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Best DNA testing kits in 2020: 23andMe, AncestryDNA and more compared – CNET

With a simple sample of your saliva or swab of your cheek, a DNA testing kit can be used to research familial origin or ancestry and determine paternity. Formerly a niche pursuit, home DNA testing is now an easy way to map out your family tree.

The kits have become quite affordable over the past few years, too, with a wide range of DNA testing companies -- from trailblazers such asAncestryand23andMeto upstarts such as LivingDNA-- selling testing kits.

You can learn a lot from DNA testing. In addition to deepening your understanding of ancestry, some services will introduce you to living relatives around the world, through a common ancestor, or use markers to shed light on your predisposition to specific health issues and diseases. Others will even test dog DNA and give you insight into your dog's health and breed makeup. Here we present to you our roundup of the nine best DNA test kits and services -- what they offer, how they work and how much they cost.

We'll update this story as we continue our in-depth testing of these services. In the meantime, the ones included here are the most popular DNA testing services as determined by Google keyword search rankings.

Looking for more in-depth info on DNA testing services in general? Jump to our explainer.

Named for the 23 chromosomes found in human cells, 23andMe offers a battery of tests, including some that analyze health risks like Type 2 diabetes and Alzheimer's disease. (It was these tests thatattracted attention from the FDA.)

23andMe earns points for the depth of its medical tests, as well as the size of its match database. Purchasers of this DNA kit should note that the basic DNA test is $99 but that medical results cost another $99.

The added expense may be worth the money; the additional information includes genetic health risk information, wellness reports, trait reports, and carrier status reports, which indicate whether a particular DNA profile may be a genetic carrier of a disease or disability.

Your DNA information is gathered using a saliva sample from Autosomal DNA testing, which, once analyzed, is stored forever on 23andMe's servers. The service also provides for a chromosome browser and comparison, as long as any possible matches approve your access. The service's matrilineal and patrilineal line testing can geolocate your ancestry DNA in more than 1,000 regions.

(Appropriate for a genomics company, 23andMe's executive ranks contain some interesting familial relationships: CEO and co-founder Anne Wojcicki is the former wife of Google co-founder Sergey Brin and sister of YouTube CEO Susan Wojcicki.)

Ancestry DNA has a vibrant genealogical community and offers a wide range of databases, research resources and family matching features. The Ancestry DNA test provides analysis segments of your DNA results and traces its origins to 500 geographic regions throughout Europe, Africa and Asia -- the most detailed of any of the services we've profiled. AncestryDNA also says that it can help you learn about up to 26 traits and attributes you've inherited from your ancestors -- all from a little bit of saliva.

Ancestry maintains a free family tree search tool, and you can add your specific results to that database. You can also download your full DNA profile and import that data into another tool -- but Ancestry doesn't offer a chromosome browser, so you can't do DNA segment comparisons. Ancestry DNA stores results in its DNA database forever.

FamilyTreeDNA is operated by Houston-based genetic testing lab Gene-by-Gene. Gene-by-Gene also operates the Genomics Research Center for National Geographics' Genographic Project, which has concluded its public participation phase.

FamilyTreeDNA offers a wide range of tests. The basic autosomal test costs $79 (plus shipping) and is conducted with a swab test sample of your cheek cells. You can add sequences and markers, and your father's line and mother's line tests, but that will step up the price considerably.

If you're interested in doing in-depth analysis, the FamilyTreeDNA offers a chromosome browser, allows raw data to be uploaded, provides support for setting different segment matching thresholds and allows up to five comparisons to be done at once. FamilyTreeDNA allows trial transfers from 23andMe and Ancestry DNA into its DNA match database; additional transfers of various datasets are available for a fee. FamilyTreeDNA promises to keep data for 25 years.

Offering DNA test kits and a range of online subscription services, MyHeritage says that its database includes more ethnicities -- that's 42 -- than any other major testing service. The free 14-day trial will let you poke around the company's massive online DNA database which includes 3.5 billion profiles in addition to information about over 100 million subscribers and their collective 46 million family trees.

Starting at $79, the company's DNA testing kits are competitively priced and cover the basics: A simple cheek swab will give you an analysis of your ethnic origins and the identification of relatives who share your DNA. In addition to MyHeritage's free basic subscription, which will let you assemble a family tree up to 250 people, there are other packages that accommodate larger trees, advanced DNA features, and more robust research tools. The company allows you to upload test data from other DNA testing services.

MyHeritage says that it has also sold more than one million DNA testing kits -- but its enormous database is largely powered by Geni.com, a genealogy social mediaaccording to the New York Times site, that has assembled "the world's largest, scientifically vetted family tree," according to the New York Times. (MyHeritage is Geni.com's parent company.)

HomeDNA is kind of like the Walmart of DNA testing, which is somewhat appropriate given that the company's testing kits are sold at Walmart stores in addition to CVS, Rite Aid and Walgreens pharmacies.

HomeDNA offers a range of DNA ancestry testing services priced between $69 to $199. Though the jury is still out about the effectiveness of specialty tests, HomeDNA also sells test kits to determine food and pet sensitivity ($99), diet and exercise strategies based on your genetic makeup ($119), paternity ($164), and even skin care ($99).

Dog owners can buy a dog DNA test to help you determine your dog's breed history for $125. You can also buy a $125 health screening for your dog or cat that includes a series of tests for genetic diseases and traits. (If you're interested in a canine DNA test for less, Wisdom Health offers a dog DNA test kit for under $80.)

Testing is done with a mouth swab. Shipping is free. And results are kept for 25 years.

African Ancestry can't compete on price or the size of its match database, but it does offer deep regional analysis. It's a worthy specialized service for individuals looking at exploring African ancestry.

Rather than a match database of individuals, African Ancestry has the world's largest database of African lineages. The company can trace your ancestry back to a region in Africa and then pinpoint its location today. It can also dive deep into history and help find original ethnic groups that may date back as long as 500 years ago.

But the tests can get quite expensive. The company sells a maternal test kit and a paternal test kit for $299 each (shipping is free). If you want to trace both your maternal and paternal lines back through this DNA database, it'll cost you about $600. Still, for African family histories, the depth of analysis is unique among the services we profiled.

The Full Genomes service is so expensive, it offers a payment plan. But the service offers the largest library of Y-chromosome SNPs around. So if you want to explore your patrilineal background, this is the most comprehensive option on the market. You can also look into your mitochondrial DNA. There's no family match database, however.

Testing is done with a cheek swab. The company charges $25 for shipping.

Living DNA is a UK-based genomics firm that offers autosomal DNA data, as well as a breakdown of matrilineal and patrilineal lines. DNA data is gathered through a mouth swab.

Living DNA has a very limited family match database, so if you're looking for a service that can match you to relatives around the world, this isn't the one for you. But Living DNA's test is quite comprehensive, analyzing multiple types of DNA: it tests 638,000 autosomal SNPs, 22,500 Y chromosome SNPs and 17,800 X chromosome SNPs, along with 4,700 mitochondrial SNPs.

And the service tracks DNA to 80 geographic regions. Those with a UK family history will see a map of where paternal and maternal ancestors lived on the islands. Though we didn't test it first hand, Living DNA says its tools allow you to upload DNA data from other services to predict relationship matches.

Nebula Genomics offers a somewhat different take on DNA testing from the other testing services we've profiled. While you can order a full test kit from the company (and you should check to see if they're running a price promotion before ordering), you can also upload an existing DNA sequence from Ancestry or 23andMe's DNA database and get Nebula's reports at a reduced price.

The company claims a very different approach to DNA testing. Where most DNA testing firms examine a subset of the DNA sequence, Nebula says it examines the whole DNA sequence. They tell us they test, "1.3 billion positions and results in one thousand times more data than tests that use microarray-based genotyping.".

While the company does not offer a family-finding match database to connect you with relatives, they do offer a unique art print based on your DNA. We're not entirely sure we'd want to showcase our DNA up on the wall along with our prints of dogs playing poker and velvet Elvis, but Nebula's prints are quite attractive.

Of more note is the depth of the company's scientific reports based on your DNA sequence. The company also tests the microbiome in your mouth, providing a detailed overview of the bacteria contained inside your mouth and what it means for your overall health.

We wouldn't necessarily recommend Nebula's kit as your first stop on your DNA testing journey, particularly if you want to connect with your ancestors and family tree. But if you want to dive deeper even than 23andMe into the medical aspects of both your DNA and your personal mouth biome, Nebula is definitely a fascinating option to explore.

If you're using a home DNA testing service, you're likely looking for one of three things:

Ancestry and family history:The first big draw of a full DNA test is that you'll get a detailed breakdown on ancestry and ethnicity, and the migration patterns of your common ancestors. Spoiler alert: Your ethnic background may be radically different than you think it is. You'll also find out what a haplogroup is.

Relative identification:With your permission, some DNA services will let you connect with relatives you never knew you had -- other folks with matching DNA who have used the service and likewise given their permission to connect to possible relations.

Health and disease info:DNA testing can also indicate which conditions for what you may have a preponderance. It's a controversial feature, to be sure. Knowing that you have a genetic predisposition to a certain form of cancer may make you more vigilant for testing, but it may also lead to increased stress -- worrying about a potential health condition that may never develop, even if you're "genetically susceptible" to it. The possibility of false positives and false negatives abound -- any such information should be discussed with your doctor before you act upon it.

Afraid of needles and drawing blood? That's not an issue with these kits, which all involve either a swab test or a little bit of spit. All you need to do is spit into a vial or rub a swab in your mouth -- all the genetic data needed for these tests is present in your saliva -- and ship the DNA sample to the company for analysis.

The reason that a saliva sample works as well as blood (or hair follicles or skin samples) is that your DNA -- which is short for deoxyribonucleic acid -- is present in all of them. It's the basic genetic code present in all of your cells that makes up your key attributes, from the color of your eyes to the shape of your ears to how susceptible you are to cholesterol.

The key terms you need to know when comparing DNA testing services are:

SNP (single nucleotide polymorphism):Genotyping is done by measuring genetic variation. One of the more common is SNP genotyping, which measures the variations of a single nucleotide polymorphism. In our service summaries below, we discuss the number of SNPs. That's because the more a company measures, the more granular the variations analyzed.

Autosomal DNA testing:An autosomal test can be administered to both men and women and traces lineage back through both the maternal and paternal bloodlines.

Y-DNA:The Y-DNA test can only be administered to men and traces DNA back through the patrilineal ancestry (basically from father to grandfather to great grandfather).

mtDNA:The mtDNA is matrilineal and lets you trace your ancestry back through your mother, grandmother and great grandmother.

Autosomal tests can get you quality genetic information going back about four or five generations. Because the Y-DNA and mtDNA tests are more focused on one side of the line, you can get information going back farther, but with fewer data about family structure.

Before you use any of the services we've highlighted below, keep these important factors in mind.

Match database size:If you're looking for living relatives, this is important. Simply put, the bigger the pool of available data, the better the chance you'll have of finding a match.

Privacy concerns:Nothing is more private than your health data, which is why you should make sure a prospective DNA testing site follows the same best-practice online security protocols you'd expect from your bank or email provider. You'll want to look for two-factor authentication, an encrypted password database and so on.

But for DNA testing providers, you should also investigate how they're sharing your genetic data -- even if anonymously -- and how long they keep the data. It's not just academic: Authoritiesrecently identified a suspect in the Golden State Killer murdersthanks to an open-source DNA and genealogy service known as GEDmatch (not profiled here).

If you're creeped out by how much information Facebook,Googleand Amazon have on you based on your online browsing habits, just remember that these DNA testing services are getting what is effectively your medical history. Make sure of their policies before turning over that valuable data. Also, even if you don't share your DNA with a service, your familial DNA data may be available if a relative shared their genetic material. The privacy issues can get very complex.

Don't expect perfect accuracy.Testing kits can give you indications, but taking a DNA test with one of these testing services won't magically produce a history book of your family's background.

Consult a doctor on any health data:Cancer. Leukemia. Heart disease. Alzheimer's disease. There are a lot of scary afflictions out there, and your DNA testing may well indicate which ones to which you are genetically predispositioned. But the data markers from DNA testing kits exist in isolation. You should consult your doctor to explore the data from any of these tests. They'll help you determine how to implement any lifestyle changes or followup testing as a result, if it's worth doing so.

CNET's Justin Jaffe contributed to this story.

Updated periodically with new information.

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

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Dallas football player overcomes OCD, anxiety and drug withdrawals to earn scholarship at SMU – WFAA.com

DALLAS "I'm sitting there, laying on the bathroom floor, thinking I'm about to die."

Gerrit Choate recounted every detail of a cold winter day in Salt Lake City, Utah.

The worst day of his life.

"The doctor said it was similar to a heroin addict going through a complete cold turkey detox," Choate said.

Gerrit experienced withdrawals for a drug Luvox, the brand name of fluvoxamine prescribed to treat obsessive-compulsive disorder (OCD).

"Come to find out from the doctors that the recommended usage is no longer than 30 days, Choate said.

Gerrit's eye widened for emphasis.

"I had been on it for about nine years."

Choate is the son of Southern Methodist University football legend Putt Choate, who still holds the school record for tackles in a season and tackles in a career.

"I grew up going to SMU games," Gerrit said. "It was always a dream to play for SMU."

Dreams turned to nightmares.

In sixth grade, Gerrit broke a bone in his lower back while playing football.

"Several doctors said I was never going to play any sport again," Gerrit said.

Devastating news for a multi-sport athlete.

"You tell that kid he can't, and he's gonna do it," declared his mom Fifi Choate.

After a year in a back brace, Choate consulted with another doctor who told him there's a slight chance he could play again.

Choate adhered to a strict rehabilitation plan and was eventually cleared to play.

The best day of his life.

The Dallas native became an All-State player at Parish Episcopal High School under head coach Scott Nady.

However, the helmet and broad shoulders masked the mental struggles Choate endured for years.

As a child, he was diagnosed with severe anxiety and OCD.

"Suddenly, he became a very anxious little boy," Fifi said. "Always needing reinforcement."

"The best way to describe it is just an overwhelming feeling that if you do don't this task, whatever it is, something bad will happen," Choate said.

The definition of OCD, according to the National Institute of Mental Health, is: a common, chronic, and long-lasting disorder in which a person has uncontrollable, reoccurring thoughts (obsessions) and/or behaviors (compulsions) that he or she feels the urge to repeat over and over.

"It was kind of difficult for me to understand," Choate's father Putt admitted.

As symptoms worsened, Chaote was prescribed Luvox.

With no Division 1 offers out of high school, Chaote walked on at Utah.

However, early into his college career, his mind and body started to shut down.

Doctors recommended Chaote take an expensive drug-gene test.

"Our insurance wouldn't even pay for it," Putt said.

According to the drug-gene test, Luvox was toxic for Choate's genetic makeup.

He had to wean off the drug as soon as possible, which would be no easy task since his body relied on it for nearly a decade.

"'I should've known as a parent.' That's what you beat yourself up the most about," Putt said. "You didn't check. You didn't look. You weren't aware. You didn't protect your child."

Fifi moved out to Utah for a couple of months to help Choate come off the medication. He would take gradually smaller doses until he was off of it completely.

One morning, Choate took his daily dose, but he had an upset stomach and vomited.

His body never digested that day's dose.

The worst day of his life.

"It was the most frightening thing I've been through," Fifi said. "He was so violently ill."

"It was just a nightmare," Choate said.

With his mother watching helplessly, Choate vomited 24 times in 12 hours in his Utah dorm.

"It was a complete shock how an anxiety medication could do this to somebody," Choate said. "Because one day off of this stuff has me going through hell."

Over time, Choate weaned off the drug and moved home to Dallas.

He stuck to a strict nutrition plan thanks to his nutritionist Jill Lane and walked on at SMU.

"Most people would've quit," Putt said. "And nobody would've blamed him."

"I just marvel at his determination," Fifi said proudly. "When life keeps knocking him down, there he goes. He's not going to accept it."

Now, Choate sees the benefit to sharing his experience with others.

"He doesn't want another child to go through the hell he had to go through," Fifi explained. "If your child has these symptoms, if they have anxiety, get them tested. Get their genetic testing done and see what their bodies can tolerate."

"It's not your typical comeback story," Choate said. "It took me a while to see it could help people and it would be selfish not to share it."

Gerrit is now a junior at SMU, where he plays linebacker and special teams.

Prior to his junior season, Choate was promoted from walk-on to scholarship athlete at his father's alma mater.

"He's a warrior," Fifi said, wiping tears from her eyes. "He takes a beating and gets back up."

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Task force to be set up to prevent genetic diseases: minister – The News International

Task force to be set up to prevent genetic diseases: minister

LAHORE : Punjab Minister for Health Prof Yasmin Rashid has said that only two per cent of children are born healthy and 98 per cent of children fall prey to one genetic disease or the other.

Considering it a highly important and sensitive subject, Punjab government decided to establish task force for the prevention of genetic diseases among children in the province, the minister announced this in a press conference held in connection with World Rare Disease Day 2020 here on Saturday. The day is observed every year all over the world on the last day of February (this year on February 29). The ceremony was arranged jointly by the Department of Pediatric Gastroenterology, Hepatology Children Hospital Lahore and an international partner institute from Germany.

Children Hospital Dean Prof Masood Sadiq, paediatric department head Prof Dr Huma Arshad Cheema and three-member team from Germany including Prof Peter Bauer MD and Dr Susan Krake also attended the ceremony.

Several children who had suffered genetic diseases were also brought by their parents from all over the country where the speakers highlighted the impact of the cousin marriage and marriages in same castes.

Prof Yasmin Rashid lauded the efforts of Prof Huma and her team to bring international renowned experts to Pakistan to observe highly important day to sensitise the government functionaries and the public about the burden of the disease on children because of marriages with cousins and in same castes.

Declaring it an alarming situation, she said that the treatment of the genetic disease is highly expensive and the Punjab government was allocating massive funds for the ailing kids.

She especially thanked to the international experts for coming Pakistan in order to educate the local doctors and create awareness among masses about the genetic diseases, saying that the establishment of a dedicated unit for the same purpose in Childrens Hospital Lahore was highly commendable.

She also acknowledged the services of Prof Huma Arshad Cheema for establishing the only department in any public sector institute in Pakistan (at Childrens Hospital Lahore).

Speaking on the occasion, Prof Masood Sadiq appreciated the efforts of the health minister for providing his hospital hefty funds Rs700 million, declaring it a major step to provide treatment to the kids uninterruptedly.

He said that the Childrens Hospital has provided free test and diagnosis facility to more than 2,000 children suffering from genetic diseases during last couple of years.

Prof Huma said that the department of pediatric and gastroenterology and Hepatology of Childrens Hospital Lahore is the only one all over the country for diagnosis and treatment of patients with the lysosomal storage disorders, inborn error of metabolism and all kinds of genetic diseases.

Declaring it a blessing for the kids in Pakistan, she said that these services were made possible in collaboration with a network of world famous scientists, physicians, charitable organisations and research centres. I am sharing with pleasure that we are able to provide treatment to the kids coming from all four provinces of Pakistan with genetic diseases, Prof Huma said.

She said that it is quite unfortunate that there is no private or public sector facility for genetic testing in Pakistan. We have to send blood samples abroad for testing and per person test cost is more than Rs200,000 and for tests of an affected children and his/her parents, we have to bear cost Rs600,000, she said. Despite the fact that the number of affected children was tremendously high in Pakistan, yet the Childrens Hospital was providing test facility absolutely free of cost, she said.

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Moffat County wolves open up a new pack of issues – The Grand Junction Daily Sentinel

A wolf pack in Moffat County has upended one of Colorados most controversial wildlife management debates, prompting voters, legislators and wildlife officials to wonder what course to chart on wolf reintroduction and management.

In mid-February, Colorado Parks and Wildlife confirmed through DNA tests on scat samples taken from Moffat County that a pack of at least four wolves was present in Colorado. The four were siblings, three females and one male.

This is the first time weve documented a group of wolves, a pack of wolves in the state since they were extirpated in the early 1940s, said Eric Odell, wildlife species conservation program manager.

These wolves were confirmed by Parks and Wildlife less than a month after a Colorado petition was certified giving voters the chance to weigh in on wolf reintroduction.

The pack coming into the state was a real reset in the conversation because, of course, before that the wolf reintroduction conversation was centered around the proposed ballot initiative, which has now gained signatures, said Colorado State Sen. Kerry Donovan, who is working on the issue in the Legislature. With the pack coming into the state, and with both sexes represented within the pack, we now have a management issue as well that the state isnt perhaps entirely prepared for.

Representatives of the Colorado Stop the Wolf Coalition have said the presence of this pack and its three females that could reproduce will make reintroduction unnecessary. But wildlife biologists say the presence of Craigs newest neighbors doesnt mean a re-established wolf population in Colorado is a done deal.

Another two wolves were observed by Parks and Wildlife staff, but genetic testing on those two has not been confirmed. More scat has been tested, but the full results have not been released. The genetics of those two animals are critical in understanding whether this pack is the start of a full wolf recovery in Colorado or an anomaly.

Its built into their social system to avoid mating with relatives, so they would not form a mated pair, University of Colorado-Denver Professor Diana Tomback said of the sibling wolves. The perpetuation of this pack is going to depend on what the genetic relationship is of the other two members.

Tomback, a conservation biologist who served on the science committee of the Rocky Mountain Wolf Project, said there are still too many unknowns to determine the future of these wolves. However, having a breeding pack in Colorado may not be enough for the species to recover, she said, without more wolves to provide genetic diversity.

If you actually go by the guidelines used by U.S. Fish and Wildlife to determine whether a population is recovered, it has to populate enough range and be there with enough population size to be able to withstand disturbances and challenges that are natural to their environment, Tomback said. From the perspective of genetic diversity, this one pack is inadequate.

Other wolves make the trek from the northern Rocky Mountains to Colorado, Odell said. Between 2004 and 2019, six gray wolves were photographed or killed in Colorado. More would have to make that journey and find this pack for the population to expand.

One pack is a start to establishing a population, but it does not meet the (U.S. Fish and Wildlife Service) definition of a wolf population two or more packs successfully reproducing for two or more years, Odell said. Genetic diversity is important, and only one pack does not provide that needed diversity.

Wolves have spread this way in the past. Gray wolves were reintroduced to Yellowstone National Park and Idaho in the mid-1990s and expanded their populations in Idaho, Wyoming and Montana. Today, more than 2,000 wolves are estimated in those three states.

In the early 2000s wolves started to pop up in Oregon, which sits across the Snake River from Idaho, said Michelle Dennehy, communications coordinator for the Oregon Department of Fish and Wildlife. One was killed crossing a road. Another was found shot. All were lone wolves, known as dispersers, that had left packs to search for mates.

In 2008, a wolf from Idaho crossed the Snake River and gave birth, starting the first pack in the state.

In 2009, Oregon confirmed a second pack, she said. By 2010, both packs were giving birth to pups.

We didnt do any translocation, Dennehy said. Everything is here naturally or reproduced naturally and weve gone from, if you just look at the numbers, from 10 in 2009 to 137 at the end of 2019.

Oregon and Colorado are not a one-to-one comparison when it comes to wolf migration though, Tomback said. The major obstacle between Idahos estimated 1,000 wolves and northeast Oregon is the Snake River. While it is a difficult river to cross, many wolves have done it. According to the latest Oregon Wolf Management Plan, Radio-collar data shows that dispersing wolves immigrate to and emigrate from Oregon, indicating that Oregon is part of a metapopulation with Idaho and Washington.

THE HARD ROAD TO COLORADO

In Wyoming, wolves in the northwestern portion of the state are managed with some hunting allowed in areas outside Yellowstone National Park. In the rest of the state, wolves are considered a nuisance species and can be killed with no limit, Odell said.

A wolf must make a 120-mile trek from the southern edge of Wyomings Wolf Trophy Game Management Area through high desert hills, sagebrush seas, canyons and across Interstate 80 to get to the Colorado border. During that trip, by Wyoming law, they can be killed without limitation.

It is a challenge and this does seem to be the first time two individuals, a male and a female have made it down and found each other and successfully reproduced, Odell, with Colorado Parks and Wildlife, said. Wolf management in Wyoming, they manage it as a game species in the northwest part of the state and, then outside of that its a varmint species, so there is quite a challenge for animals to cross that landscape.

Because of the difficulty in crossing through southern Wyoming, Odell said this pack is likely a mated pair that produced pups in or near Colorado.

Denny Behrens, Colorado Stop the Wolf Coalition co-chairman and regional director for Big Game Forever, said this natural reproduction and the known instances of wolves dispersing into Colorado in the past make further reintroduction efforts, like the initiative that will appear on the November ballot this year, moot.

Theres no need for introduction in this state, Behrens said. They are naturally dispersing out of the nonessential experimental area up in Wyoming and so its the same thing. Theyre moving into Washington and Oregon and California.

Tomback said she is skeptical that dispersing wolves will make it to Colorado frequently enough to provide the necessary genetic diversity to ensure the continued survival of the population.

If people want to reintroduce wolves into Colorado, its going to take more than waiting for this to happen, Tomback said. The last 25 years have shown that, yes, individual wolves may disperse and make it down, but theyre not able to find another wolf of the right sex and form a pack. So wolf reintroduction, scientifically, the reality is its going to take some help to get that genetic diversity and to get the numbers down where wolves can form packs with each other.

Donovan has proposed a bill to provide for the reintroduction of wolves, but only after a funding source has been identified to pay for wolf management and reimbursement to ranchers who lose livestock to wolf depredation. It also gives five years for the current wolf population to establish naturally before moving forward with reintroduction.

Donovan said she was pursuing the legislation to take a deliberative approach to the question of wolf management and reintroduction, but she said many unknowns still surround the states lone pack.

We will have to see if they settle down in a range, if they reproduce this spring, Donovan said. Right now, we dont know if we have a roaming pack of teenagers or if we have a group thats looking to settle down in Colorado.

While the first pack in Colorado is historic being the first to cross that hostile terrain, find each other and perhaps settle here for good they are only the start of what could mark the first return of a real population of wolves in the state in 80 years, Odell said. Whether through human reintroduction or from wolves dispersing from the north, the formation of more packs will be needed if wolves are going to once again range widely throughout Colorado.

With a ballot initiative coming in November, a bill proposed in the Senate and at least six wolves wandering through northwest Colorado wilderness, Donovan said looking into all the issues wolves represent is now more important than ever.

We have wolves in Colorado and we suspect that delisting could come out of D.C. sooner rather than later, Donovan said. I think it is a perfect time to look at these issues in a very thoughtful way with the folks in the room who are most excited about having wolves in the mountains again and what that means and the people in the room who are most concerned about what it means to have a federal land lease and a wolf pack as your neighbors.

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Reinventing the Blood Test – The Good Men Project

By Nigel Bowen

When John Kelly took part in a traditional rite of passage for the Irish a year-long backpacking trip around Australia he had no idea that a quarter of a century later hed be exporting innovative Australian medical technology to the world.

I visited in my early twenties and loved the quality of life. So I moved here permanently in my late twenties and continued my career in the medical device industry, explains Kelly.

In the following years, the success of his two employers ResMed and Unilife Corporation in developing easy-to-use medical devices inspired Kelly to create a game-changing medical device of his own.

The experiences of his daughter had highlighted how costly and time-consuming the process of blood test diagnosis was for both patient and medical practitioner. Typically, one trained medical professional was required to extract the blood and send it to a laboratory to be analysed by another trained medical professional. Then, the patient was required to make another appointment to learn the results regardless of the outcome.

With this in mind, Kelly resigned from his position as Unilife Corporations Chief Operating Officer in 2008 and launched a start-up called Atomo Diagnostics the following year. His goal was to create a simple and accurate blood test that was as straightforward and immediate as a home pregnancy test.

Making self-testing easy

Six years later Atomo Diagnostics has two rapid test products in market. The innovative design of the device is easy to use compared with competing products and makes it possible to test on-the-spot, allowing for self-testing in the future. With all of the various testing elements integrated within the one device rather than bits in boxes, the error margin is significantly reduced.

While it didnt take particularly long for validation via government grants, private funding, accolades and industry interest, in the early days Kelly was very aware he was taking a huge gamble.

Theres no way of knowing if these things are going to be a waste of time, effort and money until youve at least got a working prototype, notes Kelly. During that initial phase the company was funded with my money along with IDEs, a product development company I partnered with to produce the device. If the assumptions in the business plan, of which there were many, had been wrong I would have lost several hundred thousand dollars and 18 months of my life.

Kelly believes federal government grants and tax breaks played a crucial role in the success of what was to become the AtomoRapid platform. Funding at key stages of the business, from concept to commercialisation, were critical in getting the idea off the ground.

Commercialisation Australia provides funding to businesses when they really need it and if they hadnt provided it to Atomo who knows if wed have been able to develop the concept, prove it in the market and get the private investment we subsequently got, said Kelly.

Kelly took several personal measures to increases his chances of success. Despite having undergraduate qualifications in mechanical engineering and a Masters in systems engineering under his belt, Kelly embarked on an MBA at the University of Sydney in the hope of broadening his skill set and better support his company.

I had the technical background but I felt I needed to round out my executive management skills. The MBA certainly provided that, along with allowing me to expand my network, which turned out to be useful. One of my classmates now sits on the board of Atomo.

Improving the accuracy of testing

Following a huge amount of time and investment, by 2013 Atomo Diagnostics had a rapid blood test ready for testing in the field and had convinced diagnostic industry multinational BBI Solutions to help commercialise it. Seeing the opportunities to positively impact accuracy of HIV testing, the company decided to test first in the South African cities of Johannesburg, Cape Town and Pretoria. According to Kelly, errors with the existing tests result in tens of thousands of people being incorrectly diagnosed as negative for HIV each year. Those people then go without treatment and have a higher risk of unknowingly pass the disease on to others.

We picked Africa because it has the highest disease burdens globally for HIV and malaria. It was not hugely profitable compared with other tests and other developed markets, however HIV and malaria have the biggest impact on global health and deserved our focus, Kelly says.

Up against a number of competing rapid testing products, Kelly says healthcare professionals who used the all-in-one AtomoRapid HIV test found it far more straightforward than the bits in a box tests sold by other companies.

The feedback was fantastic one woman whod been testing people for 15 years used the AtomoRapid platform for one day then told her boss she never wanted to use anything else ever again, says Kelly.

It has made testing simpler and more accessible by combining the lancet [needle], capillary tube [storage unit] and test strip [results] into one device, said Phillip Smith, project leader for mobile services at the Desmond Tutu HIV Foundation in South Africa

Our counsellors found it was simpler to use than traditional tests and had a much clearer test strip, making it easier to identify the outcome. The device is so straightforward were now investigating the feasibility of allowing self-testing.

Collaboration across the globe

Across the other side of the globe in New York, the Atomo products have also been turning heads. At the 2014 Medical Design Excellence Awards the AtomoRapid HIV (1&2) integrated rapid antibody test won Best in Show, with the judges declaring that the product was earth-shaking in its potential significant impact on third-world detection of infectious disease, global public health and individual healthcare.

The award is the latest in a bevy of accolades for the device ranging from the 2012 Engineers Australia Bradfield Award to taking out first place in Anthills SMART 100 Australian Innovations for 2014.

Far from worrying about losing of hundreds of thousands of dollars as he was in the early days, Kelly is now focused on building a company that could end up with a market capitalisation of hundreds of millions of dollars and make a difference for millions of people around the globe.

Weve just launched an AtomoRapid platform that tests for malaria in Africa, weve done a deal with a US company about adapting the platform for use as a home test for blood coagulation, were talking to some pharmaceutical companies about developing custom diagnostics for things such as allergies and kidney function, and weve recently signed a Memorandum of Understanding with a large listed Chinese healthcare company that want the rights to use our technology in their country. And thats just what weve achieved with the current platform, he says, hinting at big future plans for Atomo Diagnostics.

Once we develop it further, particularly if we can incorporate multi-sensor technology, we can start doing things such as genetic testing or testing for a range of different diseases, rather than just one.

If successful, these developments would mean a global game change for patients and the healthcare industry alike.

This post was previously published on australiaunlimited.com and is republished here under a Creative Commons license.

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Scots boy is one of handful diagnosed with rare condition – and the only one still alive – Daily Record

Little Finlay Duthie is one a billion or 7.8billion as he is the only known child in the world to have a condition which could end his life at any time.

Finlay, 11, has hyper-oricemia pulmonary hypertension (HUPRA) a genetic syndrome only discovered in 2010 in three children in Palestine, who have all died.

But Finlays condition is a unique mutation of the condition which means his parents cannot tell how it will progress.

He was only diagnosed a year ago after years of genetic testing.

In the cases of the Palestinian children and the handful subsequently diagnosed around the world with HUPRA, all were dead by four years old.

HUPRA is an extremely rare mitochondrial disease.

Mitochondria are structures in cells which convert food to energy but in HUPRA patients they dont charge properly causing failure in vital organs.

Finlay will be prone to heart disease, diabetes, epilepsy and faces losing his sight and hearing.

His kidneys have already failed and dad Ross, 36, gave him one of his five years ago.

But, like all kidney transplants, the organ will not last for life so mum, Jennifer, 36, has already been tested as a match and is on stand-by. She said: We had hoped to get him to his teenage years without the transplant but his kidneys failed.

He is now on anti-rejection drugs which means his immune system is low so he gets really ill and ends up in hospital with bugs most people would shake off in a couple of days.

Finlay has had 13 operations including ones for biopsies when cancer was feared on two occasions because of the increased risk with anti-rejection drugs.

Although he is 11, he has a mental age of three because one of the other effects of the condition is global developmental delay. He also has autism. Former nurse Jennifer, who runs a toddler sensory group, added: Finlay is normally a happy little guy really cheeky and always has a smile on his face. He has no awareness of how unwell he is.

The couple, from Stirling, have another son Harrison, 15 months, who is a carrier but unaffected.

Jennifer said: Finlay has some of the same symptoms as other HUPRA patients and some different ones. We think he is the only one in the world with this variation.

It is good in a way because his variation has allowed him to live longer. But it is also a double-edged sword because we are dealing with something no one knows about.

We have no idea what the future holds.

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AmorChem invests in a new gene therapy approach to help patients with a devastating orphan disease – Business Wire

MONTREAL--(BUSINESS WIRE)--The venture capital fund AmorChem II is very proud to announce the financing of a new university project focusing on preclinical development of retinal gene augmentation to treat Peroxisome Biogenesis Disorders in the Zellweger spectrum (PBD-ZSD). The funds financing will bring together three major research groups from the Research Institute of the McGill University Hospital Centre (RI-MUHC), the University of Pennsylvania, and the University of Southern California.

This program is focused on developing and testing a gene therapy construct that may ultimately improve the well-being of patients with a disabling disorder by treating retinal degeneration. Retinal degeneration leading to blindness is a major, untreatable feature of PBD-ZSD. In fact, visual improvement is a critical symptomatic target that can substantially improve quality of life of patients. The collaborators propose to test the gene therapy targeting retinal photoreceptor cells in PEX1 animal models to study recovery of peroxisomal function, says Ins Holzbaur, Managing Partner at AmorChem.

It is particularly rewarding for us to finance such a promising and impactful program in an indication where the current standard of care is strictly supportive. This project allows AmorChem to address a major need in this multisystem disorder and enable the improvement of communication, learning, mobility and autonomy of patients with PBD-ZSD. In addition, the strategy of using retinal gene therapy could eventually open the door to using gene augmentation in other organ systems affected in this disorder, adds Elizabeth Douville, Managing Partner at AmorChem.

Three seasoned researchers are contributing a wide breadth of experience and knowledge to this project. The collaboration is led by Dr. Nancy Braverman from the RI-MUHC, internationally recognized for her work in peroxisomal diseases. In addition, the collaboration will benefit from the materials generated by Dr. Jean Bennett at the Center of Advanced Retinal and Ophthalmic Therapeutics at the University of Pennsylvania and the mammalian cell technology expertise of Dr. Joseph G Hacia from the University of Southern California.

"The innovative work by Dr. Braverman has tremendous potential to make a difference in the lives of patients with peroxisomal disorders. The partnership between the RI-MUHC, University of Pennsylvania, University of Southern California and Amorchem is an exemplary demonstration of the synergies required for translating this scientific discovery to tangible benefits for patients," adds Bruce Mazer, MD Executive Director and CSO (Interim), Research Institute of the McGill University Health Centre.

If the initial proof of concept studies are viable, AmorChem will exercise its Option to negotiate an exclusive license to the underlying technology.

About AmorChem

AmorChem (www.amorchem.com) is a leading early stage venture capital fund launched in 2011 in Montreal. The AmorChem team utilizes its deep understanding of fundamental science to uncover its therapeutic potential and focuses its core expertise in translational research to accelerate therapeutic drug discovery and development across a broad spectrum of disease areas. The fund capitalises on both its venture capital expertise and its entrepreneurial experience to spark the creation of start-up companies and help shape them into the next generation of biotech companies. With over $85M under management, AmorChem has financed over 30 university projects and started up several biotechnology companies from the fruits of this innovative research.

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IPO Weekly Recap: Yes, the IPO Market has caught the coronavirus – Nasdaq

The IPO market is feeling the effects of the coronavirus outbreak with a surge in the VIX volatility index weighing on IPO activity. Since 2015, a week in which VIX volatility has surpassed 35 has been followed by a week averaging two US IPO pricings. Our observational trend continues through 2020, as one biotech entered the public market this past week.  Six IPOs and three SPACs submitted initial filings with the SEC.

Passage Bio (PASG), a preclinical biotech developing gene therapies, priced at the high end of the range to raise $216 million at an $840 million market cap. The deal raised 72% more in proceeds than Passage Bio originally filed for. The company is furthering the research from UPenn’s Gene Therapy Program, which is headed by co-founder James Wilson. Bolstered by the recent performances of other large early stage biotech IPOs, Passage Bio finished up 23%, another sign that biotechs are pushing back against the effects of the coronavirus. Chinese medical information platform Zhongchao (ZCMD) began trading on Monday after raising $12 million in an IPO on Friday 2/21. The company was flat after its first day on the Nasdaq and is currently down 2%.

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IPO Weekly Recap: Yes, the IPO Market has caught the coronavirus

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Medical Research and Innovation at Ohio State – Columbus Monthly

From a cancer vaccine to gene insertion for those with Parkinson's, local researchers are breaking through.

Research is big business at Ohio State University, with medical funding currently exceeding a quarter of a billion dollars, according to Peter Mohler, vice dean for research at OSUs College of Medicine. Ohio State gets grants from the National Institutes of Health and other sources such as other government agencies, nonprofit foundations and industry contracts.

Funding for OSUs College of Medicine, alone, now includes some $268.5 million. What follows are some of the latest breakthroughs.

An Anticancer Vaccine

A new anticancer vaccine, called B-Vaxx, is still in the early stages of being tested but initial studies are promising. The first-ever human trial at Ohio State led by researcher Pravin Kaumaya, a professor in the college of medicines department of obstetrics and gynecology, showed that patients with metastatic or recurrent solid tumors that overexpress the HER-2 protein had a stronger immune response than they did to current treatments.

This means that B-Vaxx may be more effective in killing tumor cells in many types of aggressive breast, gastroesophageal, endometrial, ovarian, colorectal and lung cancers. Although more research and clinical trials are needed, the bottom line on this first report is that scientists have concluded that the vaccine induced patient antibodies that showed potent antitumor activity.

Hope for Parkinsons

Dr. Krystof Bankiewicz, a researcher specializing in neurodegenerative disorders, and Dr. Russell Lonser, chair of OSUs department of neurological surgery, have been working with transformational gene therapy to develop cures for Parkinsons and other neurodegenerative diseases.

A one-step solution for Parkinsons could be the insertion of a non-pathogenic virus thats been modified to do only one thing: deliver the missing gene to a specific region of the brain.

The missing gene, if implemented, stops the progression of Parkinsons. Administering it, however, is a complex procedure. An MRI scanner is used to directly implant it in the brain.

Six clinical trials regarding the gene therapy and its effects on neurodegenerative diseasesincluding Parkinsons, Alzheimers, Huntingtons and moreare underway at Ohio State. In fact, the clinical trials for pediatric patients have been so successful that registration of the therapy has been fast-tracked with the U.S. Food and Drug Administration. There is hope that the drug will be approved this year for use in children.

Brain Stimulation

A small 2018 study at Ohio State implanted electrodes into the frontal cortex of Alzheimers patients and programmed a pacemaker to deliver deep brain stimulation. DBS has already proven to be helpful for patients with Parkinsons, epilepsy and obsessive-compulsive disorder. And, it is currently being studied for addiction, chronic pain, multiple sclerosis, traumatic brain injury and more.

Two of three people showed statistical improvement, says Dr. Douglas Scharre, professor of neurology and clinical psychology at OSUs Center for Cognitive and Memory Disorders and its Center for Neuromodulation. One patient was able to plan an outing and handle money, make plans for an event and cook a simple meal. These may seem like minor improvements, but if the patient cant do it, the caregiver has to.

Atrial Fib: The Watchman

Among the 3,000 clinical trials at various stages at Ohio State in recent years has been apilot studylead by Dr. Ahmet Kilic, former OSU associate professor of cardiac surgery, on the efficacy of the Watchman, a tiny parachute-like device which is implanted into the heart to regulate the heartbeat of those who suffer from atrial fibrillation. (Kilic is now director of heart transplantation and mechanical circulatory support at Johns Hopkins Medicine.)

Along with reducing stroke risk, the Watchman allows for remote monitoring of heart function. Watchman patients also forgo the risk of excessive bleeding caused by long-term use of warfarin, such as Coumadin and other blood thinners. The implantnow in more than 100,000 peoplecan eliminate regular blood tests and food-and-drink restrictions that come with warfarin.

Expecting a Daughter?

Researchers at the Wexner Medical Center have found thatthat immune cell samples of women carrying girls produced more proteins called pro-inflammatory cytokines than those carrying boys, resulting in exacerbation of conditions such as asthma, and contributing to fatigue and achiness.

Too many of these cytokinescan really be unhelpful for our bodies functioning, explains Amanda Mitchell, lead author of the study while she was a postdoctoral researcher in the universitys Institute for Behavioral Medicine Research. Women carrying girls exhibited greater inflammatory responses when faced with some sort of immune challenge compared to women carrying boys.

Exercising and doing relaxing activities, such as meditation, are recommended. Also, eating healthy foods, including leafy greens, will better support healthy immune responses. Mitchell is now an assistant professor at the University of Louisvilles department of counseling and human development.

More Sleep EqualsHappier Marriages

According to the Centers for Disease Control and Prevention, 35 percent of Americans get less than seven hours of sleep per night, resulting in increased risk of stress-related inflammation and ensuing chronic illnesses such as cardiovascular disease, diabetes, arthritis and others.

In arecent studyat Ohio States Institute for Behavioral Medicine, married couples were asked to supply blood samples and information regarding hours they slept the previous two nights. They were then asked to resolve a conflict, with blood samples taken after the discussion. Although people who had slept less initially had no more inflammation than usual, there was a greater inflammatory response after the conflict. Furthermore, if both partners got less than seven hours of sleep the previous two nights, they were more likely to become hostile.

Couples using unhealthy resolution tactics had an even greater inflammatory response. In a marriage, sleep patterns often track together, explains Janice Kiecolt-Glaser, the senior author of the study and director of OSUs Institute for Behavioral Medicine Research. If one person is restless, or has chronic problems, that can impact the others sleep. If these problems persist over time, you can get this nasty reverberation within the couple.

Less Stress, Better Health

Dining on a Greek salad may be great, but if youre stressed, it may be no better for you than fish and chips, according to an Ohio State study published inMolecular Psychiatry. In the study, 58 women were given two different types of meals, one high in saturated fat, which has been linked to cardiovascular disease, and another with more heart-healthy, plant-based oil. The meals were similar in terms of calories and grams of fat. While inflammatory responses were predictably lower if the women were not stressed after the healthier meal, if a woman was stressed, it looked like she was eating the saturated fat meal in terms of her [inflammatory] responses, study author Kiecolt-Glaser told National Public Radio.

Even though the stressors were for everyday issues, such as dealing with a sick parent, the stress seemed to boost inflammation, increasing chances for disease and slowing the healing process. Still, more research needs to be done and there are plenty of ways to combat stress, includingdeep-breathing.

Immune Cells and Sex

An Ohio State study done on rats and reported in theJournal of Neurosciencefound that immune mast cells,usually ignored by neuroscientists, appear to play an important role in determining the gender of an animals sexual behavior.

When researchers, led by Kathryn Lenz, assistant professor of behavioral neuroscience, silenced the mast cells in male fetal rats, they found that the adult males were far less interested in having sex with females. In fact, they acted almost like females, according the study.

Newborn female rats whose mast cells were activated with a stimulating chemical did the opposite, showing more traditionally males behaviors. Lenz theorizes that if human development mirrors what was seen in this study, even relatively minor influencessuch as an allergic reaction, injury or inflammation during pregnancycould possibly steer sexual behavior and development.

On the Move: Its All Good

According to Bernadette Melnyk, chief wellness officer and dean of OSUs College of Nursing, researchers at the American College of Sports Medicine have confirmed that physical activity completed in any duration is associated with health benefits and count towards your recommended 150 minutes of weekly activity.

Traditionally, physical activity recommendations have focused on accumulating moderate-to-vigorous physical activity either in a continuous manner, such as going for a 30-minute run, or in short bouts performed throughout the day, according to theACSM. However, in 2018, thanks to the advent of digital and other activity trackers, the ACSM also recognized that most daily activity is sporadic and is typically performed in bouts that are less than 10 minutes in duration. Any such activity is now associated with favorable health-related outcomes.

Take time each day to get moving, even if only for five minutes, adds Melnyk.

Reprinted fromColumbus Monthly Health 2020.

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Biogen and Sangamo Announce Global Collaboration to Develop Gene Regulation Therapies for Alzheimer’s, Parkinson’s, Neuromuscular, and Other…

CAMBRIDGE, Mass. & BRISBANE, Calif.--(BUSINESS WIRE)--Biogen Inc. (Nasdaq: BIIB) and Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicine company, today announced that they have executed a global licensing collaboration agreement to develop and commercialize ST-501 for tauopathies including Alzheimers disease, ST-502 for synucleinopathies including Parkinsons disease, a third undisclosed neuromuscular disease target, and up to nine additional undisclosed neurological disease targets. The companies will leverage Sangamos proprietary zinc finger protein (ZFP) technology delivered via adeno-associated virus (AAV) to modulate the expression of key genes involved in neurological diseases.

As a pioneer in neuroscience, Biogen will collaborate with Sangamo on a new gene regulation therapy approach, working at the DNA level, with the potential to treat challenging neurological diseases of global significance. We aim to develop and advance these programs forward to investigational new drug applications, said Alfred Sandrock Jr., M.D., Ph.D., Executive Vice President, Research and Development at Biogen.

There are currently no approved disease modifying treatments for patients with many devastating neurodegenerative diseases such as Alzheimers and Parkinsons, creating an urgency for the development of medicines that will not just address symptoms like the current standards of care, but slow or stop the progression of disease, said Sandy Macrae, CEO of Sangamo. We believe that the promise of genomic medicine in neuroscience is to provide a one-time treatment for patients to alter their disease natural history by addressing the underlying cause at the genomic level.

Sangamos genome regulation technology, zinc finger protein transcription factors (ZFP-TFs), is currently delivered with AAVs and functions at the DNA level to selectively repress or activate the expression of specific genes to achieve a desired therapeutic effect. Highly specific, potent, and tunable repression of tau and alpha synuclein has been demonstrated in preclinical studies using AAV vectors to deliver tau-targeted (ST-501) and alpha synuclein-targeted (ST-502) ZFP-TFs.

The combination of Sangamos proprietary zinc finger technology, Biogens unmatched neuroscience research, drug development, and commercialization experience and capabilities, and our shared commitment to bring innovative medicines to patients with neurological diseases establishes the foundation for a robust and compelling collaboration, said Stephane Boissel, Head of Corporate Strategy at Sangamo. This collaboration exemplifies Sangamos commitment to our ongoing strategy to partner programs that address substantial and diverse patient populations in disease areas requiring complex clinical trial designs and commercial pathways, therefore bringing treatments to patients faster and more efficiently, while deriving maximum value from our platform.

Under the terms of the collaboration, Biogen has exclusive global rights to ST-501 for tauopathies including Alzheimers disease, ST-502 for synucleinopathies including Parkinsons disease, and a third undisclosed neuromuscular disease target. In addition, Biogen has exclusive rights to nominate up to nine additional undisclosed targets over a target selection period of five years. Sangamo will perform early research activities, costs for which will be shared by the companies, aimed at the development of the combination of proprietary CNS delivery vectors and ZFP-TFs targeting therapeutically relevant genes. Biogen will then assume responsibility and costs for the investigational new drug-enabling studies, clinical development, related regulatory interactions, and global commercialization.

Sangamo will be responsible for GMP manufacturing activities for the initial clinical trials for the first three products of the collaboration and plans to leverage its in-house manufacturing capacity. Biogen will assume responsibility for GMP manufacturing activities beyond the first clinical trial for each of the first three products.

Upon closing of this transaction, Sangamo will receive $350 million comprised of $125 million in a license fee payment and $225 million from the sale of new Sangamo stock, or approximately 24 million shares at $9.21 per share. In addition, Sangamo may receive up to $2.37 billion in other development, regulatory, and commercial milestone payments, including up to $925 million in pre-approval milestone payments and up to $1,445 million in first commercial sale and other sales-based milestone payments. Sangamo will also be eligible to receive from Biogen tiered high single-digit to sub-teen double-digit royalties on potential net commercial sales of products arising from the collaboration. Closing of the transaction is contingent on completion of review under antitrust laws, including the Hart-Scott-Rodino (HSR) Antitrust Improvements Act of 1976 in the U.S.

Conference call

Sangamo will host a conference call at 8:00 a.m. ET tomorrow, Friday, February 28, which will be open to the public via telephone and webcast. During the conference call, Sangamo will discuss the collaboration, review financial results for the fourth quarter and full year 2019, and provide a business update. The conference call dial-in numbers are (877) 377-7553 for domestic callers and (678) 894-3968 for international callers. The conference ID number for the call is 4609858. Participants may access the live webcast via a link on the Sangamo website in the Investors and Media section under Events and Presentations. A conference call replay will be available for one week following the conference call on Sangamos website. The conference call replay numbers for domestic and international callers are (855) 859-2056 and (404) 537-3406, respectively. The conference ID number for the replay is 4609858.

About Biogen

At Biogen, our mission is clear: we are pioneers in neuroscience. Biogen discovers, develops, and delivers worldwide innovative therapies for people living with serious neurological and neurodegenerative diseases as well as related therapeutic adjacencies. One of the worlds first global biotechnology companies, Biogen was founded in 1978 by Charles Weissmann, Heinz Schaller, Kenneth Murray, and Nobel Prize winners Walter Gilbert and Phillip Sharp. Today Biogen has the leading portfolio of medicines to treat multiple sclerosis, has introduced the first approved treatment for spinal muscular atrophy, commercializes biosimilars of advanced biologics, and is focused on advancing research programs in multiple sclerosis and neuroimmunology, Alzheimers disease and dementia, neuromuscular disorders, movement disorders, ophthalmology, immunology, neurocognitive disorders, acute neurology, and pain.

Biogen routinely posts information that may be important to investors on its website at http://www.biogen.com. To learn more, please visit http://www.biogen.com and follow Biogen on social media Twitter, LinkedIn, Facebook, YouTube.

About Sangamo Therapeutics

Sangamo Therapeutics is committed to translating ground-breaking science into genomic medicines with the potential to transform patients lives using gene therapy, ex vivo gene-edited cell therapy, and in vivo genome editing and gene regulation. For more information about Sangamo, visit http://www.sangamo.com.

Biogen Safe Harbor

This press release contains forward-looking statements, made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995, including statements relating to the potential benefits and results that may be achieved through Biogens proposed collaboration with Sangamo; the anticipated completion and timing of the proposed transaction; the potential benefits, safety and efficacy of ST-501 and ST-502; the potential of Biogens commercial business and pipeline programs; Biogens strategy and plans; the potential treatment of neurological diseases; and risks and uncertainties associated with drug development and commercialization. These forward-looking statements may be accompanied by words such as aim, anticipate, believe, could, estimate, expect, forecast, goal, intend, may, plan, potential, possible, will, would, and other words and terms of similar meaning. Drug development and commercialization involve a high degree of risk, and only a small number of research and development programs result in commercialization of a product. Results in early stage clinical trials may not be indicative of full results or results from later stage or larger scale clinical trials and do not ensure regulatory approval. You should not place undue reliance on these statements or the scientific data presented.

These statements involve risks and uncertainties that could cause actual results to differ materially from those reflected in such statements, including, without limitation: risks that the proposed transaction will be completed in a timely manner or at all; the possibility that certain closing conditions to the proposed transaction will not be satisfied; uncertainty as to whether the anticipated benefits of the proposed collaboration can be achieved; risks of unexpected hurdles, costs or delays; uncertainty of success in the development and potential commercialization of ST-501 and ST-502 and other undisclosed neurological targets, which may be impacted by, among other things, unexpected concerns that may arise from additional data or analysis, the occurrence of adverse safety events, failure to obtain regulatory approvals in certain jurisdictions, failure to protect and enforce Biogens data, intellectual property, and other proprietary rights and uncertainties relating to intellectual property claims and challenges; product liability claims; and third party collaboration risks. The foregoing sets forth many, but not all, of the factors that could cause actual results to differ from Biogens expectations in any forward-looking statement. Investors should consider this cautionary statement, as well as the risks factors identified in Biogens most recent annual or quarterly report and in other reports Biogen has filed with the U.S. Securities and Exchange Commission. These statements are based on Biogens current beliefs and expectations and speak only as of the date of this press release. Biogen does not undertake any obligation to publicly update any forward-looking statements, whether as a result of new information, future developments or otherwise.

Sangamo Forward Looking Statements

This press release contains forward-looking statements regarding Sangamo's current expectations. These forward-looking statements include, without limitation, statements relating to the potential to use ZFP technology delivered via AAV to repress specific genes involved in neurological diseases, the ability of genomic medicine to provide one-time treatments, other statements regarding investigational therapies and their therapeutic benefits, statements related the anticipated effectiveness of the collaboration and the timing and benefits thereof, Sangamo's sale of shares of its common stock, receipt of an upfront payment and potential receipt of development- and sales-based milestones, as well as royalties on potential future sales, and other statements that are not historical fact. These statements are not guarantees of future performance and are subject to risks and assumptions that are difficult to predict. Factors that could cause actual results to differ include, but are not limited to, risks and uncertainties related to: the research and development process; the ability to cause the agreements to become effective on the proposed terms and schedule, the ability to obtain clearance under the HSR and to satisfy the other closing conditions, and the potential for technological developments by Sangamo's competitors that will obviate Sangamo's technologies, the new, uncertain and time consuming gene regulation therapy development and regulatory process, including the risks that Sangamo and Biogen may not be successful in their research efforts under the collaboration and that, even if successful, Biogen may be unable to successfully develop and commercialize licensed products resulting from the collaboration; Sangamo's dependence on collaborative partners, including the risks that if Biogen were to breach or terminate the agreement or otherwise fail to successfully develop and commercialize licensed products resulting from the collaboration and in a timely manner, Sangamo would not obtain the anticipated financial and other benefits of the collaboration and the development and/or commercialization of Sangamo's gene editing technology could be delayed, perhaps substantially. There can be no assurance that the necessary milestones or approvals will be obtained for any of the product candidates in this collaboration. Actual results may differ from those projected in forward-looking statements due to risks and uncertainties that exist in Sangamo's operations and business environments. These risks and uncertainties are described more fully in Sangamo's filings with the U.S. Securities and Exchange Commission, including its most recent Annual Report on Form 10-K and most recent Quarterly Report on Form 10-Q. Forward-looking statements contained in this announcement are made as of this date, and Sangamo undertakes no duty to update such information except as required under applicable law.

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New ammunition uncovered by U of T researchers to develop colorectal cancer treatment – Varsity

One in 14 Ontarians can expect to be diagnosed with colorectal cancer in their lifetime. COURTESY OF ED UTHMAN/FLICKR

University of Toronto scientists have identified a key protein as a common factor in the growth of many different types of colorectal cancer tumours, according to research published in the Journal of Cell Biology. Colorectal cancer develops in the colon or rectum. In Ontario, it is also the second most fatal cancer, and one in 14 Ontarians can expect to be diagnosed with this form of cancer in their lifetime.

In past research, scientists have linked the excessive accumulation of beta-catenin, a protein with crucial functions in cell development, to the expression of genes that drive tumour proliferation. Research has associated 80 per cent of colorectal cancers with gene mutations that greatly increase the production of beta-catenin.

The co-authors of the study have identified another protein, Importin-11, as the compound that enables beta-catenin transportation to the nucleus of the human cell. Cancer therapies that inhibit this transport could be a promising way to treat colorectal cancer.

Fundamental research provides new knowledge for cancer therapies

The Varsity spoke to Dr. Stephane Angers, a co-author of the study and an associate professor at U of Ts Department of Biochemistry. Angers lab has spent a considerable amount of time studying biological pathways the series of chemical changes during cellular development that give cells their final functions.

Angers noted that Monika Mis, the lead author of the study and a PhD student, uncovered the role of Importin-11 in colorectal cancer in Angers lab. Mis used the gene-editing CRISPR-Cas9 technology to screen genes in colorectal cancer calls to identify a novel gene, IPO11, which encodes for the protein Importin-11.

Current treatment options for colorectal cancer include surgery, chemotherapy, and other radiation therapy. Although this discovery is still in its fundamental stages, blocking the transport of beta-catenin holds great promise for developing new therapies.

As Angers put it, It provides new ammunition, new possibilities, and new knowledge that could lead in the future to new therapies, but it is very much at the discovery level at this point.

More research required to develop therapies

Further research could involve drug discovery and widen the scope of Importin-11 function in various cells. Researchers may also find it valuable to analyze existing data about colorectal cancer. The goal is to understand how the mutations affect tumour formation and develop therapies that harness this knowledge.

Angers lab is also investigating other potential applications of the Wnt pathway, a specific biological pathway associated with beta-catenin. A particularly interesting aspect is its role in regenerative medicine, which is the study of restoring human cells, tissues, and organs.

We think that with new molecules that we have developed we can now activate the pathway in order to promote the regenerative abilities of tissues, noted Angers.

Tags: biology, cancer, medicine, oncology

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New ammunition uncovered by U of T researchers to develop colorectal cancer treatment - Varsity

Recommendation and review posted by Bethany Smith

How targeting tumor blood vessels may help overcome treatment resistance in glioblastoma – FierceBiotech

Tumors often fuel their growth by forming new blood vessels to provide oxygen and nutrients. Widely used drugs directed against VEGF or VEGFR inhibit this process. Unfortunately, though, those drugs have failed to rein in the aggressive brain cancer glioblastoma.

Nowscientists at the University of Pennsylvania have demonstrated that targeting a mechanism in a subset of stromal cells known as endothelial cellswhich line the inside of blood vesselsmight help overcome drug resistance in glioblastoma. They believe the finding could point to a new therapeutic strategy to make these malignant cancer cells vulnerable to chemotherapy.

The team found that a mechanism within the well-known Wnt/beta-catenin signaling pathway causes endothelial cells to act morelike stem cells, leading to an abnormal growth of blood vessels that makes brain cancer cells resistant to treatment. Blocking Wnt/beta-catenin sensitized glioblastoma (GBM) to chemotherapy in mice, according to a new study published in Science Translational Medicine.

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GBM is difficult to treat partly because the tumors themselves often harbor different mutations, which makes treatments focused on one molecular target ineffective. So the UPenn team, led by Yi Fan, M.D., Ph.D., lookedbeyond particular genetic abnormalities in different groups of cancer cells and instead focused on overcoming resistance.

Fan's team searched GBM endothelial cells for the regulatory mechanisms that control chemoresistance, and found increased activation of multiple stem cell-associated transcriptional factors. The upregulation of these factors allowed the cells to propagate and also correlated with cell resistance to the widely used chemotherapy drug temozolomide (TMZ).

RELATED: Immuno-oncology combo targeting rogue enzyme in glioblastoma extends survival in mice

Further analysis revealed that the resistance is enabled by the Wnt/beta-catenin pathway, which regulates stem cell renewal. Its abnormal activation has perviously been linked to multiple cancer types.

In mice that had their endothelial cell-specific beta-catenin knocked out, treatment with TMZ cut tumor volume by 90%, whereas treating normal mice only slowed tumor growth. In a mouse model of GBM, the researchers combined Wnt inhibitor XAV939 with TMZ and significantly extended survival when compared to animals that got either drug alone.

Theres huge demand for better treatment options for GBM, as the diseases five-year survival rate remains low at around 5% to 10%. Researchers at the MD Anderson Cancer Center recently foundthat blocking the immune-suppressing enzyme CD73 could add benefits to inhibiting the immune checkpoints PD-1 and CTLA-4 in GBM. And ateam at Cedars-Sinai developed a polymer scaffold to deliver either one of these two types of checkpoint inhibitors cross the blood-brain barrier directly to brain tumor sites.

Ziopharm Oncology used its experimental drug veledimex to boost the immune response to IL-12 gene therapy. Investigators recently found signs of positive responses of the regimen among a small group of patients with recurrent glioblastoma.

Fan believes his teams approach of using Wnt inhibitors to block endothelial cells boasts several advantages over directly attacking cancer cells. For one thing, treating these stromal cells would get at the root cause of tumor survival. Secondly, because it doesnt aim for genetic markers, it should remain effective even after tumors mutate.

Because stromal cells have a more stable genome, they will not mutate the way cancer cells do, meaning secondary resistance is unlikely, Fan said in a statement. The team now hopes to test the method in a clinical trial.

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How targeting tumor blood vessels may help overcome treatment resistance in glioblastoma - FierceBiotech

Recommendation and review posted by Bethany Smith

Precision medicine in 2020: what barriers remain for drug developers? – pharmaceutical-technology.com

]]> High costs, data management issues and recruitment problems are some of the main challenges for personalised medicine in clinical development. Credit: US Air Force/Kemberly Groue Genome sequencing costs are falling fast, opening the playing field for developing highly personalised drug candidates. Personalised medicine in the 21st century offers the promise of therapies customised based on the study of what truly makes us unique: our DNA.

The importance of the individual has been widely established in medicine since time immemorial. The well-worn adage that physicians should treat the patient, not the disease has been around since the 19th century, and the awareness of that message is far older than that. Even Hippocrates, the father of Western medicine who treated patients in the fifth century BC, stressed the importance of treating each patient as an individual.

For the sweet [medicines] do not benefit everyone, nor do the astringent ones, nor are all patients able to drink the same things, Hippocrates wrote.

Hippocrates might have tailored his rudimentary treatments based on the patients age, physique and other easily observable factors, but personalised medicine in the 21st century offers the promise of therapies customised based on the study of what truly makes us unique: our DNA.

Advancements in genomics, proteomics, data analysis and other fields both medical and technical are gradually facilitating the development of laser-focused drugs, as well as the ability to predict peoples personal risk factors for particular diseases and how individual responses to various treatments might differ.

After years of anticipation, there is now evidence that governments around the world have clocked the importance of personalised medicine and are driving efforts to the build the genetic data sets and biobanks that are required to push the science forward. Former US President Barack Obama launched the Precision Medicine Initiative to great fanfare in 2015; the scheme has since evolved into the All of Us research programme, which aims to gather health data from more than a million US volunteer-citizens to unlock new insights.

In the UK, the 100,000 Genomes Project reached its goal of sequencing 100,000 whole genomes from 85,000 NHS patients with cancer or rare diseases. Genomics England has noted that so far, analysis of this data has revealed actionable findings in around one in four rare disease patients, while about 50% of cancer cases suggest the potential for a therapy or clinical trial.

You can match a blood transfusion to a blood type that was an important discovery, said Obama at the launch of the Precision Medicines Initiative, summarising the broad appeal of personalised therapies and diagnostics. What if matching a cancer cure to our genetic code was just as easy, just as standard? What if figuring out the right dose of medicine was as simple as taking our temperature?

The stage might be set for personalised healthcare to dramatically transform public health, but few in the medical field would deny that the world is hardly ready yet. Transitioning from the traditional one-cure-fits-all treatment model to new processes that leverage patients genetics, lifestyles and environmental risk factors is an immense task that presents challenges in both the laboratory and the clinic.

Oncology is, by a landslide, the field that has been most impacted by developments in precision medicine; around 90% of the top-marketed precision treatments approved in 2018 were cancer therapies, while other therapeutic areas have lagged far behind. The majority of approved precision medicines in oncology achieve something of a halfway house between the old way and the new they fall short of being tailored to a specific individual, but they allow for more detailed stratification of patients by the oncogenic mutations of their tumours, which may be driving cancer cell survival and growth.

Common examples of these mutations are HER-2 in certain breast and stomach cancers, BRAF in melanoma and EGFR in lung cancer. High expression of these proteins at cancer sites can be targeted by precision treatments, such as Roches monoclonal antibody Herceptin (trastuzumab) for HER-2, Genentechs BRAF inhibitor Zelboraf (vemurafenib), and Roches EGFR inhibitor Tagrisso (osimertinib). Regulators such as the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA) are also increasingly approving tumour-agnostic treatments the first and most famous of which is Mercks immunotherapy Keytruda (pembrolizumab) which target specific biomarkers regardless of tumour location.

But despite the availability of a growing menu of personalised cancer treatments, actually matching patients up to the right therapy can be difficult. According to a survey of US acute care organisations conducted by Definitive Healthcare and published in December 2019, just over 20% had established precision medicine programmes. Investment in genomic testing is vital to quickly get patients on the best treatment course, but financial and operational barriers remain.

The foremost among these is the cost associated with genomic sequencing and the use of companion diagnostic devices, cited by 28% of Definitive Healthcares respondents as the biggest challenge for already-established precision medicine schemes. Lack of expertise is another obstacle, as many physicians may struggle to accurately interpret test results without specialist assistance another major cost driver for clinics and hospital departments trying to build pathology teams that are up-to-date with the newest tests. A 2018 survey of 160 oncologists by Cardinal Health found that 60% of physicians who dont use genomic tests avoid them because of the difficulty of interpreting the data.

In clinical research and development, too, there are growing pains associated with moving the pharmaceutical pipeline towards drugs targeting smaller patient sub-groups. Again, cost is a central issue companion diagnostics dont come cheap, finding and validating biomarkers to guide targeted therapies is a lengthy task, and analysing vast amounts of data often requires new teams with specialised knowledge.

The expense of incorporating a host of new processes into innovative trial designs not to mention the cost of manufacturing cell and gene therapies obviously has an impact on the list price of personalised drugs that win approval. This is most clearly seen in the eye-watering prices of some of the worlds first truly individualised cancer treatments, chimeric antigen receptor T-cell (CAR-T) therapies.

Treatments such as Novartiss Kymriah and Gileads Yescarta remove T-cells from the patients blood, modify them to target tumour cell antigens and then infuse them back into the blood stream. These therapies have achieved impressive results in rare and advanced cancers, but cost upwards of $400,000 per patient, limiting their reimbursement options among both private and public payers. Promising advances in CAR-T manufacturing and potential off-the-shelf T-cell production could help bring these costs down in the years to come, but for now the problem remains.

As for the broader clinical trial eco-system, these studies have been historically set up to assess a drug candidates safety and efficacy in an increasingly large segment of the patient population, building evidence towards the regulatory approval process. Bringing a personalised medicine through the clinical development process is a new paradigm in a number of ways; as well as the aforementioned cost drivers, there can be an extra enrolment burden to identify and recruit patients this is already a common cause of trial failure, but its all the more difficult when youre looking to access a small patient sub-group with the appropriate biological profile.

The difficulty of providing sufficient evidence of safety and efficacy can also present issues where current regulations struggle to accommodate new innovations in personalised medicine. Smaller trial designs present statistical problems in terms of understanding a drugs definitive risk-benefit profile, and while some personalised applications can be discovered as part of larger trials that fail to meet their endpoints outside of a select patient group with particular biomarkers, many current regulations dont accept post hoc analysis and would require an entirely new trial.

Personalised medicine developers desire better guidance on how best to design a successful clinical trial for a personalised therapy, because absent guidance, they risk presenting suboptimal evidence regarding stratification options, reads a 2017 study on personalised medicine barriers, published in the Journal of Law and Biosciences. Designing clinical trials for differently responding subgroups (for example, biomarker-positive and biomarker-negative groups) requires additional time and resources. Companies are reluctant to make this investment without a commensurate increase in the certainty of regulatory approval.

The increasing use of surrogate endpoints, conditional approvals and real-world data is helping to address these issues, but theyre not yet an ideal solution. Conditional approvals rely on very careful post-marketing observation and analysis, while the value of surrogate endpoints has been questioned, adding to the tension between accelerating approvals and ensuring patient safety.

The ultimate benefits of creating more personalised treatments are clear, and their advantages for human health could, in the long-term, be matched by their economic returns. After all, quickly treating patients with the right therapy for them or, even better, using knowledge of a patients genetic risk profile to prevent illness in the first place would be a huge financial gain for overburdened health systems.

Todays costs are gradually falling, as NIH data on DNA sequencing costs demonstrate. But there is still a long way to go before we can wave goodbye to the blanket drug development that has dominated modern pharma for decades, even in the advanced field of oncology, let alone other therapeutic areas. Only a sustained and holistic push from regulators, drug developers, clinicians, governments and others will be enough to bring us over the line.

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Precision medicine in 2020: what barriers remain for drug developers? - pharmaceutical-technology.com

Recommendation and review posted by Bethany Smith

United Therapeutics: Buy The Dip – Seeking Alpha

On February 26, United Therapeutics (UTHR) came out with earnings results for the fourth quarter of 2019. The companys revenues of $311.11 million were down YoY by 18.43% and fell short of the consensus by $46.06 million. The companys fourth-quarter non-GAAP EPS (earnings per share) of $1.96 also missed the consensus by $1.43.

Subsequently, the stock is down by 11.97% from $112.86 on February 25 to $100.79 on February 27. I believe that this is an investor overreaction, considering that the dip in revenues and EPS is associated with a one-time event of inventory destocking by a distributor in the fourth quarter. Earlier in 2019, this distributor had placed larger-than-normal orders for the companys products due to an error in its patient utilization data. Hence, the smaller-than-normal order in the fourth quarter was an attempt to normalize inventory levels.

Besides this small glitch, the company boasts of robust fundamentals. Today, we will see why United Therapeutics can prove to be an attractive opportunity in 2020.

United Therapeutics is a biotechnology company focused on developing novel, life-extending therapies in lung disease, oncology, and organ manufacturing indications. The company commercializes therapies including an infused formulation of the prostacyclin analog treprostinil, Remodulin; an inhaled formulation of treprostinil, Tyvaso, and a tablet dosage form of treprostinil, Orenitram; and an oral PDE-5 inhibitor, Adcirca, in PAH (pulmonary arterial hypertension) indication. The company also offers Unituxin, a monoclonal antibody for treating neuroblastoma.

United Therapeutics is now working on developing next-generation technologies for Remodulin, Tyvaso, and Orenitram. The companys R&D pipeline also has investigational novel biologics and NCEs (new chemical entities) targeting various lung, oncology, and organ manufacturing indications.

In July 2002, the company secured FDA approval for Remodulin as a continuous subcutaneous infusion for the treatment of PAH patients with NYHA Class II-IV symptoms to diminish symptoms associated with exercise. Despite the launch of a generic treprostinil in early 2019, Remodulins sales were only 2% down YoY to $587 million in 2019. In fact, the company reported the highest number of new Remodulin starts in the last ten years.

The YoY decline in Remodulin sales was attributed to a small number of patients switching to the generic drug, as well as to the fact that new patients start on lower dosages of Remodulin and then begin the process of titrating to their effective dose. Since the patient switching trend has all but trickled down, the company now expects U.S. Remodulin patient demand as well as sales to jump up YoY in 2020.

Remodulin demand has been pretty sticky for years. It is a very complex product targeting a very fragile patient population, which places a high premium on years of safety and supply chain reliability.

United Therapeutics is now getting ready to launch the subcutaneous delivery system for Remodulin injections, the RemUnity system, in July 2020. Co-developed by United Therapeutics and Deka Research and Development and cleared by FDA under the FDA 510(K) pathway, the RemUnity system is expected to reduce the risk of bolus dosing due to pump failures and provides wider arrays of notifications, alerts, and alarms than current pumps. Additionally, the RemUnity system will control Remodulin flow rates without the use of a motor. The pump is small and lightweight with a service life of at least three years.

On February 24, the company announced preliminary analysis from Phase 3 INCREASE study evaluating Tyvaso Inhalation Solution in patients suffering from WHO (World Health Organization) Group 3 pulmonary hypertension associated with PH-ILD (interstitial lung disease). The trial met its primary endpoint of demonstrating improvement in 6MWD (six-minute walk distance). In addition to increasing six-minute walk distance by 21 meters versus placebo after 16 weeks of treatment, Tyvaso also demonstrated significant improvements in each of the study's secondary endpoints, including reduction in the cardiac biomarker NT-proBNP, time to first clinical worsening event, change in peak 6MWD at Week 12, and change in trough 6MWD at week 15. Based on the INCREASE results, the company now plans to submit a supplemental new drug application to expand the Tyvasos label in mid-2020.

In June 2019, the company submitted a 505(B)(1) NDA (new drug application) for Trevyent disposable treprostinil pump system. Although the FDA first accepted the application and set PDUFA (prescription drug user fee act) date as April 27, 2020, the agency later included a mid-cycle information request noting several deficiencies in the Trevyent NDA. While the company has already sent its responses to the FDA, based on recent discussions with the FDA, the company expects a delay in the PDUFA date. The company expects this FDA approval in the next 18 months.

United Therapeutics also expects the launch of ISR (Implantable System for Remodulin) codeveloped with Medtronic (MDT) to be delayed to 2021. The delay of the commercial launch is attributable to Medtronic satisfying various conditions to its PMA approval.

United Therapeutics estimates the prevalence of PAH WHO Group 1 in the U.S. to be more than 45,000. The currently approved PAH drugs are for PAH WHO 1 class patients. According to PHA (Pulmonary Hypertension Association), WHO Group 1 refers to pulmonary arterial hypertension (PAH), which is caused when the arteries in the lungs become narrowed, thickened or stiff. The right side of the heart must work harder to push blood through these narrowed arteries. This extra stress can cause the heart to lose its ability to pump enough blood through the lungs to meet the needs of the rest of the body.

However, the prevalence of PAH WHO Group 3 patients in the U.S is even higher, of around more than 130,000 patients. These patients do not have any FDA approved treatment option. Again, according to PHA, WHO Group 3 includes PH due to chronic lung disease and/or hypoxia (low oxygen levels). These lung diseases include obstructive lung disease where the lung airways narrow and make it harder to exhale (e.g. COPD or emphysema); restrictive lung disease in which the lungs have a tough time expanding when one inhales (e.g. interstitial lung disease or pulmonary fibrosis); sleep apnea, and living in an area of high altitude for a long period of time. Arteries in the lungs tighten so that blood can only go to areas of the lungs that are receiving the most air and oxygen. This tightening leads to high blood pressure throughout the lungs.

United Therapeutics is working to increase its total patient base from around 7,500 to 25,000 in the next five years.

Besides Remodulin, Tyvaso and Orenitram also saw the highest patient demand in 2019 as compared to the last four years.

Then again, United Therapeutics expects RemUnity, Trevyent, ISR, and Smart Pump technology to have a very favorable impact on future patient demand and sales of Remodulin. The company also expects label expansion of Tyvaso based on the INCREASE study to increase the addressable U.S. population by more than 30,000 patients. This will also position the Tyvaso as a treatment option for a section of the PAH WHO Group 3 patients, those with interstitial lung disease. The company is studying Tyvaso in COPD (chronic obstructive pulmonary disorder) patients in the Phase 3 PERFECT study. Finally, the expanded label of Orenitram based on the FREEDOM-EV trial highlighting superior morbidity and mortality outcomes is also helping to attract new PAH patients.

United Therapeutics is now studying Tyvaso in PH WHO Group 3 patients, PH COPD patients, in Phase 3 trial PERFECT. The company seems to have high hopes for the success of the trial after robust results from the INCREASE study since inhaled therapies can avoid the VQ (Ventilation Perfusion Mismatch), as is seen in system therapies targeting lung diseases. The PERFECT trial is currently 20% enrolled.

Besides studying already approved products for additional indications, the company also has a slew of investigational NCEs and biologics in its pipeline.

United Therapeutics expects investigational Phase 3 asset, ralinepag, to transform the care of WHO Group I PH patients. The company is also studying a gene therapy study in the Phase 3 trial, SAPPHIRE, to make PH treatment more convenient with just a once quarterly infusion of genetically engineered autologous stem cells from the patient. The company expects enrollment to be completed in 2021, and product launch by 2025. Finally, the company is attempting its hand at the much challenging and lucrative organ manufacturing segment.

The biggest risk weighing down on United Therapeutics valuation is the potential impact of generic erosion of Remodulin. The company depends extensively on its topline on the treprostinil franchise, exposing it to significant business concentration risk.

The company is exposed to R&D failure risk and subsequent contraction in probable growth drivers. The most recent example of such a failed trial is the phase 2/3 DISTINCT study evaluating Unituxin in Injection added to irinotecan compared to irinotecan or topotecan alone in patients with relapsed or refractory SCLC (small cell lung cancer). On February 3, 2020, the company announced that this trial did not meet its primary endpoint of extending OS (overall survival).

According to finviz, the 12-month consensus target price of the company is $122.22. On February 27, Cowen analyst Chris Shibutani upgraded United Therapeutics from Market Perform to Outperform and raised the price target from $119 to $145. On February 25, Wedbush analyst Liana Moussatos raised her price target from $237 to $243 and reiterated Outperform rating for the stock. On February 13, Credit Suisse analyst Martin Auster reiterated the Outperform rating and raised his price target from $113 to $121. On January 31, JPMorgan analyst Jessica Fye revised upwards United Therapeutics rating from Neutral to Overweight and price target from $116 to $120. On December 27, Oppenheimer analyst Hartaj Singh reiterated the Outperform rating and $155 target price.

We see that the overall analyst sentiment and target price have consistently improved for the company since March 2019.

Based on the companys robust product portfolio, strong pipeline, and multiple upcoming catalysts, I believe that $145 is a fair estimate of the true value of the company. I believe that retail investors with above-average risk appetite should consider buying the company on the latest dip in 2020.

Disclosure: I/we have no positions in any stocks mentioned, and no plans to initiate any positions within the next 72 hours. I wrote this article myself, and it expresses my own opinions. I am not receiving compensation for it (other than from Seeking Alpha). I have no business relationship with any company whose stock is mentioned in this article.

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United Therapeutics: Buy The Dip - Seeking Alpha

Recommendation and review posted by Bethany Smith

cyborgs, robots, and biohackers: the first-ever survey of transhumanism – Designboom

london-based photographers david vintner and gem fletcher document individuals who form part of the transhumanism culture throughout europe, russia and the united states in their latest collaborative photo series, I want to believe an exploration of transhumanism. the five-year-long project explores the core idea behind transhumanism the belief that human beings are destined to transcend their mortal flesh through technology.

neil harbisson hears color neil harbisson was born with achromatism, a rare disease that renders him colourblind. rather than overcome achromatism, harbisson created a new sense to go beyond the human visual spectrumin 2004 he had an antenna implanted into his skull. the antenna allows him to perceive visible and invisible colours as audible vibrations, including infrareds and ultraviolets.

the photo series by vintner and fletcher illustrates three gradual stages of transhumanism from testing ground, patient zero to humanity 2.0. at the lowest tier, testing ground looks into individuals who have created wearable technology to expand their human abilities, improving everything from concentration to mental health.patient zero studies those who have taken permanent action to become half human and half robot. in the final chapter, humanity 2.0, the transhumanist subjects focus on life extension and immortality.

the work of the individuals in this book demonstrates how optimizing our brains and bodies could revolutionize and redefine humanity. as human architects, we are only limited by our imagination, explains vintner and fletcher.

kevin warrick widely considered as one of the first cyborgs.kevin warrick is a pioneering professor in cybernetics and considered by many as the worlds first cyborg. kevin instigated a series of experiments involving the neuro-surgical implantation of a device into the nerves of his left arm in order to link his nervous system directly to a computer. this enabled him to have a symbiotic connection with a robotic hand. he could control the hand using his own brain signals from anywhere in the world, as well as sense what the robot hand was feeling.

humans are now gods. we are now able to create and design humans, but do humans have the foresight to do it in the right way? questions the photographers.

cyborg arm

for many transhumanists, life extension and immortality is the goal. transhumanism started as early as 1923 and has developed over recent years through the rise of sci-fi themed books, movies and the democratization of technology. as studies on experimental genetic engineering, tissue regeneration and stem cell treatments are also becoming more apparent in todays world, transhumanists hope to extend the life of the human body anywhere from twenty to 500 years longer than the average lifespan.

vintner and fletcher are working together on releasing the photo series as a book, which can be funded on crowd-funding platform kickstarter, here.

moon ribas sensing earthquakesmoon ribas is connected to online seismographs allowing her to perceive the seismic activity of the planet through vibrations in her body. the vibration she feels depends on the intensity of the earthquake. if she is standing in newcastle, she can sense earthquakes happening everywhere from japan to greece. she describes the sensation as having two heartbeats, her biological heartbeat and the earthbeat, which has its own rhythm inside her body.

dr natasha vita-more a leading expert on human enhancement and emerging technologies

dr. aubrey de grey biomedical gerontologist and the chief science 0fficer of SENS research foundation

liz parrish founder of bioviva

dr max more president and CEO of the alcor life extension foundation

patient zero - james young after an accident that left him a double amputee, james young turned to bionics to redesign his body. obsessed with the metal gear solid, he worked with gaming giant konami and prosthetic sculptor sophie de oliveira barata to develop an advanced bionic arm inspired by the computer game.

carbon fibre bionic limb

patient zero - rob spence known as the eyeborg, rob spence lost an eye as a child while playing with his grandfather's shotgun. inspired by a love of the bionic man and his interest in documentary filmmaking, spence created an eye with a wireless video camera inside. the camera is not connected to his optic nerve but sends footage to a remote receiver. over the years, he has created several different aesthetics for the eye, from a realistic 'hidden camera' version to a terminator inspired glowing red version.

patient zero - neil harbisson neil harbisson was born with achromatism, a rare disease that renders him colourblind. rather than overcome achromatism, harbisson created a new sense to go beyond the human visual spectrumin 2004 he had an antenna implanted into his skull. the antenna allows him to perceive visible and invisible colours as audible vibrations, including infrareds and ultraviolets.

image out of rob spence's eyes

new ways of seeing - EYEsect the experimental device aims to recreate the experience of seeing the world like a chameleon, with two single steerable eyes. in changing the way we perceive the world around us, eyesect alters our version of reality enabling new ways to sense and experience our environment.

new ways of seeing - north sense created by liviu babitz and scott cohen, north sense is a small matchbox-sized artificial sense organ that delivers a short vibration every time the user faces north, similar to the biological abilities of migratory birds, extending the human perception of orientation.

new ways of seeing - aisen caro chacin / echolocation the echolocation headphones are a pair of goggles that aid human echolocation. it is designed to substitute the users vision augmenting our spatial awareness with sound, similar to the abilities of bats and whales. the device has both the potential to aid the visually impaired and provide sighted individuals with a new sense.

project info:

title:I want to believe an exploration of transhumanism

artistic director: gem fletcher

photographer: david vintiner

kick starter page: I want to believe

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cyborgs, robots, and biohackers: the first-ever survey of transhumanism - Designboom

Recommendation and review posted by Bethany Smith

Penny Stocks To Watch This Week; Coronavirus Still A Concern – Penny Stocks

Are These Penny Stocks On Your Watch List Right Now As Coronavirus Fuels Headlines?

Over the weekend, global headlines continued to see countless mentions of coronavirus cases. While many compare it to the average flu, its global spread hasnt been ignored. While the death toll among young, healthy individuals hasnt become a major concern, the fact that mortality is an issue, has been a focal point. Meanwhile, the broader markets are coming off the worst week since the financial crisis.

However, if you are an active reader of PennyStocks.com, it was actually a huge week for coronavirus penny stocks. Last week saw many of these coronavirus stocks reach highs that were well out of penny stock range. Some even blew clear past $40 per share at times.

While the S&P and Dow posed a comeback at the end of the day, we cant ignore continued concerns over the weekend. The first U.S. death has been recorded while new states are reporting their first cases. Meanwhile, weve got a big week of economic data ahead.

Coming up first, weve got China PMI data to consider. It showed the fastest contraction in factory activity ever in February. This was even worse than during the financial crisis. Meanwhile, the likelihood of a Fed rate cut just got that much greater. Slower non-farm payroll figures from Friday didnt help either.

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Consensus forecasts showed payrolls gaining 175,000, which slowed from 225,000 in January. While the U.S. economy appears increasingly vulnerable to the outbreak, the Fed has said it will act as appropriate in response to evolving risks. On another note, The Bank of Canada will also hold a policy meeting this week.

Some analysts think that the Bank could pre-emptively cut rates. This may be a further indication of whats to come for other central banks. The BoC has been known to move early in certain instances, so further market risk could be in play and in favor of certain penny stocks like gold if this is the case.

Then weve got Super Tuesday to think about. A total of 14 states will cast ballots in favor of their optimal Democratic candidate. Bernie Sanders has been said to hold the lead heading into the week. But Super Tuesday will further shed light on what American Democrats want in a candidate, this week. Given everything at work, there are certain sectors that those investing in penny stocks could be focusing on this week.

First, though gold stocks have dipped amid coronavirus concerns, it doesnt mean they will be counted out by any means. Certainly, broader sector stocks felt the brunt of the latest outbreak. However, given the financial uncertainty and economic shifts, gold stocks have historically presented safe-haven options in the market. There are several gold penny stocks that either held a trend or saw a late-Friday bounce last week.

First, IMC International Mining Inc. (IMIMF Free Report)(IMCX) was one of the only gold penny stocks that didnt pull back aggressively last week. In fact, for most of February, shares traded within a range of high $0.30s to low $0.40s. While its U.S. listing is only about a month old, its Canadian listing shows that momentum has built since the beginning of the year. Around January 8th, the stock traded around $0.25 CAD and managed to reach highs of $0.50 CAD.

In February, the company reported several big developments that could be in focus this month. Aside from appointing a new CFO, IMC International also entered into the early phases of a potential acquisition. IMC signed a letter of intent to acquire Thane Minerals, owners of the Cathedral property in northern British Columbia. The Cathedral Project is in an area called the Quesnel Terrane which is rich in mineralization perfect for gold discovery.

The fact that Cathedral is still in a relatively unexplored portion of the northern Quesnel Terrane could signal an opportunity for the company. Considering the proposed acquisition is in its early phases, the market may be watching closely to see if IMC will formally close. It would present immediate access to the Quesnel Terrane via the Catherdral property. The area hosts past and planned production of 50 billion pounds of copper and 28 million ounces of gold.

Next, Yamana Gold stock (AUY Free Report) could be one of the potentially oversold gold penny stocks to watch next week. Shares plummeted last week as coronavirus fears put a stranglehold on the market. After hitting fresh highs of $4.94 on the 24th, Yamana gold stock pulled back to lows it hadnt seen since mid-January.

Aside from last weeks market fueled sell-off, Yamana has actually performed well. That goes from a price and a fundamental perspective. The companys created a pipeline of highly viable exploration projects and created significant positions across the globe with producing assets. But the next move has Yamana focusing on the longer-term.

Yamana has always taken a long-term strategic perspective. We believe that investing in a generative exploration program today will secure our future tomorrow. We have strong prospects in mine-friendly jurisdictions that we know well, and we are confident that they will become a cornerstone of Yamanas next generation of mines.

Yamana has also further optimized its portfolio by shedding assets. For example, last month the company sold off its portfolio of royalty interests and contingent payments from the Deep Carbonates project for $65 million. This is broken up into a $20 million cash component and $45 million share position of which Yamana can further benefit from the potential success of the project.

Similar to Yamana and unlike IMC International, Hecla Mining (HL Free Report) saw its stock drop last week. For Hecla, it was a dip to new 2020 lows. However, in a similar fashion to other gold stocks, HL stock rebounded by the end of the day on Friday. Overall, the sentiment surrounding the company hasnt been bearish.

Earlier last month, the company announced fourth-quarter and full-year 2019 results. Sales for Q4 came in at $225 million with full-year sales hitting $673.3 million marking the highest in company history. Silver production from Hecla came in at 12.6 million ounces along with record gold production of 272,873 ounces. Hecla also reported record reserves for silver, lead, and zinc. Aside from this, the company was able to decrease its net debt by $136 million.

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In 2020, at current prices, we expect continued strong cash flow generation with the ongoing solid performance at Greens Creek, the ramp-up of Lucky Friday, expected improvements at Casa Berardi and the potential mine life extension at San Sebastian from the Hugh Zone, said Phillips S. Baker, Jr., President and CEO of Hecla. During after-hours trading on February 28, Hecla gold stock traded higher at $2.79, which may be something to note if its one of the names on your penny stocks watch list for this week.

After last week, theres no question that biotech penny stocks have taken a spotlight. While some stock saw their biggest drops in recent weeks, many biotech penny stocks reached record highs. In fact, many of these coronavirus penny stocks have been covered consistently for some time now. We put an article out getting everyone up to speed last week Coronavirus Madness: Stocks That Scored Big For Investors. Could these penny stocks to watch join the list this week?

First, Biocept Inc. (BIOC Free Report) was one of the biotech penny stocks discussed in January. At the time the company announced that its Target Selector assays were available to doctors. The focus is for doctors to use the product to evaluate cerebrospinal fluid of patients in checking for tumors. Earlier last month the company reported that it entered into an agreement with a California-based physician association to provide testing services to physicians and patients in that network.

Considering that testing could become a big focus during this outbreak, Biocept appears to have gained attention. On Friday, BIOC stock saw one of its biggest days yet. Shares traded from an open of $0.285 as we focused on the company. By the closing bell, the penny stock was at $0.78. While there hasnt been much coronavirus-specific news, as weve seen speculation played a big role with the latest boom in coronavirus penny stocks.

If Biocept is one of the names on your list of penny stocks, keep this information in mind. Hype may need to be factored into this equation. If BIOC is falling into the coronavirus stock category, take particular consideration in the fact that its pipeline is focused on testing for cancerous tumors. Also, keep in mind that BIOC made a dramatic move on Friday and profit-taking could also be something to keep mindful of in the week ahead.

Another one of the coronavirus penny stocks to watch might be Heat Biologics (HTBX Free Report). While most coronavirus stocks have been taking off for days, HTBX stock seems to have just caught attention on February 28. This came after a downtrend for most of the year. Theres no recent news and no new filings. But several popular message board sites are paring HTBX stock with coronavirus-related news.

Similar to Biocept, Heat Biologics focuses on cancer and T-cell stimulation. Nothing on its site nor in its previous updates were related to coronavirus or the common flu. Meanwhile, back in December, the company reported that it had dosed its first patient in a Phase 1 trial of its t-cell treatment, HS-130. Something else to note is that on the corporate site, it shows the company held a 2020 Special Meeting of Stockholders on February 27.

So, while the market digests the latest coronavirus data, HTBX could find itself in the conversation apparently. In a company 8-K filing, the results of the special meeting showed shareholders agreed to each of Heats proposals. This included an increase to authorized shares from 100m to 250m, effecting a reverse split of common stock, as well as updates to its stock incentive plan. The company also approved authorization to issue undesignated preferred stock. In regard to the reverse split, the company gave a ration within a range of 1 share of Common Stock for every 2-50 shares of Common Stock.

Finally, Oragenics Inc. (OGEN Free Report) was one of the top penny stocks to watch in February. Last Friday, that momentum continued to push shares to new 11-month highs after the market close. OGEN stock reached $0.90 after closing the regular session just under $0.75.

The biotech penny stock started moving as it approached an industry presentation date in early February. We also highlighted all of the FORM 4s filed that showed insider movement in the stock. But there could be a few things at work right now to take notice of, mainly the companys focus on infectious diseases. Oragenics reported the completion of enrollment of its Phase 2 clinical trial for AG013 in Oral Mucositis. More specifically, the company previously reported that it plans to deliver the results of its study early this year.

While coronavirus penny stocks are in focus, timing is of importance. Thats also considering the timeline given in recent CDC press conferences too. Given Fridays momentum will OGEN continue to be one of the coronavirus stocks to watch this week? On its website, the company even states:

Oragenics, in collaboration with Precigen, is in the process of developing lantibiotics, a novel class of antibiotics that are known to be active against several life-threatening infectious agents, toward the goal of commercialization for the treatment of infectious diseases in humans.

Pursuant to an agreement between Midam Ventures LLC and IMC International Mining Corp.(CSE: IMCX), Midam has been paid $200,000 for a period from January 7, 2020, to February 14, 2020. Midam has been paid an additional $200,000 and extended its period of coverage to March 14, 2020. We may buy or sell additional shares of IMC International Mining Corp.(CSE: IMCX) in the open market at any time, including before, during or after the Website and Information, to provide public dissemination of favorable Information about IMC International Mining Corp. (CSE: IMCX).Full disclaimer here.

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The Bad Economics of the U.S. Health Care System Shows Up Starkly in its Approach to Rare Diseases – TIME

If you ever end up in an emergency room, the first thing that happens is a doctor or nurse will check your critical vitals: your temperature, blood pressure, respiratory rate, and pulse. If those indicate your life is at risk, your care is prioritized over others who have already been waiting or who have been seen by a doctor but require additional testing (e.g. an x-ray or blood test) or a specialist to review their symptoms. This process, called triaging, is the global standard for allocating resources in emergency care.

Triaging is a marvel of modern healthcare if it is abundantly clear that you are on deaths door. But if theres no textbook description of your condition, it can leave you needlessly suffering while the experts try to figure it out. Thats often the case for those with rare diseases, a group of conditions that are not individually common, but combined, affect an estimated 10% of the global population, some 475 million people. An estimated 80% of the 7,000 identified rare diseases are caused by DNA mutations that occur during pregnancy, meaning most of those with this category of illness are born with it. In many cases, these babies emerge from the womb with life-threatening conditions that doctorsworking the triage systemwill immediately address. However, this also tends to lead doctors to then ignore the underlying rare diseasean unnecessary medical expenditure, in the triage system framework.

I am one of the people living with a rare disease.

I was born with a number of symptoms and signs that put my life at risk a collapsed lung, a premature exit from my mothers body after only six months, and malnutrition from a hole in my amniotic sac. These problems were all treated and resolved over a multiple month stay in the hospital dictated by the triage system. I was allocated hospital resources for my life-threatening conditions until I was deemed stable enough to go home: the point at which I wouldnt die if I left the care of the hospital.

But there was another problem that was overlooked, and which wasnt diagnosed until I was five years old.

Every bone in my body was bent and every muscle atrophied or non-existent. I couldnt move my neck away from shoulder; I couldnt straighten my legs, knees, arms, wrists, ankles, toes, hands, or fingers beyond fixed, fully bent positions.

The triage system worked at saving my life, but never addressed how I would live day to day or even physically move from a single location by myself. It never addressed the underlying issue and root of the problem: a rare orthopedic genetic disease.

The life or death triage standard is one of the primary reasons that it takes, on average, seven years for people with a rare disease to get a diagnosis in the U.S.and

I am one of the lucky ones. Serendipitously, a Because of these surgeries, and additional ones Ive had since, I could feed myself, live free from a wheelchair, go to school through the post-graduate level, and hold a full-time job.

The orthopedic surgeries I neededover 29 of them in less than 30 yearshave cost millions of dollars. And these costs grow each year as I undergo additional exploratory surgery in the absence of any cure. However, these costs are still lower than what I would have incurred had I been left as the triage system deemed stable as an infant. I would have required 24-hour in-home care my entire life. I would never have been able to use the bathroom alone, to get dressed alone or to even leave the house alone. The lifetime value of a working individual according to the US Office of Management and Budget is on average $7 million to $9 million. The cost of a full-time caregiver is on average $40,320 a year; if a rare-disease patient reaches the average US life expectancy of 78 years old, the lifetime cost of full-time care is at least $3 million.

Our healthcare system needs to weigh the long-term costs of leaving behind people with rare disease, and, more specifically, evaluate the economic consequences that follow at a global scale.

Even more so, we need to weigh the costs of creating a pipeline to fill the treatment gap facing people with rare disease., Using genomic sequencing, clinicians can holistically understand the genetic roots of rare disease and even potentially cure rare disease through gene therapy, which modifies and permanently fixes abnormal genes that cause a specific rare disease at birth.

Nonetheless, identifying the root genetic cause of rare disease is the only way to begin to cure a rare disease rather than just treating the symptoms in an ad hoc fashion. While certain pharmaceutical drugs can be developed from the findings in a genomic sequence to help mitigate or lessen symptoms, the science suggests the only way to cure a rare disease is to administer an even newer science called gene therapywhich modifies and permanently fixes genes that are abnormal. Considered to be the most expensive option, a However, this nascent science is costly: gene therapy costs around $2 million for current US Food and Drug Administration (FDA)-approved options.

Insurance plans in the US rarely pay for clinical-grade whole-genomic sequencing (which can carry a price tag of up to $9,000)let alone gene therapies. Yet if the most expensive cost to cure a rare disease is $2 million, thats still far less than $3 million for a lifetime of full-time care (which excludes additional expenses). And new studies, like one the World Economic Forum released in the lead up to this years International Rare Disease Day, show that we can ultimately save money in the long term by funding more treatments and, as an added benefit, potentially develop more cures by learning when treatments work and when treatments dont work.

We cant create clinical pathways for the more than 7,000 rare diseases overnight, but we need a standard of care that goes beyond using death as the primary barometer of focus, over-simplifies the complexity of what it means to be healthy, and only considers short term costs. An economically effective, new model could center on allocating resources with the end goal to allow people to reach a level of health that provides basic mobility or basic independencea level of health allowing economic productivity. We are living in a time of unprecedented medical innovation, and our system of coverage needs to catch up. We can do better than just keeping people alive.

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State advantageous to have an economically active citizen, than to keep disabled – The KXAN 36 News

In recent years in the world of medical genetics has been a real breakthrough. New medicines through which have learned to treat disease, cure which recently even anticipated. As far as the development of the Russian medical genetics corresponds to the global trends?

Sergey Kutsev: In the world, indeed, occurred very serious changes in the field of genetics. They happen literally before our eyes, and we are talking primarily about new approaches in the treatment of orphan genetic diseases. First of all there is to say about classical gene therapy. She has nearly 30 years, and the long development has led to the fact that, for example, a few years ago appeared the first drug for the treatment of a very rare hereditary disease (1 case per 1 million), which is characterized by increase in triglycerides in the blood. The disease is accompanied by acute pancreatitis and can lead to death. Unfortunately, not all of the goals the developers of the drug have been achieved, widespread it is not found, and the high cost of treatment was unjustified.

However, development of such drugs continued. The result is a drug that can be called a real breakthrough in the world of genetics, used for treatment of spinal muscular atrophy. From this disease the child dies, as a rule, in the second year of life. So today, a single administration of this drug leads to an almost complete cure of such a severe hereditary disease. Therefore, despite the high cost of treating one child more than $2 million the price of the drug is justified.

And now of course we see great prospects of this direction, because in addition to these drugs at present, there are already a number of advanced clinical trials for the treatment of the same gene-therapy methods and other hereditary orphan diseases, among which phenylketonuria, hemophilia, some hereditary diseases WSOena.

If we talk about the domestic developments, I would say that our pharmaceutical industry is now turning its attention to the problems of patients with orphan diseases, and we follow the global developments. With regard to diagnosis and early diagnosis very important for treatment of rare diseases, it in our country is organized on a global level. And the issue here is not only in the availability of genetic diagnosis, but also in the fact that in addition to conventional methods, such as molecular genetics, we use the latest technology of massive parallel sequencing (sequencing of the new generation). These technologies allow us to diagnose a wide range of hereditary diseases.

In April last year at the Federal level was approved by the scientific-technical program of development of genetic technologies. As far as its content corresponds, in your opinion, the needs of medical genetics in Russia?

Sergey Kutsev: the Program of development of genetic technologies is quite extensive. Very important for us section on the creation in Russia of centers of genomic research at the international level. To date, three such centres: one of them is engaged in Biosafety (this refers to infectious agents), the second genetic technology in agriculture, and the third genetic technologies in medicine. Under the program, as a basic guideline deals with the editing of the genome. This decision of the government we support with both hands, because it is aimed at fostering research in the field is very promising technology, the technology of genome editing. This is the next phase in medical genetics. It is how to get the cells directly from the patient to edit the genome, that is, to correct the defect that arose in these cells, and transplanting them back to the cells subsequently multiply and, accordingly, began to produce the necessarynecessary body substances. This approach is very promising, it relates to the gene-cell technology. And, of course, the development of these technologies it is necessary to invest now, because thats the future.

You have repeatedly said that the qualification of Russian geneticists are highly valued on a global level. At the same time, the personnel issue is one of the most serious in todays genetics in Russia. Suffice it to say, doctors-geneticists, who are involved in the identification of orphan diseases in the country, only about 350. How is this problem solved at the Federal level?

Sergey Kutsev: the Shortage of personnel in genetics in Russia is pretty decent. In some regions of the country there are only one to two doctors-genetics in the region. Frames for genetics to prepare necessary. Needed professionals, especially doctors-genetics, doctors genetics laboratory.

in addition, there is a problem in the education of doctors of all specialties neurologists, nephrologists, ophthalmologists, cardiologists and so on. In practice, any doctor can meet a genetic disease. Therefore, genetics, ranging from inheritance laws to the clinical manifestations of some diseases, doctors of different specialties just have to know. The fact that undergraduate education of doctors is paid to genetics now, few hours of study. On the other hand, specialists for teaching genetics in more than 50 medical universities in the country is also not enough.

What is the output?

Sergey Kutsev: In early February at the meeting of the academic Council approved my proposal to create a large scientific and educational centre on the basis of our Medico-genetic scientific center named after academician N. P.Bochkova. We planned for 2020, the establishment of more than 15 departments in our centre and already certain areas, among which neurogenetics, ophthalmic genetics, cardiogenetics and so on. These chairs will be required to take the advanced training of physicians of different SPEcelesta in the field of genetics. So now we are talking about postgraduate education simply because the urgent need to train people who are already in practice, who would now be able to work and to identify patients with hereditary diseases at an early stage. Of course, it is necessary to engage students, education at undergraduate level, but I think now we have to extinguish the fire. And gradually increase the share of education in genetics for students of all medical universities. For refresher training were going to have about 100 of geneticists who could teach. And of course, intend to look for partners among medical universities in the country.

what are the main directions currently your genetic center?

Sergey Kutsev: Our institution is primarily a scientific center, which is the study of the etiology, pathogenesis, development of diagnostic approaches, prevention hereditary, including orphan diseases. Despite the fact that we diagnose a disease, we can diagnose any of the hereditary diseases described in the world practice.

our Second destination medicine. Every year we take more than 11 thousand patients. And when you consider that advise usually the whole family, it is more than 20 thousand people per year. Speaking of laboratory studies, in the year we spend more than 70 thousand genetic research, and on this indicator are the largest centre in Europe. According to my calculations, about 90% of diagnoses of hereditary diseases in the country were set in the walls of our institution.

And third we focus on education. To us comes more and more residents and graduate students. Now, if you count in all the courses, internship and graduate school trained more than 50 people. Thats a lot. Im sure it will be a high level of doctors and scientists.

You have repeatedly talked about the need to expand screening to 41 nosology. What has been achieved in this direction today? How effective it can be in terms of preventing the development of illness and subsequent disability of the patient?

Sergey Kutsev: At the end of December last year, we almost agreed on a program of extending screening up to 39 diseases. Of course, it is difficult to expand screening to more than 1.65 million newborns per year. But this program will allow us to annually identify more than 2 thousand children, you can help. To make it clear those children who could die in the first year of life or during childhood, or become disabled. If we identify hereditary diseases in the first days of life, in most cases we can help them to avoid death and disability.

If we talk about pharmacoeconomics, the treatment of children in these cases are relatively inexpensive and can become a large financial burden for the state. The treatment of one such patient is estimated to be worth about 500 thousand rubles.

So even those who say that the treatment of orphan diseases is expensive and costly, I can assure you that the state advantageous to have an economically active citizen than many years, to include the disabled. This path is calculated and passed in many States. In addition, the screening in this case is one of the mechanisms capable of improving the demographic indicators of the country. We hope that an expanded screening across the country will be able to start in the last quarter of 2021.

the impression that in the last year or two orphan diseases are increasingly get into the focus of attention of both society and government. In particular, it greatly expanded the list of orphan diseases of the WNV program. How do you assess the prospects for federalization, and that more needs to be done to improve the situation with rare diseases in Russia?

Sergey Kutsev: Attention to orphan diseases from the state the actubut I grew up. First, there is more understanding that this is a great medical and economic problem that needs to be addressed. By the way, according to one study, about 40% of the seats in childrens hospitals today takes children with a serious hereditary, including orphan, diseases. And expansion of screening in this case is one of the tools to change these statistics for the better. Secondly, a social problem that society can not bear to look on the suffering of children, especially when there are ways to help children with serious illnesses to remain in society and thrive together with their peers.

In my opinion, the most important thing now is to improve the skills of physicians-geneticists, laboratory geneticists, and strengthen medical genetic service in all regions. This will help to put the correct diagnosis in patients with orphan diseases. Next, we need to address questions about the creation of domestic products, modern technological approaches, classical gene therapy to develop genome editing.

on the question of the extension of the list of high-cost nosologies (VZN) that is financed at the Federal level, I am sure that gradually we have to include all orphan diseases for which treatment today there are drugs. So today on the queue can be diseases such as disease, Niemann-pick, Fabry, and others. Federalization would not only reduce the financial burden on the regions, but also will allow to solve the problem of early diagnosis of orphan diseases, and therefore, the assistance provided and the patient and his family will become more effective. In the end, with timely start of treatment, patients with these diagnoses remain economically active, reduced social burden on the budget.

in addition, under this system, a much higher control over the correctness of appointments, expensive medications and monitoring their effectiveness. A fixed price for drugs under centralized procurement havedischarges are much lower than what we saw in procurement in the regions. I believe that the government will not be able to get away from this problem and definitely solve it. Theres no other way.

Photo: iStockRare optimized

one of last years sittings of the chamber of regions, the Chairman of the Federation Council Valentina Matvienko said that the purchase of medicines for treatment of orphan (rare) diseases will be funded from the Federal budget in 2021. There is the prospect that in 2020 we will close another five diseases and in 2021 will complete the transfer of funding for the procurement of medicines for people with orphan diseases in full at the Federal level, promised the Chairman of the Federation Council.

Since the formation of the list it was 7 nosologies. Last year, the list of WNV were added to the 5 diseases that have moved from the so-called List-24, containing a list of orphan diseases, drugs for the treatment of which can be purchased at the expense of regional budgets. The necessity of transferring diseases from the List of 24 at the Federal level due to the fact that regions do not always cope with the task timely and complete provide patients with appropriate drugs, there were difficulties and the threat of treatment interruption for orphan patients. According to the Executive Director of the Union of patients and patient organisations on rare diseases Denis Belyakov, despite the fact that the availability of therapy with the diagnosis from the regional list of 24 nosology in Russia enshrined in law, the situation with treatment is not very favourable: there were cases, when, for example, the patient diagnosed with Fabry prescribed therapy is needed, but the regional authorities have not always managed to Finance the provision of medication and treatment was interrupted.

today in the regional list there were 19 diseases, among which, besides the above-mentioned Fabry disease, Niemann-pick disease, arterial LeJonker hypertension, idiopathic thrombocytopenic purpura (ITP), phenylketonuria (PKU) and others. This is a severe genetic disease, leading to disability and often to death.

Experts say that the introduction of rare diseases in the WNV program will allow patients to get necessary drugs fully. In addition, the federalization of orphan diseases provides pricing transparency in the procurement of products and flexibility of a regional supply of medicines. It is very important that patients even with severe orphan diagnoses whose treatment begins in a timely manner, throughout their life are able to remain economically active members of society, is no different from a healthy person.

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Searching for the ‘big break’ that could turn stem cells into a weapon against dementia – Genetic Literacy Project

Recent developments in the field of stem cell research are paving a path towards a radical shift in the way we diagnose and treat dementia. Stem cells have excited scientists for years and research groups across the globe are using them to advance modern medicine. Using stem cells to aid the fight against dementia is perhaps one of the most critical applications of the technology. Dementia is the leading cause of death in the UK, sixth in US and fifth globally, with an estimated 50 million people currently affected.

The term dementia does not relate to a single disease, but more an array of symptoms that can arise from multiple conditions. The most common is Alzheimers disease (AD) which accounts for up to 80% of all cases. Dementia itself is caused by the death of cells that make up the complex circuitry of our brains and an eventual loss of large portions of the brain. Patients suffering with dementia often exhibit the same general symptoms such as confusion, memory loss and an inability to perform day to day functions. It is a debilitating condition that often strikes the most vulnerable members of society and, consequently, many research groups around the globe work to try to understand dementia-causing diseases to provide better diagnostic and treatment platforms.

In 2007, a research group at Kyoto University in Japan published a study with the potential to change the face of research into dementia along with many other fields. Professor Shinya Yamanaka and his research team developed a method whereby stem cells (cells that can be transformed/differentiated into cells from any tissue) could be generated from a sample of skin. The study, which resulted in a 2012 Nobel Prize for Prof. Yamanaka, demonstrated that skin cells could be isolated from a patient and genetically reprogrammed into induced pluripotent stem cells (iPSCs). In short, this technology made it possible to generate and study brain cells from a patient with dementia without having to remove any of their brain. All they would need to do is provide scientists with a sample of skin.

Since this development, research groups around the globe have started using iPSCs from many patients with dementia in order to understand the biological mechanisms that underlie disease. Dr Eric Hill runs a research group at Aston University in the UK that specializes in iPSCs for dementia research and he had the following to say about the technology:

Its really exciting because it allows us to study cells with genetic mutations that are patient specific. We can get a much better picture of what is actually happening in the brains of these patients. We can now generate all the different cell types found in the human brain and understand how they function together and map the changes that result in disease.

The latter was perhaps most powerfully demonstrated in a study published by a team at the University of North Carolina, led by Professor Hansang Cho. The team was able to generate three key cell subtypes that play important roles in brain function; study the impact of mutations associated with Alzheimers disease; and even replicate some of the core malfunctions found to trigger disease in the brains of patients.

Studies like this are of significance because a large part of the focus in dementia research is on trying to understand how such changes in function arise. When a patient is diagnosed with a disease such as Alzheimers it is often too late for effective treatment. Scientists, instead, seek to elucidate those early changes in brain cell function in order to diagnose patients earlier to give more time for treatment. It is very much a case of prevention being better than a cure. Dr Hill provided an encouraging statement regarding this:

When we generate brain cells from iPSCs the cells we get are developmentally very young. What is interesting is the fact we still see differences between cells from dementia patients versus healthy patients suggesting we could find markers to help us detect and prevent disease some years before it develops.

Despite such promise, however, iPSCs have yet to provide the field of dementia research with that big break. Multiple treatments have progressed into clinical trials since the technology first emerged but no therapies have been approved. Drugs that show promise in the lab fail to deliver on their potential in patient clinical trials, sending researchers back to square one.

We should not be disheartened by this, however, and should instead view it as space into which the technology of using iPSCs to study dementia can grow. A lot of drugs fail in clinical trials because the platforms used to run initial tests dont provide scientists with a wide enough perspective of how those drugs will influence human cells. Additionally, many preclinical studies use animals with dementia-causing disease artificially induced into them. Studies like this often fail to translate into humans because the initial data is not from a human perspective. This is where researchers like Dr. Hill think iPSCs can provide us with an advantage:

iPSCs could provide us with much better platforms for screening drugs to treat and prevent these diseases. They can really add to what we already have, and while we might not be able to grow a full human brain, we can generate the cells that provide the building blocks for one. They give us the chance to screen new therapies more efficiently, better test their effectiveness and reduce the amount of animal use in dementia research.

Dr Hill is not alone in seeing the promise of using iPSCs to find better treatments for preventing the progression of dementia. Multiple research groups around the world have shown the potential of iPSC-derived brain cells for studying the effectiveness of new therapies.

In the last 12 months we have observed a wave of new studies using iPSCs to try to develop better treatments for diseases like Alzheimers, Parkinsons, Huntingtons disease and ALS. From studies in the University of California identifying cholesterol metabolism as a potential target to treating Alzheimers to studies in Luxembourg helping us find better treatments for Parkinsons, it is easy to see why the global effort to get that big break from iPSCs continues to gain interest. We might still be waiting for that next Noble Prize-winning discovery that will improve the lives of millions of patients but the collective effort of iPSC research groups across the world brings us a step closer with every study they publish. Dementia may, one day, be a thing of the past and iPSC research will likely be a significant part in getting us there.

Sam Moxon has a PhD in regenerative medicine and is currently involved in dementia research. He is a freelance writer with an interest in the development of new technologies to diagnose and treat degenerative diseases. Follow him on Twitter @DrSamMoxon

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On the Road to 3-D Printed Organs – The Scientist

For years, scientists have predicted that 3-D printingwhich has been used it to make toys, homes, scientific tools and even a plastic bunny that contained a DNA code for its own replicationcould one day be harnessed to print live, human body parts to mitigate a shortage of donor organs. So far, researchers also used 3-D printing in medicine and dentistry to create dental implants, prosthetics, and models for surgeons to practice on before they make cuts on a patient. But many researchers have moved beyond printing with plastics and metalsprinting with cells that then form living human tissues.

No one has printed fully functional, transplantable human organs just yet, but scientists are getting closer, making pieces of tissue that can be used to test drugs and designing methods to overcome the challenges of recreating the bodys complex biology.

A confocal microscopy image showing 3-Dprinted stem cells differentiating into bone cells

The first 3-D printer was developed in the late 1980s. It could print small objects designed using computer-aided design (CAD) software. A design would be virtually sliced into layers only three-thousandths of a millimeter thick. Then, the printer would piece that design into the complete product.

There were two main strategies a printer might use to lay down the pattern: it could extrude a paste through a very fine tip, printing the design starting with the bottom layer and working upward with each layer being supported by the previous layers. Alternatively, it could start with a container filled with resin and use a pointed laser to solidify portions of that resin to create a solid object from the top down, which would be lifted and removed from the surrounding resin.

When it comes to printing cells and biomaterials to make replicas of body parts and organs, these same two strategies apply, but the ability to work with biological materials in this way has required input from cell biologists, engineers, developmental biologists, materials scientists, and others.

So far, scientists have printed mini organoids and microfluidics models of tissues, also known as organs on chips. Both have yielded practical and theoretical insights into the function of the human body. Some of these models are used by pharmaceutical companies to test drugs before moving on to animal studies and eventually clinical trials. One group, for example, printed cardiac cells on a chip and connected it to a bioreactor before using it to test the cardiac toxicity of a well-known cancer drug, doxorubicin. The team showed that the cells beating rate decreased dramatically after exposure to the drug.

However, scientists have yet to construct organs that truly replicate the myriad structural characteristics and functions of human tissues. There are a number of companies who are attempting to do things like 3-D print ears, and researchers have already reported transplanting 3-D printed ears onto children who had birth defects that left their ears underdeveloped, notes Robby Bowles, a bioengineer at the University of Utah. The ear transplants are, he says, kind of the first proof of concept of 3-D printing for medicine.

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Bowles adds that researchers are still a ways away from printing more-complex tissues and organs that can be transplanted into living organisms. But, for many scientists, thats precisely the goal. As of February 2020, more than 112,000 people in the US are waiting for an organ transplant, according to the United Network for Organ Sharing. About 20 of them die each day.

For many years, biological engineers have tried to build 3-D scaffolds that they could seed with stem cells that would eventually differentiate and grow into the shapes of organs, but to a large extent those techniques dont allow you to introduce kind of the organization of gradients and the patterning that is in the tissue, says Bowles. There is no control over where the cells go in that tissue. By contrast, 3-D printing enables researchers with to very precisely direct the placement of cellsa feat that could lead to better control over organ development.

Ideally, 3-D printed organs would be built from cells that a patients immune system could recognize as its own, to avoid immune rejection and the need for patients to take immunosuppressive drugs. Such organs could potentially be built from patient-specific induced pluripotent stem cells, but one challenge is getting the cells to differentiate into the subtype of mature cell thats needed to build a particular organ. The difficulty is kind of coming together and producing complex patternings of cells and biomaterials together to produce different functions of the different tissues and organs, says Bowles.

To imitate the patterns seen in vivo, scientists print cells into hydrogels or other environments with molecular signals and gradients designed to coax the cells into organizing themselves into lifelike organs. Scientists can use 3-D printing to build these hydrogels as well. With other techniques, the patterns achieved have typically been two-dimensional, Eben Alsberg, a bioengineer at the University of Illinois, tells The Scientist in an email. Three-dimensional bioprinting permits much more control over signal presentation in 3D.

So far, researchers have created patches of tissue that mimic portions of certain organs but havent managed to replicate the complexity or cell density of a full organ. But its possible that in some patients, even a patch would be an effective treatment. At the end of 2016, a company called Organovo announced the start of a program to develop 3-D printed liver tissue for human transplants after a study showed that transplanted patches of 3-D printed liver cells successfully engrafted in a mouse model of a genetic liver disease and boosted several biomarkers that suggested an improvement in liver function.

Only in the past few years have researchers started to make headway with one of the biggest challenges in printing 3-D organs: creating vasculature. After the patches were engrafted into the mouses liver in the Organovo study, blood was delivered to it by the surrounding liver tissue, but an entire organ would need to come prepared for blood flow.

For any cells to stay alive, [the organ] needs that blood supply, so it cant just be this huge chunk of tissue, says Courtney Gegg, a senior director of tissue engineering at Prellis Biologics, which makes and sells scaffolds to support 3-D printed tissue. Thats been recognized as one of the key issues.

Mark Skylar-Scott, a bioengineer at the Wyss Institute, says that the problem has held back tissue engineering for decades. But in 2018, Sbastian Uzel, Skylar-Scott, and a team at the Wyss Institute managed to 3-D print a tiny, beating heart ventricle complete with blood vessels. A few days after printing the tissue, Uzel says he came into the lab to find a piece of twitching tissue, which was both very terrifying and exciting.

For any cells to stay alive, [the organ] needs that blood supply, so it cant just be this huge chunk of tissue.

Courtney Gegg, Prellis Biologics

Instead of printing the veins in layers, the team used embedded printinga technique in which, instead of building from the bottom of a slide upwards, material is extruded directly into a bath, or matrix. This strategy, which allows the researchers to print free form in 3-D, says Skylar-Scott, rather having to print each layer one on top of the other to support the structure, is a more efficient way to print a vascular tree. The matrix in this case was the cellular material that made up the heart ventricle. A gelatin-like ink pushed these cells gently out of the way to create a network of channels. Once printing was finished, the combination was warmed up. This heat caused the cellular matrix to solidify, but the gelatin to liquify so it could then be rinsed out, leaving space for blood to flow through.

But that doesnt mean the problem is completely solved. The Wyss Institute teams ventricle had blood vessels, but not nearly as many as a full-sized heart. Gegg points out that to truly imitate human biology, an individual cell will have to be within 200 microns of your nearest blood supply. . . . Everything has to be very, very close. Thats far more intricate than what researchers have printed so far.

Due to hurdles with adding vasculature and many other challenges that still face 3-Dprinted tissues, laboratory-built organs wont be available for transplant anytime soon. In the meantime, 3-D printing portions of tissue is helping accelerate both basic and clinical research about the human body.

Emma Yasinski is a Florida-based freelance reporter. Follow her on Twitter@EmmaYas24.

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On the Road to 3-D Printed Organs - The Scientist

Recommendation and review posted by Bethany Smith

Global Autologous Stem Cell and Non-Stem Cell Based Therapies Market Provides An In-Depth Insight Of Sales Analysis -Regenexx, Genzyme – Fashion…

Global Autologous Stem Cell and Non-Stem Cell Based Therapies Market 2020-2025 Research Report is spread throughout 100+ pages and offers exclusive important statistics, informative data, key traits and competitive landscape details on this area of interest sector.

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Top Manufacturers Listed in the Atmospheric Water Generator Market Report are:

U.S. STEM CELL, INC.Brainstorm Cell TherapeuticsCytoriDendreon CorporationFibrocellLion BiotechnologiesCaladrius BiosciencesOpexa TherapeuticsOrgenesisRegenexxGenzymeAntriaRegeneusMesoblastPluristem Therapeutics IncTigenixMed cell EuropeHolostemMiltenyi Biotec

By Types:

Embryonic Stem CellResident Cardiac Stem CellsAdult Bone MarrowDerived Stem CellsUmbilical Cord Blood Stem Cells

By Applications:

Neurodegenerative DisordersAutoimmune DiseasesCancer and TumorsCardiovascular Diseases

Covering Region:

1. South America Backup Software Market Covers Colombia, Brazil, and Argentina.2. North America Backup Software Market Covers Canada, United States, and Mexico.3. Europe Backup Software Market Covers UK, France, Italy, Germany, and Russia.4. The Middle East and Africa Backup Software Market Covers UAE, Saudi Arabia, Egypt, Nigeria, and South Africa.5. Asia Pacific Backup Software Market Covers Korea, Japan, China, Southeast Asia, and India.

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Global Autologous Stem Cell and Non-Stem Cell Based Therapies Market Provides An In-Depth Insight Of Sales Analysis -Regenexx, Genzyme - Fashion...

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AgeX Therapeutics Researchers Publish Paper on the Age Reprogramming of Super-Centenarian Cells – Yahoo Finance

Cells of 114-year-old converted to young pluripotent stem cells

Evidence of reversal of the telomere aging clock in a supercentenarian

Supports hypothesis of no upper age limit for reprogramming cellular aging

Introduces possibility of identifying the underlying biology of extreme human lifespan and healthspan

AgeX Therapeutics, Inc. ("AgeX"; NYSE American: AGE), a biotechnology company focused on developing therapeutics for human aging and regeneration, announced a new paper co-authored by two AgeX scientists that could lead to new insights into the fundamental mechanisms of aging and why super-centenarians not only live the longest, but also experience extraordinary healthspans; an extension of the healthy years of life that compresses morbidity to a very short period near the end of life. The paper, "Induced pluripotency and spontaneous reversal of cellular aging in supercentenarian donor cells," is published online in the peer-reviewed scientific journal "Biochemical and Biophysical Research Communications" from Elsevier. The senior author is Dana Larocca, PhD, VP of Discovery Research at AgeX, and the first author is Jieun Lee, PhD, Scientist at AgeX.

"Clearly, we can learn a lot about aging and longevity from the longest of the long-lived, the supercentenarians, and we hope that this paper accelerates such research," commented Dr. Larocca. "Now that we have converted the cells of one of the longest-lived people in history, a deceased 114-year-old American woman, to a young pluripotent state, researchers can do so with cells from other supercentenarians. The goal is to understand specifically how these "extreme agers" manage to avoid the major chronic illnesses of aging better than any other age group including centenarians. We can essentially put their cells in a time machine and revert them to an earlier state, then study their biology to help unlock the mysteries of super-longevity. Scientists have long wondered, and now we know that we can indeed reset the developmental state and cellular age in the oldest of the old."

Story continues

By way of comparison, the paper also describes undertaking a similar process with cells from two other donors: an eight-year-old with a rapid-aging syndrome commonly known as Progeria, and a 43-year-old, healthy disease-free control (HDC) subject. The paper notes that the supercentenarians cells reverted to induced pluripotent stem (iPS) cells at the same rate as the HDC subject and the Progeria patient. However, there may be some negative impact of extreme age on telomere resetting as this did not occur as frequently in the supercentenarian as in the other two donors.

The donated cells were from "the longevity collection," a cell bank established by the NIHs National Institute on Aging.

About AgeX Therapeutics

AgeX Therapeutics, Inc. (NYSE American: AGE) is focused on developing and commercializing innovative therapeutics for human aging. Its PureStem and UniverCyte manufacturing and immunotolerance technologies are designed to work together to generate highly-defined, universal, allogeneic, off-the-shelf pluripotent stem cell-derived young cells of any type for application in a variety of diseases with a high unmet medical need. AgeX has two preclinical cell therapy programs: AGEX-VASC1 (vascular progenitor cells) for tissue ischemia and AGEX-BAT1 (brown fat cells) for Type II diabetes. AgeXs revolutionary longevity platform induced Tissue Regeneration (iTR) aims to unlock cellular immortality and regenerative capacity to reverse age-related changes within tissues. AGEX-iTR1547 is an iTR-based formulation in preclinical development. HyStem is AgeXs delivery technology to stably engraft PureStem cell therapies in the body. AgeX is developing its core product pipeline for use in the clinic to extend human healthspan and is seeking opportunities to establish licensing and collaboration agreements around its broad IP estate and proprietary technology platforms.

For more information, please visit http://www.agexinc.com or connect with the company on Twitter, LinkedIn, Facebook, and YouTube.

Forward-Looking Statements

Certain statements contained in this release are "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995. Any statements that are not historical fact including, but not limited to statements that contain words such as "will," "believes," "plans," "anticipates," "expects," "estimates" should also be considered forward-looking statements. Forward-looking statements involve risks and uncertainties. Actual results may differ materially from the results anticipated in these forward-looking statements and as such should be evaluated together with the many uncertainties that affect the business of AgeX Therapeutics, Inc. and its subsidiaries particularly those mentioned in the cautionary statements found in more detail in the "Risk Factors" section of AgeXs Annual Report on Form 10-K and Quarterly Reports on Form 10-Q filed with the Securities and Exchange Commissions (copies of which may be obtained at http://www.sec.gov). Subsequent events and developments may cause these forward-looking statements to change. AgeX specifically disclaims any obligation or intention to update or revise these forward-looking statements as a result of changed events or circumstances that occur after the date of this release, except as required by applicable law.

View source version on businesswire.com: https://www.businesswire.com/news/home/20200228005122/en/

Contacts

Media Contact for AgeX:

Bill Douglass Gotham Communications, LLCbill@gothamcomm.com (646) 504-0890

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AgeX Therapeutics Researchers Publish Paper on the Age Reprogramming of Super-Centenarian Cells - Yahoo Finance

Recommendation and review posted by Bethany Smith

Dont feed sickness – Times of India

When an animal, such as a dog or cat, is sick or unwell, its first natural instinct is to refuse food. Their body automatically cuts down appetite till they recover. They may drink water, but nothing else. This is bodys intelligence taking over to heal and recover.Intuitive eatingHumans arent any different. At least, intuitively. All of us are products of nature and we have been designed the same. When we call in sick, the first thing that happens is that our appetites shut down too. Our taste of mouth changes and our sensitivity to smell and taste is numbed. This is our bodys intelligence and subtle biofeedback trying to tell us that its time we give our body some rest.

This is fasting in a way and has been practised since the longest time across all religions. Fasting is inbuilt in us and is considered medicinal if you actually go back and read its history. It is our bodys uncomplicated way of cleaning and healing. Unfortunately, we have moved too far from nature and our natural instincts. We are constantly eating and munching even when sick, thereby compromising our own immunity and masking the inbuilt mechanism to heal.Take a break

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Dont feed sickness - Times of India

Recommendation and review posted by Bethany Smith

Global Stem Cell Market Poised for Strong Growth as Global Regenerative Medicine Market Poised to Reach US$45 billion by 2025 – P&T Community

DUBLIN, Feb. 27, 2020 /PRNewswire/ -- The "Stem Cell Banking - Market Analysis, Trends, and Forecasts" report has been added to ResearchAndMarkets.com's offering.

The growing interest in regenerative medicine which involves replacing, engineering or regenerating human cells, tissues or organs, will drive market growth of stem cells. Developments in stem cells bioprocessing are important and will be a key factor that will influence and help regenerative medicine research move into real-world clinical use. The impact of regenerative medicine on healthcare will be comparable to the impact of antibiotics, vaccines, and monoclonal antibodies in current clinical care. With the global regenerative medicine market poised to reach over US$45 billion by 2025, demand for stem cells will witness robust growth.

Another emerging application area for stem cells is in drug testing in the pharmaceutical field. New drugs in development can be safely, accurately, and effectively be tested on stem cells before commencing tests on animal and human models. Among the various types of stem cells, umbilical cord stem cells are growing in popularity as they are easy and safe to extract. After birth blood from the umbilical cord is extracted without posing risk either to the mother or the child. As compared to embryonic and fetal stem cells which are saddled with safety and ethical issues, umbilical cord is recovered postnatally and is today an inexpensive and valuable source of multipotent stem cells. Until now discarded as waste material, umbilical cord blood is today acknowledged as a valuable source of blood stem cells. The huge gap between newborns and available cord blood banks reveals huge untapped opportunity for developing and establishing a more effective banking system for making this type of stem cells viable for commercial scale production and supply. Umbilical cord and placenta contain haematopoietic blood stem cells (HSCs). These are the only cells capable of producing immune system cells (red cells, white cells and platelet).

HSCs are valuable in the treatment of blood diseases and successful bone marrow transplants. Also, unlike bone marrow stem cells, umbilical cord blood has the advantage of having 'off-the-shelf' uses as it requires no human leukocyte antigen (HLA) tissue matching. Developments in stem cell preservation will remain crucial for successful stem cell banking. Among the preservation technologies, cryopreservation remains popular. Development of additives for protecting cells from the stresses of freezing and thawing will also be important for the future of the market. The United States and Europe represent large markets worldwide with a combined share of 60.5% of the market. China ranks as the fastest growing market with a CAGR of 10.8% over the analysis period supported by the large and growing network of umbilical cord blood banks in the country. The Chinese government has, over the years, systematically nurtured the growth of umbilical cord blood (UCB) banks under the 'Developmental and Reproductive Research Initiation' program launched in 2008. Several hybrid public-private partnerships and favorable governmental licensing policies today are responsible for the current growth in this market.

Competitors identified in this market include:

Companies Mentioned

For more information about this report visit https://www.researchandmarkets.com/r/vgxw4q

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

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View original content:http://www.prnewswire.com/news-releases/global-stem-cell-market-poised-for-strong-growth-as-global-regenerative-medicine-market-poised-to-reach-us45-billion-by-2025-301012457.html

SOURCE Research and Markets

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Global Stem Cell Market Poised for Strong Growth as Global Regenerative Medicine Market Poised to Reach US$45 billion by 2025 - P&T Community

Recommendation and review posted by Bethany Smith

Jimbo Fisher’s Kidz1stFund, Aggie Corps of Cadets team up for Be the Match bone marrow donor registry drive – Bryan-College Station Eagle

A bone marrow donor registry drive on Texas A&M Universitys campus brought more than 400 students to Duncan Dining Hall on Friday.

Participants swabbed their mouths to provide DNA samples and have their names added to Be the Matchs national bone marrow registry, which will help people in need of bone marrow connect with donors. The event was a collaborative effort between the A&M Corps of Cadets, the Kidz1stFund and Be the Match.

If anyone is matched, Community Engagement Representative for Be the Match Gulf Coast Benita Davis said they will need to have additional blood work done before donating.

A&M senior and Cadet 1st Lt. Mitchell Moore said his attendance on Friday was motivated in part by his interest in the medical field, since he is aiming to go to medical school.

Its minimum to no risk for you and not too much time, Moore said about donating. A small time on your part can make a huge difference and extend someones life by years.

A&M football coach Jimbo Fisher and Candi Fisher started Kidz1stFund in 2011 to raise money for research about fanconi anemia, which is a rare blood disorder that their teenage son was diagnosed with. Fisher stopped by Fridays event to speak with organizers and meet participants.

Its amazing how many great people there are in this world who are willing to help other people, Fisher said. These young men and women out here are actually saving lives.

Davis said donating is not as painful as many people often think, especially since about 80% of donations can be made with the nonsurgical method of giving peripheral blood stem cells. The other 20% of donations involve marrow being removed from the hip while the donor is asleep under general anesthesia. According to the Be the Match website, about one in 430 people on the registry end up donating.

The cadets in attendance on Friday were a reflection of who they are as people and students, according to Amy Thompson, assistant commandant, marketing and communications for the Corps of Cadets.

Selfless service is an A&M core value its also a core value for the Corps of Cadets, Thompson said. The commandant really supports and encourages cadets to seek out opportunities to be leaders in selfless service. This is one of those opportunities where we can do that on a very large scale and make a huge impact.

Corps Squadron 1 commander and A&M senior Jacob Svetz donated using stem cells about two years ago. He said he thinks everyone should sign up for the registry.

The few pin pricks that you get Its such a miniscule amount of pain compared to what that family and individual are suffering through, Svetz said. To put yourself into a position to be able to help that for me, it doesnt make sense not to.

Be the Match On Campus President and A&M senior Paige Boone said the organization hosts drives every month. The next one will be March 25 at A&Ms Rudder Plaza. Home swab kits are also an option. To get started, visit join.bethematch.org or text CORPS to 61474. Anyone ages 18 to 44 can participate.

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Jimbo Fisher's Kidz1stFund, Aggie Corps of Cadets team up for Be the Match bone marrow donor registry drive - Bryan-College Station Eagle

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