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Stem Cell Alopecia Treatment Market Growth Trends, Key Players, Competitive Strategies and Forecasts to 2026 – Jewish Life News

By adminJune 13, 2020Stem Cell Treatment

Stem Cell Alopecia Treatment Market Overview

The Stem Cell Alopecia Treatment market report presents a detailed evaluation of the market. The report focuses on providing a holistic overview with a forecast period of the report extending from 2018 to 2026. The Stem Cell Alopecia Treatment market report includes analysis in terms of both quantitative and qualitative data, taking into factors such as Product pricing, Product penetration, Country GDP, movement of parent market & child markets, End application industries, etc. The report is defined by bifurcating various parts of the market into segments which provide an understanding of different aspects of the market.

The overall report is divided into the following primary sections: segments, market outlook, competitive landscape and company profiles. The segments cover various aspects of the market, from the trends that are affecting the market to major market players, in turn providing a well-rounded assessment of the market. In terms of the market outlook section, the report provides a study of the major market dynamics that are playing a substantial role in the market. The market outlook section is further categorized into sections; drivers, restraints, opportunities and challenges. The drivers and restraints cover the internal factors of the market whereas opportunities and challenges are the external factors that are affecting the market. The market outlook section also comprises Porters Five Forces analysis (which explains buyers bargaining power, suppliers bargaining power, threat of new entrants, threat of substitutes, and degree of competition in the Stem Cell Alopecia Treatment) in addition to the market dynamics.

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Leading Stem Cell Alopecia Treatment manufacturers/companies operating at both regional and global levels:

Stem Cell Alopecia Treatment Market Scope Of The Report

This report offers past, present as well as future analysis and estimates for the Stem Cell Alopecia Treatment market. The market estimates that are provided in the report are calculated through an exhaustive research methodology. The research methodology that is adopted involves multiple channels of research, chiefly primary interviews, secondary research and subject matter expert advice. The market estimates are calculated on the basis of the degree of impact of the current market dynamics along with various economic, social and political factors on the Stem Cell Alopecia Treatment market. Both positive as well as negative changes to the market are taken into consideration for the market estimates.

Stem Cell Alopecia Treatment Market Competitive Landscape & Company Profiles

The competitive landscape and company profile chapters of the market report are dedicated to the major players in the Stem Cell Alopecia Treatment market. An evaluation of these market players through their product benchmarking, key developments and financial statements sheds a light into the overall market evaluation. The company profile section also includes a SWOT analysis (top three companies) of these players. In addition, the companies that are provided in this section can be customized according to the clients requirements.

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Stem Cell Alopecia Treatment Market Research Methodology

The research methodology adopted for the analysis of the market involves the consolidation of various research considerations such as subject matter expert advice, primary and secondary research. Primary research involves the extraction of information through various aspects such as numerous telephonic interviews, industry experts, questionnaires and in some cases face-to-face interactions. Primary interviews are usually carried out on a continuous basis with industry experts in order to acquire a topical understanding of the market as well as to be able to substantiate the existing analysis of the data.

Subject matter expertise involves the validation of the key research findings that were attained from primary and secondary research. The subject matter experts that are consulted have extensive experience in the market research industry and the specific requirements of the clients are reviewed by the experts to check for completion of the market study. Secondary research used for the Stem Cell Alopecia Treatment market report includes sources such as press releases, company annual reports, and research papers that are related to the industry. Other sources can include government websites, industry magazines and associations for gathering more meticulous data. These multiple channels of research help to find as well as substantiate research findings.

Table of Content

1 Introduction of Stem Cell Alopecia Treatment Market

1.1 Overview of the Market1.2 Scope of Report1.3 Assumptions

2 Executive Summary

3 Research Methodology of Verified Market Research

3.1 Data Mining3.2 Validation3.3 Primary Interviews3.4 List of Data Sources

4 Stem Cell Alopecia Treatment Market Outlook

4.1 Overview4.2 Market Dynamics4.2.1 Drivers4.2.2 Restraints4.2.3 Opportunities4.3 Porters Five Force Model4.4 Value Chain Analysis

5 Stem Cell Alopecia Treatment Market, By Deployment Model

5.1 Overview

6 Stem Cell Alopecia Treatment Market, By Solution

6.1 Overview

7 Stem Cell Alopecia Treatment Market, By Vertical

7.1 Overview

8 Stem Cell Alopecia Treatment Market, By Geography

8.1 Overview8.2 North America8.2.1 U.S.8.2.2 Canada8.2.3 Mexico8.3 Europe8.3.1 Germany8.3.2 U.K.8.3.3 France8.3.4 Rest of Europe8.4 Asia Pacific8.4.1 China8.4.2 Japan8.4.3 India8.4.4 Rest of Asia Pacific8.5 Rest of the World8.5.1 Latin America8.5.2 Middle East

9 Stem Cell Alopecia Treatment Market Competitive Landscape

9.1 Overview9.2 Company Market Ranking9.3 Key Development Strategies

10 Company Profiles

10.1.1 Overview10.1.2 Financial Performance10.1.3 Product Outlook10.1.4 Key Developments

11 Appendix

11.1 Related Research

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Stem Cell Alopecia Treatment Market Growth Trends, Key Players, Competitive Strategies and Forecasts to 2026 - Jewish Life News

COVID-19: Potential impact on Animal Stem Cell Therapy Market top key players, size, Analysis, growth, research, Types, Regions and Forecast from…

By adminJune 13, 2020Stem Cell Treatment

The report on the Animal Stem Cell Therapy market provides a birds eye view of the current proceeding within the Animal Stem Cell Therapy market. Further, the report also takes into account the impact of the novel COVID-19 pandemic on the Animal Stem Cell Therapy market and offers a clear assessment of the projected market fluctuations during the forecast period. The different factors that are likely to impact the overall dynamics of the Animal Stem Cell Therapy market over the forecast period (2019-2029) including the current trends, growth opportunities, restraining factors, and more are discussed in detail in the market study.

As per the presented market report, the global Animal Stem Cell Therapy market is projected to attain a CAGR growth of ~XX% during the assessment period and surpass a value of ~US$XX by the end of 20XX. Further, the report suggests that the growth of the Animal Stem Cell Therapy market hinges its hope on a range of factors including, emphasis on innovation by market players, surge in the investments pertaining to R&D activities, and favorable regulatory policies among others.

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Competition Landscape

The report provides critical insights related to the business operations of prominent companies operating in the Animal Stem Cell Therapy market. The revenue generated, market presence of different companies, product range, and the financials of each company is included in the report.

Regional Landscape

The regional landscape section of the report provides resourceful insights related to the scenario of the Animal Stem Cell Therapy market in the key regions. Further, the market attractiveness of each region provides players a clear understanding of the overall growth potential of the Animal Stem Cell Therapy market in each region.

End-User Analysis

The report provides a detailed analysis of the various end-users of the Animal Stem Cell Therapy along with the market share, size, and revenue generated by each end-user.

Segment by Type, the Animal Stem Cell Therapy market is segmented intoDogsHorsesOthers

Segment by Application, the Animal Stem Cell Therapy market is segmented intoVeterinary HospitalsResearch Organizations

Regional and Country-level AnalysisThe Animal Stem Cell Therapy market is analysed and market size information is provided by regions (countries).The key regions covered in the Animal Stem Cell Therapy market report are North America, Europe, Asia Pacific, Latin America, Middle East and Africa. It also covers key regions (countries), viz, U.S., Canada, Germany, France, U.K., Italy, Russia, China, Japan, South Korea, India, Australia, Taiwan, Indonesia, Thailand, Malaysia, Philippines, Vietnam, Mexico, Brazil, Turkey, Saudi Arabia, UAE, etc.The report includes country-wise and region-wise market size for the period 2015-2026. It also includes market size and forecast by Type, and by Application segment in terms of sales and revenue for the period 2015-2026.Competitive Landscape and Animal Stem Cell Therapy Market Share AnalysisAnimal Stem Cell Therapy market competitive landscape provides details and data information by players. The report offers comprehensive analysis and accurate statistics on revenue by the player for the period 2015-2020. It also offers detailed analysis supported by reliable statistics on revenue (global and regional level) by players for the period 2015-2020. Details included are company description, major business, company total revenue and the sales, revenue generated in Animal Stem Cell Therapy business, the date to enter into the Animal Stem Cell Therapy market, Animal Stem Cell Therapy product introduction, recent developments, etc.The major vendors covered:Medivet Biologics LLCVETSTEM BIOPHARMAJ-ARMU.S. Stem Cell, IncVetCell TherapeuticsCelavet Inc.Magellan Stem CellsKintaro Cells PowerAnimal Stem CareAnimal Cell TherapiesCell Therapy SciencesAnimacel

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Real World Evidence Shows OS Improvements in Older Patients With AML From 2000 to 2016 – Cancer Therapy Advisor

By adminJune 13, 2020Stem Cell Treatment

Only adult patients with acute myeloid leukemia (AML) aged 50 years and older showed significant improvements in overall survival (OS) from 2000 to 2016, according to results of a retrospective, population-based study conducted in Denmark. These findings were presented during the Virtual Edition of the 25th European Hematology Association (EHA) Annual Congress.

AML is a clinically and genetically diverse disease associated with a poor prognosis. The estimated median OS for patients diagnosed with AML who are younger than 60 years is less than 3 years and for patients who are diagnosed at age 60 years or older it is less than 1 year. With a median age at diagnosis of approximately 70 years, AML is typically diagnosed in older adults.

Over the last several years, a number of targeted therapies have been approved (eg, FLT3 and IDH inhibitors) or reapproved (eg, gemtuzumab ozogamicin, an anti-CD33 antibody drug conjugate) for the treatment of patients with AML, with the development of novel agents largely driven by an increased understanding of the molecular landscape of the disease. While few new treatments were approved in the setting of AML in the 15 years preceding these recent drug approvals, a number of advancements in routine clinical practice regarding the delivery of chemotherapy, supportive care, and the use of allogeneic hematopoietic stem cell transplantation (ASCT) were made during that period of time.

The primary aim of this study was to examine the temporal change in OS for adult patients with AML included in the Danish National Acute Leukemic Registry who were diagnosed with the disease between 2000 and 2016. In addition, trends in treatment strategy since 2000 were also examined.

Of the 3825 patients included in the overall study cohort, the 2-year OS rates for patients diagnosed with AML between 2000 to 2006, 2007 to 2011, and 2012 to 2016 were not significantly different at 26%, 24%, and 25%, respectively. However, an analysis of patient age at the time of AML diagnosis within these time periods showed an increase in the proportion of patients aged 60 years and older from 2000 to 2006 and 2012 to 2016. While only 27% of patients were 75 years or older in 2000 to 2006, this percentage increased to 36% in 2012 to 2016.

Age-standardized survival analyses showed improvements in OS over time for the subgroup of patients aged 50 years and older at the time of their AML diagnosis. Specifically, the respective rates of 2-year OS in 2002 and 2016 were 41% and 58% for patients aged 50 to 59 years at AML diagnosis (P =.03). The same rate was 21% and 32% for those diagnosed with AML at age 60 to 75 years (P =.009). In contrast, no significant change in 2-year OS rate was observed for patients aged 40 to 49 years (P =.4), and there was a very limited increase in the rate of 2-year OS observed for patients older than 75 years at time of AML diagnosis (P =.046).

For each patient included in the analysis, the most intensive therapy received within 30 days of diagnosis was classified as either intensive (ie, corresponding to therapy that could induce disease remission), nonintensive (eg, low-dose cytarabine or azacitidine), or palliative/no treatment.

Intensive therapy was administered to 53%, 49%, and 44% of study patients during the years 2000 to 2006, 2007 to 2011, and 2012 to 2016, respectively. However, within this group of 1872 patients, there was a substantial increase in the proportion of patients in the 50 to 59 year age subgroup receiving intensive therapy 82.5% during 2000 to 2006 and 92.3% during 2012 to 2016. There was also an increase in the proportion of patients achieving complete remission (CR) following administration of the first or second course of intensive chemotherapy when comparing 2012 to 2016 with 2000 to 2016 (76% vs 71%, respectively; P =.09). These findings translated into a significant improvement in OS (P =.004), which, on age-adjusted survival analyses, was observed for patients between 50 and 75 years treated with intensive therapy.

Whereas only 8% of patients achieving a CR with intensive chemotherapy were referred for ASCT from 2000 to 2006, with a corresponding median age of 46 years, this percentage increased to 28% from 2012 to 2016 (P <.001) and the median age of these patients was 58 years.

Nonintensive chemotherapy was the most intensive therapeutic approach within 30 days of AML diagnosis for 14% of patients from 2012 to 2016 compared with only 3% of patients from 2000 to 2006, and this temporal trend was particularly notable in patients older than 75 years at the time at AML diagnosis, with 21% undergoing nonintensive therapy in the more recent time interval compared with only 3.8% in 2000 to 2006.

Palliative treatment/no treatment was the most intensive therapeutic approach in approximately 40% of patients in between 2000 to 2006 and 2012 to 2016.

In summarizing the results of this study, the presenting author Lasse Jakobsen, PhD, of the Department of Hematology at Aalborg University Hospital in Denmark, stated that when looking specifically at patients between 50 and 75 years of age, we observed a large increase in 2-year OS, which may be attributable to increased use of stem cell transplantation and non-palliative treatment. He also noted that an improvement in selection for both intensive and nonintensive therapies may also contribute to the increase.

Although we observed a significant improvement in OS among the very elderly patients, this improvement was rather limited and these patients still have a very poor outcome, Dr Jakobsen concluded.

Read more of Cancer Therapy Advisors coverage of the EHA virtual meeting by visiting the conference page.

Reference

Jakobsen LH, Roug AS, vlisen AK, et al. Temporal trends in overall survival among adult non-APL AML patients in the period 2000-2016: A Danish population-based study. Presented at: Virtual Edition of the 25th European Hematology Association (EHA) Annual Congress; June 2020. Abstract S145.

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Real World Evidence Shows OS Improvements in Older Patients With AML From 2000 to 2016 - Cancer Therapy Advisor

Omeros’ Narsoplimab Pivotal Trial Data to be Shared as an Oral Presentation at Annual European Hematology Association Congress – Business Wire

By adminJune 13, 2020Stem Cell Treatment

SEATTLE--(BUSINESS WIRE)--Omeros Corporation (Nasdaq: OMER): The results of Omeros pivotal trial of narsoplimab for the treatment of hematopoietic stem cell transplant-associated thrombotic microangiopathy (HSCT-TMA) will be shared as an oral presentation at the virtual edition of the 25th annual European Hematology Association Congress.

The presentation, entitled Narsoplimab (OMS721) for the Treatment of Adult Hematopoietic Stem Cell Transplant-Associated Thrombotic Microangiopathy, will be delivered by Alessandro Rambaldi, M.D., Professor of Hematology at the University of Milan and Head of the Hematology and Bone Marrow Transplant Unit at ASST Papa Giovanni XXIII in Bergamo, Italy.

Due to the worldwide impact of COVID-19, the 25th Congress of the European Hematology Association (EHA) will be held as a virtual meeting. Dr. Rambaldis oral presentation can be accessed on demand by registered meeting attendees on the EHA Virtual Congress platform beginning Friday, June 12, at 08:30 a.m. CEST / 2:30 a.m. EDT. Also on Friday, the presentation slides will be publicly available on Omeros website at https://www.omeros.com/scientific-publications/.

About HSCT-TMA

Hematopoietic stem cell transplant-associated thrombotic microangiopathy (HSCT-TMA) is a significant and often lethal complication of stem cell transplants. This condition is a systemic, multifactorial disorder caused by endothelial cell damage induced by conditioning regimens, immunosuppressant therapies, infection, GvHD, and other factors associated with stem cell transplantation. Endothelial damage, which activates the lectin pathway of complement, plays a central role in the development of HSCT-TMA. The condition occurs in both autologous and allogeneic transplants but is more common in the allogeneic population. In the United States and Europe, approximately 25,000 to 30,000 allogeneic transplants are performed annually. Recent reports in both adult and pediatric allogeneic stem cell transplant populations have found an HSCT-TMA incidence of approximately 40 percent, and high-risk features may be present in up to 80 percent of these patients. In severe cases of HSCT-TMA, mortality can exceed 90 percent and, even in those who survive, long-term renal sequalae are common. There is no approved therapy or standard of care for HSCT-TMA.

About Narsoplimab

Narsoplimab, also known as OMS721, is an investigational human monoclonal antibody targeting mannan-binding lectin-associated serine protease-2 (MASP-2), a novel pro-inflammatory protein target and the effector enzyme of the lectin pathway of complement. Importantly, inhibition of MASP-2 does not appear to interfere with the antibody-dependent classical complement activation pathway, which is a critical component of the acquired immune response to infection. Omeros controls the worldwide rights to MASP-2 and all therapeutics targeting MASP-2.

Phase 3 clinical programs are in progress for narsoplimab in HSCT-TMA, in immunoglobulin A (IgA) nephropathy, and in atypical hemolytic uremic syndrome (aHUS). The FDA has granted narsoplimab breakthrough therapy designations for HSCT-TMA and for IgA nephropathy; orphan drug status for the prevention (inhibition) of complement-mediated thrombotic microangiopathies, for the treatment of HSCT-TMA and for the treatment of IgA nephropathy; and fast track designation for the treatment of patients with aHUS. The European Medicines Agency has granted orphan drug designation to narsoplimab for treatment in HSCT and for treatment of primary IgA nephropathy.

About Omeros Corporation

Omeros is a commercial-stage biopharmaceutical company committed to discovering, developing and commercializing small-molecule and protein therapeutics for large-market as well as orphan indications targeting inflammation, complement-mediated diseases, disorders of the central nervous system and immune-related diseases, including cancers. In addition to its commercial product OMIDRIA (phenylephrine and ketorolac intraocular solution) 1% / 0.3%, Omeros has multiple Phase 3 and Phase 2 clinical-stage development programs focused on complement-mediated disorders and substance abuse. In addition, Omeros has a diverse group of preclinical programs including GPR174, a novel target in immuno-oncology that modulates a new cancer immunity axis recently discovered by Omeros. Small-molecule inhibitors of GPR174 are part of Omeros proprietary G protein-coupled receptor (GPCR) platform through which it controls 54 new GPCR drug targets and their corresponding compounds. The company also exclusively possesses a novel antibody-generating platform.

Combined irradiation and chemotherapy better prepares children for stem cell transplantation than chemotherapy alone – DOTmed HealthCare Business News

By adminJune 13, 2020Stem Cell Treatment

THE HAGUE, Netherlands, June 12, 2020 /PRNewswire/ -- Treatment of childhood cancer is a success story, particularly for acute lymphoblastic leukemia (ALL). More than 90% of ALL patients below 18 years of age are rescued with contemporary chemotherapy. However, the remaining 10% have resistant or reoccurring leukemia and require alternative treatment regimens. One of the most powerful leukemia therapies is hematopoietic stem cell transplantation from a donor (allogeneic HSCT). Approximately 50-80% of pediatric ALL patients that receive allogeneic HSCT are cured, 20% experience leukemic reoccurrence (relapse), and 10% die from complications.

Allogeneic HSCT is a multistep procedure:

Identify a suitable donor, i.e., a compatible sibling or unrelated person.

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Therefore, a large consortium of pediatric transplant experts initiated a global study to investigate whether chemotherapy-based conditioning could substitute TBI. The study is called FORUM (For Omitting Radiation Under Majority Age) and had to be stopped because chemotherapy-based conditioning had significantly poorer outcomes (i.e., lower overall survival rates) than the combination of TBI and chemotherapy. The researchers will now perform prospective monitoring to better define the advantages and limitations of various conditioning approaches.

Presenter: Dr Christina PetersAffiliation: Stem Cell Transplantation Unit, St. Anna Children's Hospital, Vienna, AustriaAbstract: #S102 TBI OR CHEMOTHERAPY BASED CONDITIONING FOR CHILDREN AND ADOLESCENTS WITH ALL: A PROSPECTIVE RANDOMIZED MULTICENTER-STUDY "FORUM" ON BEHALF OF THE AIEOP-BFM-ALL-SG, IBFM-SG, INTREALL-SG AND EBMT-PD-WP

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Combined irradiation and chemotherapy better prepares children for stem cell transplantation than chemotherapy alone - DOTmed HealthCare Business News

Sure, Bit Bio got some significant cash for its cell coding work. But it’s the insiders who are backing them that will garner the attention -…

By adminJune 13, 2020Stem Cell Treatment

You can count the R&D execs at AbbVie among the believers in Genmabs bispecific platform tech.

Moving beyond the Allergan buyout, AbbVie refocused on its cancer drug pipeline, shelling out $750 million in cash and promising up to $3.15 billion more in milestones 60% for development and regulatory goals to ally itself on a slate of 7 development and discovery programs.

At the front of the queue is the early-stage drug epcoritamab, a CD3xCD20 bispecific from its DuoBody collection. Theres also DuoHexaBody-CD37 and DuoBody-CD3x5T4. And then AbbVie gets to pick and choose from among the discovery work at Genmab for 4 more, with AbbVie adding in its own contributions in the pairing up to come.

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Sure, Bit Bio got some significant cash for its cell coding work. But it's the insiders who are backing them that will garner the attention -...

Janus-Faced PCL2? Alzheimer’s Risk Protein Toggles TREM2 and TLR Pathways – Alzforum

By adminJune 13, 2020Induced Pluripotent Stem Cells

12 Jun 2020

Rare variants in TREM2 and PCLG2 influence a persons odds of developing Alzheimers disease, but that is far from all the two genes have in common. According to a study published June 8 in Nature Neuroscience, phospholipase C 2 acts downstream of TREM2 in a signaling pathway that supports critical microglial functions. Using human microglia derived from induced pluripotent stem cells, researchers led by Joseph Lewcock at Denali Therapeutics in South San Francisco reported that knocking out either gene product prevented the immune cells from efficiently processing lipids and neuronal debris. The researchers also found that, independently of TREM2, PLC2 is involved in a pro-inflammatory side hustle dictated by toll-like receptors, which, it so happens, is exacerbated by intracellular lipid build-up. Taken together, the findings strongly implicate faulty microglial lipid handling in the etiology of AD, and support therapeutic strategies that aim to rev up TREM2 signaling.

Using an impressive array of experimental conditions in gene-edited iPSC-microglia, [the authors] demonstrate that PLC2 is a downstream effector of TREM2 and a regulator of lipid metabolism. This exciting discovery directly connects PLC2 to well-established AD pathways involving APOE, TREM2, and microglial activation, commented Rik van der Kant, Vrije University, Amsterdam (full comment below). Florent Ginhoux of the Agency for Science, Technology and Research in Singapore, agreed. The study elegantly links TREM2 and PLC2 signaling pathways, and offers mechanistic insight into how variants in these genes affect the pathophysiology of AD, Ginhoux wrote (full comment below).

Double Dealing. When triggered by TREM2, PLC2 supports lipid metabolism and survival (left). When triggered by TLRs, PLC2 triggers inflammation. In TREM2 KO microglia (right), lipids accumulate and this exacerbates the pro-inflammatory, TLR-driven pathway. [Courtesy of Andreone et al., Nature Neuroscience, 2020.]

Since the discovery, in 2012, that rare variants in the coding region of TREM2 triple the risk of AD, researchers have pegged the receptor as supporting myriad microglial functions, including phagocytosis, walling off A plaques, and promoting an anti-inflammatory, neuroprotective environment (May 2016 news; Apr 2017 conference news;Jul 2018 conference news).

Separately, researchers discovered a rare variant in phospholipase C 2 (PLCG2) that protects against AD (Aug 2017 conference news on Sims et al., 2017). PLCs are a large family of intracellular enzymes that cleave the membrane phospholipid phosphatidylinositol-4,5-bisphosphate (PIP2) to diacylglycerol (DAG) and inositol-1,4,5-trisphosphate (IP3), a process that facilitates calcium signaling. In the brain, the 2 isoform is predominantly expressed by microglia, and initial studies suggest that the protective variant munches phospholipids with more gusto than the common one does (Zhang et al., 2014; May 2019 news).

Might the functions of TREM2 and PLC2 intersect in microglia? To study this question, co-first authors Benjamin Andreone and Laralynne Przybyla derived human microglia. They wove together elements from three recently developed protocols to coax so-called induced microglia (iMGs) from induced pluripotent stem cells (Muffat et al., 2016; Pandya et al., 2017; McQuade et al., 2018). They then used CRISPR to wipe out expression of TREM2 or PLCG2 in these cell-based models.

Under normal conditions, iMGs missing either TREM2 or PLCG2 appeared healthy and viable. When the going got toughi.e., when growth factors were depleted from the culture mediaboth types of knockout suffered a similar fate, dying sooner than their wild-type counterparts. The transcriptomes of each of the two iMG knockouts also differed from those of wild-type cells in similar ways. Specifically, half of the genes differentially expressed in TREM2 KO iMGs were similarly affected in PLCG KO iMGs. These common genes were part of signal transduction pathways downstream of DAP12, the adaptor protein that mediates TREM2 signaling. Using biochemical approaches, the researchers ultimately pieced together a signaling cascade by which lipids activate TREM2, leading to the phosphorylation of Syk2, which directly interacts with PLC2, unleashing its phospholipase activity and downstream signaling events.

Disabling the pathway, either by knocking out TREM2 or PLC2, had a dramatic impact on the processing of lipids, including cholesterol-laden myelin. All microglial lines in this study readily engulfed this type of fluorescently labeled debris; however, while wild-type cells had largely disposed of it after four days, TREM2 or PLCG2 knockouts were still chock-full of it by then. Tellingly, perhaps, the knockout cells failed to ramp up expression of several lipid processing genes in response to the myelin challenge.

Choking on Lipids? Wild-type microglia (left) readily digested lipids after treatment with myelin, while microglia lacking PLCG2 (middle) and TREM2 (right) accumulated the lipids. [Courtesy of Andreone et al., Nature Neuroscience, 2020.]

Lipidomics experiments revealed that the knockouts became burdened with a backlog of several subtypes of unprocessed lipid, including free cholesterol, cholesteryl esters, and myelin-derived ceramides. Similarly, in co-culture experiments with iPSC-derived neurons, both types of microglial knockout were unable to properly digest detritus from injured axons.

How might AD risk variants shift these phenotypes? The researchers generated iMGs that expressed the R47H variant of TREM2, or the protective P522R variant of PLCG2. As might be expected from prior findings on these variants, the R47H-TREM2 iMGs processed lipids more sluggishly than wild-type, whereas the P522R-PLCG2 microglia more deftly disposed of them than wild-type. Together, the findings support the idea that TREM2 and PLCG2 variants influence AD risk via lipid metabolism.

Lest a reader be tempted to tie a neat little bow on this set of results, here comes the twist: PLC2 also takes marching orders from toll-like receptors. This was previously reported in peripheral immune cells. The Denali researchers found the same in iMGs, as PLCG2 knockouts failed to mount a pro-inflammatory response to the TLR2 ligand zymosan.

Interestingly, the same pro-inflammatory cytokines that were down in response to zymosan in PLCG2 knockout iMGs were up in TREM2 knockout iMGs. For example, compared with wild-type iMGs treated with zymosan, PLCG2 knockouts secreted 50 percent less IL-1, while TREM2 knockouts secreted 64 percent more.

The same pattern emerged when the researchers used the TLR4 ligand LPS to trigger the microglial NLRP3 inflammasome, which itself has been tied to AD (Nov 2019 news). Loading up the microglia with myelin prior to triggering the inflammasome dramatically enhanced the inflammatory response in TREM2 KO iMGs, the scientists report. This implies that intracellular lipid accumulation may exacerbate damaging inflammatory pathways. The findings dovetail with those of a recent study that tied lipid droplet-accumulating microglia (LAM) in the aging hippocampus to neuroinflammation (Aug 2019 news).

Overall, the findings cast PLC2 as a two-faced player in microglia. When triggered via TREM2, this phospholipase facilitates processing of lipids and microglial survival. When tripped off by TLRs, it ramps up potentially damaging pro-inflammatory responses. And when lipids build up, as might occur in the aging brain, they exacerbate the pro-inflammatory pathway, Andreone told Alzforum. He believes the balance between these two PLC2 signaling pathways could dictate whether microglia help or harm.

The findings lend support to a therapeutic strategy of agonizing TREM2 signaling, Lewcock told Alzforum. That the protective PLC2 variant enhances lipid processing in microglia fits with the idea that even people whose TREM2 functions normally could stand to benefit from a boost in this pathway. Activating PLC2 is also a potential strategy, Lewcock said, although it would come with the risk of rousing its pro-inflammatory side. More work is needed to dissect how the PLC2 protective variant influences signaling downstream of TREM2 versus TLRs.

This is a very important paper, wrote Christian Haass at the German Center for Neurodegenerative Diseases in Munich. Haass noted that its findings fit with fresh data from his and other groups, but also cautioned that the molecular signature of a protective subpopulation of microglia needs to be defined in much greater detail (full comment below).

Denali is collaborating with Haass group to develop an activating antibody for TREM2, which will come with a blood-brain barrier transport vehicle to shuttle it into the brain (May 2019 conference news;May 2020 news).AL002, a TREM2-activating antibody developed by Alector and Abbvie, entered early clinical trials last year (see clinicaltrials.gov).Jessica Shugart

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Stem Cell Banking Market will Generate Massive Revenue to $6,956 million by 2023 | Cord Blood Registry, ViaCord, Cryo-Cell, China Cord Blood…

By adminJune 13, 2020Embryonic Stem Cells

The global stem cell banking market was valued at $1,986 million in 2016, and is estimated to reach $6,956 million by 2023, registering a CAGR of 19.5% from 2017 to 2023. Stem cell banking is a process where the stem cell care isolated from different sources such as umbilical cord and bone marrow that is stored and preserved for future use. These cells can be cryo-frozen and stored for decades. Private and public banks are different types of banks available to store stem cells.

Top Companies Covered in this Report: Cord Blood Registry, ViaCord, Cryo-Cell, China Cord Blood Corporation, Cryo-Save, New York Cord Blood Program, CordVida, Americord, CryoHoldco, Vita34

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Increase in R&D activities in regards with applications of stem cells and increase in prevalence of fatal chronic diseases majorly drive the growth of the global stem cell banking market. Moreover, the large number of births occurring globally and growth in GDP & disposable income help increase the number of stem cell units stored, which would help fuel the market growth. However, legal and ethical issues related to stem cell collections and high processing & storage cost are projected to hamper the market growth. The initiative taken by organizations and companies to spread awareness in regards with the benefits of stem cells and untapped market in the developing regions help to open new avenues for the growth of stem cell banking market in the near future.

The global stem cell banking market is segmented based on cell type, bank type, service type, utilization, and region. Based on cell type, the market is classified into umbilical cord stem cells, adult stem cells, and embryonic stem cells. Depending on bank type, it is bifurcated into public and private. By service type, it is categorized into collection & transportation, processing, analysis, and storage. By utilization, it is classified into used and unused. Based on region, it is analyzed across North America, Europe, Asia-Pacific, and LAMEA.

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Table Of Content

CHAPTER 1: INTRODUCTION

CHAPTER 2: EXECUTIVE SUMMARY

CHAPTER 3: MARKET OVERVIEW

CHAPTER 4: STEM CELL BANKING MARKET, BY CELL TYPE

CHAPTER 5: STEM CELL BANKING MARKET, BY BANK TYPE

CHAPTER 6: STEM CELL BANKING MARKET, BY SERVICE TYPE

CHAPTER 7: STEM CELL BANKING MARKET, BY UTILIZATION

CHAPTER 8: STEM CELL BANKING MARKET, BY REGION

CHAPTER 9: COMPANY PROFILES

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Stem Cell Banking Market will Generate Massive Revenue to $6,956 million by 2023 | Cord Blood Registry, ViaCord, Cryo-Cell, China Cord Blood...

Majority of Evaluable Patients Across Genotypes Achieve Transfusion Independence and Maintain It with Near-Normal Hemoglobin Levels in Phase 3 Studies…

By adminJune 13, 2020Stem Cell Clinic

89% of evaluable patients (17/19) with transfusion-dependent -thalassemia who do not have a 0/0 genotype achieved transfusion independence with 11.9 g/dL median weighted average total hemoglobin (Hb) level in HGB-207

Data from exploratory analyses of HGB-207 show improved markers of blood cell production and bone marrow function in patients who achieved transfusion independence

85% of patients (11/13) with a 0/0 genotype or IVS-I-110 mutation in HGB-212 have been transfusion-free for at least 7 months

bluebird bio, Inc. (Nasdaq: BLUE) today announced that new data from ongoing Phase 3 studies of betibeglogene autotemcel (beti-cel; formerly LentiGlobin for -thalassemia gene therapy) show pediatric, adolescent and adult patients with a range of genotypes of transfusion-dependent -thalassemia (TDT) achieve and maintain transfusion independence with hemoglobin (Hb) levels that are near-normal (10.5 g/dL). These data are being presented at the Virtual Edition of the 25th European Hematology Association (EHA25) Annual Congress.

"With more than a decade of clinical experience evaluating gene therapy in patients with transfusion dependent -thalassemia across a wide range of ages and genotypes, we have built the most comprehensive understanding of treatment outcomes in the field," said David Davidson, M.D., chief medical officer, bluebird bio. "Seeing patients achieve transfusion independence and maintain that positive clinical benefit over time with robust hemoglobin levels reflects our initial vision of the potential of beti-cel. The accumulating long-term data demonstrating improvements in bone marrow histology, iron balance and red cell biology support the potential of beti-cel to correct the underlying pathophysiology of transfusion-dependent -thalassemia."

A total of 60 pediatric, adolescent and adult patients across genotypes of TDT have been treated with beti-cel in the Phase 1/2 Northstar (HGB-204) and HGB-205 studies, and the Phase 3 Northstar-2 (HGB-207) and Northstar-3 (HGB-212) studies as of March 3, 2020. In studies of beti-cel, transfusion independence is defined as no longer needing red blood cell transfusions for at least 12 months while maintaining a weighted average Hb of at least 9 g/dL.

TDT is a severe genetic disease caused by mutations in the -globin gene that results in significantly reduced or absent adult hemoglobin (HbA). In order to survive, people with TDT maintain Hb levels through lifelong, chronic blood transfusions. These transfusions carry the risk of progressive multi-organ damage due to unavoidable iron overload.

"Patients with transfusion-dependent -thalassemia do not make enough healthy red blood cells and cannot live without chronic transfusions; for patients that means a lifetime of necessary visits to a hospital or clinic and reliance on an often unreliable blood supply, which compounds the challenges of managing this disease," said presenting study author Professor John B. Porter, MA, M.D., FRCP, FRCPath, University College London Hospital, London, UK. "These results showing patients free from transfusions and maintaining near-normal hemoglobin levels after treatment with beti-cel is a positive outcome for people living with transfusion-dependent -thalassemia. In addition, we now have more data that provide further evidence that most of these patients have a measurable improvement in markers of healthy red blood cell production."

Beti-cel is a one-time gene therapy designed to address the underlying genetic cause of TDT by adding functional copies of a modified form of the -globin gene (A-T87Q-globin gene) into a patients own hematopoietic (blood) stem cells (HSCs). This means there is no need for donor HSCs from another person, as is required for allogeneic HSC transplantation (allo-HSCT). Once a patient has the A-T87Q-globin gene, they have the potential to produce HbAT87Q, which is gene therapy-derived Hb, at levels that eliminate or significantly reduce the need for transfusions.

As of March 3, 2020, all 23 patients in HGB-207 were treated and have been followed for a median of 19.4 months. These patients ranged in age from four to 34 years, including eight pediatric (<12 years of age) and 15 adolescent/adult (>12 years of age) patients. Only 19 patients were evaluable for transfusion independence; four additional patients do not yet have sufficient follow-up to be assessed for transfusion independence.

Eighty-nine percent of evaluable patients (17/19) achieved transfusion independence, with median weighted average total Hb levels of 11.9 g/dL (min-max: 9.4 12.9 g/dL) over a median of 19.4 months of follow-up to date (min-max: 12.3 31.4 months). These 17 patients previously required a median of 17.5 transfusions per year (min-max: 11.5 37 transfusions per year).

Improved iron levels, as measured by serum ferritin and hepcidin levels (proteins involved in iron storage and homeostasis), were observed and trends toward improved iron management were seen. Over half of patients stopped chelation therapy, which is needed to reduce excess iron caused by chronic blood transfusions. Seven out of 23 patients began using phlebotomy for iron reduction.

Analysis of Healthy Red Blood Cell Production

In exploratory analyses, biomarkers of ineffective erythropoiesis (red blood cell production) were evaluated in patients who achieved transfusion independence in HGB-207.

The myeloid to erythroid (M:E) ratio in bone marrow from patients who achieved transfusion independence increased from a median of 1:3 (n=17) at baseline to 1:1.2 (n=16) at Month 12. Improvement of the M:E ratio, the ratio of white blood cell and red blood cell precursors in the bone marrow, suggests an improvement in mature red blood cell production. Images illustrating the bone marrow cellularity at baseline, Month 12 and Month 24 are available in the EHA25 presentation (abstract #S296): "Improvement in erythropoiesis in patients with transfusion-dependent -thalassemia following treatment with betibeglogene autotemcel (LentiGlobin for -thalassemia) in the Phase 3 HGB-207 study".

Additionally, biomarkers of erythropoiesis continue to demonstrate a trend toward normalization in patients who achieved transfusion independence, including improved levels over time of erythropoietin, a hormone involved in red blood cell production; reticulocytes, immature red blood cells; and soluble transferrin receptor, a protein measured to help evaluate iron status. The continued normalization of red blood cell production over time among some patients who achieved transfusion independence supports the disease-modifying potential of beti-cel in patients with TDT.

Northstar-3 (HGB-212) Efficacy

As of March 3, 2020, 15 patients (genotypes: 9 0/0, 3 0/ +IVS1-110, 3 homozygous IVS-1-110 mutation) were treated and had a median follow-up of 14.4 months (min-max: 1.124.0 months). Median age at enrollment was 15 (min-max: 4 33 years).

Six of eight evaluable patients achieved transfusion independence, with median weighted average total Hb levels of 11.5 g/dL (min-max: 9.5 13.5 g/dL), and continued to maintain transfusion independence for a median duration of 13.6 months (min-max: 12.2 21.2 months) as of the data cutoff.

Eighty-five percent of patients (11/13) with at least seven months of follow-up had not received a transfusion in more than seven months at time of data cutoff. These 11 patients previously required a median of 18.5 transfusions per year (min-max: 11.0 39.5 transfusions per year). In these patients, gene therapy-derived HbAT87Q supported total Hb levels ranging from 8.814.0 g/dL at last visit.

Betibeglogene autotemcel Safety

Non-serious adverse events (AEs) observed during the HGB-207 and HGB-212 trials that were considered related or possibly related to beti-cel were tachycardia, abdominal pain, pain in extremities, leukopenia, neutropenia and thrombocytopenia. One serious event of thrombocytopenia was considered possibly related to beti-cel.

In HGB-207, serious events post-infusion in two patients included three events of veno-occlusive liver disease and two events of thrombocytopenia. In HGB-212, serious events post-infusion in two patients included two events of pyrexia.

Additional AEs observed in clinical studies were consistent with the known side effects of HSC collection and bone marrow ablation with busulfan, including SAEs of veno-occlusive disease.

In both Phase 3 studies, there have been no deaths, no graft failure, no cases of vector-mediated replication competent lentivirus or clonal dominance, no leukemia and no lymphoma.

The presentations are now available on demand on the EHA25 website:

About betibeglogene autotemcel

The European Commission granted conditional marketing authorization (CMA) for betibeglogene autotemcel (beti-cel; formerly LentiGlobin gene therapy for -thalassemia), marketed as ZYNTEGLO gene therapy, for patients 12 years and older with transfusion-dependent -thalassemia (TDT) who do not have a 0/0 genotype, for whom hematopoietic stem cell (HSC) transplantation is appropriate, but a human leukocyte antigen (HLA)-matched related HSC donor is not available. On April 28, 2020, the European Medicines Agency (EMA) renewed the CMA for ZYNTEGLO, supported by data from 32 patients treated with ZYNTEGLO, including three patients with up to five years of follow-up.

TDT is a severe genetic disease caused by mutations in the -globin gene that result in reduced or significantly reduced hemoglobin (Hb). In order to survive, people with TDT maintain Hb levels through lifelong chronic blood transfusions. These transfusions carry the risk of progressive multi-organ damage due to unavoidable iron overload.

Beti-cel adds functional copies of a modified form of the -globin gene (A-T87Q-globin gene) into a patients own hematopoietic (blood) stem cells (HSCs). Once a patient has the A-T87Q-globin gene, they have the potential to produce HbAT87Q, which is gene therapy-derived hemoglobin, at levels that may eliminate or significantly reduce the need for transfusions.

Non-serious adverse events (AEs) observed during clinical studies that were attributed to beti-cel included abdominal pain, thrombocytopenia, leukopenia, neutropenia, hot flush, dyspnea, pain in extremity and non-cardiac chest pain. Two serious adverse events (SAE) of thrombocytopenia was considered possibly related to beti-cel.

Additional AEs observed in clinical studies were consistent with the known side effects of HSC collection and bone marrow ablation with busulfan, including SAEs of veno-occlusive disease.

The CMA for beti-cel is valid in the 27 member states of the EU as well as UK, Iceland, Liechtenstein and Norway. For details, please see the Summary of Product Characteristics (SmPC).

The U.S. Food and Drug Administration (FDA) granted beti-cel orphan drug designation and Breakthrough Therapy designation for the treatment of transfusion-dependent -thalassemia. Beti-cel is not approved in the U.S.

Beti-cel continues to be evaluated in the ongoing Phase 3 Northstar-2 and Northstar-3 studies. For more information about the ongoing clinical studies, visit http://www.northstarclinicalstudies.com or clinicaltrials.gov and use identifier NCT02906202 for Northstar-2 (HGB-207) and NCT03207009 for Northstar-3 (HGB-212).

bluebird bio is conducting a long-term safety and efficacy follow-up study (LTF-303) for people who have participated in bluebird bio-sponsored clinical studies of betibeglogene autotemcel or LentiGlobin for SCD. For more information visit: https://www.bluebirdbio.com/our-science/clinical-trials or clinicaltrials.gov and use identifier NCT02633943 for LTF-303.

About bluebird bio, Inc.

bluebird bio is pioneering gene therapy with purpose. From our Cambridge, Mass., headquarters, were developing gene therapies for severe genetic diseases and cancer, with the goal that people facing potentially fatal conditions with limited treatment options can live their lives fully. Beyond our labs, were working to positively disrupt the healthcare system to create access, transparency and education so that gene therapy can become available to all those who can benefit.

bluebird bio is a human company powered by human stories. Were putting our care and expertise to work across a spectrum of disorders including cerebral adrenoleukodystrophy, sickle cell disease, -thalassemia and multiple myeloma using three gene therapy technologies: gene addition, cell therapy and (megaTAL-enabled) gene editing.

bluebird bio has additional nests in Seattle, Wash; Durham, N.C.; and Zug, Switzerland. For more information, visit bluebirdbio.com.

Follow bluebird bio on social media: @bluebirdbio, LinkedIn, Instagram and YouTube.

ZYNTEGLO, LentiGlobin, and bluebird bio are trademarks of bluebird bio, Inc.

bluebird bio Forward-Looking Statements

This release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995. Any forward-looking statements are based on managements current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to: the risk that the COVID-19 pandemic and resulting impact on our operations and healthcare systems will affect the execution of our development plans or the conduct of our clinical studies; the risk that the efficacy and safety results observed in the patients treated in our prior and ongoing clinical trials of beti-cel may not persist; and the risk that the efficacy and safety results from our prior and ongoing clinical trials will not continue or be repeated with additional patients in our ongoing or planned clinical trials or in the commercial context; the risk that the FDA will require additional information regarding beti-cel, resulting in a delay to our anticipated timelines for regulatory submissions, including submission of our BLA. For a discussion of other risks and uncertainties, and other important factors, any of which could cause our actual results to differ from those contained in the forward-looking statements, see the section entitled "Risk Factors" in our most recent Form 10-Q, as well as discussions of potential risks, uncertainties, and other important factors in our subsequent filings with the Securities and Exchange Commission. All information in this press release is as of the date of the release, and bluebird bio undertakes no duty to update this information unless required by law.

View source version on businesswire.com: https://www.businesswire.com/news/home/20200612005084/en/

Contacts

Media:Catherine Falcetti, 339-499-9436cfalcetti@bluebirdbio.com

Investors:Ingrid Goldberg, 410-960-5022igoldberg@bluebirdbio.com

Elizabeth Pingpank, 617-914-8736epingpank@bluebirdbio.com

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Majority of Evaluable Patients Across Genotypes Achieve Transfusion Independence and Maintain It with Near-Normal Hemoglobin Levels in Phase 3 Studies...

Cord Blood Banking Leader Cryo-Cell Enters into Patent Option Agreement with Duke University – Global Banking And Finance Review

By adminJune 13, 2020Stem Cell Clinic

OLDSMAR, Fla., June 11, 2020 Cryo-Cell International, Inc. (OTC:QB Markets Group Symbol: CCEL) (the Company), the worlds first private cord blood bank to separate and store stem cells in 1992, announced that effective June 9, 2020, the Company has entered into a patent option agreement with Duke University. The six-month exclusive option agreement gives Cryo-Cell an option to obtain a license to manufacture and sell products based on Dr. Joanne Kurtzbergs patents. Please see the Companys Form 8-K filed with the Securities and Exchange Commission on June 11, 2020 for more details.

David Portnoy, Chairman of the Board and Co-CEO, said, Cryo-Cell is honored to have entered into this option agreement with Dr. Kurtzberg and Duke University and believes that this is a significant step in the transformation of the Company. We look forward to Duke becoming a major shareholder and partner of the Company for many years to come.

We are excited to enter into this exclusive option agreement with Cryo-Cell and are looking forward to working together to bring novel cord blood and birthing tissue based cellular therapeutics to the clinic, said Joanne Kurtzberg, MD, who is the Jerome S. Harris distinguished professor of pediatrics and a pioneer in cell therapies based on umbilical cord blood. Dr. Joanne Kurtzberg is an internationally renowned expert in pediatric hematology/oncology, pediatric blood and marrow transplantation, umbilical cord blood banking and transplantation, and novel applications of cord blood in the emerging fields of cellular therapies and regenerative medicine.

About Cryo-Cell International, Inc.

Founded in 1989, Cryo-Cell International, Inc. is the worlds first private cord blood bank. More than 500,000 parents from 87 countries have entrusted Cryo-Cell International with their babys cord blood and cord tissue stem cells. In addition to its family bank, Cryo-Cell International has a public banking program in partnership with Duke University. Cryo-Cells public bank has provided cord blood for more than 600 transplantations and operates cord blood donation sites across the U.S in prominent hospitals such as CedarsSinai Hospital in Los Angeles and Baptist Hospital in Miami. Cryo-Cells mission is to provide clients with state-of-the-art cord blood and cord tissue cryopreservation services, raise awareness of the opportunity for expectant parents to bank or donate their babys cord blood and support the advancement of regenerative medicine. Cryo-Cell operates in a facility that is FDA registered, cGMP-/cGTP-compliant and licensed in all states requiring licensure. Besides being AABB accredited as a cord blood facility, Cryo-Cell was also the first U.S. (for private use only) cord blood bank to receive FACT accreditation for adhering to the most stringent cord blood quality standards set by any internationally recognized, independent accrediting organization. In addition, Cryo-Cell is ISO 13485:2003certified by TV, an internationally recognized, quality assessment organization. Cryo-Cell is a publicly traded company, OTCQB:CCEL. For more information, please visit http://www.cryo-cell.com.

Forward-Looking Statement

Statements herein the terms believes, intends, projects, anticipates, expects, and similar expressions as used are intended to reflect forward-looking statements of the Company. The information contained herein is subject to various risks, uncertainties and other factors that could cause actual results to differ materially from the results anticipated in such forward-looking statements or paragraphs, many of which are outside the control of the Company. These uncertainties and other factors include the impact of the COVID-19 pandemic on our sales, operations and supply chain, the success of the Companys global expansion initiatives and product diversification, the Companys actual future ownership stake in future therapies emerging from its collaborative research partnerships, the success related to its IP portfolio, the Companys ability to enter into a definitive license agreement with Duke, the Companys future competitive position in stem cell innovation, future success of its core business and the competitive impact of public cord blood banking on the Companys business, the Companys ability to minimize future costs to the Company related to R&D initiatives and collaborations and the success of such initiatives and collaborations, the success and enforceability of the Companys menstrual stem cell technology license agreements and umbilical cord blood license agreements and their ability to provide the Company with royalty fees, the ability of the reproductive tissue storage to generate new revenues for the Company and those risks and uncertainties contained in risk factors described in documents the Company files from time to time with the Securities and Exchange Commission, including the most recent Annual Report on Form 10-K, Quarterly Reports on Form 10-Q and any Current Reports on Form 8-K filed by the Company. The Company disclaims any obligations to subsequently revise any forward-looking statements to reflect events or circumstances after the date of such statements.

Contact: David Portnoy, Chairman and Co-CEO Cryo-Cell International, Inc. 813-749-2100 [emailprotected]

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Cord Blood Banking Leader Cryo-Cell Enters into Patent Option Agreement with Duke University - Global Banking And Finance Review