Scientists in Australia claim to have discovered what could be a life-long cure for potentially fatal allergies to peanuts, shellfish and other food.

The researchers said they had been able to turn off the allergic response in tests on mice using gene therapy to desensitise the bodys immune system, and suggested this could also be used to treat asthma.

They predicted human trials could begin in just five or six years.

Commenting on the study, a leading British expert said scientists had managed to cure allergies in mice before without this leading to an effective human treatment, but added that the new research could lead to the "Holy Grail" of allergy treatment.

He was sceptical about the researchers' claims their technique might be effective against asthma, but Asthma UK said it was "a very exciting step forward".

Allergies occur when the immune system over-reacts to something that is usually harmless. In the journal JCI Insight, the Australian researchers reported they had used genetic techniques to prevent this from happening in mice who were allergic to the protein in egg whites.

In a video about the new research, Professor Ray Steptoe, of Queensland University, said: We can actually turn off the response. What that means is the disease is stopped in its tracks.

What we do is we stop the underlying disease that causes these symptoms. That could revolutionise treatment for severe allergies. It would prevent, we think, some of the life-threatening allergic episodes that occur for people who are allergic to foods for instance.

That would make a huge difference for people with severe allergies what that would mean is they would no longer be in fear of life-threatening incidents if they were to go to a restaurant and be exposed to shellfish and they werent aware that was in the food.

Kids with peanut allergies could go to school without any fear of being contaminated from other kids food.

We envisage in the future, with this approach, that they could go to the doctors rooms, get a single treatment and that would give them permanent protection from future allergic attacks or asthma attacks.

He added that the researchers hoped human trials could begin in five to six years, estimated it would take a similar period after that for the treatment to be available to patients.

Professor Adnan Custovic, an allergy expert at Imperial College London, expressed particular caution about the claim the treatment would be effective against asthma as the condition is caused by a completely different mechanism to the one behind food allergies.

But he added: This is one of the potentially exciting approaches to treating allergies.

Its sort of approach, where you try to switch off the allergic response, is kind of the Holy Grail, but a mouse model is not the same as a human model.

We can cure allergies in mice but we cannot do it in humans the mechanisms are not identical. Only time will tell whether this approach will be a viable one.

And he criticised the degree of optimism about the technique expressed by the Australian team.

My real problem with this sort of bombastic statements like this is people with asthma it gives them hope which very often is not realistic, Professor Custovic said.

However Dr Erika Kennington, head of research at Asthma UK, was more optimistic.

This is potentially a very exciting step forward in asthma research," she said.

"Allergen immunotherapy exposing people to small amounts of an allergen in order to build up tolerance is currently the only disease-altering treatment available for asthma but it can have significant side effects in some people, and every other existing asthma treatment and medication works by reducing or relieving the symptoms.

"These findings suggesting a novel approach to reversing allergic disease are therefore very welcome.

We also know that there are certain allergy triggers that cause asthma flare ups, which makes this research important in possibly reducing the risk of life-threatening asthma attacks."

But Dr Kennington also pointed to the difference between animal and human trials.

A lot more research is needed to see if the same results can be achieved in people before we can say that a cure for asthma is around the corner," she said.

In the study of the allergic mice, the researchers inserted a gene into blood stem cells that controls the immune response to the egg white.

The genetically modified cells were then injected into the mices bone marrow, where they produced new blood cells that were able to turn off the allergic response.

The researchers hope to create a similar form of gene therapy that works on humans after a single injection.

We havent quite got it to the point where its as simple as getting a flu jab, so we are working on making it simpler and safer so it could be used across a wide cross-section of affected individuals, Dr Steptoe said.

Dr Louisa James, British Society for Immunology spokesperson and an immunologistat Queen Mary University of London, said allergies were "far more complex than can be replicated in an animal model".

"Patients with severe allergies often react to several different types of allergen and symptoms can develop over several years," she said.

"Although the results are encouraging and heading in the right direction, it is too early to predict whether this form of therapy could ever be used to treat allergies in humans.

"As the authors state in their paper 'gene-therapy is not yet suitable for clinical application to mild disease in young individuals'.

"There are simply too many open questions around the translation of these findings from animal models into humans.Would the cells engineered to produce allergens produce the same response in humans? How would other immune cells that play a critical role in human allergy be affected? What are the mechanisms that switch off the immune response and are they comparable in humans?

This approach holds promise, and further research is certainly warranted, but claims that a single injection could switch off allergies are over-optimistic at this time.

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Allergy treatment: Scientists claim breakthrough that could lead to ... - The Independent

Doctors are reporting unprecedented success from a new cell and gene therapy for multiple myeloma, a blood cancer that's on the rise. Although it's early and the study is small 35 people every patient responded and all but two were in some level of remission within two months.

In a second study of nearly two dozen patients, everyone above a certain dose responded.

Experts at an American Society of Clinical Oncology conference in Chicago, where the results were announced Monday, say it's a first for multiple myeloma and rare for any cancer treatment to have such success.

Chemotherapy helps 10 to 30 percent of patients; immune system drugs, 35 to 40 percent at best, and some gene-targeting drugs, 70 to 80 percent, "but you don't get to 100," said Dr. Len Lichtenfeld, deputy chief medical officer of the American Cancer Society.

"These are impressive results" but time will tell if they last, he said.

ABOUT THE DISEASE

Multiple myeloma affects plasma cells, which make antibodies to fight infection. More than 30,000 cases occur each year in the United States, and more than 115,000 worldwide. It's the second fastest growing cancer for men and the third for women, rising 2 to 3 percent per year, according to the National Cancer Institute. About 60,000 to 70,000 Americans have it now.

Nine new drugs have been approved for it since 2000 but they're not cures; only about half of U.S. patients live five years after diagnosis.

With cell therapy, "I can't say we may get a cure but at least we bring hope of that possibility," said Dr. Frank Fan. He is chief scientific officer of Nanjing Legend Biotech, a Chinese company that tested the treatment with doctors at Xi'an Jiaotong University.

HOW IT WORKS

The treatment, called CAR-T therapy , involves filtering a patient's blood to remove immune system soldiers called T cells. These are altered in a lab to contain a gene that targets cancer and then given back to the patient intravenously.

Doctors call it a "living drug" a one-time treatment to permanently alter cells that multiply in the body into an army to fight cancer. It's shown promise against some leukemias and lymphomas, but this is a new type being tried for multiple myeloma, in patients whose cancer worsened despite many other treatments.

THE STUDIES

In the Chinese study, 19 of 35 patients are long enough past treatment to judge whether they are in complete remission, and 14 are. The other five had at least a partial remission, with their cancer greatly diminished. Some are more than a year past treatment with no sign of disease.

Most patients had a group of side effects common with this treatment, including fever, low blood pressure and trouble breathing. Only two cases were severe and all were treatable and temporary, doctors said.

The second study was done in the U.S. by Bluebird Bio and Celgene, using a cell treatment developed by the National Cancer Institute. It tested four different dose levels of cells in a total of 21 patients. Eighteen are long enough from treatment to judge effectiveness, and all 15 who got an adequate amount of cells had a response. Four have reached full remission so far, and some are more than a year past treatment.

WHAT EXPERTS SAY

The results are "very remarkable" not just for how many responded but how well, said Dr. Kenneth Anderson of Dana-Farber Cancer Institute in Boston.

"We need to be looking for how long these cells persist" and keep the cancer under control, he said.

Dr. Carl June, a University of Pennsylvania researcher who received the conference's top science award for his early work on CAR-T therapy, said "it's very rare" to see everyone respond to a treatment. His lab also had this happen all 22 children testing a new version of CAR-T for leukemia responded, his colleagues reported at the conference.

"The first patients we treated in 2010 haven't relapsed," June said.

Dr. Michael Sabel of the University of Michigan called the treatment "revolutionary."

"This is really the epitome of personalized medicine," extending immune therapy to more types of patients, he said.

NEXT STEPS

Legend Biotech plans to continue the study in up to 100 people in China and plans a study in the U.S. early next year. The treatment is expected to cost $200,000 to $300,000, and "who's going to pay for that is a big issue," Fan said.

"The manufacturing process is very expensive and you can't scale up. It's individualized. You cannot make a batch" as is done with a drug, he said.

Nick Leschly, Bluebird's chief executive, said the next phase of his company's study will test what seems the ideal dose in 20 more people.

Marilynn Marchione can be followed at http://twitter.com/MMarchioneAP

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A first: All respond to gene therapy in a blood cancer study - ABC News

A consultant studies a mammogram. The drug olaparib could slow cancer growth by three months, researchers have found. Photograph: Rui Vieira/PA

A type of inherited and incurable breast cancer that tends to affect younger women could be targeted by a new therapy, researchers have found.

A small study presented at the worlds largest cancer conference found treating patients with the drug olaparib could slow cancer growth by three months and be less toxic for patients with inherited BRCA-related breast cancer.

Researchers said there was not enough data to say whether patients survived longer as a result of the treatment.

We are in our infancy, said Dr Daniel Hayes, president of the American Society of Clinical Oncology and professor of breast cancer research at the University of Michigan. This is clearly an advance; this is clearly proof of concept these can work with breast cancer.

Does it look like its going to extend life? We dont know yet, he said.

The drug is part of the developing field of precision medicine, which targets patients genes to tailor treatment.

It is a perfect example of how understanding a patients genetics and the biology of their tumor can be used to target its weaknesses and personalize treatment, said Andrew Tutt, director of the Breast Cancer Now Research Centre at The Institute of Cancer Research.

Olaparib is already available for women with BRCA-mutant advanced ovarian cancer, and is the first drug to be approved that is directed against an inherited genetic mutation. The study was the first to show olaparib can slow growth of inherited BRCA-related breast cancer. The drug is not yet approved for that use.

People with inherited mutations in the BRCA gene make up about 3% of all breast cancer patients, and tend to be younger. The median age of women in the olaparib trial was 44 years old.

BRCA genes are part of a pathway to keep cells reproducing normally. An inherited defect can fail to stop abnormal growth, thus increasing the risk of cancer. The study examined the effectiveness of olaparib against a class of BRCA-related cancers called triple negative. Olaparib is part of a class of four drugs called PARP-inhibitors that work by shutting down a pathway cancer cells use to reproduce.

The study from Memorial Sloan Kettering Cancer Center in New York randomly treated 300 women with advanced, BRCA-mutated cancer with olaparib or chemotherapy. All the participants had already received two rounds of chemotherapy.

About 60% of patients who received olaparib saw tumors shrink, compared with 29% of patients who received chemotherapy. That meant patients who received olaparib saw cancer advance in seven months, versus four months for only chemotherapy.

Researchers cautioned it is unclear whether olaparib extended life for these patients, and that more research was needed to find out which subset of patients benefit most from olaparib.

Original post:

New therapy offers hope against incurable form of breast cancer - The Guardian

By Laurie McginleyThe Washington Post

The oncologist was blunt: Stefanie Joho's colon cancer was raging out of control and there was nothing more she could do. Flanked by her parents and sister, the 23-year-old felt something wet on her shoulder. She looked up to see her father weeping.

"I felt dead inside, utterly demoralized, ready to be done," Joho remembers.

But her younger sister couldn't accept that. When the family got back to Joho's apartment in New York's Flatiron district, Jess opened her laptop and began searching frantically for clinical trials, using medical words she'd heard but not fully understood. An hour later, she came into her sister's room and showed her what she'd found.

"I'm not letting you give up," she told Stefanie. "This is not the end."

That search led to a contact at Johns Hopkins University, and a few days later, Joho got a call from a cancer geneticist co-leading a study there.

"Get down here as fast as you can!" Luis Diaz said. "We are having tremendous success with patients like you."

What followed is an illuminating tale of how one woman's intersection with experimental research helped open a new frontier in cancer treatment with approval of a drug that, for the first time, targets a genetic feature in a tumor rather than the disease's location in the body.

The breakthrough, now made official by the Food and Drug Administration, immediately could benefit some patients with certain kinds of advanced cancer that aren't responding to chemotherapy. Each should be tested for that genetic signature, scientists stress.

"These are people facing death sentences," said Hopkins geneticist Bert Vogelstein. "This treatment might keep some of them in remission for a long time."

A pivotal small trial

In August 2014, Joho stumbled into Hopkins for her first infusion of the immunotherapy drug Keytruda. She was in agony from a malignant mass in her midsection, and even with the copious amounts of OxyContin she was swallowing, she needed a new fentanyl patch on her arm every 48 hours. Yet within just days, the excruciating back pain had eased. Then an unfamiliar sensation hunger returned. She burst into tears when she realized what it was.

As months went by, her tumor shrank and ultimately disappeared. She stopped treatment this past August, free from all signs of disease.

The small trial in Baltimore was pivotal, and not only for the young marketing professional. It showed that immunotherapy could attack colon and other cancers thought to be unstoppable. The key was their tumors' genetic defect, known as mismatch repair (MMR) deficiency akin to a missing spell-check on their DNA. As the DNA copies itself, the abnormality prevents any errors from being fixed. In the cancer cells, that means huge numbers of mutations that are good targets for immunotherapy.

The treatment approach isn't a panacea, however. The glitch under scrutiny which can arise spontaneously or be inherited is found in just 4 percent of cancers overall. But bore in on a few specific types, and the scenario changes dramatically. The problem occurs in up to 20 percent of colon cancers and about 40 percent of endometrial malignancies cancer in the lining of the uterus.

In the United States, researchers estimate that initially about 15,000 people with this defect may be helped by this immunotherapy. That number is likely to rise sharply as doctors begin using it earlier on eligible patients.

Joho was among the first.

Even before Joho got sick, cancer had cast a long shadow on her family. Her mother has Lynch syndrome, a hereditary disorder that sharply raises the risk of certain cancers, and since 2003, Priscilla Joho has suffered colon cancer, uterine cancer and squamous cell carcinoma of the skin.

Stefanie's older sister, Vanessa, had already tested positive for Lynch syndrome, and Stefanie planned to get tested when she turned 25. But at 22, several months after she graduated from New York University, she began feeling unusually tired. She blamed the fatigue on her demanding job. Her primary-care physician, aware of her mother's medical history, ordered a colonoscopy.

When Joho woke up from the procedure, the gastroenterologist looked "like a ghost," she said. A subsequent CT scan revealed a very large tumor in her colon. She'd definitely inherited Lynch syndrome.

She underwent surgery in January 2013 at Philadelphia's Fox Chase Cancer Center, where her mother had been treated. The news was good: The cancer didn't appear to have spread, so she could skip chemotherapy and follow up with scans every three months.

By August of that year, though, Joho started having relentless back pain. Tests detected the invasive tumor in her abdomen. Another operation, and now she started chemo. Once again, in spring 2014, the cancer roared back. Her doctors in New York, where she now was living, switched to a more aggressive chemo regimen.

"This thing is going to kill me," Joho remembered thinking. "It was eating me alive."

Genetics meets immunology

Joho began planning to move to her parents' home in suburban Philadelphia: "I thought, 'I'm dying, and I'd like to breathe fresh air and be around the green and the trees.' "

Her younger sister wasn't ready for her to give up. Jess searched for clinical trials, typing in "immunotherapy" and other terms she'd heard the doctors use. Up popped a trial at Hopkins, where doctors were testing a drug called pembrolizumab.

"Pembro" is part of a class of new medications called checkpoint inhibitors that disable the brakes that keep the immune system from attacking tumors. In September 2014, the treatment was approved by the FDA for advanced melanoma and marketed as Keytruda. The medication made headlines in 2015 when it helped treat former President Jimmy Carter for melanoma that had spread to his brain and liver. It later was cleared for several other malignancies.

Yet researchers still don't know why immunotherapy, once hailed as a game changer, works in only a minority of patients. Figuring that out is important for clinical as well as financial reasons. Keytruda, for example, costs about $150,000 a year.

By the time Joho arrived at Hopkins, the trial had been underway for a year. While an earlier study had shown a similar immunotherapy drug to be effective for a significant proportion of patients with advanced melanoma or lung or kidney cancer, checkpoint inhibitors weren't making headway with colon cancer. A single patient out of 20 had responded in a couple of trials.

Why did some tumors shrink while others didn't? What was different about the single colon cancer patient who benefited? Drew Pardoll, director of the Bloomberg-Kimmel Institute for Cancer Immunotherapy at Hopkins, and top researcher Suzanne Topalian took the unusual step of consulting with the cancer geneticists who worked one floor up.

"This was the first date in what became the marriage of cancer genetics and cancer immunology," Pardoll said.

In a brainstorming session, the geneticists were quick to offer their theories. They suggested that the melanoma and lung cancer patients had done best because those cancers have lots of mutations, a consequence of exposure to sunlight and cigarette smoke. The mutations produce proteins recognized by the immune system as foreign and ripe for attack, and the drug boosts the system's response.

And that one colon-cancer patient? As Vogelstein recalls, "We all said in unison, 'He must have MMR deficiency!' " because such a genetic glitch would spawn even more mutations.

When the patient's tumor tissue was tested, it was indeed positive for the defect.

The researchers decided to run a small trial, led by Hopkins immunologist Dung Le and geneticist Diaz, to determine whether the defect could predict a patient's response to immunotherapy. The pharmaceutical company Merck provided its still-experimental drug pembrolizumab. Three groups of volunteers were recruited: 10 colon cancer patients whose tumors had the genetic problem; 18 colon cancer patients without it; and 7 patients with other malignancies with the defect.

The first results, published in 2015 in the New England Journal of Medicine, were striking. Four out of the 10 colon cancer patients with the defect and 5 out of the other 7 cancer patients with the abnormality responded to the drug. In the remaining group, nothing. Since then, updated numbers have reinforced that a high proportion of patients with the genetic feature benefit from the drug, often for a lengthy period. Other trials by pharmaceutical companies have shown similar results.

The Hopkins investigators found that tumors with the defect had, on average, 1,700 mutations, compared with only 70 for tumors without the problem. That confirmed the theory that high numbers of mutations make it more likely the immune system will recognize and attack cancer if it gets assistance from immunotherapy.

For Joho, now 27 and living in suburban Philadelphia, the hard lesson from the past few years is clear: The cancer field is changing so rapidly that patients can't rely on their doctors to find them the best treatments.

"Oncologists can barely keep up," she said. "My sister found a trial I was a perfect candidate for, and my doctors didn't even know it existed."

Her first several weeks on the trial were rough, and she still has some lasting side effects today joint pain in her knees, minor nausea and fatigue.

"I have had to adapt to some new limits," she acknowledged. "But I still feel better than I have in five years."

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IT'S A START: Newly approved gene therapy may help 4 percent of cancer patients - Sarasota Herald-Tribune

In Brief Researchers have successfully used immunotherapy to "turn off" asthma and allergic responses in animals. This work will eventually be used to create one-shot treatments that permanently silence allergies. Erasing Asthma

Scientists from the University of Queensland have used gene therapy to turn off the immune response responsible for asthma. The team believes their technique may also be able to permanently silence severe allergy responses to common allergens such as bee venom, peanuts, and shellfish. Thus far, the research has been successful in animal trials, and if it can be replicated in human trials, it may provide a one-time treatment for asthma and allergy patients.

The technique erases the memory of the cells which cause allergic reactions using genetically modified stem cells that are resistant to allergens. We have now been able wipe the memory of these T-cells in animals with gene therapy, de-sensitizing the immune system so that it tolerates the [allergen] protein, lead researcher Ray Steptoe said in a press release. We take blood stem cells, insert a gene which regulates the allergen protein and we put that into the recipient. Those engineered cells produce new blood cells programmed to express the protein and target specific immune cells, which turn off the allergic response.

According to the Centers for Disease Control (CDC), about 1 in 12 people (25 million) in the U.S.have asthma, and these numbers are increasing annually. As of 2007, the last year for which the CDC has data, asthma cost the U.S. approximately $56 billion in costs for medical bills, lost work and school days, and early deaths. According to the World Health Organization (WHO), 235 million people worldwide have asthma, which is the most common chronic childhood disease, occurring in all countries regardless of level of development.

The researchers findings must now besubjected to further pre-clinical investigation, with the aim of replicating the results in the laboratory using human cells. The longer term goal will be a one-time gene therapy injection that would replace short-term allergy treatments, which vary in their effectiveness. We havent quite got it to the point where its as simple as getting a flu jab so we are working on making it simpler and safer so it could be used across a wide cross-section of affected individuals, Dr. Steptoe said in the press release.

More:

A New Gene Therapy Could Hold the Key to Curing Allergies and Asthma - Futurism

In a very competitive bidding process Sangamo Therapeutics (SGMO) has inked a significant global partnership with Pfizer (PFE), who brings a wealth of value to SGMO, and notably validates the latter companys gene therapy platform, explains biotech expert John McCamant, editor The Medical Technology Stock Letter.

After repeated delays at SGMO under the previous management, the new senior team is executing at a very high level with 4 clinical trials either under way and/or about to start for the first time in company history.

With the consensus often suggests SGMOs proprietary technology has been made obsolete with the emergence of CRISPR, this Pfizer collaboration totally refutes that notion and reminds investors that SGMO remains a gene therapy/gene editing leader, with the largest patent portfolio in the field.

In our view, this could be just the beginning for SGMO as we see further deals of this size or even larger.

PFE will provide $70 million in upfront cash. The deal is broadly based for hemophilia A and provides up to another $300 million in development funds for the lead compound SB-525 and another $175 million for related and follow-up compounds.

Lastly, SGMO will receive tiered double-digit royalties on net sales. Additionally, the company will be collaborating with Pfizer on manufacturing and technical operations utilizing viral delivery vectors.

Importantly, the joint venture also gives SMGO access to the Pfizer global hemophilia infrastructure, one that already has other hemophilia compounds under development.

Gene editing, along with cell and gene therapies are basically the most advanced technologies targeting a growing and competitive segment for many serious diseases that are otherwise not treatable/druggable with conventional pharmaceuticals.

SMGO has several first and next-generation platform technologies and they are still optimizing their development programs which will include both adults and children.

The new management of SGMO have really turned this company around, progressing in the lab, the clinic and this large collaboration with Pfizer validates this progress in one of the most competitive, blockbuster markets in the drug industry today.

With four clinical trials either underway and/or starting over the next twelve months, investors will hear a steady stream of clinical updates and potentially a major partnership for Alzheimers disease from SGMO .

Before today, the stock was trading at just a $312 million valuation likely based on the years of stops and starts, the previous managements reputation and the competitive noise around other technologies/companies.

With the recent slew of important, fundamental announcements positive FDA designations, encouraging Alzheimers data and now the big PFE deal validating the platform in our view, the 40% after-market run-up in SGMO shares is only the beginning. SGMO is a buy under $8 with a target price of $16.

Subscribe to John McCamant's The Medical Technology Stock Letter here

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Sangamo: Making Gains in Gene Therapy - Moneyshow.com (registration)

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Gene Therapy Has Been Used to 'Switch Off' Asthma Symptoms - ScienceAlert

The team hopes to develop a single, injected, gene therapy treatment that could eliminate many severe allergic responses (Credit: University of Queensland)

Scientists may be one step closer to discovering a way to genetically "turn off" allergic responses with a single injection. A team of researchers at the University of Queensland has developed a new process that has successfully silenced a severe allergic response in mice, using blood stem cells engineered with a gene that can target specific immune cells.

The big challenge previous allergy researchers faced was that immune cells, known as T-cells, tended to develop a form of "memory" so that once someone developed an immune response to an allergen, it would easily recur upon future contact. The key was finding a way to erase that "memory" response to the protein in the allergen causing the immune reaction.

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"We take blood stem cells, insert a gene which regulates the allergen protein and we put that into the recipient," says Professor Ray Steptoe, explaining the new process developed by his team at The University of Queensland. "Those engineered cells produce new blood cells that express the protein and target specific immune cells, 'turning off' the allergic response."

The team's initial clinical investigations looked at an experimental asthma allergen, with the new process found to successfully terminate established allergic responses in sensitized laboratory mice. While the initial research has focused on a very specific asthma allergen, Professor Steptoe believes the process could be applied to many other severe allergic responses, such as peanuts, bee venom and shell fish.

The long-term goal of the research would be to develop a therapy that could cure specific allergies with a single injection, much like a vaccine.

"We haven't quite got it to the point where it's as simple as getting a flu jab," says Professor Steptoe, "so we are working on making it simpler and safer so it could be used across a wide cross-section of affected individuals."

The team is realistic about the time it will take before this discovery results in practical benefits for allergy sufferers, with at least five years more laboratory work needed before even human trials can be conducted. But this new discovery could mean that, within 10 or 15 years, asthma and other lethal allergic responses might be eliminated with a single, one-time treatment.

The findings were recent published in the journal JCI Insight.

Watch Professor Ray Steptoe from The University of Queensland discuss his team's findings in the video below.

Source: The University of Queensland

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Gene therapy could wipe immune memory and "turn off" severe allergies - New Atlas

Xconomy Texas

San Antonio Most diabetes treatments work by giving the body the insulin it needs to break down sugar. But that approach deals with the symptoms of diabetes. In recent years, scientists and companies have taken aim at the root cause of the condition by attempting to stimulate or replace the cells in the pancreas responsible for producing insulin in the first place. One of them is a San Antonio researcher hoping to use gene therapya potentially one-time, long lasting treatmentto do the trick.

When cells in the pancreas, known as beta cells, either get destroyed by the immune system or stop producing enough insulin, the result is type 1 or type 2 diabetes. Companies large and small-from European diabetes drug giant Novo Nordisk to privately held startups ViaCyte, of San Diego, and Semma Therapeutics, of Cambridge, MAwant to engineer stem cells that develop into pancreatic beta cells to help a patient produce insulin.

Other researchers, such as Bruno Doiron, a scientist and assistant professor at the University of Texas Health Science Center at San Antonio, have different ideas. Doiron has developed an injectible treatment consisting of three molecules glucokinase, a second that targets a protein known as PTP1B, and a third that targets a protein called Pdx-1, a so-called transcription factor that regulates genesthat, when infused into the body, are meant to help stimulate the formation of new beta cells. Doiron has tried the method on mice, and based on some encouraging early results, intends to move the work forward through a startup company.

You have to prove you can translate that to a large animal model, he says.

The San Antonio company, Syner-III, got its name because of the synergistic use of three molecules to generate the beta cells, he says. Those molecules are administered via a gene therapy procedure: theyre stuffed into a modified virus and injected directly into the pancreas in a one-time treatment, where they are meant to stimulate beta cell production. The work was published in the peer-reviewed journal Current Pharmaceutical Biotechnology in 2016.

Doiron hopes to raise as much as $10 million to complete preclinical testing.

Others, including Novartis, are considering different ways of boosting beta cell production. Researchers from the Swiss company published findings in Nature Communications that showed a group of compounds called aminopyrazines could be packed into a pill and similarly lead to more beta cells, and more insulin, in mice. Such attempts are fraught with failure, however. In an article on its own website, Novartis notes that researchers have succeeded in producing beta cells in mice many times, but havent been able to reproduce those results in humans.

The potential payoff, however, is huge. Some 29.1 million Americans have diabetes, and 1.25 million of them have type 1 diabetes, according to the American Diabetes Association. Doiron believes the therapy may be able to help both types. While stem cell research has had its share of failures and competition continues to increase in insulin therapysuch as pumps that automatically deliver the treatmentDoiron says a gene therapy, if successful, could result in a longer-lasting, more effective treatment.

When I use your own body to produce medicine, that drastically changes the field, he says.

David Holley is Xconomy's national correspondent based in Austin, TX. You can reach him at dholley@xconomy.com

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With Gene Therapy for Diabetes, San Antonio Researcher Eyes Funding - Xconomy

The basic physical and functional unit of heredity is the genes. It is a specific sequence of sources that programs instructions on how to produce proteins. Although genes gets a lot interest because of lack of open understanding of its nature, it is the protein that is the main element that perform most of life functions and even consists of the majority of the cellular structures. When the genes are changed, the programmed or encoded proteins become unable to accomplish the usual function expected from it and genetic disorders arise.

Gene therapy is the replacement of genetic material to an impaired cell. It then corrects the flaw in the genome of a patient. Gene therapy can also be applied to a developed lifetime disease of an individual such as cancer or recurring infection. It gives a specific trait or attribute to the cell giving it strength to combat the disease.

Generally, gene therapy is normally used in acute diseases when the cells of a particular organ in the body cannot function the normal way because it does not have sufficient required protein to perform a specific bodily task. In order to replace the faulty protein, a gene transfer vector or a gene transfer agent is altered so that it contains the gene that encodes for this protein. The altered vector is then administered to the patient. The gene transfer vector delivers the altered gene to the cell, which in turn, the cells mechanism converts the healing gene to correct the problem. In essence, it fixes the malfunctioning cell.

To summarize, gene therapy is a procedure for correcting defective genes that are responsible for the development of a disease. There are several approaches that maybe used in correcting faulty genes:

* The most common is the insertion of a normal gene into a non-specific location inside the genome to replace the non-functional gene.

* An abnormal gene is being substituted by a normal gene using homologous recombination or DNA crossover.

* The abnormal gene is repaired by undoing the genetic damage that happened long time ago. It is also called selective reverse mutation, which returns the gene to its normal function.

The gene transfer vector or a gene transfer agent, which is the carrier to deliver the therapeutic gene to the patients target cells is basically a virus that has been altered genetically to carry a normal human DNA. Many scientists tried to take advantage of this discovery and manipulate the virus genome to aid in delivering the healing genes and remove the disease causing ones.

Some of the few different types of viruses that are used as gene therapy vectors are retroviruses, adenoviruses, adeno-associated viruses and herpes simplex viruses.

There are also factors that have kept gene therapy from becoming the perfect treatment for genetic diseases. Some of the discovered factors are:

* Short life of gene therapy

* Immune response

* Risk of viral vectors that once inside the patients body, it may recover its ability to cause disease.

* Multigene disorders

Human gene therapy has triggered many issues since it was known. The promise of the technology is very great but the reality of it is somehow overwhelming. Human gene therapy must be seriously and cautiously evaluated.

It will be, as technology evolves.

Dave Kotecki is an innovative businessman who has a passion for new advancements in medical technology. His principle if you cant sell, youll fail is one of the benchmarks of his ability to practice what he learned in the internet industry. To learn how you can profit from the discovery of customized nutritional products, click here .

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How Helpful is Gene Therapy? - Good Herald

May 30, 2017 08:05 ET | Source: Abeona Therapeutics Inc

NEW YORK and CLEVELAND, May 30, 2017 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (Nasdaq:ABEO), a leading clinical-stage biopharmaceutical company focused on developing novel gene therapies for life-threatening rare diseases, announced today that the FDA has granted Rare Pediatric Disease Designation for Abeonas EB-101 gene therapy program for patients with dystrophic epidermolysis bullosa (DEB), including recessive dystrophic epidermolysis bullosa (RDEB), which are life-threatening genetic skin disorders characterized by skin blisters and erosions that cover the body.

These designations are granted to drugs with high promise that may address areas of unmet medical need for children with rare diseases. RDEB is a debilitating and life threatening inherited disorder with no approved treatment options available for patients today," stated Timothy J. Miller, Ph.D., President & CEO of Abeona Therapeutics Inc. Building upon the already granted FDA and EMA Orphan Drug Disease Designations for the EB-101 gene therapy program, receiving the Rare Pediatric Disease Designation is another important validation of the science and clinical approach to developing a novel gene therapy for RDEB patients.

Typically, wounds on patients with RDEB, also known as "butterfly skin" syndrome, can remain unhealed for months to years due to the inability of the skin to stay attached to the underlying dermis and can cover a large percentage of the body. In the ongoing Phase 1/2 clinical trial, EB-101 was administered to non-healing chronic wounds on each subject and assessed for wound healing at predefined time points over years. The primary endpoints of the clinical trial assess safety and evaluate wound healing after EB-101 administration compared to control untreated wounds. Secondary endpoints include expression of collagen C7 and restoration of anchoring fibrils at three and six months post-administration.

About Rare Pediatric Disease Designation: The rare pediatric disease designation indicates that the FDA may give the company a pediatric priority review voucher if the drug is approved for the pediatric indication. That voucher could then be used by the company for another drugany drugto be given a priority review. A priority review mandates that the FDA will review a BLA drug submission within six months instead of the standard 10 months. Normally, a priority review designation would only be given to a drug that is for a serious condition and has demonstrated the potential to be a significant improvement in safety and effectiveness. The priority review voucher may be used by the sponsor, sold or transferred.

EB-101 Gene Therapy Program Highlights:

About EB-101: EB-101 is an autologous, ex-vivo gene therapy in which COL7A1 is transduced into autologous keratinocytes for the treatment of Recessive Dystrophic Epidermolysis Bullosa (RDEB). RDEB is a subtype of an inherited genetic skin disorder characterized by chronic skin blistering, open and painful wounds, joint contractures, esophageal strictures, pseudosyndactyly, corneal abrasions and a shortened life span. Patients with RDEB lack functional type VII collagen owing to mutations in the gene COL7A1 that encodes for C7 and is the main component of anchoring fibrils, which stabilize the dermal-epidermal basement membrane. Patients are being enrolled in the ongoing Phase 2 portion of the Phase 1/2 clinical trial (NCT01263379). The EB-101 program has also been granted orphan drug designation by the FDA and European Medicines Agency (EMA).

About Epidermolysis Bullosa (EB): EB is a group of devastating, life-threatening genetic skin disorders that is characterized by skin blisters and erosions all over the body. The most severe form, recessive dystrophic epidermolysis bullosa (RDEB), is characterized by chronic skin blistering, open and painful wounds, joint contractures, esophageal strictures, pseudosyndactyly, corneal abrasions and a shortened life span. Patients with RDEB lack functional type VII collagen (C7) owing to mutations in the gene COL7A1 that encodes for C7 and is the main component of anchoring fibrils that attach the dermis to the epidermis. EB patients suffer through intense pain throughout their lives, with no effective treatments available to reduce the severity of their symptoms. Along with the life-threatening infectious complications associated with this disorder, many individuals often develop an aggressive form of squamous cell carcinoma (SCC).

About Abeona: Abeona Therapeutics Inc. is a clinical-stage biopharmaceutical company developing gene therapies for life-threatening rare genetic diseases. Abeona's lead programs include ABO-102 (AAV-SGSH), an adeno-associated virus (AAV) based gene therapy for Sanfilippo syndrome type A (MPS IIIA) and EB-101 (gene-corrected skin grafts) for recessive dystrophic epidermolysis bullosa (RDEB). Abeona is also developing ABO-101 (AAV-NAGLU) for Sanfilippo syndrome type B (MPS IIIB), ABO-201 (AAV-CLN3) gene therapy for juvenile Batten disease (JNCL), ABO-202 (AAV-CLN1) for treatment of infantile Batten disease (INCL), EB-201 for epidermolysis bullosa (EB), ABO-301 (AAV-FANCC) for Fanconi anemia (FA) disorder and ABO-302 using a novel CRISPR/Cas9-based gene editing approach to gene therapy for rare blood diseases. In addition, Abeona has a plasma-based protein therapy pipeline, including SDF Alpha (alpha-1 protease inhibitor) for inherited COPD, using its proprietary SDF (Salt Diafiltration) ethanol-free process. For more information, visit http://www.abeonatherapeutics.com.

Investor Contact: Christine Silverstein Vice President, Investor Relations Abeona Therapeutics Inc. +1 (212)-786-6212 csilverstein@abeonatherapeutics.com

Media Contact: Andrea Lucca Vice President, Communications & Operations Abeona Therapeutics Inc. +1 (212)-786-6208 alucca@abeonatherapeutics.com

This press release contains certain statements that are forward-looking within the meaning of Section 27a of the Securities Act of 1933, as amended, the expected receipt of a Priority Review Voucher and that involve risks and uncertainties. These statements include, without limitation, our plans for continued development and internationalization of our clinical programs, that patients will continue to be identified, enrolled, treated and monitored in the EB-101 clinical trial, and that studies will continue to indicate that EB-101 is well-tolerated and may offer significant improvements in wound healing. These statements are subject to numerous risks and uncertainties, including but not limited to continued interest in our rare disease portfolio, our ability to enroll patients in clinical trials, the impact of competition; the ability to develop our products and technologies; the ability to achieve or obtain necessary regulatory approvals; the ability to secure licenses for any technology that may be necessary to commercialize our products; the impact of changes in the financial markets and global economic conditions; and other risks as may be detailed from time to time in the Company's Annual Reports on Form 10-K and other reports filed by the Company with the Securities and Exchange Commission. The Company undertakes no obligations to make any revisions to the forward-looking statements contained in this release or to update them to reflect events or circumstances occurring after the date of this release, whether as a result of new information, future developments or otherwise.

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Abeona Therapeutics Receives Rare Pediatric Disease Designation ... - GlobeNewswire (press release)

Spark Therapeutics has posted fresh data from a phase 1/2 trial of its hemophilia B gene therapy, SPK-9001. The data suggest Sparks tapering courses of steroids successfully arrested the factor IX (FIX) declines seen in two patients who appeared to have an immune reaction to the Spark100 vector capsid.

Philadelphia-based Spark first reported on the two patients in December. At that time, the fall in FIX activityfrom 71% to 68% and, more dramatically, 32% to 12%raised concerns about the safety of the treatment. Spark downplayed these concerns, noting that the patient who received steroids more promptly only experienced a 3% decline in FIX activity. The latest set of data backed up Sparks confidence.

Both patients have now completed their tapering courses of steroids. After completing the courses of steroids, both patients saw their alanine aminotransferase levels return to baseline. The FIX activity of the subject whose level fell to 12% has since stabilized. Spark said the patient has been at around 15% over the nine weeks since finishing the course of steroids, a level likely to result in improved clinical outcomes. The other patients FIX activity is back up above 70%.

Our analysis suggests that a tapering course of oral corticosteroids has been well-tolerated and may help control potential capsid immune responses following SPK-9001 infusion, Spark CSO Katherine High, M.D., said in a statement.

Those data could lessen concerns that immune responses will scuttle the gene therapy, which has consistently delivered higher FIX activity levels than its main rival, uniQures AMT-060. UniQure sees safety as an area in which it may have an edge over Spark, although questionmarks hang over the elevated liver enzymes seen in some patients in the study.

Other aspects of the Spark data drop are free from red flags. Mean steady-stage FIX activity in the 10 participants who are 12 weeks or more post treatment stood at 33% at the last count. That is up on the 28% Spark reported in its December update. And with the range of responses spanning from 14% to 81%, it is also well clear of the 3% to 12.7% uniQure recorded in its high-dose cohort in December.

UniQure has argued FIX activity levels are less important than clinical benefits, such as cessation of bleeding and reduction in infusions. Sparks latest data show SPK-001 continuing to perform well against these measures. At the last count, the annualized bleeding rate was down 96%. The fall in the annualized infusion rate was 99%.

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Spark hemophilia B gene therapy clears another early test - FierceBiotech

Xconomy San Diego

Christopher Reinhard will tell you there is nothing unusual about the 20-plus years hes spent getting an experimental heart therapy to late-stage clinical trials.

Very rarely will you get a short story on development of a drug, said Reinhard (above), who is a principal investor and the CEO of San Diego-based Angionetics. Two decades is about what you would expect for a new drug-making method, Reinhard said.

That may be true, but it doesnt begin to convey the tortuous path that Reinhard has followed to get Angionetics where it is today. The biotech is starting a phase 3 trial in the next few months that seeks to enroll some 320 patients with myocardial ischemiawhen clogged coronary arteries reduce the flow of oxygen-rich blood to the heart.

To treat the disease, Angionetics isnt testing some new type of cholesterol-lowering drug, or a stent to help open clogged arteries.

Rather, its attempting a risky and less-proven methodgene therapy, in which new genetic instructions are transported into the body to help produce a specific protein. Gene therapies have been in development for decades, but are only now starting to come of age thanks to a variety of technological advances. Two therapies are approved in Europe, from UniQure (NASDAQ: QURE) and GlaxoSmithKline, both for ultra rare immune and metabolic diseases. Spark Therapeutics (NASDAQ: ONCE) this year is expected to file the first ever U.S. approval application for a gene therapy, a treatment for a form of childhood blindness.

Angionetics gene therapy, Ad5FGF-4 (Generx), is intended to stimulate the growth of new blood vessels in the heart. A catheter inserted through the groin delivers the genescarried within modified virusesinto heart cells, where they are supposed to produce a protein, fibroblast growth factor-4, that helps grow new blood vessels.

The hope is to ease chest pain and relieve the effects of clogged coronary arteries by stimulating the growth of new blood vessels in areas in the heart where there is insufficient blood flow. Were just taking the heart and trying to enhance its ability to grow more blood vessels, Reinhard said.

Angionetics image highlights growth of collateral blood vessels (Image by Bryan Christie Design, used with permission)

While gene therapies are more advanced than ever, and several experimental treatments aimed at heart disease and heart failure are being tested, none have yet succeeded. A heart failure gene therapy from San Diegos Celladon, for instance, failed in 2015.

Still, the potential prize is substantial. Of the estimated 16.5 million Americans with coronary heart disease, Angionetics Reinhard said about half experience heart-related chest pain.

The current standard of care offers two principal methods of treatment. The first course of therapy is usually to prescribe drugs like nitrates that temporarily dilates blood vessels to Next Page

Bruce V. Bigelow is the editor of Xconomy San Diego. You can e-mail him at bbigelow@xconomy.com or call (619) 669-8788

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Angionetics Nears Key Gene Therapy Trial for Coronary Heart Disease - Xconomy

New step toward the treatment of myotubular myopathy gene ... Science Daily A team of researchers has demonstrated the efficacy of administration of a therapeutic vector by a single intravenous injection and identified the dose that ... and more »

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New step toward the treatment of myotubular myopathy gene ... - Science Daily

Tocagen has filed to raise up to $86 million in an IPO to take its breakthrough-designated brain cancer gene therapy combination through the first part of a phase 2/3 trial. The San Diego, California-based biotech expects to deliver data from the phase 2 in the first half of next year, having wrapped up enrollment last month.

That trial is assessing the combination of gene therapy Toca 511 and prodrug Toca FC in patients with first or second recurrence of glioblastoma or anaplastic astrocytoma who are undergoing resection.

Subjects receive either standard of care or the Toca 511-Toca FC combination. Toca 511 is a retroviral replicating vector that encodes cytosine deaminase (CD). Its administration is intended to equip cancer cells to produce CD, a prodrug activator enzyme. Tocagen then gives patients the prodrug, Toca FC, an extended-release formulation of approved antifungal agent 5-fluorocytosine that is inactive until exposed to CD. Tocagen hopes Toca FC will cross the blood-brain barrier, be activated by CD and then kill both cancer cells and immunosuppressive cells.

Among the 24 patients who received the higher Toca 511 doses in a phase 1 and met the inclusion criteria for the phase 2/3, Toca saw three complete responses and two partial responses. At the time of the last data update, the responders were still alive 24 to 43 months after entering the study. The median overall survival for the 24 patients is 14.3 months.

The question facing Tocagen is whether that will translate into improved overall survival when the experimental regimen is pitted against standard of care options including Mercks Temodal and Roches Avastin. Tocagen is hoping Wall Street will provide the money it needs to start to answer the question.

Tocagen is yet to set the terms for its IPO, but listed $86 million as its proposed maximum offering. A chunk of the anticipated haul is earmarked for manufacturing scale-up and validation of Toca 511 and Toca FC. Tocagen plans to set aside another tranche to complete the ongoing phase 2, leaving some cash left over to wrap up a phase 1b of the combination in other indications, including newly-diagnosed brain cancer and a clutch of other solid tumors.

If Tocagen hits its fundraising goal, the IPO cash will see it through at least the next 12 months, taking it up to the delivery of phase 2 data.

Tocagen has financed its progress to date through a series of low-profile venture roundsmost recently a $28.8 million investment in 2015and by seeking donations via a brain cancer nonprofit.

Management must now persuade public investors to part with their cash. This year, Braeburn Pharmaceuticals and Visterra have both pulled IPOs after getting a frosty reception on Wall Street. Another company, ObsEva, hit its range but subsequently saw its stock slide. Tocagen will be hoping its experience has more in common with Jounce Therapeutics, which raised $102 million before seeing its stock go on a 30% tear in its first months on the market.

Jounce benefited from the starpower of a $2.6 billion pact with Celgene and a pitch that positions it at the forefront of the second wave of immuno-oncology. Tocagen lacks attributes with such pulling power, but in co-founder and R&D chief Harry Gruber, M.D. it has a name that could turn the heads of investors.

Gruber is gene therapy specialist who has played a role in getting a handful of biotechs started over the past 30 years, including Gensia and Viagene. Gensia ultimately became part of Teva through a $3.4 billion buyout, while Viagene accepted a $95 million bid from Chironnow part of Novartisafter a bumpy few years trying to develop gene therapies in the early 1990s.

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Tocagen seeks $86M IPO to fund cancer gene therapy trial ... - FierceBiotech

by Stuart Tomlinson, KATU News

Even before her first birthday, Kristin Kelly Bretanas parents noticed a fluttering in her eyes. As she grew, Bretana said she was accident-prone and had trouble seeing colors.

I have difficulty seeing far away, and I have difficulty with a lot of light saturation and Im almost completely color blind, Bretena said. And I say almost because there are certain situations where I can see a color if the saturations high enough.

Eventually her doctors gave her the news: She had achromatopsia, a rare genetic disease that results when you have a mutation in a gene that codes for a protein thats very important for the function of the cones.

To see, our retinas use rods and cones. Rods we use at nighttime and in dim light, cones are what we use during the daytime and for our color vision, says Dr. Mark Pennesi, OHSU Associate Professor of Ophthalmology whos now treating Bretana for the disease with gene therapy.

With achromatopsia the rods are actually ok, but the cones have no function at all," Pennesi said. "These patients have severe vision loss, theyre legally blind and they have no color vision at all. When they go outside they can barely see. They have to wear very dark sunglasses, but in dim light they actually see very well.

Pennesi said by taking a modified virus and stripping out all the things that make you sick, doctors then inject a normal copy of the missing or diseased gene back into the eye. The modified gene can then perhaps either stop the progression of the disease, or even restore function.

We actually go inside of the eye with a very, very fine needle and create a little pocket of fluid underneath the retina and inject that modified virus, Pennesi said. The virus then attaches to the retinal cells and inserts that DNA into the cells so they can start making the protein again.

Bretana said she has come to accept her limitations.

Its something I am not allowed to forget whether it be crossing the street and realizing that I am putting my life into a persons hands who is driving down the road because I cannot see inside the cab of a car, she said. It doesnt bother me nearly as much as it used to when I was a child and struggling with issues like self-identity and self-efficacy and what-not.

Pennesi said the therapy can be used for other diseases of the eye. OHSU currently has 7 gene therapy studies underway.

Its actually a very exciting time because many of these patients for decades have been told theres nothing that can be done, Pennesi said.

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OHSU gene therapy studies offer hope - and vision - to patients with eye disease - KATU

Pfizer has confirmed it assessing potential sites in North Carolina for a gene therapy production plant.

Reports in the US press suggested Pfizer is considering either expanding its existing facility in Sanford, North Carolina or building the plant at a site nearby.

Kim Bencker, head of communications at Pfizer Global supply, told us in an emailed statement We recently announced that were moving forward with scoping potential sites in Sanford for our new gene therapy site.

She added: This work is still in the preliminary stages and we arent able to share additional detail at this time.

The move follows a little over a year after the US drug manufacturer acquired Bamboo, a North Carolina-based gene therapy developer.

The deal included a recombinant Adeno-Associated Virus (rAAV) vector design and production technology, a Phase I candidate for Giant Axonal Neuropathy and a preclinical programme targeting Duchenne Muscular Dystrophy (DMD).

Pfizer also gained a 11,000sq ft gene therapy manufacturing facility in Chapel Hill that Bamboo bought from the University of North Carolina in 2016.

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Pfizer mulls sites for NC gene therapy plant year after Bamboo buy - BioPharma-Reporter.com

A number of recent headlines imply a recent case study just publishedproves that gene therapy has cured sickle cell diseasea genetic disorder that incurs tremendous pain, suffering and diminished life expectancy.

Due to such limited progress in management of this condition, this team of researcherstook samples from the bone marrow of a patient with severe diseaseUsing a lentiviral vector, they transferred an anti-sickling gene into the patients stem cellswhich get put back into the patient in the hope they will multiply and replace the cells made with the defective gene.

[T]he team concludes their patient had complete clinical remission with correction of hemolysis and biologic hallmarks of the disease. Furthermore, after fifteen months, the antisickling protein remained high at approximately 50% and the patient had no crises or hospitalizations. Before, the patient required regular transfusions.

ACSHs Senior Fellow in Molecular Biology, Dr. Julianna LeMieux, puts the promise of gene therapy into even greater context for this and other disease entities:This is an incredibly promising result, even with the obvious caveat that it is only one person. Sickle Cell is a disease that is ripe for genetic advances[T]his one success story is incredibly encouraging for the sickle cell community and for moving the field of curing diseases using genetic editing forward.

[The study can be found here.]

The GLP aggregated and excerpted this blog/article to reflect the diversity of news, opinion, and analysis. Read full, original post:Did Gene Therapy Cure Sickle Cell Disease?

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Sickle cell cure? Patient in complete remission following gene therapy - Genetic Literacy Project

JaCeon Golden has only ever known the inside of hospitals. But the treatment hes receiving may have implications far beyond his as-yet isolated life.

Round-faced and big-eyed, with a perpetual pout that belies his sunny nature, he looks as healthy as any other 5-month-old. But JaCeon was born without a functioning immune system. Even the most banal of infections a cold, a diaper rash could be deadly.

Earlier this year, JaCeon became the first baby at UCSF Benioff Childrens Hospital at Mission Bay to undergo an experimental gene therapy treatment that, doctors hope, will nudge his body to build a new, robust immune system.

From right: Dannie Hawkins checks on her nephew Ja'Ceon Golden, who is being held by patient care assistant Grace Deng at UCSF Benioff Children's Hospital on Wednesday, March 8, 2017, in San Francisco, Calif. Golden, who is five months old, is diagnosed with severe combined immunodeficiency disease (SCID). He is a patient at UCSF, where he stays in a sterile room. The hospital is working on a new gene therapy treatment for SCID. Hawkins brought her nephew Golden from New Mexico for the experimental treatment.

From right: Dannie Hawkins checks on her nephew Ja'Ceon Golden, who...

So far, his results are promising. In a few weeks, JaCeons great aunt, whos also his guardian, hopes to introduce him to the world outside.

Am I going to see him smile when we walk out of here? Dannie Hawkins, 52, said with a glance at the baby, being fed from a bottle by a nurse wearing a gown and gloves. Hows he going to do in the free world?

It will be a while months, probably years before JaCeon is able to fully integrate with that wide world: go to school and birthday parties, ride a public bus, swim in a community pool. But that those activities may be in his future at all is extraordinary.

The treatment given to JaCeon is the result of decades of research into gene therapy that included a string of striking failures that led many doctors to abandon the pursuit altogether.

Gene therapy long had been considered a potential treatment for severe combined immunodeficiency disorder, or SCID, the condition JaCeon was born with, and some other genetic syndromes. The idea is to replace a single gene thats causing trouble.

Even as many doctors gave up on the promise of gene therapy, teams of stubborn scientists kept plugging away. And a few years ago, their experiments started to work, propelled by advances in the understanding of stem cells in this case, a type called hematopoietic stem cells that live in bone marrow and are responsible for generating blood and immune cells and improved methods of delivering genetic repairs.

JaCeon Golden is treated by patient care assistant Grace Deng (center) and pediatric oncology nurse Kat Wienskowski.

JaCeon Golden is treated by patient care assistant Grace Deng...

Now human gene therapy is being tested in trials at UCLA, where a team has treated 20 children with one type of SCID, and at UCSF in collaboration with St. Jude Childrens Research Hospital in Memphis. Both trials are funded by grants from the California Institute for Regenerative Medicine, the states stem cell agency, located in Oakland.

Researchers are studying similar therapies in hopes of curing genetic syndromes like sickle cell disease. And the stem cell agency is funding gene therapy research into potential treatments for HIV, brain cancer and Huntingtons disease, among others.

Gene therapy has been shown to work, the efficacy has been shown. And its safe, said Sohel Talib, a senior science officer at the state stem cell agency. The confidence has come. Now we have to follow it up.

JaCeon was born at a hospital in Las Cruces, N.M., and diagnosed with SCID just after birth as part of a standard newborn screening. He was flown to UCSF, one of a handful of facilities with expertise in SCID, when he was 3 weeks old. His great-aunt joined him about a month later, in November.

The immune disorder is commonly known as bubble baby disease, because until fairly recently kids born with it had to live in isolation, often in plastic bubbles in hospital rooms or their own homes to protect them from infections.

Babies born with SCID have a genetic mutation that leaves their immune system unable to develop disease-fighting cells. Without treatment, most will die within a year. Since the 1970s, some babies with SCID were cured with a bone-marrow transplant. But to be effective, a perfect match was required, almost always from a sibling, and only about a fifth of kids have such a match.

Ja'Ceon Golden is held by patient care assistant Grace Deng, as Deng bottle feeds Golden at UCSF Benioff Children's Hospital on Wednesday, March 8, 2017, in San Francisco, Calif. Golden, who is five months old, is diagnosed with severe combined immunodeficiency disease (SCID). He is a patient at UCSF, where he stays in a sterile room. The hospital is working on a new gene therapy treatment for SCID. Golden was brought from New Mexico for the experimental treatment.

Ja'Ceon Golden is held by patient care assistant Grace Deng, as...

The rest could undergo a bone marrow transplant from a partial match in JaCeons case, his great-aunt was one but even when that treatment was successful, kids were left with fragile immune systems that required constant maintenance with antibiotics and other boosts.

Gene therapy, though, may prove as effective as a bone marrow transplant from a perfect match.

The procedure starts with doctors harvesting stem cells from a babys own bone marrow, usually taken from the hip. In JaCeons case, his stem cells were sent in January to St. Jude in Memphis, where scientists are perfecting the gene-therapy delivery mechanism.

Sending away JaCeons stem cells was probably the most stressful time of my life, short of my own kids maybe being born, said Dr. Morton Cowan, the lead investigator of the UCSF trial, who has worked in SCID research for more than 30 years.

JaCeons stem cells were flown east over the first big weekend of major storms in California. Flights were being canceled around the clock, and doctors only had a window of about 36 hours to get the fresh cells to the labs in Memphis.

The trip was successful, but not without a hitch. After the cells were engineered and were being sent back to California, the material for a few heart-stopping hours got lost in the mail.

In a couple of months, Cowan said, he hopes to be able to do the gene-therapy delivery at UCSF labs, avoiding the travel headaches.

For now, that still happens at St. Jude. Doctors used a virus in fact, HIV, the virus that causes AIDS to deliver the gene therapy to JaCeons stem cells. The virus is neutered, with all of the disease-causing pieces inside removed.

Whats left is a missile-like shell designed to infiltrate a cell and deliver whatever payload doctors have inserted inside in this case, a healthy gene that will restore the stem cells ability to build normal immune cells.

Back in San Francisco, the cells were infused into JaCeon via a port in his chest. Because theyre his own cells, there was no fear his body would reject them.

He did have to undergo mild chemotherapy to kill off some of his own bone marrow and make room for the re-engineered stem cells to roost, but UCSF has been developing a technique for limiting the dosage of chemotherapy given in gene therapy procedures.

JaCeon suffered no obvious side effects from either the stem cell infusion or the chemotherapy drugs, doctors said.

Hes just thriving. Hes just hes great, Cowan said. He added, We cant open the Champagne just yet, but early tests show the new gene is active, and JaCeon has had an uptick of certain immune cells.

The infusion procedure took just 20 minutes, and JaCeon slept through it, but it felt momentous nonetheless.

It had been difficult to decide to enroll JaCeon in the trial, Hawkins said. Since she was a partial match for a bone marrow transplant, she had the option of giving him the traditional and well-tested therapy.

Shed said to his doctors, So youre telling me hes a guinea pig? They told her, she recalls, If it works, he can open the door for other kids.

That night, as Hawkins slept on the decision, I kept waking up, waking up, all night long, she said. If there was a possibility he could save someone else ... she added, and then broke off in tears.

She spends about six hours with JaCeon every day, beginning each morning with a bath in sterile water, brought by nurses in special tubs. Shes constantly wiping down his toys, clothes, bedding and stuffed animals.

Ive changed a lot of diapers in my time, but this is way more complicated than with other kids, Hawkins said, demonstrating the multistep process she uses to prevent diaper rash.

Im not going to say its been easy, she said. But hes doing fine. I wouldnt have it any other way.

Erin Allday is a San Francisco Chronicle staff writer. Email: eallday@sfchronicle.com

Twitter: @erinallday

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Amid advances in gene therapy, 'bubble baby' in SF gains hope - San Francisco Chronicle

Pfizer has confirmed it assessing potential sites in North Carolina for a gene therapy production plant.

Reports in the US press suggested Pfizer is considering either expanding its existing facility in Sanford, North Carolina or building the plant at a site nearby.

Kim Bencker, head of communications at Pfizer Global supply, told us in an emailed statement We recently announced that were moving forward with scoping potential sites in Sanford for our new gene therapy site.

She added: This work is still in the preliminary stages and we arent able to share additional detail at this time.

The move follows a little over a year after the US drug manufacturer acquired Bamboo, a North Carolina-based gene therapy developer.

The deal included a recombinant Adeno-Associated Virus (rAAV) vector design and production technology, a Phase I candidate for Giant Axonal Neuropathy and a preclinical programme targeting Duchenne Muscular Dystrophy (DMD).

Pfizer also gained a 11,000sq ft gene therapy manufacturing facility in Chapel Hill that Bamboo bought from the University of North Carolina in 2016.

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Pfizer scopes out sites for gene therapy plant in North Carolina - BioPharma-Reporter.com