with the Securities and Exchange Commission (the SEC). Lineages forward-looking statements are based upon its current expectations and involve assumptions that may never materialize or may prove to be incorrect. All forward-looking statements are expressly qualified in their entirety by these cautionary statements. Further information regarding these and other risks is included under the heading Risk Factors in Lineages periodic reports with the SEC, including Lineages Annual Report on Form 10-K filed with the SEC on March 14, 2019 and its other reports, which are available from the SECs website. You are cautioned not to place undue reliance on forward-looking statements, which speak only as of the date on which they were made. Lineage undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made, except as required by law.

View source version on businesswire.com: https://www.businesswire.com/news/home/20191210005404/en/

Excerpt from:
Lineage Cell Therapeutics and AgeX Therapeutics Announce Issuance of US Patent for Method of Generating Induced Pluripotent Stem Cells - BioSpace

AgeX Therapeutics and Lineage Cell Therapeutics Announce Issuance of U.S. Patent for Method of Generating Induced Pluripotent Stem Cells – Yahoo…

AgeX Therapeutics, Inc. (NYSE American: AGE) and Lineage Cell Therapeutics, Inc. (NYSE American and TASE LCTX), announced today that the United States Patent and Trademark Office (USPTO) has issued U.S. Patent No. 10,501,723, entitled "Methods of Reprogramming Animal Somatic Cells" covering what is commonly designated "induced Pluripotent Stem (iPS) cells. The issued claims include methods to manufacture pluripotent cells capable of becoming any cell in the body. The patent has an early priority date, having been filed before the first scientific publication of Shinya Yamanaka, for which he won the Nobel Prize for Physiology or Medicine in 2012.

"This patent broadly describes multiple techniques for reprogramming cells of the body back to the all-powerful stem cell state," said Dr. Michael D. West, CEO of AgeX and first inventor on the patent. "Perhaps more significantly, it includes certain factors that address some of the difficulties currently encountered with iPS cells. It also reflects the foundational work our scientists have undertaken to apply reprogramming technology to age-reversal, specifically, induced Tissue Regeneration (iTR) which is currently a focus of AgeX product development." A video describing the significance of the patent in AgeXs product development is available on the AgeX website.

"The issuance of this patent highlights Lineages dominant position in the field of cell therapy," stated Brian M. Culley, CEO of Lineage. "Our efforts to develop new treatments rely on well-characterized and NIH-approved human cell lines. These lines are not genetically manipulated, which avoids the safety concerns associated with genetic aberrations arising from the creation of iPS cells. We believe the Lineage cell lines provide the safest option for our current clinical-stage programs, particularly in immune-privileged anatomical sites such as the eye (OpRegen for the treatment of dry AMD) and spinal cord (OPC1, for the treatment of spinal cord injury). However, the vast intellectual property estate which underlies our cell therapy platform has never been limited to these particular cell lines. As one example, this newly-issued patent provides us with proprietary methods for producing induced pluripotent stem cells, or, as it was practiced by us prior to Yamanaka, Analytical Reprogramming Technology (ART). In certain settings, an ART/iPS approach might offer important advantages, such as for an autologous treatment or when the selection of preferential attributes from a series of iPS lines is desirable. Questions as to which stem cell technology is preferred ultimately will be answered by clinical safety and efficacy and likely will be indication-specific, so we believe it is in the best interest of our shareholders to generate patented technology which enables us to pursue programs in either or both formats which we believe will ensure the highest probability of success."

Induced Pluripotent Stem Cells (iPS) are typically derived from adult skin or blood cells which have been "reprogrammed" or "induced" to retrace their developmental age and regain the potential to form all of the young cell and tissue types of the body. In 2010 inventors of the -723 patent issued today demonstrated that this reversal of developmental aging even extended to the telomere clock of cell aging. This reprogramming technology provides an alternate source of starting material for the manufacture of potentially any type of human cell needed for therapeutic purposes. Because iPSCs can be derived directly from adult tissues, they can be used to generate pluripotent cells from patients with known genetic abnormalities for drug discovery or as an alternative source of cell types for regenerative therapies.

U.S. Patent No. 10,501,723, entitled "Methods of Reprogramming Animal Somatic Cells" was assigned to Advanced Cell Technology of Marlborough, Massachusetts (now Astellas Institute for Regenerative Medicine) and licensed to Lineage and sublicensed to AgeX Therapeutics for defined fields of use. Inventors of the patent include Michael D. West, CEO of AgeX and previous CEO of Advanced Cell Technology, Karen B. Chapman, Ph.D., and Roy Geoffrey Sargent, Ph.D.

About AgeX Therapeutics

AgeX Therapeutics, Inc. (NYSE American: AGE) is focused on developing and commercializing innovative therapeutics for human aging. Its PureStem and UniverCyte manufacturing and immunotolerance technologies are designed to work together to generate highly-defined, universal, allogeneic, off-the-shelf pluripotent stem cell-derived young cells of any type for application in a variety of diseases with a high unmet medical need. AgeX has two preclinical cell therapy programs: AGEX-VASC1 (vascular progenitor cells) for tissue ischemia and AGEX-BAT1 (brown fat cells) for Type II diabetes. AgeXs revolutionary longevity platform induced Tissue Regeneration (iTR) aims to unlock cellular immortality and regenerative capacity to reverse age-related changes within tissues. AGEX-iTR1547 is an iTR-based formulation in preclinical development. HyStem is AgeXs delivery technology to stably engraft PureStem cell therapies in the body. AgeX is developing its core product pipeline for use in the clinic to extend human healthspan and is seeking opportunities to establish licensing and collaboration agreements around its broad IP estate and proprietary technology platforms.

Story continues

For more information, please visit http://www.agexinc.com or connect with the company on Twitter, LinkedIn, Facebook, and YouTube.

About Lineage Cell Therapeutics, Inc.

Lineage Cell Therapeutics is a clinical-stage biotechnology company developing novel cell therapies for unmet medical needs. Lineages programs are based on its proprietary cell-based therapy platform and associated development and manufacturing capabilities. With this platform Lineage develops and manufactures specialized, terminally-differentiated human cells from its pluripotent and progenitor cell starting materials. These differentiated cells are developed either to replace or support cells that are dysfunctional or absent due to degenerative disease or traumatic injury or administered as a means of helping the body mount an effective immune response to cancer. Lineages clinical assets include (i) OpRegen, a retinal pigment epithelium transplant therapy in Phase I/IIa development for the treatment of dry age-related macular degeneration, a leading cause of blindness in the developed world; (ii) OPC1, an oligodendrocyte progenitor cell therapy in Phase I/IIa development for the treatment of acute spinal cord injuries; and (iii) VAC2, an allogeneic cancer immunotherapy of antigen-presenting dendritic cells currently in Phase I development for the treatment of non-small cell lung cancer. Lineage is also evaluating potential partnership opportunities for Renevia, a facial aesthetics product that was recently granted a Conformit Europenne (CE) Mark. For more information, please visit http://www.lineagecell.com or follow the Company on Twitter @LineageCell.

Forward-Looking Statements

Certain statements contained in this release are "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995. Any statements that are not historical fact including, but not limited to statements that contain words such as "will," "believes," "plans," "anticipates," "expects," "estimates" should also be considered forward-looking statements. Forward-looking statements involve risks and uncertainties. Actual results may differ materially from the results anticipated in these forward-looking statements and as such should be evaluated together with the many uncertainties that affect the business of AgeX Therapeutics, Inc. and its subsidiaries, particularly those mentioned in the cautionary statements found in more detail in the "Risk Factors" section of AgeXs Annual Report on Form 10-K and Quarterly Reports on Form 10-Q filed with the Securities and Exchange Commissions (copies of which may be obtained at http://www.sec.gov). Subsequent events and developments may cause these forward-looking statements to change. AgeX specifically disclaims any obligation or intention to update or revise these forward-looking statements as a result of changed events or circumstances that occur after the date of this release, except as required by applicable law.

View source version on businesswire.com: https://www.businesswire.com/news/home/20191210005435/en/

Contacts

Media Contact for AgeX:Bill Douglass Gotham Communications, LLCbill@gothamcomm.com (646) 504-0890

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AgeX Therapeutics and Lineage Cell Therapeutics Announce Issuance of U.S. Patent for Method of Generating Induced Pluripotent Stem Cells - Yahoo...

Patent Granted To Lineage & AgeX – Anti Aging News

Lineage Cell Therapeutics and AgeX Therapeutics have been awarded a United States Patent and Trademark Office patent for Methods Of Reprogramming Animal Somatic Cells.

The issuance of this patent highlights Lineages dominant position in the field of cell therapy, stated Brian M. Culley, CEO of Lineage. Our efforts to develop new treatments rely on well-characterized and NIH-approved human cell lines. These lines are not genetically manipulated, which avoids the safety concerns associated with genetic aberrations arising from the creation of iPS cells. We believe the Lineage cell lines provide the safest option for our current clinical-stage programs, particularly in immune-privileged anatomical sites such as the eye (OpRegen for the treatment of dry AMD) and spinal cord (OPC1, for the treatment of spinal cord injury). However, the vast intellectual property estate which underlies our cell therapy platform has never been limited to these particular cell lines. As one example, this newly-issued patent provides us with proprietary methods for producing induced pluripotent stem cells, or, as it was practiced by us prior to Yamanaka, Analytical Reprogramming Technology (ART). In certain settings, an ART/iPS approach might offer important advantages, such as for an autologous treatment or when the selection of preferential attributes from a series of iPS lines is desirable. Questions as to which stem cell technology is preferred ultimately will be answered by clinical safety and efficacy and likely will be indication-specific, so we believe it is in the best interest of our shareholders to generate patented technology which enables us to pursue programs in either or both formats which we believe will ensure the highest probability of success.

This patent broadly describes multiple techniques for reprogramming cells of the body back to the all-powerful stem cell state, said Dr Michael D West, CEO of AgeX and first inventor on the patent. Perhaps more significantly, it includes certain factors that address some of the difficulties currently encountered with iPS cells. It also reflects the foundational work our scientists have undertaken to apply reprogramming technology to age-reversal, specifically, induced Tissue Regeneration (iTR) which is currently a focus of AgeX product development.

Patent 10,501,723 covers induced pluripotent stem cells which includes methods to manufacture iPSs cells that are capable of becoming any cell within the body. This patent has an early priority date having been filed before the first scientific publication, and was assigned to Advanced Cell Technology of Marlborough, Massachusetts and licenced to Lineage as well as being sublicensed to Age X for defined fields of use.

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Patent Granted To Lineage & AgeX - Anti Aging News

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

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.

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Summary

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

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

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

Novel Cell Sorting and Separation Markets, 2030 – P&T Community

DUBLIN, Oct. 7, 2019 /PRNewswire/ -- The "Novel Cell Sorting and Separation Market: Focus on Acoustophoresis, Buoyancy, Dielectrophoresis, Magnetophoretics, Microfluidics, Optoelectronics, Traceless Affinity and Other Technologies, 2019-2030" report has been added to ResearchAndMarkets.com's offering.

The Novel Cell Sorting and Separation Market: Focus on Acoustophoresis, Buoyancy, Dielectrophoresis, Magnetophoretics, Microfluidics, Optoelectronics, Traceless Affinity, and Other Technologies, 2019-2030' report features an extensive study of the current landscape and future outlook of the growing market for novel cell sorting and separation technologies (beyond conventional methods). The study presents detailed analyses of cell sorters, cell isolation kits, and affiliated consumables and reagents, that are based on the aforementioned technologies.

Advances in the fields of cell biology and regenerative medicine have led to the development of various cell-based therapies, which, developers claim, possess the potential to treat a variety of clinical conditions. In 2018, it was reported that there were more than 1,000 clinical trials of such therapies, being conducted across the globe by over 900 industry players.

Moreover, the total investment in the aforementioned clinical research efforts was estimated to be around USD 13 billion. Given the recent breakthroughs in clinical testing and the discovery of a variety of diagnostic biomarkers, the isolation of one or multiple cell types from a heterogenous population has not only become simpler but also an integral part of modern clinical R&D. The applications of cell separation technologies are vast, starting from basic research to biological therapy development and manufacturing.

However, conventional cell sorting techniques, including adherence-based sorting, membrane filtration-based sorting, and fluorescence- and magnetic-based sorting, are limited by exorbitant operational costs, time-consuming procedures, and the need for complex biochemical labels. As a result, the use of such techniques has, so far, been restricted in the more niche and emerging application areas.

Amongst other elements, the report features:

Companies Mentioned

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Novel Cell Sorting and Separation Markets, 2030 - P&T Community

Stem Cell-Derived Cells Market to Record an Exponential CAGR by 2025 – NewsVarsity

Stem cell-derived cells are ready-made human induced pluripotent stem cells (iPS) and iPS-derived cell lines that are extracted ethically and have been characterized as per highest industry standards. Stem cell-derived cells iPS cells are derived from the skin fibroblasts from variety of healthy human donors of varying age and gender. These stem cell-derived cells are then commercialized for use with the consent obtained from cell donors. These stem cell-derived cells are then developed using a complete culture system that is an easy-to-use system used for defined iPS-derived cell expansion. Majority of the key players in stem cell-derived cells market are focused on generating high-end quality cardiomyocytes as well as hepatocytes that enables end use facilities to easily obtain ready-made iPSC-derived cells. As the stem cell-derived cells market registers a robust growth due to rapid adoption in stem cellderived cells therapy products, there is a relative need for regulatory guidelines that need to be maintained to assist designing of scientifically comprehensive preclinical studies. The stem cell-derived cells obtained from human induced pluripotent stem cells (iPS) are initially dissociated into a single-cell suspension and later frozen in vials. The commercially available stem cell-derived cell kits contain a vial of stem cell-derived cells, a bottle of thawing base and culture base.

The increasing approval for new stem cell-derived cells by the FDA across the globe is projected to propel stem cell-derived cells market revenue growth over the forecast years. With low entry barriers, a rise in number of companies has been registered that specializes in offering high end quality human tissue for research purpose to obtain human induced pluripotent stem cells (iPS) derived cells. The increase in product commercialization activities for stem cell-derived cells by leading manufacturers such as Takara Bio Inc. With the increasing rise in development of stem cell based therapies, the number of stem cell-derived cells under development or due for FDA approval is anticipated to increase, thereby estimating to be the most prominent factor driving the growth of stem cell-derived cells market. However, high costs associated with the development of stem cell-derived cells using complete culture systems is restraining the revenue growth in stem cell-derived cells market.

Browse Full Report at https://www.persistencemarketresearch.com/market-research/stem-cell-derived-cells-market

The global Stem cell-derived cells market is segmented on basis of product type, material type, application type, end user and geographic region:

Segmentation by Product Type Stem Cell-Derived Cell Kits Stem Cell-Derived Definitive Endoderm Cell Kits Stem Cell-Derived Beta Cell Kits Stem Cell-Derived Hepatocytes Kits Stem Cell-Derived Cardiomyocytes Kits Accessories

Segmentation by End User Hospitals Research and Academic Institutions Biotechnology and Pharmaceutical Companies Contract Research Organizations/ Contract Manufacturing Organizations

Get Sample Copy of this report at https://www.persistencemarketresearch.com/samples/28780?source=atm

The stem cell-derived cells market is categorized based on product type and end user. Based on product type, the stem cell-derived cells are classified into two major types stem cell-derived cell kits and accessories. Among these stem cell-derived cell kits, stem cell-derived hepatocytes kits are the most preferred stem cell-derived cells product type. On the basis of product type, stem cell-derived cardiomyocytes kits segment is projected to expand its growth at a significant CAGR over the forecast years on the account of more demand from the end use segments. However, the stem cell-derived definitive endoderm cell kits segment is projected to remain the second most lucrative revenue share segment in stem cell-derived cells market. Biotechnology and pharmaceutical companies followed by research and academic institutions is expected to register substantial revenue growth rate during the forecast period.

North America and Europe cumulatively are projected to remain most lucrative regions and register significant market revenue share in global stem cell-derived cells market due to the increased patient pool in the regions with increasing adoption for stem cell based therapies. The launch of new stem cell-derived cells kits and accessories on FDA approval for the U.S. market allows North America to capture significant revenue share in stem cell-derived cells market. Asian countries due to strong funding in research and development are entirely focused on production of stem cell-derived cells thereby aiding South Asian and East Asian countries to grow at a robust CAGR over the forecast period.

Some of the major key manufacturers involved in global stem cell-derived cells market are Takara Bio Inc., Viacyte, Inc. and others.

The report covers exhaustive analysis on: Stem cell-derived cells Market Segments Stem cell-derived cells Market Dynamics Historical Actual Market Size, 2014 2018 Stem cell-derived cells Market Size & Forecast 2019 to 2029 Stem cell-derived cells Market Current Trends/Issues/Challenges Competition & Companies involved Stem cell-derived cells Market Drivers and Restraints

Regional analysis includes North America Latin America Europe East Asia South Asia Oceania The Middle East & Africa

Report Highlights: Shifting Industry dynamics In-depth market segmentation Historical, current and projected industry size Recent industry trends Key Competition landscape Strategies of key players and product offerings Potential and niche segments/regions exhibiting promising growth A neutral perspective towards market performance

Get Full Report Access of this report at https://www.persistencemarketresearch.com/checkout/28780?source=atm

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Stem Cell-Derived Cells Market to Record an Exponential CAGR by 2025 - NewsVarsity

Stem Cell-Derived Cells Market to Record an Exponential CAGR by 2025 – Commerce Gazette

Stem cell-derived cells are ready-made human induced pluripotent stem cells (iPS) and iPS-derived cell lines that are extracted ethically and have been characterized as per highest industry standards. Stem cell-derived cells iPS cells are derived from the skin fibroblasts from variety of healthy human donors of varying age and gender. These stem cell-derived cells are then commercialized for use with the consent obtained from cell donors. These stem cell-derived cells are then developed using a complete culture system that is an easy-to-use system used for defined iPS-derived cell expansion. Majority of the key players in stem cell-derived cells market are focused on generating high-end quality cardiomyocytes as well as hepatocytes that enables end use facilities to easily obtain ready-made iPSC-derived cells. As the stem cell-derived cells market registers a robust growth due to rapid adoption in stem cellderived cells therapy products, there is a relative need for regulatory guidelines that need to be maintained to assist designing of scientifically comprehensive preclinical studies. The stem cell-derived cells obtained from human induced pluripotent stem cells (iPS) are initially dissociated into a single-cell suspension and later frozen in vials. The commercially available stem cell-derived cell kits contain a vial of stem cell-derived cells, a bottle of thawing base and culture base.

The increasing approval for new stem cell-derived cells by the FDA across the globe is projected to propel stem cell-derived cells market revenue growth over the forecast years. With low entry barriers, a rise in number of companies has been registered that specializes in offering high end quality human tissue for research purpose to obtain human induced pluripotent stem cells (iPS) derived cells. The increase in product commercialization activities for stem cell-derived cells by leading manufacturers such as Takara Bio Inc. With the increasing rise in development of stem cell based therapies, the number of stem cell-derived cells under development or due for FDA approval is anticipated to increase, thereby estimating to be the most prominent factor driving the growth of stem cell-derived cells market. However, high costs associated with the development of stem cell-derived cells using complete culture systems is restraining the revenue growth in stem cell-derived cells market.

Get Sample Copy of this report at https://www.persistencemarketresearch.com/samples/28780?source=atm

The global Stem cell-derived cells market is segmented on basis of product type, material type, application type, end user and geographic region:

Segmentation by Product Type Stem Cell-Derived Cell Kits Stem Cell-Derived Definitive Endoderm Cell Kits Stem Cell-Derived Beta Cell Kits Stem Cell-Derived Hepatocytes Kits Stem Cell-Derived Cardiomyocytes Kits Accessories

Segmentation by End User Hospitals Research and Academic Institutions Biotechnology and Pharmaceutical Companies Contract Research Organizations/ Contract Manufacturing Organizations

Request to View TOC at https://www.persistencemarketresearch.com/toc/28780?source=atm

The stem cell-derived cells market is categorized based on product type and end user. Based on product type, the stem cell-derived cells are classified into two major types stem cell-derived cell kits and accessories. Among these stem cell-derived cell kits, stem cell-derived hepatocytes kits are the most preferred stem cell-derived cells product type. On the basis of product type, stem cell-derived cardiomyocytes kits segment is projected to expand its growth at a significant CAGR over the forecast years on the account of more demand from the end use segments. However, the stem cell-derived definitive endoderm cell kits segment is projected to remain the second most lucrative revenue share segment in stem cell-derived cells market. Biotechnology and pharmaceutical companies followed by research and academic institutions is expected to register substantial revenue growth rate during the forecast period.

North America and Europe cumulatively are projected to remain most lucrative regions and register significant market revenue share in global stem cell-derived cells market due to the increased patient pool in the regions with increasing adoption for stem cell based therapies. The launch of new stem cell-derived cells kits and accessories on FDA approval for the U.S. market allows North America to capture significant revenue share in stem cell-derived cells market. Asian countries due to strong funding in research and development are entirely focused on production of stem cell-derived cells thereby aiding South Asian and East Asian countries to grow at a robust CAGR over the forecast period.

Some of the major key manufacturers involved in global stem cell-derived cells market are Takara Bio Inc., Viacyte, Inc. and others.

The report covers exhaustive analysis on: Stem cell-derived cells Market Segments Stem cell-derived cells Market Dynamics Historical Actual Market Size, 2014 2018 Stem cell-derived cells Market Size & Forecast 2019 to 2029 Stem cell-derived cells Market Current Trends/Issues/Challenges Competition & Companies involved Stem cell-derived cells Market Drivers and Restraints

Regional analysis includes North America Latin America Europe East Asia South Asia Oceania The Middle East & Africa

Report Highlights: Shifting Industry dynamics In-depth market segmentation Historical, current and projected industry size Recent industry trends Key Competition landscape Strategies of key players and product offerings Potential and niche segments/regions exhibiting promising growth A neutral perspective towards market performance

Purchase this report at https://www.persistencemarketresearch.com/checkout/28780?source=atm

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Stem Cell-Derived Cells Market to Record an Exponential CAGR by 2025 - Commerce Gazette

Interview: BIOLIFE4D is The First US Company to Bioprint a Mini-Heart (for Cardiotoxicity Testing) – 3DPrint.com

After quite a few teaser pictures on their social media platforms since August, BIOLIFE4D finally announced one of the biggest milestones for the company: they successfully 3D printed a tiny heart. But how small is the mini heart?Actually, it is about one quarter the size of a human heart.

The ability to 3D bioprint a mini-heart now gives the biotech firm a roadmap to achieve their ultimate goal: bioprinting a full-scale human heart viable for transplant. It is now a matter of optimizing processes and scaling up the technology for the pioneering company headquartered in Illinois.

Ravi Birla

With the structure of a full-sized heart and four internal chambers, the mini heart is replicating partial functional metrics compared to a full-sized heart as close as anyone has gotten to producing a fully functional heart through 3D bioprinting.The scientific milestone was accomplished at the companysresearch facility at JLABS in Houston, led by Ravi Birla, Chief Science Officer of BIOLIFE4D

3DPrint.com asked Birla about their achievement to understand how functional it is and how this project could lead to a fully beating organ in the future.

The functional performance of our mini-heart is not the same as a normal mammalian heart, though this is a future objective of the research, explained Birla. Our mini-heart is intended for use in drug cardiotoxicity screening, which means that the bar that it must achieve is less than the bar required for a viable transplanted organ. This is why the performance requirements for our mini-heart do not need to mimic a fully-functional animal heart at this point.

Bioprinting at BIOLIFE4D

As we move forward we will be optimizing our bioink as well as the bioprinting parameters which are needed for optimal functional performance, suggeted the expert, who alsopreviously served as the Associate Director of the Department of Stem Cell Engineering at the Texas Heart Institute in Houston.

So how did they do it? First on their list was developing a proprietary bioink using a very specific composition of different extracellular matrix compounds that closely replicate the properties of the mammalian heart. There is still no formal name to the bioinkas it was developed in-house and for now, it is currently intended for BIOLIFE4D use only.

Then, they got around creating a novel and unique bioprinting algorithm, consisting of printing parameters optimized for the whole heart. Coupling its proprietary bioink with patient-derived cardiomyocytes and its enabling bioprinting technology, BIOLIFE4D was able to bioprint a heart. Birla suggested that because of the strategic partnerships that they have developed, they have access to and utilize most of the commercially available printers which are on the market, but the mini-heart was essentially biofabricated in their labs using a CELLINK INKREDIBLE+.

We currently used a commercial source of human cells, through the expected use of the technology in using patient derived autologous cells, claimed Birla. Utilizing patient specific cells is really a cornerstone to our technology.

Currently those lucky enough to receive a donor heart transplant are really only trading one disease for another. The donor heart will save their life, but to prevent rejection the patient needs to take a large regiment of immunosuppressant therapy which causes many significant challenges for the patient. By bioengineering the heart out of the patients own cells we eliminate the need for that immunosuppressant therapy which could allow for a much better quality of life for the patient, he continued.

With this platform technology in place, BIOLIFE4D is now well-positioned to build upon it and work towards the development of a full-scale human heart. This latest milestone also positions the company as one of the top contenders at the forefront of whole heart bioengineering, a field that is rapidly advancing.

However, beyond the scientific advancements the mini-heart represents, this is also an opportunity to provide the pharmacological industry and drug discovery companies a new tool for cardiotoxicity testing of new drugs and compounds. Until now the model used for predicting the cardiotoxicity effects of a new drug or compound was essentially limited to the animal model.But BIOLIFE4D intends to ultimately provide the mini-hearts as a more reliable model of predicting cardiotoxicity, claiming that there is no better predictor of how a human heart will react than a human heart. This also represents an opportunity to reduce the number of animals used for testing purposes, something which is already banned in quite a few regions,including India, the European Union, New Zealand, Israel, and Norway.

We are already working closely with companies that provide cardiotoxicity testing services to the Pharma and drug discovery industries. All drugs, new compounds and anything else that currently undergoes cardiotoxicity testing requirements prior to entering the human market could be candidates for the mini-heart. After all, what would provide a better predictive model of how a human heart will respond than a human heart (albeit a scaled-down version)? revealed Birla.

The mini-heart has many of the features of a human heart even though BIOLIFE4D has not been able to recreate the full functionality of a human heart yet.

While we have bioengineered mini-hearts, and this in itself is a major accomplishment, a significant advancement in the field of whole heart engineering and moves us closer to bioprinting human hearts for transplantation, this accomplishment does not provide us with a specific time-line or a significant guidance on when the fully funcitional heart will be available.

According to Birla, the most difficult part to 3D print a human heart at this point is the valves, due to the complex tri-leaflet geometry. But as they begin to scale up, they can anticipate that the complex vasculature that is needed to keep an organ viable could prove to be a big challenge.

Birla is convinced that the algorithm used as a fundamental part of the mini-heart could change the way labs will bioprint organs in the future.Weused very specific and highly customized printing parameters to bioprint the mini-heart which we have customized for our use in our lab and for our specific purposes. Some of the process ultimately could be leveraged for the bioengineering of other organs, but our overall process to bioengineer a human heart is unique to a heart.

One of the huge advantages BIOLIFE4D enjoys is that they have been able to form strategic partnerships with various major research institutions and hospitals to provide them access to some of the most state-of-the-art facilities and equipment. Nevertheless, because of the highly confidential nature of their work, most of it is done in-house at the labs and by their own researchers.

The successful demonstration of a mini heart is the latest in a string of scientific milestones from BIOLIFE4D as it seeks to produce the worlds first 3D bioprinted human heart viable for transplant. Earlier in 2019, they successfully 3D bioprinted various individual heart components, including valves, ventricles, blood vessels, and in June of 2018 they 3D bioprintedhuman cardiac tissue(a cardiac patch).

The company states that their innovative 3D bioprinting process provides the ability to reprogram a patients own white blood cells to iPS cells, and then to differentiate those iPS cells into different types of cardiac cells needed to 3D bioprint individual cardia components and ultimately, a human heart viable for transplant.

This is crucial for a company that seeks to disrupt how heart disease and other cardiac impairments are treated, particularly by improving the transplant process so that in the future they can eliminate the need for donor organs. Heart disease is the number one cause of death of men and women in the United States each year. Heart diseases even claim more lives each year than all forms of cancer combined, yet countless individuals who need transplants are left waiting as there are not enough donors to meet demand and every 30 seconds, someone dies in the US of a heart disease-related event.

While we have come a long way, and we are moving forward at a fast pace, we just dont know how long it will take to achieve a full-scale heart. We have to keep in mind that mother nature had millions of years to perfect this process inside our bodies, while we just arent sure exactly how long it is going to take us to perfect the process outside of the body, concluded Birla.

At BIOLIFE4D, they know there are still challenges on the way to the full-size human heart viable for transplantation, however, this achievement signals that they are on the right path. They highlighted that their success, as well as the significant advancements they have been able to achieve already,are a result of an incredible team effort,a multi-disciplinary group of researchers working on the project, from bioengineers to life scientists.Their team consists of people with specific skill sets and areas of expertise, all working hard to bring this incredible life-saving technology to the market in the shortest time possible.

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Interview: BIOLIFE4D is The First US Company to Bioprint a Mini-Heart (for Cardiotoxicity Testing) - 3DPrint.com

Genetic Tests Offered (all nonprofit) | The John and …

ABCA4 Retinal Degeneration Autosomal Recessive and Autosomal DominantPlease submit parental samples (no charge) in addition to the patient's sample; requisition needed for each. ABCA4 & ELOVL4 (Leu263 del5tttCTTAA) First Tier Testing$463 12-14 weeks Allele-Specific Testing Followed by Conventional Sequencing 81479 Second Allele Testing$1,611 14-16 weeks Conventional Sequencing 81479AchromatopsiaAutosomal RecessiveCNGA3(exon 8) & CNGB3(Exon 10)First Tier Testing$2338-10 weeksConventional Sequencing81479Autosomal Recessive & X-linkedCNGA3, CNGB3, CNNM4, GNAT2, KCNV2, NBAS, OPN1LW, PDE6C, PDE6H & RPGRExome Testing$220014-16 weeksConventional Sequencing & Next Generation Sequencing81479Autosomal Dominant Neovascular Inflammatory Vitreoretinopathy (ADNIV) Autosomal Dominant CAPN5 $373 12-14 weeks Conventional Sequencing 81479Bardet-Biedl SyndromeAutosomal RecessiveBBS1 (Met390Arg) BBS10 (Leu90 ins1T)First Tier Testing$1408-10 weeksAllele-Specific Testing81479ARL6, BBS1, BBS2, BBS4, BBS5, BBS7, BBS9, BBS10, BBS12, CEP290, INPP5E, LZTFL1, MKS1, MKKS, SDCCAG8, TRIM32 & TTC8Exome Testing$220014-16 weeksAllele-Specific Testing Followed by Conventional Sequencing and Next Generation Sequencing81479Best Disease Autosomal Dominant BEST1 (Full coding region) $373 12-14 weeks Conventional Sequencing 81406Blue Cone Monochromacy X-Linked OPNL1W - Locus Control Region $429 6-8 weeks Deletion Detection (Males Only) 81479Choroideremia X-Linked CHM (Full coding region) $485 14-16 weeks Conventional Sequencing 81479Cone-Rod DystrophyAutosomal DominantCRX (full coding region), GUCA1A(Leu151Phe) & GUCY2D (Exon 13)$2618-10 weeksConventional Sequencing81404, 81479Congenital Stationary Night BlindnessAutosomal Dominant, Autosomal Recessive & X-LinkedCACNA1F, GRM6, PDE6B & TRPM1First Tier Testing$2338-10 weeksConventional Sequencing81479CABP4, CACNA1F, GNAT1, GPR179, GRK1, GRM6, LRIT3, NYX, PDE6B, RDH5, RHO, SAG, SLC24A1, TRPM1Exome Testing$220014-16 weeksConventional Sequencing & Next Generation Sequencing81479Corneal Dystrophy-Stromal Autosomal Dominant TGFBI (Exons 4 & 11-14) $205 12-14 weeks Conventional Sequencing 81479EnhancedS-Cone Syndrome Autosomal RecessivePlease submit parental samples (no charge) in addition to the patient's sample; requisition needed for each. NR2E3 (Exons 2-8) $314 14-16 weeks Conventional Sequencing 81479Jewish Retinal Degeneration Panel - Leber Congenital Amaurosis, Retinitis Pigmentosa and Usher Syndrome Autosomal Recessive DHDDS (Lys42Glu), LCA5 (Gln279Stop), MAK (Lys429 Alu Insertion), PCDH15 (Arg245Stop), USH3A (Asn48Lys) $205 4 weeks Conventional Sequencing 81400, 81479Juvenile Open Angle Glaucoma Autosomal Dominant MYOC (full coding region) $205 12-14 weeks Conventional Sequencing 81479Juvenile X-Linked Retinoschisis X-Linked RS1 (full coding region) $233 10-12 weeks Conventional Sequencing 81479Leber Congenital AmaurosisAutosomal Recessive Please submit parental samples (no charge) in addition to the patient's sample; requisition needed for each.AIPL1, CEP290, CRB1, CRX, GUCY2D, IQCB1, LCA5, LRAT, NMNAT1, RD3, RDH12, RPE65 (entire coding region), RPGRIP1, SPATA7, TULP1First Tier Testing$95714-16 weeksAllele-Specific Testing Followed by Conventional Sequencing81404, 81406, 81408, 81479Exome Testing$28006-8 monthsAllele-Specific Testing Followed by Conventional Sequencing and Next Generation Sequencing81479Leber Hereditary Optic Neuropathy Mitochondrial 3460, 11778, 14484 $140 6-8 weeks Allele-Specific Testing 81401Malattia Leventinese Autosomal Dominant EFEMP1 (Arg345Trp mutation) $140 6-8 weeks Allele-Specific Testing 81479Norrie Disease X-Linked NDP (full coding region) $121 8-10 weeks Conventional Sequencing 81404North Carolina Macular Dystrophy Autosomal Dominant PRDM13, IRX1 $243 6-8 weeks Allele-Specific Testing and Conventional Sequencing 81479Pattern Dystrophy Autosomal Dominant RDS (full coding region) $149 8-10 weeks Conventional Sequencing 81404Primary Open Angle Glaucoma Autosomal Dominant MYOC (full coding region) $205 12-14 weeks Conventional Sequencing 81479Retinitis Pigmentosa Autosomal Dominant C1QTNF5, IMPDH1, MAK, NR2E3, PRPF3, PRPF31, PRPF8, RDH12, RDS, RHO, RP1, RP9, SNRNP200, TOPORS $320 8-10 weeks Allele-Specific Testing Followed by Conventional Sequencing 81404, 81479Retinitis Pigmentosa Autosomal Recessive ABCA4, CC2D2A, CERKL, CLRN1, CNGA1, CRB1, DHDDS, EYS, FAM161A, FLVCR1, IDH3B, IMPG2, LRAT, MAK, NR2E3, NRL, PDE6A, PDE6B, PDE6G, PROM1, RBP3, RDH12, RGR, RLBP1, RPE65, SAG, TTPA, TULP1, USH2A, ZNF513 $833 12 14 weeks Allele-Specific Testing Followed by Conventional Sequencing 81408, 81479Retinitis Pigmentosa X-Linked RP2, RPGR $865 12-14 weeks Conventional Sequencing 81479Sorsby Dystrophy Autosomal Dominant TIMP3 (Exons 1 & 5) $121 8-10 weeks Conventional Sequencing 81479Stargardt like Macular Dystrophy Autosomal Dominant ELOVL4 (Leu263 del5tttCTTAA) $140 6-8 weeks Allele-Specific Testing 81479Stargardt Disease Autosomal Recessive and Autosomal DominantPlease submit parental samples (no charge) in addition to the patient's sample; requisition needed for each. ABCA4 & ELOVL4 (Leu263 del5tttCTTAA) First Tier Testing$463 12-14 weeks Allele-Specific Testing Followed by Conventional Sequencing 81408, 81479 Second Allele Testing$1,611 14-16 weeks Conventional Sequencing 81408, 81479Usher SyndromeAutosomal RecessiveCDH23, CLRN1, MYO7A, PCDH15, USH1C, USH1G & USH2AFirst Tier Testing$5758-10 weeksAllele-Specific Testing Followed by Conventional Sequencing81400, 81407, 81408, 81479Second Allele Testing$575-$1,626 10-12 weeksConventional Sequencing81400, 81407, 81408, 81479ABHD12, CDH23, CIB2, CLRN1, DFNB31, GPR98, HARS, MYO7A, PCDH15, USH1C, USH1G & USH2AExome Testing$220014-16 weeksAllele-Specific Testing Followed by Conventional Sequencing and Next Generation Sequencing81400, 81407, 81408, 81479X-Linked Familial Exudative Vitreoretinopathy (XL-FEVR) X-Linked NDP (full coding region) $121 8-12 weeks Conventional Sequencing 81479

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Genetic Tests Offered (all nonprofit) | The John and ...

The addition of human iPS cell-derived neural progenitors …

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

Cardiomyocyte

Neural progenitor

Spheroid

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The addition of human iPS cell-derived neural progenitors ...

iPS Cells for Disease Modeling and Drug Discovery

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

12:50 PLENARY KEYNOTE SESSION

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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