Gene Therapies Make it to Clinical Trials – Discover Magazine

After years of ethical debates and breakthroughs in the lab, CRISPR has finally made its way to clinical trials. Researchers are now looking at whether the DNA-editing tool, as well as more conventional gene therapies, can effectively treat a wide array of heritable disorders and even cancers.

Theres been a convergence of the science getting better, the manufacturing getting much better, and money being available for these kinds of studies, says Cynthia Dunbar, a senior investigator at the National Heart, Lung, and Blood Institute. Its truly come of age.

CRISPR formally known as CRISPR-Cas9 has been touted as an improvement over conventional gene therapy because of its potential precision. CRISPR (clustered regularly interspaced short palindromic repeats) is a genetic code that, contained in a strand of RNA and paired with the enzyme Cas9, acts like molecular scissors that can target and snip out specific genes. Add a template for a healthy gene, and CRISPRs cut can allow the cell to replace a defective gene with a healthy one.

In April, scientists at the University of Pennsylvania announced they had begun using CRISPR for cancer treatments. The first two patients one with multiple myeloma, the other with sarcoma had cells from their immune systems removed. Researchers used CRISPR to genetically edit the cells in the lab, and then returned them back into their bodies.

On the other side of the country, Mark Walters, a blood and bone marrow transplant specialist at the University of California, San Francisco, Benioff Childrens Hospital in Oakland, is gearing up for trials that will use CRISPR to repair the defective gene that causes sickle cell disease. With CRISPR, once youve made that type of correction, [that cell] is 100 percent healthy, says Walters.

Another team is tackling the same disease using a type of hemoglobin, a protein in red blood cells, thats normally made only in fetuses and newborn babies. Researchers found that some adults continue to produce these proteins throughout their lives, and when those adults also have sickle cell disease, their symptoms are mild. So the international team used CRISPR to disable the gene that interferes with production of this hemoglobin, resuming its production and protecting the adult patients against sickle cell disease.

Several other CRISPR studies are in the works to treat a range of inherited disorders, including hemophilia and SCID-X1 (also known as X-linked severe combined immunodeficiency, the so-called bubble boy disease in which babies are born without a functioning immune system).

At St. Jude Childrens Research Hospital, a gene therapy trial cured Gael Jesus Pino Alva (pictured with his mother, Giannina) of SCID-X1, the bubble boy disease. (Credit: St. Jude Children's Research Hospital/Peter Barta)

The past year also saw success in a handful of experiments on conventional gene therapy. Instead of using CRISPR to repair disease-causing genes, these treatments use hollowed-out viruses to ferry healthy versions of genes into cells. Millions of these altered cells are released into the bloodstream or bone marrow in hopes that enough will land in the right places. But because scientists cant predict where the circulating genes may end up, this shotgun approach has had unintended, sometimes fatal, consequences including, in an earlier study, inadvertently activating leukemia-causing genes in patients treated for SCID-X1.

But in 2019, researchers learned that using a different type of virus one related to HIV to transport the genes may prevent these side effects. In an April study, researchers at St. Jude Childrens Research Hospital in Memphis, Tennessee, and UCSF Benioff Childrens Hospital in Oakland collected bone marrow from eight newborns with SCID-X1. They loaded corrective genes into the disabled HIV-related virus, which carried them into the patients bone marrow stem cells. The infants also received low doses of busulfan, a chemotherapy that gave the doctored stem cells room to grow. So far, we havent seen anything worrisome, says Ewelina Mamcarz, a pediatric oncologist at St. Jude who led the research team. The study recently added its 12th patient.

Gene therapy does have its momentum [back], says Mamcarz, reflecting on the fields setback after the earlier studys leukemia side effects. Theres so much that still needs to be done, and so many questions, she says. [But] this is how medicine evolves. We always want to be better than we were a week ago.

In the future, the hope is that gene therapy technologies will move beyond mending simple genetic mistakes and be used to combat big killers like diabetes or heart disease. [Those diseases are] more challenging, but a lot of them would benefit from knocking out a bad gene, says Dunbar.

For now, though, researchers are optimistic about the progress thats already been made. All of this has been very encouraging, says Dunbar. [And] for sickle cell in the U.S. and hemophilia in the developed world, these diseases may soon be solved.

[This story originally appeared in print as "Gene Therapy Gets Clinical."]

Original post:
Gene Therapies Make it to Clinical Trials - Discover Magazine

What is HLH, and what role did it play in the death of a healthy 34-year-old ESPN reporter? – FirstCoastNews.com WTLV-WJXX

How does a healthy 34-year-old die after being diagnosed with pneumonia?

That's the question at the heart of the tragic and sudden death of an ESPN reporter earlier this week.

According to college football reporter Edward Aschoff's fiance, Katy Berteau, about a week after being diagnosed with a severe form of pneumonia, he began treatment for a "presumed diagnosed" of hemophagocytic lymphohistiocytosis (HLH). Three days after being moved to the ICU, Berteau tweeted from Aschoff's account on Thursday night, he died.

HLH is a rare and complicated condition that's not entirely well-understood by researchers, but it basically stems from your immune system severely overreacting to an infection (such as pneumonia) or another illness.

"It just gets overworked and starts fighting regular tissues, so usually those tissues are organs, like your spleen or your liver," Atlanta area doctor Will Epps said. "So it can result in liver failure or organ failure and unfortunately end up expiring from it."

Here's a general outline:

According to the Genetic and Rare Diseases Information Center at the National Institutes of Health, HLHcomes in two forms: A genetic form and an acquired form.

St. Jude Children's Research Hospitalsays the genetic form is more common in young children, while the acquired form, sometimes called the secondary form, usually affects older children and adults, such as Aschoff.

What happens, broadly, is this: Faced with a severe infection, such as pneumonia (or other severe conditions like cancer), a person's immune system overreacts and, as theJohns Hopkins School of Medicine describes it, certain while blood cells called histiocytes and lymphocytes "attack your other blood cells."

"These abnormal blood cells collect in your spleen and liver, causing these organs to enlarge," Johns Hopkins says.

(According to the NIH, HLH may also be associated with a separate genetic condition X-linked lymphoproliferative disease - XLP - when it results from an inappropriate immune response.)

It can cause death in a matter of weeks, according to researchers.

A2012 paper in the medical journal "Clinical Advances in Hematology & Oncology,"outlines adult HLH extensively.

"It is useful to think of HLH as the severe end of a spectrum of hyperinflammatory diseases in which the immune system causes damage to host tissues," the paper's authors, Dr. Roman Leonid Kleynberg and Dr. Gary J. Schiller, wrote.

That paper estimated HLH occurs in only 1.2 cases per one million individuals every year, making it extremely rare.

Specifically amongst children, St. Jude estimates HLH is diagnosed in fewer than 1 out of every 50,000 - 100,000 children per year.

The condition's mortality rate is difficult to pin down because it can fluctuate based on what caused it. Many sources say a common cause is Epstein-Barr virus, for example, and the 2012 paper reported the mortality rate associated with that 18-24 percent. Other causes can have lower mortality rates.

Generally, doctors try both to treat the underlying trigger for HLH and address the immune response.

"Treating that is tamping that down, either through steroids or with a chemotherapeutic agent which tends to attack or lower that immune system," Epps said.

John Hopkins Medicine also details antibiotic and antiviral drugs being used, or if drug treatment fails doctors may turn to stem cell transplants.

There is no known way to prevent HLH.

More here:
What is HLH, and what role did it play in the death of a healthy 34-year-old ESPN reporter? - FirstCoastNews.com WTLV-WJXX

2019: The year gene therapy came of age – INQUIRER.net

For decades, the DNA of living organisms such as corn and salmon has been modified, but Crispr, invented in 2012, made gene editing more widely accessible. Image: YinYang/IStock.com via AFP Relaxnews

In the summer, a mother in Nashville with a seemingly incurable genetic disorder finally found an end to her suffering by editing her genome.

Victoria Grays recovery from sickle cell disease, which had caused her painful seizures, came in a year of breakthroughs in one of the hottest areas of medical research gene therapy.

I have hoped for a cure since I was about 11, the 34-year-old told AFP in an email.

Since I received the new cells, I have been able to enjoy more time with my family without worrying about pain or an out-of-the-blue emergency.

Over several weeks, Grays blood was drawn so doctors could get to the cause of her illness stem cells from her bone marrow that were making deformed red blood cells.

The stem cells were sent to a Scottish laboratory, where their DNA was modified using Crispr/Cas9 pronounced Crisper a new tool informally known as molecular scissors.

The genetically edited cells were transfused back into Grays veins and bone marrow. A month later, she was producing normal blood cells.

Medics warn that caution is necessary but, theoretically, she has been cured.

This is one patient. This is early results. We need to see how it works out in other patients, said her doctor, Haydar Frangoul, at the Sarah Cannon Research Institute in Nashville.

But these results are really exciting.

In Germany, a 19-year-old woman was treated with a similar method for a different blood disease, beta thalassemia. She had previously needed 16 blood transfusions per year.

Nine months later, she is completely free of that burden.

For decades, the DNA of living organisms such as corn and salmon has been modified.

But Crispr, invented in 2012, made gene editing more widely accessible. It is much simpler than preceding technology, cheaper and easy to use in small labs.

The technique has given new impetus to the perennial debate over the wisdom of humanity manipulating life itself.

Its all developing very quickly, said French geneticist Emmanuelle Charpentier, one of Crisprs inventors and the cofounder of Crispr Therapeutics, the biotech company conducting the clinical trials involving Gray and the German patient.

Cures

Crispr is the latest breakthrough in a year of great strides in gene therapy, a medical adventure started three decades ago, when the first TV telethons were raising money for children with muscular dystrophy.

Scientists practicing the technique insert a normal gene into cells containing a defective gene.

It does the work the original could not such as making normal red blood cells, in Victorias case, or making tumor-killing super white blood cells for a cancer patient.

Crispr goes even further: instead of adding a gene, the tool edits the genome itself.

After decades of research and clinical trials on a genetic fix to genetic disorders, 2019 saw a historic milestone: approval to bring to market the first gene therapies for a neuromuscular disease in the United States and a blood disease in the European Union.

They join several other gene therapies bringing the total to eight approved in recent years to treat certain cancers and an inherited blindness.

Serge Braun, the scientific director of the French Muscular Dystrophy Association, sees 2019 as a turning point that will lead to a medical revolution.

Twenty-five, 30 years, thats the time it had to take, he told AFP from Paris.

It took a generation for gene therapy to become a reality. Now, its only going to go faster.

Just outside Washington, at the National Institutes of Health (NIH), researchers are also celebrating a breakthrough period.

We have hit an inflection point, said Carrie Wolinetz, NIHs associate director for science policy.

These therapies are exorbitantly expensive, however, costing up to $2 million meaning patients face grueling negotiations with their insurance companies.

They also involve a complex regimen of procedures that are only available in wealthy countries.

Gray spent months in hospital getting blood drawn, undergoing chemotherapy, having edited stem cells reintroduced via transfusion and fighting a general infection.

You cannot do this in a community hospital close to home, said her doctor.

However, the number of approved gene therapies will increase to about 40 by 2022, according to MIT researchers.

They will mostly target cancers and diseases that affect muscles, the eyes and the nervous system.

Bioterrorism

Another problem with Crispr is that its relative simplicity has triggered the imaginations of rogue practitioners who dont necessarily share the medical ethics of Western medicine.

Last year in China, scientist He Jiankui triggered an international scandal and his excommunication from the scientific community when he used Crispr to create what he called the first gene-edited humans.

The biophysicist said he had altered the DNA of human embryos that became twin girls Lulu and Nana.

His goal was to create a mutation that would prevent the girls from contracting HIV, even though there was no specific reason to put them through the process.

That technology is not safe, said Kiran Musunuru, a genetics professor at the University of Pennsylvania, explaining that the Crispr scissors often cut next to the targeted gene, causing unexpected mutations.

Its very easy to do if you dont care about the consequences, Musunuru added.

Despite the ethical pitfalls, restraint seems mainly to have prevailed so far.

The community is keeping a close eye on Russia, where biologist Denis Rebrikov has said he wants to use Crispr to help deaf parents have children without the disability.

There is also the temptation to genetically edit entire animal species malaria-causing mosquitoes in Burkina Faso or mice hosting ticks that carry Lyme disease in the US.

The researchers in charge of those projects are advancing carefully, however, fully aware of the unpredictability of chain reactions on the ecosystem.

Charpentier doesnt believe in the more dystopian scenarios predicted for gene therapy, including American biohackers injecting themselves with Crispr technology bought online.

Not everyone is a biologist or scientist, she said.

And the possibility of military hijacking to create soldier-killing viruses or bacteria that would ravage enemies crops?

Charpentier thinks that technology generally tends to be used for the better.

Im a bacteriologist weve been talking about bioterrorism for years, she said. Nothing has ever happened.IB/JB

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Stem Cells Market is Expected to Expand at an Impressive Rate by 2023 – News Cast Report

A Comprehensive research study conducted byKD Market Insightson Stem Cells Market By Product (Adult Stem Cells, Human Embryonic Stem Cells, Induced Pluripotent Stem Cells, Others), By Application (Regenerative Medicines, Drug Discovery and Development, Others), By Technology (Cell Acquisition, Cell Production, Cryopreservation, Expansion and Sub-Culture), By End-User (Biopharmaceutical, Biotechnology Industry, Research Institutes), By Treatment Type (Allogeneic Stem Cell Therapy, Auto logic Stem Cell Therapy, Syngeneic Stem Cell Therapy), By Banking Type (Public, Private) & Global Region Market Size, Share, Trends and Forecast 2018-2023 report offers extensive and highly detailed historical, current and future market trends in the global and regional/market. TheStem Cells Marketreport includes market size, growth drivers, barriers, opportunities, trends and other information which helps to find new opportunities in this market for the growth of the business through new technologies and developments.

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Stem Cells Market is Expected to Expand at an Impressive Rate by 2023 - News Cast Report

ESPN reporter diagnosed with pneumonia, HLH. What is HLH? – Crossroads Today

Edward Aschoff, a college football reporter for ESPN, died Tuesday on his 34th birthday, according to ESPN Edward Aschoff, a college football reporter for ESPN, died Tuesday on his 34th birthday, according to ESPN Related stories

BRISTOL, Conn. - When ESPN reporter Edward Aschoff died, he had been diagnosed with multifocal pneumonia and a rare disease known as HLH, his fiance tweeted.

Aschoff was first admitted to the hospital and diagnosed with pneumonia in many parts of his lungs but was brought back to the emergency room when antibiotic treatment failed and he got worse, Katy Berteau said.

"After many tests - bone marrow and lung biopsies - treatment was started for a presumed diagnosis of HLH," she tweeted. "Within 3 days of being moved into the ICU, he passed."

HLH, hemophagocytic lymphohistiocytosis, is a rare disease that affects the immune system.

She did not provide any further details about the manner of Aschoff's death, which occurred on his 34th birthday.

Other people, including Aschoff himself, expressed surprise about the seriousness of the illness in a young man in apparently good health.

"Anyone ever had multifocal (bilateral) pneumonia in their early 30s as some who never gets sick and has a very good immune system? Asking for two friends ... my lungs," he tweeted on December 5.

More questions have come up about his second diagnosis, HLH. It is unclear if Aschoff had HLH or pneumonia first, if one came from the other, and exactly how he died so quickly.

Here is what we know about the diseases Aschoff's had:

Pneumonia is when air sacs in the lungs fill with fluid or pus. It can be caused by a virus, bacteria or a fungus, causing a fever and respiratory problems.

It can occur in one or both lungs, and multifocal means the pneumonia occurs in multiple places.

Thousands of people die around the world each year of pneumonia, but most healthy people can fight it off, especially with antibiotics and antiviral medications. The people most at risk are the young, elderly, frail or immune-compromised.

HLH is a rare disease that affects the immune system, making certain white blood cells attack other blood cells and enlarging the spleen and liver, according to Johns Hopkins Medicine.

It can be inherited or acquired, Johns Hopkins said. About a quarter of cases are passed down through families, and the rest come from infections, a weakened immune system and cancer.

Symptoms can include coughing, difficulty breathing, fever, headaches, rashes, swollen lymph nodes, jaundice and digestive problems, according to Johns Hopkins.

There is treatment for HLH, and acquired forms may clear when properly treated, Johns Hopkins said. If familial HLH goes untreated, it is usually fatal.

Treatments include chemotherapy, immunotherapy, steroids, antibiotic drugs and antiviral drugs. Stem cell transplants can cure HLH in most cases if drug treatments don't work, Johns Hopkins said.

There is no way to prevent HLH, the medical center said.

Here is the original post:
ESPN reporter diagnosed with pneumonia, HLH. What is HLH? - Crossroads Today

2019: the year gene therapy came of age – Times of India

WASHINGTON: In the summer, a mother in Nashville with a seemingly incurable genetic disorder finally found an end to her suffering - by editing her genome.Victoria Gray's recovery from sickle cell disease, which had caused her painful seizures, came in a year of breakthroughs in one of the hottest areas of medical research - gene therapy.'; var randomNumber = Math.random(); var isIndia = (window.geoinfo && window.geoinfo.CountryCode === 'IN') && (window.location.href.indexOf('outsideindia') === -1 ); console.log(isIndia && randomNumber "I have hoped for a cure since I was about 11," the 34-year-old told AFP in an email.

"Since I received the new cells, I have been able to enjoy more time with my family without worrying about pain or an out-of-the-blue emergency."

Over several weeks, Gray's blood was drawn so doctors could get to the cause of her illness - stem cells from her bone marrow that were making deformed red blood cells.

The stem cells were sent to a Scottish laboratory, where their DNA was modified using Crispr/Cas9 - pronounced "Crisper" -- a new tool informally known as molecular "scissors."

The genetically edited cells were transfused back into Gray's veins and bone marrow. A month later, she was producing normal blood cells.

Medics warn that caution is necessary but, theoretically, she has been cured.

"This is one patient. This is early results. We need to see how it works out in other patients," said her doctor, Haydar Frangoul, at the Sarah Cannon Research Institute in Nashville.

"But these results are really exciting."

In Germany, a 19-year-old woman was treated with a similar method for a different blood disease, beta thalassemia. She had previously needed 16 blood transfusions per year.

Nine months later, she is completely free of that burden.

For decades, the DNA of living organisms such as corn and salmon has been modified.

But Crispr, invented in 2012, made gene editing more widely accessible. It is much simpler than preceding technology, cheaper and easy to use in small labs.

The technique has given new impetus to the perennial debate over the wisdom of humanity manipulating life itself.

"It's all developing very quickly," said French geneticist Emmanuelle Charpentier, one of Crispr's inventors and the cofounder of Crispr Therapeutics, the biotech company conducting the clinical trials involving Gray and the German patient.

Crispr is the latest breakthrough in a year of great strides in gene therapy, a medical adventure started three decades ago, when the first TV telethons were raising money for children with muscular dystrophy.

Scientists practising the technique insert a normal gene into cells containing a defective gene.

It does the work the original could not -- such as making normal red blood cells, in Victoria's case, or making tumor-killing super white blood cells for a cancer patient.

Crispr goes even further: instead of adding a gene, the tool edits the genome itself.

After decades of research and clinical trials on a genetic fix to genetic disorders, 2019 saw a historic milestone: approval to bring to market the first gene therapies for a neuromuscular disease in the US and a blood disease in the European Union.

They join several other gene therapies - bringing the total to eight - approved in recent years to treat certain cancers and an inherited blindness.

Serge Braun, the scientific director of the French Muscular Dystrophy Association, sees 2019 as a turning point that will lead to a medical revolution.

"Twenty-five, 30 years, that's the time it had to take," he told AFP from Paris.

"It took a generation for gene therapy to become a reality. Now, it's only going to go faster."

Just outside Washington, at the National Institutes of Health (NIH), researchers are also celebrating a "breakthrough period."

"We have hit an inflection point," said Carrie Wolinetz, NIH's associate director for science policy.

These therapies are exorbitantly expensive, however, costing up to $2 million - meaning patients face grueling negotiations with their insurance companies.

They also involve a complex regimen of procedures that are only available in wealthy countries.

Gray spent months in hospital getting blood drawn, undergoing chemotherapy, having edited stem cells reintroduced via transfusion - and fighting a general infection.

"You cannot do this in a community hospital close to home," said her doctor.

However, the number of approved gene therapies will increase to about 40 by 2022, according to MIT researchers.

They will mostly target cancers and diseases that affect muscles, the eyes and the nervous system.

Another problem with Crispr is that its relative simplicity has triggered the imaginations of rogue practitioners who don't necessarily share the medical ethics of Western medicine.

Last year in China, scientist He Jiankui triggered an international scandal - and his excommunication from the scientific community - when he used Crispr to create what he called the first gene-edited humans.

The biophysicist said he had altered the DNA of human embryos that became twin girls Lulu and Nana.

His goal was to create a mutation that would prevent the girls from contracting HIV, even though there was no specific reason to put them through the process.

"That technology is not safe," said Kiran Musunuru, a genetics professor at the University of Pennsylvania, explaining that the Crispr "scissors" often cut next to the targeted gene, causing unexpected mutations.

"It's very easy to do if you don't care about the consequences," Musunuru added.

Despite the ethical pitfalls, restraint seems mainly to have prevailed so far.

The community is keeping a close eye on Russia, where biologist Denis Rebrikov has said he wants to use Crispr to help deaf parents have children without the disability.

There is also the temptation to genetically edit entire animal species - malaria-causing mosquitoes in Burkina Faso or mice hosting ticks that carry Lyme disease in the US.

The researchers in charge of those projects are advancing carefully, however, fully aware of the unpredictability of chain reactions on the ecosystem.

Charpentier doesn't believe in the more dystopian scenarios predicted for gene therapy, including American "biohackers" injecting themselves with Crispr technology bought online.

See the original post:
2019: the year gene therapy came of age - Times of India

This Is How Human Head Transplants Could Be Achieved, According To A Neurosurgeon – IFLScience

The idea of transplanting a human head onto another persons body may sound like the stuff of science fiction and thats because it is. But while penning a fictional story about the worlds first cranial exchange, neuroscientist Bruce Mathew came up with an idea that he says could soon become a real-life procedure.

Speaking to The Telegraph, Mathew who was previously the clinical lead for neurosurgery at Hull University Teaching Hospitals NHS Trust explained that a head could be grafted onto another body if the entire spinal cord is transplanted along with it.

Initially our intention was to just brainstorm an idea and it seemed rather silly, but then I realized, it actually isnt. If you transplant the brain and keep the brain and spinal cord together its actually not impossible, he said.

The spinal cord is the most profound thing imaginable. You need to keep the brain connected to the spinal cord. The idea that you cut the spinal cord is utterly ridiculous."

Obviously this is not an easy thing to do, and while recent advances have opened up the possibility of reattaching individual severed nerves, the prospect of connecting an entire spinal column is still some way out of reach.

Yet with surgical technologies improving at a rapid rate, Mathew says it is not entirely unrealistic to think that it will probably happen in the next 10 years.

At the moment, you can connect one or two nerves, but with robotics and artificial intelligence well soon be able to do 200 nerves, he explained.

Of course, there are likely to be many complications with such a procedure, as the recipients body will probably reject such a large amount of donor material. While Mathew hasnt figured out all the solutions in detail, he says that transferring gut bacteria along with the head and spinal cord, and stem cell transplants, may help to ensure that the transplant is accepted.

At present, Mathew has no plans to take his idea any further than the pages of his science fiction novel, although Italian neurosurgeon Sergio Canavero has spent the last few years actively attempting to achieve a human head transplant.

In 2017 he announced that he had successfully transplanted the head of one human corpse onto another, and previously claimed to have grafted a donor head onto a monkey although the animal never regained consciousness and would probably have been paralyzed if it had, as the spinal cord remained unattached.

Despite this, Canavero apparently has a willing human donor, and of course, there are thousands of people (and sometimes just heads) across the world cryonically frozenin the hope medicine and technology of the future will be able to revive them. Perhaps it will happen one day, but we're not quite there yet.

See the original post here:
This Is How Human Head Transplants Could Be Achieved, According To A Neurosurgeon - IFLScience

Taiwan Healthcare+ Expo 2019: A 360 exposure to MedTech and Therapeutic world – BSA bureau

Taiwan Healthcare provides convenient access to certified and high-quality medical services supported by its burgeoning biotechnology industry.

The Taiwan Healthcare+ Expo T2019 was an enriching exposure to advanced medicine and digital technology which had an engaging encounter with practitioners of the healthcare sectors, medical professionals and MedTech explorers from around the world during 3rd to 6th Dec 2019, at Taipei Nangang Exhibition Center.

Expo 2019 witnessed more than 173,705 visitors, 2800+ international professionals, 52 hospital & medical institutes, 28 industry associations, 12 medical conferences and much more knowledge sharing platforms. Expo also convened 25,000 professional visitors including hospital decision-makers, buyers, distributors, Venture capitalists, company executives as 600 exhibitors hosting in 1,850 booths from 17 countries.

Healthcare + Expo Taiwan was the key Asia Pacific forum for interdisciplinary collaboration between the medical, health, IT / electronics, and bio-pharmaceutical sectors, which provides a common stage for the development of medical technology, innovation and new ventures.

Medicine and technology being the key drivers, Healthcare+ Expo have seen rapid growth in size and significance of procurement, networking, and marketing since its inception in 2015. The expo encourages global participation from buyers, enterprises, Hospital management executives, health industry BD executives, distributors, medical agencies, and venture capitalists to showcase smart healthcare and Hospital equipments. More than 2800 international professionals from Europe (4.1%), Southeast/South Asia (47.2%), Northeast Asia (11.3%), America (9.3%), China, Hong Kong, Macao (20%), Middle East (2%) explored the offering of modern health care technology.

Australia-Taiwan Biomedical & Digital Health Forum:

A forum organized by Australian authorities, Chine Medical University Hospital and Research Center for Biotechnology and Medicine Policy explored the landscape in bio-innovations and digital health development in Australia and Taiwan, with a view to identifying and developing opportunities for collaborations. The Australian and Taiwanese speakers shared their expertise and insights in their areas of specialization to unleash the potential MedTech capabilities to leverage innovative health technologies and solutions.

The Global leading medical system innovator and prime players in therapeutic solutions viz. Hitachi, IBA, Varian and Sumitomo launched the symposium featuring the latest developments, applications and business models of advanced therapies

Exhibition Themes

The expo had 7 exhibition themes, hospitals/medical institute (32%), smart healthcare (20%), preventive medicine/ health management (17%), depicting bio-innovation (12%), biotech/ pharmaceuticals (7%), genetic medicine/cell therapy (7%), Bio agriculture (5%).

Hospitals and medical institutes: Halthcare+ Expo 2019 hosted the Hospitals and medical institutes from the diversified sectors which offer advanced medical technology and surgery solutions, medical specialities, speciality diagnosis, nursing care services, hospital management, and health IT system.

Smart healthcare and hospital equipments: Advances in healthcare AI and technology are uplifting the medical facilities with smart hospital solutions and system, mobile healthcare devices, sensors and wearable products, hospital equipments, diagnosis, monitoring device and surgical products.

Preventive medicine/health management: Focusing majorly on Personal health management, health evaluation/ promotion services, disease prevention supplies, health supplements, sport medicine, assistive technologies, training products, long term care organisations, assisted living and nursing homes, speciality clinical, pharmacies, medical insurance, home healthcare, telemedicine, home-use medical devices and rehabilitation product

Bio innovation: Innovative technologies from academic and research institutes and start-ups, R&D services, collaborations, and incubator services, financial, intellectual property, commercialisation, and regulatory consulting services

Biotech /pharmaceutical: The key industries for the production of any active pharmaceutical ingredients (APIs), CMOs, CROs, CDMOs, new drugs, biologics, vaccines and generics, new drug development, testing, manufacturing equipment, and pharmaceutical plant construction services

Genetic medicine/ cell therapy: An elevated interest in precision medicine has encouraged the procedures like genetic testing, screening, and disease risk assessment, biomarker-based diagnostics, reagents, and instruments, cell therapies, stem cell storage services, regenerative medicine, and related reagents, instruments

Bio-agricultural: Development of natural ingredients and cultivation and production of organic products, bio-detection reagents, products, and services for plants, animals, and food safety, animal feed activities, drugs and vaccines and plant growth regulators

An interactive platform beneficiary to every attendee

Global professionals gathered at the Taiwan Expo 2019 with the purpose of sourcing the products and their service (24.1%), source partnering (18.8%), purchase products and their services (6.1%), to develop a business network (15.5%) and to gather market and sector intelligence (31.5%).

Bio B2B platform: Taiwan Expo B2B platform was designedaccurately for bio-business professionalsto find value-added partnerships in Taiwan in fields ranging from APIs, pharmaceuticals, and medical devices, to bio-agricultural and health supplement companies. Bio B2B connected professionals and industrialists seeking new pipelines and technology platform, innovative and high-quality bio-medical products, manufacturing-distributing or contract research services with the suitable bio-medical companies in Taiwan that fit their specific needs.Expo 2019 assisted the aspirants in compiling, selecting, and making available remarkable achievements and technological developments originated in Taiwan in medicine and biotechnology sectors.

A hope to Patients: The Expo platform was a demo guide to the world-class medical facilities Taiwan Healthcare network can offer to patients. Taiwan is known for its high-quality niche medical services such as liver transplantation, craniofacial surgery, artificial reproductive technology, comprehensive minimally-invasive surgeries, and many more with high success rate.

A Professional manual to medical practitioners:The Expo exhibits were enriching to practising healthcare professionals to grab an opportunity with hospital collaboration projects, medical & clinical partnerships and hospital management cooperation with leading medical centres in Taiwan. Medical professionals evaluated the opportunity for direct collaboration request with a medical team.

Dr Shih-Sheng Chang, Chief Secretary, China Medical University Hospital, Taichung, said, The annual Taiwan Healthcare Expo presents cutting-edge biotechnologies and their applications in the medical field. It also provides a platform for hospitals, medical device companies, pharmaceutical companies as well as technology companies to exchange the latest information. This year CMUH was honoured to present developments on stem cell therapies, immune cell therapies, medical AI system application, and 3D printing. It successfully attracted more than 3000 visitors.

Dr Hsiu-Ling Hsiao, CSO, Ever Supreme (ES) Bio-Technology, as one of the exhibitor says, The annual Taiwan Healthcare Expo provided the opportunities for our company to present our accomplishments and portfolio to the public. It also helps us to be visible and to get a connection with international experts and customers. In compliance with the new policy of Regulations Governing Specific Cellular Therapeutic Technology (RGSCTT) in Taiwan, ES is the first biotechnology company approved to use Dendritic Cells (DC) to treat all stages of 8 cancers.

Visitors to the Expo were encouraged to take diagnosis test to learn their health status, consult the medical experts or experience the latest development of healthcare services, purchase health beneficiary products, nutrition, healthcare aids, medical alert systems, medical supplies, patient care, mobile services for health and sports gears and many more interactive activities.

Also, Refer:

Taiwan Healthcare+ Expo B2B opportunities

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Taiwan Healthcare+ Expo 2019: A 360 exposure to MedTech and Therapeutic world - BSA bureau

Lawmakers Present Mother & Daughter With the Signed Bill They Inspired – River Journal Staff

Dreams Law will require New Yorkers with a Central Venous Line to have access to safe and continued care when discharged from a hospital

This week, Assemblywoman Sandy Galef and Senator David Carlucci along with health advocates presented 14-year-old, stem cell transplant survivor, Dream Shepherd and her mother, Diana Lemon, with a pen certificate, officially recognizing Dreams Law (A.212/S.474)was signed by Governor Cuomo.

Dreams Law guarantees safe and continued care for patients with a Central Venous Line after being discharged from the hospital, something not afforded to Dream following her release.

Dreams story is one of strength in the face of adversity, but is also a story of the medical system failing New York families, said Assemblywoman Galef. Senator Carlucci and I advocated for this bill because no family should be left in the lurch when dealing with a life-threatening illness. I am so pleased that the Governor is supportive of this bill and has expanded its coverage.

New Yorkers should be able to leave a hospital with the care they need when at home, saidSenator David Carlucci. Central Venous Lines can easily cause infection, and thankfully because of Diana, Dream is here today. We cannot let insurance companies do this to another family. Dreams Law will better protect New Yorkers, and I thank Assemblywoman Galef and the Governor for their support of the bill.

As a parent my goal is to raise my children up to be victors over any circumstances put before them and thats what has been done. Four long years of lobbying Dreams law, its my intention that this legislation will empower caregivers and give adequate care for patients that need it the most, said Diana Lemon, Dreams mother.

I have no doubt this law will improve health outcomes for children with pediatric cancer, sickle cell disease, children who have had transplants and any other child who is discharged home with a central venous line, saidRhonda Ryan, Director of the Family Support Program for Friends of Karen. I believe there will be a decreased risk of infections and fewer hospitalizations as a result of this law. We are grateful at Friends of Karen that this important law has been passed. Our professional staff will inform the families with whom we work that parents or guardians now have the opportunity for professional nursing care in the home if they so choose.

In December of 2014, Dream had a stem cell transplant to battle Sickle Cell Disease and doctors put in a Central Venous Line that went to Dreams heart to administer medication. Upon discharge, Dreams insurance would not cover a home nurse to care for the Central Venous Line because they said it was not medically necessary.

Dreams mother could not afford a caretaker and without the proper medical knowledge had to care for her daughter. In NYS Health facilities, only registered nurses are allowed to administer medications with the use of a Central Venous Line, and according to the New England Journal of Medicine about 28,000 people die each year in Intensive Care Units due to blood stream infections from a Central Venous Line.

In the wake of this harrowing experience, Diana and Dream made it their mission to work with lawmakers so that no other family would find themselves in a similar position.

Westchester County Executive George Latimer added, I would like to extend my sincerest thanks to Governor Andrew Cuomo for his support of this legislation, that will truly help to give some of our most vulnerable patients the best possible care. Dreams Law will ensure that all patients who are discharged from local hospitals will continue to have access to specialized treatment from a skilled caretaker, guaranteeing the best chance for recovery from devastating illnesses.

Noah Doherty, General Manager Accucare Nursing & Home Care, said, Im proud to be part of a State where our elected officials understand the importance of private duty nursing in the home. Infusion therapy at home can save the life of a medically fragile child. Id like to thank Senator David Carlucci and Assemblywoman Sandy Galef for their tireless efforts with Dreams Law.

Galef and Carlucci are now working to amend Dreams Law to apply to all New Yorkers leaving the hospital, regardless of condition or further care needed so appropriate patient care is part of any discharge plan from a hospital.

Dreams Law will take effect immediately and can be amended when the legislative session begins in January.

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Lawmakers Present Mother & Daughter With the Signed Bill They Inspired - River Journal Staff

Sorrento Announces Dr. Robin Smith as New Member of the Board of Directors – GlobeNewswire

SAN DIEGO, Dec. 16, 2019 (GLOBE NEWSWIRE) -- Sorrento Therapeutics, Inc. (Nasdaq: SRNE, "Sorrento") announced today that Robin L. Smith, MD, MBA has been appointed to its Board of Directors.

Dr. Smith is a business leader, entrepreneur, medical doctor, author and philanthropist who has held various C-Suite and board positions in the biopharmaceutical industry. Her experience includes significant contributions on numerous board committees for public medical, digital and health care entities. She currently serves on the boards of directors of Celularity, Inc., a pioneer company focused on cancer and regenerative medicine, Seelos Therapeutics, Inc. (NASDAQ: SEEL), Spiritus Therapeutics, which she co-founded, the International Board of Sanford Health, and is the Chairman, President and founder of the Cura Foundation.

Dr. Smith has been recognized for her extensive leadership in the biopharmaceutical industry winning an array of industry awards and business recognitions. She received the Regenerative Medicine Foundation (RMF) 2019 Stem Cell and Regenerative Medicine Action Award for International Diplomacy in 2019, the 2018 HEALinc Future Health Humanitarian Award, the Business Intelligence Groups Woman of the Year Award in 2018 and the 2018 Gold Stevie Award for Woman of the Year (Government or Non-Profit). In April 2016, Pope Francis awarded Dr. Smith Dame Commander with Star Pontifical Equestrian Order of Saint Sylvester Pope and Martyr and was awarded the Lifetime Achievement in Healthcare and Science by The National Museum of Catholic Art and Library. Dr. Smith is a 2016 recipient of the Women of Power and Influence Awards and winner of the 2014 Brava Award.

She received her B.A. degree from Yale University and her M.D. degree from the Yale School of Medicine. Dr. Smith holds an M.B.A. degree from the Wharton School of Business and completed the Stanford University Directors Program and received an honorary Doctor of Science degree from Thomas Jefferson Medical College

Sorrento is an exciting clinical stage, antibody-centric, biopharmaceutical company with unique assets and a commendable commitment to developing new therapies for individuals with life threatening illnesses. The company has an incredibly talented management team and innovative scientists. I am honored to join their board and appreciate the opportunity to work with the team and other board members, commented Dr. Smith.

Dr. Henry Ji, Chairman and CEO, commented, We are very excited to have Robin join our board. She brings deep medical, business knowledge and diversity at a critical time for our company. I believe Dr. Smith will be an invaluable addition to our board and can be a great contributor to a bright future for Sorrento Therapeutics.

About Sorrento Therapeutics, Inc.

Sorrento is a clinical stage, antibody-centric, biopharmaceutical company developing new therapies to turn malignant cancers into manageable and possibly curable diseases. Sorrento's multimodal multipronged approach to fighting cancer is made possible by its extensive immuno-oncology platforms, including key assets such as fully human antibodies (G-MAB library), antibody-drug conjugates (ADC) as well as CAR-T and oncolytic virus (Seprehvir).

Sorrento's commitment to life-enhancing therapies for cancer patients is also demonstrated by its effort to advance a first-in-class (TRPV1 agonist) RTX and ZTlido. RTX is completing a phase IB trial in terminal cancer patients. ZTlido was approved by US FDA on February 28, 2018.

For more information visit http://www.sorrentotherapeutics.com

Media and Investor Relations

Alexis Nahama, SVP Corporate Development

Telephone: 1.858.203.4120

Email: mediarelations@sorrentotherapeutics.com

ZTlido and G-MAB are trademarks owned by Scilex Pharmaceuticals Inc. and Sorrento, respectively.

Seprehvir, is a registered trademark of VirttuBiologics Limited, a wholly owned subsidiary of TNK Therapeutics, Inc. and part of the group of companies owned by Sorrento Therapeutics, Inc.

All other trademarks are the property of their respective owners.

2019 Sorrento Therapeutics, Inc. All Rights Reserved.

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Sorrento Announces Dr. Robin Smith as New Member of the Board of Directors - GlobeNewswire