Category Archives: Gene Medicine

Developed at the University of Pennsylvania and licensed to Novartis, CTL019, or tisagenlecleucel, is what scientists are calling a living drug.

A gene therapy to treat a form of blood cancer is just inches away from regulatory approval in the USputting the world in the throes of a paradigm shift in medicine. If the Food and Drug Administration (FDA) accepts the recommendation of one of its panels and gives the nod to Novartiss CTL019, the treatment would become the first-ever gene therapy for any disease to be made available commercially. The small matter of the record aside, the treatments efficacy in treating B-cell acute lymphoblastic leukaemia that has resisted conventional treatment or relapsed in patients as young as 3 years of age is reason enough to celebrate.

Developed at the University of Pennsylvania and licensed to Novartis, CTL019, or tisagenlecleucel, is what scientists are calling a living drugthough it is strictly not a drugbecause it involves engineering specific genes in the patients T lymphocytes (T-cells) that are a part of the bodys cell-mediated immunity, to attack B-cells that are also a part of the bodys immune system, but become malignant in leukaemia. First, a patients T-cells are harvested in millions, and a disabled form of the Human Immunodeficieny Virus is used to transfer the new genetic material to the T-cells genome. The altered T-cells are then intravenously returned to the patients body.

Due to the modification, T-cells recognise CD-19, a surface protein present on B-cells, as an antigen and mount an immune response. A study of 63 patients who received the treatment between April 2015 and August 2016 is the main evidence of CTL019s efficacy52 went into remission while 11 died; 11 of the 52 who had gone into remission had relapsed by November 2016 while, 29 are still in remission. However, there are a few concerns that need addressing. The FDA panel particularly pointed at the severe side-effectssince the modified T-cells dont differentiate between healthy B-cells and cancerous ones, the immune system collapsesand unclear long-term effects. For the moment, though, the opportunistic infections that may assail the patient are the main worry.

Oncologists, however, sound positive, given the immediate side-effects can be stemmedpatients receiving CTL019 are given immunoglobulin infusions every few monthsbut a greater length of time must pass before long-term effects, if any, are evident. Given how concentrated management of the therapy along with side-effects is necessary, Novartis will make it available in just 30-35 medical centres in the US initially. The good news is Novartis is not the only horse in the race in a pole positionKite Pharma, a US-based company, has applied for FDA approval for its Chimeric Antigen Receptor T-cell (CAR-T) therapy that involves similar genetic engineering of T-cells.

Given how difficult it is to ensure consistencyin some cases, the body fails to respondand scale with biologics, the challenges for all such gene therapies are daunting. CTL019s success, however, has an important message for Indiait is perhaps time to shed the stubborn resistance to genetic engineering when a developed nation is ready to adopt it, long-term risks included.

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First gene therapy in the world is here, heralds new era in medicine - Financial Express

Last week, Beijing-based biotech company Sinogene introduced Longlong to the world, a cute beagle puppy that is the first dog ever cloned using gene-editing. Longlong was born from a surrogate mother on May 26th, and even though he looks and acts like any other puppy, hes genetically identical to another dog, 2-year-old Apple. Like Longlong, Apple was born in a laboratory and used to research human diseases.

Lai Liangxue, the lead scientist at Sinogene, thinks animal cloning, although controversial, is necessary for learning about human disease prevention. LaitoldSixth Tonethat Longlongs success means that Chinese biotech companies will be able to conduct biomedical research on their own clones which is also much cheaper than gene-editing.

Meanwhile, Shi Zhensheng, a researcher and professor at China Agriculture University, believes that cloning dogs will benefit both man and mans best friend. The gene-edited dogs have great advantages in helping scientists to research human medicine and genetic diseases, also promoting the study on veterinary medicine, he said, according toCGTN.

Several pet owners have already come forward, Zhao said, seeking to bring beloved family dogs back to life. In South Korea, this sort of thing costs up to $100,000. The cloning of Longlong cost the company 10 million yuan ($1.5 million), a price that Zhao hopes to cut significantly in the coming years.Our price will be half of that, he said. We hope to popularize [such cloning] for the public.

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First gene-edited cloned dog may open gates to more cloning and help fight human diseases - Genetic Literacy Project

Conventional methods for treating diseases, including the 200 or so cancer varieties that are known to science, normally involve taking drugs or undergoing treatments that do what your own body cannot. Gene therapy has come along and, despite being a nascent medical field, has changed everything.

Through a range of different methods, the genes of the patients own cells are edited with breath-taking precision using synthetic viruses or CRISPR; these augmented cells are then reintroduced back into their bodies and their immune systems do the rest of the work. Trialled all over the world, this type of therapy is shown to cure children of seemingly incurable blood cancer, nullify someones sickle cell disease, and more.

Up until this point, gene therapy like this has remained experimental, meaning that it was only available to people taking part in a clinical trial. As reported by The New York Times, however, this is to no longer be the case: the Food and Drug Administration (FDA) is about to officially give the go-ahead for gene therapy to be put on the market for the very first time.

In this case, the therapy will be used to treat people suffering from an aggressive type of blood cancer named B-cell acute lymphoblastic leukemia. It will be made available to children and young adults aged between three and 25 who have been temporarily cured of the disease only to find that it has returned with a vengeance.

The treatment will be patient specific: Their own cells will be shipped off to be altered, frozen, then sent back to be reintroduced into the patient at a later date.

Gene therapy treatment has been used on a range of children across the globe to cure them of this form of leukemia. Although they suffered from quite severe side effects, they were not life-changing and, most importantly, these kids are cancer-free years on.

The family of the first child to have ever been treated in this way, Emily Whitehead, were present at the FDA panel discussing whether or not to authorize the treatment for mainstream medical use. Apart from individual testimonies, a key study showing that this method pushes 82.5 percent of patients into remission was also considered.

The panel was apparently convinced that this gene therapy works where other treatments fail and that thousands of childrens lives will be saved if its approved, so they unanimously recommended that it should be available. Its now certain that the FDA chiefs will green light the treatment.

Although there are still questions about potentially dangerous long-term effects of the procedure, this decision is a landmark one, opening up a brand new world in the fight against cancer, and giving hope to thousands of children around the world.

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Revolutionary Cancer-Killing Gene-Editing Treatment To Be Approved For Mainstream Use - IFLScience

We believe that when this treatment is approved it will save thousands of childrens lives around the world, Emilys father, Tom Whitehead, told the panel. I hope that someday all of you on the advisory committee can tell your families for generations that you were part of the process that ended the use of toxic treatments like chemotherapy and radiation as standard treatment, and turned blood cancers into a treatable disease that even after relapse most people survive.

The main evidence that Novartis presented to the F.D.A. came from a study of 63 patients who received the treatment from April 2015 to August 2016. Fifty-two of them, or 82.5 percent, went into remission a high rate for such a severe disease. Eleven others died.

Its a new world, an exciting therapy, said Dr. Gwen Nichols, the chief medical officer of the Leukemia and Lymphoma Society, which paid for some of the research that led to the treatment.

The next step, she said, will be to determine what we can combine it with and is there a way to use it in the future to treat patients with less disease, so that the immune system is in better shape and really able to fight. She added, This is the beginning of something big.

At the meeting, the panel of experts did not question the lifesaving potential of the treatment in hopeless cases. But they raised concerns about potentially life-threatening side effects short-term worries about acute reactions like those Emily experienced, and longer-term worries about whether the infused cells could, years later, cause secondary cancers or other problems.

Oncologists have learned how to treat the acute reactions, and so far, no long-term problems have been detected, but not enough time has passed to rule them out.

Patients who receive the treatment will be entered in a registry and tracked for 15 years.

Treatments involving live cells, known as biologics are generally far more difficult to manufacture than standard drugs, and the panelists also expressed concerns about whether Novartis would be able to produce consistent treatments and maintain quality control as it scaled up its operation.

Another parent at the meeting, Don McMahon, described his son Connors grueling 12 years with severe and relapsing leukemia, which started when he was 3. Mr. McMahon displayed painful photographs of Connor, bald and intubated during treatment. And he added that chemotherapy had left his son infertile.

A year ago, the family was preparing for a bone marrow transplant when they learned about the cell treatment, which Connor then underwent at Duke University. He has since returned to playing hockey. Compared with standard treatment, which required dozens of spinal taps and painful bone marrow tests, the T-cell treatment was far easier to tolerate, Mr. McMahon said, and he urged the panel to vote for approval.

A third parent, Amy Kappen, also recommended approval, even though her daughter, Sophia, 5, had died despite receiving the cell treatment. But it did relieve her symptoms and give her a few extra months. Sophias disease was far advanced, and Ms. Kappen thought that if the treatment could have been given sooner, Sophia might have survived.

We hope that more families have a longer time with their children fighting this evil disease, and our children deserve this chance, she said.

The treatment was developed by researchers at the University of Pennsylvania and licensed to Novartis.

Use will not be widespread at first because the disease is not common. It affects only 5,000 people a year, about 60 percent of them children and young adults. Most children are cured with standard treatments, but in 15 percent of cases like Emilys and Connors the disease does not respond, or it relapses.

Analysts predict that these individualized treatments could cost more than $300,000, but a spokeswoman for Novartis, Julie Masow, declined to specify a price.

Although the figure may seem high, people with cancer often endure years of expensive treatment and repeat hospital stays that can ultimately cost even more.

Because the treatment is complex and patients need expert care to manage the side effects, Novartis will initially limit its use to 30 or 35 medical centers where employees will be trained and approved to administer it, the company said.

As to whether the treatment, known as CTL019 or tisagenlecleucel (pronounced tis-a-gen-LEK-loo-sell), will be available in other countries, Ms. Masow said by email: Should CTL019 receive approval in the U.S., it will be the decision of the centers whether to receive international patients. We are working on bringing CTL019 to other countries around the world. She added that the company would file for approvals in the European Union later this year.

By late November 2016, 11 of the 52 patients in the study who went into remission relapsed. Twenty-nine were still in remission. Eleven others had further treatments, like bone marrow transplants. One patient was not available for assessment. Three who had relapses died, and one who did not relapse died from a new treatment given during remission. The median duration of remission is not known because it has not been reached: Some patients were still well when last checked.

Researchers are still debating about which patients can safely forgo further treatment, and which might need a bone marrow treatment to give the best chance of a cure.

The treatment requires removing millions of a patients T-cells a type of white blood cell often called soldiers of the immune system and genetically engineering them to kill cancer cells. The technique employs a disabled form of H.I.V., the virus that causes AIDS, to carry new genetic material into the T-cells to reprogram them. The process turbocharges the T-cells to attack B-cells, a normal part of the immune system that turn malignant in leukemia. The T-cells home in on a protein called CD-19 that is found on the surface of most B-cells.

The altered T-cells are then dripped back into the patients veins, where they multiply and start fighting the cancer.

Dr. Carl H. June, a leader of the University of Pennsylvania team that developed the treatment, calls the turbocharged cells serial killers. A single one can destroy up to 100,000 cancer cells.

Because the treatment destroys not only leukemic B-cells but also healthy ones, which help fight germs, patients need treatment to protect them from infection. So every few months they receive infusions of immune globulins.

In studies, the process of re-engineering T-cells for treatment sometimes took four months, and some patients were so sick that they died before their cells came back. At the meeting, Novartis said the turnaround time was now down to 22 days. The company also described bar-coding and other procedures used to keep from mixing up samples once the treatment is conducted on a bigger scale.

Michael Werner, a lawyer and expert on gene and cell technologies and regulation, and a partner at Holland and Knight in Washington, said that results so far proved that T-cell treatment works.

The fact that it can be done means more people will go into the field and more companies will start developing these products. He added, I think were in for really exciting times.

A version of this article appears in print on July 13, 2017, on Page A1 of the New York edition with the headline: F.D.A. Panel Urges New Living Drug To Fight Cancer.

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FDA Panel Recommends Approval for Gene-Altering Leukemia Treatment - New York Times

Dr. Erin Kinney used to assess patients nutritional needs with an analysis of their eating habits and family and medical histories. Nowadays the Arnold naturopathic doctor is delving deeper and also analyzing her patients' genetic makeup.

She is part of a growing number of dieticians, nutritionists and holistic doctors practicing nutritional genetics, or looking at how variations in genes can modify the affects of nutrients on health. The health care providers are using the information to help patients figure out which foods they should eat or avoid, and that best suits their biological makeup.

"We are now learning that maybe you have a certain genetic disposition to not making enough of a certain enzyme," said Kinney, who is also president of the Maryland Naturopathic Doctors Association. "It is really changing the way we are going to be able to tailor our treatments for patients."

Dieticians for years have used genetics on a limited basis, looking at mutations in one particular gene that may make patients prone to disease or more likely to have certain health conditions. For example, patients who are lactose intolerant can't break down the lactose found in dairy products because of a mutation in the lactose gene. So they suffer with gas and other uncomfortable digestive problems after drinking milk or eating cheese or ice cream.

The sequencing of the human genome - or the mapping of every gene in the body - has enabled not just dieticians, but all doctors, to use genetics in a more comprehensive way. A doctor can look at a patient's entire genetic makeup to determine, for instance, if they have multiple gene mutations that would make them more prone to obesity or cardiovascular disease.

Algerina Perna / Baltimore Sun

Most human genes are the same from person to person, but some have variations. The most common type of variation is known as a SNP (pronounced "snips"), or single nucleotide polymorphisms, and accounts for difference in physical characteristics of people, including eye and hair color or blood type. Different SNPs can also determine if someone will be more prone to developing certain diseases.

"The genome is the gold mine where genes can be targeted and we can learn more about how nutrients can play a role in people's health," said Braxton Mitchell, a genetic epidemiologist at the University of Maryland School of Medicine who studies the genetics of complex diseases.

But Mitchell and others caution the field is still emerging and that ongoing research is needed to sort out the many ways genetics could be used to guide nutrition and food choices.

The Academy of Nutrition and Dietetics wrote a position paper in 2014, published in the organization's journal, that said nutritional genomics is not ready for widespread use.

"The practical application of nutritional genomics for complex chronic disease is an emerging science and the use of nutrigenetic testing to provide dietary advice is not ready for routine dietetics practice," the position paper said. "Registered dietitian nutritionists need basic competency in genetics as a foundation for understanding nutritional genomics; proficiency requires advanced knowledge and skills."

A spokesman for the organization said it still stands behind that statement.

"It is pretty complicated right now because genes are complicated," said Ginger Hultin, a dietician and spokeswoman for the Academy of Nutrition and Dietetics. "It is not generally enough to just look at a single gene variation. There is so much to look at on genes and you also have to consider a person's lifestyle and how they interact with the environment."

The Maryland University of Integrative Health held its first ever symposium last month to encourage what they called "knowledge sharing" on the interaction between genetics and dietary factors. The conference, held in partnership with the Maryland Naturopathic Doctors Association, was focused on giving health care providers practical advice on how to incorporate nutritional genomics testing, counseling and treatment into clinical practice.

Christy Williamson, an adjunct professor at the university who also owns a genetic nutritional company, said that variations in genes might mean that two people with diabetes might need different diets customized for their biological makeup. Other people might have a gene variation that causes them to need more exercise to get enough molecular oxygen to lose weight.

There is also the possibility that a mutation might not have any affect on the body.

"Certain things are epigenetically activated," Williamson said. "That doesn't mean everybody who has a gene variation will get that particular disease. Genetics kind of loads the gun and the environment pulls the trigger."

Lifestyle can also offset the affects of the gene variation. If your gene makes you more prone to gain weight, for instance, extra exercise could help you fend off weight gain.

"You can have bad genes, but if you have a good lifestyle you can change the way your genes are expressed," Kinney said. "Your body can get back into balance despite that your body has these gene mutations."

Companies like 23andMe, Vitagene and Nutrigenomix will map a patient's genome using a swab of saliva. Kinney then uses a system called Opus 23 Pro that analyzes the data.

Kristen Kissik, an Annapolis dance and yoga instructor, was one of those patients who had her genome analyzed. Kissik wanted to make sure she was doing everything she could to ensure she had the most optimal health. She had been eating foods she knew would help reduce inflammation, improve her thyroid functions and ease her gluten sensitivity.

"The stuff I got almost affirmed my intuition," Kissik said of the results her genome analysis. "I know I had an issue and it is in my genes."

Mitchell, the University of Maryland epidemiologist, believes nutritional genomics has the potential to one day help decide which diets are best for people. He predicts it could also eventually lead to new treatments for various diseases and medical conditions. But more research needs to be done to validate the potential benefits.

"There is a lot going on in the field," he said. "We have to be skeptical when see results from early studies. We want to see results repeated before we believe them."

amcdaniels@baltsun.com

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Genetics playing a growing role in intersection of nutrition and health - Baltimore Sun

July 13, 2017 by Nora Dunne Localization of pro-fibrotic hormones in heart muscle cells (green), which synthesize and release transforming growth factor-beta (red) following cardiac injury. Nuclei are shown in blue. Credit: Northwestern University

Northwestern Medicine scientists have identified a novel molecular mechanism that regulates scar formation in the heart, a common manifestation of aging and nearly every form of heart disease. The discovery was published in the journal Circulation.

The scientists showed that heart muscle cells called cardiomyocytes are a principal source of the molecular signals that drive scarring in the heart, a process known as cardiac fibrosis.

"Historically, these signals were thought to arise from other cell types in the heart," explained Panagiotis (Peter) Flevaris, MD, PhD, '12, '17 GME, instructor of Medicine in the Division of Cardiology and first author of the paper. "We also identified the gene that serves as the master regulator of the synthesis and release of pro-fibrotic signals from heart muscle cells across different species."

First, Flevaris and colleagues showed that a familial mutation in that gene, which encodes the protein plasminogen activator inhibitor-1 (PAI-1), leads to spontaneous cardiac fibrosis in otherwise healthy individuals in an Old Order Amish community. The findings in the human population confirmed previous observations in mice lacking the gene for PAI-1.

Importantly, the scientists found that a protein called bone morphogenetic protein 7 (BMP7) can prevent the generation of fibrotic signals by cardiomyocytes and may be able to serve as a future therapy for cardiac fibrosis.

"Tissue fibrosis is the leading cause of organ failure, but persists as one of the most pressing global health problems due to lack of effective pharmacotherapies. Currently, the only cure for cardiac fibrosis is heart transplantation," said Flevaris, who completed this research during a post-doctoral fellowship in the Department of Medicine's Physician-Scientist Training Program, working with senior author Douglas Vaughan, MD, Irving S. Cutter Professor of Medicine and chair of Medicine.

"Supported by clinical and functional data, our work provides exciting new evidence that modulation of cardiomyocyte signals by BMP7 represents a unique therapeutic strategy to prevent cardiac fibrosis," Flevaris added. "This discovery not only provides a novel paradigm for how the heart muscle generates signals following injury, but also has the potential to transform healthcare for the heart failure population at large."

Explore further: Study shows cardiac fibrosis reversal through gene targeting in heart failure models

More information: Panagiotis Flevaris et al. PAI-1 Controls Cardiomyocyte TGF- and Cardiac Fibrosis, Circulation (2017). DOI: 10.1161/CIRCULATIONAHA.117.028145

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Root of cardiac fibrosis defined - Medical Xpress

July 12, 2017 Killer T cells surround a cancer cell. Credit: NIH

Early phase Northwestern Medicine research has demonstrated a potential new therapeutic strategy for treating deadly glioblastoma brain tumors.

The strategy involves using lipid polymer based nanoparticles to deliver molecules to the tumors, where the molecules shut down key cancer drivers called brain tumor initiating cells (BTICs).

"BTICs are malignant brain tumor populations that underlie the therapy resistance, recurrence and unstoppable invasion commonly encountered by glioblastoma patients after the standard treatment regimen of surgical resection, radiation and chemotherapy," explained the study's first author, Dr. Dou Yu, research assistant professor of neurological surgery at Northwestern University Feinberg School of Medicine.

The findings were published in the journal Proceedings of the National Academy of Sciences.

Using mouse models of brain tumors implanted with BTICs derived from human patients, the scientists injected nanoparticles containing small interfering RNA (siRNA)short sequences of RNA molecules that reduce the expression of specific cancer promoting proteinsdirectly into the tumor. In the new study, the strategy stopped tumor growth and extended survival when the therapy was administered continuously through an implanted drug infusion pump.

"This major progress, although still at a conceptual stage, underscores a new direction in the pursuit of a cure for one of the most devastating medical conditions known to mankind," said Yu, who collaborated on the research with principal investigator Dr. Maciej Lesniak, Michael J. Marchese Professor of Neurosurgery and chair of neurological surgery.

Glioblastoma is particularly difficult to treat because its genetic makeup varies from patient to patient. This new therapeutic approach would make it possible to deliver siRNAs to target multiple cancer-causing gene products simultaneously in a particular patient's tumor.

In this study, the scientists tested siRNAs that target four transcription factors highly expressed in many glioblastoma tissuesbut not all. The therapy worked against classes of glioblastoma BTICs with high levels of those transcription factors, while other classes of the cancer did not respond.

"This paints a picture for personalized glioblastoma therapy regimens based on tumor profiling," Yu said. "Customized nanomedicine could target the unique genetic signatures in any specific patient and potentially lead to greater therapeutic benefits."

The strategy could also apply to other medical conditions related to the central nervous systemnot just brain tumors.

"Degenerative neurological diseases or even psychiatric conditions could potentially be the therapeutic candidates for this multiplexed delivery platform," Yu said.

Before scientists can translate this proof-of-concept research to humans, they will need to continue refining the nanomedicine platform and evaluating its long-term safety. Still, the findings from this new research provide insight for further investigation.

"Nanomedicine provides a unique opportunity to advance a therapeutic strategy for a disease without a cure. By effectively targeting brain tumor initiating stem cells responsible for cancer recurrence, this approach opens up novel translational approaches to malignant brain cancer," Lesniak summed up.

Explore further: Cold virus, stem cells tested to destroy deadly brain cancer

More information: Dou Yu et al, Multiplexed RNAi therapy against brain tumor-initiating cells via lipopolymeric nanoparticle infusion delays glioblastoma progression, Proceedings of the National Academy of Sciences (2017). DOI: 10.1073/pnas.1701911114

Early phase Northwestern Medicine research has demonstrated a potential new therapeutic strategy for treating deadly glioblastoma brain tumors.

To enhance the visibility of organs as they are scanned with magnetic resonance imaging (MRI), patients are usually injected with a compound known as a contrast agent before going into the scanner. The most commonly used ...

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Nanomedicine opens door to precision medicine for brain tumors - Phys.Org

Kolon Life Science filed for a Biologics License Application (BLA) for Invossa-K Inj. with MFDS in August 2016 based on efficacy results from its Phase III clinical trials conducted at 12 major university hospitals in Korea. Invossa-K Inj. will be manufactured by Kolon Life Science, and marketed by Mundipharma and Kolon Pharmaceuticals for the Korean market.

"We are excited to launch the world's first cell and gene therapy for knee osteoarthritis and potentially the world's first disease-modifying osteoarthritis drug (DMOAD)," stated Mr. Woosok Lee, CEO of TissueGene. "This approval is the first critical step towards a global launch for this innovative, novel cell and gene therapy technology that will address one of the most pressing unmet medical need affecting millions of people suffering from osteoarthritis worldwide."

Invossa is a first-in-class cell and gene therapy drug designed to conveniently and effectively treat osteoarthritis of the knee through a single intra-articular injection. Clinical trials completed in Korea and on-going in the US have demonstrated pain relief, increased mobility, and potentially game-changing improvements in joint structure offering substantial relief and convenience for osteoarthritis patients who would otherwise be in need of surgery.

Through its national US Phase III clinical trials, TissueGene will be using the results to seek a DMOAD designation for Invossa from the US Food and Drug Administration (FDA), potentially making Invossa the first and only cell and gene therapy for osteoarthritis of the knee.

In November last year, Kolon Life Science signed a license agreement with Mitsubishi Tanabe Pharmaceutical Corporation, and Mitsubishi Tanabe Pharma is proceeding with the preparation of clinical trials through its exclusive development and commercialization rights in Japan.

TissueGene, Inc. TissueGene, Inc., is a Maryland-based regenerative medicine company specializing in cell and gene therapy. TissueGene's lead product is Invossa, an allogeneic, cell and gene therapy for osteoarthritis of the knee that is preparing for Phase III clinical trials in the US pursuant to a Special Protocol Assessment (SPA) agreement reached with the U.S. Food and Drug Administration. Information about the trials can be found at the NIH registry, ww.clinicaltrials.gov. For additional information about TissueGene, Inc., please visit http://www.tissuegene.com.

Kolon Life Science Kolon Life Science has been developing innovative cell and gene therapies including Invossa K Inj., the world's first cell-mediated gene therapy for osteoarthritis, since its founding in 2000. In addition to its biopharmaceuticals business, the company is also engaged in the business of providing active pharmaceuticals ingredients (API), eco-chemicals including antimicrobials for personal-care and industrial applications, as well as water-treatment solutions. For more information, please visit http://www.kolonls.co.kr/eng

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Korea Approves the World's First Cell and Gene Therapy for Knee Osteoarthritis - PR Newswire (press release)

Published Wednesday, Jul. 12, 2017, 7:35 am

Front Page Events New way to shut off genes speeds battle against genetic diseases

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Gene editing using the CRISPR system is transforming genetic research and could be poised to transform the treatment of genetic diseases such ashemophiliaandcystic fibrosis. Now aUniversity of Virginia School of Medicineresearcher has found a safer and highly efficient way to use the system to silence genes.

Mazhar Adli, PhD, and his team have developed a technique to prevent genes from carrying out their function without causing the extensive DNA damage the current approach requires. This is important because silencing genes allows scientists to understand what individual genes do and identify the ones that cause disease. Gene silencing also may one day let doctors better treat and even cure genetic diseases with abnormal gene activity.

Our genes the blueprints for life are segments of the long double strands of DNA in our cells. To silence genes using the current approach, the CRISPR system cuts both strands. Doing this too often causes the cells to die a major limitation for CRISPR. Even cuts that are not fatal to cells can have unintended effects that result from the bodys efforts to repair the broken DNA.

Adlis approach, on the other hand, avoids cutting the DNA altogether. Instead, it takes advantage of the fact that DNA is made of four main building blocks: cytosine, adenine, guanine and thymine. Adlis method lets scientists use CRISPR to convert one building block into another to artificially create what are called stop codons the off switches that naturally occur at the end of genes. Turn cytosine into thymine, for example, and the whole gene is silenced, meaning there is no protein production from that gene.

We found around 17,000 genes we can target this way, and, as you know, we have roughly 20,000 genes, Adli explained.So a very large fraction of the genome we can target with this CRISPR stop approach.

He noted the benefits of the new techniques genetic alchemy: Its about as efficient as what we used previously, he said. But more importantly, it is safer. It doesnt cause cell death. Further, the new approach is compatible with high-throughput screening that lets scientists do their research much more quickly. You can delete basically every single gene [of certain types] in a population of cells and then watch the entire population to see whats going to happen to them, he said. So with a single experiment you can interrogate the function of thousands and thousands of genes.

Adli, of UVAs Department of Biochemistry and Molecular Genetics, is making his technique available for free to scientists around the world. Any lab that uses CRISPR should have the capability to use it, he said. He expects it will initially be used for research in labs like his, but, with the first human tests of gene editing now beginning, that may change in the future.

Adlis team has outlined the new technique in an article in the scientific journalNature Methods. The article was written by Cem Kuscu, Mahmut Parlak, Turan Tufan, Jiekun Yang, Karol Szlachta, Xiaolong Wei, Rashad Mammadov and Adli.

The work was supported by a V Scholar award from the V Cancer Research Foundation and a pilot project award from the UVA Cancer Center.

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New way to shut off genes speeds battle against genetic diseases - Augusta Free Press

Breathing in a New Gene Therapy to treat Pulmonary Hypertension

Newswise (New York, NY July 12, 2017) Mount Sinai has partnered with Theragene Pharmaceuticals, Inc. to advance a novel airway-delivered gene therapy for treating pulmonary hypertension (PH), a form of high blood pressure in blood vessels in the lungs that is linked to heart failure. If the therapy succeeds in human clinical trials, it will provide patients for the first time with a way to reverse the damage caused by PH.

This gene therapy technique comes from the research of Roger J. Hajjar, MD, Professor of Medicine and Director of the Cardiovascular Research Center at the Icahn School of Medicine at Mount Sinai, and has been proven effective in rodent and pig animal models. PH is a deadly disease that disproportionately affects young adults and women; 58 percent of cases are found in young adults and 72 percent are women. There is currently no effective cure for PH, and about 50 percent of people who are diagnosed will die from the disease within five years.

PH is a rare (15-50 cases per million people), rapidly progressing disease that occurs when blood pressure is too high in vessels leading from the heart to the lungs. The high pressure is caused by abnormal remodeling of the lung blood vessels, characterized by a proliferation of smooth muscle cells and a thickening and narrowing of these vessels, and can lead to failure of the right ventricle of the heart and premature death. Abnormalities in calcium cycling within the vascular cells play a key role in the pathophysiology of pulmonary hypertension, along with deficiencies in the sarcoplasmic reticulum calcium ATPase pump (SERCA2a) protein which regulates intracellular calcium within these vascular cells and prevents them from proliferating within the vessel wall. Downregulation of SERCA2a leads to the proliferative remodeling of the vasculature. This gene therapy, delivered via an inhaled aerosolized spray, aims to increase the expression of SERCA2a protein, and has been shown in rodents and pigs to improve heart and lung function, as well as reduce and even reverse cellular changes caused by PH.

This is a devastating disease, and our work in collaboration with many laboratories across the country has allowed us to identify a specific molecular target and use gene therapy to improve cardiovascular and lung parameters in experimental models of PH. We look forward to starting first-in-human studies using this approach in affected patients, said Dr. Hajjar, the senior author of the studies, highlighting that clinical trials will be underway in the next two years.It may take several years before a product is commercially available for PH patients.

We are excited about the potential for SERCA2a gene therapy as a new modality in treating this serious disease, said Jon Berglin, Chief Executive Officer of Theragene Pharmaceuticals, Inc. We look forward to develop and advance this promising product into the clinic.

This represents another critical advancement in a potentially transformative therapeutic breakthrough by Mount Sinai scientists, demonstrating our commitment to improving health outcomes. We are thrilled to be working with Theragene Pharmaceuticals, and continue to strengthen our expertise in partnering health care innovations with industry, said Erik Lium, PhD, Senior Vice President of Mount Sinai Innovation Partners, the commercialization arm of the Icahn School of Medicine at Mount Sinai.

About Mount Sinai Innovation Partners (MSIP)MSIP is responsible for driving the real-world application and commercialization of Mount Sinai discoveries and the development of research partnerships with industry. The aim is to translate these innovations into healthcare products and services that benefit patients and society. MSIP is responsible for the full spectrum of commercialization activities required to bring the Icahn School of Medicine and the Mount Sinai Health Systems inventions to life. These activities include evaluating, patenting, marketing and licensing new technologies, engaging commercial and non-profit relationships for sponsored research, material transfer and confidentiality, as well as fostering an ecosystem of entrepreneurship within our research and health system communities. For more information, visit http://www.ip.mountsinai.org.

About Theragene Pharmaceuticals, Inc.Theragene is a biopharmaceutical company developing cutting-edge science for the treatment of debilitating diseases. The Companys diverse portfolio consists of preclinical and clinical oncology and cardiology platforms utilizing next generation gene therapy and immunotherapy methods.

Original post:
Breathing in a New Gene Therapy to Treat Pulmonary Hypertension - Newswise (press release)