Here’s what to know about Sickle Cell Disease in kids – Loma Linda University Health

With September being Sickle Cell Disease Awareness Month, Loma Linda University Childrens Health wants to help educate the community about SCD one of the most common yet overlooked genetic disorders in the world.

Each year, approximately 1,000 babies in the U.S. and 500,000 worldwide are born with the disease, according to the Sickle Cell Disease Association of America.

Akshat Jain, MD, MPH, a global sickle cell disease expert at Childrens Hospital, is passionate about establishing awareness and proper care for children suffering from SCD and Sickle Cell Trait, especially the diverse patient population in San Bernardino County.

There are many barriers to receiving care for those with SCD in our community, Jain says. One barrier specifically is lack of awareness surrounding the disease coupled with lack of awareness surrounding the treatment options available at Childrens Hospital.

In sickle cell disease, a persons red blood cells have an irregular cell shape, Jain says. Instead of round discs, theyre in a crescent or sickle shape.

Due to their shape, texture and inflexibility, the cells become clumped together. This grouping causes a blockage in a childs blood vessels, hindering blood-flow. This blockage may cause varying levels of pain and potentially organ damage long-term.

Jain says some of the signs and symptoms of SCD include:

Jain says that many children with SCD develop symptoms in their first year of life. SCD is commonly diagnosed during newborn screening tests, which check for the abnormal hemoglobin found in SCD. Additionally, if both parents of a child are known carriers of a SCD trait, their child will have a 25% chance of having the disease, Jain says.

Some of the emergent issues needing immediate medical care in kids with SCD disease include:

Treatments for SCD include pain medicines for pain management, adequate hydration, blood transfusions, vaccines and antibiotics, and some medicines. Currently, stem cell transplant from bone marrow is the recognized cure for SCD.

Childrens Hospital, with Jain working as a lead on the team, performed the institutions first stem cell transplant in 2019, curing a then 11-year-old girl who had suffered from SCD since birth. Since then, the team has successfully performed the transplant on several pediatric patients.

Patients with SCD at Childrens Hospital are placed into a treatment and care program where Jain and his team offer non-traditional services such as individualized patient treatment plans and direct access to the care team in case of an emergent event. Additionally, the program is working toward offering curative gene therapy for both sickle cell and hemophilia patients.

The bottom line is children and families suffering from this disease need to know that theyre not alone, Jain says. Here at Childrens Hospital, we are here to manage and fight this disease alongside of you.

Learn more about our treatments for sickle cell disease at our Specialty Team Centers.

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Here's what to know about Sickle Cell Disease in kids - Loma Linda University Health

Improved Induction Therapies Could Eliminate the Need for Transplant in Myeloma – OncLive

As induction therapies continue to improve the depth of response (DOR) in patients with multiple myeloma, it may be possible to eliminate the need forautologous stem cell transplant (ASCT) in this population, provided that deep responses with up-front therapy can be achieved, according to Jeffrey Wolf, MD.

There are a lot of reasons I would like to get rid ofautologous stem cell transplants, Wolf said. Newer drugs and newer regimens are going to allow us to get there, [provided] we [conduct] the right studies.

There has been notable growth of induction therapies within the myeloma space, particularly with regard to triplet regimens, such as lenalidomide (Revlimid), bortezomib (Velcade), and dexamethasone (RVd), as well as carfilzomib (Kyprolis), lenalidomide, and dexamethasone (KRd). Daratumumab (Darzalex) is also making great progress by quickly moving to the frontline setting for patients who are transplant ineligible, as well as for older patients who appear to tolerate the agent well, Wolf explained.

Its possible that as our induction therapies improve over the next few years, we may be able to eliminateautologous stem cell transplantation as a form ofconsolidationif we [can] get deep responses, such as minimal residual disease (MRD) [negativity] with just our initial induction therapy, said Wolf.

In an interview with OncLive, Wolf, a clinical professor within the Department of Medicine at University of California, San Francisco (UCSF) and director of the Myeloma Program at the UCSF Helen Diller Family Comprehensive Cancer Center, discusses treatment options for patients with myeloma, in addition to how MRD can be used to inform clinical decisions and improve patient outcomes.

OncLive: Could you discuss the evolution of induction therapy in multiple myeloma?

Wolf: Induction therapy for myeloma has evolved tremendously over the past few years, [especially] triplet induction [regimens, such as] RVd or KRd,based on recent studies. Daratumumab is certainly making a rapid move to be included in [frontline] therapy, especially in patients who are not eligible for transplant. It seems [that if the agent is] appropriate for them, it could be appropriate for younger patients, as well, but we dont have an FDA approval [in younger patients yet].

The goal [of treatment] is DOR, [which] is measured byMRD.

What are some of the strategies that are being used for transplant-eligible and -ineligible patients with newly diagnosed disease?

One of the main studies I presented [during my talk] was the ENDURANCE trial, which was just presented at the 2020 American Society of Clinical Onccology Virtual Meeting. The trial was what we thought of as somewhat of a flawed study that [claimed] RVd and KRd were equivalent for progression-freesurvival (PFS) [in transplant-eligible patients]. The problem is that most patients move on to transplant. What we really should have been looking at was KRd plus transplant versus RVd plus transplant. [The studys design] eliminated patients from the analysis when they underwent transplant.

In the transplant-ineligible setting, there are a lot more studies to refer to, including the studies that moved daratumumab to the frontline setting. Initially, we learned that maintenance lenalidomide was necessary even in patients who are not posttransplant. Another study showed thatdaratumumab added to bortezomib, melphalan, and prednisone(VMP) really improved PFS and overall survival (OS). Of course, [there is also the study that evaluated]daratumumab plus lenalidomide and dexamethasone (Rd) versusRd, [whereby] daratumumab adds tremendous depth and durability.

This morning I put 2 older patients on that regimen. They tolerateddaratumumab quite well and can get a DOR that is equivalent to that of a patient who undergoes transplant.

How are you navigating among the agents that are currently available in practice?

It seems complicated because there are so many options, but it tends to sort itself out by circumstance. For example, this morning I saw a transplant-ineligible patient who didnt getMedicare Part D and, therefore, I couldnt give them oral therapies such aslenalidomide. Their referring doctor gave them cyclophosphamide, bortezomib, and dexamethasone (CyBorD), which is all [intravenous]. This morning I decided to switch them tosubcutaneous daratumumab, bortezomib,dexamethasone.

Most of these regimens will give you the same responses. I often start with preexisting conditions to decide which drugs I eliminate and which drugs Im going to use instead.

What is the role of transplant in this space right now? Will transplant retain its role in future?

I started out as a transplanter 40 years ago, so its hard to say this, but Ive been thinking for the past decade that were on the verge of getting rid ofautologous stem cell transplant from myeloma. If our induction therapies are so effective, we may be able to eliminate transplant. Id like to see us conduct trials, such as the MASTER trial, led by Luciano Costa, MD, PhD, of the University of Alabamas Birmingham School of Medicine, in which patients who achieve MRD [negativity] with induction therapy do not proceed to transplant. Were going to have to do those kinds of studies to eliminate transplant. Its a fairly primitive kind of therapy in that we give high-dose therapy to wipe out as much myeloma as we can, and in doing so wipe out vulnerable bone marrow. Then, we have to [transplant] frozen stem cells from the patient. [The alkylator] leads to increased risk of secondary malignancies. Patients lose their hair, they get sick for 2 or 3 months, and theyre immunocompromised, which is not a good thing to be these days.

How might MRD be used to optimize patient outcomes?

In the world of myeloma, experts are divided over whether to use MRD to make decisions. I happen to be in the group [in favor of MRD]. Its no different than usingmonoclonal spike (m spike) orlight chain measurements to make decisions. Were already using MRD in chronic lymphocytic leukemia andacute lymphocytic leukemia to make decisions. The only restriction in myeloma is that, so far, weve only been able to [evaluate MRD with] bone marrow and not blood. If we can start measuring MRD in myeloma [through] blood, it would be as commonly used as light chain or m spike measurements to make these decisions. Itll help us immeasurably because most of the time were getting patients into complete remission (CR)which it really isnt CRand then we keep them on regimens for maintenance.

For example, when we dont know what were accomplishing, [its worth asking], Are patients getting better? Are patients staying the same? Is the MRD going up while were treating patients with drugs that clearly arent working? Should we be switching therapies? For patients who are MRD [negative] year after year, is there any reason why we have them on these therapies that are so costly and have so many adverse effects (AEs) and secondary malignancies? There are all kinds of reasons as to why we should be using MRD for measurement. I would argue that the only reason why we arent using MRD is because we have to [evaluate] it in bone marrow, which is a little more uncomfortable compared with blood, and maybe because the studies havent been completed yet. Its only a matter of 1 or 2 years before we will be using MRD to make clinical decisions. In terms of frontline therapies, using MRD might allow us to [avoid] transplant or indicate when weve given enough treatment and is time to move to maintenance. There a lot of possibilities to using MRD instead of just historically saying, this is how we do it.

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Improved Induction Therapies Could Eliminate the Need for Transplant in Myeloma - OncLive

Incyte and MorphoSys to Host Investor Event to Discuss the Unmet Need and Global Opportunities for Tafasitamab in Non-Hodgkin Lymphomas – Business…

WILMINGTON, Del. & PLANEGG & MUNICH, Germany--(BUSINESS WIRE)--Incyte (Nasdaq:INCY) and MorphoSys AG (FSE: MOR; Prime Standard Segment; MDAX & TecDAX; NASDAQ:MOR) today announced that the companies intend to host a conference call and webcast to discuss global development, unmet need and commercial opportunities for tafasitamab.

Dr. Gilles Salles will join Incyte and MorphoSys leadership as an expert speaker. Dr. Salles was the principal investigator and first author of the ICML 2019 and EHA 2020 data presentations, as well as first author of the 2020 Lancet Oncology publication of the L-MIND trial investigating tafasitamab in combination with lenalidomide for the treatment of adult patients with relapsed or refractory diffuse large B-cell lymphoma.

The conference call and webcast will be held on Tuesday, September 29, 2020 from 9:00 11:00 a.m. EDT / 3:00 5:00 p.m. CEST. The live webcast and replay will be available via http://www.morphosys.com and investor.incyte.com.

To access the conference call, U.S. domestic callers please dial 877-423-0830. Callers outside of the U.S. please dial +49 69201744220 or +44 2030092470. When prompted, provide the conference pin number, 83557299#.

About Tafasitamab

Tafasitamab is a humanized Fc-modified cytolytic CD19 targeting monoclonal antibody. In 2010, MorphoSys licensed exclusive worldwide rights to develop and commercialize tafasitamab from Xencor, Inc. Tafasitamab incorporates an XmAb engineered Fc domain, which mediates B-cell lysis through apoptosis and immune effector mechanism including antibody-dependent cell-mediated cytotoxicity (ADCC) and antibody-dependent cellular phagocytosis (ADCP).

Monjuvi (tafasitamab-cxix) is approved by the U.S. Food and Drug Administration in combination with lenalidomide for the treatment of adult patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL) not otherwise specified, including DLBCL arising from low grade lymphoma, and who are not eligible for autologous stem cell transplant (ASCT).This indication is approved under accelerated approval based on overall response rate. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s).

In January 2020, MorphoSys and Incyte entered into a collaboration and licensing agreement to further develop and commercialize tafasitamab globally. Monjuvi is being co-commercialized by Incyte and MorphoSys in the United States. Incyte has exclusive commercialization rights outside the United States.

A marketing authorization application (MAA) seeking the approval of tafasitamab in combination with lenalidomide in the EU has been validated by the European Medicines Agency (EMA) and is currently under review for the treatment of adult patients with relapsed or refractory DLBCL, including DLBCL arising from low grade lymphoma, who are not candidates for ASCT.

Tafasitamab is being clinically investigated as a therapeutic option in B-cell malignancies in a number of ongoing combination trials.

Monjuvi is a registered trademark of MorphoSys AG.

XmAb is a registered trademark of Xencor, Inc.

Important Safety Information

What are the possible side effects of MONJUVI?

MONJUVI may cause serious side effects, including:

The most common side effects of MONJUVI include:

These are not all the possible side effects of MONJUVI.

Call your doctor for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088.

Before you receive MONJUVI, tell your healthcare provider about all your medical conditions, including if you:

You should also read the lenalidomide Medication Guide for important information about pregnancy, contraception, and blood and sperm donation.

Tell your healthcare provider about all the medications you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements.

Please see the full Prescribing Information for Monjuvi, including Patient Information, for additional Important Safety Information.

About Incyte

Incyte is a Wilmington, Delaware-based, global biopharmaceutical company focused on finding solutions for serious unmet medical needs through the discovery, development and commercialization of proprietary therapeutics. For additional information on Incyte, please visit Incyte.com and follow @Incyte.

About MorphoSys

MorphoSys is a commercial-stage biopharmaceutical company dedicated to the discovery, development and commercialization of exceptional, innovative therapies for patients suffering from serious diseases. The focus is on cancer. Based on its leading expertise in antibody, protein and peptide technologies, MorphoSys, together with its partners, has developed and contributed to the development of more than 100 product candidates, 27 of which are currently in clinical development. In 2017, Tremfya, marketed by Janssen for the treatment of plaque psoriasis, became the first drug based on MorphoSys' antibody technology to receive regulatory approval. In July 2020 the U.S. Food and Drug Administration approved the companys proprietary product Monjuvi (tafasitamab-cxix) in combination with lenalidomide for the treatment of adult patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL) not otherwise specified, including DLBCL arising from low grade lymphoma, and who are not eligible for autologous stem cell transplant (ASCT). Headquartered near Munich, Germany, the MorphoSys group, including the fully owned U.S. subsidiary MorphoSys US Inc., has ~500 employees. More information at http://www.morphosys.com.

Tremfya is a registered trademark of Janssen Biotech.

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Incyte and MorphoSys to Host Investor Event to Discuss the Unmet Need and Global Opportunities for Tafasitamab in Non-Hodgkin Lymphomas - Business...

Jazz Pharmaceuticals Launches Initiative to Help Educate People Living with Rare Form of Leukemia – BioSpace

DUBLIN, Sept. 17, 2020 /PRNewswire/ -- Jazz Pharmaceuticals plc (Nasdaq: JAZZ) today announced the launch of Find the Right Fit (FindTheRightFit-sAML.com), a U.S. patient education program developed in consultation with the Myelodysplastic Syndromes (MDS) Foundation, Inc. and the Cancer Support Community focused on empowering people affected by secondary acute myeloid leukemia (sAML) and MDS.

Within the hematology oncology community, certain patient groups face especially difficult odds. In particular, patients newly diagnosed with sAML and related blood disorders have few resources focused on disease education and managing daily life that are dedicated to this community. As a subtype of AML, which has the lowest survival rate of all leukemias1, sAML has a particularly poor prognosis.2,3

"During Blood Cancer Awareness Month, we reflect on the unwavering strength of patients, caregivers, families and healthcare providers battling or impacted by blood cancer," said Kim Sablich, executive vice president and general manager of North America at Jazz Pharmaceuticals. "Find the Right Fit was created with the understanding that an sAML diagnosis can be overwhelming, but that education can help inform optimal treatment plans that work best for each individual patient."

Find the Right FitNagivating sAML offers patients and their caregivers a powerful collection of tools, including articles, videos, and patient stories, intended to educate on the science behind sAML, provide information on which subtypes patients should be tested for, and offer resources regarding treatment options and coping strategies. The program also shares the stories of patients, which are featured to help those impacted by sAML relate to the various experiences and perspectives that exist within the community.

"In addition to the support available through the Cancer Support Community and the Myelodysplastic Syndrome Foundation, Inc., we are pleased that Find the Right Fit adds resources and information to help inform and inspire people during this critical point in time," said Linda Bohannon, MSM,BSN,RN,president of the Cancer Support Community.

Making up approximately 30 percent of AML cases, sAML can spread quickly and requires a specialized treatment approach, which is why it is important that patients know what to discuss with their doctors in order to pursue the route best suited for their unique needs.3-7 By listening to advocacy groups, patients and their care teams, Jazz hopes to continue evolving the Find the Right Fit program to better serve those living with an sAML or MDS diagnosis.

"With 16 years of experience at the MDS Foundation, I understand how overwhelming a cancer diagnosis can be for a family," said Tracey Iraca, executive director of the MDS Foundation, Inc. "The foundation is proud to partner with Jazz on the launch of Find the Right Fit Navigating sAML, to help provide patients with vital information to navigate a rare disease diagnosis."

About AML Acute myeloid leukemia (AML) is a type of cancer in which the bone marrow makes abnormal myeloblasts (a type of white blood cell), red blood cells, or platelets.3 It can sometimes spread to other parts of the body including the lymph nodes, liver, spleen, central nervous system (brain and spinal cord), and testicles.8 AML is a relatively rare disease representing 1.1 percent of all new cancer cases.9 It is estimated that more than 19,500 people will be diagnosed with AML in the United States this year with the potential for more than 11,000 people to die from the disease.10 The median age at diagnosis is 68 years old,9 with rising age associated with a progressively worsening prognosis.11 There is also a reduced tolerance for intensive chemotherapy as patients age.12 AML has the lowest survival rate of any other form of leukemia.9 Patients with newly diagnosed therapy-related AML or AML with myelodysplasia-related changes may have a particularly poor prognosis.13-15 A hematopoietic stem cell transplant may be a curative treatment option for patients.16

AboutJazz Pharmaceuticals Jazz Pharmaceuticalsplc(Nasdaq: JAZZ) is a global biopharmaceutical company dedicated to developing and commercializing life-changing medicines that transform the lives of patients with serious diseases - often with limited or no options. We have a diverse portfolio of marketed medicines and novel product candidates, from early- to late-stage development, in key therapeutic areas. Our focus is in neuroscience, including sleep and movement disorders, and in oncology, including hematologic malignancies and solid tumors. We actively explore new options for patients including novel compounds, small molecule advancements, biologics and innovative delivery technologies. Jazz is headquartered inDublin, Irelandand has employees around the globe, serving patients in more than 90 countries. For more information, please visitwww.jazzpharmaceuticals.comand follow@JazzPharmaon Twitter.

About the MDS Foundation The MDS Foundation, Inc. is an international non-profit advocacy organization whose mission is to support and educate patients and healthcare providers with innovative research into the fields of MDS, Acute Myeloid Leukemia and related myeloid neoplasms in order to accelerate progress leading to the diagnosis, control and cure of these diseases.

About the Cancer Support Community As the largest professionally led nonprofit network of cancer support worldwide, the Cancer Support Community (CSC) is dedicated to ensuring that all people impacted by cancer are empowered by knowledge, strengthened by action and sustained by community. This global network of 175 locations, including CSC and Gilda's Club centers, healthcare partnerships and satellite locations, delivers more than $50 million in free support services to patients and families.

Media Contact: Jacqueline Kirby, Vice President, Corporate Affairs & Government Relations Ireland +353 1 697 2141 U.S. +1 215 867 4910

Investor Contact: Kathee Littrell, Vice President, Investor Relations Ireland +353 1 634 7887 U.S. +1 650 496 2717

References:

View original content to download multimedia:http://www.prnewswire.com/news-releases/jazz-pharmaceuticals-launches-initiative-to-help-educate-people-living-with-rare-form-of-leukemia-301133085.html

SOURCE Jazz Pharmaceuticals plc

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Jazz Pharmaceuticals Launches Initiative to Help Educate People Living with Rare Form of Leukemia - BioSpace

Heart tissue regeneration: a "cell-less" therapy may be the key – Emergency-Live

This study for the regeneration of the heart tissue started reporting: It is a recent piece of news, their approach, which accelerated recovery from heart attack in pigs, could address issues with safety and effectiveness that have prevented whole-cell heart therapies from reaching clinical adoption. In recent years, researchers have explored the possibility of using transplants of heart cells grown from induced pluripotent stem cells to heal cardiac tissue in the aftermath of events such as heart attacks.

However, transplanted heart cells often fail to engraft within the recipient and perish after a few days. Clinicians also remain worried that the cells that do engraft could cause severe health issues like arrythmia and even contribute to the formation of tumors in the long run. Instead of transplanting whole cells, Gao et al. tackled these issues by only administering exosomes, or tiny containers for proteins and DNA that are secreted by cells.

Specifically, they isolated exosomes from three types of human heart cells smooth muscle cells, cardiomyocytes, and endothelial cells and injected them into the hearts of pigs after heart attack. Pigs that received the exosomes recovered more heart function and showed smaller scars compared with untreated animals and improved as well as pigs that received whole cell transplants. Gao et al. say that the acellular exosomes could enable physicians to exploit the cardioprotective and reparative properties of hiPSC-derived cells while avoiding complexities associated with cell storage, transportation, and immune rejection.

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Heart tissue regeneration: a "cell-less" therapy may be the key - Emergency-Live

Researchers Discover a Way To Create Induced Tropoblast Stem Cells – Technology Networks

In vitro culture of induced trophoblast stem cells (pink) wrapping clusters of naive induced pluripotent stem cells (cyan). Credit: Monash Biomedicine Discovery Institute

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An international collaboration involving Monash University and Duke-NUS researchers have made an unexpected world-first stem cell discovery that may lead to new treatments for placenta complications during pregnancy.

While it is widely known that adult skin cells can be reprogrammed into cells similar to human embryonic stem cells that can then be used to develop tissue from human organs - known as induced pluripotent stem cells (iPSCs) - the same process could not create placenta tissue.

iPSCs opened up the potential for personalised cell therapies and new opportunities for regenerative medicine, safe drug testing and toxicity assessments, however little was known about exactly how they were made.

An international team led by ARC Future Fellow Professor Jose Polo from Monash University's Biomedicine Discovery Institute and the Australian Research Medicine Institute, together with Assistant Professor Owen Rackham from Duke-NUS in Singapore, examined the molecular changes the adult skin cells went through to become iPSCs. It was during the study of this process that they discovered a new way to create induced trophoblast stem cells (iTSCs) that can be used to make placenta cells.

This exciting discovery, also involving the expertise of three first authors, Dr. Xiaodong Liu, Dr. John Ouyang and Dr. Fernando Rossello, will enable further research into new treatments for placenta complications and the measurement of drug toxicity to placenta cells, which has implications during pregnancy.

"This is really important because iPSCs cannot give rise to placenta, thus all the advances in disease modelling and cell therapy that iPSCs have brought about did not translate to the placenta," Professor Polo said.

"When I started my PhD five years ago our goal was to understand the nuts and bolts of how iPSCs are made, however along the way we also discovered how to make iTSCs," said Dr Liu.

"This discovery will provide the capacity to model human placenta in vitro and enable a pathway to future cell therapies," commented Dr Ouyang.

"This study demonstrates how by successfully combining both cutting edge experimental and computational tools, basic science leads to unexpected discoveries that can be transformative," Professor Rackham said.

Professors Polo and Rackham said many other groups from Australian and international universities contributed to the study over the years, making it a truly international endeavour.

Reference:Liu, X., Ouyang, J.F., Rossello, F.J. et al. Reprogramming roadmap reveals route to human induced trophoblast stem cells. Nature (2020). https://doi.org/10.1038/s41586-020-2734-6

This article has been republished from the following materials. Note: material may have been edited for length and content. For further information, please contact the cited source.

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Researchers Discover a Way To Create Induced Tropoblast Stem Cells - Technology Networks

Impact of Corona on Stem Cell Therapy Market Key Manufactures and Business Overview till 2026 | Osiris Therapeutics, NuVasive, Chiesi Pharmaceuticals…

The globalStem Cell Therapy Marketis carefully researched in the report while largely concentrating on top players and their business tactics, geographical expansion, market segments, competitive landscape, manufacturing, and pricing and cost structures. Each section of the research study is specially prepared to explore key aspects of the global Stem Cell Therapy market. For instance, the market dynamics section digs deep into the drivers, restraints, trends, and opportunities of the global Stem Cell Therapy market. With qualitative and quantitative analysis, we help you with thorough and comprehensive research on the global Stem Cell Therapy market. We have also focused on SWOT, PESTLE, and Porters Five Forces analyses of the global Stem Cell Therapy market.

Leading players of the global Stem Cell Therapy market are analyzed taking into account their market share, recent developments, new product launches, partnerships, mergers or acquisitions, and markets served. We also provide an exhaustive analysis of their product portfolios to explore the products and applications they concentrate on when operating in the global Stem Cell Therapy market. Furthermore, the report offers two separate market forecasts one for the production side and another for the consumption side of the global Stem Cell Therapy market. It also provides useful recommendations for new as well as established players of the global Stem Cell Therapy market.

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Major Players:

Osiris Therapeutics NuVasive Chiesi Pharmaceuticals JCRPharmaceutical Pharmicell Medi-post Anterogen Molmed Takeda (TiGenix)

Segmentation by Product:

Autologous Allogeneic

Segmentation by Application:

Musculoskeletal Disorder Wounds & Injuries Cornea Cardiovascular Diseases Others

Regions and Countries:U.S, Canada, France, Germany, UK, Italy, Rest of Europe, India, China, Japan, Singapore, South Korea, Australia, Rest of APAC, Brazil, Mexico, Argentina, Rest of LATAM, Saudi Arabia, South Africa, UAE.

Report Objectives

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Table of Contents

Report Overview:It includes major players of the global Stem Cell Therapy market covered in the research study, research scope, and Market segments by type, market segments by application, years considered for the research study, and objectives of the report.

Global Growth Trends:This section focuses on industry trends where market drivers and top market trends are shed light upon. It also provides growth rates of key producers operating in the global Stem Cell Therapy market. Furthermore, it offers production and capacity analysis where marketing pricing trends, capacity, production, and production value of the global Stem Cell Therapy market are discussed.

Market Share by Manufacturers:Here, the report provides details about revenue by manufacturers, production and capacity by manufacturers, price by manufacturers, expansion plans, mergers and acquisitions, and products, market entry dates, distribution, and market areas of key manufacturers.

Market Size by Type:This section concentrates on product type segments where production value market share, price, and production market share by product type are discussed.

Market Size by Application:Besides an overview of the global Stem Cell Therapy market by application, it gives a study on the consumption in the global Stem Cell Therapy market by application.

Production by Region:Here, the production value growth rate, production growth rate, import and export, and key players of each regional market are provided.

Consumption by Region:This section provides information on the consumption in each regional market studied in the report. The consumption is discussed on the basis of country, application, and product type.

Company Profiles:Almost all leading players of the global Stem Cell Therapy market are profiled in this section. The analysts have provided information about their recent developments in the global Stem Cell Therapy market, products, revenue, production, business, and company.

Market Forecast by Production:The production and production value forecasts included in this section are for the global Stem Cell Therapy market as well as for key regional markets.

Market Forecast by Consumption:The consumption and consumption value forecasts included in this section are for the global Stem Cell Therapy market as well as for key regional markets.

Value Chain and Sales Analysis:It deeply analyzes customers, distributors, sales channels, and value chain of the global Stem Cell Therapy market.

Key Findings:This section gives a quick look at important findings of the research study.

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Our research base consists of a wide spectrum of premium market research reports. Apart from comprehensive syndicated research reports, our in-house team of research analysts leverages excellent research capabilities to deliver highly customized tailor-made reports. The market entry strategies presented in our reports has helped organizations of all sizes to generate profits by making timely business decisions. The research information including market size, sales, revenue, and competitive analysis offered, is the product of our excellence in the market research domain.

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STEM CELL BANKING Market Outlook, Recent Trends and Growth Forecast 2020-2026 – The Research Process

The report on NEUROSCIENCE ANTIBODIES AND ASSAYS market is a comprehensive evaluation of the market. It does so via in-depth qualitative insights, historical data, and verifiable projections about NEUROSCIENCE ANTIBODIES AND ASSAYS market size. The estimates featured in the report have been derived using proven research methodologies and assumptions. By doing so, the research report serves as a repository of analysis and information for every facet of the NEUROSCIENCE ANTIBODIES AND ASSAYS market, including but not limited to: Regional markets, technology, types, and applications.

The report provides insights on the following sections:

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As moving to the next segment NEUROSCIENCE ANTIBODIES AND ASSAYS Market report also evaluates the key opportunities in the market and outlines the factors that are and will be driving the growth of the NEUROSCIENCE ANTIBODIES AND ASSAYS industry. The major vendors in the NEUROSCIENCE ANTIBODIES AND ASSAYS market aggressively focused on enhancing their contributions to meet the business goal.

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The report also presents the market competition landscape and a corresponding detailed analysis of the major vendor/manufacturers in the market. The key manufacturers covered in this report:

Some of the Highlights about Table of Content of NEUROSCIENCE ANTIBODIES AND ASSAYS Market

1 NEUROSCIENCE ANTIBODIES AND ASSAYS Market overview

2 Executive Summary

3 Market Drivers, Challenges and Trends

4 Marketing, Distributors and Customer

5 Key Players Analysis

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STEM CELL BANKING Market Outlook, Recent Trends and Growth Forecast 2020-2026 - The Research Process

Global Animal Stem Cell Therapy Market Size, Share, Development Trend, Demand in Industry Growth Drivers and Challenges 2020-2025 – Express Journal

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The Top 10 Biotech Companies Brewing at… – Labiotech.eu

Here are the top biotech companies you will find in Oxford, a city with an old scientific tradition and an enormous output of biotech applications.

Oxford is well known for its university, one of the oldest in Europe and considered to be one of the best in the world. Recently, the University of Oxford has been the center of attention thanks to an experimental Covid-19 vaccine that the university is developing in partnership with big pharmaceutical companies. If successful, the vaccine, already in phase III testing, could be one of the first to get approval for this new disease.

The University of Oxford has hosted thousands of bright minds over the years. Thanks to its emphasis on technology transfer, the university has also helped a large number of them turn their ideas into successful spinout companies. The environment created around the university has also attracted many talents and businesses to the city, making it the ideal melting pot for new and promising ideas.

Biotech is one of the fortes of the innovation seen in the city of Oxford. So we consulted with local experts to put together a list of the most remarkable companies in the city, be it for their size, innovation, or influence in the sector.

Founded in 2008, Immunocore is one of just a few private biotech companies in Europe that are estimated to be worth over 1B. The company is tackling multiple forms of cancer as well as infectious and autoimmune diseases using T-cell receptor (TCR) technology. TCRs are proteins on the surface of immune T cells that are responsible for identifying a threat that must be destroyed, such as cancerous or infected cells. Immunocore aims to patients with engineered TCRs to circumvent the mechanisms by which these threats evade the immune system, restoring its ability to fight disease.

The company is collaborating on several projects with Genentech, AstraZeneca, Eli Lilly, and GSK. Its most advanced program is a treatment for uveal melanoma that is currently in phase III trials. Other programs target solid tumors, hepatitis, HIV, and type 1 diabetes.

Adaptimmune Therapeutics was founded at the same time as Immunocore with the goal of exploiting TCR technology in the form of T-cell therapy. The company engineers the TCRs naturally present on the patients own immune T cells to improve their ability to identify cancerous cells.

Adaptimmune is now getting ready to start late-stage clinical trials in multiple cancer types. Thanks to a deal with Astellas Pharma, the company is also gearing up to start clinical testing of a version of its T-cell therapy that doesnt require engineering each dose individually for each patient, using donor cells instead. Adaptimmune also has several partnerships with companies including GSK, Noile-Immune Biotech, and Alpine Immune Sciences.

Oxford Biodynamics was spun out of Oxford University in 2007 with the goal of developing liquid biopsy tests that can perform a diagnosis from just a drop of blood. The company specializes in epigenetics, that is changes to the structure of our DNA that determines which genes are switched on or off.

Oxford Biodynamics works in a wide range of indications, including cancer, diabetes, Alzheimers, multiple sclerosis, and rheumatoid arthritis among many others. The tests are not only designed to diagnose a disease; they can also be made to determine which patients are going to benefit the most from a specific drug, and how likely the disease is to progress faster or relapse.

With these tests, the company is supporting the development of personalized medicine approaches and helping drug developers increase their chances of succeeding in clinical trials. Partners include big pharma such as Pfizer and EMD, as well as universities and research institutes.

Chronos Therapeutics started out in 2009 as a spinout of the University of Oxford with the goal of developing drugs for age-related conditions. The companys lead program targets amyotrophic lateral sclerosis (ALS), the most common motor neuron disease, for which there are no treatments able to stop its progression.

Over time, the company has expanded its portfolio into other indications, particularly those that affect the brain, by acquiring assets from third parties. They include programs targeting fatigue caused by multiple sclerosis, addictive behaviors such as binge eating and alcohol use disorder, and post-traumatic stress disorder.

Evox Therapeutics is developing a drug delivery technology based on exosomes nanoparticles that our bodies naturally use to transport molecules. The company engineers exosomes to carry different types of drugs, such as proteins, RNA, or small drugs. The key advantage is that these natural carriers are able to reach targets that conventional drug delivery systems cant, such as the brain.

Founded in 2016, the company has signed big deals with Takeda and Eli Lilly. Its programs are all in preclinical testing and mostly target rare diseases. One of them targets the rare liver disorder argininosuccinic aciduria and is scheduled to enter clinical trials in 2021.

Exscientia is a pioneer in the application of artificial intelligence to drug discovery. The company uses AI to identify potential drug candidates and optimize their structure to maximize their chances of success in clinical trials. The goal is to speed up and reduce the costs of the drug discovery process.

Earlier this year, the companys drug candidate for obsessive-compulsive disorder became one of the first AI-designed drugs to enter clinical trials. While a drug typically takes five years from identification until clinical trials, this one did so in just a year.

Exscientia is partnered with Bayer, BMS, Sanofi, and GSk among others. In the wake of the Covid-19 pandemic, the company set out to go through a database of 15,000 approved and investigational drugs that had already passed safety testing to find candidates that can then be fast-tracked to clinical testing in Covid-19 patients.

Oxford Biomedica was set up in 1995 as a developer and provider of lentiviral vectors for gene and cell therapy. These vectors allow the permanent introduction of a desired DNA sequence into a target cell, be it in the test tube or directly in the patients body.

The technology of Oxford Biomedica is regularly used by companies such as Novartis, Sanofi, Boehringer Ingelheim, Imperial Innovations, and Orchard Therapeutics. Notably, the vectors developed by the company are used in Novartis Kymriah, the first CAR T-cell therapy approved in Europe and the US as a cancer treatment.

Oxford Biomedica also has a preclinical pipeline of proprietary programs in a wide range of applications, including cancer, eye disease, ALS, and liver disease. Last year, the company struck a deal with Microsoft to reduce the complexity and costs of gene and cell therapy manufacturing using artificial intelligence.

Founded in 2016, Arctoris aims to bring the benefits of automation to cancer research. Through the companys services, a researcher could just order an experiment online and spend their valuable time designing experiments and analyzing results rather than performing the repetitive tasks needed to complete them.

Arctoris aims to contribute to reducing the costs of drug discovery, which are increasing every year as treatments become personalized and results more difficult to replicate. In the context of the Covid-19 pandemic, Arctoris has established assays that allow scientists the possibility of running Covid-19 experiments remotely.

Founded in 2015, Orbit Discovery is a drug discovery company focusing on the identification of peptide drugs. The company has developed a technology that significantly improves on conventional methods of drug screening such as phage display or mRNA display.

The technology consists of fusing peptides to the DNA sequence encoding them and presenting them to live cells. This method allows the screening of peptide targets that were previously missed by other technologies, and to study their effect on live cells to better predict their function.

The company has already identified several candidates in the areas of cardiology, immunology, and cancer, and is working with partners such as Zealand Pharma in their preclinical development.

OxStem is a drug discovery company with an unusual approach to stem cell treatments. Instead of using stem cells as a therapy, the firm focuses on developing small molecule drugs that can reprogram the patients own stem cells to treat a wide range of diseases related to aging, including cancer and diabetes as well as neurological, cardiovascular, and ocular conditions.

Founded in 2013, the strategy of OxStem is to spin out companies that specialize in each disease area to focus on the development of the drugs found by the parent company, with five subsidiaries set up so far.

Tags: Adaptimmune, Immunocore, Oxford BioMedica, United Kingdom

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The Top 10 Biotech Companies Brewing at... - Labiotech.eu