Results From Pivotal Phase 2/3 Study of Emapalumab in Patients With Primary HLH Published in New Eng – PharmiWeb.com

STOCKHOLM, May 7, 2020 /PRNewswire/ -- Sobi announced today that the results from the pivotal phase 2/3 study evaluating the efficacy and safety of emapalumab in patients with primary haemophagocytic lymphohistiocytosis (HLH) were published in the New England Journal of Medicine on 7 May 2020. Emapalumab is the first therapy approved by the US Food & Drug Administration (FDA) for primary HLH and is under review by the European Medicines Agency (EMA).

Primary HLH is a rare syndrome that typically presents in infancy but can also be seen in adults and is associated with high morbidity and mortality. This life-threatening disease is characterised by immune dysregulation and uncontrolled hyperinflammation. The treatment objective is to suppress the hyperinflammation and control the acute features of the disease in order to successfully bring patients to haematopoietic stem cell transplantation (HSCT).

"The publication of the results in this highly respected medical journal is a testament to the medical importance of the emapalumab findings for patients with primary HLH," says Milan Zdravkovic, Head of Research & Development and Chief Medical Officer at Sobi. "The results further advance our understanding of primary HLH and the role of interferon gamma in its pathogenesis. Our hope is to contribute to the improvement of care and treatment for patients suffering from this potentially fatal disease."

The results with emapalumab in primary HLH published in the New England Journal of Medicine highlight the overall response rate of 63 percent in previously treated patients at the end of up to 8 weeks of treatment (compared to the pre-specified null hypothesis of 40 percent (p=0.02)). In the previously treated group, 70 percent of patients were able to proceed to transplantation. The most commonly reported adverse reactions ( 20 per cent) were infections, hypertension, infusion-related reactions and fever.

Michael Jordan, Professor of Pediatrics at the Cincinnati Children's Hospital Medical Center in the US and coordinating Principal Investigator of the study (US), confirms the importance of making advances in finding new therapies for HLH and emphasises the importance of the publication of the results: "The findings from the study are encouraging for those affected by this devastating disease."

Professor Franco Locatelli, Head of the Department of Onco-Haematology, Bambino Ges Children's Hospital IRCCS, Sapienza University of Rome, Italy, and coordinating Principal Investigator (EU), adds: "Emapalumab represents a prototype model molecularly targeted therapy and an important step towards improving outcomes for this severe and life-threatening disease."

This pivotal clinical study is the first study in primary HLH to prospectively assess and report treatment responses using predefined comprehensive objective clinical and laboratory criteria. Preclinical data have shown the central role of interferon gamma (IFN) in the pathogenesis of this disease1. Emapalumab is a monoclonal antibody that binds to and neutralises IFN. It was approved by the US Food & Drug Administration (FDA) on the basis of this clinical study for the treatment of primary HLH in adult and paediatric (newborn and older) patients with refractory, recurrent or progressive disease, or intolerance to conventional HLH therapy, and received Breakthrough Designation prior to review.

About emapalumab

Emapalumab is a monoclonal antibody that binds to and neutralises interferon gamma (IFN). In the US, emapalumab is indicated for paediatric (newborn and older) and adult primary haemophagocytic lymphohistiocytosis (HLH) patients with refractory, recurrent or progressive disease, or intolerance to conventional HLH therapy. Emapalumab is the first and only medicine approved in the US for primary HLH, a rare syndrome of hyperinflammation that usually occurs within the first year of life and can rapidly become fatal unless diagnosed and treated. The FDA approval is based on data from the phase 2/3 studies (NCT01818492 and NCT02069899). Emapalumab is indicated for administration through intravenous infusion over one hour twice per week until haematopoietic stem cell transplantation (HSCT). For more information please see http://www.gamifant.com including the full US Prescribing Information. Emapalumab is under review for primary HLH by the European Medicines Agency (EMA).

About SobiTM

Sobi is a specialised international biopharmaceutical company transforming the lives of people with rare diseases. Sobi is providing sustainable access to innovative therapies in the areas of haematology, immunology and specialty indications. Today, Sobi employs approximately 1,400 people across Europe, North America, the Middle East, Russia and North Africa. In 2019, Sobi's revenues amounted to SEK 14.2 billion. Sobi's share (STO:SOBI) is listed on Nasdaq Stockholm. You can find more information about Sobi at sobi.com.

For more information please contactPaula Treutiger, Head of Communication & Investor Relations+ 46-733-666-599paula.treutiger@sobi.com

Linda Holmstrm, Corporate Communication & Investor Relations+46-708-734-095linda.holmstrom@sobi.com

1. Jordan et al. Blood 2004;104:735-43.

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Results From Pivotal Phase 2/3 Study of Emapalumab in Patients With Primary HLH Published in New Eng - PharmiWeb.com

Characterization and Immunomodulation of Canine Amniotic Membrane Stem | SCCAA – Dove Medical Press

Alessandra de Oliveira Pinheiro,1 Valria M Lara,1 Aline F Souza,1 Juliana B Casals,2 Fabiana F Bressan,1 Paulo Fantinato Neto,1 Vanessa C Oliveira,1 Daniele S Martins,1 Carlos E Ambrosio1

1Department of Veterinary Medicine, Faculty of Animal Science and Food Engineering, University of So Paulo, Pirassununga, So Paulo, Brazil; 2Private Veterinary Practice, Pirassununga, So Paulo, Brazil

Correspondence: Carlos E AmbrosioDepartment of Veterinary Medicine, Faculty of Animal Science and Food Engineering, University of So Paulo, FZEA- Av. Duque de Caxias Norte, 225, ZMV, Pirassununga 13635-900, So Paulo, BrazilTel +55 19 3565-4113 Email ceambrosio@usp.br

Purpose: Amniotic membrane stem cells have a high capacity of proliferation, cell expansion, and plasticity, as well as immunomodulatory properties that contribute to maternal-fetal tolerance. Owing to the lack of research on human amniotic membrane at different gestational stages, the canine model is considered ideal because of its genetic and physiological similarities. We aimed to characterize the canine amniotic membrane (CAM) cell lineage in different gestational stages and evaluate the expression of immunomodulatory genes.Materials and Methods: Twenty CAMs from early (20 30 days) (n=7), mid- (31 45 days) (n=7), and late gestation (46 63 days) (n=6) stages were studied. The cell features were assessed by cell viability tests, growth curve, colony-forming units, in vitro differentiation, cell labeling for different immunophenotypes, and pluripotent potential markers. The cells were subjected to RT-PCR and qPCR analysis to determine the expression of IDO, HGF, EGF, PGE2, and IL-10 genes.Results: CAM cells exhibited a fibroblastoid morphology and adherence to plastic with an average cell viability of 78.5%. The growth curve indicated a growth peak in the second passage and we obtained an average of 138.2 colonies. Osteogenic, chondrogenic, and adipogenic lineages were confirmed by in vitro differentiation assays. Cellular immunophenotyping experiments confirmed the presence of positive mesenchymal markers (CD90 and CD105) and the low or negative expression of hematopoietic markers (CD45 and CD34). Qualitative analysis of the immunomodulatory functions indicated the expression of the IDO, HGF, EGF5, and PGE2 genes. When stimulated by interferon-gamma, CAM cells exhibited higher IDO levels throughout gestation.Conclusion: The CAMs from different gestational stages presented features consistent with mesenchymal stem cell lineage; better results were observed during the late gestation stage. Therefore, the gestational stage is a key factor that may influence the functionality of therapies when using fetal membrane tissues from different periods of pregnancy.

Keywords: canine stem cells, immunomodulation, fetal annexes

This work is published by Dove Medical Press Limited, and licensed under a Creative Commons Attribution License.The full terms of the License are available at http://creativecommons.org/licenses/by/4.0/.The license permits unrestricted use, distribution, and reproduction in any medium, provided the original author and source are credited.

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Characterization and Immunomodulation of Canine Amniotic Membrane Stem | SCCAA - Dove Medical Press

Navigating cancer as a young adult: ‘I’m trying to figure out who I am’ – Stanford Medical Center Report

When David Llano was diagnosed with leukemia in June 2014, the news came as a shock.

Then 17, Llano had just finished his junior year of high school, and he was looking forward to a summer of hanging out with friends. Instead, he was immediately hospitalized at Lucile Packard Children's Hospital Stanford.

For the next few months, he was extremely ill, as he received chemotherapy treatments and eventually a stem cell transplant.

I wrote about his experience in a feature story for Stanford Medicine magazine. As the article explains, teens and young adults with cancer face biological and psychosocial challenges distinct from those of other cancer patients, both in treatment and as they recover.

During my reporting, I spoke with Stanford child and adolescent psychologist Emily Ach, PhD, who frequently works with teens who have cancer.

"The rift between the patient's experience and what's happening with their peer group is a real challenge to figure out," she told me.

Llano said he felt this rift when he returned to high school four months after his diagnosis. From the story:

The first day back at school was harder -- weirder, really -- than he expected.

"People I didn't even know would go up to me and hug me, be really touchy with me," he said. "All the kids pitied me."

His experience isn't unusual, psychologist Ach said. "Maybe at college reentry, kids are more sensitive and appropriate, but not necessarily," she said. "In high school, they're pretty reliably not, and being different in any way is really hard."

When Ach is helping patients prepare to return to school, she reminds them that although many aspects of having cancer are outside of their control, they get to choose how much information about their illness to share.

"Kids vary widely in terms of how open they want to be," Ach told me. For instance, some teens are comfortable responding to a question about a scar by saying, "Oh, I was treated for leukemia. That's where I had a port for my chemo." Others might prefer to say, "I don't really want to talk about it. I was sick, and I had a medical procedure."

Regardless of the specifics, planning responses for awkward questions helps, Ach said. "For a lot of kids, coming up with that on the spot is really hard, and the potential to be caught off-guard is so anxiety-provoking," she said.

Like many young cancer patients, Llano initially had trouble seeing what his future would hold: "You don't really have an identity," he said. "You're like, 'I have cancer and I'm trying to figure out who I am.'"

He kept up a few close friendships from before his illness, but decided to complete his senior year of high school at the hospital school at Packard Children's. There, other students had their own challenging medical journeys and wouldn't be surprised by what he'd been through.

Llano also became active in peer advising at the hospital, helping other teens navigate the challenges of cancer.

He is now in good health and planning a career as a child-life specialist.

Photography, including image of cancer survivor David Llano at his Sunnyvale home, by Timothy Archibald

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Lineage Cell Therapeutics Reports New Data With OpRegen for the Treatment of Dry AMD With Geographic Atrophy – BioSpace

CARLSBAD, Calif.--(BUSINESS WIRE)-- Lineage Cell Therapeutics, Inc. (NYSE American and TASE: LCTX), a clinical-stage biotechnology company developing novel cell therapies for unmet medical needs, today announced that updated results from a Phase I/IIa study of its lead product candidate, OpRegen, a retinal pigment epithelium (RPE) cell transplant therapy currently in development for the treatment of dry age-related macular degeneration (AMD), were published online via the ARVOLearn platform as part of the 2020 Association for Research in Vision and Ophthalmology (ARVO) Meeting. The presentation entitled, Phase I/IIa Clinical Trial of Human Embryonic Stem Cell (hESC)-Derived Retinal Pigmented Epithelium (RPE, OpRegen) Transplantation in Advanced Dry Form Age-Related Macular Degeneration (AMD): Interim Results (Abstract # 3363764), was presented by Christopher D. Riemann, M.D., Vitreoretinal Surgeon and Fellowship Director, Cincinnati Eye Institute (CEI) and University of Cincinnati School of Medicine. Dr. Riemanns presentation is available on the Media page of the Lineage website. Lineage will also host a live call with Dr. Riemann, on Monday, May 11, 2020 at 5:00 p.m. ET/2:00 p.m. PT to further discuss the results of treatment with OpRegen. Interested parties can access the call on the Events and Presentations section of Lineages website.

This update is significant as it builds on our earlier reports of gains in visual acuity and provides a more comprehensive picture of treatment with OpRegen for dry AMD, with meaningful improvements in the progression of geographic atrophy, visual acuity, and reading speed observed in our first Cohort 4 patient and first Orbit SDS with thaw-and-inject formulation dosed patient, stated Brian M. Culley, Lineage CEO. As dry AMD is a slow and progressive disease, it takes many months to observe changes to retinal anatomy or visual acuity. With the benefit of longer follow-up, we now can report that some OpRegen treated patients are able to see better, have less growth in their area of GA, and are able to read faster, all of which represent significant enhancements to vision and quality of life metrics. In addition to these individual results, the pooled data continues to suggest a treatment effect in both visual acuity and GA progression. Notably, we also are reporting additional evidence that OpRegen cells remain present for at least 4 years and hope that longer follow-up periods will reinforce a growing body of evidence that OpRegen is well-tolerated and can provide sustained and clinically meaningful benefits with a single dose of RPE cells. Our near-term objective is to treat and monitor the final four patients in Cohort 4 of the current study and utilize these data to direct our clinical, regulatory, and partnership discussions. Our goal is to combine the best cell line, the best production process, and the best delivery system, to position OpRegen as the front-runner in the race to address the unmet need in the potential billion-dollar dry AMD market.

As a principal investigator on the OpRegen clinical study, I am excited to present this most recent update, where all Cohort 4 patients treated with OpRegen had improved Best Corrected Visual Acuity up to one year or at their last visit, demonstrating a substantial treatment response, stated Christopher D. Riemann, M.D. The pooled Cohort 4 data demonstrate a significant, greater than 10-letter sustained visual acuity improvement over the entire followup period. Reading center assessments of GA also suggest a reduction in GA progression in the OpRegen treated eye when compared to fellow eye in Cohort 4. I am encouraged by the results observed in patients treated to date with OpRegen and I look forward to dosing patients in this study at CEI.

KOL Call Information and Webcast

Lineage will host a conference call with Dr. Riemann, on Monday, May 11, 2020 at 5:00 p.m. ET/2:00 p.m. PT to further discuss the results following treatment with OpRegen. A live webcast of the conference call will be available online in the Events and Presentations section of Lineages website. Interested parties may also access the conference call by dialing (866) 888-8633 from the U.S. and Canada and (636) 812-6629 from elsewhere outside the U.S. and Canada and should request the Lineage Cell Therapeutics Call. A replay of the webcast will be available on Lineages website for 30 days and a telephone replay will be available through May 19, 2020, by dialing (855) 859-2056 from the U.S. and Canada and (404) 537-3406 from elsewhere outside the U.S. and Canada and entering conference ID number 6597936.

About Lineage Cell Therapeutics, Inc.

Lineage Cell Therapeutics is a clinical-stage biotechnology company developing novel cell therapies for unmet medical needs. Lineages programs are based on its robust proprietary cell-based therapy platform and associated in-house development and manufacturing capabilities. With this platform Lineage develops and manufactures specialized, terminally differentiated human cells from its pluripotent and progenitor cell starting materials. These differentiated cells are developed to either replace or support cells that are dysfunctional or absent due to degenerative disease or traumatic injury or administered as a means of helping the body mount an effective immune response to cancer. Lineages clinical programs are in markets with billion dollar opportunities and include three allogeneic (off-the-shelf) product candidates: (i) OpRegen, a retinal pigment epithelium transplant therapy in Phase 1/2a development for the treatment of dry age-related macular degeneration, a leading cause of blindness in the developed world; (ii) OPC1, an oligodendrocyte progenitor cell therapy in Phase 1/2a development for the treatment of acute spinal cord injuries; and (iii) VAC2, a cancer immunotherapy of antigen-presenting dendritic cells in Phase 1 development for the treatment of non-small cell lung cancer. For more information, please visit http://www.lineagecell.com or follow the Company on Twitter @LineageCell.

Forward-Looking Statements

Lineage cautions you that all statements, other than statements of historical facts, contained in this press release, are forward-looking statements. Forward-looking statements, in some cases, can be identified by terms such as believe, may, will, estimate, continue, anticipate, design, intend, expect, could, plan, potential, predict, seek, should, would, contemplate, project, target, tend to, or the negative version of these words and similar expressions. Such statements include, but are not limited to, statements relating to Lineages objectives with respect to OpRegen. Forward-looking statements involve known and unknown risks, uncertainties and other factors that may cause Lineages actual results, performance or achievements to be materially different from future results, performance or achievements expressed or implied by the forward-looking statements in this press release, including risks and uncertainties inherent in Lineages business and other risks in Lineages filings with the Securities and Exchange Commission (the SEC). Lineages forward-looking statements are based upon its current expectations and involve assumptions that may never materialize or may prove to be incorrect. All forward-looking statements are expressly qualified in their entirety by these cautionary statements. Further information regarding these and other risks is included under the heading Risk Factors in Lineages periodic reports with the SEC, including Lineages Annual Report on Form 10-K filed with the SEC on March 12, 2020 and its other reports, which are available from the SECs website. You are cautioned not to place undue reliance on forward-looking statements, which speak only as of the date on which they were made. Lineage undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made, except as required by law.

View source version on businesswire.com: https://www.businesswire.com/news/home/20200506005264/en/

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Lineage Cell Therapeutics Reports New Data With OpRegen for the Treatment of Dry AMD With Geographic Atrophy - BioSpace

Orgenesis leads the charge at an exciting time for cell and gene therapy – Proactive Investors USA & Canada

OrgenesisInc () is a global biotech groupfocused on unlocking the potential of personalized therapies and closed processing systems through its cell and gene therapy platform.

The Germantown, Maryland-based companys aim is to provide life-changing treatments at the point-of-care to patients at low cost. It's Cell and Gene Therapy Biotech (CGT) platform has three key elements. The first revolves around point-of-care therapeutics, which consists of a pipeline of licensed cell and gene therapies and scientific knowhow. The second aspect relates to point-of-care technologies, which include a suite of in-licensed technologies engineered to create customized processing systems for affordable therapies.

Finally, the third component rests on a point-of care network, which is a collaborative, international ecosystem of leading research institutes and hospitals committed to supplying cell and gene therapies at the patient bedside. It is an intricate web of affiliated pre-clinical and clinical-stage biopharmaceutical companies, research institutions and hospitals through which Orgenesis is able to in-license technologies or advanced therapy medicinal products (ATMPs) and co-develop them with its partners.

On February 11, 202, Orgenesis completed the sale of subsidiary Masthercell Global Inc, a contract development manufacturing organization (CDMO), to Somerset, New Jersey-based Catalent Pharma Solutions, for around $127 million.

The successful sale has spotlighted Orgenesis boss Vered Caplans considerable leadership skills. She has since been named one of the top 20 inspirational leaders in the field of advanced medicine by The Medicine Maker, which creates an annual Power List of the worlds top drugmakers.

Caplan acquired Masthercell in March 2015 and grew the CDMO segment revenue from a run-rate of just $3 million to a run-rate of around $30 million at the end of 2019, reflecting a compound annual growth rate of 59% under her leadership, and a sale price of more than five times the initial purchase price of around $25 million. Caplan has indicated that she plans to use theMasthercell sale proceeds to grow the groups evolving point-of-care cell therapy business and develop advanced therapy medicinal products.

Orgenesis posted strong financial results for the year ended December 31, 2019, driven by its rapidly expanding point-of-care cellular therapy platform and new collaborations.

Gross profit jumped by 92% to $15 million in fiscal 2019, compared to $7.8 million for fiscal 2018. Gross profit soared 92% to $15 million in FY2019, compared to $7.8 million for FY 2018. The companys growth strategy is working as the point-of-care platform generated $3.1 million in sales, compared to nothing in FY 2018.

The company had cash and equivalents of $11.4 million at the end of 2019, which did not include the $127 million from the sale of its CDMO business in February.

Orgenesis is using theMasthercell sale proceeds to expand the companys point-of-care cell therapy business. Currently, the costs of cell and gene therapies are prohibitive, as illustrated by CAR-T therapies, which cost hundreds of thousands of dollars per patient, per year. To lower costs, Orgenesis is switching from a high-cost centralized manufacturing model to a localized point-of-care model.

The biotech is currently focused on therapies which span a wide range of treatments, such as cell-based immunotherapies, treatments for metabolic and neurodegenerative diseases and tissue regeneration.

At the start of April, Orgenesis teamed up with regenerative medicine and cell therapy firm RevaTis on a new joint venture to produce certain stem cells. The two firms plan to leverage Orgenesiss autologous Cell and Gene Therapy Biotech platform to advance clinical trials. Under the deal, RevaTis and Orgenesis will use the formers patented technique to obtain muscle-derived mesenchymal stem cells (mdMSC) as a source of exosomes and various other cellular products. Orgenesis and RevaTis are hoping to build on RevaTiss early success in animals to develop therapies and advance human trials using Orgenesiss expertise and point-of-care platform, which include automated systems, 3D printing and bioreactor technologies.

Meanwhile, Orgenesis and ExcellaBio have developed a breakthrough manufacturing process for so-called bioxomes, which are synthetically made exosomes or extracellular vesicles (EVs). The latter are what transfer DNA, RNA, and proteins to other cells, thereby altering the function of targeted cells. Until now, exosome/EV production has been based on conventional, complex and costly methods of ultracentrifugation or ultrafiltration. However, the two companies have demonstrated the scale up of Bioxomes through a proprietary technique, while generating consistent and repeatable results, including uniform particles sizes.

Orgenesisrecently completed the acquisition of Tamir Biotechnology and its broad spectrum antiviral platform, ranpirnase in a cash and stock deal for roughly $21 million. The company will use ranpirnase to target human papillomavirus (HPV), which causes genital warts. A topical version of ranpirnase was evaluated in Phase 1/2 clinical trials and it demonstrated a clear clinical effect and a good safety profile, the group noted.

Going forward, Orgenesis said it plans to move the program through a Phase 2b trial in the US. Additionally, the company will undergo a new clinical trial targeting anal dysplasia, a precusor to anal cancer driven by the HPV virus. Ranpirnase is a member of the superfamily of enzymes that cause the degradation of RNA and mediate biological activities, including cell death.

Orgenesis CEO Vered Caplan noted that the company has dramatically transformed since the beginning of 2020 helped by the Masthercell sale.

The sale was an important step in our strategy to leverage our unique capabilities that directly address the key challenges facing the cell and gene therapy industry, Caplan said in a statement.

Our goal is to transform the delivery of cell and gene therapy through our point-of-care therapeutics, technologies and network, thereby lowering costs and unlocking the power of cell and gene through a more decentralized and integrated approach, she added.

Contact the author Uttara Choudhury at[emailprotected]

Follow her onTwitter:@UttaraProactive

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End-use Industries of Stem Cell Assay Product Witness Unparalleled Slowdown Induced by Global Outbreak of COVID-361 – 3rd Watch News

In 2029, the Stem Cell Assay market is spectated to surpass ~US$ xx Mn/Bn with a CAGR of xx% over the forecast period. The Stem Cell Assay market clicked a value of ~US$ xx Mn/Bn in 2018. Region is expected to account for a significant market share, where the Stem Cell Assay market size is projected to inflate with a CAGR of xx% during the forecast period.

In the Stem Cell Assay market research study, 2018 is considered as the base year, and 2019-2029 is considered as the forecast period to predict the market size. Important regions emphasized in the report include region 1 (country 1, country2), region 2 (country 1, country2), and region 3 (country 1, country2).

The report on the Stem Cell Assay market provides a birds eye view of the current proceeding within the Stem Cell Assay market. Further, the report also takes into account the impact of the novel COVID-19 pandemic on the Stem Cell Assay market and offers a clear assessment of the projected market fluctuations during the forecast period.

Get Free Sample PDF (including COVID19 Impact Analysis, full TOC, Tables and Figures) of Market Report @ https://www.marketresearchhub.com/enquiry.php?type=S&repid=2543934&source=atm

Global Stem Cell Assay market report on the basis of market players

The report examines each Stem Cell Assay market player according to its market share, production footprint, and growth rate. SWOT analysis of the players (strengths, weaknesses, opportunities and threats) has been covered in this report. Further, the Stem Cell Assay market study depicts the recent launches, agreements, R&D projects, and business strategies of the market players including

The following manufacturers are covered:GE HealthcarePromega CorporationThermo Fisher ScientificMerck KGaACell BiolabsHemogenixStemcell TechnologiesBio-Rad LaboratoriesBio-Techne CorporationCellular Dynamics International

Segment by RegionsNorth AmericaEuropeChinaJapanSoutheast AsiaIndia

Segment by TypeDermatology Stem Cell AssayCardiovascular Stem Cell AssayCentral Nervous System Stem Cell AssayOncology Stem Cell AssayOther

Segment by ApplicationRegenerative Medicine & Therapy DevelopmentDrug Discovery and DevelopmentClinical ResearchOther

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Research Methodology of Stem Cell Assay Market Report

The global Stem Cell Assay market study covers the estimation size of the market both in terms of value (Mn/Bn USD) and volume (x units). Both top-down and bottom-up approaches have been used to calculate and authenticate the market size of the Stem Cell Assay market, and predict the scenario of various sub-markets in the overall market. Primary and secondary research has been thoroughly performed to analyze the prominent players and their market share in the Stem Cell Assay market. Further, all the numbers, segmentation, and shares have been gathered using authentic primary and secondary sources.

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End-use Industries of Stem Cell Assay Product Witness Unparalleled Slowdown Induced by Global Outbreak of COVID-361 - 3rd Watch News

DIABETES RESEARCH INSTITUTE FOUNDATION RECEIVES $3 MILLION GIFT FROM NORTH AMERICA’S BUILDING TRADES UNIONS TO FUND CELL THERAPY FOR COVID-19 -…

Miami, Fla., May 06, 2020 (GLOBE NEWSWIRE) -- The Diabetes Research Institute Foundation (DRIF) today announced a donation of $3 million from North Americas Building Trades Unions (NABTU), to support specific and urgent research on the use of umbilical cord mesenchymal stem cells to treat COVID-19-related inflammation. The generous gift will accelerate needed clinical trials during this challenging time.

On April 5, 2020, the U.S. Food and Drug Administration (FDA) approved mesenchymal stem cell treatments for seriously ill COVID-19 patients as an expanded access compassionate use. Subsequently, Dr. Camillo Ricordi, Director of the Diabetes Research Institute and Cell Transplant Program, opened the first-of-its-kind clinical trial on the use of umbilical cord mesenchymal stem cells (UC-MSC) to treat COVID-19-related inflammation. The critical study will assess the safety and efficacyincluding clinical outcomes and a variety of pulmonary, biochemical, and immunological testsof the intravenous administration of UC-MSCs in patients with severe cases of COVID-19.

NABTU and its members are proud to stand behind the work of Dr. Ricordi, the Diabetes Research Institute team and their partners around the world as they work to find a cure for diabetes and protect the lives of people affected by COVID-19, said NABTU President Sean McGarvey. As COVID-19 cases show severe implications for high-risk individuals and essential workers, Dr. Ricordis DRI research is essential to treating patients who are facing this life-threatening virus. Our work doesnt stop with this $3 million commitment; NABTU and our members have already begun organizing friends and supporters to raise the $30 million Dr. Ricordi says is required to effectively treat the millions of people who need it and will receive it at no cost to them or their families.

For 35 years, North Americas Building Trades Unions membership has supported the Diabetes Research Institute Foundation, and that longstanding commitment has had a significant impact on our quest for a cure for diabetes, said DRI Foundation CEO Sean Kramer. The continued generosity of NABTUs members over the years has helped fund and develop many clinical trials, including the DRIs prior research on UC-MSCs in patients with type 1 diabetes, but todays gift will truly help all Americans in the midst of this pandemic.

As it has been for decades, whenever we need them, the building trades stand in the breach for their countries and communities, Dr. Ricordi said. This generous donation is another example of how this amazing organization leads by example and how leadership and action can make a difference on the path of cures.

The relationship between DRIF and NABTU began with the Blueprint for Cure in 1984, an unprecedented campaign that supported funding of a state-of-the-art center where scientists would have every tool necessary to conduct research to find a cure for diabetes, a disease afflicting many union members and citizens in North America. Through this initiative, the members of the unions funded and built the Diabetes Research Institute facility in Miami, the most comprehensive diabetes research center in the world. Today, NABTU and its members have donated nearly $60 million to support the DRIFs efforts to find a cure for diabetes.

The DRIF is grateful for NABTUs dedication to our mission and cause, and we are especially appreciative of todays gift, Kramer added. Simultaneously, a therapy for COVID-19 and a cure for type one diabetes are within reach.

To learn more about the DRI mission and research for a cure, visit DiabetesResearch.org.

About the Diabetes Research Institute and Foundation

The mission of the Diabetes Research Institute Foundation is to provide the Diabetes Research Institute with the funding necessary to cure diabetes now. The Diabetes Research Institute at the University of Miami Miller School of Medicine leads the world in cure-focused research. As one of the largest and most comprehensive research centers dedicated to curing diabetes, the DRI is aggressively working to develop a biological cure by restoring natural insulin production in people living with the disease. Researchers have already shown that transplanted islet cells allow patients to live without the need for insulin therapy. Some study participants have maintained insulin independence for more than 10 years. The DRI is now building upon these promising outcomes through its BioHub strategy, a multi-pronged approach that addresses the major challenges standing in the way of a cure. For more information, please visit DiabetesResearch.org or call 800-321-3437.

About NABTU

North Americas Building Trades Unions is an alliance of 14 national and international unions in the building and construction industry that collectively represent over 3 million skilled craft professionals in the United States and Canada. Each year, our unions and our signatory contractor partners invest over $1.6 billion in private-sector money to fund and operate over 1,900 apprenticeship training and education facilities across North America that produce the safest, most highly trained, and productive, skilled craft workers found anywhere in the world. NABTU is dedicated to creating economic security and employment opportunities for its construction workers by safeguarding wage and benefits standards, promoting responsible private capital investments, investing in renowned apprenticeship and training, and creating pathways to the middle class for women, communities of color and military veterans in the construction industry. For more information, please visit http://www.nabtu.org, and to learn more about the building trades efforts during this pandemic, please follow the hashtag #buildingtradeswhateverittakes.

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FDA Approves Tabrecta, the First Targeted Drug for Patients with Non-Small Cell Lung Cancer and MET exon 14 – Curetoday.com

Tabrecta (capmatinib) will treat patients with metastatic non-small cell lung cancer that has a mutation leading to MET exon 14 skipping. The drug is the first targeted option for patients with lung cancer and this type of mutation.

Tabrecta is the first therapy approved by the FDA specifically to treat NSCLC with mutations that lead to epithelial-mesenchymal transition (EMT), which is MET exon 14 skipping.

Tabrecta is approved for patients who are new to treatment and also those who have received previous therapies, regardless of prior treatment type.

Along with the drug approval, the FDA gave the green light to a companion diagnostic, the FoundationOne CDx assay, which can identify these mutations in patients.

In epithelialmesenchymal transition(EMT), the cells that line an organ lose their polarity and ability to adhere to other cells, giving them the ability to invade tissues and organs. MET exon 14 skipping means that a segment of RNA that should prompt the production of a specific protein stops sending those messages.

The spread of cancer consists of a sequential series of events and MET exon 14 skipping is recognized as a critical event in this process, the FDA stated in a press release about the approval. Mutations leading to MET exon 14 skipping are found in 3% to 4% of patients with lung cancer, the agency stated.

Lung cancer is increasingly being divided into multiple subsets of molecularly defined populations with drugs being developed to target these specific groups, said Dr. Richard Pazdur, director of the FDAs Oncology Center of Excellence and acting director of the Office of Oncologic Diseases in the FDAs Center for Drug Evaluation and Research, in the release.

Taken orally, Tabrecta works by blocking a key protein that drives metastatic NSCLC in these patients. The FDA approved it based on the results of a clinical trial involving patients with NSCLC who had mutations leading to MET exon 14 skipping; their tumors did not express the proteins EGFR or ALK.

The evaluated study population included 28 patients who had never undergone treatment for NSCLC and 69 previously treated patients. The overall response rate (ORR; the percentage of participants who experienced a prespecified amount of tumor shrinkage) for the 28 participants was 68%, with 4% having a complete response and 64% having a partial response.

The ORR for the 69 participants was 41%, with all having a partial response. Of the responding participants who had never undergone treatment for NSCLC, 47% had a duration of response lasting 12 months or longer compared with 32.1% of the responding participants who had been previously treated.

Common side effects for patients taking Tabrecta included swelling of the legs, nausea, fatigue, vomiting, shortness of breath and decreased appetite.

Tabrecta may cause serious side effects including scarring or inflammation of the lungs. It may also cause damage to liver cells or harm a developing fetus or newborn baby. Patients may be more sensitive to sunlight when they take Tabrecta and should take precautions to cover their skin and use sunscreen.

Tabrecta was approved under theFDAs accelerated approval, breakthrough designation and priority review programs, which provide for a quicker review of drugs that treat serious or life-threatening diseases and represent a meaningful advantage over existing treatments.

Continued approval for this indication may be contingent upon verification of these results in confirmatory clinical trials.

Check back for what you need to know regarding this approval.

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FDA Approves Tabrecta, the First Targeted Drug for Patients with Non-Small Cell Lung Cancer and MET exon 14 - Curetoday.com