Category Archives: Cell Medicine

[Editors Note: Kristin Marguerite Collier is an Assistant Professor of Internal Medicine at the University of Michigan Medical School where she practices general Internal Medicine. She serves as an Associate Program Director of the Internal Medicine Residency Program and is the Director of the Programs Primary Care Track. In addition, she is the Director of the University of Michigan Medical School Program on Health, Spirituality and Religion. She spoke to Charles Camosy about Mary, Jesus, and pregnancy.]

Camosy: So here we are at the end of Advent, with Marys pregnancy with Jesus at the front of our minds. Whats the first thing which comes to your mind when you think about this?

Collier: The first thing that comes to my mind is love that God so loved the world that he sent his only begotten son so that whoever believes in him should not perish but have eternal life. The fact that the creator of the universe chose a woman to bear his son to be our Savior is beautiful and awe-inspiring. Out of these emotions, flows a sense of gratitude and peace as I await with solemnity, during Advent, the birth and coming of our Lord Jesus Christ.

Obviously medical science is quite interested in studying and continuing to learn about pregnancy. What are some things that Crux readers might not know about the science behind the relationship between mother and prenatal child?

Through advances in prenatal imaging and the field of immunology, the truly wondrous miracle that is pregnancy is now being more fully understood. Two aspects of pregnancy that your readers might be interested in knowing more about relate to the placenta and something known as fetomaternal microchimerism.

As many of your readers may know, the placenta is the organ through which the mother and prenatal child interface. The placenta is an organ that is attached to the inside of the uterus and connects to the prenatal child through the childs umbilical cord.

What is not as well known about this organ is that the placenta is the only organ in human biology that is made by two persons, together, in cooperation. The placenta is built from tissue that is part from mom, and part from the growing baby. Because of this, the placenta is referred to as a feto-maternal organ. It is the only organ made by two people, in cooperation with providence. It is the first time mom and her baby come together, albeit at the cellular level, to do something in cooperation.

Whenever I think of this, I picture the ceiling of the Sistine Chapel, where Michelangelo depicts God and Man reaching out for one another, hands about to touch and perhaps entwine. In the creation of the placenta, cells from the trophoblast, which are from the embryo, reach down towards the mothers uterine wall while at the same time, the spiral arteries from the mothers uterus are reaching up towards the embryo. This process leads to the creation of the placenta.

The placenta is the only purposely transient organ in humans and unlike the rest of our organs, acts as many organs in one. The placenta functions to eliminate waste, like the kidneys would do, facilitates transfer of oxygen and carbon dioxide, like the lungs would do, and provides nutrients, like a GI tract would do. It even has endocrine and immune function. What used to be discarded as just the afterbirth is now regarded as a magnificently complex shared organ that supports the formation of the prenatal child.

The placenta is such an important organ that the National Institutes of Health (NIH) has established, under its Eunice Kennedy Shriver National Institute of Child Health and Human Development arm, the Human Placenta Project (HPP). The website says The placenta is arguably one of the most important organs in the body. It influences not just the health of a woman and her fetus during pregnancy, but also the lifelong health of both mother and child. The aim of the HPP is to better understand, through research, the amazing placenta with the ultimate goal of improving the health of children and mothers. The research done by the HPP continues to demonstrate that a childs prenatal and postnatal health is inextricably linked to the health of the placenta.

In addition to the placenta, mother and prenatal child interact at a cellular level in something known as fetomaternal microchimerism. In Greek Mythology, the chimera is a fire breathing monster comprised of three species in one a lions head, a goats body and a serpents tail. In science, microchimerism is the presence of a small population of genetically distinct and separately derived cells within an individual. During pregnancy, small numbers of cells traffic across the placenta. Some of the prenatal childs cells cross into the mother, and some cells from the mother cross into the prenatal child. The cells from the prenatal child are pluripotent and integrate into tissues in her mothers body and start functioning like the cells around them. This integration is known as feto-maternal microchimerism.

The presence of these cells is amazing for several reasons. One is that these cells have been found in various maternal organs and tissues such as the brain, the breast, the thyroid and the skin. These are all organs which in some way are important for the health of both the baby and her mother in relationship. The post-partum phase is when there is need, for example, for lactation. The fetomaternal microchimeric cells have been shown to be important in signaling lactation. These cells have been found in the skin, for example, in Cesarean section incisions where they are helping to produce collagen. Baby is helping mom heal after delivery by the presence of her cells! It would be one thing for these cells to come into the mother and be inert, but is a whole other thing entirely that these cells are active and aid mom for example in helping to produce milk for her baby and helping her heal. These cells may even affect how soon the mother can get pregnant again and therefore can affect spacing of future siblings.

Usually, foreign or other cells are detected by the host immune system and are destroyed. The fact that these fetal cells survive and then are allowed to integrate into maternal tissue speaks to a cooperation between the mother and her child at the level of the cell that parallels that seen in the development of the placenta, suggesting that the physical connection between mom and baby is even deeper and more beautiful than previously thought. Research in fetomaternal microchimerism suggests that the presence of these cells may favorably affect the future risk of malignancy. The presence of these cells in the maternal breast may help protect mom from breast cancer years after the babys birth.

To think that a physical presence of the baby in her mother is helping protect her from cancer at the level of the cell, speaks to a radical mutuality at the cellular level that we are just beginning to understand. Some of the effects of fetomaternal microchimerism, however, may be detrimental in some cases. This research is still underway. The big takeaway is that the science of microchimerism supports the fact that some human beings carry remnants of other humans in their bodies. Thus, we arent the singular-autonomous individuals we think of ourselves as being.

This is astonishing! Why do you think these facts are not more widely known? Why arent pregnant women, or new mothers, informed of stuff like this?

I agree. It is truly astonishing. Im not entirely clear why this type of information isnt more widely known or made available in information given to women as they go through their pregnancy. I can only hypothesize on reasons why. In my opinion, pregnancy, like so many other conditions experienced by people, has been purely medicalized and distilled down to practical facts. Having had the blessing of four pregnancies myself, I remember the information that I was given. I received information about what I, as a pregnant woman, may experience regarding changes in my body or other common symptoms related to pregnancy, and was given a timeline with expected growth of the baby at each stage. I recall very little communication by the medical staff that spoke to wonder or awe in the process. I know that most women experience an immense sense of wonder and awe during their pregnancy that sometimes isnt celebrated and discussed perhaps as much as it should be. The implicit message I fear women get sometimes is that billions of women before you and after you have and will become pregnant and this isnt as special and amazing as you personally think it is. But yes it is! As Einstein said, you either believe everything in this life is a miracle or nothing is.

Lets turn back to theology. Especially in light of Marys pregnancy with Jesus, what theological implications are there for these new things we are learning?

I am not a theologian, however, I think the existence of fetomaternal microchimerism speaks to an interconnectedness at the cellular level by Gods design. As I wrote about in a piece for Church Life Journal earlier this year, we see relationship at the heart of the Scriptures. We see that God, the maker of all things, is in a covenant bond with his people. We would be wrong to think that God, being the maker of all things, and being relational in his very nature, would not reveal in our biology a relational nature.

As a non-Catholic Christian, what implications does this medical science have, in your view, for Catholic veneration of Mary as Mother of God?

Jesus Christ redeemed every stage and aspect of our bodies through which he has passed. The Word became flesh in Marys uterus. Therefore, the uterus is a sacred space because it held our Lord and Savior. If we consider the biological reality of fetomaternal microchimerism, we can assume that some of Jesus cells transferred across the placenta in Marys womb into the Blessed Mother. What we could take from this is that even when Jesus physically left his mother, part of him remained in her and remains in her forever. This further magnifies her position as the glorious Theotokas.

I can also imagine that fetomaternal microchimerism can carry significant meaning not only for Catholic women, but for all women who have lost prenatal children or children after birth because we know that mothers have always thought, in some way, their children, even after death, were still with them. Now we see, through the lens of fetomaternal microchimerism, that they still are.

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Medical discoveries about pregnancy could shed light on Mary as Mother of God - Crux: Covering all things Catholic

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

Gene therapy is fighting to enter mainstream medicine. With sickle cell disease, the fight is heating up.

Roughly two years ago, the FDA made the historic decision to approve the first gene therapy in the US, finally realizing the therapeutic potential of hacking our biological base code after decades of cycles of hope and despair. Other approvals soon followed, including Luxturna to target inherited blindness and Zolgensma, a single injection that could save children with a degenerative disease from their muscles wasting away and dying before the age of two.

Yet despite their transformative potential, gene therapy has only targeted relatively rareand often fataldisorders. Thats about to change.

This year, a handful of companies deployed gene therapy against sickle-cell anemia, a condition that affects over 20 million people worldwide and 100,000 Americans. With over a dozen therapies in the run, sickle-cell disease could be the indication that allows gene therapy to enter the mainstream. Yet because of its unique nature, sickle-cell could also be the indication that shines an unflinching spotlight on challenges to the nascent breakthrough, both ethically and technologically.

You see, sickle-cell anemia, while being one of the worlds best-known genetic diseases, and one of the best understood, also predominantly affects third-world countries and marginalized people of color in the US. So far, gene therapy has come with a hefty bill exceeding millions; few people afflicted by the condition can carry that amount. The potential treatments are enormously complex, further upping costs to include lengthy hospital stays, and increasing potential side effects. To muddy the waters even more, the disorder, though causing tremendous pain and risk of stroke, already has approved pharmaceutical treatments and isnt necessarily considered life-threatening.

How we handle gene therapies for sickle-cell could inform many other similar therapies to come. With nearly 400 clinical trials in the making and two dozen nearing approval, theres no doubt that hacking our genes will become one of the most transformative medical wonders of the new decade. The question is: will it ever be available for everyone in need?

Even those uninterested in biology have likely heard of the disorder. Sickle-cell anemia holds the crown as the first genetic disorder to be traced to its molecular roots nearly a hundred years ago.

The root of the disorder is a single genetic mutation that drastically changes the structure of the oxygen-carrying protein, beta-globin, in red blood cells. The result is that the cells, rather than forming their usual slick disc-shape, turn into jagged, sickle-shaped daggers that damage blood vessels or block them altogether. The symptoms arent always uniform; rather, they come in crisis episodes during which the pain becomes nearly intolerable.

Kids with sickle-cell disorder usually die before the age of five; those who survive suffer a lifetime of debilitating pain and increased risk of stroke and infection. The symptoms can be managed to a degree with a cocktail of drugsantibiotics, painkillers, and a drug that reduces crisis episodes but ups infection risksand frequent blood transfusions or bone marrow transplants. More recently, the FDA approved a drug that helps prevent sickled-shaped cells from forming clumps in the vessels to further combat the disorder.

To Dr. David Williams at Boston Childrens Hospital in Massachusetts, the availability of these treatmentshowever inadequatesuggests that gene therapy remains too risky for sickle-cell disease. Its not an immediately lethal diseaseit wouldnt be ethical to treat those patients with a highly risky experimental approach, he said to Nature.

Others disagree. Freeing patients from a lifetime of risks and pain seems worthy, regardless of the price tag. Inspired by recent FDA approvals, companies have jumped onto three different treatments in a bitter fight to be the first to win approval.

The complexity of sickle-cell disease also opens the door to competing ideas about how to best treat it.

The most direct approach, backed by Bluebird Bio in Cambridge, Massachusetts, uses a virus to insert a functional copy of the broken beta-globin gene into blood cells. This approach seems to be on track for winning the first FDA approval for the disorder.

The second idea is to add a beneficial oxygen-carrying protein, rather than fixing the broken one. Here, viruses carry gamma-globin, which is a variant mostly present in fetal blood cells, but shuts off production soon after birth. Gamma-globin acts as a repellent that prevents clotting, a main trigger for strokes and other dangerous vascular diseases.

Yet another idea also focuses on gamma-globin, the good guy oxygen-carrier. Here, rather than inserting genes to produce the protein, the key is to remove the breaks that halt its production after birth. Both Bluebird Bio and Sangamo Therapeutics, based in Richmond, California, are pursing this approach. The rise of CRISPR-oriented companies is especially giving the idea new promise, in which CRISPR can theoretically shut off the break without too many side effects.

But there are complications. All three approaches also tap into cell therapy: blood-producing cells are removed from the body through chemotherapy, genetically edited, and re-infused into the bone marrow to reconstruct the entire blood system.

Its a risky, costly, and lengthy solution. Nevertheless, there have already been signs of success in the US. One person in a Bluebird Bio trial remained symptom-free for a year; another, using a CRISPR-based approach, hasnt experienced a crisis in four months since leaving the hospital. For about a year, Bluebird Bio has monitored a dozen treated patients. So far, according to the company, none has reported episodes of severe pain.

Despite these early successes, advocates worry about the actual impact of a genetic approach to sickle-cell disease.

Similar to other gene therapies, the treatment is considered a last-line, hail Mary solution for the most difficult cases of sickle cell disease because of its inherent risks and costly nature. Yet end-of-the-line patients often suffer from kidney, liver, and heart damages that make chemotherapy far too dangerous.

Then theres the problem of global access. Some developing countries, where sickle-cell disease is more prevalent, dont even have consistent access to safe blood transfusions, not to mention the laboratory equipment needed for altering blood-producing stem cells. Recent efforts in education, early screening, and prevention have also allowed people to live longer and reduce the stigma of the disorder.

Is a $1 million price tag ever attainable? To combat exhorbitant costs, Bluebird Bio is offering an installment payment plan for five years, which can be terminated anytime the treatment stops working. Yet for patients in South Africa, India, or Cambodia, the costs far exceed the $3 per month price tag for standard treatment. Even hydroxyurea, the newly-approved FDA drug to reduce crisis pain episodes, is just a fraction of the price tag that comes with gene therapy.

As gene therapy technologies are further refined and their base cost reduced, its possible that overall costs will drop. Yet whether these treatments will be affordable in the long run remains questionable. Even as scientists focus on efficacy rather than price tag, NIH director Dr. Francis Collins believes not thinking about global access is almost unethical. There are historical examples for optimism: vaccines, once rather fringe, now touch almost every corner of our world with the help of scientific knowledge, advocacy groups, andfundamentallyproven efficacy.

With the rise of gene therapy, were now in an age of personalized medicine beyond imagination. Its true that perhaps sickle-cell disease genetic therapies arent quite there yet in terms of safety and efficacy; but without tackling access issues, the therapy will be stymied in its impact for global good. As genetic editing tools become more powerful, gene therapy has the potential to save even more livesif its made accessible to those who need it most.

Image Credit: Image by Narupon Promvichai from Pixabay

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Gene Therapy for Sickle-Cell Anemia Looks Promisingbut It's Riddled With Controversy - Singularity Hub

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Discovery promises to aid production of protein-based drugs, vaccines, other biomaterials

Tubes of green fluorescent protein glow more brightly when they contain more of the protein. Researchers at Washington University School of Medicine have found a way to increase protein production up to a thousandfold, a discovery that could aid production of proteins used in the medical, food, agriculture, chemical and other industries.

Medicines such as insulin for diabetes and clotting factors for hemophilia are hard to synthesize in the lab. Such drugs are based on therapeutic proteins, so scientists have engineered bacteria into tiny protein-making factories. But even with the help of bacteria or other cells, the process of producing proteins for medical or commercial applications is laborious and costly.

Now, researchers at Washington University School of Medicine in St. Louis have discovered a way to supercharge protein production up to a thousandfold. The findings, published Dec. 18 in Nature Communications, could help increase production and drive down costs of making certain protein-based drugs, vaccines and diagnostics, as well as proteins used in the food, agriculture, biomaterials, bioenergy and chemical industries.

The process of producing proteins for medical or commercial applications can be complex, expensive and time-consuming, said Sergej Djuranovic, PhD, an associate professor of cell biology and physiology and the studys senior author. If you can make each bacterium produce 10 times as much protein, you only need one-tenth the volume of bacteria to get the job done, which would cut costs tremendously. This technique works with all kinds of proteins because its a basic feature of the universal protein-synthesizing machinery.

Proteins are built from chains of amino acids hundreds of links long. Djuranovic and first author Manasvi Verma, an undergraduate researcher in Djuranovics lab, stumbled on the importance of the first few amino acids when an experiment for a different study failed to work as expected. The researchers were looking for ways to control the amount of protein produced from a specific gene.

We changed the sequence of the first few amino acids, and we thought it would have no effect on protein expression, but instead, it increased protein expression by 300%, Djuranovic said. So then we started digging in to why that happened.

The researchers turned to green fluorescent protein, a tool used in biomedical research to estimate the amount of protein in a sample by measuring the amount of fluorescent light produced. Djuranovic and colleagues randomly changed the sequence of the first few amino acids in green fluorescent protein, generating 9,261 distinct versions, identical except for the very beginning.

The brilliance of the different versions of green fluorescent protein varied a thousandfold from the dimmest to the brightest, the researchers found, indicating a thousandfold difference in the amount of protein produced. With careful analysis and further experiments, Djuranovic, Verma and their collaborators from Washington University and Stanford University identified certain combinations of amino acids at the third, fourth and fifth positions in the protein chain that gave rise to sky-high amounts of protein.

Moreover, the same amino-acid triplets not only ramped up production of green fluorescent protein, which originally comes from jellyfish, but also production of proteins from distantly related species like coral and humans.

The findings could help increase production of proteins not only for medical applications, but in food, agriculture, chemical and other industries.

There are so many ways we could benefit from ramping up protein production, Djuranovic said. In the biomedical space, there are many proteins used in drugs, vaccines, diagnostics and biomaterials for medical devices that might become less expensive if we could improve production. And thats not to mention proteins produced for use in the food industry theres one called chymosin that is very important in cheese-making, for example the chemical industry, bioenergy, scientific research and others. Optimizing protein production could have a broad range of commercial benefits.

Verma M, Choi J, Cottrell KA, Lavagnino Z, Thomas EN, Pavlovic-Djuranovic S, Szczesny P, Piston DW, Zaher HS, Puglisi JD, Djuranovic S. A short translational ramp determines the efficiency of protein synthesis. Nature Communications. Dec. 18, 2019. DOI: 10.1038/s41467-019-13810-1

This work is supported by the National Institutes of Health (NIH), grant numbers R01 R01GM112824, R01GM51266, R01GM113078, R01DK115972 and T32GM007067; the Skandalaris Center LEAP Award; JDRF, award number 3-APF-2018-573-A-N; and Stanford University Bio-X Fellowship.

SD holds US Provisional Patent #62/540,897 Methods to modulate protein translation efficiency. This patent is owned by Washington University and managed by the Washington University Office of Technology Management (reference numberT061889)

Washington University School of Medicines 1,500 faculty physicians also are the medical staff of Barnes-Jewish and St. Louis Childrens hospitals. The School of Medicine is a leader in medical research, teaching and patient care, ranking among the top 10 medical schools in the nation by U.S. News & World Report. Through its affiliations with Barnes-Jewish and St. Louis Childrens hospitals, the School of Medicine is linked to BJC HealthCare.

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Scientists find way to supercharge protein production - Washington University School of Medicine in St. Louis

NEW YORK, Dec. 17, 2019 /PRNewswire/ -- The global 3D cell culture market is projected to grow at a CAGR of 15.7% during the forecast period.

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The 3D cell culture market is projected to reach USD 1,846 million by 2024 from USD 892 million in 2019, at a CAGR of 15.7%. The growth in this market is primarily driven by the increasing focus on developing alternatives to animal testing, growing focus on personalized medicine, increasing incidence of chronic diseases, and the availability of funding for research. On the other hand, the lack of infrastructure for 3D cell-based research and the high cost of cell biology research are expected to limit market growth during the forecast period.

The microfluidics-based 3D cell cultures segment is projected to grow at the highest CAGR during the forecast period.Based on product, the 3D cell culture market is segmented into scaffold-based, scaffold-free, microfluidics-based, and magnetic & bioprinted 3D cell cultures.The microfluidics-based segment is expected to register the highest CAGR during the forecast period.

Funding initiatives from various government and private investors are among the key factors driving the growth of this market.

The cancer and stem cell research segment accounted for the largest share of the 3D cell culture market in 2018.On the basis of application, the 3D cell culture market is segmented into cancer & stem cell research, drug discovery & toxicology testing, and tissue engineering & regenerative medicine.The cancer & stem cell research segment accounted for the largest share of the market in 2018.

The increasing prevalence of cancer and significant funding initiatives for cancer research from the government as well as the private sector are some of the major factors driving the growth of this application segment.

Europe to witness high growth during the forecast period.Based on region, the 3D cell culture market is segmented into North America, Europe, Asia Pacific, and the Rest of the World (RoW). The European market is expected to grow at the highest CAGR owing to the growth of the pharmaceutical and biotechnology industry, increasing incidence of cancer, growing number of venture capital investments, strategic expansion of market players in the region, recent commercialization of microfluidic-based products, increasing presence of major market players, and the large number of research activities in the region.

The primary interviews conducted for this report can be categorized as follows: By Company Type: Tier 1: 50%, Tier 2: 30%, and Tier 3: 20% By Designation: C-level: 37%, D-level: 29%, and Others: 34% By Region: North America: 38%, Europe: 23%, Asia: 30%, and the RoW: 9%

List of companies profiled in this report Thermo Fisher Scientific (US) Corning Incorporated (US) Merck (Germany) Lonza AG (Switzerland) REPROCELL Incorporated (Japan) TissUse (Germany) InSphero (Switzerland) Synthecon (US) 3D Biotek (US) CN Bio (UK) Hamilton Company (US) MIMETAS (Netherlands) Emulate (US) Hrel Corporation (US) QGel SA (Switzerland) SynVivo (US) Advanced BioMatrix (US) Greiner Bio-One International (Austria) PromoCell (Germany)

Research Coverage:The report provides an overview of the 3D cell culture market.It aims at estimating the market size and growth potential of this market across different segments such as product, application, end user, and region.

The report also includes an in-depth competitive analysis of the key players in the market, along with their company profiles, recent developments, and key market strategies.

Key Benefits of Buying the Report:The report will help the market leaders/new entrants in the 3D cell culture market by providing them with the closest approximations of revenues for the overall market and its subsegments.This report will help stakeholders to understand the competitive landscape better and gain insights to position their businesses and help companies adopt suitable go-to-market strategies.

The report also helps stakeholders understand the pulse of the market and provide them with information regarding key market drivers and opportunities.

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The 3D cell culture market is projected to reach USD 1,846 million by 2024 from USD 892 million in 2019, at a CAGR of 15.7% - PRNewswire

SOUTH SAN FRANCISCO, Calif. and CAMBRIDGE, Mass., Dec. 18, 2019 (GLOBE NEWSWIRE) -- Global Blood Therapeutics , Inc. (GBT) (NASDAQ: GBT) and Syros Pharmaceuticals, Inc. (NASDAQ: SYRS) today announced that they have entered into a collaboration to discover, develop and commercialize novel therapies for sickle cell disease (SCD) and beta thalassemia. Under the agreement, Syros will use its leading gene control platform to identify therapeutic targets and discover drugs that induce fetal hemoglobin, and GBT will receive an option to obtain an exclusive worldwide license to develop, manufacture and commercialize products resulting from the collaboration.

The discovery and development of novel therapeutic approaches to treat sickle cell disease has been a driving force for GBT since we were founded, said Ted W. Love, M.D., president and CEO of GBT. We believe that Syros approach to inducing fetal hemoglobin is one of the most promising ways to identify the next generation of therapies to treat sickle cell disease and beta thalassemia at a fundamental level upstream of serious complications such as organ damage, organ failure and early death. We will continue to seek the best scientific approaches to transform the treatment of these devastating lifelong diseases.

Using its gene control platform to elucidate mechanisms controlling gamma globin gene expression, Syros identified components of LRF (leukemia/lymphoma-related factor) and the NuRD (nucleosome remodeling and histone deacetylation) complex that could serve as potential targets to switch on the gamma globin gene, which is normally silenced a few months after birth. By turning on gamma globin expression, GBT and Syros aim to induce the production of fetal hemoglobin, which is known to exert protective effects on the red blood cells of patients with SCD and beta thalassemia and mitigate the clinical manifestation of these diseases.

We believe it is possible to provide a functional cure for patients with sickle cell disease or beta thalassemia by switching on the gamma globin gene with an oral medicine, said Nancy Simonian, M.D., CEO of Syros. Partnering with GBT, an established leader in sickle cell disease with proven research, development, manufacturing and commercialization capabilities, allows us to expand and accelerate our program, exploring multiple approaches in parallel with the aim of bringing much-needed new therapies to market for patients with sickle cell disease and beta thalassemia as quickly as possible.

Syros drug discovery program in SCD was highlighted recently in an oral presentation at the 61st American Society of Hematology (ASH) Annual Meeting, as well as in an ASH press briefing. In that presentation, Syros described its discovery of a fetal hemoglobin repressor that, when knocked down in primary cells and an erythroid cell line expressing adult hemoglobin, induced fetal hemoglobin in nearly 100% of cells and increased total fetal hemoglobin levels to 40%, exceeding levels that are associated with a functional cure in SCD patients.

Terms of the AgreementUnder the terms of the agreement, GBT will pay Syros $20 million upfront and fund up to $40 million in preclinical research for at least three years. Should GBT exercise its option under the agreement, Syros could receive up to $315 million in option exercise, development, regulatory, commercialization and sales-based milestones per product candidate and product resulting from the collaboration. Syros would also receive mid- to high-single digit royalties on sales of products resulting from the collaboration. In addition, Syros would have the option to co-promote the first product resulting from the collaboration in the United States.

About GBTGBT is a biopharmaceutical company dedicated to the discovery, development and delivery of life-changing treatments that provide hope to underserved patient communities. Founded in 2011, GBT is delivering on its goal to transform the treatment and care of sickle cell disease (SCD), a lifelong, devastating inherited blood disorder. The company has introduced Oxbryta (voxelotor), the first FDA-approved treatment that directly inhibits sickle hemoglobin polymerization, the root cause of SCD. GBT is also advancing its pipeline program in SCD with inclacumab, a p-selectin inhibitor in development to address pain crises associated with the disease. In addition, GBTs drug discovery teams are working on new targets to develop the next generation of treatments for SCD. To learn more, please visit http://www.gbt.com and follow the company on Twitter @GBT_news.

About Syros PharmaceuticalsSyros is redefining the power of small molecules to control the expression of genes. Based on its unique ability to elucidate regulatory regions of the genome, Syros aims to develop medicines that provide a profound benefit for patients with diseases that have eluded other genomics-based approaches. Syros is advancing a robust pipeline of development candidates, including SY-1425, a first-in-class oral selective RAR agonist in a Phase 2 trial in a genomically defined subset of acute myeloid leukemia patients, and SY-5609, a highly selective and potent oral CDK7 inhibitor in investigational new drug application-enabling studies in cancer. Syros also has multiple preclinical and discovery programs in oncology and monogenic diseases, including sickle cell disease. For more information, visit http://www.syros.com and follow us on Twitter (@SyrosPharma) and LinkedIn.

Forward-Looking StatementsCertain statements in this press release are forward-looking within the meaning of the Private Securities Litigation Reform Act of 1995, including statements containing the words will, anticipates, plans, believes, forecast, estimates, expects and intends, or similar expressions. These forward-looking statements are based on the current expectations of GBT and Syros, and actual results could differ materially. Statements in this press release may include statements that are not historical facts and are considered forward-looking within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. GBT and Syros each intend these forward-looking statements, including statements regarding the ability of the parties to discover, develop and commercialize novel therapies for SCD and beta thalassemia under the collaboration, the scientific and therapeutic potential of Syros gene control platform and approach to inducing fetal hemoglobin, the exercise by GBT of its option under the collaboration agreement, the potential milestone payments and royalties due to Syros under the collaboration agreement, and Syros option to co-promote the first product resulting from the collaboration in the United States, to be covered by the safe harbor provisions for forward-looking statements contained in Section 27A of the Securities Act and Section 21E of the Securities Exchange Act, and GBT and Syros make this statement for purposes of complying with those safe harbor provisions. These forward-looking statements reflect the current views of GBT and Syros about their respective plans, intentions, expectations, strategies and prospects, which are based on the information currently available to the companies and on assumptions the companies have made. Neither GBT nor Syros can give any assurance that the plans, intentions, expectations or strategies will be attained or achieved, and, furthermore, actual results may differ materially from those described in the forward-looking statements and will be affected by a variety of risks and factors that are beyond the control of GBT and Syros including, without limitation, the timing and progress of, and any data generated from, the parties research and development activities under the collaboration, and the amount and timing of resources devoted by each of the parties to activities under the collaboration, along with those risks set forth in GBT and Syros respective Annual Reports on Form 10-K for the fiscal year ended December 31, 2018, and most recent Quarterly Reports on Form 10-Q filed with the U.S. Securities and Exchange Commission, as well as discussions of potential risks, uncertainties and other important factors in the companies subsequent filings with the U.S. Securities and Exchange Commission. Except as required by law, neither GBT nor Syros assumes any obligation to update publicly any forward-looking statements, whether as a result of new information, future events or otherwise.

Contact Information:

Global Blood Therapeutics (GBT)

MediaSteven Immergut650-410-3258media@gbt.com

InvestorsStephanie Yao650-741-7730investor@gbt.com

Syros Pharmaceuticals

MediaNaomi Aoki617-283-4298naoki@syros.com

InvestorsHannah Deresiewicz212-362-1200hannah.deresiewicz@sternir.com

Original post:
GBT and Syros Partner to Discover, Develop and Commercialize Novel Therapies for Sickle Cell Disease and Beta Thalassemia - BioSpace

Bacterial nano-syringe injects the toxic protein.

Pathogens can use a range of toxins to damage their host organism. Bacteria, such as those responsible for causing the deadly Plague, use a special injection mechanism to deliver their poisonous contents into the host cell. Stefan Raunser, Director at the Max Planck Institute for Molecular Physiology in Dortmund, together with his team, has already produced a detailed analysis of this toxins sophisticated mechanism. They have now succeeded in replacing the toxin in this nano-syringe with a different substance. This accomplishment creates a basis for their ultimate goal to use bacterial syringes as drug transporters in medicine.

As soon as bacteria have entered a host organism, they deploy their lethal weapon. Just like a syringe needle, they insert a channel through the outer protective layer of the host cell. The toxic protein contained in the capsule is then injected and attacks the cells structural framework. Within just a few minutes, the cell dies.

Raunsers team discovered this lethal mechanism by using cryo-electron microscopy, a technique employed by only a few research groups in the world. What this technique reveals is the three-dimensional structure of proteins in near-atomic resolution.

Targeted injection into body cells

Stefan Raunsers researchers have now found a way to replace the toxin in the bacterias nano-syringe with different proteins, and then inject them into cells. For the exchange to work, however, the proteins must fulfil certain criteria: they must be a particular size, be positively charged, and must not interact with the toxin capsule. With this technique, we have taken the first step towards our ultimate goal of using these nano-syringes in medicine to introduce drugs into body cells in a targeted manner, says Raunser, describing the successful research results.

To transfer its toxic charge into the host cell, the injection mechanism must first dock with the cell. But the bacteria must trick the host cell- by pretending that the toxin is a substance that can be safely absorbed similar to the famous trojan horse trick. To do this, they have areas that are recognized by sensors on the cell surface.

We are currently looking for the toxins docking stations. Once we have found them and understood how the toxin binds to the cell surface, we aim to specifically modify the injection mechanism so that it can recognize cancer cells. We could then inject a killer protein exclusively into tumour cells. This would open up completely new possibilities in cancer medicine with minimal side effects, predicts Raunser.

More here:
Protein Injections in Medicine - Global Health News Wire

XconomyNational

More than 250 miles above the Earths surface aboard the International Space Station, a first-in-kind study of neurodegenerative disease is expected to reveal never-before-seen cell interactions.

The National Stem Cell Foundation (NSCF) is funding the study, which is the result of a bi-coastal collaboration between the New York Stem Cell Foundation (NYSCF) Research Institute and Aspen Neuroscience, a San Diego startup developing personalized cell therapies for Parkinsons disease.

Collaborating with the New York Stem Cell Foundation (NYSCF) Research Institute on the other side of the country, the two teams have been working together for more than two years, exchanging and sharing technology to develop patient-derived, induced pluripotent stem cell (iPSC) organoid models.

The 3D human organoid models were launched to the International Space Station earlier this month for research in microgravity, with the goal of furthering our understanding of neurogenerative diseases back on earth.

The models incorporate microglia, the inflammatory cells of the immune system that are implicated in the development of Parkinsons, multiple sclerosis, and other neurodegenerative diseases, explains Paula Grisanti, CEO of NSCF.

Studying the 3D models in microgravity, researchers are able to observe cell interaction, gene expression, and other developments not seen in a regular lab.

Its not possible for you to have this same 3D model of cell interaction on Earth. This will be the first time in space where we can that in 3D, Grisanti tells Xconomy.

Cells behave differently in space, though its not completely understood why. Cartilage grows faster and bigger, proteins fold differently, and cells mature more rapidly. Being able to see this happen in real-timethe models will be filmed for the full 30 dayswill offer researchers unprecedented insight into neurodegenerative disease.

To see how those cells talk to each other for 30 days when they are up on the international space station will allow scientists to see the point at which things start to go awry in those diseases and hopefully identify a new place or a new point at which you could intervene with a cell or gene therapy that may or may not currently exist, says Grisanti.

The research will touch back down to earth in early January at which time both labs will analyze the models to determine what exactly happened during their time in space. All data will be published for full dissemination.

(Paul Kuehl, Jason Rexroat, Gentry Barnett, Valentina Fossati, Jason Stein, Scott Noggle, Jana Stoudemire. Image courtesy of Space Tango)

NSCF has budgeted for a year of post-flight research after which the researchers will send the models back to the space station for a second flight to confirm what they saw and test new hypotheses, explains Grisanti. A second year of post-flight research also is funded, as is a second flight at the end of 2020.

We know were going to see something new because it has never been done before, says Grisanti, who explains that the budget and project will continue to be extended as long as new theories and opportunities are being developed.

The December flight was the second for the research teams at Apsen and NYSCF. A preliminary flight was conducted in July 2019 to test the hardware systems and prepare for the SpaceX CRS-19 launch.

Aspen has also been pressing ahead with its own research on solid ground. Last week, the company closed a $6.5 million seed round led by Domain Associates and Axon Ventures.

Aspens cell therapy approach was developed by its co-founders, Jeanne Loring, professor emeritus and founding director of the Center for Regenerative Medicine at The Scripps Research Institute and Andres Bratt-Leal, a former post-doctoral researcher in Lorings lab. Also serving as Aspens chief scientific officer, Jeanne Loring was in May named Xconomys Stem Cell Pioneer of the Year.

(Main image: Experiment loaded for launch at Kennedy Space Center. Courtesy of Space Tango)

Melissa Fassbender is an Xconomy editor based in Chicago. You can reach her at mfassbender@xconomy.com.

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Orbiting Organoids: Research in Space to Unveil New Neurodegeneration Insight - Xconomy

UC San Diego School of Medicine researchers developed a mathematical equation to predict when healthy liver cells become cancerous before tumors are visible in a standard clinical setting.

By combining RNA sequencing, bioinformatics and mathematical modeling, University of California San Diego School of Medicine and Moores Cancer Center researchers identified a sudden transcriptomic switch that turns healthy liver tissue cancerous. The finding was used to develop a quantitative analytical tool that assesses cancer risk in patients with chronic liver disease and to predict tumor stages and prognosis for patients with liver cancer.

In the December 16, 2019 online edition of the Proceedings of the National Academy of Science (PNAS), Gen-Sheng Feng, PhD, professor of in the Department of Pathology and Section of Molecular Biology, Division of Biological Sciences at UC San Diego, and team describe developing a tumorigenic index score that identifies a shift from healthy to malignant cells.

Because we do not have an effective drug to treat liver cancer in its late stages, early detection of liver cancer, when a tumor is less than 10 millimeters, allows oncologists to better treat, surgically remove and kill cancer cells, said Feng, senior author on the paper. For the first time, we have a mathematical equation that can predict when healthy liver cells become cancerous and, importantly, we are able to detect cancer cells before tumors are visible in a standard clinical setting.

The new analytical tool focused on the analysis of transcription factor clusters. Transcription factors are proteins that bind to specific DNA sequences in order to direct which genes should be turned on or off in a cell. By quantitatively measuring changes in transcription factors together with downstream target genes as a unit (transcription factor clusters), the research group interrogated RNA-sequencing data collected in the pre-cancer and cancer stages of mouse models with different forms of liver cancer and chronic liver diseases like steatosis, fibrosis and cirrhosis.

The analysis found 61 transcription factor clusters that were either up- or down-regulated in mice with cancer, even identifying transcription factors that have not been previously reported in liver cancer.

Gaowei Wang, PhD, a computational biologist and postdoctoral fellow in Fengs lab, helped design a comprehensive analysis of a liver cell transcriptome the entire collection of RNA sequences in a cell. This allowed the team to compare expression of transcription factor clusters in healthy livers and those with chronic liver diseases at various stages to identify when cells became cancerous in mice.

After developing the math model using mouse data, researchers applied the analytical tool to a public database to re-analyze human patient data and were able to identify which people had cancer and which had chronic liver disease. In patients with cirrhosis, who are at high risk of developing cancer, they could see a positive tumor index score and in some cases tumor nodules that were not yet visible in the clinic.

This mathematical approach can be developed into a risk assessment and early diagnostic tool of liver cancer development for a larger population of people living with chronic liver disease, particularly those with cirrhosis, said Feng. The analysis of individuals at high risk may have an important application in precision medicine. And, with further development and optimization, this tool might be modified to predict the development of other cancers.

According to the American Cancer Society, more than 700,000 new cases of liver cancer are diagnosed globally and 600,000 deaths occur each year, making it among the leading causes of cancer death in the world. In 2019, an estimated 42,000 new cases of liver cancer will be diagnosed and 31,000 people will die in the United States alone.

Further testing is needed before it can be used in a clinical setting. The next step is to analyze liver biopsies, with the ultimate goal of using blood samples to predict risk and stage liver cancer, said Feng.

Co-authors include: Xiaolin Luo, Yan Liang, Kota Kaneko, Hairi Li and Xiang-Dong Fu, all from UC San Diego.

Funding for this research came, in part, from the National Institutes of Health (R01CA188506, R01CA176012).

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Math Equation Predicts and Detects Liver Cancer - UC San Diego Health

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Ribon Therapeutics, a clinical stage biotechnology company developing first-in-class therapeutics targeting novel enzyme families activated under cellular stress conditions, today announced the appointment of Neal Rosen, M.D., Ph.D., to its scientific advisory board (SAB). Dr. Rosen is a Member of the Molecular Pharmacology Program and the Department of Medicine at Memorial Sloan Kettering Cancer Center (MSK).

Neals experience identifying and elucidating new molecular signaling pathways and understanding how to transform these early discoveries into effective drug development strategies fits perfectly with the work were undertaking at Ribon, said Victoria Richon, Ph.D., President and Chief Executive Officer, Ribon Therapeutics. We see great untapped potential for the development of novel, first-in-class therapeutics that target the novel stress response pathways that many cancers rely on for survival, and working with experts such as Neal, who have successfully forged new paths in drug development will be invaluable.

Targeting stress response pathways to treat cancer has great potential, and shares similarities with the discovery and therapeutic targeting of kinase pathways, an area I have spent much of my career working to better understand and advance, commented Dr. Rosen. It is now understood that stress response pathways play a wide range of roles aiding cancer development and growth. I look forward to working with this truly pioneering team.

Ribon has done tremendous work taking its foundational discoveries and translating them into development programs, as evidenced by how quickly the Company was able to advance their lead program, RBN-2397, into the clinic, said Jim Audia, Ph.D., Chairman of the Ribon SAB. At this point in the Company's development, Neal is a fantastic addition to Ribons already stellar SAB, bringing his vast experience in the development and understanding of novel cancer targets and therapeutics to Ribon's developing pipeline.

Dr. Rosen's major interests involve identification and study of the key molecular events and growth signaling pathways responsible for the development of human cancers, and the use of this information for the development of mechanism-based therapeutic strategies. Dr. Rosen has pioneered the concepts that feedback inhibition of physiologic signaling is an important consequence of oncogene activation that shapes the phenotype of cancer cells and that relief of this feedback in tumors treated with inhibitors of oncoprotein-activated signaling causes adaptive resistance to these drugs. Recent work from the Rosen laboratory includes the elucidation of the underlying mechanisms whereby mutated BRAF genes cause cancer and the discovery that these mutations may be divided into three different classes that determine the effective strategies for their treatment. These studies predicted several of the cellular mechanisms whereby tumors develop acquired resistance and adaptive resistance to standard therapy and the discovery and development of new drugs that will reverse this resistance. Recently, the Rosen laboratory has also focused on the development of the first direct inhibitor of RAS, a gene involved in the development of 25% of human cancers. This work, in addition to other recent studies by the Rosen laboratory on the consequences of relief of negative feedback by oncoprotein inhibitors, has led to multiple clinical trials of combination therapies at Memorial Sloan Kettering and other cancer centers in the United States and internationally that have shown promising early results. He is the incumbent of the Enid A. Haupt Chair in Medical Oncology at MSK and the recipient of the Lifetime Achievement Award from the Society for Melanoma Research.

Dr. Rosen received his undergraduate degree in chemistry from Columbia College and an M.D. and Ph.D. in Molecular Biology from the Albert Einstein College of Medicine. He completed a residency in Internal Medicine at the Brigham and Womens Hospital, and postdoctoral training and a fellowship in Medical Oncology at the National Cancer Institute. He was on the senior staff of the Medicine Branch at the NCI prior to joining the faculty of MSK.

RBN-2397 Inhibiting PARP7, a Key MonoPARP Cancer Dependency

Ribons lead program, RBN-2397, is focused on inhibiting overactive PARP7 in tumors, which has been shown to play a key role in cancer survival. Ribons research has discovered that many cancer cells rely on PARP7 for intrinsic cell survival, and that PARP7 allows cancer cells to hide from the immune system. Ribon has demonstrated that inhibition of PARP7 with RBN-2397 can potently inhibit the growth of cancer cells and restore interferon signaling, effectively releasing the brake cancer uses to hide from the immune system and suppress both innate and adaptive immune mechanisms. In several cancer models, RBN-2397 demonstrated durable tumor growth inhibition, potent antiproliferative activity and restoration of interferon signaling. Ribon plans to initially develop RBN-2397 in squamous cell carcinoma of the lung, where research has shown PARP7 to be genetically amplified. The company also plans to explore RBN-2397 for the treatment of additional cancers, including cancers of the aerodigestive tract, pancreatic cancer and ovarian cancer.

PARP7 is a member of the monoPARP family of proteins, which are key regulators of stress responses that enable cancer cells to survive and also evade immune detection, and emerging science has linked their activity with disease development. MonoPARPs are a family of 12 enzymes that are functionally and structurally distinct from the more well-known polyPARPs, such as PARP1/2. MonoPARPs function across a variety of stress responses relevant to disease development in cancer, inflammatory conditions and neurodegenerative diseases. Ribon has built an integrated technology platform to interrogate monoPARPs to develop first-in-class, small molecule therapeutics.

About Ribon Therapeutics

Ribon Therapeutics is a biotechnology company developing first-in-class therapeutics targeting novel enzyme families activated under cellular stress conditions that contribute to disease. We are exploring novel areas of biology to develop effective treatments for patients with limited therapeutic options. Leveraging a chemical biology approach and our proprietary discovery platform, we are building a pipeline of selective, small molecule inhibitors to numerous NAD+ utilizing enzymes, beginning with monoPARPs, which have applications across multiple therapeutic areas. Our lead program is RBN-2397, a first-in-class PARP7 inhibitor in development for the treatment of cancer. Ribon is located in Cambridge, Massachusetts. For more information, please visit http://www.ribontx.com.

Read more from the original source:
Ribon Therapeutics Strengthens Scientific Advisory Board with Appointment of Neal Rosen, M.D., Ph.D. - Business Wire