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News Royal Oak native meets woman she saved with vital stem cell donation Jenn Schanz 11 – WXYZ

When you see Kelly Schneider and Alex Barr together, you'd think they go way back; they laugh at the same moments, seem to have inside jokes, and generally send off a vibe that they're old friends.

"Our families are both Middle Eastern, so we just have this connection. Weve just been gabbing and eating like we know each other," Schneider told Action News at her mother's house in Bloomfield Hills on Sunday.

It's where Barr and her mother came from the Boston area to meet Schneider and her family or the very first time in person, and to say thank you, since sharing something pretty personal back in August of 2018.

"I mean, she kind of is morphing into me now that she has my DNA. Thats how this works, right?" Schneider joked.

About a year earlier, in the summer of 2017 Barr, then in graduate school in the Boston area, learned she had Leukemia for the second time.

"It was just unreal. Like I couldnt even process it," Barr told Action News.

Barr didn't know it then, but Schneider had already signed up with Be The Match, and a national bone marrow registry, when she learned a close friend was diagnosed with cancer.

"We went and got tested and we donated blood. And unfortunately she did not survive. But after 4 or 5 months after she passed away, I got a call from Be The Match.

That call was on behalf of Barr, hoping Schneider might be willing to donate life-saving bone marrow.

Soon after, Schneider was getting treatment to donate stem cells from her bone marrow, to save Barr's life, who was still a stranger at the time.

All Schneider knew then was that her donation was going to help a 24-year-old from Michigan.

"How could you not? If someone needs it?" She said.

"When you hear bone marrow donation that sounds scary like theyre going to drill into your bone or something," Barr said, noting that it really wasn't as intense of a procedure as some people may think.

In this case, Schneider had to get a series of shots, the stem cells were collected, and then shipped to Boston for Barr, who is now in remission.

Its incredible. Like, I cant even describe. And I know that I would do the same," Barr said.

First, the two communicated communicated through the registry.

"We had been talking back and forth like online since September. I could tell that we would really hit it off," Barr told Action News.

Then, they decided to meet in person at Schneider's mother's house.

Not only do the two now share some of the same DNA, they keep finding other things they have in common.

Like a photo of Barr's cousin, which looks strikingly similar to Schneider.

I look like her! she said, pointing at the photo Barr brought with her.

For both Barr and Schneider, this full-circle experience is a reminder of how important the the Be The Match registry is, for the thousands of people waiting to find their life-saving donor, and just possibly, a life-long connection too.

Barr, who is now a healthy 26-year-old, is working in the health field. She works in the Hemostasis and Thrombosis Division at Beth Israel Deaconess Medical Center (BIDMC), hoping to help others who have been diagnosed with potentially terminal diseases.

Barr said her experience beating Leukemia inspired her to go into the medical field as a biologist to study diseases of the blood.

She is a currently also a volunteer with Be The Match, and conducts her own registry drives as living proof of how important the registry is and how bone marrow donations can save lives.

Click here to join the register or the learn more about the Be The Match.

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News Royal Oak native meets woman she saved with vital stem cell donation Jenn Schanz 11 - WXYZ

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Mixed ethnic backgrounds make it that much harder to find a bone marrow transplant – MLive.com

ANN ARBOR, MI They searched the world for a match.

Bennett Sevack needed a bone marrow transplant. He was diagnosed with myelodysplastic syndrome after treatment for chronic lymphocytic leukemia. But there was no match for the Ann Arbor resident in the international registry.

So, his family started making calls.

Doctors first tried his siblings and cousins. When that didnt work, his sisters, friends and family spent six weeks calling and sharing flyers with synagogues around the world to encourage people to join the bone marrow registry, hoping someone with Sevacks Sephardic and Ashkenazi Jewish background would be willing to donate.

By fall, a partial match was found in an Italian obstetrics hospital in an umbilical cord blood sample. Soon after, another cord blood match was found in the U.S. It was enough to perform a transplant.

Sevack has gained an intense appreciation for donors.

You can save somebodys life..." he said. Its a blessing to do.

Finding an acceptable bone marrow donor is a challenge in general, but its that much harder for people of color and patients of complex ethnic backgrounds like Sevack, according to health care providers and families whove embarked on far-reaching searches for a match.

A white person has a 77% shot at finding a matching donor, according to Be The Match, a widely used donor registry. A black persons chances are 23%. For people of mixed backgrounds, the search can become more complex.

Part of the issue is the relatively narrow population of donors in the registry. Another part is the science: Certain HLA types, the protein that needs to match in bone marrow transplants, are more unique to specific backgrounds.

A closer look at plans for the new $920M University of Michigan hospital

Sevacks transplant was completed in October 2019 more than three years after his initial diagnosis, and four months after getting news that he would definitely need a transplant.

He spent eight weeks healing from his transplant and two weeks healing from a bout of graft-versus-host disease in the hematology oncology clinic on the seventh floor of Ann Arbors C.S. Mott Childrens Hospital.

On the same floor, a 9-year-old Ann Arbor-area patient undergoing chemotherapy for acute myeloid leukemia is in the same predicament that Sevack once faced. She may need a bone marrow transplant as well, but a donor match her ethnic background is Cantonese, Hispanic and Caucasian has yet to be found in the registry.

The search

Genetics play a major role in finding a good match, said Mark Vander Lugt, a pediatric bone marrow transplant specialist at C.S. Mott working with the 9-year-old. People get four HLA types from each parent, meaning donors need to match in at least six spots, though sometimes a stronger match is required.

You get combinations that are hard to find in the registry because of that, Vander Lugt said.

Not everyone needs a transplant once theyre diagnosed. Doctors often treat blood cancers and related illnesses with chemotherapy first killing off the bad cells and waiting to see if healthy cells regenerate. If they dont, a transplant replaces the bone marrow, where blood stem cells are made.

Mothers breast cancer pushes Ypsilanti woman to get preventative mastectomy

The registry is so low on certain populations that some began taking matters into their own hands. Athena Asklipiadis founded Mixed Marrow, a nonprofit that conducts outreach to encourage multi-ethnic bone marrow and blood cell donations. She said the misconceptions of donation is one of the largest challenges in getting people registered. She started by reaching out to Facebook groups and college clubs on the West Coast, where she is based. Eventually, families began reaching out to her to help in the donor search.

People use this phrase, When theres a cure for cancer or If theres a cure for cancer, Asklipiadis said. But these are actual blood diseases and cancers that are curable. Theyre curable by the selflessness of a donor and thats the beautiful thing about it.

Sevacks sister Laurel Bernstein was part of the group of relatives and supporters who called synagogues in search of donors. She estimated they called hundreds of people and added more than 1,000 to the donor registry while searching for Sevacks match.

The nine of us all felt so energized by what happened, Bernstein said. "We had not finished calling the list, even though we got the match. We went through to the end of the list to help pay it forward, to help other people, to get that much more DNA into the database.

The transplant

For Sevack, it was umbilical cord blood that saved his life. DNA from the two samples created enough HLA type matches to begin the transplant.

Vander Lugt said the preferred transplant method is still donor transplants, because cord blood units are smaller and in more limited supply than donor cells.

Misconceptions of transplants are another roadblock. A bone marrow harvest in which a donor undergoes surgery to collect marrow from the hip is widely believed to be painful. But experts describe the process as a mild discomfort with a two-to-three day recovery time, instead.

And direct harvest is no longer the only method. Many hospitals also practice apheresis, which collects the peripheral blood that can generate new bone marrow through a process that looks like dialysis. Theres no surgery involved.

Choosing between the two collection processes is based on multiple factors, including the health of the patient undergoing general anesthesia and doctor and donor preference, Vander Lugt said.

The value

Fear of the unknown is what affected Sevack the most going into treatment. He said he had an anxiety breakdown the morning of his transplant, when he realized what was about to happen.

My heart was to the moon, Sevack said. I was fully dressed and every part of me was drenched. In about eight minutes I had gone through this journey of fear because the reality of what was going to come was upon me. (My wife) calmed me down, I changed my clothes and this journey began. Theres been plenty of times when Ive been scared to death.

Finding a transplant match taught his wife Phyllis Sevack the importance of facing each day separately. Fighting cancer was about healing every day making big battles smaller.

The reality of it hits all at once, Phyllis Sevack said. "I wouldnt want anybody to be afraid to go through it because it is every day take it day by day. And as soon as Bennett got over that hes not doing everything on one day, (he got better mentally).

Now at home, Bennett Sevack may take a year to fully heal. He looks forward to small improvements in his quality of life, like an expanded diet and doing his own grocery shopping at Meijer, he said.

Even the routine blood and platelet donations he received in treatment were invaluable to him. They saved his life.

Where to donate blood in Washtenaw County during shortage

Every day, theyd give me sacks of stuff that made me sick to think about it, Sevack said. It was someones donation that was given, that was literally giving me life. People who donate would do an amazing service to someone. You may not know who that is but its somebody.

Potential donors can begin the registration process by filling out a questionnaire about their medical history at bethematch.org. A cheek swab will confirm whether you meet the medical guidelines for donation. You may remain on the registry for years before a match is found. More information is available here.

Wolverines for Life and the Michigan Medicine Bone Marrow Transplant department are hosting a registration drive from 10:30 a.m. to 5 p.m. Tuesday, Feb. 18 at the Pierpoint Commons lobby, 2101 Bonisteel Blvd., Ann Arbor.

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Mixed ethnic backgrounds make it that much harder to find a bone marrow transplant - MLive.com

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FHSU partners with Be the Match for bone marrow registry event – hays Post

Brynn Niblock, FHSU junior in pre-med from Hoxie, swabs her cheek as part of the sign up for the Be the Match bone marrow registry Feb. 6 at Gross Memorial Coliseum.

By CRISTINA JANNEYHays Post

Usually Tiger basketball games are a time to have fun cheer the home team, eat some popcorn but students and community members at Feb. 6 game took a few minutes to stop and potentially save a life.

FHSU student health sponsored a Be the Match bone marrow registry drive.

Potential donors ages 18 to 44 answered a list of qualifying health questions on their smartphones and then swabbed their cheeks to be matched with a potential cancer sufferer in need of bone marrow transplants.

Kathy Pyke of Hays knows too the well the importance of the registry. Pyke was at Gross Memorial Coliseum the night of the drive as a volunteer handing out information to potential donors.

Her husband, Tom, was diagnosed with leukemia on March 1, 2014. Family members were tested, and they were not matches. Doctors were also unable to find a bone marrow match on the national registry. There were 6.2 million people in the registry at the time.

In lieu of a bone marrow transplant, Pyke was given donated umbilical cord blood.

Initially the treatment improved Pyke's condition. However, he ultimately died as a result of the disease on Feb. 12, 2015 at the age of 62.

Kathy said the family was rocked by Tom's illness. He was playing golf and went fishing the week before he was diagnosed with cancer.

Kathy said she wishes she could be on the registry to help another family, but her age prevents her from doing so.

"Not only for my husband," she said of the importance of the registry. "I did pray there had been a match. We stayed at the Hope Lodge that was run by the American Cancer Society in Kansas City. There were 45 apartments there and everyone there has someone who has cancer plus a caregiver in it. You just see so many lives being touched. ...

"If this is something that can help somebody, it is just an easy thing to do."

Kathy said she had a good friend who had a family member sign up for the registry, and he was able to donate to someone who had cancer in England.

Pyke said she would also like to see more hospitals participate in the cord blood bank, which is what helped her husband. At the time of Tom's illness, HaysMed was not participating in the umbilical cord blood bank.

Michelle Toogood, BSN, RN, supervisor of Hays Meds Women's andInfant Care Center/NICU, said parents wishing to participate in cord blood donation should initiate the process prior to delivery. HaysMed staff will then aid in the collection of the specimen.

"I just can't express how much people need to do this," Pyke said of signing up for the registry. "It is just so easy to swab test and they could potentially save more than one person's life. It is so easy to do and so important."

If you are identified as a match to someone suffering from cancer, you would be contacted through the registry and asked if you are willing to donate,Amanda McCord, RN at the FHSU student health center.

"Finding the perfect match is essential for people who are fighting this type of cancer," McCord said. "The closer the match the better their chances of remission and beating whatever cancer they are fighting."

There are over 70 diseases that can be treated by bone marrow transplants, according to Be the Match.

Physicians will usually look for matches among relatives first, but only 70 percent of the time are matches made from family members, McCord said.

Statistics also indicate minority patients are less likely to find matches than Caucasian patients. Be the Match is trying to boost minority participation as there are fewer minority participants in the registry at this time, McCord said.

Donating bone marrow is a little bit different for every donor, McCord said.

Most give through a Peripheral Blood Stem Cell (PBSC) donation. A machine draws blood from one arm, extracts the cells it needs, and returns the remaining blood through your other arm, according to the Be the Match website.

Others give through a marrow donation. Liquid marrow is withdrawn from the back of your pelvic bone with a needle. In this case, youll receive anesthesia and feel no pain during the procedure, the Be the Match website said.

According to Be the Match,PBSC donors may experience headaches or body aches several days before collection, but these disappear shortly after donation. Most donors feel completely recovered within a few weeks.

If you missed the Be the Match event at FHSU last week, you can contact Be the Match though its website, and the organization will send you the cheek swab kit to sign up for the registry.

The Be the Match website also has information on the donation process and a link to make monetary donations to the Be the Match program.

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FHSU partners with Be the Match for bone marrow registry event - hays Post

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8-year-old bone marrow recipient and donor celebrate in emotional reunion – WFAA.com

DALLAS It is more blessed to give than to receive. That's what a Bible verse says. And watching the smiles of a now healthy 8-year-old boy, that's what a recent bone marrow donor will gladly tell you too.

"I felt like the earth underneath my legs was pulled out and like I didn't know what to do," said Anitha Nagilla of Frisco of the 2017 chronic myeloid leukemia diagnosis for her then 6-year-old son Akshaj.

After chemotherapy and other treatments, he would need a bone marrow transplant to regenerate his immune system. But no one in his family, including his older sister, was a close enough match. Also, the likelihood of someone with Asian Indian heritage having a matched, available donor is only 41%.

The Nagilla family moved to north Texas from southern India 13 years ago.

WFAA

Every three minutes, someone is diagnosed with a blood cancer, like leukemia or lymphoma. For many patients, finding a bone marrow donor who is a match is their only chance for a cure. Finding a match can happen anywhere in the world at any time.

In nearby Colleyville, another immigrant, unknowingly, was about to help save the Akshaj's life.

WFAA

"Typical immigration story. Parents moved for a better life for their children and for themselves," said Dr. Prasanthi Ganesa, an oncologist who works at the Center for Cancer and Blood disorders in Fort Worth.

Dr. Ganesa moved to the U.S. as a 10-year-old child, also from southern India. She now works with adult cancer patients.

But as a medical student at Texas A&M, she and a group of friends decided to join the nationalBone Marrow Registry. The donation of blood, stem cells, and bone marrow can help people recover from a variety of cancer-related illnesses.

"At that time it was pretty simple," Dr. Ganesa said of joining the registry approximately 17 years ago. "You fill out some paperwork. It's a (saliva) swab and they have my information. So I've been on it, really not thinking about it, until I got the call. It was a surprise. It was a delightful surprise."

The surprise was that she was a match for a young boy, the same age as her own youngest child. After an outpatient procedure where marrow was extracted from a location on each hip, she went home, then back to work, and waited to hear what happened to whoever it was that received her donation.

Dr. Prasanthi Ganesa

Friday morning at Children's Medical Center in Dallas, she met him face to face.

In a meeting room all decorated for Valentine's Day, they celebrated what is also known as "National Donor Day."

"Hi buddy, how are you?" Dr. Ganesa said as she reached out to hug 8-year-old Akshaj Nagilla. "Oh my goodness," she said as they embraced.

"Definitely is on the list of one of my happiest proudest moments in my life," Dr. Ganesa said. "And Akshaj I have you to thank for that. You know what this means right? We're gonna have to be friends forever," she said as the audience of doctors, nurses, and family and friends laughed and applauded.

Being on the marrow registry is easy. All it takes is a swab of saliva. And minority donors are needed the most. Dr. Ganesa was one of two marrow donors for Akshaj. She supplied the first donation.

Akshaj initially recovered but relapsed in the fall of 2018, when he received a second transplant from a different donor, also of Asian Indian heritage. But his family credits the first donation with starting his long road to recovery.

"She being close here in Dallas is undoubtedly remarkable, in that it happened to our family," Anitha Nagilla said.

"She saved my son's life directly. But she saved some other lives as well in the family," Nagilla said of the impact on her entire extended family. "She is a lifesaver for others as well."

Friday morning at their first meeting, the Nagilla family presented Dr. Ganesa with a gold bracelet and a card with a personal message from Akshaj inside. "I'm doing good and wish you well always", he wrote.

"How wonderful is this," Dr. Ganesa said as they embraced again.

The usually shy 8-year-old also mustered up the courage to climb up to a podium and address the entire crowd, but mostly his message was for Dr. Ganesa.

"I am very thankful because I am still alive," he said.

"I just encourage everybody of Indian origin, of southeast Asian origin, any minority group to get yourself on the registry because it could save a life," Dr. Ganesa said.

"It feels really good to be able to say I gave a part of myself and I saved this person's life. I think it's the ultimate meaning of being a human being. We are here to love all serve all. And what an opportunity that I have had. So I am so grateful that I've had that opportunity," Dr. Ganesa said.

She says she feels like she received more than she gave. As she, her own two sons and the Nagilla family gathered for a group hug, her own extended family maybe just got a lot bigger too.

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8-year-old bone marrow recipient and donor celebrate in emotional reunion - WFAA.com

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Bride saves babys life by donating her bone marrow then meets her for the first time as a flower girl at he – The Sun

WHEN student Hayden Ryals saw a call for blood stem cell donors, she didn't think twice about signing up.

But she was shocked when she received a call just a year later, asking for her help to save a one-year-old girl's life.

6

Hayden, now 27, from Alabama, had the operation and instantly felt bonded to little Skye.

And two years later, the pair met for the first time, when Skye was invited to be a flower girl at Hayden's wedding.

Speaking exclusively to Fabulous Digital, Hayden tells her story...

Since I was about 16, I have always given blood. It seemed like such a small thing to do to help people. Donating only takes 10 minutes.

6

Then I went to University in Auburn, Alabama. I was walking to a lecture one day and saw a stall from Be the Match. I wandered over to see what they were about.

The lady at the stall told me they were looking for bone marrow donors. I just needed a swab in my cheek, so they would have my tissue, and then I would be put on the register. You only get called if you're a match.

I happily said yes, it only took two minutes, and didnt think much more about it.

Almost a year to the day later, in April 2016, I got a phone call. It was from Be the Match.

When I spoke to her mum Talia, it was emotional. 'Youve saved my daughters life,' she said through tears

"You have been matched with a one-year-old little girl," I was told.

At first I thought it was a mistake, surely I wouldnt be able to do something so big? But it was true, she needed me to save her life.

I had been feeling so down in life, wondering what I was supposed to be doing, I felt a surge of joy at being able to help someone.

6

I found out the little girl, Skye Savren-McCormick, wasdiagnosed withjuvenile myelomonocytic leukemia, a rare and aggressive form of blood cancer. She lived in California, miles away from me.

I immediately knew I wanted to help her.

I sent her parents a letter, through a co-ordinator, telling them I wouldnt back out.

They already felt like family to me. It was strange how connected I felt to a little girl I had never met.

6

In July 2016, we did the transfer. I went to a hospital three hours away in Birmingham Alabama and was put under general anaesthetic for an hour.

They put a hollow needle into my hip bone to extract my bone marrow. It really wasnt bad, and I didnt feel much pain afterwards.

I was told Skye responded well, but had to wait a year to contact Skye's parents.

When I eventually spoke to her mum Talia, it was emotional. "Youve saved my daughters life," she said through tears.

6

Around this time, I started dating my childhood friend Adrian Ryals, now 34. He was in the US Air Force, and was posted to Korea for a year in 2017.

Just before he left, Adrian popped the question. Delighted, we set a wedding date for June 2018.

In March 2018, I sent Skye a birthday present and asked if she would consider being my flower girl.

Becoming a blood stem cell donor

Every 20 minutes, someone is diagnosed with blood cancer in the UK.

For many, blood stem cell donation is their only chance of survival.

It's difficult to find a match, because of the millions of different stem cell combinations, so the DKMS are constantly on the look out for donors.

To become a blood stem cell donor, you just need to request a kit and do a five-minute cheek swab at home.

You can check your eligibility, and request a kit, here.

The odds are you may never be called upon.

Most blood stem cells are collected through a needle in the arm, just like giving blood.

Bone marrow collection involves stem cells being collected from the back of the hip under general anaesthetic. It takes one to two hours and most donors return to normal activities within a week. This method is only used in 10% of cases.

I knew it was a big ask, she lived a six-hour flight away, but she was so special to me.

If there was a chance she could come, I wanted her at my wedding.

Skye still lived in hospital at the time and had never flown. But in May 2018, the doctor gave her the green light to be able to come to the wedding.

6

Talia, Skye and her dad Todd flew down the Thursday before the wedding.

As we arrived at the church where we were having the rehearsal, they were already there waiting.

I was breathless and speechless to finally meet this little human, and know I was fortunate enough to have been able to help her.

I just ran up to her, got on my knees and gave her a big hug. She wasnt shy with me, as we had spoken on the phone a few times before.

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Skye didnt really understand what I had done for her, but she knew I was somebody special.

I was so honoured to have Skye be my flower girl, it made the wedding even more beautiful. I feel so lucky to have her in my life.

Earlier this week, we reported on a mother-of-the-bride who demanded her ginger bridesmaid dyed her hair for the wedding - because it "clashed" with her hair.

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Bride saves babys life by donating her bone marrow then meets her for the first time as a flower girl at he - The Sun

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Stem Cell Banking Market 2020-Global Leading Players, Industry Updates, Future Growth, Business Prospects, Forthcoming Developments and Future…

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Stem cells are cells which have self-renewing abilities and segregation into numerous cell lineages. Stem cells are found in all human beings from an early stage to the end stage. The stem cell banking process includes the storage of stem cells from different sources and they are being used for research and clinical purposes. The goal of stem cell banking is that if any persons tissue is badly damaged the stem cell therapy is the cure for that. Skin transplants, brain cell transplantations are some of the treatments which are cured by stem cell technique.

Explore Key Industry Insights In 60 Tables And 220 Figures From The 350 Pages Of Report, Global Stem Cell Banking MarketBy Source (Placental Stem Cells (PSCs), Human Embryo-Derived Stem Cells (hESCs), Bone Marrow-Derived Stem Cells (BMSCs), o Dental Pulp-Derived Stem Cells (DPSCS), Adipose Tissue-Derived Stem Cells (ADSCs) and Other Stem Cell Sources), By Application (Personalized Storage, Clinical, Research), Service Type (Sample Collection and Transportation, Sample Processing, Sample Analysis, Sample Preservation and Storage), Geography (North America, Europe, Asia Pacific, Latin America and The Middle East and Africa) Industry Trends and Forecast to 2026.

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Segmentation: Global Stem Cell Banking Market

By Source

By Application

Research

By Service Type

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The global Stem Cell Banking market is highly fragmented and the major players have used various strategies such as product (software) launches, agreements, joint ventures, partnerships, acquisitions, and others to increase their footprints in this market. The report includes market shares of Stem Cell Banking market for global, Europe, North America, Asia Pacific and South America.

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Research Methodology: Global Stem Cell Banking Market

Data collection and base year analysis is done using data collection modules with large sample sizes. The market data is analysed and forecasted using market statistical and coherent models. Also market share analysis and key trend analysis are the major success factors in the market report. To know more please request an analyst call or can drop down your enquiry.

The key research methodology used by DBMR research team is data triangulation which involves data mining, analysis of the impact of data variables on the market, and primary (industry expert) validation. Apart from this, other data models include Vendor Positioning Grid, Market Time Line Analysis, Market Overview and Guide, Company Positioning Grid, Company Market Share Analysis, Standards of Measurement, Top to Bottom Analysis and Vendor Share Analysis. To know more about the research methodology, drop in an inquiry to speak to our industry experts.

Customization of the Report

All segmentation provided above in this report is represented at country level.

All products covered in the market, product volume and average selling prices will be included as customizable options which may incur no or minimal additional cost (depends on customization)

Table of Content: Global Stem Cell Banking Market

Continued..

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Stem Cell Banking Market 2020-Global Leading Players, Industry Updates, Future Growth, Business Prospects, Forthcoming Developments and Future...

Recommendation and review posted by Bethany Smith

50 children diagnosed with cancer in Estonia every year – ERR News

The youngest children diagnosed with cancer are just one day old. The most frequent type of cancer diagnosed is leukemia, ETV's current affairs show "Aktuaalne kaamera" reported.

"Leukemia is a disease of hematopoietic stem cells; hematopoietic stem cells are the cells which derive from all the cells that circulate in our blood - red blood cells, thrombocytes, white blood cells. Every one of them has a function and if they're not present, they're not able to fill the function," Ain Kaare, Head of the Department of Hematology and Bone Marrow Transplantation in University of Tartu said.

Kaare said that what helps children to cope with cancers is hope and optimism, and they are able to enjoy it, when they are feeling good.

These courageous children make an effort over years in order to get better. Kaare noted that there are children in the clinic who have undergone hematopoietic stem cell transplantation three times in a row, which often adults don't survive.

"In my work office, I have a picture on a wall by a boy who was five at the time, where he has written that "doctor Kaare is my friend". In the picture, there was I with a bag in my hand and in that bag there were supposed to be the hematopoietic stem cells that we transplanted to him," Kaare said.

Kaili Lellep, president of the Estonian Association of Parents of Children with Cancer (Vhihaigete laste vanemate liit) said that a child with a cancer needs a supportive parent around all the time to comfort them and explain everything if necessary, and to get their thoughts away from their illness. Children with cancer often stay in hospital for months.

On Saturday, golden ribbons and the golden light on Tallinn's TV tower and at Tallinn's Children Hospital (Tallinna lastehaigla) sent a message to hospital wards and homes alike, that seriously ill children are not alone and are being thought of, hoping that tomorrow they will be better. The golden ribbon is well-known all over the world.

"This ribbon symbolises support for children with cancer and support for the suffering of children with cancer," Lellep said.

--

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Originally posted here:
50 children diagnosed with cancer in Estonia every year - ERR News

Recommendation and review posted by Bethany Smith

Cord Stem Cell banking Market 2020-2026 Booming With Healthy CAGR || Key Players Cryo-Save AG, Lifecell, StemCyte India Therapeutics Pvt. Ltd,…

Global Cord stem cell banking market is estimated to reach USD 13.8 billion by 2026 registering a healthy CAGR of 22.4%. The increasing number of parents storing their childs cord blood, acceptance of stem cell therapeutics, high applicability of stem cells are key driver to the market.

Request for FREE sample copy or PDF Herehttps://www.databridgemarketresearch.com/request-a-sample?dbmr=global-cord-stem-cell-banking-market&raksh

Few of the major market competitors currently working in the globalcord stem cell banking marketareCBR Systems, Inc., Cordlife, Cells4Life Group LLP, Cryo-Cell International, Inc., Cryo-Save AG, Lifecell, StemCyte India Therapeutics Pvt. Ltd, Viacord, SMART CELLS PLUS., Cryoviva India, Global Cord Blood Corporation, National Cord Blood Program, Vita 34, ReeLabs Pvt. Ltd., Regrow Biosciences Pvt. Ltd. , ACROBiosystems., Americord Registry LLC., New York Blood Center, Maze Cord Blood, GoodCell., AABB, Stem Cell Cryobank, New England Cryogenic Center, Inc. among others

The data and information included in this Global Cord Stem Cell banking business report helps businesses take sound decisions and plan about the advertising and sales promotion strategy more successfully. This Cord Stem Cell banking market research report is generated by taking into account a range of objectives of market research that are vital for the clients success. This report also includes strategic profiling of key players in the market, systematic analysis of their core competencies, and draws a competitive landscape for the Healthcare industry. The Global Cord Stem Cell banking business report includes market shares for global, Europe, North America, Asia Pacific and South America.

Market Definition: Global Cord Stem Cell Banking Market

Cord stem cells banking is nothing but the storing of the cord blood cell contained in the umbilical cord and placenta of a newborn child. This cord blood contains the stem cells which can be used in future to treat disease such as leukemia, thalassemia, autoimmune diseases, and inherited metabolic disorders, and few others.

Segmentation: Global Cord Stem Cell Banking Market

Cord Stem Cell banking Market : By Storage Type

Cord Stem Cell banking Market : By Product Type

Cord Stem Cell banking Market : By Service Type

Cord Stem Cell banking Market : By Indication

Cord Stem Cell banking Market : By Source

Cord Stem Cell banking Market : By Geography

Browse Detailed TOC, Tables, Figures, Charts and Companies @https://www.databridgemarketresearch.com/toc?dbmr=global-cord-stem-cell-banking-market&raksh

Key Developments in the Cord Stem Cell banking Market:

Cord Stem Cell banking Market : Drivers

Cord Stem Cell banking Market : Restraint

Competitive Analysis: Global Cord Stem Cell Banking Market

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

Key benefits of buying the Cord Stem Cell banking Market Report:

This Cord Stem Cell banking Market report will enable both of the sides in market be an established firm or a relative new entrant. It helps the established firms to know about the moves which are being performed by their competitors and also helps the new entrants by educating them about the market situations and the industry trends. This Cord Stem Cell banking Market report is quite fruitful in helping to understand the market definition and all the aspects of the market including the CAGR value and key profiles.

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Cord Stem Cell banking Market 2020-2026 Booming With Healthy CAGR || Key Players Cryo-Save AG, Lifecell, StemCyte India Therapeutics Pvt. Ltd,...

Recommendation and review posted by Bethany Smith

Power Moves: Bobbie Knight taking helm at Miles College is the latest in a lifetime of leadership – Yellowhammer News

Becoming president ofMiles College the first female chief executive in the schools 122-year history wasnt part of Bobbie Knights retirement plan.

After 37 years with Alabama Power, where she held several leadership positions, including vice president of Public Relations and vice president of the companys Birmingham Division, Knight wasnt in the market for a new, full-time job.

Indeed, Knight had plenty going on even after her 2016 retirement from the power company.

In 2017, she was elected to Miles board of trustees and co-chaired newly elected Birmingham Mayor Randall Woodfins transition team. Then, in 2018, she was appointed to the Birmingham Airport Authority, where her colleagues immediately elected her chair. She also had her own consulting company, not to mention other, ongoing volunteer civic obligations.

But when longtime Miles President George French announced last year that he was leaving to become president of Clark Atlanta University, the Miles board of trustees quickly turned to Knight to serve as interim president of the 1,700-student college in Fairfield near Birmingham.

I was absolutely floored, Knight said.

I deliberated long and hard after I got over the initial shock of being asked to consider this opportunity and I have continuously prayed for the wisdom, strength and courage it will take to lead this institution with integrity, compassion and a servants heart, Knight said during a press conference announcing her appointment.

Bobbie Knight shares her plans for Miles College from Alabama NewsCenter on Vimeo.

During this transition, the job before me is clear; first, to serve the students of Miles College by ensuring they receive a quality education, that they are equipped with the tools they need to be successful here and in the future and that they enjoy a safe and fulfilling campus life. Second, my job is to maintain a fiscally sound institution. I have a business background and my plan is to use business principles and practices to keep this institution financially strong.

It didnt take long for Knight to make a mark.

In January, Miles announced it had received its single largest contribution from an individual donor in school history $1 million.

The donation came from a celebrity more often associated with another Alabama institute of higher learning: Charles Barkley, the former Auburn University and NBA basketball great and television commentator.

Barkley singled out Knight in his comments about the donation. Ive gotten to know Bobbie Knight over the last year and it was really something I wanted to do, Barkley said in a statement. To have a female president is a big deal and I want to help Bobbie be as successful as she can be.

Knight said that even though Barkley didnt attend Miles or any other historically black college or university, he understands how vitally important HBCUs have been in this country.

Barkleys donation drew national attention, and Knight hoped it would set the stage for more contributions as Miles embarked on a $100 million fundraising campaign. Before the month was over, the school announced it had received a $50,000 contribution to its football program from Tampa Bay Buccaneers quarterback and Hueytown native Jameis Winston.

Having someone of Jameis stature selflessly contribute to our growth here at Miles gives credence to what we are trying to accomplish, which is to give our student-athletes the best collegiate experience possible, Knight said in a news release.

That Barkley cited his relationship with Knight in making his donation is hardly the first time Knight has been recognized for her skills and for making a difference.

Knight grew up in the Birmingham neighborhood of Zion City, one of five children. Her mother worked as a pastry chef in the long-closed Pizitz department store bakery. Her dad was an inspector atStockham Valves and Fittings, at that time an important member of Birminghams heavy industrial sector. He passed away when Knight was 14.

Bobbie truly comes from humble means, said Robert Holmes, a retired Alabama Power executive and longtime civic leader who serves as vice chair of the Samford University board of trustees. Holmes watched Knight rise through the company ranks, starting with an evening shift in customer service and moving through positions of increasing importance.

She has an unparalleled work ethic, Holmes said, noting how Knight went back to school to get a law degree while working full-time.

After becoming a vice president at the power company, Knight was chosen among 21 women worldwide for the annual Leadership Foundation Fellows Program of theInternational Womens Forum. The exclusive fellowship for female executives included study at Harvard University and the Judge School of Business at Cambridge University in England.

Knight has been honored with numerous other accolades through the years, including Outstanding Alumni in Public Relations by the University of Alabama School of Communications and recipient of the Womens History Award from the Birmingham Chapter of the NAACP.

She has served on numerous civic and nonprofit boards, including Red Mountain Theatre, VOICES for Alabamas Children, the Alabama Literacy Council and United Way of Central Alabama. She helped to create Birminghams Railroad Park as a member of its founding board and served as chair of the board of the Birmingham Civil Rights Institute.

When Bobbie gets engaged in projects, she gets engaged, said Norm Davis, a retired financial services executive who has known Knight for 25 years.

Bobbie is very strategic in her thinking and her actions, said Davis, who was working with French on plans for Miles fundraising campaign when French announced his move to Atlanta.

Shes just done everything right, he said about Knights new role as college president. Shes one of those people that, when she sees something where she can make a difference, she is always willing to roll up her sleeves and go to work.

He recalls observing Knight on a scalding summer afternoon, watching practice for the Miles marching band. She is all over the campus, engaging the kids. She is working on strengthening the graduation rate, recruiting students, building relationships.

She continues to build the community, Davis added, noting that he and Knight both believe a vibrant Miles College can serve as an economic engine in Fairfield and for western Jefferson County.

I think we have the opportunity to make a huge difference in this region. Thats what I see, Knight said.

She is going to leave Miles better than how she found it, Holmes said, citing Knights passion for the community that raised her.

Bobbie wants to give back to the city, and the county and the state, from where weve both gotten so much from, said Holmes, also a Birmingham native. She is a living example of what one can do.

Power Moves, an ongoing series by Alabama NewsCenter, celebrates the contributions of multicultural leaders in Alabama. VisitAlabamaNewsCenter.comthroughout the year for inspiring stories of those working to elevate the state.

(Courtesy of Alabama NewsCenter)

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Power Moves: Bobbie Knight taking helm at Miles College is the latest in a lifetime of leadership - Yellowhammer News

Recommendation and review posted by Bethany Smith

Diverse businesses ‘critical’ to success of Birmingham – Yellowhammer News

In 2017, she was elected to Miles board of trustees and co-chaired newly elected Birmingham Mayor Randall Woodfins transition team. Then, in 2018, she was appointed to the Birmingham Airport Authority, where her colleagues immediately elected her chair. She also had her own consulting company, not to mention other, ongoing volunteer civic obligations.

But when longtime Miles President George French announced last year that he was leaving to become president of Clark Atlanta University, the Miles board of trustees quickly turned to Knight to serve as interim president of the 1,700-student college in Fairfield near Birmingham.

I was absolutely floored, Knight said.

I deliberated long and hard after I got over the initial shock of being asked to consider this opportunity and I have continuously prayed for the wisdom, strength and courage it will take to lead this institution with integrity, compassion and a servants heart, Knight said during a press conference announcing her appointment.

Bobbie Knight shares her plans for Miles College from Alabama NewsCenter on Vimeo.

During this transition, the job before me is clear; first, to serve the students of Miles College by ensuring they receive a quality education, that they are equipped with the tools they need to be successful here and in the future and that they enjoy a safe and fulfilling campus life. Second, my job is to maintain a fiscally sound institution. I have a business background and my plan is to use business principles and practices to keep this institution financially strong.

It didnt take long for Knight to make a mark.

In January, Miles announced it had received its single largest contribution from an individual donor in school history $1 million.

The donation came from a celebrity more often associated with another Alabama institute of higher learning: Charles Barkley, the former Auburn University and NBA basketball great and television commentator.

Barkley singled out Knight in his comments about the donation. Ive gotten to know Bobbie Knight over the last year and it was really something I wanted to do, Barkley said in a statement. To have a female president is a big deal and I want to help Bobbie be as successful as she can be.

Knight said that even though Barkley didnt attend Miles or any other historically black college or university, he understands how vitally important HBCUs have been in this country.

Barkleys donation drew national attention, and Knight hoped it would set the stage for more contributions as Miles embarked on a $100 million fundraising campaign. Before the month was over, the school announced it had received a $50,000 contribution to its football program from Tampa Bay Buccaneers quarterback and Hueytown native Jameis Winston.

Having someone of Jameis stature selflessly contribute to our growth here at Miles gives credence to what we are trying to accomplish, which is to give our student-athletes the best collegiate experience possible, Knight said in a news release.

That Barkley cited his relationship with Knight in making his donation is hardly the first time Knight has been recognized for her skills and for making a difference.

Knight grew up in the Birmingham neighborhood of Zion City, one of five children. Her mother worked as a pastry chef in the long-closed Pizitz department store bakery. Her dad was an inspector atStockham Valves and Fittings, at that time an important member of Birminghams heavy industrial sector. He passed away when Knight was 14.

Bobbie truly comes from humble means, said Robert Holmes, a retired Alabama Power executive and longtime civic leader who serves as vice chair of the Samford University board of trustees. Holmes watched Knight rise through the company ranks, starting with an evening shift in customer service and moving through positions of increasing importance.

She has an unparalleled work ethic, Holmes said, noting how Knight went back to school to get a law degree while working full-time.

After becoming a vice president at the power company, Knight was chosen among 21 women worldwide for the annual Leadership Foundation Fellows Program of theInternational Womens Forum. The exclusive fellowship for female executives included study at Harvard University and the Judge School of Business at Cambridge University in England.

Knight has been honored with numerous other accolades through the years, including Outstanding Alumni in Public Relations by the University of Alabama School of Communications and recipient of the Womens History Award from the Birmingham Chapter of the NAACP.

She has served on numerous civic and nonprofit boards, including Red Mountain Theatre, VOICES for Alabamas Children, the Alabama Literacy Council and United Way of Central Alabama. She helped to create Birminghams Railroad Park as a member of its founding board and served as chair of the board of the Birmingham Civil Rights Institute.

When Bobbie gets engaged in projects, she gets engaged, said Norm Davis, a retired financial services executive who has known Knight for 25 years.

Bobbie is very strategic in her thinking and her actions, said Davis, who was working with French on plans for Miles fundraising campaign when French announced his move to Atlanta.

Shes just done everything right, he said about Knights new role as college president. Shes one of those people that, when she sees something where she can make a difference, she is always willing to roll up her sleeves and go to work.

He recalls observing Knight on a scalding summer afternoon, watching practice for the Miles marching band. She is all over the campus, engaging the kids. She is working on strengthening the graduation rate, recruiting students, building relationships.

She continues to build the community, Davis added, noting that he and Knight both believe a vibrant Miles College can serve as an economic engine in Fairfield and for western Jefferson County.

I think we have the opportunity to make a huge difference in this region. Thats what I see, Knight said.

She is going to leave Miles better than how she found it, Holmes said, citing Knights passion for the community that raised her.

Bobbie wants to give back to the city, and the county and the state, from where weve both gotten so much from, said Holmes, also a Birmingham native. She is a living example of what one can do.

Power Moves, an ongoing series by Alabama NewsCenter, celebrates the contributions of multicultural leaders in Alabama. VisitAlabamaNewsCenter.comthroughout the year for inspiring stories of those working to elevate the state.

(Courtesy of Alabama NewsCenter)

More here:
Diverse businesses 'critical' to success of Birmingham - Yellowhammer News

Recommendation and review posted by Bethany Smith

A Decade Of Change: How Tech Evolved In The 2010s And What’s In Store For The 2020s – Seeking Alpha

Significant technological advancements and societal shifts occurred during the 2010's decade. Yet many of these developments became so quickly engrained in our daily lives that they often went relatively unnoticed, and their impact all but forgotten. Over this next decade, the 2020s, we expect similar rapid and meaningful advancements to occur. Moore's law suggests that over a 10-year period, semiconductors will advance by 32 times, bringing about mesmerizing innovation in the digital age that should not only change technology but society as well. In this piece, we review the technological advancements over the last decade and anticipate what revolutionary changes may be in store for us over the next 10 years.

Mobile upgraded from 3G to 4G networks

Social Media Brought Our Lives Online

The Dawn of Genomics and Precision Medicine

Electric vehicles (EVs) became competitive with internal combustion engine (ICE) vehicles

AI and Big Data Took Off

Data storage moved to the cloud

5G becomes the new wireless standard

5G's means more than just a faster internet. It proliferates the mass-adoption of connected devices in homes, cities and enterprises, including those involved in agriculture, healthcare, manufacturing, and infrastructure. Increasingly, these industries rely on connected devices to gather and analyze data, making processes more efficient, reducing downtime, and freeing up time to focus on new products and initiatives.

The continued decline of computing costs and improvements in network infrastructure and artificial intelligence are key drivers of the emergence of edge computing - data processing conducted locally without transmission across a large network. Some technologies require instantaneous data interpretation and can't rely on networks with higher latency. Autonomous vehicles, robotic surgery, gaming, and smart factories, for example, can require near simultaneous data transmission. Edge computing reduces latency to 5-10 milliseconds from 25-35 milliseconds for early deployed 5G devices.10 A study estimates the economic impact of edge computing to reach $4.1 trillion by 2030.11

Transportation goes electric, autonomous, and shared

Shared autonomous and electric vehicles are expected to cost $0.35 per mile by 2030, less than half the cost of owning a personal sedan ($0.88 per mile).12 By 2030, new car models equipped with self-driving technology are expected to be commonplace. This transition will allow drivers to take their hands off the wheel while their vehicles drive autonomously, or to hail driverless shared robotaxis. Considering that the average American spends 18 days' worth of time driving each year, greater than the average number of vacation days per year (10 days), the productivity and leisure benefits of autonomous vehicles could be enormous.

Robotics enters new industries

Improvements in artificial intelligence and dexterity are the result of advanced 3D vision capabilities and end-of-arm tooling for precise movements. While the auto manufacturing segment remains robotics' largest end-market, emerging segments like healthcare and hospitality can benefit from smarter, more capable robots that can conduct surgery, check on patients, cook food, or deliver items to hotel rooms. Agriculture will also see a robotics-driven revolution with automated spraying, monitoring, and picking likely resulting in reduced costs and improved crop yields. The global stock of industrial robots is expected to reach 20 million by 2030, representing a 24% CAGR as robots enter more industries.13

Omnichannel-commerce: Not a zero-sum game between e-commerce and brick-and-mortar

The separation between e-commerce and brick and mortar retail will blur as digital and physical shopping experiences become one in the same. Augmented reality will give consumers the ability to try on and order clothes from the comfort of their homes, but physical stores with reduced footprints will feature unique consumer experiences, immediate purchases, and try-before-you-buy optionality. Coupled with IoT technologies that track consumer behavior and build datasets around it, and advances in AI, retailers could be able to predict consumer behavior at almost 100% accuracy. We also expect to see e-commerce as a value add for small- and mid-sized businesses, providing a medium for them to sell products outside of their locality and giving a means of accessing global consumer-bases.

As the population ages, there will be increasing pressure to reduce costs, improve patient outcomes, and optimize physicians' time. All of this is possible by leveraging telemedicine and artificial intelligence - two technologies that are set to disrupt healthcare. Telemedicine offers an alternative solution to in-person doctor visits, providing diagnostic and clinical services to patients around-the-clock, from the convenience of one's home or workplace. Currently, telemedicine utilization rates remain below 10% in the U.S., but penetration is likely to grow as technology reduces costs and adds convenience.14 Further digitalization of healthcare will also enable new use cases for AI. Recent developments like AI-driven, real-time MRI interpretation could mean enhanced diagnoses and treatments across other healthcare verticals, in addition to its use in telemedicine as an AI chatbot for triage or basic medical advice.

Advances in genomics could offer the first realistic opportunity to cure certain illnesses previously deemed fatal. Scientists have identified more than 50,000 genetic diseases caused by a single gene mutation in humans, known as Mendelian diseases. These are likely to be the first diseases treated through gene editing techniques.15 As the medical and scientific communities' understanding of gene expression and protein pathways improves and genomic sequencing costs continue to fall, we expect a vast increase in the number of tested and proven genomic therapies.

The 2010s were a decade of phenomenal innovation, led largely by the transition to mobile and the rise of data, which accelerated the growth of AI, e-commerce, social media, and biotechnology. In the 2020s, additional foundational changes will take place as data latency shortens and AI algorithms improve. Such progress should enable new technologies to flourish, including autonomous vehicles, conversational AI, the massive internet of things, and augmented and virtual reality. As new technologies are introduced to the market place, they will have a profound impact on our lives, workplaces, and even investments as they reshape the economy.

Related ETFs

BOTZ: The Global X Robotics & Artificial Intelligence ETF (NASDAQ:BOTZ) seeks to invest in companies that potentially stand to benefit from increased adoption and utilization of robotics and artificial intelligence (AI), including those involved with industrial robotics and automation, non-industrial robots, and autonomous vehicles.

SNSR: The Global X Internet of Things ETF (NASDAQ:SNSR) enables investors to access a potential high growth theme through companies at the leading edge of IoT, an approach which transcends classic sector, industry and geographic regions to target this emerging theme. In a single trade, SNSR delivers access to dozens of companies with high exposure to emerging IoT technology.

CLOU: The Global X Cloud Computing ETF (NASDAQ:CLOU) seeks to invest in companies positioned to benefit from the increased adoption of cloud computing technology, including companies whose principal business is in offering computing Software-as-a-Service (SaaS), Platform-as-a-Service (PaaS), Infrastructure-as-a-Service (IaaS), managed server storage space and data center real estate investment trusts, and/or cloud and edge computing infrastructure and hardware.

AIQ: The Global X Future Analytics Tech ETF (NASDAQ:AIQ) seeks to invest in companies that potentially stand to benefit from the further development and utilization of artificial intelligence (AI) technology in their products and services, as well as in companies that provide hardware facilitating the use of AI for the analysis of big data.

DRIV: The Global X Autonomous & Electric Vehicles ETF (NASDAQ:DRIV) seeks to invest in companies involved in the development of autonomous vehicle technology, electric vehicles ("EVs"), and EV components and materials. This includes companies involved in the development of autonomous vehicle software and hardware, as well as companies that produce EVs, EV components such as lithium batteries, and critical EV materials such as lithium and cobalt.

GNOM: The Global X Genomics & Biotechnology ETF (NASDAQ:GNOM) seeks to invest in companies that potentially stand to benefit from further advances in the field of genomic science, such as companies involved in gene editing, genomic sequencing, genetic medicine/therapy, computational genomics, and biotechnology.

SOCL: The Global X Social Media ETF (NASDAQ:SOCL) seeks to provide investment results that correspond generally to the price and yield performance, before fees and expenses, of the Solactive Social Media Total Return Index.

Footnotes

1. Ken'sTechTips, "Download Speeds: What Do 2G, 3G, 4G & 5G Actually Mean?," Nov 23, 2018.

2. Vox, "Digital advertising in the US is finally bigger than print and television," Feb 20, 2019.

3. Statista, "Number of global social network users 2010-2021," Aug 14, 2019.

4. Note: Estimates from Statista correspond to yearly averages (Statista publishes monthly or quarterly figures). Estimates from TNW correspond to the first figure provided for the corresponding year (TNW published an animation with interpolated data). Estimates for Facebook, Twitter, Instagram, WhatsApp, and Pinterest are taken from Statista. All other series come from TNW. TNW does not provide details regarding their underlying sources but our analysis suggests their estimates are consistent with primary sources and reports such as company earnings press releases, official company websites, and published articles. Statista uses monthly active users to measure social media platform usage. Statista specifies "Facebook measures monthly active users (MAUs) as users that have logged in during the past 30 days. Users are counted separately for Facebook and other apps. Statista specifies "Figures do not include Instagram or WhatsApp users unless they would otherwise qualify as such users, respectively, based on their other activities on Facebook."

5. Bloomberg, "The CRISPR revolution & hyper-personalized medicine," Jan 15, 2018.

6. BloombergNEF, "A Behind the Scenes Take on Lithium-ion Battery Prices," Mar 5, 2019.

7. Global X, "What's Driving the Electric Vehicle, Lithium, and Battery Markets in 2019?," May 21, 2019.

8. Fenner, "Alibaba's AI Outguns Humans in Reading Test

9. Bondcap, "Internet Trends 2019," Jun 11, 2019.

10. Financier Worldwide, "The emergence of edge computing," Dec 2019.

11. Chetan Sharma Consulting, "The Edge Internet Economy Forecast to be Worth Over $4.1 Trillion," Aug 21, 2019.

12. Rocky Mountain Institute, "Peak Car Ownership," 2016.

13. Oxford Economics, "How Robots Change the World," Jun 2019.

14. Teladoc, "Investor Presentation: Canaccord Growth Conference," Aug 8, 2019.

15. WIRED, "Gene Editing is Trickier Than Expected-but Fixes Are in Sight," Feb 28, 2019.

Investing involves risk, including the possible loss of principal. There is no guarantee the strategies discussed will be successful. International investments may involve risk of capital loss from unfavorable fluctuation in currency values, from differences in generally accepted accounting principles or from economic or political instability in other nations. Emerging markets involve heightened risks related to the same factors as well as increased volatility and lower trading volume. Narrowly focused investments may be subject to higher volatility. There are additional risks associated with investing in lithium and the lithium mining industry. The investable universe for thematic ETFs may be limited. The funds are non-diversified.

Healthcare, Pharmaceutical, Biotechnology and Medical Device companies can be affected by government regulations, expiring patents, rapid product obsolescence, and intense industry competition. The risks related to investing in cloud computing companies include disruption in service caused by hardware or software failure, interruptions or delays in service by third-party data center hosting facilities and maintenance providers, security breaches involving certain private, sensitive, proprietary and confidential information managed and transmitted by cloud computing companies, and privacy concerns and laws, evolving Internet regulation and other foreign or domestic regulations that may limit or otherwise affect the operations of such companies.

Investing in securities engaged in the social media industry include disruption in service caused by hardware or software failure; interruptions or delays in service by third-parties; security breaches involving certain private, sensitive, proprietary and confidential information managed and transmitted by social media companies; and privacy concerns and laws, evolving Internet regulation and other foreign or domestic regulations that may limit or otherwise affect the operations of such companies.

Information Technology companies can be affected by rapid product obsolescence, and intense industry competition. Risks include disruption in service caused by hardware or software failure; interruptions or delays in service by third-parties; security breaches involving certain private, sensitive, proprietary and confidential information managed and transmitted; and privacy concerns and laws, evolving Internet regulation and other foreign or domestic regulations that may limit or otherwise affect the operations.

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Editor's Note: The summary bullets for this article were chosen by Seeking Alpha editors.

Originally posted here:
A Decade Of Change: How Tech Evolved In The 2010s And What's In Store For The 2020s - Seeking Alpha

Recommendation and review posted by Bethany Smith

Surge in the Adoption of Stem Cell-Derived Cells to Fuel the Growth of the Stem Cell-Derived Cells Market Through the Assessment Period 2019 2029 -…

The comprehensive report published by Persistence Market Research offers an in-depth intelligence related to the various factors that are likely to impact the demand, revenue generation, and sales of the Stem Cell-Derived Cells Market. In addition, the report singles out the different parameters that are expected to influence the overall dynamics of the Stem Cell-Derived Cells Market during the forecast period 2019 2029.

As per the findings of the presented study, the Stem Cell-Derived Cells Market is poised to surpass the value of ~US$ XX by the end of 2029 growing at a CAGR of ~XX% over the assessment period. The report includes a thorough analysis of the upstream raw materials, supply-demand ratio of the Stem Cell-Derived Cells in different regions, import-export trends and more to provide readers a fair understanding of the global market scenario.

ThisPress Release will help you to understand the Volume, growth with Impacting Trends. Click HERE To get SAMPLE PDF (Including Full TOC, Table & Figures) athttps://www.persistencemarketresearch.co/samples/28780

The report segregates the Stem Cell-Derived Cells Market into different segments to provide a detailed understanding of the various aspects of the market. The competitive analysis of the Stem Cell-Derived Cells Market includes valuable insights based on which, market players can formulate impactful growth strategies to enhance their presence in the Stem Cell-Derived Cells Market.

Key findings of the report:

The report aims to eliminate the following doubts related to the Stem Cell-Derived Cells Market:

Get Access To TOC Covering 200+ Topics athttps://www.persistencemarketresearch.co/toc/28780

key players in stem cell-derived cells market are focused on generating high-end quality cardiomyocytes as well as hepatocytes that enables end use facilities to easily obtain ready-made iPSC-derived cells. As the stem cell-derived cells market registers a robust growth due to rapid adoption in stem cellderived cells therapy products, there is a relative need for regulatory guidelines that need to be maintained to assist designing of scientifically comprehensive preclinical studies. The stem cell-derived cells obtained from human induced pluripotent stem cells (iPS) are initially dissociated into a single-cell suspension and later frozen in vials. The commercially available stem cell-derived cell kits contain a vial of stem cell-derived cells, a bottle of thawing base and culture base.

The increasing approval for new stem cell-derived cells by the FDA across the globe is projected to propel stem cell-derived cells market revenue growth over the forecast years. With low entry barriers, a rise in number of companies has been registered that specializes in offering high end quality human tissue for research purpose to obtain human induced pluripotent stem cells (iPS) derived cells. The increase in product commercialization activities for stem cell-derived cells by leading manufacturers such as Takara Bio Inc. With the increasing rise in development of stem cell based therapies, the number of stem cell-derived cells under development or due for FDA approval is anticipated to increase, thereby estimating to be the most prominent factor driving the growth of stem cell-derived cells market. However, high costs associated with the development of stem cell-derived cells using complete culture systems is restraining the revenue growth in stem cell-derived cells market.

The global Stem cell-derived cells market is segmented on basis of product type, material type, application type, end user and geographic region:

Segmentation by Product Type

Segmentation by End User

The stem cell-derived cells market is categorized based on product type and end user. Based on product type, the stem cell-derived cells are classified into two major types stem cell-derived cell kits and accessories. Among these stem cell-derived cell kits, stem cell-derived hepatocytes kits are the most preferred stem cell-derived cells product type. On the basis of product type, stem cell-derived cardiomyocytes kits segment is projected to expand its growth at a significant CAGR over the forecast years on the account of more demand from the end use segments. However, the stem cell-derived definitive endoderm cell kits segment is projected to remain the second most lucrative revenue share segment in stem cell-derived cells market. Biotechnology and pharmaceutical companies followed by research and academic institutions is expected to register substantial revenue growth rate during the forecast period.

North America and Europe cumulatively are projected to remain most lucrative regions and register significant market revenue share in global stem cell-derived cells market due to the increased patient pool in the regions with increasing adoption for stem cell based therapies. The launch of new stem cell-derived cells kits and accessories on FDA approval for the U.S. market allows North America to capture significant revenue share in stem cell-derived cells market. Asian countries due to strong funding in research and development are entirely focused on production of stem cell-derived cells thereby aiding South Asian and East Asian countries to grow at a robust CAGR over the forecast period.

Some of the major key manufacturers involved in global stem cell-derived cells market are Takara Bio Inc., Viacyte, Inc. and others.

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Tags: Stem CStem Cell-Derived Cells MarketStem Cell-Derived Cells Market DynamicsStem Cell-Derived Cells Market GrowthStem Cell-Derived Cells Market KeyplayersStem Cell-Derived Cells Market Trends

Originally posted here:
Surge in the Adoption of Stem Cell-Derived Cells to Fuel the Growth of the Stem Cell-Derived Cells Market Through the Assessment Period 2019 2029 -...

Recommendation and review posted by Bethany Smith

Isolated Extramedullary Relapse in Acute Lymphoblastic Leukemia: What Can We Do Before and After Transplant? – Cancer Network

Santiago Riviello-Goya, MD1; Aldo A. Acosta-Medina, MD2; Sergio I. Inclan-Alarcon, MD3; Sofa Garcia-Miranda, MD2; and Christianne Bourlon, MD, MHSc2

1Department of Medicine, Instituto Nacional de Ciencias Mdicas y Nutricin Salvador Zubirn, Mexico City, Mexico; 2Department of Hematology, Instituto Nacional de Ciencias Mdicas y Nutricin Salvador Zubirn, Mexico City, Mexico; 3Cancer Center, Centro Mdico ABC, Mexico City, Mexico

A 43-year-old male with a history of B-cell acute lymphoblastic leukemia (ALL), who underwent allogeneic hematopoietic stem cell transplantation (HSCT) 5 months prior, presented to the emergency department with a 5-day history of progressive bilateral lower extremity weakness. On physical examination, there were no additional neurologic findings; sensory function and urethral and anal sphincter tone were preserved.

Initial clinical laboratory testing showed peripheral blood cell counts, a peripheral blood smear, and a comprehensive metabolic panel within normal limits. Neuroimaging by computed tomography (CT) and magnetic resonance showed no evidence of acute intracranial processes or lesions suggestive of leukemic relapse. A lumbar puncture for cerebrospinal fluid (CSF) analysis was performed and documented the presence of lymphoid-appearing blasts (Figure 1). Flow cytometry (FC) confirmed central nervous system (CNS) infiltration by B-lineage lymphoid blasts (CD34+, CD45+, CD22+, CD19+, and CD10+) (Figure 2). Bone marrow aspirate and biopsy, including FC evaluation, were negative for systemic relapse. Bone marrow chimerism was 98%.

With a diagnosis of isolated extramedullary leukemic relapse (iEMR), the patient was initiated on weekly intrathecal chemotherapy and was weaned off graft-versus-host disease (GVHD) prophylaxis, achieving CSF clearance after 4 weeks of therapy. Against Hematology service recommendations, the patient declined systemic therapy and received only whole brain radiation therapy (24 Gy in 12 fractions).

The patient experienced remission of neurologic symptoms; however, after 5 months, he developed bilateral testicular tenderness and enlargement. An ultrasound was performed and was suggestive of leukemic infiltration (Figure 3). Chemotherapy with methotrexate and L-asparaginase in addition to radiotherapy to the testes (24 Gy in 12 fractions) was given without complications.

One year after initial CNS iEMR, the patient developed overt bone marrow relapse (BMR), as evidenced by development of bone pain throughout the lumbosacral region, and the appearance of multiple blastic and lytic lesions throughout the appendicular and axial skeleton. A positron emission tomography-CT scan documented abdominal lymphadenopathy (Figure 4). With this rapidly progressive picture, the patient was transitioned to supportive care and died 2 months later.

Is the risk of iEMR following HSCT modified by the choice of conditioning regimen? If so, which of the following approaches would have been the best choice to prevent iEMR in this patient?

A. There is no role of conditioning therapy in preventing iEMRB. Reduced intensity of regimen to favor graft-versus-leukemia (GVL) effectC. Nonmieloablative regimens including fludarabineD. Mieloablative regimens including total body irradiation (TBI)

CORRECT ANSWER: D. Mieloablative regimens including total body irradiation (TBI).

Allogeneic HSCT is an effective treatment for ALL, which can achieve long-term remission and even a potential cure.1 Antineoplastic activity is dependent on both high-dose chemotherapy and graft alloreactivity, with the latter manifested in the GVL effect, and undesirably yet inherently, in GVHD.2 Despite recent advances in allogeneic HSCT strategies, disease relapse is common and remains the most important cause of death in this population. Relapse is reported in 30% to 40% of patients but can increase to 60% in patients who are in a second complete remission (CR) at time of HSCT.2,3

Risk factors for relapse in patients with ALL who have undergone HSCT include disease- and transplant-related features. Reported high-risk disease characteristics include: hyperleukocytosis at diagnosis (white blood cell count >30 x109/L for B-lineage ALL and >100 x109/L for T-lineage ALL); cytogenetics associated with poor outcomes, including chromosome 11 translocations and t(9;22); a short remission timespan; more than a first CR; and a failed or delayed remission after induction therapy.4 In the HSCT population, transplant-related factors should be considered, including alternative donors other than those who are matched related and matched unrelated, the type of conditioning regimen, and the development of GVHD.2

ALL relapse following HSCT most commonly involves the medullary compartment, with a cumulative incidence of 41% at 5 years. Conversely, extramedullary relapse (EMR) is uncommon, with a 5-year cumulative incidence of 11.0% and 5.8% for EMR and iEMR, respectively.5 Due to the rarity of EMR, its prognostic impact remains controversial and the ideal management strategies are a subject of active study.

EMR is associated with poor clinical outcomes; however, the subgroup of patients with iEMR (as presented in this patient case) is gaining attention due to its increasing frequency, its role heralding a systemic relapse, and its clinical behavior showing better survival outcomes compared with BMR and EMR.6-8

Isolated EMR is defined as the presence of clonal blasts in any tissue other than the medullary compartment; bone marrow evaluation must show less than 5% of clonal blasts and a full donor chimerism. Most commonly affected sites include the skin, soft tissues, lymph nodes, and immune sanctuaries including the CNS and testes.1,5,9 Because prevention rather than treatment of relapse is related to improved survival outcomes, it is important to define subgroups of patients who may benefit fromearly intervention with a personalized transplant strategy.

Higher rates of iEMR have been linked to patients of younger age. This is thought to be secondary to: (1) a higher incidence of ALL compared with acute myeloid leukemia (AML) in this age subgroup, the former of which is most associated with EMR; (2) the relative overrepresentation of myelomonocytic/monocytic phenotypes in AML presenting in young individuals; and (3) the higher likelihood of a history of EMR in children compared with adults.1,10

A history of extramedullary (EM) disease, which has consistently been found to impact the development of iEMR, is preexistent in up to half of patients. In 2 out of 3 cases of EMR, disease affects the site of original EM involvement, possibly due to low efficacy of both high-dose chemotherapy and the GVL effect.1,5 An exception to this is CNS involvement, despite being a risk factor for subsequent CNS iEMR, which is commonly reported de novo, reflecting the protective effect of regularly administered prophylaxis to patients at high risk of CNS infiltration.11

The effect of GVHD on risk of iEMR is highly nuanced. Despite its well-known role as a protective factor for BMR, the same effect does not appear to hold true for iEMR.12 Initial reports in this population showed no differences in relapse-free survival regardless of acute or chronic GVHD (cGVHD) or a positive association between extensive cGVHD and iEMR development.10,13 This has led to investigators to postulate that the underlying physiopathology differs among different types of relapse, with decreased expression of human leukocyte antigen (HLA) minor histocompatibility antigens and adhesion molecules and decreased penetration of both immune cells and high-dose chemotherapy to EM sites.14 These mechanisms lead to decreased effectivness of T-cell dependent cytotoxicity of donor lymphocytes as compared with the medullary compartment, with subsequent clone selection and escape, enabling the development of iEMR.6

With the increased use of alternative donors, this has been contested in the haploidentical setting, with a recent report showing significantly increased rates of iEMR in patients who do not develop cGVHD. It is suggested that the role of GVL, coupled with GVHD, in this HLA-mismatched setting could partially explain the added benefit of GVHD in this subgroup. This report also evidenced increased tumor chemosensitivity in patients with EMR compared with BMR, possibly explained by reduced concentrations of conditioning therapy at EM sites.9

Cytogenetics associated with poor outcomes and advanced disease at the time of HSCT were described as risk factors for iEMR in initial cohort studies.1,5,10,15,16 However, recent publications that include alternative-donor HSCT recipients have reported that a haploidentical source could overcome this negative impact.9

The influence of type of conditioning regimen on likelihood of iEMR has been studied only retrospectively, mainly comparing TBI-based versus chemotherapy-based approaches. The landmark paper by Simpson et al showed a significantly elevated rate of iEMR in patients receiving busulfan-based conditioning. This finding has been related to the lack of penetration of drugs into the immune sanctuaries with chemotherapy-only regimens.17

Multiple approaches, including combination and single treatment for iEMR, have been described. Combination therapy including systemic chemotherapy plus local radiotherapy (or in CNS disease, radiation to the craniospinal axis, intrathecal chemotherapy, and systemic chemotherapy) has been associated with higher response rates than single-treatment strategies.9 Nonetheless, the best responses have been observed when combination therapy is followed by a cellular therapy (eg, second allogeneic HSCT, donor leukocyte infusion, and donor stem cell infusion), leading to CR rates of greater than 80%.5,13 Whether this increase in CR rate translates to an increase in survival outcomes remains debatable due to conflicting results in the current literature for iEMR.

Financial Disclosure: The authors have no significant financial interest in or other relationship with the manufacturer of any product or provider of any service mentioned in this article.

Corresponding author:

Christianne Bourlon, MD, MHScVasco de Quiroga No. 15.Belisario Domnguez Seccin XVI

Tlalpan, C.P. 14080, Ciudad de Mxico, Mxico

E-mail: chrisbourlon@hotmail.com

References:

1. Ge L, Ye F, Mao X, et al. Extramedullary relapse of acute leukemia after allogeneic hematopoietic stem cell transplantation: different characteristics between acute myelogenous leukemia and acute lymphoblastic leukemia. Biol Blood Marrow Transplant. 2014;20(7):1040-1047. doi: 10.1016/j.bbmt.2014.03.030.

2. Pavletic SZ, Kumar S, Mohty M, et al. NCI First International Workshop on the Biology, Prevention, and Treatment of Relapse after Allogeneic Hematopoietic Stem Cell Transplantation: report from the Committee on the Epidemiology and Natural History of Relapse following Allogeneic Cell Transplantation. Biol Blood Marrow Transplant. 2010;16(7):871-890. doi: 10.1016/j.bbmt.2010.04.004.

3. Devillier R, Crocchiolo R, Etienne A, et al. Outcome of relapse after allogeneic stem cell transplant in patients with acute myeloid leukemia. Leuk Lymphoma. 2013;54(6):1228-1234. doi: 10.3109/10428194.2012.741230.

4. Hoelzer D, Bassan R, Dombret H, Fielding A, Ribera JM, Buske C; ESMO Guidelines Committee. Acute lymphoblastic leukaemia in adult patients: ESMO Clinical Practice Guidelines for diagnosis, treatment and follow-up. Ann Oncol. 2016;27(suppl 5):v69-v82. doi: 10.1093/annonc/mdw025.

5. Shem-Tov N, Saraceni F, Danylesko I, et al. Isolated extramedullary relapse of acute leukemia after allogeneic stem cell transplantation: different kinetics and better prognosis than systemic relapse. Biol Blood Marrow Transplant. 2017;23(7):1087-1094. doi: 10.1016/j.bbmt.2017.03.023.

6. Lee JH, Choi SJ, Lee JH, et al. Anti-leukemic effect of graft-versus-host disease on bone marrow and extramedullary relapses in acute leukemia. Haematologica. 2005;90(10):1380-1388.

7. Xie N, Zhou J, Zhang Y, Yu F, Song Y. Extramedullary relapse of leukemia after allogeneic hematopoietic stem cell transplantation. Medicine (Baltimore). 2019;98(19):e15584. doi: 10.1097/MD.0000000000015584.

8. Shi JM, Meng XJ, Luo Y, et al. Clinical characteristics and outcome of isolated extramedullary relapse in acute leukemia after allogeneic stem cell transplantation: a single-center analysis. Leuk Res. 2013;37(4):372-377. doi: 10.1016/j.leukres.2012.12.002.

9. Mo XD, Kong J, Zhao T, et al. Extramedullary relapse of acute leukemia after haploidentical hematopoietic stem cell transplantation: incidence, risk factors, treatment, and clinical outcomes. Biol Blood Marrow Transplant. 2014;20(12):2023-2028. doi:10.1016/j.bbmt.2014.08.023.

10. Harris AC, Kitko CL, Couriel DR, et al. Extramedullary relapse of acute myeloid leukemia following allogeneic hematopoietic stem cell transplantation: incidence, risk factors and outcomes. Haematologica. 2013;98(2):179-184. doi: 10.3324/haematol.2012.073189.

11. Hamdi A, Mawad R, Bassett R, et al. Central nervous system relapse in adults with acute lymphoblastic leukemia after allogeneic hematopoietic stem cell transplantation. Biol Blood Marrow Transplant. 2014;20(11):1767-1771. doi: 10.1016/j.bbmt.2014.07.005.

12. Giralt SA, Champlin RE. Leukemia relapse after allogeneic bone marrow transplantation: a review. Blood. 1994;84(11):3603-3612.

13. Solh M, DeFor TE, Weisdorf DJ, Kaufman DS. Extramedullary relapse of acute myelogenous leukemia after allogeneic hematopoietic stem cell transplantation: better prognosis than systemic relapse. Biol Blood Marrow Transplant. 2012;18(1):106-112. doi: 10.1016/j.bbmt.2011.05.023.

14. Kolb HJ. Graft-versus-leukemia effects of transplantation and donor lymphocytes. Blood. 2008;112(12):4371-4383. doi: 10.1182/blood-2008-03-077974.

15. Lee KH, Lee JH, Choi SJ, et al. Bone marrow vs extramedullary relapse of acute leukemia after allogeneic hematopoietic cell transplantation: risk factors and clinical course. Bone Marrow Transplant. 2003;32(8):835-842. doi: 10.1038/sj.bmt.1704223.

16. Clark WB, Strickland SA, Barrett AJ, Savani BN. Extramedullary relapses after allogeneic stem cell transplantation for acute myeloid leukemia and myelodysplastic syndrome. Haematologica. 2010;95(6):860-863.

17. Simpson DR, Nevill T, Shepherd JD, et al. High incidence of extramedullary relapse of AML after busulfan/cyclophosphamide conditioning and allogeneic stem cell transplantation. Bone Marrow Transplant. 1998;22(3):259-264. doi: 10.1038/sj.bmt.1701319.

Excerpt from:
Isolated Extramedullary Relapse in Acute Lymphoblastic Leukemia: What Can We Do Before and After Transplant? - Cancer Network

Recommendation and review posted by Bethany Smith

The Alliance for Regenerative Medicine Releases Agenda for 2020 Cell & Gene Meeting on the Mediterranean – Yahoo Finance

WASHINGTON, D.C., Feb. 13, 2020 (GLOBE NEWSWIRE) -- via NEWMEDIAWIRE -- The Alliance for Regenerative Medicine (ARM), the international advocacy organization representing the cell and gene therapy and broader regenerative medicine sector, today released the agenda for its second annual Cell & Gene Meeting on the Mediterranean. The event will be held from April 15-17, 2020 in Barcelona, Spain.

The event, modeled after ARMs highly successful Cell & Gene Meeting on the Mesa, is expected to attract more than 500 attendees, including senior executives from leading cell therapy, gene therapy, and tissue engineering companies worldwide, large pharma and biotech representatives, institutional investors, academic research institutions, patient foundations, disease philanthropies, and members of the life science media community.

The agenda includes:

Plenary Session:

Keynote Address:

Panels:

Throughout the two-day event, participants can also attend presentations by more than 50 publicly traded and emerging private companies, highlighting clinical and commercial progress in cell therapy, gene and gene-modified cell therapy, tissue engineering, biomaterials and more. In addition to their presentations, representatives from these organizations will also be available for one-on-one partnering opportunities throughout the conference.

2020 presenting companies include:Adaptimmune, AGTC, Ambys Medicines, AskBio, Aspect Biosystems, Atara Biotherapeutics, Autolus Therapeutics, Avectas, AVROBIO, Axovant Gene Therapies, bluebird bio, Bone Therapeutics, Cabaletta Bio, Caribou Biosciences, Celavie Biosciences, Cellatoz Therapeutics, Cellect Biotherapeutics, CEVEC, Cryoport, Cynata Therapeutics, Flexion Therapeutics, Fraunhofer IZI, Genethon, GenSight Biologics, Healios, Iovance Biotherapeutics, Kiadis Pharma, Kytopen, LogicBio Therapeutics, MeiraGTx, Minerva Biotechnologies, MolMed, Novadip Biosciences, Orchard Therapeutics, Oxford Biomedica, PDC*line Pharma, Polyplus-transfection, Precision BioSciences, Promethera Biosciences, PTC Therapeutics, Recombinetics, REGENXBIO, ReNeuron, Rexgenero, Sangamo, SmartPharm Therapeutics, Standards Coordinating Body for Regenerative Medicine, Theradaptive, ThermoGenesis, Tmunity Therapeutics, Ultragenyx Pharmaceutical, VERIGRAFT, and Vineti.

For full details on the agenda and further information about the event, please visit http://www.meetingonthemed.com.

Registration is complimentary for credentialed members of the media. For members of the media interested in attending, please contact Kaitlyn Donaldson Dupont at kdonaldson@alliancerm.org or Consilium Strategic Communications at ARM@consilium-comms.com.

About the Alliance for Regenerative Medicine

The Alliance for Regenerative Medicine (ARM) is an international multi-stakeholder advocacy organization that promotes legislative, regulatory, and reimbursement initiatives necessary to facilitate access to life-giving advances in regenerative medicine worldwide. Founded in 2009, ARM works to increase public understanding of the field and its potential to transform human healthcare, providing business development and investor outreach services to support the growth of its 350+ member organizations worldwide. ARM represents the interests of therapeutic developers, academic research institutions, major medical centers, investors, and patient groups that comprise the broader regenerative medicine community and is the prominent international advocacy organization in this field.

ARM has 70+ members across 15 countries in Europe. ARM aims to work closely with European stakeholders, leveraging its membership to create a supportive commercial and regulatory environment to create better conditions for the development and commercialization of ATMPs in Europe; develop strong stakeholder support around proposed solutions to improve patient access to ATMPs; promote clear, predictable and efficient regulatory framework across Europe; and promote international convergence of key regulations and guidance. For more information, visit alliancerm.org.

Kaitlyn Donaldson Dupont8037278346kdonaldson@alliancerm.org

Original post:
The Alliance for Regenerative Medicine Releases Agenda for 2020 Cell & Gene Meeting on the Mediterranean - Yahoo Finance

Recommendation and review posted by Bethany Smith

Biologists rush to re-create the China coronavirus from its DNA code – MIT Technology Review

The world is watching with alarm as China struggles to contain a dangerous new virus, now being called SARS-CoV-2. It has quarantined entire cities, and the US has put a blanket ban on travellers whove been there. Health officials are scrambling to understand how the virus is transmitted and how to treat patients.

But in one University of North Carolina lab, theres a different race. Researchers are trying to create a copy of the virus. From scratch.

Led by Ralph Baric, an expert in coronaviruseswhich get their name from the crown-shaped spike they use to enter human cellsthe North Carolina team expects to recreate the virus starting only from computer readouts of its genetic sequence posted online by Chinese labs last month.

The remarkable ability to boot up viruses from genetic instructions is made possible by companies that manufacture custom DNA molecules, such as Integrated DNA Technology, Twist Bioscience, and Atum. By ordering the right genes, which cost a few thousand dollars, and then stitching them together to create a copy of the coronavirus genome, its possible to inject the genetic material into cells and jump-start the virus to life.

The ability to make a lethal virus from mail-order DNA was first demonstrated 20 years ago. Its enough of a bioterrorism concern that companies carefully monitor who is ordering which genes. But its also an important way to respond to a sudden outbreak, since synthetic virus recipes give researchers powerful ways to study treatments, vaccines, and how mutations could make it more dangerous.

When a synthetic virus is better than the real thing

Barics North Carolina lab, which specializes in engineering viruses, has previously butted heads with Washington agencies over the work, which has included synthesizing new, never before seen coronaviruses that can infect mice. In 2014, the National Institutes of Health froze funding to several labs, including Barics, over concerns that such research was too risky. The funding was later reinstated.

For the China virus, Baric said in a telephone interview, his team placed an order for matching DNA from a manufacturer last month. Their first step was to go online and look at genetic sequences of the virus. They then compared several available sequences, which differ slightly, and picked a consensus version to have manufactured.

Once Baric gets his DNA, something that could take a month, he plans to inject the genetic instructions into cells. If things go as planned, the cells should begin making actual infectious viral particles.

CDC

By rolling their own germs, scientists can get hold of viruses even if they cant obtain them directly from a country, especially one thats in the grip of an epidemic. Baric says so far samples of the live virus from patients have not been made widely available from China. This is the future in terms of how the medical research community responds to a new threat, says Baric.

The real virus and the synthetic one should be basically identical. But with the synthetic one, we have a DNA copy that we can go back to over and over and over again, to make genetically identical viruses, says Timothy Sheahan, a researcher at UNC who works with Baric. Starting from these copies, scientists can remove genes, add others, and figure out things like what makes the germ spread and how it gains access to human cells. Sheahan wants to try infecting mice with the virus and giving them various drugs to see what stops it.

Artificial copies may also help scientists keep up with the outbreaks unpredictable path. I worry this virus is going to mutate in the course of the epidemic, and this would allow me to study what effects those mutations have, says Stanley Perlman, a microbiologist who works on coronaviruses at the University of Iowa. The synthetic virus is just a substitute for the actual virus, but with the DNA clone you can manipulate it and find the weak points and develop a therapy.

During past outbreaks, scientists would have had to wait months or years to get a look at the germ behind an outbreak. But with SARS-CoV-2 it took only weeks until its genetic sequence was posted online. Immediately, some scientists began analyzing the genetic data, comparing it to viruses from bats, snakes and pangolins; they concluded it could have begun circulating last November.

Biotech companies, governments, and universities also quickly started ordering physical copies of particular genes found in the virus. DNA manufacturers say they have been deluged with orders for virus parts, including those useful for verifying diagnostic tests and others needed to make potential vaccines.

Its been a pretty dramatic uptick, starting with the publication of the genome, says Adam Clore, technical director of synthetic biology at IDT, based in Iowa, and one of the worlds largest sellers of DNA. Its high priority. There are a number of institutions that are devoting nearly all their energy working on detection or vaccines.

Still, most researchers need only one or two genes from the virus to carry forward work on tests and vaccines. Barics lab in North Carolina is the only one in the US known to be trying to re-create the virus completely from ordered DNA parts.

How to keep deadly viruses out of the wrong hands

It was in the early 2000s that scientists first showed that synthetic DNA strands could be used to resurrect viruses just from their genetic code. A team in New York State did it with polio, producing infectious material from DNA they ordered online.

The technology immediately created bio-weapon worries. What if terrorists used the technique to resurrect smallpox? That hasnt happened, but it does mean that scourges like polio, smallpoxand now the Chinese coronaviruscannot now ever be truly wiped out. Researchers at the US Centers for Disease Control and Prevention (CDC) drove that point home in 2005 when they resurrected the influenza virus that killed tens of millions in 1918-1919.

To keep the technology out of the hands of evil-doers, companies that manufacture DNA banded together a few years ago to limit access to dangerous genes. The big US players have all agreed to compare incoming DNA orders to a database of about 60 lethal germs and toxins called select agents so that only authorized labs can ever obtain the DNA needed to resurrect them.

CDC

At our request, Battelle, a scientific R&D company whose software ThreatSEQ can make those comparisons, ran the scenario of someone trying to order a copy of SARS-CoV-2. According to Craig Bartling, a senior research scientist at Battelle, the software flagged both the entire virus, and most of its genes individually, at the highest threat level. Bartling says the alerts went off because the virus is highly similar to the original SARS, a related virus that sparked a global outbreak starting in 2002.

Research into the new virus is seen as risky enough that manufacturers of DNA hurried last week to meet and formulate a policy about who should be able buy complete versions of the new germs genome. In a statement released on February 11, the International Gene Synthesis Consortium, a trade group, struck a cautious position. It said it would treat the new Chinese virus as if it were SARS, a germ added to the select agent list in 2012 and whose possession is tightly monitored by the US government.

That means anyone who wants a complete synthetic copy of SARS-CoV-2 would need to undergo specific and detailed vetting and prove they are already registered by the CDC to work with SARS, as the North Carolina researchers are.

However, companies that manufacture DNA still have discretion over what they sell and to whom, and not all of them think they should make the whole genome of this virus. Claes Gustaffson, founder and chief commercial officer of Atum, a DNA supplier in California, says hes gotten orders from eight companies for parts of the virus genome and has personally approved a request by a US government agency to make 90% of its geneslikely to create an attenuated (i.e., harmless) version of it.

They probably want to figure out how to make a vaccine as quickly as possible, says Gustaffson. But if someone wanted the whole thing, I wouldnt make it. Some things, like polio, you dont want to make, no matter who is asking.

UNC Gillings School of Public Health

Not everyone thinks synthesizing the new coronavirus is particularly dangerous. I dont really see a huge amount of risk, says Nicholas G. Evans, who studies biothreats at the University of Massachusetts, Lowell. Right now, a lot of people are spending a lot of time on how this coronavirus works. I think the risks are outweighed by the benefits.

The outbreak, which appears to have begun in a live animal market in the city of Wuhan, had caused more than 64,000 cases and 1,350 deaths in China by February 14, so its even worse than SARS, which killed 774 people.

Still, the US has not yet declared the new virus to be a select agent. According to Baric, the decision to add a new virus to the most-dangerous list is not made in the expanding outbreak, because it slows down research.

Scaring people

For now, only a very few sophisticated centers can actually re-boot a virus; theres no chance a nut working from a garage could do it. We are at the point where the best of the best can start to synthesize this new virus contemporaneously with the outbreak. But that is just a few labs, says Evans. Fortunately, we are still far from the point when lots of people can synthesize anything.

The advanced state of synthetic virus research, and the ability to genetically engineer germs, inevitably feeds fears, and conspiracy theories. Social media and some blog sites have been full of groundless speculation that the new virus was accidentally released from a Chinese bioweapon lab located outside of Wuhan. Theres no evidence that is the case, and substantial evidence it is not, but the rumor caused a diplomatic breach with China after it was repeated in the US Congress by a senator, Tom Cotton of Arkansas.

Baric says he doesnt see a particular danger to synthesizing the new virus at this stage of the outbreak, especially because the virus is still circulating in the wild. The important thing is to figure out what it does and stop it. Whether you get it from a cell or synthesize it, it ends up the same thing, says Baric.

Continued here:
Biologists rush to re-create the China coronavirus from its DNA code - MIT Technology Review

Recommendation and review posted by Bethany Smith

Global Viral Vector & Plasmid DNA Manufacturing Market 2019 Industry Research, Segmentation, Key Players Analysis and Forecast to 2024 – Nyse…

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Global Viral Vector & Plasmid DNA Manufacturing Market 2019 Industry Research, Segmentation, Key Players Analysis and Forecast to 2024 - Nyse...

Recommendation and review posted by Bethany Smith

Therapies poised to reshape the treatment landscape for hematologic disorders – BioWorld Online

Millions of people are affected by blood disorders, and the prevalence is expected to grow as our population ages.

It is not surprising that, according to the American Society of Hematology, the FDA approved several new therapies or new indications for previously approved therapies in 2019 for people living with non-malignant blood disorders. Those included two disease-modifying treatments for sickle cell disease and the first anticoagulant for venous thromboembolism management in children.

In the wings and poised to reshape the treatment landscape for hematologic disorders are two potential blockbuster drugs - Valrox (valoctocogene roxaparvovec) for hemophilia A and vadadustat for anemia related to chronic kidney disease - that are among the 11 included in the Cortellis Drugs to Watch analysis predicted to achieve annual sales of $1 billion by 2024.

Hemophilia A

Hemophilia A, also called factor VIII (FVIII) deficiency or classic hemophilia, is a genetic disorder caused by missing or defective factor VIII, a clotting protein. People with that disorder are at risk for painful and/or potentially life-threatening bleeds from even modest injuries. According to the U.S. CDC, the condition occurs in approximately one in 5,000 live births, and about 20,000 people are living with hemophilia in the U.S. Accurate data on the worldwide incidence of hemophilia is estimated at more than 400,000.

People suffering from the most severe form of hemophilia A often experience painful, spontaneous bleeds into their muscles or joints and that group makes up approximately 43% of the hemophilia A population. The standard of care (SOC) for such individuals is a prophylactic regimen of replacement factor VIII infusions administered intravenously up to two to three times per week or 100 to 150 infusions per year. Despite those treatments, many people continue to experience bleeds, resulting in progressive and debilitating joint damage, which can have a major impact on their quality of life.

One and done treatment

San Rafael, Calif.-based Biomarin Pharmaceutical Inc. has submitted a BLA to the FDA for its investigational AAV gene therapy, Valrox, for adults with hemophilia A. The therapy is designed to deliver functional copies of the FVIII gene into patients cells, enabling them to make the previously missing or defective FVIII protein. If approved, Valrox would be the first potentially curative (one and done) approach to hemophilia A, eliminating the need for blood transfusions and FVIII replacement therapy after a single infusion. Subject to completion of the agency's filing review, the company anticipates the BLA review to start in February.

The FDA has granted breakthrough therapy and orphan drug designations; and the EMA has validated the company's marketing authorization application with the review under accelerated assessment.

According to the Cortellis Drugs to Watch analysis, the filings were based on interim data from the phase III GENEr8-1 study as well as three-year phase I/II trial data. In GENEr8-1, Valrox met the prespecified criteria for U.S. and EU regulatory review, with eight patients in a 20-patient cohort achieving FVIII levels of at least 40 IU/dl at 23 to 26 weeks; the ongoing trial has the goal of evaluating superiority of Valrox to the current standard of care, prophylactic therapy. The high-dose cohort in the phase I/II study showed 100% resolution of target joints, a 96% reduction in mean annualized FVIII usage, and all patients remained off FVIII prophylaxis. No thrombotic events or development of FVIII inhibitors have been reported to date.

Transformative for patients

"People with severe hemophilia A continue to experience clinically relevant breakthrough bleeds despite the current standard of care and can be limited in their physical activities," noted John Pasi, chief investigator for the phase I/II study and a principal investigator for the phase III study. "Valoctocogene roxaparvovec represents a potentially transformative investigative therapy that could improve patients' quality of life, including consequences of bleeding, physical functioning, role functioning, emotional impact, treatment concern and worry."

In January, the company published three years of follow-up data in the phase I/II study showing the median use of exogenous factor VIII at the 6e13-vg/kg dose was reduced from 138.5 infusions per year to zero infusions per year in year three. In the year before study entry, the mean annualized number of factor VIII infusions per participant was 136.7+22.4; at the end of year three, the mean annualized use of exogenous factor VIII decreased by 96% to a mean of 5.5+9.4 infusions.

"As a treating physician, I am excited about the potential of the field of gene therapy to make a meaningful difference in the lives of people with hemophilia A," Pasi noted on the results.

First-mover advantage

In a presentation at the annual J.P. Morgan Healthcare Conference in January 2020, Biomarin Chairman and CEO Jean-Jacques Bienaime was excited to reveal that the company is ramping up productivity at its Novato, Calif., plant, more than doubling its capacity to 10,000 doses annually. He noted that the capacity upgrade is important as the firm wants to be able to supply the gene therapy market as quickly as possible "because first-mover advantage in gene therapy is fundamental in a sense that every time you treat a patient that patient is off the market."

Gene therapy certainly has the potential to revolutionize treatment for patients with hemophilia A. However, according to the Canadian Hemophilia Society, Never have so many coagulation therapies been in development. In addition to the large number of therapies recently introduced, we have identified another 18 new therapies in development or soon to be marketed, including six clotting factor concentrates, four bypassing therapies to treat patients with inhibitors, two non-factor coagulation products and five gene therapy products.

If approved, Valrox will certainly face competition from well-established FVIII replacement therapies, in addition to those that are nearing the market, the Cortellis analysis suggests.

Recently approved therapies such as Roche Holding AG's Hemlibra (emicizumab) and Bayer AG's Jivi (antihemophilic factor [recombinant] pegylated) are improving the options available to those patients. The hemophilia market is conservative in adopting new therapies, the analysis notes, and patients may be reluctant to switch to new products if their current replacement therapy works well. However, an unmet need remains, as approximately 75% of hemophilia A patients do not respond adequately to their treatment or even do not receive treatment at all. Despite a relatively low worldwide prevalence of patients, with orphan pricing, hemophilia A represents a large market.

In addition, Valrox is not without its own challenges, the Cortellis analysis notes. The drug fell short in the initial cohort of the phase III GENEr8-1 trial: of 17 evaluable patients, three failed to achieve FVIII levels above 5 IU/dl. The mean and median FVIII levels across the cohort were also lower than seen in phase I/II, at 33 and 36 IU/dl, respectively. Failure to improve the response once full data are available may impact the potential commercial uptake

The market

The global market for hemophilia A last year was estimated to be close to $10 billion, and Bienaime in his presentation said that an estimated 121,000 patients are located in the territories covered by the company. Although no pricing has yet been set for the gene therapy, the company has done a lot of payer research. He indicated that payers attribute a high value to the physiological correction of hemophilia A, and it appears the U.S. payer community would be comfortable with a price between $2 million and $3 million.

Given the fact that wholesale acquisition pricing for Hemlibra in non-inhibitor adult patients is between $600,000 to $800,000 per year, Biomarin has looked at pricing of between $1 million and $5 million. Since by launch date it will have four years of data on Valrox, the price could be set at four times the average price of Hemlibra ($700,000), establishing a price of $2.8 million. That would make it the most expensive one-time therapy, topping spinal muscular atrophy gene therapy Zolgensma (onasemnogene abeparvovec-xioi) from Novartis AG at $2.1 million. The company says that this first gene therapy for any form of inherited hemophilia could save health care systems more than $20 million over a typical patients lifetime.

For that reason, even if the penetration is modest, Valrox could post significant revenue. The Cortellis analysis predicts sales of $17.45 million forecast for this year, rising to $1.297 billion in 2024. However, while Biomarin is in the pole position with its gene therapy, potential competition in the pipeline from FVIII gene therapies in development may temper sales forecasts in the mid- to long term. Spark Therapeutics Inc.'s SPK-8011 entered phase III development in February 2019, having shown a 94% reduction in bleeds and a 95% reduction in FVIII infusions in a previous phase I/II study. Other direct competitors in phase I/II development include Ultragenyx Pharmaceutical Inc.s DTX-201, Sparks SPK-8016, Shire plcs SHP-654 and University College London (UCL)/St. Jude Children's Research Hospitals AAV2/8-HLP-FVIII-V3.

Anemia in chronic kidney disease

Anemia is one of the many complications of chronic kidney disease (CKD), which worsens as kidney disease progresses; most patients whose CKD has progressed to kidney failure have significant anemia. It is estimated that CKD affects 200 million people worldwide. The anemia in CKD is currently treated with injectable recombinant erythropoiesis-stimulating agents (ESAs), which are often associated with inconsistent hemoglobin responses and safety risks.

The need for more effective and safer therapies has led to the discovery of hypoxia-inducible factor prolyl hydroxylase (HIF-PH) enzyme inhibitors, a new class of agents for the treatment of anemia in CKD (see sidebar story). Those agents work by stabilizing the HIF complex and stimulating endogenous erythropoietin production even in patients with end-stage kidney disease.

Several HIF-PH enzyme inhibitors are currently in development targeting the estimated $3.5 billion renal anemia market, including Cambridge, Mass.-based Akebia Therapeutics Inc.s vadadustat, which has advanced to late-stage clinical trials. In July, its strategic partner, Osaka-based Mitsubishi Tanabe Pharma Corp., filed an NDA with the Japanese Ministry of Health, Labour and Welfare, seeking approval for the product as a treatment for anemia due to CKD.

The filing is based on data from four studies in Japanese patients: an active-controlled study in non-dialysis-dependent CKD anemia (J01), another active-controlled study but in dialysis-dependent patients with CKD anemia (J03), and two single-arm studies in patients with peritoneal and hemodialysis-dependent CKD anemia (J02 and J04). Vadadustat demonstrated non-inferiority with respect to hemoglobin level versus the active comparator (darbepoetin alfa) in both dialysis- and non-dialysis-dependent patients (J01 and J03 studies; 11.66 vs 11.93 g/dL, and 10.61 vs 10.65 g/dL, respectively) and showed therapeutic effect in the single-arm studies.

If approved, the company expects commercial launch during mid-2020, and filings in the U.S. and the EU are planned, those territories covered by Akebias alliance with Otsuka Pharmaceutical Co., Ltd.

Competitors

Within the HIF-PH inhibitor class itself, and particularly from Fibrogen Inc.s Evrenzo (roxadustat), the first-in class HIF-PH inhibitor, vadadustat will face direct competition. Fibrogen has partnered with Astellas Pharma Inc. and Astrazeneca plc for the development and marketing of Evrenzo, which has been approved in Japan (in dialysis patients) and China (in dialysis- and nondialysis-dependent patients) in the third quarter of 2019. An additional filing in Japan in non-dialysis patients is expected in the short term upon completion of a second pivotal study in that setting. The drug has also been filed in the U.S (for both patient populations) and filings in the EU are expected by March, according to Cortellis, following positive top-line data from the ALPS (nondialysis-dependent) and HIMALAYAS (dialysis-dependent) studies.

The analysis indicates vadadustat would also face competition from other well-established therapeutic approaches in CKD such as intravenous iron replacement products and blood transfusions that offer rapid increases in Hb levels.

Future direct competition will also come from Glaxosmithkline plcs HIF-PH inhibitor daprodustat, which was submitted for approval in Japan in August last year.

The Cortellis analysis cites sales forecasts in 2024 of $1.188 billion for Evrenzo and $286 million for daprodustat. For vadadustat, sales of $2 million are forecast for this year, rising to $1.589 billion in 2024.

See a related article:

Out of basic science, a blockbuster: Vadadustat

To read more about the Cortellis Drugs to Watch potential blockbusters, visit BioWorlds collection of articles which are freely available.

More:
Therapies poised to reshape the treatment landscape for hematologic disorders - BioWorld Online

Recommendation and review posted by Bethany Smith

Edited Transcript of RARE earnings conference call or presentation 13-Feb-20 10:00pm GMT – Yahoo Finance

Novato Feb 14, 2020 (Thomson StreetEvents) -- Edited Transcript of Ultragenyx Pharmaceutical Inc earnings conference call or presentation Thursday, February 13, 2020 at 10:00:00pm GMT

Ultragenyx Pharmaceutical Inc. - Senior Director of IR & Corporate Communications

* Emil D. Kakkis

Ultragenyx Pharmaceutical Inc. - President, CEO & Director

Ultragenyx Pharmaceutical Inc. - CFO & Executive VP

* Andrea R. Tan

Sanford C. Bernstein & Co., LLC., Research Division - VP

Ladies and gentlemen, thank you for standing by. And welcome to the Ultragenyx Fourth Quarter and Full Year 2019 Financial Results Conference Call. (Operator Instructions)

I would now like to hand the conference to your speaker today, Danielle Keatley. Please go ahead.

Danielle Keatley, Ultragenyx Pharmaceutical Inc. - Senior Director of IR & Corporate Communications [2]

Thank you. Good afternoon, and welcome to the Ultragenyx Pharmaceutical financial results and corporate update conference call for the fourth quarter and full year 2019. We've issued a press release detailing our financial results, which you can find on our website at ultragenyx.com.

I'm Danielle Keatley, Senior Director of Investor Relations. And with me today are Emil Kakkis, Chief Executive Officer and President; and Shalini Sharp, Chief Financial Officer.

I'd like to remind investors that this call will include forward-looking statements within the meaning of the safe harbor provisions of the Private Securities Litigation Reform Act of 1995, including, but not limited to, the types of statements identified as forward-looking in our quarterly report on Form 10-Q that was filed on November 6, 2019, our annual report on Form 10-K that will be filed soon; and our subsequent periodic reports filed with the SEC, which will all be available on our website in the Investors section.

These forward-looking statements represent our views only as of the date of this call and involves substantial risks and uncertainties, including many that are beyond our control.

Please note that actual results could differ materially from those projected in any forward-looking statement.

For a further description of the risks and uncertainties that could cause actual results to differ materially from those expressed in the forward-looking statements as well as risks relating to our business, see our periodic reports filed with the SEC.

I'll now turn the call over to Emil.

Emil D. Kakkis, Ultragenyx Pharmaceutical Inc. - President, CEO & Director [3]

Thank you, Daniel. I'll start with our commercial performance in the fourth quarter of 2019. Shalini will then summarize our financial results for the quarter and the year. I'll come back at the end to discuss the progress across our clinical and preclinical programs and our outlook for the rest of the year.

Starting with Crysvita, which has been the main focus of our commercial efforts and the primary driver of revenue in 2019. Performance in the fourth quarter built on the momentum in the first 5 quarters of launch, this was reflected by continued increases in depleted start forms and the number of patients on reimbursed therapy.

In the U.S., we ended the year with approximately 1,590 completed start forms, 160 more than the third quarter. We also ended the year with approximately 1,330 patients on reimbursed therapy, a 200 patient increased versus the end of the third quarter.

As the commercial team continues to work to penetrate the adult market, we believe Crysvita will continue to be one of the most successful rare disease programs launched.

Our 2020 Crysvita revenue guidance of $125 million to $140 million further reflects the confidence we have in Crysvita and our commercial team's ability to execute. Now that the early launch period is over, going forward, we do not plan on providing specific launch metrics, but we'll focus on revenue for Crysvita. To put our launch progress in perspective, we mapped out the top rare disease launches through their first 6 quarters over the last 15 years. We found that Crysvita is one of the top rare disease launches based on top line total product sales. We've also generated a substantial revenue of setting a price that is substantially lower than any of the other top rare disease drugs. As a result, we have successfully ensured that payer view our pricing as responsible, allowing us to reach more patients, especially adult and achieve a positive financial outcome for the company. This approach is core to our philosophy about improving access and total revenue by moderating rare disease pricing.

Turning to Canada, we are seeing continued prescribing interest from physicians and the number of reimbursed patients with private insurance has exceeded our expectations. More than half of Canadians have supplemental private insurance today and the number of pediatric and adult patients were receiving Crysvita through their private insurance drug plans continues to grow. We also continue to pursue public reimbursement in Canada, which will take more time.

Moving to Latin America. In Argentina and Colombia, the number of patients are reimbursed, named patient treatment continues to increase, and the feedback has been very positive.

In Brazil, the demand has been strong, with a significant number of patients successfully navigating the cumbersome legal process and a few receiving reimbursed treatment to date. We're also seeking pricing and full reimbursement approval by the Ministry of Health to enable more rapid access for patients in Brazil. Ultimately, we believe there is significant potential for Crysvita in Latin America, with growing demand in multiple countries for the product.

Briefly turning to Mepsevii. The therapy approved in the United States, Europe and Brazil, and demand continues to build gradually as typical for enzyme replacement therapies. We are also continuing reimbursement discussions with various government health authorities throughout the world.

With that, I'll turn the call over to Shalini, who will provide a financial update.

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Shalini Sharp, Ultragenyx Pharmaceutical Inc. - CFO & Executive VP [4]

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Thank you, Emil. And good afternoon, everyone. Earlier today, we issued a press release that included a financial update, which I will briefly summarize.

Ultragenyx's total net revenue for the 12-month period ending December 31, 2019, was $103.7 million. And for the fourth quarter of 2019 was $35.6 million. The following is a product-by-product breakdown of these figures.

For Crysvita, during the year ended December 31, 2019, we recognized total revenue of $87.3 million. This includes $74.9 million in collaboration revenue in the U.S. profit share territory in Canada and $8.1 million in royalty revenue in the European territory from our collaboration and license agreement with our partner, Kyowa Kirin or KKC. Net product sales for Crysvita in other regions totaled $4.3 million. Total Crysvita revenue recognized Ultragenyx for the 3 months ended December 31, 2019, was $29.9 million. This includes $26.1 million in collaboration revenue in the North American profit share territory, $2.2 million in royalty revenue on KKC sales in the European territory and $1.6 million in net product revenue in other regions.

Recall, there was a significant order that was placed on the last day of the third quarter of 2019, which was recognized in the fourth quarter due to shipping terms. Depending on ordering patterns, we continue to expect fluctuations in our quarter-to-quarter revenue recognition from time to time.

In Latin America, full reimbursement takes place on a country-by-country basis and can take some time, which can be further complicated by economic and political instability. Total 2019 Crysvita sales in North America, Europe and Latin America, which are shared with KKC were approximately $104 million for the fourth quarter and approximately $316 million for the full year 2019.

Mepsevii product revenue for the fourth quarter of 2019 was $4.4 million and was $12.6 million for the year. Due to the rarity of MPS 7, we expect revenues for this product to be somewhat irregular from quarter-to-quarter and to build very gradually as is typical for enzyme replacement therapies.

UX007 named patient revenue in the fourth quarter was $1.2 million and was $3.3 million for the year. We also recognized $0.1 million in revenue this quarter and $0.5 million for the year from our research agreement with Bayer.

As we have stated previously, we continue to expect revenues from this agreement to be minimal going forward. Our total operating expenses were $130 million for the fourth quarter of 2019. For the past several quarters, up to 20% of our operating expenses, excluding expenses related to business development transactions like genetics and Arcturus has consisted of noncash items.

Our research and development costs were $83.1 million. We expect our R&D cost to increase moderately over the time as we continue advancing product candidates from early preclinical development into early and pivotal clinical studies.

Our SG&A costs in Q4 were $41.9 million. We expect SG&A to increase moderately over time as we support our commercial programs simultaneously launching across multiple geographies.

Our cost of sales were $5.1 million for the fourth quarter of 2019. This includes a $3.8 million reserve on Mepsevii inventory that did not meet our quality standards.

Recalling the third quarter of 2019, there was a $1.9 million reserve for a similar issue. We expect that a majority of these reserves will be recovered from our supplier, and we do not currently anticipate any supply interruptions or future reserves related to this issue.

Net loss for the fourth quarter of 2019 was $93.8 million or [$1. 62] per share, basic and diluted compared with a net loss of $87.8 million or $1.73 per share basic and diluted for the fourth quarter of 2018. For the year ended December 31, 2019, net loss was $402.7 million or $7.12 per share, basic and diluted, compared with a net loss for the same period in 2018 of $197.6 million or $3.97 per share basic and diluted.

The net loss for the fourth quarter of 2019 and for the year ended December 31, 2019, includes unrealized gains of $1.4 million and $13.4 million, respectively, from their fair value adjustment on the investment in our Arcturus equity securities.

The net loss for the full year ended 2018 was reduced by $170.3 million due to sales of priority review vouchers.

For the year ended December 31, 2019, cash used in operations was $345.4 million. This includes $20 million for the GeneTx upfront payment in the third quarter of 2019, $15.6 million for the amended Arcturus license rights in the second quarter of 2019 as well as adjustments for significant noncash charges, including stock-based compensation expense of $82 million.

We ended the fourth quarter of 2019 with $760.4 million in cash, cash equivalents and available for sale investments. This includes proceeds of $320 million we received from the sale of the company's royalty interest in Crysvita in the European territory.

Moving to our guidance for 2020. We continue to expect the Crysvita revenue to Ultragenyx in our territories to be between $125 million and $140 million. Those territories include North America, Latin America and Turkey and exclude the EU royalty, as this was monetized in the transaction that was completed with Royalty Pharma that was announced in December 2019. We expect the pace of our revenue growth to significantly exceed the pace of expense growth. And therefore, we are projecting a greater than 20% decrease in net cash burn, which includes net cash used in operations as well as capital expenditures.

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Emil D. Kakkis, Ultragenyx Pharmaceutical Inc. - President, CEO & Director [5]

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Thank you, Shalini. I'll spend a few minutes on our clinical and preclinical programs before turning to the upcoming catalysts. I'll start with Crysvita for tumor-Induced Osteomalacia a rare disease for which approximately half of the patients have tumors that cannot be surgically removed and leading them with no other current treatment options.

In December of last year, we submitted a supplemental biologic license application ahead of our anticipated timing. We expect to hear back from FDA on submission acceptance and review designation later this month.

Turning to UX007 for LC-FAOD. A devastating set of diseases with a high mortality rate despite newborn screening and current use of MCT oil. The FDA is currently reviewing the new drug application and set a PDUFA date for July 31, 2020. As we've discussed before, the FDA does not currently plan to hold an advisory committee meeting to discuss the application. The review process continues on track, and we expect to review decision by the PDUFA date.

In addition to progress in U.S., we've also submitted a marketing authorization application to regulatory authorities in Brazil, and we continue discussion with other regulatory authorities in the EU and Canada.

Based on our experiences, we know that there are a lot of patients with LC-FAOD, who are not doing well on current treatment of MCT oil and are seeking new treatment options. In France alone, for example, there were originally only a few doctors requesting UX007 via the ATU named patient program. Now there are approximately 20 physicians treating 34 patients for the LC-FAOD, who are using UX007 through that named patient program. We expect there to be significant interest in the product, if approved, but as with many inborn air products, we believe, will build steadily and will take time.

In the developed world, there are approximately 8,000 to 14,000 patients with LC-FAOD and we own the worldwide rights to the product.

Moving to DTX301, our gene therapy program for ornithine transcarbamylase deficiency or OTC deficiency. OTC deficiency is an X-linked urea cycle disorder that limits the body's ability to detoxify ammonia into urea. These patients can quickly deteriorate into full metabolic crisis, causing neurologic deficits, hospitalization, coma, in some cases, death.

In January, we reported positive data from dose Cohort 3 and longer-term data from the first 2 cohorts of our OTC study.

In Cohort 3, we are seeing responses from all 3 patients. 2 of the patients are confirmed responders, and the third patient appears to be responder as well, but we will wait until we have longer-term data to confirm this.

In total, up to 6 of the 9 patients have responded. Importantly, 3 patients have come off their ammonia scavenger medications and liberalize their diet. We consider these patients complete responders and these patients appear to be metabolically cured.

Based on these data and the favorable safety profile, we believe the Cohort 3 1e13 GC per kilogram dose is the appropriate dose level. We're seeing a more consistent response across the patients, and we believe this higher dose has achieved the adequate level of therapeutic effect. From here, we will enroll a fourth cohort at the same dose of Cohort 3, this time using prophylactic steroids rather than reactive steroids. We believe this will enhance the level of expression, also provide more consistent expression. We expect data in the second half of 2020 from this cohort. If positive will proceed to dose 3 more patients and simultaneously discuss the design of the Phase III study and end points with regulators.

Based on our ongoing conversation with FDA, we expect that ammonia will be a primary endpoint. The FDA considers ammonia validated clinical endpoint, and they've approved other products based on ammonia.

Switching to DTX401, our gene therapy program in glycogen storage disease type Ia, a disease that leads to severe and sometimes life-threatening hypoglycemia. Patients with GSDIa today have to take cornstarch every 3 to 4 hours, which can keep glucose levels up. But it does not address the disease and its long term consequences. While cornstarch therapy has same lives and improved health, it is not a normal life by any measure and patients or their parents live in fear of death, if they miss a single dose of cornstarch.

Today, we've shown data from the first 2 cohorts of all 6 patients demonstrating a meaningful clinical response to the therapy at the 2e12 and 6e12 dose levels. This includes improvement in glucose control, shown by timed hypoglycemia reductions in cornstarch requirements for all patients. In the second dose cohort, all patients showed a meaningful reduction in glycogen storage and improvements in metabolism. These days data indicate that Cohort 2 dose is showing greater transgene expression and our view that these patients have greatly improved glucose control. They are weighing down their starch requirements, and we think we have a treatment that could change the future of GSDIa patients.

We've now moved to a confirmatory cohort of 3 patients at the same dose and are simultaneously having discussions with the FDA about the Phase III study. We expect to have data from the confirmatory cohort in the first half of 2020. And we could be in a position then to begin Phase III in the second half of 2020.

I will also touch on our agreement with the [gene genetics] Biotherapeutics to advance GTX-102, an antisense oligonucleotide for the treatment of Angelman syndrome. Angelman is a devastating neurologic disease that affects approximately 60,000 patients worldwide, and there are no approved treatment options today. Disease is not neurodegenerative. So there is potential to reverse some disease symptoms, which include speech, cognitive impairment, seizures, ataxia and sleep dysfunction. As a result, we think Angelman's one of the disease in neurology that could benefit most from a treatment. The disease mechanism is well understood, and ASOs (inaudible) can target the disease directly. We believe that the team at GeneTx has developed a very potent and specific differentiated antisense oligonucleotide. We are excited to partner with this group.

The IND for this program is now active, and GeneTx has received IRB our Institutional Review Board approval for the first study site. We expect enrollment in the Phase I/II study to begin in the coming months. Following the acceptance of IND, we paid a $25 million milestone to obtain the option to maintain the option to acquire the company until the earlier of 30 months after the first patient's dose or 90 days after the results are available from the Phase I/II study.

The last product I'll discuss is DTX201 for hemophilia A. Our program is partnered with Bayer and used in material for our proprietary HeLa manufacturing platform.

At the European Association of Hemophilia and Allied Disorders meeting last week, Bayer presented data on first 2 low dose cohorts of the Phase I/II study. All 4 patients showed a response with 3 of the 4 patients showing clinically meaningful increase in Factor VIII levels. One patient Cohort 1 achieved clinically meaningful Factor VIII levels and has experienced only 4 bleeds posttreatment compared to 99 bleeds the prior year. Both patients in dose Cohort 2 achieved clinically meaningful Factor VIII levels up to 24 and 30 weeks.

Patient 4 on Cohort 2 has been bleed-free and treatment-free for up to 7 months of the data cutoff. The same patient had a mild ALT/AST elevations that were managed with a short tapering course of steroids. And the other patients have not required steroids at all. A third high-dose cohort has been dosed, and we expect to see additional updates this year.

While Bayer is responsible for the clinical execution of the program, we are pleased to see that our HeLa manufacturing platform validated and looked forward to continued progress with the program. As a reminder, we are eligible to see milestones and royalty payments from Bayer for this program.

I'll spend a few minutes now discussing a number of important milestones in the coming months that will continue to drive our progress, and then we can move to Q&A.

For Crysvita, in 2020, we expect revenue between $125 million to $140 million across North America, Latin America and Turkey, representing a 58% to 77% increase versus 2019 in the same territories. This will be driven by continued strong performance in the U.S. and expansion of our reach in Latin America through named patient sales and pending regulatory decisions as well as growth in Canada. With our Rare transmission for Crysvita for the treatment of TIO, we are looking to expand procedures in this additional patient population, while there are fewer patients with TIO, there's often a very urgent need for treatment. If approved in this indication, we believe Crysvita therapy will be adopted over phosphate therapy.

For UX007, we will continue to work with the FDA, to view our NDA, working towards the PDUFA date of July 31, 2020. The review is progressing well, and we look forward to being able to provide the stream to many more patients with LC-FAOD. For the gene therapy programs, we have shown strong data for our 2 programs in GSDIa and OTC. In the both phase, we believe we had found the appropriate dose.

The GSDIa program will have a data readout from the confirmatory cohort in the first half, and the OTC program will read out in the second half. We are simultaneously having discussions with FDA about the Phase III studies for both programs.

The Bayer hemophilia A program is writing us with the first clinical data using material from our proprietary HeLa platform, our Wilson disease program will use this HeLa manufacturing system when it enters the clinic, and we are targeting an IND for this program by the end of 2020. We'll also provide more updates on the GTX102 ASO program for Angelman as the program begins to enroll patients.

To summarize briefly, our commercial team continues to execute at an extremely high level, making Crysvita one of the top rare disease launches. The continued efforts with Crysvita and Mepsevii as well as 2 more potential launches this year set us up to grow -- substantially grow our commercial business.

In 2019, we had annual revenue exceeding $100 million for the first time, with substantial growth expected in 2020. We're now well capitalized with $760 million in cash and equivalents, we combined with the financial discipline we are applying and expect to reduce net cash burn in 2020. This puts us in good position to drive our clinical programs forward.

Our gene therapy programs are advancing to a confirmatory dose cohorts through Phase III studies and the Angelman, Wilson disease programs are both large indication opportunities that are nearing clinic with diverse set of early-stage product candidates to follow. We have become a diversified rare disease company, we'll continue to grow. We're constantly innovating, adapting where disease drug developments, strategies, trial designs, and endpoints, working with regulators, establish a more efficient model for rare disease drug development as well as evolving the way we commercialize product in these indications and efficiently manage the cost structure. These are just some of the things we do each day, and we have the foundation while we build an exceptional rare disease company.

With that, let's move to your questions. Operator, can you please provide the instructions for the Q&A portion of the call.

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Questions and Answers

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Operator [1]

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(Operator Instructions) And our first question will come from the line of Gena Wang from Barclays.

--------------------------------------------------------------------------------

Huidong Wang, Barclays Bank PLC, Research Division - Research Analyst [2]

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I wanted to congratulate you on the great quarter. I have 2 questions. The first one is regarding the Crysvita 2020 revenue guidance. Just wondering the 58% to 77% growth is mainly driven by U.S. growth or geographic expansion? How much growth assumption was building for Latin America.

My second question is regarding the Angelman Syndrome. Could you walk through the Phase I trial design in terms of initial dose? And how would your dose escalate? And what kind of data will lead your decision to acquire GeneTx?

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Emil D. Kakkis, Ultragenyx Pharmaceutical Inc. - President, CEO & Director [3]

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Great. So on the Crysvita launch growth, Shalini do you want to answer that particular one, how the numbers...

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Shalini Sharp, Ultragenyx Pharmaceutical Inc. - CFO & Executive VP [4]

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Link:
Edited Transcript of RARE earnings conference call or presentation 13-Feb-20 10:00pm GMT - Yahoo Finance

Recommendation and review posted by Bethany Smith

Global Gene Therapy Market Is Expected To Reach Around USD 2269 Million By 2024 – TheInfobiz

A leading research firm, Zion Market Research added a latest industry report on "Global Gene Therapy Market" consisting of 110+ pages during the forecast period and Gene Therapy Market report offers a comprehensive research updates and information related to market growth, demand, opportunities in the global Gene Therapy Market.

According to the report the Global Gene Therapy Market Is Expected To Reach Around USD 2,269 Million By 2024

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The Gene Therapy Market report mainly includes the major company profiles with their annual sales & revenue, business strategies, company major products, profits, industry growth parameters, industry contribution on global and regional level.This report covers the global Gene Therapy Market performance in terms of value and volume contribution. This section also includes major company analysis of key trends, drivers, restraints, challenges, and opportunities, which are influencing the global Gene Therapy Market. Impact analysis of key growth drivers and restraints, based on the weighted average model, is included in this report to better equip clients with crystal clear decision-making insights.

The Gene Therapy Market research report mainly segmented into types, applications and regions.The market overview section highlights the Gene Therapy Market definition, taxonomy, and an overview of the parent market across the globe and region wise.To provide better understanding of the global Gene Therapy Market, the report includes in-depth analysis of drivers, restraints, and trends in all major regions namely, Asia Pacific, North America, Europe, Latin America and the Middle East & Africa, which influence the current market scenario and future status of the global Gene Therapy Market over the forecast period.

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Global Gene Therapy Market Is Expected To Reach Around USD 2269 Million By 2024 - TheInfobiz

Recommendation and review posted by Bethany Smith

Extraordinary growth in Gene therapy market is expected to reach US$ 5,609.9 million by 2027, growing at an estimated CAGR of 8.2% over the forecast…

Absolute Markets Insights has added comprehensive analytical data to its massive repository titled Gene therapy market. The report highlights significant market players operating in global regions such as North America, Latin America, Europe, Asia-Pacific, and India. Different graphical presentation techniques such as charts, graphs, table, and pictures have been used while curating the report.

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In terms of revenue, global gene therapy market was valued at US$ 919.6 million in 2018 and is anticipated to reach US$ 5,609.9 million by 2027, growing at a CAGR of 8.2% over the forecast period. Market participants are adopting partnerships or acquisition as their strategy to strengthen their foothold. For instance, Pfizer Inc. acquired Medivation, Inc. and Bamboo Therapeutics, Inc. to develop a focused product for the treatment of patients with rare diseases related to neuromuscular and central nervous system. Companies are building relationships with community and patients to understand the disease and design therapies accordingly.

Lethal diseases like cancer can be treated using gene therapy by inserting the antisense strands to revert the effect of the oncogenes using bio engineered vectors. Recently, scientists developed bionic chip to transfer DNA to cells using electroporation technique. During the forecast period, nanoparticles will play an important role in gene delivery systems to increase the efficiency of transfection of the non-viral carriers, thereby, fuelling the gene therapy market.

Due to drastic shift in treatment patterns, gene therapy treatment is considered one of the reliable cures for lethal diseases. The vectors or the DNA carriers are safer and have improved in terms of carrying genes without rejection which help the companies to attract venture capitalists to invest more in gene therapy market. Most of the research companies are focusing on development of gene carriers for the successful gene delivery. One of the prominent used vectors among the gene vehicle family is adeno associated virus. Cancer and Sensory disorders are the major area of concern that need to be fixed and hence drug development related to these disease is driving the gene therapy market.

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Gene Therapy Market:

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Extraordinary growth in Gene therapy market is expected to reach US$ 5,609.9 million by 2027, growing at an estimated CAGR of 8.2% over the forecast...

Recommendation and review posted by Bethany Smith

Are stem cells really the key to making humans live longer? – Wired.co.uk

To some longevity acolytes, stem cells promise the secret to eternal youth. For a hefty fee, you can pay a startup to extract your own stem cells and cryogenically freeze them, in the hope that they can one day be used in a treatment to help extend your life.

Other firms let you bank stem cells from your babys umbilical cord and placenta after childbirth, if youre convinced the high cost represents an insurance policy against future illness. Or you can follow the example of Sandra Bullock and Cate Blanchett and opt for an anti-ageing cream made with stem cells derived from the severed foreskins of newborn babies in South Korea.

Stem cells are the parent cells which give rise to other cells in our bodies. Since scientists first isolated human embryonic stem cells in a lab and grew them over 20 years ago, they have been mooted as a source of great hope for regenerative medical treatments, including for age-related degenerative conditions such as Parkinsons, Alzheimers, heart disease and stroke.

But apart from a few small-scale examples, the only stem cell-based medical treatment practised in clinics uses haematopoietic stem cells found in the blood and bone marrow which only produce blood cells for transplants in blood cancer patients. These cells are taken from a patients sibling or an unrelated donor, before being infused into a patients blood, or theyre taken from a patients own blood before being reinfused. The procedure has been used to treat blood malignancies for almost half a century, and recently multiple sclerosis too. So how likely is it that the predictions about stem cells' longevity-enhancing powers will become a reality?

In September 2019, Google banned ads for unproven or experimental medical techniques such as most stem cell therapy, citing a rise in bad actors attempting to take advantage of individuals by offering untested, deceptive treatments [that can often] lead to dangerous health outcomes. The decision was welcomed by the International Society for Stem Cell Research, which emphasised that most stem cell interventions remain experimental. Selling treatments before well-regulated clinical trials have been done, the body said, [threatens public] confidence in biomedical research and undermines the development of legitimate new therapies.

Its easy to see how less scrupulous companies can exploit the allure of stem cells, which seem to occupy a place in our collective consciousness as a kind of magical elixir. High hopes for stem cell-based therapies have grown since 2006, when the Japanese biologist Shinya Yamanaka created a new technology to reprogram adult cells, such as skin cells, into a similar state to embryonic stem cells, which are pluripotent, meaning they can develop into any tissue in the body. The Nobel prize-winning breakthrough was hailed as a major step in the study of stem cells without the need for controversial embryo research, and towards the use of these human induced pluripotent stem cells to regenerate damaged or diseased organs or effectively grow new spare parts which could treat the life-limiting and life-shortening illnesses associated with ageing.

Gerontologist Aubrey de Grey, whose Strategies for Engineered Negligible Senescence (SENS) research foundation aims to eliminate ageing-related diseases, thinks the chances well soon have stem cell based therapies are high. For anything that's in clinical trials, you're talking about maybe five years before it's available to the general public, he says, citing stem cell treatments for Parkinsons disease, currently being tested in phase two clinical trials, as one of the developments he thinks is likely to come soonest.

However, given that these trials involve a relatively small number of participants and most clinical trials ultimately fail, his predictions might be overly optimistic. Often described as a maverick, De Grey believes that humans can live forever and there is a 50 per cent chance medical advances of which stem cell therapies will play an important part will make this a reality within the next 17 years. Though living forever, he says, is not the ultimate goal but a rather large side effect of medicine which will successfully prevent or repair the damage that comes with ageing.

For New Jersey-based Robert Hariri, who co-founded Human Longevity Inc, which set its sights more modestly on making 100 the new 60, stem cells derived from placentas present especially exciting opportunities. A biomedical scientist, surgeon and entrepreneur, Hariri says his current venture Celularity which is focused on engineering placental cells, including stem cells, to create drugs for cancer and other conditions is not as concerned about the actual age number, but about preserving human performance as we age and treating the degenerative diseases that rob us of our quality of life.

Many of those working in the field, however, remain cautious in their optimism. Researchers have highlighted the potential risks of giving pluripotent cells to patients, whether they are induced or embryonic, as these cells can develop cancer-causing mutations as they grow.

Davide Danovi, a scientist at Kings College Londons Centre for Stem Cells & Regenerative Medicine, says the path to stem cell-based therapy is very long and full of hurdles. The supply chain involves challenges, he says. On the one hand, allogeneic treatments those with stem cells derived from one individual and expanded into big batches to create cells to treat many individuals have the advantage of being similar to the traditional pharmaceutical business models. The product is clear, its something that comes in a vial and can be scaled up and mass produced, Danovi says. But this treatment can present a greater risk of rejection from the patient, as opposed to the more bespoke autologous option which is more expensive and time-consuming as it involves extracting a patients own stem cells before reprogramming them.

Danovi is most excited by the potential of stem cells to treat age-related macular degeneration. In 2017 Japanese scientist Masayo Takahash led a team that administered transplants of artificially grown retinal cells created from induced pluripotent stem cells taken from donors to five patients with the eye condition, which can cause blindness, and theyre reported to be doing well. The eye, he says seems to be a place where immunity plays less of a role relative to other issues, so you can host cells which come from another individual with fewer problems [of rejection]. But, with other organs such as the liver, he says there are major conceptual problems with creating enough tissue. Its like the clean meat burger - you're talking about a production that is, in many cases, not easy to reach with the current technology.

Hariri believes placentas will solve some of the production challenges crucially, theyre an abundant commodity, with the vast majority thrown out after childbirth. His interest was sparked 20 years ago when his oldest daughter was in the womb: When I saw her first ultrasound in the first trimester, the placenta had already developed into a relatively sizable organ, even though she was just a peanut-sized embryo. Id been taught that the placenta was nothing more than an interface, but [if that was the case], you would expect that it would grow at the same rate as the embryo. His curiosity piqued, he began to see the placenta not as an interface but as a biological factory, where stem cells could be expanded and differentiated to participate in the development of that foetus. That intrigued me and I started to collect placentas and just, you know, basically disassemble them.

Placentas have numerous benefits, he says they dont carry the same ethical controversy as embryonic stem cells, for one thing. Scientists working on embryonic stem cells have to destroy an early embryo, and that option yields them a dozen cells, which have to be culture-expanded in the laboratory into billions of cells. In contrast, the placenta houses, billions and hundreds of billions of cells, which can be expanded as well, but you're starting out with a dramatically larger starting material.

Increasingly, scientists in the anti-ageing sphere are focusing on an approach that seems like the opposite of planting fresh stem cells into our bodies. Experts such as Ilaria Bellantuono at Sheffield Universitys Healthy Lifespan Institute are working towards creating senolytics medication that could kill off our senescent cells, the zombie cells that accumulate in tissues as we age and cause chronic inflammation. I think stem cells are very good for specific disease, where the environment is still young, Bellantuono says, but the data in animal models tells us that senolytics are actually able to delay the onset and reduce the severity of multiple diseases at the same time for example, there is evidence for osteoarthritis, osteoporosis, cardiovascular disease, Alzheimer's, Parkinson's, and diabetes. She explains that while human trials are still in their early stages, senolytics are likely to be more cost-effective than stem cell therapy and the status quo of older patients taking multiple pills for multiple diseases, which can interact with each other. Besides, she adds, they may actually work in tandem with stem-cell based therapies in the future, with senolytics creating a more hospitable environment in tissues to allow stem cells to do their work.

And as for the so-called penis facial? Its far from the only ultra-expensive stem cell skincare making bold anti-ageing claims but youre probably better off saving your money, as you are with the experimental medical treatments on offer. Stem cells are definitely exciting but theyre not the key to eternal youth. At least, not yet.

Robert Harari will be one of the speakers at WIRED Health in London on March 25, 2020. For more details, and to book your ticket, click here

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Are stem cells really the key to making humans live longer? - Wired.co.uk

Recommendation and review posted by Bethany Smith

Sensorized skin helps robots understand where the hell they are – The Next Web

Clumsy robots have been offered hope of improving their coordination after MIT researchers found a new way to help them find their bearings.

The systemgives soft robots a greater awareness of their movements by analysing motion and position data through a sensorized skin.

It works by collecting feedback fromsensors on the robots body.A deep learning model then analyses the data to estimate the robots 3D configuration.

[Read:Scientists used stem cells to create a new life-form: Organic robots]

The sensors are comprised of conductive silicone sheets, which the researchers cut into patterns inspired by kirigami a variation of origami that that involvescutting as well as folding paper. These patterns make the material sufficiently flexible and stretchable to be applied to soft robots.

A deep neural network then captures signals from sensorsto predict the best configuration for the robot.

The system aims to overcome the problem of controlling soft robots that can move in countless direction by giving themproprioception an awareness of their position and movements.It could eventually make artificial limbs better at handling objects.

The researchers used the system to teach an elephant trunk-shaped robot to predict its own position as it rotated and extended.

We want to use these soft robotic trunks, for instance, to orient and control themselves automatically, to pick things up and interact with the world, said MIT researcherRyan Truby, who co-wrote a paper describing how the system works. This is a first step toward that type of more sophisticated automated control.

Truby admits that the system can not yet capture subtle or dynamic motion. But it could at least reduce the clumsiness that has embarrassed robotkind for decades.

Youre here because you want to learn more about artificial intelligence. So do we. So this summer, were bringing Neural to TNW Conference 2020, where we will host a vibrant program dedicated exclusively to AI. With keynotes by experts from companies like Spotify, RSA, and Medium, our Neural track will take a deep dive into new innovations, ethical problems, and how AI can transform businesses. Get your early bird ticket and check out the full Neural track.

Published February 13, 2020 17:10 UTC

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Sensorized skin helps robots understand where the hell they are - The Next Web

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What makes your hair turn white faster – The Star Online

People usually start gradually greying in their thirties. Once across the age of 50, one will be hard-pressed to successfully disguise ones white hair without paying monthly visits to a hairdresser.

However, medical reports suggest the process of hair colour loss, which scientists call canities or achromotrichia, can be greatly accelerated by persistent acute stress or severe trauma.

Some historians have speculated that the French Queen Marie Antoinettes hair turned white when she heard she was to be guillotined at the height of the French Revolution in 1793.

For the longest time, its been said that stress makes the hair turn white, but until now, there was no scientific basis for this belief.

Our study proved that the phenomenon does indeed occur and we identified the mechanisms involved.

In addition, we discovered a way of interrupting the process of hair colour loss due to stress, said Thiago Mattar Cunha, a researcher affiliated with the Center for Research on Inflammatory Diseases (CRID) a Research, Innovation and Dissemination Center funded by FAPESP and hosted by the University of So Paulos Ribeiro Preto Medical School in Brazil.

The study was conducted in partnership with a group led by Hsu Ya-Chieh, a professor of regenerative biology at Harvard University in the United States.

According to Cunha, the results, published recently in the journal Nature, were partly serendipitous.

We were conducting a study on pain using black C57 mice, a dark-furred laboratory strain, he said.

In this model, we administered a substance called resiniferatoxin to activate a receptor expressed by sensory nerve fibres and induce intense pain.

Some four weeks after systemic injection of the toxin, a PhD student observed that the animals fur had turned completely white.

The experiment was repeated several times until the CRID researchers concluded that the phenomenon was indeed due to the application of resiniferatoxin, a naturally-occurring chemical found in resin spurge (Euphorbia resinifera), a cactus-like plant native to Morocco.

We set out to check the hypothesis that the loss of fur colour resulted from pain-induced stress, Cunha said.

We designed a very simple experiment to see if the phenomenon was dependent on activation of sympathetic nerve fibres.

He explained that the sympathetic nervous system is directly affected by stress.

This division of the autonomic nervous system consists of nerves that branch from the spine and run throughout the body.

It controls the organisms fight or flight response to imminent danger, triggering the release of adrenaline and cortisol to make the heart beat faster, blood pressure rise, respiration accelerate and the pupils dilate, among other systemic effects.

After injecting resiniferatoxin into the mice, we treated them with guanethidine, an anti-hypertensive capable of inhibiting neurotransmission via sympathetic fibres.

We observed that the process of fur colour loss was blocked by the treatment, Cunha said.

In another experiment, neurotransmission was interrupted by the surgical removal of sympathetic fibres.

In this case too, fur colour was not lost in the weeks following pain induction.

These and other experiments conducted by our group demonstrated the participation of sympathetic innervation in achromotrichia and confirmed that pain is a powerful stressor in this model.

But it remained to detail the mechanisms involved, he explained.

Maturing too quickly

Cunha spent a period at Harvard as a visiting professor in 2018-19 with a scholarship from the joint programme Harvard holds with CAPES, the Brazilian Education Ministrys Office for Faculty Development.

In conversations with colleagues, he heard that a Harvard group had made similar discoveries to those of his group at So Paulo, and that their findings were also partly accidental.

Professor Hsu Ya-Chieh invited me to join a project in which the phenomenon was being investigated in more detail.

Shes a leading researcher on processes that control skin stem cell differentiation, Cunha said.

His group already knew by then that pain-related stress was somehow making the melanocyte stem cells in the hair follicle bulb mature too soon.

These cells are responsible for yielding melanin-producing cells. Melanin is the pigment primarily responsible for skin and hair colour.

In a young individual, the cells are undifferentiated like all stem cells, but with ageing, they gradually differentiate.

Once the process is complete, they stop producing the melanocytes that produce melanin, Cunha explained.

We used various methodologies to show that intense sympathetic activity speeds up differentiation significantly.

In our model therefore, pain accelerated the ageing of melanocyte stem cells.

When we started to study this, I expected that stress was bad for the body but the detrimental impact of stress that we discovered was beyond what I imagined, Prof Hsu said.

After just a few days, all of the pigment-regenerating stem cells were lost. Once theyre gone, you cant regenerate pigment anymore. The damage is permanent.

Study lead author and postdoctoral fellow Zhang Bing added: Acute stress, particularly the fight-or-flight response, has been traditionally viewed to be beneficial for an animals survival.

But in this case, acute stress causes permanent depletion of stem cells.

Other systems in the organism are probably affected by intense stress in a similar manner to the hair follicle bulb.

We dont know for sure what the implications are, Cunha said.

Im currently working with other researchers on an investigation of the effects of sympathetic activity in other stem cell subpopulations.

Altered gene expression

RNA (ribonucleic acid) sequencing was one of the methodologies used to explore the mechanisms that promote melanocyte stem cell differentiation.

The researchers used this technology to compare the gene expression profiles of mice that received the injection of resiniferatoxin developing pain, stress and fur colour loss with those of mice injected with a placebo.

We looked for genes whose expression was most altered after stress induction, and one caught our attention: the gene that encodes a protein called CDK (cyclin-dependent kinase).

This is an enzyme that participates in cell cycle regulation, Cunha said.

When the researchers repeated the pain induction procedure and treated the mice with a CDK inhibitor, they found that melanocyte stem cell differentiation was prevented, as was fur colour loss.

This finding shows that CDK participates in the process and could, therefore, be a therapeutic target, he said.

Its too soon to know whether it will actually become a target someday in clinical practice, but its worth exploring further.

In another experiment, the researchers demonstrated that when the sympathetic system is robustly activated, the fibres that innervate hair follicle bulbs release noradrenaline very near the melanocyte stem cells.

We showed that melanocyte stem cells express the protein ADRB2 (beta-2 adrenergic receptor), which is activated by noradrenaline, and we discovered that the stem cells differentiate when this receptor is activated by noradrenaline, Cunha said.

To confirm the finding, the researchers repeated the experiment using mice that had been genetically modified so as not to express ADRB2.

As suspected, their fur did not turn white after they were injected with resiniferatoxin.

In another test, we injected noradrenaline directly into the skin of the mouse.

As a result, the fur around the site of the injection turned white, Cunha said.

Finally, the group treated a primary culture of human melanocytes (melanin-producing cells obtained directly from the skin of a volunteer) with noradrenaline, which as noted earlier, is released by the sympathetic nerve fibres in hair follicles.

The result was an increase in expression of CDK similar to that observed in mice.

According to Cunha, the researchers do not yet know if there will be future aesthetic applications for their findings, such as the development of a drug that prevents the hair colour loss associated with ageing.

It would be necessary to see if a CDK inhibitor has side effects, and if so, whether they would be outweighed by the aesthetic benefit, he said. Agncia FAPESP

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What makes your hair turn white faster - The Star Online

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The ghost behind grey – THE WEEK

Marie Antoinette, the last queen of France, is best remembered for her reckless extravagance and her tragic death. French revolutionaries executed her with a guillotine in 1793 for treason. Popular belief is that her hair turned grey the night before her death.

There are other stories and anecdotes like this which suggest that stressful experiences are correlated with the phenomenon of hair greying. Now, for the first time, Harvard University scientists have found the real mechanism behind it.

Published in the journal Nature, the study was initiated with the aim of analysing the effect of stress on various tissues. Hair and skin are the tissues that are visible from outside. So, the researchers started with effects of stress on hair colour.

Their initial hypothesis was that stress initiates an immune attack on pigment-producing cells in the hair follicles. However, when they tested it on mouse, they found those lacking immune cells (nude mouse) also exhibited hair greying. Then, their attention turned to a hormone called cortisol. It is responsible for regulating a wide range of processes through the body, including metabolism and the immune response. In response to stress, extra cortisol is released to help the body to respond appropriately. Surprisingly, when the researchers removed the adrenal gland from the miceto prevent production of cortisol-like hormone aldosteroneand triggered stress, their hair still turned grey.

Finally, the researchers tested the sympathetic nerves that branch out to each hair follicle. The sympathetic nerve system is responsible for the bodys fight-or-flight response. They found that in a stressful condition, the sympathetic nerves release a chemical called norepinephrine, which is taken up by certain stem cells in the hair follicle. Stem cells act as a reservoir for pigment-producing cells. During hair regeneration, some of these stem cells are converted into pigment-producing cells to give colour to new hair strands.

When these stem cells take norepinephrine, they are activated excessively and all of them get converted into pigment-producing cells. This would prematurely deplete the reservoir for pigment-producing cells. Once all of them are consumed, pigment regeneration would stop, resulting in permanent damage. The fight-or-flight response has been traditionally viewed as beneficial. But now it is proved that it has its own detrimental effects, too.

The study established how neurons interact at the cellular and molecular level to link stress with hair greying. The findings are expected to put light on the broader effects of stress on various body parts. The scientists will initiate new studies that seek to modify or block the damaging effects of stress.

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The ghost behind grey - THE WEEK

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Fountain of Youth within reach without surgery – KIIITV.com

CORPUS CHRISTI, Texas In today's world, millions are spent on cosmetic surgery to keep that healthy glow everywhere we go. However, if you don't have thousands of dollars to pay for it, there are other, more safe alternatives that are non-surgical.

A few years ago I started noticing that no matter how much sleep I got, I just didn't look as fresh as I did when I was younger. I thought maybe it was time to talk to a professional about how I could get back that youthful appearance.

Stress, the daily rigor of life, and gravity all take their toll on the face, and there's nothing wrong with admitting you need a little help to look and feel a little better.

I visited with Dr. Vijay Bingdingdavale, a local cosmetic surgeon, to address my concerns and explore some options. The first thing he suggested was injections to relax my forehead area.

"That'll lift the eyebrows as well. What happens is when we inject these two areas, your eyebrows come a little bit higher, and giving you more of a refreshed look," Dr. Bingdingdavale said.

Then adding fullness to the upper cheeks would bring some balance to my face.

"You see how when you have a little bit more cheek fullness it harmonizes the face? It lifts this and fill this in as well," Dr. Bingdingdavale said.

Using fat transfer as opposed to artificial fillers has an additional benefit.

"We see this a lot, because there are stem cells in the fat, it makes the overlying skin more refreshed and more young-looking," Dr. Bingdingdavale said.

In the end, that's what we all want -- a more refreshed appearance even if we don't get our eight hours every night.

You can catch Dr. Vijay Bingdingdavale on First Edition on Sundays discussing skin care and healthy living.

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Fountain of Youth within reach without surgery - KIIITV.com

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