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GEMoaB Announces Internationally Renowned Experts to Newly Formed Strategic and Scientific Advisory Board – Yahoo Finance

DRESDEN, Germany, March 12, 2020 /PRNewswire/ -- GEMoaB, a biopharmaceutical company focused on the development of next generation immunotherapies for hard-to-treat cancers, today announced the appointment of five distinguished scientific, clinical and public affairs leaders to its inaugural Strategic and Scientific Advisory Board. The group will provide counsel to support the continued development of the company's proprietary immune-oncology platforms and help to shape the company's broader strategic and scientific decisions and plans.

GEMoaB Logo (PRNewsfoto/GEMoaB GmbH)

"We are thrilled to have this group of experts join our Strategic and Scientific Advisory Board," said Michael Pehl, CEO of GEMoaB. "We look forward to working closely with our Strategic and Scientific Advisory Board members as we continue to build a fully integrated and leading biopharmaceutical company and accelerate our UniCAR, RevCAR and ATAC pipeline efforts to bring them to cancer patients in need."

Members of GEMoaB's Strategic and Scientific Advisory Board are:

Professor Dr. Gerhard Ehninger Gerhard is GEMoaB's co-founder and Chief Medical Officer and will chair the Strategic and Scientific Advisory board. He is a pioneer in the field of cancer cell therapies and has dedicated his career to clinical and translational oncology research. Gerhard was Head of Hematology & Oncology, University Hospital 'Carl Gustav Carus', Technical University Dresden, Germany as well as the former President of the German Society of Hematology and Oncology (DGHO). Furthermore, Gerhard is co-founder of the German Bone Marrow Donor Registry (DKMS), Chief Executive Officer and founding shareholder at Cellex Gesellschaft fr Zellgewinnung mbH and founding shareholder of GEMoaB Monoclonals GmbH.

Professor Dr. Michael BachmannDr. Bachmann is an internationally leading expert in tumor immunology and founding shareholder of GEMoaB Monoclonals GmbH. Dr. Bachmann is Director of the Institute for Radiopharmaceutical Cancer Research, Helmholtz-Center Dresden, Germany as well as Head of Radioimmunology, Helmholtz-Center Dresden, Germany. In addition, Dr. Bachmann is Head of Tumor Immunology, University Cancer Center (UCC), University Hospital 'Carl Gustav Carus', Technical University Dresden, Germany and Deputy Head of the Working Group Tumor Immunology of the German Society for Immunology (Deutsche Gesellschaft fr Immunologie, DGfI).

Professor Dr. Bob Lwenberg Dr. Lwenberg's unique scientific career has focused on the pathobiology, molecular diagnostic, clinical and translational research of acute myeloid leukemia. Dr Lwenberg is Professor of Hematology and is the former Chairman of the Department of Hematology at Erasmus University Medical Center, Rotterdam, the Netherlands. Dr. Lwenberg was one of the founders and has served as President of the European Hematology Association (EHA). He has been president of the International Society of Experimental Hematology and the International Society of Hematology. He is former Chairman of the Scientific Advisory Board and current member of the Board of the European School of Hematology (Paris). He founded and subsequently served as the first president of the Dutch-Belgian Cooperative Group on Hemato-Oncology in Adults (HOVON), one of the leading cooperative clinical trial consortia in hemato-oncology in Europe. Between 2013-2020, Dr. Lwenberg was the Editor-in-Chief of Blood, the official journal of the American Society of Hematology. Bob Lwenberg is an elected member of the Royal Academy of Sciences and Arts of the Netherlands.

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Dr. Thomas de Maizire Dr. de Maizire is a member of the German Parliament, Member of the Finance Committee of the German Parliament, former German Federal Minister and has throughout his distinguished career served in multiple key public and governmental roles in Germany. Dr. de Maizire has been Head of State Chancellery of Mecklenburg-Vorpommern, Minister of State and Head of State Chancellery of Saxony, Minister of State of Finance of Saxony, Minister of State of Justice of Saxony, Minister of State of the Interior of Saxony, Federal Minister and Head of Federal Chancellery of Germany, German Federal Minister of the Interior and German Federal Minister of Defence.

Professor Dr. Katy Rezvani Dr. Rezvani is the Director of Translational Research, Medical Director of the MD Anderson GMP and Cell Therapy Laboratory and Chief, Section of Cellular Therapy, Department of Stem Cell Transplant and Cellular Therapy, MD Anderson Cancer Center in Houston/Texas, USA. Dr. Rezvani joined the faculty at the MDACC in 2012 from the Hammersmith Hospital in London, where she was Director of the allogeneic adult stem cell transplant program, Medical Director of the GMP facility and Director of the Transplant Immunology Research Laboratory. Dr. Rezvani has an active research laboratory program in transplantation immunology where the focus of her research group is to study the role of natural killer cells (NK) cells in mediating immunity against leukemia, and to understand the mechanisms of tumor-induced NK cell dysfunction

"Our efforts are focused on maximizing the potential of engineered cellular therapies in hematology and oncology," said Professor Dr. Gerhard Ehninger, GEMoaB's co-founder and Chief Medical Officer. "The deep expertise and past experiences of all of our Strategic and Scientific Advisory Board members will bolster GEMoAB's ability to positively impact patients' lives."

About GEMoaB

GEMoaB is a privately-owned, clinical-stage biopharmaceutical company that isaiming to become a globally leading biopharmaceutical company. By advancing its proprietary UniCAR, RevCAR and ATAC platforms, the company will discover, develop, manufacture and commercialize next generation immunotherapies for the treatment of cancer patients with a high unmet medical need.

GEMoaB has a broad pipeline of product candidates in pre-clinical and clinical development for the treatment of hematological malignancies as well as solid tumors. Its clinical stage assets GEM333, an Affinity-Tailored Adaptor for T-Cells (ATAC) with binding specificity to CD33 in relapsed/refractory AML, and GEM3PSCA, an ATAC with binding specificity to PSCA for the treatment of castrate-resistant metastatic prostate cancer and other PSCA expressing late-stage solid tumors, are currently investigated in Phase I studies and globally partnered with Bristol-Myers Squibb/Celgene. A Phase IA dose-finding study of the first UniCAR asset, UniCAR-T-CD123 for treatment of relapsed/refractory AML and ALL has been initiated, UniCAR-T-PSMA against CRPC and other PSMA-expressing late-stage solid tumors, is planned to be tested in a Phase I study initiated by H2 2020.

Manufacturing expertise, capability and capacity are key for developing cellular immunotherapies for cancer patients. GEMoaB has established a preferred partnership with its sister company Cellex in Cologne, a world leader in manufacturing hematopoietic blood stem cell products and a leading European CMO for CAR-T cells, co-operating in that area with several large biotech companies.

About UniCAR

GEMoaB is developing a rapidly switchable universal CAR-T platform, UniCAR, to improve the therapeutic window and increase efficacy and safety of CAR-T cell therapies in more challenging cancers, including solid tumors. Standard CAR-T cells depend on the presence and direct binding of cancer antigens for activation and proliferation. An inherent key feature of the UniCAR platform is a rapidly switchable on/off mechanism (less than 4 hours after interruption of TM supply) enabled by the short pharmacokinetic half-life and fast internalization of soluble adaptors termed targeting modules (TMs). These TMs provide the antigen-specificity to activate UniCAR gene-modified T-cells (UniCAR-T) and consist of a highly flexible antigen-binding moiety, linked to a small peptide motif recognized by UniCAR-T.

About ATAC

GEMoaB's platform of Affinity-Tailored Adaptors for T-Cells (ATAC) is characterized by high binding affinity to tumor antigens and lower affinity to the CD3 antigen on effector T-cells, preventing T-cell auto-activation in pre-clinical models. Safety and tolerability of the treatment are also increased by the relatively short serum half-life (60 min). The use of fully humanized antibodies reduces the risk of immunogenicity even in case of chronic dosing. Half-life extended ATACs are in pre-clinical development.

More information can be found at http://www.gemoab.com.

Forward-looking Statements

This announcement includes forward-looking statements that involve risks, uncertainties and other factors, many of which are outside of our control, that could cause actual results to differ materially from the results and matters discussed in the forward looking statements. Forward looking statements include statements concerning our plans, goals, future events and or other information that is not historical information.

The Company does not assume any liability whatsoever for forward-looking statements. The Company assumes that potential partners will perform and rely on their own independent analyses as the case may be. The Company will be under no obligation to update the Information.

GEMoaB Monoclonals GmbHTatzberg 4701307 DresdenGERMANY

For further information please contactConstanze Medackc.medack@gemoab.com; Tel.: +49 351 4466-45027

Investor ContactMichael Pehlm.pehl@gemoab.com; Tel.: +49 351 4466-45030

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SOURCE GEMoaB

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GEMoaB Announces Internationally Renowned Experts to Newly Formed Strategic and Scientific Advisory Board - Yahoo Finance

Adult Stem Cells

By: Ian Murnaghan BSc (hons), MSc - Updated: 21 Feb 2019| *Discuss

Although stem cells have defining characteristics, they do have different sources. Adult stem cells, also called somatic stem cells, possess the same basic characteristics of all stem cells. An adult stem cell is an unspecialised cell that is capable of:

Another goal is to develop insulin-producing cells for diabetes. With heart attacks causing enormous morbidity and mortality each year, it is also hoped that adult stem cells can repair damage to the heart.

The use of adult stem cells is more widely accepted, particularly by the public, because it does not require destruction of an embryo as with embryonic stem cells. Adult stem cells also don't have the same immunological challenges as embryonic stem cells because they are harvested from the patient. This means that a person's body is less likely to reject the stem cells because they are compatible with that person's unique physiological makeup.

Overall, adult stem cells don't pose the same ethical concerns and controversy in comparison with embryonic stem cells, but their practical challenges are numerous. As scientists continue to seek ways to effectively harvest adult stem cells, the public can await new treatments for some of the more serious and common diseases.

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Adult Stem Cells

Australia’s Mesoblast plans to evaluate its stem cell therapy in patients infected with COVID-19 – BioWorld Online

PERTH, Australia Australian stem cell therapy company Mesoblast Ltd. plans to evaluate its allogeneic mesenchymal stem cell (MSC) candidate, remestemcel-L, in patients with acute respiratory distress syndrome (ARDS) caused by coronavirus (COVID-19) in the U.S., Australia, China and Europe.

The company is in active discussions with various governments, regulatory authorities, medical institutions and pharmaceutical companies to implement these activities.

What people are dying of is acute respiratory distress syndrome, which is the bodys immune response to the virus in the lungs, and the immune system goes haywire, and in its battle with the virus it overreacts and causes severe damage to the lungs, Mesoblast CEO Silviu Itescu told BioWorld.

Were going to be evaluating whether an injection of our cells intravenously can tone down the immune system just enough so it gets rid of the virus but doesnt destroy your lungs at the same time.

Recently published results from an investigator-initiated clinical study conducted in China reported that allogeneic MSCs cured or significantly improved functional outcomes in all seven treated patients with severe COVID-19 pneumonia.

We have now looked at our own data in lung disease in adults where half the patients had the same kind of inflammation in the lungs as you get with coronavirus, and our cells significantly reduced the inflammation and significantly improved lung function, Itescu said, noting that he is awaiting emergency use authorization to treat patients under a clinical trial protocol.

In a post-hoc analyses of a 60-patient randomized controlled study in chronic obstructive pulmonary disease (COPD), remestemcel-L infusions were well-tolerated, significantly reduced inflammatory biomarkers, and significantly improved pulmonary function in those patients with elevated inflammatory biomarkers.

Since the same inflammatory biomarkers are also elevated in COVID-19, those data suggest that remestemcel-L could be useful in the treatment of patients with ARDS due to COVID-19. The COPD study results have been submitted for presentation at an international conference, with full results to be submitted for publication shortly.

Mortality in COVID-19-infected patients with the inflammatory lung condition is reported to approach 50% and is associated with older age, co-morbidities such as diabetes, higher disease severity, and elevated markers of inflammation.

Current therapeutic interventions do not appear to be improving in-hospital survival, and remestemcel-L has potential for use in the treatment of ARDS, which is the principal cause of death in COVID-19 infection.

Itescu said he didnt know of any other stem cell companies that were doing this. He said that other companies could try the approach from a research perspective but that Mesoblast has all the patents locked down.

The companys intellectual property portfolio encompasses more than 1,000 patents or patent applications in all major markets and includes the use of MSCs obtained from any source for patients with ARDS, and for inflammatory lung disease due to coronavirus (COVID-19), influenza and other viruses.

Remestemcel-L is being studied in numerous clinical trials across several inflammatory conditions, including in elderly patients with lung disease and adults and children with steroid-refractory acute graft-vs.-host disease (aGVHD).

Mesoblasts stem cell therapy is currently being reviewed by the FDA for potential approval in the treatment of children with steroid-refractory aGVHD. The company submitted the final module of a rolling BLA in January.

Remestemcel-L is being developed for rare pediatric and adult inflammatory conditions. It is an investigational therapy comprising culture-expanded MSCs derived from the bone marrow of an unrelated donor and is administered in a series of intravenous infusions.

The stem cell therapy is believed to have immunomodulatory properties to counteract the inflammatory processes that are implicated in several diseases by down-regulating the production of pro-inflammatory cytokines, increasing production of anti-inflammatory cytokines, and enabling recruitment of naturally occurring anti-inflammatory cells to involved tissues, according to Mesoblast.

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Australia's Mesoblast plans to evaluate its stem cell therapy in patients infected with COVID-19 - BioWorld Online

Astronauts growing new organs on International Space Station – The Independent

Astronauts are growing the beginnings of new organs on board the International Space Station.

The experiment is an attempt to grow human tissue by sending adult human stem cells into space, and allowing them to grow in space.

Eventually, it is hoped, the stem cells will develop into bone, cartilage and other organs. If that is successful, the discoveries could be used to try and grow organs for transplant, the scientists involved say.

Sharing the full story, not just the headlines

The experiment uses weightlessness as a tool, according to Cara Thiel, one of the two researchers from the University of Zurich who are conducting the research. The lack of gravity on board the International Space Station will be used to encourage the stem cells to grow into tissue in three dimensions, rather than the single-layerstructures that form on Earth.

It is being conducted by the astronauts on board the International Space Station using a mobile mini-laboratory that was sent on a SpaceX rocket last week. The experiment will last for a month, during which scientists will watch to see how the stem cells grow.

Mystic Mountain, a pillar of gas and dust standing at three-light-years tall, bursting with jets of gas flom fledgling stars buried within, was captured by Nasa's Hubble Space Telelscope in February 2010

Nasa/ESA/STScI

The first ever selfie taken on an alien planet, captured by Nasa's Curiosity Rover in the early days of its mission to explore Mars in 2012

Nasa/JPL-Caltech/MSSS

Death of a star: This image from Nasa's Chandra X-ray telescope shows the supernova of Tycho, a star in our Milky Way galaxy

Nasa

Arrokoth, the most distant object ever explored, pictured here on 1 January 2019 by a camera on Nasa's New Horizons spaceraft at a distance of 4.1 billion miles from Earth

Getty

An image of the Large Magellanic Cloud galaxy seen in infrared light by the Herschel Space Observatory in January 2012. Regions of space such as this are where new stars are born from a mixture of elements and cosmic dust

Nasa

The first ever image of a black hole, captured by the Event Horizon telescope, as part of a global collaboration involving Nasa, and released on 10 April 2019. The image reveals the black hole at the centre of Messier 87, a massive galaxy in the nearby Virgo galaxy cluster. This black hole resides about 54 million light-years from Earth

Getty

Pluto, as pictured by Nasa's New Horizons spacecraft as it flew over the dwarf planet for the first time ever in July 2015

Nasa/APL/SwRI

A coronal mass ejection as seen by the Chandra Observatory in 2019. This is the first time that Chandra has detected this phenomenon from a star other than the Sun

Nasa

Dark, narrow, 100 meter-long streaks running downhill on the surface Mars were believed to be evidence of contemporary flowing water. It has since been suggested that they may instead be formed by flowing sand

Nasa/JPL/University of Arizona

Morning Aurora: Nasa astronaut Scott Kelly captured this photograph of the green lights of the aurora from the International Space Station in October 2015

Nasa/Scott Kelly

Mystic Mountain, a pillar of gas and dust standing at three-light-years tall, bursting with jets of gas flom fledgling stars buried within, was captured by Nasa's Hubble Space Telelscope in February 2010

Nasa/ESA/STScI

The first ever selfie taken on an alien planet, captured by Nasa's Curiosity Rover in the early days of its mission to explore Mars in 2012

Nasa/JPL-Caltech/MSSS

Death of a star: This image from Nasa's Chandra X-ray telescope shows the supernova of Tycho, a star in our Milky Way galaxy

Nasa

Arrokoth, the most distant object ever explored, pictured here on 1 January 2019 by a camera on Nasa's New Horizons spaceraft at a distance of 4.1 billion miles from Earth

Getty

An image of the Large Magellanic Cloud galaxy seen in infrared light by the Herschel Space Observatory in January 2012. Regions of space such as this are where new stars are born from a mixture of elements and cosmic dust

Nasa

The first ever image of a black hole, captured by the Event Horizon telescope, as part of a global collaboration involving Nasa, and released on 10 April 2019. The image reveals the black hole at the centre of Messier 87, a massive galaxy in the nearby Virgo galaxy cluster. This black hole resides about 54 million light-years from Earth

Getty

Pluto, as pictured by Nasa's New Horizons spacecraft as it flew over the dwarf planet for the first time ever in July 2015

Nasa/APL/SwRI

A coronal mass ejection as seen by the Chandra Observatory in 2019. This is the first time that Chandra has detected this phenomenon from a star other than the Sun

Nasa

Dark, narrow, 100 meter-long streaks running downhill on the surface Mars were believed to be evidence of contemporary flowing water. It has since been suggested that they may instead be formed by flowing sand

Nasa/JPL/University of Arizona

Morning Aurora: Nasa astronaut Scott Kelly captured this photograph of the green lights of the aurora from the International Space Station in October 2015

Nasa/Scott Kelly

If it is successful, they hope to switch from a small laboratory to bigger production. From there, they could use the process to generate tissue for transplants by taking cells from patients, or generating organ-like materialthat could be used to test drugs, either ensuring that it works for a specific patients or reducing the number of animals used in experiments.

On Earth, tissue grows in monolayer cultures: generating flat, 2D tissue. But investigations both in space and Earth suggest that in microgravity, cells exhibit spatially unrestricted growth and assemble into complex 3D aggregates, saidOliver Ullrich, who is also leading the research.

Previous research has involved simulated ad real experiments, mostly using tumour cells, and placing real human stem cells into microgravity simulators. But for the next stage of the research there is no alternative to the ISS, he says, because 3D tissue formation of this kind requires several days or even weeks in microgravity.

After the month-long experiment, the scientists will get the samples back and expect to see successful formation oforganoids smaller, more simple versions of organs inside the test tubes. The test tubes were launched with stem cells and are expected to return to Earth with organ-like tissue structures inside, said Professor Ullrich.

Scientists are still not sure why the conditions of the International Space Station lead to the assembly of complex 3D tissue structures. Professor Ullrich and other scientists are still continuing to research how the gravitational force and the molecular machinery in the cell interact to create new and different kinds of tissue on Earth and in space.

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Astronauts growing new organs on International Space Station - The Independent

How to build a body from scratch, Altered Carbon-style – SYFY WIRE

The world of Netflix's Altered Carbon is one where consciousness is no longer tethered to the physical body. It can be, and regularly is, uploaded into "cortical stacks," which are implanted at the base of the neck. In the event of death, a persons consciousness can be reloaded into a new body, known as a "sleeve." For those less fortunate, like protagonist Takeshi Kovacs, that might mean receiving a body thats not your own. In one particularly existential example from the series first episode, it might even mean a young child being uploaded into the body of an adult.

For those with means, however, the mind can be placed into a swiftly made, identical clone, allowing them to return to their lives with little interruption. We've covered what it might take to create a digital copy of a persons mind before (spoiler: it wouldnt be easy), but Altered Carbon's techno-immortality requires a second piece: the swift creation of replacement bodies.

One of the major hurdles that has kept real-world cloning from being the game changer everyone suspected it might be after the birth of Dolly, the first successfully cloned mammal, is the relatively slow development of human bodies. If you wanted to clone a 50-year-old human and get them back to the same stage of development, it would take you 50 years. That's a little too slow to make use of in the same way science fiction does.

We don't have the means to artificially age a body at a rapid pace, but what if we were able to shortcut these limitations to put it plainly, what would it take to build an adult body from scratch?

BONES

If you want to build a person from scratch, you must first make the universe. Carl Sagan said something like that, I think. Just after that, though, youll need a skeleton. Without bones, youll be left with little more than a Cronenbergian nightmare, cool in its own way, but not what were shooting for.

Today, if you have trouble with your bones, your options are limited. The first option, and in most cases the best one, is to let the bone heal itself. Your body is pretty resilient and capable of repairing most day-to-day injuries, even the ones accompanied by a sickening crack. If the injury is really bad, things get a little more medieval. Surgeons might use a series of metal plates and screws to hold your bones in place and give them time for your bodys healing processes to do their work. But those solutions only work for relatively minor injuries where the bone tissue is at least moderately intact.

When it comes to bone replacements, things are a little tougher.

Again, we can return to metal. Like the Wolverine, you might have part of your skeleton replaced or covered over with metal. This might be sufficient in specific cases, but it all feels a little crude.

Ramille Shah, Ph.D., headed a team out of Northwestern's McCormick School of Engineering to create a new material capable of instigating rapid bone regeneration. The team used 3D printers (the invention that never stops giving) and a mixture of 90 percent hydroxyapatite, a natural element of human bones, and 10 percent medical polymer to build bone constructs.

The result is a bit of artificial bone modeled in whatever shape the patient needs. It is porous, allowing for blood vessels and other tissues to easily integrate. The elastibone (perhaps the worst superhero name, trademark pending) stimulates bone regeneration and degrades over time. The intent is for the artificial structure to dissipate, leaving actual bone in its place. A technology like this would go a long way to repairing complex bone defects in all manner of patients, but is particularly promising in pediatrics, where the patients are still growing.

But, in order to truly build a bone from scratch, well need something even better. Thats where Nina Tandon and EpiBone come in.

This technology would work by taking a sample of fatty tissue, something readily available if your plan is to build a copy of an existing person, and use it to extract stem cells. Those cells would then be applied to a 3D printed scaffold of a cows bone which has been scrubbed of all its living cells. Those undifferentiated stem cells would then be placed into a bioreactor (something which sounds made up but is very real) and coaxed into growing into a fully formed bone in just a few weeks. Given enough bioreactors, and enough cows (pour one out for our fallen bovine brethren) you could feasibly grow an entire skeleton in the time it takes for you to finally fold the laundry thats been sitting in the corner of your room.

Now that youve got a skeleton, youre going to need some

ORGANS

For a long time, there weren't many ways to get a new organ if you needed one. The most commonly used method (we hope) was to get your name on a list and wait for a donor. The unfortunate reality of organ donation is that there are more people who need organs than there are organs available. Even when an organ does become available, the odds are against you that theyll match your bodys preferences, and even if you get a match, theres always the threat of rejection.

Organ transplants are a veritable miracle procedure and, while we sometimes take them for granted, they are evidence of our living in truly wizardly times in medical science. But science is never content with the status quo and humanity is forever wondering if we can further laugh in the face of nature. The preferred solution would be to develop a way to craft bespoke organs, made from the recipients' own cells.

Growing cells in a petri dish is old hat. Weve been doing that for longer than many of us have been alive. The trouble is, you can take a heart cell and induce it to multiply in a dish, but all you end up with is a dish-shaped collection of heart cells. That might be good for studying cellular biology, not so good for pumping blood through a person.

A collection of cells does not an organ make. You need something more a scaffold. Each of your organs is a complex collection of various cell types clinging to a protein structure. You can think of that structure as the framing around which the rest of a house is built. Without it, you've got little more than some insulation and drywall tossed into a haphazard stack. You need that scaffold.

There are hopes that one day well be able to build them via (drum roll please) 3D printing, but were not there yet. The level of minute detail involved is beyond our current ability. So, we have to borrow from nature.

Scientists are able to take an existing organ and strip it of its surface cells by pumping detergent through it (good for removing pesky stains and unwanted biological material). Whats left is a ghostly protein structure ready for seeding.

All that's left is to take tissue samples from the recipient and seed them onto the structure, pop it into one of those handy bioreactors, and let the cells get to work. Eventually, youll end up with an organ made of the patients own tissues. Current tests are pretty impressive, but were still a ways off from having a functioning process. The number of different tissue types involved in complex organs is a barrier and the complexity of small structures like circulatory vessels is another. Still, the technology is promising and would not only allow us to build any and all organs in record time, it would solve the organ transplant shortage and save countless lives.

So, now youve got a rigid skeleton filled with juicy oozing organs. Your neighbors are starting to wonder about the smell coming from your garage and youre grateful this abominable creature is not yet sentient because it would very likely go running for the hills. At least it would if it had

MUSCLES

Look, we all know its been a while since youve been to the gym. You bought a membership for the new year and you went a few times. You really meant well but life happened and, somehow, it all got away from you. We get it. It happens to the best of us.

While you might not have the muscle mass you wish you had, you still have quite a lot. The average persons body is comprised of somewhere between 35 and 40 percent muscle, give or take. Thats a lot. Even after all of your efforts with bioreactors, youve only managed to make 60 percent of a person. Its nothing to be scoffed at, but you arent done yet.

In order to complete next steps, youre going to need more tissue samples and a few friends from Duke University.

Using human cells that were no longer stem cells but not yet muscle cells, Nenad Bursac and Lauran Madden, an associate professor of biomedical engineering and a postdoctoral researcher, respectively, were able to successfully create functioning muscle tissues in a lab.

They grew the tissue samples and, using a 3D scaffold and a nutritive gel, ended up with working muscle fibers. These bundles of muscle fibers included receptors capable of taking in external stimuli and contracted when acted on by electricity.

For their part, the intent is not to build novel muscular structures, but to test the efficacy of drugs to treat diseases. According to Bursac, drug tests in the laboratory matched results seen in living patients. Those patients with muscular ailments could provide a tissue sample, that sample could then be grown into fiber bundles and used to test various drug treatments, ex vivo, to find a workable treatment without all the trial and error usually required.

Thanks to Bursac and the team at Duke, youve now built almost all of Takeshi Kovacs. Hes twitching and moving around on the table. He might be screaming a little, thanks to those vat-grown lungs and hes still oozing a bit. Most of all, hes embarrassed by his nakedness. A lots changed in the intervening centuries, but not the need for

SKIN

Youve got your terrible Frankensteinian gift all put together, all thats left is the wrapping. Here, too, is an area were moderately familiar with. When a patient loses skin through injury, a graft can be taken from elsewhere and used to replace the damaged tissue. It gets the job done, some skin is better than no skin of course, but theres still room for improvement.

More recently, bioengineers have had some success in growing sheets of epithelial tissue for implantation but they lacked oil and sweat glands. Again, close, but not quite. Until

A study undertaken at the RIKEN Center for Developmental Biology, led by Takashi Tsuji took cells from the gums of mice and used chemicals to revert them to a stem-cell-like state. The cells were used to grow complex skin tissues.

Once the tissues were ready, they were transplanted onto living mice and were found to develop normally. Not only did those tissues function as a protective barrier, the primary function of skin, but they also succeeded in developing hair follicles and sweat glands. Even more importantly, they successfully integrated with surrounding tissue systems like muscle groups and nerves.

There are, of course, other tissue types weve not covered, each of them important to the successful functioning of a body, but if these emerging technologies are any indication, were well on our way in those areas as well.

So, youve done it. Youve made a full-grown human from scratch in months rather than decades. All thats left is to upload a mind and youre well on your way to cyberpunk chicanery. Go forth, Kovacs, we're rooting for you. And dont mess up this body, please. It was really hard to make. Thanks.

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How to build a body from scratch, Altered Carbon-style - SYFY WIRE

2nd person cured of HIV thanks to stem cell transplant – Medical News Today

Researchers in the United Kingdom have confirmed that a stem cell transplant has cured a second person of HIV.

In 2007, Timothy Ray Brown became the first person ever whom doctors declared to be cured of HIV. At the time, they referred to him publicly as the Berlin patient.

His journey toward a cure was not straightforward. After having received an HIV diagnosis in the 1990s, Mr. Brown received antiretroviral treatment the usual course of action for an HIV infection.

However, later on, he also received a diagnosis of acute myeloid leukemia, for which he eventually required a stem cell transplant.

As he was looking for a suitable donor match, his doctor had the idea to try an experiment. He looked for a donor with a specific genetic mutation that made them practically immune to HIV.

Receiving stem cells from this donor, it turned out, not only treated Mr. Browns leukemia but also cured the HIV infection.

Now, as a study featuring in The Lancet shows, another person has officially been cured of HIV, also thanks to a stem cell transplant.

In this second case, the person received a stem cell transplant with cells that did not express the CCR5 gene, which produces a protein that helps the virus enter cells.

The cells without the CCR5 gene were part of a bone marrow transplant, which the person was undergoing as a treatment for Hodgkin lymphoma.

Following the transplant, and at 30 months after the person ceased antiretroviral therapy, doctors confirmed that the HIV viral load remained undetectable in blood samples.

This finding means that whatever traces of the viruss genetic material might still be in the system, they are so-called fossil traces, meaning that they cannot lead to further replication of the virus.

The specialists confirmed that HIV also remained undetectable in samples of cerebrospinal fluid, semen, intestinal tissue, and lymphoid tissue.

We propose that these results represent the second ever case of a patient to be cured of HIV, says the studys lead author, Prof. Ravindra Kumar Gupta, from the University of Cambridge in the U.K.

Our findings show that the success of stem cell transplantation as a cure for HIV, first reported 9 years ago in the Berlin patient, can be replicated.

Prof. Ravindra Kumar Gupta

However, Prof. Gupta emphasizes that [i]t is important to note that this curative treatment is high risk and only used as a last resort for patients with HIV who also have life threatening hematological [blood] malignancies.

Therefore, this is not a treatment that would be offered widely to patients with HIV who are on successful antiretroviral treatment, the researcher goes on to caution.

Commenting on these findings, other researchers involved in the study express the hope that, in the future, scientists may be able to use state-of-the-art gene editing tools as part of interventions meant to treat and cure HIV.

Dr. Dimitra Peppa, who is from the University of Oxford in the U.K. and co-authored the study, notes that [g]ene editing using the CCR5 has received a lot of attention recently.

Nevertheless, she points out, there is still a long way to go before such therapies may become viable.

There are still many ethical and technical barriers e.g., gene editing, efficiency, and robust safety data to overcome before any approach using CCR5 gene editing can be considered as a scalable cure strategy for HIV, she says.

Continued here:
2nd person cured of HIV thanks to stem cell transplant - Medical News Today

Insights Into the $8.8 Billion Cell Therapy Industry, 2020-2027 – Rising Adoption of Regenerative Medicine, Introduction of Novel Platforms &…

DUBLIN, March 11, 2020 /PRNewswire/ -- The "Cell Therapy Market Size, Share & Trends Analysis Report by Use-type (Research, Commercialized, Musculoskeletal Disorders), by Therapy Type (Autologous, Allogeneic), by Region, and Segment Forecasts, 2020 - 2027" report has been added to ResearchAndMarkets.com's offering.

The global cell therapy market size is expected to reach USD 8.8 billion by 2027 at a CAGR of 5.4%, over the forecast period.

Cellular therapies hold a great therapeutic promise across various clinical applications. This has resulted in substantial global investments in research and clinical translation. Moreover, rapid advances in stem cell research hold the potential to fulfill the unmet demand of pharmaceutical entities, biotech entities, and doctors in disease management. These factors have boosted revenue growth for the market.

Currently, there are a limited number of FDA-approved commercial stem and non-stem cell therapies in the market. Furthermore, LAVIV (Azficel-T), manufactured and commercialized by Fibrocell Technologies, witnessed revenue wind-down in the past years. Key developers are making substantial investments in the adoption of advanced technologies to address the aforementioned challenges.

The introduction of proprietary cell lines is recognized as the primary means by which a single cell can be exploited for the production of a robust portfolio of candidates. Companies are leveraging new technologies not only for the expansion of their product portfolio but also for establishing out-licensing or co-development agreements with other entities to support their product development programs.

For instance, MaxCyte has more than 40 high-value cellular therapy partnership programs within immune-oncology, regenerative medicine, and gene editing, including fifteen clinical-stage programs. Increase in the number of collaborations between entities for product commercialization is anticipated to accelerate market revenue to a major extent in the coming years.

In Asia-Pacific, the market is anticipated to witness significant growth over the forecast period. This is attributed to rising awareness cellular therapies among patients and healthcare entities in chronic disease management. In addition, availability of therapeutic treatment at lower prices is also driving the regional market. Japan is likely to witness fast growth over the forecast period attributed to increasing research activities on regenerative medicine.

Further key findings from the report suggest:

Key Topics Covered

Chapter 1 Executive Summary

Chapter 2 Research Methodology

Chapter 3 Cell Therapy Market Variables, Trends & Scope3.1 Market Segmentation & Scope3.1.1 Market driver analysis3.1.1.1 Rise in number of clinical studies pertaining to the development of cellular therapies3.1.1.2 Rising adoption of regenerative medicine3.1.1.3 Introduction of novel platforms and technologies3.1.2 Market restraint analysis3.1.2.1 Ethical concerns related to stem cell research3.1.2.2 Clinical issues pertaining to development & implementation of cell therapy3.1.2.2.1 Manufacturing issues3.1.2.2.2 Genetic instability3.1.2.2.3 Stem cell culture condition3.1.2.2.4 Stem cell distribution after transplant3.1.2.2.5 Immunological rejection3.1.2.2.6 Challenges associated with allogeneic mode of transplantation3.2 Penetration & Growth Prospect Mapping For Therapy Type, 20193.3 Cell Therapy Market (Stem & Non-stem Cells)-Swot Analysis, by Factor (Political & Legal, Economic and Technological)3.4 Industry Analysis - Porter's3.5 Cell Therapy Market (Stem & Non-stem Cells)-Regulatory Landscape

Chapter 4 Cell Therapy Market (Stem & Non-stem Cells) Categorization: Use-type Estimates & Trend Analysis4.1 Cell Therapy Market (Stem & Non-stem Cells): Use-type Movement Analysis4.2 Clinical-use4.3 Research-use

Chapter 5 Cell Therapy Market (Stem & Non-stem Cells) Categorization: Therapy Type Estimates & Trend Analysis5.1 Cell Therapy Market (Stem & Non-stem Cells): Therapy Type Movement Analysis5.2 Allogeneic Therapies5.3 Autologous Therapies

Chapter 6 Cell Therapy Market (Stem & Non-stem Cells) Categorization: Regional Estimates & Trend Analysis, by Product6.1 Cell Therapy Market (Stem & Non-stem Cells) Share by Regional, 2019 & 20276.2 North America6.3 Europe6.4 Asia-Pacific6.5 Latin America6.6 MEA

Chapter 7 Competitive Landscape7.1 Strategy Framework7.2 Company Profiles7.2.1 Kolon TissueGene, Inc.7.2.2 JCR Pharmaceuticals Co. Ltd.7.2.3 MEDIPOST7.2.4 Osiris Therapeutics, Inc.7.2.5 Stemedica Cell Technologies, Inc.7.2.6 Cells for Cells7.2.7 NuVasive, Inc.7.2.8 Fibrocell Science, Inc.7.2.9 Vericel Corporation7.2.10 Pharmicell Co. Ltd.7.2.11 Anterogen Co. Ltd.7.2.12 Celgene Corporation

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Insights Into the $8.8 Billion Cell Therapy Industry, 2020-2027 - Rising Adoption of Regenerative Medicine, Introduction of Novel Platforms &...

Stem cells to help the heart – Science Magazine

Shinya Yamanaka's 2006 discovery of induced pluripotent stem cells (iPSCs) ignited a revolution in the field of stem cell biology (1). For the first time, nearly all human somatic tissues could be produced from iPSCs reprogrammed from blood or skin cells, in a process that took only weeks. This advance was particularly crucial for obtaining surrogate tissues from cell types that are otherwise difficult to procure and do not readily expand in vitro, such as cardiac or neural cells. Additionally, many ethical concerns are avoided, because this technology uses a patient's own genetic material to create iPSCs rather than relying on embryonic stem cells. In the aftermath of Yamanaka's discovery, entire biomedical industries have developed around the promise of using human iPSCs (hiPSCs) and their derivatives for in vitro disease modeling, drug screening, and cell therapy (2).

The hiPSC technology has had a particularly notable impact in cardiac regenerative medicine, a field where scientists and clinicians have been working to devise new methods to better understand how cardiovascular disease manifests and how to restore cardiovascular function after disease strikes (3). The heart is limited in its ability to regenerate lost cardiomyocytes (beating heart muscle cells), following an adverse event such as a heart attack (4). Cardiomyocytes derived from hiPSCs (hiPSC-CMs) may represent a potential replacement option for dead cells in such a scenario. However, certain issues remain to be addressed, such as whether hiPSC-CMs can integrate with host myocardial tissue in the long term (5).

While using hiPSC-CMs for in vivo cell therapy may become practical in the future, employing hiPSC-CMs for high-throughput drug discovery and screening is becoming a reality in the present (6). Cardiovascular diseases can be recapitulated in a dish with patient-specific hiPSC-CMs. For example, if a patient exhibits a cardiac arrhythmia caused by a genetic abnormality in a sarcomeric protein or ion channel, that same rhythm problem can be recapitulated in vitro (7). Thanks to advances in hiPSC differentiation protocols, hiPSC-CMs can now be mass-produced to study cardiovascular disease mechanisms in vitro (8).

My graduate thesis in the laboratories of Joseph Wu and Sean Wu at Stanford University focused on in vitro applications of hiPSC-CMs for cardiovascular disease modeling and for high-throughput screening of chemotherapeutic compounds to predict cardiotoxicity. I initially embarked on a project using hiPSC-CMs to model viral myocarditis, a viral infection of the heart, caused by the B3 strain of coxsackievirus (9). I began by demonstrating that hiPSC-CMs express the receptors necessary for viral internalization and subsequently found that hiPSC-CMs were highly susceptible to coxsackievirus infection, exhibiting viral cytopathic effect within hours of infection. I also identified compounds that could alleviate coxsackievirus infection on hiPSC-CMs, a translationally relevant finding, as there remains a shortage of treatments for viral myocarditis.

Using a genetically modified variant of coxsackievirus B3 expressing luciferase, I developed a screening platform for assessing the efficacy of antiviral compounds. Pretreatment with interferon-, ribavirin, or pyrrolidine dithiocarbamate markedly suppressed viral replication on hiPSC-CMs by activating intracellular antiviral response and viral protein clearance pathways. These compounds alleviated viral replication in a dose-dependent fashion at low concentrations without causing cellular toxicity.

I next sought to use hiPSC-CMs to screen anticancer chemotherapeutic compounds for their off-target cardiovascular toxicities (10). Cardiotoxicity represents a major cause of drug withdrawal from the pharmaceutical market, and several chemotherapeutic agents can cause unintended cardiovascular damage (11). Using cultured hiPSC-CMs, I evaluated 21 U.S. Food and Drug Administrationapproved tyrosine kinase inhibitors (TKIs), commonly prescribed anticancer compounds, for their cardiotoxic potential. HiPSC-CMs express the major tyrosine kinase receptor proteins such as the insulin, insulin-like growth factor (IGF), vascular endothelial growth factor (VEGF), and platelet-derived growth factor (PDGF) receptors, lending validity to this cellular model.

Initially, human induced pluripotent stem cells (hiPSCs) can be produced by reprogramming skin or blood cells by nonviral or viral reprogramming methods. Cardiac differentiation protocols allow for the creation of cardiomyocytes derived from hiPSCs (hiPSC-CMs) for downstream applications, including in vitro disease modeling, drug screening, and regenerative cell therapy.

With data from a battery of cellular apoptosis, contractility, electrophysiology, and signaling assays, I generated a cardiac safety index to help align in vitro toxicity data to clinical drug safety guidelines (12). From the safety index, I determined that a subclass of VEGF receptor 2/PDGF receptorinhibiting tyrosine kinase inhibitors, some of which exhibit toxicity clinically, also elicited cardiotoxicities in hiPSC-CMs. These manifested as substantial alterations in cellular electrophysiology, contractility, and viability when administered at clinically relevant concentrations. I also discovered that cotreatment with either IGF or insulin partially rescued TKI-induced toxicity by up-regulating antiapoptotic signaling pathways. This work could prove useful for groups aiming to develop effective screening platforms to assess new chemotherapeutic compounds for cardiotoxic side effects.

I also collaborated with the Center for the Advancement of Science in Space (CASIS) to send a sample of hiPSC-CMs to the International Space Station. As humankind ventures beyond our home planet, it is imperative that we better understand how the heart functions for long periods of time in microgravity. Analysis of these hiPSC-CMs revealed microgravity-induced alterations in metabolic gene expression and calcium handling (13).

In recent years, the stem cell field has experienced an explosion of studies using hiPSC-CMs as a model cellular system to study cardiovascular biology. As improvements in hiPSC-CM mass production continue, we will see a rise in studies using these cells for disease modeling and drug screening. Thus, although hiPSC-CM technology is in its infancy, it holds great potential to improve cardiovascular health.

PHOTO: COURTESY OF A. SHARMA

FINALIST

Arun Sharma

Arun Sharma received his undergraduate degree from Duke University and a Ph.D. from Stanford University. Having completed a postdoctoral fellowship at the Harvard Medical School, Sharma is now a senior research fellow jointly appointed at the Smidt Heart Institute and Board of Governors Regenerative Medicine Institute at the Cedars-Sinai Medical Center in Los Angeles. His research seeks to develop in vitro platforms for cardiovascular disease modeling and drug cardiotoxicity assessment. http://www.sciencemag.org/content/367/6483/1206.1

Excerpt from:
Stem cells to help the heart - Science Magazine

Induced Pluripotent Stem Cells Market Expected to Witness the Highest Growth 202 – ITResearchBrief.com

Recently, Market Study Report, LLC, added a research on the ' Induced Pluripotent Stem Cells market' which encompasses significant inputs with respect to market share, market size, regional landscape, contributing players, and revenue projection of this industry vertical. The report also educates investors regarding the existing tends, prime challenges, and current expansion strategies applied by the key organizations that constitute the hyperactive competitive gamut of this business sphere.

This research report on Induced Pluripotent Stem Cells market is an in-depth assessment of this business space, along with a brief overview of its different market segments. The study details the entire market scenario through a basic summary of the Induced Pluripotent Stem Cells market with respect to its current position and industry size, in terms of revenue and volume. The research also encompasses the important insights about the geographical outlook of the market, coupled with an elaborate study of the competitive backdrop of the Induced Pluripotent Stem Cells market.

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Induced Pluripotent Stem Cells Regional Market Analysis

Induced Pluripotent Stem Cells Segment Market Analysis (by Type)

Induced Pluripotent Stem Cells Segment Market Analysis (by Application)

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Amyotrophic Lateral Sclerosis Treatment Market Share Forecast to 2027: Orion Pharma, Kringle Pharma, Inc., Aquestive Therapeutics, Apotex Inc, Bausch…

Global Amyotrophic Lateral Sclerosis Treatment Market By Disease Type (Benign Focal Amyotrophy of ALS, Infantile Spinal Muscular Atrophy, Juvenile Spinal Muscular Atrophy, Kugelberg-Welander Disease, Primary Lateral Sclerosis, Progressive Bulbar Palsy, Spinal Muscular Atrophy, Upper Motor Neuron Disease, Werdnig-Hoffman Disease, Wohlfart-Disease), Drug Type (Riluzole and Edaravone (Radicava)), By Distribution Channel (Hospital Pharmacies, and Retail & Online Pharmacies), Treatment type (Chemotherapy, Stem cell therapy) End- User (Hospitals, Homecare, Specialty Clinics, Others), Geography (North America, South America, Europe, Asia-Pacific, Middle East and Africa) Industry Trends & Forecast to 2026

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Few of the major competitors currently working in the amyotrophic lateral sclerosis treatment market are Sanofi (France), Mitsubishi Tanabe Pharma Corporation (Japan), BrainStorm Cell Limited (US), ViroMed Co., Ltd (South Korea), Ionis Pharmaceuticals (US), Genervon Biopharmaceuticals, LLC (US), Biogen (US), ORPHAZYME A/S (Denmark), Orion Pharma (Finland), Kringle Pharma, Inc. (Japan), Aquestive Therapeutics (US), Apotex Inc (Canada), Bausch Health Companies Inc. (Canada), Neuralstem, Inc. (US), Implicit Bioscience (Australia), F. Hoffmann-La Roche Ltd (Switzerland), TREEWAY (Netherlands), CYTOKINETICS, INC. (US), AB Science (France), Advanz Pharmaceutical (Canada) and few among others.

Market Analysis:Global Amyotrophic Lateral Sclerosis Treatment Market

Global amyotrophic lateral sclerosis treatment market is expected to rise gradually to an estimated value of USD 3.6 billion by 2026, registering a CAGR of 21.6% in the forecast period of 2019-2026 with the annual sales of USD 0.75 billion in the year of 2018. This rise in market value can be attributed to the increasing awareness and concerns regarding the health of patients.

Market Definition:Global Amyotrophic Lateral Sclerosis Treatment Market

Amyotrophic lateral sclerosis (ALS) is a neurodegenerative disease, also known as motor neuron disease (MND) and Lou Gehrigs disease that affects nerve cells (motor neurons) of brain, brain stem and spinal cord that controls voluntary muscles. This disease is progressive in nature.

According to Centers for Disease Control and Prevention, there were approximately 14,000 15,000 people in US in 2016 having ALS, almost more than half of these cases could have been avoided with the availability of proper medical treatment. This significant number is expected to act as a driver to the market growth.

Market Drivers

Market Restraints

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Segmentation: Global Amyotrophic Lateral Sclerosis Treatment Market

By Disease Type

ByDrug Type

By Distribution Channel

By Treatment Type

By End- User

ByGeography

Key Developments in the Market:

Competitive Analysis:

Global amyotrophic lateral sclerosis treatment market is highly fragmented and the major players have used various strategies such as new product launches, expansions, agreements, joint ventures, partnerships, acquisitions, and others to increase their footprints in this market. The report includes market shares of amyotrophic lateral sclerosis treatment market for global, Europe, North America, Asia-Pacific, South America and Middle East & Africa.

Primary Respondents

Demand Side: Doctors, Surgeons, Medical Consultants, Nurses, Hospital Buyers, Group Purchasing Organizations, Associations, Insurers, Medical Payers, Healthcare Authorities, Universities, Technological Writers, Scientists, Promoters, and Investors among others.

Supply Side: Product Managers, Marketing Managers, C-Level Executives, Distributors, Market Intelligence, and Regulatory Affairs Managers among others.

Reasons to Purchase this Report

Customization of the Report:

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Amyotrophic Lateral Sclerosis Treatment Market Share Forecast to 2027: Orion Pharma, Kringle Pharma, Inc., Aquestive Therapeutics, Apotex Inc, Bausch...