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Plinabulin Combo May Be Superior Therapy for Prevention of Chemotherapy-Induced Neutropenia – Targeted Oncology

By daniellenierenberg

Treatment with plinabulin in combination with pegfilgrastim (Neulasta) achieved the primary end point and several key secondary end points in the phase 3 PROTECTIVE (Study 106) clinical trial, which evaluated the combination compared with pegfilgrastim alone for superiority in chemotherapy-induced neutropenia prevention, according to the interim results announced by BeyondSpring, developer of the drug.

These results demonstrate significant enhancement to granulocyte colony-stimulating factors (G-CSFs) with the combination compared with pegfilgrastim alone for the prevention of grade 4 or severe neutropenia, supporting the potential for superiority with the combination compared with pegfilgrastim alone.

These interim results from the PROTECTIVE-2 Phase 3 study, which compares the Plinabulin-Neulasta combination to Neulasta alone, have the potential to be clinically meaningful for cancer patients receiving chemotherapy, stated Douglas W. Blayney, MD, professor of Medicine at Stanford Medical School and global principal investigator of Plinabulins chemotherapy-induced neutropenia studies. Since most infections, hospitalizations, and other complications of chemotherapy-induced neutropenia occur in the first week after chemotherapy, it is particularly gratifying to see the combinations clinical benefit demonstrated.

The primary end point was the rare of severe neutropenia prevention in cycle 1 (P <.01). The key secondary end points included duration of severe neutropenia in cycle 1 (P <.05) and duration of severe neutropenia in the first 8 days of cycle 1 (P <.05). The achievement of these end points demonstrates the agents ability to provide early protection against severe neutropenia induced by chemotherapy.

The interim analysis of the double-blind, active-controlled, global PROTECTIVE-2 study was pre-specified for the first 120 patients accrued. The study also has procedures in place for the prevention of potential bias after the planned interim analysis, and BeyondSpring opted to be informed by independent statisticians on whether the pre-specified P values were met rather than the exact P values.

The study was designed to assess both the safety and efficacy of plinabulin in patients with breast cancer treated with docetaxel, doxorubicin, and cyclophosphamide (TAC) in a 21-day cycle. Plinabulin was administered in a 40 mg dose on day 1 with a 6 mg dose of pegfilgrastim on day 2, while pegfilgrastim was administered at the same 6 mg dose on day 2 in the control arm. TAC is considered a high-risk chemotherapy regimen associated with neutropenia.

PROTECTIVE-2 is a superiority study for chemotherapy-induced neutropenia efficacy compared with pegfilgrastim alone and is currently enrolling patients. Grade 4 neutropenia rate for TAC and pegfilgrastim is observed in 83% to 93% of patients, which represents a severe unmet medical need.

The study drug and G-CSF have complementary mechanisms of action for preventing chemotherapy-induced neutropenia. Plinabulin is a first-in-class differentiated immune and stem cell modulator, which is currently in late-stage clinical development as a potential treatment for increasing overall survival in patients with cancer in addition to its ability to alleviate chemotherapy-induced neutropenia.

Plinabulin is also under evaluation in a phase 3 study as a direct anticancer drug as treatment of patients with nonsmall cell lung cancer and 2 phase 3 studies for the prevention of chemotherapy-induced neutropenia. The data for this agent in preventing chemotherapy-induced neutropenia highlights its ability to boost the number of hematopoietic stem or progenitor cells (HSPcs), as well as lineage-/cKIT+/Sca1+ cells in mice. Plinabulins effects on HSPcs may explain its ability to treat chemotherapy-induced neutropenia as well as its ability to reduce chemotherapy-induced thrombocytopenia and increase circulating CD34-positive cells.

To be included in the study, patients should be candidates for TAC in either the adjuvant or neoadjuvant setting, have early-stage breast cancer, and have received no prior chemotherapy. Patients must also have an ECOG performance status of 0 or 1 and a life expectancy of at least 3 months. Patients cannot be included in the study if they have a history of myelogenous leukemia, myelodysplastic syndrome, or sickle cell disease; use of CYP3A4, CYP2D6 or P-glycoprotein inhibitors and inducers within 14 days prior to receiving the study drug; or have received an investigational agent or tumor vaccine within 2 weeks, any concurrent anticancer therapies, or a bone marrow or stem cell transplantation.

These results could help to confirm the patient benefit of Plinabulins different mechanism of action from the G-CSF-based agents, such as Neulasta, Blayney stated. Plinabulin appears to have chemotherapy-induced neutropenia protection in Week 1, and G-CSFs have protection in Week 2 of chemotherapy cycles. The combination should logically provide significantly better protection than Neulasta alone as shown in the interim readout. We are well on our way to confirming that the combination offers protection throughout the chemotherapy cycle, which is an unmet medical need.

Reference

BeyondSpring Announces Positive Topline Interim Results from PROTECTIVE-2 (Study 106) Phase 3 Trial Evaluating Superiority of Plinabulin in Combination with Neulasta for Chemotherapy-Induced Neutropenia Prevention. NewsRelease. BeyondSpring. June 15, 2020. Accessed June 15, 2020. https://bit.ly/2YDJbiQ

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Children’s Hospital Los Angeles Ranked No. 1 Children’s Hospital in the Western U.S., No. 5 Nationally for Second Straight Year – Newswise

By daniellenierenberg

Newswise LOS ANGELES (June 16, 2020) Children's Hospital Los Angeles (CHLA) ranks again among the nations premier destinations for pediatric care, according to the U.S. News & World Report Best Children's Hospitals annual list released today.

CHLA not only retained its national No. 5 ranking in U.S. News Honor Roll of Best Childrens Hospitalswhich recognizes institutions with the most outstanding pediatric clinical careit continued its four-year streak of being the highest-scoring childrens hospital in the entire Western United States.

To make U.S. News & World Reports prestigious Best Childrens Hospitals Honor Roll, one must demonstrate the strongest achievements in clinical excellence, with a matchless team of expert, compassionate specialists committed to research and education as well as protocols that drive safety and quality and consistently lead to the best health outcomes for patients, says CHLA President and Chief Executive Officer Paul S. Viviano. "This honor affirms the work of every CHLA team member and our belief that when parents choose Childrens Hospital Los Angeles, they are choosing the best care for kids."

Every year, U.S. News scores nearly every major hospital and health system in the country and ranks them according to performance benchmarks, peer review, certifications, and other data provided by the hospital and third-party measurements of excellence. Children's hospitals are ranked separately from other facilities due to the specialized expertise, equipment and facilities required to care for infants, children and youth.

This year, U.S. News surveyed 118 pediatric medical centers, including hospitals that are freestanding or part of a larger institution. CHLA improved its ranking over last year in seven of the 10 pediatric specialty categories the survey considers, including a number two ranking for Neonatal Care. In all, the hospital earned top-10 recognition in seven of those categories:

CHLA has an organization-wide commitment to providing our patients the care they need no matter their circumstances, says CHLA Chief Medical Officer James Stein, M.D., MSc. "Our clinical teams often treat the most acute cases that are outside the scope or expertise of other childrens hospitals in California, and being named a Top-5 childrens hospital in the U.S. is a testament to the clinicians and staff who work every day to make sure each child receives the best care and experience possible.

Founded in 1901, Children's Hospital Los Angeles is a pediatric academic medical center built around its mission of creating hope and building healthier futures for children. Renowned for its world-class clinical care, leading-edge research and one of the largest and most successful pediatric training programs in the countryall while being the pediatric safety net hospital for the entire regionCHLA now sees more than 600,000 patient visits annually between its main hospital and five neighborhood care clinics.

CHLA physicians, nurses and clinical staff provide compassionate and lifesaving pediatric care for patients ranging from infants to young adults, hailing from all 50 states and more than 75 countries. Clinical care is led by physicians who are faculty members of the Keck School of Medicine of USC. Many of the hospital's achievements in care are made possible through a cohesive relationship between clinical experts at the bedside and the basic, translational, and clinical research conducted in The Saban Research Institute of CHLA.

In the past year, CHLA has had several notable achievements, including:

U.S. News and World Report works with research firm RTI International to develop its annual Best Children's Hospitals list, a collaboration between hospitals and the magazine to benchmark the performance of childrens hospitals for the benefit of parents and their children. The survey evaluates hundreds of data points, including patient survival and surgical complication rates; staffing, technology and special services; infection prevention and delivery of care; reputation among peer physicians nationwide (i.e. Where would the best pediatric specialists send their kids?); how involved parents are in their childrens care; and many other measurements of excellence.

U.S. News Media Group, the parent of U.S. News & World Report, announced the 2020-21 hospital rankings online at 12:01 a.m. EST on Tuesday, June 16. For additional information, please visit the Best Childrens Hospitals Honor Roll and specialty rankings page at usnews.com/childrenshospitals.

About Children's Hospital Los Angeles Founded in 1901,Children's Hospital Los Angelesis ranked the topchildrens hospital in California andfifth in the nation for clinical excellence with its selection to the prestigious U.S. News & World Report Honor Roll of childrens hospitals. Clinical care is led by physicians who are faculty members of the Keck School of Medicine of USC through an affiliation dating from 1932.The hospitalalso leads thelargest pediatric residency training program at a freestanding childrens hospital of its kind in the western United States.TheSaban Research Instituteof Childrens Hospital Los Angeles encompasses basic, translational and clinical research conducted at CHLA. The hospitals Global Health Program facilitates services for international patients from more than 75 countries. To learn more, follow us on Facebook, Instagram, LinkedIn and Twitter, and visit our blog for families (CHLA.org/blog) andour research blog (ResearCHLABlog.org).

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2025 Projection: Thalassemia Treatment Market Analysis by SWOT, Investment, Future Growth and Major Key Players 2020 to 2026 – Cole of Duty

By daniellenierenberg

COVID-19 impact will also be included and considered for forecast.

Global Thalassemia Treatment Market research report provides detail information about Market Introduction, Market Summary, Global market Revenue (Revenue USD), Market Drivers, Market Restraints, Market Opportunities, Competitive Analysis, Regional and Country Level.

Thalassemia Treatment Market Size Covers Global Industry Analysis, Size, Share, CAGR, Trends, Forecast And Business Opportunity.

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Global Thalassemia Treatment Market 2018-2024Brandessence Market Research is working on a new report titleGlobal Thalassemia Treatment Market: Global Size, Trends, Competitive, Historical & Forecast Analysis, 2018-2024?. Rise in number of altered Thalassemia genes, increase in awareness about the disease and high adoption of chelation therapy & blood transfusion for treatment by doctors as well as patients arelikely to enhance the growth of Global Thalassemia Treatment Market.

Scope of Global Thalassemia Treatment Market Reports

Thalassemia is aninherited blood disorder in which the body makes an abnormal form of hemoglobin.People having Thalassemia disease are unable to make sufficient hemoglobin which causes severe anemic conditions.Hemoglobin is found in red blood cells and transports oxygen to all parts of the body. When there is insufficient hemoglobin in the red blood cells, oxygen cannot get to all parts of the body. Organs demand oxygen and are unable to function properly.There are two primary types of Thalassemia disease such as Alpha Thalassemia disease and Beta Thalassemia disease.Alpha Thalassemia results in a formation of additional beta globins, which leads to the formation of beta-globin tetramers (4) called Hemoglobin H.

Beta Thalassemia causesadditionalformation of alpha globins, which develops alpha globin tetramers (a4) that store in the erythroblast (immature red blood cell).Thalassemia is caused by mutations in the DNA of cells that make hemoglobin.

Factors that increase risk of Thalassemia include Family history of thalassemia and certain ancestry.Possible complications of Thalassemia includeIron overload, Infections, Bone deformities, splenomegaly, slowed growth rate of child and Heart problems.

Thalassemia signs and symptoms include Fatigue, Weakness, Pale or yellowish skin, Facial bone deformities, slow growth, abdominal swelling, Dark urine, chest pain,cold hands and feet, poor feeding, greater susceptibility to infections. Diagnosis of Thalassemia includesa complete blood count (CBC), a reticulocyte count,Iron count, Genetic testing and prenatal testing. Treatment of Thalassemia depends on the type and severity of Thalassemia such as Blood transfusions, Bone marrow or stem cell transplant, Surgery and Gene therapy.

Global Thalassemia Treatment Market has been segmented on the basis ofType of Treatment, Diagnosis, End usersand Geography. On the basis of Type of TreatmentGlobal Thalassemia Treatment Market is classified into Blood Transfusion, Chelating Therapy, Bone Marrow Transplant, Stem Cell Transplant, Surgery, Gene Therapy and Others.On the basis of DiagnosisGlobal Thalassemia Treatment Market is classified into Perinatal Testing, Prenatal Testing, Pre-Implantation and Other.On the basis of the End user the Global Thalassemia Treatment Market is classified into Hospitals, Biotechnological Laboratories, Diagnostic Laboratories, Educational Research Institutes, Pharmaceutical Industries and others.

The regions covered in Global Thalassemia Treatment Market report are North America, Europe, Asia-Pacific and Rest of the World. On the basis of country level, Global Melanoma Drug Market sub divided in to U.S., Mexico, Canada, U.K., France, Germany, Italy, China, Japan, India, South East Asia, GCC, Africa, etc.

Key Players for Global Thalassemia Treatment Market Reports

Global Thalassemia Treatment Market reports cover prominent players like Bluebird bio Inc., Acceleron Pharma Inc., Novartis AG, Celgene Corporation, Shire plc, Bellicum Pharmaceuticals, GlaxoSmithKline Plc, Celgene,Lonza group, Alnylam Pharmaceuticals Inc., Calimmune Inc., CRISPR Therapeutics, Editas Medicine Inc., Errant Gene Therapeutics LLC, Gamida Cell Ltd, Gilead Sciences Inc., Incyte Corp, Ionis Pharmaceuticals Inc., IRBM Science Park SpA, Johnson & Johnson, Kiadis Pharma NV, La Jolla Pharmaceutical Company, Merck & Co Inc., PharmaEssentia Corp, Protagonist Therapeutics Inc., Sangamo Therapeutics Inc., Zydus Cadila Healthcare Ltd, Genorama Ltd, HiMedia Laboratories, DiagCor Bioscience Inc. Ltd and Tosoh Bioscience Inc.

Global Thalassemia Treatment Market Dynamics

Increase in awareness about the disease and technological expansions are likely to raise the adoption of gene therapies. Also Rising Prevalence of Thalassemia, Increase in Pharmaceutical R&D Spending, Increasing Spending on Stem Cell Research, Rising Healthcare Expenditure and Rising Asian Population will boost theGlobal Thalassemia Treatment Market. Treatment of Thalassemia is mostly restricted to regular blood transfusions and iron chelation therapy.Moreover, High operation cost of sophisticated clinical and preclinical imaging systems, High cost of maintenance andless life span of accessoriesalso restraining theGlobal Thalassemia Treatment Market.Yearlyspending for treatment of Thalassemia ranged from $ 108 to 432, depending on type of treatment with average cost per blood transfusion was $ 5.22.2. Average 18.5%14.3 of the total annual income was spent on the treatment for Thalassemia. Drugs prescribed for Thalassemia mostly cures symptoms and side effects such as anemia, iron overload, slow growth of children and vitamin deficiency.Occurrence of Thalassemia is reported to increase steadily over the years across different regions. This can be due to population migration, intermarriages, genetic as well as environmental factors prompting the condition and its implications.Systematic Drugs under Pipeline, Rising Scope for Gene Therapy and increasing awareness towards Thalassemia are some opportunities in the forecast period for theGlobal Thalassemia Treatment Market.

Global Thalassemia Treatment MarketRegional Analysis

North America have largest share ofGlobal Thalassemia Treatment Market. It is mainly driven by quickly increasing immigrant population from tropical regions, rising number of population with Thalassemia carrier gene and rise in birth rates due to variation of genes among the population in the U.S.There are some prenatal tests available on the market to determine the possibility of alpha thalassemia including both invasive and non-invasive technique.

The alpha thalassemia testing market has aemergent trend in the countries with traditional groups like Mediterranean countries, African countries and few countries in Asia Pacific. Furthermore, in Asia Pacific region the growth in similar community marriage practices and high fertility ratewith alpha thalassemia patients have been detected. This is expected to raiseacceptance of blood transfusion and chelation therapy treatments during the forecast period.A latestimprovement in the testing of alpha Thalassemia may determine the risk of the disease by in vitro examination of the embryo. While there are various such tests available in theGlobal Thalassemia Treatment Market but lack of awareness leads to the neglect and delayed diagnosis of the diseased state.

Most frequently prone area for alpha thalassemia is Mediterranean countries, African countries, and Southeast Asian countries. Thalassemia trait practically affects 6% to 35% of the population in these ethnic groups. Middle East & Africa is likely to be the fastest risingGlobal Thalassemia Treatment Market during the forecast period.

Key Benefits for Global Thalassemia Treatment Market Reports

Global Thalassemia Treatment Market report covers in depth historical and forecast analysis.Global Thalassemia Treatment Market research report provides detail information about Market Introduction, Market Summary, Global market Revenue (Revenue USD), Global market sale (K Units), Global market Drivers, Market Restraints, Market opportunities, Competitive Analysis, Regional and Country Level.Global Thalassemia Treatment Market report helps to identify opportunities in market place.Global Thalassemia Treatment Market report covers extensive analysis of emerging trends and competitive landscape.Global Thalassemia Treatment Market Segmentation

Global Thalassemia Treatment Market: By Type of Treatment Analysis

Blood TransfusionChelating TherapyBone Marrow TransplantStem Cell TransplantSurgeryGene TherapyOtherGlobal Thalassemia Treatment Market: By Diagnosis Analysis

Perinatal TestingPrenatal TestingPre-ImplantationOtherGlobal Thalassemia Treatment Market: By End user Analysis

HospitalsBiotechnological LaboratoriesDiagnostic LaboratoriesEducational Research InstitutesPharmaceutical IndustriesOtherGlobal Thalassemia Treatment Market: By Regional & Country Analysis

North AmericaU.S.MexicoCanadaEuropeUKFranceGermanyItalyAsia PacificChinaJapanIndiaSoutheast AsiaLatin AmericaBrazilThe Middle East and AfricaGCCAfricaRest of Middle East and AfricaNeed a PDF of the global market report? Visit: https://industrystatsreport.com/Request/Sample?ResearchPostId=57&RequestType=Methodology

Table of Content:

Market Overview: The report begins with this section where product overview and highlights of product and application segments of the Global Thalassemia Treatment Market are provided. Highlights of the segmentation study include price, revenue, sales, sales growth rate, and market share by product.

Competition by Company: Here, the competition in the Worldwide Global Thalassemia Treatment Market is analyzed, By price, revenue, sales, and market share by company, market rate, competitive situations Landscape, and latest trends, merger, expansion, acquisition, and market shares of top companies.

Company Profiles and Sales Data: As the name suggests, this section gives the sales data of key players of the Global Thalassemia Treatment Market as well as some useful information on their business. It talks about the gross margin, price, revenue, products, and their specifications, type, applications, competitors, manufacturing base, and the main business of key players operating in the Global Thalassemia Treatment Market.

Market Status and Outlook by Region: In this section, the report discusses about gross margin, sales, revenue, production, market share, CAGR, and market size by region. Here, the Global Thalassemia Treatment Market is deeply analyzed on the basis of regions and countries such as North America, Europe, China, India, Japan, and the MEA.

Application or End User: This section of the research study shows how different end-user/application segments contribute to the Global Thalassemia Treatment Market.

Market Forecast: Here, the report offers a complete forecast of the Global Thalassemia Treatment Market by product, application, and region. It also offers global sales and revenue forecast for all years of the forecast period.

Research Findings and Conclusion: This is one of the last sections of the report where the findings of the analysts and the conclusion of the research study are provided.

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We publish market research reports & business insights produced by highly qualified and experienced industry analysts. Our research reports are available in a wide range of industry verticals including aviation, food & beverage, healthcare, ICT, Construction, Chemicals and lot more. Brand Essence Market Research report will be best fit for senior executives, business development managers, marketing managers, consultants, CEOs, CIOs, COOs, and Directors, governments, agencies, organizations and Ph.D. Students.

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2025 Projection: Thalassemia Treatment Market Analysis by SWOT, Investment, Future Growth and Major Key Players 2020 to 2026 - Cole of Duty

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Race Oncology confirms positive results from Bisantrene drug trial on patients with advanced acute myeloid leukaemia – Small Caps

By daniellenierenberg

Specialty pharmaceutical company Race Oncology (ASX: RAC) has reported positive data from an investigator-initiated Phase II clinical trial of historical cancer drug Bisantrene on patients with relapsed or refractory acute myeloid leukaemia.

Conducted at the Sheba Medical Centre in Israel, the open-label, single-agent trial studied 10 patients who, on average, had failed three prior lines of treatment.

Bisantrene was found to be well tolerated with no unexpected or serious toxicities, the company reported.

After a single course of treatment, the drug demonstrated an overall clinical response rate of 40%, with one patient progressing to complete remission and three achieving partial remission.

One patient was bridged to allogeneic stem cell transplantation (where cells are donated to the patient from a genetically-matched donor) and there were no removals or withdrawals from the study during treatment.

The drug also had marked activity in four patients with extramedullary (outside of the bone marrow) acute myeloid leukaemia such as leukemia cutis, chloromas, and central nervous system disease which has historically been difficult to treat.

The most frequently reported serious adverse events were thrombocytopaenia (low blood platelets) and mucositis (mouth ulcers), both of which were expected side effects of anthracyline and anthracene chemotherapeutics.

One patient experienced transient grade one kidney toxicity and there were no liver toxicities observed.

Race said there were no anaphylactoid-type reactions observed in any patient over the course of treatment. This type of reaction was a serious adverse event regularly observed in the historical trials.

Relapsed or refractory acute myeloid leukaemia remains a significant therapeutic challenge.

While meaningful gains have been achieved in recent years with the introduction of new targeted drugs, published studies claim the clinical outcomes remain unsatisfactory.

Bisantrene is a small molecule cancer drug related to anthracyclines the most widely-used class of chemotherapy drugs.

Unlike anthracyclines, it has a greatly reduced risk of cardiotoxicity (heart damage), meaning it can be used with patients who have reached their cardiotoxic limit with anthracyclines, or cannot tolerate anthracyclines due to existing heart conditions, age or other factors.

Bisantrene was tested in more than 40 phase II clinical trials during the 1980s and 1990s with up to $200 million put into its development, before it was lost in a series of big pharmaceutical mergers.

Race clinical advisory board chairman and international authority in clinical leukaemia and stem cell research, Professor Borje Andersson, said the trial confirmed historical results which used a different formulation of Bisantrene.

While Bisantrene had been demonstrated in the 1980s as an effective salvage drug against acute myeloid leukaemia, the [clinical] data we had was old, he said.

It was important for us to study it by todays standards using the current formulation, so we could confirm whether our strategy of repurposing this drug is sound.

We also wanted to confirm that Bisantrene could still generate a meaningful response rate in a highly-frail patient population with heavily pre-treated acute myeloid leukaemia, Professor Andersson added.

The study saw a reduction in the leukaemic disease burden and an overall response rate in 40% of the patients.

While we must study the drug further, it appears that with this kind of response, Bisantrene-based therapy may have the potential to serve as an important bridge to allogeneic stem cell transplantation in patients who otherwise have few therapeutic options, Professor Andersson said.

Race chief scientific officer Dr Daniel Tillett said a key focus of Phase II clinical trial was to determine Bisantrenes safety in a modern context.

These results are pleasing from a safety and activity perspective, particularly given the clinically-challenging patient population included in the trial, he said.

It was encouraging to see the drugs tolerability profile compared favourably with other commonly-used chemotherapy agents such as anthracyclines, while the side effects were in keeping with what we would expect to see with all chemotherapeutics of this class, Dr Tillett added.

Professor Andersson said the trial found Bisantrene to be an agent with an acceptable safety profile and promising anti-leukaemic activity.

As this was an open-label, single-agent trial, we can be confident that it was the Bisantrene exposure which generated the positive results, he said.

The patient cohort had advanced acute myeloid leukaemia and had previously failed an average of three lines of therapy, so they were always going to be tough to treat, but a 40% overall response rate after only a single course of treatment markedly exceeded our expectations.

It is a hugely promising result and one which reinforces our development plans for this drug, Professor Andersson added.

A follow-up study combining Bisantrene with other anti-leukaemic drugs is currently in the advanced planning stages.

At mid-morning, shares in Race Oncology were up 53.97% to $0.485.

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Hematopoietic Stem Cells Transplantation (HSCT) Market Trends and Forecast Analysis by Business Manufactures and Product Type-Kite Pharma, Thermo…

By daniellenierenberg

The Hematopoietic Stem Cells Transplantation (HSCT) Market research report enhanced worldwide Coronavirus COVID19 impact analysis on the market size (Value, Production and Consumption), splits the breakdown (Data Status 2014-2020 and 6 Year Forecast From 2020 to 2026), by region, manufacturers, type and End User/application. This Hematopoietic Stem Cells Transplantation (HSCT) market report covers the worldwide top manufacturers like (Kite Pharma, Thermo Fisher Scientific, CellGenix Technologie Transfer, Cesca Therapeutics, R&D Systems) which including information such as: Capacity, Production, Price, Sales, Revenue, Shipment, Gross, Gross Profit, Import, Export, Interview Record, Business Distribution etc., these data help the consumer know about the Hematopoietic Stem Cells Transplantation (HSCT) market competitors better. It covers Regional Segment Analysis, Type, Application, Major Manufactures, Hematopoietic Stem Cells Transplantation (HSCT) Industry Chain Analysis, Competitive Insights and Macroeconomic Analysis.

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Hematopoietic Stem Cells Transplantation (HSCT) Market report offers comprehensive assessment of 1) Executive Summary, 2) Market Overview, 3) Key Market Trends, 4) Key Success Factors, 5) Hematopoietic Stem Cells Transplantation (HSCT) Market Demand/Consumption (Value or Size in US$ Mn) Analysis, 6) Hematopoietic Stem Cells Transplantation (HSCT) Market Background, 7) Hematopoietic Stem Cells Transplantation (HSCT) industry Analysis & Forecast 20202026 by Type, Application and Region, 8) Hematopoietic Stem Cells Transplantation (HSCT) Market Structure Analysis, 9) Competition Landscape, 10) Company Share and Company Profiles, 11) Assumptions and Acronyms and, 12) Research Methodology etc.

Scope of Hematopoietic Stem Cells Transplantation (HSCT) Market:Hematopoietic stem cell transplants (HSCT) present to a valid treatment for several congenital and other hematopoietic system disorders, post chemotherapy, and immune sensitive diseases. HSCT is also preferred for replacement of cellular components and deficient cells. The indications for HSCT thus are wide; the most frequent indication as per reported by Worldwide Network for Blood and Marrow Transplantation Group (WNBT) (2013) is lymphoproliferative disorder (53.2% of all HSCT), 12% of whom received allogeneic and the rest received autologous transplant. Plasma cell disorders are the most frequent indication in this group. A multitude of literature published by researchers and organizations demonstrate that autologous transplant own a greater edge against allogeneic HSCT.

Over 30 years of studies in the field of blood-forming stem cells i.e. hematopoietic stem cells (HSC), researchers have developed significant understanding to use HSCs as a therapy. At present, no type of stem cell, adult, embryonic or fetal has attained such sufficient status. Hematopoietic stem cell transplantation (HSCT) is now routinely used for treating patients with malignant and non-malignant disorders of blood and the immune system. Currently, researchers have observed that through animal studies HSCs have the ability to form other cells such as blood vessels, muscles, and bone. Further application of this approach it may eventually be able to treat a wide array of conditions and replace ailing tissues. However, despite the vast experience with HSCs, researchers face major barriers in expanding their use beyond the replacement of immune and blood cells.

Hematopoietic stem cells are unable to proliferate and differentiate in-vitro. Researchers have yet to evolve an accurate method to differentiate stem cells from other cells derived from blood or bone marrow. Once such technical barriers are overcome, the avenues for realizing the full potential of HSCT. The type of transplant a person receives depends on several different factors, including the type and course of the disease, availability of suitable donors, and the patients overall health. There are three different sources of hematopoietic stem cells such as bone marrow, peripheral blood stem cells, and umbilical cord blood. The stem cell source used for a given transplant depends upon the underlying disease, the type of transplant (allogeneic or autologous), and size of the patient.

On the basis on the end users/applications,this report focuses on the status and outlook for major applications/end users, shipments, revenue (Million USD), price, and market share and growth rate foreach application.

Leukemia Lymphoproliferative Disorders Solid Tumors Non-Malignant Disorders Others

On the basis of product type, this report displays the shipments, revenue (Million USD), price, and market share and growth rate of each type.

Autologous Transplant Allogenic Transplant

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Geographically, the report includes the research on production, consumption, revenue, Hematopoietic Stem Cells Transplantation (HSCT) market share and growth rate, and forecast (2020-2026) of the following regions:

Important Hematopoietic Stem Cells Transplantation (HSCT) Market Data Available In This Report:

Strategic Recommendations, Forecast Growth Areasof the Hematopoietic Stem Cells Transplantation (HSCT) Market.

Challengesfor the New Entrants,TrendsMarketDrivers.

Emerging Opportunities,Competitive Landscape,Revenue Shareof Main Manufacturers.

This Report Discusses the Hematopoietic Stem Cells Transplantation (HSCT) MarketSummary; MarketScopeGives A BriefOutlineof theHematopoietic Stem Cells Transplantation (HSCT) Market.

Key Performing Regions (APAC, EMEA, Americas) Along With Their Major Countries Are Detailed In This Report.

Company Profiles, Product Analysis,Marketing Strategies, Emerging Market Segments and Comprehensive Analysis of Hematopoietic Stem Cells Transplantation (HSCT) Market.

Hematopoietic Stem Cells Transplantation (HSCT) Market ShareYear-Over-Year Growthof Key Players in Promising Regions.

What is the (North America, South America, Europe, Africa, Middle East, Asia, China, Japan)production, production value, consumption, consumption value, import and exportof Hematopoietic Stem Cells Transplantation (HSCT) market?

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Hematopoietic Stem Cells Transplantation (HSCT) Market Trends and Forecast Analysis by Business Manufactures and Product Type-Kite Pharma, Thermo...

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Cell Therapy Manufacturing Market: Regional Analysis and Strategies in Forecasted Period 2020-2030 – 3rd Watch News

By daniellenierenberg

Prophecy Market Insights has recently published a Cell Therapy Manufacturing report which represents the latest industry data and future trends, allowing users to recognize the products and driving revenue growth and profitability of the market.

The report offers a broad analysis of key segments, key drivers, regions, and leading market players. The report contains an analysis of different geographical areas and presents a competitive scenario to promote leading market players, new entrants, and investors determine emerging economies. The key highlights offered in the report would benefit market players to formulate strategies for the future and gain a strong position in the Cell Therapy Manufacturing market.

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The Cell Therapy Manufacturing report begins with a brief introduction which contains a market overview of the industry followed by its market size and research scope. Further, the report provides an overview of market segmentation, for example- type, application, and region. The drivers, restraints, and opportunities for the market are also mentioned, along with current policies and trends in the industry. The Cell Therapy Manufacturing market also covers PEST analysis for the market. Thisanalysisprovides information based on four external factors (political, economic, social and technological) in relation to your business situation. Basically, it helps to understand how these factorswillaffect the performance and activities of your business in the long-term. The report describes the growth rate of each segment in-depth with the help of charts and tables. Moreover, various regions related to the growth of the Cell Therapy Manufacturing market are analyzed in the report. These regions include North America, Europe, Asia-Pacific, Middle East and Africa, and Latin America.

Segmentation Overview:

Cell Therapy Manufacturing market report states the overview, historical data along with size, share, growth, demand, and revenue of the global industry. In this research report, there is an accurate analysis of the current and upcoming opportunities in the market by explaining the fastest and largest growing segments across regions. The survey report includes vast investigation of the geographical scene of the Cell Therapy Manufacturing market, which is manifestly arranged into the localities

Australia, New Zealand, Rest of Asia-Pacific

The study presents the performance of each player active in the Cell Therapy Manufacturing market. It also provides a summary and highlights the current advancements of each player in the market along with its SWOT analysis. The information provided in the research report is a great source for study investors and stakeholders interested in the market. In addition, the report offers insights on buyers, suppliers, and merchants in the market. There is a comprehensive analysis of consumption, market share, and growth rate of each application is offered for the historic period.

Cell Therapy ManufacturingMarket Key Players:

harmicell, Merck Group, Dickinson and Company, Thermo Fisher, Lonza Group, Miltenyi Biotec GmBH, Takara Bio Group, STEMCELL Technologies, Cellular Dynamics International, Becton, Osiris Therapeutics, Bio-Rad Laboratories, Inc., Anterogen, MEDIPOST, Holostem Terapie Avanazate, Pluristem Therapeutics, Brammer Bio, CELLforCURE, Gene Therapy Catapult EUFETS, MaSTherCell, PharmaCell, Cognate BioServices and WuXi AppTec.

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

By daniellenierenberg

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

Allogeneic HSCT is a multistep procedure:

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

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

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

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

By daniellenierenberg

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

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

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

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

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Table Of Content

CHAPTER 1: INTRODUCTION

CHAPTER 2: EXECUTIVE SUMMARY

CHAPTER 3: MARKET OVERVIEW

CHAPTER 4: STEM CELL BANKING MARKET, BY CELL TYPE

CHAPTER 5: STEM CELL BANKING MARKET, BY BANK TYPE

CHAPTER 6: STEM CELL BANKING MARKET, BY SERVICE TYPE

CHAPTER 7: STEM CELL BANKING MARKET, BY UTILIZATION

CHAPTER 8: STEM CELL BANKING MARKET, BY REGION

CHAPTER 9: COMPANY PROFILES

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BrainStorm Receives $500K to Support ALS Study of NurOwn Therapy – ALS News Today

By daniellenierenberg

The ALS Association and I AM ALS have awarded BrainStorm Cell Therapeutics $500,000 to support an amyotrophic lateral sclerosis (ALS) biomarker study based on the biotechnology companys pivotal trial into its NurOwn therapy.

Specifically, the combined grant $400,000 is from the ALS Association will be used to gain insights from data as well as tissue and blood samples collected from patients enrolled in the Phase 3 clinical trial (NCT03280056) that is measuring NurOwns safety and effectiveness.

As part of the award, BrainStorm agreed to share data and samples with the ALS community to potentially advance other ALS research, and to have trial results independently validated.

By assessing how NurOwn interacts with brain and spinal cord targets, the hope is the study produces a deeper understanding of crucial biomarkers associated with treatment response. A biomarker is any measurable body substance that changes over time, and that correlates with treatment response.

If it works the way its intended, the study is expected to help inform scientists broader understanding of ALS biomarkers.

This grant to BrainStorm marks an important step forward in establishing how exactly NurOwn works in the body, Calaneet Balas, ALS Association president and CEO, said in a press release. This research is also important to our overall pursuit of identification and validation of ALS biomarkers. We hope NurOwn is ultimately proven effective in treating ALS, and we stand ready to support BrainStorm in its plan to apply for a biologics license for NurOwn.

NurOwn is a cell-based therapy that usesmesenchymal stem cells (MSCs), which are extracted from a patients own bone marrow. These cells have the ability to generate different cell types. After extraction, MSCs are expanded in the lab and matured into cells that produce high levels ofneurotrophic factors, which are compounds that promote nervous tissue growth and survival. The converted cells are then reintroduced into the body via an injection into muscles or the spinal canal.

A Phase 2 trial (NCT02017912) found NurOwn to be safe and to significantly slow disease progression in a subset of ALS patients with fast-advancing disease. The Phase 3 trial is evaluating the safety and effectiveness of three administrations every two months of NurOwn into the spinal canal, when compared with a placebo.

The Phase 3 trials chief goal is to confirm the effectiveness of NurOwn as measured by the amyotrophic lateral sclerosis functional rating scale (ALSFRS-R), a score of abilities such as swallowing, speech, handwriting, or walking.

Despite the COVID-19 pandemic, patient dosing in the trial is expected to be complete by next month. The study is fully enrolled with 200 participants across six U.S. sites. All patients have been given at least two of three doses. A summary of important findings will be announced later this year.

This critical research study involves one of the largest and most robust clinical trial collectors of [cerebrospinal fluid] biomarkers, said Chaim Lebovits, BrainStorm CEO. Data generated from this study will increase our understanding of how NurOwn therapy impacts ALS disease progression, and may identify patients who benefit the most from this form of therapy. We also hope that this research study will benefit the broader ALS community as we collectively advance toward our shared goal of delivering much-needed treatments, he said.

Danielle Carnival, CEO of I AM ALS, said the ALS community is at a pivotal time in terms of treatment research.

We need to move with urgency in all of our efforts to deliver treatments and cures for ALS, she said. This biomarker research will help us more expeditiously understand the effectiveness of NurOwn, while possibly unlocking discoveries that provide clues for other promising treatments.

Mary M. Chapman began her professional career at United Press International, running both print and broadcast desks. She then became a Michigan correspondent for what is now Bloomberg BNA, where she mainly covered the automotive industry plus legal, tax and regulatory issues. A member of the Automotive Press Association and one of a relatively small number of women on the car beat, Chapman has discussed the automotive industry multiple times of National Public Radio, and in 2014 was selected as an honorary judge at the prestigious Cobble Beach Concours dElegance. She has written for numerous national outlets including Time, People, Al-Jazeera America, Fortune, Daily Beast, MSN.com, Newsweek, The Detroit News and Detroit Free Press. The winner of the Society of Professional Journalists award for outstanding reporting, Chapman has had dozens of articles in The New York Times, including two on the coveted front page. She has completed a manuscript about centenarian car enthusiast Margaret Dunning, titled Belle of the Concours.

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Ana holds a PhD in Immunology from the University of Lisbon and worked as a postdoctoral researcher at Instituto de Medicina Molecular (iMM) in Lisbon, Portugal. She graduated with a BSc in Genetics from the University of Newcastle and received a Masters in Biomolecular Archaeology from the University of Manchester, England. After leaving the lab to pursue a career in Science Communication, she served as the Director of Science Communication at iMM.

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EHA25Virtual: Combined Irradiation and Chemotherapy Better Prepares Children for Stem Cell Transplantation than Chemotherapy Alone – P&T Community

By daniellenierenberg

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

Allogeneic HSCT is a multistep procedure:

For high-risk leukemia, the gold standard conditioning procedure is a combination of total body irradiation (TBI) and high dose chemotherapy. This approach is very effective in controlling leukemia in the conditioning step, but patients may experience highly negative consequences of this procedure later in life: sterility, growth retardation, lung problems, and secondary cancer.

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

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

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Westerleigh resident is alive because of stem cell therapy by his doctor — for free. Heres his story. – SILive.com

By daniellenierenberg

Stephen Raffone had difficulty breathing. He coughed up sputum and was wheezing. Doctors told him he had chronic obstructive pulmonary disease (COPD), a condition that causes blocked airflow from the lungs.

As a result, he was being treated for stage 4 COPD.

His doctor was also treating him for cellulitis, an inflammatory and painful bacterial skin infection where extremities appear red and swollen and the area can feel hot and tender to the touch, as well as poor circulation.

My legs were beginning to get ulcerated and they were breaking down, said Raffone.

He was administered the Roman Catholic Churchs Last Rites three times several years ago when he was a patient in Richmond University Medical Center.

Raffone, who is now 63, was in need of a lung transplant.

He was a heavy smoker and it took its toll. However, because he was in a weakened state due to other serious health conditions, doctors told him hed never survive the surgery.

The Westerleigh resident, who has been in need of 24-hour care for the last several years, requires the assistance of two nurses who rotate 12-hour shifts.

One, a close family friend, suggested Raffone see a medical specialist who performs stem cell therapy, a procedure where the patients own stem cells are removed, treated and returned to his or her own body after a conditioning regimen.

She contacted Dr. Alexandre M. Scheer of Scheer Medical Wellness and he agreed to see Raffone.

Dr. Alexandre M. Scheer (Courtesy/Stephen Raffone)Staten Island Advance

But since Raffone was unable to leave his home, Scheer visited Raffone for a consultation and to evaluate his condition.

Fast forward a year and a half and Scheer has continued with those visits almost every Saturday free of charge also underwriting the cost for treatments, as well as Uber rides from Manhattan to Staten Island, in order to perform the stem cell procedure.

RAFFONES NURSE SPEAKS

One of Raffones nurses recounted Raffones journey.

She explained that when they started to explore stem cell therapy she placed calls to several doctors, but the biggest thing that jumped out at her was the astronomical cost.

But there was something about Dr. Scheer. And I just knew he was the right one, said the registered nurse for more than 30 years. "He wasnt interested in money. His goal is his patients outcome. Stephen did pay for the first set of treatments, but since then, Dr. Scheer has not taken a dime.

When the patient began treatments, the first therapy was a tremendous boost and then every week after that he was treated for seven weeks. In the beginning, the doctor visited every week and brought whatever supplies was needed. The PRP (platelet rich plasma) treatments are daily.

I draw the blood, I spin the blood," she said. We have a small centrifuge here so it separates the blood. The PRP is given by a nebulizer. It takes about 30 minutes. And once a week he gets a protein enriched plasma, which takes about a half hour, she added.

He has chronic venous ulcerations of the both lower extremities from the knee down, she said.

Raffone has end stage COPD. But since he started the treatments, hes gone to the hospital only once. And he has tested negative for antibody COVID-19.

RAFFONES TREATMENT BEGINS

Raffone was required to install the centrifuge machine with needles and plasma tube, a laboratory device used for the separation of fluids, gas or liquid, based on density. Separation is achieved by spinning a vessel containing material at high speed.

Initially, Dr. Scheer sent a plastic surgeon to my home to perform liposuction, a type of fat-removal procedure used in plastic surgery, where they separate the fat and preserve the stem cells, Raffone said. They did this four times weekly at the beginning. Dr. Scheer has been visiting my home pretty much each week since Sept. 22, 2018. But right now the stem cell therapy is done once a month."

They draw blood out and spin it. Its all done through IV. Right now stem cell infusion is done once a month and daily through a nebulizer. Dr. Scheer does it on Saturday and my nurse and dear friend to Dr. Scheer does it during the week. My house looks like a hospital. Dr. Scheer is keeping me alive and everything is healing up so well, said Raffone.

Stephen Raffone's left leg before stem cell treatment. (Courtesy/Stephen Raffone)Staten Island Advance

Raffone says he wanted to come forward with his account at this time because hes so grateful and especially today when so many negative stories are in the news.

We need some good stories. There are very few people like Dr. Scheer, especially now during the COVID-19 crisis, he said.

My nurse draws the blood and puts it in a centrifuge when the doctor cant make it from the city. But Dr. Scheer is still coming to my house in spite of the COVID-19 crisis," Raffone continued.

Raffone has been confined to a bed one that he says turns you from side to side and upside down. But Dr. Scheer is confident that when restrictions are lifted and physical therapy sessions resume, Raffone will be able to walk.

The stem cell therapy is not only helping to combat Raffones COPD, but it has also helped him with cellulitis on his leg.

Stephen's Raffone healed left leg after stem cell therapy. (Courtesy/Stephen Raffone)Staten Island Advance

Scheer, a staunch supporter of stem cell therapy, has a background in neurosurgery and regenerative medicine. He performs surgery at several surgical centers in Manhattan.

It has to do with the amount of cells your bone marrow," he said. What we do is . . . saturate the body with stem cells. It suppresses the inflammatory response. COVID-19 also is an inflammatory disease. The COVID-19 kills the lungs. So you dont have oxygen going through. The stem cells protect, so you have continual oxygen transfer.

Dr. Scheer, who practices at Sheer Medical Wellness in Manhattan, says you can regenerate yourself.

I want my patients to be fine. I will pay for the patient. Im happy Stephens alive. And then my life is made. Stephen will now be able to walk after physical therapy. He was on 12 liters of oxygen daily. Hes now on two liters. I know his nurse very well and thats how we connected. The stem cell treatment is the appropriate treatment for him. I pay out of pocket because I know the right treatment for his condition," he added.

Dr. Scheer points out in China and in Israel stem cell therapy is the treatment they use for COVID- 19.

Its where you take Eastern and Western medicine and put it together. The patients body and will to live and having the right outlook on life has a lot to do with proper health. Our group is so big. We have 40 different doctors in my practice. Im the medical director, he said. Stem cell treatment is the future of medicine. At $10,000 a treatment, its very expensive. And the number depends on the issue at hand.

THE INITIAL CALL

When Scheer spoke to Raffone, He said I cant get out of bed,' the doctor said. "I drove to Staten Island and I got to know Stephen and his family very well. Its not a one-time treatment. Im seeing him on a weekly basis. There is a relationship that occurs. And thats what matters and thats what keeps people alive. Hope is what keeps them alive. And Im doing this since 2001. The treatment involves platelet enriched plasma that suppresses inflammatory reactions in the lungs. Whats happening is youre able to suppress the inflammatory reaction. His legs and his heart are getting better as well. This is a treatment until we can get him walking.

Scheer says Raffone must undergo physical therapy in oder for him to walk around freely.

And hell be able to travel to my office. Im not giving up on him. Im paying out of pocket. A quarter of my patients, I pay for. Stephen has gone through so much. Hes alive because of stem cell therapy. And due to his lung condition with COVID, he has not contracted it."

Scheer says its been a team effort, with multiple doctors coming into play.

Stephen is keeping himself alive. Im just the tool that can help. I just do the best I can for as many people as I can.

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New CRISPR, gene therapy results strengthen potential for treatment of blood diseases – BioPharma Dive

By daniellenierenberg

Three people with the inherited blood diseases sickle cell and beta thalassemia remain free of burdensome blood transfusions and their worst symptoms, months after receiving an infusion of genetically modified stem cells.

One of the three, a young woman with a severe form of beta thalassemia, has now been followed for over a year since she was treated, while the second, a woman in her 30s with sickle cell disease, is more than nine months removed from her infusion. They are the first two patients in pioneering studies of a therapy, developed by CRISPR Therapeutics and Vertex, that's based on the gene editing technology known as CRISPR.

Both patients continue to respond to treatment, bolstering evidence of genetic medicine's potential to permanently alter the course of devastating hereditary conditions like sickle cell and transfusion-dependent beta thalassemia. A gene therapy developed by Bluebird Bio has shown similar potential.

First results from the two studies, disclosed last November, were "taking the promise of CRISPR and turning that into a reality," said Samarth Kulkarni, CRISPR Therapeutics' CEO, in an interview. The additional data and follow-up now available "show these effects can be long-lasting and durable."

And in beta thalassemia, the first patient's experience is now supported by results from another patient who was treated about five months ago. This individual has also been able to stop receiving blood transfusions.

Taken together, the two patients responses are "proof of concept," CRISPR Therapeutics and Vertex claim, that their approach to treating beta thalassemia has the potential to be curative.

In sickle cell, the companies are also hopeful. The one patient for whom they have data has not had a vaso-occlusive crisis, a painful episode caused by the disease's characteristic sickling of red blood cells, since her treatment.

"The clinical manifestation of the disease is different, but we see consistent outcomes across both diseases," said Bastiano Sanna, Vertex's head of cell and genetic therapies, in an interview.

Three other beta thalassemia patients and one other sickle cell disease patient have been treated in the two studies of CRISPR Therapeutics and Vertex's therapy, dubbed CTX001. If results continue to look positive, CTX001 could be another powerful way to help people for whom treatment options have long been limited.

CRISPR, an easy-to-use method of genetic surgery that's derived from a bacterial defense system, has become a mainstay in labs across the world for all types of experiments. Its potential use as a human therapeutic has drawn closer as companies harnessing the technology CRISPR Therapeutics, Editas Medicine and Intellia Therapeutics have advanced their research. CRISPR Therapeutics is the first of the three to deliver results from a clinical trial.

CRISPR and Vertex unveiled their updated results at the European Hematology Association's virtual meeting on Friday. Also being presented were the latest data from Bluebird's gene therapy, known as LentiGlobin.

Bluebird is much further along, having treated 60 patients with beta thalassemia and 37 with sickle cell disease across six different studies.

Updated results from three of those studies showed 23 of 27 evaluable patients with beta thalassemia were transfusion independent for at least a year following treatment. And in sickle cell, no serious vaso-occlusive crises were observed in the 18 patients who had at least six months of follow-up. An episode was previously reported in one patient several months after LentiGlobin treatment, but was judged to be non-serious.

One sickle cell patient died suddenly 20 months following infusion with LentiGlobin, Bluebird reported Friday. Both the treating physician and an independent study committee concluded the death, ruled to be cardiovascular in nature, was unlikely to be related to the gene therapy.

Both beta thalassemia and sickle cell are diseases caused by mutations in the beta globin gene, faulty DNA that results in either absent or warped hemoglobin. Without enough hemoglobin, patients' red blood cells can't carry needed oxygen throughout the body. And those with sickle cell have abnormal hemoglobin that makes red blood cells fragile and stiff, causing them to stick in blood vessels.

Both diseases require chronic blood transfusions, and can lead to organ damage and reduced lifespans. Treatment options are limited, although that's now changing. The Food and Drug Administration, over the past few years, has approved Reblozyl, for beta thalassemia, and Oxbryta and Adakveo, for sickle cell.

Adakveo reduces the frequency of vaso-occlusive crises, while Reblozyl and Oxbryta are chronic medicines meant to boost patients' hemoglobin levels.

CRISPR Therapeutics and Vertex, along with Bluebird, are trying to accomplish the same goal but in more dramatic fashion: raising hemoglobin levels high enough so patients can stop blood transfusions and, in sickle cell, avoid pain crises altogether.

CRISPR and Vertex use CRISPR/cas9 gene editing to modify the DNA of stem cells extracted from a patient's bone marrow. The cells are engineered to produce a type of hemoglobin that's present at birth but normally replaced soon after. Once returned to the body and engrafted in the bone marrow, these CRISPR'd cells substitute this so-called fetal hemoglobin for the missing adult hemoglobin.

In the three patients treated so far, that appears to be what's happened. Both beta thalassemia patients are producing hemoglobin at levels considered normal. The sickle cell patient now has enough fetal hemoglobin to dilute the effects of sickled hemoglobin, potentially helping to preserve red blood cells.

Crucially, CRISPR and Vertex shared data for the first time indicating a high percentage of edited cells are present in each patient's bone marrow, supporting their confidence that the effects of treatment might last.

Bluebird, by contrast, doesn't edit the DNA of extracted stem cells, but rather inserts a modified gene into those cells. Once infused and engrafted in a patient, the cells can produce gene therapy-derived hemoglobin.

In most beta thalassemia and sickle cell patients treated with Bluebird's LentiGlobin, hemoglobin levels rose to normal or near-normal levels.

LentiGlobin is already approved for certain beta thalassemia patients in Europe as Zynteglo. In the U.S., Bluebird has hit delays and pushed back when it expects to submit an application to the middle of next year. A filing for an accelerated approval in sickle cell would likely follow sometime in the second half of 2021.

CRISPR and Vertex, meanwhile, plan to enroll more patients into their two studies, which they hope could serve as sufficient for an approval application if positive, Kulkarni said.

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New CRISPR, gene therapy results strengthen potential for treatment of blood diseases - BioPharma Dive

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Regenerative Therapy Options for Horses With Osteoarthritis – TheHorse.com

By daniellenierenberg

Biologic, or regenerative, therapies have altered the way many equine veterinarians treat problematic joints. Some of the most mainstream and popular modalities they currently use to manage osteoarthritis (OA) in horses are autologous conditioned serum, autologous protein solution, platelet-rich plasma, and mesenchymal stem cells.

Most biologic therapies involve collecting and concentrating the horses natural anti-inflammatory and regenerative proteins or cells so they can be injected into an area of pathology (disease or damage) in the same horse.

Autologous conditioned serum is a cell-free extract of whole blood that has been processed to contain high concentrations of interleukin-1 receptor antagonist protein (IRAP), a naturally occurring anti-inflammatory protein within the body. It is marketed under the trade names IRAP and IRAP II.

When preparing ACS, veterinarians collect venous blood in a proprietary syringe system that encourages porous glass beads to bind with white blood cells. During an incubation process the bound white cells release high concentrations of IRAP. The veterinarian then draws the serum off into small portions and freezes it for future injection into arthritic joints. In clinical studies of ACS, researchers have reported improved synovial membrane (joint surface lining) health, stimulation of natural IRAP production, and improved lameness.

Platelet rich plasma is blood plasma thats been centrifuged or filtrated to have a higher concentration of platelets than whole blood. Many horse owners are familiar with PRP and its use in tendon and ligament injuries; however, veterinarians are using it more regularly for treating joint disease.

One of platelets roles in the body is to modulate tissue healing. They do so by releasing growth factors and signaling molecules that initiate repair and promote anabolic (supporting tissue growth) effects. Veterinarians have capitalized on this ability by injecting high concentrations of platelets directly into damaged or inflamed regions. Because many PRP systems allow for stallside preparation, it is a convenient option for immediate treatment without the hassle of incubation or culturing in the lab, as is the case with ACS and stem cell preparation, respectively.

Historically, equine veterinarians have primarily used PRP to help treat soft tissue injuries. More recent work has led to intra-articular (in the joint) use with promising results. Although researchers have demonstrated how platelet-derived products work in vitro (in the lab) and veterinarians have seen promising anecdotal results in vivo (in the live horse), theyve yet to produce evidence-based confirmation of its clinical efficacy.

Mark Revenaugh, DVM, owner of Northwest Equine Performance, in Mulino, Oregon, says the main factors standing between researchers ability to gather objective data and establish a consensus on PRPs efficacy are the high variability among preparation systems, individual patient reactivity to the product, and an unknown ideal concentration of platelets for particular injuries.

Most practitioners cant always check how many platelets are being used, he says. Depending on the system, one veterinarian may be using 100,000 platelets/milliliter and another veterinarian may be using 1 billion platelets/milliliter. These are not the same treatments, even though both are called PRP. I would love to see an industry standard develop.

Overall, PRPs positive anecdotal results and relatively easy preparation make it a useful option for treating osteoarthritis (OA) in horses.

Autologous protein solution (marketed under the trade name Pro-Stride) is essentially a hybrid of ACS and PRP. Its two-step stallside preparation process involves separating whole blood and sequestering white blood cells and platelets in a small fraction of plasma. The veterinarian then concentrates the separate blood components by filtration, leaving a solution of white blood cells, platelets, and serum proteins that provides the anti-inflammatory mediators of IRAP and the platelet-derived growth factors of PRP.

In a 2014 study out of The Ohio State University, researchers revealed that an intra-articular APS injection can significantly improve lameness, weight-bearing symmetry, and range of joint motion in horses that dont have severe lameness or significant compromise to the joint structure.

Mesenchymal stems cells are adult stem cells that can direct regeneration and repair of damaged tissue. Veterinarians have used this type of stem cell as a treatment strategy for equine soft tissue injury for some time; its only recently that veterinarians have begun using them to treat OA, and its not fully clear how they work in this capacity. Researchers working on early stem cell studies hoped to establish evidence that stem cells injected into regions of injury would develop into the respective tissue. While this hypothesis proved to be incorrect, continued research has revealed that these cells might instead have anti-inflammatory effects and the ability to recruit other stem cells to the area that could, in fact, heal damaged tissue.

The two most common forms of mesenchymal stem cells are adipose (fat)-derived and bone-marrow-derived. Some study results have shown that bone marrow sources yield smaller concentrations thanbut are superior toadipose sources in their ability to differentiate into musculoskeletal tissue. Some encouraging data supporting the use of mesenchymal stem cells for treating OA exists, but researchers have only published a small number of studies with promising results. Equine veterinarians have used MSCs to treat intra-articular soft tissue injury (meniscal and cruciate damagecartilaginous tissues and ligaments that support the stifle), with successful anecdotal results. Theyve reported more variable outcomes when using it for primary intra-articular injuries.

Carter Judy, DVM, Dipl. ACVS, staff surgeon at Alamo Pintado Equine Medical Center, in Los Olivos, California, says he currently prefers to use PRP and APS for OA treatment over MSCs. However, he admits there is much to be discovered. What will be interesting to see is how manipulating the cells and providing them with different signals and markers can make their efficacy much more potent and focused, he says.

When weighing treatment options for horses with OA, veterinarians should base their decision to use a certain biologic modality on its cost, availability, and how a horse has responded previously.

Our knowledge base of how the biologics work is improving, but we are in the infancy of understanding, Judy says. Much of the use is based on the clinical response as much as is it on the scientific data.

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

By daniellenierenberg

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

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

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

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

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

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

Northstar-2 (HGB-207) Efficacy

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

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

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

Analysis of Healthy Red Blood Cell Production

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

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

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

Northstar-3 (HGB-212) Efficacy

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

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

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

Betibeglogene autotemcel Safety

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

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

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

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

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

About betibeglogene autotemcel

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

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

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

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

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

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

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

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

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

About bluebird bio, Inc.

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

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

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

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

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

bluebird bio Forward-Looking Statements

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

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

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COVID 19 to Lead the Sales of Myelofibrosis Treatment to Register Stellar Growth in the Next 10 Years – Cole of Duty

By daniellenierenberg

Myelofibrosis or osteomyelofibrosis is a myeloproliferative disorder which is characterized by proliferation of abnormal clone of hematopoietic stem cells. Myelofibrosis is a rare type of chronic leukemia which affects the blood forming function of the bone marrow tissue. National Institute of Health (NIH) has listed it as a rare disease as the prevalence of myelofibrosis in UK is as low as 0.5 cases per 100,000 population. The cause of myelofibrosis is the genetic mutation in bone marrow stem cells. The disorder is found to occur mainly in the people of age 50 or more and shows no symptoms at an early stage. The common symptoms associated with myelofibrosis include weakness, fatigue, anemia, splenomegaly (spleen enlargement) and gout. However, the disease progresses very slowly and 10% of the patients eventually develop acute myeloid leukemia. Treatment options for myelofibrosis are mainly to prevent the complications associated with low blood count and splenomegaly.

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The global market for myelofibrosis treatment is expected to grow moderately due to low incidence of a disease. However, increasing incidence of genetic disorders, lifestyle up-gradation and rise in smoking population are the factors which can boost the growth of global myelofibrosis treatment market. The high cost of therapy will the growth of global myelofibrosis treatment market.

The global market for myelofibrosis treatment is segmented on basis of treatment type, end user and geography:

As myelofibrosis is considered as non-curable disease treatment options mainly depend on visible symptoms of a disease. Primary stages of the myelofibrosis are treated with supportive therapies such as chemotherapy and radiation therapy. However, there are serious unmet needs in myelofibrosis treatment market due to lack of disease modifying agents. Approval of JAK1/JAK2 inhibitor Ruxolitinib in 2011 is considered as a breakthrough in myelofibrosis treatment. Stem cell transplantation for the treatment of myelofibrosis also holds tremendous potential for market growth but high cost of therapy is foreseen to limits the growth of the segment.

On the basis of treatment type, the global myelofibrosis treatment market has been segmented into blood transfusion, chemotherapy, androgen therapy and stem cell or bone marrow transplantation. Chemotherapy segment is expected to contribute major share due to easy availability of chemotherapeutic agents. Ruxolitinib is the only chemotherapeutic agent approved by the USFDA specifically for the treatment of myelofibrosis, which will drive the global myelofibrosis treatment market over the forecast period.

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Geographically, global myelofibrosis treatment market is segmented into five regions viz. North America, Latin America, Europe, Asia Pacific and Middle East & Africa. Northe America is anticipated to lead the global myelofibrosis treatment market due to comparatively high prevalence of the disease in the region.

Some of the key market players in the global myelofibrosis treatment market are Incyte Corporation, Novartis AG, Celgene Corporation, Mylan Pharmaceuticals Ulc., Bristol-Myers Squibb Company, Eli Lilly and Company, Taro Pharmaceuticals Inc., AllCells LLC, Lonza Group Ltd., ATCC Inc. and others.

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Market Analysis and Technological Opportunities of Cell Therapy Manufacturing Market till 2030 – Medic Insider

By daniellenierenberg

Prophecy Market Insights Cell Therapy Manufacturing market research report provides a comprehensive, 360-degree analysis of the targeted market which helps stakeholders to identify the opportunities as well as challenges. The research report study offers keen competitive landscape analysis including key development trends, accurate quantitative and in-depth commentary insights, market dynamics, and key regional development status forecast 2020-2029. It incorporates market evolution study, involving the current scenario, growth rate, and capacity inflation prospects, based on Porters Five Forces and DROT analyses.

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An executive summary provides the markets definition, application, overview, classifications, product specifications, manufacturing processes; raw materials, and cost structures.

Market Dynamics offers drivers, restraints, challenges, trends, and opportunities of the Cell Therapy Manufacturing market

Segment Level Analysis in terms of types, product, geography, demography, etc. along with market size forecast

Regional and Country- level Analysis different geographical areas are studied deeply and an economical scenario has been offered to support new entrants, leading market players, and investors to regulate emerging economies. The top producers and consumers focus on production, product capacity, value, consumption, growth opportunity, and market share in these key regions, covering

The comprehensive list of Key Market Players along with their market overview, product protocol, key highlights, key financial issues, SWOT analysis, and business strategies. The report dedicatedly offers helpful solutions for players to increase their clients on a global scale and expand their favour significantly over the forecast period. The report also serves strategic decision-making solutions for the clients.

Competitive landscape Analysis provides mergers and acquisitions, collaborations along with new product launches, heat map analysis, and market presence and specificity analysis.

Segmentation Overview:

Cell Therapy ManufacturingMarket Key Companies:

harmicell, Merck Group, Dickinson and Company, Thermo Fisher, Lonza Group, Miltenyi Biotec GmBH, Takara Bio Group, STEMCELL Technologies, Cellular Dynamics International, Becton, Osiris Therapeutics, Bio-Rad Laboratories, Inc., Anterogen, MEDIPOST, Holostem Terapie Avanazate, Pluristem Therapeutics, Brammer Bio, CELLforCURE, Gene Therapy Catapult EUFETS, MaSTherCell, PharmaCell, Cognate BioServices and WuXi AppTec.

The Cell Therapy Manufacturing research study comprises 100+ market data Tables, Graphs & Figures, Pie Chat to understand detailed analysis of the market. The predictions estimated in the market report have been resulted in using proven research techniques, methodologies, and assumptions. This Cell Therapy Manufacturing market report states the market overview, historical data along with size, growth, share, demand, and revenue of the global industry.

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The study analyses the manufacturing and processing requirements, project funding, project cost, project economics, profit margins, predicted returns on investment, etc. This report is a must-read for investors, entrepreneurs, consultants, researchers, business strategists, and all those who have any kind of stake or are planning to foray into the Cell Therapy Manufacturing industry in any manner.

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Market Analysis and Technological Opportunities of Cell Therapy Manufacturing Market till 2030 - Medic Insider

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Aprea Therapeutics Presents Results From French Phase Ib/II Clinical Trial of APR-246 (Eprenetapopt) and Azacitidine in Patients with TP53 Mutant…

By daniellenierenberg

BOSTON, June 12, 2020 (GLOBE NEWSWIRE) -- Aprea Therapeutics, Inc.(Nasdaq: APRE), a biopharmaceutical company focused on developing and commercializing novel cancer therapeutics that reactivate mutant tumor suppressor protein, p53, today announced the oral presentation of updated data from its French Phase 1b/2 clinical trial at the 25th European Hematology Association Annual Meeting (EHA). The trial is evaluating the safety and efficacy of APR-246 (eprenetapopt) in combination with azacitidine (AZA) for the treatment of TP53 mutant myelodysplastic syndromes (MDS) and acute myeloid leukemia (AML). The clinical trial is sponsored by the Groupe Francophone des Mylodysplasies (GFM).

As of the April 1, 2020 data cutoff, the overall response rate (ORR) in 28 evaluable MDS patients was 75%, with a 57% complete remission (CR) rate, by International Working Group (IWG) criteria. With a median duration of follow-up of 9.7 months, the median overall survival (OS) for all enrolled patients (n=52) was 12.1 months and in MDS patients (n=34) was 12.1 months. For patients who remained on treatment for 3 or more cycles of treatment the median OS was higher at 13.7 months versus 2.8 months for patients who were on treatment for fewer than 3 cycles. Relative to baseline, mutant TP53 variant allele frequency (VAF) was decreased in responding patients by 3 cycles of treatment, including 20 (51%) patients who achieved mutant TP53 negativity by next-generation sequencing (NGS).

The data from this ongoing trial of eprenetapopt with azacitidine continue to be very encouraging in these most difficult-to-treat TP53 mutant MDS and AML patients, who not only have at least one TP53 mutation but the majority of whom also have high risk cytogenetic abnormalities, said Thomas Cluzeau, M.D., co-lead investigator for the GFM trial. We continue to observe ORR and CR rates in these patients that are substantially higher than the GFMs experience with azacitidine monotherapy. Furthermore, with increased duration of follow-up, we now also see the emergence of highly encouraging overall survival that appears to be better than azacitidine alone or in combination with others agents in this very high-risk molecular group of patients with a TP53 mutation.

Details of the on-demand oral presentation are as follows:

Title: APR-246 Combined with Azacitidine in TP53 Mutated Myelodysplastic Syndromes (MDS) and Acute Myeloid Leukemia. A Phase 2 Study by the Groupe Francophone des Mylodysplasies (GFM)

Oral Abstract Session: Novel treatments for MDS I

Abstract: S181

About the Clinical Trial

Eligible patients in the Phase Ib/II clinical trial include hypomethylating agent (HMA) nave, TP53 mutated MDS and AML. All enrolled patients were to receive APR-246 as a 4,500 mg fixed dose IV daily for 4 days and AZA over 7 days in 28-day cycles. The primary endpoint of the trial is CR rate.

AboutAprea Therapeutics, Inc.

Aprea Therapeutics, Inc.is a biopharmaceutical company headquartered inBoston, Massachusettswith research facilities inStockholm, Sweden, focused on developing and commercializing novel cancer therapeutics that reactivatemutant tumor suppressor protein, p53. The Companys lead product candidate is APR-246 (eprenetapopt), a small molecule in clinical development for hematologic malignancies, including myelodysplastic syndromes (MDS) and acute myeloid leukemia (AML). APR-246 has received Breakthrough Therapy, Orphan Drug and Fast Track designations from the FDA for MDS, and Orphan Drug designation from the European Commission for MDS, AML and ovarian cancer. For more information, please visit the company website atwww.aprea.com.

The Company may use, and intends to use, its investor relations website at https://ir.aprea.com/ as a means of disclosing material nonpublic information and for complying with its disclosure obligations under Regulation FD.

About Myelodysplastic Syndromes

Myelodysplastic syndromes (MDS) represents a spectrum of hematopoietic stem cell malignancies in which bone marrow fails to produce sufficient numbers of healthy blood cells. Approximately 30-40% of MDS patients progress to acute myeloid leukemia (AML) and mutation of the p53 tumor suppressor protein is thought to contribute to disease progression. Mutations in p53 are found in up to 20% of MDS and AML patients and are associated with poor overall prognosis.

About p53 and APR-246 (eprenetapopt)

The p53 tumor suppressor gene is the most frequently mutated gene in human cancer, occurring in approximately 50% of all human tumors. These mutations are often associated with resistance to anti-cancer drugs and poor overall survival, representing a major unmet medical need in the treatment of cancer.

APR-246 (eprenetapopt) is a small molecule that has demonstrated reactivation of mutant and inactivated p53 protein by restoring wild-type p53 conformation and function and thereby induce programmed cell death in human cancer cells. Pre-clinical anti-tumor activity has been observed with APR-246 in a wide variety of solid and hematological cancers, including MDS, AML, and ovarian cancer, among others. Additionally, strong synergy has been seen with both traditional anti-cancer agents, such as chemotherapy, as well as newer mechanism-based anti-cancer drugs and immuno-oncology checkpoint inhibitors. In addition to pre-clinical testing, a Phase 1/2 clinical program with APR-246 has been completed, demonstrating a favorable safety profile and both biological and confirmed clinical responses in hematological malignancies and solid tumors with mutations in the TP53 gene.

Forward-Looking StatementCertain information contained in this press release includes forward-looking statements, within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended, related to our clinical trials, regulatory submissions and projected cash position. We may, in some cases use terms such as predicts, believes, potential, continue, anticipates, estimates, expects, plans, intends, targeting, confidence, may, could, might, likely, will, should or other words that convey uncertainty of the future events or outcomes to identify these forward-looking statements. Our forward-looking statements are based on current beliefs and expectations of our management team that involve risks, potential changes in circumstances, assumptions, and uncertainties. Any or all of the forward-looking statements may turn out to be wrong or be affected by inaccurate assumptions we might make or by known or unknown risks and uncertainties. These forward looking statements are subject to risks and uncertainties including risks related to the success and timing of our clinical trials or other studies, risks associated with the coronavirus pandemic and the other risks set forth in our filings with theU.S. Securities and Exchange Commission. For all these reasons, actual results and developments could be materially different from those expressed in or implied by our forward-looking statements. You are cautioned not to place undue reliance on these forward-looking statements, which are made only as of the date of this press release. We undertake no obligation to publicly update such forward-looking statements to reflect subsequent events or circumstances.

Source:Aprea Therapeutics, Inc.

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Aprea Therapeutics Presents Results From French Phase Ib/II Clinical Trial of APR-246 (Eprenetapopt) and Azacitidine in Patients with TP53 Mutant...

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Bone Marrow Processing Systems Market : Industry Trends and Developments 2018 2025 – Cole of Duty

By daniellenierenberg

Bone marrow aspirationand trephine biopsy are usually performed on the back of the hipbone, or posterior iliac crest. An aspirate can also be obtained from the sternum (breastbone). For the sternal aspirate, the patient lies on their back, with a pillow under the shoulder to raise the chest. A trephine biopsy should never be performed on the sternum, due to the risk of injury to blood vessels, lungs or the heart.

The need to selectively isolate and concentrate selective cells, such as mononuclear cells, allogeneic cancer cells, T cells and others, is driving the market. Over 30,000 bone marrow transplants occur every year. The explosive growth of stem cells therapies represents the largest growth opportunity for bone marrow processing systems.

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Europe and North America spearheaded the market as of 2018, by contributing over 74.0% to the overall revenue. Majority of stem cell transplants are conducted in Europe, and it is one of the major factors contributing to the lucrative share in the cell harvesting system market.

In 2018, North America dominated the research landscape as more than 54.0% of stem cell clinical trials were conducted in this region. The region also accounts for the second largest number of stem cell transplantation, which is further driving the demand for harvesting in the region.

Asia Pacific is anticipated to witness lucrative growth over the forecast period, owing to rising incidence of chronic diseases and increasing demand for stem cell transplantation along with stem cell-based therapy. Japan and China are the biggest markets for harvesting systems in Asia Pacific.

Emerging countries such as Mexico, South Korea, and South Africa are also expected to report lucrative growth over the forecast period. Growing investment by government bodies on stem cell-based research and increase in aging population can be attributed to the increasing demand for these therapies in these countries.

Major players operating in the global bone marrow processing systems market are ThermoGenesis (Cesca Therapeutics inc.), RegenMed Systems Inc., MK Alliance Inc., Fresenius Kabi AG, Harvest Technologies (Terumo BCT), Arthrex, Inc. and others.

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Bone Marrow Processing Systems Market : Industry Trends and Developments 2018 2025 - Cole of Duty

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EHA25Virtual: Combined Irradiation and Chemotherapy Better Prepares Children for Stem Cell Transplantation than Chemotherapy Alone – PR Newswire UK

By daniellenierenberg

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

Allogeneic HSCT is a multistep procedure:

For high-risk leukemia, the gold standard conditioning procedure is a combination of total body irradiation (TBI) and high dose chemotherapy. This approach is very effective in controlling leukemia in the conditioning step, but patients may experience highly negative consequences of this procedure later in life: sterility, growth retardation, lung problems, and secondary cancer.

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

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

Embargo: Please note that our embargo policy applies to all selected abstracts in the Press Briefings. For more information click here.

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SOURCE European Hematology Association (EHA)

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EHA25Virtual: Combined Irradiation and Chemotherapy Better Prepares Children for Stem Cell Transplantation than Chemotherapy Alone - PR Newswire UK

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BrainStorm to Present at the Raymond James Human Health Innovations Conference – Yahoo Finance

By daniellenierenberg

Management to Host One-on-One Investment Meetings

NEW YORK, June 11, 2020 /PRNewswire/ --BrainStorm Cell Therapeutics Inc.(NASDAQ: BCLI), a leading developer of adult stem cell therapies for neurodegenerative diseases, today announced Chaim Lebovits, CEO and Ralph Kern, MD, MHSc, President and Chief Medical Officer, will present a corporate overview on Thursday, June 18 at 9:00 am EST, during theRaymond James Human Health Innovations Conference, a virtual event connecting institutional investors with company management teams that will be held June 15-18, 2020.

Mr. Lebovits and Dr. Kern will update conference participants on the Company's investigational therapeutic, NurOwn, that is currently in a fully enrolled phase 3 study for the treatment of ALS and a phase 2 study for the treatment of progressive multiple sclerosis. Additionally, they will present an overview of the Company's financial position and pipeline. After the presentation, the management team will participate in a question and answer session with institutional investors.

Mr. Lebovits and Dr. Kern will be joined by David Setboun, PhD, MBA, Chief Operating Officer, Stacy Lindborg, PhD, Head of Global Clinical Research, and Preetam Shah, PhD, MBA, Chief Financial Officer, for a series of one-on-one meetings, with select institutional investors arranged by Raymond James.

Participants can view the presentation via the event link and those unable to join will have access to an archived link on the Company's Events and Presentation webpage after the conclusion of the conference.

EVENT: Raymond James Human Health Innovations Conference

PRESENTATION: Thursday, June 18th at 9:00 am EST

LINK: https://bit.ly/2YmZf8u

About NurOwn

NurOwn (autologous MSC-NTF) cells represent a promising investigational therapeutic 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. BrainStorm has fully enrolled a Phase 3 pivotal trial of autologous MSC-NTF cells for the treatment of amyotrophic lateral sclerosis (ALS). BrainStorm also recently receivedU.S.FDA acceptance to initiate a Phase 2 open-label multicenter trial in progressive MS and enrollment began inMarch 2019.

AboutBrainStorm 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 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 theU.S. Food and Drug Administration(U.S.FDA) and theEuropean Medicines Agency(EMA) in ALS. BrainStorm has fully enrolled a Phase 3 pivotal trial in ALS (NCT03280056), investigating repeat-administration of autologous MSC-NTF cells at sixU.S.sites supported by a grant from theCalifornia Institute for Regenerative Medicine(CIRM CLIN2-0989). The pivotal study is intended to support a filing forU.S.FDA approval of autologous MSC-NTF cells in ALS. BrainStorm also recently receivedU.S.FDA clearance to initiate a Phase 2 open-label multicenter trial in progressive Multiple Sclerosis. The Phase 2 study of autologous MSC-NTF cells in patients with progressive MS (NCT03799718) started enrollment inMarch 2019.

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Safe-Harbor Statement

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 causeBrainStorm Cell Therapeutics Inc.'sactual 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, BrainStorm's need to raise additional capital, BrainStorm's ability to continue as a going concern, regulatory approval of BrainStorm's NurOwn treatment candidate, the success of BrainStorm's 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 BrainStorm's NurOwn treatment candidate to achieve broad acceptance as a treatment option for ALS or other neurodegenerative diseases, BrainStorm's ability to manufacture and commercialize the NurOwn treatment candidate, obtaining patents that provide meaningful protection, competition and market developments, BrainStorm's 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 athttp://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

Investor Relations:Preetam Shah, MBA, PhDChief Financial OfficerBrainStorm Cell Therapeutics Inc.Phone: +1-862-397-1860pshah@brainstorm-cell.com

Media:

Sean LeousWestwicke/ICR PRPhone: +1-646-677-1839sean.leous@icrinc.com

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SOURCE Brainstorm Cell Therapeutics Inc

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BrainStorm to Present at the Raymond James Human Health Innovations Conference - Yahoo Finance

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