Gambling addiction is a clinically recognized problem that requires special therapy and medical intervention in most cases to achieve full recovery. The premise of gambling is simple you risk something you have in the hopes of adding to the value of your original stake.

However, by repeatedly engaging with products recognized as gambling, many people are prone to developing gambling addiction or showing symptoms of compulsive gambling without realizing it. If you find yourself addicted to gambling, this article is your path to recovery and understanding pathological gambling better.

The official gambling addiction definitionstates that an individual must experience an uncontrollable urge to gamblewith increasing amounts despite the high risk involvedand possibly serious consequences for ones own well-being or the well-being of family members, friends, and loved ones. Gambling can be addictive because it targets the brains reward system and leads to the release of dopamine, the hormone connected with happiness.

However, not all people are at risk of developing compulsive or pathological gambling. In fact, most people are able to resist, but a good number of people are experiencing problem gamblingor are at risk of becoming compulsive gamblers themselves.

Today, gambling addiction or compulsive gambling is a mental health issue, and the addiction at its core is equated to abuseof substancessuch as alcoholand drugs. Gambling affects more men than women, but it is a genderless disorder with a high incidence across the human population. Different countries around the world recognize gambling addiction as a serious mental problem that needs specialist intervention.

The term compulsion used to describe an independent aberration but today compulsive gambling is equated to or synonymous with gambling addiction.

Problem gambling begins discreetly and privately. In most cases, an individual is unaware that they are developing an addiction to compulsive gambling right to the point where the condition starts interfering with day-to-day chores and responsibilities. Anxietyor depressionis another concomitant symptom of burgeoning or pathological gambling addiction. Similar to other addictions, as in the case of substance addicts who overuse drugs or alcohol, problem gambling is driven on by a chemical component in the brain.

Compulsive gamblers feel rewarded for pursuing an activity that feels good, but this feeling of elation or high, is connected to a chemical response from the brain which floods the frontal cortex with dopamine. Addictive substances may trick the brain into releasing these hormones that are ten levels above normal levels.

Studies have established that gambling products can elicit the same chemical response from peoples brains, leading to high-dopamine production and spiraling into problem gambling without the knowledge of the individual.

Impulsivity and reward-seeking behavior become norms without explanation and perfectly ordinary individuals begin to behave as problem gamblers, showing symptoms and signs of gambling addiction that appear almost without a warning. Today gambling addiction is also referred to as gambling disorderand it encompasses an effort by the scientific community, including the American Psychiatric Association, to establish the reasons behind addictive behavior, in gambling and beyond.

By now, research has shown that addicts, regardless of their chosen form of addiction, whether that is substance abuse problems or gambling products, have a genetic predisposition that gives insight into how these problems develop, evolve, and how they can be eventually treated to the benefit of problem gamblers. Gambling is officially recognized as an impulse control disorderand this is good news because treatment professionals are tasked with making feel better. But first, they need to know how to read the symptoms.

Gambling addiction in patients tends to be approached differently. To start helping, though, experts must first identify gambling addictionsignsand symptoms, which is essential to getting an early start in treatment. These symptoms will vary between individuals in terms of intensity and some may remain hidden until very late in the addiction.

From the standpoint of most problem gamblers, gambling addiction is described as a complete loss of controlover the individuals ability to control spending habits, even though the potentially ruinous consequences are well-understood by the majority of pathological gamblers.

Therefore, problem gambling will always pivot around core behavioral aberrations that can be predicted, studied, and counterweightedin therapy or by the individual themselves depending on how advanced the compulsion is.

To spot a symptom, you may use our manual, which will help you help determine if you are currently showing compulsive gambling symptoms to a point where they need professional help.

Even if you are not a problem gambler, it is always important to monitor your condition, especially if you have a history of substance abuse in your family or recognize addictive patterns in your own behavior. Here are several points that constitute symptoms and signs of gambling addiction.

Thanks to clinical experience and government-funded research, we understand the downsides of gambling a little better. Moreover, studying gambling has allowed health expertsto come with a very detailed breakdown and create accessible help materials that will assist you in identifying a problem if there is one. More importantly, remember to answer truthfully to the following questions for accurate results:

The National Healthcare System (NHS) in the United Kingdom recommends using a four-answer system where you will score differently based on your answers. Your answers should be never, sometimes, most of the time, almost always and you should score 0, 1, 2, and 3 for each respectively. If your score is higher than 8, the NHS suggests that you may be a problem gambler or you may have a gambling addiction.

Pathological gamblingis not an easy thing to deal with, and this is why governments continue to spend money on research and to fund specialist clinic centers. While some individuals may find it easier to distance themselves from compulsive gambling, for others fighting addiction is a life-long struggle.

Compulsion has a powerful pull and most health experts try to find a way that individuals can stay away from temptation, and resist your craving to gamble. With this said, stopping gambling is easy, but replacing it with a positive, constructive emotion can take time, effort, and grit.

The good news is that despite the difficulties, maintaining recovery is very possible, although it may require lifestyle changes and constant effort of will on your part. Certain pathologies, such as mental disorders, can be channeled into something positive. Start small and remember that exerting control over behavior is essential to long-lasting effects. Self-help is a good place to start, but you may seek professional help over time.

When you are a diagnosed gambling addict, the best thing you can do is to stop gambling. To do so, you must put some distance between gambling products and yourself. However, this is difficult at a time when the Internet makes it very easy for gambling firms to have targeted outreach through data use. Because of this, gambling companies know exactly who wishes to gamble based on social media and browser history.

Thankfully, there is a solution. You can put considerable distance between yourself and temptations. Avoid searching for gambling, and channel addiction elsewhere. Flag every ad you get on social media or Google that targets you, explaining that you are a recovering gambling addict and such content must not be displayed to you.

Avoid visiting casinos, whether in person or online and ask your friends and family members to respect your wishes in not bringing topics that are associative with your gambling addiction or that may trigger a strong urge to gamble.

Gambling addiction can be controlled by a system of checks and balances whereby you create a social circle that will help you keep yourself in check. Networking and joining groups with other people experiencing the same issues as you are, or better yet, who have recovered completely, is a good way to begin your recovery.

In addiction vernacular, individuals who take care of struggling addicts are known as mentors. Peer groups can have a strong and positive influence on your life, creating a sort of achievement system which you want to unlock, and that influences your life for the better.

When the desire to gamble becomes too strong, make sure to reach out to family, friends, or your designated mentor. Relapses are common, so do not beat yourself up if you do happen to give in on occasion, but know that your recovery depends on unwavering determination.

Because addiction takes time to tackle, and then effort and consistency to maintain recovery, many people create if-scenarios where they agree to complete a certain part of their treatment if they are allowed to experience the subject of their addiction.

Such if-statements are dangerous and they do not address the issue at hand, but rather postpone the urge to gamble and lead to pent-up desire to play. The best way to deal with any compulsive or pathological gambling is to be aware of your situation and not predicate your recovery on future rewards.

In todays world, avoiding exposure to gambling productscan be difficult. Clever algorithms target you to the point where you almost cannot avoid running into a piece of advertisement, regardless of the medium that is used to deliver the message. However, this is where smart and powerful self-help tools come in handy.

Online gambling may seem all-pervasive, but advanced gambling markets have rolled out solutions that will literally block your access from every licensed operator in the country that is part of the exclusion program. Such programs exist in all European regulated markets, the United Kingdom, the United States, and Australia.

They are admittedly developed better in some regions than others, but ultimately they are used to treat pathological gambling. Governments still have to put more effort into helping gambling addicts, but the good news is that considerable efforts have already been made.

Gambling addicts have many ways out of this difficult position. The main challenge addicts face has to do with the fact that they are reluctant to admit they have a problem. Forcing therapy or treatment options on individuals rarely work, as an addict much more likely to resist help unless they have opted into it.

To this end, all recovery and treatment processes are predicated on the willingness of participants. An individual who acknowledges that they have a gambling problem can start experimenting with ways that allow him or her to control their failings.

Cognitive-behavioral therapy is a good approach to mental health. The therapy is predicated on finding the underlying reason for your behavior and why certain things trigger you. Most commonly, the conclusion is that certain experiences elicit a stronger response in your brain that sends a dopamine rush.

By teaching yourself to study, analyze, and understand these individual bursts of hormones, you can master and model your behavior in a way that is positive and healthy. There are many private and government treatment centers that offer this type of therapy.

Sometimes, an individual may experience a range of mental health problems that relate to and tie into gambling addiction. In fact, conditions such as OCDand ADHDmay be the originator of negative emotions that make you seek validation or quick thrill by engaging in reckless gambling behavior. Therefore some mood stabilizers and anti-depressants can go a long way in helping you deal with your gambling problem. Remember to not resort to medication on your own and seek professional help.

Self-help for gambling addiction is becoming very popular. Some people prefer to try and deal with the issue on their own. This is a perfectly valid approach and individuals who are strong-willed and in the habit of addressing their behavior analytically stand to benefit. Joining peer groups is a way to have a natural self-check and

Relapses are a problem gamblers bigger fear. Unfortunately, they also happen quite often, which is not in itself something to be worried about. Mental health and achieving a healthy mindset towards your gambling problem are important, and a relapse is not an end of your road to recovery.

Just the opposite, you need to develop the right attitude towards this momentary setback. Instead of hiding it, fearing stigma the same way you probably first hid your gambling addiction, its best to open up to a loved one, your support group, or a health specialist.

Dont let the cravings simmer and fester. Talk out your issue or urge to gamble with someone whom you trust and who has proven a moral bulwark for your recovery. The support of people whom you have come to trust and appreciate is essential to genuinely pushing past your addiction.

If you do relapse, dont spend too much time agonizing over this. It happens, and the fact you feel guilty means that you are advancing in your recovery. Talk out what you have achieved by relapsing and whether it was worth it.

Compare how you felt during recovery with how you felt when you satisfied your urge. More often than not, you will establish that your addiction is now physically and mentally unpleasant and your desire to gamble is much lesser than before you started treatment.

Relapses arent too bad when they happen, and if anything, they are a quick reality-check if your treatment is working.

Addiction simply means that you have enough energy and determination, notwithstanding the chemical factor involved. You can channel your determination to pursue an activity into something that has a far more beneficial effect on you.

Of course, moderation will be essential, as the goal is to master addictive behavior, not encourage it in one form or another. You can take up any hobby that has to do with sports, music, art, books or anything you wish, really.

Even 15 minutes of exercise a day can help you achieve much better mental health. Exercise releases dopamine and boosts your cognitive and mental abilities, leading to a healthier lifestyle free of cravings to achieve momentary satisfaction.

Remind yourself that your pursuit of the almighty dollar is illusionary and that gambling is rigged and statistically stacked against you. You cannot realistically expect the win simply because the odds are against you, and this is always the case.

Who is at a risk to develop a gambling addiction? Gambling problems come in many forms, but understanding the type of personality that it would take to go over the tipping point is important to understand impulse control disorders in the first place.

Common risk factors are now identified, but they are by no means final or a blueprint. However, gambling studies have proven with a fair degree of accuracy that there are several ways to understand who is the most at risk from pathological gambling.

Maybe you have been told that you ought to hide your gambling problems for the sake of decency. Perhaps that used to be right, but support and reaching out to others is the best way to tackle gambling addictions.

Today, treatment professionals encourage their patients to share stories so that people who are only beginning to deal with their issues may know they are not alone. While your suffering may seem personal and exclusive, your emotions are universal and human, and by sharing, you can cope and move on with your life.

No problem gambling story should be buried or covered up. By opening up, you will be helping others to see a way out as well as assist researchers and professionals who are genuinely concerned to study and understand problem gambling and a gamblers mind closer. All this health information can be put to good use.

To convince you that you are not in this alone, we have put together the stories of people whom you may know, but to whom you will feel a unique connection after hearing about their own experience with gambling. Read the personal gambling stories of people who have been through the crucible of problem gambling:

Pathological gamblers need help to address a gambling problem. This can be a loved one or trained medical professionals, a helpline, or a peer group. Whatever your choice and approach to tackling gambling addictions, you can rest assured that help is readily available.

Pathological gambling is a condition that should be taken seriously and deserves the attention of health experts. Thankfully, attitudes towards problem gamblers have evolved and your struggle is recognized as a valid medical concern.

With the help of your family, friends, peer groups, a helpline, or treatment facility, you can make sure that you deal with your impulses and control your cravings.

By following your healthcare professional advice and building a safe environment for yourself, you can not only learn to live with gambling addiction but outlive it. You can do better, and your friends, family, and people you love believe in you.

Problem gambling, also known as compulsive gambling, pathological gambling, or gambling addiction in laymens terms, is a mental health issue that affects close to 0.5% of the population. Its characterized by the compulsive need to spend money on gambling and gambling without a stop.

Yes, gambling addiction is a type of impulse control disorder and is a recognized mental health problem. Sufferers cannot control themselves from making potentially perilous decisions that impact their livelihoods, mental, and physical well-being.

Find a therapist, use self-exclusion tools, and join a support group. Fighting gambling addiction and avoiding relapsing is a lifetime battle. With proper nurturing and guidance, you can avoid returning to pathological gambling, though.

Healthcare facilities, free clinics, non-for-profit organizations all offer help in dealing with problem gambling. Pathological gamblers can find numerous organizations willing to help, including GamblersAnonymous, GamCare, GamStop, and more.

Cognitive-behavioral therapy, peer groups, gambling addiction treatment programs are among the most popular choices. Self-help is another viable option with free clinics offering pro-bono help to problem gamblers.

Gambling addiction begins with anxiety, overspending, and constant preoccupation about gambling games. Individuals experiencing gambling addiction may lie to, borrow, or steal money from friends, family, and employers to fuel their habit.

Go here to read the rest:
What Is Gambling Addiction and Problem Gambling? - GamblingNews.com

Recommendation and review posted by Bethany Smith

Good afternoon, and welcome health colleagues to the first European Alliance for Personalised Medicine (EAPM) update of the week, as we move delightedly towards Christmas. EAPM has just released a leading paper on gene therapy more of this below, along with the customary updates, writes EAPM Executive Director Denis Horgan.

ATMPs push forward prospects for tackling severe disease

EAPM has released a paper on gene therapy, based on its recent policy discussion, Propelling Healthcare with Advanced Therapy Medicinal Products.Challenges confront the sector, complicating the translation from research into patient access. Scientific, clinical development and regulatory issues are compounded by limited experience with clinical and commercial use, limited manufacturing know-how, high costs, and difficulties in accessing development funding and investment.

Pricing and reimbursement and market access issues are an additional challenge, particularly in Europe, where unfamiliarity with the technology and uncer- tainty over the use of real-world evidence induce caution among clinicians, health technology assessment bodies and payers. There is a need for a review of the suitability of the regula- tory and market access framework for these products, focused development of data, public/ private partnerships, and fuller collaboration governments, doctors, insurers, patients, and pharmaceutical companies.

This paper makes specific recommendations for allstakeholders, ranging from early dialogue on potential products, linking of clinical data, and patient registries or standardization of control frameworks, to a comprehensive approach to evidencegeneration, assessment, pricing, and payment for ATMPs.The paper isavailable here.

5.1 billion secured for EU Health Programme

On 14 December, the biggest ever EU Health Programme and the rules to distribute its 5.1 billion funds were agreed. Negotiators of the European Parliament and EU member states struck a deal on the law setting up the European Unions so called 'EU4Health' Programme from 2021 onwards.

"The COVID-19 pandemic has revealed that Europe was not equipped to deal with a serious health crisis. During the first peak of the pandemic, we not only lacked doctors, nurses and medical staff, but also medicines and medical equipment. It must never happen again that a doctor must choose who lives or dies because the hospital does not have resources to help all," said the EPP Group's Cristian Silviu Buoi MEP, the lead negotiator for the European Parliament and a stronger support of EAPM.

"Therefore, the programme will allocate funds for the establishment of a reserve for essential crisis-relevant products, medical and healthcare staff, in synergy and complementarity with other EU instruments," he added. Requisite to the health programme that Buoi negotiated is an increase in health funding in the new EU long-term budget.

The EPP Group also wanted the programme to support the training of health staff, reduce health inequalities, support digitalization in the health-care sector, finance a masterplan against cancer and bring back the production of medicines to Europe.

WHO does not envisage COVID-19 vaccines being made mandatory

The World Health Organization (WHO) does not foresee countries making it mandatory for citizens to take the new COVID-19 vaccines which have been developed, an official said. I dont think we envisage any countries creating a mandate for vaccinations, Kate OBrien, the WHOs director of immunization vaccines and biologicals, told a news conference.

Pressure mounts on EU drug regulator to approve COVID-19 vaccine

Europes drug regulator is under increasing pressure to quickly approve the COVID-19 vaccine developed by Germanys BioNTech, officials said, as inoculations get started in Britain and the United States. The push underscores the frictions between regulators and governments wishing to curb the pandemic that has killed more than 1.6 million people worldwide. Four EU sources said that the European Medicines Agency (EMA) has been under pressure from the European Commission and EU governments to approve vaccines more quickly. One EMA official said on 14December that pressure had increased on the agency from EU governments through usual channels of communications after 2 December, when the British regulator granted an emergency authorization to the Pfizer/BioNTech vaccine.

UK health secretary announces discovery of new coronavirus variant

UK Health SecretaryMatt Hancock appeared in the House of Commons on Monday (14 December) to give an urgent statement to MPs on the coronavirus pandemic in the UK. He said experts haveidentified a new variant of COVID-19 which may be responsible for the "faster spread" in south-east England.

Hancock said: "Over the last few days, thanks to our world-class genomic capability in the UK, we have identified a new variant of coronavirus which may be associated with the faster spread in the south of England."

He added: "Initial analysis suggests that this variant is growing faster than the existing variants. We've currently identified over 1,000 cases with this variant predominantly in the South of England although cases have been identified in nearly 60 different local authority areas."

When asked if the new variant will impact the effectiveness of the vaccine, Hancock said: "The medical advice that we have is that it is highly unlikely that this new variant will impinge the vaccine and the impact of the vaccine. But we will know that in the coming days and weeks as the new strand is cultured at Porton Down and then the tests conducted upon it.

WHO a touch more circumspect

The World Health Organization is aware of the new variant of COVID-19 that has emerged in Britain, but there is no evidence the strain behaves differently to existing types of the virus, it said onMonday (14 December).We are aware of this genetic variant reported in 1,000 individuals in England, the WHOs top emergencies expert Mike Ryan told a news briefing in Geneva. Authorities are looking at its significance. We have seen many variants, this virus evolves and changes over time.

FDA authorizes vaccine following extreme Trump pressure

The US Food and Drug Administration (FDA) on Friday (11 December) gave emergency use authorization to the nations first coronavirus vaccine, launching what scientists hope will be a critical counteroffensive against a pathogen that has killed more than 290,000 Americans, shredded the nations social and political fabric and devastated the economy.

The historic authorization of thevaccine from Pfizer and BioNTech for people age 16 and older, just 336 days after the genetic blueprint of a novel coronavirus was shared online by Chinese scientists, sets in motion a highly choreographed and complex distribution process aimed at speeding vaccines throughout the United States to curb the pandemic.

US President Donald Trump had put the FDA under extreme pressure to approve thevaccine, describing the organization as a slow, old turtle in a tweet. The FDA action came after White House Chief of Staff Mark Meadows on Friday told FDA commissioner Stephen Hahn to be prepared to submit his resignation if the agency did not clear the vaccine by days end, according to people familiar with the situation who spoke on the condition of anonymity because they were notauthorized to discuss what happened.

And that is everything for the start of the week from EAPM dont forget to check our gene therapy paperhere,have an excellent week.

See original here:
EAPM Advancing gene therapy with Advanced Therapy Medicinal Products - EU Reporter

Recommendation and review posted by Bethany Smith

Locanabio, a San Diego gene therapy company focused on treatments for severe neurodegenerative diseases such as Huntingtons and Lou Gehrigs disease, has raised $100 million in a second round of venture capital funding.

The Torrey Pines Mesa company will use the money for further pre-clinical and clinical development of its proprietary RNA-targeting system to fight degenerative diseases including myotonic dystrophy type 1 and retinal disease, along with Huntingtons and genetic ALS.

Locanabios approach is to combine two methods for treating diseases gene therapy and RNA modification. The platform consists of several RNA-targeting systems that are combined with gene therapy delivery to modify dysfunctional RNA.

The capabilities of the platform could allow Locanabio to develop treatments for a wide range of genetic diseases beyond those on its current roadmap.

This financing positions us to accelerate our efforts to advance multiple promising programs into (new drug) studies in 2021 and to further develop our novel RNA-targeting platform, which has the potential to be a major new advance in medicine that can bring hope to patients with many devastating genetic diseases, said Chief Executive Jim Burns in a statement.

Burns joined Locanabio in December 2019 from Casebia, where he served as the chief executive and led the team in developing CRISPR-based therapeutics to treat blood disorders, blindness and heart disease. Before that, he spent the bulk of his career at Sanofi-Genzyme, where he held several leadership roles.

This latest financing was led by Vida Ventures. Other new investors participating include RA Capital Management, Invus, Acuta Capital Partners and an investment fund associated with SVB Leerink.

Prior investors ARCH Venture Partners, Temasek, Lightstone Ventures, UCB Ventures and Google Ventures also participated. Lonanabio previously raised $55 million in May 2019.

As part of the funding round, Rajul Jain, a medical doctor and director of Vida Ventures, will join Locanabios board of directors.

The unique approach in RNA targeting using gene therapy to deliver RNA binding proteins developed by Locanabio represents the next frontier of genetic medicine with the ability to target the root cause of a range of genetic diseases, said Jain in a statement. They have built a strong management team to execute this bold vision, and we are proud to support them.

See the article here:
San Diego's Locanabio raises $100 million for treatments aimed at degenerative diseases - The San Diego Union-Tribune

Recommendation and review posted by Bethany Smith

Good afternoon, and welcome health colleagues to the first European Alliance for Personalised Medicine (EAPM) update of the week, as we move delightedly towards Christmas. EAPM has just released a leading paper on gene therapy more of this below, along with the customary updates, writes EAPM Executive Director Denis Horgan.

ATMPs push forward prospects for tackling severe disease

EAPM has released a paper on gene therapy, based on its recent policy discussion, Propelling Healthcare with Advanced Therapy Medicinal Products.Challenges confront the sector, complicating the translation from research into patient access. Scientific, clinical development and regulatory issues are compounded by limited experience with clinical and commercial use, limited manufacturing know-how, high costs, and difficulties in accessing development funding and investment.

Pricing and reimbursement and market access issues are an additional challenge, particularly in Europe, where unfamiliarity with the technology and uncer- tainty over the use of real-world evidence induce caution among clinicians, health technology assessment bodies and payers. There is a need for a review of the suitability of the regula- tory and market access framework for these products, focused development of data, public/ private partnerships, and fuller collaboration governments, doctors, insurers, patients, and pharmaceutical companies.

This paper makes specific recommendations for allstakeholders, ranging from early dialogue on potential products, linking of clinical data, and patient registries or standardization of control frameworks, to a comprehensive approach to evidencegeneration, assessment, pricing, and payment for ATMPs.The paper isavailable here.

5.1 billion secured for EU Health Programme

On 14 December, the biggest ever EU Health Programme and the rules to distribute its 5.1 billion funds were agreed. Negotiators of the European Parliament and EU member states struck a deal on the law setting up the European Unions so called 'EU4Health' Programme from 2021 onwards.

"The COVID-19 pandemic has revealed that Europe was not equipped to deal with a serious health crisis. During the first peak of the pandemic, we not only lacked doctors, nurses and medical staff, but also medicines and medical equipment. It must never happen again that a doctor must choose who lives or dies because the hospital does not have resources to help all," said the EPP Group's Cristian Silviu Buoi MEP, the lead negotiator for the European Parliament and a stronger support of EAPM.

"Therefore, the programme will allocate funds for the establishment of a reserve for essential crisis-relevant products, medical and healthcare staff, in synergy and complementarity with other EU instruments," he added. Requisite to the health programme that Buoi negotiated is an increase in health funding in the new EU long-term budget.

The EPP Group also wanted the programme to support the training of health staff, reduce health inequalities, support digitalization in the health-care sector, finance a masterplan against cancer and bring back the production of medicines to Europe.

WHO does not envisage COVID-19 vaccines being made mandatory

The World Health Organization (WHO) does not foresee countries making it mandatory for citizens to take the new COVID-19 vaccines which have been developed, an official said. I dont think we envisage any countries creating a mandate for vaccinations, Kate OBrien, the WHOs director of immunization vaccines and biologicals, told a news conference.

Pressure mounts on EU drug regulator to approve COVID-19 vaccine

Europes drug regulator is under increasing pressure to quickly approve the COVID-19 vaccine developed by Germanys BioNTech, officials said, as inoculations get started in Britain and the United States. The push underscores the frictions between regulators and governments wishing to curb the pandemic that has killed more than 1.6 million people worldwide. Four EU sources said that the European Medicines Agency (EMA) has been under pressure from the European Commission and EU governments to approve vaccines more quickly. One EMA official said on 14December that pressure had increased on the agency from EU governments through usual channels of communications after 2 December, when the British regulator granted an emergency authorization to the Pfizer/BioNTech vaccine.

UK health secretary announces discovery of new coronavirus variant

UK Health SecretaryMatt Hancock appeared in the House of Commons on Monday (14 December) to give an urgent statement to MPs on the coronavirus pandemic in the UK. He said experts haveidentified a new variant of COVID-19 which may be responsible for the "faster spread" in south-east England.

Hancock said: "Over the last few days, thanks to our world-class genomic capability in the UK, we have identified a new variant of coronavirus which may be associated with the faster spread in the south of England."

He added: "Initial analysis suggests that this variant is growing faster than the existing variants. We've currently identified over 1,000 cases with this variant predominantly in the South of England although cases have been identified in nearly 60 different local authority areas."

When asked if the new variant will impact the effectiveness of the vaccine, Hancock said: "The medical advice that we have is that it is highly unlikely that this new variant will impinge the vaccine and the impact of the vaccine. But we will know that in the coming days and weeks as the new strand is cultured at Porton Down and then the tests conducted upon it.

WHO a touch more circumspect

The World Health Organization is aware of the new variant of COVID-19 that has emerged in Britain, but there is no evidence the strain behaves differently to existing types of the virus, it said onMonday (14 December).We are aware of this genetic variant reported in 1,000 individuals in England, the WHOs top emergencies expert Mike Ryan told a news briefing in Geneva. Authorities are looking at its significance. We have seen many variants, this virus evolves and changes over time.

FDA authorizes vaccine following extreme Trump pressure

The US Food and Drug Administration (FDA) on Friday (11 December) gave emergency use authorization to the nations first coronavirus vaccine, launching what scientists hope will be a critical counteroffensive against a pathogen that has killed more than 290,000 Americans, shredded the nations social and political fabric and devastated the economy.

The historic authorization of thevaccine from Pfizer and BioNTech for people age 16 and older, just 336 days after the genetic blueprint of a novel coronavirus was shared online by Chinese scientists, sets in motion a highly choreographed and complex distribution process aimed at speeding vaccines throughout the United States to curb the pandemic.

US President Donald Trump had put the FDA under extreme pressure to approve thevaccine, describing the organization as a slow, old turtle in a tweet. The FDA action came after White House Chief of Staff Mark Meadows on Friday told FDA commissioner Stephen Hahn to be prepared to submit his resignation if the agency did not clear the vaccine by days end, according to people familiar with the situation who spoke on the condition of anonymity because they were notauthorized to discuss what happened.

And that is everything for the start of the week from EAPM dont forget to check our gene therapy paperhere,have an excellent week.

Continue reading here:
Italy needs new restrictions to avoid third, devastating COVID-19 wave - PM to paper - EU Reporter

Recommendation and review posted by Bethany Smith

PHOENIX, Dec. 16, 2020 /PRNewswire/ --Creative Medical Technology Holdings Inc., (OTC CELZ) announced today positive preclinical data supporting the utilization of its ImmCelz cell based immunotherapy for treatment of stroke. In an animal model of ischemia stroke, the middle cerebral artery ligation model, administration of ImmCelz resulted in 34% reduction in infarct volume, whereas control bone marrow mesenchymal stem cells reduced infarct volume by 21%. Additionally, improvements in functional recovery where observed using the Rotarod test. At 28 days after induction of stroke the animals receiving ImmCelz had superior running time (92% of non-stroke controls) compared to animals which received bone marrow mesenchymal stem cells (73% of non-stroke control). Animals that received saline had a running time that was 50% of non-stroke controls.

"The regenerative potential of immune cells that have been programmed by stem cells is a fascinating and novel area of research." Said Dr. Amit Patel, coinventor of ImmCelz, and board member of the Company. "Conceptual advantages of using reprogrammed T cells include higher migratory ability due to smaller size, as well as ability to replicate and potentially form "regenerative memory cells."

"This data, which is covered by our previous filed patents, such as no. 15/987739, Generation of autologous immune modulatory cells for treatment of neurological conditions, demonstrate that immune modulation via this stem cell based method may be a novel and superior way of addressing the $30 billion dollar market for stroke therapeutics1." Said Dr. Thomas Ichim, coinventor of the patent and Chief Scientific Officer of the Company. "The fact that this technology, which has priority back to 2017, is demonstrating such stunning results, motivates us to consider filing an Investigational New Drug Application for use in stroke."

Creative Medical Technology Holdings possesses numerous issued patents in the area of cellular therapy including patent no. 10,842,815 covering use of T regulatory cells for spinal disc regeneration, patent no. 9,598,673 covering stem cell therapy for disc regeneration, patent no. 10,792,310 covering regeneration of ovaries using endothelial progenitor cells and mesenchymal stem cells, patent no. 8,372,797 covering use of stem cells for erectile dysfunction, and patent no. 7,569,385 licensed from the University of California covering a novel stem cell type.

"While stroke historically has been a major area of unmet medical need, the rise in stroke cases , as well as the fact that younger people are increasingly falling victim to stroke, strongly motivates us to accelerate our developmental programs and to continue to explore participation of Big Pharma in this space." Said Timothy Warbington, President and CEO of the Company. "We are eager to replicate the existing experiments start compiling the dossier needed to take ImmCelz into humans using the Investigational New Drug Application (IND) route through the FDA."

About Creative Medical Technology Holdings

Creative Medical Technology Holdings, Inc. is a commercial stage biotechnology company specializing in stem cell technology in the fields of urology, neurology and orthopedics and trades on the OTC under the ticker symbol CELZ. For further information about the company, please visitwww.creativemedicaltechnology.com.

Forward Looking Statements

OTC Markets has not reviewed and does not accept responsibility for the adequacy or accuracy of this release. This news release may contain forward-looking statements including but not limited to comments regarding the timing and content of upcoming clinical trials and laboratory results, marketing efforts, funding, etc. Forward-looking statements address future events and conditions and, therefore, involve inherent risks and uncertainties. Actual results may differ materially from those currently anticipated in such statements. See the periodic and other reports filed by Creative Medical Technology Holdings, Inc. with the Securities and Exchange Commission and available on the Commission's website atwww.sec.gov.

Timothy Warbington, CEOCEO@ CreativeMedicalHealth.com

Creativemedicaltechnology.comwww.StemSpine.comwww.Caverstem.comwww.Femcelz.com

1 Stroke Management Market Size Forecasts 2026 | Statistics Report (gminsights.com)

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Creative Medical Technology Holdings Announces Successful Application of ImmCelz Immunotherapy for Treatment of Stroke - KPVI News 6

Recommendation and review posted by Bethany Smith

Cancer researchers have created a new class of drugs to selectively target and destroy myeloid leukaemia cells with TET gene mutations.

Photomicrograph of bone marrow biopsy showing myeloblasts of acute myeloid leukemia (AML), a cancer of white blood cells.

Researchers have developed a novel class of targeted cancer drug that may be highly effective for the treatment of myeloid leukaemias. According to the team, their synthetic molecule, called TETi76, was able to selectively kill cells with TET2 gene mutations, one of the most common driver mutations in myeloid leukaemias.

Myeloid leukaemias are cancers derived from stem and progenitor cells in the bone marrow that give rise to all normal blood cells. These malignancies are normally treated with chemotherapy, either alone or in combination with targeted drugs; however, the significant side-effects associated with this treatment mean a more selective/targeted treatment is desirable.

In a new study published inBlood Cancer Discovery, researchers from the Cleveland Clinics Taussig Cancer Institute and Lerner Research Institute, both US, describe a new pharmacological strategy to preferentially target and eliminate leukaemia cells with TET2 mutations.

In preclinical models, we found that a synthetic molecule called TETi76 was able to target and kill the mutant cancer cells both in the early phases of disease what we call clonal haematopoiesis of indeterminate potential, or CHIP and in fully developed TET2 mutant myeloid leukaemia, said Dr Jaroslaw Maciejewski, a practicing haematologist and chair of the Cleveland Clinic Department of Translational Hematology & Oncology Research, who has been investigating the TET2 gene for the last decade.

TET genes encode DNA dioxygenase enzymes, which remove chemical groups from DNA molecules. Their activity ultimately changes what genes are expressed and can contribute to the development and spread of disease.

TET genes act as tumour suppressors, so loss-of-function mutations are common in haematological cancers, like leukaemias. While all members of the TET family are dioxygenases, TET2 is the most powerful. Genetic TET2 deficiency has been shown to skew differentiation of blood cells and clonal expansion of progenitor and stem cells. However, its related genes TET1 and TET3 provide residual enzymatic activity, sufficient to facilitate the survival of these progenitor cells harbouring cancerous mutations, thereby promoting the spread of the cancer, even when TET2 is inactive.

In their study, the research team designed TETi76 to replicate and amplify the effects of a natural molecule called 2-hydroxyglutarate (2HG), which inhibits the enzymatic activity of TET genes. They hoped to selectively eliminate TET2 mutant leukaemia cells centres by targeting their reliance on this residual DNA dioxygenase activity.

We took lessons from the natural biological capabilities of 2HG, explained Dr Babal Kant Jha, Maciejewskis collaborator from the Department of Translational Hematology & Oncology Research. We studied the molecule and rationally designed a novel small molecule, synthesised by our chemistry group headed by Dr James Phillips. Together, we generated TETi76 a similar, but more potent version capable of inhibiting not just TET2, but also the remaining disease-driving enzymatic activity of TET1 and TET3.

The researchers studied TETi76s effects in both preclinical disease and xenograft models (where human cancer cells are implanted into preclinical models). In both models, treatment with the novel TET inhibitor suppressed the clonal evolution of TET2 mutant cells.

While the team cautioned that additional studies would be critical to investigate the small molecules cancer-fighting capabilities in patients, Dr Jha said we are optimistic about our results, which show not just that TETi76 preferentially restricts the growth and spread of cells with TET2 mutations, but also gives survival advantage to normal stem and progenitor cells.

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Novel class of targeted cancer therapies could treat myeloid leukaemias - Drug Target Review

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Victoria Gray (second from left) with children Jamarius Wash, Jadasia Wash and Jaden Wash. Now that the gene-editing treatment has eased Gray's pain, she has been able be more active in her kids' lives and looks forward to the future. "This is really a life-changer for me," she says. Victoria Gray hide caption

Victoria Gray (second from left) with children Jamarius Wash, Jadasia Wash and Jaden Wash. Now that the gene-editing treatment has eased Gray's pain, she has been able be more active in her kids' lives and looks forward to the future. "This is really a life-changer for me," she says.

The last thing a lot of people want to do these days is get on a plane. But even a pandemic would not stop Victoria Gray. She jumped at the chance to head to the airport this summer.

"It was one of those things I was waiting to get a chance to do," says Gray.

She had never flown before because she was born with sickle cell disease. She feared the altitude change might trigger one of the worst complications of the devastating genetic disease a sudden attack of excruciating pain.

But Gray is the first person in the United States to be successfully treated for a genetic disorder with the help of CRISPR, a revolutionary gene-editing technique that makes it much easier to make very precise changes in DNA.

About a year after getting the treatment, it was working so well that Gray felt comfortable flying for the first time. She went to Washington, D.C., to visit her husband, who has been away for months on deployment with the National Guard.

"It was exciting. I had a window. And I got to look out the window and see the clouds and everything," says Gray, 35, of Forest, Miss.

Gray wore a mask the whole time to protect herself against the coronavirus, kept her distance from other people at the airport, and arrived happily in Washington, D.C., even though she's afraid of heights.

"I didn't hyperventilate like I thought I would," Gray says, laughing as she recounts the adventure in an interview with NPR.

NPR has had exclusive access to follow Gray through her experience since she underwent the landmark treatment on July 2, 2019. Since the last time NPR checked in with Gray in June, she has continued to improve. Researchers have become increasingly confident that the approach is safe, working for her and will continue to work. Moreover, they are becoming far more encouraged that her case is far from a fluke.

At a recent meeting of the American Society for Hematology, researchers reported the latest results from the first 10 patients treated via the technique in a research study, including Gray, two other sickle cell patients and seven patients with a related blood disorder, beta thalassemia. The patients now have been followed for between three and 18 months.

All the patients appear to have responded well. The only side effects have been from the intense chemotherapy they've had to undergo before getting the billions of edited cells infused into their bodies.

The New England Journal of Medicine published online this month the first peer-reviewed research paper from the study, focusing on Gray and the first beta thalassemia patient who was treated.

"I'm very excited to see these results," says Jennifer Doudna of the University of California, Berkeley, who shared the Nobel Prize this year for her role in the development of CRISPR. "Patients appear to be cured of their disease, which is simply remarkable."

Another nine patients have also been treated, according to CRISPR Therapeutics in Cambridge, Mass., and Vertex Pharmaceuticals in Boston, two companies sponsoring the research. Those individuals haven't been followed long enough to report any results, officials say.

But the results from the first 10 patients "represent an important scientific and medical milestone," says Dr. David Altshuler, Vertex's chief scientific officer.

The treatment boosted levels of a protein in the study subjects' blood known as fetal hemoglobin. The scientists believe that protein is compensating for defective adult hemoglobin that their bodies produce because of a genetic defect they were born with. Hemoglobin is necessary for red blood cells to carry oxygen.

Analyses of samples of bone marrow cells from Gray six months after getting the treatment, then again six months later, showed the gene-edited cells had persisted the full year a promising indication that the approach has permanently altered her DNA and could last a lifetime.

"This gives us great confidence that this can be a one-time therapy that can be a cure for life," says Samarth Kulkarni, the CEO of CRISPR Therapeutics.

Gray and the two other sickle cell patients haven't had any complications from their disease since getting the treatment, including any pain attacks or hospitalizations. Gray has also been able to wean off the powerful pain medications she'd needed most of her life.

Prior to the treatment, Gray experienced an average of seven such episodes every year. Similarly, the beta thalassemia patients haven't needed the regular blood transfusions that had been required to keep them alive.

"It is a big deal because we we able to prove that we can edit human cells and we can infuse them safely into patients and it totally changed their life," says Dr. Haydar Frangoul at the Sarah Cannon Research Institute in Nashville. Frangoul is Gray's doctor and is helping run the study.

For the treatment, doctors remove stem cells from the patients' bone marrow and use CRISPR to edit a gene in the cells, activating the production of fetal hemoglobin. That protein is produced by fetuses in the womb but usually shuts off shortly after birth.

The patients then undergo a grueling round of chemotherapy to destroy most of their bone marrow to make room for the gene-edited cells, billions of which are then infused into their bodies.

"It is opening the door for us to show that this therapy can not only be used in sickle cell and thalassemia but potentially can be used in other disorders," Frangoul says.

Doctors have already started trying to use CRISPR to treat cancer and to restore vision to people blinded by a genetic disease. They hope to try it for many other diseases as well, including heart disease and AIDS.

The researchers stress that they will have to follow Gray and many other patients for a lot longer to be sure the treatment is safe and that it keeps working. But they are optimistic it will.

Gray hopes so too.

"It's amazing," she says. "It's better than I could have imagined. I feel like I can do what I want now."

The last year hasn't always been easy for Gray, though. Like millions of other Americans, she has been sheltering at home with three of her children, worrying about keeping them safe and helping them learn from home much of the time.

"I'm trying to do the things I need to do while watch them at the same time to make sure they're doing the things they need to do," Gray says. "It's been a tough task."

But she has been able do other things she never got to do before, such as watch her oldest son's football games and see her daughter cheerleading.

"This is really a life-changer for me," she says. "It's magnificent."

She's now looking forward to going back to school herself, learning to swim, traveling more when the pandemic finally ends, and watching her children grow up without them worrying about their mother dying.

"I want to see them graduate high school and be able to take them to move into dorms in college. And I want to be there for their weddings just everything that the normal people get to do in life. I want to be able to do those things with my kids," she says. "I can look forward now to having grandkids one day being a grandmama."

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1st Patients To Get CRISPR Gene-Editing Treatment Continue To Thrive - NPR

Recommendation and review posted by Bethany Smith

Bone Regeneration Material Market: Introduction

Bone-regeneration techniques, either with autografts or allografts, represent a challenge for reconstructive surgery. Biomaterials are temporary matrices for bone growth and provide a specific environment and architecture for tissue development. Depending on the specific intended application of the matrix, whether for structural support, drug-delivery capability, or both, certain material categories may be more or less well suited to the final structure.

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Key Drivers and Restraints of Global Bone Regeneration Material Market

Increase in prevalence of degenerative joint diseases boost the market. Worldwide estimates of degenerative joint diseases indicate that 9.6% men and 18.0% women above 60 years have symptomatic osteoarthritis. According to expert opinions presented in the EULAR committee report, radiographic evidence of knee osteoarthritis in men and women over 65 years of age is found in 30% of the population.

In the absence of disease modifying therapy, a large number of patients with osteoarthritis progress to advance joint destruction. Surgery with bone grafts and substitutes play a major role in the management of osteoarthritis to avoid advanced joint destruction. According to the American College of Rheumatology, advances in biomaterial and tissue engineering are expected to create new opportunities to integrate surgical approaches in osteoarthritis.

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Increase in the number of orthopedic surgeries also fuels the market. According to the American Academy of Orthopaedic Surgeons (AAOS), approximately 129,000 total knee arthroplasty (TKA) surgeries were performed in the U.S. in 1990, and the number has increased to over 600,000 in 2010. The AAOS has projected that 3 million TKA procedures would be performed by 2030 in the U.S. alone. Moreover, spinal surgeries are becoming increasingly popular, and approximately 432,000 spinal fusions are performed in the U.S. each year. Bone grafts and substitutes are extensively used for the surgeries mentioned above. This is likely to fuel the bone regeneration material market.

Bone graft and substitutes are a long-term solution to bone problem treatment; however, these are expensive. No two patients or their customized bone grafts and substitutes treatments are exactly alike. Hence, the number of appointments, procedures, and costs vary accordingly. Surgeons charge US$ 35,000 to US$ 40,000 for a complex posterolateral lumbar spine fusion bone graft surgery. Most surgeons refer patients to specialty surgeons, neurologists, or orthopedic physicians, which increases the cost of procedure. Asia is price-sensitive and displays inhibitions with respect to investing in bone graft and substitutes, which are often only affordable to the elite population; therefore offering a comparatively smaller market.

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Cell-based Segment to Expand Significantly

Based on product type, the global bone regeneration material market can be divided into ceramic-based, polymer-based, growth factor-based, cell-based and others

The ceramic-based segment dominated the global market in 2019. It is projected to sustain its position during the forecast period. Ceramic-based bone grafts are widely used to reduce the need for iliac crest bone grafting. Rise in geriatric population with oral health issues across the world has augmented the number of bone graft surgeries performed in the last few years.

However, the cell based segment is projected to expand at a notable CAGR during the forecast period. Bone tissue engineering (BTE) using bone marrow stem cells has been suggested as a promising technique for reconstructing bone defect in order to overcome the drawbacks of bone graft materials.

Orthopedic surgery segment to dominate global bone regeneration material market

Based on application, the global bone regeneration material market can be segregated into orthopedic surgery, bone trauma, dental surgery and others.

In terms of revenue, the orthopedic surgery segment accounted for a prominent share of the market in 2019 owing to a rise in the geriatric population and increase in cases of orthopedic diseases. According to WHO, between 2015 and 2050, the proportion of the world's population over 60 years would nearly double from 12% to 22%. The number of people aged 60 years and older is estimated to outnumber children younger than 5 years by 2020. As per MVZ Gelenk-Klinik data, more than 2400 orthopedic surgical procedures are performed per year at the Gelenk Klinik Orthopaedic Hospital.

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North America to dominate global bone regeneration material market

In terms of region, the global bone regeneration material market can be divided into: North America, Europe, Asia Pacific, Latin America, and Middle East & Africa

North America accounted for a significant share of the bone regeneration material market in 2019, followed by Europe. Usage of new and innovative products in both premium and value segments among various bone grafts substitutes is projected to boost the bone regeneration material market in several countries in Europe and North America in the next few years. According to the Centers for Disease Control and Prevention (CDC), the total number of inpatient surgeries carried out in the U.S. were 51.4 million in 2014; of these 719,000 were total knee replacements and 332,000 were total hip replacement.

The market in developing countries in Asia Pacific is estimated to expand at a significant CAGR during the forecast period. The market in Asia Pacific is driven by an increase in population and time taken to accept new technologies. Increase in the number of patients and geriatric population are major factors that are expected to propel the market in Japan during the forecast period. According to the Gerontological Society of America, Japan has the highest proportion of geriatric population in the world. Hence, demand for orthopedic surgeries is estimated to be higher in Japan than that in other countries in Asia Pacific.

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Key Manufacturers Operating in Market

The global bone regeneration material market was highly fragmented in 2019. Key manufacturers operating in the global market are:

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Bone Regeneration Material Market: Cell-based Segment to Expand Significantly - BioSpace

Recommendation and review posted by Bethany Smith

If youre like 95% of American adults, you had chickenpox as a kid. Before the United States started its widespread vaccination program in 1995, there were roughly four million cases of chickenpox every year. So, most people suffered through an infection with this highly contagious virus and its itchy, whole-body rash.

But unlike many childhood viruses, the varicella-zoster virus that causes chickenpox doesnt clear from the body when the illness ends. Instead it hangs around, taking up residence and lying dormant in the nerves, sometimes for decades, with the immune system holding it in check. In some people, it lives there harmlessly for the rest of their life. But in others, the virus can suddenly emerge and strike again, this time appearing as a different condition known as shingles.

Like chickenpox, shingles also causes a blistering rash, but this time it generally appears as a painful band around one side of your ribcage or on one side of your face. The first symptom for many people is pain or a burning sensation in the affected area. You may also have fever, a headache, and fatigue. Along with the rash and other temporary symptoms, shingles can also bring unpleasant, long-lasting, and sometimes permanent complications, such as skin infections, nerve pain in the area where the rash appeared, or even vision loss.

Experts dont fully understand this. One theory is that shingles occurs when your immune system loses its ability to keep the virus in check.

After you get chickenpox, your immune system is able to recognize the varicella-zoster virus thanks to specialized immune system cells, called B and T cells, that are able to remember the virus and quickly marshal an attack on it. Factors that weaken the immune system increase your risk of developing shingles. These include

While you may not be able to control certain factors that might trigger shingles, there are strategies you can use to prevent shingles. The most important is vaccination. Research shows that the shingles vaccine Shingrix is 90% effective in preventing an outbreak of shingles. Even if you do get shingles after being vaccinated, Shingrix greatly reduces your risk of developing persistent pain in the affected area, known as post-herpetic neuralgia.

In addition to getting vaccinated, its always a good idea to take steps to keep your body healthy, such as choosing healthy foods, staying active, and getting sufficient sleep. Its not clear if healthy lifestyle habits like these can prevent shingles, but even if they dont, theyre worthwhile because they will benefit your body in many other ways.

Link:
Shingles: What triggers this painful, burning rash? - Harvard Health Blog - Harvard Health

Recommendation and review posted by Bethany Smith

Global Orthopedic Regenerative Medicine Market Growth To Increase Manifold By 2027 A fundamental overview of the Orthopedic Regenerative Medicine Market niche is provided in the Orthopedic Regenerative Medicine Market report accompanying definitions, classifications, applications along with industry chain framework. The Orthopedic Regenerative Medicine market report provides a broad assessment of the required market dynamics and the latest trends. It also highlights regional markets, prominent market players, multiple market segments [products, applications, end users, and key regions] and subsectors that broadly consider numerous departments along with applications.

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Top Key Players Global Orthopedic Regenerative Medicine Market Competition: Curasan, Inc., Carmell Therapeutics Corporation, Anika Therapeutics, Inc., Conatus Pharmaceuticals Inc., Histogen Inc., Royal Biologics, Ortho Regenerative Technologies, Inc., Swiss Biomed Orthopaedics AG, Osiris Therapeutics, Inc., and Octane Medical Inc.

The market can be segmented into:By Procedure Cell TherapyTissue EngineeringBy Cell TypeInduced Pluripotent Stem Cells (iPSCs)Adult Stem CellsTissue Specific Progenitor Stem Cells (TSPSCs),Mesenchymal Stem Cells (MSCs)Umbilical Cord Stem Cells (UCSCs)Bone Marrow Stem Cells (BMSCs)By SourceBone MarrowUmbilical Cord BloodAdipose TissueAllograftsAmniotic FluidBy ApplicationsTendons RepairCartilage RepairBone RepairLigament RepairSpine RepairOthers

Furthermore, the report acknowledges that in this growing and immediately intensifying market situation, the most recent advertising and marketing details are critical to determining the performance of the forecast period and making an essential choice for the profitability and growth of the Orthopedic Regenerative Medicine market. Do it. Additionally, the report covers various factors influencing the growth of the Orthopedic Regenerative Medicine market during the forecast period. Additionally, this particular analysis will also determine its impact on individual segments of the market.

To identify the growth opportunities in the Orthopedic Regenerative Medicine market, the report has been segmented into regions that are growing faster than the overall market. This region was focused on regions with slower growth rates than the global market. Each geographic segment of the Orthopedic Regenerative Medicine market has been independently investigated, with price, distribution and demand data specifically for North America (US, Canada and Mexico), Europe (Germany, France, UK, Russia and Italy), and Asia-region markets. . Pacific (China, Japan, Korea, India and Southeast Asia), South America (Brazil, Argentina, Colombia, etc.), Middle East and Africa (Saudi Arabia, UAE, Egypt, Nigeria and South Africa).

Research purpose:

Provides strategic profiling of key players in the market, comprehensively analyzes core competencies, and derives the competitive landscape of the market.

Provides insight into factors influencing market growth. Orthopedic Regenerative Medicine market analysis based on various factors such as price analysis, supply chain analysis, Porter Five Force analysis, etc.

It provides detailed analysis of the market structure with forecasts for various segments and sub-segments of the global Orthopedic Regenerative Medicine market.

Provides a country level analysis of the market in relation to its current market size and future outlook.

Provides country-level analysis of the market by application, product type and sub-segment.

Provides historical and forecast revenue for the market segment and sub-segments in relation to the four major regions and countries in North America, Europe, Asia and other countries.

Track and analyze competitive developments such as joint ventures, strategic alliances, new product development and research and development in the global Orthopedic Regenerative Medicine Market.

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The chapters covered in the research report are as follows:

Chapter 1, 2: Targets of the Global Orthopedic Regenerative Medicine Market, encompassing market introduction, product images, market summary, and development scope.

Chapter 3, Chapter 4: Global Market Competition, Sales Volume, and Market Profit by Manufacturer.

Chapters 5, 6, 7: Global Supply (Production), Consumption, Exports, Imports by Regions such as USA, Asia Pacific, China, India, Japan. From 2015 to 2024, we conduct regional market research based on regional sales rate and market share.

Chapters 8, 9, 10: Global Market Analysis by Application, Cost Analysis, Marketing Strategy Analysis, Distributor/Trader

Chapters 11, 12: Market Information and Research Conclusions, Appendix and Data Sources.

The market report also primarily identifies additional useful and useful information about the industry, including the Orthopedic Regenerative Medicine market development trends analysis, return on investment, and feasibility analysis. Additionally, SWOT analysis is distributed in the report to analyze the growth of key global market players in the Orthopedic Regenerative Medicine market industry.

In addition, the research report investigates:

Competitors and manufacturers in the global market

By product type, application and growth factor

Industry status and outlook for major applications / end users / usage areas

Thanks for reading this article. You can also get individual chapter sections, such as North America, Europe, or Asia, or regional versions of the report.

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Impact of Covid-19 On Orthopedic Regenerative Medicine Market Business Overview and Forecast to 2027 | Curasan, Inc., Carmell Therapeutics...

Recommendation and review posted by Bethany Smith

The allogeneic off-the-shelf CD22-directed T-cell product, UCART22, showed early signs of activity and no evidence of unexpected toxicities at 2 dose levels for adult patients with relapsed/refractory CD22-positive B-cell acute lymphoblastic leukemia, according to the results of a study presented during the 2020 ASH Annual Meeting.1

In the phase 1 BALLI-01 (NCT04150497) dose-escalation and dose-expansion study, 2 patients at the 1 x 105 cells/kg dose achieved a complete remission (CR) with incomplete hematologic recovery on day 28. One of these patients attained a minimal residual disease (MRD)positive CR at day 42 followed by subsequent inotuzumab ozogamicin (Besponsa) and then transplant.

One patient at dose level 2, 1 x 106 cells/kg, experienced a significant bone marrow blast reduction at day 28, followed by disease progression.

No patients experienced dose-limiting toxicities (DLTs), immune effector cellassociated neurotoxicity syndrome (ICANS), graft-versus-host disease (GVHD), adverse effects (AE) of special interest (AESI), a UCART22-related AE that was grade 3 or higher, or a serious AE (SAE).

UCART22 showed no unexpected toxicities at the doses of 1 x 105 cells/kg and 1 x 106 cells/kg with fludarabine and cyclophosphamide lymphodepletion, lead study author Nitin Jain, MD, an assistant professor in the Department of Leukemia, The University of Texas MD Anderson Cancer Center, said in a virtual presentation during the meeting. Host immune recovery was observed early, and the addition of alemtuzumab [Lemtrada] to fludarabine and cyclophosphamide lymphodepletion is currently being explored with the goal to achieve deeper and more sustained T-cell depletion and to promote expansion and persistence of UCART22.

Standard treatment for adult patients with B-cell ALL includes multiagent chemotherapy with or without allogeneic stem cell transplant. However, 30% to 60% of patients with newly diagnosed B-cell ALL who achieve a CR will relapse, and the expected 5-year survival rate for those with relapsed/refractory disease is approximately 10%.

Previously, UCART19, when paired with lymphodepletion using fludarabine, cyclophosphamide, and alemtuzumab, was found to show efficacy in this patient population.2

CD22 is an FDA-approved therapeutic target in B-cell ALL. UCART22 is an immediately available, standardized, manufactured agent with the ability to re-dose, and its CAR expression redirects T cells to tumor antigens, Jain explained.

Moreover, through its mechanism of action, TRAC becomes disrupted using Transcription activator-like effector nucleases (Talen) technology to eliminate TCR from cell surface and reduce the risk of GVHD. CD52 is also disrupted with the use of Talen to eliminate sensitivity to lymphodepletion with alemtuzumab. Finally, there is a CD20 mimotope for rituximab (Rituxan) as a safety switch, Jain added.

UCART22 has also demonstrated in vivo antitumor activity in immune-compromised mice that were engrafted with CD22-positive Burkitt lymphoma cells in a dose-dependent manner.

In the dose-escalation/dose-expansion BALLI-01 study, investigators are enrolling up to 30 patients in a modified Toxicity Probability Interval design. There are 3 cohorts, which have 2 to 4 patients on each cohort: 1 x 105 cells/kg (dose level 1), 1 x 106 cells/kg (dose level 2), and 5 x 106 cells/kg. The focus of the dose-escalation phase of the trial was to determine the maximum-tolerated dose (MTD) and the recommended phase 2 dose (RP2D) before heading into the dose-expansion portion of the trial.

To be eligible for enrollment, patients must have been between 18 and 70 years old, have acceptable organ function, an ECOG performance status of 0 or 1, at least 90% of B-cell ALL blast CD22 expression, and had previously received at least 1 standard chemotherapy regimen and at least 1 salvage regimen.

End points of the trial included safety and tolerability, MTD/R2PD, investigator-assessed response, immune reconstitution, and UCART22 expansion and persistence.

The lymphodepletion regimens were comprised of fludarabine (at 30 mg/m2 x 4 days) plus cyclophosphamide (1 g/m2 x 3 days); the study has since been amended to include the regimen of fludarabine (at 30 mg/m2 x 3 days), cyclophosphamide (500 g/m2 x 3 days), and alemtuzumab (20 mg/day x 3 days) and is currently enrolling patients.

Following screening, lymphodepletion, and UCART22 infusion, patients underwent an observation period for DLTs with a primary disease evaluation at 28 days; additional efficacy evaluations occurred at 56 days and 84 days. Patients were followed for 2 years and continued to be assessed for long-term follow-up.

As of July 1, 2020, 7 patients were screened, of which 1 patient failed and 6 were therefore enrolled on the study. One patient discontinued therapy before receiving UCART22 due to hypoxia from pneumonitis that was linked with lymphodepletion. Five patients were treated with UCART22 at dose level 1 (n = 3) and dose level 2 (n = 2).

The median age of participants was 24 years (range, 22-52), 3 of the 5 patients were male, and 3 had an ECOG performance status of 0. The median number of prior therapies was 3 (range, 2-6), and there were a median 35% bone marrow blasts (range, 10%-78%) prior to lymphodepletion.

Three patients had complex karyotype and 2 had diploid cytogenetics. One patient each had the following molecular abnormalities: CRLF2, CRLF2 and JAK2, CDKN2A loss, KRAS and PTPN11, and IKZF1. Only 1 patient had undergone haploidentical transplant. Four patients previously received prior CD19- or CD22-directed therapy, including blinatumomab (Blincyto), inotuzumab ozogamicin (Besponsa), and CD19-directed CAR T-cell therapy. At study entry, 3 patients had refractory disease and 2 patients had relapsed disease.

Grade 3 or higher treatment-emergent AEs (TEAEs), which were unrelated to study treatment, included hypokalemia, anemia, increased bilirubin, and acute hypoxic respiratory failure. Also not related to UCART22, 3 patients experienced 4 treatment-emergent SAEs: porta-hepatis hematoma, sepsis, bleeding, and sepsis in the context of disease progression. No treatment discontinuations due to a treatment-related TEAE were reported.

The patient who achieved a CR followed by transplant was a 22-year-old male who had undergone 2 prior treatments for B-cell ALL and received UCART22 at a dose of 1 x 105 cells/kg. He did not experience CRS, ICANS, GVHD, nor a SAE, and all TEAEs were grade 1.

Jain also noted that host T-cell constitution was observed in all patients within the DLT observation period. UCART22 was also not detectable through flow cytometry or molecular analysis, the latter of which was at dose level 1 only.

References:

1. Jain N, Roboz GJ, Konopleva M, et al. Preliminary results of BALLI-O1: a phase I study of UCART22 (allogeneic engineered T cells expressing anti-CD22 chimeric antigen receptor) in adult patients with relapsed/refractory anti-CD22+ B-cell acute lymphoblastic leukemia (NCT04150497). Presented at: 2020 ASH Annual Meeting and Exposition; December 4-8, 2020; Virtual. Abstract 163.

2. Benjamin R, Graham C, Yallop D, et al. Preliminary data on safety, cellular kinetics and anti-leukemic activity of UCART19, an allogeneic anti-CD19 CAR T-cell product, in a pool of adult and pediatric patients with high-risk CD19+ relapsed/refractory b-cell acute lymphoblastic leukemia. Blood. 2018;132(suppl 1):896. doi:10.1182/blood-2018-99-111356.

Read the original post:
Early Signs of Activity and Tolerability Found in Allogeneic Product UCART22 for Patients with Relapsed/Refractory CD22+ B-Cell ALL - Cancer Network

Recommendation and review posted by Bethany Smith

As interest in chimeric antigen receptor (CAR) T-cell therapy continues to grow with more promising data coming out and approvals from the FDA in various hematologic malignancies, the role of this cellular therapy has yet to be defined in multiple myeloma, but recent data have inspired hope for this therapy in the relapsed/refractory population.

The B-cell maturation antigen (BCMA)directed CAR T-cell therapy idecabtagene vicleucel (ide-cel; bb2121) has generated excitement in this population following the submission of a Biologics License Application (BLA) in March 2020, seeking approval of ide-cel in patients with multiple myeloma who have received at least 3 prior therapies, including an immunomodulatory drug (IMiD), a proteasome inhibitor (PI), and an anti-CD38 antibody, and a Priority Review designation granted in September 2020. Following delays in the review due to coronavirus disease 2019, the Prescription Drug User Fee Act action date has been set as March 27, 2021.

Deep and durable responses were observed with ide-cel as treatment of heavily pretreated patients with relapsed/refractory multiple myeloma, according to updated results from the CRB-401 study presented by Yi Lin, MD, PhD, assistant professor of oncology and associate professor of medicine at Mayo Clinic, during the 2020 American Society of Hematology (ASH) Annual Meeting. The efficacy and safety findings were consistent with prior findings and supported a favorable clinical risk-benefit profile at target dose levels 150 x 106.1

The median overall survival with ide-cel was 34.2 months (95% CI, 19.2-not evaluable) among all patients in this triple-classexposed population, and half of the patients who had ongoing responses achieved a duration of response >2 years. The median progression-free survival (PFS) was 8.8 months (95% CI, 5.9-11.9). The objective response rate (ORR) overall was 75.8%, which included complete responses (CRs) in 38.7%.

These results from CRB-401 are comparable to the findings from the pivotal phase 2 KarMMa study (NCT03361748), which were presented earlier this year during the 2020 American Society of Clinical Oncology (ASCO) Virtual Scientific Program and support the Biologics License Application. The median OS for this study was 19.4 months, and the median PFS was 8.8 months. The ORR was 73%, which included a CR rate of 33%, and the median duration of response was 10.7 months.2

Ide-cel is being explored in several ongoing studies as well, including the phase 2 KarMMa-2 (NCT-3601078), phase 3 KarMMa-3 (NCT03651128), and phase 1 KarMMa-4 (NCT04196491) clinical trials. These phase 2 and 3 studies are evaluating ide-cel in patients with triple-classexposed disease, and the phase 1 study will explore the use of this CAR T-cell therapy in patients with high-risk newly diagnosed multiple myeloma.

These data have also set the stage for other BCMA-directed CAR T-cell therapies in development for the treatment of patients with multiple myeloma.

In an interview with Targeted Oncology, Lin discussed the updated findings from the CRB-401 study of ide-cel as treatment of patients with relapsed/refractory multiple myeloma.

TARGETED ONCOLOGY: What historical data have we seen with BCMA-directed CAR T-cell therapy in patients with relapsed/refractory multiple myeloma?

Lin: With the CAR T approach in multiple myeloma, the very first case report was actually with CD19-targeted CAR T because there was already experience with that particular antigen in leukemia and lymphomas. There's some ongoing effort in terms of dual targeting with CD19 and BCMA, but BCMA very quickly emerged as an ideal candidate for the myeloma space. This is an antigen that is more uniformly expressed on plasma cells, including myeloma cells, and maybe a small subset of mature B cells, but otherwise BCMA is not expressed on healthy tissues.

There have been some single-center clinical trials with the BCMA-targeted CAR T approach prior to the CRB-401 study, both with National Cancer Institute and the University of Pennsylvania with slightly different constructs. With those early phase 1 studies, there was a little bit more toxicity seen, although there was certainly some response, but the response wasn't particularly durable. CRB-401 is the first in a series of now industry-sponsored multicenter studies, in which we are now seeing a much more encouraging durable response rate and also a more favorable side effect profile as well. At ASH this year, I presented the longer follow-up on the phase 1 CRB-401 study. There is a pivotal phase 2 KarMMa study using the same CAR T construct that had been presented at ASCO earlier this year.

TARGETED ONCOLOGY: Please describe the design of the trial and what was different about the study.

Lin: The CRB-401 study has 2 parts. The first part is the dose-escalation part, and the second part is the dose expansion. The dose escalation is basically testing the range of a fixed dose of 50 million all the way up to 800 million of ide-cel CAR T cells in a relatively small number of patients, basically looking for signs of severe side effects to identify a safe dose. The dose expansion cohort is where we take the more promising doses in terms of response, and also safety profile, and test them in more patients to get a better safety signal, which is then moved forward for phase 2 testing in the KarMMa study.

In the dose-expansion portion of CRB-401, we required that each patient must have had exposure to an anti-CD38 antibody. That was allowed in a dose escalation but not required for everybody. [To be included in the study,] the patient must have had become refractory to the most recent line of treatment before they came on the study. The other thing that was different was that in the dose-escalation cohort, all patients had their myeloma cells in the bone marrow reviewed centrally by immunohistochemistry staining, and they were required to have at least 50% of these cells having BCMA expression in a dose-expansion cohort, to better understand the clinical efficacy and safety profiles of this treatment. We also included some patients that had BCMA expression below that to even levels that were not detectable by immunohistochemistry.

TARGETED ONCOLOGY: What were the results from this study?

Lin: The study [included] a total of 62 patients. The results from the first 33 patients were already published in the New England Journal of Medicine last year, and this year at ASH, data were presented for outcomes of the entire 62-patient cohort, with a median follow-up of now 18.1 months. What we have seen so far is in this entire treated patient cohort these are patients with very high-risk features of myeloma, and close to a third of these patients had high-risk cytogenetics, 37% of these patients had extra modularity plasma effect, and almost half of these patients needed some type of systemic therapy while their CAR T cells are being made. These patients, on average, had 6 lines of prior therapy, and in close to 70% or higher, these patients are either triple-refractory or were refractory to the most recent line of therapy.

For this group of patients that was treated overall, the safety signal was very tolerable, which is not surprising with CAR T therapy because these patients also do get lymphodepletion chemotherapy as part of the treatment with CAR T. We do see that low blood count is the most common side effect, including the more severe low blood counts, but on average, the recovery of these blood counts can be seen well under the first 3 months after CAR T infusion. The other most common side effects that we need to watch for with CAR T are cytokine release syndrome (CRS) and neurotoxicity. What we have seen in this study is that, on average, about 76% of these patients had some type of CRS. However, those that had grade 3 or higher, that is only [seen] in 6.5% of the patients, so much lower, and that's also reflected in the relative lower use of tocilizumab and steroids, as well, to manage the side effects. About 35% of these patients had some type of neurologic side effect, but again, only 1 patient had a more severe form of neurotoxicity. Compared to what we have seen with the CAR T experience in the lymphoma/leukemia space, this is a very, very encouraging safety profile.

We have also now seen that the ORR is quite high. It's 75.8% with a CR and stringent CR rate of about 38.7%. Many of these patients that had bone marrow that were evaluable for minimal residual disease (MRD) response were MRD-negative. We are seeing, since we tested many doses, that there is a dose-related increase in response with increasing [the] dose, and we have also seen that the duration of response is 10.3 months. When we look at the dose that was tested as well in those expansions [in] the 150 to 450 range, what we have seen is that the duration of response is comparable, so not significantly decreased, for patients with high-risk features like those with extramedullary disease for older patients, as well as patients who needed to get bridging therapy during treatment. The median PFS is 8.8 months, and the median OS is 34.2 months.

So far, the response rate, duration of response, and PFS seem to be comparable to what we also now see in the KarMMa study, which has less follow-up, but we are seeing a very nice median OS for a treatment in which we're just giving a 1 dose infusion and no follow-up maintenance therapy.

TARGETED ONCOLOGY: In terms of CAR T-cell therapy, how do you see this strategy impacting this patient population in the future?

Lin: I think there's definitely a role for this in the practice. The BLA for ide-cel has been submitted to the FDA, so we're anticipating review sometime in early 2021. This is very exciting because this could very well be the first CAR T for multiple myeloma. I think this would definitely be a treatment option for these patients. Based on how KarMMa is designed, we anticipate that the FDA approval will be in the space of patients who [have] had at least 3 lines of prior therapy and have been exposed to the currently approved 3 main backbones of treatmenta PI, IMiD, and the CD38 antibody. The full detail is pending final FDA review and the label. However, in that space, certainly looking at the demographic of the patient that's been treated so far as CRB-401 and KarMMa, that's a wider group of patients. Based on the fact that this is a treatment that is a basically living active cells, I perceive that the earlier that patient could get this therapy in the earliest possible approved indication, there would likely be potentially more benefit for the patients.

TARGETED ONCOLOGY: Do you think there is hope for this treatment in other hematologic malignancies outside of lymphomas and leukemias as well?

Lin: That is actually a very interesting question because what we're seeing in terms of the severity of CRS and neurotoxicity is a reflection of our evolving learning about how to manage the toxicity, as well. There is a component to the CAR design, the disease, the nature of the disease, the kinetics of the CAR T actions, in the manifestation of these symptoms. What we are seeing now, with even the prior CAR and next-generation CAR coming on, we will likely see an ongoing improvement in terms of a reduction of severity of these symptoms and also in the ways that we could manage these symptoms.

The fact that myeloma would be the next disease that has an FDA-approved CAR T also relates to the fact that the BCMA antigen is more restricted on the cell type where the malignancy is involved, similar to CD19 for lymphoid malignancy. We are seeing that there are some challenges, for example with acute myeloid leukemia or myeloid neoplasms where a number of antigens could overlap with stem cells, which we wouldn't want to try to hurt. There are some novel CAR approaches to try to overcome that, and those are in very early phase testing, so we'll need to see how those results evolve.

References

1. Lin Y, Raje NS, Berdeja JG, et al. Idecabtagene vicleucel (ide-cel, bb2121), a BCMA-directed CAR T cell therapy, in patients with relapsed and refractory multiple myeloma: updated results from phase 1 CRB-401 study. Presented at: 2020 ASH Annual Meeting & Exposition; December 5-8, 2020; Virtual. Abstract 131.

2. Munshi NC, Anderson Jr LD, Jagannath S, et al. Idecabtagene vicleucel (ide-cel; bb2121), a BCMA-targeted CAR T-cell therapy, in patients with relapsed and refractory multiple myeloma (RRMM): Initial KarMMa results.J Clin Oncol. 2020;38(suppl):8503. doi:10.1200/JCO.2020.38.15_suppl.8503

See original here:
Updated Findings Show Continued Efficacy for CAR T-Cell Therapy in Heavily Pretreated Myeloma - Targeted Oncology

Recommendation and review posted by Bethany Smith

Joceline Colvert was diagnosed with relapsing remitting Multiple Sclerosis in her early 20s and says she spent the first eight years researching and managing her condition while trying to mention it as little as possible to others and completing her Sound Production degree.

I spent most of my late 20s and early 30s finding ways to manage relapses, the symptoms of which have included whole body numbness, loss of the use of both hands, right eye blindness, vertigo and double vision, she said. Thankfully these symptoms did resolve however left scarring on my nerves. This results in reduced vision in my formerly blind eye and hands that dont function very well with repetitive tasks.

This semi-denial worked for me until about 2010 when I started to become a bit limpy which I did my best to hide. After a couple of memorable falls and fractures I decided to face up to being slightly rickety and got a hiking pole that I used occasionally in public. Since then Ive needed to get used to being visibly disabled, and switch between two hiking poles for very short distances and a wheelchair everywhere else.

Joceline, who lives with her husband and her five beloved cats and dogs, says she is not eligible for Haematopoietic Stem Cell Transplantation (HSCT), on the NHS which is the first treatment I have ever got excited about and believe could work. It could be truly life-changing.

As a result she is trying to raise money to fund the treatment herself.

HSCT is a procedure that aims to reset the faulty immune system which, in my case, is attacking my nervous system from within, Joceline said. Stem cells will be taken from my bone marrow or blood before my immune system is wiped out with chemotherapy. My cells are then reintroduced into my blood, where they grow a new immune system which will hopefully no longer attack my nerves or have any memory of MS.

The aim of HSCT is to completely halt progression, putting MS into remission with no requirement for immunosuppressant drug therapy. The success rate for relapsing remitting MS is 80% - 90% which is absolutely phenomenal compared to the limited available drug treatments, which only aim to slow down disability.

HSCT is available on the NHS, however there is a very strict criteria for which I do not qualify. The expense of the treatment and the increased pressures on the public purse mean the NHS will only treat patients who have been diagnosed for fewer than 15 years.

I have been diagnosed for 18 years.

I had prepared myself for this possibility and, for the last year, have been researching treatment with The National Pirogov Medical Centre Russia (Moscow). Russia has been pioneering in their use of HSCT to treat MS and are world renowned for their expertise and care. Im excited to have a treatment date in March 2021 which fills me with hope for a future free from progression. I need your help to get there.

Joceline, who loves making stop-motion animation puppets and props and playing musical instruments, says the treatment will cost 40,800, and the flights 800.

She has launched a Go Fund Me page at https://gf.me/u/y538k2 which has already seen donations of more than 26,000.

I am incredibly grateful for any help you can give towards enabling me to access this life-changing treatment, she said.

After almost two decades of managing MS flare-ups and their consequences, its hard to put into words just what a future without them would mean to me.

Thank you for reading this and for any help you can put towards this goal.

Read this article:
Haywards Heath woman's bid to fund stem cell treatment to combat MS - Mid Sussex Times

Recommendation and review posted by Bethany Smith

Dublin, Dec. 09, 2020 (GLOBE NEWSWIRE) -- The "Alopecia Market Share, Growth & Analysis, By Disease, By Application, By Sales Distribution, By Gender And Segment Forecasts, 2016-2026" report has been added to ResearchAndMarkets.com's offering.

The growing prevalence of diseases that trigger alopecia, such as hyperthyroidism, hypopituitarism, lupus, acute stress disorder, and diabetes, are stimulating market growth.

Market Size - USD 9.08 billion in 2019, Market Growth - CAGR of 5.1%, Market Trends - Growing focus on aesthetic appearance and rise in disposable incomes.

The Global Alopecia Market size is expected to reach 13.65 Billion from USD 9.08 Billion in 2019, delivering a CAGR of 5.1% through 2027. The market growth is driven by the growing prevalence of chronic disorders, such as celiac disease, hypothyroidism, hyperthyroidism, Hodgkin's disease, acute stress disorder, hypopituitarism, Hashimoto's disease, lupus, diabetes, Addison's disease, and others, which trigger alopecia in patients.

Changing lifestyle habits, such as overconsumption of alcohol and tobacco and growing stress levels, are resulting in an increased number of alopecia cases globally. Increasing focus on physical appearance and rising disposable incomes are favoring industry growth.

The increasing prevalence of oral treatments, licensed topical treatments, such as minoxidil, finasteride, and surgical procedures, such as hair transplantation or replacement, is expected to boost alopecia market growth over the forecast period.

Further key findings from the report suggest

Key report features:

Key Topics Covered:

Chapter 1. Market Synopsis

Chapter 2. Executive Summary

Chapter 3. Indicative Metrics

Chapter 4. Alopecia Market Segmentation & Impact Analysis

Chapter 5. Alopecia Market By Disease Insights & Trends

Chapter 6. Alopecia Market By Sales distribution Insights & Trends

Chapter 7. Alopecia Market By Gender Insights & Trends

Chapter 8. Alopecia Market By Application Insights & Trends

Chapter 9. Alopecia Market Regional Outlook

Chapter 10. Competitive Landscape

Chapter 11. Company Profiles

For more information about this report visit https://www.researchandmarkets.com/r/jrlv6p

About ResearchAndMarkets.comResearchAndMarkets.com is the world's leading source for international market research reports and market data. We provide you with the latest data on international and regional markets, key industries, the top companies, new products and the latest trends.

Research and Markets also offers Custom Research services providing focused, comprehensive and tailored research.

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Global Alopecia Market is Forecast to Deliver a CAGR of 5.1% Between 2019 and 2027 - GlobeNewswire

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To get sample Copy of the report, along with the TOC, Statistics, and Tables please visit @https://www.databridgemarketresearch.com/request-a-sample/?dbmr=global-induced-pluripotent-stem-cells-market

Some of the key players profiled in the report areFUJIFILM Holdings Corporation, Astellas Pharma Inc, Fate Therapeutics, Bristol-Myers Squibb Company, ViaCyte, Inc., CELGENE CORPORATION, Vericel Corporation, KCI Licensing, Inc, STEMCELL Technologies Inc., Japan Tissue Engineering Co., Ltd., Organogenesis Holdings Inc, Lonza, Takara Bio Inc., Horizon Discovery Group plc, Thermo Fisher Scientific.

Global Induced Pluripotent Stem Cells (iPSCs) Market Scope and Market Size

Induced pluripotent stem cells (iPSCs) market is segmented of the basis of derived cell type, application and end- user. The growth amongst these segments will help you analyse meagre growth segments in the industries, and provide the users with valuable market overview and market insights to help them in making strategic decisions for identification of core market applications.

Key Developments in the Market:

In March 2018, Kaneka Corporation announced that they have acquired a patent in the Japan for the creation of the method to mass-culture pluripotent stem cells including iPS cells and ES cells. This will help the company to use the technology to produce high quality pluripotent stem cells which can be used in the drug and cell therapy.

In March 2015, Fujifilm announced that they have acquired Cellular Dynamics International. The main aim of the acquisition is to expand their business in the iPS cell-based drug discovery support service with the use of CDS technology. It will help them to product high- quality automatic human cells with the help of the induced pluripotent stem cells. This will help the company to be more competitive in the drug discovery and regenerative medicine.

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Global Induced Pluripotent Stem Cells (iPSCs) Market Drivers:

Increasing R&D investment activities is expected to create new opportunity for the market.

Increasing demand for personalized regenerative cell therapies among medical researchers & healthcare is expected to enhance the market growth. Some of the other factors such as increasing cases of chronic diseases, growing awareness among patient, rising funding by government & private sectors and rising number ofclinical trialsis expected to drive the induced pluripotent stem cells (iPSCs) market in the forecast period of 2020 to 2027.

High cost of the induced pluripotent stem cells (iPSCs) and increasing ethical issues & lengthy processes is expected to hamper the market growth in the mentioned forecast period.

Table of Contents:

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About Data Bridge Market Research

An absolute way to forecast what future holds is to comprehend the trend today!

Data Bridge set forth itself as an unconventional and neoteric Market research and consulting firm with unparalleled level of resilience and integrated approaches. We are determined to unearth the best market opportunities and foster efficient information for your business to thrive in the market. Data Bridge endeavors to provide appropriate solutions to the complex business challenges and initiates an effortless decision-making process.

Data bridge is an aftermath of sheer wisdom and experience which was formulated and framed in the year 2015 in Pune. We ponder into the heterogeneous markets in accord with our clients needs and scoop out the best possible solutions and detailed information about the market trends. Data Bridge delve into the markets across Asia, North America, South America, Africa to name few.

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Global Induced Pluripotent Stem Cells (iPSCs) Market Expectable to Exceed Global Market Revenue, Size, Segments and Market Competition Trend to...

Recommendation and review posted by Bethany Smith

Commercial constellations set new standards for the industry in numbers but GEO comsat and government still drive the biggest demand in manufacturing and launch revenues

In its latest analysis of satellite manufacturing and launch services, Satellites to be Built and Launched by 2029, analyst Euroconsult anticipates almost a quintupling in satellite demand in the next decade with an average of 1,250 satellites to be launched on a yearly basis. In comparison to the 260 yearly satellites launched in the previous decade, this skyrocketing number cements the structural changes occurring in the market and the industry, not only in the number of satellites but also in terms of satellite missions and operators, both governmental and commercial.

The satellite industry will indeed experience a quick and radical transformation when it comes to satellite numbers. However, despite this spike in satellite demand, we are looking at half of the market concentrated around a handful of mega-constellations. In addition, some being vertically integrated means that their procurement will not be done on an open competition basis. Nevertheless, GEO comsat remains the leading segment pulling 1/3 of the market revenues, but here too we anticipate -20% drop in operational assets by 2029. argued Maxime Puteaux, Editor-in-Chief of this research product and Principal advisor at Euroconsult.

Several key market trends are catalysing the satellite industrys structural changes.

Firstly, for the first time in a single year more than 1000 satellites were launched, of which 70% from Starlink alone. This symbolic threshold will become a new standard for the next ten years with significant annual variations mainly driven by the replacement of the commercial constellations.

Secondly, the orders of GEO comsat have been exceptionally high in 2020 at 18 units, of which 13 are for the accelerated C-band clearance plan of the FCC in the US. In addition, proof of a structural slowdown of that market in satellite numbers, GEO comsat replacement is also challenged by fleet rationalisation approaches, in-orbit life extension and transitioning of some of the traffic to upcoming NGSO constellations. Manufacturers GEO comsat product portfolios are diversifying, ranging from a few hundred kilograms to 6 Tons VHTS. Digital payloads become the rule for a data-centric market (rather than a broadcasting market).

Thirdly, beyond the commercial space momentum, governments will remain the first customers with 80% satellite manufacturing and launch revenues for the period. Investments by defence operators is driven by security applications and a growing endorsement of smallsats, COTS and constellations while civil agencies focus on large Earth observation systems.

Lastly, access to the space industry is diversifying with a few smallsat-dedicated launchers now operational and more expected to perform maiden flights in 2021. A new generation of GTO-capable launchers is expected to enter the market within the next two years with a design-to-cost approach. Meanwhile, SpaceX masters reusability and executes Starlinks launches at marginal cost, with Falcon 9 recovery and reuse becoming a standard endorsed by customer.

Original post:
Commercial constellations don't live up to the hype: Euroconsult - SatelliteProME.com

Recommendation and review posted by Bethany Smith

The globalAntioxidant Supplement Marketresearch report enlists the vital and practical information with regards to market situation. The present scenario of Antioxidant Supplement market, along with its previous performance as well as future scope are covered in the report. This eases the users understanding of the market thoroughly, while also gaining knowledge about market opportunities and the dominant players Puritan, GNC, Jarrow Formulas, Bulkpowders, NOW, Biocare, Life Extension, Vibrant Health, Cytoplan, AST R-ALA in the Antioxidant Supplement market.

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The Global Antioxidant Supplement Market Research Report Details

The beginning of the report summarizes the market with the definition of the overall Antioxidant Supplement market.

The following section includes Antioxidant Supplement market segmentation Medical Grade, Food Grade. Segmentation is done on the basis of application, type, end-user industries, and several such factors among others.

We have strived to include sub-segments Medical, Food, Cosmetics, Others in segmentation section, wherever possible. Also included are details regarding the dominant segments in the worldwide Antioxidant Supplement market.

The global Antioxidant Supplement market has also been classified on the basis of regions. On the basis of the regional diversification, details regarding market share and size have also been obtained.

In the succeeding part, growth factors for the Antioxidant Supplement market have been elucidated. This section also explains the technological advancements made to improve market size and position. Also enlisted is the information pertaining to the end-use industries for the Antioxidant Supplement market.

Read Detailed Index of full Research Study at::https://www.marketresearchstore.com/report/global-antioxidant-supplement-market-report-2020-industry-analysis-759225

Antioxidant Supplement Market COVID-19 Impact Analysis

As the world is still dealing with COVID-19 situation, many of the countries have slowly started to revive its economic situation by starting its trade and businesses. There has been enormous loss in these few months both in terms of economy and human lives. As the WHO has already suggested that there are very less chances that the virus will completely go, hence we will have start living with it. Many of the drug companies are getting positive response of their COVID-19 vaccines, but there is still time for its availability in the global market.

There are 15 Sections to show the global Antioxidant Supplement market

Sections 1, Definition, Specifications and Classification of Antioxidant Supplement , Applications of Antioxidant Supplement , Market Segment by Regions;Section 2, Assembling Cost Structure, Crude Material and Providers, Assembling Procedure, Industry Chain Structure;Sections 3,Technical Data and Manufacturing Plants Analysis of Antioxidant Supplement , Capacity and Commercial Production Date, Manufacturing Plants Distribution, R&D Status and Technology Source, Raw Materials Sources Analysis;Sections 4, Generally Market Analysis, Limit Examination (Organization Fragment), Sales Examination (Organization Portion), sales Value Investigation (Organization Section);Sections 5 and Six, Regional Market Investigation that incorporates United States, China, Europe, Japan, Korea and Taiwan, Antioxidant Supplement segment Market Examination (by Sort);Sections 7 and Eight, The Antioxidant Supplement Segment Market Analysis (by Application) Major Manufacturers Analysis of Antioxidant Supplement ;Sections Nine, Market Trend Analysis, Regional Market Trend, Market Trend by Product Type Medical Grade, Food Grade Market Trend by Application Medical, Food, Cosmetics, Others;Sections 10, Regional Promoting Type Investigation, Worldwide Exchange Type Examination, Inventory network Investigation;Sections 11, The Customers Examination of global Antioxidant Supplement;Sections 12, Antioxidant Supplement Research Findings and Conclusion, Appendix, system and information source;Sections 13, 14 and 15, Antioxidant Supplement deals channel, wholesalers, merchants, traders, Exploration Discoveries and End, appendix and data source.

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Particulars Of The Global Antioxidant Supplement Market Research Report

Further part in the report enlists the restraining factors for the Antioxidant Supplement market growth. The restraints are explained comprehensively and with details in order that the client can comprehend how these factors are affecting the global Antioxidant Supplement market and how such factors can be tackled effectively using suitable measures.

Also, regional study and analysis of global Antioxidant Supplement market focused on in the report. Here, the major regions with Antioxidant Supplement market establishment have been explained thoroughly. Due to this, our clients will have clarity in understanding the booming markets as well as the potential Antioxidant Supplement markets in the near future.

The concluding section relates to the conclusions and observations regarding the global Antioxidant Supplement market.

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Global Antioxidant Supplement Market Proceeds To Witness Huge Upswing Over Assessment Period by 2025 - The Courier

Recommendation and review posted by Bethany Smith

Three years ago, pictures of bulk carriers queued off the coast of Mackay in central Queensland were framed as evidence of a renaissance in the coal industry.

There were more than 70 coal ships in the offshore gridlock in December 2017. This year there are just 12 waiting equalling a record low mark set at the height of the coronavirus pandemic.

At the worlds biggest coal export port in Newcastle, no China-bound ships are waiting or scheduled to load before Christmas. More than 50 ships carrying Australian coal are reportedly waiting off the Chinese coast.

In the face of falling coal prices and volumes, the industry and governments have remained bullish about coals long-term prospects. They say twin pressures of the pandemic slowdown and Chinas ban on Australian coal will ultimately pass.

In an apparent show of faith, the Queensland government took a 9.9% stake in the float of Dalrymple Bay Infrastructure (DBI) one of two large terminals in Mackay, the main hub for exports from the Bowen Basin. The investment was welcomed by the resources sector as a clear vote of confidence ... in the role of resources in Queenslands Covid-19 recovery and economic growth for decades to come.

DBI was launched on the Australian stock exchange this week with government backing and the broader market surging. It was the second biggest Australian IPO this year (the largest, tech company Nuix, gained 63% on debut last week). DBI also promised investors a handsome 7% dividend.

When the stocks hit the market, DBI tanked, down 16%. It gained no ground the following day. Investors were not buying the pitch that coal has a rosy future.

Its a pivotal moment, says Tim Buckley, an energy markets expert at the Institute of Energy Economics and Financial Analysis.

The financial markets do move so much faster than the real world, they are all about constantly reevaluating the risk-return and growth prospects.

Theres no long-term growth prospect at all for the [coal] industry. Its like trying to catch a falling knife.

Australias biggest super fund, AustralianSuper, has committed to hitting net zero emissions across its $200bn investment portfolio by 2050 but has not specifically ruled out investing in coal projects.

Nonetheless, its chief executive, Ian Silk, seems less than enthusiastic about the idea.

The economic outlook for coal stocks generally is incredibly bleak, for obvious reasons, he says.

He says Aussie would approach any particular coal project on its financial merits.

But its pretty plain by the way were so underweight coal that thats not an attractive sector, he says.

Silk is not alone. Institutional investors the big pension funds and other piles of money that provide much of the capital businesses need to operate have increasingly turned away from coal and other fossil fuels.

Norways Government Pension Fund Global, which at US$1.2tn is so big it holds about 1.5% of all the shares in listed companies in the world, has strict rules forbidding it from investing in companies that produce more than 20m tonnes of thermal coal a year or produce power of more than 10,000MW a year from burning coal.

As a result, in May it excluded from investment two big multinationals that mine coal in Australia, Glencore and Anglo American, as well as Australian power company AGL Energy.

It also put BHP on notice that it could dump its stake in the Big Australian if it didnt get out of thermal coal.

In Australia, the big banks have displayed an increasing unwillingness to lend to coal, with ANZ in October saying it would not write new loans to businesses with more than 10% exposure to thermal coal and existing customers with more than 50% exposure would need to show it specific, time bound and public diversification strategies to continue receiving the banks cash.

The harder line from banks followed warnings from their regulator, the Australian Prudential Regulation Authority, that they needed to consider climate risks when making decisions.

Apra member Geoff Summerhayes laid down the regulators position in a speech in 2017 that was met with howls of dismay and derision by some.

This week, he said the criticism was good impetus for me to actually go harder, because its very much a financial risk with real prudential implications.

It is easy to blame the failure of Australian coal prospects to re-emerge from the pandemic on the situation in China. However, there are growing signals the industry is heading into its final bust cycle.

A few days before the Dalrymple Bay terminal was floated, the largest coal producer in Australia and the western world, Glencore, released its annual investor update and critically announced new plans for a managed decline of its coal business and net-zero emissions by 2050.

While those plans are ultimately long-term (and also play to the companys strategic interest by seeking to keep prices at viable levels by constraining supply) they also show the company expects volumes to drop substantially up to 20% in the next few years, compared to previous projections.

The same investor update last year envisaged Glencore would produce 140m tonnes of coal in 2022. Now the company only expects to mine 115m tonnes that year. It might consider mine-life extension projects but has no plans to develop new coal mines.

Coal is also on the nose at Australias two big mining companies, BHP and Rio Tinto, which have turned away from the black rock and towards red ones as the iron ore price continues to soar.

Rio Tinto sold its last Australian coal mine in 2018 and, under pressure from investors, BHP has promised to get out of thermal coal burned in power plants within two years but so far has found no buyers.

Of particular concern to miners in Queensland is the way financial markets have treated metallurgical (or coking) coal, which is used in steelmaking. More than 80% of the exports from Dalrymple Bay are metallurgical coal.

In the days before the DBI float, company chief executive Anthony Timbrell told the Australian Financial Review it would seek to emphasise the difference between metallurgical and thermal (energy producing) coal.

I guess its our job to draw out that story and remind people of the complexity, Timbrell said.

Thermal coal is the primary target of environmental activists; while metallurgical coal is less susceptible in the immediate-term to a global energy pivot towards renewables.

BHP has also been keen to draw the distinction, which is in its financial interests as metallurgical coal attracts a higher price than thermal coal.

However, as excitement builds around the prospect of (as yet, not commercialised) steelmaking alternatives like green hydrogen, the financial markets increasingly appear to be making little differentiation between the classes of coal.

Buckley points to a graph comparing the US and Australian metallurgical coal producer Coronado with Australian company Whitehaven, which largely mines thermal coal.

Since Coronado was listed in 2018, both Coronado and Whitehavens shares have dived almost in harmony by about 65%. The All Ordinaries is up about 20% over the same period.

The financial markets are no longer really differentiating between coking coal and thermal coal, Buckley said.

Dalrymple Bay is a really interesting bellwether for Queensland. Having already been priced down, having failed to get institutional support, taxpayers effectively did a bailout.

The vendor (Brookfield Asset Management) is the most successful investor in energy infrastructure, and you dont buy from the most successful energy investor in the world and think youre getting a bargain.

This isnt a resources sector problem either, this is a coal problem. The Australian resource sector is having the best year in history, iron ore prices are at phenomenal highs. Its the fossil fuel sector thats on its knees.

The coal company run by John Canavan, the brother of Queensland senator Matt Canavan, went under earlier this month.

The company, ICRA Rolleston, is a junior joint venture partner with Glencore in the Rolleston thermal coal mine in Queensland. Glencore will continue to operate the mine but a court case finalised last month showed how the collapse in the coal price had turned the mine into a loss-making venture.

John Canavans share of the mines costs were about $14m more than sales revenue in August. Glencore expected another $4m shortfall by the end of the year.

The Queensland Exploration Council (an offshoot of the Resources Council) this week released a report card showing some growth in spending by coal speculators during 2019-20 and said there was definitely a feeling of growth and optimism in the sector. In the detail of its report, though, for the first time in four years the QEC downgraded its view on coal prices, saying these has become cause for concern.

In its prospectus for potential investors, DBI warned about a series of risks that included its customers collapsing due to low coal prices, or long-term decline in global coal demand.

Its most significant new investor, the Queensland government, released a study in September that stated there is a substantial degree of uncertainty about assumptions used to underpin long-term market projections, including about the price of coking coal.

Queensland Treasurys analysis highlights that Queenslands future coal demand will continue to be primarily linked to key economies in north-east and south-east Asia. In particular, the future demand for Queenslands metallurgical coal likely hinges on demand from the worlds two largest coal consumers, China and India.

DBIs warnings included that ongoing political tensions between Australia and China could ultimately result in a decline in coal exports from the port.

Demand for metallurgical coal or coal generally may reduce over a period of time due to a variety of reasons, including reduced demand from key coal export markets, such as China, Japan, Taiwan, South Korean and India.

In addition to Chinas import restrictions, China, Japan and South Korea Australias three largest coal customers each announced aggressive pivots towards net zero emissions this year.

Adding to DBIs troubles is the nature of its business where contracts with exporters are regulated by a competition authority. The ports capacity is fully allocated. Unable to raise prices or attract new customers, its pitch to investors has been about expanding its capacity to grow the business, even as shipments are being shunned by China, export volumes contract, coal companies collapse and other Queensland ports face severe debt problems.

Of those terminals, Wiggins Island at Gladstone and the Abbot Point terminal near Bowen, owned by Adani, have both been operating at well below design capacity for all of this decade, a point Buckley and others say shows any expansion of Dalrymple Bay is not viable.

Abbot Point where Adanis debts are estimated in excess of $1.5bn typically has a queue of about three coal ships. Earlier this week, there were no ships waiting to enter the port.

Link:
The end of coal? Why investors aren't buying the myth of the industry's 'renaissance' - The Guardian

Recommendation and review posted by Bethany Smith

Researchers in California aim to develop a new CRISPR-based test for COVID-19 that can be read anytime, anywhereby turning a basic smartphone camera into a microscope capable of detecting the coronavirus genetic material.

The team consists of scientists from the University of California, San Francisco, UC Berkeley, and the Gladstone Institutesincluding a collaboration with Jennifer Doudna, president of the Innovative Genomics Institute, and winner of the 2020 Nobel Prize in Chemistry for co-discovering CRISPR-Cas genome editing, the technology that underpins the test.

"It has been an urgent task for the scientific community to not only increase testingbut also to provide new testing options," said Melanie Ott, director of the Gladstone Institute of Virology and one of the leaders of a study evaluating the test, published in Cell. "The assay we developed could provide rapid, low-cost testing to help control the spread of COVID-19."

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The smartphone diagnostic aims to provide a positive or negative result in less than 30 minutes, as well as gauge the amounts of SARS-CoV-2 virus present in a nasal swab sample.

"When coupled with repeated testing, measuring viral load could help determine whether an infection is increasing or decreasing," said UC Berkeley bioengineer Daniel Fletcher, a Chan Zuckerberg Biohub investigator. "Monitoring the course of a patient's infection could help health care professionals estimate the stage of infection and predict, in real time, how long is likely needed for recovery."

RELATED: Jennifer Doudna's new CRISPR company will tackle disease detection

Most current molecular tests for COVID-19 are based on PCRa method that requires the virus RNA to be converted into DNA before the diagnostics can work. It also needs to amplify that DNA, by repeatedly making copies to capture a detectable signal, which calls for specialized chemical reagents and laboratory equipment.

Alternatively, the researchers approach uses CRISPR proteins designed to hunt directly for the virus RNA, skipping the conversion and amplification steps.

"One reason we're excited about CRISPR-based diagnostics is the potential for quick, accurate results at the point of need," said Doudna, whose CRISPR-focused company Mammoth Biosciences was tapped earlier this year by GlaxoSmithKline to develop an over-the-counter COVID-19 test.

"This is especially helpful in places with limited access to testing, or when frequent, rapid testing is needed. It could eliminate a lot of the bottlenecks we've seen with COVID-19," she said.

The new test uses a Cas13 protein tagged with a molecule that glows once its cut, as part of the genetic-snipping that occurs when it matches up with a specific piece of RNA. When more of the virus genome is present, more cuts occurcreating a brighter glow that can be picked up by a smartphone camera used with a darkened box.

RELATED: Stanford develops CRISPR 'lab on a chip' for detecting COVID-19

The team of researchers had originally been pursuing the quick testing method as a potential diagnostic for HIV, but pivoted to the coronavirus as the pandemic began to spread this year.

"We knew the assay we were developing would be a logical fit to help the crisis by allowing rapid testing with minimal resources," co-first author Parinaz Fozouni, a UCSF graduate student working in Ott's lab at Gladstone. "Instead of the well-known CRISPR protein called Cas9, which recognizes and cleaves DNA, we used Cas13, which cleaves RNA."

The study also found that samples with high concentrations of the virus produced a signal much faster, with positive results for someone who potentially more likely to be contagious delivered in under 5 minutes.

"Recent models of SARS-CoV-2 suggest that frequent testing with a fast turnaround time is what we need to overcome the current pandemic," said Ott. "We hope that with increased testing, we can avoid lockdowns and protect the most vulnerable populations."

In addition to being widely available and cheaper compared to lab equipment, smartphones could also make use of their GPS and digital connectivity to help track the spread of infections in various regions.

"We hope to develop our test into a device that could instantly upload results into cloud-based systems while maintaining patient privacy, which would be important for contact tracing and epidemiologic studies," Ott said. "This type of smartphone-based diagnostic test could play a crucial role in controlling the current and future pandemics."

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Pairing CRISPR with a smartphone camera, this COVID-19 test finds results in 30 minutes - FierceBiotech

Recommendation and review posted by Bethany Smith

Identifying and isolating individuals who may be contagious with the coronavirus is key to limiting the spread of the disease. But even months into the pandemic, many patients are still waiting days to receive COVID-19 test results.

Scientists at UC Berkeley and Gladstone Institutes have developeda new CRISPR-based COVID-19 diagnostic testthat, with the help of a smartphone camera, can provide a positive or negative result in 15 to 30 minutes. Unlike many other tests that are available, this test also gives an estimate of viral load, or the number of virus particles in a sample, which can help doctors monitor the progression of a COVID-19 infection and estimate how contagious a patient might be.

Monitoring the course of a patients infection could help health care professionals estimate the stage of infection and predict, in real time, how long is likely needed for recovery and how long the individual should quarantine, said Daniel Fletcher, a professor of bioengineering at Berkeley and one of the leaders of the study.

The technique was designed in collaboration with Dr. Melanie Ott, director of the Gladstone Institute of Virology, as well as Berkeley professor Jennifer Doudna, who is a senior investigator at Gladstone, president of the Innovative Genomics Institute and a Howard Hughes Medical Institute investigator. Doudna recently won the 2020 Nobel Prize in Chemistry for co-discovering CRISPR-Cas genome editing, the technology that underlies this work.

Most COVID-19 diagnostic tests rely on a method called PCR, short for polymerase chain reaction, which searches for pieces of the SARS-CoV-2 viral RNA in a sample. These PCR tests work by first isolating the viral RNA, then converting the RNA into DNA and then amplifying the DNA segments making many identical copies so that the segments can be more easily detected.

The new diagnostic test takes advantage of the CRISPR Cas13 protein, which directly binds and cleaves RNA segments. This eliminates the DNA conversion and amplification steps and greatly reduces the time needed to complete the analysis.

One reason were excited about CRISPR-based diagnostics is the potential for quick, accurate results at the point of need, Doudna said. This is especially helpful in places with limited access to testing or when frequent, rapid testing is needed. It could eliminate a lot of the bottlenecks weve seen with COVID-19.

In the test, CRISPR Cas13 proteins are programmed to recognize segments of SARS-CoV-2 viral RNA and then combined with a probe that becomes fluorescent when cleaved. When the Cas13 proteins are activated by the viral RNA, they start to cleave the fluorescent probe. With the help of a handheld device, the resulting fluorescence can be measured by the smartphone camera. The rate at which the fluorescence becomes brighter is related to the number of virus particles in the sample.

Recent models of SARS-CoV-2 suggest that frequent testing with a fast turnaround time is what we need to overcome the current pandemic, Ott said. We hope that with increased testing, we can begin to reopen economies and protect the most vulnerable populations.

Now that the CRISPR-based assay has been developed for SARS-CoV-2, it could be modified to detect RNA segments of other viral diseases, like the common cold, influenza or even human immunodeficiency virus. The team is currently working to package the test into a device that could be made available at clinics and other point-of-care settings and that one day could even be used in the home.

The eventual goal is to have a personal device, like a mobile phone, that is able to detect a range of different viral infections and quickly determine whether you have a common cold or SARS-Cov-2 or influenza, Fletcher said. That possibility now exists, and further collaboration between engineers, biologists and clinicians is needed to make that a reality.

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New CRISPR-based COVID-19 test uses smartphone cameras to spot virus RNA - University of California

Recommendation and review posted by Bethany Smith

A solid clinical trial update convinced several Wall Street analysts to raise their price targets for CRISPR Therapeutics (NASDAQ:CRSP) on Thursday. Those new expectations include a bold $170 target from investment bank Needham -- about 16% higher than the biotech's closing price Wednesday.

The candidate generating all this enthusiasm is CTX001, an experimental therapy for two hemoglobin-based disorders: beta-thalassemia and sickle cell disease. CRISPR Therapeutics is developing it in partnership with Vertex Pharmaceuticals (NASDAQ:VRTX). This single-application treatment involves reengineering a patient's own stem cells to produce fetal hemoglobin once they've been reinfused, and it appears to work as intended.

Image source: Getty Images.

At the latest update, seven beta-thalassemia patients had been under post-therapy observation for least three months, and so far, none have required transfusions. Among three patients with sickle cell disease who also have been observed for at least three months, none have experienced a vaso-occlusive crisis since undergoing the therapy.

CRISPR Therapeutics has also administered CTX001 to 10 patients who hadn't had their three-month observations in time for their data to be included in the company's recent presentation. In the summer of 2021, though, we should know if CTX001 has a shot at competing against LentiGlobin, an experimental treatment from bluebird bio (NASDAQ:BLUE).

LentiGlobin aims to treat the same limited groups of patients as CTX001, and it's been in development longer. Recently, bluebird bio published results from BCL11A, a treatment that more closely resembles CTX001, and the early data looks competitive.

There is a good chance that CTX001 and experimental cancer treatments rolling through CRISPR Therapeutics' pipeline will drive the stock to $170 and beyond. However, based on the potential for competition from bluebird bio, and considering the usual issues that clinical-stage biotechs must navigate, the company faces significant risks. Investors who buy this stock should only do so within a well-diversified portfolio.

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Is CRISPR Therapeutics Heading for $170? - The Motley Fool

Recommendation and review posted by Bethany Smith

Demonstrates modularity of Intellias in vivo liver insertion technology to durably restore protein, compared to traditional gene therapy

Single-course administration of genome editing system provides potentially curative approach to AAT deficiency

CAMBRIDGE, Mass., Dec. 12, 2020 (GLOBE NEWSWIRE) -- Intellia Therapeutics, Inc. (NASDAQ:NTLA), is presenting the first demonstration of physiological protein levels of human alpha-1 antitrypsin (AAT) in non-human primates (NHPs) following a single administration. Compared to traditional adeno-associated virus (AAV) gene therapy, Intellias targeted liver gene insertion technology has the ability to achieve therapeutic levels of protein expression, in a stable and durable manner, after a single course of treatment. The company is presenting these data today at the Alpha-1 Foundations 20th Gordon L. Snider Critical Issues Workshop: The Promise of Gene-Based Interventions of Alpha-1 Antitrypsin Deficiency.

Our new data reinforce the promise for Intellia to potentially cure a variety of rare genetic diseases requiring the restoration of a functional protein in the liver with a single-course therapy, said Intellia President and Chief Executive Officer John Leonard, M.D. Weve now demonstrated our platforms modularity and translatability to multiple targets of interest by inserting genes to durably produce unprecedented levels of protein in NHPs for hemophilia B and AAT deficiency. In parallel with advancing to the clinic treatments for other severe diseases, we will continue preclinical studies that further validate our wholly owned, CRISPR-based AAT deficiency treatment strategies for achieving normal AAT protein levels.

Presentation Details

Title: CRISPR/Cas9-Mediated Targeted Gene Insertion of SERPINA1 to Treat Alpha-1 Antitrypsin DeficiencySession: Gene EditingTime: 3:15 p.m. ETPresenting Author: Sean Burns, M.D., senior director of Intellias Disease Biology and Pharmacology group

Intellia is advancing multiple genome editing strategies that may treat both lung and liver manifestations of AAT deficiency (AATD), which occur due to mutations in the SERPINA1 gene. The normal human AAT protein levels Intellia achieved following targeted insertion of the human SERPINA1 gene remained stable through 11 weeks in an ongoing NHP study. The observed levels of human AAT protein produced from the liver may be therapeutically sufficient to restore protease inhibition to protect the lungs and liver from improperly regulated neutrophil elastase activity. The NHP data build on previous resultsshowing that consecutive in vivo genome editing (knockout plus insertion) achieved therapeutically relevant results in an AATD mouse model.

The findings being presented today reinforce recent data showing the use of the same proprietary insertion technology for targeted gene insertion ofFactor 9 resulted in circulating human Factor IX, a blood-clotting protein that is missing or defective in hemophilia B patients, that ranged from normal levels (50-150%)1 to supratherapeutic levels in a six-week NHP study. Intellia and Regeneron, the lead party, are co-developing potential hemophilia A and B CRISPR/Cas9-based treatments using their jointly developed targeted transgene insertion capabilities. Intellia is continuing to develop its proprietary platform to advance its wholly owned research programs, such as AATD. Click here to register for the Alpha-1 Foundations virtual workshop and here to view Intellias presentation on the companys website.

____________________________1 National Hemophilia Foundation

About Alpha-1 Antitrypsin Deficiency and Intellias Genome Editing Treatment Approach

The SERPINA1 gene normally encodes the alpha-1 antitrypsin (AAT) protein produced in the liver that is then secreted to protect the lungs. SERPINA1 mutations can cause AAT deficiency (AATD), a rare, genetic disease that commonly manifests in lung dysfunction, as well as in liver disease in some patients. Intellias targeted in vivo insertion platform uses a hybrid delivery system combining a non-viral lipid nanoparticle (LNP), which encapsulates CRISPR/Cas9 components, with an adeno-associated virus (AAV) carrying a donor DNA template to enable therapeutic protein production. One of the editing strategies Intellia is studying as a potential single-course AATD treatment is using the companys SERPINA1 gene insertion approach to restore normal human AAT protein levels. Intellia also is investigating a consecutive genome editing approach, in which the PiZ allele, the most prevalent disease-causing mutation of SERPINA1, is knocked out and the normal human SERPINA1 gene is inserted.

AboutIntellia TherapeuticsIntellia Therapeuticsis a leading genome editing company, focused on the development of proprietary, potentially curative therapeutics using the CRISPR/Cas9 system. Intellia believes the CRISPR/Cas9 technology has the potential to transform medicine by both producing therapeutics that permanently edit and/or correct disease-associated genes in the human body with a single treatment course, and creating enhanced engineered cells that can treat oncological and immunological diseases. Intellias combination of deep scientific, technical and clinical development experience, along with its leading intellectual property portfolio, puts it in a unique position to unlock broad therapeutic applications of the CRISPR/Cas9 technology and create new classes of therapeutic products. Learn more aboutIntellia and CRISPR/Cas9 atintelliatx.com. Follow us on Twitter@intelliatweets.

Forward-Looking Statements This press release contains forward-looking statements of Intellia Therapeutics, Inc. (Intellia or the Company) within the meaning of the Private Securities Litigation Reform Act of 1995. These forward-looking statements include, but are not limited to, express or implied statements regarding Intellias beliefs and expectations regarding its: plans to advance and complete preclinical studies, including any necessary non-human primate studies, for its hemophilia A, hemophilia B, and other in vivo and ex vivo research and development programs, such as its AATD research program; development of a proprietary LNP/AAV hybrid delivery system, as well as its modular platform to advance its complex genome editing capabilities, such as gene insertion, as well as knockout editing capabilities; advancement and expansion of its CRISPR/Cas9 technology to develop human therapeutic products, as well as its ability to maintain and expand its related intellectual property portfolio; ability to demonstrate its platforms modularity and replicate or apply results achieved in preclinical studies, including those in its hemophilia A and hemophilia B programs and its AATD research program, in any future studies, including human clinical trials; ability to develop other in vivo or ex vivo cell therapeutics of all types using CRISPR/Cas9 technology; expectations of the potential impact of the coronavirus disease 2019 pandemic on strategy, future operations and timing of its clinical trials or IND submissions; ability to optimize the impact of its collaborations on its development programs, including but not limited to its collaborations with Regeneron, including its co-development programs for hemophilia A and hemophilia B; statements regarding the timing of regulatory filings regarding its development programs; use of capital, expenses, future accumulated deficit and other 2020 financial results or in the future; and ability to fund operations at least through the next 24 months.

Any forward-looking statements in this press release are based on managements current expectations and beliefs of future events, and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to: risks related to Intellias ability to protect and maintain its intellectual property position; risks related to Intellias relationship with third parties, including its licensors and licensees; risks related to the ability of its licensors to protect and maintain their intellectual property position; uncertainties related to the authorization, initiation and conduct of studies and other development requirements for its product candidates; the risk that any one or more of Intellias product candidates will not be successfully developed and commercialized; the risk that the results of preclinical studies or clinical studies will not be predictive of future results in connection with future studies; and the risk that Intellias collaborations with Regeneron or its other collaborations will not continue or will not be successful. For a discussion of these and other risks and uncertainties, and other important factors, any of which could cause Intellias actual results to differ from those contained in the forward-looking statements, see the section entitled Risk Factors in Intellias most recent annual report on Form 10-K as well as discussions of potential risks, uncertainties, and other important factors in Intellias other filings with the Securities and Exchange Commission. All information in this press release is as of the date of the release, and Intellia undertakes no duty to update this information unless required by law.

Intellia Contacts:

Media:Lynnea OlivarezDirectorExternal Affairs & Communications+1 956-330-1917lynnea.olivarez@intelliatx.com

Investors:Lina LiAssociate DirectorInvestor Relations+1 857-706-1612lina.li@intelliatx.com

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Intellia Therapeutics Achieves Normal Human Alpha-1 Antitrypsin Protein Levels in Non-Human Primates Through Targeted Gene Insertion for the Treatment...

Recommendation and review posted by Bethany Smith

Called de-extinction, the resurrection of lost species is one of the many applications to be revolutionized by the new gene-editing technology CRISPR-Cas9.

[A]ll scientists need are organic remnantssuch as pieces of bonethat contain fragments of DNA. Those fragments allow geneticists to discover the complete genome of the extinct animal (a process scientists refer to as sequencing). Once they have this recipe for the extinct species, CRISPR enables scientists to edit the DNA of its closest living relative to create a genome that, as edited, approximates the genetic code of the extinct species. Think of the living animals DNA as version 2.0 of a piece of software: the goal is to get back to version 1.0. You compare all of the millions of lines of code to spot differences, and then painstakingly edit the lines with differences to restore the code to its original state.

Once the DNA has been edited to reintroduce the key traits of the extinct plant or animal, the edited DNA is inserted into the nucleus of a reproducing cell. The resulting individual may not be genetically identical to the extinct species, but the key traits that made the extinct species unique are reintroduced.

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De-extinction: Why CRISPR gene editing might be the most revolutionary development in science ever - Genetic Literacy Project

Recommendation and review posted by Bethany Smith

Bacteria readily acquires a sequence of other species DNA into their own, in specific areas that we now call CRISPR. In the lab, CRISPR was synthesized by linking together two guide RNA sequences into a format that would provide the target information and allow us to edit multiple genes simultaneously.

Cancer is a genetic disease, it works by creating certain changes to genes that control the way our cells function, especially how they grow and divide. Some rare cancers, sarcomas in particular, have been treated using CRISPR, which is why the gene-editing tool seems like a good diagnostic and therapeutic tool in the future of cancer treatments.

Obtaining rare cancerous tumors for research is difficult, but luckily organizations such as Pattern.org and the Rare Cancer Research Foundation (RCRF) come into play. These sister groups perform a matching program that enables patients to directly donate their tumor tissue and medical data to research. All the data generated by the project is freely available to the research community and is dedicated to open science.

Using Pattern.org, the Broad Institute of MIT and Harvard has created over 40 next-generation de-identified cancer models, Ms. Barbara Van Hare, Director of Foundation partnerships at RCRF said, These models and associated data will be shared within the worldwide research community.

After procuring these rare disease samples, Dr. Jesse Boehm from Eli and Edythe L. Broad Institute might have the answer to decipher the genetic landscape of cancer cells and use that to our advantage. Dr. Boehm is the scientific director of the Broad Institutes Cancer Dependency Map Initiative where he works on the cancer cell line factory project and the cancer dependency map.

Cancer samples are broken apart into cell models and are coaxed into growing in different conditions over a year-long time period. The data from these new cell models are then shared broadly with the world. This is a pipeline activity called the cell line factory. It is a part of an international effort to create a large reference data set, that is called the cancer dependency map.

The cancer dependency map has a two-pronged approach, first by testing cell lines against drugs and then pooled CRISPR screening. First, all cell lines are systematically tested against all drugs developed for any disease. Some known drugs have shown to be effective against certain cancers, clinical trials are swift as these are existing therapies.

There are 20,000 proteins in the human genome and only 6000 drug therapies. Only five percent of human genes can be targeted with drugs. The cancer dependency Map is completed with the help of CRISPR, Dr Boehm said.

Pooled CRISPR screening is used and 100,000 CRISPRs target every gene in the genome. Every cell is challenged with all these CRISPRs and at the end of the experiment, the abundance is compared to the beginning of the experiment.

CRISPR is used to snip genes,the DNA repairs creating a broken gene. Cells that are required for viability die and drop out of the population. CRISPRs are bar coded, so if by the end of the experiment the CRISPR is absent, it targets the gene that the cell needed to survive. The genes that drop out are good drug targets, most of these make way for drug discovery projects right away.

CRISPR is such a sharp tool, it inspires a lot more confidence than its predecessors, Dr Boehm said. He uses the analogy of Google Maps for this project: It needs to tell clinicians what to do and where to go, but for it to be relevant-the data needs to be dense enough in that area.

An additional therapy for cancers involves making four genetic modifications to T cells (immune cells that can kill cancer). It basically adds genes to T cells to fight cancer. One of these is a synthetic gene that gives the T cells a protein that can identify cancer cells better. CRISPR is also used to mute three genes that limit the cells cancer-killing abilities (Stadtmauer et al. 2020). With these limiting genes removed, the T cells are less inhibited to fight cancer.

These therapeutics and the Cancer dependency map will take a few decades to develop but will prove to be a very sharp tool in our arsenal against rare cancers when complete.

References:

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How CRISPR could help us discover and treat rare cancers - ZME Science

Recommendation and review posted by Bethany Smith

THURSDAY, Dec. 10, 2020 (HealthDay News) -- Use of the ex vivo CRISPR-Cas9-based gene-editing platform to edit the erythroid enhancer region of BCL11A in hematopoietic stem and progenitor cells, producing CTX001, results in increased hemoglobin (Hb) among patients with transfusion-dependent -thalassemia (TDT) and sickle cell disease (SCD), according to a study published online Dec. 5 in the New England Journal of Medicine to coincide with the annual meeting of the American Society of Hematology, held virtually from Dec. 5 to 8.

Haydar Frangoul, M.D., from the Sarah Cannon Center for Blood Cancer at the Children's Hospital at TriStar Centennial in Nashville, Tennessee, and colleagues presented safety and efficacy results from patients with at least three months of follow-up from two first-in-human studies of CTX001 for TDT and SCD. Data were included for seven patients with TDT and three with SCD.

The researchers found increases in total Hb and fetal Hb among all patients over time. Patients with TDT stopped receiving packed red blood cell transfusions soon after CTX001 infusion; the first patients with TDT who received CTX001 remained transfusion-free for more than 15 months. Since CTX001 infusion, the patients with SCD have had no vaso-occlusive crises (VOCs); the first patient to receive CTX001 remained VOC-free for more than one year. The safety profile after CTX001 infusion was generally consistent with busulfan myeloablation in all 10 patients. One patient with TDT had four serious adverse events related or possibly related to CTX001.

"By gene editing the patient's own stem cells we may have the potential to make this therapy an option for many patients facing these blood diseases," Frangoul said in a statement.

Several authors disclosed financial ties to pharmaceutical companies, including CRISPR Therapeutics and Vertex Pharmaceuticals, which sponsored the trial.

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ASH: CRISPR-Cas9 Gene Editing Promising in TDT, SCD - HealthDay News

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