Page 3«..2345..1020..»

Archive for the ‘Gene Therapy Research’ Category

Renova Therapeutics co-founder awarded highest research honor … – PR Newswire (press release)

SAN DIEGO, Sept. 6, 2017 /PRNewswire/ -- Renova Therapeutics, a biotechnology company developing gene and peptide-based treatments for cardiovascular and metabolic diseases, today announced that the company's co-founder Dr. H. Kirk Hammond is the recipient of the 2017 William S. Middleton Award, the highest biomedical laboratory research award in the U.S. Department of Veterans Affairs (VA).

The Middleton Award is given annually to recognize outstanding achievements in biomedical research. Dr. Hammond, a Professor of Medicine at UC San Diego and a cardiologist with the VA San Diego Healthcare System, received the award for his contributions to the understanding of mechanisms of cardiovascular disease and novel gene transfer treatments for angina and heart failure. Dr. Hammond is also investigating gene transfer for type 2 diabetes.

Dr. Hammond has authored more than 100 peer-reviewed publications related to cardiovascular disease and is an inventor on nine patents. He devised and led the Phase 2 clinical trial of AC6 gene transfer for the treatment of patients with heart failure and reduced ejection fraction. Results of the trial indicated that, through a one-time administration, AC6 gene transfer safely increased heart function beyond optimal heart failure therapy (JAMA Cardiology). This study was funded by the National Institutes of Health (NIH), the Gene Therapy Resource Program and Renova Therapeutics, via an NIH public-private partnership.

"If successful, these trials could lead to the first registration of a gene therapy product for treating heart disease," said Dr. Rachel Ramoni, VA's Chief Research and Development Officer. "Dr. Hammond is clearly a pioneer of intracoronary gene therapy and novel patient delivery mechanisms that will have a broad impact on the health care of veterans."

AC6 gene transfer is being developed by Renova Therapeutics as RT-100, its lead gene therapy candidate advancing to a Phase 3 clinical trial known as FLOURISH.

About heart failureHeart failure is a chronic disease characterized by the inability of the heart to pump sufficient blood to meet the body's demands. It is a progressive and fatal chronic condition, and symptoms worsen over time. Heart failure afflicts more than 28 million people globally and is the only cardiovascular disease that is increasing in prevalence. In the United States, it is the most common cause for emergency hospital admissions in patients 65 and older.

About Renova Therapeutics Renova Therapeutics is developing definitive, one-time gene therapies and peptide infusion treatments to restore the health of people suffering from chronic diseases. The first indications the company is pursuing are gene therapy treatments for heart failure and type 2 diabetes, two of the most common and devastating chronic diseases in the world. The company's lead product, RT-100, is a treatment that delivers a therapeutic gene directly to the heart during a routine outpatient procedure and has the potential to increase heart function in millions of patients with heart failure. The company's product pipeline also includes a groundbreaking gene therapy in preclinical stage for sufferers of type 2 diabetes, as well as a peptide infusion therapy for the treatment of acute decompensated heart failure. Renova Therapeutics was founded in 2009 and is led by an experienced management team in biopharmaceuticals and gene therapy. For additional information about the company, please visit


View original content with multimedia:

SOURCE Renova Therapeutics


Read the rest here:
Renova Therapeutics co-founder awarded highest research honor ... - PR Newswire (press release)

Upcoming Duchenne Gene Therapy Trial to Be Focus of PPMD-hosted Webinar on Wednesday, Sept. 6 – Muscular Dystrophy News

Parent Project Muscular Dystrophy (PPMD) will host a one-hourwebinarat 2 p.m. Eastern Time on Wednesday, Sept. 6, that will focus on an upcoming clinical trial exploring gene therapy for Duchenne muscular dystrophy.

The webinar will be led by Dr. Jerry Mendell, who, together with fellow researcher Dr. Louise Rodino-Klapac, received a $2.2 million grant from PPMD in January 2017 for their gene therapy research project at the Nationwide Childrens Hospital in Columbus, Ohio.

The project is now approaching its first human trial, expected to begin in the next few months. Mendell will talk about how the trial is designed, including inclusion and exclusion criteria for participation. He will also share planned timelines.

Those wishing to participate are asked to register and submit questions in advance. Follow this link for more information about registering and submitting questions.

The grant was the first in the nonprofits Gene Transfer Initiative, which intends to support research into gene-therapy-based solutions. The webinar is part of a series that intends to present researchers and companies that focus on gene therapy for Duchenne.

Such therapies include gene transfer techniques, in which a small but functional version of the dystrophin gene, referred to as micro-dystrophin, is delivered with the help of a non-infectious virus. Other approaches use gene editing with the help of the CRISPR-Cas9 system (a naturally occurring bacterial defense system that has been adapted into a gene-editing tool).

The Nationwide Childrens Hospital trial will focus on the delivery of micro-dystrophin.

But while the webinar series will present research projects in various stages of progress, it spent the first parton Aug. 15 discussing what these approaches really mean, allowing patients and families to better understandthe complex science behind the therapeutic approaches. By understanding the science, PPMD hopes that families can make better choices once these therapies reach clinical trials.

PPMD also felt prompted to bring gene therapies to Duchenne patients with the recent FDA approval of Kymriah, the first gene therapy to be approved in the U.S.

While Kymriah is a cancer immunotherapy using a different approach than that likely to be used in Duchenne, the approval constitutes another piece of evidence showing the tremendous strides this technology has made since the 1990s and its early days of research, PPMDs Abby Bronson wrote in a blog post.

[The Kymriah] approval means that there are regulatory and commercial pathways for cell and gene based therapy. It means that you can put living DNA into a human and it can do its job, Bronson wrote in her blog. And it means that years of scientists making seemingly incremental advances can all come together and result into a giant step forward. A step forward that we believe will move this technology in a direction that will eventually benefit our community, our children.

Read the original post:
Upcoming Duchenne Gene Therapy Trial to Be Focus of PPMD-hosted Webinar on Wednesday, Sept. 6 - Muscular Dystrophy News

Has the Era of Gene Therapy Finally Arrived? – Scientific American (blog)

In 1990, geneticist William French Anderson injectedcells with altered genes into a four-year-old girl with severe immunodeficiency disorder. This was the first sanctioned test of gene therapy, in which genetic material is used to treat or prevent disease.

If were lucky, Anderson told The Chicago Tribune, with this little girl weve opened the door for genetic engineering to attack major killers and cripplers, particularly AIDS, cancer and heart disease.

Gene therapy has never fulfilled these grand hopes. In the decades since Andersons experiment, thousands of clinical trials of gene therapies have been carried out. But the first gene therapy was only approved for sale in the U.S. this week. The Food and Drug Administration announced its approval of Kymriah, a gene therapy produced by Novartis for a form of childhood leukemia. A few gene therapies have previously become available in China and Europe.

An FDA press release emphasizes the historic nature of the approval. Were entering a new frontier in medical innovation with the ability to reprogram a patients own cells to attack a deadly cancer, FDA Commissioner Scott Gottlieb says.

As I have noted in previousposts (see Further Reading), the hype provoked by genetic research has always outrun the reality. Gene-therapy proponents have long predicted that it will eliminate diseases such as cystic fibrosis and early-onset breast cancer, which are traceable to a defective gene, as well as disorders with more complex genetic causes.Enthusiasts also envisioned genetically engineered "designer babies" who would grow up to be smarter than Nobel laureates and more athletic than Olympians.

Gene therapy turned out to be extremely difficult, because it can trigger unpredictable, fatal responses from the body's immune system.The National Institutes of Health warnsthat gene therapy can have very serious health risks, such as toxicity, inflammation, and cancer.

Kymriah is a case in point. The FDA press release warns that Kymriah can cause life-threatening immune reactions and neurological events, as well as serious infections, low blood pressure (hypotension), acute kidney injury, fever, and decreased oxygen (hypoxia). According to The New York Times, the FDA is requiring that hospitals and doctors be specially trained and certified to administer [Kymriah], and that they stock a certain drug needed to quell severe reactions.

Kymriah illustrates another problem with gene therapy: high cost. Novartis is charging $475,000 for Kymriah. As a recent Reuters article notes, over the past five years two gene therapies have been approved for sale in Europe, one for a rare blood disease and the other for the bubble-boy immunodeficiency disorder. The therapies cost $1 million and $700,000, respectively. So far, the companies that make the therapies have achieved a total of three sales.

As journalist Horace Freeland Judson points out in this excellent 2006 overview, The Glimmering Promise of Gene Therapy, biology and economics have conspired against gene therapy. Judsonnotes that most individual diseases caused by single-gene defectsthe kind that seem most likely to be cured by gene therapyare rare. (Sickle-cell anemia and some other hemoglobin disorders are among the few exceptions.)

Judson adds that because different diseases have different genetic mechanisms and affect different types of tissue, each presents a new set of research problems to be solved almost from scratch. As the millions burned away, it became clear that even with success, the cost per patient cured would continue to be enormous. And success had shown itself to be always glimmering and shifting just beyond reach.

The advent of CRISPR, a powerful gene-editing technique, has inspired hopes that gene therapy might finally fulfillexpectations. Researchers recently employed CRISPRin human embryos to counteract a mutation that causes heart disease. Potentially, The New York Times reported last month, the method could apply to any of more than 10,000 conditions caused by specific inherited mutations.

CRISPR has also renewed concerns about the ethics of engineering people with enhanced physical and mental traits. These concerns are grossly premature. As Science noted recently, CRISPR poses some of the same risks as other gene therapies. The methodstill has a long way to go before it can be used safely and effectively to repairnot just disruptgenes in people.And in fact questions have now been raised about the CRISPR research on embryos mentioned above.

Some day, applied genetics might live up to its hype, but that day is far from arriving.

Further Reading:

Could Olympians Be Tweaking Their Genes?

Have researchers really discovered any genes for behavior?

My Problem with Taboo Behavioral Genetics? The Research Stinks!

Hype of Feel-Good Gene Makes Me Feel Bad.

New York TimesHypes "Infidelity Gene."

Quest for Intelligence Genes Churns out More Dubious Results.

Warrior Gene Makes Me Mad.

Should Research on Race and IQ Be Banned?

Read more:
Has the Era of Gene Therapy Finally Arrived? - Scientific American (blog)

Opinion: How investors should play gene-therapy stocks – MarketWatch

For a few thousand people around the world, reaching the age of 20 is a landmark to dread, not to celebrate.

Coping since birth with Leber Congenital Amaurosis (LCA), anyone with this genetic eye disorder who hasnt already lost their sight can expect to be legally blind before they reach 21 years of age.

Characterized by deep-set eyes that are prone to involuntarily, jerky movements, LCA is caused by a fault in one or more of about 14 genes so far identified. There is no proven treatment, although that may soon change.

In late August, biotech company Spark Therapeutics Inc. ONCE, +1.70% was granted a priority review of a treatment for LCA that may make it the first gene therapy approved for use in the U.S. by the Food and Drug Administration (FDA).

Read: Novartis CAR-T therapy was the first to be approved in the U.S.

The Philadelphia-based company will by Jan. 12 discover whether the FDA will issue a biologics license for Luxturna, which can replace the faulty RPE65 gene that causes LCA with a properly functioning copy. Should it be approved, victims of this disease will soon be able to receive a single injection that may permanently restore functional eyesight.

Gene therapys payoffs

While traditional research is usually focused on unlocking a way to treat one condition, gene therapies such as Luxturna may be game changers because they are based on platforms that can be adapted and used to tackle multiple inherited disorders.

Using similar techniques, Spark is also working on a functional cure for hemophilia, a disease that afflicts about 20,000 people in the U.S. and around 400,000 globally for which the market is worth about $8.5 billion in the U.S. and European Union.

In-human trials of SPK-8011 recently showed that Sparks therapy has the potential to lift the Factor VIII protein necessary for normal blood clotting to functional and sustained levels. In short, as with the Luxturna, the therapy has the potential to offer a one-shot cure.

That would be seismic for hemophiliacs, whose main option today is regular infusions of Factor VIII protein. Unfortunately, within a few days almost none of the protein remains in the body and the hemophiliacs blood is again unable to clot normally. Spark is also developing a treatment for hemophilia B, a much smaller market.

A new dawn

Biotech companies have reached this point because research has advanced to the stage where weve figured out how to identify the genetic causes of disease and how to apply that knowledge to develop therapies that will replace defective genes to provide a lasting cure.

Voyager Therapeutics Inc. VYGR, +24.70% is focused on gene therapies for neurological disorders such as Parkinsons, Huntingtons, Lou Gehrigs disease or ALS, Friedreichs ataxia (which damages the nervous system), Alzheimers and chronic pain.

In addition to cancer immunotherapy and the more controversial gene editing, bluebird bio Inc. BLUE, +0.84% has eight gene therapy programs, including research into adrenoleukodystrophy, or ALD, a deadly brain disorder that mostly affects boys and men; beta thalassemia; and sickle cell, none of which have a cure.

Should Spark, or another company such as BioMarin Pharmaceutical Inc. BMRN, -0.72% or Sangamo Therapeutics Inc. SGMO, -4.43% which are also working on hemophilia, succeed with its gene therapy, it could adversely impact suppliers of traditional Factor VIII protein infusions, such as Shire PLC SHP, +0.89% which had revenue from hemophilia treatments of $870.9 million in the first quarter of 2017.

Cost problems

Cost has been a headwind for the two gene therapies so far approved. In April, Fierce Pharma reported that uniQure NV QURE, +4.42% would not ask the European Medicines Agency to renew its marketing authorization for Glybera, the worlds most expensive drug at $1 million, when it expires in October, because in the four years after it gained approval in 2012 it was used commercially and paid for once, according to the MIT Technology Review.

Europes other approved gene therapy has fared no better. GlaxoSmithKline Plc GSK, +0.28% said in July it is seeking a buyer for Strimvelis, a treatment for a rare inherited immune deficiency, which took a year after approval to gain its first patient.

Perhaps the solution is a new payments system for ultra-expensive and long-lasting gene therapies, based on annuities for each additional time period of a treatments effectiveness.

But how do you measure cost? In December, Biogen Inc. BIIB, +0.48% gained FDA approval for Spinraza, a treatment for spinal muscular atrophy, the leading genetic cause of infant death in the U.S. Spinraza is priced at $375,000 a year for life (after $750,000 in the first year of therapy), while a one-shot gene therapy being developed by AveXis Inc. AVXS, +1.89% for SMA may provide a cure to someone who could go on to live 80 or more years. What sort of a premium for AveXis approach is justified?

Pricing is not dissuading biotech companies. There are about 7,000 genetic diseases, and the whole pharmaceutical and biotech industry is now working to solve each of those problems.

Investors seeking to benefit from a potential medical moonshot should consider allocating capital on a long-term basis to well-managed gene therapy companies with transformative assets that give them a competitive advantage.

Ethan Lovell is co-portfolio manager of the Janus Henderson Investors Global Life Sciences strategy.

Read the rest here:
Opinion: How investors should play gene-therapy stocks - MarketWatch

Lasker Awards honor Planned Parenthood and research on preventing and fighting cancer – Los Angeles Times

Planned Parenthood, the embattled nonprofit health provider that specializes in reproductive health, has won the 2017 Lasker Award for public service.

The Albert and Mary Lasker Foundation also honored Dr. Douglas Lowy and John Schiller of the National Cancer Institute with its clinical research award for work that led to the development of a vaccine against human papillomavirus, which causes cervical cancer. Molecular biologist Michael N. Hall received the foundations basic medical research award for laying the scientific groundwork for advances in the treatment of cancer, diabetes, neurodegenerative disorders and diseases of aging.

The Lasker Awards, announced Wednesday, are given annually to recognize advancements in the prevention and treatment of disease. Each award carries an honorarium of $250,000. Dozens of past winners have gone on to win the Nobel Prize.

The Lasker-Bloomberg Public Service Award to Planned Parenthood comes at a time when the international organizations global mission has come under budgetary assault on Capitol Hill. The healthcare provider offers cancer screenings, testing for sexually transmitted diseases, birth control services and general care to millions of people each year. But the organization also provides abortion services, which makes it a frequent target of some lawmakers and others with antiabortion views.

Approximately one in five women in the U.S. have received its assistance at some point during their lives, the Lasker Foundation said in its award citation. Without Planned Parenthood, many individuals would not have access to high-quality and affordable health care.

Lowy and Schillers research on infection-fighting antibodies led to the development of a vaccine against human papillomavirus. The virus, also known as HPV, causes the worlds second-most common cause of malignancy in women, cervical cancer. In 2014, that work led President Obama to award Lowy and Schiller the National Medal of Technology and Innovation.

All of this years honorees acknowledged the changed political environment in which they conduct their activities. All warned that their work and other work like it would be squelched if the Trump administrations proposed restrictions on womens healthcare and cuts to basic biomedical research funding are adopted by Congress.

Planned Parenthood President Cecile Richards noted that her organizations founders, Dr. Margaret Sanger and Dr. Bessie Moses, were the first women to be awarded the Lasker prize for medicine for their contributions to contraception at a time when it was illegal in the United States. She marveled that more than 65 years later, the U.S. government has reprised its hostility to the policies that the work of Sanger and Moses made possible.

Were at a moment in the U.S. where there are major political efforts to get a rollback of reproductive care and reproductive rights, Richards said.

The scientists honored by the Lasker Awards offered more indirect criticism. They suggested that amid deep budget cuts in federal funding for biomedical research, scientists will not have the latitude to pursue research on subjects whose significance in not yet understood.

Basic science is the engine that drives important breakthroughs in public health, said Schiller, whose work led to the development of the first vaccine to prevent a cancer.

Its not clear which basic discoveries are going to lead to public health breakthroughs, he added. Its an example where we cant be too top-down in our research enterprise. You cant dictate which discoveries will be made.

Evan Vucci / Associated Press

National Cancer Institute researchers Douglas Lowy, left, and John Schiller, shown here with President Obama, have been awarded the Lasker Award for clinical research.

National Cancer Institute researchers Douglas Lowy, left, and John Schiller, shown here with President Obama, have been awarded the Lasker Award for clinical research. (Evan Vucci / Associated Press)

That was certainly the case for Hall, an American and Swiss scientist based at the University of Basel in Switzerland whose work has been translated into therapies for a variety of diseases.

He won his Lasker Award for his discovery of a protein called TOR (short for target of rapamycin) that tells cells when to grow, divide and survive. The gene that expresses TOR is found in organisms ranging in complexity from yeast to humans, and it often mutates in cancer cells. In mammals, who have a version called mTOR, its also a key player in activation of the immune system.

Halls elucidation of how TOR works has led to the use of a class of targeted cancer drugs called mTOR inhibitors, including rapamycin and mimics such as the drug everolimus (marketed as Afinitor), in the treatment of certain aggressive cancers of the kidney, breast or brain.

Faulty signaling in the mTOR network is implicated not only in cancer, but in a range of other diseases linked to aging, such as diabetes and brain diseases. That has led many to believe that understanding how TOR works will lead to insights that could extend the human lifespan.

Among the insights already gleaned: that in mice, at least, calorie restriction lengthens lifespan by inhibiting the activity of mTOR.

The basic research honored by this years Lasker Award was part of an international race among scientists to unravel a mystery: why (and how) did the drug rapamycin, an antifungal medication that emerged from soil harvested on Easter Island, also have the ability to suppress the proliferation of both cancer cells and immune cells in mammals?

Hall and his colleagues identified and sequenced the TOR1 and TOR2 genes in yeast, and published the result in the journal Cell in 1993.



After California got rid of personal exemptions for vaccines, medical exemptions went way up

PSA screening for prostate cancer saves lives after all, study says

See the article here:
Lasker Awards honor Planned Parenthood and research on preventing and fighting cancer - Los Angeles Times

Stevenage-based Cell and Gene Therapy Catapult gets 12 million … – Comet 24

PUBLISHED: 18:08 31 August 2017 | UPDATED: 18:08 31 August 2017

Mia Jankowicz

The Gene and Cell Therapy Catapult is due to open in Autumn 2017. Picture: Daniel Buman

daniel burman

Email this article to a friend

To send a link to this page you must be logged in.

The Cell and Gene Therapy Catapult is making its home at the Stevenage Bioscience Catalyst campus in Gunnels Wood Road, and is due to open in autumn 2017.

Now an extra 12 million in government funds will go towards fitting out the buildings second floor.

The centre had already attracted 55 million of funding in 2014 from the Department for Business, Innovation and Skills (now the Department for Business, Energy & Industrial Strategy).

The extra funds will double the centres capacity and at full capacity it is predicted to bring 1.2 billion in revenue by 2020.

Chief executive officer Keith Thompson explained to the Comet that Stevenage was a good fit for the site, with the towns closeness to airports as well as the presence of other scientific expertise all big positives.

We went through a very rigorous search across the UK for our site, said Mr Thompson.

Theres a strong pedigree of pharmaceuticals around the area.

Stevenages workforce also stands to benefit, with the potential creation of around 180 support jobs.

The Cell & Gene Therapy Catapult has a mission to accelerate the UKs cell therapy industry and to make Stevenage an industry world leader.

Currently, one problem holding up cell research globally is the low availability of the large numbers of cells needed to perform large-scale clinical trials.

The 7,200-square-metre facility will allow UK businesses that are developing new cell therapy treatments to use its labs to manufacture cells for clinical trials at a large scale.

Cell and gene therapies are showing potential worldwide to combat numerous illnesses.

At the frontier of medical science, cell therapy is a technique which involves the injection of living cells into the human body in order to repair the direct causes of genetic diseases.

For example, the Cell & Gene Therapy Catapult played a large role in the creation of modified cells that are trained to recognise a certain protein in leukaemia cells, and then attack and destroy the cancerous cells.

To find out more visit

Here is the original post:
Stevenage-based Cell and Gene Therapy Catapult gets 12 million ... - Comet 24

News of NoteGene therapy to protect the heart; boosting chemo with cardio drugs; reversing memory loss – FierceBiotech

Could junk DNA protect our hearts?

Scientists at the University of California at Los Angeles and the Howard Hughes Medical Institute reported that they successfully used gene therapy to lower cholesterol in mouse models of familial hypercholesterolemia. The gene they used, called LeXis, was once considered junk DNA because it seemed to serve no purpose. But when the researchers gave the mice LeXis and then fed them a high-cholesterol diet for 15 weeks (think cheeseburgers and fries), their cholesterol went down, artery blockages opened up andless fat appeared to build upin their livers. The research was published in the journal Circulation. Release

Researchers at the Francis Crick Institute have discovered that acute myeloid leukemia (AML) causes bone marrow to leak blood, which in turn impedes the proper delivery of chemotherapy. So they tried mixing chemo with experimental drugs designed to treat heart and blood vessel disorders, and the results were promising. In mouse models of AML and in human tissue samples, the heart drugs stopped the leaks and the chemo became more effective, the researchers reported in the journal Cancer Cell. They believe the findings may point to a potential new combination of treatments for AML. Release

Researchers at Columbia University have completed mouse studies suggesting that a hormone produced by bone cells, osteocalcin, may be useful in reversing memory loss that occurs as part of aging. They gave aged mice continuous infusions of the hormone for two months and observed improvements on two different memory tests. Similar results were seen when the mice were given plasma from young mice, which have naturally high levels of osteocalcin. They plan to do more research to determine whether their findings, published in the Journal of Experimental Medicine, can be translated to drug therapies for people. Release

Read more here:
News of NoteGene therapy to protect the heart; boosting chemo with cardio drugs; reversing memory loss - FierceBiotech

Gene therapy using ‘junk DNA’ could lower risk for heart disease – UCLA Newsroom


Scientists from UCLA and the Howard Hughes Medical Institute successfully used a gene that suppresses cholesterol levels as part of a treatment to reduce plaque in mice with a disorder called familial hypercholesterolemia. In a preclinical study, researchers found that the gene, LeXis, lowered cholesterol and blockages in the arteries, and the treatment appeared to reduce the build-up of fat in liver cells.

Familial hypercholesterolemia is an inherited condition characterized by extremely high levels of low-density lipoprotein cholesterol (commonly referred to as bad cholesterol) and an increased risk of early heart disease.

The LeXis gene belongs to a unique group of genes that until recently were considered junk DNA because scientists believed they served little purpose. However, evidence from the human genome project led to the discovery that genes like LeXis are actually active. The study of these genes,now referred to as long noncoding ribonucleic acids, or lncRNAs, is a rapidly evolving area in biology.

Researchers wanted to test whether a single injection of LeXis could slow the development of heart disease. To do so, they gave the mice either LeXis or a control gene, and fed them a 15-week diet consisting of food high in sodium and cholesterol the mouse equivalent of fast-food hamburgers and french fries. Researchers then measured the progression of heart disease.

In the next phase of the study, researchers intend to confirm the findings in larger animals and test the therapy in combination with currently available treatments.

Although previous research has shown that lncRNAs can be important, this is the first study to show that they could potentially be used to treat a human disease using gene therapy. Junk genes could one day offer a framework for treating people with familial hypercholesterolemia and other conditions that are otherwise very difficult to treat.

The papers authors are Xiaohui Wu, Zhengyi Zhang and Dr. Tamer Sallam of UCLA; and Dr. Peter Tontonoz, Marius Jones and David Salisbury of the Howard Hughes Medical Institute.

The study waspublished onlinein the journal Circulation.

The research was supported by grants from the National Heart, Lung, and Blood Institute; the American College of Cardiology; and the Lauren B. Leichtman and Arthur E. Levine UCLA Cardiovascular Discovery Fund.

Learn more about the cardiovascular research theme at UCLA.

Continue reading here:
Gene therapy using 'junk DNA' could lower risk for heart disease - UCLA Newsroom

A ‘historic’ cancer treatment designed by Penn researchers just got approved by the FDA – The Daily Pennsylvanian

National Eye Institute / CC 2.0

Penn Medicine researchers made huge strides in the medical world Wednesday when the Food and Drug Administration approved a gene-altering cancer treatment that they designed. It's the first of its kind to be approved.

The therapy is marketed as Kymriah and made by Novartis, but was originally developed at Penn by Carl June, a Penn Medicine professor in immunotherapy, and his team, The New York Times reported. The treatment, which is the first-of-its-kind in the United States, uses the patient's genetically altered immune cells to fight the disease.

The FDA called the gene therapy a "historic" act.

Timothy Cripe, an oncologist with Nationwide Children's Hospitalin Columbus, Ohio, referred to the research as the "most exciting thing I've seen in my lifetime," The Washington Post reported.

The treatment is meant for children and young adults with B-cell acute lymphoblastic leukemia, especially those who don't respond well to traditional treatment methods.

According to the New York Times, the first child to receive the therapy was Emily Whitehead in 2012. Whitehead was severely ill from leukemia in 2012, but after treatment, has been free from cancer for more than five years.

Penn researchers have been working on approving this treatment method for years. In 2011, the results of the CAR-T cell therapy, as the treatment was initially called, were published in the New England Journal of Medicine and Science Translational Medicine by June and his team. It was the first demonstration of the use of gene transfer therapy to create serial killer T cells targeting cancerous tumors, according to a press release by the Penn medical school.

A year later, the University partnered with Swiss pharmaceutical company Novartis to continue research on immunotherapy research. At the time, the collaboration was the largest academic-industry agreement in Penns history.

In 2016, Penn Medicine, along with five other peer institutions, partnered with The Parker Institute for Cancer Immunotherapy after receiving a $250 million grant to develop new techniques for cancer treatment.

Novartis said the gene-therapy would cost $475,000 and would be available at an initial network of 20 approved medical centers, as the treatment is hard to administer.

I have to keep pinching myself to see that this happened, June said to The New York Times. It was so improbable that this would ever be a commercially approved therapy, and now its the first gene therapy approved in the United States. Its so different from all the pharmaceutical models. I think the cancer world is forever changed.

Continued here:
A 'historic' cancer treatment designed by Penn researchers just got approved by the FDA - The Daily Pennsylvanian

‘Hit-and-run’ gene therapy nanoparticles could enhance CAR-T … – FierceBiotech

Personalized cancer treatments known as CAR-T cells (chimeric antigen receptor T cells) have dominated the headlines lately, thanks to Novartis tisagenlecleucel, which won an early approval from the FDA for the treatment of leukemia on Aug. 30. But CAR-T treatments are labor-intensive and expensive to make, and they can attack healthy tissues in the body, leading to dangerous side effects.

Scientists at the Fred Hutchinson Cancer Research Center have developed a tool that they believe could address both those shortcomings of CAR-T and other forms of cell engineering. They have invented nanoparticles that deliver proteins to cells, which in turn edit those cells genes temporarily. Lead author and bioengineer Matthias Stephan describes it as hit-and-run gene therapy, and he believes the technique will streamline the manufacturing of cell-based therapies.

Heres how it works: The nanoparticles home in on specific cells, such as the T cells in the immune system. They then deposit messenger RNA (mRNA) to those cells, which triggers short-term changes in the proteins the genes produce. The technology does not permanently change the DNA, but it makes enough of an impact on it to produce a therapeutic outcome.

RELATED: Can CAR-T cancer treatments be fine-tuned to avoid toxic side effects?

Whats more, the nanoparticles can be freeze-dried and then activated with a small amount of water. They really let you fulfill all your wishes as a genetic engineer because you can pack in all your different [gene-therapy] components and further improve the therapeutic potential of your cell product without additional manufacturing steps, Stephan said in an article posted on Fred Hutchs website.

Stephans team proved out their concept by testing the nanoparticles in three different cell-engineering applications, one of which was CAR-T. Currently, CAR-T treatments are made by giving T cellsgenes that teach them to destroy cancer cells. The Fred Hutch scientists used their nanoparticles to remove a different gene from T cellsone that normally prompts them to attack healthy tissue.

Then they tried enhancing the CAR-T cells in a different manner. They temporarily gave them genes that have the potential to make central memory T cells, which are able to survive over the long term, remembering their cancerous targets and attacking them should they ever resurface.

The scientists tested their engineered CAR-T cells in mouse models of leukemia and found that the animals that received them lived twice as long as mice that got conventional CAR-T cells. They also tested the nanoparticles in two other cancer-related applications of gene therapy.

Despite all the excitement over CAR-T, concerns about side effects continue to dog the field. A dangerous immune reaction known as a cytokine storm has been seen in trials of both Novartis treatment and Axi-Cel, a CAR-T from Kite Pharma, which is being acquired by Gilead. The third player in the CAR-T field, Juno Therapeutics, saw its late-stage trials delayed when some patients died of neurological side effects.

Fred Hutch scientists have been working on other techniques for improving CAR-T. In December, a set of researchers there who receive funding from Juno announced positive results from a trial of a fine-tuned CAR-T treatment in patients with chronic lymphocytic leukemia (CLL). Instead of using just one type of CAR-T, the team combined two specially selected cell subtypes into one treatment. They also announced they had identified biomarkers that they believe can be used to predict which patients are likely to have severe reactions to the treatment.

Stephans team is now collaborating with several companies to fine-tune CAR-T treatments for cancer, according to Fred Hutch. And they believe their freeze-dried nanoparticles may prove useful in developing treatments for a range of other diseases, too, including HIV and blood disorders caused by defective hemoglobin.

Excerpt from:
'Hit-and-run' gene therapy nanoparticles could enhance CAR-T ... - FierceBiotech

Ipswich resident Talia Duff’s fight for life continues, family hopes to raise funds for research – Wicked Local Ipswich

By Bryan Sater

The urgent race to cure a rare disease that attacks the muscular system does not happen overnight.

Researchers have made significant progress toward saving 11-year-old Talia Duffs life. Duff suffers from CMT4J, a degenerative genetic disease similar to ALS. But plenty of work remains.

While the Duff family pushes the science of gene therapy to save Talia, fundraising efforts in the community continue and local teens have a plan to help Talia with the challenges of her daily life.

Working toward a cure

The potential cure for Talia exists in the form of gene therapy, where a healthy gene replaces the mutated gene (FIG4), which causes CMT4J.

Scientists at Jackson Laboratory, in Bar Harbor, Maine, have harvested a benign virus, like the common cold, stripped some of its DNA, and attached it to a healthy copy of the FIG4 gene.

Called a vector, this manipulated virus delivers the healthy FIG4 gene into the body by infecting cells in a targeted location, such as motor neurons in the spinal cord.

Theoretically, gene therapy not only stops the disease, but could allow peripheral nerves to heal and give Talia back some of her lost strength.

The gene therapy that could save Talias life could also have a wide-reaching effect on how rare disease are treated throughout the world. Potentially, others who suffer from a one-gene mutation defect could benefit by replicating the same sort of find and replace method that gene therapy proscribes.

Currently, research on mice genetically-altered to have CMT4J is being conducted at Jackson Labs. The mice have received the viral vector and are being studied for their response. Results of the experiment have not yet been published, but the Duffs scientific team expects to make an announcement in the coming weeks regarding the status of the test-subject mice.


While gene therapy has been identified as the potential cure for CMT4J, paying for the process is still a major obstacle. The money raised so far covered the cost of the viral vector creation, but as CMT4J advances its assault on Talias respiratory system, the Duffs continue to face increasing urgency in their fundraising efforts.

Pharmaceutical companies are not inclined to pipeline drugs for a disease that affects less than two dozen people worldwide. And normal scientific channels could take more than a decade, time which Talia does not have.

The Duffs did get some help from Jackson Labs, as the facility allocated money it received from a National Institute of Health grant toward some of the pre-clinical trial expenses.

However, the Duffs still need to raise between $1 million and $2 million in the coming year in order to fund a clinical trial in humans. They have raised nearly $300,000 so far and fundraising efforts are ongoing.

On Oct. 20, the Dare to Be Rare Gala will be Cure CMT4Js biggest fundraiser yet. Between sponsorship, ticket sales and live and silent auctions, the organization hopes to raise nearly $100,000.

Local fundraisers continue to chip away as well, including last weeks Tidbits for Talia at the Mayflower restaurant, flower sales at Ipswich Flowers, ongoing sales of brightly-colored shoelaces in the community, and outreach to major donors for significant gifts.

The amazing Ipswich community has contributed so much to our cause both financially and emotionally, said Talias mother, Jocelyn. Their support means so much to our family. It also lends tremendous credibility as we continue to pursue corporate and private donors beyond Ipswich. We hope they will feel compelled to give once they learn of Ipswich's generosity and the incredible work being done by our scientists in the lab."

Robotic arm

A group of high school students is doing what they can to help improve Talias quality of life. As CMT4J has progressed, Talia has lost most use of her extremities, including her arms and legs, compromising her ability to do simple things, such as lift a fork to her mouth or hold a book to read.

Enter the Ipswich High School Robotics Team. The students have established a four-phase plan to develop a robotic arm to help Talia with some of these regular tasks. They have secured a $10,000 Payne Grant to fund the project and have put together an initial prototype for the robotic arm that Talia will be able to use.

The team intends to accomplish two things with this project, said Rick Gadbois, team mentor, who met Jocelyn Duff at a rare disease conference last fall. Students will invent a device that helps Talia with daily activities, and will also publicize the effort to bring attention to CMT4J, to get funding for clinical research, for treatment and for a cure.

Talias health

While CMT4J weakened her limbs to the point where she depends entirely on a wheelchair, it has now begun to attack Talias respiratory system as well. The involuntary muscles that precipitate breathing are weakening, preventing her from coughing or taking deep breaths.

Three times per day, Talia must submit to a cough-assist machine, which, said Jocelyn Duff, forces air down her lungs and then vacuums it right back out in order to clear out any secretions in the lungs.

In addition, Talia now sleeps with a BIPAP machine, which delivers focused pressure to her lungs while she sleeps. The purpose is to maintain the oxygen saturation in her body at night, thus helping her sleep better and providing her more energy during the day.

Throughout it all, Talia continues to fight, and according to her mother, She is still her amazing, resilient, brave self.

Talias summer

Despite her condition, Talia was still able to enjoy summer as much as any 11-year-old. Her family vacationed in Maine, where she got to kayak, one of her favorite activities.

She took in a Red Sox game with a fellow CMT4J patient who visited from Seattle. The pair went onto the field at Fenway for batting practice and met Sox first baseman Mitch Moreland, who chatted with them.

Talia also began preparing for the start of sixth grade next week by going to locker night at the middle school and gathering her back-to-school supplies.

I think she is excited about school starting again this year, seeing her old friends and making some new ones, said Jocelyn Duff.

Visit link:
Ipswich resident Talia Duff's fight for life continues, family hopes to raise funds for research - Wicked Local Ipswich

Global Cartilage Repair Market 2017-2021 – Gene Therapy and Stem Cell Therapy is the latest Market Trend Making … – Business Wire (press release)

DUBLIN--(BUSINESS WIRE)--The "Global Cartilage Repair Market 2017-2021" report has been added to Research and Markets' offering.

The global cartilage repair market to grow at a CAGR of 11.59 % during the period 2017-2021.

The treatment of articular cartilage has evolved tremendously in the past decade. Reparative and restorative methods have been developed to address the significant source of morbidity in the young and active patients. Articular cartilage injury can be focal, which is localized or systemic. Procedures are being developed not only to alleviate the symptoms associated with articular cartilage defects but also to limit the progression of cartilage damages into degenerative diseases.

According to the report, one of the major drivers for this market is Rising incidence of accidental injuries. Globally, the road traffic injuries are increasing, with post complicated symptoms such as weakening of tendons, cartilage tear, and orthopedic issues.

The latest trend gaining momentum in the market is Gene therapy and stem cell therapy. Gene therapy is one of the promising fields in the cartilage repair. Many clinical studies have been performed for cartilage repair. The researchers are trying to develop gene therapy for cartilage repair and currently been investigated for clinical application.

Further, the report states that one of the major factors hindering the growth of this market is Product side effects. Surgeons use cartilage repair products such as tissue scaffold to improve the recovery. These products once grafted in the body may cause serious complications, resulting in their increased scrutiny for safety and efficacy. In many autologous chondrocyte implantation, there were common complications such as graft rejection, symptomatic hypertrophy, disturbed fusion and delamination.

Key vendors

Other prominent vendors

Key Topics Covered:

For more information about this report visit

See original here:
Global Cartilage Repair Market 2017-2021 - Gene Therapy and Stem Cell Therapy is the latest Market Trend Making ... - Business Wire (press release)

$91M Deal: CSL Acquires California Stem Cell Gene Therapy Developer – NBC 10 Philadelphia

CSL Behring, a Montgomery County-based global developer of biotherapeutic products, has entered into a deal to buy Calimmune Inc. for $91 million.

The deal also includes the potential for Calimmune to earn additional performance based milestone payments of up to $325 million over a period currently anticipated to be around eight years or more following the closing of the transaction. The transaction is expected to close within the next two weeks.

Calimmune, a biotechnology company specializing in hematopoietic stem cell gene therapy, has research and development facilities in Pasadena, Calif., and Sydney, Australia. [Hematopoietic stem cells are responsible for the production of all cellular blood components.]

The acquisition provides CSL Behring of King of Prussia, Pa., with Calimmunes pre-clinical asset, CAL-H, an experimental gene therapy for the treatment of sickle cell disease and beta-thalassemia. Officials at CSL Behring, a division of CSL Ltd. of Australia, said CAL-H complements CSL Behrings current product portfolio and its "deep expertise" in hematology.

To read the full story, click here.

For more business news, visit Philadelphia Business Journal.

Published at 9:28 PM EDT on Aug 28, 2017

$91M Deal: CSL Acquires California Stem Cell Gene Therapy Developer - NBC 10 Philadelphia

RegenxBio to Acquire Dimension Tx in Combo of Gene Therapy Companies – Xconomy

Xconomy Boston

Dimension Therapeutics has agreed to be acquired by RegenxBio, the gene therapy developer that originally helped form the company four years ago.

RegenxBio (NASDAQ: RGNX), based in Rockville, MD, will pay $3.41 per share in the all-stock deal valued at approximately $86 million. By comparison, Cambridge, MA-based Dimension (NASDAQ: DMTX) went public at $13 per share in 2015. Dimensions closing stock price on Thursday was $1.20.

The acquisition agreement comes two months after Dimension laid off a quarter of its staff and shifted its priorities away from its experimental hemophilia B gene therapy, DTX101. Dimension made those moves in the wake of early clinical trial results that suggested the therapy prompted a possible immune system response. While immunosuppressive steroids can tamp down these responses, such treatment can also diminish the effect of gene therapy. Dimensions stock price tumbled by nearly 50 percent on those January results.

Gene therapies aim to treat inherited disorders by transplanting normal genes that correct or fix genes that are missing or defective. Fixing the gene is meant to address the root cause of a disease and offer a long-lasting treatment, and perhaps even a cure. The FDA has not yet approved any gene therapies, but at one point, Dimension was in the mix of companies aiming to bring these treatments to the market. Spark Therapeutics (NASDAQ: ONCE) has since emerged as the company likely to receive the first U.S. gene therapy approval. The Philadelphia biotechs lead gene therapy candidate, a potential treatment for an inherited form of blindness, is currently being reviewed by the FDA. Spark has also reported progress in early-stage clinical trials for its hemophilia B gene therapy candidate.

Dimensions gene therapies deliver healthy genes using a modified virus, a gene delivery technology that was developed by RegenexBio. In 2013, RegenxBio joined with Fidelity Biosciences to form Dimension. Dimensions scientific and technical advisory board was led by James Wilson, a University of Pennsylvania geneticist who was RegenxBios scientific founder.

In acquiring Dimension, RegenxBio will add to its pipeline two Dimension gene therapy candidates: One compound, called DTX301, is being prepared to start clinical testing in patients who have ornithine transcarbamylase (OTC) deficiency, a disorder characterized by the lack of an enzyme key to breaking down and removing nitrogen from the body. The other compound, DTX401, is in development as a potential treatment for glycogen storage disease type 1a (GSD1a), an inherited disorder that leads to the buildup of a complex sugar called glycogen. RegenxBio will also acquire other preclinical compounds in development, as well as intellectual property that Dimension developed using RegenxBios technology.

The merger agreement prohibits Dimension from seeking a better deal, and the company has agreed to unspecified certain restrictions on responding to any proposals that may come its way, according to a securities filing. Dimension shareholders still need to sign off on the deal, which has received approval from the boards of directors of both companies. But if Dimension calls off the deal, it must pay RegenxBio a $2.85 million termination fee, according to the filing.

The companies expect to close the acquisition by the end of 2017. Upon closing, Dimension will become a subsidiary of RegenxBio; Dimension shareholders will own approximately 10.9 percent of the combined company, according to the agreement.

Image from Depositphotos.

Frank Vinluan is editor of Xconomy Raleigh-Durham, based in Research Triangle Park. You can reach him at fvinluan [at]

Read the rest here:
RegenxBio to Acquire Dimension Tx in Combo of Gene Therapy Companies - Xconomy

Global Gene Therapy Partnering Terms and Agreements 2010 to … – Business Wire (press release)

DUBLIN--(BUSINESS WIRE)--The "Global Gene Therapy Partnering Terms and Agreements 2010 to 2017" report has been added to Research and Markets' offering.

The Global Gene Therapy Partnering Terms and Agreements 2010-2017 report provides an understanding and access to the gene therapy partnering deals and agreements entered into by the worlds leading healthcare companies.

The report provides a detailed understanding and analysis of how and why companies enter gene therapy partnering deals. The majority of deals are early development stage whereby the licensee obtains a right or an option right to license the licensors gene therapy technology or product candidates. These deals tend to be multicomponent, starting with collaborative R&D, and commercialization of outcomes.

This report provides details of the latest gene therapy, oligonucletides including aptamers agreements announced in the healthcare sectors.

Global Gene Therapy Partnering Terms and Agreements includes:

In Global Gene Therapy Partnering Terms and Agreements, the available contracts are listed by:

Key Topics Covered:

Executive Summary

Chapter 1 - Introduction

Chapter 2 - Trends in Gene therapy dealmaking

Chapter 3 - Leading Gene therapy deals

Chapter 4 - Most active Gene therapy dealmakers

Chapter 5 - Gene therapy contracts dealmaking directory

Chapter 6 - Gene therapy dealmaking by technology type

Chapter 7 - Partnering resource center

For more information about this report visit

See the original post:
Global Gene Therapy Partnering Terms and Agreements 2010 to ... - Business Wire (press release)

Cambridge gene therapy firm Dimension Therapeutics to be acquired – Boston Business Journal

Boston Business Journal
Cambridge gene therapy firm Dimension Therapeutics to be acquired
Boston Business Journal
One of the three gene therapy biotechs in Cambridge, Dimension Therapeutics, has agreed to be acquired by a Maryland company in an all-stock transaction that values Dimension at just $86 million a fraction of its value a year ago. ... team and of ...

Read the rest here:
Cambridge gene therapy firm Dimension Therapeutics to be acquired - Boston Business Journal

Birth control research is moving beyond the pill – Science News Magazine

Mention the pill, and only one kind of drug comes to mind. The claim that oral contraceptives have on that simple noun testifies to the pills singular effect in the United States. Introduced in 1960, the pill gave women reliable access to birth control for the first time. The opportunity to delay having children opened the door to higher education and professional careers for many women.

More than 50 years later, the most commonly used form of reversible contraception in this country is still the pill. Additional methods have been developed for women such as implants, patches, vaginal rings and injectables but most do basically the same thing as the pill: use synthetic versions of sex steroid hormones to suppress ovulation. The method has proved its merit, but the current crop of contraceptives doesnt work for everyone. Some women cant tolerate the side effects stemming from manipulation of the hormones. Others cant use hormonal contraceptives at all, because of underlying health conditions.

In a survey, 62 percent of U.S. women ages 15 to 44 reported using contraception in 2011 to 2013. The pill was the most popular form of birth control, followed by female sterilization (which permanently blocks the fallopian tubes). Rounding out the top five methods were the male condom, long-acting reversible contraception (like intrauterine devices and implants) and male sterilization (vasectomy). In the survey, if women used more than one method, only the most effective method was counted.

And whats new for men? Their main mode of contraception, the condom, has been around for at least 400 years, perhaps longer. Alternatively, men who want to take the lead on family planning can go the surgical route with a vasectomy.

The dearth of alternatives is not due to a lack of research. Reproductive biologists and other researchers have made many exciting discoveries since the pill was introduced. But taking a promising finding in cells or in mice to human testing is hard for any drug. And for contraceptives, theres an extra wrinkle: Youre developing products for very healthy people, so you have to make sure [the drugs] are incredibly safe, and the side effect profile is acceptable, says Diana Blithe, a biochemist and chief of the contraceptive development program at the National Institute of Child Health and Human Development in Bethesda, Md.

Even with the long road to human testing, odds are that by the time the pill turns 75, there will be new options stocking the contraceptive cabinet. Researchers are currently exploring a method that keeps womens eggs in a state of suspended animation for later use. For men, there could be nonhormonal methods that stop sperm from developing and launching their epic journey. The impact of these novel methods might ripple out into society much as the pills once did.

There were 6.1 million pregnancies in the United States in 2011. Forty five percent of them, or a whopping 2.8 million, were not intentional, according to a 2016 report in the New England Journal of Medicine.

Unplanned pregnancies can have consequences for parents and kids, studies find. Womens education can be cut short. Unwanted pregnancies are linked to delayed prenatal care probably because moms dont realize theyre pregnant as well as low birth weight in infants. Postpartum depression is more common for mothers who did not intend to have a baby than for those who did.

The numbers also suggest that the contraceptives available arent meeting everyones needs. Some methods are expensive. And some users have health concerns or just dont stick with an option. In 2008, about 40 percent of unintended pregnancies were in couples that used contraception, but inconsistently, according to the Guttmacher Institute, a reproductive health research and policy organization in New York City.

Proportion of U.S. pregnancies in 2011 that were unplanned

From 2011 to 2013, the most popular reversible contraceptive choice for women ages 15 to 44 was the pill, with use at nearly 26 percent. The pill and other hormonal contraceptives contain the female sex steroid hormones estrogen and progesterone, or progesterone alone, usually in synthetic forms. These hormones prevent ovulation by suppressing the brains release of follicle-stimulating hormone and luteinizing hormone.

Some women find that hormonal contraceptives work well; other women experience side effects such as headaches, nausea, mood changes and acne. Oral contraceptives also increase the risk of blood clots, taking the drugs off the table for women with a history of blood clots, stroke or cardiovascular disease. The pill is also a no-go for women with severe hypertension or who have ever had breast cancer.

Relying on hormones to halt sperm production can also work. A new hormone-based gel for men, applied to the skin, is in human testing. It combines the male sex steroid testosterone with a synthetic progesterone. Plans are under way for couples to test the gel as their only form of birth control. But giving men hormones can come with side effects, such as reduced muscle mass and a drop in sexual function.

Discoveries that are beginning to explain the earliest stages of egg development and the finishing touches of sperm growth may lead to steroid-free alternatives.

Hormonal contraception disrupts ovulation, and the egg that was scheduled for departure from an ovary dies. But what if there was a method that preserved the egg for later?

When women are born, their ovaries have a full set of oocytes, or eggs a million or so. Each is housed within a sac of cells called a follicle. The outer portion of each ovary is filled with the earliest, dormant form of these egg-carrying follicles, called primordial follicles. The sleeping cells are waiting to be woken up, so they can begin growing in preparation for ovulation. But why the alarm clock goes off for one primordial follicle and not another is an open question, says reproductive biologist David Ppin of Massachusetts General Hospital and Harvard Medical School.

You could potentially preserve that pool of eggs for later in life, theoretically.

David Ppin

Todays hormonal contraceptives act on ovarian follicles that are already growing, and once that starts, there is no going back if ovulation doesnt happen, the egg dies. Aiming contraception at the sleeping eggs could mean putting off pregnancy, while holding on to the eggs. By preventing that first wake-up call, actually, you keep the egg, Ppin says. You could potentially preserve that pool of eggs for later in life, theoretically.

Meet the biological agent that could keep eggs asleep: Mllerian-inhibiting substance, or MIS. Also known as anti-Mllerian hormone, MIS is not a sex steroid hormone. It is produced in the developing testes and prevents male embryos from growing female reproductive parts. In adult female mice, MIS can also be a perpetual snooze button for primordial follicles, Ppin and colleagues, including Mass General and Harvard pediatric surgeon Patricia Donahoe, reported in the Feb. 28 Proceedings of the National Academy of Sciences.

Hundreds of follicles are estimated to be in various stages of development at any given time. The active growers release MIS locally, which limits the number of primordial follicles that wake up. This process allows the body to control and maintain the supply of eggs over a womans reproductive life span.

Primordial follicles, the sacs that house immature eggs, reside in the outermost region of the ovary. When follicles wake up, they begin to develop and move farther into the ovary. When a womans monthly menstrual cycle begins, follicle-stimulating hormone prompts additional growth of certain developing follicles. A dominant follicle matures. Luteinizing hormone helps the mature follicle open up, and the egg is ovulated and released into the fallopian tube. New experimental approaches to birth control aim to keep the primordial follicles dormant, so they can be available later in a womans life.

In their study, Ppin, Donahoe and colleagues used a virus to introduce a modified version of the MIS gene into certain cells in mice. This permanent change gave the mice a higher dose of MIS protein than is found normally in females. The follicles that had already been growing completed their development, but after that, no new follicles were activated, leaving a collection of sleeping-beauty primordial follicles.

When the researchers paired female mice treated with the gene therapy with males, the females were still able to become pregnant and have healthy babies within the first six weeks, because of those follicles that had already started growing in the ovaries. Once that supply was used up, the females were infertile.

Youre just stopping the horses that havent yet come out of the gate, Donahoe says.

To test a nonpermanent approach, the team gave normal female mice the MIS protein as a twice-daily shot. Activation of primordial follicles stopped. When treatment ended, the ovaries got back to business and follicles began growing again.

Ppin and Donahoe see several uses for MIS as a contraceptive. The permanent gene therapy method could be a nonsurgical contraceptive approach for pets or stray animals. The research team is working with the Cincinnati Zoo to study this method in cats.

Frequent shots of the MIS protein are too expensive for broad use, but they could help protect the reserve of ovarian follicles in young cancer patients. Growing follicles are dividing quite rapidly, so they are very sensitive to chemotherapy, Ppin says. Chemo can kill off the growing follicles, which means there is no more MIS to stop activation of other primordial follicles. Too many follicles wake up, which can deplete a womans egg supply. In mice given chemotherapy drugs, MIS-treated animals were left with more primordial follicles than untreated animals, the researchers found.

Still eager to make an MIS-like contraceptive for all women that is cheap and easy to use, perhaps as a pill, the researchers are searching libraries of small molecules to find one that mimics the action of MIS. Maybe it would be an already existing [U.S. Food and Drug Administration] approved medication thats the first screen we are performing or maybe its a very simple molecule, very cheap to synthesize, Ppin says.

Story continues below image

In the ovary of a normal mouse (left), a large follicle is shown at a late stage of development (a light pink oocyte surrounded by follicular cells, inset). In the ovary of a mouse treated with Mllerian-inhibiting substance, follicle development ceased and only primordial follicles were found (arrows, right).

In men, vitamin A does more than promote healthy eyes. Its essential for sperm production, too. The testes take up vitamin A from carrots and other foods and convert it to retinoic acid. The acid binds to the retinoic acid receptor, which is found in cells throughout the body.

In the 1990s, scientists reported that when they disrupted the gene for one version of the retinoic acid receptor, referred to as alpha, in mice, the animals are fine, but the males are sterile, says geneticist Debra Wolgemuth of Columbia University Medical Center. Wolgemuth and her colleagues, who study the biology of sperm production, set out to find a drug that could interfere with the receptor, rather than permanently knocking out the gene.

Wolgemuth came across a paper from 2001 by a group studying a drug that could bind to all three versions of the receptor, including alpha. The drug inactivates the receptor and shuts down the series of events that typically follow. Although tests in rats showed the drug could be taken orally and broken down safely by the body, the researchers highlighted one notable side effect. They called it testicular toxicity, Wolgemuth says.

Rather than a negative, Wolgemuth saw the toxicity as a sign of a potential male contraceptive. With molecular biologist Sanny Chung of Columbia and colleagues, she gave the drug to male mice for seven days, then examined their testes.

Sperm go through many stages of development as they transition from round germ cells to their final shape with a characteristic head and tail. Before sperm are released to begin their journey through the male reproductive system, says Wolgemuth, they line up like little soldiers in a battalion to leave the testes.

Story continues below image

In healthy mice, normal sperm line up at the center of a part of the testes known as the seminiferous tubule, ready for release (left, arrows). Mice treated with a drug that blocks whats known as the retinoic acid receptor have defective sperm that dont line up (right, arrows).

In mice treated with the drug, the sperm dont align properly, Wolgemuth and colleagues reported in 2011 in Endocrinology. The sperm arent released, so they die in the testes. The researchers found no evidence of harm to other organs. Male mice given the drug once a day for four weeks became infertile by the end of treatment and remained that way for four weeks after treatment stopped. By 12 weeks after treatment, the mice regained their mojo and successfully mated with females.

Later, the team gave mice a smaller dose of the drug for 16 weeks, over a quarter of their reproductive lives, notes Chung. The treated mice became sterile, but once off the drug, they soon became papas to healthy pups that grew into fertile adults, the researchers wrote in Endocrinology last year.

Next step: Wolgemuth plans to test the drug in nonhuman primates. Her group is also collaborating with a team of medicinal chemists to look for compounds that target only the alpha version of the retinoic acid receptor. Even though the tested drug did not lead to side effects, having an option that doesnt interfere with the other two versions of the receptor would be ideal, says Wolgemuth.

Another nonhormonal male contraceptive is the result of a long research career dedicated to such a product. In the late 1960s, Joseph Tash had two tours as a summer student in an obstetrics and gynecological department at Michael Reese Hospital in Chicago. He saw how heavily the burden of birth control fell to women. I felt it was important to try to expand the contraceptive and family planning choices to men, he says.

In 2013, the compound H2-gamendazole became the first nonhormonal contraceptive to receive FDA regulatory guidance, a crucial thumbs-up along the drug development road. Its a kind of checklist of the testing conditions and experiments necessary to proceed with preclinical and human trials.

Tash, now at the University of Kansas Medical Center in Kansas City, and colleagues began with an anticancer drug that, during clinical trials, severely cut down on sperm production. But there were a lot of side effects, Tash says, which would be totally unacceptable to otherwise healthy males. So the researchers designed similar drugs to minimize the side effects, including H2-gamendazole. Rats given a single oral dose of the drug once a week for six weeks became sterile after two weeks of use. By 10 weeks after the dosing stopped, all of the animals were fully fertile again.

The drug interferes with the last stage of sperm development, when the cells acquire their familiar sperm features. At this stage, as well as throughout the developmental process, sperm are tended to by Sertoli cells, which feed and support the growing sperm. The sperm are actually tethered to the Sertoli cells to prevent them from leaving the reef before they can swim.

H2-gamendazole disrupts the junctions between the sperm and the Sertoli cells, releasing the sperm prematurely and leading to their destruction. The testes have a built-in cleaning system, so to speak, that gets rid of the abnormal sperm, Tash says.

Tashs team has also tested H2-gamendazole in mice, rabbits, dogs and monkeys. In each animal, there was a block in sperm production just exactly like we see in the rats, Tash says. The team has also found that the drug can be taken as a pill and is rapidly taken up by the testes, at levels 10 to 20 times higher than in other tissues. I think this explains to a large extent why we havent seen any remarkable side effects, Tash says.

The work on H2-gamendazole, yet to be published, led to the FDAs regulatory guidance, a show of confidence in the drug. If Tash and colleagues can demonstrate to the FDA that the drug is safe and well tolerated, that might pique the interest of pharmaceutical companies to handle the final stages of testing and to take the drug to market. Its going to have to be a squeaky clean compound for pharma to become interested, Tash says.

Birth control methods born of these projects might shake things up outside the bedroom. If further testing finds that eggs kept asleep by an MIS-based contraceptive remain healthy and viable, delaying pregnancy may not necessarily lead to reduced fertility. A lot of women 35 and older are faced with reduced fertility, Ppin says. A method to target the activation of primordial follicles so you could keep them for later I think that would be beneficial.

Any new contraceptive options for men could shift the conversation men and women have about birth control. A multinational survey published in 2005 found more than half of men would be willing to use a new method of male birth control. There is an increasing number of men who are willing to help carry that burden, Tash says.

When that first product gets out there for men, Blithe adds, I think that will be a turning point.Any new contraceptive options for men could shift the conversation men and women have about birth control. A multinational survey published in 2005 found more than half of men would be willing to use a new method of male birth control. There is an increasing number of men who are willing to help carry that burden, Tash says.

This story appears in the Sept. 2, 2017 Science News with the headline, "Access denied: Scientists seek innovative ways to block the union of egg and sperm."

More here:
Birth control research is moving beyond the pill - Science News Magazine

Apic Bio Launches to Advance First-in-Class Gene Therapy for … – Business Wire (press release)

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Apic Bio, Inc., a pre-clinical stage gene therapy company leveraging its proprietary platform to advance therapies to treat rare diseases with complex mechanisms, in particular Alpha-1 Antitrypsin Deficiency (Alpha 1), launched today with an initial investment led by the venture philanthropy arm of the Alpha-1 Foundation and a private investor with the disease.

Its lead product, APB-101, targets the liver via an AAV delivered Dual Function Vector (df-AAV) whereby the Z-AAT protein is silenced and M-AAT protein is augmented. APB-101 has achieved a pre-clinical proof of concept with efficacy demonstrated in vitro and in vivo. It is currently undergoing pre-clinical GLP toxicology studies in non-human primates. Patients living with Alpha 1 lack sufficient levels of circulating AAT protein to protect lung tissue against damage from proteases, and experience the accumulation of mutant AAT polymers in the liver. Clinically, the deficiency is manifested by progressive emphysema and the accumulation presents a significant risk of liver cirrhosis.

John Reilly, Co-Founder & President said: We are grateful to TAP and A1AT Investors, LLC who have supported the successful start of Apic Bio by providing the first tranche of our seed financing round allowing us to secure key intellectual property rights and operational support. With such strong support from the advocacy and patient community, we are confident that we will identify the right corporate partners to help us achieve our business development goals and bring this exciting new therapy to patients.

The df-AAV platform allows treatment of other diseases with complex mechanisms where the mutant gene product must be reduced and the normal gene product must be augmented.

Dr. Chris Mueller, Co-founder and Chief Scientific Officer of Apic Bio said: We are encouraged by the feedback that we have received during our pre-IND meeting with the FDA that there is a clear path for us to conduct a first-in-human Phase 1/2 clinical study. Furthermore, we are very much looking forward to demonstrating the benefit of APB-101 to patients that have been living with alpha-1 and have had very little hope for a cure. Our data suggests this is a liver sparing approach for gene augmentation which may exceed the therapeutic and safety margins when compared to a strict gene augmentation without gene silencing that may exacerbate the underlying liver disease.

TAP is very pleased to provide this funding to Apic Bio. Their cutting-edge work on a therapy that addresses both the liver and lung disease brings us closer to finding a cure for Alpha-1 Antitrypsin Deficiency, thus fulfilling our mission, said Jean-Marc Quach, CEO for The Alpha-1 Project.

Todays launch of Apic Bio has been a long time coming for the hundreds of thousands of people who are challenged by Alpha 1, said Ed Krapels, who has been living with Alpha 1 and is the new companys first individual investor. Now that we are moving forward, we hope to work with patients, their advocates and researchers to make a cure readily available. Krapels added.

About Apic Bio: Apic Bio, Inc. is a spin-off from the University of Massachusetts Medical School (UMMS) and is based upon nearly 30 years of gene therapy research by its scientific founders Christian Mueller, PhD, Associate Professor of Pediatrics and a member of the Horae Gene Therapy Center at the University of Massachusetts Medical School, Terence R. Flotte, MD, the Celia and Isaac Haidak Professor in Medical Education, dean of the School of Medicine and provost and executive deputy chancellor of the University of Massachusetts Medical School; and colleagues at the Horae Gene Therapy Center. Their research is funded in part by an $11M grant from the National Heart, Lung, and Blood Institute (NHLBI).

View original post here:
Apic Bio Launches to Advance First-in-Class Gene Therapy for ... - Business Wire (press release)

Comic and Telethon Host Jerry Lewis Dies At 91 – WebMD

Aug. 21, 2017 -- Jerry Lewis, a consummate performer on stage and screen who used his fame to raise billions of dollars toward a cure for muscular dystrophy and other neuromuscular diseases, died Sunday at his home in Las Vegas. He was 91.

Born Joseph Levitch on March 16, 1926, in Newark, NJ, to vaudeville parents, Lewis wrote, appeared in, and directed 80-plus movies and TV shows over 5 decades in show business. He was memorable for his goofball antics and rubbery face (The Nutty Professor, The Bellboy, and The Ladies Man) and for his capacity for self-parody. Early in his career, he formed half of a comedy team with the late Dean Martin, with whom he hosted "The Martin and Lewis Radio Show" and made 16 films. In 1956, Lewis recorded an album (Jerry Lewis Just Sings) that made the Top 20 on the Billboard charts.

For many people, Lewis will be remembered best for raising awareness and money for the Muscular Dystrophy Association (MDA) during the 50-plus years he hosted the nationally televised Labor Day weekend telethon. In all, the shows raised more than $2 billion.

From 1956 until 2010, Lewis was the face of muscular dystrophy, a relatively rare neuromuscular disease that often begins in childhood and progressively robs a person of mobility. Lewis would wrap up the 21 1/2-hour annual show with a heartfelt version of Youll Never Walk Alone. He would typically sing it in a voice hoarse from hours of urging viewers to contribute to the cause, and probably from smoking on air throughout the broadcast.

In 1977, Lewis was nominated for a Nobel Prize for his 50 years of fighting muscular dystrophy.

(We) will be forever grateful to Jerry Lewis, a world-class humanitarian.

The reason for his stepping down as host of the telethon isnt clear -- The Hollywood Reporter wrote that he was unceremoniously dumped -- but Lewis never seemed to have talked about it publicly.

The MDA, on its website, praised Lewis efforts, saying it will be forever grateful to Jerry Lewis, a world-class humanitarian, for his indefatigable and inspiring work on behalf of kids and families with neuromuscular diseases, and for the countless dollars his commitment helped raise for critical research and services.

Perhaps Lewis left because the telethon, which had shrunk to a 2-hour show, had outlived its usefulness. In May 2015, the MDA announced it would end the telethon because of the expense and the realities of viewership and concentrate its fundraising on social media and other web-based channels.

The MDA raises money for medical research on 40 neuromuscular diseases, including Duchenne/Becker muscular dystrophy, the most prevalent form of MD, and amyotrophic lateral sclerosis (ALS), or Lou Gehrigs disease. In 2015, the organization said it was focusing support on gene therapy research and new drugs.

While Lewis did not suffer from a neuromuscular disease, he struggled with health issues for years. He had type 1 diabetes and pulmonary fibrosis, a condition in which tissue deep in the lungs scar and stiffen, making it more difficult for oxygen to get into the blood and causing shortness of breath. He suffered two heart attacks. He also had prostate cancer surgery in 1982. He had chronic back pain, which led to an addiction to the prescription painkiller Percodan, which he successfully replaced with an implanted device that dulls nerve impulses. In 2003, he had to wean himself off of steroids used to treat his lung disease.

Lewis last performances on stage were in March 2014, when he sold out two shows at La Mirada Theatre in California. He was 88 at the time, although he appeared in the 2016 film The Trust, with Nicolas Cage and Elijah Wood.

Lewis was divorced from Patti Lewis, with whom he had six children, and married SanDee Pitnick in 1983. They adopted a daughter together. In 2009, Lewis youngest son, Joseph, who struggled with drug addiction, committed suicide at age 45. In an interview he gave to The Hollywood Reporter in June 2014, Lewis was broken up by Josephs death, saying, To this day I don't understand it because it's unfair -- not unfair to me, but unfair to him. That he went that way made the unfairness stupidity. But he was my son and he's gone, and there's not a lot I can do about that. I beat myself a thousand times.

He was beloved throughout the world, but the French were particularly enchanted by Lewis. In 2006, for his 80th birthday, Lewis was awarded a medal and induction as a commander into the Legion of Honor, which is considered the highest decoration in France. It is akin to being knighted by the queen in England.

Lewis has two stars on the Hollywood Walk of Fame and received the Jean Hersholt Humanitarian Award at the 2009 Oscars ceremony. He received many other honors throughout this life, both for his humanitarian work and his work as a TV and movie star, and as a producer and director.

IMDb: Jerry Lewis Biography. Jerry Lewis.

Muscular Dystrophy Association.

The Hollywood Reporter: At Home With Jerry Lewis as He Opens Up About Son's Death, Skirmishes With Fans.

The Associated Press: Jerry Lewis telethon ends decades-long run, fundraising awareness for Muscular Dystrophy Association.

CDC: Facts About Muscular Dystrophy.

Pulmonary Fibrosis Foundation.

View original post here:
Comic and Telethon Host Jerry Lewis Dies At 91 - WebMD

Pacific Biosciences Is Advancing Genomics – Seeking Alpha

Pacific Biosciences of California (PACB) is a $480 million market cap company focused on development of innovative technologies and systems that impact diagnosis and treatment of disease and improve the world's food and energy supply. The company developed the Single Molecule, Real-Time (SMRT) Sequencing genomics technology, which enables real-time analysis of DNA synthesis. The company's technology can be used in human biomedical research to resolve heritability and variant types across populations or disease states.

Applications in plant sciences and agriculture include crop and livestock research acceleration via sequencing and transcriptome analysis. PACB technologies can also be used to characterize viruses and microbes of infectious disease, enabling the design of better vaccines and treatments.

So why would the reader be interested in PACB? The market for gene therapy applications are increasing at a CAGR of more than 20%, according to an analysis at Global Market Insights. In 2015, the gene therapy market was reported at over $800 million, and is expected to rise to $1.4 billion by 2024. The following figure depicts what this 20% growth looks like for a potential successful long investor. Nice, isn't it?

But gene therapies alone don't tell the whole story of market applications for genetic research tools. Cell therapy is an industry that plays a large role in applications for genetic analysis. The combined markets for cell and gene therapies are expected to rise from approximately $8 billion in 2018 to $12 billion in 2020. Strong Bio hopes that these predictive trend models can be useful for investors, and rather than focus on a company's pipeline in every article, it is striving to meet the needs of investors by focusing on some backstage players that have immense potential in advancing medical and agricultural research, such as PACB.

The rise in markets for gene therapies and cell therapies is expected to be driven by rapid technological advancement and increased adoption of new genomic techniques. Obviously, the largest market space in cell therapy and gene therapy application is cancer, with inflammatory disease also comprising a significant component of these potential markets. The World Health Organization predicts the number of new cancer cases will rise by as much as 70% over the next 20 years.

That is a lot of patients, and will fuel industry growth. In addition, favorable FDA regulation stances will serve cancer genetics industry growth in a positively weighted manner. Cancer genetics programs will begin to mainstream infrastructure to provide assays, informatics, and gene testing education to patients at hospital sites. Cancer will become a treatment regime reminiscent of chronic disease models, targeting specific molecules involved in specific patient's pathology.

The company sells its SMRT technology as a package product called Sequel System. SMRT supports numerous sequencing applications, bringing unique and novel depth and quality to genetic research. It is applicable in whole genome analysis for total genetic composition data of organisms including microbes, humans, plants, and animals. For instance, the company recently reported improving existing sequencing information for the maize plant genome, including fixing mistakes, reducing gaps, increasing sequence contiguity up to 50-fold, and adding difficult to reach (centromere region) sequences.

SMRT can be applied to produce in-depth analysis of genetic variations in disease models, using targeted approaches. It provides true long read lengths and highest consensus accuracy available, revealing a full spectrum of genetic variation for microbes and virus, and heterologous cell populations such as escaping cancer cell genomes. PACB SMRT technology offers RNA isoform sequencing functions that can produce full-length transcripts (eliminating the need for assembly, useful in transcriptome analyses). Epigenetic characterization of DNA modifications in prokaryotic and eukaryotic models are also possible.

The company recently announced an agreement by Novogene (China) to purchase ten Sequel systems in addition to ten already purchased earlier this year. To date Novogene is the largest user of PACB Sequel systems, and the reordering of technology indicates customer satisfaction and increased productivity.

Novogene and PACB have agreed to co-market and promote genomic applications. The company cites high demand by Novogene as impetus to double its production capacity to meet the orders, driven by a Chinese precision medicine initiative to sequence variants in 1000 individuals. The company is also participating in other world genome discovery projects.

Net loss for 2Q 2017 was $25.5 million, compared to $18.5 million for the second quarter of 2016. Operating expenses for 2Q 2017 was $32.4 million, compared to $28.7 million for 2Q 2016. The company reported $20.1 million in 2Q 2017 product, service, and other revenue compared to $17.2 million for 2Q, 2016. Cash and cash equivalents at end Q2 2017 was $102.6 million, compared to $72.0 million at December 31, 2016. In June 2017, the company did an offering of approximately 15 million shares at $3.10 per share, raising approximately $46 million.

Strong Bio wants to emphasize that the era of gene therapy is upon us, and several candidates are likely to be approved as potential first gene therapy products approved by FDA later this year. Emerging supportive technologies stand to benefit as the medical system and biotechnology investment centers begin to realize the fruits of such endeavors.

PACB is right in there for the upcoming revolution. To give the reader an idea of the power of its technology on client bottom line, the Sequel system was able to do the maize genomic work at a cost of around $20,000 per maize line, compared to the nearly $30 million in cost for the original maize genome reference.

It is well-positioned in the industry, with its technology being referenced in over 35 presentations at annual AGBT 2017 conference, demonstrating customer value. Over 135 references to PACB technology were made in the 2017 annual PAG conference. The stock has jumped a bit after announcing the sale of 10 Sequel units, but would be attractive on a pullback into the gap to the $3.00 offering range, and is thus watchlisted as an exciting prospect. It is also possible that some consolidation in this space could occur, leading to potential merger and acquisition action. Yahoo consensus target of 4 analysts is $5.95 per share.

Risks for investment in PACB include industry dynamics that the high cost of developing new treatments impedes growth in the gene therapy industry. Moreover, no gene therapy products have yet been approved by the FDA. However, advancements in rare diseases using the gene therapy/genomic approach are coming soon. It is clear that cell therapies that involve genomic research will offset the slow-to-start gene therapy development costs impediment, and serve as a catalyst for profitable efforts to fund gene therapies as they enter the market over the coming months and years.

Another risk for investors is that the company is not yet close to a cash-neutral revenue stream, so further dilution could be possible. There is some competition in the genomics industry as well, but Strong Bio regards SMRT as a front-runner in the space. One could have argued 20 years ago that there is no obvious value in sequencing genomes, but given the breakthroughs that are upon us in the upcoming gene therapy era, the time to place investments is rapidly drawing near. The reward to risk ratio for PACB is compelling.

Disclosure: I/we have no positions in any stocks mentioned, and no plans to initiate any positions within the next 72 hours.

I wrote this article myself, and it expresses my own opinions. I am not receiving compensation for it (other than from Seeking Alpha). I have no business relationship with any company whose stock is mentioned in this article.

Originally posted here:
Pacific Biosciences Is Advancing Genomics - Seeking Alpha

Scientists foresee Russian gene therapy for HIV cure may be registered in 5-10 years – TASS

MOSCOW, August 17. /TASS/. A Russian gene therapy drug for individuals infected with HIV called Dinavir is undergoing pre-clinical trials, and the drug has already proved its efficiency on cells. The pre-clinical tests on animal models, clinical trials and the registration procedure may take up to 10 years, senior research fellow at the Epidemiology Central Research Institute of Rospotrebnadzor (the Federal Service on Surveillance for Customers Rights Protection and Human Well-Being) Dina Glazkova told TASS.

"This is not about the next year, but rather in five years, at the earliest. It takes up to 10 years on the average," she said.

Glazkova reiterated that the registration is made after the clinical trials. "Again, the clinical trials are costly, and the drug production is costly as well," the scientist added.

Dinavir proved to be safe while tested on cells, in vitro. A Phase II pre-clinical trial will utilize animal models to test the efficiency and safety of treatment. A Phase I clinical trial will be carried out on humans to test safety of the therapy and will take up not less than a year.

"Phase II takes up two to three years, and it is unclear how much will be required from us. Phase I is about safety, and it takes a few patients: five, maybe ten. Phase II is when we have to prove that the drug works in these five to ten [patients] and that it had a positive effect on them. Phase III is when we enroll a lot of patients [in the trial] to show that the five were cured not by accident and that it [the gene therapy] really works," Glazkova explained.

The gene therapy for HIV treatment is being developed by a group of researches at the Epidemiology Central Research Institute of Rospotrebnadzor.

See the original post here:
Scientists foresee Russian gene therapy for HIV cure may be registered in 5-10 years - TASS

Why three little virus-free pigs matter – The Messenger (subscription)

We've done some genetics. We've done DNA. We've done GMOs. We've done some immunology. We've done science literacy. Now, let's get down in the weeds a little bit and put all of that together to look at just exactly why virus-free piglets are news.

Aren't piglets just the cutest things? The three particular precious petite porkers in the picture have recently been making the rounds of the national news, usually under a headline that says something like "Scientists create virus-free pigs." This is an excellent example of a headline that really fails to do what a headline is supposed to, which is capture the reader's interest. Virus-free pigs. Big whoop.

If, however, you got past the lame headlines and you've read any of those stories, you know that this is sort of a big deal, on several different levels, and we'll use what we've already talked about in some of my earlier articles about genes, viruses, DNA, genetic engineering, and the value of science literacy to explore why these three little pigs are important.

These pigs are really just like any other pigs, except they were born without any genes in their genomes that code for a number of viruses found in all other pigs, called porcine endogenous retroviruses, or PERVs (yeah, I know. Not that kind of perv). Let's talks about what the term means and what PERVs are. The word "porcine" just means "related to pigs." "Endogenous" means "something that is normally found in or originating from within an organism." For instance, insulin is an endogenous human hormone, because it is produced by the pancreas and is normally present in people. "Retroviruses" are a group of viruses that use RNA (ribonucleic acid) instead of DNA (deoxyribonucleic acid) as their genetic material.

When a virus of any kind infects a cell, the reproductive machinery of the cell, which is normally used to make proteins and copy the DNA of the cell so that the cell can divide and reproduce, is hijacked by the virus. The virus causes the cell to make new copies of the viral genome and then to use the viral genes to make viral proteins. When the cell makes the new viral proteins, they are assembled into new viruses and the new viruses then can go out to infect other cells. Usually, the cell is destroyed in the process. Sometimes, though, the viral genes just get integrated into the genome of the infected host cell and the cell goes on living and reproducing as normal, only now, every time the cell divides, the new cell also has a copy of the viral genes in its DNA.

Sometimes, the viral genes just sit there, causing no

problems. This is called a "latent virus." It may sit there forever doing nothing, or sometimes, something happens to activate the latent virus and the viral genes start to be reproduced, viral proteins start to be made by the infected cells and that may cause disease. The Herpes simplex family of viruses is an example of latent viruses. Other examples of latent viruses are called retroviruses. In a retrovirus, the RNA from the retrovirus is used as a template to make DNA in the infected cell, and then the DNA becomes integrated into the host cell's genome. HIV is an example of a retrovirus that affects humans.

If you look at the genome of almost any organism, particularly complex organisms, like most of us, there is a lot of what is called "non-coding DNA." Non-coding DNA is exactly that -- it doesn't code for any specific proteins. There is some disagreement on whether this non-coding DNA has any function at all, but the amount of it is pretty amazing. Somewhere between 80 percent and 98 percent of the human genome is non-coding. This was a bit of a surprise when the Human Genome Project was going on in the 1990s.

The Human Genome Project was a very ambitious, very wide-ranging effort to identify all the genes in the human genome and map the location where each gene would be found on our chromosomes. The early expectation was that the human genome, based on the amount of DNA it contains (along with human ego), would contain hundreds of thousands of, possibly a million, individual genes. After all, something as marvelous as we are would obviously have the most genes of any creature, right? Wrong. The initial findings of the genome project was that humans have about 30,000 individual genes that code for proteins. This low number was quite the surprise. After all, there is a single-celled protozoan that has over 60,000 genes. There is a plant that has a genome almost three times the size of the human genome. Talk about you rude awakenings! Here we are, thinking how complex and wonderful we are, and there are flowers and pond scum with larger, more complex genomes than ours!

So, what does this have to do with our story today? Well, with the discovery that the vast majority of the human genome, and the genomes of most complex organisms, for that matter, doesn't code for proteins, it begs the question, "then why is it there and where did it come from?" Both of those are good questions that haven't been fully answered, but part of that "extra" DNA is probably DNA that originated long, long ago in our evolutionary history as viral DNA that got integrated into our own genome. The same is likely true for much of the PERV DNA in pigs.

In the case of PERVs, the viruses are found in most of the pig's cells, including the sperm and egg cells used in reproduction. Because they are in the reproductive cells, newborn pigs are already infected with the virus. The PERVs don't normally cause any disease in the pigs, as they usually remains latent in pig cells. The problem with PERVs is that they can be transferred to humans and infect human cells when pig organs or tissues are transplanted into people and PERVs can potentially cause disease in humans.

This is why scientists bothered to try to make virus-free pigs. Pigs have long been used as a source of organs and tissues for transplantation into humans. One reason for this is that the anatomy and physiology of pigs is very similar to that of humans, and so many of their organs and tissues are very similar to those of humans. Pigs and people are also of similar size, so swapping out parts works pretty well because, for instance, a heart valve from a pig is just about the same size as a heart valve from a human. Producing pigs that have tissues free of PERVs is a big step into making pig organs more available and safer for transplantation into humans.

The cute little virus-free piggies in the picture were created using a couple of genetic engineering techniques that are both revolutionary and controversial. The first technique is a new technology called CRISPR (pronounced "crisper"). It stands for "Clustered, Regularly Interspaced Short Palindromic Repeats." I'm not going to go into what all that means. What I will say is that it takes advantage of a genetic mechanism used by bacteria to avoid being infected by viruses. Yes, bacteria can be, and often are, infected by viruses. The CRISPR technology allows scientists to target specific gene sequences in the DNA of a cell, cut it out and replace it with a new gene sequence. CRISPR is hugely valuable in genetic research and has great promise in therapeutic use to treat genetic diseases. Theoretically, CRISPR could be used to cut out defective genes in a patient with a genetic disease and replace the bad gene with a good one. That sort of application is quite a way off. In the case of our pigs, however, CRISPR was used to cut out the genes for all the PERVs found in pig cells that were grown in a dish. The result of that was pig cells that were completely free of PERVs.

The second controversial technique that was used is called "somatic cell nuclear transfer." A somatic cell is just a term for any of the regular, non-reproductive cells found in an organism. In this technique, the nucleus of a cell, where all the genetic material is located, is removed. That nucleus is then transferred into another cell, from which the nucleus has also been removed, essentially turning the recipient cell into a genetic copy of the donor cell. If the recipient cell is a reproductive cell, like an egg cell, and the egg is fertilized, the genes contained in the donor cell will be present in all the cells that develop from the fertilized egg. In this case, the nuclei from the cells grown in the dish that were modified to be PERV-free were injected into fertilized pig eggs. The eggs were then implanted into surrogate mother pigs and they developed into our piglets.

This technology has incredible potential in transplant therapeutics, as well as gene therapy to correct some horrible diseases. Somatic cell nuclear transfer also has another name -- cloning. Dolly the Sheep, if you remember her, was the first mammal to be produced through cloning. The term "cloning" brings up all sorts of late-night horror movie terrors and visions of genetically engineered babies and so on. In reality, cloning is not fearsome or evil. It is just a fairly simple, very powerful tool in the field of genetic research.

However, here is where the science literacy part of the story comes into play. These techniques were used, in this case, as a step toward improving options for transplanted organs and tissues. It is theoretically possible, however, that these same techniques could be used for less clearly beneficial ends. It could, for instance, be further developed and adapted to be used to modify human embryos to create "designer babies." Clearly, this is an issue with profound bioethical considerations. It is important that we, as a human society, understand this science, and that includes you.

This technology, like other forms of genetic engineering, stem cell-based therapeutics, artificial intelligence, GMOs, and other equally powerful, potentially transformative science, could be hugely valuable in improving the human condition if used properly, but the consequences of abuse of the technology are also huge. We must be part of a well-informed populace to make reasoned, rational decisions on how we want our science to be used.

Already, the scientific communities of the U.S., UK, China, and others have set strict guidelines on what types of research along the lines of that which produced our virus-free pigs is permissible, but as science moves forward, there will need to be more discussion. The benefits and consequences of these technologies are so huge that we must discuss them from a position of knowledge and understanding, not from one of fear, ignorance and emotion. This is why it is so vitally important for everyone to be scientifically literate.

Michael J. Howard, Ph.D., is the vice president fo education and research at Baptist Health Madisonville. He can be reached by email at or via Twitter at @madville_sci.

View original post here:
Why three little virus-free pigs matter - The Messenger (subscription)

Listening for the Public Voice – Slate Magazine


On Aug. 3, the scientific article in Nature finally gave us some facts about the much-hyped experiments that involved editing the genomes of human embryos at the Center for Embryonic Cell and Gene Therapy at Oregon Health and Science University. The story had broken in late July in Technology Review, spurring profuse hand-wringing and discussion. But until we saw the scientific paper, it was not clear what cells and methods were used, what genes were edited, or what the results were.

Now we know more, and while the paper demonstrates the possibility of genome editing of human embryos, it raises more questions than it answers. It is a useful demonstration of technical promise, though not an immediate prelude to the birth of a genome-edited baby. But the process by which the news emerged is also an ominous harbinger of the discombobulated way the debate about genetically altering human embryos is likely to unfold. We need open, vigorous debate that captures the many, often contradictory, moral views of Americans. Yet what we are likely to get is piecemeal, fragmented stories of breakthroughs with incomplete details, more sober publication in science journals that appear later, news commentary that lasts a few days, and very little systematic effort to think through what policy should be.

The science underlying this news cycle about human genome editing builds on a technique first developed six years ago by studying how bacteria alter DNA. CRISPR genome editing is the most recent, and most promising, way to introduce changes into DNA. It is faster, easier, and cheaper than previous methods and should eventually be more precise and controllablewhich is why it may one day be available for clinical use in people.

Though headlines about the study discussed designer babies, researchers prefer to emphasize how these techniques could help stop devastating genetic disorders. The Oregon experiments with human embryo cells corrected disease-associated DNA variants associated with heart muscle wasting that can cause heart failure. The treated embryos were alive for only a few days and were never intended to become a human baby. They were, however, human embryos deliberately created for the research.

U.S. guidance in this area is sparse and reflects the lack of societal consensus. In 1994, when the federal government was contemplating funding for research involving human embryos, the NIH Embryo Research Panel concluded that just this kind of experiment was ethically appropriate. But within hours of that reports release, then-President Bill Clinton announced he did not agree with creating embryos in order to do research on them.

The United States currently has just two policies relevant to genomic editing of human embryos. The first blocks federal funding: On April 28, 2015, Francis Collins, director of the National Institutes of Health, stated, NIH will not fund any use of gene-editing technologies in human embryos. This is not embedded in statute or formal executive order, but members of Congress are fully aware of it and it is, in effect, a federal policy. NIH can (and does) fund genome editing of nonembryonic cells that might be used to treat cancer and for other possible therapeutic purposes, but not embryonic cells that would have their effect by creating humans with germline alterations.

Second, Congress has prohibited the Food and Drug Administration from reviewing research in which a human embryo is intentionally created or modified to include a heritable genetic modification. This language comes from a rider to FDAs annual appropriations. Yet use of human embryonic cells for treatment should be subject to FDA regulation. So this language in effect means alterations of embryonic cells cannot be done in the United States if there is any intent to treat a human being, including implantation of an altered embryo into a womans uterus. This will remain true so long as the rider is included in FDAs annual appropriations. The federal government thus has two relevant policies, both of which take federal agencies out of the action: One removes NIH funding, and the other precludes FDA oversight of genome-edited human embryos.

This leaves privately funded research that has no direct therapeutic purpose, such as with the Oregon experiments. The funding came from OHSU itself; South Korean Basic Research Funds; the municipal government of Shenzhen, China; and several private philanthropies (Chapman, Mathers, Helmsley, and Moxie). The research complies with recommendations to study the basic cellular processes of genome editing, keeping an eye on possible future clinical use but only so long as the work does not attempt to create a human pregnancy.

By coincidence, on the same day the Nature paper came out, the American Journal of Human Genetics also published a thoughtful 10-page position statement about germline genome editing from the American Society for Human Genetics endorsed by many other genetic and reproductive medicine organizations from all over the world. It reviews recommendations of the National Academies of Sciences, Engineering, and Medicine, several international and U.S.-based organizations and commissions, and makes several recommendations of its own, concluding it is inappropriate to perform germline gene editing that culminates in human pregnancy, but also there is no reason to prohibit in vitro germline genome editing on human embryos and gametes, with appropriate oversight and consent from donors, to facilitate research on the possible future clinical applications. Indeed, the statement argues for public funding. Finally, it urges research to proceed only with compelling medical rationale, strong oversight, and a transparent public process to solicit and incorporate stakeholder input.

So is there a problem here? It is truly wonderful that medical and scientific organizations have addressed genome editing. It is, however, far from sufficient. Reports and scientific consensus statements inform the policy debate but cannot resolve it. All of the reports on genome editing call for robust public debate, but the simple fact is that embryo research has proven highly divisive and resistant to consensus, and it is far from clear how to know when there is enough thoughtful deliberation to make policy choices. Its significant that none of the reports have emerged from a process that embodied such engagement. The Catholic Church, evangelical Christians, and concerned civic action groups who view embryo research as immoral are not likely to turn to the National Academies of Sciences, Engineering and Medicine, the American Society for Human Genetics, the Hinxton Group, the Nuffield Council on Bioetics, or other scientific and medical organizations for their primary counsel. They may well listen to scientists, but religious and moral doctrine will get greater weight. Yet religious groups highly critical of embryo research are part of the political systemand whether we embrace this sort of genome editing in the United States is a political question, not a purely technical one.

Reports and scientific consensus statements inform the policy debate but cannot resolveit.

Addressing the political questions will be extremely difficult. The U.S. government is poorly positioned to mediate the policy debate in a way that recognizes and addresses our complex moral pluralism. NIH and FDA are two of the most crucial agencies, but current policies remove them from line authority, and with good reason, given that engaging in this debate could actually endanger the agencies other vital missions. International consensus about genome editing of human embryos remains no more likely than about embryo research in general: Some countries ban it while others actively promote and fund it. Private foundations dont have the mandate or incentive to mediate political debate about a controversial technology that rouses the politics of abortion. What private philanthropic organization would willingly take on such a thankless and politically perilous task, and what organization would be credible to the full range of constituencies?

So who can carry out the public engagement that everyone seems to agree we need? The likely answer is no one. This problem occurs with all debate about fraught scientific and technical innovations, but its particularly acute when it touches on highly ossified abortion politics.

The debate about genomic editing of human embryos is unlikely to follow the recommendations for systematic forethought proposed by illustrious research bodies and reports. Given the reactions weve seen to human embryonic stem-cell research in the past two decades, we have ample reason for pessimism. Rather, debate is more likely to progress by reaction to events as researchers make newsoften with the same lack of information we lived with for the last week of July, based on incomplete media accounts and quotes from disparate experts who lacked access to the details. Most of the debate will be quote-to-quote combat in the public media, leavened by news and analysis in scientific and medical journals, but surrounded by controversy in religious and political media. It is not what anyone designing a system would want. But the recommendations for robust public engagement and debate feel a bit vacuous and vague, aspirations untethered to a concrete framework.

Our divisive political system seems fated to make decisions about genomic editing of human embryos mainly amidst conflict, with experts dueling in the public media rather than through a thoughtful and well-informed debate conducted in a credible framework. As the furor over the Oregon experiments begins to dissipate, we await the event that will cause the next flare-up. And so it will continue, skipping from news cycle to news cycle.

History shows that sometimes technical advances settle the issues, at least for most people and in defined contexts. Furor about in vitro fertilization after Louise Brown, the first test tube baby, was born in 1978 gave way to acceptance as grateful parents gave birth to more and more healthy babies and welcomed them into their families. Initial revulsion at heart transplants gave way in the face of success. Anger about prospects for human embryonic stem-cell research might similarly attenuate if practical applications emerge.

Such historical examples show precisely why reflective deliberation remains essential, despite its unlikely success. Momentum tends to carry the research forward. Yet at times we should stop, learn more, and decide actively rather than passively whether to proceed, when, how, and with what outcomes in mind. In the case of genome editing of human embryos, however, it seems likely that technology will make the next move.

This article is part of Future Tense, a collaboration among Arizona State University, New America, and Slate. Future Tense explores the ways emerging technologies affect society, policy, and culture. To read more, follow us on Twitter and sign up for our weekly newsletter.

Read more from the original source:
Listening for the Public Voice - Slate Magazine

Cancer Gene Therapy Market – Forecasts and Opportunity Assessment by Technavio – Business Wire (press release)

LONDON--(BUSINESS WIRE)--According to the latest market study released by Technavio, the global cancer gene therapy market is expected to grow at a CAGR of almost 21% during the forecast period.

This research report titled Global Cancer Gene Therapy Market 2017-2021 provides an in-depth analysis of the market in terms of revenue and emerging market trends. This market research report also includes up to date analysis and forecasts for various market segments and all geographical regions.

The rising prevalence rate of cancer has been a huge challenge for the global economies as the disease leads to high rate of mortality and economic losses. The current treatment options available come with many drawbacks such as severe side effects and relapse of cancer. These factors have led to high investment in the R&D for development of various novel therapies with cancer gene therapy being one of the major ones of them. The therapy mainly uses three types of treatment options namely oncolytic virotherapy, gene transfer therapy, and gene-induced immunotherapy.

This report is available at a USD 1,000 discount for a limited time only: View market snapshot before purchasing

Buy 1 Technavio report and get the second for 50% off. Buy 2 Technavio reports and get the third for free.

Technavios healthcare and life sciences research analysts categorize the global cancer gene therapy market into the following segments by therapy. They are:

Looking for more information on this market? Request a free sample report

Technavios sample reports are free of charge and contain multiple sections of the report including the market size and forecast, drivers, challenges, trends, and more.

Oncolytic virotherapy

Oncolytic virotherapy is one of the fastest growing treatment modality. In this therapy, the anti-cancer cells specifically destroy the cancer cells without causing harm to the normal cells. Each virus has a specific cellular tropism that determines which tissue will be preferentially infected by the virus and thus will further lead to the disease.

According to Sapna Jha, a lead oncology research analyst from Technavio, The oncolytic virotherapy has shown encouraging results in the pre-clinical studies. The novel treatment option holds great opportunity to make a significant effect on quality and length of the life of the individual. Adenovirus is the most commonly used virus in oncolytic virotherapy.

Gene transfer

Gene transfer or gene insertion is one of the most exciting and emerging cancer treatment methods. The therapy is expected to be the fastest growing type of therapy in the cancer gene therapy market. This is a radical new treatment method that involves the introduction of a new gene into the cancer cell or the surrounding tissues.

Genes with different functions have been proposed for this therapy; some of them include antiangiogenesis genes, cellular stasis genes, and suicide genes. Many different viral vectors are used to deliver these genes, Adenovirus being most common of them. Other than viral vectors, certain non-viral methods are also studied in the various clinical trial, which includes oligodendromer DNA coatings and naked DNA transfer, adds Sapna.

Gene-induced immunotherapy

Immunotherapy works on the concept of boosting the immune system of the individual to target and destroy cancer cells. However, traditional immunotherapy has shown limited success rate in the field. Various gene therapy techniques are being used to overcome this limitation.

The next-generation gene-induced immunotherapy vaccines are already in clinical trial. Gene-induced immunotherapy is a type of gene therapy where genetically engineered genes are used to generate an immune response against cancer. Growing knowledge and understanding of mechanisms regulating the initiation and maintenance of cytotoxic immune response has led to the designing of several genetic immunization strategies.

The top vendors highlighted by Technavios research analysts in this report are:

Browse Related Reports:

About Technavio

Technavio is a leading global technology research and advisory company. Their research and analysis focuses on emerging market trends and provides actionable insights to help businesses identify market opportunities and develop effective strategies to optimize their market positions.

With over 500 specialized analysts, Technavios report library consists of more than 10,000 reports and counting, covering 800 technologies, spanning across 50 countries. Their client base consists of enterprises of all sizes, including more than 100 Fortune 500 companies. This growing client base relies on Technavios comprehensive coverage, extensive research, and actionable market insights to identify opportunities in existing and potential markets and assess their competitive positions within changing market scenarios.

If you are interested in more information, please contact our media team at

Go here to see the original:
Cancer Gene Therapy Market - Forecasts and Opportunity Assessment by Technavio - Business Wire (press release)

Gene Therapy | Pfizer: One of the world’s premier …

Gene therapy is a technology aimed at correcting or fixing a gene that may be defective. This exciting and potentially transformative area of research is focused on the development of potential treatments for monogenic diseases, or diseases that are caused by a defect in one gene.

The technology involves the introduction of genetic material (DNA or RNA) into the body, often through delivering a corrected copy of a gene to a patients cells to compensate for a defective one, using a viral vector.

The technology involves the introduction of genetic material (DNA or RNA) into the body, often through delivering a corrected copy of a gene to a patients cells to compensate for a defective one, using a viral vector.

Viral vectors can be developed using adeno-associated virus (AAV), a naturally occurring virus which has been adapted for gene therapy use. Its ability to deliver genetic material to a wide range of tissues makes AAV vectors useful for transferring therapeutic genes into target cells. Gene therapy research holds tremendous promise in leading to the possible development of highly-specialized, potentially one-time delivery treatments for patients suffering from rare, monogenic diseases.

Gene therapy research holdstremendous promise

Pfizer aims to build an industry-leading gene therapy platform with a strategy focused on establishing a transformational portfolio through in-house capabilities, and enhancing those capabilities through strategic collaborations, as well as potential licensing and M&A activities.

We're working to access the most effective vector designs available to build a robust clinical stage portfolio, and employing a scalable manufacturing approach, proprietary cell lines and sophisticated analytics to support clinical development.

In addition, we're collaborating with some of the foremost experts in this field, through collaborations with Spark Therapeutics, Inc., on a potentially transformative gene therapy treatment for hemophilia B, which received Breakthrough Therapy designation from the US Food and Drug Administration, and 4D Molecular Therapeutics to discover and develop targeted next-generation AAV vectors for cardiac disease.

Gene therapy holds the promise of bringing true disease modification for patients suffering from devastating diseases, a promise were working to seeing become a reality in the years to come.

Continue reading here:
Gene Therapy | Pfizer: One of the world's premier ...