Archive for May, 2020

The Pixion Market Research offers complete overview of the Global Bone Marrow Transplant market with marketing knowledge on the basis of recorded data for marketing decision makers. Report also focuses on all the important aspects of the industry such as new models, opportunities and trends which enable more effective marketing decision making and theories with empirical insights from marketing study. Hence the report is beneficial for the readers as it informs about the crucial parameters and market developments in order to take steps accordingly and make marketing strategies.

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The Research makes some important proposals for new projects of the industry before evaluating its feasibility. The report is also inclusive of different business models, quantitative analysis on the basis of various analytical tools. Hence the market size of the Global Bone Marrow Transplant market is estimated over the forecast period. CAGR for the estimated period of time is forecasted in terms of revenue.

There are some key segments covered in this report such as product type, application, competitive landscape and key geographies.

This report focuses on the outlook of the industry on the basis of key applications and end users of the market.

Geographical analysis is one of the most important feature of any industry. This section majorly focuses of the key regions and countries which have good market of the industry. The major trends and developments taking place in the key regions are covered in this report. Hence, geographical analysis provides a deep insight about the opportunities and possibilities of generating revenue for the new entrants in the market.

The Report provides the industry analysis, estimation and extraction of the data based on the historic database for future status. It also covers the growth aspects of the market along with the restraining factors which are likely to impact on the overall the growth of market in the estimated forecast period. In addition, it also covers the demand and supply of the market research study in the estimated forecast period. Detailed study of the market players with their profile, sales analysis and competitive landscape is provided in the report. Furthermore, partnership, collaboration and mergers in the industry are mentioned for the ease of the study of the Global Bone Marrow Transplant industry.

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The impact of Coronavirus outbreak on the market industry is explained in this report. Covid-19 outbreak has climbed rapidly and it is likely pull the market down further in the upcoming years. Thus the complete overview of the pandemic is studied to help you understand the economic impact of the virus so far. Therefore, the strategies and solutions are discussed based on assessments made by different analysts and industry experts in order to stabilize the industry condition and grow further to maintain the status in the market.

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COVID-19 Impact On Global Bone Marrow Transplant Market New Study Of Trend And Forecast Report 2020-2026 - Cole of Duty

The Covid-19 (coronavirus) pandemic is impacting society and the overall economy across the world. The impact of this pandemic is growing day by day as well as affecting the supply chain. The COVID-19 crisis is creating uncertainty in the stock market, massive slowing of supply chain, falling business confidence, and increasing panic among the customer segments. The overall effect of the pandemic is impacting the production process of several industries including Life science Industry, and many more. Trade barriers are further restraining the demand- supply outlook. As government of different regions have already announced total lockdown and temporarily shutdown of industries, the overall production process being adversely affected; thus, hinder the overall Stem Cell Therapy market globally. This report on Stem Cell Therapy market provides the analysis on impact on Covid-19 on various business segments and country markets. The report also showcase market trends and forecast to 2027, factoring the impact of Covid -19 Situation.

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Stem cell therapy is a technique which uses stem cells for the treatment of various disorders. Stem cell therapy is capable of curing broad spectrum of disorders ranging from simple to life threatening. These stem cells are obtained from different sources, such as, adipose tissue, bone marrow, embryonic stem cell and cord blood among others. Stem cell therapy is enables to treat more than 70 disorders, including degenerative as well as neuromuscular disorders. The ability of a stem cell to renew itself helps in replacing the damaged areas in the human body.

MARKET DYNAMICSIncrease in the number of stem cell banking facilities and rising awareness on the benefits of stem cell for curing various disorders are expected to drive the market during the forecast period. Rise in number of regulations to promote stem cell therapy and increase in number of funds for research in developing countries are expected to offer growth opportunities to the market during the coming years.

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The research provides answers to the following key questions:

The study conducts SWOT analysis to evaluate strengths and weaknesses of the key players in the Stem Cell Therapy market. Further, the report conducts an intricate examination of drivers and restraints operating in the market. The report also evaluates the trends observed in the parent market, along with the macro-economic indicators, prevailing factors, and market appeal according to different segments. The report also predicts the influence of different industry aspects on the Stem Cell Therapy market segments and regions.

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Stem Cell Therapy Market Segmented by Region/Country: North America, Europe, Asia Pacific, Middle East & Africa, and Central & South America

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COVID-19 Impact on STEM CELL THERAPY MARKET 2020 TO 2027-EXPANDING WORLDWIDE WITH TOP PLAYERS FUTURE BUSINESS SCOPE AND INVESTMENT ANALYSIS REPORT -...

Hair loss is not something usually for one to worry about, however, it can be upsetting for some. At times, hair loss can be only temporary and can be caused by an illness, genetics, stress, weight loss or an iron deficiency. In others, hair loss is a normal part of getting older but what can be done to help halt the process?Dr Earim Chaudryspoke exclusively toExpress.co.ukto discuss all thingshair-relatedand what are some of the best supplements one can take to help with hair loss.

Dr Chaudry said: Approximately 30percentof men by the age of 30 and 70percentby the age of 70 are affected by Male Pattern Baldness (MPB) but it can appear as early as teenage years, particularly if there is a strong family history of early baldness.

First, a little background on male pattern baldness and how it happens.

Androgen hormones communicate to hair follicles through specific androgen receptors located at the root of the hair follicle. Interestingly, these receptors are only present on hair follicles located at the front and crown of the scalp.

They are not present on the back and sides which is why these areas are not normally affected by the process of MPB.

Genetic factors determine how active these receptors are and therefore how sensitive the hair follicles are to the effect of androgens. The gene that codes for the androgen receptor (AR) has been confirmed to play a role in MPB.

The androgen receptor (AR) gene is located on the X chromosome, which in men is inherited from the mother.

This explains the common belief that MPB is inherited from the maternal side of the family. New research supports that there are far more genetic factors, which can come from one or both parents that contribute to the overall risk of MPB.

Equally, early-onset MPB has been independently linked to metabolic syndrome and other risk factors for heart disease.

When asked what happens during the hair loss process, Dr Chaudry replied: MPBis the most common form of hair loss, caused by your genetics primarily.

It accounts for 95percentof male hair loss and is treatable. In MPB hair follicles gradually shrink, your hair becomes thinner and sheds more quickly.

At the hair follicle, there is an enzyme called 5-alpha-reductase (5AR) which changes testosterone into DHT (a more potent hormone). Hair follicles susceptible to MPB are more sensitive to DHT, and it causes them to shrink.

The hair at the back of the head is more resistant to the effects of DHT and because of receding hairlines and the crown usually are the areas of MPB."

When it comes to the best supplements one can take to help with hair loss, Dr Chaudry recommends: Part of the B family, biotin is one of several vitamins that convert nutrients from food into energy.

It plays an important role in the healthiness of your hair, as well as your skin and nails, and is one of the only natural remedies for hair loss that is backed by science.

Biotin deficiency is extremely rare, so the chances are it will not be the root cause of your hair.

However, some studies have shown that taking biotin helps speed up and increase the rate of hair growth in certainindividuals."

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Hair loss treatment: Expert reveals what you should be taking to help stimulate hair - Express

Back in autumn 2018, investors began piling into U.S.-based India Globalization Capital, Inc., (IGC) which had announced in September of that year the startup of a CBD beverage called Nitro G. The company had been touting a distribution deal with a partner in Malaysia that would make the business go. IGC eventually raised $30 million in a secondary offering on the New York Stock Exchange.

Due diligence: By October 29 of that year the NYSE had delisted IGCs common stock, flagging the company for promoting ventures the success of which is problematical after analysts pointed to a flaw in the companys plans. As a class action lawsuit filed against IGC in November 2018 described the strategic weakness: Plaintiffs alleged that the company failed to disclose to investors that cannabis was illegal and punishable by death in Malaysia.

Thats only one of the more colorful among pending federal class action lawsuits against cannabis enterprises in U.S. courts in which investors are crying over the money they lost blindly chasing risky stocks.

Meanwhile, as the hemp industry wobbles like any other toddler, everybody from independent farmers to big, loud public companies find themselves before the judge these days.

The claims such as those against IGC are standard fare in the public markets, particularly those companies traded as penny stocks. They generally fall into the made materially false and misleading statements category, with companies and their executives sued for allegedly lying about this or that, or forgetting to disclose some fundamental problem, causing stock prices to drop. Here are some other legal cases in the hemp and CBD sectors, most of which continue to wind their way through federal courts under U.S. securities laws:

Chewing gum conundrum: Plaintiffs allege U.S. CBD maker CV Sciences made misleading public statements about the status of a patent application for a CBD chewing gum. The patent was later rejected, hitting CVs share price. (Filed August 2018)

Some property in Jamaica: Investors allege Canada-based Aphria, Inc. paid C$193 million (US$137 million) for a bundle of assets in Jamaica, Argentina and Colombia that have virtually zero worth. Plaintiffs say the deals real purpose was to help executives siphon money from the company through a web of other deals. (Filed December 2018)

Pumpin it: Plaintiffs allege 22nd Century Group, which says it makes low-THC cannabis products, used an aggressive promotional campaign to push the companys stock price while failing to pursue U.S. Food & Drug Administration approval for any new products. The companys shares plummeted after 22nd Centurys CEO resigned amid rumors about the alleged price-pumping scheme. (Filed January 2019)

What licenses? A plaintiff group is seeking up to $4 million from cannabis producer Sundial Growers, Inc. of Canada through a lawsuit alleging the company misled them regarding the potential for CBD sales through its acquisition of Bridge Farm, a UK-based ag firm. The plaintiffs say Sundial claimed the British company held hemp farming and export licenses when in fact it did not. Sundial is also facing a number of suits in the marijuana space.

Puff puff-ery: Greenlane Holdings, Inc. has been targeted with a lawsuit for allegedly failing to disclose that the city of San Francisco was planning to ban e-cigarettes, after touting its relationship with JUUL, a San Francisco-based e-cigarette maker, causing a drop in Greenlanes share price. (Filed September 2019)

Ka-ching! Paragon Coin, Inc. of the U.S, which says it is incorporating blockchain technology into the cannabis industry, is alleged to have sold tokens that were not registered as securities. The lawsuit is yet unsettled, but SEC has in the meantime settled other charges against Paragon over its initial coin offerings, imposing a $250,000 fine and ordering investors compensated. (Filed January 2018)

How do you feel now?: Investors allege that Zynerba Pharmaceuticals, a CBD maker, mischaracterized the results of clinical trials of a transdermal CBD gel, Zygel, causing shares to suffer. Plaintiffs claim the company reported that the gel was well-tolerated among subjects who participated in the trial, while in truth nearly all patients in the trial reported mild to serious adverse effects, and eight patients discontinued the trial altogether. (Filed October 2019)

Authentic losses: Petitioners say Canadian cannabis giant Tilray violated federal securities laws by inflating the potential of a revenue sharing agreement signed with Authentic Brands Group (ABG) in March 2019. The plaintiffs say Q4 2019 results later showed charges of $112.1 million related to impairment of the ABG agreement. Tilray reported net 2019 losses of $321.2 million, or $3.20 per share. (Filed April 2020)

Gelatinous: Shareholders say Canopy Growth, also of Canada, misled them on the potential for a line of soft gels and oils, saying on a conference call that demand for the products was strong as late as November 2019. That eventually led to a restructuring charge of CA$32.7 million (US$23.2 million) due to poor sales, excessive returns and a bloated inventory. (Filed November 2019)

A marathon 5 years running: Hemp Inc. is the target of an ongoing federal case by the U.S. Securities & Exchange Commission (SEC) in which it is charged with violating securities laws. The original 2016 SEC complaint against the company and three executives charges them with committing a long-running fraud and running an illegal scheme to sell unregistered stock. A Nevada federal judge in April 2020 upheld sanctions against the three executives.

While public companies had their share of legal troubles, private enterprises also are finding themselves the subjects of claims by stakeholders as litigation in the cannabis, CBD and hemp sectors proliferates. The cases reflect the wide range of potential pitfalls at this formative stage of the industry:

Is that mold? Nevada-based Industrial Hemp Manufacturing LLC (a subsidiary of Hemp Inc. see above) sued American Hemp Seed Genetics LLC (AHSG), an Oregon company for $700,000 over a seed contract signed in April 2018, claiming that an initial shipment for which it paid $70,000 contained dead and moldy seeds, while others had a germination rate of only 24%. IHM was to sell $700,000 worth of American Hemp Seed Genetics seed under an arrangement between the two companies. AHSG in March 2020 lost an appeal to dismiss the lawsuit on jurisdictional grounds, so the case continues.

Up in smoke again: Oregonized Hemp Co. LLC and owner Justin Pitts filed a $2.5 million lawsuit against Josephine and Jackson counties in Oregon, alleging that police seized more than two tons of legal industrial hemp from his warehouse, then later destroyed the crop thinking it was marijuana. At least 5,000 pounds of plant material was seized from the greenhouse during the raid, which Pitts said was legal industrial hemp with THC concentrations at or below 0.3 percent. The lawsuit, filed in April 2020, charges civil rights and due process violations.

Pet supplies: CBD supplier Sage Fulfillment sued the pet product distributor Earth Animal Ventures in Connecticut for allegedly reneging on a purchase agreement, claiming revenue losses of $4.8 million in a total $5.95 million deal. In the suit, filed in April 2020, Sage claims Earth Animal failed to live up to its commitment to buy a minimum of 400,000 units of its CBD gel and applicator after purchasing only 75,000 units.

Facing down the Sheriff: Licensed California hemp grower Apothio LLC filed a federal lawsuit against Kern County Sheriff Donny Youngblood, California Department of Fish and Wildlife Director Charles Bonham, their agencies and other county and state officials over the destruction of nearly 500 acres of hemp plants the grower says are worth $1 billion, according to the lawsuit. The federal suit, filed in April, called authorities actions one the largest wholesale destruction of personal property by government entities in the history of the United States.

Troubles in Kentucky: Bankrupt hemp processor GenCanna faces a number of lawsuits after the Kentucky company collapsed late last year:

Integrity/Architecture, Lexington, Kentucky; general contractors Pinnacle Inc., Benton, Kentucky; and Crawford Sales, a door maker based in Evansville, Indiana, say GenCanna owes them a combined $50,000..

Lexington Certified Public Accounting firm Dean Dorton claims GenCanna owes it more than $500,000.

Agro firm Furnwood Farms is in arbitration with GenCanna after filing a lawsuit seeking $5 million last October due to losses resulting from the late delivery, and questionable quality, of cultivation seeds as well as changes in the wording of agreements made by GenCanna.

A group of construction and other contractors filed liens against property owned by the Industrial Authority of Mayfield-Graves County which is under lease to GenCanna. The contractors claim in their lawsuit, filed in October 2019, that GenCanna owes them $13 million related to a processing plant construction project in Mayfield, Kentucky.

Seed deal fails to sprout: Wisconsin hemp seed seller Legacy Hemp LLC is suing Canadian hemp firm Terramax after it tried to terminate a distribution deal for industrial hemp seed in the U.S., claiming Terramax is trying to move distribution rights to another hemp company. The conflict involves questions over which states Legacy was granted rights for distribution of Terramax X-59 hemp seeds. The suit was filed in March 2020.

Organizational matter: In another case in Kentucky, a group of farmers in April filed a $69 million class action lawsuit in U.S. federal courts, alleging Bluegrass Bio-Extracts, Owensboro, Kentucky, and owners Gerald Edds and Bruce Peters, committed fraud and violated the U.S. Racketeer Influenced Corrupt Organizations (RICO) Act by breaching a contract to buy their 2019 hemp crops.

Slanderous affair: American cannabis research firm New Frontier Data sued UK research group Prohibition Partners (PP Intelligence Ltd) and cannabis investor Andy Defrancesco in 2019 for assault libel and slander in a dispute involving rights to research and written analysis in a report on cannabis investments. The case is reported closed, but no details are publicly available.

Problem from the start: GX Farms of California sued H.E.M.P. Group of Colorado for $17 million in January 2020 over a batch of hemp seeds that failed to germinate as promised. GX said it paid $364,000 for 520,000 seeds in spring 2019. According to the suit, GX Farms paid another $25,000 to a broker who promised a germination rate of 99%. GX Farms alleges it lost $3.5 million to $17 million in potential income.

And one at the end: Oregon agri-company Jefferson State Farms has sued a cooperative for $11 million, claiming the group failed to harvest its crops as promised. Jefferson State alleges unlawful trade practices and breach of contract against Palex Enterprises, Hemp Warehouse and Great Horizons LLC, and four individuals. The farm company said it paid the co-op $136,000 as a down payment to schedule its hemp harvest, which never happened. Jefferson State claims $86,000 of that down payment has yet to be returned.

Plow it under: CBD maker Elemental Processing of Kentucky filed a $44 million lawsuit against HP Farms of Oregon, claiming HP sold it a batch of 6 million seeds that were mostly male, causing a massive crop failure in 2019 as farmers to whom Elemental distributed the seeds had to plow their hemp fields under. Elemental says it paid about $352,000 in advance for the seeds and had agreed to pay $3.5 million more or 15% of its profits from the harvested flowers. (Filed September 2019).

Heavy metal: A group of plaintiffs are seeking class action against American Shaman LLC of Missouri, claiming CBD they purchased from the company contained lead and other contaminants. The complaint accuses American Shaman of falsely advertising its CBD products as being free of heavy metals and insecticides. (Filed May 2020)

SOURCES: Goodwin Procter, Law 360, Reuters, Bloomberg Law

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Death penalty, chewing gum, a property in Jamaica: Lawuits run the gamut - HempToday

The majority of the worlds mammoth remains is discovered in Russia every year. Yet, some people prefer to believe that we dont even need them as evidence because these animals are still very much alive and well.

In one of the most remote parts of Russia, in the vast Siberian taiga, one can go a hundred miles without meeting a fellow traveler. Could these territories harbor living mammoths to this day?

Some people in Russia believe its possible. Some TV programs devoted to the unknown run investigations into this question, while locals continue to occasionally circulate stories of mammoth sightings. According to one rumor from the 1940s, military pilots flew over the dense taiga in Yakutia and spotted a small herd of animals, very similar to wooly mammoths. In 1978, a similar story happened to a group of people on the Indigirka river in Yakutia. They claimed that one early morning, they woke up to about a dozen mammoths, calmly drinking from the river.

Some Russians believe that mammoths can still be found living in dense Siberian taiga.

These accounts, however, are nothing more than urban legends, like the Yeti or the Loch Ness monster, thinks Nikita Zimov, Russian ecologist and director of the Pleistocene Park in Yakutia, a project that aims to revive the natural habitat of mammoths - the northern subarctic steppe grassland ecosystem, which existed here thousands of years ago. The ultimate dream is to see mammoths walk the Earth again. (So, it stands to reason that Zimov would gladly have supported a living mammoth theory, had he really thought it possible.)

This is complete nonsense. Mammoths in the mainland of Eurasia became extinct, or rather were wiped out (by people), 9,000 years ago. The last mammoths lived on Wrangel Island until 3,380 years ago, he argues. Plus, there is no such thing as unexplored Siberian taiga anymore - over the past 200-300 years, people traversed it many times over.

If one wants to find uniquely preserved mammoth fossils, Russia is the place to go!

Why is that? Well, the permafrost in the northernmost parts of Russia is uniquely suited to the task (as if a mammoth died just yesterday), and when it melts away, the erosion of river banks or other natural factors end up exposing these remains, first for locals, and then scientists, to see.

According to the Mammoth Museum in Yakutsk, 75 percent of the world's known mammoth and related graves with preserved soft tissue were found in Yakutia.

The presentation of a stuffed young mammoth male in St. Petersburg. The remains of a 15-year-old teenage mammoth were discovered in August 2012 at the mouth of the Yenisei River in Taimyr and are estimated to be about 30,000 years old.

Mammoths used to live in the area spanning from modern Spain to Canada [there was a natural bridge that connected Eurasia to North America back then]. Where it emerged as a species I cant say, but a major part of its habitat was on the territory of todays Russia - therefore, it wouldnt be an exaggeration to call Russia a sort of a motherland of mammoths, thinks Zimov.

Yet, according to him, the number of remains found in Russia isnt a telltale sign that it harbored the largest population on the planet. It only indicates that their remains have been much better preserved here thanks to permafrost. The density of the mammoth population could have been higher on the territory of modern Spain, for example, the expert believes.

The mummified remains of mammoth "Yuka" on display in Vladivostok. Yuka, the best preserved Siberian woolly mammoth to be found so far, was discovered in the permafrost of Ust-Yana Ulus in Russia's Yakutia in 2010.

The idea of bringing the beast back from the dead seems tempting, but it looks like Russian scientists are not too involved in this area of genetic research, despite so much frozen DNA being unearthed in the country.

According to Zimov, there are three main teams who are most preoccupied with this question right now - one headed by George Church from Harvard, and two from Japan and South Korea. They have all been researching the problem since the mid-90s.

75 percent of the world's known mammoth and related graves with preserved soft tissue were found in Yakutia.

Our scientists from the Mammoth Museum in Yakutsk are also participating in this research, but their work is mostly about finding the genetic material and transporting it to labs in Japan or Korea, Zimov explains, adding that Russian genetics as a science hasnt been developing as fast as it has in other countries.

He notes that there is much more talk in the media about mammoth cloning than there is evidence to its potential effectiveness. Id personally be very happy if we saw mammoths in our Pleistocene Park. But for now, this is more like waiting for a miracle, he concludes.

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The Motherland of Mammoths: Can the woolly beasts still be found alive in Siberia? - Russia Beyond

Some diseases exhibit a clear sex bias, occurring more often, hitting harder or eliciting different symptoms in men or women.

For instance, the autoimmune conditions lupus and Sjgren's syndrome affect nine times more women than men, while schizophrenia affects more men and tends to cause more severe symptoms in men than in women.

Likewise, early reports suggest that despite similar rates of infection, men are dying from COVID-19 more often than women, as happened during previous outbreaks of the related diseases SARS and MERS.

For decades, scientists have tried to pinpoint why some diseases have an unexpected sex bias. Behavior can play a role, but that explains only a piece of the puzzle. Hormones are commonly invoked, but how exactly they contribute to the disparity is unclear. As for genes, few, if any, answers have been found on the X and Y sex chromosomes for most diseases.

Now, work led by researchers in the Blavatnik Institute at Harvard Medical School and at the Broad Institute of MIT and Harvard provides a clear genetic explanation behind the sex bias observed in some of these diseases.

The team's findings, reported May 11 inNature, suggest that greater abundance of an immune-related protein in men protects against lupus and Sjgren's but heightens vulnerability to schizophrenia.

The protein, called complement component 4 (C4) and produced by the C4 gene, tags cellular debris for prompt removal by immune cells.

The team's key findings:

"Sex acts as a lens that magnifies the effects of genetic variation," said the study's first author, Nolan Kamitaki, research associate in genetics in the lab of Steven McCarroll at HMS and the Broad.

"We all know about illnesses that either women or men get a lot more, but we've had no idea why," said Steven McCarroll, the Dorothy and Milton Flier Professor of Biomedical Science and Genetics at HMS and director of genomic neurobiology at the Stanley Center for Psychiatric Research at the Broad. "This work is exciting because it gives us one of our first handles on the biology."

McCarroll is co-senior author of the study with Timothy Vyse of King's College London.

Although C4 variation appears to contribute powerfully to disease risk, it is only one among many genetic and environmental factors that influence disease development.

The study's results are informing the ongoing development of drugs that modulate the complement system, the authors said.

"For example, researchers will need to make sure that drugs that tone down the complement system do not unintentionally increase risk for autoimmune disease," said McCarroll. "Scientists will also need to consider the possibility that such drugs may be differentially helpful in male and female patients."

On a broader level, the work offers a more solid foundation for understanding sex variation in disease than has been available before.

"It's helpful to be able to think about sex-biased disease biology in terms of specific molecules, beyond vague references to 'hormones,'" McCarroll said. "We now realize that the complement system shapes vulnerability for a wide variety of illnesses."

Cell sweeper

In 2016, researchers led by Aswin Sekar, a former McCarroll lab member who is a co-author of the new study, made international headlines when they revealed that specific C4 gene variants underlie the largest common genetic risk factor for developing schizophrenia.

The new work suggests that C4 genes confer both an advantage and disadvantage to carriers, much as the gene variant that causes sickle cell disease also protects people against malaria.

"C4 gene variants come with this yin and yang of heightened and reduced vulnerability in different organ systems," said McCarroll.

The findings, when combined with insights from earlier work, offer insights into what may be happening at the molecular level.

When cells are injured, whether from a sunburn or infection, they leak their contents into the surrounding tissue. Cells from the adaptive immune system, which specialize in recognizing unfamiliar molecules around distressed cells, spot debris from the cell nuclei. If these immune cells mistake the flotsam for an invading pathogen, they may instigate an attack against material that isn't foreign at all--the essence of autoimmunity.

Researchers believe that complement proteins help tag these leaked molecules as trash so they're quickly removed by other cells, before the adaptive immune system pays too much attention to them. In people with lower levels of complement proteins, however, the uncollected debris lingers longer, and adaptive immune cells may become confused into acting as if the debris is itself the cause of problem.

As part of the new study, Kamitaki and colleagues measured complement protein levels in the cerebrospinal fluid of 589 people and blood plasma of 1,844 people. They found that samples from women aged 20 through 50 had significantly fewer complement proteins--including not only C4 but also C3, which activates C4--than samples from men of the same age.

That's the same age range in which lupus, Sjgren's and schizophrenia vulnerabilities differ by sex, Kamitaki said.

The results align with previous observations by other groups that severe early-onset lupus is sometimes associated with a complete lack of complement proteins, that lupus flare-ups can be linked to drops in complement protein levels and that a common gene variant associated with lupus affects the C3 receptor.

"There were all these medical hints," said McCarroll. "Human genetics helps put those hints together."Two flavors

The bulk of the findings arose from analyses of whole genomes from 1,265 people along with single nucleotide polymorphism (SNP) data from 6,700 people with lupus and 11,500 controls.

C4 genes and proteins come in two types, C4A and C4B. The researchers found that having more copies of the C4A gene and higher levels of C4A proteins was associated with greater protection against lupus and Sjgren's, while C4B genes had a significant but more modest effect. On the other hand, C4A was linked with increased risk of schizophrenia, while C4B had no effect on that illness.

In men, common combinations of C4A and C4B produced a 14-fold range of risk for lupus and 31-fold range of risk for Sjgren's, compared to only 6-fold and 15-fold ranges in women, respectively.

The researchers didn't expect the genes' effects to be so strong.

"Large genetic effects tend to come from rare variants, while common gene variants generally have small effects," said McCarroll. "The C4 gene variants are common, yet they are very impactful in lupus and Sjgren's."

Still, complement genes don't tell the full story of lupus, Sjgren's or schizophrenia risk, none of which are caused entirely by genetics.

"The complement system contributes to the sex bias, but it's only one of probably many genetic and environmental contributors," said Kamitaki.

Answers from diversity

Complement genes and another family of immune-related genes, called human leukocyte antigen or HLA genes, are interspersed throughout the same complex stretch of the human genome. HLA variants have been shown to raise risk of developing other autoimmune diseases, including type 1 diabetes, celiac disease and rheumatoid arthritis, and researchers had long believed that something similar was happening with lupus and Sjgren's.

The culprit, however, remained stubbornly hard to pin down, because specific variants in HLA genes and C4 genes always seemed to appear together in the same people.

Kamitaki and colleagues overcame this hurdle by analyzing DNA from a cohort of several thousand African American research participants. The participants' DNA contained many more recombinations between complement and HLA genes, allowing the researchers to finally tease apart the genes' contributions.

"It became quite clear which gene was responsible," said McCarroll. "That was a real gift to science from African American research participants. The question had been unsolved for decades."

The discovery provides further proof that the field of genetics would benefit from diversifying the populations it studies, McCarroll said.

"It will really help for genetics to expand more strongly beyond European ancestries and learn from genetic variation and ancestries all over the world," he said.

C4 variation could contribute to sex-based vulnerabilities in other diseases not yet analyzed, the authors said. It's not yet clear whether C4 pertains to the sex bias seen in COVID-19.

"We don't know the mechanism yet for why men seem to get sicker from COVID-19," said McCarroll. "Complement molecules are potentially important in any immune or inflammatory condition, and in COVID-19, it seems the immune response can be part of a downward spiral in some patients. But we don't know the key details yet."

It also remains to be seen how the differing effects of complement genes apply to people with intersex traits, also known as disorders or differences of sex development, who don't always fit textbook genetic or biological definitions of male and female.

"That is important to understand," said McCarroll.

Reference:Kamitaki et al.Complement genes contribute sex-biased vulnerability in diverse disorders. Nature, 2020; DOI: 10.1038/s41586-020-2277-x.

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Sex, Genes and Vulnerability - Technology Networks

American lobsters have occasionally escaped or been released into European waters after being imported for the seafood market.Experts have long feared they could threaten European lobsters by introducing disease or establishing as an invasive species.

Hybridization when a pure species is threatened at a genetic level via interbreeding with a different but related species had been less of a concern because lab studies suggested European and American lobsters were reluctant to mate.

However, when an American lobster female was found bearing eggs in a fjord in Sweden, University of Exeter researchers tested the offspring and found they were clearly distinct from both European and American lobsters.

We had just developed a genetic test for seafood traceability that could separate any American lobsters mislabelled as more expensive European equivalents once theyve been cooked and shell colouration is no longer a useful indicator of the species, said Dr Charlie Ellis, of the University of Exeter.

What we found when we tested these offspring is that they came out exactly in the middle of this separation half American and half European so these lobsters were hybrids.

This has potentially concerning implications for the lobster industry and conservation efforts, and Dr Ellis says further research is required to assess the extent of the threat.

Until recently, it was thought that American and European lobsters would avoid crossbreeding, but this introduced American female has mated with a native European male, probably because she was unable to find an American male, he said.

We now need to check whether any mature adult hybrids are fertile, because if they are then they have the ability to spread these unwanted American genes far and wide across our native lobster stocks.

Working with collaborators from the University of Gothenburg who originally found the hybrid egg clutch, the researchers say their study, published in the journal Scientific Reports, highlights the vital use of genetics to distinguish hybrid lobsters which might look almost identical to a pure strain.

It is particularly concerning that we seem to have found American lobster genes in one of our lobster reserves, said Linda Svanberg of the Gothenburg team.

The better news is we now have this genetic tool to test lobsters or their eggs for hybridisation, added Dr Jamie Stevens, leader of the research which was funded by an EU grant through the Agritech Cornwall scheme, so we can use it track the spread of these alien genes to assess how big a threat this presents to our native lobster species.

The team advise that, for a range of conservation reasons including potential contact with American lobsters, it is important that the general public never release a marketed lobster back into the wild, even our native species.

Dr Tom Jenkins said: Although we appreciate that all animal-lovers have concern for the fate of individual animals, in this case the rescue of one animal might endanger the health of the entire wild population, so once a lobster has entered the seafood supply chain thats where it should stay.ReferenceEllis et al. (2020). Crossing the pond: genetic assignment detects lobster hybridisation. Scientific Reports. DOI: https://doi.org/10.1038/s41598-020-64692-z

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Seafood Traceability Captures Lobsters Crossing the Pond - Technology Networks

BOTHELL, Wash.--(BUSINESS WIRE)-- Seattle Genetics, Inc., Inc. (Nasdaq:SGEN) today announced that the Swiss Agency for Therapeutic Products (Swissmedic) has granted approval for TUKYSA (tucatinib) tablets in combination with trastuzumab and capecitabine, for the treatment of patients with metastatic HER2-positive breast cancer, who have previously received two or more anti-HER2 regimens in any setting, including trastuzumab, pertuzumab and trastuzumab-emtansine (TDM1).

The application for TUKYSA approval was reviewed by Swissmedic as part of Project Orbis, an initiative of the U.S. Food and Drug Administration (FDA) Oncology Center of Excellence that provides a framework for concurrent submission and review of oncology drugs among participating international regulatory agencies in Canada, Australia and Singapore. On April 17, the FDA approved TUKYSA in the U.S. under the FDAs Real-Time Oncology Review (RTOR) pilot program, four months prior to its action date, and represented the first new drug approved under Project Orbis.

Were grateful to Swissmedic for their collaboration through FDAs Project Orbis in approving this important new medicine in Switzerland, said Jennifer Stephens, Vice President of Regulatory Affairs at Seattle Genetics. We're committed to bringing new targeted therapies to patients, and we are excited about this important first step toward making TUKYSA available to patients in Switzerland.

TUKYSA is an oral, small molecule tyrosine kinase inhibitor (TKI) of HER2, a protein that contributes to cancer cell growth.i,ii

The approval is based on results from the pivotal trial HER2CLIMB, a randomized (2:1), double-blind, placebo-controlled trial that enrolled 612 patients with HER2-positive unresectable locally advanced or metastatic breast cancer who had previously received, either separately or in combination, trastuzumab, pertuzumab, and ado-trastuzumab emtansine (T-DM1). The study results were published in The New England Journal of Medicine in December 2019.

About HER2-Positive Breast Cancer

Patients with HER2-positive breast cancer have tumors with high levels of a protein called human epidermal growth factor receptor 2 (HER2), which promotes the growth of cancer cells. In 2018, more than two million new cases of breast cancer were diagnosed worldwide, including 522,513 in Europe. iii Between 15 and 20 percent of breast cancer cases are HER2-positive.iv Historically, HER2-positive breast cancer tends to be more aggressive and more likely to recur than HER2-negative breast cancer.v,vi,vii Up to 50 percent of metastatic HER2-positive breast cancer patients develop brain metastases over time.viii,ix,x

About TUKYSA (tucatinib)

TUKYSA is an oral medicine that is a tyrosine kinase inhibitor of the HER2 protein. In vitro (in lab studies), TUKYSA inhibited phosphorylation of HER2 and HER3, resulting in inhibition of downstream MAPK and AKT signaling and cell growth (proliferation), and showed anti-tumor activity in HER2-expressing tumor cells. In vivo (in living organisms), TUKYSA inhibited the growth of HER2-expressing tumors. The combination of TUKYSA and the anti-HER2 antibody trastuzumab showed increased anti-tumor activity in vitro and in vivo compared to either medicine alone.xi In the U.S., TUKYSA is approved in combination with trastuzumab and capecitabine for adult patients with advanced unresectable or metastatic HER2-positive breast cancer, including patients with brain metastases, who have received one or more prior anti-HER2-based regimens in the metastatic setting.

Important U.S. Safety Information

Warnings and Precautions

If diarrhea occurs, administer antidiarrheal treatment as clinically indicated. Perform diagnostic tests as clinically indicated to exclude other causes of diarrhea. Based on the severity of the diarrhea, interrupt dose, then dose reduce or permanently discontinue TUKYSA.

Monitor ALT, AST, and bilirubin prior to starting TUKYSA, every 3 weeks during treatment, and as clinically indicated. Based on the severity of hepatoxicity, interrupt dose, then dose reduce or permanently discontinue TUKYSA.

Adverse Reactions

Serious adverse reactions occurred in 26% of patients who received TUKYSA. Serious adverse reactions in 2% of patients who received TUKYSA were diarrhea (4%), vomiting (2.5%), nausea (2%), abdominal pain (2%), and seizure (2%). Fatal adverse reactions occurred in 2% of patients who received TUKYSA including sudden death, sepsis, dehydration, and cardiogenic shock.

Adverse reactions led to treatment discontinuation in 6% of patients who received TUKYSA; those occurring in 1% of patients were hepatotoxicity (1.5%) and diarrhea (1%). Adverse reactions led to dose reduction in 21% of patients who received TUKYSA; those occurring in 2% of patients were hepatotoxicity (8%) and diarrhea (6%).

The most common adverse reactions in patients who received TUKYSA (20%) were diarrhea, palmar-plantar erythrodysesthesia, nausea, fatigue, hepatotoxicity, vomiting, stomatitis, decreased appetite, abdominal pain, headache, anemia, and rash.

Lab Abnormalities

In HER2CLIMB, Grade 3 laboratory abnormalities reported in 5% of patients who received TUKYSA were: decreased phosphate, increased ALT, decreased potassium, and increased AST. The mean increase in serum creatinine was 32% within the first 21 days of treatment with TUKYSA. The serum creatinine increases persisted throughout treatment and were reversible upon treatment completion. Consider alternative markers of renal function if persistent elevations in serum creatinine are observed.

Drug Interactions

Use in Specific Populations

For more information, please see the full Prescribing Information for TUKYSA here.

About Seattle Genetics

Seattle Genetics, Inc. is a global biotechnology company that discovers, develops and commercializes transformative cancer medicines to make a meaningful difference in peoples lives. ADCETRIS (brentuximab vedotin) and PADCEVTM (enfortumab vedotin-ejfv) use the companys industry-leading antibody-drug conjugate (ADC) technology. ADCETRIS is approved in certain CD30-expressing lymphomas, and PADCEV is approved in certain metastatic urothelial cancers. TUKYSATM (tucatinib), a small molecule tyrosine kinase inhibitor, is approved in certain HER2-positive metastatic breast cancers. The company is headquartered in Bothell, Washington, with locations in California, Switzerland and the European Union. For more information on our robust pipeline, visit http://www.seattlegenetics.com and follow @SeattleGenetics on Twitter.

Forward Looking Statements

Certain statements made in this press release are forward looking, such as those, among others, relating to the therapeutic potential of TUKYSA including its efficacy, safety and therapeutic uses including the potential use of TUKYSA in combination with trastuzumab and capecitabine for the treatment of patients with metastatic HER2-positive breast cancer, who have previously received two or more anti-HER2 regimens in any setting, including trastuzumab, pertuzumab and trastuzumab-emtansine (TDM1) and the potential to bring TUKYSA to patients in Switzerland. Actual results or developments may differ materially from those projected or implied in these forward-looking statements. Factors that may cause such a difference include reimbursement processes, the extent of reimbursement, the possibility that adverse events or safety signals may occur, the possibility that the ultimate utilization and adoption of TUKYSA by prescribing physicians may be limited, including due to impacts related to the COVID-19 pandemic, the possibility of difficulties in supplying and commercializing a new therapeutic agent, and the possibility of adverse regulatory actions. More information about the risks and uncertainties faced by Seattle Genetics is contained under the caption Risk Factors included in the companys Quarterly Report on Form 10-Q for the quarter ended March 31, 2020 filed with the Securities and Exchange Commission. Seattle Genetics disclaims any intention or obligation to update or revise any forward-looking statements, whether as a result of new information, future events or otherwise, except as required by law.

i TUKYSA [package insert]. Bothell, WA: Seattle Genetics, Inc.ii Anita Kulukian, Patrice Lee, Janelle Taylor, et al. Preclinical Activity of HER2-Selective Tyrosine Kinase Inhibitor Tucatinib as a Single Agent or in Combination with Trastuzumab or Docetaxel in Solid Tumor ModelsMol Cancer Ther 2020;19:976-987.iii Breast. Globocan 2018. World Health Organization. 2019. https://gco.iarc.fr/today/data/factsheets/cancers/20-Breast-fact-sheet.pdf iv Slamon D, Clark G, Wong S, et al. Human breast cancer: correlation of relapse and survival with amplification of the HER-2/neu oncogene. Science. 1987; 235(4785): 177-82.v Loibli S, Gianni L. HER2-positive breast cancer. Lancet. 2017; 389(10087): 2415-29.vi Slamon D, Clark G, Wong S, et al. Human breast cancer: correlation of relapse and survival with amplification of the HER-2/neu oncogene. Science. 1987; 235(4785): 177-82.vii Breast Cancer HER2 Status. American Cancer Society website. http://www.cancer.org/cancer/breast-cancer/understanding-a-breast-cancer-diagnosis/breast-cancer-her2-status.html. Accessed March 9, 2020.viii Freedman RA, Gelman RS, Anders CK, et al. TBCRC 022: a phase II trial of neratinib and capecitabine for patients with human epidermal growth factor receptor 2-positive breast cancer and brain metastases. J Clin Oncol. 2019;37:1081-1089.ix Olson EM, Najita JS, Sohl J, et al. Clinical outcomes and treatment practice patterns of patients with HER2-positive metastatic breast cancer in the post-trastuzumab era. Breast. 2013;22:525-531.x Bendell JC, Domchek SM, Burstein HJ, et al. Central nervous system metastases in women who receive trastuzumab-based therapy for metastatic breast carcinoma. Cancer. 2003;97:2972-2977.xi TUKYSA [package insert]. Bothell, WA: Seattle Genetics, Inc.

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Seattle Genetics Announces the Approval of TUKYSA (tucatinib) in Switzerland for the Treatment of Patients with Metastatic HER2-Positive Breast Cancer...

Statistics show men are more likely to die from Covid-19 and it seems that is due to fundamental physical differences between the sexes.

With twice as many men as women dying from Covid-19, the pandemic is proving that man flu is not a myth and when it comes to immunity women really are the stronger sex.

But having a hyper-vigilant and very aggressive immune system has its downsides too it puts women at greater risk of auto-immune conditions such as multiple sclerosis, rheumatoid arthritis and coeliac disease.

Now, some experts believe these sex-based divides in health and immunity hold the key to conquering not only the coronavirus, but many other health challenges too.

When the first figures emerged from China they showed that although coronavirus infection rates were almost the same for men and women, two-thirds of those dying were men.

Initially, this was put down to the fact that around 62% of Chinese men have smoked, compared to just 3% of women there.

But this theory began to unravel as similar patterns in Covid-19 deaths emerged in Europe and America, where many more women smoke.

Professor Sarah Hawkes, director of the Centre for Gender and Global Health at University College London, says: What Covid-19 is throwing into stark relief is that there are differences, but it is important to understand the difference between sex and gender sex is biological, while gender relates to socially constructed differences.

Gender differences in lifestyle factors such as smoking, diet and alcohol consumption explain some of the health disparities between men and women.

But it is biology, particularly having two X chromosomes, that ensures women really are the fittest when it comes to survival.

Dr Sharon Moalem, an award-winning genetics researcher and best-selling author who has spent decades studying this phenomenon, explains: X chromosomes contain around 1,000 different genes and many are involved in the immune system.

"But the male Y chromosome has only around 70 genes, which are mostly involved in sperm production.

As a result, women not only have two different versions of many immune system genes, these genes also work together and swap genetic information.

Dr Moalem explains: Its like having a tool-box which has two of every tool; two different size screwdrivers, two different hammers, two sizes of wrench.

On top of this, female cells work together, side by side. They have the ability to make a tool and share it with their sister cell, she adds.

Hormones play a part too. Dr Kyle Sue, clinical assistant professor in paediatrics and family medicine at the University of Alberta in Canada, says: Oestrogen tends to be protective, in that it increases the work the immune system is able to do to fight infection, whereas testosterone seems to do the opposite.

And these differences mean that men and women are dealt very different hands when it comes to health.

The XX-factor gives females an advantage before they are even born. Early miscarriage is more common when women have conceived a boy, while pregnancy complications such as pre-eclampsia and placental problems are also more common when women are carrying a boy.

Around 55% of babies born before 32 weeks are boys and this rises to around 60% for very premature babies. Boys who are born prematurely are also more likely to suffer lasting damage such as cerebral palsy and cognitive damage.

One reason for this could be that premature girls have higher levels of catecholamine, a fight-or-flight hormone which primes the body for physical activity.

The most obvious example of female survival superiority is life expectancy. The Office of National Statistics calculates that, on average, a toddler will live to be 79.3 years old if theyre a boy, but 82.9 years if theyre a girl.

Similarly, a 65-year-old man can expect to live for another 18.6 years, but a woman the same age will have another 21 years. Only one in five men makes it to their 90th birthday, compared to one in three women.

Danish researchers who studied death records from seven famines and epidemics (including the Irish potato famine) found women are the life-expectancy champions.

The statistics revealed they lived longer in every case.

Men have a 29% higher risk of developing heart disease and are more likely to have cardiac disease before their 50s. In part this is because of lifestyle: they are more likely to be overweight, drink to excess and smoke. But hormones are a factor too.

Until menopause, women are protected by their higher levels of oestrogen, with studies confirming the hormone reduces oxidative stress, blood pressure and fibrosis, as well as improving the elasticity of blood vessels.

Conversely, testosterone appears to increase cardiac risk by suppressing levels of heart-friendly HDL cholesterol.

Yet despite this, men are more likely to survive their first heart attack.

As heart disease is still seen as a male problem, women often delay seeking help and, when they do, they are 50% more likely to be misdiagnosed.

Women are also less likely to receive bypass surgery, stents and other procedures. Symptoms vary, too. Men usually report crushing chest pain, while women sometimes describe this as tightness or dismiss it as indigestion.

Women are also more likely to experience symptoms such as pain in the arms, nausea and sweating, which are not immediately associated with heart attack.

Having an aggressive immune system puts women at greater risk of auto-immune disorders.

Multiple sclerosis is three times more common in women than men; for every nine women with lupus, only one man is affected; and, under the age of 50, the incidence of rheumatoid arthritis is four to five times higher in women.

But Dr Moalem says women still have an edge. Even though women are more likely to be affected by auto-immune conditions, compared to the men who get them, women do much better.

"For instance, one study found men had an average MS Severity Score of 5.11 while for women the average was just 3.02.

Studies of vaccines, including the MMR, the BCG for tuberculosis and the combined tetanus diphtheria and pertussis shot show women have a much stronger immune response, which gives them better protection.

A large American study of flu jabs found women got two or three times more protection than men from the same dose.

This super-response means women are also more likely to experience side-effects prompting some experts to argue they should have lower doses.

Women are more likely to seek help for pain problems and appear to be more susceptible to specific problems such as migraine and neuropathic pain.

There is also evidence that men and women feel pain in different ways, which could have important implications for pain relief.

Unlimited exercise outdoors is to be allowed, with the previous 'once-a-day' rule scrapped

People will be allowed to sit on parks and beaches, provided they stay two metres away from other people

Boris Johnson said in his speech on Sunday night that people will be able to drive to other destinations from Wednesday, presumably to exercise there, although more detailed plans are likely to follow

From May 13, people will be able to meet one friend or relative from a different household in a public place, provided they stay two metres apart. Gatherings of more than two people remain banned, so people cannot meet both their parents, for example

Golf courses and tennis courts will reopen from May 13, with social distancing relatively easy to achieve, although you can play only with people in your household. Team sports such as football are not likely to be encouraged, as social distancing is difficult and more people than the average household would be required

Angling and water sports are also to be allowed to resume in England from Wednesday. Again, you must be within your own household and two metres from other people

Rather than a change to the rules, the government is now encouraging people to return to work if they cannot do so from home. But they also urge people to drive, walk or cycle to work rather than use public transport, if possible

For instance, the diabetes drug metformin blocks pain from nerve damage but only in men.

Some studies suggest women have a higher pain threshold.

When they have access to self-administered opioids following surgery, men give themselves higher doses even though studies show women need higher doses of morphine to get the same relief.

Men have a higher proportion of muscle, so burn more calories.

On average men need 2,500kcal a day, while women need 2,000. Women are more efficient at storing fat and typically have six to 11% more body fat.

Its an evolutionary safeguard to ensure they could conceive and breastfeed when food was scarce.

Because women have more body fat and less water in their bodies, if a man and woman of the same weight drink the same amount of alcohol, the woman has a higher blood alcohol reading.

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Deadly coronavirus has proven that women are the stronger sex - Mirror Online

Researchers of a new study have now pinpointed the specific gene responsible for Cape honey bees unique capability for virgin births.

Although this sexless reproductive trait has been known for over a hundred years, it was only through modern genetic tools that researchers were able to discover the responsible gene.

The Cape honey bee is a subspecies of the western honey bee often found along the southern coast of South Africa.

After a 30-year-long search, researchers were able to find the gene, named GB45239, on the bees 11th chromosome. The studys breakthroughs were detailed in the journal Current Biology.

Scientists have been looking for this gene for the last 30 years. Now that we know its on chromosome 11, we have solved a mystery, the studys co-author Benjamin Oldroyd, professor of behavioral genetics at the University of Sydney in Australia, said in a news release.

Researchers said the discovery of this particular gene could propel new insights into the evolution of diverse reproductive strategies among animals.

Sex is a weird way to reproduce and yet it is the most common form of reproduction for animals and plants on the planet, Oldroyd said. Its a major biological mystery why there is so much sex going on and it doesnt make evolutionary sense. Asexuality is a much more efficient way to reproduce, and every now and then we see a species revert to it.

The presence of the GB45239 gene enables Cape honey bee workers to lay eggs that produce only females. The ability to asexually birth daughters is known as thelytokous parthenogenesis.

In such a society where asexual reproduction dominates, males arent necessarily needed. However, having female workers capable of essentially producing duplicates of themselves can cause problems.

Cape workers can become genetically reincarnated as a female queen and that prospect changes everything, Oldroyd said. Instead of being a cooperative society, Cape honey bee colonies are riven with conflict because any worker can be genetically reincarnated as the next queen. When a colony loses its queen the workers fight and compete to be the mother of the next queen.

If scientists can ever find a way to turn on and off the GB45239 gene, the method could be used to combat a variety of asexually reproducing pest species.

If we could control a switch that allows animals to reproduce asexually, that would have important applications in agriculture, biotechnology and many other fields, Oldroyd said.

Ethen Kim Lieser is a Tech Editor who has held posts at Google, The Korea Herald, Lincoln Journal Star, AsianWeek and Arirang TV.

Image: Reuters

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Researchers Discover Gene Responsible for Honey Bee Virgin Births - The National Interest

When a woman named Shanawdithit died from tuberculosis in Newfoundland nearly 200 years ago, it was widely believed that her death marked a tragic end to her peoples existence.

For centuries, the Beothuk had thrived along the rocky shores of the island, taking on a near-mythical status as descendants of the first people encountered by Norse explorers in what is now Canada. But their population was devastated by decades of starvation and diseases, and when she died in 1829, Shanawdithit was believed to be the last of her line.

New research from Memorial University, however, has found Beothuk DNA probably still exists in people alive today a discovery that would rewrite the history of the Newfoundlands early inhabitants, even as it confirms the accuracy of local First Nations oral tradition.

Weve got good evidence that we have genetic continuity from the Beothic into modern persons, said biologist Dr Steve Carr.

But while the finding would trigger a rethink for historians, the notion is not surprising to local Indigenous groups.

Mikmaq oral history has long asserted a shared ancestry with the original inhabitants of Newfoundland, and local First Nations have worked closely with Carr to help lend genetic evidence to their own traditions.

There were always connections or friendly relations going back more than 200 years ago and when you mingle that way, periodically, things would happen, said Chief Misel Joe of the Miawpukek Mikamawey Mawiomi, a Mikmaq First Nation in Newfoundland.

Historians believe the Beothuk are descended from a group that braved the ocean to cross from Labrador to Newfoundland thousands of years ago and whose distinct culture emerged around 1500 CE. At one point, as many as 2,000 Beothuk lived in communities scattered around Newfoundland.

For generations, they largely resisted and avoided relations with European settlers; the few interactions between the two were defined by violent encounters.

Early European settlements on the coast cut off Beothuk access to critical salmon and seals forcing them to move further inland where they sustained themselves on caribou before finally succumbing to starvation and disease.

But Carrs research suggests it was only a cultural extinction; their genetic legacy lives on.

In his study, Carr used DNA samples from Shanawdithits aunt and uncle Demasduit and Nonosbawsut whose skulls were sent to the University of Edinburgh in the 1850s. After a long campaign by Chief Joes community, the remains were repatriated to Newfoundland from National Museums Scotland in March.

After running samples through a genetics database, Carr was able to find his smoking gun a man in Tennessee who was genetically similar to Nonosbawsut, but had no known Indigenous ancestry.

With only a small amount of data to work with, Carr hopes more samples will further demonstrate a connection.

Its easy to obtain the DNA sequence from somebody and you can count the number of similarities. Thats a very easy thing to do. But to reconstruct the patterns of a relationship is a very challenging problem, said Carr, adding that further research into the known movement and connections between the Beothuk and Mimaq was still required.

The findings also illustrate the way in which genetic uniqueness in this case the distinct sequence of Beothuk mitochondrial genomes can persist intact for generations. While humans share an immense amount of DNA that traces back millennia, said Carr, the intent of his research lay in teasing out the subtle and distinguishing differences between known groups.

For years, academia has ignored the oral histories of Indigenous peoples, said Chief Joe.

Academics are hard people to convince. They often have this mindset that this the way it was no matter what information we give them to the contrary, he said.

He described a frustrating experience in a land claims court, where the adjudicator suggested the Mikmaq first arrived in Newfoundland in the 1700s.

But we have an oral history of British sailors meeting our people and asking for directions. We drew them a map on birch bark. If this is the first time we had ever been on the land, how could we draw a map? said Joe.

Its convenient for government, for everyone, to ignore people who had no written history

The community is excited to keep working with Carr on further testing, said Joe, to further strengthen the evidence of shared ancestry.

This is a big thing for us, he said. But it all comes from something we already knew.

This article was amended on 11 May 2020. An earlier version incorrectly said that the skulls of Demasduit and Nonosbawsut were taken to the Royal Museum in Scotland in 1828.

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Canada: DNA discovery lends weight to First Nations ancestral story - The Guardian

WILMINGTON, Del.--(BUSINESS WIRE)--AstraZeneca and Merck & Co., Inc., Kenilworth, NJ, US (Merck: known as MSD outside the US and Canada) today announced LYNPARZA (olaparib) in combination with bevacizumab has been approved in the US for the maintenance treatment of adult patients with advanced epithelial ovarian, fallopian tube or primary peritoneal cancer who are in complete or partial response to first-line platinum-based chemotherapy and whose cancer is associated with homologous recombination deficiency (HRD) positive status defined by either a deleterious or suspected deleterious BRCA mutation, and/or genomic instability. Patients will be selected for therapy based on an FDA-approved companion diagnostic for LYNPARZA.

The approval by the US Food and Drug Administration (FDA) was based on a biomarker subgroup analysis from the Phase III PAOLA-1 trial which showed LYNPARZA in combination with bevacizumab reduced the risk of disease progression or death by 67% (equal to a hazard ratio of 0.33). The addition of LYNPARZA also improved progression-free survival (PFS) to a median of 37.2 months versus 17.7 months with bevacizumab alone in patients with HRD-positive advanced ovarian cancer.

Approximately one in two women with advanced ovarian cancer has an HRD-positive tumor. For patients with advanced ovarian cancer, the primary aim of 1st-line treatment is to delay disease progression for as long as possible with the intent to achieve long-term remission.

Isabelle Ray-Coquard, principal investigator of the PAOLA-1 trial and medical oncologist, Centre Lon Brard and President of the GINECO group, said: Ovarian cancer is a devastating disease. The magnitude of benefit in HRD-positive patients in the PAOLA-1 trial is impactful. The combination of LYNPARZA and bevacizumab now provides women with HRD-positive advanced ovarian cancer with a new standard of care and I look forward to seeing this translate into clinical practice.

Dave Fredrickson, Executive Vice President, Oncology Business Unit, said: This approval represents another milestone for LYNPARZA in patients with ovarian cancer. The median progression-free survival of more than three years offers new hope for more women to delay relapse in this difficult-to-treat disease. These results further establish that HRD-positive is a distinct subset of ovarian cancer, and HRD testing is now a critical component for the diagnosis and tailoring of treatment for women with advanced ovarian cancer.

Roy Baynes, Senior Vice President and Head of Global Clinical Development, Chief Medical Officer, Merck Research Laboratories, said: Advances in understanding the role of biomarkers and PARP inhibition have fundamentally changed how physicians treat this aggressive type of cancer. Todays approval based on the PAOLA-1 trial highlights the importance of HRD testing at diagnosis to identify those who may benefit from LYNPARZA in combination with bevacizumab as a 1st-line maintenance treatment.

Fatal adverse reactions occurred in 1 patient due to concurrent pneumonia and aplastic anemia. Serious adverse reactions occurred in 31% of patients who received LYNPARZA/bevacizumab. Serious adverse reactions in >5% of patients included hypertension (19%) and anemia (17%).

The most common adverse reactions (Grades 1-4) occurring in 10% of patients treated with LYNPARZA/bevacizumab and at 5% frequency compared to placebo/bevacizumab were: nausea (53%), fatigue (including asthenia) (53%), anemia (41%), lymphopenia (24%), vomiting (22%) and leukopenia (18%). In addition, the most common adverse reactions (10%) for patients receiving LYNPARZA/bevacizumab irrespective of the frequency compared with the placebo/bevacizumab arm were: diarrhea (18%), neutropenia (18%), urinary tract infection (15%), and headache (14%).

In addition, venous thromboembolic events occurred more commonly in patients receiving LYNPARZA/bevacizumab (5%) than in those receiving placebo/bevacizumab (1.9%).

The full results from the Phase III PAOLA-1 trial can be found inThe New England Journal of Medicine.

Financial considerationsFollowing this approval for LYNPARZA in the US, AstraZeneca will receive from Merck $100m in Collaboration Revenue, anticipated to be booked by the Company during the second quarter of 2020.

IMPORTANT SAFETY INFORMATION

CONTRAINDICATIONS

There are no contraindications for LYNPARZA.

WARNINGS AND PRECAUTIONS

Myelodysplastic Syndrome/Acute Myeloid Leukemia (MDS/AML): Occurred in <1.5% of patients exposed to LYNPARZA monotherapy, and the majority of events had a fatal outcome. The duration of therapy in patients who developed secondary MDS/AML varied from <6 months to >2 years. All of these patients had previous chemotherapy with platinum agents and/or other DNA-damaging agents, including radiotherapy, and some also had a history of more than one primary malignancy or of bone marrow dysplasia.

Do not start LYNPARZA until patients have recovered from hematological toxicity caused by previous chemotherapy (Grade 1). Monitor complete blood count for cytopenia at baseline and monthly thereafter for clinically significant changes during treatment. For prolonged hematological toxicities, interrupt LYNPARZA and monitor blood count weekly until recovery.

If the levels have not recovered to Grade 1 or less after 4 weeks, refer the patient to a hematologist for further investigations, including bone marrow analysis and blood sample for cytogenetics. Discontinue LYNPARZA if MDS/AML is confirmed.

Pneumonitis: Occurred in <1% of patients exposed to LYNPARZA, and some cases were fatal. If patients present with new or worsening respiratory symptoms such as dyspnea, cough, and fever, or a radiological abnormality occurs, interrupt LYNPARZA treatment and initiate prompt investigation. Discontinue LYNPARZA if pneumonitis is confirmed and treat patient appropriately.

Embryo-Fetal Toxicity: Based on its mechanism of action and findings in animals, LYNPARZA can cause fetal harm. A pregnancy test is recommended for females of reproductive potential prior to initiating treatment.

FemalesAdvise females of reproductive potential of the potential risk to a fetus and to use effective contraception during treatment and for 6 months following the last dose.

MalesAdvise male patients with female partners of reproductive potential or who are pregnant to use effective contraception during treatment and for 3 months following the last dose of LYNPARZA and to not donate sperm during this time.

ADVERSE REACTIONSFirst-Line Maintenance BRCAm Advanced Ovarian Cancer

Most common adverse reactions (Grades 1-4) in 10% of patients in clinical trials of LYNPARZA in the first-line maintenance setting for SOLO-1 were: nausea (77%), fatigue (67%), abdominal pain (45%), vomiting (40%), anemia (38%), diarrhea (37%), constipation (28%), upper respiratory tract infection/influenza/nasopharyngitis/bronchitis (28%), dysgeusia (26%), decreased appetite (20%), dizziness (20%), neutropenia (17%), dyspepsia (17%), dyspnea (15%), leukopenia (13%), UTI (13%), thrombocytopenia (11%), and stomatitis (11%).

Most common laboratory abnormalities (Grades 1-4) in 25% of patients in clinical trials of LYNPARZA in the first-line maintenance setting for SOLO-1 were: decrease in hemoglobin (87%), increase in mean corpuscular volume (87%), decrease in leukocytes (70%), decrease in lymphocytes (67%), decrease in absolute neutrophil count (51%), decrease in platelets (35%), and increase in serum creatinine (34%).

ADVERSE REACTIONSFirst-Line Maintenance Advanced Ovarian Cancer in Combination with Bevacizumab

Most common adverse reactions (Grades 1-4) in 10% of patients treated with LYNPARZA/bevacizumab compared to a 5% frequency for placebo/bevacizumab in the first-line maintenance setting for PAOLA-1 were: nausea (53%), fatigue (including asthenia) (53%), anemia (41%), lymphopenia (24%), vomiting (22%) and leukopenia (18%). In addition, the most common adverse reactions (10%) for patients receiving LYNPARZA/bevacizumab irrespective of the frequency compared with the placebo/bevacizumab arm were: diarrhea (18%), neutropenia (18%), urinary tract infection (15%) and headache (14%).

In addition, venous thromboembolic events occurred more commonly in patients receiving LYNPARZA/bevacizumab (5%) than in those receiving placebo/bevacizumab (1.9%).

Most common laboratory abnormalities (Grades 1-4) in 25% of patients for LYNPARZA in combination with bevacizumab in the first-line maintenance setting for PAOLA-1 were: decrease in hemoglobin (79%), decrease in lymphocytes (63%), increase in serum creatinine (61%), decrease in leukocytes (59%), decrease in absolute neutrophil count (35%) and decrease in platelets (35%).

ADVERSE REACTIONSMaintenance Recurrent Ovarian Cancer

Most common adverse reactions (Grades 1-4) in 20% of patients in clinical trials of LYNPARZA in the maintenance setting for SOLO-2 were: nausea (76%), fatigue (including asthenia) (66%), anemia (44%), vomiting (37%), nasopharyngitis/upper respiratory tract infection (URI)/influenza (36%), diarrhea (33%), arthralgia/myalgia (30%), dysgeusia (27%), headache (26%), decreased appetite (22%), and stomatitis (20%).

Study 19: nausea (71%), fatigue (including asthenia) (63%), vomiting (35%), diarrhea (28%), anemia (23%), respiratory tract infection (22%), constipation (22%), headache (21%), decreased appetite (21%) and dyspepsia (20%).

Most common laboratory abnormalities (Grades 1-4) in 25% of patients in clinical trials of LYNPARZA in the maintenance setting (SOLO-2/Study 19) were: increase in mean corpuscular volume (89%/82%), decrease in hemoglobin (83%/82%), decrease in leukocytes (69%/58%), decrease in lymphocytes (67%/52%), decrease in absolute neutrophil count (51%/47%), increase in serum creatinine (44%/45%), and decrease in platelets (42%/36%).

ADVERSE REACTIONSAdvanced gBRCAm Ovarian Cancer

Most common adverse reactions (Grades 1-4) in 20% of patients in clinical trials of LYNPARZA for advanced gBRCAm ovarian cancer after 3 or more lines of chemotherapy (pooled from 6 studies) were: fatigue/asthenia (66%), nausea (64%), vomiting (43%), anemia (34%), diarrhea (31%), nasopharyngitis/upper respiratory tract infection (URI) (26%), dyspepsia (25%), myalgia (22%), decreased appetite (22%), and arthralgia/musculoskeletal pain (21%).

Most common laboratory abnormalities (Grades 1-4) in 25% of patients in clinical trials of LYNPARZA for advanced gBRCAm ovarian cancer (pooled from 6 studies) were: decrease in hemoglobin (90%), mean corpuscular volume elevation (57%), decrease in lymphocytes (56%), increase in serum creatinine (30%), decrease in platelets (30%), and decrease in absolute neutrophil count (25%).

ADVERSE REACTIONSgBRCAm, HER2-Negative Metastatic Breast Cancer

Most common adverse reactions (Grades 1-4) in 20% of patients in OlympiAD were: nausea (58%), anemia (40%), fatigue (including asthenia) (37%), vomiting (30%), neutropenia (27%), respiratory tract infection (27%), leukopenia (25%), diarrhea (21%), and headache (20%).

Most common laboratory abnormalities (Grades 1-4) in 25% of patients in OlympiAD were: decrease in hemoglobin (82%), decrease in lymphocytes (73%), decrease in leukocytes (71%), increase in mean corpuscular volume (71%), decrease in absolute neutrophil count (46%), and decrease in platelets (33%).

ADVERSE REACTIONSFirst-Line Maintenance gBRCAm Metastatic Pancreatic Adenocarcinoma

Most common adverse reactions (Grades 1-4) in 10% of patients in clinical trials of LYNPARZA in the first-line maintenance setting for POLO were: fatigue (60%), nausea (45%), abdominal pain (34%), diarrhea (29%), anemia (27%), decreased appetite (25%), constipation (23%), vomiting (20%), back pain (19%), arthralgia (15%), rash (15%), thrombocytopenia (14%), dyspnea (13%), neutropenia (12%), nasopharyngitis (12%), dysgeusia (11%), and stomatitis (10%).

Most common laboratory abnormalities (Grades 1-4) in 25% of patients in clinical trials of LYNPARZA in the first-line maintenance setting for POLO were: increase in serum creatinine (99%), decrease in hemoglobin (86%), increase in mean corpuscular volume (71%), decrease in lymphocytes (61%), decrease in platelets (56%), decrease in leukocytes (50%), and decrease in absolute neutrophil count (25%).

DRUG INTERACTIONS

Anticancer Agents: Clinical studies of LYNPARZA in combination with other myelosuppressive anticancer agents, including DNA-damaging agents, indicate a potentiation and prolongation of myelosuppressive toxicity.

CYP3A Inhibitors: Avoid concomitant use of strong or moderate CYP3A inhibitors. If a strong or moderate CYP3A inhibitor must be co-administered, reduce the dose of LYNPARZA. Advise patients to avoid grapefruit, grapefruit juice, Seville oranges, and Seville orange juice during LYNPARZA treatment.

CYP3A Inducers: Avoid concomitant use of strong or moderate CYP3A inducers when using LYNPARZA. If a moderate inducer cannot be avoided, there is a potential for decreased efficacy of LYNPARZA.

USE IN SPECIFIC POPULATIONS

Lactation: No data are available regarding the presence of olaparib in human milk, its effects on the breastfed infant or on milk production. Because of the potential for serious adverse reactions in the breastfed infant, advise a lactating woman not to breastfeed during treatment with LYNPARZA and for 1 month after receiving the final dose.

Pediatric Use: The safety and efficacy of LYNPARZA have not been established in pediatric patients.

Hepatic Impairment: No adjustment to the starting dose is required in patients with mild or moderate hepatic impairment (Child-Pugh classification A and B). There are no data in patients with severe hepatic impairment (Child-Pugh classification C).

Renal Impairment: No dosage modification is recommended in patients with mild renal impairment (CLcr 51-80 mL/min estimated by Cockcroft-Gault). In patients with moderate renal impairment (CLcr 31-50 mL/min), reduce the dose of LYNPARZA to 200 mg twice daily. There are no data in patients with severe renal impairment or end-stage renal disease (CLcr 30 mL/min).

INDICATIONS

LYNPARZA is a poly (ADP-ribose) polymerase (PARP) inhibitor indicated:

First-Line Maintenance BRCAm Advanced Ovarian Cancer

For the maintenance treatment of adult patients with deleterious or suspected deleterious germline or somatic BRCA-mutated (gBRCAm or sBRCAm) advanced epithelial ovarian, fallopian tube, or primary peritoneal cancer who are in complete or partial response to first-line platinum-based chemotherapy. Select patients for therapy based on an FDA-approved companion diagnostic for LYNPARZA.

First-Line Maintenance HRD Positive Advanced Ovarian Cancer in Combination with Bevacizumab

In combination with bevacizumab for the maintenance treatment of adult patients with advanced epithelial ovarian, fallopian tube or primary peritoneal cancer who are in complete or partial response to first-line platinum-based chemotherapy and whose cancer is associated with homologous recombination deficiency (HRD) positive status defined by either:

Select patients for therapy based on an FDA-approved companion diagnostic for LYNPARZA.

Maintenance Recurrent Ovarian CancerFor the maintenance treatment of adult patients with recurrent epithelial ovarian, fallopian tube, or primary peritoneal cancer, who are in complete or partial response to platinum-based chemotherapy.

Advanced gBRCAm Ovarian CancerFor the treatment of adult patients with deleterious or suspected deleterious germline BRCA-mutated (gBRCAm) advanced ovarian cancer who have been treated with 3 or more prior lines of chemotherapy. Select patients for therapy based on an FDA-approved companion diagnostic for LYNPARZA.

gBRCAm, HER2-Negative Metastatic Breast CancerFor the treatment of adult patients with deleterious or suspected deleterious gBRCAm, human epidermal growth factor receptor 2 (HER2)-negative metastatic breast cancer who have been treated with chemotherapy in the neoadjuvant, adjuvant, or metastatic setting. Patients with hormone receptor (HR)-positive breast cancer should have been treated with a prior endocrine therapy or be considered inappropriate for endocrine therapy. Select patients for therapy based on an FDA-approved companion diagnostic for LYNPARZA.

First-Line Maintenance gBRCAm Metastatic Pancreatic CancerFor the maintenance treatment of adult patients with deleterious or suspected deleterious gBRCAm metastatic pancreatic adenocarcinoma whose disease has not progressed on at least 16 weeks of a first-line platinum-based chemotherapy regimen. Select patients for therapy based on an FDA-approved companion diagnostic for LYNPARZA.

Please click here for complete Prescribing Information, including Patient Information (Medication Guide).

NOTES TO EDITORS

About Ovarian CancerApproximately 22,000 women in the United States are diagnosed with ovarian cancer (including ovarian, fallopian tube and primary peritoneal cancers) each year. Ovarian cancer ranks fifth in cancer deaths among women, accounting for more deaths than any other cancer of the female reproductive system. The risk of developing ovarian cancer is increased in women with specific inherited genetic abnormalities, including BRCA1/2 mutations.

Most women are diagnosed with advanced (Stage III or IV) ovarian cancer and have a five-year survival rate of approximately 30%. Approximately 50% of ovarian cancers are HRD-positive, including BRCA1/2 mutation. Some 22% of ovarian cancers have a BRCA1/2 mutation.

About PAOLA-1PAOLA-1 is a double-blind, Phase III trial testing the efficacy and safety of LYNPARZA (300 mg twice daily) in combination with bevacizumab vs. bevacizumab alone, as a 1st-line maintenance treatment for newly diagnosed advanced FIGO Stage III-IV high-grade serous or endometrioid ovarian, fallopian tube, or peritoneal cancer patients who had a complete or partial response to 1st-line treatment with platinum-based chemotherapy and bevacizumab. AstraZeneca and Merck announced in August 2019 that the trial met its primary endpoint of PFS.

Simultaneously, the Myriad Genetics myChoice CDx test has been approved in the US as a companion diagnostic for LYNPARZA in this new indication.

About Homologous Recombination DeficiencyHRD, which defines a sub-group of ovarian cancer, encompasses a wide range of genetic abnormalities, including BRCA mutations. As with BRCA gene mutations, HRD interferes with normal cell DNA repair mechanisms and confers sensitivity to PARP inhibitors including LYNPARZA.

About LYNPARZALYNPARZA (olaparib) is a first-in-class PARP inhibitor and the first targeted treatment to potentially exploit DNA damage response (DDR) pathway deficiencies, such as BRCA mutations, to preferentially kill cancer cells. Inhibition of PARP with LYNPARZA leads to the trapping of PARP bound to DNA single-strand breaks, stalling of replication forks, their collapse and the generation of DNA double-strand breaks and cancer cell death. LYNPARZA is being tested in a range of tumor types with defects and dependencies in the DDR pathway.

LYNPARZA, which is being jointly developed and commercialized by AstraZeneca and Merck, has a broad and advanced clinical trial development program, and AstraZeneca and Merck are working together to understand how it may affect multiple PARP-dependent tumors as a monotherapy and in combination across multiple cancer types. LYNPARZA is being tested in a range of DDR-deficient tumor types and is the foundation of AstraZenecas industry-leading portfolio of compounds targeting DDR mechanisms in cancer cells.

About the AstraZeneca and Merck Strategic Oncology CollaborationIn July 2017, AstraZeneca and Merck & Co., Inc., Kenilworth, NJ, US, known as MSD outside the United States and Canada, announced a global strategic oncology collaboration to co-develop and co-commercialize certain oncology products, including LYNPARZA, the worlds first PARP inhibitor, for multiple cancer types. Working together, the companies will develop these products in combination with other potential new medicines and as monotherapies. Independently, the companies will develop these oncology products in combination with their respective PD-L1 and PD-1 medicines.

About AstraZeneca in OncologyAstraZeneca has a deep-rooted heritage in Oncology and offers a quickly growing portfolio of new medicines that has the potential to transform patients lives and the Companys future. With at least six new medicines to be launched between 2014 and 2020, and a broad pipeline of small molecules and biologics in development, we are committed to advance Oncology as a key growth driver for AstraZeneca focused on lung, ovarian, breast and blood cancers. In addition to our core capabilities, we actively pursue innovative partnerships and investments that accelerate the delivery of our strategy, as illustrated by our investment in Acerta Pharma in hematology.

By harnessing the power of four scientific platforms Immuno-Oncology, Tumor Drivers and Resistance, DNA Damage Response and Antibody Drug Conjugates and by championing the development of personalized combinations, AstraZeneca has the vision to redefine cancer treatment and one day eliminate cancer as a cause of death.

About AstraZenecaAstraZeneca is a global, science-led biopharmaceutical company that focuses on the discovery, development and commercialization of prescription medicines, primarily for the treatment of diseases in three main therapy areas - Oncology, Cardiovascular, Renal & Metabolism and Respiratory. The Company also is selectively active in the areas of autoimmunity, neuroscience and infection. AstraZeneca operates in over 100 countries and its innovative medicines are used by millions of patients worldwide. For more information, please visit http://www.astrazeneca-us.com and follow us on Twitter @AstraZenecaUS.

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LYNPARZA (Olaparib) Approved in US as 1st-line Maintenance Treatment With bevacizumab For HRD-Positive Advanced Ovarian Cancer - Business Wire

A decade ago, scientists won the nobel prize for connecting the dots between telomeres and aging. Telomeres are the protective caps on the ends of the strands of DNA called chromosomes. Telomeres shorten every time a cell divides. When they become very short, they trigger cell crisis and cell death.

Since this discovery was made, researchers have found new insights into telomeres and their connection to wrinkled skin, as well as how their length might be extended.

Scientists have uncovered new information on telomeres that could help combat the effects of inflammation and aging. Researchers from the University of Pittsburg recently discovered how oxidative stress plays a critical in the link between telomeres and cancer. I dont want to blind you with science (telomeres are a complex area), but every breakthrough into telomeres will ultimately equal a breakthrough in understanding how we age and how we can keep cells including our skin cells healthy.

Telomeres are composed of repeated sequences of DNA. The results from this study suggest that the mechanism by which oxidative stress accelerates telomere shortening is by damaging the DNA precursor molecules, not the telomere itself. This will have a big impact on appreciating how to manage oxidative stress to prevent aging and diseases such as cancer.

At Stanford University School of Medicine, scientists claim they can now lengthen teleomeres. Skin cells with telomeres lengthened by the procedure were able to divide up to 40 more times than untreated cells (source).

However, longer teleomeres is not necessarily associated with a longer life (source). Indeed, those naysayers say that good diet and an exercise regimen will do that. Yet, it does seem that preventing further shortening of the telomeres could be beneficial, especially for aging skin. Telomeres are likened to the tips of shoelaces that stop them unraveling and so you really want to be thinking about how to stabilize your telomeres. Happily, advances in skin care can help you out. There are three approaches to consider: special ingredients that target telomeres, ingredients that prevent oxidative damage (known, of course, as antioxidants) and stem cells.

Target your telomeres

One very interesting ingredient is called astragalus, and although it is rare, we do have a couple of Truth In Aging finds with it. But first, what is it and how does it work? In 2008, a UCLA AIDS Institute study found that a chemical they called TAT2 from the astragalus root, which is frequently used in Chinese herbal therapy, can prevent or slow the progressive shortening of telomeres. It can be found in Prana Reishi Mushroom Shield ($42 in the shop) and ExPrtise Effective Anti-Aging Face Serum ($120 in the shop).

Another ingredient to look for is treprenone, also marketed under the name of Renovage. Its promise is to stabilize telomeres, so at the very least they won't shorten. Maintaining telomere length extends the Hayflick Limit (the rate at which cells turn over before conking out completely) by one third. Youll find treprenone (Renovage) in Your Best Face Correct ($150 in the shop) Your Best Face Boost ($65 in the shop.

Amp up with antioxidants

The research is clear: Preventing oxidative damage is the job of an antioxidant. Free radicals are charged chemical particles of oxygen that enter into destructive chemical bonds with organic substances such as proteins, as explained by Gerald Imber, MD. Antioxidants limit the production of free radicals and therefore help prevent oxidative stress. There are many sources in plants, and vitamins are also antioxidants.

There are 33 antioxidants in Skin 2 Skin Aging Intervention Cream ($73 in the shop), including the universal antioxidant alpha lipoic acid. The powerful antioxidants in Your Best Face Rescue ($145 in the shop) include spin trap, an advanced form of vitamin C and EGCG. Bee venom has some 18 active compounds include antioxidants, peptides with powerful anti-inflammatory actions, and enzymes. It is the key ingredient in LaCrme Beaut Luxurious Bee Venom Rich Face Treatment Cream ($202 in the shop).

Look for plant stem cells

Plant stem cells never age. British scientists found that plant stem cells were much more sensitive to DNA damage than other cells. Once they sense damage, they trigger death of these cells before it spreads and causes more. In addition, they have the ability to stimulate cell renewal and replace specific cells in need of repair.

Ao Skincare Raw Nourish AM Treatment ($65 in the shop) has the powerful antioxidant astaxanthin and plant stem cells.

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New Study Suggests the Consequences of Oxidative Stress on Telomeres - Truth In Aging

Healthcare Business

Chris Lange

Aytu BioScience Inc. (NASDAQ: AYTU) has been a stock market darling since it broke onto the COVID-19 testing scene. As a result, the stock has absolutely exploded this year. Although many were concerned at the onset of the year about Nasdaq compliance, the stock price quickly quieted these concerns when COVID-19 hit the scene.

Aytu BioScience stock traded as low as $0.34 a share in February before it came out with a test to help fight the pandemic. Since then, the shares have reached as high as $2.99, but they have normalized and found a trading range between $1.25 and $1.65.

Testing for the coronavirus has become a lucrative field. Other companies specifically dealing with this include Quest Diagnostics Inc. (NYSE: DGX), Co-Diagnostics Inc. (NASDAQ: CODX) and Becton, Dickinson and Co. (NYSE: BDX). Their stocks have seen solid gains since they entered the coronavirus test business as well.

Aytu BioScience is a specialty pharmaceutical company. It focuses on the development and commercialization of novel products in the fields of hypogonadism (low testosterone), cough and upper respiratory symptoms, insomnia, and male infertility.

What has set this company apart from the rest is that it has secured an exclusive distribution agreement for the right to commercialize a rapid COVID-19 test. The company said that the test has been licensed from L.B. Resources in Hong Kong. It licensed North American rights from a product developer named Zhejiang Orient Gene Biotech.

The test is intended for professional use, at point of care. What stands out here is that the test is said to deliver clinical results in two to 10 minutes, wherever it is administered.

Late in April, the company announced that it has partnered with Sterling Medical Devices to finalize the development of the Healight platform technology. Basically, this tech is a novel endotracheal catheter that acts as a potential treatment for the coronavirus.

Management believes the Healight platform technology has the potential to affect outcomes positively for critically ill patients infected with coronavirus and other infections. Aytu, with support of the team at Cedars-Sinai, is working with the U.S. Food and Drug Administration (FDA). The aim is to determine an expedited regulatory process with the potential to enable near-term use of the technology initially as a coronavirus intervention for critically ill intubated patients.

Aytu BioScience held its 2020 annual meeting in late April. A few key developments there will have an impact on the direction of the company. To start, a few new directors will get their turn on the board.

In the meeting, stockholders elected Joshua Disbrow, Steven Boyd, Gary Cantrell, Carl Dockery, John Donofrio Jr., Michael Macaluso and Ketan Mehta to the companys board of directors for one-year terms.

Separately, stockholders approved an advisory vote on executive compensation and ratified the appointment of Plante & Moran as the independent auditor for the company for the 2020 year. Stockholders also approved the board of directors to authorize a reverse split of the companys common stock.

However, the board did not elect to proceed with a reverse stock split at that time. This decision is the result of Aytu regaining compliance with Nasdaq listing requirements, including having a $1.00 closing bid price for the companys common stock. Additionally, the board noted that effecting a reverse split would not be in the best interest of shareholders at the time.

Check out the companys 8-K from this meeting on the U.S. Securities and Exchange Commission website.

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COVID-19 Testing Continues to Lift Aytu BioScience Stock - 24/7 Wall St.

Advancing your induced pluripotent stem cells or human embryonic stem cell therapy research to clinical applications requires careful material selection because the quality of starting materials significantly impact the properties of your final stem cell therapy product. Gibco CTS products have been developed to ease the transition from stem cell therapy research to clinical applications by providing high quality GMP manufactured, commercial scale ancillary materials with a high degree of qualification, traceability and regulatory documentation. In an effort to help you maximize the potential of your stem cell research and therapy, and simplify the transition to clinic-ready processes, we offer an extensive selection of research use stem cell research products with complementary CTS formulations. Our CTS products are used in commercially approved cell therapies as well as over 100 clinical trials and are backed by our professional regulatory support and over 30 years of GMP manufacturing experience.

Induced pluripotent stem cells (iPSCs) and embryonic stem cells (ESCs) , sometimes collectively referred to as pluripotent stem cells (PSCs), are cells that have the ability to renew themselves indefinitely and differentiate into almost any cell type when exposed to the right microenvironment. These unique properties enable the application of induced pluripotent stem cells and embryonic stem cells in disease modeling, drug discovery, drug toxicity testing, and cell therapy. Strikingly, most embryonic stem cell and induced pluripotent stem cell applications have the potential to improve human health, none more directly so than ESC or iPSC therapy. The most intuitive approach for ES or iPS cell therapy is to transplant PSC-derived cells for the direct replacement of damaged or degenerated cells or tissue. However, there are many other approaches to ES or iPS stem cell therapy such as transplanting PSC-derived cells that then release signals triggering endogenous repair mechanisms.

At Thermo Fisher Scientific, we support the development of your human embryonic stem cell therapy or induced pluripotent stem cell therapy from the earliest stages of research and all the way to the clinic. We offer high-quality products across the iPS cell therapy workflow from reprogramming to differentiation. Most Gibco media and supplements for culture and differentiation are manufactured under GMP conditions at sites that use methods and controls that conform to current Good Manufacturing Practices (cGMP) for medical devices. These manufacturing sites are ISO 13485 and ISO 9001certified, and the rigorous practices we adhere to at these sites help ensure the consistency, reliability, and high quality of a wide variety of iPSC therapy workflow reagents.

To further help you maximize the potential of your research and streamline your transition to the clinic, we offer Gibco Cell Therapy Systems (CTS) equivalents for many of our research-use products. In addition to GMP manufacturing, Gibco CTS products undergo quality control testing and are accompanied by appropriate documentation so you can transition your cell therapy to the clinic with confidence.

*Adherence to supplier related responsibilities of USP<1043>

First off-the-shelf reprogramming system manufactured in accordance with GMP requirements. CTS CytoTune 2.1 kit offers high-efficiency Sendai delivery of reprogramming factors.

Click image to enlarge

Efficient reprogramming from adult human dermal fibroblasts, T cells, and CD34+ cells. These data demonstrate that the CytoTune-iPS 2.1 kit can be used to successfully reprogram human dermal fibroblasts (HDFa), T cells, and CD34+ cells.

Gibco CTS Essential 6 Medium is a xeno-free, feeder-free, cGMP-manufactured medium which supports the spontaneous or directed differentiation of human pluripotent stem cells (PSCs) and the reprogramming of somatic cells.

Based on the widely cited Gibco Essential 8 Medium, Gibco CTS Essential 8 Medium is the first globally available human- and animal originfree culture medium for human pluripotent stem cells (hPSCs) and is designed to meet international regulatory requirements for cell therapy.

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Using Applied Biosystems TaqMan hPSC Scorecard Panel analysis, Gibco CTS Essential 8 Medium and research-use-only Essential 8 Medium were shown to support comparable expression of PSC markers and lineage markers in undifferentiated PSCs and PSC-derived embryoid bodies.

CTS Vitronectin (VTN-N) Recombinant Human Protein is a defined matrix for feeder-free culture of iPSCs. Designed in the laboratory of James Thomson, this recombinant protein is intended for use with the CTS Essential 8 culture system.

CTS RevitaCell Supplement (100X) is an animal-origin-free, chemically defined supplement used with PSCs for post-thaw recovery or in combination with CTS Essential 8 Medium for single cell passaging. To minimize both the loss of cell viability and differentiation of PSCs, use the CTS PSC Cryopreservation Kit.

CTS Versene is a gentle non-enzymatic cell dissociation reagent for use in routine clump passaging of PSCs while maintaining viability over multiple passages.

For the cryopreservation and recovery of PSCs, the CTS PSC Cryopreservation Medium and CTS RevitaCell Supplement minimize the loss of cell viability and maximize post-thaw recovery when used in combination. Both reagents are included in the CTS PSC Cryopreservation Kit.

The CTS PSC Cryopreservation Medium is a xeno-free solution for the cryopreservation of pluripotent stem cells (PSCs). Both CTS PSC Cryopreservation Medium and CTS RevitaCell supplement are included in the CTS PSC Cryopreservation Kit that helps minimize loss of cell viability and maximize post-thaw recovery.

CTS KnockOut SR XenoFree Medium is a defined, xeno-free serum replacement based on the traditional Gibco KnockOut Serum Replacement, which has been cited in more than 2,000 publications and trusted for over 20 years.

Maintenance of pluripotency using CTS KNOCKOUT SR XenoFree Medium. Following 10 passages in either KSR (left lane) or KSR XenoFree CTS (right lane) on HFF attached with CELLstart substrate, BG01v gene expression was examined (top). Gene expression of embryoid bodies generated from the same P10 BG01v/HFF cultures (bottom).

Your choice of chemically defined human- and animal origin-free basal media for pluripotent stem cell culture. Based on traditional DMEM and DMEM/F12 formulations, these basal media are:

We offer full customization options to help meet your unique specifications for any project. Flexibility is yours in creating your own Gibco custom cell culture medium

Intended use of the products mentioned on this page vary. For specific intended use statements please refer to the product label.

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Induced Pluripotent Stem Cell (iPSC) Media and Reagents ...

Early next year, a small clinical trial will begin in Japan, marking the first time reprogrammed stem cells will be deployed to help regenerate injured hearts. A team led by Osaka University cardiac surgeon Yoshiki Sawa will implant sheetseach consisting of 100 million stem-cell derived cardiomyocytesonto the hearts of three patients with advanced heart failure.

Its a big deal theyve gotten approval to do this, remarks Jalees Rehman, associate professor of medicine and pharmacology at the University of Illinois at Chicago.

The cardiac study is one of the first few clinical applications of induced pluripotent stem (iPS) cells, the first being an iPS cell-based treatment of macular degeneration of the eye, which also took place in Japan.While it is a big deal to pioneer such a technology clinically, the trial also has its risks, unknowns, and critics.

Japans health ministry conditionally approved the heart experiment in May, with the goal of assessing the safety of the procedure. If the first trial and a later one enrolling 10 patients prove successful, the treatment will be made commercially available soon under a new fast-track system in Japan designed to speed up the development of regenerative therapies.

Since the trial was announced, several Japanese researchers have voiced their concerns in comments and correspondences in Nature. One of them, Akira Akayabashi, a professor of biomedical ethics at Tokyo University, notes that the trail participants will receive iPS-derived cells from a donor, instead of from their own tissue, and will have to be placed on immunosuppressants for three months to prevent rejection. It will add extra burden of using immune suppressants to heart failure patients who are already suffering, he tells The Scientist.

Im really happy there are countries in the world that are really prioritizing the role of stem cells.

Phillip Yang, Stanford University

Sawa says that creating cardiomyocytes derived from a patients own cells is not always an option, because the reprogramming process takes a long time. Additionally, providing off-the-shelf treatments is a more feasible route to address heart failure, he says. Cell therapy [using a patients own cells] seems to be not suitable for industrialization, he says.

While preclinical work with iPS cells has proven effective in improving heart function in mice, pig, and monkey models, its not quite clear by which mechanism the cells are promoting muscle regeneration. Its still unknown whether these cells actually integrate into the heart and become beating heart cells, or whether they just release factors and help existing heart cells, Rehman says.

Sawas research in pigs suggests that iPS cellderived cardiomyocytes promote regeneration of the heart by secreting certain cytokines that stimulate the native heart muscle to grow, he explains. In contrast to skeletal myoblasts taken from patients thighswhich he is testing in another clinical trial for heart failurethe cardiac cells derived from iPS cells have additional cytokines [that] seem to be very helpful, he tells The Scientist.

The use of the cell sheets [in humans] is intriguing, says Phillip Yang, an associate professor of cardiovascular medicine at Stanford University. Ultimately, [the procedure] would help find out if this method of application will work or not, he says. However, if the secretions are what helps repair the heart, he questions why implanting the cells is necessary. Instead, Yang and others are studying potential regenerative therapies by isolating and injecting these regenerative factors into pigs hearts.

Yang says he is most concerned with the survival of the reprogrammed cardiomyocytes within the heart. The likelihood of a cell, a very fragile, iPS-derived cell, surviving in an area where there is a large amount of injury, lack of blood, lack of oxygen supply is very low, he says. If they disappear quickly, the treatment wont be effective.

Another concern is that implanting the cells as a sheet will likely involve open-heart surgery. Because they are making a sheet of cells, they cannot just inject it into the heart, Rehman says, adding that injecting the cells is a much less invasive approach. And given that the patients are already suffering from severe heart failure, is the surgery itself going to be a risk for them? he asks.

There are other risks associated with introducing the new cells into the heart, Rehman says. They could potentially become tumorigenic, because they originate from a very proliferative cell type. The possibility of heart rhythm problems could also be an issue, he says. This became apparent when researchers at the University of Washington injected monkey hearts with cardiomyocytes derived from human embryonic stem cells.

Regardless of the concerns, Yang is excited to see the outcome of Sawas trial, and the new information it will bring to the field.

Im really happy there are countries in the world that are really prioritizing the role of stem cells, Yang says. Since stem cell researcher Shinya Yamanaka received the Nobel prize for the development of iPS cells in 2012, the field has been booming in Japan. Four years ago, the Japanese government decided to put more than $1 billion USD towards regenerative medicine research, a quarter of which goes to a project to develop stocks of iPS cells for biomedical research.

If there was a really effective treatment for heart failurewhich is still the number one cause of hospital admission in this country and so forthI think that would be great, Yang says. But whether iPS-derived cardiomyocytes is the answer, and much better than anything else, that Im not so sure.

Correction (November 14): A previous version of this article incorrectly stated that this study is the second-ever clinical applications of iPS cells, when it is in fact one of several.The Scientistregrets the error.

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First iPS Cell Trial for Heart Disease Raises Excitement ...

People who are overweight and suffering from joint pain caused by osteoarthritis are often reluctant to exercise, even though physical activity can boost muscle strength and relieve pain. A new study suggests gene therapy may someday be a good option for those peopleand it may help them shed pounds, too.

Researchers at the Washington University School of Medicine gave young mice a single injection of the gene that makes follistatin, a protein that normally blocks another protein called myostatin, which modulates muscle growth. The therapy caused a significant buildup of muscle mass in the mice while also preventing obesity, the team reported in the journal Science Advances.

We've identified here a way to use gene therapy to build muscle quickly, said senior investigator Farshid Guilak, Ph.D., professor of orthopaedic surgery and director of research at Shriners Hospitals for Children St. Louis, in a statement. 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."

In fact, the mice didnt just build muscle, they also nearly doubled their strength without exercising any more than they usually did. Despite being fed a high-fat diet, they had fewer metabolic issues and stronger hearts than did animals that did not receive the follistatin gene. Their joints were healthier, with less cartilage damage and inflammatory markers than their untreated counterparts, the researchers reported.

Whats more, the Washington University team discovered that the gene therapy promoted the beiging of white fat, meaning it turned some unhealthy white adipose tissue into brown fat, which positively correlates with increased triglyceride clearance, normalized glucose level, and reduced inflammation, the researchers wrote in the study. Therefore, delivering the follistatin gene could serve as a very promising approach to induce beiging of [white adipose tissue] in obesity, they wrote.

RELATED: Could gene therapy be the solution to obesity and diabetes?

This is not the first time gene therapy has been proposed as a potential treatment for obesity and other metabolic diseases. Australian researchers demonstrated that removing the gene RCAN1 from mice, for example, helped turn white fat into brown fat. And a team in South Korea used the gene editing system CRISPR to remove the FABP4 gene from mice that had been fed a high-fat diet, resulting in a 20% loss of body weight and a reduction in insulin resistance.

The Washington University teams approach is distinctive in that it focuses on building muscle. But the researchers noted theyll have to do further studies to determine whether the gene therapy has any negative effect on heart muscle. Even though heart health improved in the mice, any thickening of the hearts walls could be dangerous over time.

Still, Guilak and his colleagues believe that follistatin gene therapy could be a promising approach to treating several conditions, including muscular dystrophy and other diseases that cause muscle wasting, they said in the study.

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Gene therapy cuts fat and builds muscle in sedentary mice on unhealthy diets - FierceBiotech

California-based Kriya Therapeutics announced on Tuesday that it concluded an $80.5 million Series A financing round, led by QVT, Dexcel Pharma, Foresite Capital, Bluebird Ventures, Narya Capital, Amplo, Paul Manning, and Asia Alpha. The company, which was founded in 2019, has a pipeline that includes multiple AAV-based gene therapies for the treatment of type 1 and type 2 diabetes, as well as obesity.

"There have been numerous successful gene therapies focused on rare monogenic diseases in recent years," said Shankar Ramaswamy, M.D., Co-Founder, Chairman, and CEO of Kriya Therapeutics. "We see tremendous potential to expand the field and apply gene therapy to highly prevalent serious diseases. We are focused on designing gene therapies using algorithmic tools, scalable infrastructure, and proprietary technology to optimize the efficacy and durability of our treatments. We look forward to accelerating the development of our pipeline, platform technologies, and internal GMP manufacturing capability with the funds raised in this Series A financing."

Kriya focuses on developing gene therapies for conditions that impact millions of patients. Its goal is to design one-time gene therapies to express therapeutic proteins within specific human tissues. Kriyas pipeline includes KT-A112, KT-A522, and KT-A832, all of which are investigational gene therapies.

Kriyas leadership team is composed of experts who have experience designing, developing and manufacturing successful gene therapies as well.

"Kriya is building a leading team and cutting-edge infrastructure to engineer best-in-class gene therapies for severe chronic conditions and accelerate their advancement into human clinical trials," said Roger Jeffs, Ph.D., Co-Founder and Vice Chairman of Kriya. "The company is committed to incorporating the latest advancements in the field into the design and development of its therapeutic constructs. Through its R&D laboratory capabilities in the Bay Area and in-house process development and manufacturing infrastructure in Research Triangle Park, I believe that Kriya will be uniquely positioned to become a leader in the gene therapy field."

Another company with a focus on gene therapy that recently came out of stealth mode is Dyno Therapeutics. On Monday, the Massachusetts-based organization announced that it is now eligible for more than $2 billion in upfront payments, research support and various milestones and options fees through its research-and-development and collaboration deals.

Dyno, which launched in 2018 with approximately $9 million in financing, has a technology platform built on the intellectual property that came from the laboratory of George Church. Church, who is the Robert Winthrop Professor of Genetics at Harvard Medical School, is a cofounder of Dyno.

At Dyno, we see a vast opportunity to expand the treatment landscape for gene therapies, said Eric D. Kelsic, co-founder and chief executive officer of Dyno. The success of gene therapy relies on the ability of vectors to safely and precisely deliver a gene to the intended target cells and tissues. Our approach addresses the major limitations of naturally occurring AAV vectors and creates optimized, disease-specific vectors for gene therapies with great curative potential. Our portfolio of R&D programs and newly-announced collaborations with leading gene therapy developers reflect the applicability of our AI-powered approach to improve treatments for patients and expand the number of treatable diseases with gene therapies.

Dyno has announced partnerships with Novartis and Sarepta Therapeutics thus far. With Sarepta, Dyno will design and discover novel AAV capsids to improve gene therapy for muscle diseases. Along with Novartis, Dyno will focus on developing improved AAV vectors for gene therapies for ocular diseases.

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Kriya Therapeutics to Focus on Gene Therapy with $80.5M in Funding - BioSpace

A gene therapy developer emerging from stealth mode said today it could generate more than $2 billion through collaborations launched with Novartis and Sarepta Therapeutics to develop treatments based on a delivery platform developed in the lab of George Church, PhD, of Harvard Medical School.

Dyno Therapeutics said it will partner with Novartis on research, development, and commercialization of new gene therapies for eye diseases incorporating improved adeno-associated virus (AAV) vectors. Separately, Dyno will collaborate with Sarepta on gene therapies for muscle diseases.

Both collaborations will apply Dynos CapsidMap platform, which uses artificial intelligence (AI) to design novel capsids that confer improved functional properties to AAV vectors. At the core of CapsidMap, according to Dyno, are advanced search algorithms applying machine learning and the companys large quantities of experimental data.

CapsidMap is designed to integrate DNA library synthesis and next generation DNA sequencing to measureinvivogene delivery properties in high throughput.The capsid platform is designed to expand the universe of diseases treatable via gene therapy by improving upon present-day AAV vectors, which are limited by delivery, immunity, packaging size, and manufacturing challenges.

Our portfolio of R&D programs and newly-announced collaborations with leading gene therapy developers reflect the applicability of our AI-powered approach to improve treatments for patients and expand the number of treatable diseases with gene therapies, Eric Kelsic, PhD, a former postdoc of Church who is Dynos CEO and Co-founder, said in a statement. We see a vast opportunity to expand the treatment landscape for gene therapies.

Dyno has an exclusive option to enter into a license agreement with Harvard University for the CapsidMap technology, which was developed in the lab of Church, a co-founder of Dyno and chairman of its scientific advisory board. Church is the Robert Winthrop Professor of Genetics at Harvard Medical School and a Core Faculty member at Harvards Wyss Institute for Biologically Inspired Engineering.

Church and other co-founders and members of his lab at HMS and the Wyss Institute carried out work that underpin Dynos approach to AAV capsid engineering, described in Comprehensive AAV capsid fitness landscape reveals a viral gene and enables machine-guided design, a paper published November 29, 2019, in the journal Science.

In that paper, Church, Kelsic, and two co-authors detailed how they mutated one-by-one each of the 735 amino acids within the AAV2 capsid, including all possible codon substitutions, insertions and deletions at each position. They generated a virus library containing about 200,000 variants and identified capsid changes that both maintained AAV2s viability and improved its homing potential (tropism) to specific organs in mice.

Unexpectedly, the team also discovered a new accessory protein hidden within the capsid-encoding DNA sequence that binds to the membrane of target cells, playing a significant role in viral production.

Our comprehensive, machine-guided design strategy generated viable mutants in a principled and high-throughput manner and is generalizable to other proteins and engineering challenges, the researchers reported. Applied to AAV, such methods now enable the systematic optimization of natural capsids into synthetic variants with enhanced properties for emerging gene therapies.

Under its partnership with Novartis, Dyno will be responsible for using AI technology and its suite of machine learning and experimental tools for the design and discovery of novel AAV capsids. Novartis agreed to conduct preclinical, clinical, and commercialization activities for the gene therapy product candidates created with the novel AAV capsids.

Dyno and Novartis did not disclose specific financial terms of their collaboration. They did say, however, that Novartis agreed to pay Dyno an upfront fee plus committed research funding and license fees. Dyno will be eligible to receive clinical, regulatory and sales milestone payments. Dyno will also receive royalties on worldwide net sales of any commercial products developed through the partnership.

Many eye diseases are ideally suited to being treated with gene therapies, and more opportunities can be opened with new and improved AAV vectors, Kelsic added. With their extensive ophthalmologic expertise, Novartis is an ideal partner to leverage Dynos platform to design AI-powered vectors to expand the impact of gene therapies for ocular diseases.

Under its collaboration with Sarepta, Dyno agreed to oversee the design and discovery of novel AAV capsids with improved functional properties for gene therapy, while Sarepta agreed to take responsibility for conducting preclinical, clinical and commercialization activities for gene therapy product candidates using the novel capsids.

Our agreement with Dyno provides us with another valuable tool to develop next-generation capsids for gene therapies to treat rare diseases, stated Doug Ingram, Sareptas president and CEO. By leveraging Dynos AI platform and Sareptas deep expertise in gene therapy development, our goal is to advance next-generation treatments with improved muscle-targeting capabilities.

Sarepta agreed to pay Dyno a total $40 million in in upfront, option, and license payments during the research phase of their collaboration. Should Sarepta develop and commercialize multiple candidates for multiple muscle diseases, Dyno said, it will be eligible for additional significant future milestone payments. Dyno will also receive royalties on worldwide net sales of any commercial products developed through the collaboration.

Dyno was launched in late 2018 with a $9 million financing co-led by Polaris Partners and CRV. Alan Crane, a co-founder of Dyno and entrepreneur partner at Polaris Partners, and Dylan Morris, general partner at CRV, have joined Dynos board, with Crane serving as Dynos executive chairman.

Dyno said it did not anticipate the need for additional fundraising, based on the significant financial resources made available through its collaborations.

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Dyno Inks Up to $2B+ in Gene Therapy Partnerships with Novartis, Sarepta - Genetic Engineering & Biotechnology News

NEW YORK--(BUSINESS WIRE)--Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) (Rocket), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare disorders, today presents new clinical data supporting longer-term efficacy and durability of gene therapy for Fanconi Anemia (FA) and Leukocyte Adhesion Deficiency-I (LAD-I) at the 23rd Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT) being held virtually May 12-15, 2020. Two oral presentations highlight updates from the companys Phase 1/2 study of RP-L201 for the treatment of severe LAD-I and the Phase 1/2 study of RP-L102 Process A for the treatment of FA.

The latest additional data from both our LAD-I and FA programs demonstrate sustained engraftment and durable clinical impact, said Jonathan D. Schwartz, M.D., Chief Medical Officer and Senior Vice President of Rocket. These results further support the viability of gene therapy in LAD-I and FA, disorders in which bone marrow transplant is the primary curative option and is associated with high rates of toxicity.

Patients with severe LAD-I have neutrophil CD18 expression of less than 2% of normal, with extremely high mortality in early childhood, said Dr. Schwartz. In this first patient treated with RP-L201 using Process B, an increase from less than 1% to 47% CD18 expression sustained over six months demonstrates that RP-L201 has the potential to correct the neutrophil deficiency that is the hallmark of LAD-I. We are also pleased with the continued visible improvement of multiple disease-related skin lesions. These results lend further support to the applicability of Process B across the lentiviral portfolio. The second patient has also recently been treated, and we look forward to completing the Phase 1 portion of the registrational trial for this program.

Dr. Schwartz continued, In our FA program, patients followed for a year or more after treatment with RP-L102 Process A continue to demonstrate durable engraftment and hematologic correction, without the use of pre-treatment conditioning regimens. All six patients who received minimally adequate drug product and were followed for more than one year display sustained and progressive engraftment. Notably, hemoglobin levels have normalized to baseline in two patients treated. Todays update not only gives us confidence as we transition to our improved Process B drug product, but also supports the potential of gene therapy in the absence of any conditioning regimen as a definitive hematologic treatment for FA. The ability to treat patients without the side effects associated with allogeneic transplant or the use of genotoxic conditioning, and to restore blood cell counts is a major milestone for the FA scientific community.

Details on Rockets oral presentations at ASGCT:

Title: A Phase 1/2 Study of Lentiviral-mediated Ex-vivo Gene Therapy for Pediatric Patients with Severe Leukocyte Adhesion Deficiency-I (LAD-I): Initial Results from the First Treated Patient Session: HSPC Gene Therapies for Blood and Immune DisordersPresenter: Donald B. Kohn, M.D., Professor of Microbiology, Immunology and Molecular Genetics, Pediatrics (Hematology/Oncology), Molecular and Medical Pharmacology, member of the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at UCLA and principal investigator of the Phase 1 trialDate: Tuesday May 12, 2020Time: 4:30 p.m. - 4:45 p.m. EDT

Additional results from the first patient treated with RP-L201 for LAD-I continue to demonstrate evidence of safety and potential efficacy. Analyses of peripheral vector copy number (VCN) and CD18-expressing neutrophils were performed six months post treatment with RP-L201 to evaluate engraftment and phenotypic correction. The patient demonstrated peripheral blood VCN levels of 1.3 and CD18-expression of 47%, which is sustained from the 45% expression observed three months post treatment; pretreatment CD18 expression was <1%. The drug product VCN was 3.8. Additionally, the patient continues to display visible improvement of skin lesions. No safety or tolerability issues related to RP-L201 administration have been identified to date.

RP-L201 was in-licensed from the Centro de Investigaciones Energticas, Medioambientales y Tecnolgicas (CIEMAT), Centro de Investigacin Biomdica en Red de Enfermedades Raras (CIBERER) and Instituto de Investigacin Sanitaria Fundacin Jimnez Daz (IIS-FJD). The lentiviral vector was developed in a collaboration between The University College of London (UCL) and CIEMAT.

Title: Updated Results of a European Gene Therapy Trial in Fanconi Anemia Patients, Subtype ASession: HSPC Gene Therapies for Blood and Immune DisordersPresenter: Juan A. Bueren, Ph.D., Scientific Director of the FA gene therapy program and Head of the Hematopoietic Innovative Therapies Division at CIEMAT in Spain / CIBERER / IIS-FJD Date: Tuesday May 12, 2020Time: 4:45 p.m. - 5:00 p.m. EDT

Nine pediatric patients have been enrolled and treated in the Phase 1/2 clinical trial of RP-L102 Process A for the treatment of Fanconi Anemia, seven of whom are evaluable at or beyond the one year mark following treatment. The first four patients (02002, 02004, 02005 and 02006) exhibit robust and durable engraftment, continued hematologic correction and blood count stabilization. Importantly, hemoglobin levels for patients 02002 and 02006 have increased to a healthy, normal range; these patients received more optimal product consistent with the minimal dose criteria established for the Process B registrational program. Two additional patients (02008 and 02013) who have been followed for a year or more after treatment display early evidence of engraftment, as measured by increases in peripheral blood VCNs. Patient 02007 received a lower than optimal dose and is beginning to demonstrate preliminary signs of engraftment. Blood counts are not yet available in these patients. Two patients, patients 01003 and 02009, have not been included in this analysis. Patient 02009 is only six months post treatment and will continue to be followed. Patient 01003 received a drug product that did not meet full release criteria due to a technical issue this was a one-time lab-specific issue that was addressed. To date, no patients in this trial have undergone allogeneic bone marrow transplant.

RP-L102 is being developed in conjunction with CIEMAT, CIBERER and IIS-FJD.

The presentations will be made available on Rockets website at http://www.rocketpharma.com/asgct-presentations/ following presentation at the conference.

About Leukocyte Adhesion Deficiency-ISevere Leukocyte Adhesion Deficiency-I (LAD-I) is a rare, autosomal recessive pediatric disease caused by mutations in the ITGB2 gene encoding for the beta-2 integrin component CD18. CD18 is a key protein that facilitates leukocyte adhesion and extravasation from blood vessels to combat infections. As a result, children with severe LAD-I are often affected immediately after birth. During infancy, they suffer from recurrent life-threatening bacterial and fungal infections that respond poorly to antibiotics and require frequent hospitalizations. Children who survive infancy experience recurrent severe infections including pneumonia, gingival ulcers, necrotic skin ulcers, and septicemia. Without a successful bone marrow transplant, mortality in patients with severe LAD-I is 60-75% prior to the age of 2 and survival beyond the age of 5 is uncommon. There is a high unmet medical need for patients with severe LAD-I.

Rockets LAD-I research is made possible by a grant from the California Institute for Regenerative Medicine (Grant Number CLIN2-11480). The contents of this press release are solely the responsibility of Rocket and do not necessarily represent the official views of CIRM or any other agency of the State of California.

About Fanconi AnemiaFanconi Anemia (FA) is a rare pediatric disease characterized by bone marrow failure, malformations and cancer predisposition. The primary cause of death among patients with FA is bone marrow failure, which typically occurs during the first decade of life. Allogeneic hematopoietic stem cell transplantation (HSCT), when available, corrects the hematologic component of FA, but requires myeloablative conditioning. Graft-versus-host disease, a known complication of allogeneic HSCT, is associated with an increased risk of solid tumors, mainly squamous cell carcinomas of the head and neck region. Approximately 60-70% of patients with FA have a Fanconi Anemia complementation group A (FANCA) gene mutation, which encodes for a protein essential for DNA repair. Mutation in the FANCA gene leads to chromosomal breakage and increased sensitivity to oxidative and environmental stress. Increased sensitivity to DNA-alkylating agents such as mitomycin-C (MMC) or diepoxybutane (DEB) is a gold standard test for FA diagnosis. Somatic mosaicism occurs when there is a spontaneous correction of the mutated gene that can lead to stabilization or correction of a FA patients blood counts in the absence of any administered therapy. Somatic mosaicism, often referred to as natural gene therapy provides a strong rationale for the development of FA gene therapy because of the selective growth advantage of gene-corrected hematopoietic stem cells over FA cells1.

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

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

Rocket Cautionary Statement Regarding Forward-Looking StatementsVarious statements in this release concerning Rocket's future expectations, plans and prospects, including without limitation, Rocket's expectations regarding its guidance for 2020 in light of COVID-19, the safety, effectiveness and timing of product candidates that Rocket may develop, to treat Fanconi Anemia (FA), Leukocyte Adhesion Deficiency-I (LAD-I), Pyruvate Kinase Deficiency (PKD), Infantile Malignant Osteopetrosis (IMO) and Danon Disease, and the safety, effectiveness and timing of related pre-clinical studies and clinical trials, may constitute forward-looking statements for the purposes of the safe harbor provisions under the Private Securities Litigation Reform Act of 1995 and other federal securities laws and are subject to substantial risks, uncertainties and assumptions. You should not place reliance on these forward-looking statements, which often include words such as "believe," "expect," "anticipate," "intend," "plan," "will give," "estimate," "seek," "will," "may," "suggest" or similar terms, variations of such terms or the negative of those terms. Although Rocket believes that the expectations reflected in the forward-looking statements are reasonable, Rocket cannot guarantee such outcomes. Actual results may differ materially from those indicated by these forward-looking statements as a result of various important factors, including, without limitation, Rocket's ability to monitor the impact of COVID-19 on its business operations and take steps to ensure the safety of patients, families and employees, the interest from patients and families for participation in each of Rockets ongoing trials, our expectations regarding when clinical trial sites will resume normal business operations, our expectations regarding the delays and impact of COVID-19 on clinical sites, patient enrollment, trial timelines and data readouts, our expectations regarding our drug supply for our ongoing and anticipated trials, actions of regulatory agencies, which may affect the initiation, timing and progress of pre-clinical studies and clinical trials of its product candidates, Rocket's dependence on third parties for development, manufacture, marketing, sales and distribution of product candidates, the outcome of litigation, and unexpected expenditures, as well as those risks more fully discussed in the section entitled "Risk Factors" in Rocket's Quarterly Report on Form 10-Q for the quarter ended March 31, 2020, filed May 8, 2020 with the SEC. Accordingly, you should not place undue reliance on these forward-looking statements. All such statements speak only as of the date made, and Rocket undertakes no obligation to update or revise publicly any forward-looking statements, whether as a result of new information, future events or otherwise.

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Rocket Pharmaceuticals Presents Positive Updates on FA and LAD-I Gene Therapy Programs at the 23rd Annual Meeting of the American Society of Gene and...

SOUTH SAN FRANCISCO, Calif.--(BUSINESS WIRE)--Atara Biotherapeutics, Inc. (Nasdaq: ATRA), a pioneer in T-cell immunotherapy leveraging its novel allogeneic EBV T-cell platform to develop transformative therapies for patients with severe diseases including solid tumors, hematologic cancers and autoimmune diseases, today announced the appointment of immunology and cell & gene therapy expert Maria Grazia Roncarolo, MD, to the Board of Directors.

Dr. Roncarolo is the George D. Smith Professor in Stem Cell and Regenerative Medicine, Professor of Pediatrics and Medicine, Director of the Center for Definitive and Curative Medicine, and Co-Director of the Institute for Stem Cell Biology and Regenerative Medicine at Stanford University.

In 2014, Dr. Roncarolo established the Stanford Center for Definitive and Curative Medicine. The center, which is dedicated to the development of innovative stem cell and gene therapies for patients with currently incurable diseases, spans a wide range of bench and clinical research activities from basic biology through translational research and features its own GMP cell processing and Phase 1 study units.

I am thrilled to have one of the worlds leading experts in immunology and T cells, Dr. Roncarolo, bringing to our Board her experience and strategic vision in cell therapy and gene editing as well as her passion for transformative immunotherapies, said Pascal Touchon, President and Chief Executive Officer of Atara. She has dedicated her life to caring for patients with severe immunological and hematological diseases and has an impressive record in translating scientific discoveries in cell and gene therapy into novel treatments which aligns very well with Ataras mission.

Dr. Roncarolo has served as the primary investigator in several landmark trials involving the development of innovative stem cell- and gene-based therapies. She worked at DNAX Research Institute for Molecular and Cellular Biology in Palo Alto for several years, where she contributed to the discovery of novel cytokines, cell-signaling molecules that are part of the immune response. She studied the role of these cytokines in inducing immunological tolerance and in promoting stem cell growth and differentiation. As Director of the Telethon Institute for Cell and Gene Therapy and the San Raffaele Scientific Institute in Milan, Dr. Roncarolo was the principal investigator leading the successful gene therapy trial in SCID, a severe life threatening disorder in which patients lack an enzyme critical to DNA synthesis.

Beyond studying new therapies, Dr. Roncarolo has also helped elucidate drivers of disease at the molecular and cellular level, as she has investigated the mechanisms of immune-mediated diseases throughout her career and helped advance the understanding of immunological tolerance. Dr. Roncarolo was the recipient of the outstanding achievement award from the European Society of Gene and Cell Therapy (ESGCT) in 2010 and from the American Society of Gene and Cell Therapy (ASGCT) in 2017. She is currently the president of the Federation of Clinical Immunology Societies.

About Atara Biotherapeutics

Atara Biotherapeutics, Inc. (@Atarabio) is a pioneer in T-cell immunotherapy leveraging its novel allogeneic EBV T-cell platform to develop transformative therapies for patients with severe diseases including solid tumors, hematologic cancers and autoimmune disease. With our lead program in Phase 3 clinical development, Atara is the most advanced allogeneic T-cell immunotherapy company and intends to rapidly deliver off-the-shelf treatments to patients with high unmet medical need. Our platform leverages the unique biology of EBV T cells and has the capability to treat a wide range of EBV-associated diseases, or other severe diseases through incorporation of engineered CARs (chimeric antigen receptors) or TCRs (T-cell receptors). Atara is applying this one platform to create a robust pipeline including: tab-cel (tabelecleucel) in Phase 3 development for Epstein-Barr virus-driven post-transplant lymphoproliferative disease (EBV+ PTLD); ATA188, a T-cell immunotherapy targeting EBV antigens as a potential treatment for multiple sclerosis; and multiple next-generation chimeric antigen receptor T-cell (CAR T) immunotherapies for both solid tumors and hematologic malignancies. Improving patients lives is our mission and we will never stop working to bring transformative therapies to those in need. Atara is headquartered in South San Francisco and our leading-edge research, development and manufacturing facility is based in Thousand Oaks, California. For additional information about the company, please visit atarabio.com and follow us on Twitter and LinkedIn.

Forward-Looking Statements

This press release contains or may imply "forward-looking statements" within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934. Because such statements deal with future events and are based on Atara Biotherapeutics' current expectations, they are subject to various risks and uncertainties and actual results, performance or achievements of Atara Biotherapeutics could differ materially from those described in or implied by the statements in this press release. These forward-looking statements are subject to risks and uncertainties, including those discussed in Atara Biotherapeutics' filings with the Securities and Exchange Commission (SEC), including in the Risk Factors and Managements Discussion and Analysis of Financial Condition and Results of Operations sections of the Companys most recently filed periodic reports on Form 10-K and Form 10-Q and subsequent filings and in the documents incorporated by reference therein. Except as otherwise required by law, Atara Biotherapeutics disclaims any intention or obligation to update or revise any forward-looking statements, which speak only as of the date hereof, whether as a result of new information, future events or circumstances or otherwise.

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Atara Biotherapeutics Announces Appointment of Cell & Gene Therapy Expert Maria Grazia Roncarolo, MD to Board of Directors - Business Wire

DUBLIN Dyno Therapeutics Inc., an early stage gene therapy firm applying artificial intelligence to advanced capsid engineering, has entered partnerships with Novartis AG and Sarepta Therapeutics Inc., in ophthalmic indications and muscle diseases, respectively, which have more than $2 billion in biobucks attached. Further financial details are scant, although it could receive more than $40 million in up-front, option and license payments during the research phase of the Sarepta alliance. The two deals are evidence of the market appetite for improved adeno-associated viral vectors. We had a significant amount of interest even before the company was formed, Dyno CEO and co-founder Eric Kelsic told BioWorld.

The phase III pivotal trial of mavacamten, an oral, allosteric cardiac myosin modulator for treating symptomatic, obstructive hypertrophic cardiomyopathy, from Myokardia Inc., of Brisbane, Calif., hit its primary and all secondary endpoints. The data show mavacamten was well-tolerated and demonstrated safety results comparable to placebo. Ninety-eight percent of patients enrolled completed the study. The company stock (NASDAQ:MYOK) was met by enthusiasm midday Monday, as shares swelled by 62%. Myokardia said it plans to submit an NDA in the first quarter of 2021. Myokardia is developing mavacamten to treat conditions in which excessive cardiac contractility and impaired diastolic filling of the heart are the underlying cause.

After being hit with the major market meltdown during March, public biopharmaceutical companies developing new medicines put that behind them with a dramatic surge in valuations in April. As a result, the BioWorld Drug Developers index recorded an almost 20% increase during the period, with that momentum continuing into early May.

By delivering the protein follistatin via gene therapy, researchers at Washington University in St. Louis were able to increase skeletal muscle mass, decrease inflammation and reverse obesity-related arthritis in mice who developed osteoarthritis as a result of a high-fat diet. They reported their results in the May 8, 2020, online issue of Science Advances.

HONG KONG The Japanese government is tightening its grip on its listed companies, including those working on promising COVID-19 treatments. On May 8, the Japanese Ministry of Finance releaseda list of 518 companies that would be subject to stricter restrictions on receiving foreign investments. Starting on June 7, foreign investors buying a stake of 1% or more in Japanese firms will be pre-screened, compared with the previous limit of 10%. In a statement, the ministry said the move was related to the degree of impact of the investment on maintaining the basis of production and technologies in the business sectors that relate to protection of national security, maintenance of public order, or safeguard of public safety.

DUBLIN Tolerogenixx GmbH is on track to move its cell-based immune tolerance induction therapy for kidney transplant recipients into a 200-patient phase IIb trial, following the publication of promising data from a phase Ib trial in 10 patients, in which all participants had a successful transplant at one-year follow-up, including those in a high-dose group on a reduced immunosuppression regimen.

Having a COVID-19 therapy approved through an emergency use authorization (EUA) is not the same as having access to it, even if its free. With one-third of the COVID-19 cases confirmed globally as of today and 28.5% of the deaths, the U.S. is getting 40% of the 1.5 million doses of remdesivir Gilead Sciences Inc. is donating worldwide. The federal government last week began doling out the 607,000 doses to the states, which are then charged with distributing them to the hospitals with the greatest need. But given the supply and manufacturing timeline for the first drug granted an EUA to treat severe COVID-19 cases, more than 300,000 eligible U.S. patients likely will not have access to the drug through the end of July, said Brian Abrahams, a senior analyst with RBC Capital Markets LLC.

HONG KONG South Koreas Hanmi Pharmaceutical Co. Ltd. has filed a new drug approval application for Rolontis (eflapegrastim) with the countrys Ministry of Food and Drug Safety. Rolontis, a biologic to treat neutropenia, is the first of its kind in South Korea and, according to Hanmi, the first to be developed using the companys Lapscovery platform.

About 15 months after closing its multibillion-dollar acquisition of Loxo Oncology Inc., Eli Lilly and Co. has secured an accelerated FDA approval for the first of the deal's headline assets, the RET kinase inhibitor selpercatinib, now branded as Retevmo. The green light, following a priority review, allows for marketing of the drug as a treatment for three types of tumors non-small-cell lung cancer, medullary thyroid cancer and other types of thyroid cancers in patients whose tumors have a rearranged during transfection alteration. The decision arrived well ahead of an earlier-projected third-quarter decision by the agency.

Three biopharma drugs are up for FDA approval this week, including one new chemical entity, dasotraline, from Sunovion Pharmaceuticals Inc. to treat binge eating disorder, and two other candidates that are part of supplemental filings for expanded oncology indications. Sunovions drug and Blueprint Medicines Corp.s Ayvakit (avapritinib) to treat fourth-line gastrointestinal stromal tumors (GIST), have PDUFA dates set for May 14, while Clovis Oncology Inc.s Rubraca (rucaparib) for prostate cancer is scheduled for May 15.

The articles from BioWorlds ongoing coverage of the COVID-19 coronavirus outbreak are available at http://www.bioworld.com/coronavirus. Note that we have added three critical tables which are constantly updated:

4D Pharma, 9 Meters, Abbvie, ADC, Amag, Appili, Artara, Astrazeneca, Bellerophon, Biomarin, Bluebird, Bridgebio, Burning Rock, Cellect, Cocrystal, Crispr, Daicchi, Dar, Faron, Histogen, Hummingbird, Infinity, Inmune, Intercept, Jazz, Kiniksa, Kura, Lilly, Merck, Mimedx, Myokardia, Navidea, Noxxon, Orexo, Oyster Point, Pliant, Plus, Portage, Protagonist, Protalix, Regeneron, Relief, Ritter, Strongbridge, Themis, Tiburio, Tolerogenixx, Tracon, Vertex

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Novartis, Sarepta join Dyno's enterprise to boldly go to new gene therapy frontier - BioWorld Online

The Cell and Gene Therapy Catapult (CGT Catapult) and Kyoto, Japan-based CiRA Foundation are launching a new collaborative research project focused on induced pluripotent stem (iPS) cell characterisation.

With the move, the groups are hoping to further the application of iPS cell technologies for the manufacture of regenerative medicine products.

The potential of distinct iPS cell lines for differentiation into specific cell types is usually biased towards some cell line-specificity which, the parties note, is very difficult to predict. As such, in order to select an appropriate iPS cell line for clinical trials it is necessary to differentiate several candidate cell lines, which is time-consuming.

CGT Catapult and CiRA plan to explore novel methods of evaluating cell differentiation and aim to establish reliable tests to predict the potential of iPS cell to differentiation bias, a capability that would help to advance the use of iPS cells for regenerative medicine products.

We are honoured to collaborate with CiRA Foundation, an organisation with world-leading capabilities in iPS cell technology, and to be the first group to utilise CiRAs innovative iPS cell lines outside of Japan, said CGT's chief executive Matthew Durdy

This is a truly exciting project to help further the application and manufacture of iPS cells into cell therapies. We look forward to progressing this promising research together, which has potential benefits for the global advanced therapies industry.

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Cell and Gene Therapy Catapult links with Japan's CiRA Foundation - PharmaTimes

For its first quarter ended March 31, 2020, the Maryland-based company, reported revenue of $1.9 million, compared to $0.4 million a year earlier

Inc (), a global biotech company focused on accelerating and transforming the delivery of cell and gene therapies, posted first-quarter results on Monday that saw its revenue soar 348% year-over-year driven by its Cell and Gene Therapy (CGT) Biotech platform.

For its first quarter ended March 31, 2020, the Germantown, Maryland-based company, reported revenue of $1.9 million, compared to $0.4 million in the first quarter of 2019.

Orgenesis achieved net income of $75.6 million, or $4.23 per share, reflecting the sale of subsidiary Masthercell Global Inc, a contract development manufacturing organization (CDMO).

READ:Orgenesis boss Vered Caplan makes top 20 list of inspirational leaders in advanced medicine

On February 11, Orgenesis completed the successful sale of its CDMO business to Somerset, New Jersey-based Catalent Pharma Solutions, for around $127 million.

As a result, Orgenesis reported cash and equivalents of $107.1 million as of March 31, 2020.

In a statement accompanying the numbers, Orgenesis CEO Vered Caplan said: Step by step, our CGT Biotech Platform is gaining traction within the market, as illustrated by the year-over-year growth.

In the first quarter of 2020, revenue increased to $1.9 million, or nearly an $8 million revenue run rate compared to $3.1 million for all of 2019. We believe our CGT Biotech Platform is poised for growth this year through industry partnerships that are currently underway with leading research institutes and hospitals around the world, she added.

The companys CGT Biotech platform consists of three core elements:point-of care Therapeutics, point-of care Technologies, and point-of care Network.

Caplan also noted that earlier this year, the company struck collaboration agreements with two leading healthcare research institutes in the US.

We plan to utilize our point-of-care Network to support their growing development and processing needs in order to advance and accelerate cell and gene-based clinical therapeutic research, said Caplan.

Orgenesis is using the Masthercell sale proceeds to expand the companys point-of-care cell therapy business. The biotech is currently focused on therapies which span a wide range of treatments.

In addition to our POCare Network, we are building our pipeline of POCare Therapeutics and Technologies, with an ultimate goal of providing life-changing treatments to large numbers of patients at reduced costs within the point-of-care setting, said Caplan.

Specifically, we are focusing on immune-oncology, metabolic and autoimmune diseases, as well as anti-viral therapies.

Orgenesis also recently completed the acquisition of Tamir Biotechnology and its broad-spectrum antiviral platform, ranpirnase in a cash and stock deal for roughly $21 million. The company will use ranpirnase to target human papillomavirus (HPV), which causes genital warts.

Ranpirnase has demonstrated clinical efficacy against HPV and other hard to target viruses based on its unique mechanism of action of killing the virus and modulating the immune system, said Caplan.

Going forward, Orgenesis plans to move the program through a Phase 2b trial in the US.

Meanwhile, the Orgenesis boss said the company has received a nod from regulators to keep research alive at its labs during the coronavirus (COVID-19) pandemic.

We are leveraging all our knowledge and expertise in the field of cell and gene therapy, including anti-viral technologies, in an attempt to find potential COVID-19 cures and therapies, said Caplan.

Importantly, we have a strong balance sheet and are strategically positioned to bring a variety of therapies to market in a cost-effective, high-quality and scalable manner.

At the start of April, Orgenesis teamed up with regenerative medicine and cell therapy firm RevaTis on a new joint venture to produce certain stem cells. The two firms plan to leverage Orgenesiss autologous CGT Biotech platform to advance clinical trials.

Under the deal, RevaTis and Orgenesis will use the formers patented technique to obtain muscle-derived mesenchymal stem cells (mdMSC) as a source of exosomes and various other cellular products.

Our plan is to combine RevaTis patented technique to obtain mdMSCs through a minimally invasive muscle micro-biopsy with our own automated/closed-systems, 3D printing, and bioreactor technologies, said Caplan.

The goal of this JV is to lower the costs and accelerate the timeline of bringing these innovative therapies through the clinic and into commercialization.

Contact the author Uttara Choudhury at [emailprotected]

Follow her on Twitter: @UttaraProactive

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Orgenesis sees 1Q revenue rocket driven by its Cell and Gene Therapy Biotech platform - Proactive Investors USA & Canada

Amber Freeds singular mission is to help scientists develop a treatment for her son. But the pandemic has put the success of that mission into question.

Freed is a mother to twins Riley and Maxwell, who just turned 3 this year. Riley is a healthy toddler. But Maxwell has a rare genetic disease that has led to delays in his development. Hes nonverbal, and he has a movement disorder. The condition responsible for these symptoms is called SLC6A1, after the gene thats affected, and Maxwell was only the 34th person in the world to get this diagnosis. if left untreated, the disease could soon cause debilitating epilepsy.

When doctors broke the news of the diagnosis to Freed, they told her there was no treatment or cure for her son, and that her family would have to prepare to live with Maxwells condition. But Amber didnt accept that.

Every instinct in my body said, Youll have your whole lifetime to cry for yourself, Freed said. At this exact moment, you put your feelings and sadness aside. This isnt about you. This is about Maxwell. And you fight like the third monkey on the loading deck to Noahs Ark, and its starting to rain.

This is how Freeds quest began. She taught herself microbiology, so she could understand her sons condition. She lobbied scientists to take up the research, which wasnt on their radar given how rare the disease is. She convinced scientists in China to make genetically engineered mice that could be sent to the US and act as a model of Maxwells disease. And she fundraised a necessary step given the high cost of the gene therapy research.

All told, shes raised more than $1 million. What were doing will be the building blocks of all gene therapies to come after us, Freed told Arielle Duhaime-Ross on an episode of Reset in January.

Even though she was racing against time, she was optimistic that Maxwell would be treated this year, as experiments on the genetically modified mice were about to begin.

Then the pandemic hit.

Covid-19 has brought most non-coronavirus medical research to a halt, and closed labs.

I never anticipated or thought something like this could happen, Freed said, but also it was a state of devastation like I felt when Maxwell was originally diagnosed that lightning hit us. The unthinkable happened. And here, lightning has hit again. A black swan event for the world that no one could have anticipated.

Freed came back to Reset to talk about the impact of the pandemic on Maxwell and her family as well as rare disease research as a whole. Listen to the full episode below.

The episode also features a conversation with BuzzFeed science reporter Dan Vergano, who first wrote about Ambers story and who talks about how the closure has impacted thousands of other kids with rare diseases, as well as clinical trials for other types of medical research.

Listen and read more:

Listen to the original Reset story from January

Dan Verganos reporting on this story for BuzzFeed

Back in January, Dans piece on how the mice were being used to develop a treatment

Milestones for Maxwell, Amber Freeds website

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A treatment for this toddlers rare genetic condition was in sight. Then the pandemic hit. - Vox.com