Category Archives: Pennsylvania Stem Cells

TORONTO--(BUSINESS WIRE)-- Vasomune Therapeutics, Inc., a therapeutic biopharmaceutical company developing a novel first-in-class medicine for the treatment of COVID-19 pneumonia and pathogen-induced ARDS, today announced the formation of a Clinical and Scientific Advisory Board. The Board is composed of key opinion leaders in their respective fields of medicine and translational therapeutic development.

We are very excited to be working with this esteemed group of medical and scientific advisors as we transition to the clinic with our novel therapeutic approach to COVID-19 that involves targeting the vasculature, said Douglas A. Hamilton, President and Chief Executive Officer, Vasomune.

Members of the Vasomune Clinical and Scientific Advisory Board are:

David Andrews, PhD Dr. Andrews is Director of and Senior Scientist in Biological Sciences at Sunnybrook Research Institute, Professor of Biochemistry and Medical Biophysics at University of Toronto and a Tier 1 Canada Research Chair in Membrane Biogenesis. His research areas include regulation of apoptosis at mitochondrial level, high throughput and image-based high-content screening using automated imaging and analysis of cells in monolayer and 3D cultures, gene knockdown and screening of libraries of small molecules. Dr. Andrews participated in the start-up of MBI Fermentas and Isogenica, and his group performs collaborative and contract research for a variety of biotech companies. He holds licensed patents in regulation of translation and in vitro evolution.

Carolyn S. Calfee, MD, MAS Dr. Calfee, MD, MAS is Professor of Medicine and Anesthesia at the University of California, San Francisco (UCSF), where she attends intensive care units. She completed her undergraduate studies at Yale University and medical school at the University of Pennsylvania before moving to UCSF for her residency, chief residency and fellowship training, as well as her Masters degree in Clinical Research. Her primary academic focus is the pathogenesis and treatment of acute respiratory distress syndrome (ARDS). Her main research interests include molecular subphenotypes of ARDS and precision medicine in critical care; the role of environmental exposure, including smoking, air pollution and novel tobacco products in susceptibility to lung injury; and novel treatments for ARDS.

Eddie Fan, PhD, MD Dr. Fan is an Associate Professor in the Interdepartmental Division of Critical Care Medicine and the Institute of Health Policy, Management and Evaluation at the University of Toronto and a Staff Intensivist at the University Health Network/Mount Sinai Hospital. He is also currently the Medical Director of the Extracorporeal Life Support Program at Toronto General Hospital. Dr. Fans research has focused on advanced life support for acute respiratory failure and patient outcomes from critical illness. Dr. Fan obtained his undergraduate degree from the University of Toronto, his medical degree from the University of Western Ontario and a PhD in Clinical Investigation from Johns Hopkins University.

Michael Julius, PhD Dr. Julius is a professor emeritus in the Department of Immunology at the University of Toronto. His four decades of research experience at Stanford University, the Basel Institute for Immunology, the Institute Pasteur, McGill University and the University of Toronto, where he chaired the Department of Immunology, is dedicated to understanding the biochemistry and genetics of lymphocyte activation. Dr Julius has in-depth knowledge of multiple therapeutic areas, including neuroscience, cancer, cardiovascular and immune system; and expertise across multiple platforms, including high-content cellular analyses, artificial intelligence, health informatics, and imaging-guided interventions and therapeutics. Most recently, Dr. Julius was recruited to the position of Vice President, Research, at Sunnybrook Health Sciences Centre where he created an international hub for life sciences dedicated to both discovery and commercialization with an annual budget of $125M. This initiative achieved a functional integration of researchers, clinicians, business and patients towards moving discoveries into the clinic; and has spun-off 15 startup companies. Dr. Julius received his BSc at McGill University and his PhD at Stanford University, and has authored 230 publications.

Dana McClintock, MD Dr. McClintock is the Chief Medical Officer of Alladapt Immunotherapeutics, Inc., a role she has held since 2018. Prior to joining Alladapt, Dr. McClintock was Global Head of Innovation for Immunology, Infectious Disease and Ophthalmology, at Genentech/Roche. Prior to this position, Dr. McClintock held roles of increasing responsibility at Genentech/Roche, including Global Head of Pipeline and Portfolio Planning for Immunology, Infectious Disease and Ophthalmology, as well as Interim Global Co-Head of Ophthalmology. Earlier in Dr. McClintocks pharmaceutical career, she was deeply involved in respiratory, immunology and ophthalmology clinical development activities across a range of phases, from IND-enabling work and early clinical trials through post-marketing commitments and medical affairs activities. Dr. McClintock was involved in key clinical development activities for omalizumab (Xolair) and ranibizumab (Lucentis) as along with other pipeline molecules. Prior to joining the pharmaceutical industry, Dr. McClintocks academic research focus was in ARDS, with publications evaluating ventilator parameters as well plasma biomarkers of epithelial and endothelial cell injury. Dr. McClintock received a BA in Chemistry from Duke University and an MD from the University of Virginia. Dr. McClintock completed her training in Internal Medicine and Pulmonary and Critical Care Medicine, both at the UCSF.

Renato Skerlj, PhD Dr. Skerlj has 25 years of experience leading the discovery and development of small molecule drugs to treat rare diseases, cancer, infection and neurodegenerative diseases, and deep scientific expertise in the research and development of innovative, genetically-targeted treatments. Currently, he is Senior Vice President of Research and Development at X4 Pharmaceuticals in Cambridge, MA, and previously held drug discovery and development leadership roles at Cambridge-based Lysosomal Therapeutics, Inc. Prior to that, he was interim Head of Small Molecule Discovery at Genzyme, and was part of the executive team at AnorMED, a publicly-traded company that was acquired by Genzyme in 2006. Dr. Skerlj is an inventor of both plerixafor, a stem cell mobilizer approved by the U.S. Food and Drug Administration (FDA) in 2008, and ertapenem, an anti-bacterial approved by the FDA in 2001, and has been responsible for delivering multiple drug candidates into early clinical research. He has authored 65 publications and holds 50 patents. Dr. Skerlj received his PhD in Synthetic Organic Chemistry from the University of British Columbia and completed postdoctoral fellowships at the University of Oxford and Ohio State University.

About AV-001

AV-001 is an investigational medicine designed to activate the Tie2 receptor and restore normal barrier defense in the vasculature. Following trauma or infection, the bodys host vascular response can become unchecked, leading to vascular leak and ultimately organ failure and death. Vasomune is developing AV-001 for the treatment of COVID-19 pneumonia and pathogen-induced moderate-to-severe ARDS. ARDS is a life-threatening condition that can develop after pneumonia, trauma, shock and sepsis, and is also the leading cause of death for patients infected with COVID-19. Prior to the onset of the coronavirus pandemic, the combined annual incidence of ARDS was approximately 370,000 patients per year in the US and EU with an average mortality rate of 40%. At present, there are no effective therapeutics to treat ARDS.

About Vasomune Therapeutics

Vasomune Therapeutics is a private early stage biopharmaceutical company developing the next generation of medicines to harness the bodys ability to defend against illness. The company is transitioning in the near term to the clinic with a novel therapeutic approach to ARDS that involves targeting a signaling molecule in the vasculature responsible for regulating barrier defense. Vascular dysfunction is associated with the pathology of several disease states, including COVID-19 pneumonia, acute respiratory distress syndrome (ARDS), acute lung injury (ALI), acute kidney injury (AKI), hemorrhagic shock, sepsis and stroke. Vasomunes head office and laboratory is located in Toronto, Canada with US offices in San Mateo, CA. For more information about the company and its product candidates, please visit http://www.vasomune.com or email the President and CEO of Vasomune at dhamilton@vasomune.com.

View source version on businesswire.com: https://www.businesswire.com/news/home/20200511005286/en/

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Vasomune Therapeutics Announces Clinical and Scientific Advisory Board - BioSpace

CHINESE scientists have been accused of being real-life Dr Frankensteins who play God by cloning apes and editing the genes of babies.

Some of their work has been dubbed monstrous while other cutting edge research could lead to cures for Parkinson's and Alzheimer's.

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It's important to note that the US and the UK are not immune from conducting tests on animals and in fact carry out THOUSANDS of experiments on primates every year.

However, China has become the capital of research on apes and monkeys believing that our closest relatives hold the key to understanding brain disorders that destroy lives.

Incredibly, the Institute of Neuroscience (ION) in Shanghai, cloned five infant monkeys last year from an adult macaque who had been genetically-edited.

The result was baby primates intentionally born with a mutation that disrupts their wake-sleep cycle.

By giving the monkeys new drugs to treat their pre-existing brain disorders, the scientists hope to develop treatments for illnesses such as Alzheimer's disease.

It's no wonder the ION has been dubbed the "Cern of primate neurobiology".

The Institute successfully cloned two macaque monkeys in 2018 - a world first giving the experts confidence to push ahead with further experiments.

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Heaping praise on the research, the Chinese Academy of Sciences said: "The achievement heralds a new era in which China can produce batches of standardised monkey clones, which will serve as animal models in the research of the brain's cognitive functions, early diagnoses and interventions of diseases, as well as research and development of drugs.

While China allows genetic manipulation on animals it has banned the use of gene-editing on humans but that hasn't stopped some of its scientists "playing God" with unborn children.

Scientist He Jiankui, 35, rocked the scientific world when he revealed he had altered the embryos of twin girls in 2018.

In December last year, it was revealed that a third child born to a different mum had also been gene-edited.

The rogue expert said he used a tool called Crispr to disable a gene that allows the AIDS virus to enter cells in a bid to make the children immune from the disease.

But why have such experiments been dubbed monstrous by others within the scientific community?

Experts claim gene-editing in people could "divide humans into subspecies" and can cause mutations, genetic problems and even cancer.

Dr Kiran Musunuru, an expert in this area from the University of Pennsylvania, called the experiment unconscionable an experiment on human beings that is not morally or ethically defensible.

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Professor Julian Savulescu, of the University of Oxford, said: If true, this experiment is monstrous.

The embryos were healthy. No known diseases. Gene editing itself is experimental and is still associated with off-target mutations, capable of causing genetic problems early and later in life, including the development of cancer.

There are many effective ways to prevent HIV in healthy individuals: for example, protected sex.

Last December, Mr Jiankui was jailed for three years after news of the third child's birth was revealed.

He was convicted of practising medicine without a licence and fined 330,000 by a court in Shenzhen, the Xinhua news agency reported.

One of the most controversial experiments to date was the creation of embryos that were part human and part primate.

Last year, Spaniard Juan Carlos Izpisa Belmonte led a team of Chinese researchers with the end goal of creating monkeys which have entirely human organs such as kidneys or livers.

The organs will then be used for human transplants.

Based in China, the team made the chimeras a single organism with cells from more than one genotype - by injecting human stem cells into a fresh monkey embryo.

Biologist Belmonte previously tried adding human cells to embryos of pigs but the disturbing experiment was not successful.

However, because primates are genetically related to humans, the chances of the new research being successful is much greater.

The scientists also use gene-editing technology to disable certain cell formations in the animals to give the human cells a better chance of thriving.

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In the US and other western democracies, such research is banned however in China, experts are allowed to push the boundaries of scientific ethics.

Importantly, no Frankenstein monster has been born as a result of this research... not yet anyway.

Instead, the hybrid embryos are allowed to develop for around two weeks so their progress can be studied.

Mr Belmonte defended his work with the Chinese, saying: History shows us time and time again that, over time, our ethical and moral standards change and mutate, like our DNA, and what yesterday was ethically unacceptable, if this really represents an advance for the progress of humanity, today it is already an essential part of our lives."

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A similar experiment involved two piglets who were born with monkey cells in December at the State Key Laboratory of Stem Cell and Reproductive Biology in Beijing.

The so-called 'pig-monkey chimeras' died a week later.

Away from China, one of the most sinister experiments took place at the University of Munich where two monkeys were given transplanted pig hearts.

The poor creatures died after six months in a study which was deemed a success.

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Chinas scientists accused of playing God by creating monstrous cloned apes and primates with human organs - The Sun

PENNSYLVANIA, USA Brianna Boriosi, 30, and her husband Marc both are considered essential employees because they work in inpatient drug and alcohol treatment services. They are now self-quarantining at home because they both tested positive for COVID-19.

What makes their case more complicated and worrisome is that Brianna is 7-and-a-half months pregnant, due June 24. Of course she immediately called her doctors.

They told me I was their first positive pregnancy case with COVID-19 in their practice, Brianna said.

Almost immediately after she found out she was positive she was getting calls from the Pennsylvania Department of Health.They wanted to know what the circumstances were leading up to getting infected, but also they hoped Brianna would give consent for test samples.

Once the babys born, they talked about maybe taking some of the umbilical cord or the blood in there, Brianna said. As well as the placenta, and if Im willing, looking into possibly donating stem cells.

It didnt take long for her to say yes.

There was a possibility that the baby will either be immune or have some of these antibodies.

While doctors believe the virus will not get passed onto her unborn child, scientists hope what they find through tests can help them to get to a vaccine and the cure.

Anything I can do to help or donate or whatever, Im definitely all about it.

Her positive test could lead to keeping everyone negative for the virus in the future. Brianna wants that for their baby as well as their two-year-old son.

Because both Brianna and Marc are positive, doctors presume their son is as well. The couple is thankful that he hasnt shown any symptoms, and they hope and pray it stays that way.

RELATED: Feds to track, publicly share info on nursing home COVID-19 outbreaks

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RELATED: Reports suggest many may have had coronavirus with no symptoms

Contact reporter John Charlton atjcharlton@whas11.com. Follow him onTwitter (@JCharltonNews) andFacebook.

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Pregnant and positive | Scientists look for COVID-19 immunity in the womb - WHAS11.com

Why Dr. Thomas E. Young?

Pennsylvania Stem Cell Centers, with three offices in the greater Philadelphia region, is a leading and innovative provider of adult stem cell therapies in Pennsylvania. Working as an affiliate of the Cell Surgical Network (the worlds largest regenerative medicine clinical research organization), Dr. Thomas E. Young is committed to helping patients who suffer from degenerative and inflammatory conditions. Therapies using ones own stem cells may be the next great advancement in medicine.

Because Pennsylvania Stem Cell Centers only uses adult stem cells harvested directly from the patients own fat stores, an extremely high number of stem cells are usually available almost immediately. Regenerative healing naturally takes time and we do not expect immediate improvement in symptoms. Patients will be followed closely through our research registry and data, carefully collected to help establish effectiveness of our protocols.

Stem Cell Therapy is used to promote the repair of degenerative or injured tissue by harnessing the natural healing power of mesenchymal stem cells that form the musculoskeletal tissues in the body including bone, joint cartilage, ligaments, and tendons. The mission at PA Stem Cell Treatment Centers is to promote natural healing using autologous cellular therapies for the cure and treatment of musculoskeletal disorders, to relieve pain, and to improve functionality and quality of life.

PA Stem Cell Treatment Centers is highly committed to clinical research and the advancement of regenerative medicine.

Read this article:
Why Choose Us - Pennsylvania Stem Cell Center

Moderna (MRNA) put mRNA technology on the map, however, this technology has not yet proven itself convincingly in clinical trials. The concept of making your own body behave like a drug manufacturing engine is interesting, but it has its hurdles. One hurdle is the body's own immune system, which may reject the mRNA; the other is whether the technology will produce enough proteins to make a difference - with the human body, you never know what will trigger a cascade of unpredictable events that could foil the "best laid plans of men," however smart. Delivery into cells is another issue. Everything always boils down to human trials - and here, Moderna is not there yet.

Most of the biotech investing rules I follow tell me Moderna is an absolute avoid. One, a $12bn market valuation based on a pipeline of near 20 candidates. These are based on a single all-curing drug platform. Not a single drug candidate is beyond phase 2. Two, the current price of the one-year old IPO is at 52-week and all time highs based on a fear and hope around a sudden pandemic like coronavirus. Three, a corollary of point one, there is little phase 2 efficacy data anywhere, therefore the platform, however promising, is absolutely unproven. However, on the other hand, another critical business rule I also follow is "follow the cash," and this early stage biopharma has $2bn of it. This is more cash than the entire current market cap of a company like Amarin (AMRN) with an approved and blockbuster potential product.

In order to understand this anomaly, I looked into the science behind the company, because the valuation of a pre-market pre-approval stage biopharma is mostly based on the science. And the science does look promising.

First, let us understand that the company says that mRNA-based therapeutic protein synthesis is the next step to recombinant protein technology, which has spawned an industry worth over $200bn. However, recombinant technology cannot create certain major types of proteins - intracellular and membrane proteins which represent as much as two-thirds of the proteins in humans. This is a major part of what mRNA technology can do.

There are various competitive advantages to mRNA over recombinance - for one, since the proteins are made naturally, there's less chance of rejection and immunogenicity. Another advantage, as the company says, "A vast number of potential mRNA medicines can be developed, therefore, with only minor changes to the underlying chemical structure of the molecule or manufacturing processes, a significant advantage over small molecule or protein therapeutics."

Moderna was founded in 2010 and IPO-ed in late 2018. Reading through the 10-K, what struck me was that there's a huge number of programs, all of them early stages, each demonstrating, to some extent, the development of critical antibodies upon using the drug candidate. However, instead of developing any particular program to fruition, including BLA and approval, this company focuses on advancing the entire pipeline at the same time.

Here's a snapshot of the pipeline:

Source

Next, let me present a set of 6 slides, each for one of the modalities above, which shows the latest available trial data for that drug candidate:-

Source - 10-K

Now, we have multiple programs progressing through phase 2 - which is really the datapoint that first gets us interested (or not) in a company. Then, just today, we read about the company's plans to start a phase 3 trial "soon." However, like we said, we still couldn't find enough that could justify this huge $11.8bn valuation. I mean, the science is good, in theory, but this sort of high-grade technology has so many pitfalls it really doesn't make sense to have too much expectation until we see phase 3 data.

The above 6 slides basically show that in the lab and in primates and in healthy subjects, there's constructive antibody activity on dosing with these mRNA medicines. Some of the measures of these activities are promising, for example, for mRNA-1944, "participants had measured antibody levels exceeding the levels of antibody expected to be protective against chikungunya infection (> 1 g/mL) following a single dose, with the middle and high doses projected to maintain antibody levels above protective levels for at least 16 weeks." But this was a phase 1 study in healthy volunteers, and while promising, like I said, this alone does not justify the valuation.

Sometimes, companies like these justify their valuations on the basis of their founders, or the founding technology; Juno comes to mind. Again, nothing like that was clearly apparent to me on reading either the 10-K or the Corporate Presentation. Besides a lot of basic and advanced genetic science, I could not figure out who is behind the science; admittedly, though, MRNA does have a vast patent estate comprising more than 550 patents worldwide, applied for and granted.

A much better overview of the history of the science is found here. There are basically five key figures behind it; the original science was developed by University of Pennsylvania scientist Katalin Karik, but her startup didn't go anywhere directly. "Later, in 2010 Harvard University scientist Derrick Rossi used modified mRNA to encode proteins that reprogrammed adult cells into embryonic-like stem cells. Harvard cardiovascular scientist Kenneth Chien, now at the Karolinska Institute, and Massachusetts Institute of Technologys famed serial entrepreneur Robert Langer spotted mRNAs therapeutic potential and joined Rossi in pitching a stem cell company to the venture capital firm Flagship Pioneering." This led to Moderna.

In recent times, under the coronavirus pandemic, Moderna has suppressed the rest of its pipeline and is focusing almost entirely on mRNA-1273, its candidate for treating COVID-19. Although mRNA can in theory target multiple types of diseases, vaccines are still their easiest application, since "the mRNA needs to produce only a small amount of protein for the vaccine to work, and setting off the bodys RNA immune sensors a little wont hurt." The company already had multiple viral vaccine targets under development, including one on MERS-COV in the lab, so it is understandable that a little tweak could set things off in coronavirus targeting.

From its press release, the latest that is happening in this regard is

On March 27, 2020, the NIH announced that Emory University in Atlanta will begin enrolling healthy adult volunteers ages 18 to 55 years in the NIH-led Phase 1 study of mRNA-1273.

According to a PR dated 4/7/2020, Moderna will host a virtual Vaccines Day for analysts and investors on 4/14/2020. The Vaccines Day will include presentations from Stphane Bancel, Chief Executive Officer, Tal Zaks, Chief Medical Officer, and key opinion leaders with a focus on mRNA vaccines and the Companys core prophylactic vaccines modality.

According to another PR dated 4/8/2020, Lorence Kim, M.D., the company's Chief Financial Officer, will participate in the 19th Annual Needham Healthcare Conference on 4/15/2020.

Currently, everything in this $12bn behemoth hinges around producing a working vaccine for SARS-COV-2. There are pitfalls - efficacy, timeline, positioning, market - that could determine how it all works out. Success or failure here could determine what happens to the company as a whole, because the market seems to be in an over-expectant mode right now.

In 2018, when the company IPO-ed, CEO Bancel said This is a 20-year job...We believe we are just starting. It seems to this author that $12bn is just a little too much to start with for something that may be promising, but still unproven. The science looks good - although there's a lot of secrecy behind it as of now - so if these prices go down for whatever reason, I would be much more interested. The company's vast and diverse collaborations - with AstraZeneca (NYSE:AZN), Merck (NYSE:MRK) and others - does build confidence that big pharma is looking at it favorably.

Thanks for reading. At the Total Pharma Tracker, we do more than follow biotech news. Using our IOMachine, our team of analysts work to be ahead of the curve.

That means that when the catalyst comes that will make or break a stock, we've positioned ourselves for success. And we share that positioning and all the analysis behind it with our members.

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

Additional disclosure: I own AMRN.

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Moderna: A $12 Billion Brand Built On Hope And mRNA - Seeking Alpha

13 January 2020

Promising results from clinical trials give hope for using CRISPR/Cas9 genome editing to treat various heritable diseases and cancer in humans.

It has been seven years since the discovery that the CRISPR/Cas9 defence system, used by microbes to destroy viruses, could be re-engineered to edit the human genome. Since then researchers have carried out an array of experiments to explore potential applications.

Biophysist Dr He Jiankui sparked global controversy concerning the ethics of genome editing when he used CRISPR to genetically modify embryos, resulting in the birth of the first genome-edited babies (see BioNews 977).

Yet researchers worldwide have at the same time been investigating the use of CRISPR for non-heritable changes, modifying the genes in non-embryonic cells to treat a wide range of diseases.

'There's been a lot of appropriate caution in applying this to treating people, but I think we're starting to see some of the results of that work,' said Dr Edward Stadtmauer, a haematologist at the University of Pennsylvania, Philadelphia.

Over a dozen new clinical trials testing CRISPRtherapy on diseases such as cancer, HIV and sickle cell anaemia were listed on the clinicaltrials.gov database last year. One trial in its early stages used CRISPR to treat sickle cell anaemia and beta-thalassaemia, both genetic blood disorders that result in the production of an abnormal form of the oxygen-carrying protein, haemoglobin.

Two patients with these disorders were treated by CRISPR Therapeutics in Cambridge, Massachusetts, and Vertex Pharmaceuticals in Boston, Massachusetts, using CRISPR to inactivate a gene that switches off the production of an alternative form of haemoglobin. Preliminary results of the study suggest that this therapy improved some of the symptoms but the participants will need to be followed for a longer period to be sure.

Results from two other trials, one in which genome-edited blood cells were transplanted into a man to treat HIV infection, and the other in which they were transplanted into three people to treat some forms of cancer, were less successful. In both cases, the transplanted cells flourished in the bone marrow of recipients, without any serious safety concerns, but did not produce a clear medical benefit. The study has been placed on hold while researchers explore ways to boost that percentage, says Hongkui Deng, a stem-cell researcher at Peking University, Beijing, China and a lead author of the work.

Other researchers are trying to move beyond editing cells in vitro. In July 2019 a clinical trial was launched to treat Leber congenital amaurosis 10 (LCA10), a rare genetic disease that causes blindness. The trial, launched by two pharmaceutical companies, Editas Medicine in Cambridge, Massachusetts, and Allergan in Dublin, Ireland, will be the first trial that uses CRISPR to edit cells inside of the body. The researchers are testing AGN-151587 (EDIT-101), which is a novel CRISPR treatment delivered via adeno-associated virus (AAV) directly to the eye's light-sensing photoreceptor cells to remove the mutation that causes LCA10.

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The potential use of CRISPR to treat disease is gaining momentum - BioNews

This undated screen grab shows the cell-division of two fertilized human embryos during the first 24 hours of embryonic development following IVF treatment at a private clinic in London. ( Jim Dyson/Getty Images )

LOS ANGELES - Some of the scientific advancements of the 2010s have been truly mind-blowing, and perhaps none more so than the leaps and bounds weve made in the realm of reproduction.

This was not only the decade in which the first three-parent baby was born, it was the era when a rogue scientist chose to make edits to a set of twin girls DNA, making real the long-imagined scenario of genetically altering human beings while simultaneously thrusting the deeply complicated ethical discussions surrounding this practice into the limelight.

These are the five most life-altering breakthroughs in reproduction from the past decade.

In 2018, Chinese biophysics researcher He Jiankui announced that he had used the gene-editing tool CRISPR to modify the genes of two twin girls before birth. He and his team said that their goal was to make the girls immune to infection by HIV through the elimination of a gene called CCR5.

When the news broke, many mainstream scientists criticized the attempt, calling it too unsafe to try. Where some people saw the potential for a new kind of medical treatment capable of eradicating genetic disease, others saw a window into a dystopian future filled with designer babies and framed by a new kind of eugenics.

At the time, Dr. Kiran Musunuru, a University of Pennsylvania gene-editing expert, said Hes work was unconscionable... an experiment on human beings that is not morally or ethically defensible.

Other experts believe Hes work could propel the field of gene editing forward.

The twins, known as Lulu and Nana, have continued to make headlines since their birth. The gene modification that He claims to have carried out may have caused some unintended mutations in other parts of the genome, which could have unpredictable consequences for their health long term something many scientists who argue against Hes work cite as a reason to hold off on using gene-editing technology on humans.

Only time will tell what will happen to Lulu and Nana and if the edits to their DNA ultimately help or hurt them, but their story pushed the topic of human gene-editing and the ethics surrounding it to the forefront of the global scientific community.

In 2016, a technique called mitochondrial transfer was used successfully for the first time to create a three-parent baby grown from a fathers sperm, a mothers cell nucleus and a third donors egg that had the nucleus removed.

This technique was developed to prevent the transmission of certain genetic disorders through the mothers mitochondria. The majority of a three-parent babys DNA would come from his parents in the form of nuclear DNA, and only a small portion would come from the donor in the form of mitochondrial DNA.

A team led by physician John Zhang at the New Hope Fertility Center in New York City facilitated the birth of the first three-parent baby in April 2016.

Using human pluripotent stem cells, researchers were able to make the precursors of human sperm or eggs. In other words, they reprogrammed skin and blood stem cells to become an early-state version of what would eventually become either sperm or an egg.

"The creation of primordial germ cells is one of the earliest events during early mammalian development," Dr. Naoko Irie, first author of the paper from the Wellcome Trust/Cancer Research UK Gurdon Institute at the University of Cambridge told Science Daily. "It's a stage we've managed to recreate using stem cells from mice and rats, but until now few researches have done this systematically using human stem cells. It has highlighted important differences between embryo development in humans and rodents that may mean findings in mice and rats may not be directly extrapolated to humans."

A 2018 study showed that gene editing can allow two same-sex mice to conceive pups, and two female mice were able to successfully create healthy pups that then went on to reproduce themselves.

A team of researchers at the Chinese Academy of Sciences in Beijing, led by developmental biologist Qi Zhou, were able to use gene editing to produce 29 living mice from two females, seven of which went on to have their own pups. They were able to produce 12 pups from two male parents, but those offspring were not able to live more than two days.Whether or not the method can one day be used in same-sex human reproduction is still up for debate.

For the first time ever, Chinese scientists were able to clone two primates using the technique that produced Dolly the sheep, the first mammal to be cloned from an adult somatic cell via nuclear transfer.

The two cloned female macaques were named Zhong Zhong and Hua Hua, and their successful birth opened up the possibility of using the same cloning method to one day clone humans.

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What a time to be alive: Reproductive breakthroughs of the 2010s that changed life as we know it - FOX 10 News Phoenix

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WASHINGTON DC, United States (AFP) 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.

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

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 co-founder 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 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 tumour-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 US$2 million meaning patients face gruelling 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 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.

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.

I'm a bacteriologist we've been talking about bioterrorism for years, she said. Nothing has ever happened.

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2019: The year gene therapy came of age - Jamaica Observer

Trevena Inc. (NASDAQ:TRVN) and VistaGen Therapeutics Inc. (NASDAQ:VTGN), are influenced by contrast since they are both players in the Biotechnology. These factors are particularly influence the analyst recommendations, profitability, risk, dividends, institutional ownership, earnings and valuation of the two firms.

Earnings and Valuation

We can see in table 1 the earnings per share (EPS), gross revenue and valuation of Trevena Inc. and VistaGen Therapeutics Inc.

Profitability

Table 2 provides us the net margins, return on assets and return on equity of both companies.

Volatility and Risk

Trevena Inc.s current beta is 2.39 and it happens to be 139.00% more volatile than S&P 500. From a competition point of view, VistaGen Therapeutics Inc. has a -0.48 beta which is 148.00% less volatile compared to S&P 500.

Liquidity

The Current Ratio and a Quick Ratio of Trevena Inc. are 3.6 and 3.6. Competitively, VistaGen Therapeutics Inc. has 4.9 and 4.9 for Current and Quick Ratio. VistaGen Therapeutics Inc.s better ability to pay short and long-term obligations than Trevena Inc.

Analyst Recommendations

The table delivered features the ratings and recommendations for Trevena Inc. and VistaGen Therapeutics Inc.

Trevena Inc.s upside potential currently stands at 394.84% and an $3.5 average target price. Competitively VistaGen Therapeutics Inc. has an average target price of $22, with potential upside of 4,957.47%. The results from earlier shows that analysts opinion suggest that VistaGen Therapeutics Inc. seems more appealing than Trevena Inc.

Institutional & Insider Ownership

The shares of both Trevena Inc. and VistaGen Therapeutics Inc. are owned by institutional investors at 32.2% and 20.4% respectively. 1% are Trevena Inc.s share owned by insiders. Competitively, VistaGen Therapeutics Inc. has 0.2% of its share owned by insiders.

Performance

Here are the Weekly, Monthly, Quarterly, Half Yearly, Yearly and YTD Performance of both pretenders.

For the past year Trevena Inc. has 120.93% stronger performance while VistaGen Therapeutics Inc. has -66% weaker performance.

Summary

On 8 of the 11 factors Trevena Inc. beats VistaGen Therapeutics Inc.

Trevena, Inc., a biopharmaceutical company, develops various therapies based on breakthrough science to benefit patients and healthcare providers confronting serious medical conditions. Its product candidates include oliceridine injection, a -receptor G protein pathway selective modulator, which is in Phase III clinical trials for the management of moderate-to-severe acute pain where intravenous administration is preferred; TRV250, a G protein biased ligand targeting the d-receptor, which is in preclinical development for the treatment of migraine; and TRV734, a small molecule G protein biased ligand at the mu-receptor that is in Phase I clinical trials for the treatment of moderate to severe acute and chronic pain, as well as TRV027 for the treatment of acute heart failure. Trevena, Inc. was founded in 2007 and is headquartered in King of Prussia, Pennsylvania.

VistaGen Therapeutics, Inc., a clinical-stage biopharmaceutical company, engages in developing and commercializing medicines for depression and other central nervous system (CNS) disorders. The company's lead product candidate is AV-101, which is in Phase II development stage, an adjunctive treatment used for major depressive disorder. It also focuses on potential commercial applications of its human pluripotent stem cell (hPSC) technology platform to discover, rescue, develop, and commercialize new chemical entities (NCEs) for CNS and other diseases; and regenerative medicine involving hPSC-derived blood, cartilage, heart, and liver cells. In addition, the company develops CardioSafe 3D, an in vitro cardiac bioassay system for predicting human heart toxicity of small molecule NCEs. VistaGen Therapeutics, Inc. has licensing, sublicensing, and collaboration agreements with BlueRock Therapeutics, LP; U.S. National Institutes of Health; Cato Research Ltd.; and University Health Network. The company was founded in 1998 and is headquartered in South San Francisco, California.

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Trevena Inc. (TRVN)'s Financial Results Comparing With VistaGen Therapeutics Inc. (NASDAQ:VTGN) - The Broch Herald

When the call came, Dimitri Georgiadis would have exactly four hours to hug his two sons, zip up his bag and motor 253 miles from Syracuse to Philadelphia.

If he wasted even 15 minutes, the donated lung would be gone.

Word spread quickly through St. Sophias Greek Orthodox Church community. Does anyone know a pilot?

Ted Limpert immediately said yes. Limpert is a Syracuse city court judge, handling every kind of charge from midnight mishaps to human trafficking.

Before that, he was a part-time fighter pilot one of the first to scramble into action when the planes hit the World Trade Center.

Hes flown 2,200 hours in an F-16 and 750 hours in the A-10 Warthog as a member of the 174th Air National Guard fighter wing.

It was Limpert who flew a jet over Clinton Square, cracking the silence on the first anniversary of the Sept. 11, 2001 attacks.

He has retired from the military, but he still owns an airplane, parked at a small airport in Cortland.

This was the plan: Limpert would collect Georgiadis and his wife Eleni, drive to his plane, fly about an hour to the North Philadelphia airport. They would get a car, even if it meant calling an ambulance, and drive through Philadelphia traffic to Temple University Hospital, where a donated lung would be waiting to replace the ones threatening Georgiadis life.

It would just be another mission, he thought.

We called it TOT, time over target, Limpert said. You can be early, but you cant be late.

Georgiadis, 55, was diagnosed with pulmonary fibrosis in 2006. As he got older, the disease grew more and more aggressive. He worked 18 years at Di Lauros Bakery in Syracuse. Then he went to work in the maintenance department, plowing snow and fixing faucets, for the Onondaga County Parks Department.

Georgiadis went on oxygen and disability. He moved up the list for a lung transplant. It would be done at Temple University Hospital, where he was being treated.

He was planning to drive himself to the hospital. Doctors said the call could come in the middle of the night, when accidents are most likely to claim the lives of donors.

It would be a stretch for a man on oxygen and in shock to make it over the foggy hills of Pennsylvania, through Philadelphia traffic and to the hospital in time.

Eleni Georgiadis started making phone calls.

All summer, I was calling different pilots, she said. Everybody we called, they said they couldnt guarantee me. They said well put you on the list, but we cant guarantee.

Even when she offered to pay, there was no guarantee, she said.

Then, a friend from church asked a friend of Limperts for help. The families were strangers.

He said Absolutely, Eleni Georgiadis said.

For two months, Limpert kept the car gassed up. He constantly checked weather conditions. He carried his phone everywhere, to the bench and to bed.

If he left town, he texted Eleni Georgiadis.

When hed come back, hed say Green Light. Your pilot is here. Ready for call, she said.

On Monday, Nov. 18, Limpert checked the weather one more time before heading for bed. It didnt look good.

At about 9 p.m., the call came.

He had not packed an overnight bag, even though his wife told him to. She handed him a Red Bull and he was out the door.

There was too much fog, they would have to drive to Philadelphia.

Limpert typed the hospitals address into his GPS app. It said the arrival time was 1:06 a.m.

That would be too late.

Limpert, a judge, declined to put a number on his speed down I-81, through Scranton and onto I-76.

I was proceeding with traffic, he said, laughing.

But there was no traffic, he said.

Limpert had thought to consult with police officer friends in advance and he was ready to call in the state police for an escort, if necessary.

In the two months leading up to the call, Limpert got to know the Georgiadises. He invited the couple to the Cortland Airport to introduce them to his airplane.

They climbed into the four-seater Mooney, tried on the headsets, made sure they were up for a noisy and bumpy ride. Other times, they shared Sunday dinner, homemade baklava, calzones and stories. They talked about their Greek heritage and their children.

Heres what Limpert did not say:

He had his own type of transplant last year.

In January 2018, a doctor at the VA Medical Center noticed elevated protein levels during a routine blood check and referred him to an oncologist. The eventual diagnosis: Multiple myeloma.

Limpert did six months of chemotherapy with so few side effects, he kept it a secret from his children and colleagues. By July, he couldnt hide the treatment any longer. He needed a stem cell transplant.

Doctors at Upstate Medical University harvested 17 million cells, then pushed 5 million of them back into his body. He was in the hospital for two weeks. The rest are in the bank for future treatment.

On the late night drive to Philadelphia, Limpert thought about his own illness, the way he relied on others to pull him through.

In the car, he encouraged the couple to focus on the 10-to-12-hour surgery they faced.

I didnt tell them, Limpert said. I thought they needed to focus on their own health issues.

The GPS sent the three on a ride down the Schuylkill Expressway a highway that often functions more like a parking lot. Limpert took the risk.

We pulled in at 12:43 a.m., Limpert said. We still had 15 minutes to spare.

The lung patient, his wife and their fighter pilot took a selfie. The Georgiadises rushed into the hospital.

Limpert looked at the can of Red Bull unopened in his car. He decided to drive back to Syracuse.

Limpert arrived in his office for arraignments by 9 a.m. Every wall in his chamber has a photo or drawing of a fighter jet. There are commendations and photos from the seven times he was deployed to Southwest Asia, starting with Operation Desert Storm and ending with Operation Enduring Freedom.

This was pretty easy, he said.

Georgiadis surgery was a success.

So far, so good, he said in an interview Tuesday from the hospital. He will be in the hospital for at least two months. He expressed a tearful thank you to the donor, whose identity is not known. They will write a letter to the donors family to see if they want to connect and share their stories.

In the past week, they have stayed in constant touch with Limpert, who they now consider family.

He took a huge burden off our shoulder, Dimitri Georgiadis said. The biggest one ever.

In Greek, Theodore means The Gift of God, Eleni Georgiadis said.

We call Ted our angel, she said.

Contact the author: Michelle Breidenbach | mbreidenbach@syracuse.com | 315-470-3186.

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253 miles in 4 hours: A Syracuse mans race for a lung transplant, and the angel who helped him - syracuse.com