Stem Cells Market- What Are The Main Factors That Contributing Towards Industry Growth? – Industry Mirror

Stem Cells Market AnalysisAccording to Market Research, the Global Stem Cells Market was valued at USD 5.88 Billion in 2018 and is expected to witness a growth of 10.32% from 2019-2026 and reach USD 12.96 Billion by 2026.

What is Stem Cells Market?Stem cellscan be defined as unspecialized cells that develop into the specialized cells and make up different types of tissue in the human body. Since stem cells are unspecialized type of cells and are capable of renewing themselves through cell division. Stem cells can be Pluripotent as well as Multipotent.

Pluripotent stem cells are stem cells usually found in embryos which give rise to all the cells found in the human body, while multipotent stem cells, which are found in adults or in babies umbilical cords, have a more restricted ability. Their development is limited to cells that form the organ system that they are originated from. When a stem cell undergoes division, each new cell possess a potential either to remain a stem cell or develop into another type of cell with a more specialized function, such as a muscle cell, a red blood cell, or a brain cell.

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Stem Cells Market OutlookStem cell research is considered as one of the most intriguing areas of contemporary biology, but, as with many expanding fields of scientific inquiry, research on stem cells stimulates scientific queries as rapidly as it produces new discoveries. Until recently, scientists used to primarily work with two types of stem cells from animals and humans: embryonic stem cells and non-embryonic somatic or adult stem cells.

Since the advent of stem cells, one of the crucial benefits of stem cell research is the accessibility of cell lines and that they can be acquired ethically. The demands for pluripotent stem cells are increasing owing to the fact that it differentiates in various cell types in the human body. Pluripotent stem cells tend to have various applications in the medical treatment. Growing awareness regarding the stem cells and establishment of stem cell banks is expected to fuel the market growth rate.

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Ethical issues related to pluripotent stem cells could hamper the growth of stems cells in the global market as research with these cells require disrupting an artificially-fertilized embryo at the 5-14 day stage. Another factor which is limiting the growth of stem cells market is unknown long-term consequences.

Global Stem Cells Market SegmentationThe Global Stem Cells Market is classified on the basis of Product, Treatment Type, Therapeutic Application and Region. The gist of breaking down the market into various segments is to gather the information about various aspects of the market.

On the basis of Products, the market is bifurcated on the basis of Adult Stem Cells, Human Embryonic Cells, and Induced Pluripotent Stem Cell. Adult stem cells accounts for a major share in the global stem cells market. Even though embryonic stem cells have a wide range of applications, the market growth rate for this sub-segment is substantial owing to the ethical issues faced by this sub-segment in the global market.

In terms of Therapeutic Application, the market study encompasses various aspects such ca Regenerative Medicine, Neurological Disorders, Orthopedic Treatments, Oncology Disorders, Diabetes, Injuries & Wounds and Cardiovascular Disorders. Growing awareness regarding regenerative medicine is expected to make this sub-segment hold a potential market share globally. Growing healthcare expenditure and presence of major industry players makes North America hold major share in the global market.

Stem Cells Market Competitive LandscapeThe Stem Cells Market study report offers a valuable insight with an emphasis on global market including some of the major players such asBioTime Inc., Cytori Therapeutics, Inc., STEMCELL Technologies Inc., Astellas Pharma Inc., U.S. Stem Cell, Inc., Osiris Therapeutics, Inc., Takara Bio Inc., Caladrius Biosciences, Inc., Cellular Engineering Technologies Inc., and BrainStorm Cell Therapeutics Inc. Our market analysis also entails a section solely dedicated for such major players wherein our analysts provide an insight to the financial statements of all the major players, along with its product benchmarking and SWOT analysis. The competitive landscape section also includes key development strategies, market share and market ranking analysis of the above mentioned players globally.

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Analyst View:As per our sources following trends were observed in terms of most popular sources of stem cells:

Stem cells from adult bone marrow were observed to be the most popular source. Scope of stem cell therapy is increasing with growing number of applications. Clinical research has advanced to a great magnitude towards preventing, identifying and handling devastating diseases. Various applications of stem cells in regeneration such as Cardiac Regeneration, Hepatic Regeneration, Regeneration of Neural Tissue, etc. have come up lately. This suggests that the market for stem cells will grow significantly over the forecast period.

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What a time to be alive: Reproductive breakthroughs of the 2010s that changed life as we know it – FOX 10 News Phoenix

This undated screen grab shows the cell-division of two fertilized human embryos during the first 24 hours of embryonic development following IVF treatment at a private clinic in London. ( Jim Dyson/Getty Images )

LOS ANGELES - Some of the scientific advancements of the 2010s have been truly mind-blowing, and perhaps none more so than the leaps and bounds weve made in the realm of reproduction.

This was not only the decade in which the first three-parent baby was born, it was the era when a rogue scientist chose to make edits to a set of twin girls DNA, making real the long-imagined scenario of genetically altering human beings while simultaneously thrusting the deeply complicated ethical discussions surrounding this practice into the limelight.

These are the five most life-altering breakthroughs in reproduction from the past decade.

In 2018, Chinese biophysics researcher He Jiankui announced that he had used the gene-editing tool CRISPR to modify the genes of two twin girls before birth. He and his team said that their goal was to make the girls immune to infection by HIV through the elimination of a gene called CCR5.

When the news broke, many mainstream scientists criticized the attempt, calling it too unsafe to try. Where some people saw the potential for a new kind of medical treatment capable of eradicating genetic disease, others saw a window into a dystopian future filled with designer babies and framed by a new kind of eugenics.

At the time, Dr. Kiran Musunuru, a University of Pennsylvania gene-editing expert, said Hes work was unconscionable... an experiment on human beings that is not morally or ethically defensible.

Other experts believe Hes work could propel the field of gene editing forward.

The twins, known as Lulu and Nana, have continued to make headlines since their birth. The gene modification that He claims to have carried out may have caused some unintended mutations in other parts of the genome, which could have unpredictable consequences for their health long term something many scientists who argue against Hes work cite as a reason to hold off on using gene-editing technology on humans.

Only time will tell what will happen to Lulu and Nana and if the edits to their DNA ultimately help or hurt them, but their story pushed the topic of human gene-editing and the ethics surrounding it to the forefront of the global scientific community.

In 2016, a technique called mitochondrial transfer was used successfully for the first time to create a three-parent baby grown from a fathers sperm, a mothers cell nucleus and a third donors egg that had the nucleus removed.

This technique was developed to prevent the transmission of certain genetic disorders through the mothers mitochondria. The majority of a three-parent babys DNA would come from his parents in the form of nuclear DNA, and only a small portion would come from the donor in the form of mitochondrial DNA.

A team led by physician John Zhang at the New Hope Fertility Center in New York City facilitated the birth of the first three-parent baby in April 2016.

Using human pluripotent stem cells, researchers were able to make the precursors of human sperm or eggs. In other words, they reprogrammed skin and blood stem cells to become an early-state version of what would eventually become either sperm or an egg.

"The creation of primordial germ cells is one of the earliest events during early mammalian development," Dr. Naoko Irie, first author of the paper from the Wellcome Trust/Cancer Research UK Gurdon Institute at the University of Cambridge told Science Daily. "It's a stage we've managed to recreate using stem cells from mice and rats, but until now few researches have done this systematically using human stem cells. It has highlighted important differences between embryo development in humans and rodents that may mean findings in mice and rats may not be directly extrapolated to humans."

A 2018 study showed that gene editing can allow two same-sex mice to conceive pups, and two female mice were able to successfully create healthy pups that then went on to reproduce themselves.

A team of researchers at the Chinese Academy of Sciences in Beijing, led by developmental biologist Qi Zhou, were able to use gene editing to produce 29 living mice from two females, seven of which went on to have their own pups. They were able to produce 12 pups from two male parents, but those offspring were not able to live more than two days.Whether or not the method can one day be used in same-sex human reproduction is still up for debate.

For the first time ever, Chinese scientists were able to clone two primates using the technique that produced Dolly the sheep, the first mammal to be cloned from an adult somatic cell via nuclear transfer.

The two cloned female macaques were named Zhong Zhong and Hua Hua, and their successful birth opened up the possibility of using the same cloning method to one day clone humans.

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Gene Therapy Market 2019-2027 / Trends, Growth, Opportunities And Top Key – Market Research Sheets

The report covers the forecast and analysis of the gene therapy market on a global and regional level. The study provides historical data from 2015 to 2018 along with a forecast from 2019 to 2027 based on revenue (USD Million). The study includes drivers and restraints of the gene therapy market along with the impact they have on the demand over the forecast period. Additionally, the report includes the study of opportunities available in the gene therapy market on a global level.

In order to give the users of this report a comprehensive view of the gene therapy market, we have included a competitive landscape and an analysis of Porters Five Forces model for the market. The study encompasses a market attractiveness analysis, wherein all the segments are bench marked based on their market size, growth rate, and general attractiveness.

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The report provides company market share analysis to give a broader overview of the key players in the market. In addition, the report also covers key strategic developments of the market including acquisitions & mergers, new service launches, agreements, partnerships, collaborations & joint ventures, research & development, and regional expansion of major participants involved in the market on a global and regional basis.

The study provides a decisive view of the gene therapy market by segmenting the market based on the type, vector type, therapy area, and regions. All the segments have been analyzed based on present and future trends and the market is estimated from 2019 to 2027. The regional segmentation includes the current and forecast demand for North America, Europe, Asia Pacific, Latin America, and the Middle East and Africa.

Gene therapy is utilized for treating neurodegenerative disorders like Alzheimer, amyotrophic lateral sclerosis, and spinal muscular atrophy. Gene therapy is one of the key treatment kinds that will propel the market growth over the forecast period. Moreover, gene therapy also finds lucrative applications in precision medicine. In addition to this, a rise in the occurrence of cancer is prompting the demand to treat the disease through gene therapy.

Based on the type, the market can be segregated into Germ Line Gene Therapy and Somatic Gene Therapy. In terms of vector type, the gene therapy industry can be divided into Viral Vectors, Non-Viral Vectors, and Human Artificial Chromosome. On the basis of therapy area, the market for gene therapy can be classified into Cancer, Neurological Diseases, Infectious Diseases, Genetic Disorders, Rheumatoid Arthritis, and Others.

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The key players included in this market are Advanced Cell & Gene Therapy, Audentes Therapeutics, Benitec Biopharma, Biogen, Blubird Bio, Inc., Bristol-Myers Squibb Company, CHIESI Farmaceutici SPA, Eurofins Scientific, Geneta Science, Genzyme Corporation, Gilead, GlaxoSmithKline PLC, Human Stem Cells institute, Novartis AG, Orchard Therapeutics, Pfizer Inc., Sangamo therapeutics, Spark therapeutics, and Voyager Therapeutics.

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Gene Therapy Market 2019-2027 / Trends, Growth, Opportunities And Top Key - Market Research Sheets

Cell Therapy Industry Applications 2019-Size by Type (Allogenic Therapies), by Technique (Stem Cell Therapy), Global Market Growth by Demand Analysis…

TheGlobal Cell Therapy Marketwas estimated to be valued at USD XX million in 2018 and is projected to reach USD XX million by 2026, at a CAGR of XX% during 2019 to 2026.

Cell therapy involves the administration of somatic cell preparations for the treatment of diseases or traumatic damages. The objective of this study is to provide long term treatment through a single injection of therapeutic cells.

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Growing aging patient population, the rise in cell therapy transplantations globally, and rising disease awareness drive the growth of the market. However, stringent regulatory policies may restrain growth of the market in the forecast period.

The Global Cell Therapy Industry is primarily segmented based on different type, technique, cell source, technology, end users and region

On the basis of type, the market is split into:

On the basis of technique, the market is split into:

On the basis of cell source, the market is split into:

On the basis of technology, the market is split into:

On the basis of end user, the market is split into:

Moreover, the market is classified based on regions and countries as follows:

Key players profiled in the report includes:

These organizations are focusing on growth strategies, such as new product launches, expansions, acquisitions, and agreements & partnerships to expand their operations across the globe.

GlobalCell TherapyIndustry isspread across 121 pages, profiling 10 companies and supported with tables and figures. Inquire more or share a questions if any before the purchase on this report @https://www.orianresearch.com/enquiry-before-buying/1281174

What you can expect from our report: Total Addressable Market [ Present Market Size forecasted to 2026 with CAGR ] Regional level split [North America, Europe, Asia Pacific, South America, Middle East & Africa] Country wise Market Size Split [Important countries with major market share] Market Size Breakdown by Product/ ServiceTypes [ ] Market Size by Application/Industry verticals/ End Users [ ] Market Share and Revenue/Sales of 10-15 Leading Players in the Market Production Capacity of Leading Players whenever applicable Market Trends Emerging Technologies/products/start-ups, PESTEL Analysis, SWOT Analysis, Porters Five Forces, etc. Pricing Trend Analysis Average Pricing across regions Brandwise Ranking of Major Market Players globally

Key Benefits of the Report:

Target Audience:

Research Methodology:

The market is derived through extensive use of secondary, primary, in-house research follows by expert validation and third party perspective, such as, analyst reports of investment banks. The secondary research is the primary base of our study wherein we conducted extensive data mining, referring to verified data products, such as, white papers, government and regulatory published articles, technical journals, trade magazines, and paid data products.

For forecasting, regional demand & supply factors, recent investments, market dynamics including technical growth scenario, consumer behavior, and end use trends and dynamics, and production capacity were taken into consideration. Different weightages have been assigned to these parameters and quantified their market impacts using the weighted average analysis to derive the market growth rate.

The market estimates and forecasts have been verified through exhaustive primary research with the Key Industry Participants (KIPs), which typically include:

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Table of Content

1. Introduction2. Research Methodology3. Executive Summary4. Global Cell Therapy Market Overview5. Global Cell Therapy Market, by Type6. Global Cell Therapy Market, by Technique7. Global Cell Therapy Market, by Cell Source8. Global Cell Therapy Market, by Technology9. Global Cell Therapy Market, by End Users10. Global Cell Therapy Market by Region11. Competitive Landscape12. Company Profiles13. Cell Therapy Manufacturing Cost Analysis14. Key InsightsEnd of the reportDisclaimer

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Cell Therapy Industry Applications 2019-Size by Type (Allogenic Therapies), by Technique (Stem Cell Therapy), Global Market Growth by Demand Analysis...

Stem Cell Therapies Market research Likely to Emerge over a Period of 2015-2025 – PharmiWeb.com

VALLEY COTTAGE, N.Y. Stem cells are undifferentiated biological cells, and having remarkable potential to divide into any kind of other cells. When a stem cell divides, each new cell will be a new stem cell or it will be like another cell which is having specific function such as a muscle cell, a red blood cell, brain cell and some other cells.

There are two types of stem cells

Stem cells harvested from umbilical cord blood just after birth. And this cells can be stored in specific conditions. Stem cells also can be harvest from bone marrow, adipose tissue.

Embryonic cells can differentiate into ectoderm, endoderm and mesoderm in developing stage. Stem cells used in the therapies and surgeries for regeneration of organisms or cells, tissues.

Stem cells are used for the treatment of Gastro intestine diseases, Metabolic diseases, Immune system diseases, Central Nervous System diseases, Cardiovascular diseases, Wounds and injuries, Eye diseases, Musculoskeletal disorders.

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Harvesting of Adult cell is somewhat difficult compare to embryonic cells. Because Adult cells available in the own body and it is somewhat difficult to harvest.

Stem Cell TherapiesMarket: Drivers and Restraints

Technology advancements in healthcare now curing life threatening diseases and giving promising results. Stem Cell Therapies having so many advantages like regenerating the other cells and body organisms. This is the main driver for this market. These therapies are useful in many life threatening treatments. Increasing the prevalence rate of diseases are driven the Stem Cell Therapies market, it is also driven by increasing technology advancements in healthcare. Technological advancements in healthcare now saving the population from life threatening complications.

Increasing funding from government, private organizations and increasing the Companies focus onStem cell therapiesare also driven this market

However, Collecting the Embryonic Stem cells are easy but Collecting Adult Stem cell or Somatic Stem cells are difficult and also we have to take more precautions for storing the collected stem cells.

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Stem Cell TherapiesMarket: Segmentation

Stem Cell Therapies are segmented into following types

Based on treatment:

Based on application:

Based on End User:

Stem Cell TherapiesMarket: Overview

With rapid technological advantage in healthcare and its promising results, the use of Stem Cell Therapies will increase and the market is expected to have a double digit growth in the forecast period (2015-2025).

Stem Cell TherapiesMarket: Region- wise Outlook

Depending on geographic regions, the global Stem Cell Therapies market is segmented into seven key regions: North America, South America, Eastern Europe, Western Europe, Asia Pacific excluding Japan, Japan and Middle East & Africa.

The use of Stem Cell Therapies is high in North America because it is highly developed region, having good technological advancements in healthcare setup and people are having good awareness about health care. In Asia pacific region china and India also having rapid growth in health care set up. Europe also having good growth in this market.

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Stem Cell TherapiesMarket: Key Players

Some of the key players in this market are

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Orgenesis and Theracell to launch point-of-care cell and gene therapy centers in HYGEIA Group"s hospitals – Proactive Investors USA & Canada

CEO Vered Caplan says the move would enable the development and delivery of cell and gene therapies onsite at HYGEIA's hospitals in Greece

Inc (), a developer of advanced cell therapies, revealed Friday that it struck a strategic partnership agreement between the Theracell joint venture and the large HYGEIA Group which runs three hospitals in Greece.

In a statement, the Germantown, Maryland-based company said that under the terms of the agreement, the joint venture will implement point-of-care cell therapy platform for clinical development and commercialization of cell and gene therapies within the HYGEIA Groups network of hospitals in Greece.

and TheraCell Advanced Biotechnology earlier formed a joint venture to advance point-of-care platform in Greece, the Balkan region and some Middle Eastern countries.

The point-of-care platform is designed to collect, process and supply cells within the patient care setting for various treatments.

The main goal is to reduce the cost and complexity of supplying cell and gene therapies, said , as well as boostquality by integrating automated processing units and proprietary technologies.

Significantly, HYGEIA is the first hospital network in the region to implement Orgenesis point-of-care cell therapy platform. The partnership aims to provide the HYGEIA Group with resources to advance clinical development and deliver personalized, advanced therapies across its network for a range of diseases in oncology, hematology, orthopedics, nephrology, dermatology and diabetes.

This partnership with the HYGEIA Group further validates the significant value proposition of our point-of-care platform, as it enables the development and delivery of cell and gene therapies onsite at hospitals, said Orgenesis CEO Vered Caplan.

We believe this platform has the potential to transform the cell and gene therapy market, by bringing life-saving therapies to market in a much more time and cost-effective manner, she added.

The Orgenesis boss said Theracell had proved to be an ideal partner with extensive experience and capabilities in autologous cell therapy and regenerative medicine, and strong operations in Greece and relationships in the region.

We are in active discussions to establish PoCare locations and partnerships with hospitals and healthcare networks in other countries and regions across the world, said Caplan.

Greeces HYGEIA Group operates three hospitals with a capacity of 1,261 beds, 52 operating rooms, 19 delivery rooms and 10 intensive care units.

HYGEIA Group CEO Andreas Kartapanis said thanks to the partnership with Theracell and Orgenesis it would be the first hospital network in Greece to provide advanced cell and gene therapies.

We believe this partnership will provide us a strong competitive advantage in this rapidly developing field. More importantly, this partnership will benefit patients that will now have greater access to these important therapies, said Kartapanis.

For the fiscal third quarter ended September 30, Orgenesis generated meaningful revenue, over $1 million, through its rapidly advancing point-of-care cellular therapy platform.

Meanwhile, TheraCell has experience in the isolation, processing and application of adipose-derived stem cells, as well as somatic cells. It has developed a patented platform for tissue engineering and cell therapies in the areas of dermatology, articular cartilage defects, and chronic kidney injury.

Contact Uttara Choudhury at[emailprotected]

Follow her onTwitter:@UttaraProactive

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Orgenesis and Theracell to launch point-of-care cell and gene therapy centers in HYGEIA Group"s hospitals - Proactive Investors USA & Canada

Rocket Pharmaceuticals Announces First Patient Treated in Global Registrational Phase 2 Study of RP-L102 Process B for Fanconi Anemia – BioSpace

NEW YORK--(BUSINESS WIRE)-- Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) (Rocket), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, today announces that the first patient in the global Phase 2 registration-enabling study of RP-L102 Process B received investigational therapy. RP-L102 is the Companys lentiviral vector (LVV)-based gene therapy for the treatment of Fanconi Anemia (FA).

The initiation of Rockets first Phase 2 trial is an important milestone for the company as well as patients throughout the world battling FA, said Kinnari Patel, Pharm.D., MBA, Chief Operating Officer and Head of Development of Rocket. With the recent feedback received from the FDA and EMA of MMC-resistance as the primary endpoint, we are optimistic about the prospect of benefiting patients and, if the data are positive, working towards BLA and MAA submissions.

The registrational package will include twelve patients from the U.S. and EU, two from the U.S. Phase 1 study and 10 additional patients from the global Phase 2 study (NCT04069533). Patients will receive a single intravenous infusion of RP-L102 that utilizes fresh cells and Process B which incorporates a modified stem cell enrichment process, transduction enhancers, as well as commercial-grade vector and final drug product. Improved mitomycin-C (MMC) resistance in bone marrow colony forming (progenitor) cells is the primary endpoint, and may also serve as a surrogate endpoint for accelerated approval. Additional outcome measures include stability or increase in blood counts with no significant worsening in anemia, neutropenia or thrombocytopenia and peripheral blood and bone marrow genetic correction, as demonstrated by progressive increases in vector copy number (VCN) over the months subsequent to infusion.

Lucile Packard Childrens Hospital Stanford and Hospital Infantil Universitario Nio Jess are serving as the lead clinical sites and University of Minnesota is conducting centralized evaluation of bone marrow MMC-resistance and engaging in advisory activities for the global trial of RP-L102. RP-L102 was in-licensed from the Centro de Investigaciones Energticas, Medioambientales y Tecnolgicas (CIEMAT), Centro de Investigacin Biomdica en Red de Enfermedades Raras (CIBERER), Instituto de Investigacin Sanitaria Fundacin Jimnez Daz (IIS-FJD) and Fundacion para la Investigacion Biomedica Hospital Infantil Universitario Nio Jesus (FIB-HIUNJ).

About Fanconi Anemia

Fanconi Anemia (FA) is a rare pediatric disease characterized by bone marrow failure, malformations and cancer predisposition. The primary cause of death among patients with FA is bone marrow failure, which typically occurs during the first decade of life. Allogeneic hematopoietic stem cell transplantation (HSCT), when available, corrects the hematologic component of FA, but requires myeloablative conditioning, which is highly toxic for the patient. HSCT is frequently complicated by graft versus host disease and also increases the risk of solid tumors, particularly upper aerodigestive tract squamous cell carcinomas. Approximately 60-70% of patients with FA have a FANCA gene mutation, which encodes for a protein essential for DNA repair. Mutations in the FANCA gene leads to chromosomal breakage and increased sensitivity to oxidative and environmental stress. Chromosome fragility induced by DNA-alkylating agents such as mitomycin-C (MMC) or diepoxybutane (DEB) is the gold standard test for FA diagnosis. These assays can further differentiate FA patients from mosaic patients. Somatic mosaicism occurs when there is a spontaneous reversion mutation that can lead to a mixed chimerism of corrected and uncorrected bone marrow cells leading to stabilization or correction of an FA patients blood counts in the absence of any administered therapy. Somatic mosaicism provides strong rationale for the development of FA gene therapy and demonstrates the selective advantage of gene-corrected hematopoietic cells in FA1.

1Soulier, J.,et al. (2005) Detection of somatic mosaicism and classification of Fanconi anemia patients by analysis of the FA/BRCA pathway. Blood 105: 1329-1336

About Rocket Pharmaceuticals, Inc.

Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) (Rocket) is advancing an integrated and sustainable pipeline of genetic therapies that correct the root cause of complex and rare childhood disorders. The companys platform-agnostic approach enables it to design the best therapy for each indication, creating potentially transformative options for patients contending with rare genetic diseases. Rocket's clinical programs using lentiviral vector (LVV)-based gene therapy are for the treatment of Fanconi Anemia (FA), a difficult to treat genetic disease that leads to bone marrow failure and potentially cancer, Leukocyte Adhesion Deficiency-I (LAD-I), a severe pediatric genetic disorder that causes recurrent and life-threatening infections which are frequently fatal, and Pyruvate Kinase Deficiency (PKD) a rare, monogenic red blood cell disorder resulting in increased red cell destruction and mild to life-threatening anemia. Rockets first clinical program using adeno-associated virus (AAV)-based gene therapy is for Danon disease, a devastating, pediatric heart failure condition. Rockets pre-clinical pipeline program is for Infantile Malignant Osteopetrosis (IMO), a bone marrow-derived disorder. For more information about Rocket, please visit http://www.rocketpharma.com.

Rocket Cautionary Statement Regarding Forward-Looking Statements

Various statements in this release concerning Rocket's future expectations, plans and prospects, including without limitation, Rocket's expectations regarding the safety, effectiveness and timing of product candidates that Rocket may develop, to treat Fanconi Anemia (FA), Leukocyte Adhesion Deficiency-I (LAD-I), Pyruvate Kinase Deficiency (PKD), Infantile Malignant Osteopetrosis (IMO) and Danon disease, and the safety, effectiveness and timing of related pre-clinical studies and clinical trials, may constitute forward-looking statements for the purposes of the safe harbor provisions under the Private Securities Litigation Reform Act of 1995 and other federal securities laws and are subject to substantial risks, uncertainties and assumptions. You should not place reliance on these forward-looking statements, which often include words such as "believe," "expect," "anticipate," "intend," "plan," "will give," "estimate," "seek," "will," "may," "suggest" or similar terms, variations of such terms or the negative of those terms. Although Rocket believes that the expectations reflected in the forward-looking statements are reasonable, Rocket cannot guarantee such outcomes. Actual results may differ materially from those indicated by these forward-looking statements as a result of various important factors, including, without limitation, Rocket's ability to successfully demonstrate the efficacy and safety of such products and pre-clinical studies and clinical trials, its gene therapy programs, the pre-clinical and clinical results for its product candidates, which may not support further development and marketing approval, the potential advantages of Rocket's product candidates, actions of regulatory agencies, which may affect the initiation, timing and progress of pre-clinical studies and clinical trials of its product candidates, Rocket's and its licensors ability to obtain, maintain and protect its and their respective intellectual property, the timing, cost or other aspects of a potential commercial launch of Rocket's product candidates, Rocket's ability to manage operating expenses, Rocket's ability to obtain additional funding to support its business activities and establish and maintain strategic business alliances and new business initiatives, Rocket's dependence on third parties for development, manufacture, marketing, sales and distribution of product candidates, the outcome of litigation, and unexpected expenditures, as well as those risks more fully discussed in the section entitled "Risk Factors" in Rocket's Quarterly Report on Form 10-Q for the quarter ended September 30, 2019, filed November 8, 2019. Accordingly, you should not place undue reliance on these forward-looking statements. All such statements speak only as of the date made, and Rocket undertakes no obligation to update or revise publicly any forward-looking statements, whether as a result of new information, future events or otherwise.

View source version on businesswire.com: https://www.businesswire.com/news/home/20191206005080/en/

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Rocket Pharmaceuticals Announces First Patient Treated in Global Registrational Phase 2 Study of RP-L102 Process B for Fanconi Anemia - BioSpace

Stem Cell Therapy Industry 2019 Global Market Size, Trends, Revenue, Growth Prospects, Key Companies and Forecast by 2023 – Techi Labs

Bone marrow transplant is the most widely used stem-cell therapy, but some therapies derived from umbilical cord blood are also in use. Research is underway to develop various sources for stem cells, and to apply stem-cell treatments for neurodegenerative diseases and conditions, diabetes, heart disease, and other conditions.

With the ability of scientists to isolate and culture embryonic stem cells, and with scientists growing ability to create stem cells using somatic cell nuclear transfer and techniques to create induced pluripotent stem cells, controversy has crept in, both related to abortion politics and to human cloning. Additionally, efforts to market treatments based on transplant of stored umbilical cord blood have been controversial.

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Scope of the Report:

This report studies the Stem Cell Therapy market status and outlook of Global and major regions, from angles of players, countries, product types and end industries; this report analyzes the top players in global market, and splits the Stem Cell Therapy market by product type and applications/end industries.

USA is a huge market, and the total sum of the industry is more than 24 Million US dollars in 2014. At the same time, this industry continuously increases, with the development of global economy.

According to the research, the most potential market in the main countries of stem cell therapy industry is China, determined by the rising level of medical care. Besides, South America, Middle East should also be focused by the investors. They are the potential consumers of stem cell therapy.

Complete report on Stem Cell Therapy Market report spread across 139 pages, profiling 18 companies and supported with tables and figures.

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Development policiesand plans are discussed as well as manufacturing processes and cost structures are also analyzed. This report also states import/export consumption, supply and demand Figures, cost, price, revenue and gross margins. The report focuses on global major leading Stem Cell Therapy Industry players providing information such as company profiles, product picture and specification, capacity, production, price, cost, revenue and contact information. Upstream raw materials and equipment and downstream demand analysis is also carried out. The Stem Cell Therapy industry development trends and marketing channels are

Analysis ofStem Cell Therapy Industry Key Manufacturers:

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This report studies the top producers and consumers, focuses on product capacity, production, value, consumption, market share and growth opportunity in these key regions, covering:

Market Segment by Type, covers

Market Segment by Applications, can be divided into

Table of Contents

There are 15 Chapters to deeply display the global Banknote-Printing Machine market.

Chapter 1, to describe Banknote-Printing Machine Introduction, product scope, market overview, market opportunities, market risk, market driving force

Chapter 2, to analyze the top manufacturers of Banknote-Printing Machine, with sales, revenue, and price of Banknote-Printing Machine, in 2016 and 2017;

Chapter 3, to display the competitive situation among the top manufacturers, with sales, revenue and market share in 2016 and 2017;

Chapter 4, to show the global market by regions, with sales, revenue and market share of Banknote-Printing Machine, for each region, from 2013 to 2018;

Chapter 5, 6, 7, 8 and 9, to analyze the market by countries, by type, by application and by manufacturers, with sales, revenue and market share by key countries in these regions;

Chapter 10 and 11, to show the market by type and application, with sales market share and growth rate by type, application, from 2013 to 2018;

Chapter 12, Banknote-Printing Machine market forecast, by regions, type and application, with sales and revenue, from 2018 to 2023;

Chapter 13, 14 and 15, to describe Banknote-Printing Machine sales channel, distributors, traders, dealers, Research Findings and Conclusion, appendix and data source.

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Stem Cell Therapy Industry 2019 Global Market Size, Trends, Revenue, Growth Prospects, Key Companies and Forecast by 2023 - Techi Labs

Brave new world? Why the public might be ready for gene-edited babies – Genetic Literacy Project

Should we be able to genetically design our children before they are born giving them the eye and hair color we prefer, deciding their sex and height, and even genetically manipulating their intelligence? Most, including top scientists and genetic counselors, say no. But what if the genetic manipulation was done in order to cure a debilitating or potentially fatal illness before the individual was even born? And what if this one genetic treatment could save the individuals descendants from inheriting the disorder, too? A 2018 meeting of non-scientists in Germany indicated that public opinion may be moving in the yes direction.

Though gene editing technology isnt yet at the point where we can genetically alter unborn individuals at the clinical level by applying gene editing tools like CRISPR/Cas9 to embryos, sperm or egg cells the technology is rapidly advancing.

Debate over the subject was rekindled last year, when a Chinese researcher announced that he used CRISPR to create the worlds first gene-edited babies. According to the Associated Press, He Jiankui said he altered embryos for seven couples during fertility treatments. The treatment was designed to protect the children of an HIV-positive parent by disabling a gene that would have allowed the virus to enter their cells. The trial resulted in one pregnancy twins who were born in November. According to the Associated Press, the researcher said:

I feel a strong responsibility that its not just to make a first, but also make it an example. Society will decide what to do next.

The gene-editing community has grappled with the unexpected announcement in the following months. It was not well received by other researchers, with many of them condemning the action. Feng Zhang, one of the inventors of CRISPR, called for an moratorium on gene-edited babies. Zhang wrote:

Not only do I see this as risky, but I am also deeply concerned about the lack of transparency surrounding this trial. All medical advances, gene editing or otherwise and particularly those that impact vulnerable populations, should be cautiously and thoughtfully tested, discussed openly with patients, physicians, scientists, and other community members, and implemented in an equitable way.

Its not unusual to see articles and reports in the media focusing on the worst possible scenarios when it comes to genome editing, as is often the case with new or particularly disruptive technologies. The mere mention of the topic tends to incite fears of a New Eugenics master race or exacerbation of medical inequalities along class lines where the poor are barred from genetic interventions and the rich have easy access. Still others, such as some Catholic and Muslim groups, worry that genetic manipulation of any sort is interfering with divine plans.

These kinds of fears have been gradually loosening their hold on our imaginations, while the coolly-measured possibilities of gene editing nothing more than a new and innovative medical tool are winning over. And as with any new technology, it comes with a particular set of risks and benefits that must be taken into consideration. The slow change in public attitude toward gene editing is evident in groups such as Germanys Citizens Delphi Germline Therapy project at Karlsruhe Institute of Technology (KIT).

After debating detailed information on the risks and benefits over the course of a few months, and undergoing a rigorous participation process that combined aspects of Citizens Jury with the Delphi survey method, the group has called for the loosening of bans on germline gene editing research in Germany. Their final report was presented at Berlin Science Week in late 2018. And though their verdict only applies to the current ban in Germany, which includes a ban on basic research in germline cells, the groups decision could have an impact on regulations around the world. Indeed, the participants were keen on having Germany play a more active role in the development of international gene editing guidelines.

Regarding the Citizens Jury and Delphi survey methods used, Ralf Grtker, who developed and carried out the project in collaboration with the Department of Science Communication at the Institute for German Studies at KIT, stated, The process is geared towards working on a complex topic with a group of laypeople, empower them to make an informed judgment, and eventually reach recommendations for politicians. Though the group only consisted of 26 German citizens, the group was representative of the population, and the topic of gene editing was thoroughly investigated from a variety of angles.

The group acknowledged the risks of genome editing, such as unknown and off-target effects, but ultimately agreed that the way forward was to open the doors to germline gene editing research at the national and global levels. Their conclusions also echoed those made by genetics experts in a position statement published in the August 2017 issue of the American Journal of Human Genetics, which stated, At this time, given the nature and number of unanswered scientific, ethical, and policy questions, it is inappropriate to perform germline gene editing that culminates in human pregnancy.

But to answer those scientific, ethical, and policy questions, basic research is crucial. And lines of communication need to be open between countries, and between scientists and non-scientists. Without these measures, misunderstandings and missed clinical opportunities will proliferate, along with the dangers of underground gene editing and gene editing tourism. Several governments around the world are grappling with heated and unresolved debates regarding germline gene editing, which in several cases appear to be impeding research.

Though a great deal of cooperation and knowledge exchange is taking place between countries, other nations have yet to move away from all-out bans on germline gene editing. Canada, for example, has criminalized germline gene editing under the 2004 Assisted Human Reproduction Act. Penalties for the crime of germline gene tampering include a $500,000 CAD fine and up to 10 years of jail time.

Following a presentation at the annual Till & McCulloch Meetings of stem-cell and regenerative-medicine researchers, Bartha Knoppers, a health policy expert at McGill University in Montreal, said, Canadas policy has simply shut down discussion (about gene editing). We need to start to talk. Some Canadian scientists worry that Canada is lagging behind in gene editing research, unable to fully participate in the global conversation taking place on this important subject.

At the other end of the spectrum are countries like Japan, where a proposal allowing for the use of gene editing tools in human embryos has been drafted. The new guidelines would restrict manipulation of human embryos for reproduction, though this restriction would likely not be legally binding.

Though several countries have formal bans in place restricting germline gene editing, many of these same countries allow for somatic (or non-reproductive) gene editing applications, which is when gene editing tools are applied to adult cells. Somatic gene edits are not passed on to future generations, so there is less concern about the possible repercussions.

A version of this article previously ran on the GLP on Nov 27, 2018.

Kristen Hovet covers genetics, medical innovations, and the intersection of sociology and culture. Follow her on her website or Twitter @kristenhovet

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Brave new world? Why the public might be ready for gene-edited babies - Genetic Literacy Project

Stem Cell Therapies Market research to Witness a Healthy Growth during 2015 2025 – Lake Shore Gazette

Stem cells are undifferentiated biological cells, and having remarkable potential to divide into any kind of other cells. When a stem cell divides, each new cell will be a new stem cell or it will be like another cell which is having specific function such as a muscle cell, a red blood cell, brain cell and some other cells.

There are two types of stem cells

Stem cells harvested from umbilical cord blood just after birth. And this cells can be stored in specific conditions. Stem cells also can be harvest from bone marrow, adipose tissue.

Embryonic cells can differentiate into ectoderm, endoderm and mesoderm in developing stage. Stem cells used in the therapies and surgeries for regeneration of organisms or cells, tissues.

Stem cells are used for the treatment of Gastro intestine diseases, Metabolic diseases, Immune system diseases, Central Nervous System diseases, Cardiovascular diseases, Wounds and injuries, Eye diseases, Musculoskeletal disorders.

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Harvesting of Adult cell is somewhat difficult compare to embryonic cells. Because Adult cells available in the own body and it is somewhat difficult to harvest.

Stem Cell TherapiesMarket: Drivers and Restraints

Technology advancements in healthcare now curing life threatening diseases and giving promising results. Stem Cell Therapies having so many advantages like regenerating the other cells and body organisms. This is the main driver for this market. These therapies are useful in many life threatening treatments. Increasing the prevalence rate of diseases are driven the Stem Cell Therapies market, it is also driven by increasing technology advancements in healthcare. Technological advancements in healthcare now saving the population from life threatening complications.

Increasing funding from government, private organizations and increasing the Companies focus onStem cell therapiesare also driven this market

However, Collecting the Embryonic Stem cells are easy but Collecting Adult Stem cell or Somatic Stem cells are difficult and also we have to take more precautions for storing the collected stem cells.

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Stem Cell TherapiesMarket: Segmentation

Stem Cell Therapies are segmented into following types

Based on treatment:

Based on application:

Based on End User:

Stem Cell TherapiesMarket: Overview

With rapid technological advantage in healthcare and its promising results, the use of Stem Cell Therapies will increase and the market is expected to have a double digit growth in the forecast period (2015-2025).

Stem Cell TherapiesMarket: Region- wise Outlook

Depending on geographic regions, the global Stem Cell Therapies market is segmented into seven key regions: North America, South America, Eastern Europe, Western Europe, Asia Pacific excluding Japan, Japan and Middle East & Africa.

The use of Stem Cell Therapies is high in North America because it is highly developed region, having good technological advancements in healthcare setup and people are having good awareness about health care. In Asia pacific region china and India also having rapid growth in health care set up. Europe also having good growth in this market.

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Stem Cell TherapiesMarket: Key Players

Some of the key players in this market are

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Stem Cell Therapies Market research to Witness a Healthy Growth during 2015 2025 - Lake Shore Gazette