Back in the pre-pandemic era, I was really looking forward to April 8. On that date,Carl Zimmerwas going to give a talk at my school, Stevens Institute of Technology, about his latest book,She Has Her Mothers Laugh. For decades, Zimmer has reported on biology in The New York Times and other publications and in books,13 so far.Mothers Laughtells the epic tale of our attempts to plumb the mysteries of heredity and to improve ourselves with that knowledge. The book is a marvelous work of historyZimmers account of the early days of eugenics in the U.S. is especially grippingas well as a detailed, up-to-date report on CRISPR and other advances that add urgency to old debates about human enhancement. Zimmer is an engaging story-teller and insatiable reporter, who visits scientists in their labs and even volunteers to be a subject. As a result, while discussing the remarkable diversity of creatures dwelling on and in our bodies, he can tell you that his own bellybutton harbors a bacterium,Marimonas, also found in the Mariana Trench. In lieu of Carls April 8 talk, here he answers questions about genetics and related topics. John Horgan

Horgan:How did you end up in the science-writing racket, anyway? Any regrets?

Zimmer:I feel incredibly lucky to have this job. It wasn't anything I thought about with any foresight. I loved to write, and I loved science. A couple years out of college, I got a job as an assistant copy editor at the science magazineDiscover. There, I got a great training in how to fact-check and report on science. I stayed there for ten years before heading out on my own.

Horgan:Why the focus on biology? When you started out, wasnt physics going to solve everything?

Zimmer:As a junior reporter atDiscover, I had to write about all sorts of stuff--astronomy, geoscience, physics, technology, and so on. But I found that biology was always the field that managed to surprise me the most. Evolution has gone off in such crazy directions in the past four billion years, and the tools biologists have to study life have grown incredibly powerful over the past few decades.

Horgan:I sometimes worry Im too mean to scientists. Do you ever worry youre too nice?

Zimmer:As a fact-checker, you learn that no one should be given a pass. When I report on a story, I talk with outside experts to see if researchers I'm writing about are really delivering on what they claim. And it's also important to keep up with what social scientists and philosophers have to say--because science doesn't happen in a vacuum and can have dangerous consequences.

Horgan:Whats the biggest thing thats happened in science since you started writing about it?

Zimmer:DNA sequencing. It changed everything, from the study of Neanderthals to tracking the covid-19 pandemic.

Horgan:In 2009you quit the online chat show Bloggingheads.tv, on whichwe once spoke, because it gave a platform to creationists. Have your feelings about creationism evolved over the past decade?

Zimmer:No. Creationists have not done any good science since then, while evolutionary biology has leapt forward in dramatic fashion.

Horgan:Whenever I criticize scientificracism, orsexism, people call me an unscientific social justice warrior. I knowthis happens to you, too. How do you deal with these people?

Zimmer:People try to deflect from weak arguments by accusing their opponents of being contemptible.

Horgan:Is CRISPR living up to its hype? If so, will it help gene therapy, finally, take off?

Zimmer:CRISPR is already a mainstay of scientific research, for testing how genes work and how mutations affect health. It's already into clinical trials for diseases like sickle cell anemia just few years after its invention. We have yet to see how well it will work in those applications. But it's unquestionably one of the most important advances in the history of biology.

Horgan:By the time I reached the end ofShe Has Her Mothers Laugh, I wasnt sure whether you think genetic enhancement of humans is feasible, or desirable. Could you clarify?

Zimmer:I think anyone who pretends to have a simple answer is wrong. The answer depends not only on the complexity of biology, but also on what we really want from genetic enhancement. We are already carrying out genetic enhancement when parents with Huntington's disease pick embryos for IVF without the mutation. But I'm skeptical that any manipulation will affect, say, intelligence--certainly not more than what a decent education and a healthy childhood can offer.

Horgan:Will there be any more revolutions in our understanding of heredity?

Zimmer:It's not possible to predict revolutions that haven't happened. But I think that scientists will learn a lot about how epigenetic changes can be carried down through generations--if not in humans, then in other animals and plants.

Horgan:Will our knowledge ever be so complete that the nature/debate finally ends?

Zimmer:I can't rule it out, but it won't be easy. It's relatively easy to study how genes influence variation, but the environment is so vast and complex it may not submit to simple experiments with clear results. Still, there are some very impressive experiments that are grappling with these challenges.

Horgan:Are radical life extension, and possibly immortality, feasible?

Zimmer:I'm not holding my breath. Aging is the result of so many factors that it's hard to see how any simple intervention can change it much. Immortality just seems biologically silly to me.

Horgan:I cant resist asking: what do you think of the U.S. response to the coronavirus?

Zimmer:A disaster.

Further Reading:

Was Darwin Wrong?

How Can We Curb the Spread of Scientific Racism?

Should Research on Race and IQ Be Banned?

My Problem with Taboo Behavioral Genetics? The Science Stinks!

Quest for Intelligence Genes Turns Out More Dubious Results

Have Researchers Really Discovered Any Genes for Behavior?

Defending Stephen Jay Goulds Crusade Against Biological Determinism

Darwin Was Sexist, and So Are Many Modern Scientists

Do Women Want to be Oppressed?

Google Engineer Fired for Sexist Memo Isnt a Hero

See also my free, online bookMind-Body Problems: Science, Subjectivity & Who We Really Are, also available as a Kindle e-book and paperback.

Go here to read the rest:
Will the Nature/Nurture Debate Ever End? - Scientific American

Recommendation and review posted by Bethany Smith

Global Cryonics Technology Market 2020 to 2026, is a comprehensive report which provides a detailed overview of the major driver, opportunities, challenges, current market trends and strategies impacting the global Cryonics Technology market in conjunction with calculation and forecast of size, share, and growth rate analysis. Combining the analysis capabilities and knowledge integration with the relevant findings, the report has foretold the robust future growth of the Cryonics Technology market all told its geographical and merchandise segments.

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Key Players of the Global Cryonics Technology Market

Praxair, Cellulis, Cryologics, Cryotherm, KrioRus, VWR, Thermo Fisher Scientific, Custom Biogenic Systems, Oregon Cryonics, Alcor Life Extension Foundation, Osiris Cryonics, Sigma-Aldrich, Southern Cryonics.

Segmentation by product type

Slow freezingVitrificationUltra-rapid

Segmentation by application

Animal husbandryFishery scienceMedical sciencePreservation of microbiology cultureConserving plant biodiversity

Market Segment by Regions, regional analysis covers 2019-2025:

North America(United States, Canada and Mexico)Europe(Germany, France, UK, Russia and Italy)Asia-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)

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

Competition by Company:Here, the competition in the global Cryonics Technology Market is analyzed, taking into consideration price, revenue, sales, and market share by company, market concentration rate, competitive situations and trends, expansion, merger and acquisition, and market shares of top 5 and 10 companies.

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

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

Application or End User:This part of the research study shows how different application segments contribute to the global Cryonics Technology Market.

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

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

Appendix:Here, we have provided a disclaimer, our data sources, data triangulation, market breakdown, research programs and design, and our research approach.

The research includes historic data from 2015 to 2020 and forecasts until 2026 which makes the reports an invaluable resource for industry executives, marketing, sales and product managers, consultants, analysts, and other people looking for key industry data in readily accessible documents with clearly presented tables and graphs.

We also offer customization on reports based on specific client requirement

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Excerpt from:
Research Report, Growth Trends and Competitive Analysis 2020-2026 Cole Reports - Cole of Duty

Recommendation and review posted by Bethany Smith

The Department of Defense has begun to ratchet up spending to recapitalize the U.S. strategic nuclear triad and its supporting infrastructure, as several programs move from research and development into the procurement phase. The projected Pentagon expenditures are at least $167 billion from 2021-2025. This amount does not include the large nuclear warhead sustainment and modernization costs funded by the Department of Energy, projected to cost $81 billion over the next five years.

Nuclear forces require modernization, but that will entail opportunity costs.In a budget environment that offers little prospect of greater defense spending, especially in the COVID19 era, more money for nuclear forces will mean less funding for conventional capabilities.

That has potentially negative consequences for the security of the United States and its allies.While nuclear forces provide day-to-day deterrence, the Pentagon leadership spends most of its time thinking about how to employ conventional forces to manage security challenges around the world.The renewed focus on great power competition further elevates the importance of conventional forces.It is important to get the balance between nuclear and conventional forces right, particularly as the most likely path to use of nuclear arms would be an escalation of a conventional conflict.Having robust conventional forces to prevail in or deter a conventional conflict in the first place could avert a nuclear crisis or worse.

Nuclear Weapons and Budgets

For the foreseeable future, the United States will continue to rely on nuclear deterrence for its security and that of its allies (whether we should be comfortable with that prospect is another question).Many U.S. nuclear weapons systems are aging, and replacing them will cost money, lots of money.The Pentagons five-year plan for its nuclear weapons programs proposes $29 billion in fiscal year 2021, rising to $38 billion in fiscal year 2025, as programs move from research and development to procurement.The plan envisages a total of $167 billion over five years.And that total may be understated; weapons costs increase not just as they move to the procurement phase, but as cost overruns and other issues drive the costs up compared to earlier projections.

The Pentagon knew that the procurement bow wave of nuclear weapons spending would hit in the 2020s and that funding it would pose a challenge.In October 2015, the principal deputy undersecretary of defense said Were looking at that big bow wave and wondering how the heck were going to pay for it and probably thanking our stars that we wont be here to have to answer the question.

The Pentagons funding request for fiscal year 2021 includes $4.4 billion for the new Columbia-class ballistic missile submarine that will replace Ohio-class submarines, which will begin to be retired at the end of the decade; $1.2 billion for the life extension program for the Trident II submarine-launched ballistic missile (SLBM); $1.5 billion for the Ground Based Strategic Deterrent, an intercontinental ballistic missile (ICBM) to replace the Minuteman III ICBM; $2.8 billion for the B-21 stealth bomber that will replace the B-1 and B-2 bombers; $500 million for the Long-Range Standoff Missile that will arm B-52 and B-21 bombers; and $7 billion for nuclear command, control and communications systems.

The Pentagon funds primarily go to delivery and command and control systems for nuclear weapons.The National Nuclear Security Administration at the Department of Energy bears the costs of the warheads themselves. It seeks $15.6 billion for five nuclear warhead life-extension and other infrastructure programs in fiscal year 2021, the first year of a five-year plan totaling $81 billion.The fiscal year 2021 request is nearly $3 billion more than the agency had earlier planned to ask, which suggests these programs are encountering significant cost growth.

Some look at these figures and the overall defense budget (the Pentagon wants a total of $740 billion for fiscal year 2021) and calculate that the cost of building and operating U.S. nuclear forces will amount to only 6-7 percent of the defense budget.That may be true, but how relevant is that figure?

By one estimate, the cost of building and operating the F-35 fighter program for the U.S. Air Force, U.S. Navy and U.S. Marines over the programs lifetime will be $1 trillion.Amortized over 50 years, that amounts to $20 billion per year or only 2.7 percent of the Defense Departments fiscal year 2021 budget request.The problem is that these percentages and lots of other small percentages add up.When one includes all of the programs, plus personnel and readiness costs as well as everything else that the Pentagon wants, the percentages will total to more than 100 percent of the figure that Congress is prepared to appropriate for defense.

Opportunity Costs

The defense budget is unlikely to grow.Opportunity costs represent the things the Pentagon has to give up or forgo in order to fund its nuclear weapons programs.The military services gave an indication of these costs with their unfunded priorities lists, which this year total $18 billion.These show what the services would like to buy if they had additional funds, and that includes a lot of conventional weapons.

The Air Force, for example, would like to procure an additional twelve F-35 fighters as well as fund advance procurement for an additional twelve F-35s in fiscal year 2022.It would also like to buy three more tanker aircraft than budgeted.

The Army is reorienting from counter-insurgency operations in places such as Afghanistan and Iraq to facing off against major peer competitors, that is, Russia and China.Its wish list includes more long-range precision fires (artillery and short-range surface-to-surface missiles), a new combat vehicle, helicopters and more air and missile defense systems.

The Navy would like to add five F-35s to its aircraft buy, but its bigger desire is more attack submarines and warships, given its target of building up to a fleet of 355 ships. The Navy termed a second Virginia-class attack submarine its top unfunded priority in fiscal year 2021.It has set a requirement for 66 attack submarines and currently has about 50.However, as older Los Angeles-class submarines retire, that number could fall to 42. Forgoing construction of a Virginia-class submarine does not help to close that gap.

Moreover, the total number of Navy ships, now 293, will decline in the near term, widening the gap to get to 355.The Navys five-year shipbuilding program cut five of twelve planned Arleigh Burke-class destroyers, and cost considerations have led the Navy to decide to retire ten older Burke-class destroyers rather than extend their service life for an additional ten years.This comes when China is rapidly expanding its navy, and Russian attack submarines are returning on a more regular cycle to the Atlantic Ocean.

The Navy has said that funding the first Columbia-class ballistic missile submarine forced a cut-back in the number of other ships in its fiscal 2021 shipbuilding request.The decision not to fund a second Virginia-class attack submarine appears to stem directly from the unexpected $3 billion plus-up in funding for the National Nuclear Security Administrations fiscal year 2021 programs.

These are the opportunity costs of more nuclear weapons:fewer dollars for aircraft, ships, attack submarines and ground combat equipment for conventional deterrence and defense.

Nuclear War and Deterring Conventional Conflict

The principal driving factor behind the size of U.S. nuclear forces comes from Russian nuclear forces and doctrine.Diverse and effective U.S. nuclear forces that can deter a Russian nuclear attack should suffice to deter a nuclear attack by any third country.In contrast to the Cold War, the U.S. military no longer seems to worry much about a bolt from the bluea sudden Soviet or Russian first strike involving a massive number of nuclear weapons designed to destroy the bulk of U.S. strategic forces before they could launch.That is because, under any conceivable scenario, sufficient U.S. strategic forcesprincipally on ballistic missile submarines at seawould survive to inflict a devastating retaliatory response.

The most likely scenario for nuclear use between the United States and Russia is a regional conflict fought at the conventional level in which one side begins to lose and decides to escalate by employing a small number of low-yield nuclear weapons, seeking to reverse battlefield losses and signal the strength of its resolve.Questions thus have arisen about whether Russia has an escalate-to-deescalate doctrine and whether the 2018 U.S. nuclear posture review lowers the threshold for use of nuclear weapons.

If the United States and its allies have sufficiently robust conventional forces, they can prevail in a regional conflict at the conventional level and push any decision about first use of nuclear weapons onto the other side (Russia, or perhaps China or North Korea depending on the scenario).The other side would have to weigh carefully the likelihood that its first use of nuclear weapons would trigger a nuclear response, opening the decidedly grim prospect of further nuclear escalation and of things spinning out of control.The other sides leader might calculate that he/she could control the escalation, but that gamble would come with no guarantee. It would appear a poor bet given the enormous consequences if things go wrong.Happily, the test has never been run.

This is why the opportunity costs of nuclear weapons programs matter.If those programs strip too much funding from conventional forces, they weaken the ability of the United States and its allies to prevail in a conventional conflictor to deter that conflict in the first placeand increase the possibility that the United States might have to employ nuclear weapons to avert defeat.

Read the original:
Weapons, Opportunity Costs, COVID19 and Avoiding Nuclear War - The National Interest

Recommendation and review posted by Bethany Smith

Adeles significant slim down has been credited to a combination of exercise and eating sirtfoods. So what is the sirt diet and could it work for you?

COVID-19 may still be daily overwhelming the internet, but for a few hours last week all the talk on social mediturned to just how singer Adele could havelost so much weight.

And the answer? Apparently (allegedly) shes been following the sirtfood diet, detailed in a book of the same name written by Aidan Goggins and Glen Matten.

The sirt in sirtfood stands for sirtuins a group of genes and the proteins they encode that play a key role in cellular homeostasis. Sirtuins have been implicated in the stress response, DNA repair and energy metabolism, but the area where they've whipped up more scientific interest (and hype) than any other isin relation to extending lifespan.

The finding that sirtuins were linked to longevity in yeast led to an explosion of research that has yielded ambiguous results. There have been some signs of life extension and improved long-term health, including an anti-diabetic potential in laboratory animals, but nothing thats been reliably shown to extend life in mammals.

The central tenet of the sirtfood diet is that eating certain polyphenol-rich foods red wine and dark chocolate are frequently citedas examples to make the diet seem sexier can stimulate sirtuins (coined your skinny genes by the authors) and create weight loss.

Activating sirtuins through diet is an intriguing theory, but theres little evidence to support it, and even less evidence that doing so would lead to significant weight loss in practice.

The diet promises a weight loss of 7 pounds in 7 days and that longer-term you will improve your resistance to disease while gaining incredible energy and glowing health.

If you dont gnaw off your arm in hunger before you get slim that is.

Phase one of the diet is three miserable days of consuming just 1000 calories including three green juices, followed by four more days when youre permitted 1500 calories but still have to gag on drink green juice.

Phase two lasts for two weeks and isn't a whole lot more appealing, while longer-term you have to continue consuming the same list of foods repeatedly.

Polyphenol-rich food such as apples and citrus, green leafy vegetables, berries and olive oil are all perfectly healthy but having to focus on them at every meal is faddish and restrictive.

And thats the real reason the sirt diet works for those that do stick to it despite sounding seductively sciencey the restraints mean its just another not-particularly-well-disguised route to cutting calories.

If it works for you and it worked for Adele, then great. But lets not pretend sirt diet success stories are down to anything other than eating less.

Excerpt from:
Eat Like a Bird and Weight Loss Will Be a Dead Sirt - American Council on Science and Health

Recommendation and review posted by Bethany Smith

The exploration drive approval allows for development within a nominated corridor as it is recognised that the exact location will vary as the mineralisation is further identified.

Alkane Resources Limited () has received approval from the NSW Government Resources Regulator to develop an exploration drive from theexisting Wyoming One underground operations at Tomingley Gold Project to Roswell and San Antonio deposits, around four kilometres to the south.

The company believes this is an important milestone towards a future potential underground mine at Roswell and San Antonio that could be developed independently of an open cut operation.

While being an exploration drive, itwill be sized such that it could also accommodate production in the future.

Alkane managing director Nic Earner said: Alkane has the equipment purchased, personnel recruited and land acquired to allow this development to proceed and we will now incorporate the timing of the drive development into the Tomingley Gold Operations budget schedules, as well as our regional exploration plans.

Given the prospectivity of the region around our existing operations, this approval provides Alkane with all the exploration and, in time, production options, to extend the life of mine at Tomingley well into the future.

The Tomingley Gold Project (TGP) in Central West NSW contains Alkanes Tomingley Gold Operations (TGO), an open pit mine with a 1 million tonnes per annum processing facility that is transitioning to underground.

Over the past two years, Alkane has conducted an extensive regional exploration program around the TGP with the objective of defining additional resources that have the potential to be mined either via open pit or underground operations and fed to the TGO processing facility.

Thisprogram has yielded broad, shallow, high-grade intercepts that demonstrate potential for material project life extension in excess of 10 years, and show that a return to open pit mining and/or underground extension is possible with appropriate resource confirmation, landholder agreement and regulatory approvals.

The drive is conceptual in nature to allow visualisation of the potential development.

At both the Roswell and San Antonio prospects, inferred resources have been defined of 7.02 million tonnes at 1.97 g/t gold for 445,000 ounces and 7.92 million tonnes at 1.78 g/t for 453,000 ounces respectively.

The resources are now being prepared into conceptual mine plans that can be used to progress NSW Government mining approvals.

Development of the underground exploration drive will allowdrilling positions, bulk-samplingand assessment of the corridor between the existing operations, as the Roswell and San Antonio deposits are not as readily accessible from surface.

Visit link:
Alkane Resources gets green light for underground exploration drive to reach new gold deposits - Proactive Investors Australia

Recommendation and review posted by Bethany Smith

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

Key companies operating in the global Reishi Mushroom Supplements market include ,Natures Way Reishi,Solaray Reishi Mushroom,Life Extension Reishi Extract,Host Defense Reishi,Terrasoul Superfoods,Swanson Reishi Mushroom,Aloha Medicinals,Mushroom Science,Planetary Herbals

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Segmental Analysis

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

Global Reishi Mushroom Supplements Market Segment By Type:

,Organic Reishi Mushroom Supplements,Inorganic Reishi Mushroom Supplements Reishi Mushroom Supplements

Global Reishi Mushroom Supplements Market Segment By Application:

,Online Sales,Offline Sales

Competitive Landscape

It is important for every market participant to be familiar with the competitive scenario in the global Reishi Mushroom Supplements industry. In order to fulfil the requirements, the industry analysts have evaluated the strategic activities of the competitors to help the key players strengthen their foothold in the market and increase their competitiveness.

Key companies operating in the global Reishi Mushroom Supplements market include ,Natures Way Reishi,Solaray Reishi Mushroom,Life Extension Reishi Extract,Host Defense Reishi,Terrasoul Superfoods,Swanson Reishi Mushroom,Aloha Medicinals,Mushroom Science,Planetary Herbals

Key questions answered in the report:

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TOC

1 Study Coverage1.1 Reishi Mushroom Supplements Product Introduction1.2 Market Segments1.3 Key Reishi Mushroom Supplements Manufacturers Covered: Ranking by Revenue1.4 Market by Type1.4.1 Global Reishi Mushroom Supplements Market Size Growth Rate by Type1.4.2 Organic Reishi Mushroom Supplements1.4.3 Inorganic Reishi Mushroom Supplements1.5 Market by Application1.5.1 Global Reishi Mushroom Supplements Market Size Growth Rate by Application1.5.2 Online Sales1.5.3 Offline Sales1.6 Coronavirus Disease 2019 (Covid-19): Reishi Mushroom Supplements Industry Impact1.6.1 How the Covid-19 is Affecting the Reishi Mushroom Supplements Industry

1.6.1.1 Reishi Mushroom Supplements Business Impact Assessment Covid-19

1.6.1.2 Supply Chain Challenges

1.6.1.3 COVID-19s Impact On Crude Oil and Refined Products1.6.2 Market Trends and Reishi Mushroom Supplements Potential Opportunities in the COVID-19 Landscape1.6.3 Measures / Proposal against Covid-19

1.6.3.1 Government Measures to Combat Covid-19 Impact

1.6.3.2 Proposal for Reishi Mushroom Supplements Players to Combat Covid-19 Impact1.7 Study Objectives1.8 Years Considered 2 Executive Summary2.1 Global Reishi Mushroom Supplements Market Size Estimates and Forecasts2.1.1 Global Reishi Mushroom Supplements Revenue 2015-20262.1.2 Global Reishi Mushroom Supplements Sales 2015-20262.2 Reishi Mushroom Supplements Market Size by Region: 2020 Versus 20262.2.1 Global Reishi Mushroom Supplements Retrospective Market Scenario in Sales by Region: 2015-20202.2.2 Global Reishi Mushroom Supplements Retrospective Market Scenario in Revenue by Region: 2015-2020 3 Global Reishi Mushroom Supplements Competitor Landscape by Players3.1 Reishi Mushroom Supplements Sales by Manufacturers3.1.1 Reishi Mushroom Supplements Sales by Manufacturers (2015-2020)3.1.2 Reishi Mushroom Supplements Sales Market Share by Manufacturers (2015-2020)3.2 Reishi Mushroom Supplements Revenue by Manufacturers3.2.1 Reishi Mushroom Supplements Revenue by Manufacturers (2015-2020)3.2.2 Reishi Mushroom Supplements Revenue Share by Manufacturers (2015-2020)3.2.3 Global Reishi Mushroom Supplements Market Concentration Ratio (CR5 and HHI) (2015-2020)3.2.4 Global Top 10 and Top 5 Companies by Reishi Mushroom Supplements Revenue in 20193.2.5 Global Reishi Mushroom Supplements Market Share by Company Type (Tier 1, Tier 2 and Tier 3)3.3 Reishi Mushroom Supplements Price by Manufacturers3.4 Reishi Mushroom Supplements Manufacturing Base Distribution, Product Types3.4.1 Reishi Mushroom Supplements Manufacturers Manufacturing Base Distribution, Headquarters3.4.2 Manufacturers Reishi Mushroom Supplements Product Type3.4.3 Date of International Manufacturers Enter into Reishi Mushroom Supplements Market3.5 Manufacturers Mergers & Acquisitions, Expansion Plans 4 Breakdown Data by Type (2015-2026)4.1 Global Reishi Mushroom Supplements Market Size by Type (2015-2020)4.1.1 Global Reishi Mushroom Supplements Sales by Type (2015-2020)4.1.2 Global Reishi Mushroom Supplements Revenue by Type (2015-2020)4.1.3 Reishi Mushroom Supplements Average Selling Price (ASP) by Type (2015-2026)4.2 Global Reishi Mushroom Supplements Market Size Forecast by Type (2021-2026)4.2.1 Global Reishi Mushroom Supplements Sales Forecast by Type (2021-2026)4.2.2 Global Reishi Mushroom Supplements Revenue Forecast by Type (2021-2026)4.2.3 Reishi Mushroom Supplements Average Selling Price (ASP) Forecast by Type (2021-2026)4.3 Global Reishi Mushroom Supplements Market Share by Price Tier (2015-2020): Low-End, Mid-Range and High-End 5 Breakdown Data by Application (2015-2026)5.1 Global Reishi Mushroom Supplements Market Size by Application (2015-2020)5.1.1 Global Reishi Mushroom Supplements Sales by Application (2015-2020)5.1.2 Global Reishi Mushroom Supplements Revenue by Application (2015-2020)5.1.3 Reishi Mushroom Supplements Price by Application (2015-2020)5.2 Reishi Mushroom Supplements Market Size Forecast by Application (2021-2026)5.2.1 Global Reishi Mushroom Supplements Sales Forecast by Application (2021-2026)5.2.2 Global Reishi Mushroom Supplements Revenue Forecast by Application (2021-2026)5.2.3 Global Reishi Mushroom Supplements Price Forecast by Application (2021-2026) 6 North America6.1 North America Reishi Mushroom Supplements by Country6.1.1 North America Reishi Mushroom Supplements Sales by Country6.1.2 North America Reishi Mushroom Supplements Revenue by Country6.1.3 U.S.6.1.4 Canada6.2 North America Reishi Mushroom Supplements Market Facts & Figures by Type6.3 North America Reishi Mushroom Supplements Market Facts & Figures by Application 7 Europe7.1 Europe Reishi Mushroom Supplements by Country7.1.1 Europe Reishi Mushroom Supplements Sales by Country7.1.2 Europe Reishi Mushroom Supplements Revenue by Country7.1.3 Germany7.1.4 France7.1.5 U.K.7.1.6 Italy7.1.7 Russia7.2 Europe Reishi Mushroom Supplements Market Facts & Figures by Type7.3 Europe Reishi Mushroom Supplements Market Facts & Figures by Application 8 Asia Pacific8.1 Asia Pacific Reishi Mushroom Supplements by Region8.1.1 Asia Pacific Reishi Mushroom Supplements Sales by Region8.1.2 Asia Pacific Reishi Mushroom Supplements Revenue by Region8.1.3 China8.1.4 Japan8.1.5 South Korea8.1.6 India8.1.7 Australia8.1.8 Taiwan8.1.9 Indonesia8.1.10 Thailand8.1.11 Malaysia8.1.12 Philippines8.1.13 Vietnam8.2 Asia Pacific Reishi Mushroom Supplements Market Facts & Figures by Type8.3 Asia Pacific Reishi Mushroom Supplements Market Facts & Figures by Application 9 Latin America9.1 Latin America Reishi Mushroom Supplements by Country9.1.1 Latin America Reishi Mushroom Supplements Sales by Country9.1.2 Latin America Reishi Mushroom Supplements Revenue by Country9.1.3 Mexico9.1.4 Brazil9.1.5 Argentina9.2 Central & South America Reishi Mushroom Supplements Market Facts & Figures by Type9.3 Central & South America Reishi Mushroom Supplements Market Facts & Figures by Application 10 Middle East and Africa10.1 Middle East and Africa Reishi Mushroom Supplements by Country10.1.1 Middle East and Africa Reishi Mushroom Supplements Sales by Country10.1.2 Middle East and Africa Reishi Mushroom Supplements Revenue by Country10.1.3 Turkey10.1.4 Saudi Arabia10.1.5 U.A.E10.2 Middle East and Africa Reishi Mushroom Supplements Market Facts & Figures by Type10.3 Middle East and Africa Reishi Mushroom Supplements Market Facts & Figures by Application 11 Company Profiles11.1 Natures Way Reishi11.1.1 Natures Way Reishi Corporation Information11.1.2 Natures Way Reishi Description, Business Overview and Total Revenue11.1.3 Natures Way Reishi Sales, Revenue and Gross Margin (2015-2020)11.1.4 Natures Way Reishi Reishi Mushroom Supplements Products Offered11.1.5 Natures Way Reishi Recent Development11.2 Solaray Reishi Mushroom11.2.1 Solaray Reishi Mushroom Corporation Information11.2.2 Solaray Reishi Mushroom Description, Business Overview and Total Revenue11.2.3 Solaray Reishi Mushroom Sales, Revenue and Gross Margin (2015-2020)11.2.4 Solaray Reishi Mushroom Reishi Mushroom Supplements Products Offered11.2.5 Solaray Reishi Mushroom Recent Development11.3 Life Extension Reishi Extract11.3.1 Life Extension Reishi Extract Corporation Information11.3.2 Life Extension Reishi Extract Description, Business Overview and Total Revenue11.3.3 Life Extension Reishi Extract Sales, Revenue and Gross Margin (2015-2020)11.3.4 Life Extension Reishi Extract Reishi Mushroom Supplements Products Offered11.3.5 Life Extension Reishi Extract Recent Development11.4 Host Defense Reishi11.4.1 Host Defense Reishi Corporation Information11.4.2 Host Defense Reishi Description, Business Overview and Total Revenue11.4.3 Host Defense Reishi Sales, Revenue and Gross Margin (2015-2020)11.4.4 Host Defense Reishi Reishi Mushroom Supplements Products Offered11.4.5 Host Defense Reishi Recent Development11.5 Terrasoul Superfoods11.5.1 Terrasoul Superfoods Corporation Information11.5.2 Terrasoul Superfoods Description, Business Overview and Total Revenue11.5.3 Terrasoul Superfoods Sales, Revenue and Gross Margin (2015-2020)11.5.4 Terrasoul Superfoods Reishi Mushroom Supplements Products Offered11.5.5 Terrasoul Superfoods Recent Development11.6 Swanson Reishi Mushroom11.6.1 Swanson Reishi Mushroom Corporation Information11.6.2 Swanson Reishi Mushroom Description, Business Overview and Total Revenue11.6.3 Swanson Reishi Mushroom Sales, Revenue and Gross Margin (2015-2020)11.6.4 Swanson Reishi Mushroom Reishi Mushroom Supplements Products Offered11.6.5 Swanson Reishi Mushroom Recent Development11.7 Aloha Medicinals11.7.1 Aloha Medicinals Corporation Information11.7.2 Aloha Medicinals Description, Business Overview and Total Revenue11.7.3 Aloha Medicinals Sales, Revenue and Gross Margin (2015-2020)11.7.4 Aloha Medicinals Reishi Mushroom Supplements Products Offered11.7.5 Aloha Medicinals Recent Development11.8 Mushroom Science11.8.1 Mushroom Science Corporation Information11.8.2 Mushroom Science Description, Business Overview and Total Revenue11.8.3 Mushroom Science Sales, Revenue and Gross Margin (2015-2020)11.8.4 Mushroom Science Reishi Mushroom Supplements Products Offered11.8.5 Mushroom Science Recent Development11.9 Planetary Herbals11.9.1 Planetary Herbals Corporation Information11.9.2 Planetary Herbals Description, Business Overview and Total Revenue11.9.3 Planetary Herbals Sales, Revenue and Gross Margin (2015-2020)11.9.4 Planetary Herbals Reishi Mushroom Supplements Products Offered11.9.5 Planetary Herbals Recent Development11.1 Natures Way Reishi11.1.1 Natures Way Reishi Corporation Information11.1.2 Natures Way Reishi Description, Business Overview and Total Revenue11.1.3 Natures Way Reishi Sales, Revenue and Gross Margin (2015-2020)11.1.4 Natures Way Reishi Reishi Mushroom Supplements Products Offered11.1.5 Natures Way Reishi Recent Development 12 Future Forecast by Regions (Countries) (2021-2026)12.1 Reishi Mushroom Supplements Market Estimates and Projections by Region12.1.1 Global Reishi Mushroom Supplements Sales Forecast by Regions 2021-202612.1.2 Global Reishi Mushroom Supplements Revenue Forecast by Regions 2021-202612.2 North America Reishi Mushroom Supplements Market Size Forecast (2021-2026)12.2.1 North America: Reishi Mushroom Supplements Sales Forecast (2021-2026)12.2.2 North America: Reishi Mushroom Supplements Revenue Forecast (2021-2026)12.2.3 North America: Reishi Mushroom Supplements Market Size Forecast by Country (2021-2026)12.3 Europe Reishi Mushroom Supplements Market Size Forecast (2021-2026)12.3.1 Europe: Reishi Mushroom Supplements Sales Forecast (2021-2026)12.3.2 Europe: Reishi Mushroom Supplements Revenue Forecast (2021-2026)12.3.3 Europe: Reishi Mushroom Supplements Market Size Forecast by Country (2021-2026)12.4 Asia Pacific Reishi Mushroom Supplements Market Size Forecast (2021-2026)12.4.1 Asia Pacific: Reishi Mushroom Supplements Sales Forecast (2021-2026)12.4.2 Asia Pacific: Reishi Mushroom Supplements Revenue Forecast (2021-2026)12.4.3 Asia Pacific: Reishi Mushroom Supplements Market Size Forecast by Region (2021-2026)12.5 Latin America Reishi Mushroom Supplements Market Size Forecast (2021-2026)12.5.1 Latin America: Reishi Mushroom Supplements Sales Forecast (2021-2026)12.5.2 Latin America: Reishi Mushroom Supplements Revenue Forecast (2021-2026)12.5.3 Latin America: Reishi Mushroom Supplements Market Size Forecast by Country (2021-2026)12.6 Middle East and Africa Reishi Mushroom Supplements Market Size Forecast (2021-2026)12.6.1 Middle East and Africa: Reishi Mushroom Supplements Sales Forecast (2021-2026)12.6.2 Middle East and Africa: Reishi Mushroom Supplements Revenue Forecast (2021-2026)12.6.3 Middle East and Africa: Reishi Mushroom Supplements Market Size Forecast by Country (2021-2026) 13 Market Opportunities, Challenges, Risks and Influences Factors Analysis13.1 Market Opportunities and Drivers13.2 Market Challenges13.3 Market Risks/Restraints13.4 Porters Five Forces Analysis13.5 Primary Interviews with Key Reishi Mushroom Supplements Players (Opinion Leaders) 14 Value Chain and Sales Channels Analysis14.1 Value Chain Analysis14.2 Reishi Mushroom Supplements Customers14.3 Sales Channels Analysis14.3.1 Sales Channels14.3.2 Distributors 15 Research Findings and Conclusion 16 Appendix16.1 Research Methodology16.1.1 Methodology/Research Approach16.1.2 Data Source16.2 Author Details

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Reishi Mushroom Supplements Market Research Report: Probable Key Development To Be Observed Market States And Outlook Across By 2026 - Weekly Wall

Recommendation and review posted by Bethany Smith

Several accidents at the power station reveal serious issues in maintenance, safety systems

In the past five years, two major accidents and a minor accident occurred at the Neyveli Lignite Thermal Power Station (NLTPS) a lignite-based thermal power station located near the lignite mines of Neyveli in Tamil Nadus Cuddalore district.

Another accident at the plant owned by Neyveli Lignite Corporation (NLC), an undertaking of the Union government revealed serious issues in maintenance and safety systems in the old units running at NLTPS, for which the senior management should be held responsible.

NLC has a history of management issues. There have been frequent strikes by workers over several issues over the years. These strikes may be one of the reasons for poor operational practices at NLTPS.

Continuous delays in the commissioning of new units forced NLTPS to run its old units, scheduled to retire between 2011-15. Efficient and safe operating life of thermal power plant is considered to be around 25 years. At NLTPS, however, a large number of units are 25-57 years old.

Unit 6, where the incident occurred, is more than 26 years old and is to be retired due to unavailability of space for pollution control technologies, according to the National Electricity Plan, 2018.

NLTPS has distinct phases, where new units are usually commissioned, with auxillary utilities (like coal handling, water treatment etc) being common for different units in each phase.Phase one consists of units 1-6 (50 x 6 megawatts or MW) and 7-9 (100 x 3 MW), commissioned during 1962-70. All these units were scheduled to be retired between 2011-15.

This plan, however, could not be executed due to a continuous delay in commissioning of the new capacity.

The present total capacity of the first stage is 500 MW (unit 7, with a capacity of 100 MW, was retired in 2019). The expansion of phase one included two units of 210 MW capacity each.

NLTPS phase two has units 1-7 (210 x 7 MW) commissioned during 1988-93. Stage two expansion has two units of 250 MW capacity, each commissioned in 2014-15.

Two new units of 500 MW each, that were scheduled to arrive in 2014-15, were continuously delayed. One of them was added in 2019 and the other is yet to come.

These two units were critical as retirement of the old plant was based on the commissioning of these units. The retirement of old units (40-50-years-old) was continuously delayed due to delay in the commissioning of new units.

Source: CSE

History of disorder

2011:It was planned for NLTPS phase one to be decommissioned between 2011 and 2014. In 2011, however, the period of operations was extended for five years and still has not been retired.

2013-17: Frequent strikes in NLC on dilution of stakes and wages issues, with the matter in court till February, 2020.

May 20, 2014: Blast at a pipeline of a boiler in NLTPS phase one unit seven (100 MW) killed two people and injured four others. An NLC probe committee blamed an engineer for the incident and said it was satisfied with the built-in safety protection available in the scheme. It was claimed that a life extension programme and periodical residual life assessment studies were conducted to ensure safe operation of the plant.

June 2019:One person was killed and two others seriously injured in an explosion of the safety valve at one of the units in NLTPS phase two.

May 5, 2020: Minor fire incident at the conveyor belt in NLTPS phase two.

May 7, 2020: The incident occurred in the NLTPS phase two, unit 6 boiler of NLC India's second thermal power station. The pressure build-up inside the boiler caused the blast. Eight people, who were working in the area, were injured and taken to NLC's hospital.

There may be many technical reasons including excessive accumulation of ash and improper fuel combustion for a blast in a boiler, caused by uneven heat transfer at certain locations, said Ashu Gupta, a former general manager at National Thermal Power Corporation Limited.

All these issues are handled by power plants in day-to-day operations andmaintenance, he said. This blast is clearly a result of a failure of built-in checks and safety systems that are in place in all the power plants, Gupta added.

Such incidents are the blatant failure of maintenance and operational safety interlocks and should not be linked to the novel coronavirus disease (COVID-19) outbreak, according to Amit Kumar Singh, a power sector consultant.

There might be various technical reasons for the blast, but the real reasons will be revealed after the investigation report comes out, according to several experts.

Over the years, Delhi-based non-profit Centre for Science and Environment (CSE) has been pushing hard to expedite the retirement of old units.

The execution of government plans for the retirement of old plants are continuously delayed. These old plants require significant investment for operation and safety, with poor management at the plant level making these units prone to such serious incidents.

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Original post:
All is not well at the Neyveli thermal power station in Cuddalore - Down To Earth Magazine

Recommendation and review posted by Bethany Smith

CAMBRIDGE, Mass., May 12, 2020 (GLOBE NEWSWIRE) -- Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading genome editing company focused on developing curative therapeutics using CRISPR/Cas9 technology bothin vivoandex vivo,is presenting three oral presentations and two poster presentations at the 23rd Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT), taking place virtually from May 12-15, 2020. Intellia researchers are presenting new data in support of NTLA-5001, the companys engineered cell therapy candidate for the treatment of acute myeloid leukemia (AML). Intellia is also providing an update on NTLA-2002, its newest development candidate for the treatment of hereditary angioedema (HAE).

At Intellia, we are applying our CRISPR/Cas9 technology to develop new processes that can produce enhanced engineered cell therapies to treat severe cancers, such as AML, that traditional approaches cannot address. Our proprietary platform provides a powerful tool to generate more potent TCR-directed cells, that can treat blood cancers initially and potentially solid tumors. The data being presented today validate Intellias approach of reducing AML tumor cell blasts, and our plans to enter the clinic with NTLA-5001 next year, said Intellia President and CEO John Leonard, M.D. We are also pleased to present data that support our recently announced HAE development candidate, NTLA-2002, Intellias second systemic therapy employing our in vivo knockout approach and modular delivery platform.

Data Presentations on Intellias First Engineered Cell Therapy Development Candidate, NTLA-5001 for the Treatment of AML, and Proprietary Cell Engineering Process

NTLA-5001 is Intellias first engineered T cell receptor (TCR) T cell therapy development candidate, which targets the Wilms Tumor 1 (WT1) intracellular antigen for the treatment of AML. NTLA-5001 is being developed in collaboration with Chiara Boninis team at IRCCS Ospedale San Raffaele to treat AML patients regardless of the genetic subtype of a patients leukemia. AML is a cancer of the blood and bone marrow that is rapidly fatal without immediate treatment and is the most common type of acute leukemia in adults(Source:NIH SEER Cancer Stat Facts: Leukemia AML).

Intellias proprietary process is a significant improvement over standard engineering processes commonly used to introduce nucleic acids into cells. Intellias process enabled multiple gene edits using CRISPR/Cas9, while maintaining cell products with high expansion potential and minimal undesirable chromosomal translocations. CRISPR/Cas9 was used to insert a WT1-directed TCR in locus, while eliminating the expression of the endogenous TCRs, with the goal of producing homogeneous T cell therapies like NTLA-5001.

Intellias novel approach with NTLA-5001 can overcome the challenges of standard T cell therapy, including risks of reduced specificity associated with mixed expression and mispairing of endogenous and transgenic TCRs (tgTCRs); graph-versus-host disease (GvHD) risks, which could lead to an attack on the patients healthy cells; and reduced efficacy tied to lower tgTCR expression per T cell. Intellias unprecedented process is expected to streamline cell engineering and manufacturing, yielding a homogenous product comprising WT1-targeted T cells with high anti-tumor activity. Data highlights from todays presentation include the following:

Intellias cell engineering efforts are focused on its initial clinical investigation of NLTA-5001 on AML, while continuing preclinical studies exploring the potential for targeting WT1 in solid tumors. The company confirmed plans last week to submit an IND or IND-equivalent for NTLA-5001 for the treatment of AML in the first half of 2021.

The presentation titled, Enhanced tgTCR T Cell Product Attributes Through Process Improvement of CRISPR/Cas9 Engineering, will be made today by Aaron Prodeus, Ph.D., senior scientist, Cell Therapy, and can be found here, on the Scientific Publications & Presentations page of Intellias website. These data were a follow-on to the study presented at Keystone Symposias Engineering the Genome Conference from this past February.

In Vivo Data Supports Intellias Novel TCR Candidate

A second presentation on engineered cell therapy progress, in collaboration with IRCCS Ospedale San Raffaele, showed in vivo data demonstrating the potential of TCR-edited T cells to effectively target WT1 tumor cells in AML. In addition to the previously disclosed results of effective in vitro recognition of primary AML tumor cells by edited WT1-specific cytotoxic T cells (CD8 T cells), new data indicate that the selected TCR also enables T helper cells (CD4 T cells) to react to WT1-expressing tumor cells, providing cytokine support. This distinguishes Intellias TCR from other therapeutic TCR candidates, which either exclusively activate toxic CD8 T cells or require the co-transfection of CD8 into CD4 T cells to render them functional.

Using a mouse model carrying disseminated human primary AML, researchers observed a significant therapeutic effect, including decreased AML tumor burden. In addition, no signs of GvHD were observed in mice treated with the WT1-specific T cells. The data show that tgTCR-engineered cells have targeted anti-cancer activity in a challenging model of systemic AML, demonstrating the therapeutic potential of Intellias engineered TCR T cell approach.

The presentation titled, Exploiting CRISPR-Genome Editing and WT1-Specific T Cell Receptors to Redirect T Lymphocytes Against Acute Myeloid Leukemia, will be given today by Eliana Ruggiero, Ph.D., Experimental Hematology Unit, Division of Immunology, Transplantation and Infectious Diseases, IRCCS Ospedale San Raffaele, Italy. Notably, ASGCT meeting organizers selected this presentation as one of six to receive the ASGCT Excellence in Research Award this year.

Continued Progress on Intellias Second In Vivo Development Candidate, NTLA-2002 for the Treatment of HAE

Intellia is presenting development data updates on its potential HAE therapy, NTLA-2002, which utilizes the companys systemic in vivo knockout approach, including its proprietary lipid nanoparticle (LNP) system. HAE is a rare genetic disorder characterized by recurring and unpredictable severe swelling attacks in various parts of the body, and is significantly debilitating or even fatal in certain cases. NTLA-2002 aims to prevent unregulated production of bradykinin by knocking out the prekallikrein B1 (KLKB1) gene through a single course of treatment to ameliorate the frequency and intensity of these swelling attacks.

The KLKB1 gene knockout in an ongoing non-human primate (NHP) study resulted in a sustained 90% reduction in kallikrein activity, a level that translates to a therapeutically meaningful impact on HAE attack rates(Source: Banerji et al., NEJM, 2017). This kallikrein activity reduction was sustained for at least six months, demonstrating the same high level of efficacy and durability seen in earlier rodent studies.

The short talk titled, CRISPR/Cas9-Mediated Gene Knockout of KLKB1 to Treat Hereditary Angioedema, will be given by Jessica Seitzer, director, Genomics, Intellia on Fri., May 15, 2020, when it will be made available here, on the Scientific Publications & Presentations page of Intellias website. The presented data include results from ongoing collaborations with researchers at Regeneron, and the program is subject to an option by Regeneron to enter into a Co/Co agreement, in which Intellia would remain the lead party. Intellia expects to submit an IND or IND-equivalent to initiate a Phase 1 trial for NTLA-2002 in the second half of 2021.

About Intellia Therapeutics

Intellia Therapeuticsis a leading genome editing company focused on developing proprietary, curative therapeutics using the CRISPR/Cas9 system. Intellia believes the CRISPR/Cas9 technology has the potential to transform medicine by permanently editing disease-associated genes in the human body with a single treatment course, and through improved cell therapies that can treat cancer and immunological diseases, or can replace patients diseased cells. The combination of deep scientific, technical and clinical development experience, along with its leading intellectual property portfolio, puts Intellia in a unique position to unlock broad therapeutic applications of the CRISPR/Cas9 technology and create a new class of therapeutic products. Learn more aboutIntellia Therapeuticsand CRISPR/Cas9 atintelliatx.comand 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: planned submission of an investigational new drug (IND) application or similar clinical trial application for NTLA-2001 for the treatment of transthyretin amyloidosis (ATTR) in mid-2020 and its planned dosing of first patients in the second half of 2020; plans to submit an IND application for NTLA-5001, its first T cell receptor (TCR)-directed engineered cell therapy development candidate for its acute myeloid leukemia (AML) program in the first half of 2021; plans to submit an IND or similar clinical trial application for its hereditary angioedema (HAE) program in the second half of 2021; plans to advance and complete preclinical studies, including non-human primate studies for its ATTR program and HAE programs, and other animal studies supporting other in vivo and ex vivo programs, including its AML 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; further development of its proprietary cell engineering process for multiple sequential editing; presentation of additional data at upcoming scientific conferences, and other preclinical data in 2020; 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 ATTR, AML, and HAE programs, in any future studies, including human clinical trials; ability to develop other in vivo or ex vivo cell therapeutics of all types, and those targeting WT1 in AML in particular, using CRISPR/Cas9 technology; ability to optimize the impact of its collaborations on its development programs, including but not limited to its collaborations with Novartis or Regeneron Pharmaceuticals, Inc., and Regenerons ability to enter into a co-development and co-promotion agreement for the HAE program; statements regarding the timing of regulatory filings regarding its development programs.

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 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 Novartis or Regeneron or its other ex vivo 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+1 956-330-1917 lynnea.olivarez@intelliatx.com

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

The rest is here:
Intellia Therapeutics Reports Progress on CRISPR/Cas9 AML Cancer Therapy Using Proprietary Cell Engineering Process at the 23rd Annual Meeting of the...

Recommendation and review posted by Bethany Smith

ZUG, Switzerland and CAMBRIDGE, Mass. and BOSTON, May 11, 2020 (GLOBE NEWSWIRE) -- CRISPR Therapeutics (Nasdaq: CRSP) and Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that the U.S. Food and Drug Administration (FDA) granted Regenerative Medicine Advanced Therapy (RMAT) designation to CTX001, an investigational, autologous, gene-edited hematopoietic stem cell therapy, for the treatment of severe sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT).

RMAT designation is another important regulatory milestone for CTX001 and underscores the transformative potential of a CRISPR-based therapy for patients with severe hemoglobinopathies, said Samarth Kulkarni, Ph.D., Chief Executive Officer of CRISPR Therapeutics. We expect to share additional clinical data on CTX001 in medical and scientific forums this year as we continue to work closely with global regulatory agencies to expedite the clinical development of CTX001.

The first clinical data announced for CTX001 late last year represented a key advancement in our efforts to bring CRISPR-based therapies to people with beta thalassemia and sickle cell disease and demonstrate the curative potential of this therapy, said Bastiano Sanna, Ph.D., Executive Vice President and Chief of Cell and Genetic Therapies at Vertex. We are encouraged by these recent regulatory designations from the FDA and EMA, which speak to the potential impact this therapy could have for patients.

Established under the 21st Century Cures Act, RMAT designation is a dedicated program designed to expedite the drug development and review processes for promising pipeline products, including genetic therapies. A regenerative medicine therapy is eligible for RMAT designation if it is intended to treat, modify, reverse or cure a serious or life-threatening disease or condition, and preliminary clinical evidence indicates that the drug or therapy has the potential to address unmet medical needs for such disease or condition. Similar to Breakthrough Therapy designation, RMAT designation provides the benefits of intensive FDA guidance on efficient drug development, including the ability for early interactions with FDA to discuss surrogate or intermediate endpoints, potential ways to support accelerated approval and satisfy post-approval requirements, potential priority review of the biologics license application (BLA) and other opportunities to expedite development and review.

In addition to RMAT designation, CTX001 has received Orphan Drug Designation from the U.S. FDA for TDT and from the European Commission for TDT and SCD. CTX001 also has Fast Track Designation from the U.S. FDA for both TDT and SCD.

About CTX001CTX001 is an investigational ex vivo CRISPR gene-edited therapy that is being evaluated for patients suffering from TDT or severe SCD in which a patients hematopoietic stem cells are engineered to produce high levels of fetal hemoglobin (HbF; hemoglobin F) in red blood cells. HbF is a form of the oxygen-carrying hemoglobin that is naturally present at birth and is then replaced by the adult form of hemoglobin. The elevation of HbF by CTX001 has the potential to alleviate transfusion requirements for TDT patients and painful and debilitating sickle crises for SCD patients. CTX001 is the most advanced gene-editing approach in development for beta thalassemia and SCD.

CTX001 is being developed under a co-development and co-commercialization agreement between CRISPR Therapeutics and Vertex.

About the CRISPR-Vertex CollaborationCRISPR Therapeutics and Vertex entered into a strategic research collaboration in 2015 focused on the use of CRISPR/Cas9 to discover and develop potential new treatments aimed at the underlying genetic causes of human disease. CTX001 represents the first treatment to emerge from the joint research program. CRISPR Therapeutics and Vertex will jointly develop and commercialize CTX001 and equally share all research and development costs and profits worldwide.

About CRISPR TherapeuticsCRISPR Therapeutics is a leading gene editing company focused on developing transformative gene-based medicines for serious diseases using its proprietary CRISPR/Cas9 platform. CRISPR/Cas9 is a revolutionary gene editing technology that allows for precise, directed changes to genomic DNA. CRISPR Therapeutics has established a portfolio of therapeutic programs across a broad range of disease areas including hemoglobinopathies, oncology, regenerative medicine and rare diseases. To accelerate and expand its efforts, CRISPR Therapeutics has established strategic partnerships with leading companies including Bayer, Vertex Pharmaceuticals and ViaCyte, Inc. CRISPR Therapeutics AG is headquartered in Zug, Switzerland, with its wholly-owned U.S. subsidiary, CRISPR Therapeutics, Inc., and R&D operations based in Cambridge, Massachusetts, and business offices in San Francisco, California and London, United Kingdom. For more information, please visit http://www.crisprtx.com.

CRISPR Forward-Looking StatementThis press release may contain a number of forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including statements regarding CRISPR Therapeutics expectations about any or all of the following: (i) the status of clinical trials (including, without limitation, the expected timing of data releases) and discussions with regulatory authorities related to product candidates under development by CRISPR Therapeutics and its collaborators, including expectations regarding the benefits of RMAT designation; (ii) the expected benefits of CRISPR Therapeutics collaborations; and (iii) the therapeutic value, development, and commercial potential of CRISPR/Cas9 gene editing technologies and therapies. Without limiting the foregoing, the words believes, anticipates, plans, expects and similar expressions are intended to identify forward-looking statements. You are cautioned that forward-looking statements are inherently uncertain. Although CRISPR Therapeutics believes that such statements are based on reasonable assumptions within the bounds of its knowledge of its business and operations, forward-looking statements are neither promises nor guarantees and they are necessarily subject to a high degree of uncertainty and risk. Actual performance and results may differ materially from those projected or suggested in the forward-looking statements due to various risks and uncertainties. These risks and uncertainties include, among others: the potential impacts due to the coronavirus pandemic, such as the timing and progress of clinical trials; the potential for initial and preliminary data from any clinical trial and initial data from a limited number of patients (as is the case with CTX001 at this time) not to be indicative of final trial results; the potential that CTX001 clinical trial results may not be favorable; that future competitive or other market factors may adversely affect the commercial potential for CTX001; uncertainties regarding the intellectual property protection for CRISPR Therapeutics technology and intellectual property belonging to third parties, and the outcome of proceedings (such as an interference, an opposition or a similar proceeding) involving all or any portion of such intellectual property; and those risks and uncertainties described under the heading "Risk Factors" in CRISPR Therapeutics most recent annual report on Form 10-K, and in any other subsequent filings made by CRISPR Therapeutics with the U.S. Securities and Exchange Commission, which are available on the SEC's website at http://www.sec.gov. Existing and prospective investors are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date they are made. CRISPR Therapeutics disclaims any obligation or undertaking to update or revise any forward-looking statements contained in this press release, other than to the extent required by law.

About VertexVertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases. The company has multiple approved medicines that treat the underlying cause of cystic fibrosis (CF) a rare, life-threatening genetic disease and has several ongoing clinical and research programs in CF. Beyond CF, Vertex has a robust pipeline of investigational small molecule medicines in other serious diseases where it has deep insight into causal human biology, including pain, alpha-1 antitrypsin deficiency and APOL1-mediated kidney diseases. In addition, Vertex has a rapidly expanding pipeline of genetic and cell therapies for diseases such as sickle cell disease, beta thalassemia, Duchenne muscular dystrophy and type 1 diabetes mellitus.

Founded in 1989 in Cambridge, Mass., Vertex's global headquarters is now located in Boston's Innovation District and its international headquarters is in London, UK. Additionally, the company has research and development sites and commercial offices in North America, Europe, Australia and Latin America. Vertex is consistently recognized as one of the industry's top places to work, including 10 consecutive years on Science magazine's Top Employers list and top five on the 2019 Best Employers for Diversity list by Forbes. For company updates and to learn more about Vertex's history of innovation, visit http://www.vrtx.com or follow us on Facebook, Twitter, LinkedIn, YouTube and Instagram.

Vertex Special Note Regarding Forward-Looking StatementsThis press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, including, without limitation, the information provided regarding the status of, and expectations with respect to, the CTX001 clinical development program and related global regulatory approvals, and expectations regarding the RMAT designation. While Vertex believes the forward-looking statements contained in this press release are accurate, these forward-looking statements represent the company's beliefs only as of the date of this press release and there are a number of factors that could cause actual events or results to differ materially from those indicated by such forward-looking statements. Those risks and uncertainties include, among other things, that the development of CTX001 may not proceed or support registration due to safety, efficacy or other reasons, and other risks listed under Risk Factors in Vertex's annual report and quarterly reports filed with the Securities and Exchange Commission and available through the company's website at http://www.vrtx.com. Vertex disclaims any obligation to update the information contained in this press release as new information becomes available.

(VRTX-GEN)

CRISPR Therapeutics Investor Contact:Susan Kim, +1 617-307-7503susan.kim@crisprtx.com

CRISPR Therapeutics Media Contact:Rachel EidesWCG on behalf of CRISPR+1 617-337-4167 reides@wcgworld.com

Vertex Pharmaceuticals IncorporatedInvestors:Michael Partridge, +1 617-341-6108orZach Barber, +1 617-341-6470orBrenda Eustace, +1 617-341-6187

Media:mediainfo@vrtx.com orU.S.: +1 617-341-6992orHeather Nichols: +1 617-961-0534orInternational: +44 20 3204 5275

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CRISPR Therapeutics and Vertex Pharmaceuticals Announce FDA Regenerative Medicine Advanced Therapy (RMAT) Designation Granted to CTX001 for the...

Recommendation and review posted by Bethany Smith

Treatment of metastatic hormone receptor (HR)-positive, HER2-negative breast cancer is traditionally executed with the use of endocrine therapy. With the emergence of targeted therapies for the treatment of this disease, however, the landscape is rapidly shifting, and more therapies are under active research.

In the current landscape, oncologists have made room for CDK4/6 inhibition in combination with endocrine therapy, a combination that has been proven to improve upon progression-free and overall survival in this patient population.

At this point in time, we're grateful to have CDK4/6inhibition as our standard of care, but there is so much work going on and so many exciting new agents. What I what to think about is what lies ahead in the next decade in terms of the introduction of a lot of these newer agents into our clinics, Erica Mayer, MD, toldTargeted Oncology.

A new clinical trial is underway (PACE, NCT03147287) to further investigate CDK4/6 inhibition in combination with endocrine therapy after chemotherapy. In 3 arms of fulvestrant monotherapy, fulvestrant plus palbociclib, and fulvestrant plus palbociclib with avelumab (Bavencio), the study is primarily assessing progression-free survival (PFS), with overall response rate and treatment-emergent adverse events as secondary end points.

In an interview withTargeted Oncology, Mayer, senior medical oncologist, Breast Oncology Center, Dana-Farber Cancer Institute, discussed advances in the treatment landscape of HR-positive, HER2-negative breast, primarily with endocrine therapy and CDK4/6 inhibition.

TARGETED ONCOLOGY: Can you discuss advances in endocrine therapy in recent years and explain what you see for the future of endocrine therapy in advanced HR+, HER-negative breast cancer?

Mayer: Over the past several decades, we have primarily been using endocrine monotherapy to treat metastatic hormone receptor positive breast cancer. However, over the past several years weve had the introduction of new targeted therapies that we used in combination with an endocrine back bone, and the introduction of these agents has substantially improved progression free and overall survival for patients with metastatic hormone receptor positive HER2-negative disease. We had the introduction of the mTOR inhibitor everolimus, and the CDK4/6 inhibitors, palbociclib (Ibrance), ribociclib (Kisqali), and abemaciclib (Verzenio), as well as the most recent from 2019, a PI3 kinase inhibitor, alpelisib (Piqray).

At this point in time, there are at least 8 large randomized studies that have looked at the addition of a CDK4/6 inhibitor to back on endocrine therapy, either in the first- or second-line settings. There are trials that have used all 3 available agents and the diversity of endocrine back bone. Importantly and consistently, there is a substantial improvement in hazard ratio across the board of 0.5 to .055,which suggests a doubling and PFS with the addition of the CDK4/6 inhibitor, and that has become our standard of care for the majority of patients in the first-line or second-line settings.

In the past year, weve seen overall survival (OS) data from these agents and again, suggesting that there may be an OS advantage from the use CDK4/6 inhibitors and supporting the upfront use of these agents. The big question that has come up, though, is after a CDK4/6 inhibitor, if the cancer is progressing, what do we give next? What are our next best strategies? Should we be continuing on a CDK4/6 inhibitor like we would do with trastuzumab in HER2-positive breast cancer, or should we be switching to a different endocrine therapy with a new targeted partner? Also, how can we best select that targeted partner?

In terms of the idea of maintaining CDK4/6 inhibition, there are multiple studies ongoing trying to address this question. We are fortunate to lead a study called PACE, which is a randomized study open throughout the United States, in which patients who have progressed on a CDK4/6 inhibitor are then randomized to 1 of 3 arms, fulvestrant alone, which could be standard of care and many patients, fulvestrant with the CDK4/6 inhibitor, palbociclib, or a triplet combination of fulvestrant, palbociclib, and avelumab, which is an immunotherapy agent. This study is based off of some strong preclinical data and is actively accruing. Were hoping that will help address this question.

Beyond that, there has been research to understand mechanisms of resistance to CDK4/6 inhibitors and dissect that from known mechanisms of resistance to endocrine therapy. Ideally, we would love to think of a framework where we could, in real time, test the tumor or even test circulating tumor DNA to identify the mutation that is the cause of the resistance. Then we can apply a new targeted therapy with the next line of endocrine treatment to overcome that resistance. There are several candidates, therapies, and mechanisms which have been identified.

At this point in time, were grateful to have CDK4/6 inhibition as our standard of care, but there is so much work going on and so many exciting new agents. What I what to think about is what lies ahead in the next decade in terms of the introduction of a lot of these newer agents into our clinics.

TARGETED ONCOLOGY: With all the different agents that are available in the space, how do you determine which patients are eligible for which therapies?

Mayer: The paradigm of which treatment we give at which step in time has been something that's been changing a bit recently. As I mentioned, the first line of therapy that most patients will now be receiving would be in endocrine therapy with a CDK4/6 inhibitor, and I think it is important to note that in years past when patients showed evidence of what we think of as visceral disease, there may have been a tendency to start those patients with chemotherapy. Thats not necessary. We can give them endocrine therapy with a CDK4/6 inhibitor, which has a high response rate and a very favorable PFS and is much better tolerated than chemotherapy. This needs to be the standard of care for the vast majority of patients.

Following that, we're increasingly going to see the introduction of tumor genomics to help us identify mutations. We have 1 actionable mutation right now, which is a mutation in PIK3CA. For patients who have that mutation, the use of alpelisib may be an attractive way to go. However, for those who don't have a PIK3CA mutation, at this point in time, we have our standard endocrine therapies, and we also have everolimus. With ongoing trials were going to start to see some interesting new options for the other categories of mutations.

TARGETED ONCOLOGY: What is the key takeaway?

Mayer: The key takeaway is that none of these advances could have happened without laboratory discovery, translation to the clinic, and the completion of clinical trials. I think that for us to move as a field we need to help design the best possible trials. We need to support them all by structuring and running the trials. Importantly, all providers and patients need to work together to enter these trials and complete them and get answers these questions.

TARGETED ONCOLOGY: What accessible are some of these available agents?

Mayer: The majority of agents that I have discussed in the standard setting are FDA-approved.

These drugs that should be available to any patient, wherever they are in the United States. The availability outside the United States varies depending on each country's approval status.

Clinical trials are a challenge that our community needs to work on. Many clinical trials are available at the large academic medical centers and increasingly, trials could be open through our cooperative networks at community cancer centers or at satellite hospitals close to the larger centers. We still have many patients in this country who live very far away from institutions that have trials available for them, and I think we need to work harder to community oncologists and every breast cancer patient.

Visit link:
Improving Upon Treatment Standards in HR+ Breast Cancer - Targeted Oncology

Recommendation and review posted by Bethany Smith

Getting your period during a pandemic is like a movie scene where characters acknowledge their plight and say, At least things cant get worse. Getting a yeast infection and period double-whammy right now, though? Thats the moment that comes right after that scene, when it starts to rain.

As unpleasant as it may be to experience the irritation of a yeast infection alongside period cramps and other symptoms, your period shouldnt have too much of an effect on how you treat and get rid of your yeast infection. Hopefully, this is particularly comforting to hear when heading to your health care providers office might not be as simple as it used to be, and stressing over anything can feel more intense than normal. If you suspect youre suddenly dealing with both a yeast infection and your period, keep reading for some insight on why this might be happening and what you can do about it.

The fungus that most often causes yeast infections is known as Candida albicans, and it occurs naturally in the body, including in the vagina. Usually, bacteria called Lactobacillus prevent this fungus from growing out of control. But sometimes this yeast has a chance to grow unchecked and cause a yeast infection, which is usually characterized by itching, irritation, and a cottage cheese-like discharge.

This overgrowth can happen for a few reasons, including using antibiotics that throw off your vaginal flora, hormone fluctuations, health conditions like uncontrolled diabetes, or even lifestyle habits like regularly wearing your sweaty workout clothes for way too long, according to the Mayo Clinic. In any case, rest assured that yeast infections are incredibly common. In fact, 75 percent of women report getting a yeast infection at some point, with many experiencing at least two infections in their lifetimes, the Mayo Clinic notes. Basically, youre in great (and probably also very annoyed) company.

While most people dont regularly get yeast infections simultaneously with their periods, its definitely possible to have both at once, Taraneh Shirazian, M.D., a gynecologist and assistant professor in the Department of Obstetrics and Gynecology at NYU Langone Health, tells SELF. This is because hormones, vaginal pH, and bacteria levels can all fluctuate in the time leading up to your period, making it easier for yeast to grow too much, H. Frank Andersen, M.D., a clinical education director and ob/gyn in the Department of Medical Education and Clinical Sciences at Washington State University, tells SELF.

More specifically, an increase in estrogen in the days before your period could predispose you to a yeast infection/menstruation combo. A significant enough uptick in estrogen is a known risk factor for yeast infections; high levels of estrogen appear to lower vaginal pH, causing it to become more acidic in a way that makes it easier for yeast to overgrow to the point of infection.

Even if youre on a combined hormonal contraceptive that suppresses this kind of natural hormonal fluctuation, the estrogen in your birth control itself can also increase your risk of a yeast infection, the Mayo Clinic says. There is also research to indicate that levels of Lactobacillus drop during your period, which causes vaginal pH to become more acidic.

Luckily, Dr. Andersen notes that theres no reason to worry that your period will make the yeast infection any worse, symptomatically speaking. You might be more annoyed, of course (see the aforementioned rain metaphor), but the fact that youre menstruating shouldnt extend or exacerbate the infection. With treatment and time, your yeast infection should conclude as usual, Dr. Shirazian says.

Normally, when you notice the first signs of a yeast infection, youre probably tempted to run to the drug store, pick up an antifungal suppository, and call it a day. But you should consider at least calling your ob/gyn before trying to treat the infection at home, especially given our current reality.

Continued here:
How to Manage a Yeast Infection and Period Symptoms at the Same Time - Self

Recommendation and review posted by Bethany Smith

The Latest Growth Report on Endometriosis Therapies Market size | Industry Segment by Applications (Hospital,Clinic andOther), by Type (Hormonal Contraceptives,Gonadotropin-releasing Hormone (Gn-RH) Agonists ,Progestin Therapy andAromatase Inhibitors), Regional Outlook, Industry Demand, Latest Trends, Endometriosis Therapies Industry Share, Research Growth Forecast & Revenue by Manufacturers, The Leading Company Profiles, Growth Forecasts 2026.

The Endometriosis Therapies market study offers a thorough examination of the industry space primarily by evaluating the major parameters that define the market dynamics production and consumption. In term of production, the report incorporates information pertaining the product manufacturing, its revenue, along with gross margins of the manufacturers. The unit costs offered by the producers across various geographies are also provided in the report.

Request Sample Copy of this Report @ https://www.aeresearch.net/request-sample/185076

With respect to the consumption facet, the research report details the product consumption share and volume while listing out the individual sale prices. It also lays out the import and export graphs across various geographies and concludes by predicting the production and consumption patterns of the Endometriosis Therapies market during over the projection period.

A brief outline of the regional outlook:

A gist of the product spectrum:

An outline of the application spectrum:

Brief summary of the competitive landscape:

To sum it up, assessment on the upstream raw materials, downstream buyers, and distribution channels has been documented in the Endometriosis Therapies market report.

Table of Contents:

Executive Summary: It includes key trends of the Endometriosis Therapies market related to products, applications, and other crucial factors. It also provides analysis of the competitive landscape and CAGR and market size of the Endometriosis Therapies market based on production and revenue.

Production and Consumption by Region: It covers all regional markets to which the research study relates. Prices and key players in addition to production and consumption in each regional market are discussed.

Key Players: Here, the report throws light on financial ratios, pricing structure, production cost, gross profit, sales volume, revenue, and gross margin of leading and prominent companies competing in the Endometriosis Therapies market.

Market Segments: This part of the report discusses about product type and application segments of the Endometriosis Therapies market based on market share, CAGR, market size, and various other factors.

Research Methodology: This section discusses about the research methodology and approach used to prepare the report. It covers data triangulation, market breakdown, market size estimation, and research design and/or programs.

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Endometriosis Therapies Market Growth Is Skyrocketing Beyond Predictions - News by aeresearch

Recommendation and review posted by Bethany Smith

With his narrow face, prominent teeth and large eyes, Joey Hoofdman always looked very different from his siblings.

As well as that, his strong work ethic and keen sense of order was greatly at odds with the rest of his chaotic family.

Feeling like such an outsider made Joey miserable throughout his life. But when he began digging around for answers about his identity, little did he anticipate the scale of the genetic and ethical minefield he was about to step into.

While he was growing up in Rotterdam, Holland, Joeys parents never hid the fact they had needed help conceiving him. His dad who already had two kids from a previous marriage had gone through a vasectomy reversal for Joeys mum.

They told me they went to a doctor because of my father, explains Joey, 33. They undid the procedure because my mum really wanted to have children of their own.

Joeys parents insisted the fertility treatment theyd received had been straightforward his mother had been artificially inseminated with his fathers sperm. But Joey was unconvinced by this explanation: I was just so different from my brother and sister.

While Joeys father drank, argued, and was constantly in debt selling the kids belongings for cash, Joey by contrast was sensible, calm and early on began saving for a better life.

Eventually, sick of feeling so isolated, at just 15 Joey met his first boyfriend, moved in with him, and left his family home for ever. It was the best decision I ever made, he admits.

By the time Joey was 19, he was a successful gym owner and in a stable, loving relationship. But even though hed been able to escape the chaos of his family home, he was dogged with unease about his identity.

It nagged away at him until his father died in 2012 and believing hed never get to the bottom of it Joeys mental health spiralled.

Close friends saw it was really bothering me. It became a depression when my father passed away. I thought it was grief, but it never stopped. I felt, Maybe Im never going to know what happened?

'After three or four years, I was still in a state of depression and I didnt want to get out of bed. It was the worst feeling Id ever had.

When Joey considered suicide, his psychiatrist urged him to confront his mother. So, in March 2017, after making her a coffee and sitting her in the garden, Joey begged her for answers.

Is my father my biological father? Did something happen to you? Or am I from a neighbour or somebody else? Mum was quite upset when I asked her that. She couldnt believe I thought she might have been with another man. It distressed her, me thinking she had been unfaithful, but I just said, Mum, please. I have to know for sure.

Joey asked her to take him through exactly how he was conceived, and she said shed seen the vials of sperm labelled with the name of Joeys father.

When pressed on the name of the clinic and doctor, his mums patchy recollections led Joey to do an online search, which brought up a suburban clinic near where he grew up and the doctor Jan Karbaat.

Joey was shocked to see many complaints about missing paperwork and lost vials, suspicions that women had been given the wrong sperm, and also a court case. The government had shut the clinic in 2009. It was a horror story. Joeys mother was so upset by these findings, she threw her son out of the house there and then.

Continuing to Google Jan Karbaat sitting in his car, Joey spotted a photo of the fertility doctor behind the scandal, and got goosebumps, certain he was looking at his biological father.

It was like an electric shock. I couldnt see his face, only myself. I had the same smile, the same eyes. I sent the picture to friends, they said, Is that you, Joey?

Desperate for answers, Joey tracked down Karbaat, who was living locally. But when he knocked on his door, he was told the doctor, then 89, was too sick for visitors. A week later, he died.

Robbed of the opportunity to confront him, Joey searched for answers elsewhere. He discovered a Facebook group whose members suspected they were all Karbaats children.

Joey quickly built a rapport with Moniek Wassenaar, and noticed her Amsterdam home was strikingly similar in taste to his own. We decided to see if we were really related and did a test we were indeed half-siblings. That was the first match. It was very emotional.

Then, in June 2017, Joey put his results on a genealogy site that matches DNA results with others around the world. It paired him with Inge Herlaar, 39, who he rang, saying, I think Im your half-brother. Then came a match with Marsha Elvers, 38, another half-sister.

At the time of Karbaats death in 2017, he was thought to have had 22 recognised children from several marriages, and was suspected of secretly fathering 49 more with his patients without their permission.

In early 2019 a court decision was ruled allowing Karbaats DNA to be released for testing. Receiving the confirmation he was Karbaats son was momentous for Joey. When I saw it written down, I broke, he admits. It was almost too much to process.

Joey has at least 61 known siblings and the family keeps growing. With dark humour theyve called themselves the Karbastards. Now, Im no longer angry but I am curious, says Joey. I have the puzzle in my head, but I cant put the pieces together.

Maybe he did it because of the money, maybe he had a God complex. Thats what everyone says. But I think, at the start, he just wanted to help mothers have children.

Last month, Joey visited Karbaats grave in Rotterdam. It was the closest I could get to him. That was the most important thing so I could have peace of mind. Im not responsible for his wrongdoings and his actions, but I need to forgive him to move on. I deserve that.

Shockingly, Jan Karbaat is not the only doctor to have committed fertility fraud but no fertility doctor has ever been put behind bars for using their own sperm.

Donald Cline

In May 2015 it emerged that physician Donald Cline, whod featured in People magazine, had used his own sperm in the 1970s and 1980s while treating women in Indianapolis.

Cline was exposed after one of the people conceived at his clinic took a 23andMe DNA test, only to discover several unexpected half-siblings, whose mothers had all been Clines patients.

Norman Barwin

A pillar of the Jewish community in Ottawa, Barwin was head of the Canadian Fertility Society and had received the Queens Golden Jubilee Medal. Like Karbaat, he was first accused of chaotic record keeping, which led to women being given the wrong sperm.

But in 2016 it emerged hed been using his own sperm. Hes currently being sued by a group of his biological children, and their legal mothers and fathers, his former patients.

Cecil Jacobson

This twisted doc nicknamed The Sperminator by a TV doc about him fathered over 70 children in Virginia. His case inspired a 1993 book, Babymaker.

He also injected women with the pregnancy hormone HCG, so they produced symptoms and believed themselves pregnant.

He would give them ultrasounds and tell them that bowels or faecal matter were foetuses, before later telling them their baby had died.

The Immaculate Deception podcast is available weekly on Apple, Spotify and all podcast providers

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Doctor secretly fathered at least 60 children with patients without their permission - Mirror Online

Recommendation and review posted by Bethany Smith

Henry Budzynski, a 65-year-old airplane mechanic, is one of a small segment of society that is the most vulnerable to the coronavirus: those whose immune systems are compromised. He is undergoing cancer treatment and says, I cant afford the virus right now."

He stays home as much as he can, but every Wednesday, Henry Budzynski drives to a doctors office in Mechanicsville, where an inch-long needle is stuck into his skin just above his belly button, and a shot of chemotherapy is injected into the fatty tissue around his stomach.

Because of the cancer called amyloidosis that inhabits his body, his immune system is 40% to 50% the strength of a healthy persons. Later this month, after he checks himself into the 10th floor of the Massey Cancer Center for a high dose of chemotherapy and a stem cell transplant, his immune system temporarily will cease to exist altogether.

The timing undergoing such a procedure during a pandemic isnt ideal, but he has no other choice.

Budzynski, a 65-year-old airplane mechanic, is one of a small segment of society that is the most vulnerable to the coronavirus: those whose immune systems are compromised. Should he contract COVID-19, his body would be less prepared to fight off the virus, and he would be less likely to survive. Chemotherapy, certain types of cancer and organ transplants can cause ones immune system to be weakened.

I cant afford the virus right now, Budzynski said.

But the immunocompromised are also, in many ways, the most prepared to combat the pandemic. They were comfortable with social distancing before there was a name for it. Theyre adept at keeping their hands clean and watching what they touch.

Its been months since Budzynski stepped foot into a Lowes, Home Depot or Food Lion. When its time to buy groceries, he drives to the store with his wife, Rosemary, and waits in the car while she shops. Riding in his vehicle, which he designated a clean space, is off limits to everyone but Rosemary and their daughter.

Wednesday is a milestone in Budsynzkis treatment. After a year of weekly chemo shots to different points on his abdomen, this will be the final injection. His upcoming treatment, he said, should extend his life another 20 years.

While Budzynski cant afford to wait out the pandemic, other cancer patients have decided that COVID-19 poses a greater threat than their cancer. Surgery often means entering the same building where the virus is treated and interacting with potential vectors.

Even if COVID patients are corded off, the virus can still spread. One study by the U.S. Centers for Disease Control and Prevention found that the coronavirus could be found on half of the shoes of medical staffers. The virus was even discovered on hospital floors not walked by COVID patients.

Thats all my patients need to hear, said Dr. Kelly Hagan, a hematologist-oncologist with the Virginia Cancer Institute.

One of Hagans patients, an 88-year-old woman, was recently diagnosed with breast cancer. In a normal world, her surgery would have been scheduled immediately. Instead, she asked to delay it until July or August, and Hagan accepted. Six months ago, the doctor said, such hesitancy never would have been tolerated.

Hagan has found herself asking questions shes never asked in her career: Are some patients better off without immediate treatment? She examines the cancer, and how aggressively it spreads. She asks herself if the patient can survive two months without intervention. She studies the comorbidities, or risk factors, of the patient.

These questions, she said, involve the art of medicine more than its science.

Dr. Eric Douglas, with his dog, Jayden, could not visit hospitals even before the pandemic. He has a disease that causes cancerous cells to replace his disease-fighting white blood cells. A cold can spiral into pneumonia

Dr. Eric Douglas couldnt visit hospitals even before the pandemic. The cancer inside him, Waldenstroms macroglobulinemia, causes cancerous cells to replace the disease-fighting white blood cells that make up his bone marrow. A simple cold can spiral into pneumonia.

Disease can overtake his body without any explanation for its origin. In 2015, he nearly died of septic shock, and he doesnt know why.

His cancer forced him to leave his career in anesthesiology, but Douglas, 64, accepted the transition as an opportunity to remake himself. He went back to school and earned masters degrees in theology and religion. He now teaches part time at Randolph-Macon College and is on staff at New Hanover Presbyterian Church.

When the pandemic reached Virginia, he pulled down the stairs to his attic and retrieved two boxes of surgical masks he had stored in his home. He had purchased them during the 2009 swine flu epidemic, and he strapped one over his ears recently when he drove to UPS to mail a package a sweater knitted by his wife for his 1-week-old grandson he had not yet met. He sat in his Toyota RAV4 and waited until every customer left the building.

When he returned to his car, he wiped down his hands with sanitizer and drove home. But his children were upset when they found out he had taken a risk by leaving the house, so he hasnt gone anywhere since. Once a week, hell sit in his car, turn the key and let the engine idle for five minutes just to keep it running.

Among those who cannot delay his next procedure is Jerry Deans, 71, who was diagnosed with prostate cancer in 1999 and has undergone a litany of treatments since.

Later this month, hell lie on a table for 30 minutes at the Sarah Cannon Cancer Institute in Henrico County while a focused beam of radiation is projected into the T12 vertebra of his spine and his left hip. If all goes well, he wont feel a thing, and he wont experience a single side effect.

But waiting was never an option. Putting off radiation would mean risking his bones fracturing and his spine collapsing.

It needs to be done, Deans said. Its not an emergency, but its pretty urgent.

Because the cancer institute is in a separate building from the hospital, he wont be treated under the same roof as COVID patients.

When the pandemic struck, Deans was undergoing chemotherapy treatments. He began taking precautions in January, wearing gloves and no longer hugging others, long before they became common practices.

When the radiation therapy is behind him, he and his wife, Patsi, and his dog, Zoey, will take their camper north to the Potomac River for a small vacation. Patsi and Zoey, he said, are his hugging partners.

Isolation might be the hardest part for Angela Bowman, 61. She misses having physical human contact, and theres a new baby in the family she hasnt met yet. Before the pandemic, shed walk the malls at Short Pump or Virginia Center Commons and window-shop. She enjoyed going to the grocery store and finding a good sale.

Her friends will bring her groceries and leave them on the front stoop, or she might sneak into a grocery store on the way home from her chemotherapy treatment for non-Hodgkins lymphoma. Her friends have reliably called and visited in creative ways.

Two friends parked their car in her driveway, and called on the phone so they could speak to her and see her at the same time. Another came for coffee and sat on the back deck, a good distance away.

For Jim Price, 65, life in lockdown is familiar. He learned to social distance in 2013, when he had a liver transplant. Thats when he realized the importance of washing his hands and avoiding touching public doorknobs.

For Jim Price, 65, life in lockdown feels familiar. Its similar to the summer of 2013, when he received a liver transplant and spent the next eight weeks at home. Few visitors were allowed to enter his home, and if they did, they were all asked to scrub their hands in Purell, which had been purchased in bulk from Costco.

In the years since, he has become adept at washing his hands longer and more often. He keeps small containers of hand sanitizer in his car. He avoids touching public doorknobs whenever possible.

To help his body accept its new organ, Price takes an anti-rejection medicine that suppresses his immune system. For the past two months, he has lived life the way he did after his transplant, staying home, watching Bonanza or The Andy Griffith Show on television and reading fiction by authors like Dean Koontz. He also works full time as a lumber salesman.

You get this gift, this opportunity to stay alive, Price said. So you want to limit the number of opportunities to screw up.

Thanks to the coronavirus, healthy people are seeing the world from the perspective of someone whos immunocompromised, said Faith Jones, 38, who was diagnosed with lupus when she was in college.

Because of her condition, shes prone to developing pneumonia. When she felt feverish and stopped breathing normally last month, she went to the University of Virginia to be tested for COVID. The test came back negative.

She wants people to know that when they stay at home, they arent just protecting themselves. They are protecting people like her, and they are protecting the elderly.

Its not just about you, she said.

The residents of Deltaville in Middlesex County have taken the pandemic seriously, said Anne Cooper, 68. The customers at the local market generally wear masks, and at the hardware store theres a Purell station and a sign that says Use it or leave. Campgrounds have remained closed to keep outsiders away. The average age in Middlesex, she said, is 58.

Cooper administers chemotherapy pills at home for her breast cancer, twice every morning and twice every evening. She visits the market once a week, but its easy to avoid crowds in Deltaville, she said.

Twice a week, she receives physical therapy for the sciatica in her spine that causes hip and leg pain. She waits in the parking lot until her name is called, walks directly to the treatment room and interacts only with the therapist attending to her.

Still, in the back of her mind, she wonders who the therapist has interacted with and what germs he or she might have caught.

Sometimes, it scares me that Ive survived cancer for 17 years, and this might kill me in four months, she said. I leave it in the hands of the Lord.

Originally posted here:
Those with compromised immune systems are the most vulnerable to COVID-19. They are also the most prepared. - Richmond.com

Recommendation and review posted by Bethany Smith

Megan Dunn takes a long look at the art in the Auckland Hospital collection and finds out that yes, there is art, even in intensive care but the price of seeing it is everything.

I got the pamphlet in the mail back in January. It says after someone close to you dies it is natural to:

The day the pamphlet arrived I splayed it open and looked at the photo inside: a network of hands holding other hands. Cropped close. Just linked hands held in support of one another. Beneath the photo: we grieve as deeply as we love.

I am unable to return to normal services.

Painting from collection donated by the Art Komiti of Aucklands Paremoremo Prison

I had noticed art in waiting rooms before, not art with a capital A, not the kind of art that an art writer such as myself would bother to write about, because art like so much else in our society has its hierarchy. Contemporary art is a high-stakes game; who you write about is as important as what you say. Who cares about the art in the waiting room? Lets abbreviate: who cares?

Me. Ive had cause to think about it because last year Mums cancer returned from outer space. Shed been in remission for 17 years, which might be a national record for multiple myeloma, a cancer of the blood. Ive never really understood what her cancer is, just that she beat it first time round, and, last July, the cancer cell count was up. Then she started chemo.

Im not coping, Im just not coping, Mum called after the first round, in tears.

Have you eaten anything?

No. I dont feel like eating.

It was the dex. The specialist, a blonde in a miniskirt and long black boots, said, Thatll be the dex. Short for dexamethasone, a steroid. Another bitter pill to swallow. At that point Mum had to take up to 10 pills a day. I looked at the specialist. I was angry. Mums not coping. I had flown to Auckland to join Mum for her next haematology appointment.

But what art could I prescribe? Optimism matters, but art isnt always soothing or kind.

Im lucky I have a chance of getting better, Mum said. True. Her prognosis was good.

We went upstairs for her second round of chemo. The nurse in blue gown, gloves and a shower cap wheeled it around in a mauve container, hooked it on a drip, and inserted the port into Mums vein.

Other patients stretched out on beds like La-Z-Boys, skin drained. They too were waiting, but looked like they didnt have a chance of getting better.

I stared into the little office next to Mums chair. Harried papers on the desk; a PC lit up like a bright face, ready to supply details, cheer, whatever counts most. On the wall, an original painting of a phutukawa tree. I call it original but the concept wasnt. The sea peeked out of the background, the sky bluer than the sea. The oils had been used with little mixing, but I didnt mind that painting of the beach in summer rudely lit up by the phutukawa tree, the needles burst open in the leaves, red as fireworks.

The nurses are very nice here, Mum said.

Niceness counts like stem cells it has a tally.

That was quick, I said.

It really doesnt take long.

I wondered how long the painting took the artist. Guessing from the paint application, not long, but longer than it takes a dose of chemo to run into your veins.

I wanted to ask the nurse: who bought that painting? Who is the artist? Who put that there? But I was too shy. Why stop this real raw moment, for an intellectual aside, a detour into the art we look at it when were waiting to get better or to see how much worse it can get?

The painting of the phutukawa tree in summer yields its bouquet. Just be.

***

Mum and I got lost en route to blood work. The hospital has an art collection in its corridors that must be maintained by someone, however irregularly. We passed a Claudia Pond Eyley print black lines of plants, bright colours, a Pacific infusion abandoned along a corridor. A trio of photographs of flowers. Where are we? Is it this the way? They need to give better directions. Then, in a gangway, a pair of large, textured, brown abstracts, neither good nor bad, just out of time, like photos of your parents when they were young, in the fashions of the day. Those sideburns. What chops! Mums perm. Dads flares. A big Pat Hanly painting called Vacation was by the escalator. I meant to come back, retrace my steps, and find all this art again. And I did come back, but by then everything had changed.

I dont want to write what comes next, because I dont want the wait to be over. Waiting is a comfort in its own sad way. Just wait and see.

The sea. Waves lap in and out. I see it whenever I attend my doctors surgery above Courtenay Place. Piha is by the Korean-born photographer Jae Hoon Lee. Its moved around the surgery over the years. Now its in the waiting room above a line of plastic chairs. Whoever bought it must have thought it was soothing and anaesthetic, a balm for a worried soul like mine.

What do you want to see when you go for a smear test, or hold a baby that wont go the fuck to sleep, or wait with your partner for test results, a prognosis, a new vaccine?

I thought Ill wait to write about the art at the doctors surgery or the hospital. Or the dentist, though I have not been to the dentist in ages, no cash, so Ill wait. How much art is in your life? How many fillings? Do you have art at home? If so, what? Where did you get it? At what price?

In the waiting room, I dont blame you if you dont want to look at something difficult and ugly or think about something hard. If youre just after a good view, Arent we all?

At my doctors surgery Pihahangs near a plastic container for pamphlets, ruffled in waves.

For every problem there is a pamphlet.

The pamphlet that arrived in the mail in January is titled Department of Critical Care Medicine Bereavement Follow-up Service.

Piha in situ at Courtenay Medical, Wellington

The waiting room isnt just literal, but it is literal too. I know because the last time I was there I got a hot chocolate from the machine. Warm, syrupy. No art, just a TV on the wall.

At the haematology department, I watched a dad sitting with his young daughter on his lap. Her mind looked far away. She waited with the patience of one who has waited before. Then her mum appeared in a turban. I looked at this young mother, I had no idea what cancer of the blood she had, but I really hoped she had a chance of getting better.

Two in 100 people die, Mum said. Back in December she was waiting for her stem-cell transplant. She would be in hospital for two to three weeks. The transplant would take her immunity to zero that was where the risk of infection crept in but then the white cells would ingraft and her count would go back up. Shed signed the forms, accepting the 2% chance she would die. If theres not a bed on Thursday, it will be next week.

I wondered: Will there be any art in her room? And if so, what will it say?

The phutukawa painting in the office of the haematology department says shush shush, that lulling noise of waves from the beach. Dont worry, relax. Its OK. Look at the view. But the Jae Hoon Lee photograph in my doctors surgery says to me your hurt is timeless, the sea will exist whether we do or not, release your grip, whatever happens next will be surgically safe, emotions are never still, time is an inlet, the sea runs in and out.

***

I always knew that waiting was part of the problem. What if you wait too long? Then you cant get around to what you were going to say because you are:

***

Outside Ward 82, ICU (acute surgery). On the intercom, the number one has been buzzed off, pushed too many times. All other numbers present and correct. My mother is in bed 17, I tell the intercom, after Ive pressed the number one that isnt there and waited. Then I walked through vaguely yellow corridors lined with three framed prints, each composition a rectangle yellow, blue, orange lined with holes down the middle. They reminded me of paeans to the common household sponge. I stop at the hand-gel pump and sanitised. The art at the hospital is sanitised too but Im beyond caring. Too much caring and you move though it and pass out on the other side somewhere in the vicinity of bed 17.

At the end of the corridor past the hand gel, the Chen family have donated a small square print, red with black scribble, in honour of the ICU team. I clocked the gold engraved plaque, their appreciation registered on the wall. The painting not unlike the size of a fire alarm, but there is no glass to break open, the call has already been raised.

I got the call from the registrar on December 22 to say that Mums stem-cell plant was not going as expected and she had been admitted to ICU.

Ive been thinking about what I will do when I get better, Mum said, the night I arrived. She sat propped up in bed, on oxygen. Her face flushed, swollen, but superficially OK.

I sat next to her bed. Oh yes, what do you think?

Im going to come to your book launch.

I smiled. My book of personal essays about art and life, already way behind schedule.

What else? I asked.

I might meet someone new. I might travel.

Where would you like to go? I asked.

She paused. Maybe Africa, she said. I could go on a safari.

I nodded. Mum found it hard to walk up the small pronounced hill to our house in Wellington, sweated easily, mopped her forehead. Then shed wait, slightly panting, for her breath to right itself again, restart.

That night, I slept in the hospital. They say you can stay in my room at the Motutapu Ward, Mum said. There was no art in her room, but a wall-to-ceiling poster of a forest, kauri trees, dense, shady, green.

In the morning I got buzzed back into ICU. Mums arms and legs, twitched, calling out; face red, body puffed up; trying to unpick the PICC line from her arm, in among the beeping and the rising heart monitor, the oxygen exhaling. She doesnt recognise me, is raving to the charge nurse, who held her hand and looked at me and explained, Im just going some gentle reorientation work.

I must have seemed stunned.

Do you want to speak outside the room? the charge nurse asked.

I nodded. In the room next door to bed 17, I wailed, What is happening? Oh, what is happening? The charge nurse held me in place and comforted me, when there was no comfort to give.

Next the intensivist arrived in her blue scrubs, removed her surgical mask, wiped her hands with gel, introduced herself as Kylie. She wanted a family meeting.

The whnau room: the painting was donated by the Art Komiti of Aucklands Paremoremo Prison

The whnau room at ICU contains another Claudia Pond Eyley print on one wall and a multi-panel painting of a New Zealand landscape on the other. The mountains, a lake, smooth and even and still. A perennial view of nature, so calm, so undisturbing to see. I cant blame the hospital for containing so many paintings of the view sky, sand, sea, soothing, stretching, somehow infinite. A vista of comfort, comprehended.

I sat down on one side of the big meeting table, Kylie on the other. What is your understanding of your mothers condition? she asked. I rattled off the facts while an accompanying nurse took notes.

Mum had come in for a stem-cell transplant. She had caught pneumonia at a point in the process when she had no natural immunity. She had developed delirium. Her pre-existing heart condition had been set off: arterial fibrillation. She needed oxygen support to breathe.

This is what the 2% of risk looks like? I asked.

Yes. Kylie explained that they couldnt provide sedatives like morphine as that would risk compromising Mums breathing further. They could put Mum on a ventilator if she deteriorated but that would not be a good sign and would come with an extra level of risk. What helped patients with delirium was familiar voices.

I asked her, Are you holding anything back?

Just that this is very life threatening. Her eyes, an expression, I can now only call grave.

Well, it seemed apt. I liked Kylie. The intensivist understood how much intensity was required.

The job title intensivist seemed funny to me because in high school I was always told I was too intense. We have to find a happy medium, a teenage girlfriend once told me. So I got into art because it seemed like a place where intensity went and didnt have to turn down a notch. But art isnt the only place for intensity. At the intensive care unit (acute) on the eighth floor of Auckland Hospital the intensivists are also at work.

All day the nurse at bed 17 and I spooned jelly or orange juice into her mouth, helped her not frantically unpick her PICC line, I just want to go for a walk! No, no dear, you cant get up. Mum, you just need to rest. Rest. A familiar voice on replay. Youll feel so much better if you rest.

In the night I noticed the print of a lone bugle boy down some New Orleans alley, presumably playing jazz. No plaque. Who bought it? What family? Who was lost?

In ICU the beeping was persistent, insistent. The constant sound of inhaling, exhaling. Poke your tongue out. Ahhhh. Good girl.

Were concerned about the delirium. In ICU, delirium can be intensified, especially in older people, by the strange sounds, lights, faces.

I forgot the ducks! I keep meaning to mention the duck painting, a watercolour, a good one too, of some ducks paddling around their pond, giving no quacks in ICU. The duck pond was donated by another family, with a brass plaque. I should have jotted down the name.

The eyeball is so moveable, up and over, it can even see things that arent there. Mum said her family prayer, over and over, eyes roving. We consecrate to Thee, O Jesus of Love My aunt clutched her hands and said the prayer with her.

One night in ICU I passed a large rectangular collage of brightly coloured red and pink buttons like some budget Damien Hirst pill painting. Never passed it again. Beep, beep.

Her oxygen levels are saturating nicely.

How much?

Fifty-five percent, then down to 48%.

Dont obsess about the blood count.

Look at the patient. Look at the face.

The face her not her.

My daughter in the car on the way to the hospital singing: Pop bang crack goes the Christmas cracker, pop bang, crack goes the Christmas cracker, we will pull it off POP.

Eyes popped, snapped.

Shes been restless.

You need to sleep.

Sleep.

I stop and start, keep typing the next line, then deleting it. I dont want to get to the end of this. I dont want to remember all the family meetings in the whnau room waiting. I dont want to chart the order of those disordered days.

What was I going to say about art?

***

I spent the first weekend after Mums stem-cell transplant in her room at the Motutapu Ward. Motutapu means sacred or sanctuary. Shed just had the big dose of chemo and was quiet, but not yet unwell. My aunt was going to join her on Monday Mums 69th birthday for what would be the worst week of the process. I sat on the bed, Mum on the La-Z-Boy. We said not a whole lot.

At one point, I think I can manage a walk.

We passed the two nurses stations. En route I pointed out the art. We stopped by a faded print of sunflowers, beneath glass, but the artist was no Van Gogh. The first time Id passed the sunflowers, I hadnt rated them at all. But that was before Id read that they were by Chris Corlett, a 17-year-old who died of acute lymphoblastic leukaemia 20 years ago. Sunflowers of Hope does look like it was painted by a teenage boy. Theres something gnarly about them. Large and abundant. Full of life. Except for the leaves Chris paid special attention to the leaves stippled with decay, a bit heavy metal.

Sunflowers of Hope, Chris Cortlett, Auckland Hospital, 2019.

We stood reading the accompanying framed text about the foundation Chris had started to build up a database of 100,000 bone-marrow donors.

Courage, charisma, strength of character, sincerity whatever it is that makes some people inspirational and very special, Chris Corlett had it.

Another Claudia Pond Eyley print near the kitchen and on the door a sign that read: Dont use if youre come from a red room. I was confused by it when Id made Mum a cup of tea earlier and had to ask the nurse, Is this a red room? (Mums room was not a red room.)

I felt the light weight of Mums hand on my arm as we looped around to reception. I showed her my favourite painting, tucked in a corner. Its colours were so bold that from the corner of my eye I first suspected Matisse. Then was embarrassed when I realised the artist is Harriet, aged six, who donated Flowers for the Leukaemia Ward in memory of her father, Ned.

Harriet may not be Matisse but for a six-year-old her vase of flowers is a masterpiece of colour and compression.

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Art in the waiting room - The Spinoff

Recommendation and review posted by Bethany Smith

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Approach may lead to new treatment approach for osteoarthritis, obesity

Researchers at Washington University School of Medicine in St. Louis found that gene therapy in mice helped build strength and significant muscle mass quickly, while reducing the severity of osteoarthritis. The gene therapy also prevented obesity, even when the mice were fed a high-fat diet.

Exercise and physical therapy often are recommended to help people who have arthritis. Both can strengthen muscle a benefit that also can reduce joint pain. But building muscle mass and strength can take many months and be difficult in the face of joint pain from osteoarthritis, particularly for older people who are overweight. A new study in mice at Washington University School of Medicine in St. Louis, however, suggests gene therapy one day may help those patients.

The research shows that gene therapy helped build significant muscle mass quickly and reduced the severity of osteoarthritis in the mice, even though they didnt exercise more. The therapy also staved off obesity, even when the mice ate an extremely high-fat diet.

The study is published online May 8 in the journal Science Advances.

Obesity is the most common risk factor for osteoarthritis, said senior investigator Farshid Guilak, PhD, the Mildred B. Simon Research Professor of Orthopaedic Surgery and director of research at Shriners Hospitals for Children St. Louis. Being overweight can hinder a persons ability to exercise and benefit fully from physical therapy. Weve identified here a way to use gene therapy to build muscle quickly. It had a profound effect in the mice and kept their weight in check, suggesting a similar approach may be effective against arthritis, particularly in cases of morbid obesity.

With the papers first author, Ruhang Tang, PhD, a senior scientist in Guilaks laboratory, Guilak and his research team gave 8-week-old mice a single injection each of a virus carrying a gene called follistatin. The gene works to block the activity of a protein in muscle that keeps muscle growth in check. This enabled the mice to gain significant muscle mass without exercising more than usual.

Even without additional exercise, and while continuing to eat a high-fat diet, the muscle mass of these super mice more than doubled, and their strength nearly doubled, too. The mice also had less cartilage damage related to osteoarthritis, lower numbers of inflammatory cells and proteins in their joints, fewer metabolic problems, and healthier hearts and blood vessels than littermates that did not receive the gene therapy. The mice also were significantly less sensitive to pain.

One worry was that some of the muscle growth prompted by the gene therapy might turn out to be harmful. The heart, for example, is a muscle, and a condition called cardiac hypertrophy, in which the hearts walls thicken, is not a good thing. But in these mice, heart function actually improved, as did cardiovascular health in general.

Longer-term studies will be needed to determine the safety of this type of gene therapy. But, if safe, the strategy could be particularly beneficial for patients with conditions such as muscular dystrophy that make it difficult to build new muscle.

In the meantime, Guilak, who also co-directs the Washington University Center for Regenerative Medicine and is a professor of biomedical engineering and of developmental biology, said more traditional methods of muscle strengthening, such as lifting weights or physical therapy, remain the first line of treatment for patients with osteoarthritis.

Something like this could take years to develop, but were excited about its prospects for reducing joint damage related to osteoarthritis, as well as possibly being useful in extreme cases of obesity, he said.

Tang R, Harasymowicz NS, Wu CL, Collins KH, Choi YR, Oswald SJ, Guilak F. Gene therapy for follistatin mitigates systemic metabolic inflammation and post-traumatic arthritis in high-fat diet-induced obesity. Science Advances, published online May 8, 2020.

This work was supported by the Shriners Hospitals for Children, the National Institute of Arthritis and Musculoskeletal and Skin Diseases, the National Institute on Aging and the Office of the Director of the National Institutes of Health (NIH). Grant numbers AR50245, AR48852, AG15768, AR48182, AG 46927, AR073752, OD10707, AR060719, AR057235. Additional funding was provided by the Arthritis Foundation and the Nancy Taylor Foundation for Chronic Diseases.

Washington University School of Medicines 1,500 faculty physicians also are the medical staff of Barnes-Jewish and St. Louis Childrens hospitals. The School of Medicine is a leader in medical research, teaching and patient care, ranking among the top 10 medical schools in the nation by U.S. News & World Report. Through its affiliations with Barnes-Jewish and St. Louis Childrens hospitals, the School of Medicine is linked to BJC HealthCare.

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Gene therapy in mice builds muscle, reduces fat Washington University School of Medicine in St. Louis - Washington University School of Medicine in...

Recommendation and review posted by Bethany Smith

PHILADELPHIA, May 07, 2020 (GLOBE NEWSWIRE) -- Passage Bio, Inc. (NASDAQ: PASG), a genetic medicines company focused on developing transformative therapies for rare, monogenic central nervous system (CNS) disorders and the Gene Therapy Program (GTP) at the University of Pennsylvania (UPenn) today announced the expansion of their collaboration agreement to include an additional five programs and extending Passage Bios period to exercise new programs for an additional three years (through 2025). Additionally, Passage Bio will fund discovery research at GTP and will receive exclusive rights, subject to certain limitations, to technologies resulting from the discovery program for Passage Bio products developed with GTP, such as novel capsids, toxicity reduction technologies and delivery and formulation improvements.

Our collaboration with the GTP gives us access not only to the best discovery, technology, and research available but also to pioneering expertise in the field of gene therapy, including pre-clinical development and manufacturing experience that will help guide our programs as we move into clinical development, said Bruce Goldsmith, Ph.D., president and chief executive officer of Passage Bio. Expanding this collaboration provides us with the opportunity to not only deepen our pipeline but also strengthen our own expertise and capabilities as we strive to develop transformative gene therapies for patients. We are tremendously proud of the progress we have accomplished to date through this partnership and look forward to continuing this momentum in the years to come.

This expansion builds upon the original collaboration, which successfully established a strong partnership between Passage and GTP. Under the expanded agreement, Passage will pay $5 million annually to Penn to fund research across numerous technology applications for gene therapy. In addition to five additional program options and an extension of the relationship through 2025, Passage will receive exclusive rights, subject to certain limitations, to IP arising from this research and related indications that are applicable to the products it develops with GTP.

The partnership between GTP and Passage Bio continues to be extremely strong and productive as we collaborate to bring our gene therapy products to patients. We are extremely excited to expand the reach of our CNS products and discovery research through this continued collaboration, said James Wilson, M.D., Ph.D. director of the Gene Therapy Program at the University of Pennsylvania and chief scientific advisor of Passage Bio. As a co-founder of the company, I am also deeply committed to the growth and success of Passage. I believe that the expansion of this strong collaboration further establishes Passage Bios leadership in gene therapy and I look forward to continuing to work with our dedicated teams to reach these shared goals of helping patients with rare, monogenic CNS disorders.

About Passage Bio Passage Bio is a genetic medicines company focused on developing transformative therapies for rare, monogenic central nervous system disorders with limited or no approved treatment options. The company is based in Philadelphia, PA and has a research, collaboration and license agreement with the University of Pennsylvania and its Gene Therapy Program (GTP). The GTP conducts discovery and IND-enabling preclinical work and Passage Bio conducts all clinical development, regulatory strategy and commercialization activities under the agreement. The company has a development portfolio of six product candidates, with the option to license eleven more, with lead programs in GM1 gangliosidosis, frontotemporal dementia and Krabbe disease.

Forward Looking StatementThis press release contains forward-looking statements within the meaning of, and made pursuant to the safe harbor provisions of, the Private Securities Litigation Reform Act of 1995, including, but not limited to: our expectations about our collaborators and partners ability to execute key initiatives and the benefits and obligations associated with our arrangements with our collaborators and partners; and the ability of our lead product candidates to treat the underlying causes of their respective target monogenic CNS disorders. These forward-looking statements may be accompanied by such words as aim, anticipate, believe, could, estimate, expect, forecast, goal, intend, may, might, plan, potential, possible, will, would, and other words and terms of similar meaning. These statements involve risks and uncertainties that could cause actual results to differ materially from those reflected in such statements, including: our ability to develop, obtain regulatory approval for and commercialize our product candidates; the timing and results of preclinical studies and clinical trials; the risk that positive results in a preclinical study or clinical trial may not be replicated in subsequent trials or success in early stage clinical trials may not be predictive of results in later stage clinical trials; risks associated with clinical trials, including our ability to adequately manage clinical activities, unexpected concerns that may arise from additional data or analysis obtained during clinical trials, regulatory authorities may require additional information or further studies, or may fail to approve or may delay approval of our drug candidates; the occurrence of adverse safety events; failure to protect and enforce our intellectual property, and other proprietary rights; failure to successfully execute or realize the anticipated benefits of our strategic and growth initiatives; risks relating to technology failures or breaches; our dependence on collaborators and other third parties for the development of product candidates and other aspects of our business, which are outside of our full control; risks associated with current and potential delays, work stoppages, or supply chain disruptions caused by the coronavirus pandemic; risks associated with current and potential future healthcare reforms; risks relating to attracting and retaining key personnel; failure to comply with legal and regulatory requirements; risks relating to access to capital and credit markets; and the other risks and uncertainties that are described in the Risk Factors section in documents the company files from time to time with theSecurities and Exchange Commission(SEC), and other reports as filed with theSEC. Passage Bio undertakes no obligation to publicly update any forward-looking statement, whether written or oral, that may be made from time to time, whether as a result of new information, future developments or otherwise.

For further information, please contact:

Investors:Sarah McCabeStern Investor Relations, Inc.212-362-1200sarah.mccabe@sternir.com

Media:Emily MaxwellHDMZ312-506-5220emily.maxwell@hdmz.com

Financial Disclosure: The University of Pennsylvania and Dr. James Wilson are both co-founders of Passage Bio and hold equity interests in the company. Dr. Wilson is also the chief scientific advisor of the Company. Penn and GTP are the recipients of significant sponsored research support from the Company under research programs directed by Dr. Wilson. Penn has licensed or optioned numerous technologies to Passage Bio under an existing license and these ongoing sponsored research activities, and both Penn and Dr. Wilson stand to receive additional financial gains in the future under these licensing arrangements.

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Passage Bio Announces Expansion of Gene Therapy Collaboration with University of Pennsylvania - GlobeNewswire

Recommendation and review posted by Bethany Smith

Scientists at the Washington University School of Medicine in St. Louis, believe that gene therapy could one day be used as fat reducing therapy.

In a new study conducted on mice, scientists found that gene therapy helped build significant muscle mass quickly and reduced the severity of osteoarthritis in the mice without exercising more. Surprisingly, the therapy also staved off obesity, even when the mice ate an extremely high-fat diet.

Senior investigator Farshid Guilak, Ph.D., the Mildred B. Simon Research Professor of Orthopaedic Surgery and director of research at Shriners Hospitals for ChildrenSt. Louis said,Obesity is the most common risk factor for osteoarthritis. Being overweight can hinder a persons ability to exercise and benefit fully from physical therapy. Weve identified here a way to use gene therapy to build muscle quickly. It had a profound effect on the mice and kept their weight in check, suggesting a similar approach may be effective against arthritis, particularly in cases of morbid obesity.

Scientists gave 8-week-old mice a single injection of a virus conveying a gene called follistatin. The gene works to obstruct the action of a protein in muscle that keeps muscle growth in check. This empowered the mice to gain significant muscle mass without exercising more than usual.

Even without additional exercise, and while continuing to eat a high-fat diet, the muscle mass of these super mice more than doubled, and their strength nearly doubled, too. The mice also had less cartilage damage related to osteoarthritis, lower numbers of inflammatory cells and proteins in their joints, fewer metabolic problems, and healthier hearts and blood vessels than littermates that did not receive the gene therapy. The mice also were significantly less sensitive to pain.

During the study, scientists were concerned that some of the muscle growth might lead to being harmful. But, they found that heart function improved, as did cardiovascular health in general.

Although scientists think that long-term studies are required to determine the safety of this type of gene therapy, but, if safe, the strategy could be particularly beneficial for patients with conditions such as muscular dystrophy that make it challenging to build new muscle.

Guilak said,More traditional methods of muscle strengthening, such as lifting weights or physical therapy, remain the first line of treatment for patients with osteoarthritis. Something like this could take years to develop. Still, were excited about its prospects for reducing joint damage related to osteoarthritis, as well as possibly being useful in extreme cases of obesity.

Link:
Study suggests effective fat-reducing therapy - Tech Explorist

Recommendation and review posted by Bethany Smith

A new business intelligence report released by CMI with the title Global Gene Therapy for Rare Disease Market Research Report 2020-2027 is designed covering micro level of analysis by manufacturers and key business segments. The Global Gene Therapy for Rare Disease Market survey analysis offers energetic visions to conclude and study market size, market hopes, and competitive surroundings. The research is derived through primary and secondary statistics sources and it comprises both qualitative and quantitative detailing.

Whats keeping Kite Pharma, Inc. (Gilead Sciences, Inc.), Novartis International AG, Juno Therapeutics Inc. (Celgene Corporation), Bluebird Bio, Inc., Spark Therapeutics, Inc., uniQure N.V, Orchard Therapeutics Plc., PTC Therapeutics, Inc., and BioMarin Pharmaceutical Inc. Ahead in the Market? Benchmark yourself with the strategic moves and findings recently released by CMI.

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

Market Overview of Global Gene Therapy for Rare Disease

If you are involved in the Global Gene Therapy for Rare Disease industry or aim to be, then this study will provide you inclusive point of view. Its vital you keep your market knowledge up to date segmented by Applications and major players. If you have a different set of players/manufacturers according to geography or needs regional or country segmented reports we can provide customization according to your requirement.

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Historical year 2015 2019

Base year 2019

Forecast period** 2020 to 2027 [** unless otherwise stated]

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Regions included:

North America (United States, Canada, and Mexico)

Europe (Germany, France, UK, Russia, and Italy)

Asia-Pacific (China, Japan, Korea, India, and Southeast Asia)

South America (Brazil, Argentina, Colombia)

Middle East and Africa (Saudi Arabia, UAE, Egypt, Nigeria, and South Africa)

Read Detailed Index of full Research Study at https://www.coherentmarketinsights.com/market-insight/gene-therapy-for-rare-disease-market-2321

Important Features that are under offering & key highlights of the report:

Detailed overview of Gene Therapy for Rare Disease market

Changing market dynamics of the industry

In-depth market segmentation by Type, Application etc

Historical, current and projected market size in terms of volume and value

Recent industry trends and developments

Competitive landscape of Gene Therapy for Rare Disease market

Strategies of key players and product offerings

Potential and niche segments/regions exhibiting promising growth

A neutral perspective towards Gene Therapy for Rare Disease market performance

Market players information to sustain and enhance their footprint

Buy This Complete A Business Report: https://www.coherentmarketinsights.com/insight/buy-now/2321

Major Highlights of TOC:

Chapter One: Global Gene Therapy for Rare Disease Market Industry Overview

1.1 Gene Therapy for Rare Disease Industry

1.1.1 Overview

1.1.2 Products of Major Companies

1.2 Gene Therapy for Rare Disease Market Segment

1.2.1 Industry Chain

1.2.2 Consumer Distribution

1.3 Price & Cost Overview

Chapter Two: Global Gene Therapy for Rare Disease Market Demand

2.1 Segment Overview

2.1.1 APPLICATION 1

2.1.2 APPLICATION 2

2.1.3 Other

2.2 Global Gene Therapy for Rare Disease Market Size by Demand

2.3 Global Gene Therapy for Rare Disease Market Forecast by Demand

Chapter Three: Global Gene Therapy for Rare Disease Market by Type

3.1 By Type

3.1.1 TYPE 1

3.1.2 TYPE 2

3.2 Gene Therapy for Rare Disease Market Size by Type

3.3 Gene Therapy for Rare Disease Market Forecast by Type

Chapter Four: Major Region of Gene Therapy for Rare Disease Market

4.1 Global Gene Therapy for Rare Disease Sales

4.2 Global Gene Therapy for Rare Disease Revenue & market share

Chapter Five: Major Companies List

Chapter Six: Conclusion

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Key questions answered

o Who are the Leading key players and what are their Key Business plans in the Global Gene Therapy for Rare Disease market?

o What are the key concerns of the five forces analysis of the Global Gene Therapy for Rare Disease market?

o What are different prospects and threats faced by the dealers in the Global Gene Therapy for Rare Disease market?

o What are the strengths and weaknesses of the key vendors?

Thanks for reading this article; you can also get individual chapter wise section or region wise report version like North America, Europe or Asia.

About the Author:

Coherent Market Insights is a prominent market research and consulting firm offering action-ready syndicated research reports, custom market analysis, consulting services, and competitive analysis through various recommendations related to emerging market trends, technologies, and potential absolute dollar opportunity.

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Name: Mr.ShahCoherent Market Insights 1001 4th Ave,#3200 Seattle, WA 98154, U.S.Phone: US +1-206-701-6702/UK +44-020 8133 4027[emailprotected]

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Gene Therapy for Rare Disease Market 2020 Coronavirus (Covid-19) Business Impact 2026 Growth Trends by Manufacturers, Regions, Type and Application,...

Recommendation and review posted by Bethany Smith

The smid-cap biotech earnings deluge hit Wall Street in the week ended May 9, positively impacting the stocks in the sector. The iShares NASDAQ Biotechnology Index (NASDAQ: IBB) gained about 6% for the week.

Large-cap pharma names AstraZeneca plc (NYSE: AZN) and Novartis AG (NYSE: NVS) received FDA nods for their heart failure and lung cancer therapies, respectively. After receiving emergency use authorization from the FDA in the U.S., Gilead Sciences, Inc.'s (NASDAQ: GILD) remdesivir obtained full regulatory approval in Japan.

Moderna Inc (NASDAQ: MRNA) received the OK toproceedwith the Phase 2 trial of its mRNA coronavirus vaccine candidate mRNA-1273.

The following are the key events and catalysts that biotech investors need to watch in the coming week.

Clovis Oncology Inc (NASDAQ: CLVS) awaits the FDA nod for an expanded indication for its cancer therapy Rubraca. The sNDA seeks approval of Rubraca as a monotherapy treatment for patients with BRCA1/2-mutant recurrent, metastatic castrate-resistant prostate cancer.

Abeona Therapeutics Inc (NASDAQ: ABEO) is due to present updated interim results from the Transpher A and Transpher B studies, Phase 1/2 trials of ABO-102 and ABO-101, respectively, in mucopolysaccharidosis type IIIA, aka as Sanfilippo syndrome type.

Rocket Pharmaceuticals Inc (NASDAQ: RCKT will present updated data from Phase 1/2 FANCOLEN-I study, which is evaluating the safety and efficacy of infusion of autologous CD34 + cells transduced with a lentiviral vector carrying the FANCA gene in patients with Fanconi anemia subtype A, and updates from the Phase 1 LAD-I study that is evaluating its investigational gene therapy RP-L201 to treat severe Leukocyte Adhesion Deficiency-I.

Avrobio Inc (NASDAQ: AVRO) is scheduled to make an oral presentation on new data from the Phase 2 trial of AVR-RD-01 for Fabry (Wednesday). The company will also make an oral presentation of new data from the collaborator-sponsored Phase 1/2 clinical trial of AVR-RD-04 in cystinosis. Another oral presentation on new data from a preclinical research program for a gene therapy for Pompe disease is also scheduled for Wednesday.

Ultragenyx Pharmaceutical Inc (NASDAQ: RARE) is due to present updated data from the first three cohorts of a Phase 1/2 study of DTX301 in treating ornithine transcarbamylase deficiency (Wednesday). The company will also present updated data from the confirmatory cohort from a Phase 1/2 study of DTX401 in glycogen storage disease Type 1a (Friday).

Pfizer Inc. (NYSE: PFE) will present Phase 1b data for PF-06939926 in Duchenne muscular dystrophy on Friday.

Krystal Biotech Inc (NASDAQ: KRYS) will present a poster on KB407, an HSV-1 based gene therapy vector, for the treatment of cystic fibrosis.

See Also: Gilead Works To 'Maximize Global Supply' Of Coronavirus Candidate Remdesivir Amid Threat Of Patent Loss

Genocea Biosciences Inc (NASDAQ: GNCA) will host a KOL symposium with a live Q&A for analysts and investors to reflect on the progress of the T cell therapy landscape and provide an in-depth profile of GEN-011 Genocea's neoantigen cell therapy. (Tuesday)

Krystal Biotech is due to present at the SID meeting withresults froma Phase 1/2 study of in vivo gene therapy KB105 for treating autosomal recessive congenital ichthyosis as well as results of a Phase 1/2 trial that is evaluating in vivo correction of dystrophic epidermolysis bullosa by direct cutaneous COL7A1 gene replacement.

Caladrius Biosciences Inc (NASDAQ: CLBS) will present at the SCAI meeting Thursday with full data from the ESCaPE-CMD study of CLBS16 for the treatment of coronary microvascular dysfunction.

Constellation Pharmaceuticals Inc (NASDAQ: CNST) said abstracts of a presentation due at the June 11-14 European Hematology Association meeting will be made available Thursday. The abstract pertains to an interim update from the MANIFEST Phase 2 study that is evaluating CPI-0610 in myelofibrosis.

Auris Medical Holding Ltd (NASDAQ: EARS) is due to release top-line data in early May from the Phase 1b trial that is evaluating AM-201 in healthy volunteers. AM-201 is the company's investigational drug for the prevention of antipsychotic-induced weight gain and somnolence.

Monday

Tuesday

Wednesday

Thursday

Friday

ORIC Pharmaceuticals Inc (NASDAQ: ORIC)

2020 Benzinga.com. Benzinga does not provide investment advice. All rights reserved.

Excerpt from:
The Week Ahead In Biotech: Virtual Conference Presentations, Clovis PDUFA Date In The Spotlight - Benzinga

Recommendation and review posted by Bethany Smith

PTC Therapeutics is acquiring Censa Pharmaceuticals in a cash and stock deal. PTC, based in South Plainfield, New Jersey, focuses on rare diseases. Censa, based in Wellesley, Massachusetts, is developing CNSA-001 for phenylketonuria and other metabolic diseases.

Under the terms of the deal, PTC is paying Censa $10 million in cash and up to 850,000 shares of PTC common stock, which as of Tuesday, May 5, was trading at $48 per share, totaling about $41 million. In addition, there are development and regulatory milestones for the two most advanced programs that can reach $217.5 million. There is also receipt of a priority review voucher and $30 million to be paid in either cash or PTC common stock after completing the enrollment of a Phase III clinical trial for CNSA-001, $109 million in development and regulatory milestones for each extra indication for CNSA-001, net sales milestones up to about $160 million and a contingent value payment of a percentage of annual net sales in the single to low double digits.

Results from a Phase II clinical trial of CNSA-001 demonstrated significant and clinically relevant reductions in phenylalanine levels compared to current first-line treatment, said Stuart W. Peltz, PTCs chief executive officer. We believe that CNSA-001 has the potential to address the majority of PKU patients whose condition is not adequately managed by current treatments. We look forward to initiating a Phase III study in PKU so that patients diagnosed with this devastating condition can have a new oral treatment option as soon as possible.

Phenylketonuria (PKU) is an inborn metabolic disorder that results in decreased metabolism of the amino acid phenylalanine. If untreated, it can cause intellectual disability, seizures, behavioral issues, and mental disorders. It is caused by a mutation in the gene that codes for the enzyme needed to metabolize phenylalanine. Patients with PKU must eat a diet that limits phenylalanine, found mostly in protein.

There are two drugs, both commercialized by BioMarin Pharmaceutical, approved for PKU. Sapropterin hydrochloride (Kuvan), an oral formulation, was approved in 2007 with a diet low in phenylalanine. In a Phase II trial, Censas drug was superior to Kuvan.

BioMarins Palynziq (pegvaliase-pqpz) was approved in 2018 by the U.S. Food and Drug Administration (FDA). It is an injectable enzyme therapy approved for adults with PKU whose blood phenylalanine concentrations remain high on current treatment. BioMarin planned to begin clinical testing of a gene therapy for the disease this year, but the COVID-19 pandemic has delayed the launch, although the company indicates it hopes to begin in the second half of 2020.

CNSA-001 is an oral formulation of synthetic sepiapterin, a precursor to intracellular tetrahydrobiopterin. Intracellular tetrahydrobiopterin is a critical enzymatic cofactor involved in the metabolism and synthesis of various metabolic products.

PTCs pipeline includes early research in gene therapy, nonsense mutation, splicing and oncology. The therapies are focused on Huntingtons disease, Angelman syndrome, relapsed/refractory acute leukemias, ovarian cancer and others. The company markets Emflaza, a treatment for Duchenne muscular dystrophy (DMD) in the U.S., and markets another DMD drug, Translarna, in Europe. It also markets Tegsedi for polyneuropathy of hereditary transthyretin-mediated amyloidosis in Latin America.

PTC, with Roche, is awaiting an approval by the FDA this year for risdiplam for spinal muscular atrophy (SMA).

I am proud of the team at Censa and its achievements to date demonstrating the potential role of CNSA-001 in treating diseases of the BH4 pathway, said Jonathan Reis, president and chief executive officer of Censa. It is the right time to have an excellent fully integrated, patient-focused biotechnology organization like PTC Therapeutics take over the late-stage development of CNSA-001 so that this promising compound becomes available to patients in the near future.

Read this article:
PTC Therapeutics to Acquire Censa for $10 Million in Cash, $41 Million in Stock and More - BioSpace

Recommendation and review posted by Bethany Smith

SHANGHAI, China, May 10, 2020 (GLOBE NEWSWIRE) -- Burning Rock and Illumina (NASDAQ: ILMN), a global leader in gene sequencing and array-based technologies, jointly announced today that they are joining forces to promote the development and standardization of NGS-based cancer therapy selection in China based on Illuminas NextSeqTM 550Dx system.

In 2015, Illuminas sequencing technology and Burning Rocks development and commercial capabilities were for the first time combined, providing Chinas precision oncology market with advanced NGS-based cancer therapy selection solutions in the past five years. In 2020, as the first genetic testing company in China to achieve development of in vitro diagnostic (IVD) tests for both circulating tumor DNA (ctDNA) and tissue based on the NextSeqTM 550Dx system agreement with Illumina, Burning Rock will further expand and deepen the application of NGS technologies in the field of cancer therapy selection.

The value of NGS application in precision medicine and companion diagnostics has been widely recognized by clinical experts and cancer patients. Compared with traditional genetic testing methods, NGS-based cancer therapy selection allows patients to understand the mutation of multiple genes related to cancer treatment, providing doctors and patients with one-stop targeted therapy and immunotherapy drugs testing solutions, ultimately saving time and preserving samples.

In July 2018, Burning Rocks innovative product "Human EGFR / ALK / BRAF / KRAS gene mutation detection kit (reversible end termination sequencing method)" based on Illumina sequencing system became the first NGS-based reagent kit to be approved by National Medication Products Administration (NMPA). Since then, tumor NGS testing can be officially used in Chinese hospitals. In the future, Burning Rock will continue to seek NMPA approvals for its IVD products based on NextSeqTM 550Dx and other sequencing systems to promote the implementation of tumor NGS products in hospitals and benefit more cancer patients.

Through our long-term, close and pleasant cooperation with Illumina, we have delivered the world's leading NGS-based therapy selection solutions for clinical oncology, and improved the development and application of NGS-based cancer therapy selection in China. said Mr. Han Yusheng, founder and CEO of Burning Rock. Today we are delighted to announce that Burning Rock and Illumina will further deepen cooperation based on the NextSeqTM 550Dx system, to provide more high-quality molecular diagnostic solutions for clinical oncology treatment and promote the standardization of NGS-based cancer therapy selection in China.

Burning Rock is one of the leading precision oncology companies in China, said Joydeep Goswami, Senior Vice President of Corporate Development and Strategic Planning at Illumina, said. I am pleased to see that during the close cooperation with Illumina in the past five years, Burning Rock has continuously developed tumor diagnosis solutions that meet the needs of the local market. The new agreement is a testament to our strong cooperation in the past, and also the beginning of a more in-depth cooperation.

Li Qing, General Manager of Greater China at Illumina, said: Burning Rock has brought hope to countless Chinese patients by providing a series of tumor molecular diagnostic solutions. And we are very happy to be involved. In the future, I firmly believe that genetic testing technology will further change the current treatment paradigm for cancer and provide critical support to conquer this disease at an early date.

About Burning RockBurning Rock, whose mission is to Guard Life via Science, focuses on the application of next generation sequencing (NGS) technology in the field of precision oncology. Its business consists of i) NGS-based therapy selection testing for late-stage cancer patients, with the leading market share in China and over 185,000 tissue and liquid-based tests completed cumulatively, and ii) NGS-based cancer early detection, which has moved beyond proof-of-concept R&D into the clinical validation stage.

About IlluminaIllumina is improving human health by unlocking the power of the genome. Our focus on innovation has established us as the global leader in DNA sequencing and array-based technologies, serving customers in the research, clinical and applied markets. Our products are used for applications in the life sciences, oncology, reproductive health, agriculture and other emerging segments. To learn more, visit http://www.illumina.comand follow @illumina.

Read more from the original source:
Burning Rock deepens cooperation with Illumina to promote development and standardization of NGS-based cancer therapy selection in China -...

Recommendation and review posted by Bethany Smith

Research report on Viral Vector Manufacturing Market size | Industry Segment by Applications, by Type, Regional Outlook, Market Demand, Latest Trends, Viral Vector Manufacturing Industry Share & Revenue by Manufacturers, Company Profiles, Growth Forecasts 2025. Analyzes current market size and upcoming 5 years growth of this industry.

Report Covers Global Industry Analysis, Size, Share, CAGR, Trends, Forecast And Business Opportunity.

Download Premium Sample Copy Of This Report: https://brandessenceresearch.biz/Request/Sample?ResearchPostId=473&RequestType=Sample

Global Viral Vector Manufacturing Market to reach USD 1.24 billion by 2025.

Global Viral Vector Manufacturing Market valued approximately USD 225.44 million in 2016 is forecasted to grow with a healthy growth rate of more than 20.84% over the forecast period 2018-2025. The major factors speculated to augment the markets are availability of funding for the advancement of gene therapy, and increasing frequency of cancer, genetic disorders & infectious diseases. The risk of undesirable outcomes including mutagenesis are major challenges for the global market. Viral vectors are tools commonly used by molecular biologists to deliver genetic material into cells. This process can be performed inside a living organism (in vivo) or in cell culture (in vitro). Molecular biologists first harnessed this machinery in the 1970s.Global Viral Vector Manufacturing Market is segmented based on Type, Disease, Application, and Industry. The Adeno-associated subsegment segment of Type segment is forecasted to grow with highest CAGR while the Cancers subsegment is expected to dominate in terms of market share. Gene Therapy subsegment is also expected to achieve highest growth rate whereas Biopharmaceutical & Pharmaceutical Companies subsegment would remain dominant in market share size.The regional analysis of Global Viral Vector Manufacturing Market is considered for the key regions such as Asia Pacific, North America, Europe, Latin America and Rest of the World. North America is the leading region across the world. Whereas, owing to rising no. of research activities in countries such as China, India, and Japan, Asia Pacific region is also expected to exhibit higher growth rate / CAGR over the forecast period 2018-2025. The objective of the study is to define market sizes of different segments & countries in recent years and to forecast the values to the coming eight years. The report is designed to incorporate both qualitative and quantitative aspects of the industry within each of the regions and countries involved in the study. Furthermore, the report also caters the detailed information about the crucial aspects such as driving factors & challenges which will define the future growth of the market. Additionally, the report shall also incorporate available opportunities in micro markets for stakeholders to invest along with the detailed analysis of competitive landscape and product offerings of key players. The detailed segments and sub-segment of the market are explained below:

By Type:

oAdenoviraloRetroviraloAdeno-associatedoOthers

By Disease:

oGenetic DisordersoInfectious diseasesoCancersoOthers

By Application:

oVaccinologyoGene Therapy

By End-Use:

oResearch InstitutesoBiopharmaceutical & Pharmaceutical CompaniesoOthers

By Regions:oNorth AmericaoU.S.oCanadaoEuropeoUKoGermanyoAsia PacificoChinaoIndiaoJapanoLatin AmericaoBraziloMexicooRest of the World

Furthermore, years considered for the study are as follows:

Historical year 2015, 2016Base year 2017Forecast period 2018 to 2025

The industry is seeming to be fairly competitive. Some of the leading market players CGT Catapult, Lonza, uniQure, Merck, Cobra Biologics, Oxford BioMedica, FUJIFILM Diosynth Biotechnologies, Novasep, Spark Therapeutics, Kaneka Eurogentec, Brammer Bio, Massbiologics, Finvector Vision Therapies, Regenxbio, Thermo Fisher Scientific, Inc., Sanofi, Shenzhen SiBiono GeneTech Co., Ltd., and so on. The fierce competitiveness has made these players spend in product developments to improve the customers requirements.

Target Audience of the Viral Vector Manufacturing Market Study:

oKey Consulting Companies & AdvisorsoLarge, medium-sized, and small enterprisesoVenture capitalistsoValue-Added Resellers (VARs)oThird-party knowledge providersoInvestment bankersoInvestors

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Table of Content:

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

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

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

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

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

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

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

About Us:

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

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Recommendation and review posted by Bethany Smith

For people with rare diseases, a single gene therapy treatment could restore normal function and alleviate the burden of ongoing care, as Dr Ian Winburn tells Kennas Fitzsimons

Dr Ian Winburn

Gene therapy is the next generation of medicine that targets the underlying cause of genetic diseases. It has the potential to offer patients a really transformational clinical benefit and improve quality of life.

Thats according to Dr Ian Winburn, Global Medical Lead, Haemophilia, Endocrine and Inborn Errors of Metabolism (IEM), Rare Diseases, Pfizer Biopharmaceuticals Group. Dr Ian Winburn, Global Medical Lead, Haemophilia, Endocrine and IEM, Pfizer Biopharmaceuticals Group, pictured right during his presentation on gene therapy at BioPharma Ambition held in Dublin Castle on March 4. Pic: Conor McCabe Photography.

Formerly a clinician in the UK National Health Service (NHS), Dr Winburn trained in general surgery and completed a PhD on novel drug discovery in renal transplantation before moving into industry 10 years ago, where he worked in the area of inflammation and immunology before leading the European haemophilia team.

Dr Winburn is now working to develop innovative gene therapies with the potential to restore normal function to patients with rare diseases, possibly with just a single treatment, changing the way people manage their disease.

Gene therapy: What is it?Gene therapy uses genes as medicine. It works by introducing functioning copies of missing or defective genes into the body and can target the underlying cause of a disease at the cellular level.

There are various types of gene therapies, such as the gene editing technique, CRISPR (clustered regularly interspaced short palindromic repeats), as well as epigenetic approaches that look at ways in which genes may be turned on or turned off.

Pfizer Rare Disease is focusing on an in-vivo approach that utilises a recombinant adeno-associated virus (AAV) to deliver the gene therapy.

This approach works by targeting the missing or non-functional gene in an individuals DNA and adding a copy of it with a functioning gene that, in turn, produces a functioning protein.

The functioning gene serves as a blueprint for the tissue to create the missing or non-functioning protein that is causing a disease.

Dr Winburn said: Gene therapy is in the branch of genetic medicine, where you can think about approaches that look to add a gene to a host cell, and that gene goes on and codes for a protein. That protein its coding for can replace a missing protein. So, in the example of haemophilia, where theres a missing factor VIII or factor IX clotting factor, that protein that is either missing or is faulty could be essentially administered through a gene therapy. A gene is added to a host cell that codes for the factor VIII or factor IX and therefore replaces that protein.

The functioning gene is delivered directly to the targeted cells by means of a highly specialised viral vector. This vector, effectively, is the package that contains the gene. In simple terms, it can be likened to the cardboard boxes that online retailers use to ship products.

The manufactured vectors are protein shells modelled after viruses in which all infectious viral components have been removed, and a functioning gene is added. Different viral vectors are used to reach specific tissues in the body, such as the liver or muscle.

VectorVector is a great word because vector describes a direction, by definition, and the other way we can think about vector is a vector often carries something. There are a few approaches you can use to develop a vector. We have embraced an AAV vector that has the capacity to deliver the transgene, the gene that is going to be added to the host somatic cell. In the case of haemophilia, it is targeted at the liver, Dr Winburn says.

Rare diseases focusAbout 280 million people worldwide live with a genetic disease, and more than 80 per cent of rare diseases are genetic in origin, according to Pfizer. For people born with rare diseases, the burden of disease management can be huge. Treatment is often ongoing and may be lifelong. Gene therapy could enable patients to live without the need for ongoing treatment. This raises the prospect of relief not only from symptoms but also from the burden of disease management.

Dr Winburn elaborates on the reasons why gene therapy approaches are currently focused specifically on rare diseases that have single-gene alterations.

It tends to be rare genetic diseases that are monogenic in nature Some of the more common diseases are very much multifactorial in origin: there may be a genetic component but there are other aspects to their aetiology rather than these single, monogenic conditions that gene therapy really lends itself to.

The other aspect is that these are areas of huge unmet medical need. Often, there isnt a high standard of care with either medicines or clinical interventions that are ultimately influencing the progression and the symptoms of the disease, he says.

A lot of rare diseases often affect children by the very nature of their being of genetic origin. In some cases, children dont get the opportunity to grow up into adulthood because of these rare diseases. Having the opportunity to develop medicines where there is such a high level of unmet need and, ultimately, impact in a positive way the lives of families and their carers is a huge motivation.

Dr Winburn adds that rare diseases, collectively, are common. There are approximately 7,000 rare diseases, and the majority of these are of genetic origin. Gene therapy offers a groundbreaking technology to address these genetic diseases that have historically not had particularly strong standards of care or clinical treatment paradigms offered to them.

Gene therapy for haemophiliaPeople with the genetic disorder of haemophilia have insufficient levels of a clotting factor that helps to stop bleeding. Consequently, they bleed for longer than other people. The disease is typically treated through infusions of the missing clotting factor, with patients undergoing regular replacement therapy. Gene therapy could revolutionise this treatment model.

Its really important to put yourself in the position of a parent who has a young child who has haemophilia, Dr Winburn says.

Often, this disorder of coagulation that results in spontaneous bleeding due to the lack of functioning clotting factor first presents as early as the age of two, classically when children are becoming toddlers, when they start bumping into things and they develop bruising and the likes.

That alerts their parents attention to the possibility that there is something wrong with their clotting system and they [undergo] clinical tests and a diagnosis is made. Or, because its a genetic condition, it may run in families and parents are aware of the possibility of their newborn having haemophilia.

But if you are diagnosed, for example, at the age of two, it means that the mainstay of treatment is factor replacement. So, that commonly is an intravenous infusion possibly two to three times a week, possibly once a week, or once every other week, depending on whether its haemophilia A or B and what type of medicine is being prescribed. But its certainly frequent treatments. Again, if you put that back to a parent wholl be doing those infusions from the age of two or three that lifelong need and burden is huge.

While factor replacement enables children to live a full and active life to a degree, children with haemophilia may not necessarily get the opportunity to engage in all the activities children typically partake in as they are growing up, such as contact sports, Dr Winburn says.

There is this ongoing, lifelong burden of treatment. As those boys transition into adulthood, they often take responsibility for that and if they dont get their treatment then they will bleed spontaneously into their joints, they get problems with haemarthropathy, causing damage.

Ultimately, the incidence of joint damage and joint replacement surgeries is incredibly significant in the haemophilia population. And that is often despite optimal prophylaxis, where its being prescribed.

So, when you think about gene therapy, this is a single, one-off treatment with the potential to alleviate the need for regular infusion for a patient.

Not all patients will be eligible for a gene therapy or are being studied in gene therapy trials. This is not going to be something thats available for everybody. But for those that are eligible, and ultimately in disease where a gene therapy has been licensed by the regulator, this really does have the potential to massively impact their lives and give them a sense of normality that they havent necessarily had up until that point in time.

Future expectationsWhile gene therapy holds promise for many people with genetic diseases, it will not be an appropriate solution for every patient. The potential risks and benefits of gene therapy will be fully established through clinical trial programmes and with continued research and evaluation.

Patient safety and suitability are always primary considerations in the development of new treatments as they progress from preclinical and clinical testing through regulatory approval to potential commercial distribution. Dr Winburn stresses that, as regards the development of new gene therapies, patient safety is paramount.

Safety is always at the forefront of our thinking, it is the heart of our clinical trial programmes, it is the heart of all our regulatory work.

It is an ongoing process around evaluating safety, and particularly long-term safety, and there is a critical importance for all patients that ultimately receive a gene therapy to be followed up long-term within registries, within clinical databases, so that we can monitor and evaluate long-term safety. All our trials are designed so that safety outcomes are critically part of it and its something that we are ever watchful of.

For some patients, gene therapy is already a reality. There are currently a few rare diseases for which gene therapies are available as therapeutic options in Europe, and Dr Winburn anticipates that there may be up to 30 approved by 2023.

There is a rare congenital cause of blindness that currently has a gene therapy available and similarly a rare neurological condition that affects children also has a gene therapy available, thats spinal muscular atrophy (SMA). There is also a gene therapy for beta thalassaemia that has recently been approved and is available within Europe. In terms of haemophilia, the first gene therapy is currently under review in Europe.

It may be premature to imagine a scenario whereby gene therapy is used to treat chronic diseases more generally, but in terms of future applications for these emerging technologies it is a case of watch this space.

This is an area where we are definitely in breakthrough technology. At this moment in time, our focus has been on rare diseases. There is, of course, an interest in understanding what is possible with gene therapy in terms of where it could be utilised, Dr Winburn says.

I dont want to provide any false hopes, but I think aspirationally, there is a hope that this could certainly impact many patients and their families in a positive way.

In association with Pfizer Biopharmaceuticals Group.

Here is the original post:
Gene therapy: The 'next generation' of medicine - Irish Medical Times

Recommendation and review posted by Bethany Smith

publication date: May. 8, 2020

FDA has granted accelerated approval to Tabrecta (capmatinib) for adult patients with metastatic non-small cell lung cancer whose tumors have a mutation that leads to mesenchymal-epithelial transition exon 14 skipping as detected by an FDA-approved test.

Tabrecta is the first FDA-approved therapy to treat NSCLC with specific mutations (those that lead to mesenchymal-epithelial transition or MET exon 14 skipping).

Tabrecta is sponsored by Novartis.

FDA also approved the FoundationOne CDx assay (Foundation Medicine, Inc.) as a companion diagnostic for Tabrecta. Most patients had tumor samples that were tested for mutations that lead to MET exon 14 skipping using local tests and confirmed with the F1CDx, which is a next-generation sequencing based in vitro diagnostic device capable of detecting several mutations, including mutations that lead to MET exon 14 skipping.

Lung cancer is increasingly being divided into multiple subsets of molecularly defined populations with drugs being developed to target these specific groups, Richard Pazdur, director of the FDA Oncology Center of Excellence and acting director of the Office of Oncologic Diseases in the FDAs Center for Drug Evaluation and Research, said in a statement. Tabrecta is the first approval specifically for the treatment of patients with non-small cell lung cancer whose tumors have mutations that lead to MET exon 14 skipping. This patient population now has an option for a targeted therapy, which they didnt have prior to today.

Efficacy was demonstrated in the GEOMETRY mono-1 trial (NCT02414139), a multicenter, non-randomized, open-label, multicohort study enrolling 97 patients with metastatic NSCLC with confirmed MET exon 14 skipping. Patients received Tabrecta 400 mg orally twice daily until disease progression or unacceptable toxicity.

The main efficacy outcome measures were overall response rate (ORR) determined by a blinded independent review committee using RECIST 1.1 and response duration. Among the 28 treatment-nave patients, the ORR was 68% (95% CI: 48, 84) with a response duration of 12.6 months (95% CI: 5.5, 25.3). Among the 69 previously treated patients, the ORR was 41% (95% CI: 29, 53) with a response duration of 9.7 months (95% CI: 5.5, 13.0).

FDA approves daratumumab and hyaluronidase-fihj for multiple myeloma

FDA has approved daratumumab and hyaluronidase-fihj (Darzalex Faspro) for adult patients with newly diagnosed or relapsed/refractory multiple myeloma. This new product allows for subcutaneous dosing of daratumumab.

Darzalex Faspro is sponsored by Janssen Biotech Inc.

Daratumumab and hyaluronidase-fihj is approved for the following indications that intravenous daratumumab had previously received:

in combination with bortezomib, melphalan and prednisone in newly diagnosed patients who are ineligible for autologous stem cell transplant,

in combination with lenalidomide and dexamethasone in newly diagnosed patients who are ineligible for autologous stem cell transplant and in patients with relapsed or refractory multiple myeloma who have received at least one prior therapy,

in combination with bortezomib and dexamethasone in patients who have received at least one prior therapy,

as monotherapy, in patients who have received at least three prior lines of therapy including a proteasome inhibitor and an immunomodulatory agent or who are double-refractory to a PI and an immunomodulatory agent.

Efficacy of daratumumab and hyaluronidase-fihji (monotherapy) was evaluated in COLUMBA (NCT03277105), an open-label non-inferiority trial randomizing 263 patients to daratumumab and hyaluronidase-fihj and 259 to intravenous daratumumab (daratumumab IV). The trials co-primary endpoints were overall response rate and pharmacokinetic endpoint of the maximum Ctrough on cycle 3, day 1 pre-dose. Daratumumab and hyaluronidase-fihj was non-inferior to daratumumab IV in evaluating these two endpoints.

The ORR was 41.1% for daratumumab and hyaluronidase-fihj and 37.1% for daratumumab IV with a risk ratio of 1.11 (95% CI: 0.89, 1.37). The geometric mean ratio comparing daratumumab and hyaluronidase-fihj to daratumumab IV for maximum Ctrough was 108% (90% CI: 96,122).

Efficacy of daratumumab and hyaluronidase-fihj in combination with VMP (D-VMP) was evaluated in a single-arm cohort of PLEIADES (NCT03412565), a multi-cohort, openlabel trial. Eligible patients were required to have newly diagnosed multiple myeloma and were ineligible for transplant. The major efficacy outcome measure, ORR, was 88.1% (95% CI: 77.8, 94.7).

Efficacy of daratumumab and hyaluronidase-fihj in combination with Rd (D-Rd) was evaluated in a single-arm cohort of this trial. Eligible patients had received at least one prior line of therapy. ORR was 90.8% (95% CI: 81.0, 96.5).

FDA accepts NDA for CC-486 in AML indication

FDA has accepted a New Drug Application for CC-486, an investigational oral hypomethylating agent, for the maintenance treatment of adult patients with acute myeloid leukemia who achieved complete remission, or CR with incomplete blood count recovery, following induction therapy with or without consolidation treatment, and who are not candidates for, or who choose not to proceed to, hematopoietic stem cell transplantation.

CC-486 is sponsored by Bristol Myers Squibb. FDA granted the application Priority Review and set a Prescription Drug User Fee Act goal date of Sept. 3, 2020.

The NDA submission was based on the efficacy and safety results of the phase III QUAZAR AML-001 study, which met the primary endpoint of improved overall survival for patients receiving AML maintenance treatment with CC-486 versus placebo.

Often, newly diagnosed adult patients with AML achieve a complete response with induction therapy, however many patients will relapse and experience a poor outcome. Patients in remission are seeking treatment options that decrease the likelihood of relapse and extend overall survival, Noah Berkowitz, senior vice president of Global Clinical Development, Hematology, at Bristol Myers Squibb, said in a statement.

CC-486 is an investigational therapy that is not approved for any use in any country.

Caris Life Sciences submits two PMA applications to FDA for whole exome and whole transcriptome sequencing

Caris Life Sciences has submitted two Pre-Market Approval applications for MI Exome CDx and MI Transcriptome CDx to FDA.

MI Exome CDx, whole exome sequencing (DNA), and MI Transcriptome CDx, whole transcriptome sequencing (RNA), are precision medicine assays that include key companion diagnostic biomarkers with therapy claims, and detect all classes of alterations including genomic signatures for microsatellite instability, tumor mutation burden, and loss of heterozygosity.

MI Exome CDx is a next-generation sequencing-based test utilizing DNA isolated from formalin-fixed paraffin embedded tumor tissue specimens for the qualitative detection of genomic alterations. MI Exome CDx can identify genetic variants (single nucleotide variants, insertions and deletions), copy number alterations, MSI, TMB and LOH.

MI Transcriptome CDx is a next-generation sequencing-based test that utilizes RNA isolated from formalin-fixed paraffin embedded tumor tissue specimens for the qualitative detection of genomic and transcriptomic alterations. MI Transcriptome CDx is a broad, multi-gene panel utilized to identify gene fusions, transcript variants, genetic variants (single nucleotide variants, insertions and deletions), and gene expression changes. FDA granted MI Transcriptome CDx received Breakthrough Device designation in 2019.

See the rest here:
FDA approves Tabrecta, first targeted therapy to treat metastatic NSCLC - The Cancer Letter

Recommendation and review posted by Bethany Smith