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Actinium Pharmaceuticals Announces Iomab-ACT Program Gene Therapy Collaboration with UC Davis in Ongoing Clinical Trial for Patients with HIV-Related…

By daniellenierenberg

- Trial will replace currently used chemotherapy conditioning with apamistamab-I-131, Actinium's targeted conditioning ARC, to selectively eliminate lymphoma cancer cells and stem cells to enable engraftment of stem cell gene therapy

- Anti-HIV stem cell gene therapy intended to simultaneously treat patients' HIV-related lymphoma and develop immune cells resistant to HIV

NEW YORK, Jan. 13, 2020 /PRNewswire/ --Actinium Pharmaceuticals, Inc. (NYSE AMERICAN: ATNM) ("Actinium"), announced today that it has entered into an agreement with the University of California, Davis (UC Davis) to utilize Actinium's Antibody Radiation-Conjugate or ARC apamistamab-I-131 for targeted conditioning and replace the chemotherapy conditioning being used in an ongoing Phase 1/2 stem cell gene therapy clinical trial. In the trial, patients with relapsed or refractory HIV-related lymphoma are being treated with autologous stem cell gene therapy. This is the first gene therapy clinical trial that will utilize ARC based conditioning. The clinical trial will be conducted at UC Davis and may be expanded to additional sites in the future.

(PRNewsfoto/Actinium Pharmaceuticals, Inc.)

Dr. Mehrdad Abedi, Professor, Hematology and Oncology at UC Davis and study lead, said, "This collaboration represents an exciting combination of revolutionary technologies that could further our ability to treat patients with HIV and other life-threatening diseases with gene therapy. Despite the advances made in the field of gene therapy, the reliance on non-targeted chemotherapy and external radiation as conditioning regimens is less than optimal and poses a problem that we hope to reduce or eliminate as part of this collaboration by replacing our conditioning regimen in this study with Actinium's ARC based targeted conditioning. Advances in HIV therapies have dramatically improved patient survival, but current therapies require life-long daily use to keep the HIV virus at bay, can have severe side effects, may be overcome by HIV resistance and do not address the needs of all patients like those in this study with HIV-related lymphomas. We envision a future where a single treatment of our stem cell gene therapy can cure patients of their lymphoma and HIV leaving the patient with a new immune system that can fight, be resistant to and prevent the mutation of HIV. Apamistamab-I-131's demonstrated antitumor effect against lymphoma and ability to condition patients in a targeted manner with a demonstrated tolerable safety profile in the bone marrow transplant setting makes it an ideal conditioning agent for this patient population. Based on these factors and extensive supporting clinical data in the Iomab-B program, we selected this ARC as the conditioning agent for the next phase of our trial as we believe antibody radiation-conjugates are more advanced and hold distinct advantages over novel but unproven conditioning technologies such as Antibody Drug Conjugates and naked antibodies that are beginning to be developed albeit at the preclinical stage."

In the current clinical trial, the anti-HIV stem cell gene therapy is produced by taking a patient's own or autologous, blood forming stem cells and genetically modifying them via gene therapy with a combination of three anti-HIV genes. The intended result is for the gene modified bone marrow stem cells to produce a new immune system and newly arising immune cells that are resistant to HIV via a single treatment. Conditioning is necessary prior to adoptive cell therapies such as gene therapy to eliminate certain cell types such as immune cells and stem cells in the bone marrow so the transplanted cells can engraft. Until now, conditioning in this trial, as is typical, used a multi-drug chemotherapy regimen administered over several days. This approach is non-targeted, associated with toxicities that impairs patients and restricts the use and efficacy of cellular therapy. Apamistamab-I-131, which requires just one therapeutic administration, will displace the non-targeted chemotherapy to condition patients in a targeted manner with the goal of reducing conditioning related toxicities and improving patient outcomes. Actinium and UC David will cross-reference their respective Investigational New Drug applications and will work collaboratively to obtain necessary regulatory and institutional approvals. In this clinical collaboration, Actinium will provide drug product, support for its administration and certain trial costs. UC Davis will be responsible for the production of the anti-HIV stem cell gene therapy and overall conduct of the study and its cost.

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Dr. Dale Ludwig, Actinium's Chief Scientific Officer, said, "We are excited to be working with Dr. Abedi on this clinical study and we appreciate his recognition of the value of our Iomab-ACT targeted conditioning program may provide in support of gene stem cell therapy. This targeted approach using our CD45 ARC, enables both anti-tumor activity and effective conditioning with the potential for reduced toxicity compared to non-targeted chemotherapy and external radiation in the bone marrow transplant setting. Supported by extensive clinical investigation in 12 trials and over 300 patients, a single therapeutic dose of apamistamab-I-131 is sufficient for conditioning and, due to its dual activity, even a patient with active disease could expect to receive therapy within two weeks, which is anticipated to lead to better outcomes compared to chemotherapy, external beam radiation, or exploratory approaches such as naked antibodies or Antibody Drug Conjugates. In addition, CD45, the target of apamistamab-I-131, is ideal for targeted conditioning, as it is not expressed outside of the haemopoietic system and, because it is a poorly internalizing receptor. An ARC approach which does not require internalization of its radionuclide warhead for target cell killing, is anticipated to be more viable and more effective than Antibody Drug Conjugate approaches which need to internalize their payloads. Given the potential of this ARC targeted conditioning technology for bone marrow transplant, we are grateful to Dr. Abedi for the opportunity to advance the Iomab-ACT program into the promising field of gene stem cell therapy."

Sandesh Seth, Actinium's Chairman and Chief Executive Officer, said, "Actinium is thrilled to be working with UC Davis and honored to now be part of this important trial. It has become evident that better conditioning regimens are needed for cell and gene therapies to reach their full potential. Our team is proud to be the first company to establish a clinical stage targeted conditioning portfolio for both cell and gene therapy. We are pleased to extend our ARC technology for targeted conditioning into these rapidly advancing fields and we are committed to establishing a strong leadership position in enabling these adoptive cell therapies fully realize their great potential for improving patients' lives."

Apamistamab-I-131's demonstrated conditioning and antitumor effect in lymphoma1

Actinium's apamistamab-I-131 ARC has been studied as a targeted conditioning agent in over 300 patients in the bone marrow transplant setting in the Iomab-B Program and is currently being studied in a pivotal Phase 3 clinical (SIERRA) trial in patients with relapsed or refractory acute myeloid leukemia. Clinical proof of concept has been established with Iomab-B for targeted conditioning in high-risk, relapsed or refractory lymphoma patients prior to an autologous stem cell transplant where a favorable safety profile with no dose limiting toxicities and minimal non-hematologic toxicities observed and promising efficacy with median overall survival not reached (range: 29 months to infinity) and 31% of patients in prolonged remission at a median of 36 months follow up (range: 25 41 months)1.

1) Cassaday et al. Phase I Study of a CD45-Targeted AntibodyRadionuclide Conjugate for High-Risk Lymphoma. AACR Clin Cancer Res Published OnlineFirst September 3, 2019

About Actinium Pharmaceuticals, Inc.

Actinium Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company developing ARCs or Antibody Radiation-Conjugates, which combine the targeting ability of antibodies with the cell killing ability of radiation. Actinium's lead application for our ARCs is targeted conditioning, which is intended to selectively deplete a patient's disease or cancer cells and certain immune cells prior to a BMT or Bone Marrow Transplant, Gene Therapy or Adoptive Cell Therapy (ACT) such as CAR-T to enable engraftment of these transplanted cells with minimal toxicities. With our ARC approach, we seek to improve patient outcomes and access to these potentially curative treatments by eliminating or reducing the non-targeted chemotherapy that is used for conditioning in standard practice currently. Our lead product candidate, apamistamab-I-131 (Iomab-B) is being studied in the ongoing pivotal Phase 3 Study of Iomab-B in Elderly Relapsed or Refractory Acute Myeloid Leukemia (SIERRA) trial for BMT conditioning. The SIERRA trial is over fifty percent enrolled and promising single-agent, feasibility and safety data has been highlighted at ASH, TCT, ASCO and SOHO annual meetings. Apatmistamamb-I-131 will also be studied as a targeted conditioning agent in a Phase 1/2 anti-HIV stem cell gene therapy with UC Davis and is expected to be studied with a CAR-T therapy in 2020. In addition, we are developing a multi-disease, multi-target pipeline of clinical-stage ARCs targeting the antigens CD45 and CD33 for targeted conditioning and as a therapeutic either in combination with other therapeutic modalities or as a single agent for patients with a broad range of hematologic malignancies including acute myeloid leukemia, myelodysplastic syndrome and multiple myeloma. Ongoing combination trials include our CD33 alpha ARC, Actimab-A, in combination with the salvage chemotherapy CLAG-M and the Bcl-2 targeted therapy venetoclax. Underpinning our clinical programs is our proprietary AWE (Antibody Warhead Enabling) technology platform. This is where our intellectual property portfolio of over 100 patents, know-how, collective research and expertise in the field are being leveraged to construct and study novel ARCs and ARC combinations to bolster our pipeline for strategic purposes. Our AWE technology platform is currently being utilized in a collaborative research partnership with Astellas Pharma, Inc.

Forward-Looking Statements for Actinium Pharmaceuticals, Inc.

This press release may contain projections or other "forward-looking statements" within the meaning of the "safe-harbor" provisions of the private securities litigation reform act of 1995 regarding future events or the future financial performance of the Company which the Company undertakes no obligation to update. These statements are based on management's current expectations and are subject to risks and uncertainties that may cause actual results to differ materially from the anticipated or estimated future results, including the risks and uncertainties associated with preliminary study results varying from final results, estimates of potential markets for drugs under development, clinical trials, actions by the FDA and other governmental agencies, regulatory clearances, responses to regulatory matters, the market demand for and acceptance of Actinium's products and services, performance of clinical research organizations and other risks detailed from time to time in Actinium's filings with the Securities and Exchange Commission (the "SEC"), including without limitation its most recent annual report on form 10-K, subsequent quarterly reports on Forms 10-Q and Forms 8-K, each as amended and supplemented from time to time.

Contacts:

Investors:Hans VitzthumLifeSci Advisors, LLCHans@LifeSciAdvisors.com(617) 535-7743

Media:Alisa Steinberg, Director, IR & Corp Commsasteinberg@actiniumpharma.com(646) 237-4087

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SOURCE Actinium Pharmaceuticals, Inc.

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Helen Obando Is The Youngest To Successfully Undergo Sickle Cell Therapy – Moms

By daniellenierenberg

Sickle cell disease is a painful condition that thousands of kids have to endure. The genetic disease impacts the blood, but it can cause organ damage and other issues, including lots of pain.

Most of the time there is no end in sight, which makes it even harder on families. But the bravery of one teen is helping scientists to develop a potential cure that could change the lives of so many.

Helen Obando recently became the youngest person to ever go through a special gene therapy using stem cells.

The usual treatment for sickle cell therapy is a bone marrow transplant form a healthy sibling, but Helen's older sister Haylee also has sickle cell, so that isn't really an option for the family.

The Obandos were excited to learn about an experimental treatment that has the potential to flip the switch on the genetics and actually cure the disease.

Scientists are hoping that the new treatment could help people with a number of genetic conditions using a technique to manipulate the DNA.

Helen had to spend four weeks in the hospital after her infusion to get strong enough to go home, and they don't know yet if the treatment has worked.

The poor girl has gone through a lot. Her pelvis was harmed before she even turned 1, and at 2, her spleen had to be removed. She's had a lot of painful episodes, and while Haylee was able to match with their younger brother Ryan for a bone marrow transplant, that wasn't an option for Helen.

In the Boston Globe, Helen's mom said that she was scared of the gene therapy option when she first heard of it. But she decided that it was worth the risk to have a chance at being healthy.

Six months since the treatment, it's so far, so good. Helen's hemoglobin levels are at a point that she has never achieved. She actually has no signs of sickle cell right now, and that is just amazing.

What a brave girl to go through a risky procedure.It's a big burden for a teenager to bear, but luckily things have worked out well so far. We hope that Helen continues to find success and health in the new year.

READ NEXT:Boy Recovers From Kidney Transplant After Father Murdered His Mother

Boy Recovers From Kidney Transplant After Father Murdered His Mother

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Bomedemstat Receives Fast Track Status for Essential Thrombocythemia – Monthly Prescribing Reference

By daniellenierenberg

Home News Drugs in the Pipeline

The Food and Drug Administration (FDA) has granted Fast Track designation to bomedemstat (IMG-7289; Imago BioSciences) for the treatment of essential thrombocythemia, a myeloproliferative disorder characterized by high platelet counts.

[Essential thrombocythemia] is a quiet bone marrow cancer that can linger for years, said Hugh Young Rienhoff, Jr. MD, CEO, Imago Biosciences. In a subset of patients, the excess of platelets leads to bleeding and clotting including strokes and infractions, each having a significant impact on these patients.

Bomedemstat is believed to inhibit lysine-specific demethylase 1 (LSD1 or KDM1A), an enzyme that plays an important role in the production and function of megakaryocytes and in self-renewal of malignant hematopoietic stem cells. The investigational agent has been shown to be effective in preclinical studies across a range of myeloid malignancy models. The Company plans to initiate a phase 2 trial to assess bomedemstat in patients with essential thrombocythemia.

With only one FDA approved therapy, one that does not increase overall survival, patients are in desperate need of new options. Based on its mechanism and safety data obtained to date, we believe bomedemstat has the promise to be that new treatment, added Rienhoff.

For more information visit imagobio.com.

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BioLineRx Receives Orphan Drug Designation for Motixafortide (BL-8040) for the Treatment of Pancreatic Cancer in Europe – Olean Times Herald

By daniellenierenberg

TEL AVIV, Israel, Jan. 14, 2020 /PRNewswire/ -- BioLineRx Ltd. (NASDAQ: BLRX) (TASE: BLRX), a clinical-stage biopharmaceutical company focused on oncology, today announced that the European Commission (EC) has granted Orphan Drug Designation to its lead oncology candidate, Motixafortide (BL-8040), for the treatment of pancreatic cancer, based on a positive opinion from the Committee for Orphan Medicinal Products (COMP) of the European Medicines Agency (EMA). Last year, Motixafortide received Orphan Drug Designation for the treatment of Pancreatic Cancer from the US Food and Drug Administration (FDA).

"The Orphan Drug status we received for Motixafortide, from both the US and European regulatory bodies, is of significant strategic importance for the development of our lead product for the treatment of pancreatic cancer, an extremely difficult to treat indication with a poor response to the currently available treatments," stated Philip Serlin, Chief Executive Officer of BioLineRx."We recently reported very encouraging initial data from the triple combination arm of our ongoing Phase 2a COMBAT/KEYNOTE-202 studyin second line metastatic pancreatic cancer patients, which served as the basis for this ODD, and we believe that this designation will maximize the potential to make this new treatment available for patients in the fastest way possible."

Motixafortide is currently being evaluated in a Phase 2a study for the treatment of pancreatic cancer in combination with KEYTRUDA and chemotherapy under a collaboration agreement with Merck & Co., Inc., Kenilworth, N.J., USA (known as MSD outside the United States and Canada).

The EMA grants orphan medicinal product designation to investigational drugs intended to treat, prevent or diagnose a life-threatening or chronically debilitating disease affecting fewer than five in 10,000 people in the EU and for which no satisfactory treatment is available or, if such treatment exists, the medicine must be of significant benefit to those affected by the condition. Orphan medicinal product designation provides regulatory and financial incentives for companies to develop and market therapies, including ten years of market exclusivity, protocol assistance, fee reductions and EU-funded research.

About Motixafortide in Cancer Immunotherapy

Motixafortide is targeting CXCR4, a chemokine receptor and a well validated therapeutic target that is over-expressed in many human cancers including PDAC. CXCR4 plays a key role in tumor growth, invasion, angiogenesis, metastasis and therapeutic resistance, and CXCR4 overexpression has been shown to be correlated with poor prognosis.

Motixafortide is a short synthetic peptide used as a platform for cancer immunotherapy with unique features allowing it to function as a best-in-class antagonist of CXCR4. It shows high-affinity, long receptor occupancy and acts as an inverse agonist.

In a number of clinical and preclinical studies, Motixafortide has been shown to affect multiple modes of action in "cold" tumors, including immune cell trafficking, tumor infiltration by immune effector T cells, and reduction in immunosuppressive cells (such as MDSCs) within the tumor niche, turning "cold" tumors, such as pancreatic cancer, into "hot" (i.e., sensitizing them to immune checkpoint inhibitors and chemotherapy).

About Pancreatic Cancer

Pancreatic cancer has a low rate of early diagnosis and a poor prognosis. Its incidence rate in the US is estimated at 3.2% of new cancer cases. Each year, about 185,000 individuals globally are diagnosed with this condition, and an estimated 55,000 individuals were diagnosed with pancreatic cancer in the US during 2018. Symptoms are usually non-specific and as a result, pancreatic cancer is often not diagnosed until it reaches an advanced stage. Surgical resection does not offer adequate treatment since only 20% of patients have resectable tumors at the time of diagnosis. The overall five-year survival rate among all pancreatic cancer patients is 7-8%, which constitutes the highest mortality rate among solid tumor malignancies. The overall median survival is less than one year from diagnosis, highlighting the need for the development of new therapeutic options.

Despite advances in chemotherapeutics and immunotherapy, increases in median and overall survival rates in pancreatic cancer have been modest. Pancreatic cancer remains an area of unmet medical need, with no new approved therapies since the approval of nab-paclitaxel in combination with gemcitabine (Abraxane) for first-line treatment in 2013 and Onivyde in combination with fluorouracil and leucovorin for second-line treatment in 2015. The limited clinical benefits demonstrated by these existing standard treatment options reinforce the need for additional approaches.

About BioLineRx

BioLineRx Ltd. (NASDAQ/TASE: BLRX) is a clinical-stage biopharmaceutical company focused on oncology. The Company's business model is to in-license novel compounds, develop them through clinical stages, and then partner with pharmaceutical companies for further clinical development and/or commercialization.

The Company'slead program, Motixafortide (BL-8040), is a cancer therapy platform currently being evaluated in a Phase 2a study for the treatment of pancreatic cancer in combination with KEYTRUDA and chemotherapy under a collaboration agreement with MSD. Motixafortide is also being evaluated in a Phase 2b study in consolidation AML and a Phase 3 study in stem cell mobilization for autologous bone-marrow transplantation. In addition, the Company has an ongoing collaboration agreement with Genentech, a member of the Roche Group, evaluating Motixafortide in combination with Genentech's atezolizumab in two Phase 1b/2 solid tumor studies.

BioLineRx is developing a second oncology program, AGI-134, an immunotherapy treatment for multiple solid tumors that is currently being undergoing in a Phase 1/2a study.

For additional information on BioLineRx, please visit the Company's website at http://www.biolinerx.com, where you can review the Company's SEC filings, press releases, announcements and events. BioLineRx industry updates are also regularly updated on Facebook,Twitter, and LinkedIn.

Various statements in this release concerning BioLineRx's future expectations constitute "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995. These statements include words such as "may," "expects," "anticipates," "believes," and "intends," and describe opinions about future events. These forward-looking statements involve known and unknown risks and uncertainties that may cause the actual results, performance or achievements of BioLineRx to be materially different from any future results, performance or achievements expressed or implied by such forward-looking statements. Some of these risks are: changes in relationships with collaborators; the impact of competitive products and technological changes; risks relating to the development of new products; and the ability to implement technological improvements. These and other factors are more fully discussed in the "Risk Factors" section of BioLineRx's most recent annual report on Form 20-F filed with the Securities and Exchange Commission on March 28, 2019. In addition, any forward-looking statements represent BioLineRx's views only as of the date of this release and should not be relied upon as representing its views as of any subsequent date. BioLineRx does not assume any obligation to update any forward-looking statements unless required by law.

Contact:Tim McCarthyLifeSci Advisors, LLC+1-212-915-2564tim@lifesciadvisors.com

or

Tsipi HaitovskyPublic Relations+972-52-598-9892tsipihai5@gmail.com

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These Are the First Living Robots: Machines Made from Frog Stem Cells – Popular Mechanics

By daniellenierenberg

What happens when you cross stem cells from a frog heart and frog skin? Not muchthat is, until you program those cells to move. In that case, you've created a xenobot, a new type of organism that's part robot, part living thing.

And we've never seen anything like it before.

Researchers from Tufts University, the University of Vermont, and Harvard University have created the first xenobots from frog embryos after designing them with computer algorithms and physically shaping them with surgical precision. The skin-heart embryos are just one millimeter in size, but can accomplish some remarkable things for what they are, like physically squirming toward targets.

"These are novel living machines," Joshua Bongard, a computer scientist and robotics expert at the University of Vermont who co-led the new research, said in a press statement. "They're neither a traditional robot nor a known species of animal. It's a new class of artifact: a living, programmable organism."

By studying these curious organisms, researchers hope to learn more about the mysterious world of cellular communication. Plus, these kinds of robo-organisms could possibly be the key to drug delivery in the body or greener environmental cleanup techniques.

"Most technologies are made from steel, concrete, chemicals, and plastics, which degrade over time and can produce harmful ecological and health side effects," the authors note in a research paper published in the scientific journal Proceedings of the National Academy of Sciences. "It would thus be useful to build technologies using self-renewing and biocompatible materials, of which the ideal candidates are living systems themselves."

Xenobots borrow their name from Xenopus laevis, the scientific name for the African clawed frog from which the researchers harvested the stem cells. To create the little organisms, which scoot around a petri dish a bit like water bearsthose tiny microorganisms that are pretty much impossible to killthe researchers scraped living stem cells from frog embryos. These were separated into single cells and left to incubate.

They differentiated the stem cells into two different kinds: heart and skin cells. The heart cells are capable of expanding and contracting, which ultimately aids the xenobot in locomotion, and the skin cells provide structure. Next, using tiny forceps and an even smaller electrode, the scientists cut the cells and joined them together under a microscope in designs that were specified by a computer algorithm.

Interestingly, the two different kinds of cells did merge together well and created xenobots that could explore their watery environment for days or weeks. When flipped like a turtle on its shell, though, they could no longer move.

Other tests showed whole groups of xenobots are capable of moving in circles and pushing small items to a central location all on their own, without intervention. Some were built with holes in the center to reduce drag and the researchers even tried using the hole as a pouch to let the xenobots carry objects. Bongard said it's a step in the right direction for computer-designed organisms that can intelligently deliver drugs in the body.

Sam Kriegman, UVM

While these xenobots are capable of some spontaneous movement, they can't accomplish any coordinated efforts without the help of computers. Really, xenobots couldn't fundamentally exist without designs created through evolutionary algorithms.

Just as natural selection dictates which members of a species live and which die offbased on certain favorable or unfavorable attributes and ultimately influencing the species' characteristicsevolutionary algorithms can help find beneficial structures for the xenobots.

A team of computer scientists created a virtual world for the xenobots and then ran evolutionary algorithms to see which potential designs for the xenobots could help them move or accomplish some other goal. The algorithm looked for xenobots that performed well at those particular tasks while in a given configuration, and then bred those microorganisms with other xenobots that were considered "fit" enough to survive this simulated natural selection.

In the video above, for example, you can see a simulated version of the xenobot, which is capable of forward movement. The final organism takes on a similar shape to this design and is capable of (slowly) getting around. The red and green squares at the bottom of the structure are active cells, in this case the heart stem cells, while the blueish squares represent the passive skin stem cells.

All of this design work was completed over the course of a few months on the Deep Green supercomputer cluster at the University of Vermont. After a few hundred runs of the evolutionary algorithm, the researchers filtered out the most promising designs. Then, biologists at Tufts University assembled the real xenobots in vitro.

Anything dealing with stem cells is bound to meet at least some flack because detractors take issue with the entire premise of using stem cells, which are harvested from developing embryos.

That's compounded with other practical ethics questions, especially relating to safety and testing. For instance, should the organisms have protections similar to animals or humans when we experiment on them? Could we, ourselves, eventually require protection from the artificially produced creatures?

"When youre creating life, you dont have a good sense of what direction its going to take," Nita Farahany, who studies the ethical ramifications of new technologies at Duke University and was not involved in the study, told Smithsonian Magazine. "Any time we try to harness life [we should] recognize its potential to go really poorly."

Michael Levin, a biophysicist and co-author of the study from Tufts University, said that fear of the unknown in this case is not reasonable:

At its heart, the study is a "direct contribution to getting a handle on what people are afraid of, which is unintended consequences," Levin said.

Source: The University of Vermont

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There is a new player in adult bone healing – Baylor College of Medicine News

By daniellenierenberg

Adult bone repair relies on the activation of bone stem cells, which still remain poorly characterized. Bone stem cells have been found both in the bone marrow and in the outer layer of tissue, called periosteum, that envelopes the bone. Of the two, periosteal stem cells are the least understood.

Having a better understanding of how adult bones heal could reveal new ways of repair fractures faster and help find novel treatments for osteoporosis. Dr. Dongsu Park and his colleagues at Baylor College of Medicine investigate adult bone healing and recently uncovered a new mechanism that has potential therapeutic applications.

Previous studies have shown that bone marrow and periosteal stem cells, although they share many characteristics, also have unique functions and specific regulatory mechanisms, said Park, who is assistant professor of molecular and human genetics and of pathology and immunology at Baylor.

It is known that these two types of bone stem cells comprise a heterogeneous population that can contribute to bone thickness, shaping and fracture repair, but scientists had not been able to distinguish between different subtypes of bone stem cells and study how their different functions are regulated.

In the current study, Park and his colleagues developed a method to identify different subpopulations of periosteal stem cells, define their contribution to bone fracture repair in live mouse models and identify specific factors that regulate their migration and proliferation under physiological conditions.

The researchers discovered specific markers for periosteal stem cells in mice. The markers identified a distinct subset of stem cells that showed to be a part of life-long adult bone regeneration.

We also found that periosteal stem cells respond to mechanical injury by engaging in bone healing, Park said. They are important for healing bone fractures in the adult mice and, interestingly, they contribute more to bone regeneration than bone marrow stem cells do.

In addition, the researchers found that periosteal stem cells also respond to inflammatory molecules called chemokines, which are usually produced during bone injury. In particular, they responded to chemokine CCL5.

Periosteal stem cells have receptors molecules on their cell surface called CCR5 that bind to CCL5, which sends a signal to the cells to migrate toward the injured bone and repair it. Deleting the CCL5 or the CCR5 gene in mouse models resulted in marked defects or delayed healing. When the researchers supplied CCL5 to CCL5-deficient mice, bone healing was accelerated.

The findings suggested potential therapeutic applications. For instance, in individuals with diabetes or osteoporosis in which bone healing is slow and may lead to other complications resulting from limited mobility, accelerating bone healing may reduce hospital stay and improve prognosis.

Our findings contribute to a better understanding of how adult bones heal. We think this is one of the first studies to show that bone stem cells are heterogeneous, and that different subtypes have unique properties regulated by specific mechanisms, Park said. We have identified markers that enable us to tell bone stem cell subtypes apart and study what each subtype contributes to bone health. Understanding how bone stem cell functions are regulated offers the possibility to develop novel therapeutic strategies to treat adult bone injuries.

Find all the details of this study in the journal journal Cell Stem Cell.

Other contributors to this work include Laura C. Ortinau, Hamilton Wang, Kevin Lei, Lorenzo Deveza, Youngjae Jeong, Yannis Hara, Ingo Grafe, Scott Rosenfeld, Dongjun Lee, Brendan Lee and David T. Scadden. The authors are affiliated with one of the following institutions: Baylor College of Medicine, Texas Childrens Hospital, Pusan National University School of Medicine and Harvard University.

This study was supported by the Bone Disease Program of Texas Award and The CarolineWiess Law Fund Award, the NIAMS of the National Institutes of Health under award numbers 1K01AR061434 and 1R01AR072018 and U54 AR068069 and the NIDDK of the NIH.

By Ana Mara Rodrguez, Ph.D.

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Stem Cell Assay Market Global Competitive Analytics and Insights 2024 – Voice of Reports

By daniellenierenberg

Regenerative medicine has emerged as new paradigm in human health. It has the potential to resolve unmet medical needs. Rapid growth in the interdisciplinary field of regenerative medicine is altering the health care domain by converting fundamental science into a variety of regenerative technologies. Stem cell is an undifferentiated mass of cell that has the ability to divide indefinite times. It can be further differentiated into specialized cells such as blood cells, skin cells, neurons, heart cells, chondrocytes, and osteocytes under specific conditions. Unspecialized nature, self-renewal capability, and dedifferentiation are the unique features of stem cells. Thus, these cells are useful in different applications in pharmaceutical research and medical fields.

Report Overview @

https://www.transparencymarketresearch.com/stem-cell-assay-market.html

Stem cell research has grown significantly since 1978, when stem cells were discovered in human cord blood. Incidence of cancer is increasing across the globe due to the rise in aging population and changing lifestyle habits. This, in turn, is boosting the demand for anticancer drugs and therapies. According to the Centers for Disease Control and Prevention, 14.1 million new cancer cases were diagnosed around the globe in 2012 and around 19.3 million new cancer cases are expected to be diagnosed each year by 2025. Rise in incidences of chronic diseases is boosting the demand for research, making stem cells a highly preferred system for drug discovery due to its self-renewal capability and unspecialized nature.

Over the last decade, the application of cell-based assays has increased at a rapid pace among research institutes and pharmaceutical industries. This was primarily ascribed to the ethical issues associated with the use of animals for clinical trials. Furthermore, rise in approvals of clinical trials for stem cells based therapy, increase in funds from government organizations, and technological advancements are some of the factors driving the stem cell assay market.

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But, human embryonic stem cells, which are derived from inner cell mass of blastocyst are currently high on the political issues ethical concerns in many countries hampering the growth of the market. Additionally, lack of required infrastructure in developing countries and high cost associated with products are some of the factors restraining the stem cell assay market. Evolution of new therapies and low regulatory frameworks in emerging regions are expected to provide opportunities for market growth during the forecast period.

The global stem cell assay market has been segmented based on product, assay type, application, end-user, and region. In terms of product, the market for stem cell assay has been divided into human embryonic stem cell kits and adult stem cell kits. The adult stem cell kits segment is further divided into induced pluripotent stem cells kits, hematopoietic stem cell kits, mesenchymal stem cell kits, umbilical cord stem cell kits, and others.

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The adult stem cell kits segment is expected to account for the prominent share of the global stem cell assay market during the forecast period, led by the rise in product innovation activities and increasing focus on drug screening by biotechnology and pharmaceutical industries. Based on assay, the global stem cell assay market has been segregated into viability or cytotoxicity assay, cell identification assay, proliferation assay, differentiation assay, apoptosis assay, isolation & purification assay, and functional assay. Among these, the viability or cytotoxicity assay segment is anticipated to constitute key share of the global stem cell assay market during the forecast period, as cytotoxicity is an unavoidable stage during research.

In terms of application, the global stem cell assay market has been segmented into drug discovery & development, regenerative medicine & therapy development, and clinical research. The regenerative medicine & therapy development segment is anticipated to expand at a rapid pace during the forecast period due to the rise in incidence of Parkinsons, Alzheimers, diabetes, and cancer diseases. This is anticipated to augment the focus on the development of new therapies and innovative drugs. Evolution of new therapies is estimated to provide new opportunities for the growth of the stem cell assay market during the forecast period.

Based in end-user, the global stem cell assay market has been segregated into government research institutes, private research institutes, and industry research. The industry research segment is projected to account for the major share of the global stem cell assay market during the forecast period. Growth in adoption of stem cell assays for drug screening process and testing is likely to drive the segment in the near future.

In terms of geography, the global stem cell assay market has been divided into North America, Europe, Asia Pacific, Latin America, and Middle East & Africa. North America is expected to dominate the global stem cell assay market during the forecast period. Governmental initiatives for stem cell based research in North America are anticipated to boost the stem cell assay market in the region. The stem cell assay market in Asia Pacific is estimated to expand at a rapid pace; it is projected to overtake Europe in the near future. Development in the clinical research field and rise in patient pool are projected to augment the adoption of stem cell assay in Asia Pacific.

Key players operating in the stem cell assay market are Thermo Fisher Scientific,Merck KGaA, Promega Corporation, STEMCELL Technologies Inc., Bio-Techne Corporation, GE Healthcare, Cellular Dynamics International Inc., Hemogenix, Bio-Rad Laboratories, Inc., and Cell Biolabs Inc.

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Experts from the Mayo clinic: Spots on the skin can indicate cancer of the blood – The Times Hub

By daniellenierenberg

Experts from the Mayo clinic warned that spots on the skin may signal a blood cancer. In connection with what experts suggest as soon as possible to go to the doctor when the symptom of.

Leukemia is a cancer primarily occurs in the bone marrow as a result of mutations in blood stem cell. The consequence is the loss of the descendants of the mutated cells ability to differentiate to Mature blood cells. The danger of the disease is that the symptoms are not specific, often among the signs unexplained weight loss, fever and chills. The experts considered it important to warn you that spots on the skin can indicate cancer of the blood that allows an early identification is a deadly health hazard. Among the other important symptoms of blood cancer: swollen lymph nodes, enlarged liver or spleen, frequent nosebleeds, excessive sweating, especially at night, bone pain, constant fatigue, recurrent infections.

With regard to treatment, the experts from Mayo clinic said: chemotherapy is the main form of treatment. Biological therapy works by using methods that help the immune system to recognize cancer cells and attack them. Among the methods of struggle with a deadly disease and radiation therapy, which destroys leukemia cells and stop their growth. Among the solutions and stem cell transplantation is bone marrow transplantation. The essence of the procedure is that the blood stream is filled with healthy blood cells, which often helps to restore normal functioning of the hematopoietic system.

Natasha Kumar is a general assignment reporter at the Times Hub. She has covered sports, entertainment and many other beats in her journalism career, and has lived in Manhattan for more than 8 years. Natasha has appeared periodically on national television shows and has been published in (among others) Hindustan Times.? Times of India

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Cosmetic Skin Care Market Competitive Insights, Trends and Demand Growth 2019 to 2026 – Food & Beverage Herald

By daniellenierenberg

This Cosmetic Skin Care market report endows with a far-reaching survey of key players in the market which is based on a range of objectives of an organization such as profiling, the product outline, the quantity of production, required raw material, and the financial health of the organization. One of the sections in the report covers the evaluation of probabilities of the new investment projects and overall research conclusions are offered. Thus, the transparent, truthful and extensive market information and data included in this global industry report will definitely help develop business and improve return on investment (ROI).

Global cosmetic skin care market is set to witness a substantial CAGR of 5.5% in the forecast period of 2019- 2026. The report contains data of the base year 2018 and historic year 2017. Increasing self-consciousness among population and rising demand for anti- aging skin care products are the factor for the market growth.

Global Cosmetic Skin Care Market By Product (Anti-Aging Cosmetic Products, Skin Whitening Cosmetic Products, Sensitive Skin Care Products, Anti-Acne Products, Dry Skin Care Products, Warts Removal Products, Infant Skin Care Products, Anti-Scars Solution Products, Mole Removal Products, Multi Utility Products), Application (Flakiness Reduction, Stem Cells Protection against UV, Rehydrate the skins surface, Minimize wrinkles, Increase the viscosity of Aqueous, Others), Gender (Men, Women), Distribution Channel (Online, Departmental Stores and Convenience Stores, Pharmacies, Supermarket, Others), Geography (North America, Europe, Asia-Pacific, South America, Middle East and Africa) Industry Trends and Forecast to 2026 ;

Complete report on Global Cosmetic Skin Care Market Research Report 2019-2026 spread across 350 Pages, profiling Top companies and supports with tables and figures

Market Definition: Global Cosmetic Skin Care Market

Cosmetic skin care is a variety of products which are used to improve the skins appearance and alleviate skin conditions. It consists different products such as anti- aging cosmetic products, sensitive skin care products, anti- scar solution products, warts removal products, infant skin care products and other. They contain various ingredients which are beneficial for the skin such as phytochemicals, vitamins, essential oils, and other. Their main function is to make the skin healthy and repair the skin damages.

Key Questions Answered in Global Cosmetic Skin Care Market Report:-Our Report offers:-

Top Key Players:

Market Drivers:

Market Restraints:

Key Developments in the Market:

Customize report of Global Cosmetic Skin Care Market as per customers requirement also available.Market Segmentations:Global Cosmetic Skin Care Market is segmented on the basis of

Market Segmentations in Details:By Product

By Application

By Gender

By Distribution Channel

By GeographyNorth America

Europe

Asia-Pacific

South America

Middle East & Africa

Competitive Analysis: Global Cosmetic Skin Care Market

Global cosmetic skin care 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 cosmetic skin care market for Global, Europe, North America, Asia-Pacific, South America and Middle East & Africa.

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Sleights family’s appeal for blood stem cell donor in Whitby – The Scarborough News

By daniellenierenberg

Pete McCleave pictured with his children

Sleights residents, June and Mike McCleave's son Peter has Myeloma, a type of bone marrow cancer for which there is currently no cure.

Peter, 42, was diagnosed with the disease three years ago, and time is running short for the man who two years ago was given just seven years to live.

The family is now in a race against time to find a matching stem cell donor, who can provide the transfusion that will extend Peter's life, hopefully long enough for a cure to be found.

Mum, June, said: "We go to myeloma conferences which give details of all the updated work and drugs that are available. They are very hopeful of a cure and are working on one which involves gene therapy, meaning that good cells will attack the cancer. They reckon that in ten years there will be a cure for this."

Peter has been determined to fight the disease. He set up a campaign called 10,000 donors to encourage as many people as possible to register with DKMS, the charity dedicated to defeating blood cancer. To date 33,402 donors have registered because of this campaign and 12 donor matches have been confirmed, Pete is still waiting.

June and Mike have organised an event at Eskdale School for people to go along and take a cheek swab test to see if they are a compatible match for Peter, or others who have the disease.

The event takes place of Tuesday, January 14 from 4.00pm to 7,00pm.

Taking the test is simple and pain free, three cotton swabs (like cotton buds) collect saliva from inside the mouth and are sent for testing. It's a process which is over in seconds, with one swab collecting saliva from the left cheek, one from the right cheek and one from around the mouth.

A DKMS representative will be at the session and will take the swabs to the laboratory for analysis, you will then receive a card a few weeks later confirming you are registered as a potential donor.

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Stem Cell and Regenerative Medicine Action Awards to be Presented at World Stem Cell Summit on January 23 at the Hyatt Regency Miami – Yahoo Finance

By daniellenierenberg

2020 Honorees include Cystic Fibrosis Foundation, Emily Whitehead Foundation, Gift of Life Marrow Registry and Ret. Major General Bernard Burn Loeffke (US Military)

Miami, FL, Jan. 09, 2020 (GLOBE NEWSWIRE) -- The formal ceremony of the 2020 Stem Cell and Regenerative Medicine Action Awards will take place at a gala reception and dinner on January 23, during the 15th annual World Stem Cell Summit (WSCS) at the Hyatt Regency in Miami. Since 2005, the nonprofit Regenerative Medicine Foundation (RMF) (formerly Genetics Policy Institute) has recognized the stem cell and regenerative medicine community's leading innovators, leaders, and champions through its annual awards reception.

Bernard Siegel, Executive Director of Regenerative Medicine Foundation and founder of the World Stem Cell Summit, said, The 2020 Action Awards will recognize three important organizations that are positively impacting the emerging field of regenerative medicine. We will also honor a retired Major General, who has capped off his military and diplomatic career by promoting the cause of world peace through medicine. All of these distinguished honorees will be recognized for their devotion to improving health and developing cures through advocacy, innovation, leadership and inspiration. In addition, the wounded warrior veterans community of South Florida will also receive special recognition at the event.

Meet the 2020 Stem Cell & Regenerative Medicine Action Award Honorees:

Innovation Award: With the motto, We will not rest until we find a cure, the Cystic Fibrosis Foundation is geared towards the successful development and delivery of treatments, therapies and a cure for every person with cystic fibrosis. CF Foundation has added decades to the lives of people with the disease as a direct result of advances in treatment and care made possible through its innovative business model- venture philanthropy. The Foundation recently unveiled its Path to a Cure research agenda aimed at addressing the root genetic cause of the disease and is currently funding industry programs aimed at gene delivery with the goal of progressing into clinical studies in 2021.

Inspiration Award: Emily Whitehead Foundation is a nonprofit organization committed to raising funds to invest in the most promising pediatric cancer research. Tom and Kari Whitehead founded EWF in honor of their daughter Emily, the first child in the world to receive CAR T-cell therapy, training her own cells to fight cancer. Her inspiring story focused public attention on thepotential for cancer immunotherapy to transform cancer treatment,as well as the need to support lifesaving cancer immunotherapy research. The foundation provides support to pediatric cancer patients and promotes awareness of the disease through education and sharing other inspiring stories.

Advocacy Award: Gift of Life Marrow Registry was established in 1991 by Jay Feinberg and his family after Jay received a life-saving bone marrow transplant. Gift of Life is dedicated to saving lives and facilitating bone marrow and blood stem cell transplants for patients with leukemia, lymphoma, sickle cell and other diseases. In 2019, Gift of Life opened the worlds first apheresis center fully integrated within a registry, the Dr. Miriam and Sheldon G. Adelson Gift of Life-Be The Match Stem Cell Collection Center. With the collection center and rapidly expanding donor database, Gift of Life will launch a biobank to advance cellular therapies using allogeneically sourced cells in 2020.

Leadership Award: Ret. Major General Bernard Burn Loeffke, PhD (US Military) is a highly decorated Special Forces officer, diplomat and medical officer.He survived two helicopter crashes and was wounded in combat. After the Vietnam War, he served as the Army Attach at theU.S. Embassy in Moscow, first Defense Attach at the U.S Embassy in Beijing, a staff officer in theWhite House, and Director of the Commission onWhite House Fellows. His last command was Commanding General of Army South. After 35 years in the military, he became a medical officer traveling the world on relief missions to third and fourth world countries. Presently, at age 85, he champions the hydrocephalus and wounded warrior communities. He continues to serve as an inspiration and supporter of building peaceful international relations through medical partnerships and played a pivotal role as a keynote speaker at the inaugural 2019 World Stem Cell Summit CHINA.He is called the Peace General in Latin America. In China, he is simply known as The General, our Friend.

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To learn more about past honorees and details for sponsoring or attending the upcoming 2020 Stem Cell and Regenerative Medicine Action Awards dinner, please visit, https://www.worldstemcellsummit.com/stem-cell-action-awards/

About the World Stem Cell Summit (WSCS)

Produced by the non-profit Regenerative Medicine Foundation (RMF), and in its 15th year, the World Stem Cell Summit will take place January 21-24, 2020, in Miami, Florida in partnership with Phacilitate Leaders World, as part of Advanced Therapies Week. The Summit is the most inclusive and expansive interdisciplinary, networking, and partnering meeting in the stem cell science and regenerative medicine field. With the overarching purpose of fostering translation of biomedical research, funding, and investments targeting cures, the Summit and co-located conferences serve a diverse ecosystem of stakeholders. For more information about the upcoming World Stem Cell Summit in Miami, please visit: http://www.worldstemcellsummit.com.

About the Regenerative Medicine Foundation (RMF)

The nonprofit Regenerative Medicine Foundation fosters strategic collaborations to accelerate the development of regenerative medicine to improve health and deliver cures. RMF unites the worlds leading researchers, medical centers, universities, labs, businesses, funders, policymakers, experts in law, regulation and ethics, medical philanthropies, and patient organizations. We maintain a trusted network of leaders and pursue our mission by producing our flagship World Stem Cell Summit series of conferences and public days, honoring leaders through the Stem Cell and Regenerative Medicine Action Awards, supporting our official journal partner STEM CELLS Translational Medicine (SCTM), promoting solution-focused policy initiatives both nationally and internationally and creating STEM/STEAM educational projects. For more information about RMF, please visit: http://www.regmedfoundation.org.

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Joseph DawsonRegenerative Medicine Foundation561-906-4755joseph@regmedfoundation.org

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Roshen will issue UAH 50 mln for development of National Cancer Institute – Interfax Ukraine

By daniellenierenberg

Roshen Confectionery Corporation will allocate UAH 50 million for the overhaul of the oncohematology department and the creation of an autologous bone marrow transplantation department at the National Cancer Institute.

According to the company's press release, the project will last almost two years.

"In the building where the oncohematology and chemotherapy department is located, the roof has been leaking for many years, water leaked from the sewer under the foundation as a result, almost all the walls of the building have a fungus that is simply deadly for people with this disease. In early autumn, Roshen began the overhaul of part of the premises of the second building of the National Cancer Institute. We plan to complete the work in August 2020," Iryna Ponomarenko, the director for social projects development at Roshen Confectionery Corporation, said.

In 2018, the corporation repaired and equipped a room intended for apheresis (collection) of stem cells (for bone marrow transplantation) and donor platelets for a total of UAH 2.9 million.

In total, in 2017-2018 Roshen invested UAH 357 million in social projects.

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What is Cord Blood Stem Cells Market and What Factors will drive the Industry including Leading Players Cord Blood America Inc, Cryo-Cell…

By daniellenierenberg

The Cord Blood Stem Cells Market is predicted to worth +6500 Million USD with a CAGR of +30% over the forecast period 2020 to 2025.

Three sources of stem cells are bone marrow, peripheral blood, and cord blood. The blood in the umbilical cord is called cord blood and is collected at the time of delivery. Cord blood is an abundant source of Red Blood Cells (RBCs), white blood cells (WBCs), platelets and hematopoietic stem cells, and is extracted and stored in a private blood bank for the purpose of treating the disease in the future as needed.

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The global Cord Blood Stem Cells Market analytical report has recently published by Report Consultant to its massive repository. The research report has been summarized with informative and technical details of the dynamics of the market. It has been compiled by using some significant research methodologies such as primary and secondary research techniques. The report also elaborates on the factors which are fueling or hampering the growth of the market. It gives more focus on recent trends and technologies which are boosting the performance of the companies.

Cord Blood Stem Cells Market Key Players:

Cord Blood America Inc, Cryo-Cell International Inc, Cryo-Save AG, Cord Blood Registry Systems Inc, Viacord Inc, China Cord Blood Corporation, Cordlife Group Ltd, Vita 34 AG, Lifecell International Pvt. Ltd, Stemcyte Inc

The Cord Blood Stem Cells Market is segmented by means of storage service, application, and region.

Storage service: Public cord blood bank and Private cord blood bank

Cord blood stem cell market segmentation by application: Blood disease, Cancer, Acute leukemia, Krabbe diseases, and other diseases

Regions: North America (USA, Canada, Mexico), Europe (Germany, France, UK, Italy, Russia), Asia Pacific (China, India, Japan, South Korea, Australia, Indonesia, Malaysia), Middle East and Africa (Bahrain, Egypt, Jordan, Kuwait, Morocco, Oman, Qatar, Saudi Arabia, Syria)

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The report Describes the Cord Blood Stem Cells Market basics like definitions, classifications, applications and industry chain overview, industry policies and plans, product specifications, manufacturing processes, cost structures and so on. Then it analyzed the worlds main region market conditions, including the product price, profit, capacity, production, capacity utilization, supply, demand and industry growth rate, etc. In the end, the report introduced new project SWOT analysis, investment feasibility analysis, and investment return analysis.

Global research Cord Blood Stem Cells Market report highlights:

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What is Cord Blood Stem Cells Market and What Factors will drive the Industry including Leading Players Cord Blood America Inc, Cryo-Cell...

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The potential use of CRISPR to treat disease is gaining momentum – BioNews

By daniellenierenberg

13 January 2020

Promising results from clinical trials give hope for using CRISPR/Cas9 genome editing to treat various heritable diseases and cancer in humans.

It has been seven years since the discovery that the CRISPR/Cas9 defence system, used by microbes to destroy viruses, could be re-engineered to edit the human genome. Since then researchers have carried out an array of experiments to explore potential applications.

Biophysist Dr He Jiankui sparked global controversy concerning the ethics of genome editing when he used CRISPR to genetically modify embryos, resulting in the birth of the first genome-edited babies (see BioNews 977).

Yet researchers worldwide have at the same time been investigating the use of CRISPR for non-heritable changes, modifying the genes in non-embryonic cells to treat a wide range of diseases.

'There's been a lot of appropriate caution in applying this to treating people, but I think we're starting to see some of the results of that work,' said Dr Edward Stadtmauer, a haematologist at the University of Pennsylvania, Philadelphia.

Over a dozen new clinical trials testing CRISPRtherapy on diseases such as cancer, HIV and sickle cell anaemia were listed on the clinicaltrials.gov database last year. One trial in its early stages used CRISPR to treat sickle cell anaemia and beta-thalassaemia, both genetic blood disorders that result in the production of an abnormal form of the oxygen-carrying protein, haemoglobin.

Two patients with these disorders were treated by CRISPR Therapeutics in Cambridge, Massachusetts, and Vertex Pharmaceuticals in Boston, Massachusetts, using CRISPR to inactivate a gene that switches off the production of an alternative form of haemoglobin. Preliminary results of the study suggest that this therapy improved some of the symptoms but the participants will need to be followed for a longer period to be sure.

Results from two other trials, one in which genome-edited blood cells were transplanted into a man to treat HIV infection, and the other in which they were transplanted into three people to treat some forms of cancer, were less successful. In both cases, the transplanted cells flourished in the bone marrow of recipients, without any serious safety concerns, but did not produce a clear medical benefit. The study has been placed on hold while researchers explore ways to boost that percentage, says Hongkui Deng, a stem-cell researcher at Peking University, Beijing, China and a lead author of the work.

Other researchers are trying to move beyond editing cells in vitro. In July 2019 a clinical trial was launched to treat Leber congenital amaurosis 10 (LCA10), a rare genetic disease that causes blindness. The trial, launched by two pharmaceutical companies, Editas Medicine in Cambridge, Massachusetts, and Allergan in Dublin, Ireland, will be the first trial that uses CRISPR to edit cells inside of the body. The researchers are testing AGN-151587 (EDIT-101), which is a novel CRISPR treatment delivered via adeno-associated virus (AAV) directly to the eye's light-sensing photoreceptor cells to remove the mutation that causes LCA10.

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Imago Receives Fast Track Designation from U.S. FDA for Bomedemstat for Treatment of Essential Thrombocythemia – Yahoo Finance

By daniellenierenberg

Imago BioSciences, Inc., a clinical-stage biotechnology company developing innovative treatments for myeloid diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for the development of bomedemstat (IMG-7289) for the treatment of essential thrombocythemia (ET), a bone marrow disease associated with high platelet counts and potentially catastrophic vascular complications. Bomedemstat inhibits the enzyme LSD1 (lysine-specific demethylase 1), thus preventing excess platelet and neutrophil production.

"The Fast Track designation by the FDA recognizes the need for novel therapeutics for myeloid diseases and mirrors our own urgency in addressing these devastating conditions," said Hugh Young Rienhoff, Jr. M.D., CEO, Imago Biosciences. "ET is a quiet bone marrow cancer than can linger for years. In a subset of patients, the excess of platelets leads to bleeding and clotting including strokes and infractions, each having a significant impact on these patients. With only one FDA approved therapy, one that does not increase overall survival, patients are in desperate need of new options. Based on its mechanism and safety data obtained to date, we believe bomedemstat has the promise to be that new treatment."

The FDA grants Fast Track designation to facilitate development and expedite the review of therapies with the potential to treat a serious condition where there is an unmet medical need. A therapeutic that receives Fast Track designation can benefit from early and frequent communication with the agency, in addition to a rolling submission of the marketing application, with the objective of getting important new therapies to patients more quickly.

About Bomedemstat (IMG-7289)

Bomedemstat is a small molecule discovered by Imago BioSciences that inhibits lysine-specific demethylase 1 (LSD1 or KDM1A), an enzyme essential for production and normal function of megakaryocytes and for self-renewal of malignant hematopoietic stem or progenitor cells. Megakaryocytes are the primary producer of platelets and cytokines that drive essential thrombocythemia pathogenesis.

In non-clinical studies, bomedemstat demonstrated robust in vivo efficacy as a single agent, and in combination with other therapeutics across a range of myeloid malignancy models including the myeloproliferative neoplasms encompassing myelofibrosis, essential thrombocythemia and polycythemia vera.

The FDA has also granted Fast Track designation to bomedemstat for the treatment of myelofibrosis, which is currently being studied in an international Phase 2b study. In this study IMG-7289 was effective in reducing spleen volumes and substantially improved symptom scores in a majority of evaluable patients. For more information visit http://www.clinicaltrials.gov (NCT03136185). Additional clinical studies in hematologic disorders will begin in 2020.

About Imago BioSciences

Imago BioSciences is a clinical-stage, venture-backed pharmaceutical company whose investors include a fund managed by Blackstone Life Sciences, Frazier Healthcare Partners, Omega Funds, Amgen Ventures, MRL Ventures Fund, HighLight Capital, Pharmaron, Greenspring Associates and Xeraya Capital, as well as other corporate and venture investors.

View source version on businesswire.com: https://www.businesswire.com/news/home/20200113005164/en/

Contacts

Ian StoneCanale Communicationsian@canalecomm.com (619) 849-5388

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Stem Cell Assay Market is estimated to witness the highest growth during the forecast period 2017 2025 – Pro News Time

By daniellenierenberg

Stem Cell Assay Market: Snapshot

Stem cell assay refers to the procedure of measuring the potency of antineoplastic drugs, on the basis of their capability of retarding the growth of human tumor cells. The assay consists of qualitative or quantitative analysis or testing of affected tissues and tumors, wherein their toxicity, impurity, and other aspects are studied.

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With the growing number of successful stem cell therapy treatment cases, the global market for stem cell assays will gain substantial momentum. A number of research and development projects are lending a hand to the growth of the market. For instance, the University of Washingtons Institute for Stem Cell and Regenerative Medicine (ISCRM) has attempted to manipulate stem cells to heal eye, kidney, and heart injuries. A number of diseases such as Alzheimers, spinal cord injury, Parkinsons, diabetes, stroke, retinal disease, cancer, rheumatoid arthritis, and neurological diseases can be successfully treated via stem cell therapy. Therefore, stem cell assays will exhibit growing demand.

Another key development in the stem cell assay market is the development of innovative stem cell therapies. In April 2017, for instance, the first participant in an innovative clinical trial at the University of Wisconsin School of Medicine and Public Health was successfully treated with stem cell therapy. CardiAMP, the investigational therapy, has been designed to direct a large dose of the patients own bone-marrow cells to the point of cardiac injury, stimulating the natural healing response of the body.

Newer areas of application in medicine are being explored constantly. Consequently, stem cell assays are likely to play a key role in the formulation of treatments of a number of diseases.

Global Stem Cell Assay Market: Overview

The increasing investment in research and development of novel therapeutics owing to the rising incidence of chronic diseases has led to immense growth in the global stem cell assay market. In the next couple of years, the market is expected to spawn into a multi-billion dollar industry as healthcare sector and governments around the world increase their research spending.

The report analyzes the prevalent opportunities for the markets growth and those that companies should capitalize in the near future to strengthen their position in the market. It presents insights into the growth drivers and lists down the major restraints. Additionally, the report gauges the effect of Porters five forces on the overall stem cell assay market.

Global Stem Cell Assay Market: Key Market Segments

For the purpose of the study, the report segments the global stem cell assay market based on various parameters. For instance, in terms of assay type, the market can be segmented into isolation and purification, viability, cell identification, differentiation, proliferation, apoptosis, and function. By kit, the market can be bifurcated into human embryonic stem cell kits and adult stem cell kits. Based on instruments, flow cytometer, cell imaging systems, automated cell counter, and micro electrode arrays could be the key market segments.

In terms of application, the market can be segmented into drug discovery and development, clinical research, and regenerative medicine and therapy. The growth witnessed across the aforementioned application segments will be influenced by the increasing incidence of chronic ailments which will translate into the rising demand for regenerative medicines. Finally, based on end users, research institutes and industry research constitute the key market segments.

The report includes a detailed assessment of the various factors influencing the markets expansion across its key segments. The ones holding the most lucrative prospects are analyzed, and the factors restraining its trajectory across key segments are also discussed at length.

Global Stem Cell Assay Market: Regional Analysis

Regionally, the market is expected to witness heightened demand in the developed countries across Europe and North America. The increasing incidence of chronic ailments and the subsequently expanding patient population are the chief drivers of the stem cell assay market in North America. Besides this, the market is also expected to witness lucrative opportunities in Asia Pacific and Rest of the World.

Global Stem Cell Assay Market: Vendor Landscape

A major inclusion in the report is the detailed assessment of the markets vendor landscape. For the purpose of the study the report therefore profiles some of the leading players having influence on the overall market dynamics. It also conducts SWOT analysis to study the strengths and weaknesses of the companies profiled and identify threats and opportunities that these enterprises are forecast to witness over the course of the reports forecast period.

Some of the most prominent enterprises operating in the global stem cell assay market are Bio-Rad Laboratories, Inc (U.S.), Thermo Fisher Scientific Inc. (U.S.), GE Healthcare (U.K.), Hemogenix Inc. (U.S.), Promega Corporation (U.S.), Bio-Techne Corporation (U.S.), Merck KGaA (Germany), STEMCELL Technologies Inc. (CA), Cell Biolabs, Inc. (U.S.), and Cellular Dynamics International, Inc. (U.S.).

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About TMR Research:

TMR Research is a premier provider of customized market research and consulting services to business entities keen on succeeding in todays supercharged economic climate. Armed with an experienced, dedicated, and dynamic team of analysts, we are redefining the way our clients conduct business by providing them with authoritative and trusted research studies in tune with the latest methodologies and market trends.

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Stem Cell Assay Market is estimated to witness the highest growth during the forecast period 2017 2025 - Pro News Time

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Canine Stem Cell Therapy Market Will Make a Huge Impact in Near Future – Expert Recorder

By daniellenierenberg

A synopsis of the global canine stem cell therapy market with reference to the global healthcare pharmaceutical industry

Despite the economic and political uncertainty in the recent past, the global healthcare industry has been receiving positive nudges from reformative and technological disruptions in medical devices, pharmaceuticals and biotech, in-vitro diagnostics, and medical imaging. Key markets across the world are facing a massive rise in demand for critical care services that are pushing global healthcare spending levels to unimaginable limits.

A rapidly multiplying geriatric population; increasing prevalence of chronic ailments such as cancer and cardiac disease; growing awareness among patients; and heavy investments in clinical innovation are just some of the factors that are impacting the performance of the global healthcare industry. Proactive measures such as healthcare cost containment, primary care delivery, innovation in medical procedures (3-D printing, blockchain, and robotic surgery to name a few), safe and effective drug delivery, and well-defined healthcare regulatory compliance models are targeted at placing the sector on a high growth trajectory across key regional markets.

Parent Indicators Healthcare Current expenditure on health, % of gross domestic product Current expenditure on health, per capita, US$ purchasing power parities (current prices, current PPPs) Annual growth rate of current expenditure on health, per capita, in real terms Out-of-pocket expenditure, % of current expenditure on health Out-of-pocket expenditure, per capita, US$ purchasing power parity (current prices, current PPPs) Physicians, Density per 1000 population (head counts) Nurses, Density per 1000 population (head counts) Total hospital beds, per 1000 population Curative (acute) care beds, per 1000 population Medical technology, Magnetic Resonance Imaging units, total, per million population Medical technology, Computed Tomography scanners, total, per million population

Research Methodology

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XploreMR utilizes a triangulation methodology that is primarily based on experimental techniques such as patient-level data, to obtain precise market estimations and insights on Molecule and Drug Classes, API Formulations and preferred modes of administration. Bottom-up approach is always used to obtain insightful data for the specific country/regions. The country specific data is again analysed to derive data at a global level. This methodology ensures high quality and accuracy of information.

Secondary research is used at the initial phase to identify the age specific disease epidemiology, diagnosis rate and treatment pattern, as per disease indications. Each piece of information is eventually analysed during the entire research project which builds a strong base for the primary research information.

Primary research participants include demand-side users such as key opinion leaders, physicians, surgeons, nursing managers, clinical specialists who provide valuable insights on trends and clinical application of the drugs, key treatment patterns, adoption rate, and compliance rate.

Quantitative and qualitative assessment of basic factors driving demand, economic factors/cycles and growth rates and strategies utilized by key players in the market is analysed in detail while forecasting, in order to project Year-on-Year growth rates. These Y-o-Y growth projections are checked and aligned as per industry/product lifecycle and further utilized to develop market numbers at a holistic level.

On the other hand, we also analyse various companies annual reports, investor presentations, SEC filings, 10k reports and press release operating in this market segment to fetch substantial information about the market size, trends, opportunity, drivers, restraints and to analyse key players and their market shares. Key companies are segmented at Tier level based on their revenues, product portfolio and presence.

Please note that these are the partial steps that are being followed while developing the market size. Besides this, forecasting will be done based on our internal proprietary model which also uses different macro-economic factors such as per capita healthcare expenditure, disposable income, industry based demand driving factors impacting the market and its forecast trends apart from disease related factors.

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Standard Report Structure Executive Summary Market Definition Macro-economic analysis Parent Market Analysis Market Overview Forecast Factors Segmental Analysis and Forecast Regional Analysis Competition Analysis

Target Audience Production Companies Suppliers Channel Partners Marketing Authorities Subject Matter Experts Research Institutions Financial Institutions Market Consultants Government Authorities

Market Taxonomy

The global canine stem cell therapy market has been segmented into:

Product Type: Allogeneic Stem Cells Autologous Stem cells

Application: Arthritis Dysplasia Tendonitis Lameness Others

End User: Veterinary Hospitals Veterinary Clinics Veterinary Research Institutes

Region: North America Latin America Europe Asia Pacific Japan Middle East & Africa

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About Us

XploreMR is one of the worlds leading resellers of high-quality market research reports. We feature in-depth reports from some of the worlds most reputed market research companies and international organizations. We serve across a broad spectrum from Fortune 500 to small and medium businesses. Our clients trust us for our unwavering focus onquality and affordability. We believe high price should not be a bottleneck for organizations looking to gain access to quality information.

Contact us:XploreMR111 North Market Street, Suite 300,San Jose, CA 95113, United StatesPh.No: +16692840108E-mail id- [emailprotected]

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Canine Stem Cell Therapy Market Will Make a Huge Impact in Near Future - Expert Recorder

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Ask the Expert: What are the most common types of brain tumors in children? – The Daily Progress

By daniellenierenberg

What are the most common types of brain tumors in children, and what treatment options are available?

Brain tumors are the most common solid tumors affecting children, with approximately 4,500 new cases each year in the U.S.

As brain tumors expand or block the normal pathways in the brain, the pressures inside the skull expand. As a result, symptoms of brain tumors can include headaches, seizures, lethargy, nausea and vomiting. A child experiencing progressively worsening symptoms like these should be evaluated by a pediatrician or in the emergency room. The doctors evaluation may include a scan of the brain. If the scan shows a tumor, the next step is a consultation with a neurosurgeon.

The majority of pediatric brain tumors occur in the posterior fossa (located near the bottom of the skull and the brain stem). The most common tumors include medulloblastoma, pilocytic astrocytoma, and ependymoma. Other less common tumors can occur in the cerebral hemispheres (the two main portions of the brain) and include astrocytomas, gangliogliomas, craniopharyngiomas, and germ cell tumors.

Surgery is usually the first step in treatment when a brain tumor is discovered. The goals of surgery are to determine whether the tumor is cancerous and remove all or as much of the tumor as safely as possible. At UVa Childrens Hospital, the latest technologies are utilized to help perform surgery, including intraoperative MRI, navigation, ultrasound and minimally invasive endoscopic surgery. Based on the types of cells found in the brain tumor, additional treatments may be needed. These therapies may include chemotherapy, radiation therapy, proton therapy, stem cell rescue and bone marrow transplantation and/or supportive care for rehabilitation.

More recent treatment options have focused on precision medicine and targeted drug therapy. Targeted drug treatments can cause brain tumor cells to die by blocking abnormalities present within these cells. These drugs are changing how brain tumors are treated while improving outcomes. Current research is focused on understanding the molecular basis of tumor formation and discovery of new targets for treatment.

At UVa, we are committed to providing the best neurosurgical care for children through our multidisciplinary brain tumor team, consisting of neurosurgery, neurology, pediatric oncology and radiation oncology.

For more information, visit childrens.uvahealth.com/services/pediatric-neurosurgery.

Dr. Hasan R. Syed and Dr. John Jane Jr. are pediatric neurosurgeons at UVa Childrens Hospital.

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"I tried four natural face masks in one week. Here’s exactly what they did for my skin." – Mamamia

By daniellenierenberg

Also, I think we're all trying to be kinder to the environment with our lifestyle choices, and unlike most sheet masks on the market, it's made with100 per cent natural cotton fibres that will biodegrade six months after use. The packaging itself is recyclable too.

If you've ever watched one of those videos of a baby smiling with glee after they try chocolate for the first time, that was me with chemical exfoliation. Unlike scrubs which use psychical exfoliation to buff away at dead skin, chemical exfoliants like AHAs, glycolic acid and lactic acid dissolve dead skin and sebum which can cause breakouts and dull skin.

And when it comes to products which do just that, this cult-favouritemask is one of my personal picks.

Not only does it kind of smell, and look, like theinside of a pumpkin pie, the wonderful combo of glycolic acid, fruit stem cells, Vitamin C, Manuka honey andantioxidant-rich pumpkin puree works to give you the deliciously radiant skin you crave.

In terms of tingliness, there was less sensation than the CannaCell Glow Mask but the results were equally as impressive. My pores appeared tighter, and the texture of my skin felt smoother, brighter and just... better.

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"I tried four natural face masks in one week. Here's exactly what they did for my skin." - Mamamia

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At 16, Shes a Pioneer in the Fight to Cure Sickle Cell Disease – The New York Times

By daniellenierenberg

In the months after the gene therapy infusion at Boston Childrens, her symptoms disappeared. But doctors had given her blood transfusions while she regrew her own red blood cells, so it was not clear if the absence of symptoms was because of the gene therapy or the transfusions.

As she recovered, Helen returned to her passion: dancing. One day, she came back from her school dance group and told her mother, My legs hurt. It feels funny. Ms. Cintron smiled. Thats soreness, she explained. Helen laughed. She had only known pain from sickle cell.

Helen was scheduled for her six-month checkup on Dec. 16. By then, all the transfused cells were gone, leaving only blood made by stem cells in her own marrow. The doctors would finally tell her whether the therapy was working.

The day before, she and her parents visited the New England Aquarium in Boston. She was able to stay outside on a cold, blustery day, watching one seal bully the others, barking and fighting. When Helen mentioned that her hands were cold, Ms. Cintrons stomach clenched in fear. But it was just a normal thing to feel on a winter day.

The next morning, Dr. Esrick delivered the news. Helens total hemoglobin level was so high it was nearly normal a level she had never before achieved even with blood transfusions. She had no signs of sickle cell disease.

Now you are like me, her father told her. I jump in the pool, I run. Now you can do it, too!

Her family, accustomed to constant vigilance, is only now getting used to normal life.

On Dec. 23, Helen and her mother flew to the familys new home in Arizona.

Helen recently described her transformed outlook on Facebook.

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At 16, Shes a Pioneer in the Fight to Cure Sickle Cell Disease - The New York Times

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