SAN FRANCISCO--(BUSINESS WIRE)--

Imago BioSciences, Inc., a clinical-stage biotechnology company developing innovative treatments for malignant and life-threatening diseases of the bone marrow, today announced the appointment of James D. Watson as Chief Business Officer.

James has a long history of success in biotech financing, business development, and commercial planning. That experience will guide the strategic direction and growth of Imago, said Hugh Young Rienhoff, Jr., M.D., Chief Executive Officer of Imago BioSciences. His experience in corporate development and financings will help ensure the advancement of bomedemstat (IMG-7289) through clinical development in myelofibrosis and other indications.

Mr. Watson most recently served as Chief Business Officer at Sigilon Therapeutics where he closed a $473 million strategic partnership with Eli Lilly for a treatment for Type 1 diabetes. Prior to Sigilon, Mr. Watson was Chief Business Officer for Alvine Pharmaceuticals and led strategy, corporate development, new product planning, and finance during which he closed a transaction giving AbbVie the right to acquire Alvine for $345 million. Previously, Mr. Watson was CEO of a San Francisco-based, boutique investment bank focused on mergers, acquisitions, partnering, and raising capital for life science companies.

I am excited to join Imago BioSciences at this important stage of growth, helping the company realize its full potential. Bomedemstat has great promise for the day-to-day management of myelofibrosis and it offers the additional possibility of altering the course of this disease. Furthermore, its broader myeloproliferative neoplasm platform and expertise in life-threatening diseases of the bone marrow represent an opportunity to address areas of high unmet clinical need and to build a valuable company. said Mr. Watson.

About Bomedemstat (IMG-7289)

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

In non-clinical studies, bomedemstat demonstrated robust in vivo efficacy as a single agent and in combination with other therapeutics across a range of myeloid malignancy models including the myeloproliferative neoplasms encompassing myelofibrosis, essential thrombocythemia and polycythemia vera. The U.S. Food and Drug Administration (FDA) has granted Fast Track designation to bomedemstat for the treatment of myelofibrosis which is currently being studied in an international Phase 2b study. Additional clinical studies in hematologic disorders will begin in 2020.

About Imago BioSciences

Imago BioSciences is a clinical-stage, private therapeutics company focused on malignant and life-threatening diseases of the bone marrow. The initial clinical focus is on myeloproliferative neoplasm (MPN) disorders including myelofibrosis, essential thrombocythemia and polycythemia vera. Investors in Imago include a fund managed by Blackstone Life Sciences, Frazier Healthcare Partners, Omega Funds, Amgen Ventures, MRL Ventures Fund, HighLight Capital, Pharmaron, Greenspring Associates and Xeraya Capital.

View source version on businesswire.com: https://www.businesswire.com/news/home/20191104005265/en/

Read more:
Imago BioSciences Further Expands Executive Team with Appointment of James D. Watson as Chief Business Officer - Yahoo Finance

Bone Marrow Stem Cells Comments Off on Imago BioSciences Further Expands Executive Team with Appointment of James D. Watson as Chief Business Officer – Yahoo Finance | November 4th, 2019

The proposal was nothing fancy. Shawn Russell arrived at the pub, placed a pint on the table in front of Sarah Hodgetts and asked her to marry him.

It was out of the blue, with no ring, nothing. And I thought: Yeah, thats a really good idea, we should just get married, says Sarah. Id been in love with him for ages.

Their story began seven years earlier, in 2009, also in a pub in north London. He was sitting in his flat cap, good Yorkshireman that he was, and was reading the sports pages. I had just got off the Tube after work. From my perspective, it was love at first sight, says Sarah.

But dating wasnt easy. Shawn, a boarding school-educated boy from an Army family, who had lost his mother to leukaemia when he was two, worked as a picture editor at The Telegraph. Although Sarah was down the road in Westminster, where she worked as a civil servant (and still does), their hours were long and their schedules largely incompatible. They managed just six months initially.

It was a disaster trying to date, so we ended up with a firework display of an argument and decided there was no way we could, says Sarah over coffee near her office.

Their split didnt last, however; they had far too much in common. Besides his ridiculous sense of humour, Shawn was so into news and politics, and I worked in politics, so we couldnt not communicate. We realised we were incredibly good friends, says Sarah. Plus, she adds with a smile, he was a very attractive, 6ft 4in blue-eyed man who I was completely smitten with.

Over time, they got back together in a non-committal way, sharing weekends when their busy lives permitted. It was during this period, three-and-a-half years ago, that Shawn, then 44, proposed. The following week, he moved in with Sarah, then 41, and her son Eddy, 10, from a previous relationship, in Kings Cross, and, like all newly engaged couples, they started making plans for their future. A pair of recovering workaholics, they were going to transform their lives.

See the original post here:
I lost my fianc to leukaemia, but in my dreams hes just working the night shift - Telegraph.co.uk

Bone Marrow Stem Cells Comments Off on I lost my fianc to leukaemia, but in my dreams hes just working the night shift – Telegraph.co.uk | November 3rd, 2019

MEDIA CONTACT

Available for logged-in reporters only

Newswise Three researchers at theEli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at UCLAhave received awards totaling more than $18 million from the California Institute for Regenerative Medicine, the states stem cell agency.

The recipients are Dr. Sophie Deng, professor of ophthalmology at the UCLA Stein Eye Institute;Yvonne Chen, a UCLA associate professor of microbiology, immunology and molecular genetics; and Dr. Caroline Kuo, a UCLA assistant clinical professor of pediatrics. The awards were announced at a CIRM meeting today.

Dengs four-year, $10.3 million award will fund a clinical trial for a blinding eye condition called limbal stem cell deficiency. Limbal stem cells are specialized stem cells in eye tissue that help maintain the health of the cornea. Because of genetic defects or injuries caused by infections, burns, surgeries or other factors, some people do not have enough limbal stem cells, which results in pain, corneal scarring and blindness.

The approach she is testing involves extracting a small number of limbal stem cells from a persons eye, multiplying them in a lab, and then transplanting them back into the eye, where they could regenerate the cornea and restore vision. The research will be conducted in collaboration with theUCLAUCI Alpha Stem Cell Clinic, a partnership between UCLA and UC Irvine.

The grants awarded to Chen and Kuo are for projects that are heading toward the FDAs investigational new drug application process, which is required by the agency before a phase 1 clinical trial the stage of testing that focuses on a treatments safety.

Chens two-year, $3.2 million award will fund efforts to create a more effectiveCAR T cell therapyfor multiple myeloma, a blood cancer that affects white blood cells. The research will evaluate a specialized form of CAR T therapy that simultaneously targets two markers, BCMA and CS1, commonly found on multiple myeloma cells. CAR T therapies that target BCMA alone have been effective in clinical trials, but the presence of BCMA on multiple myeloma cells is not uniform.

Previous research has shown that the marker CS1 is present in around 90% of multiple myeloma cells. A CAR T therapy that targets both markers could potentially help more patients and reduce the likelihood of a cancer relapse.

Kuos 2 1/2-year, $4.9 million award, will support the development of a stem cell gene therapy for a deadly immunodeficiency called X-linked hyper IgM syndrome, or XHIM.

The syndrome, which is caused by a mutation in the CD40LG gene, results in invasive infections of the liver, gastrointestinal tract and lungs. Currently, the only potential cure is a bone marrow transplant from a matched donor, which carries life-threatening risks and is often less effective for XHIM patients than patients with other forms of immune deficiency. Even with current treatments, only 30% of people with the syndrome live to age 30.

Kuo will evaluate a stem cell gene therapy that corrects the genetic mutation that causes XHIM. After removing blood-forming stem cells from a person with the syndrome, the therapy would use a genetic engineering technique called CRISPR to insert a correct copy of the affected gene into the DNA of the stem cells. The corrected blood-forming stem cells would be infused back into the patient, where they could regenerate a healthy immune system.

She will collaborate with Dr. Donald Kohn, a UCLA distinguished professor of microbiology, immunology and molecular genetics who has successfully treated two other immune deficiencies bubble baby disease and X-linked chronic granulomatous disease with a similar therapy.

Read more from the original source:
Three UCLA scientists receive grants totaling more than $18 million - Newswise

Bone Marrow Stem Cells Comments Off on Three UCLA scientists receive grants totaling more than $18 million – Newswise | October 31st, 2019

Ezer Mizion, the worlds largest Jewish bone marrow registry, will host its Evening of Heroes for the Teaneck, Bergenfield, and New Milford communities on Saturday, November 9, at Congregation Keter Torah in Teaneck.

The evening begins with a musical Havdalah and mini-concert by the chasidic superstar Shulem Lemmer, the first chasidic singer to sign with Universal Records. Then Ezer Mizion will introduce IDF heroes who defend the State of Israel and have saved lives with their stem cells.

A stem cell recipient will recount the day he received a call letting him know that Ezer Mizion had identified a stem cell match for him a match that saved his life. Bret Stephens, a New York Times Pulitzer Prize-winning columnist, and Nachum Segal will give a fireside chat about innovations from Israel, including the export of more than 60 percent of Ezer Mizions stem cell transplants.

Get The Jewish Standard Newsletter by email and never miss our top storiesFree Sign Up

There will be a swabbing station for people who meet the basic criteria for donations. Israeli wines and shuk foods will be served.

The program aims to bring awareness of the organizations role in saving hundreds of lives around the world every year with its growing bone marrow registry. It has more than 1 million potential stem cell donors, and more than 550,000 of these donors are from the IDF. There is no cost to attend the adults-only evening; RSVPs are requested. For more information, go to eveningofheroes.com; email Ezer Mizions national director of development, Ryan Hyman, at ryan@ezermizionusa.org or call him at (718) 853-8400, ext. 109.

Go here to see the original:
Ezer Mizion's Evening of Heroes is November 9 in Teaneck - The Jewish Standard

Bone Marrow Stem Cells Comments Off on Ezer Mizion’s Evening of Heroes is November 9 in Teaneck – The Jewish Standard | October 31st, 2019

TEL AVIV, Israel, Oct. 31, 2019 /PRNewswire/ -- BioLineRx Ltd. (NASDAQ: BLRX) (TASE: BLRX), a clinical-stage biopharmaceutical company focused on oncology, announced today that it will deliver the following poster presentations at the Society for Immunotherapy of Cancer(SITC) 34th Annual Meeting to take place November 6-10, 2019 at the Gaylord National Hotel & Convention Center in Baltimore, Maryland:

About BL-8040

BL-8040 is a short synthetic peptide that functions as a high-affinity best-in-class antagonist for CXCR4, a chemokine receptor over-expressed in many human cancers, where it has been shown to be correlated with poor prognosis, and plays a key role in tumor growth, invasion, angiogenesis, metastasis and therapeutic resistance. CXCR4 is also directly involved in the homing and retention of hematopoietic stem cells (HSCs) and various hematological malignant cells in the bone marrow.

In a number of clinical and pre-clinical studies, BL-8040 has shown a critical role in immune cell trafficking, tumor infiltration by immune effector T cells and reduction in immunosuppressive cells within the tumor niche, turning "cold" tumors, such as pancreatic cancer, into "hot" tumors (i.e., sensitizing them to immune check point inhibitors). BL-8040-mediated inhibition of the CXCR4-CXCL12 (SDF-1) axis has also shown robust mobilization of HSCs for transplantation in hematological malignancies.

BL-8040 was licensed by BioLineRx from Biokine Therapeutics and was previously developed under the name BKT-140.

About BioLineRx

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

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

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

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

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

or

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

View original content:http://www.prnewswire.com/news-releases/biolinerx-to-present-two-posters-at-the-society-for-immunotherapy-of-cancer-sitc-2019-300948943.html

SOURCE BioLineRx Ltd.

View original post here:
BioLineRx to Present Two Posters at the Society for Immunotherapy of Cancer (SITC) 2019 - P&T Community

Bone Marrow Stem Cells Comments Off on BioLineRx to Present Two Posters at the Society for Immunotherapy of Cancer (SITC) 2019 – P&T Community | October 31st, 2019

The National Institutes of Health (NIH) and the Bill & Melinda Gates Foundation will each invest $100 million over the next four years to speed the development of affordable gene therapies for sickle cell disease (SCD) and the human immunodeficiency virus (HIV) on a global scale.

This unprecedented collaboration focuses from the get-go on access, scalability and affordability of advanced gene-based strategies for sickle cell disease and HIV to make sure everybody, everywhere has the opportunity to be cured, not just those in high-income countries, said NIH Director Francis S. Collins, MD, PhD.

Seventy-five percent of babies born with SCD live in sub-Saharan Africa. It is hoped that experimental gene therapies would advance to clinical trials in the United States and relevant African countries within the next seven to 10 years, and that safe, effective, and inexpensive gene therapies be made available globally, including in low-resource settings where the cost and complexity of these therapies make them inaccessible to many.

In recent years, gene-based treatments have been groundbreaking for rare genetic disorders and infectious diseases, Trevor Mundel, MD, PhD, president of the global health program at the Bill & Melinda Gates Foundation said in a news release.

While these treatments are exciting, people in low- and middle-income countries do not have access to these breakthroughs. By working with the NIH and scientists across Africa, we aim to ensure these approaches will improve the lives of those most in need and bring the incredible promise of gene-based treatments to the world of public health, he added.

Hemoglobin is the protein in red blood cells that binds oxygen, allowing oxygen to be transported around the body. Mutations in the HBBgene, which encodes a component of hemoglobin, result in the formation of sickle hemoglobin that causes sickle cell anemia.

Currently, gene therapies for SCD involves altering the patients own hematopoietic stem cells (bone marrow cells that divide and specialize to produce blood cells including red blood cells). Genes are introduced into the cells using a modified, harmless virus (known as a viral vector). The cells are then transplanted back into the patient where they will produce healthy red blood cells. Gene therapy has an advantage over a bone marrow transplant, as it circumvents the complications associated with a bone marrow donation.

The first goal of the collaboration between the NIH and the Gates Foundation is to develop an easy-to-administer gene-based intervention to correct the mutations in the HBBgene or deliver a functional gene that will promote the production of normal levels of hemoglobin without the need to extract cells from patients and modify them in the lab before introducing the cells back. However, this strategy, known as in vivotreatment, requires the advancement of more efficient delivery systems that can deliver the gene therapy specifically to hematopoietic stem cells.

A second goal of the collaboration will be to work together with African partners and bring potential therapies to clinical trials.

Further research is required to understand the burden of SCD in sub-Saharan Africa and to screen newborns at high risk for the disease, a task that the National Heart, Lung and Blood Institute (NHLBI) has started to tackle by building the necessary infrastructure for clinical research.

The NIH and the Gates Foundation will help boost this infrastructure to allow point-of-care screening (for example, when infants receive vaccinations), and to initiate a standard of care. This will occur outside of the official collaboration.

Our excitement around this partnership rests not only in its ability to leverage the expertise in two organizations to reduce childhood mortality rates in low-resource countries, but to bring curative therapies for sickle cell disease and HIV to communities that have been severely burdened by these diseases for generations, said Gary H. Gibbons, MD, director of the NHLBI.

A persons health should not be limited by their geographic location, whether rural America or sub-Saharan Africa; harnessing the power of science is needed to transcend borders to improve health for all, he added.

Matshidiso Rebecca Moeti, the regional director for Africa at the World Health Organization said, We are losing too much of Africas future to sickle cell disease and HIV.

Beating these diseases will take new thinking and long-term commitment. Im very pleased to see the innovative collaboration announced today, which has a chance to help tackle two of Africas greatest public health challenges, Moeti added.

Patricia holds her Ph.D. in Cell Biology from University Nova de Lisboa, and has served as an author on several research projects and fellowships, as well as major grant applications for European Agencies. She also served as a PhD student research assistant in the Laboratory of Doctor David A. Fidock, Department of Microbiology & Immunology, Columbia University, New York.

Total Posts: 94

Margarida graduated with a BS in Health Sciences from the University of Lisbon and a MSc in Biotechnology from Instituto Superior Tcnico (IST-UL). She worked as a molecular biologist research associate at a Cambridge UK-based biotech company that discovers and develops therapeutic, fully human monoclonal antibodies.

View original post here:
SCD, HIV Gene Therapy Efforts Get $200M from NIH, Gates Foundation - Sickle Cell Anemia News

Bone Marrow Stem Cells Comments Off on SCD, HIV Gene Therapy Efforts Get $200M from NIH, Gates Foundation – Sickle Cell Anemia News | October 31st, 2019

Zion Market Research published a new 110+ pages industry researchCell Isolation Market by Product (Instruments and Consumables), by Cell Type (Animal and Human), by Cell Source (Adipose Tissue, Embryonic/Cord Blood Stem Cells, and Bone Marrow), by Technique (Surface Marker-Based Cell Isolation, Centrifugation-Based Cell Isolation, and Filtration-Based Cell Isolation), by Application (Cancer Research, Biomolecule Isolation, Tissue Regeneration & Regenerative Medicine, Stem Cell Research, In Vitro Diagnostics, and Others), and By End-User (Hospitals & Diagnostic Laboratories, Research Laboratories & Institutes, Biotechnology & Biopharmaceutical Companies, and Others): Global Industry Perspective, Comprehensive Analysis, and Forecast, 20182025.

TheGlobal Cell Isolation Market Is Expected To Reach Around USD 15.16 Billion By 2025complete outline is crystal clear penned down in the GlobalCell Isolation Marketresearch report such that not only an unskilled individual but also a professional can easily extrapolate the entire Cell Isolation Market within a few seconds.The research study covers research data which makes the document a handy resource for managers, analysts, industry experts, and other key people get ready-to-access and self-analyzed study along with TOC, graphs and tables to help understand the market size, share, trends, growth drivers and market opportunities and challenges.

Download FREE PDF Sample Brochure for more Industry Insights @CLICK HERE NOW

The Cell Isolation Market research report covers major industry player profiles that include:

Becton, Dickinson, and Company, Thermo Fisher Scientific, Inc., Merck KGaA, Beckman Coulter Inc., Terumo BCT, Bio-Rad Laboratories, Inc.

This report employs the SWOT analysis technique for the assessment of the development of the most remarkable market players. It additionally considers the latest upgrades while assessing the development of leading market players. Moreover, in the global Cell Isolation Market report, the key product categories of the global Cell Isolation Market are included. The report similarly demonstrates supportive data related to the dominant players in the market, for instance, product offerings, revenue, segmentation, and business synopsis. The global Cell Isolation Market is as well analyzed on the basis of numerous regions.

Download FREE PDF Sample for more Insights@CLICK HERE NOW

Global Cell Isolation Market: Regional Analysis

To understand the competitive landscape in the market, an analysis of Porters five forces model for the market has also been included. The study encompasses a market attractiveness analysis, wherein all segments are benchmarked based on their market size, growth rate, and general attractiveness. This report is prepared using data sourced from in-house databases, secondary and primary research team of industry experts.

Browse Press Release@CLICK HERE NOW

The report answers important questions that companies may have when operating in the Global Cell Isolation Market. Some of the questions are given below:

What is the current CAGR of the Global Cell Isolation Market?

Which product is expected to show the highest market growth?

Which application is projected to gain a lions share of the Global Cell Isolation Market?

Which region is foretold to create the most number of opportunities in the Global Cell Isolation Market?

Will there be any changes in market competition during the forecast period?

Which are the top players currently operating in the global market?

How will the market situation change in the coming years?

What are the common business tactics adopted by players?

What is the growth outlook of the Global Cell Isolation Market?

Also, Research Report Examines:

Browse for more Research Reports:http://fnfresearch.com/

Thanks for reading this article; you can also get individual chapter wise section or region wise report versions like North America, Europe or Asia.

Sorry! The Author has not filled his profile.

Originally posted here:
Cell Isolation Market ||Becton, Dickinson, and Company, Thermo Fisher Scientific, Inc., Merck KGaA - Industry News Info

Bone Marrow Stem Cells Comments Off on Cell Isolation Market ||Becton, Dickinson, and Company, Thermo Fisher Scientific, Inc., Merck KGaA – Industry News Info | October 31st, 2019

Cell Harvesting MarketReport has newly added to its massive repository. Different industry-specific methods have been used for analyzing the market carefully. The informative data has been inspected through primary and secondary research techniques. The global Cell Harvesting market has been analyzed by focusing on different verticals of the businesses such as market trends, regional outlook, competitive landscape, key players, business approaches, technologies, and standard operating procedures.An exclusiveCell Harvesting Marketresearch report contains a brief on those trends which may enable the companies operating into know to strategize and the current sector to their small enterprise expansion. The investigation report analyses the market size, industry share, growth, key sections, CAGR, and drivers.

Top Companies in the GlobalCell HarvestingMarket:PerkinElmer (US), Brandel (US), TOMTEC (US), Pall Corporation (Danaher), Connectorate (Switzerland), Scinomix (US), ADSTEC (Japan), Sartorius, Terumo Corporation.

Cell harvesting usually for use in cancer or other treatment. Usually the cells are removed from the patients own bone marrow. Stem cells can be harvested from the blood or bone marrow. Umbilical cords have been saved as a future source of stem cells for the baby.

https://www.marketinsightsreports.com/reports/05161234118/global-cell-harvesting-market-size-status-and-forecast-2019-2025/inquiry?mode=46

The market engineering comprises the structured, systematic and theoretically founded procedure of analyzing, designing, introducing and also quality assuring of markets as well as their legal framework regarding simultaneously their market mechanisms and trading rules, systems, platforms and media, and their business models.

This report segments the globalCell HarvestingMarket on the basis ofTypesare:ManualAutomated

On The basis OfApplication,the GlobalCell HarvestingMarket is Segmented into:BiopharmaceuticalStem Cell Research

https://www.marketinsightsreports.com/reports/05161234118/global-cell-harvesting-market-size-status-and-forecast-2019-2025/discount?mode=46

Geographically, this report is segmented into several key Regions, with production, consumption, revenue (million USD), and market share and growth rate ofCell HarvestingMarketthese regions, from 2018 to 2025 (forecast), covering

North America,Europe,China,Japan, Southeast Asia, India, North America(USA, Canada and Mexico)Europe(Germany, France, UK, Russia and Italy)Asia-Pacific(China, Japan, Korea, India and Southeast

-Comprehensive assessment of all opportunities and risk in the market. Cell Harvesting market recent innovations and major events.-Detailed study of business strategies for growth of the market-leading players.-Conclusive study about the growth plot of Cell Harvesting market for forthcoming years.-In-depth understanding of market-particular drivers, constraints and major micro markets.-Favourable impression inside vital technological and market latest trends striking the market.

https://www.marketinsightsreports.com/reports/05161234118/global-cell-harvesting-market-size-status-and-forecast-2019-2025?mode=46

MarketInsightsReportsprovides syndicated market research on industry verticals including Healthcare, Information and Communication Technology (ICT), Technology and Media, Chemicals, Materials, Energy, Heavy Industry, etc.MarketInsightsReportsprovides global and regional market intelligence coverage, a 360-degree market view which includes statistical forecasts, competitive landscape, detailed segmentation, key trends, and strategic recommendations.

Irfan Tamboli (Head of Sales) Market Insights ReportsPhone: + 1704 266 3234 | +91-750-707-8687sales@marketinsightsreports.com|irfan@marketinsightsreports.com

Excerpt from:
Cell Harvesting Market Global Strategies and Insight driven transformation 2019-2024 - The Chicago Sentinel

Bone Marrow Stem Cells Comments Off on Cell Harvesting Market Global Strategies and Insight driven transformation 2019-2024 – The Chicago Sentinel | October 31st, 2019

By Express News Service

KOCHI:Though the impending examinations, the study leave and the hartal called by the merchants association played spoilsport, nearly 525 youngsters came forward to register themselves as stem cell donors on Tuesday at a camp set up by Smilemakers of Cusat and DATRI of St Teresas College. The organisers were expecting around 1,000 registrations.

Next, we will be holding a camp at Lulu Mall on November 14, said Ramiz Rehman of Smilemakers. Not only students but also teachers and people from outside the campus came in to register. However, one big impediment is the lack of awareness about the stem cell donation process, he said.According to him, there is a common notion that bone marrow aspiration needs to be done to extract stem cells. But this is not the case. The stem cells are extracted from the blood. There is no drilling of bones happens, said Ramiz. According to him, stem cell transfusion is the only treatment that can save the lives of those suffering from leukaemia and thalassemia.

The programme has been organised for the benefit of not only the three siblings diagnosed with thalassemia major hailing from Mattancherry, but for the thousands of patients who have registered with DATRI. Since the possibility of obtaining a match is one in 20 lakh, there is a need to have a lot of people registering as fast as possible, he said. Speaking after inaugurating the registry drive, Poornima Jayaram, actor, said: Everyone will come as one to register themselves if they put themselves in the shoes of the recipient.

Follow this link:
Stem cell registry gets good response - The New Indian Express

Bone Marrow Stem Cells Comments Off on Stem cell registry gets good response – The New Indian Express | October 30th, 2019

WHEN Laura Farmer-Maia's daughter suddenly became clingy and unhappy, she initially brushed it off as nothing but "a phase".

And given little Beatriz was just three-years-old, the last thing to cross her mind was cancer.

16

Yet, months after doctors repeatedly dismissed the symptoms as clinginess, the diagnosis was confirmed - Beatriz had an aggressive childhood cancer known as a neuroblastoma.

The horrifying news came after Beatriz's father Tiago Maia refused to leave the hospital after discovering the potential diagnosis himself on Google.

Shocked, Laura, 39, and Tiago, 40, are now urging all parents to be vigilant and check their kids for signs of the disease.

The mum, who works in advertising, first suspected something was wrong last July, when Beatriz's behaviour dramatically changed.

16

16

She said: "Before she was diagnosed, Beatriz was quite naughty but when she reached two, she suddenly became clingy and picky with her food, and had a fever all the time.

"We took her to the GP who believed it was a virus and after recurrent visits they gave her some antibiotics to cover for a potential bacterial cause, which didnt have any effect.

"Beatriz started to complain that her legs hurt so we took her to A&E, where they did some more tests and still said it might be a virus.

"Its hard to get a diagnosis right when a child is too young to explain how theyre feeling, but in the back of our minds we knew it was something bad.

"We want to spread awareness of the difficulty of diagnosing cancer in young children - if your child doesnt seem right, you should push for further tests."

16

16

Tiago pushed the GP for more tests, and blood tests showed something was wrong, so Beatriz was sent urgently to hospital.

It was there medics finally discovered a lump above her kidney and diagnosed her with neuroblastoma in September last year.

The cancer is aggressive and has a 40 per cent chance of long-term survival.

Everything moved so quickly and we all felt frightened as they carried out the tests

Tiago, originally from Portugal, added: "At the hospital, they twice said it was likely to be a virus and I refused to leave until I saw a specialist.

"I waited for three hours until a more senior doctor was free, and then Beatriz was examined by different specialists who admitted her to do all kinds of tests and observations including X-rays and ultrasounds - it was the last one that confirmed there was a lump.

"When my fears from Google turned out to be true, it was very strange because even though my life had just flipped upside down, I was almost relieved to be right - it was weird and confusing."

16

16

Beatriz was referred straight to Great Ormond Street Hospital in London, where they carried out further tests including scans, blood tests and biopsies.

She began chemotherapy just a week after being diagnosed and underwent eight gruelling rounds of chemo over the next 18 months.

Doctors then carried out a stem cell transplant to regenerate bone marrow destroyed by high dose chemo, which meant Beatriz couldnt leave the hospital for eight weeks.

Tiago, a design director, said: "I was quite scared when Beatriz was diagnosed because my mum and dad had only recently died from cancer I thought of the worst.

16

16

16

"Everything moved so quickly and we all felt frightened as they carried out the tests.

"Doctors found that the cancer had spread across her body, so she began chemotherapy just weeks after being diagnosed.

"We were told the treatment would last 18 months which was a massive shock to us.

"A week after Beatriz started chemotherapy she massively improved, but it was tricky being in hospital at first.

"Now, she still has periods of discomfort but sometimes shes happy to be in hospital because she has toys and people to come and play with her."

16

16

Laura added: "The stem cell transplant was a hard time for us all because we had to spend a lot of time apart from our other daughter, Clara, six.

"Beatriz was diagnosed in Claras first week of school and it was difficult because that was supposed to be an exciting time for her."

After more scans and hopes of an all-clear, doctors found more metastatic growths still remaining in Beatrizs head, which meant that the cancer hadnt fully cleared up and she had relapsed.

The brave youngster is now undergoing immunotherapy and is due to start a six month medical trial on the NHS at Great Ormond Street Hospital, called the Beacon Trial.

16

What is neuroblastoma?

Neuroblastoma is a type of cancer that most commonly afflicts babies and young children.

The disease develops from special nerve cells, known as neuroblasts, which get left behind from the child's development in the womb.

It mostly begins in the sufferer's adrenal glands located above the kidneys but can occur in the nerve tissue that runs along the spinal cord in the neck, chest, abdomen or pelvis.

The vicious illness can then spread to other organs like the bone, bone marrow, lymph nodes and skin.

Neuroblastoma afflicts around 100 children a year in the UK but the cause of the disease is still not known.

Its symptoms can include:

It is uncertain whether the trial will work and, even if Beatriz goes into remission, relapse rates are high but her parents are determined to do everything they can to stop the cancer from returning.

Laura and Tiago are now trying to raise 200,000 to help get their daughter into remission or to keep the cancer away if her treatment goes well.

The money is hoped to go towards further treatment, or if Beatriz gets the all-clear, a special vaccine in New York which helps keep the disease away.

Laura said: "After the stem cell transplant, the end was almost in sight but then she relapsed.

16

16

TUMOUR SHOCK My 'baby brain' turned out to be incurable tumour the size of an orange

Exclusive

TWIST OF FATE I screamed with joy at getting cancer all clear two weeks later it came back

Exclusive

KILLER CUTS Cancer campaigns axed despite UK survival rates among worst in western Europe

Comment

DEBORAH JAMES You CAN prevent cancer - two-min screening tests save lives, so why wait?

TILL DEATH DO US PART Dying bride marries love of life knowing they have just months left

Exclusive

SNAP HAPPY Woman hails 'life-changing' museum after thermal camera spots breast cancer

Exclusive

DEATH DO US PART I said 'I do' as mum lay in a coma fighting cancer - she died days later

GUT FEELING Bowel and pancreatic cancer cases soar, experts warn - signs you need to know

KRIS HALLENGA CoppaFeel! founder on the importance of positivity when dealing with cancer

"Were afraid that the cancer will get worse and worse and want to raise money to help get her into remission the ideal outcome is that the trial works and clears the disease.

"Luckily, compared to other two-year-olds, Beatriz has suffered less side effects with treatment and despite losing her curly hair shes powering through."

You can donate on Beatriz's JustGiving page here.

16

Read the original here:
Docs said our toddler was just clingy but we learned the truth on Google it was cancer - The Sun

Bone Marrow Stem Cells Comments Off on Docs said our toddler was just clingy but we learned the truth on Google it was cancer – The Sun | October 30th, 2019

MULTIPLE sclerosis campaigners have hailed a huge step forward for patients in Scotland after a stem cell therapy was recommended for use on the NHS for the first time.

Haematopoietic stem cell transplantation (HSCT) has been described as a game-changer for MS after an international clinical trial showed that it could reboot patients immune systems and halt the progress of the disease.

Some patients who had been in wheelchairs prior to treatment said their condition improved so dramatically it was like they had never been diagnosed with MS.

READ MORE: Scots MS patients 'missing out' on pioneering stem cell treatment available in England

The Scottish Health Technologies Group (SHTG) said there is now sufficient evidence for it to recommend making HSCT available on the NHS in Scotland to MS patients who have the relapsing-remitting form of the disease, and who were not responding to drug treatments.

Iain Robertson, chairman of the SHTG, said: Our committee members were able to advise that this treatment should be considered for those with this particular type of MS who have not responded to treatment with disease-modifying therapies.

We hope that our advice will be of use in helping decide the best course of treatment for these patients.

The SHTG also stressed that patients must be made aware of the demands, risks and uncertainties of the treatment, which uses chemotherapy to wipe out patients' 'faulty' immune systems before replenishing it with a transplant of stem cells harvested from their own bone marrow.

It puts patients at high risk from infections, which can be fatal, but the theory is that the treatment works by enabling patients to 'reset' their immune system to stop it attacking the central nervous system as is the case in MS.

READ MORE: Anger of Scots MS patients travelling abroad for stem cell therapy available to some on NHS England

HSCT is not considered an effective treatment for patients with the progressive form of MS, however, as stem cells cannot regrow nerves or repair damaged myelin - the protective sheath which coats nerves.

It will also be unavailable to patients with relapsing-remitting MS who no longer show signs of inflammation on an MRI brain scan.

Scotland has one of the highest rates of MS in the world, but until now Scottish patients seeking HSCT have had to travel overseas to Mexico, Russia and Israel and bankroll their own private treatment at a cost of around 40-60,000.

It has also been available privately in London since 2017, but with a 100,000 price tag.

A small number of MS patients in England have been able to access the treatment on the NHS, however, because there are clinical trials into HSCT taking place at NHS hospitals in Sheffield and London.

Morna Simpkins, director of MS Society Scotland, said: The decision from SHTG to approve HSCT for the treatment of MS is good news and could help in the development of a clear pathway, for people who could potentially benefit, to access it.

We will push to ensure that this decision leads to real change for people with MS by continuing to engage with other groups to offer the treatments, including HSCT, which are right for them.

READ MORE: Stem cells help mother with MS make 'remarkable' recovery

The SHTG said eligible patients must have equal access to the procedures regardless of where they live, but it is unlikely all health boards will be able to provide it.

The MS Society wants a centre, or centres, of excellence set up where patients from across Scotland can be referred.

Lucy Clarke from the Scottish HSCT Network said the recommendation was "a huge step forward" for people in Scotland living with MS.

Ms Clarke underwent HSCT in Russia and credits it with substantially reversing her disability.

She added: This important decision supports HSCT as a treatment option where other treatments have failed. We will continue to push so that this treatment is available to people in Scotland who need it.

A Scottish Government spokeswoman said: We are grateful to the Scottish Health Technologies Group for this important work.

"NHS Boards are expected to consider their advice on technologies in the planning and provision of its services and clinicians are expected to follow their professional judgement, working within the management structure of their Board.

We will work closely with MS Society Scotland, other third sector bodies and the clinical community to consider what the Technologies Groups findings means for provision in Scotland, including the information that needs to be available to people about eligibility and risks.

Go here to see the original:
Stem cell therapy approved for MS patients in Scotland - HeraldScotland

Bone Marrow Stem Cells Comments Off on Stem cell therapy approved for MS patients in Scotland – HeraldScotland | October 30th, 2019

DURHAM, N.C., Oct. 29, 2019 (GLOBE NEWSWIRE) -- Chimerix, Inc. (CMRX), a biopharmaceutical company focused on accelerating the development of innovative medicines to treat patients with cancer and other serious diseases, today announced that it will host a live conference call and audio webcast on Tuesday, November 5, 2019 at 8:30 a.m. ET to report financial results for the third quarter ended September 30, 2019, and to provide an operational update.

To access the live conference call, please dial (877) 354-4056 (domestic) or (678) 809-1043 (international) at least five minutes prior to the start time, and refer to conference ID 1693898. A live audio webcast of the call will also be available on the Investors section of the Company's website, http://www.chimerix.com. An archived webcast will be available on the Chimerix website approximately two hours after the event.

AboutChimerix

Chimerixis a development-stage biopharmaceutical company dedicated to accelerating the advancement of innovative medicines that make a meaningful impact in the lives of patients living with cancer and other serious diseases. The two clinical-stage development programs are dociparstat sodium (DSTAT) and brincidofovir (BCV).

Dociparstat sodium is a glycosaminoglycan biologic derived from porcine heparin that has low anticoagulant activity but retains the ability to inhibit activities of several key proteins implicated in the retention and viability of AML blasts and leukemic stem cells in the bone marrow during chemotherapy (e.g., CXCL12, selectins, HMGB1). Mobilization of AML blasts and leukemic stem cells from the bone marrow has been associated with enhanced chemosensitivity and may be a primary mechanism accounting for the observed increases in EFS and OS in Phase 2 with DSTAT versus placebo. Randomized Phase 2 data suggests that DSTAT may also accelerate platelet recovery post chemotherapy via inhibition of platelet factor 4, a negative regulator of platelet production that impairs platelet recovery following chemotherapy. BCV is a lipid conjugate DNA polymerase inhibitor in development as a medical countermeasure for smallpox.For further information, please visit the Chimerix website,www.chimerix.com

CONTACT:

Investor Relations:Michelle LaSpaluto919-972-7115ir@chimerix.com

Will OConnorStern Investor Relations212-362-1200will@sternir.com

See original here:
Chimerix to Announce Third Quarter 2019 Financial Results and Provide an Operational Update on November 5, 2019 - Yahoo Finance

Bone Marrow Stem Cells Comments Off on Chimerix to Announce Third Quarter 2019 Financial Results and Provide an Operational Update on November 5, 2019 – Yahoo Finance | October 30th, 2019

The new horror flick on Netflix, Eli, released just in time for Halloween, borrows from The Exorcist and Rosemarys Baby, with touches of The Shining. And it all takes place in what looks like Downton Abbey with the cleaning staff gone.

Eli works; its scary. But the set-up using gene therapy gone awry is unfortunate, superfluous, and even offensive. (Beware, spoilers ahead)

The film opens with 11-year-old Eli dreaming about being able to go outside without his hazmat suit and breathing without his skin reddening and blistering. He awakens and hes inside, in a bubble.

David was diagnosed, four years earlier, with a rare formof severe combined immune deficiency (SCID). It slashes his ability to make the antibodies that protect against infection, unleashes inflammation that reddens his skin, while at the same time turns his immune system against his own tissues, an autoimmune response.

The parents, caring Rose and weirdo Paul, bundle Eli up to take him to a doctor whos going to cure him with a new treatment. Once at the supposedly clean Downton Abbey haunted house, Eli has a decontamination shower.

When the boy meets the doc, she explains that his immune system makes too many bad immunoglobulins, using that word instead of antibodies because it sounds more technical.Eli quickly responds, spouting out that he has mutations in the RAG1 and RAG2 genes (recombination-activating genes).

Elis body cant make the enzymes that mix and match antibody parts, and the proportions of a bunch of immune system cells and proteins go out of whack. His condition is also called Omenn syndrome.

Dr. Horn has two nurses, and all three of them wear purple uniforms.

Good news! Dr. Horn will administer viral gene therapy! I will make you better, like my other patients, she assures the boy.

Rose gingerly begins to unwrap the blue layers that encase her son, bending down and looking like Laura Dern examining dino poo in Jurassic Park or Princess Leia releasing the hologram from R2D2. Mom and boy can finally hug!!!

At night, the house creaks. Eli wanders the spooky halls, glimpsing kids in the windows, mirrors, and reflections, including ghostly girls who look like the twins at the end of the hallway in The Shining.

A redheaded girl outside, Sadie Sink, apparently escaped from playing Max on Stranger Things, seems real.

Im allergic to the world, Eli tells her. Not exactly.

The next morning, Dr. Horn blames Elis ghost sightings on a side effect from immunosuppressants. Why is she trying to suppress an immune system already so impaired?

Next Eli, who looks so much like Tom Petty that I expected him to shriek I Wont Back Down, is strapped down to a table with a contraption holding his head in place, as Dr. Frankenstein asks her nurses to take a reading.

Dr. Horn at first seems to have gotten the basic idea of gene therapy correct: introducing a working copy of the mutant gene aboard viruses into stem cells from bone marrow. And poof! Like a magic trick! itll work, she proclaims.

She proceeds to extract a hunk of pinkish gunk after drilling into a bone, as the immobilized boy twists and grimaces on the table. Satisfied, the doc plops the glob into a Petri dish.

It burns! Eli shrieks.

That means its working, replies the doc. Within seconds, the doctored viruses have apparently hit their targets.

Then Eli awakens. It all seems a dream, but its foreshadowing.

A ghost appears in a bloody nightgown.

The house breathes at night.

A scrawny, dagger-nailed hand grabs Eli and the apparition turns into his father.

When Eli writes his name on a window, the letters rearrange to spell Lie, like Redrum becoming Murder in the mirror in The Shining. Later on, with the E written like a 3, Eli scratched into various furniture surfaces in the house becomes 317317317. What can it mean?

When Eli reports these events, Dr. Horn barks, Its the medication, as if sophisticated gene therapy has suddenly become as mundane as a tab of Tylenol. Shell have to lower the dosage because the second of the three treatments is coming up.

Treatment 2 is indeed brutal. Eli is held in a contraption like the one Hannibal Lecter wears to keep him from eating people and his head bolted like hes Frankenstein.

Were confident that the gene therapy virus is correcting the mutation, Dr. Horn declares, adding that this will burn a little bit, as she presumably delivers more.

When Eli turns red and screams, she assures him that this is supposed to happen. The virus is penetrating the blood-brain barrier, as if said barrier is a superhighway requiring that the bolts hold his head still.

I was speechless.

Barrier refers to the blood vessels in the brain that are closed to large molecules, which keeps toxins out. A widely-used gene therapy vector, AAV9 (adeno-associated virus 9), has been known for a decade to naturally cross the barrier. And that doesnt require torture hardware.

Heres a photo of one of the kids I write about receiving AAV9 gene therapy for a rare neurological disease through an intravenous delivery in her hand!

The doc then attributes Elis reaction to his body initially rejecting the new cells, like any transplant. But if his gene therapy consists of viruses traipsing across the blood-brain barrier, where did cells suddenly come from? Is it the doctoring of stem cells from bone marrow that was in Elis dream, or delivering viruses into the bloodstream?

Elis nocturnal adventures continue. Hes pushed and pulled from unseen forces as the floor turns transparent, revealing scary medical people. As he keeps bellowing the doc orders Haldol and his mom pushes Valium.

The mysterious 317 opens a key pad to an inner sanctum, which looks like the set of the second Indiana Jones film. We see insects alighting, so the place was never a clean room after all.

Eli finds a notebook with case histories of the past patients and the pieces start to fit. Perry. Agnes. Lucas.

After treatment 2, the kids eyes look haunted, their complexions gray, like Eli. After treatment 3, their heads exploded.

Then the religion clues start to fall out.

Eli discovers a photo of nuns that includes his medical team. A huge iron cross sheaths a dagger. The surgical table with Eli across it resembles Christ on the cross.

One reviewer posits that the plot is about gay conversion, pointing to a scene in which Eli literally crawls out of a closet to tell his parents the truth.

The action speeds up and twists as treatment 3 looms.

Dr. Horn dons religious garb, makes the sign of the cross, flings holy water, and babbles about Jesus and the archangel. The boy, having discovered the medical records, has become a liability.

I thought I could cure Eli. The gene therapy would have worked, if he wasnt so strong. But he cant leave here! the enraged doc yells.

But when Eli is tied down and Dr. Crazy is coming at him with the dagger pulled from the cross, he suddenly summons his inner Regan MacNeil (from The Exorcist) and stops the knife in mid-air, turns it around, and forces the doctor to stab herself. She mutters may you find peace and forgiveness in the name of the Lord, channeling Father Damien KarrasThe power of Christ compels you! as he attempts to exorcise Regan.

With the plunging of the dagger, Eli, red-eyed and screaming, rips off his restraints. His parents are thrown to the floor while the nurses and the doc, somehow still living with the dagger in her chest, try to leave.

But Eli, like Anthony in the cornfield episode of the Twilight Zone, points at them and they turn in unison and then elevate, like Regan rising from her bed. The purple ones then float around the room in an eerie circle emanating an unearthly blue glow, as if theyre on one of those centripetal force amusement park rides.

A conspiracy revealed

It turns out that all are in on whats happening, even the nice-seeming mom. And Eli realizes hes never been sick.

What has she been putting inside me? What have you been putting inside me? he shrieks at his parents, conjuring images of Rosemarysdevil spawn.

At that the nurses and doc suddenly flip upside down, the horror equivalent of Regans rotating head, and slam to the floor.

What am I?

Our son.

Eli sets the nurses and doc on fire.

Are you my dad?

I prayed every day! answers dad.

Prayed to whom? the boy bellows.

The Lord didnt answer me, but your father did, Rose utters mysteriously.

And we know.

Eli never had a SCID. Its a twist on Munchausen Syndrome by Proxy, the cause of his symptoms the holy water that mom and then doc sprinkled on him.

But the ultimate cause? Dad is the devil. At that realization, Eli makes his dads head explode.

The other kids, whose bodies indeed turn up, were Elis half-siblings, even Haley. Dad the Devil got around.

Eli is fun, fast and scary. But why did the writers have to tarnish gene therapy? Why use a genetic disease at all? And especially an ultra rare one? I cant help but wonder what motivated the writers to do this.

Ive devoted the past decade to learning about families who have rare genetic diseases and have kids who have had, or wish they could have, gene therapy, writing about them, and accompanying some of them on their journeys.

In addition to my posts here and at my blog DNA Science, I wrote the only book on gene therapy, The Forever Fix, which chronicles the efforts of a few families. The first gene therapy was FDA-approved in late 2017. The technology has indeed been like the mythical phoenix bird, arising from the ashes.

For the families, I resent the use of gene therapy as a plot point.

After viewing the film the other night, I did a final Facebook check. The first thing that popped up: a photo of an exquisite child, on a page for families dealing with Sanfilippo syndrome, a devastating neurological condition.

The boy was now free of the cruel disease, free to be at peace. He was 11. Elis age.

Genetic disease, and especially attempts to treat it, shouldnt be the stuff of horror films.

Ricki Lewis is the GLPs senior contributing writer focusing on gene therapy and gene editing. She has a PhD in genetics and is a genetic counselor, science writer and author of The Forever Fix: Gene Therapy and the Boy Who Saved It, the only popular book about gene therapy. BIO. Follow her at her website or Twitter @rickilewis

View post:
Viewpoint: Netflix's new horror movie 'Eli' is a fright. But why did they have to 'tarnish gene therapy'? - Genetic Literacy Project

Bone Marrow Stem Cells Comments Off on Viewpoint: Netflix’s new horror movie ‘Eli’ is a fright. But why did they have to ‘tarnish gene therapy’? – Genetic Literacy Project | October 30th, 2019

Gene therapy is a promising therapeutic procedure for genetic disorders or diseases in which defective genes are corrected, replaced, or inactivated.

In the case of adrenoleukodystrophy (ALD) a genetic disorder caused by mutations in the ABCD1 gene that damages the myelin sheath around nerve cells gene therapy may benefit patients prior to the onset, or during the early stages, of the disease by stopping the progression of demyelination. However, the therapy cannot be beneficial after the disease has worsened significantly.

Gene therapy works by introducing the correct gene sequence into cells. Since genetic material cannot enter the cell on its own, the correct gene sequence needs to be delivered using a vector. This vector can be a modified virus that has been engineered to remove its pathogenic genetic material so that it cannot cause disease, but is still able to transfer the correct gene sequence to the host cell.

The vector can be directly injected into the patients body or into host cells grown in the laboratory and then transplanted back into the patient. Upon successful viral transfer, the host cell should be able to produce the functional protein.

In ALD, the clinician first takes out the patients own stem cells (autologous) and then inserts the correctABCD1 gene sequence into these cells using a viral vector in the laboratory. The corrected stem cells that are able to produce the functional ALD protein are then implanted back into the patients body so they may develop into nerve cells in the brain. Since the patients own cells are being used, there are fewer risks than when donor stem cells are used.

Lenti-D,an investigational gene therapy developed by Bluebird Biois currently being studied in a Phase 2/3 clinical trial (NCT01896102) in the U.S., the U.K., and France. The study aims to evaluate the safety and effectiveness of Lenti-D in boys, up to 17 years old who havecerebral adrenoleukodystrophy (CALD). Based on the preliminary data from this study,the U.S. Food and Drug Administration (FDA)designated Lenti-D a breakthrough therapy for the treatment of CALD in May 2018.

A Phase 1/2 clinical trial (NCT02559830) is recruiting patients with ALD at the Shenzhen Second Peoples Hospital in Guangdong, China. The study aims to assess the safety and effectiveness of transplanting patient-derived bone marrow stem cells, which have been genetically-corrected using a lentiviral vector, for the treatment of ALD.

Another Phase 1/2 clinical trial (NCT03727555) at the Shenzhen Geno-Immune Medical Institute also in Guangdong, China is recruiting 10 patients with ALD. The study aims to evaluate the safety and effectiveness of a lentiviral vector carrying the healthy ABCD1 gene (TYF-ABCD1) injected directly into the patients brain for the treatment of ALD.

***

Adrenoleukodystrophy News is strictly a news and information website about the disease. It does not provide medical advice, diagnosis or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or other qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website.

Sorry! The Author has not filled his profile.

Total Posts: 0

zge has a MSc. in Molecular Genetics from the University of Leicester and a PhD in Developmental Biology from Queen Mary University of London. She worked as a Post-doctoral Research Associate at the University of Leicester for six years in the field of Behavioural Neurology before moving into science communication. She worked as the Research Communication Officer at a London based charity for almost two years.

More:
Gene Therapy - Adrenoleukodystrophy News

Bone Marrow Stem Cells Comments Off on Gene Therapy – Adrenoleukodystrophy News | October 28th, 2019

Still to be determined is how hospitals and other health care facilities will be reimbursed for the therapy and whether patients will have access to the therapy under health care proposals such as Medicare for All or a so-called public option.

One of the most promising cancer treatments to come along in years, CAR-T cell therapy uses the bodys own immune system to attack and kill cancer cells. The treatment involves bioengineering T cells, a white blood cell that fights foreign substances in the body, and equipping them with new Chimeric Antigen Receptors that target cancer cells.

CAR-T cell therapy has been approved by the U.S. Food and Drug Administration (FDA) for children with leukemia and adults with advanced lymphoma. The therapy is typically used alongside other, more traditional treatments such as surgery, chemotherapy, and radiation. Its use in combatting other forms of cancer is pending with the FDA, an agency known for its slow approval process.

Hefty Price Tag

There are currently two approved CAR-T treatments: Novartis Kymriah (tisagenlecleucel) and Gileads Yescarta (axicabtagene).

Like most newly introduced cutting-edge treatments, the two products come with a hefty price tag. A course of treatment of Kymriah costs $475,000 for pediatric and young adult patients with leukemia, and both are priced at $373,000 to treat lymphoma in adults, according tobiopharma.com. Under Centers for Medicare & Medicaid Services (CMS) regulations published in August, Medicare will reimburse hospitals for 65 percent of the treatments cost, or about $242,000, through Part B.

Although hospitals will likely welcome Medicares financial commitment, there still remains a sizable gap. Further complicating reimbursement is the lack of a separate Medicare billing code for CAR-T treatment, which will be handled via codes for bone marrow and stem cell transplants until a CAR-T billing code is developed, which could take up to three years.

CMS worked closely with the FDA and the National Cancer Institute in developing the new regulations, a time-consuming process rooted in the complexities of developing a reimbursement scheme and overseeing an innovative and evolving therapy.

At a July 31 Heritage Foundation panel discussion on Medicare for All, CMS Administrator Seema Verma discussed the challenges government programs face in approving innovative treatments.

Much of the problem is when Congress says you can cover durable medical treatment, supplies, and drugs, said Verma. Sounded great when they wrote that law 30 to 40 years ago but doesnt make sense in todays environment. All of these new treatments are coming out, and they dont fit nicely into the way the law has been constructed, and it creates problems for the agency.

Bonner R. Cohen,Ph.D.,(bcohen@nationalcenter.org)is a senior fellow at the National Center for Public Policy Research and a senior policy analyst with the Committee for a Constructive Tomorrow (CFACT).

Read the original post:
Medicare Coverage of CAR-T Cell Therapy Raises New Questions - The Heartland Institute

Bone Marrow Stem Cells Comments Off on Medicare Coverage of CAR-T Cell Therapy Raises New Questions – The Heartland Institute | October 28th, 2019

Patients with ultra-rare bone marrow disease are set to benefit from 1.15m grant from LifeArc and The Aplastic Anaemia Trust. The grant will support researchers from Kings College London and Kings College Hospital to test a personalised treatment for aplastic anaemia patients who have not responded to available therapies

The grant has been awarded to investigate the potential of a novel type of personalised cellular therapy to reverse the ultra-rare condition aplastic anaemia (AA). The results of this research could give new hope to people living with a severe, life-limiting form of this condition.

The grant will fund a clinical trial to investigate the safety and efficacy of using a patients own T-reg cells to restore the blood-making function of the bone marrow. This follows laboratory-based research from the team of scientists where T-reg cells from a patients own blood were collected, selected for activity and multiplied. In a test tube, these cells prevented the immune system from attacking the patients bone marrow stem cells.

AA is an ultra-rare life-threatening illness caused by the bone marrow failing to make enough of all three types of blood cells red blood cells, white blood cells and platelets. Only around 100-150 people in UK are diagnosed per year, affecting all ages but most commonly people between the ages of 10 to 20 years old and those over the age of 60 years.

People with the illness are at greater risk of infections, bleeding, and can experience extreme fatigue, which leaves them unable to carry out simple daily tasks that most people take for granted. Around one in three patients with severe AA fail to respond to existing drug treatments and the other option a bone marrow transplant is reliant on finding a suitable donor, requires life-long treatment with immunosuppression therapy and is unsuccessful in one in three people.

The trial at Kings College London and Kings College Hospital will run for three years and aims to recruit nine patients. A blood sample of the patients T-reg cells will be extracted, purified and grown in the lab before being given back to them in a higher concentration. As patients with AA are more susceptible to infection, this personalised treatment approach is more likely to avoid the risk of severe infection and inflammation.

Professor Ghulam Mufti, Department of Haematological Medicine at Kings College London and Kings College Hospital, and lead study investigator said: For patients with this ultra-rare disease, were looking for the first time at a personalised medicine approach where their own immune cells could be used to alter their disease. In AA there is a reduction in the number of T-regs and most of the ones that the AA patients do have are non-functional. Weve seen success in the laboratory by selecting and bolstering the number of functional T-reg cells. Now, with funding from LifeArc and the AAT, we can investigate the potential of this approach in treating AA patients who currently have very limited treatment options.

Dr Catriona Crombie, LifeArcs Head of Philanthropic Fund explained why the charity had approved the funding: LifeArc set up the Philanthropic fund to support translational research into rare diseases, where there is less interest from commercial organisations. Patients with AA can have limited treatment options; this opportunity with Kings College London, Kings College Hospital and the AAT has the potential to transform the lives of patients living with a severe form of the disease.

Grazina Berry, Chief Executive of the AAT said: AA can severely impact a persons quality of life. Through AATs close work with Kings College London and Kings College Hospital as a specialist centre of clinical care and research in AA, we identified the project with the most potential to directly benefit patients who are currently at a loss for solutions. We are delighted to have partnered with LifeArc and Kings College London and Kings College Hospital to progress this ground-breaking work, which could potentially enable people living with severe AA to once again lead a normal life.

Read more from the original source:
New funding to test personalised treatment for aplastic anaemia - Pharmafield

Bone Marrow Stem Cells Comments Off on New funding to test personalised treatment for aplastic anaemia – Pharmafield | October 26th, 2019

A gene therapy for a rare blood disorder has shown what the manufacturer calls the first evidence of long-term improvement associated with the disease.

New York-based Rocket Pharmaceuticals said Thursday that it had presented long-term follow-up data from the Phase I/II study of RP-L102, its gene therapy for Fanconi anemia, at the annual congress of the European Society of Cell and Gene Therapy in Barcelona, Spain. The company said it represented the first evidence of long-term improvement and stabilization in blood counts and durable mosaicism among patients who received the therapy without the use of the conditioning regimens normally used for allogeneic stem cell transplants, which the company calls Process A.

Shares of Rocket were up slightly on the Nasdaq following the news. RP-L102 is a lentiviral vector-based gene therapy. Most other gene therapies in development, and both of the currently marketed ones Spark Therapeutics Luxturna (voretigene neparvovec-rzyl) and Novartis Zolgensma (onasemnogene abeparvovec-xioi) are adeno-associated viral vector-based.

According to the data, representing four of nine patients, there were improved blood counts and long-term bone marrow mitomycin C (MMC) resistance, thereby indicating durable phenotypic correction. The data met or exceeded a 10 percent threshold that the company said the Food and Drug Administration and European Medicines Agency had agreed to for its upcoming Phase II registration study, for which it plans to start enrolling patients by the end of the year.

FA is a rare, genetic bone marrow failure disorder, half of whose patients are diagnosed before the age of 10, while about 10 percent of patients are diagnosed as adults, according to the National Organization for Rare Disorders. It is often associated with progressive deficiency of production of red and white blood cells and platelets in the bone marrow and can eventually lead to certain solid and liquid tumor cancers. It occurs in 1-in-136,000 births and is more common among Ashkenazi Jews, Spanish Roma and black South Africans.

These results indicate the feasibility of engraftment in FA patients using autologous, gene corrected [hematopoietic stem cells] in the absence of any conditioning regimen, said Dr. Juan Bueren, scientific director of the FA gene therapy program at Spains Center for Energy, Environmental and Technological Research, in a statement. This indicates the potential of this therapeutic approach as a definitive hematologic treatment, while avoiding the burdensome side effects associated with allogeneic transplant, including the risk of post-transplant mortality and a substantially higher risk of head and neck cancer.

Photo: virusowy, Getty Images

Read this article:
Rocket's gene therapy shows long-term efficacy in rare blood disorder - MedCity News

Bone Marrow Stem Cells Comments Off on Rocket’s gene therapy shows long-term efficacy in rare blood disorder – MedCity News | October 26th, 2019

The worlds first cell atlas of the human developmental liver has been created, giving fresh insight into how the blood and immune systems develop in the foetus.

A high-resolution resource, it will aid our understanding of normal development and efforts to tackle diseases that can form during development, such as leukaemia and immune disorders.

The cell atlas maps how the cellular landscape within the developing liver changes between the first and second trimesters of pregnancy, including how stem cell from the liver seed other tissues, supporting the high demand for oxygen required for growth.

Researchers from the Wellcome Sanger Institute in Hinxton, the Wellcome MRC Cambridge Stem Cell Institute, University of Cambridge, Newcastle University and their collaborators created the atlas by using single cell technology to analyse 140,000 liver cells and 74,000 skin, kidney and yolk sac cells.

In adults, it is bone marrow that is primarily responsible for the creation of blood and immune cells in a process called haematopoiesis.

In early embryonic life, the yolk sac and liver play a key role in creating these cells, which then seed peripheral tissues such as skin, kidney and ultimately bone marrow.

But until now, the precise process of how blood and immune systems develop in humans has been unknown.

Isolating cells from the developing liver, the researchers were able to identify them by what genes they were expressing and discover what the cells looked like.

They tagged haematopoietic cells in sections of developmental liver using heavy metal markers in order to map them to their location.

Prof Muzlifah Haniffa, a senior author of the study from Newcastle University and senior clinical fellow at the Wellcome Sanger Institute, said: Until now research in this area has been a little bit like blindfolded people studying an elephant, with each describing just a small part of it.

This is the first time that anyone has described the whole picture, how the blood and immune systems develop in such detail. Its been an extraordinary, multidisciplinary effort that is now available as a tool for the whole scientific community.

The scientists learned that during foetal development, mother haematopoietic stem cells stay in the liver. But the liver alone cannot supply enough red blood cells, so the next generation daughter cells called progenitor cells travel to other tissues, maturing in places such as the skin. Thee, they develop into red blood cells to help meet the high demand for oxygen in the developing foetus.

Dr Elisa Laurenti, a senior author from the Wellcome MRC Cambridge Stem Cell Institute and the Department of Haematology at the University of Cambridge, said: We knew that as adults age our immune system changes. This study shows how the livers ability to make blood and immune cells changes in a very short space of time, even between seven and 17 weeks post-conception.

If we can understand what makes the stem cells in the liver so good at making red blood cells, it will have important implications for regenerative medicine.

The study, published in Nature, also involved the mapping of genes involved in immune deficiencies to reveal which cells were expressing them.

It is known that gene mutations can lead to immune disorders such as leukaemia.

A better understanding of the development of healthy liver functions could aid our understanding of how to treat such conditions.

The work is part of the ambitious effort to create the first complete Human Cell Atlas.

Dr Katrina Gold, genetics and molecular sciences portfolio manager at Wellcome, said: Our immune system is vital in helping to protect us from disease, yet we know very little about how immune cells develop and behave in the early embryo. This study is hugely important, laying a critical foundation for future research that could help improve our understanding of disorders linked to the early immune system, such as childhood leukaemias.

The Human Cell Atlas has the potential to transform our understanding of health and disease and were excited to see these first discoveries from our Wellcome-funded multidisciplinary team of scientists.

Dr Sarah Teichmann, a senior author from the Wellcome Sanger Institute, University of Cambridge and co-chair of the Human Cell Atlas organising committee, said: The first comprehensive cellular map of the developmental liver is another milestone for the Human

Cell Atlas initiative.

The data is now freely available for anyone to use and will be a great resource to better understand healthy cellular development and disease-causing genetic mutations.

Read more

Asthma treatment hope as Human Cell Atlas project creates first map of lungs

Sanger Institute scientist helps unveil blueprint for extraordinary Human Cell Atlas

AstraZeneca and Cancer Research UK launch joint Functional Genomics Centre in Cambridge

Read more from the original source:
Worlds first cell atlas of developing liver created by Cambridge scientists - Cambridge Independent

Bone Marrow Stem Cells Comments Off on Worlds first cell atlas of developing liver created by Cambridge scientists – Cambridge Independent | October 26th, 2019

NEW YORK, Oct. 25, 2019 (GLOBE NEWSWIRE) -- BrainStorm Cell Therapeutics Inc. (NASDAQ: BCLI), a leader in the development of innovative autologous cellular therapies for highly debilitating neurodegenerative diseases, today announced that it will be presenting at the Dawson James Securities 5th Annual Small Cap Growth Conference, being held on October 28-29, 2019 at the Wyndham Grand Hotel in Jupiter, Florida.

Preetam Shah, PhD, MBA, Chief Financial Officer is scheduled to present on Tuesday, October 29th at 3:40 p.m. Eastern Time, in Track 2 - Preserve Ballroom B, with one-on-one meetings to be held throughout the conference.

Chaim Lebovits, President and CEO of BrainStorm said, We are pleased to have the opportunity to have Dr. Shah present at the Dawson James Small Cap Growth Conference. Dr. Shah, joined BrainStorm in September 2019, and we look forward to having him present the Companys growth strategy and future to a wide audience of accreditied investors.

About NurOwnNurOwn (autologous MSC-NTF cells) represent a promising investigational approach to targeting disease pathways important in neurodegenerative disorders. MSC-NTF cells are produced from autologous, bone marrow-derived mesenchymal stem cells (MSCs) that have been expanded and differentiated ex vivo. MSCs are converted into MSC-NTF cells by growing them under patented conditions that induce the cells to secrete high levels of neurotrophic factors. Autologous MSC-NTF cells can effectively deliver multiple NTFs and immunomodulatory cytokines directly to the site of damage to elicit a desired biological effect and ultimately slow or stabilize disease progression. NurOwn is currently being evaluated in a Phase 3 ALS randomized placebo-controlled trial and in a Phase 2 open-label multicenter trial in Progressive MS.

About BrainStorm Cell Therapeutics Inc.BrainStorm Cell Therapeutics Inc. is a leading developer of innovative autologous adult stem cell therapeutics for debilitating neurodegenerative diseases. The Company holds the rights to clinical development and commercialization of the NurOwn Cellular Therapeutic Technology Platform used to produce autologous MSC-NTF cells through an exclusive, worldwide licensing agreement. Autologous MSC-NTF cells have received Orphan Drug status designation from the U.S. Food and Drug Administration (U.S. FDA) and the European Medicines Agency (EMA) in ALS. BrainStorm has fully enrolled the Phase 3 pivotal trial in ALS (NCT03280056), investigating repeat-administration of autologous MSC-NTF cells at six sites in the U.S., supported by a grant from the California Institute for Regenerative Medicine (CIRM CLIN2-0989). The pivotal study is intended to support a BLA filing for U.S. FDA approval of autologous MSC-NTF cells in ALS. BrainStorm received U.S. FDA clearance to initiate a Phase 2 open-label multi-center trial of repeat intrathecal dosing of MSC-NTF cells in Progressive Multiple Sclerosis (NCT03799718) in December 2018 and has been enrolling clinical trial participants since March 2019. For more information, visit the company's website.

Safe-Harbor Statements Statements in this announcement other than historical data and information, including statements regarding future clinical trial enrollment and data, constitute "forward-looking statements" and involve risks and uncertainties that could cause BrainStorm Cell Therapeutics Inc.'s actual results to differ materially from those stated or implied by such forward-looking statements. Terms and phrases such as "may", "should", "would", "could", "will", "expect", "likely", "believe", "plan", "estimate", "predict", "potential", and similar terms and phrases are intended to identify these forward-looking statements. The potential risks and uncertainties include, without limitation, BrainStorms need to raise additional capital, BrainStorms ability to continue as a going concern, regulatory approval of BrainStorms NurOwn treatment candidate, the success of BrainStorms product development programs and research, regulatory and personnel issues, development of a global market for our services, the ability to secure and maintain research institutions to conduct our clinical trials, the ability to generate significant revenue, the ability of BrainStorms NurOwn treatment candidate to achieve broad acceptance as a treatment option for ALS or other neurodegenerative diseases, BrainStorms ability to manufacture and commercialize the NurOwn treatment candidate, obtaining patents that provide meaningful protection, competition and market developments, BrainStorms ability to protect our intellectual property from infringement by third parties, heath reform legislation, demand for our services, currency exchange rates and product liability claims and litigation,; and other factors detailed in BrainStorm's annual report on Form 10-K and quarterly reports on Form 10-Q available at http://www.sec.gov. These factors should be considered carefully, and readers should not place undue reliance on BrainStorm's forward-looking statements. The forward-looking statements contained in this press release are based on the beliefs, expectations and opinions of management as of the date of this press release. We do not assume any obligation to update forward-looking statements to reflect actual results or assumptions if circumstances or management's beliefs, expectations or opinions should change, unless otherwise required by law. Although we believe that the expectations reflected in the forward-looking statements are reasonable, we cannot guarantee future results, levels of activity, performance or achievements.

CONTACTS

Corporate:Uri YablonkaChief Business OfficerBrainStorm Cell Therapeutics Inc.Phone: 646-666-3188uri@brainstorm-cell.com

Media:Sean LeousWestwicke/ICR PRPhone: +1.646.677.1839sean.leous@icrinc.com

See the original post:
BrainStorm Cell Therapeutics to Present at the Dawson James Securities 5th Annual Small Cap Growth Conference - BioSpace

Bone Marrow Stem Cells Comments Off on BrainStorm Cell Therapeutics to Present at the Dawson James Securities 5th Annual Small Cap Growth Conference – BioSpace | October 26th, 2019

The build-out is estimated at $9.5M.

United Therapeutics received a building permit Tuesday for a $9.5M build-out of its cell therapy facility on the second floor of Mayo Clinics Discovery and Innovation Building.

The 21,843-square-foot space will house an automated stem cell manufacturing site, which is one of the first of its kind in the country. The Whiting-Turner Contracting Co. is the project contractor.

The technology, approved by the FDA in 2018, allows the Mayo Clinic Center for Regenerative Medicine to produce cells from the bone marrow of a stem cell donor in large enough quantities to be used as treatments in clinical trials. It allows for the treatment of multiple patients at the same time.

Construction began in 2017 on the $32.4M building at 14221 Kendall Hench Drive. It held a grand opening in August.

The first floor houses three ex-vivo lung perfusion surgical suites used for lung restoration, another form of regenerative medicine. It turns donor lungs, which previously would have previously been unusable, into viable transplant organs. United Therapeutics also collaborates with Mayo Clinic on lung restoration.

The third floor houses the Life Sciences Incubator for biotech entrepreneurs, which offers coworking space, wet labs, business resources, networking and entrepreneurial training.

Go here to see the original:
United Therapeutics Receives Permit For Cell Therapy Facility Build-Out At Mayo - Pharmaceutical Online

Bone Marrow Stem Cells Comments Off on United Therapeutics Receives Permit For Cell Therapy Facility Build-Out At Mayo – Pharmaceutical Online | October 26th, 2019