The thyroid is a butterfly-shaped gland in front of the windpipe in your neck, that secretes thyroid hormone which regulates your metabolism. The storage form of thyroid hormone is T4, which contains four iodine atoms. Once this is released into the blood, it is converted to the more active form called T3, which contains three iodine atoms.

Thyroid hormone production is regulated by TSH (thyroid stimulating hormone), which is secreted by the pituitary gland located at the base of your brain. If the thyroid is unable to produce enough thyroid hormone, the pituitary tries to rev it up by producing more TSH. The TSH level can be checked in the lab through a blood test. Normal ranges vary somewhat according to the lab, but in general are between 0.4 and 4.0. A TSH level higher than normal indicates hypothyroidism, while an abnormally low level indicates hyperthyroidism (an overactive thyroid gland).

For optimal health and vitality, its important that your TSH level remains in the normal range. Hypothyroidism, manifested by high TSH levels and low T4 levels, can cause the following: fatigue; sensitivity to cold; dry skin; constipation; weight gain; facial puffiness; hoarseness; weakness; aching; hair loss; depression; high cholesterol; slow pulse; and memory problems. Hypothyroidism in a pregnant woman results in lower IQ in the newborn, and if the hypothyroidism is severe, cretinism results, which is associated with multiple deficits including mental retardation.

The following factors can cause hypothyroidism:

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Worldwide, the most common cause is lack of iodine, which as noted above is an important component of thyroid hormone. Iodine is a naturally occurring element that occurs in varying amounts in soils throughout the world. Food grown in iodine-poor soils results in low iodine levels in humans who eat that food, resulting in hypothyroidism. In the United States and most other developed countries, iodine is added to most salt you buy at the grocery store in order to avoid iodine deficiency.

In developed countries the most common cause of low thyroid is an autoimmune disease called Hashimotos thyroiditis, where a rogue immune system attacks the thyroid gland.

Other causes of hypothyroidism include surgery or radiation for hyperthyroidism or for thyroid cancer.

Rarely, low thyroid is caused by medications such as lithium.

To make an accurate diagnosis of low thyroid in a person with an elevated TSH, a T4 level should be drawn. Sometimes a blood test for thyroid antibodies is ordered as well, to check for Hashimotos disease. In classic hypothyroidism, the T4 level is low, and the treatment is straight-forward: daily levothyroxine (T4) pills, which should be taken first thing in the morning, at least 30 minutes before eating food or taking other medications including supplements. When the TSH is a few points above normal but the T4 is normal, treatment is controversial.

Once you start thyroid replacement, your TSH should be checked in around six weeks, and if needed your dose adjusted. Once your level is stable, it just needs to be checked once a year. Pregnant women need to increase their dose. If your hypothyroidism is treated and your TSH becomes normal, but you still have symptoms such as fatigue, doctors sometimes add T3 (triiodothyronine), to cover the possibility of inadequate conversion in your body of storage T4 to active T3.

Neal Barnard, M.D., is the founding president of Physician Committee for Responsible Medicine. He has authored several books, the latest, which came out in February, called Your Body in Balance. In the chapter on thyroid, he has the following Menu for a Healthy Thyroid:

Iodine: not too much, not too little (both cause problems). There are many health reasons to avoid salt (sodium), but if you avoid iodized salt (keep in mind that sea salt, kosher salt and Himalayan salt are not iodized) you need to take an iodine supplement. The RDA of iodine for an adult is 150 mcg, 220 if pregnant, 290 if breast feeding. You can get your RDA by eating seaweed (sea vegetables) on a regular basis, and the RDA is present in many vitamin/mineral preparations or as a single iodine supplement. Avoid kelp due to too much iodine, and hijiki seaweed which can be contaminated with arsenic.

Dr. Barnard also says to avoid animal products: People who avoid meat, dairy products, and eggs have been shown in research studies to have the lowest risk of hypo- or hyperthyroidism, probably because of lower rate of autoimmune disease in people on a plant-based diet. According to Dr. Barnard, when people switch from an animal-based to a plant-based diet, they often are able to cut back on their thyroid replacement dose, and sometimes their hypothyroidism resolves.

Greg Feinsinger, M.D. is a retired family physician who has a nonprofit: Prevention and Treatment of Disease Through Nutrition. He is available by appointment for free consultations (379-5718)

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Doctor's Tip: Risks, causes, treatment and prevention of hypothyroidism - Glenwood Springs Post Independent

Recommendation and review posted by Bethany Smith

Today marks the first Fathers Day since our middle daughter, The Architect, 7, was diagnosed with type 1 diabetes (T1D). This subject comes up in my writing occasionally, but if youre just joining us, T1D (formerly known as juvenile or insulin-dependent diabetes) is an autoimmune disease that leads a persons pancreas to stop producing insulin, the hormone responsible for regulating blood sugar. It is not known what causes T1D, and unlike the much more common type 2 diabetes, it cannot be prevented or delayed with lifestyle changes.

Type 1 requires multiple daily injections of insulin through a syringe, pen or insulin pump and careful monitoring of blood glucose levels, lest the patient suffer from extremely high or low blood sugar, both of which can lead to short- and long-term complications.

In October, our lives changed forever as this diagnosis was handed down in a physicians well-rehearsed but kindly delivered speech in a hospital room on the 11th floor of Nationwide Childrens Hospital. Weve been trying to figure out the rhythm of life since then, and weve made some strides.

I say all this because during previous Fathers Days, I could have come up with a number of different adjectives to describe Mr. Roy: Hard Worker, Provider, Math Helper, Protector, Player of Board Games, Enforcer of Naps.

This year I have watched Mr. Roy acquire a new adjective: Advocate.

Merriam-Webster defines advocate as one who pleads the cause of another.

To some extent, all of us, when we become parents, are endowed with the title of advocate, but for parents of children with special needs, for example, or medical conditions, that title takes on a new meaning.

In that hospital room in October, as I struggled to the point of numbness with the burden of the unknowns that had been thrust upon us, Mr. Roy stepped into his advocacy role with vigor and grace. He didnt know any more than I did about the causes of diabetes or how to manage it; he didnt care. He knew that he loved his little girl and would stop at nothing to make sure she had everything she needed to survive.

He was the first one to give her insulin in her hospital bed, when I was too afraid Id mess it up; the one who read over all the papers and made appointments with the doctors and educators and school personnel.

Our first night home from the hospital, he was the one squeezing her finger to get a drop of blood at 3 a.m. when she was half-asleep and we had to make sure her levels werent tanking.

Every day, at every meal occasionally now, but especially in those early days he faithfully filled out the charts tracking her progress. He got on the phone with the diabetes educator when we noticed a trend that seemed off. Spoke with them late into the night when The Architect was sick and we were trying to manage blood sugar on top of sickness on top of two other kids.

More often than not, he is the one who calculates her insulin for every meal and gets her pen ready not because I cant or dont, but because he assumed the role like a bird somehow learns to fly after it leaves the nest. Hes willing to try things to improve her condition when I am paralyzed and sticking to the books.

A few months ago Mr. Roy went into a tattoo parlor on his lunch break to have the outline of a drop of blood tattooed on his forearm. Not because hes a cool, hip dad (although you are, Honey), but because he loves his girls inside and out.

In our marriage partnership, I like to think we do a pretty good job of balancing roles. Please dont get the impression from these words that I do nothing, because I, too, am learning. But to watch my husband adopt this new adjective has been a blessing I am especially celebrating this Fathers Day.

To all dads everywhere: Whatever your adjectives, thank you for the roles you take on. Its a blessing to celebrate you today.

Abbey Roy is a mom of three girls who make every day an adventure. She writes to maintain her sanity. You can probably reach her at amroy@nncogannett.com, but responses are structured around bedtimes and weekends.

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Abbey's Road: In celebration of Dad-vocates - The Newark Advocate

Recommendation and review posted by Bethany Smith

I was never one to imagine my future children. While others dream about everything from their future kids' names to their looks and passions, my thoughts have always been a bit more self-centered. I wouldn't say that I never want to have kids of my own, but as a 22-year-old graduating into an unprecedented pandemic and economic downturn, the idea of bringing another life into this world that's dependent on me for survival is more stressful than anything else. So, I didn't really give it much thought . . . until I was forced to.

About a year ago, I had to consider the idea of becoming a parent in a much more immediate way. I'm a trans woman, and one of the first steps toward transitioning is hormone therapy, which also causes infertility. At the time, all I wanted to do was start my transition and leave the body I felt so uncomfortable in behind, but instead, I had to make a decision I didn't imagine dealing with for years to come: whether I would ever have my own children.

When I first tried to start hormone therapy, it felt like an invisible force was standing in my way. I vividly remember taking the train into Chicago to the Howard Brown Health Clinic. I was running late, so I sprinted the mile walk between the train and the clinic. I got there only to find out that I was at the wrong place, and I was told to reschedule my appointment for another day. A week later, I was sitting on a sterilized hospital bed waiting for the doctor to come in and ask me why I wanted to change my body so drastically. It was scary, but I was ready for anything. I was convinced I'd have to fight for my right to access the hormones I needed because no one was going to simply hand over such life-saving drugs to a baby trans woman who still had stubble on their chin and a voice that sounded like it had something to hide.

Of course, there was no fight. My physician was on my side 110 percent. She asked me why I had decided to transition and what outcome I was looking for. I told her I didn't expect hormone therapy to change me into the femme I envisioned myself as overnight, but I wanted to fix what felt like a chemical imbalance within myself. My body was overrun with testosterone, and I was reaching for something that a daily dose of estrogen and testosterone blockers could turn from fantasy into reality.

That was when she informed me that if I truly committed to these pills, I would have to accept the fact that I would be infertile. She gave me several options for banking my sperm, but all I could hear was another obstacle in the way of my transition. My first thought was, "Who wants kids anyway?" But because I've always been a fairly responsible person, I also thought that I should respect my future self and give her the option of having her own biological children. I was torn, but the part of me that was living in the moment I had been waiting so long for told my doctor to give me the pills anyway.

I left the clinic that day unsure of my next step while also holding a month's supply of estrogen pills in my backpack. When I got home, I took one just to know the taste. I placed it under my tongue and waited for it to dissolve, but even that felt like an eternity and I swallowed the pill whole. I was just so tired of the imbalance I had felt for so long, but I also knew I might be rushing what would surely be a lifelong journey.

For the next month, I held the pills close while mulling over whether my future biological children were worth postponing my transition. I hadn't taken a pill since the first day I got them, and I started reaching out to sperm banks to see how difficult this process would be. At the time, I wasn't close to any other trans women going through hormone therapy, and the only advice I got was from trans women telling their stories on YouTube. Some who never banked their sperm before transitioning were forced to stop taking estrogen and turn away from the comfort years on hormone therapy had brought them in an effort to conceive. Even though the prospect of kids felt so far off in my mind, their stories reminded me that whenever I started my transition, I never wanted to go back to that feeling of imbalance, even for a child. I found an affordable sperm bank just west of Chicago, and in January of 2019, I banked my sperm and officially began my transition.

It's been over a year and I'm still paying to keep my sperm viable. My best chance for biological children is frozen in an egg yolk in a warehouse in Illinois. Its existence is reevaluated yearly based on my ability to pay a $250 bill, and brings with it a reminder that my connection to motherhood is tentative and sterilized. So far I haven't paid my second bill, and I question how long I'm willing to preserve my ability to conceive children. I never truly decided whether or not I wanted kids, only that to deny myself the opportunity felt foolish. Or maybe it's foolish to prolong an inevitable letting go. The only truth I know is that I'm a 22-year-old trans woman on her way to becoming. I have no idea where that journey will take me, but at the end of the day right now, I only have my choices to keep me company. And so far, I'm pretty happy with that.

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I'm a 22-Year-Old Trans Woman, and a Year Ago I Was Forced to Decide If I Ever Want to Have Kids - POPSUGAR

Recommendation and review posted by Bethany Smith

BMR can be helpful is losing weight is one of your goals.

No matter what your goals, when it comes to nutrition and body recomposition, knowledge is power. And understanding a few key principles, like how to track macros, calories and how to exercise more effectively is especially important if weight loss is your goal.

If you want to lose body fat, gain muscle or maintain your weight, an important metric you should know about is Basal Metabolic Rate (BMR). Your BMR is the minimum number of calories that your body needs to function at rest. You may think you only need energy to exercise or complete tasks, but your body has a specific energy need just to complete basic functions like breathing and regulating your hormone levels.

Your BMR is not in itself a tool for weight loss, but it can be a helpful starting point for figuring out how to adjust your diet and exercise goals. The problem with a lot of diets and weight loss programs out there is that they are "one size fits all." But every person is different, so taking one meal plan and applying it to multiple people just does not work. Your BMR is calculated based on several different factors that are personal to you, like your age, gender, current weight and activity level.

Keep reading to find out about what BMR is, how to calculate it and how it can help you with your nutrition and exercise goals.

BMR calculators use several different factors, like age and gender, to determine your BMR.

Many people use BMR as a starting point to calculate their daily calorie needs and how to best adjust them to reach their goals. In fact, many macro calculators, like the popular IIFYM, incorporate BMR into their calculations for telling you about your calorie intake and macro needs based on your goals.

One common misconception about BMR is that it is the amount of calories your body burns at rest, but that is a different metric -- Resting metabolic rate or (RMR). Your BMR is what energy your body needs to perform basic functions, while RMR is the amount of calories that your body burns while at rest. Some people use the measurements interchangeably, but they aren't necessarily the same thing.

There are many different calculators available online that can calculate your estimated BMR. Note that some of them will ask you to enter your body fat percentage, which many people do not know. If you don't, you can make an estimate or use the images provided (like IIFYM does) to guess.

Some of the best BMR calculators:

Once you start learning about your BMR, you will likely also find information on TDEE (total daily energy expenditure) since BMR is often calculated first to find TDEE.

Your BMR tells you your calorie needs, when you take that number plus how much you burn every day during normal activity and exercise, you get your TDEE. So really, TDEE is the number that you work off of for figuring out how to adjust macros or calories for body composition goals, according to IIFYM.

Weight loss is tricky, but using calculators that factor in your BMR are helpful for taking a more customized approach for your calories and macronutrient needs. The way that most macros and BMR-based calculators work is by factoring in your TDEE with your goals.

If you want to lose weight, you have to be in a calorie deficit, meaning the calculator will set your daily food intake to equal less calories than what you burn. Sometimes when you take an online quiz to find this number, you will be asked how fast you'd like to lose weight. Then the calorie deficit will be adjusted accordingly. The faster you want results, the more extreme you will have to be with cutting calories. But many experts say that slow and steady is optimal compared to trying to lose a lot of weight quickly.

If you want to maintain your weight instead of lose or gain, then knowing your BMR and TDEE can help you know how many calories you should aim to consume each day to maintain your weight. On the flipside of weight loss is gaining muscle mass. This too requires that you strategically approach your nutrition and add calories into your day (likely in the form of protein and carbs) to make sure you can gain muscle.

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The information contained in this article is for educational and informational purposes only and is not intended as health or medical advice. Always consult a physician or other qualified health provider regarding any questions you may have about a medical condition or health objectives.

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Knowing your BMR is crucial for losing weight -- here's how to find yours - CNET

Recommendation and review posted by Bethany Smith

Global Stem Cell Therapy Market report is aimed at highlighting a first-hand documentation of all the best practices in the Stem Cell Therapy industry that subsequently set the growth course active. These vital market oriented details are highly crucial to overcome cut throat competition and all the growth oriented practices typically embraced by frontline players in the Stem Cell Therapy market. Various factors and touch points that the research highlights in the report is a holistic, composite amalgamation of product portfolios of market participants, growth multiplying practices and solutions, sales gateways as well as transaction modes that coherently reflect a favorable growth prospect scenario of the market.

Get sample copy of Stem Cell Therapy Market report @https://www.adroitmarketresearch.com/contacts/request-sample/691

In addition, study report offers an array of opportunities for the players participating in the industry. This ultimately leads into the growth of the global Stem Cell Therapy market. Furthermore, report offers a comprehensive study on market size, revenue, sales, growth factors and risks involved in the growth of the market during the forecast period. The factors which are influencing the growth the market are mentioned in the report as well as the challenges which can hamper the growth of the market over the forecast period.

Full browse the report description and TOC:https://www.adroitmarketresearch.com/industry-reports/stem-cell-therapy-market

The research report encourages the readers to comprehend the importance of quality, shortcomings if any and deep investigation for every member independently by giving the global data of great importance about the market. Consequently, the research report presents the organization profiles and deals investigation of the considerable number of vendors which can assist the customers with taking better choice of the products and services. The end clients of the global Stem Cell Therapy market can be sorted based on size of the endeavour. This research report presents the open doors for the players of the global Stem Cell Therapy market. It additionally offers plans of action which can be taken and market conjectures that would be required.

Global Stem Cell Therapy market is segmented based by type, application and region.

Based on Type, the market has been segmented into:

Based on cell source, the market has been segmented into,

Adipose Tissue-Derived Mesenchymal SCsBone Marrow-Derived Mesenchymal SCsEmbryonic SCsOther Sources

Based on application, the market has been segmented into:

Based on therapeutic application, the market has been segmented into,

Musculoskeletal DisordersWounds & InjuriesCardiovascular DiseasesGastrointestinal DiseasesImmune System DiseasesOther Applications

The company profile section also focusses on companies planning expansions along with mergers & acquisitions, new initiatives, R&D updates and financial updates. But, one of the most important aspects focused in this study is the regional analysis. Region segmentation of markets helps in detailed analysis of the market in terms of business opportunities, revenue generation potential and future predictions of the market. For Stem Cell Therapy market report, the important regions highlighted are North America, South America, Asia, Europe and Middle East. The companies focused on in this report are pioneers in the Stem Cell Therapy market. The uplifting of any region in the global market is dependent upon the market players working in that region.

A qualitative and quantitative analysis of the Stem Cell Therapy market valuations for the expected period is presented to showcase the economic appetency of the global Stem Cell Therapy industry. In addition to this, the global research report comprises significant data regarding the market segmentation which is intended by primary and secondary research methodologies. This research report offers an in-depth analysis of the global Stem Cell Therapy industry with recent and upcoming market trends to offer the impending investment in the Stem Cell Therapy market. The report includes a comprehensive analysis of the industry size database along with the market prediction for the mentioned forecast period. Furthermore, the Stem Cell Therapy market research study offers comprehensive data about the opportunities, key drivers, and restraints with the impact analysis.

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Adroit Market Research is an India-based business analytics and consulting company. Our target audience is a wide range of corporations, manufacturing companies, product/technology development institutions and industry associations that require understanding of a markets size, key trends, participants and future outlook of an industry. We intend to become our clients knowledge partner and provide them with valuable market insights to help create opportunities that increase their revenues. We follow a code Explore, Learn and Transform. At our core, we are curious people who love to identify and understand industry patterns, create an insightful study around our findings and churn out money-making roadmaps.

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Ryan JohnsonAccount Manager Global3131 McKinney Ave Ste 600, Dallas,TX 75204, U.S.APhone No.: USA: +1 972-362 -8199 / +91 9665341414

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Global Stem Cell Therapy Market 2020: Size, Share, Growth Rate, Revenue and Volume, Key-Players, Top Regions and Forecast Till 2025 - Cole of Duty

Recommendation and review posted by Bethany Smith

Los Angeles, United State: Complete study of the global CRISPR And CRISPR-Associated (Cas) Genes market is carried out by the analysts in this report, taking into consideration key factors like drivers, challenges, recent trends, opportunities, advancements, and competitive landscape. This report offers a clear understanding of the present as well as future scenario of the global CRISPR And CRISPR-Associated (Cas) Genes industry. Research techniques like PESTLE and Porters Five Forces analysis have been deployed by the researchers. They have also provided accurate data on CRISPR And CRISPR-Associated (Cas) Genes production, capacity, price, cost, margin, and revenue to help the players gain a clear understanding into the overall existing and future market situation.

The research study includes great insights about critical market dynamics, including drivers, restraints, trends, and opportunities. It also includes various types of market analysis such as competitive analysis, manufacturing cost analysis, manufacturing process analysis, price analysis, and analysis of market influence factors. It is a complete study on the global CRISPR And CRISPR-Associated (Cas) Genes market that can be used as a set of effective guidelines for ensuring strong growth in the coming years. It caters to all types of interested parties, viz. stakeholders, market participants, investors, market researchers, and other individuals associated with the CRISPR And CRISPR-Associated (Cas) Genes business.

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It is important for every market participant to be familiar with the competitive scenario in the global CRISPR And CRISPR-Associated (Cas) Genes industry. In order to fulfil the requirements, the industry analysts have evaluated the strategic activities of the competitors to help the key players strengthen their foothold in the market and increase their competitiveness.

Key Players Mentioned in the Global CRISPR And CRISPR-Associated (Cas) Genes Market Research Report: , Caribou Biosciences, Addgene, CRISPR THERAPEUTICS, Merck KGaA, Mirus Bio LLC, Editas Medicine, Takara Bio USA, Thermo Fisher Scientific, Horizon Discovery Group, Intellia Therapeutics, GE Healthcare Dharmacon

Global CRISPR And CRISPR-Associated (Cas) Genes Market Segmentation by Product: Genome Editing, Genetic engineering, gRNA Database/Gene Librar, CRISPR Plasmid, Human Stem Cells, Genetically Modified Organisms/Crops, Cell Line Engineering

Global CRISPR And CRISPR-Associated (Cas) Genes Market Segmentation by Application: , Biotechnology Companies, Pharmaceutical Companies, Academic Institutes, Research and Development Institutes

The report has classified the global CRISPR And CRISPR-Associated (Cas) Genes industry into segments including product type and application. Every segment is evaluated based on growth rate and share. Besides, the analysts have studied the potential regions that may prove rewarding for the CRISPR And CRISPR-Associated (Cas) Genes manufcaturers in the coming years. The regional analysis includes reliable predictions on value and volume, thereby helping market players to gain deep insights into the overall CRISPR And CRISPR-Associated (Cas) Genes industry.

Additionally, the industry analysts have studied key regions including North America, Europe, Asia Pacific, Latin America, and Middle East and Africa, along with their respective countries. Here, they have given a clear-cut understanding of the present and future situations of the global CRISPR And CRISPR-Associated (Cas) Genes industry in key regions. This will help the key players to focus on the lucrative regional markets.

Key questions answered in the report:

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Table of Content

Table of Contents Executive Summary 1 CRISPR And CRISPR-Associated (Cas) Genes Market Overview1.1 Product Overview and Scope of CRISPR And CRISPR-Associated (Cas) Genes1.2 CRISPR And CRISPR-Associated (Cas) Genes Segment by Type1.2.1 Global CRISPR And CRISPR-Associated (Cas) Genes Production Growth Rate Comparison by Type (2014-2025)1.2.2 Genome Editing1.2.3 Genetic engineering1.2.4 gRNA Database/Gene Librar1.2.5 CRISPR Plasmid1.2.6 Human Stem Cells1.2.7 Genetically Modified Organisms/Crops1.2.8 Cell Line Engineering1.3 CRISPR And CRISPR-Associated (Cas) Genes Segment by Application1.3.1 CRISPR And CRISPR-Associated (Cas) Genes Consumption Comparison by Application (2014-2025)1.3.2 Biotechnology Companies1.3.3 Pharmaceutical Companies1.3.4 Academic Institutes1.3.5 Research and Development Institutes1.3 Global CRISPR And CRISPR-Associated (Cas) Genes Market by Region1.3.1 Global CRISPR And CRISPR-Associated (Cas) Genes Market Size Region1.3.2 North America Status and Prospect (2014-2025)1.3.3 Europe Status and Prospect (2014-2025)1.3.4 China Status and Prospect (2014-2025)1.3.5 Japan Status and Prospect (2014-2025)1.3.6 Southeast Asia Status and Prospect (2014-2025)1.3.7 India Status and Prospect (2014-2025)1.4 Global CRISPR And CRISPR-Associated (Cas) Genes Market Size1.4.1 Global CRISPR And CRISPR-Associated (Cas) Genes Revenue (2014-2025)1.4.2 Global CRISPR And CRISPR-Associated (Cas) Genes Production (2014-2025) 2 Global CRISPR And CRISPR-Associated (Cas) Genes Market Competition by Manufacturers2.1 Global CRISPR And CRISPR-Associated (Cas) Genes Production Market Share by Manufacturers (2014-2019)2.2 Global CRISPR And CRISPR-Associated (Cas) Genes Revenue Share by Manufacturers (2014-2019)2.3 Global CRISPR And CRISPR-Associated (Cas) Genes Average Price by Manufacturers (2014-2019)2.4 Manufacturers CRISPR And CRISPR-Associated (Cas) Genes Production Sites, Area Served, Product Types2.5 CRISPR And CRISPR-Associated (Cas) Genes Market Competitive Situation and Trends2.5.1 CRISPR And CRISPR-Associated (Cas) Genes Market Concentration Rate2.5.2 CRISPR And CRISPR-Associated (Cas) Genes Market Share of Top 3 and Top 5 Manufacturers2.5.3 Mergers & Acquisitions, Expansion 3 Global CRISPR And CRISPR-Associated (Cas) Genes Production Market Share by Regions3.1 Global CRISPR And CRISPR-Associated (Cas) Genes Production Market Share by Regions3.2 Global CRISPR And CRISPR-Associated (Cas) Genes Revenue Market Share by Regions (2014-2019)3.3 Global CRISPR And CRISPR-Associated (Cas) Genes Production, Revenue, Price and Gross Margin (2014-2019)3.4 North America CRISPR And CRISPR-Associated (Cas) Genes Production3.4.1 North America CRISPR And CRISPR-Associated (Cas) Genes Production Growth Rate (2014-2019)3.4.2 North America CRISPR And CRISPR-Associated (Cas) Genes Production, Revenue, Price and Gross Margin (2014-2019)3.5 Europe CRISPR And CRISPR-Associated (Cas) Genes Production3.5.1 Europe CRISPR And CRISPR-Associated (Cas) Genes Production Growth Rate (2014-2019)3.5.2 Europe CRISPR And CRISPR-Associated (Cas) Genes Production, Revenue, Price and Gross Margin (2014-2019)3.6 China CRISPR And CRISPR-Associated (Cas) Genes Production (2014-2019)3.6.1 China CRISPR And CRISPR-Associated (Cas) Genes Production Growth Rate (2014-2019)3.6.2 China CRISPR And CRISPR-Associated (Cas) Genes Production, Revenue, Price and Gross Margin (2014-2019)3.7 Japan CRISPR And CRISPR-Associated (Cas) Genes Production (2014-2019)3.7.1 Japan CRISPR And CRISPR-Associated (Cas) Genes Production Growth Rate (2014-2019)3.7.2 Japan CRISPR And CRISPR-Associated (Cas) Genes Production, Revenue, Price and Gross Margin (2014-2019) 4 Global CRISPR And CRISPR-Associated (Cas) Genes Consumption by Regions4.1 Global CRISPR And CRISPR-Associated (Cas) Genes Consumption by Regions4.2 North America CRISPR And CRISPR-Associated (Cas) Genes Consumption (2014-2019)4.3 Europe CRISPR And CRISPR-Associated (Cas) Genes Consumption (2014-2019)4.4 China CRISPR And CRISPR-Associated (Cas) Genes Consumption (2014-2019)4.5 Japan CRISPR And CRISPR-Associated (Cas) Genes Consumption (2014-2019) 5 Global CRISPR And CRISPR-Associated (Cas) Genes Production, Revenue, Price Trend by Type5.1 Global CRISPR And CRISPR-Associated (Cas) Genes Production Market Share by Type (2014-2019)5.2 Global CRISPR And CRISPR-Associated (Cas) Genes Revenue Market Share by Type (2014-2019)5.3 Global CRISPR And CRISPR-Associated (Cas) Genes Price by Type (2014-2019)5.4 Global CRISPR And CRISPR-Associated (Cas) Genes Production Growth by Type (2014-2019) 6 Global CRISPR And CRISPR-Associated (Cas) Genes Market Analysis by Applications6.1 Global CRISPR And CRISPR-Associated (Cas) Genes Consumption Market Share by Application (2014-2019)6.2 Global CRISPR And CRISPR-Associated (Cas) Genes Consumption Growth Rate by Application (2014-2019) 7 Company Profiles and Key Figures in CRISPR And CRISPR-Associated (Cas) Genes Business7.1 Caribou Biosciences7.1.1 Caribou Biosciences CRISPR And CRISPR-Associated (Cas) Genes Production Sites and Area Served7.1.2 CRISPR And CRISPR-Associated (Cas) Genes Product Introduction, Application and Specification7.1.3 Caribou Biosciences CRISPR And CRISPR-Associated (Cas) Genes Production, Revenue, Price and Gross Margin (2014-2019)7.1.4 Main Business and Markets Served7.2 Addgene7.2.1 Addgene CRISPR And CRISPR-Associated (Cas) Genes Production Sites and Area Served7.2.2 CRISPR And CRISPR-Associated (Cas) Genes Product Introduction, Application and Specification7.2.3 Addgene CRISPR And CRISPR-Associated (Cas) Genes Production, Revenue, Price and Gross Margin (2014-2019)7.2.4 Main Business and Markets Served7.3 CRISPR THERAPEUTICS7.3.1 CRISPR THERAPEUTICS CRISPR And CRISPR-Associated (Cas) Genes Production Sites and Area Served7.3.2 CRISPR And CRISPR-Associated (Cas) Genes Product Introduction, Application and Specification7.3.3 CRISPR THERAPEUTICS CRISPR And CRISPR-Associated (Cas) Genes Production, Revenue, Price and Gross Margin (2014-2019)7.3.4 Main Business and Markets Served7.4 Merck KGaA7.4.1 Merck KGaA CRISPR And CRISPR-Associated (Cas) Genes Production Sites and Area Served7.4.2 CRISPR And CRISPR-Associated (Cas) Genes Product Introduction, Application and Specification7.4.3 Merck KGaA CRISPR And CRISPR-Associated (Cas) Genes Production, Revenue, Price and Gross Margin (2014-2019)7.4.4 Main Business and Markets Served7.5 Mirus Bio LLC7.5.1 Mirus Bio LLC CRISPR And CRISPR-Associated (Cas) Genes Production Sites and Area Served7.5.2 CRISPR And CRISPR-Associated (Cas) Genes Product Introduction, Application and Specification7.5.3 Mirus Bio LLC CRISPR And CRISPR-Associated (Cas) Genes Production, Revenue, Price and Gross Margin (2014-2019)7.5.4 Main Business and Markets Served7.6 Editas Medicine7.6.1 Editas Medicine CRISPR And CRISPR-Associated (Cas) Genes Production Sites and Area Served7.6.2 CRISPR And CRISPR-Associated (Cas) Genes Product Introduction, Application and Specification7.6.3 Editas Medicine CRISPR And CRISPR-Associated (Cas) Genes Production, Revenue, Price and Gross Margin (2014-2019)7.6.4 Main Business and Markets Served7.7 Takara Bio USA7.7.1 Takara Bio USA CRISPR And CRISPR-Associated (Cas) Genes Production Sites and Area Served7.7.2 CRISPR And CRISPR-Associated (Cas) Genes Product Introduction, Application and Specification7.7.3 Takara Bio USA CRISPR And CRISPR-Associated (Cas) Genes Production, Revenue, Price and Gross Margin (2014-2019)7.7.4 Main Business and Markets Served7.8 Thermo Fisher Scientific7.8.1 Thermo Fisher Scientific CRISPR And CRISPR-Associated (Cas) Genes Production Sites and Area Served7.8.2 CRISPR And CRISPR-Associated (Cas) Genes Product Introduction, Application and Specification7.8.3 Thermo Fisher Scientific CRISPR And CRISPR-Associated (Cas) Genes Production, Revenue, Price and Gross Margin (2014-2019)7.8.4 Main Business and Markets Served7.9 Horizon Discovery Group7.9.1 Horizon Discovery Group CRISPR And CRISPR-Associated (Cas) Genes Production Sites and Area Served7.9.2 CRISPR And CRISPR-Associated (Cas) Genes Product Introduction, Application and Specification7.9.3 Horizon Discovery Group CRISPR And CRISPR-Associated (Cas) Genes Production, Revenue, Price and Gross Margin (2014-2019)7.9.4 Main Business and Markets Served7.10 Intellia Therapeutics7.10.1 Intellia Therapeutics CRISPR And CRISPR-Associated (Cas) Genes Production Sites and Area Served7.10.2 CRISPR And CRISPR-Associated (Cas) Genes Product Introduction, Application and Specification7.10.3 Intellia Therapeutics CRISPR And CRISPR-Associated (Cas) Genes Production, Revenue, Price and Gross Margin (2014-2019)7.10.4 Main Business and Markets Served7.11 GE Healthcare Dharmacon 8 CRISPR And CRISPR-Associated (Cas) Genes Manufacturing Cost Analysis8.1 CRISPR And CRISPR-Associated (Cas) Genes Key Raw Materials Analysis8.1.1 Key Raw Materials8.1.2 Price Trend of Key Raw Materials8.1.3 Key Suppliers of Raw Materials8.2 Proportion of Manufacturing Cost Structure8.3 Manufacturing Process Analysis of CRISPR And CRISPR-Associated (Cas) Genes8.4 CRISPR And CRISPR-Associated (Cas) Genes Industrial Chain Analysis 9 Marketing Channel, Distributors and Customers9.1 Marketing Channel9.1.1 Direct Marketing9.1.2 Indirect Marketing9.2 CRISPR And CRISPR-Associated (Cas) Genes Distributors List9.3 CRISPR And CRISPR-Associated (Cas) Genes Customers 10 Market Dynamics10.1 Market Trends10.2 Opportunities10.3 Market Drivers10.4 Challenges10.5 Influence Factors 11 Global CRISPR And CRISPR-Associated (Cas) Genes Market Forecast11.1 Global CRISPR And CRISPR-Associated (Cas) Genes Production, Revenue Forecast11.1.1 Global CRISPR And CRISPR-Associated (Cas) Genes Production Growth Rate Forecast (2019-2025)11.1.2 Global CRISPR And CRISPR-Associated (Cas) Genes Revenue and Growth Rate Forecast (2019-2025)11.1.3 Global CRISPR And CRISPR-Associated (Cas) Genes Price and Trend Forecast (2019-2025)11.2 Global CRISPR And CRISPR-Associated (Cas) Genes Production Forecast by Regions (2019-2025)11.2.1 North America CRISPR And CRISPR-Associated (Cas) Genes Production, Revenue Forecast (2019-2025)11.2.2 Europe CRISPR And CRISPR-Associated (Cas) Genes Production, Revenue Forecast (2019-2025)11.2.3 China CRISPR And CRISPR-Associated (Cas) Genes Production, Revenue Forecast (2019-2025)11.2.4 Japan CRISPR And CRISPR-Associated (Cas) Genes Production, Revenue Forecast (2019-2025)11.3 Global CRISPR And CRISPR-Associated (Cas) Genes Consumption Forecast by Regions (2019-2025)11.3.1 North America CRISPR And CRISPR-Associated (Cas) Genes Consumption Forecast (2019-2025)11.3.2 Europe CRISPR And CRISPR-Associated (Cas) Genes Consumption Forecast (2019-2025)11.3.3 China CRISPR And CRISPR-Associated (Cas) Genes Consumption Forecast (2019-2025)11.3.4 Japan CRISPR And CRISPR-Associated (Cas) Genes Consumption Forecast (2019-2025)11.4 Global CRISPR And CRISPR-Associated (Cas) Genes Production, Revenue and Price Forecast by Type (2019-2025)11.5 Global CRISPR And CRISPR-Associated (Cas) Genes Consumption Forecast by Application (2019-2025) 12 Research Findings and Conclusion 13 Methodology and Data Source13.1 Methodology/Research Approach13.1.1 Research Programs/Design13.1.2 Market Size Estimation13.1.3 Market Breakdown and Data Triangulation13.2 Data Source13.2.1 Secondary Sources13.2.2 Primary Sources13.3 Author List13.4 Disclaimer

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CRISPR And CRISPR-Associated (Cas) Genes Market Size, Key Trends, Challenges and Standardization, Research, Key Players, Economic Impact and Forecast...

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The report on the CRISPR Genome Editing market provides a birds eye view of the current proceeding within the CRISPR Genome Editing market. Further, the report also takes into account the impact of the novel COVID-19 pandemic on the CRISPR Genome Editing market and offers a clear assessment of the projected market fluctuations during the forecast period. The different factors that are likely to impact the overall dynamics of the CRISPR Genome Editing market over the forecast period (2019-2029) including the current trends, growth opportunities, restraining factors, and more are discussed in detail in the market study.

For top companies in United States, European Union and China, this report investigates and analyzes the production, value, price, market share and growth rate for the top manufacturers, key data from 2019 to 2025.

The CRISPR Genome Editing market report firstly introduced the basics: definitions, classifications, applications and market overview; product specifications; manufacturing processes; cost structures, raw materials and so on. Then it analyzed the worlds main region market conditions, including the product price, profit, capacity, production, supply, demand and market growth rate and forecast etc. In the end, the CRISPR Genome Editing market report introduced new project SWOT analysis, investment feasibility analysis, and investment return analysis.

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The major players profiled in this CRISPR Genome Editing market report include:

The key players covered in this studyEditas MedicineCRISPR TherapeuticsHorizon DiscoverySigma-AldrichGenscriptSangamo BiosciencesLonza GroupIntegrated DNA TechnologiesNew England BiolabsOrigene TechnologiesTransposagen BiopharmaceuticalsThermo Fisher ScientificCaribou BiosciencesPrecision BiosciencesCellectisIntellia Therapeutics

Market segment by Type, the product can be split intoGenetic EngineeringGene LibraryHuman Stem CellsOthersMarket segment by Application, split intoBiotechnology CompaniesPharmaceutical CompaniesOthers

Market segment by Regions/Countries, this report coversNorth AmericaEuropeChinaJapanSoutheast AsiaIndiaCentral & South America

The study objectives of this report are:To analyze global CRISPR Genome Editing status, future forecast, growth opportunity, key market and key players.To present the CRISPR Genome Editing development in North America, Europe, China, Japan, Southeast Asia, India and Central & South America.To strategically profile the key players and comprehensively analyze their development plan and strategies.To define, describe and forecast the market by type, market and key regions.

In this study, the years considered to estimate the market size of CRISPR Genome Editing are as follows:History Year: 2015-2019Base Year: 2019Estimated Year: 2020Forecast Year 2020 to 2026For the data information by region, company, type and application, 2019 is considered as the base year. Whenever data information was unavailable for the base year, the prior year has been considered.

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Important Information that can be extracted from the Report:

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Globally Leading Manufacturers of CRISPR Genome Editing product Scale up Production to Meet Sharp Spike in Demand Fueled by COVID-78 - Personal Injury...

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Amir Hashemi,1 Masoumeh Ezati,2 Javad Mohammadnejad,3 Behzad Houshmand,4 Shahab Faghihi5

1Department of Life Science Engineering, Faculty of New Sciences and Technologies, University of Tehran, Tehran 14395-1561, Iran; 2Tissue Engineering and Biomaterials Research Center, National Institute of Genetic Engineering and Biotechnology (NIGEB), Tehran 14965/161, Iran; 3Department of Life Science Engineering, Faculty of New Sciences and Technologies, University of Tehran, Tehran 14395-1561, Iran; 4Department of Periodontics, School of Dentistry, Shahid Beheshti University of Medical Sciences, Tehran 19857-17443, Iran; 5Tissue Engineering and Biomaterials Research Center, National Institute of Genetic Engineering and Biotechnology (NIGEB), Tehran 14965/161, Iran

Correspondence: Javad Mohammadnejad; Shahab Faghihi Tel +9821 8609-3078Tel/ Fax +98 21 44787386Fax +98(21)88497324Email mohamadnejad@ut.ac.ir; sfaghihi@nigeb.ac.ir

Background: Ineffective integration has been recognized as one of the major causes of early orthopedic failure of titanium-based implants. One strategy to address this problem is to develop modified titanium surfaces that promote osteoblast differentiation. This study explored titanium surfaces modified with TiO2 nanotubes (TiO2 NTs) capable of localized drug delivery into bone and enhanced osteoblast cell differentiation.Materials and Methods: Briefly, TiO2 NTs were subjected to anodic oxidation and loaded with Metformin, a widely used diabetes drug. To create surfaces with sustainable drug-eluting characteristics, TiO2 NTs were spin coated with a thin layer of chitosan. The surfaces were characterized via scanning electron microscopy, atomic force microscopy, and contact angle measurements. The surfaces were then exposed to mesenchymal bone marrow stem cells (MSCs) to evaluate cell adhesion, growth, differentiation, and morphology on the modified surfaces.Results: A noticeable increase in drug release time (3 days vs 20 days) and a decrease in burst release characteristics (85% to 7%) was observed in coated samples as compared to uncoated samples, respectively. Chitosan-coated TiO2 NTs exhibited a considerable enhancement in cell adhesion, proliferation, and genetic expression of type I collagen, and alkaline phosphatase activity as compared to uncoated TiO2 NTs.Conclusion: TiO2 NT surfaces with a chitosan coating are capable of delivering Metformin to a bone site over a sustained period of time with the potential to enhance MSCs cell attachment, proliferation, and differentiation.

Keywords: titania nanotubes, titanium, osteogenic differentiation, anodization, mesenchymal bone marrow stem cells, MSCs

This work is published and licensed by Dove Medical Press Limited. The full terms of this license are available at https://www.dovepress.com/terms.php and incorporate the Creative Commons Attribution - Non Commercial (unported, v3.0) License.By accessing the work you hereby accept the Terms. Non-commercial uses of the work are permitted without any further permission from Dove Medical Press Limited, provided the work is properly attributed. For permission for commercial use of this work, please see paragraphs 4.2 and 5 of our Terms.

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Chitosan Coating of TiO2 Nanotube Arrays for Improved Metformin Releas | IJN - Dove Medical Press

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2020 World Sickle Cell Day: Symptoms of sickle cell can appear around 5 months of age

World Sickle Cell Day is observed on June 19. The day is meant to raise awareness about sickle cell disease, which is a group of disorders that affect haemoglobin, the molecule in red blood cells which deliver oxygen to cells throughout the body. People with this disorder have haemoglobin S, an atypical haemoglobin molecule which can distort red blood cells into a sickle or a crescent shape. This disease is a blood disorder which is inherited and passed down from parents to the child. In 2008, the UN General Assembly recognised sickle cell disease as a public health problem, and one of the world's foremost genetic diseases.

Symptoms of sickle cell disease usually appear around five months of age. The symptoms vary from person to person and change over time.

Symptoms of sickle cell disease can appear from two to five months of age. But in milder cases, the symptoms may appear till teenage.

Sickle cell disease can cause vision problemsPhoto Credit: iStock

Also read:These Iron-Packed Foods Can Combat Anemia And Other Health Problems

One can get infected with sickle cell disease if both parents have the problem gene and pass it on to their child.

1. HbSS or sickle cell anemia is a severe kind of sickle cell disease. It occurs when child inherits sickle cell gene from each parent.

2. HbSc occurs when one parents has sickle cell gene and the other has a gene from abnormal haemoglobin.

3. Hbs beta thalassemia occurs when sickle cell gene is passed from one parent, and beta-thalassemia is passed on from the other.

If only one parent has the problem gene, then the child will not have symptoms but will have the gene known as sickle cell trait.

Also read:Anemia During Pregnancy: Symptoms, Risk Factors And Prevention Steps Every Woman Must Know

Sickle cell can be detected in an infant during newborn screening process. In case of family history, it can be detected during pregnancy.

Bone marrow or stem cell transplant is the only permanent cure for sickle cell disease. Early detection and timely treatment can help in managing symptoms, offer relief from pain and prevent severe complications.

Apart from stem cell transplant, the symptoms can be managed with the help of periodic blood transfusion, pain killers, vaccinations and antibiotics.

Also read:6 Side-Effects Of Overuse Of Antibiotics And Home Remedies By Rujuta Diwekar That Can Be As Effective

Disclaimer: This content including advice provides generic information only. It is in no way a substitute for qualified medical opinion. Always consult a specialist or your own doctor for more information. NDTV does not claim responsibility for this information.

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World Sickle Cell Day 2020: Know The Types, Symptoms, Causes And Treatment - NDTV

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The global Rheumatoid Arthritis Stem Cell Therapy market study presents an all in all compilation of the historical, current and future outlook of the market as well as the factors responsible for such a growth. With SWOT analysis, the business study highlights the strengths, weaknesses, opportunities and threats of each Rheumatoid Arthritis Stem Cell Therapy market player in a comprehensive way. Further, the Rheumatoid Arthritis Stem Cell Therapy market report emphasizes the adoption pattern of the Rheumatoid Arthritis Stem Cell Therapy across various industries.Request Sample Reporthttps://www.factmr.com/connectus/sample?flag=S&rep_id=1001The Rheumatoid Arthritis Stem Cell Therapy market report highlights the following players:The global market for rheumatoid arthritis stem cell therapy is highly fragmented. Examples of some of the key players operating in the global rheumatoid arthritis stem cell therapy market include Mesoblast Ltd., Roslin Cells, Regeneus Ltd, ReNeuron Group plc, International Stem Cell Corporation, TiGenix and others.

The Rheumatoid Arthritis Stem Cell Therapy market report examines the operating pattern of each player new product launches, partnerships, and acquisitions has been examined in detail.Important regions covered in the Rheumatoid Arthritis Stem Cell Therapy market report include:

North America (U.S., Canada)Latin America (Mexico, Brazil)Western Europe (Germany, Italy, U.K., Spain, France, Nordic countries, BENELUX)Eastern Europe (Russia, Poland, Rest Of Eastern Europe)Asia Pacific Excluding Japan (China, India, Australia & New Zealand)JapanMiddle East and Africa (GCC, S. Africa, Rest Of MEA)

The Rheumatoid Arthritis Stem Cell Therapy market report takes into consideration the following segments by treatment type:

Allogeneic Mesenchymal stem cellsBone marrow TransplantAdipose Tissue Stem Cells

The Rheumatoid Arthritis Stem Cell Therapy market report contain the following distribution channel:

HospitalsAmbulatory Surgical CentersSpecialty ClinicsHave Any Query? Ask our Industry Experts-https://www.factmr.com/connectus/sample?flag=AE&rep_id=1001

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The Rheumatoid Arthritis Stem Cell Therapy market report offers a plethora of insights which include:

Changing consumption pattern among individuals globally.Historical and future progress of the global Rheumatoid Arthritis Stem Cell Therapy market.Region-wise and country-wise segmentation of the Rheumatoid Arthritis Stem Cell Therapy market to understand the revenue, and growth lookout in these areas.Accurate Year-on-Year growth of the global Rheumatoid Arthritis Stem Cell Therapy market.Important trends, including proprietary technologies, ecological conservation, and globalization affecting the global Rheumatoid Arthritis Stem Cell Therapy market.

The Rheumatoid Arthritis Stem Cell Therapy market report answers important questions which include:

Which regulatory authorities have granted approval to the application of Rheumatoid Arthritis Stem Cell Therapy in Health industry?How will the global Rheumatoid Arthritis Stem Cell Therapy market grow over the forecast period?Which end use industry is set to become the leading consumer of Rheumatoid Arthritis Stem Cell Therapy by 2028?What manufacturing techniques are involved in the production of the Rheumatoid Arthritis Stem Cell Therapy?Which regions are the Rheumatoid Arthritis Stem Cell Therapy market players targeting to channelize their production portfolio?Get Full Access of the Report @https://www.factmr.com/report/1001/rheumatoid-arthritis-stem-cell-therapy-market

Pertinent aspects this study on the Rheumatoid Arthritis Stem Cell Therapy market tries to answer exhaustively are:

What is the forecast size (revenue/volumes) of the most lucrative regional market? What is the share of the dominant product/technology segment in the Rheumatoid Arthritis Stem Cell Therapy market? What regions are likely to witness sizable investments in research and development funding? What are Covid 19 implication on Rheumatoid Arthritis Stem Cell Therapy market and learn how businesses can respond, manage and mitigate the risks? Which countries will be the next destination for industry leaders in order to tap new revenue streams? Which new regulations might cause disruption in industry sentiments in near future? Which is the share of the dominant end user? Which region is expected to rise at the most dominant growth rate? Which technologies will have massive impact of new avenues in the Rheumatoid Arthritis Stem Cell Therapy market? Which key end-use industry trends are expected to shape the growth prospects of the Rheumatoid Arthritis Stem Cell Therapy market? What factors will promote new entrants in the Rheumatoid Arthritis Stem Cell Therapy market? What is the degree of fragmentation in the Rheumatoid Arthritis Stem Cell Therapy market, and will it increase in coming years?Why Choose Fact.MR?

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Rheumatoid Arthritis Stem Cell Therapy Market Future Innovation Strategies, Growth & Profit Analysis, Forecast by 2028 - The Cloud Tribune

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Bone marrow transplants can save patients lives by essentially giving them a new immune system to fight off cancer. But they can also cause life-threatening side effects, so their use is relegated to the sickest of patients. Orca Bio wants to change that by taking aim at how these treatments are made.

The Bay Area biotech is coming out of stealth with a $192 million series D round that will propel a pipeline of high precision allogeneic cell therapies and the manufacturing technology behind those treatments. Founded in 2016, Orca Bio zeroed in on manufacturing to make bone marrow transplants safer and more effective.

Theres a bit of a trade-off: You can have precision and a few cells, or you can have lots of cells and sacrifice precision, Orca CEO and co-founder Ivan Dimov, Ph.D., told Fierce Biotech. Most folks out there deal with less precision in order to get the sheer number of cells to treat patients We focused on technology to process extremely large numbers of cells while still having single-cell precision.

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Orcas proposition is to take donor T cells and stem cells, sort them into their different subtypes and combine them in the right mixture to treat disease.

We dont genetically modify them. But if we now take these cells and build a proprietary mix of them with single-cell precision, we can define the function of what theyre going to do, Dimov said. We can elicit powerful curative effects and control toxicities in a precise way to enhance safety and efficacy in patients that essentially need a whole new blood and immune system.

Dimov likens the processto assembling different kinds of soldiers into the right army unit to give patients so they have a new immune system to seek and destroy cancers while not seeking and destroying the patient themselves and their own tissue.

Because the manufacturing process is quick and uses donor cells, Orcas treatments could eventually reach more patients than CAR-T therapies and other engineered cell therapies can. Some cancer patients may not have enough T cells, or T cells of good enough quality, to turn into a treatment, while others simply do not live long enough for the treatment to be made.

RELATED: BIO: Meet Refuge Biotech, the company developing 'intelligent' cell therapies

The series D, drawn from Lightspeed Ventures, 8VC, DCVC Bio, ND Capital, Mubadala investment Company, Kaiser Foundation Hospitals, Kaiser Permanente Group Trust and IMRF, brings Orcas total raised to nearly $300 million. That haulwill bring its lead program, TRGFT-201, through clinical development. The program is in a phase 1/2 study in patients with blood cancers, while a second program, OGFT-001, is in a phase 1 study, also in blood cancers.

Orcas first two programs are designed for patients with terminal blood cancers, but they could move earlier in the cancer care timeline if they prove to be safer than traditional bone marrow transplants. Beyond cancer, the approach could be applied to a range of genetic disorders of the blood and immune system. The companyhasnt decided where to go next, but Dimov said the approach could be useful in treating autoimmune diseases like Crohns disease or Type 1 diabetes.

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Orca Bio breaches the surface with $192M for 'high precision' cell therapies - FierceBiotech

Recommendation and review posted by Bethany Smith

PNH and aHUS, both are extremely rare and genetic diseases. Due to PNH, destruction of red blood cells of a person occurs quite sooner than it should. It is an acquired hematopoietic stem cell disorder. Hematopoietic stem cells are developed in bone marrow and eventually turn into red blood cells, white blood cells and platelets.

The Global PNH and aHUS Market study focuses major leading industry players with information such as company profiles, product picture and specification, capacity, production, price, cost, revenue and contact information. It provides information on trends and developments, and focuses on markets and materials, capacities and technologies, and on the changing structure.

Our analysis involves the study of the market taking into consideration the impact of the COVID-19 pandemic. Please get in touch with us to get your hands on an exhaustive coverage of the impact of the current situation on the market. Our expert team of analysts will provide as per report customized to your requirement.

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Top Key Players are covered in this report: Alexion Pharmaceuticals, Alnylam Pharmaceuticals, Omeros Corporation

The report offers an exhaustive geographical analysis of the Global PNH and aHUS Market, covering important regions Such as: North America, Europe, China, Japan, Southeast Asia, India and Central & South America. It also covers key countries (regions), viz, U.S., Canada, Germany, France, U.K., Italy, Russia, China, Japan, South Korea, India, Australia, Taiwan, Indonesia, Thailand, Malaysia, Philippines, Vietnam, Mexico, Brazil, Turkey, Saudi Arabia, U.A.E, etc.

The Global PNH and aHUS Market is gaining pace and businesses have started understanding the benefits of analytics in the present day highly dynamic business environment. The market has witnessed several important developments over the past few years, with mounting volumes of business data and the shift from traditional data analysis platforms to self-service business analytics being some of the most prominent ones.

Microeconomic and macroeconomic factors which affect the PNH and aHUS Market and its growth, both positive and negative are also studied. The report features the impact of these factors on the ongoing market throughout the mentioned forecast period. The upcoming changing trends, factors driving as well as restricting the growth of the market are mentioned.

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PNH and aHUS Market with Key Findings, Regional Analysis, Key Players Profiles, Current Trends, Business Development and Upcoming Prospects 2020-2026...

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CAMBRIDGE, Mass.--(BUSINESS WIRE)--Magenta Therapeutics (Nasdaq: MGTA) and Beam Therapeutics (Nasdaq: BEAM) today announced a non-exclusive research and clinical collaboration agreement to evaluate the potential utility of MGTA-117, Magentas novel targeted ADC for conditioning of patients with sickle cell disease and beta-thalassemia receiving Beams base editing therapies. Beam is pursuing two differentiated base editing approaches to treat hemoglobinopathies: its hereditary persistence of fetal hemoglobin (HPFH) program to precisely and robustly elevate fetal hemoglobin, which could be used in treatments for both sickle cell disease and beta-thalassemia, as well as a novel approach to directly correct the sickle causing point mutation (Makassar).

Conditioning is a critical component necessary to prepare a patients body to receive the edited cells, which carry the corrected gene and must engraft in the patients bone marrow in order to be effective. Todays conditioning regimens rely on nonspecific chemotherapy or radiation, which are associated with significant toxicities. MGTA-117 precisely targets only hematopoietic stem and progenitor cells, sparing immune cells, and has shown high selectivity, potent efficacy, wide safety margins and broad tolerability in non-human primate models. MGTA-117 may be capable of clearing space in bone marrow to support long-term engraftment and rapid recovery in patients.

Beam has demonstrated the ability to edit individual DNA bases in hematopoietic stem cells at high efficiency and with little impact on the viability of edited cells relative to unedited cells using its novel base editing technology. Combining MGTA-117 with Beams HPFH and Makassar base editors could meaningfully advance the treatment of patients with sickle cell disease or beta-thalassemia.

We believe patients will benefit from a more precise process to remove hematopoietic stem cells and prepare them to receive genetic medicines. Magenta has developed targeted ADCs as the preferred modality for our conditioning programs, and we have designed MGTA-117 specifically to optimize it for use with a genetically-modified cell product delivered in a transplant setting, said Jason Gardner, D.Phil., president and chief executive officer, Magenta Therapeutics. Beams next-generation base editing technology complements our next-generation conditioning approach very well, and we are excited to combine these strengths to address the still-significant unmet medical needs of the sickle cell and beta-thalassemia patient communities.

Base editing has the potential to offer lifelong treatment for patients with many diseases, including sickle cell disease and beta-thalassemia. Our novel base editors create precise single base changes in genes without cutting the DNA, enabling durable correction of hematopoietic stem cells with minimal effects on cell viability or genomic integrity, said John Evans, chief executive officer of Beam. Combining the precision of our base editing technology with the more targeted conditioning regimen enabled by MGTA-117 could further improve therapeutic outcomes for patients suffering from these severe diseases. We look forward to partnering with the Magenta team to explore these novel technologies together.

Beam will be responsible for clinical trial costs related to development of Beams base editors when combined with MGTA-117, while Magenta will continue to be responsible for all other development costs of MGTA-117. Magenta will also continue to develop MGTA-117 in other diseases, including blood cancers and genetic diseases. Each company will retain all commercial rights to their respective technologies.

About MGTA-117

MGTA-117, Magentas most advanced conditioning program, is a CD117-targeted antibody engineered for the transplant setting and conjugated to amanitin, a toxin in-licensed from Heidelberg Pharma. It is designed to precisely deplete only hematopoietic stem and progenitor cells and has shown high selectivity, potent efficacy, wide safety margins and broad tolerability in non-human primate models, suggesting that it may be capable of clearing space in bone marrow to support long-term engraftment and rapid recovery in patients. Magenta plans to complete IND-enabling studies this year and initiate clinical studies in 2021. Magenta will continue to develop MGTA-117 in other diseases, including blood cancers and genetic diseases.

About Magenta Therapeutics

Magenta Therapeutics is a clinical-stage biotechnology company developing medicines to bring the curative power of immune system reset through stem cell transplant to more patients with autoimmune diseases, genetic diseases and blood cancers. Magenta is combining leadership in stem cell biology and biotherapeutics development with clinical and regulatory expertise, a unique business model and broad networks in the stem cell transplant world to revolutionize immune reset for more patients. Magenta is based in Cambridge, Mass. For more information, please visit http://www.magentatx.com. Follow Magenta on Twitter: @magentatx.

About Base Editing and Beam TherapeuticsBeam Therapeutics (Nasdaq: BEAM) is a biotechnology company developing precision genetic medicines through the use of base editing. Beams proprietary base editors create precise, predictable and efficient single base changes, at targeted genomic sequences, without making double-stranded breaks in the DNA. This enables a wide range of potential therapeutic editing strategies that Beam is using to advance a diversified portfolio of base editing programs. Beam is a values-driven organization focused on its people, cutting-edge science, and a vision of providing life-long cures to patients suffering from serious diseases. For more information, visit http://www.Beamtx.com.

Magenta Therapeutics Forward-Looking StatementsThis press release may contain forward-looking statements and information within the meaning of The Private Securities Litigation Reform Act of 1995 and other federal securities laws, including, without limitation, statements regarding the research and clinical collaboration agreement between Magenta and Beam, including the timing, progress and success of the collaboration contemplated under the agreement, the successful evaluation of MGTA-117 in conjunction with Beams base-editing therapies under the agreement, the anticipated cost allocation and other commercial terms under the agreement, Magentas strategy and business plan, the future development, manufacture and commercialization between Beam and Magenta as well as statements regarding expectations and plans for the anticipated timing of Magentas clinical trials and regulatory filings and the development of Magentas product candidates and advancement of Magentas preclinical programs. The use of words such as may, will, could, should, expects, intends, plans, anticipates, believes, estimates, predicts, projects, seeks, endeavor, potential, continue or the negative of such words or other similar expressions can be used to identify forward-looking statements. The express or implied forward-looking statements included in this press release are only predictions and are subject to a number of risks, uncertainties and assumptions, including, without limitation, risks set forth under the caption Risk Factors in Magentas most recent Annual Report on Form 10-K filed on March 3, 2020, as updated by Magentas most recent Quarterly Report on Form 10-Q and its other filings with the Securities and Exchange Commission, risks, uncertainties and assumptions regarding the impact of the COVID-19 pandemic to Magentas business, operations, strategy, goals and anticipated timelines, and risks, uncertainties and assumptions inherent in preclinical and clinical studies, including, without limitation, whether results from preclinical studies or earlier clinical studies will be predictive of the results of future trials and the expected timing of submissions for regulatory approval or review by governmental authorities. In light of these risks, uncertainties and assumptions, the forward-looking events and circumstances discussed in this press release may not occur and actual results could differ materially and adversely from those anticipated or implied in the forward-looking statements. You should not rely upon forward-looking statements as predictions of future events. Although Magenta believes that the expectations reflected in the forward-looking statements are reasonable, it cannot guarantee that the future results, levels of activity, performance or events and circumstances reflected in the forward-looking statements will be achieved or occur. Moreover, except as required by law, neither Magenta nor any other person assumes responsibility for the accuracy and completeness of the forward-looking statements included in this press release. Any forward-looking statement included in this press release speaks only as of the date on which it was made. We undertake no obligation to publicly update or revise any forward-looking statement, whether as a result of new information, future events or otherwise, except as required by law.

Beam Forward-Looking Statements

This press release contains forward-looking statements. Investors are cautioned not to place undue reliance on these forward-looking statements, including statements about the timing, progress and success of the collaboration contemplated under the agreement between Beam and Magenta, the successful evaluation of MGTA-117 in conjunction with Beams base-editing therapies under the agreement, the expected timing of filing INDs applications and the therapeutic applications of Beams technology. Each forward-looking statement is subject to risks and uncertainties that could cause actual results to differ materially from those expressed or implied in such statement. Applicable risks and uncertainties include the risks and uncertainties, among other things, regarding: the success in development and potential commercialization of our product candidates; Beams ability to obtain, maintain and enforce patent and other intellectual property protection for our product candidates; whether preclinical testing of our product candidates and preliminary or interim data from preclinical and clinical trials will be predictive of the results or success of ongoing or later clinical trials; that enrollment of clinical trials may take longer than expected; that Beams product candidates will experience manufacturing or supply interruptions or failures; that Beam will be unable to successfully initiate or complete the preclinical and clinical development and eventual commercialization of product candidates; that the development and commercialization of Beams product candidates will take longer or cost more than planned; the impact of COVID-19 on Beams business and the other risks and uncertainties identified under the heading Risk Factors and in Beams Annual Reports on Form 10-K for the year ended December 31, 2019 and in Beams Quarterly Report on Form 10-Q for the quarter ended March 31, 2020, and in any subsequent filings with the Securities and Exchange Commission. These forward-looking statements (except as otherwise noted) speak only as of the date of this press release. Factors or events that could cause Beams actual results to differ may emerge from time to time, and it is not possible for Beam to predict all of them. Beam undertakes no obligation to update any forward-looking statement, whether as a result of new information, future developments or otherwise, except as may be required by applicable law.

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Magenta Therapeutics and Beam Therapeutics Announce Collaboration to Evaluate Targeted Antibody-Drug Conjugate (ADC) MGTA-117 as Conditioning Regimen...

Recommendation and review posted by Bethany Smith

PUBLISHED: 18:00 18 June 2020

Simon Parkin

Imogen Roe returned home to Norfolk after 100 days in isolation in hospital. Picture: Anna Dagless

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A nine-year-old girls life was saved by an anonymous blood stem cell donor after a brave battle with leukaemia in which she asked her mum am I going to die?

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Imogen Roe, from Norwich, had just turned six when she was treated for routine tonsillitis. But her worried parents, Anna and Dean, took her back to the doctors when they noticed abnormal bruising and a rash on her body.

They were told to take Imogen to their local hospital, and were soon confronted with the devastating diagnosis.

Mum Anna, 38, said: We suspected it was a reaction to penicillin at worst. But within an hour of being at our local hospital we were told it was leukaemia, and within 24 hours we were in Addenbrookes Hospital. It all happened so fast, I dont remember feeling much other than panic and shock.

Because of the aggressiveness of her leukaemia, young Imogen began two-and-a-half years of high dosage chemotherapy.

Anna said: Imogen was such a trouper, and very co-operative which made for a very easy patient!

I cannot believe how well she just got on with things; cannulas, nose tubes, general anaesthetics, surgery - even though at times she has been very scared about procedures.

During this time the family was divided, with Imogen and her mum in Addenbrookes, whilst Dad Dean, 39, stayed at home with their other two children, Imogens twin sister Charlotte, and older brother Liam, 11.

MORE: Esm, three, wins 19-month cancer battle but cannot celebrate traditional bell ringingIn January 2019, almost three years after her diagnosis, young Imogen finished her chemo and rang her end of treatment bell to huge applause from doctors, nurses, hospital staff - and her proud, but exhausted parents.

The young family were finally able to get their lives back on track, but unfortunately more heartbreaking news was just around the corner.

Anna said: It was July 2019, almost three years to the day since Imogens original diagnosis, and we had just bought a puppy and booked a family holiday abroad with friends. Then we noticed that familiar rash on her legs, and were told to bring her to hospital.

After undergoing tests, doctors confirmed the worst Imogen had relapsed and the leukaemia had come back.

Doctors revealed that, in addition to further chemotherapy, Imogens best chance of beating the disease was to have a blood stem cell transplant from a matching donor.

Imogens siblings were both tested, and the family were delighted to be told that her sister Charlotte was a 100% match.

However, after further tests, they were confronted with shocking news after a decade of thinking Charlotte and Imogen were non-identical twins, they were in fact identical, which meant that Charlotte would not be a suitable donor after all.

Doctors began looking for Imogens potential lifesaver elsewhere searching the worldwide register of potential blood stem cell donors, hoping to find a perfect stranger who happens to be Imogens genetic twin.

With Imogens life hanging in the balance a match was found. Cord blood, donated by a new mother in the USA and frozen nine years prior, was a perfect match for Imogen. This was the only suitable match for Imogen anywhere in the world.

MORE: How support, prayers and herbal rememdies helped nurse beat coronavirusAs the frozen cord blood was prepared to be flown from America to the UK, Imogen had 10 days of conditioning treatment prior to transplant; four days of extremely strong chemotherapy, and then eight sessions of total body irradiation, to prepare her to receive the new blood stem cells.

This is an incredibly vulnerable point in any treatment plan. The new marrow should, over a few weeks, start to regenerate within the body, but for Imogen, after 36 days of daily blood tests, there was still no sign of any new cells being manufactured.

Her mum said: It was a very serious situation as without any white cells to fight off infection, Imogen was extremely vulnerable even from her isolation room, as you can pick up bugs from your own body.

Ten days post transplant she got an infection, and she went into septic shock. This led to several serious viruses, a chest infection, and bacterial infection. We had the rapid response team on standby for a transfer to intensive care, but Imogen pulled through, just as her dad arrived after making the five hour journey from our home.

Imogen remained in isolation in Bristol for 99 days, fighting off multiple infections, whilst dad Dean travelled the five hours back and forth between home and the hospital to bring Anna clean clothes and supplies.

Finally, Imogen was transferred back to Addenbrookes to continue her recovery one step closer to home.

MORE: Wife of rugby star launches new business two years after being given a month to liveOn March 13, in the midst of the Covid-19 pandemic, she was finally discharged and returned home to her mum, dad and siblings, who are now all self isolating.

It had been 170 days since she, or mum Anna, had seen any extended family.

Speaking about Imogens anonymous donor, Anna said the family were acutely aware of the luck involved in finding a match.

She said: Imogen had just one match. There are some people we know through our time in hospitals who were fortunate enough to have a selection of matches, and many others are still waiting for a match that may never come.

If you are aged between 17 and 55 and in general good health, you take the first step to register as a blood stem cell donor by registering for a home swab kit at dkms.org.uk

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Recommendation and review posted by Bethany Smith

Ahead of a planned Phase III study, Pfizer and Sangamo Therapeutics released follow-up data from its Phase I/II Alta study in hemophilia A that showed an investigational gene therapy treatment provided blood-clotting activity levels for more than one year.

This morning, the two companies updated results from a December 2019 announcement that showed a high dose treatment of the gene therapy giroctocogene fitelparvovec generated sustained factor VIII activity levels in five patients. Hemophilia A is a genetic bleeding disorder where missing or faulty blood clotting factor VIII prevents blood from clotting normally. The clotting failure can cause frequent and spontaneous bleeding that can lead to long-term joint damage and disability. For hemophilia A patients who do not have factor VIII inhibitors, typical treatment required multiple infusions of factor VIII replacements each week.

In the Alta study, the updated data showed the patients who received the 3e13 vg/kg dose produced the all-important protein on their own. No patients in the study experienced bleeding events or required FVIII infusions, Pfizer and Sangamo announced this morning. The factor VIII activity levels reflect measurements up to 61 weeks, which is based on follow-up data from the longest-treated patient in the cohort. The updated data was presented as a late-breaking oral abstract at the World Federation of Hemophilia 2020 World Congress.

Seng Cheng, chief scientific officer of Pfizers Rare Disease Research Unit, touted the results and said the research team is encouraged by the potential of the gene therapy to provide a long-term solution to hemophilia A patients. Giroctocogene fitelparvovec uses a recombinant adeno-associated virus serotype 6 vector (AAV6) encoding the complementary deoxyribonucleic acid for B domain deleted human FVIII. Pfizer and Sangamo plan to begin dosing patients with the gene therapy in the Phase III trial later this year, Cheng added.

Adding to the potential of giroctocogene fitelparvovec in this indication, Bettina M. Cockroft, chief medical officer of Sangamo, said the treatment offers a new approach to treating the disease and has the potential to provide a one-time treatment that would enable patients to produce the missing factor on their own.

These follow-up data indicate that treatment with giroctocogene fitelparvovec resulted in sustained factor levels up to 14 months following treatment and suggests the potential of this investigational gene therapy to alleviate the treatment burden of current hemophilia disease management, Cockroft said in a statement.

The therapy was generally well-tolerated, although one patient who received the high dose had a treatment-related serious adverse event that was resolved within a day. Four of the five received corticosteroids for elevated liver enzymes and three patients had subsequent ALT elevations that responded to corticosteroids.

Data for the Phase I/II study was initially presented at the American Society of Hematology annual meeting in December. Data presented at that time showed that giroctocogene fitelparvovec was generally well tolerated and resulted in sustained FVIII levels up to 44 weeks, the extent of follow-up for the longest-treated patient in the 3e13 vg/kg dose cohort at that time. Pfizer and Sangamo plan to present further follow-up data from the Alta study when all five patients in the 3e13 vg/kg dose cohort have been followed for at least one year.

Pfizer released its data a day after BioMarin showcased Phase I/II data from its gene therapy study in hemophilia A. The U.S. Food and Drug Administration is reviewing the biologics license application for BioMarins gene therapy under Priority Review, with potential action in August.

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Pfizer and Sangamo Release Updated Gene Therapy Data in Hemophilia A - BioSpace

Recommendation and review posted by Bethany Smith

Gene Therapy is a rapidly expanding market within the global biotechnology industry. Using techniques such as CRIPSR, a gene editing technology, the aim is to treat and prevent diseases. By inserting or altering harmful genes in a patient cell, it can reduce the need for surgeries or drugs.

The UK is at the forefront of advancing Gene Therapy technology accounting for 12% of global gene and cell trials [1]. Innovation hubs such as those in White City, London and Stevenage have spearheaded this development.

To carry out this experimental work, companies require specialist systems and infrastructure. Biopharma recently installed 10 Premium 212 Microbiological Safety cabinets to a Gene Therapy company based in white City, London. This is now the second company within the emerging hub of innovative life sciences start-ups to install Faster safety cabinets.

A Microbiological safety cabinet provides product, personnel and environmental protection while handling biological samples such as in tissue culture, biological testing and pathogenic samples.

The ambient air is drawn in from the slots at the stainless-steel base of the front opening and it then passes under the work surface, from where it is drawn up and blown into the plenum of the re-circulating and exhaust fan(s).

Figure 1. Diagram showing how the negative pressure ensures that all contaminated particles are kept inside the system providing user and product protection.

The bio-dynamic sealing system of the negative pressure plenum ensures that all contaminated particles are kept inside the system and are automatically drawn to the plenum or pressure chamber to be captured by the main re-circulating and exhaust H 14 HEPA/ULPA filters, thus protecting the user, product and environment.

70% of the filtered air is re-circulated (after passing through a H14 HEPA/ULPA filter), an air cleanliness in Class ISO 3, according to ISO 14644-1 laminar flow pattern downwards into the work chamber and the remaining 30% is exhausted to atmosphere through another H14 HEPA/ULPA filter.

Features that should be considered when purchasing a Class II cabinet are safety, cost (both upfront and energy cost) as well as noise and location. The Premium 212 is one of the most energy efficient cabinets in the market with an annual running cost of 0.084kWh meaning a running cost of 17.40 per year. It is also one of the quietest at 42dB meaning a lab with several cabinets side by side can be a relaxing and suitable work area for any kind of experiment. Other features include dimmable LED lighting, magnetic and removable UV sterilising lamp, electronic angled sliding sash and work surface in stainless steel AISI 316L.

At Biopharma we will always provide advise on your requirements. We regularly visit sites to help decisions such as locations and carry out site surveys. Oh and did someone say we also keep stock of most Class II cabinets.References

1. https://pharmaphorum.com/news/uk-accounts-for-12-of-cell-and-gene-therapy-trials-report/

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Choosing the right safety cabinet to advance your gene therapy research - Labmate Online

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Global Gene Therapy and Antisense Drugs Market: Snapshot

Since an escalating number of patients are being diagnosed with cancer every day, the global demand for gene therapy and antisense drugs is bound to multiply. The growing incidence of health conditions such as Parkinsons disease and high cholesterol is also boosting the demand for gene therapy and antisense drugs. Several new drugs and therapies have been making their debut in the global gene therapy and antisense drugs market of recent.

For instance, in June 2017, it was announced that a group of scientists at the University of Queensland have developed a new technique that is capable of permanently silencing severe allergies triggered by shellfish, peanuts, and venom. The treatment was found to be successful in animal trials. The technique holds considerable potential for treating asthma completely. During the same month, results of another research study for treating multiple myeloma, a type of blood cancer, were announced at a conference held by the American Society of Clinical Oncology. The new CAR-T therapy consists of filtering the blood of the patients in order to eliminate T cells which are then genetically altered and given back to the patients.

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However, the difficulty pertaining to the delivery of antisense technology to the brain can present key challenges to the expansion of the global market for gene therapy and antisense drugs. Moreover, the unavoidable toxic aftereffects associated with the technology can also inhibit the growth of the gene therapy and antisense drugs market worldwide. However, the present attempts at developing safe and efficient antisense drugs by several market participants including Ionis Pharmaceuticals, which undertook testing of the technology on transgenic mice, can promote the further expansion of the market.

Global Gene Therapy and Antisense Drugs Market: Overview

Antisense gene therapy is emerging as one of the most beneficial therapeutics for various diseases such as tumors, cancer etc. These newer therapies are based on increased knowledge of molecular events that lead to disordered cellular growth. The therapy involves using a gene silencing technique rather than a gene repairing technique for silencing the genes effect.

The research report is a valuable tool for comprehending the progression of the global gene therapy and antisense drugs market between 2017 and 2025.

Global Gene Therapy and Antisense Drugs Market: Treatment Insights

Antisense drugs attach to the mRNA of a target protein, which inhibits the protein production process. Antisense therapeutics can obstruct the expression of oncogenes and other cancer-related genes that express growth factors. Antisense gene therapy involves the utilization of various therapeutic strategies which requires a clear knowledge of the molecular anatomy of cancer related genes. Antisense gene therapy is used to treat various diseases such as hemorrhagic fever, cancer, cystic fibrosis, renal diseases, HIV/AIDS, spinal muscular atrophy, and cardiovascular diseases.

Global Gene Therapy and Antisense Drugs Market: Market Segmentation

On the basis of therapeutic area, the gene therapy and antisense drugs market is segmented into cancer, anemia, rheumatoid arthritis, cardiovascular diseases, HIV/AIDS, cystic fibrosis, diabetes mellitus and obesity, and renal diseases.

By gene transfer method, ex vivo gene transfer and in vivo gene transfer are the segments of the market. The former involves the transfer of cloned genes into cells, i.e., cells are altered outside the body before being implanted into the patient, whereas the latter involves the transfer of cloned genes directly into the patients tissues. The outcome of in vivo gene transfer technology mainly depends on the general efficacy of gene transfer and expression.

Global Gene Therapy and Antisense Drugs Market: Regional Outlook

The global gene therapy and antisense drugs market is segmented into North America, Asia Pacific, Europe, and Rest of the World. Amongst these, North America holds the leading position in the market followed by Europe. The increasing incidence of cancer and other fatal diseases, unhealthy lifestyle practices such as excessive smoking and excessive consumption of high fat content food, and increasing research efforts for treatment against cancer are the major factors driving the gene therapy and antisense drugs market in these regions.

Asia Pacific is expected to emerge as a significant market for gene therapy and antisense drugs. The high population density including a large geriatric population, expeditiously increasing demand for technologically advanced therapeutics, and increasing government support for improved healthcare infrastructure in the region is driving the growth of this regional market. Furthermore, favorable reimbursement policies and tax benefits on newer therapies will further fuel the growth of the Asia Pacific gene therapy and antisense drugs market.

Major Companies Mentioned in Report

Some of the leading companies operating in the global gene therapy and antisense drugs market are GenVec Inc., Avigen Inc., Genome Therapeutics Corp., Tekmira Pharmaceuticals Corporation, Isis Pharmaceuticals, Cell Genesys Inc., and others. These companies are profiled for their key business attributes in the report.

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Gene Therapy And Antisense Drugs Market Newer segments of application 2025 - Cole of Duty

Recommendation and review posted by Bethany Smith

Hemophilia Gene Therapy Industry Overview Competitive Analysis, Regional and Global Analysis, Segment Analysis, Market Forecasts 2026

The globalHemophilia Gene Therapy marketshave undergone huge change in the last few months. These changes were due to the outbreak of the pandemic which was first detected in the Wuhan city of China. COVID-19 which has occurred due to the coronavirus has taken many lives of people around the world. As the disease is spreading at a rapid rate many of the countries have ordered lockdown for maintaining social distancing. Due to the lockdown, many of the industries have halted their manufacturing units. There have been restrictions for cross border trading within the countries and also within the states. Owing to these conditions, trading conditions in various regions have been affected badly. The overall countries in the world are facing economic crisis thus affecting some of the major markets in the world.

Request Free Sample Copy of Hemophilia Gene Therapy Market Research Report@http://www.marketresearchstore.com/report/global-hemophilia-gene-therapy-market-report-2019-industry-607887#RequestSample

The research analysts from theMarket Research Storehave conducted a detailed study about the global Hemophilia Gene Therapy market. Owing to the above mentioned conditions, the global Hemophilia Gene Therapy market has undergone several changes on the global platform. All these updates are mentioned in the Hemophilia Gene Therapy market report study. The research analysts have conducted a thorough primary and secondary research for updating the market statistics as per the current market scenario. The detailed Hemophilia Gene Therapy market report is of over 150 pages including more than 30 tables and around 20 figures. The report includes pictorial representations of the market data in order to understand the Hemophilia Gene Therapy market in a simple and easy way.

The data that is included about the Hemophilia Gene Therapy market incorporates historical data from 2016 to 2019 and forecasts data from 2020 to 2026. The major players that are functioning in the Hemophilia Gene Therapy market areSpark Therapeutics, Ultragenyx, Shire PLC, Sangamo Therapeutics, Bioverativ, BioMarin, uniQure, Freeline Therapeutics. Details about all the market players, distributors, suppliers, and retailers are profiled in the Hemophilia Gene Therapy market report.

Read Detailed Index of full Research Study at::http://www.marketresearchstore.com/report/global-hemophilia-gene-therapy-market-report-2019-industry-607887

The Hemophilia Gene Therapy market is segmented into{Hemophilia A, Hemophilia B}; {Hemophilia A Gene Therapy, Hemophilia B Gene Therapy}. Each of the market segments is described in detail within the report. Data about the segments are represented in both qualitative and quantitative format, thus enabling to understand the market in detail.

Major Advantages for Hemophilia Gene Therapy Market:

Well-organized description of the international Hemophilia Gene Therapy market along with the ongoing inclinations and future considerations to reveal the upcoming investment areas. The all-inclusive market feasibility is examined to figure out the profit-making trends to obtain the most powerful foothold in the Hemophilia Gene Therapy industry. The Hemophilia Gene Therapy market report covers data which reveal major drivers, constraints, and openings with extensive impact analysis. The current market is quantitatively reviewed from 2019 to 2028 to pinpoint the monetary competency of the global Hemophilia Gene Therapy market. Last but not least, PORTERS Five Forces Analysis shows the effectiveness of the customers and providers from a global perspective.

If Any Inquiry of Hemophilia Gene Therapy Report:http://www.marketresearchstore.com/report/global-hemophilia-gene-therapy-market-report-2019-industry-607887#InquiryForBuying

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Global Hemophilia Gene Therapy Market 2020 with COVID-19 After Effects Analysis by Key Players | Spark Therapeutics, Ultragenyx, Shire PLC, Sangamo...

Recommendation and review posted by Bethany Smith

A new research study has been presented by Industrygrowthinsights.com offering a comprehensive analysis on the Global Gene Therapy Market where user can benefit from the complete market research report with all the required useful information about this market. This is a latest report, covering the current COVID-19 impact on the market. The pandemic of Coronavirus (COVID-19) has affected every aspect of life globally. This has brought along several changes in market conditions. The rapidly changing market scenario and initial and future assessment of the impact is covered in the report. The report discusses all major market aspects with expert opinion on current market status along with historic data. This market report is a detailed study on the growth, investment opportunities, market statistics, growing competition analysis, major key players, industry facts, important figures, sales, prices, revenues, gross margins, market shares, business strategies, top regions, demand, and developments.

The Gene Therapy Market report provides a detailed analysis of the global market size, regional and country-level market size, segment growth, market share, competitive landscape, sales analysis, impact of domestic and global market players, value chain optimization, trade regulations, recent developments, opportunity analysis, strategic market growth analysis, product launches, and technological innovations.

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Major Players Covered in this Report are: SangamoSpark TherapeuticsDimension TherapeuticsAvalanche BioCelladonVicalAdvantagene

Global Gene Therapy Market SegmentationThis market has been divided into Types, Applications, and Regions. The growth of each segment provides an accurate calculation and forecast of sales by Types and Applications, in terms of volume and value for the period between 2020 and 2026. This analysis can help you expand your business by targeting qualified niche markets. Market share data is available on the global and regional level. Regions covered in the report are North America, Europe, Asia Pacific, the Middle East & Africa, and Latin America. Research analysts understand the competitive strengths and provide competitive analysis for each competitor separately.

By Types:Ex vivoIn vivo

By Applications:Cancer DiseasesMonogenic DiseasesInfectious DiseasesCardiovascular DiseasesOthers

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Global Gene Therapy Market Regions and Countries Level AnalysisRegional analysis is a highly comprehensive part of this report. This segmentation sheds light on the sales of the Gene Therapy on regional- and country-level. This data provides a detailed and accurate country-wise volume analysis and region-wise market size analysis of the global market.

The report offers an in-depth assessment of the growth and other aspects of the market in key countries including the US, Canada, Mexico, Germany, France, the UK, Russia, Italy, China, Japan, South Korea, India, Australia, Brazil, and Saudi Arabia. The competitive landscape chapter of the global market report provides key information about market players such as company overview, total revenue (financials), market potential, global presence, Gene Therapy sales and revenue generated, market share, prices, production sites and facilities, products offered, and strategies adopted. This study provides Gene Therapy sales, revenue, and market share for each player covered in this report for a period between 2016 and 2020.

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Why Choose Us:

Table of Contents1. Executive Summary2. Assumptions and Acronyms Used3. Research Methodology4. Market Overview5. Global Market Analysis and Forecast, by Types6. Global Market Analysis and Forecast, by Applications7. Global Market Analysis and Forecast, by Regions8. North America Market Analysis and Forecast9. Latin America Market Analysis and Forecast10. Europe Market Analysis and Forecast11. Asia Pacific Market Analysis and Forecast12. Middle East & Africa Market Analysis and Forecast13. Competition Landscape

About Industrygrowthinsights:INDUSTRYGROWTHINSIGHTS has set its benchmark in the market research industry by providing syndicated and customized research report to the clients. The database of the company is updated on a daily basis to prompt the clients with the latest trends and in-depth analysis of the industry. Our pool of database contains various industry verticals that include: IT & Telecom, Food Beverage, Automotive, Healthcare, Chemicals and Energy, Consumer foods, Food and beverages, and many more. Each and every report goes through the proper research methodology, validated from the professionals and analysts to ensure the eminent quality reports.

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Gene Therapy Market Size 2020-2026 Review, Key Findings, Growth Strategy, Developing Technologies, Trends And Global Forecast By Regions - Bulletin...

Recommendation and review posted by Bethany Smith

New Jersey, United States,- The report is a must-have for business strategists, participants, consultants, researchers, investors, entrepreneurs, and other interested parties associated with the Gene Therapy Market. It is also a highly useful resource for those looking to foray into the Gene Therapy market. Besides Porters Five Forces and SWOT analysis, it offers detailed value chain assessment, comprehensive study on market dynamics including drivers, restraints, and opportunities, recent trends, and industry performance analysis. Furthermore, it digs deep into critical aspects of key subjects such as market competition, regional growth, and market segmentation so that readers could gain sound understanding of the Gene Therapy market.

The research study is a brilliant account of macroeconomic and microeconomic factors influencing the growth of the Gene Therapy market. This will help market players to make appropriate changes in their approach toward attaining growth and sustaining their position in the industry. The Gene Therapy market is segmented as per type of product, application, and geography. Each segment is evaluated in great detail so that players can focus on high-growth areas of the Gene Therapy market and increase their sales growth. Even the competitive landscape is shed light upon for players to build powerful strategies and give a tough competition to other participants in the Gene Therapy market.

The competitive analysis included in the report helps readers to become aware of unique characteristics of the vendor landscape and crucial factors impacting the market competition. It is a very important tool that players need to have in their arsenal for cementing a position of strength in the Gene Therapy market. Using this report, players can use effective business tactics to attract customers and improve their growth in the Gene Therapy market. The study provides significant details about the competitive landscape and allows players to prepare for future challenges beforehand.

Gene Therapy Market Segmentation

This market has been divided into types, applications and regions. The growth of each segment provides a precise calculation and forecast of sales by type and application, in terms of volume and value for the period between 2020 and 2026. This analysis can help you develop your business by targeting qualified niche markets. . Market share data are available at global and regional levels. The regions covered by the report are North America, Europe, Asia-Pacific, the Middle East and Africa and Latin America. Research analysts understand competitive forces and provide competitive analysis for each competitor separately.

Gene Therapy Market by Type:

YYYY

Gene Therapy Market by Application:

ZZZZ

Gene Therapy Market by Region:

North America (The USA, Canada, and Mexico)Europe (Germany, France, the UK, and Rest of Europe)Asia Pacific (China, Japan, India, and Rest of Asia Pacific)Latin America (Brazil and Rest of Latin America.)Middle East &Africa (Saudi Arabia, the UAE, South Africa, and Rest of Middle East & Africa)

The report answers important questions that companies may have when operating in the Gene Therapy market. Some of the questions are given below:

What will be the size of the Gene Therapy market in 2026?

What is the current CAGR of the Gene Therapy market?

What products have the highest growth rates?

Which application is projected to gain a lions share of the Gene Therapy market?

Which region is foretold to create the most number of opportunities in the Gene Therapy market?

Which are the top players currently operating in the Gene Therapy market?

How will the market situation change over the next few years?

What are the common business tactics adopted by players?

What is the growth outlook of the Gene Therapy market?

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Apart from hottest technological advances in the Gene Therapy market, it brings to light the future plans of dominant players in the industry

Table of Contents

Market Overview: This section comes under executive summary and is divided into four sub-sections. It basically introduces the Gene Therapy market while focusing on market size by revenue and production, market segments by type, application, and region, and product scope.

Competition by Manufacturers: It includes five sub-sections, viz. market competitive situation and trends, manufacturers products, areas served, and production sites, average price by manufacturers, revenue share by manufacturers, and production share by manufacturers.

Market Share by Region: It provides regional market shares by production and revenue besides giving details about gross margin, price, and other factors related to the growth of regional markets studied in the report. The review period considered here is 2015-2019.

Company Profiles: Each player is assessed for its market growth in terms of different factors such as markets served, gross margin, price, revenue, production, product specification, and areas served.

Manufacturing Cost Analysis: It is sub-divided into four chapters, viz. industrial chain analysis, manufacturing process analysis, manufacturing cost structure, and key raw materials analysis.

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Original post:
Gene Therapy Market 2020 Key Players, Share, Trend, Segmentation and Forecast to 2026 - Cole of Duty

Recommendation and review posted by Bethany Smith

Cell Therapy Manufacturing Market 2020 Global Industry Research report presents an in-depth analysis of the Cell Therapy Manufacturing market size, growth, share, segments, manufacturers, and technologies, key trends, market drivers, challenges, standardization, deployment models, opportunities, future roadmap and 2027 forecast.

Global Cell Therapy Manufacturing Market 2020 Industry Research Report is an expert and inside and out examination on the flow condition of the Global Cell Therapy Manufacturing industry. In addition, investigate report sorts the worldwide Cell Therapy Manufacturing market by top players/brands, area, type and the end client. This report likewise examines the different Factors impacting the market development and drivers, further reveals insight into market review, key makers, key received by them, size, most recent patterns and types, income, net edge with provincial examination and figure.

Read Summary Of Cell Therapy Manufacturing Market Report @Cell Therapy Manufacturing Market

List Of TOP KEY PLAYERS in Cell Therapy Manufacturing Market are: Pharmicell, Merck Group, Dickinson and Company, Thermo Fisher, Lonza Group, Miltenyi Biotec GmBH, Takara Bio Group, STEMCELL Technologies, Cellular Dynamics International, Becton, Osiris Therapeutics, Bio-Rad Laboratories, Inc., Anterogen, MEDIPOST, Holostem Terapie Avanazate, Pluristem Therapeutics, Brammer Bio, CELLforCURE, Gene Therapy Catapult EUFETS, MaSTherCell, PharmaCell, Cognate BioServices and WuXi AppTec.

Scope of Report:

The global Cell Therapy Manufacturing market is anticipated to rise at a considerable rate during the forecast period, between 2020 and 2027. In 2020, the market was growing at a steady rate and with the rising adoption of strategies by key players, the market is expected to rise over the projected horizon.

This report covers present status and future prospects for the Cell Therapy Manufacturing Market forecast till 2027. Market Overview, Development, and Segment by Type, Application and Region. Global Market by company, Type, Application and Geography. The report begins with an overview of the industry chain structure and describes the upstream. Besides, the report analyses Cell Therapy Manufacturing market trends, size and forecast in different geographies, type and end-use segment, in addition, the report introduces market competition overview among the major companies and companies profiles, besides, market price and channel features are covered in the report.

Cell Therapy Manufacturing Market Research Report provides exclusive vital statistics, data, information, trends and competitive landscape details in this niche sector.

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Brief Introduction to the research report.Table of Contents (Scope covered as a part of the study)Top players in the marketResearch framework (presentation)Research methodology adopted by Coherent Market Insights

Cell Therapy Manufacturing Market global industry research report is a professional and in-depth study on the market size, growth, share, trends, as well as industry analysis. According to the details of the consumption figures, the global Cell Therapy Manufacturing market forecast 2027.

Market by Region:

North America (U.S., Canada, Mexico)Europe (Germany, U.K., France, Italy, Russia, Spain etc.)Asia-Pacific (China, India, Japan, Southeast Asia etc.)South America (Brazil, Argentina etc.)The Middle East and Africa (Saudi Arabia, South Africa etc.)

Key Benefits For Stakeholders

Cell Therapy Manufacturing Market Taxonomy

On the basis of therapy type, the global cell therapy manufacturing market is segmented into:

On the basis of technology, the global cell therapy manufacturing market is segmented into:

On the basis of source, the global cell therapy manufacturing market is segmented into:

On the basis of application, the global cell therapy manufacturing market is segmented into:

On the basis of region, the global cell therapy manufacturing market is segmented into:

Main Aspects Covered In The Report:

Overview of the Cell Therapy Manufacturing market including production, consumption, status and forecast and market growth.Geographical analysis including major countries.Overview of the product type market including development.Overview of the end-user market including development.

Research Objectives:

To understand the structure of Cell Therapy Manufacturing market by identifying its various sub-segments.Focuses on the key global Cell Therapy Manufacturing manufacturers, to define, describe and analyze the sales volume, value, market share, market competition landscape, SWOT analysis and development plans in next few years. To analyze the Cell Therapy Manufacturing with respect to individual growth trends, future prospects, and their contribution to the total market. To share detailed information about the key factors influencing the growth of the market (growth potential, opportunities, drivers, industry-specific challenges and risks). To project the consumption of Cell Therapy Manufacturing submarkets, with respect to key regions (along with their respective key countries). To analyze competitive developments such as expansions, agreements, new product launches, and acquisitions in the market.To strategically profile the key players and comprehensively analyze their growth strategies.

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Cell Therapy Manufacturing Market: Factory Price and Marginal Revenue Analysis by 2026 - Surfacing Magazine

Recommendation and review posted by Bethany Smith

Application of ImmTOR plus Sareptas investigational gene therapies will be evaluated for Duchenne Muscular Dystrophy and Limb-Girdle Muscular Dystrophies

CAMBRIDGE, Mass. and WATERTOWN, Mass., June 18, 2020 (GLOBE NEWSWIRE) -- Sarepta Therapeutics (SRPT), the leader in precision genetic medicine for rare diseases, and Selecta Biosciences, Inc. (SELB) today announced that they have entered into a Research License and Option agreement granting Sarepta an option to license the rights to develop and commercialize Selectas immune tolerance platform, ImmTOR, for use in Duchenne muscular dystrophy (DMD) and certain limb-girdle muscular dystrophies (LGMDs). In advance of exercising its option, Sarepta will conduct research and evaluate the utility of ImmTOR to minimize or prevent the formation of neutralizing antibodies (NAb) to adeno-associated virus (AAV) in connection with the administration of Sareptas DMD and LGMD gene therapy candidates.

Sareptas late-stage gene therapy candidates are delivered using AAV in particular, AAVrh74. AAVrh74 was selected because of its safety profile, superior muscle tropism, empirical demonstration of high expression, and low screen-out rate for pre-existing antibodies. Currently, however, all systemic AAV-delivered constructs are one-time therapies that cannot be re-dosed due to the robust post-administration development of NAbs specific to the AAV vector. Selecta is a leader in immune tolerance and has generated strong preclinical evidence to support the potential for re-dosing patients receiving gene therapy.Selecta has reported that in preclinical studies, when used in combination with AAV gene therapy vectors,Selectas ImmTOR immune tolerance platform inhibits the development of NAbs to the vector, permitting re-dosing of the gene therapy. i

As we build our enduring gene therapy engine, we intend not only to rapidly advance treatments for rare, life-ending diseases, but at the same time, to advance the state of genetic medicine science by continually improving the utility of gene therapy. If successful, the ability to re-dose will be an enormous leap forward in the science of gene therapy and provide invaluable benefits to patients beyond those we anticipate with one-time dosing. We are encouraged by the data generated on the ImmTOR platform and excited to join with Selecta to explore the possibility of unlocking the opportunity to safely and effectively re-dose AAV-mediated gene therapies in patients with DMD and LGMDs, if needed, said Doug Ingram, President and Chief Executive Officer, Sarepta Therapeutics.

We are pleased to build on our already strong foundation of strategic partnerships and expand the clinical application of the ImmTOR platform into neuromuscular diseases. The ability to re-dose gene therapy addresses one of the major challenges of one-time therapies today, said Carsten Brunn Ph.D., President and Chief Executive Officer of Selecta Biosciences. We are excited to collaborate with a leader in genetic medicine like Sarepta, and are confident that their expertise in rare diseases combined with our immune tolerance platform has the potential to enhance the long-term therapeutic benefit to patients with these debilitating conditions.

DMD is a rare, degenerative neuromuscular disorder causing severe progressive muscle loss and premature death. LGMDs are a group of over 30 distinct diseases that cause weakness and wasting of the muscles around the hips and shoulders, eventually progressing to the arms and legs. LGMD can be caused by a single gene defect that affects specific proteins within the muscle cell, including those responsible for keeping the muscle membrane intact.

Under the terms of the research license and option agreement, Sarepta will make an initial payment to Selecta, and Selecta is eligible to receive certain pre-clinical milestone fees. If Sarepta exercises its options to enter any commercial license agreements, Selecta will be eligible for additional development, regulatory, and commercial milestone payments, as well as tiered royalties on net product sales. Additional financial details are not being disclosed.

About Selecta Biosciences, Inc.

Selecta Biosciences, Inc. is a clinical-stage biotechnology company focused on unlocking the full potential of biologic therapies based on its pioneering immune tolerance platform (ImmTOR). Selecta is committed to utilizing ImmTOR to potentially improve the efficacy of biologics, enable re-dosing of life-saving gene therapy, and create novel immunotherapies for autoimmune diseases. Selectas late-stage product candidate, SEL-212, is designed to be a monthly treatment for chronic refractory gout, a debilitating rare disease with a significant unmet medical need. SEL-212 consists of a combination of our ImmTOR platform co-administered with pegadricase, an enzyme designed to treat patients with symptomatic gout, refractory to standard uric acid lowering treatment. Selectas proprietary gene therapy product candidates are in development for certain rare inborn errors of metabolism and incorporate our ImmTOR platform with the goal of addressing barriers to repeat administration. In addition to our own pipeline of core discovery and clinical candidates, Selecta has established collaborative relationships with leading biopharmaceutical companies, including Asklepios BioPharmaceutical (AskBio) for gene therapy, and Swedish Orphan Biovitrum AB (Sobi) for SEL-212. Selecta is based in Watertown, Massachusetts. For more information, please visit http://www.selectabio.com.

Story continues

Selecta Forward-Looking Statements:

Any statements in this press release about the future expectations, plans and prospects of Selecta Biosciences, Inc. (the company), including without limitation, the companys actions regarding the monitoring and assessment of COVID-19 on the companys operations, clinical trials and manufacturing, Sareptas plans to evaluate its gene therapies in combination with the companys ImmTOR technology, the possibility of Sarepta exercising an option to enter into a commercial license agreement, the unique proprietary technology platform of the company and the unique proprietary platform of its partners, the potential of ImmTOR to enable re-dosing of AAV gene therapy, the ability of the companys ImmTOR platform to unlock the full potential of biologic therapies, the potential treatment applications for product candidates utilizing the ImmTOR platform in areas such as enzyme therapy and gene therapy, the novelty of treatment paradigms that Sarepta is able to develop in combination with the companys ImmTOR technology, the potential of any therapies developed by Sarepta in combination with the companys ImmTOR technology to fulfill unmet medical needs, the companys plan to apply its ImmTOR technology platform to a range of biologics for rare and serious diseases, the ability of Sareptas existing therapies to target the heart and skeletal muscle, expected payments to be made to the company under the Research License and Option Agreement, the potential of the ImmTOR technology platform generally and the companys ability to grow its strategic partnerships, the sufficiency of the companys cash, cash equivalents and short-term investments, and other statements containing the words anticipate, believe, continue, could, estimate, expect, hypothesize, intend, may, plan, potential, predict, project, should, target, would, and similar expressions, constitute forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including, but not limited to, the following: the uncertainties inherent in the initiation, completion and cost of clinical trials including their uncertain outcomes, the effect of the COVID-19 outbreak on any of the companys planned or ongoing clinical trials, manufacturing activities, supply chain and operations, the availability and timing of data from ongoing and future clinical trials and the results of such trials, whether preliminary results from a particular clinical trial will be predictive of the final results of that trial or whether results of early clinical trials will be indicative of the results of later clinical trials, the unproven approach of the companys ImmTOR technology, Sareptas ability to research and develop therapeutic candidates using the companys ImmTOR technology, undesirable side effects of the companys product candidates, its reliance on third parties to manufacture its product candidates and to conduct its clinical trials as well as the impact of the COVID-19 outbreak on those third parties and their ability to continue their operations, the companys inability to maintain its existing or future collaborations, licenses or contractual relationships, its inability to protect its proprietary technology and intellectual property, managements ability to perform as expected, potential delays in regulatory approvals, Sareptas ability to make up-front and milestone payments, the companys business development strategy, the availability of funding sufficient for its foreseeable and unforeseeable operating expenses and capital expenditure requirements, the companys recurring losses from operations and negative cash flows from operations raise substantial doubt regarding its ability to continue as a going concern, substantial fluctuation in the price of its common stock including stock market fluctuations that occur as a result of the COVID-19 outbreak, and other important factors discussed in the Risk Factors section of the companys most recent Quarterly Report on Form 10-Q, and in other filings that the company makes with the Securities and Exchange Commission. In addition, any forward-looking statements included in this press release represent the companys views only as of the date of its publication and should not be relied upon as representing its views as of any subsequent date. The company specifically disclaims any intention to update any forward-looking statements included in this press release.

AboutSarepta Therapeutics

At Sarepta, we are leading a revolution in precision genetic medicine and every day is an opportunity to change the lives of people living with rare disease. The Company has built an impressive position in Duchenne muscular dystrophy (DMD) and in gene therapies for limb-girdle muscular dystrophies (LGMDs), mucopolysaccharidosis type IIIA, Charcot-Marie-Tooth (CMT), and other CNS-related disorders, with more than 40 programs in various stages of development. The Companys programs and research focus span several therapeutic modalities, including RNA, gene therapy and gene editing. For more information, please visitwww.sarepta.comor follow us onTwitter,LinkedIn,InstagramandFacebook.

Sarepta Forward-Looking Statement

This press release contains "forward-looking statements." Any statements contained in this press release that are not statements of historical fact may be deemed to be forward-looking statements. Words such as "believes," "anticipates," "plans," "expects," "will," "intends," "potential," "possible" and similar expressions are intended to identify forward-looking statements. These forward-looking statements include statements regarding the parties undertakings under the agreement and potential payments and fees; the potential benefits of Sareptas gene therapy product candidates; the potential of ImmTOR to enable re-dosing of AAV gene therapy; Sareptas intention to rapidly advance treatments for rare, life-ending diseases, and to advance the state of the genetic medicine science by continually improving the utility of gene therapy; the potential of re-dosing to provide invaluable benefits to patients beyond those Sarepta anticipates with one-time dosing; the possibility of unlocking the opportunity to safely and effectively re-dose AAV-mediated gene therapies in patients with DMD and LGMDs, if needed; and the potential of the collaboration between Sarepta and Selecta to enhance the long-term therapeutic benefit to patients with these debilitating conditions.

These forward-looking statements involve risks and uncertainties, many of which are beyond our control. Known risk factors include, among others: the expected benefits and opportunities related to the collaboration between Sarepta and Selecta may not be realized or may take longer to realize than expected due to challenges and uncertainties inherent in product research and development. In particular, the collaboration may not result in any viable treatments suitable for commercialization due to a variety of reasons, including any inability of the parties to perform their commitments and obligations under the agreement; success in preclinical trials does not ensure that later clinical trials will be successful; Sarepta may not be able to execute on its business plans and goals, including meeting its expected or planned regulatory milestones and timelines, clinical development plans, and bringing its product candidates to market, due to a variety of reasons, many of which may be outside of Sareptas control, including possible limitations of company financial and other resources, manufacturing limitations that may not be anticipated or resolved for in a timely manner, regulatory, court or agency decisions, such as decisions by the United States Patent and Trademark Office with respect to patents that cover Sareptas product candidates and the COVID-19 pandemic; and even if Sareptas programs result in new commercialized products, Sarepta may not achieve the expected revenues from the sale of such products; and those risks identified under the heading Risk Factors in Sareptas most recent Annual Report on Form 10-K for the year ended December 31, 2019, and most recent Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission (SEC) as well as other SEC filings made by Sarepta which you are encouraged to review.

Any of the foregoing risks could materially and adversely affect Sareptas business, results of operations and the trading price of Sareptas common stock. For a detailed description of risks and uncertainties Sarepta faces, you are encouraged to review the SEC filings made by Sarepta. We caution investors not to place considerable reliance on the forward-looking statements contained in this press release. Sarepta does not undertake any obligation to publicly update its forward-looking statements based on events or circumstances after the date hereof.

Selecta Contacts:For Media:Joshua R. MansbachSolebury Trout+1-646-378-2964jmansbach@soleburytrout.com

For Investors:Lee M. SternSolebury Trout+1-646-378-2922lstern@soleburytrout.com

Sarepta Contacts: Investors:Ian Estepan, 617-274-4052iestepan@sarepta.com

Media:Tracy Sorrentino, 617-301-8566tsorrentino@sarepta.com

i Nature Communications,October 2018.

Original post:
Sarepta Therapeutics and Selecta Biosciences Enter Into Research License and Option Agreement for Selectas ImmTOR Immune Tolerance Platform in...

Recommendation and review posted by Bethany Smith

The Gene Therapy Products Market report is a truthful source of information which offers a telescopic view of the current market trends, situations, opportunities and status. All the data and information collected in this market document for research and analysis is represented in the form of graphs, charts or tables for the sensible understanding of users. Besides, big sample sizes have been exploited for the data collection in this report which ensembles the necessities of small, medium as well as large size of businesses. This Gene Therapy Products Market analysis report works on all the aspects of market that are required to create the finest and top-notch market research report.

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Increasing prevalence of Gene Therapy Products Market report highlights the notable industry growth drivers, challenges, opportunities and restraints for the leading key players. This market research report assesses in progress as well as future performance of the market in addition to brand-new trends in the market. This market research report also identifies and analyses the emerging trends along with major driving factors, challenges and opportunities in the market. The report includes market shares for global, Europe, North America, Asia Pacific and South America. The global Gene Therapy Products Market business report is a great resource which provides current and upcoming technical and financial details of the industry.

Global gene therapy products market is set to witness a substantial CAGR in the forecast period of 2019- 2026. The report contains data of the base year 2018 and historic year 2017. Rising cancer cases and unused potential for emerging markets are the major factors for the growth of this market.

Few of the major competitors currently working in the globalgene therapy products marketareAdaptimmune., Anchiano Therapeutics, bluebird bio, Inc., CELGENE CORPORATION, GlaxoSmithKline plc., Merck KGaA, Novartis AG, Achieve Life Sciences, Inc., Spark Therapeutics, Inc., Abeona Therapeutics, Inc, Adverum, agtc, Arbutus Biopharma, Audentes Therapeutics, AveXis, Inc., CRISPR Therapeutics, Intellia Therapeutics, Inc and Gilead Sciences,Inc. among others.

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Market Definition:Global Gene Therapy Products Market

Gene therapy or human gene therapy is a process which is used to modify gene for the treatment of any disease. Plasmid DNA, bacterial vector, human gene editing technology and viral vectors are some of the most common type of gene therapy products. The main aim of the gene therapy is to replace the dysfunctional genes. Somatic and germline are some of the most common type of the gene therapy.

Segmentation: Global Gene Therapy Products Market

Gene Therapy Products Market : By Product

Gene Therapy Products Market : By Application

Gene Therapy Products Market : ByGeography

Key Developments in the Gene Therapy Products Market:

Gene Therapy Products Market Drivers

Gene Therapy Products Market Restraints

Competitive Analysis: Gene Therapy Products Market

Global gene therapy products market is highly fragmented and the major players have used various strategies such as new product launches, expansions, agreements, joint ventures, partnerships, acquisitions, and others to increase their footprints in this market. The report includes market shares of gene therapy products market for Global, Europe, North America, Asia-Pacific, South America and Middle East & Africa.

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Gene Therapy Products Market 2020 Global Industry Trends and Forecast | Major Players are Adverum, agtc, Arbutus Biopharma, Audentes Therapeutics,...

Recommendation and review posted by Bethany Smith

Global Gene Therapy for Age-related Macular Degeneration Market Research Report Cover Covid-19 Impact

The Gene Therapy for Age-related Macular Degeneration market research report fabricated by Brand Essence Market Research is an in-depth analysis of the latest trends persuading the business outlook. The report also offers a concise summary of statistics, market valuation, and profit forecast, along with elucidating paradigms of the evolving competitive environment and business strategies enforced by the behemoths of this industry.

Download Premium Sample of the Report: https://industrystatsreport.com/Request/Sample?ResearchPostId=11998&RequestType=Sample

Latest research report on Gene Therapy for Age-related Macular Degeneration Market delivers a comprehensive study on current market trends. The outcome also includes revenue forecasts, statistics, market valuations which illustrates its growth trends and competitive landscape as well as the key players in the business.

Macular degeneration is a condition in which, macula, a part of the retina, gets damaged or deteriorated. This condition usually affects individuals who are aged 50 years and above and therefore, it is called age-related macular degeneration (AMD). AMD is the leading cause of vision loss and is directly related to the advancement of age. But smoking also plays a vital role in causing AMD. AMD is characterized by the presence of a blurred area near the center of vision that leads to distorted vision. There are two different types of AMD, including dry (atrophic) AMD (dAMD) and wet (neovascular/exudative) AMD (wAMD). The dAMD is the most common type of AMD and accounts for almost 80%-90% of the overall AMD cases.

It has been observed that age-related macular degeneration (AMD) is one of the major causes for vision loss and is characterized by the formation of a blurred area near the center of vision, a condition that mostly affects the geriatric population. According to the CDC, almost 2 million individuals in the US suffer from AMD and by 2050, this number will reach more than 5 million. This will subsequently demand the need for the development of innovative treatments for AMD, driving the markets growth.

The market research analysts have predicted that with the introduction of techniques such as fluorescein angiography, the global age-related macular degeneration market will register a CAGR of more than 7% by 2020. With the unavailability of FDA-approved treatment for dry AMD (dAMD) and the treatment of wet AMD (wAMD) involving the need of intravitreal injections for an indefinite period, gene therapy is emerging as the most-efficient approach for the treatment of age-related macular degeneration (AMD).

According to this pipeline analysis report, most of the gene therapy molecules in the pipeline are being developed for wet AMD (wAMD). Our market research analysts have also identified that most of these molecules are in the pre-clinical development stage and a considerable number of molecules have been discontinued from development.

This report focuses on the global Gene Therapy for Age-related Macular Degeneration status, future forecast, growth opportunity, key market and key players. The study objectives are to present the Gene Therapy for Age-related Macular Degeneration development in United States, Europe and China.

The key players covered in this study RetroSense Therapeutics REGENXBIO AGTC

Market segment by Type, the product can be split into Subretinal Intravitreal Unspecified

Market segment by Application, split into Monotherapy Combination Therapy

In this study, the years considered to estimate the market size of Gene Therapy for Age-related Macular Degeneration are as follows: History Year: 2014-2018 Base Year: 2018 Estimated Year: 2019 Forecast Year 2019 to 2025

Market segment by Regions/Countries, this report covers United States Europe China Japan Southeast Asia India Central & South America

The study objectives of this report are: To analyze global Gene Therapy for Age-related Macular Degeneration status, future forecast, growth opportunity, key market and key players. To present the Gene Therapy for Age-related Macular Degeneration development in United States, Europe and China. To strategically profile the key players and comprehensively analyze their development plan and strategies. To define, describe and forecast the market by product type, market and key regions.

For the data information by region, company, type and application, 2018 is considered as the base year. Whenever data information was unavailable for the base year, the prior year has been considered.

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Key Reasons to Purchase:

To gain insightful analyses of the market and a comprehensive understanding of the impact of COVID-19 is likely to have on the Gene Therapy for Age-related Macular Degeneration Market during the forecast period between 2020 and 2026, and its commercial landscapeTo learn about the market strategies that are being adopted by your competitors and other leading companiesTo understand the future outlook and prospects of the Gene Therapy for Age-related Macular Degeneration Market post COVID-19To keep you abreast with the strategies used by other players in theTo understand the changes in rules and regulations in various countries during COVID-19 and its possible effects on the market in the future.

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What's Driving on Gene Therapy for Age-related Macular Degeneration Market 2020 by Product, Application, Manufacturer, Sales and Segmentation 2025 -...

Recommendation and review posted by Bethany Smith

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The report segments the Global Cancer Therapies Market as:Global Cancer Therapies Market Size & Share, by Regions

Global Cancer Therapies Market Size & Share, by ProductsImmunological and Vaccine ApproachesAntiproliferative ApproachesPhotodynamic TherapyGene Therapy ApproachesCell Cycle and Apoptosis Approaches

Global Cancer Therapies Market Size & Share, ApplicationsHospital UseClinic UseHouseholdOther

Key PlayersAmgenAstraZenecaBristol-Myers SquibbBayer AGF. Hoffmann-La Roche LtdGlaxoSmithKline PLCJohnson & JohnsonMerck & Co. Inc.Novartis AGPfizer Inc.

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Global Cancer Therapies Market 2026 Size, Key Companies, Trends, Growth And Regional Forecasts Research - Cole of Duty

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