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Category Archives: Gene Therapy

When Will Gene Therapy Come to the U.S.?

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Several gene therapies are or will soon be in late-stage human trials. One of them could be the first to get FDA approval for sale in the U.S.

Though many gene therapies have been tested in patients around the world in hopes of curing hereditary diseases, few governments have approved their sale, and none has been approved in the United States. That could change in coming years as several therapies enter advanced trials.

A big step forward already came in November 2012, when the European Medicines Agency gave the Dutch biotech startup UniQure permission to sell its treatment. That approval came as a relief to many in the field, who had been waiting for a break in the clouds hanging over the technology since failed and fatal trials in the 1990s. You see a resurgence in terms of investors, and in truth, a number of problems have been solved, says Katherine High, a medical researcher at Childrens Hospital of Philadelphia, who is overseeing a late-stage clinical trial for a different gene therapy.

Still, experts say it is likely to be a few years before a treatment is approved in the U.S. With its European approval in hand, UniQure may have good chance of also getting the first U.S. approval, but the company says it has not yet submitted an application to the FDA.

Like most gene therapies, UniQures treatment uses a modified virus to deliver a working copy of a gene to patients who lack a healthy version. In this case, the gene is needed for the body to break down fats; without it, patients can develop painful and even fatal inflammation of the pancreas. UniQure uses a modified version of a virus that most of us already carry. The choice of virus used to deliver a gene therapy depends in part on where the treatment needs to go in the body and whether the viruses are intended to replicate themselves. Some viruses, for instance, are designed to spread throughout the body to kill cancer cells.

There are several groups that could be the first to develop a U.S.-approved gene therapy (see table). Highs team is one; they are enrolling patients in a late-stage trial of a treatment for a disorder that causes blindness at an early age. The patients in this trial have previously been given the gene therapy in one eye, and now the other will be tested.

In the experimental treatment, doctors inject a virus-borne gene just behind a patients retina. The treatment improved some patients vision to the point that they were no longer legally blind. Some patients have been stable for nearly six years. The trial is scheduled to end in April 2015.

Another possibility comes from Massachusetts-based Bluebird Bio, which has published results from patients who have seemingly been cured of a genetic blood disease (see Gene Therapy Combats Hereditary Blood Disease). The company is about to start testing its approach in a hereditary neurological disorder that is often fatal in young boys.

In a different form, gene therapy could also become an option for cancer treatment. At a meeting this summer, Amgen announced that it had met its goals for an advanced test of a gene therapy for melanoma that has spread from the skin to other parts of the body. The Amgen treatment, which was engineered from a virus that normally causes cold sores, takes a two-pronged approach to fighting cancer. The virus selectively infects cancer cells, where it replicates until the cell bursts. While growing inside the cell, the virus also produces a protein that rouses the immune system. When the cell explodes, immune cells are attracted to the tumor site to fight the disease.

In a test in patients with late-stage melanoma, 26 percent of patients whose cancer had spread saw a partial or complete tumor response for at least six months. In 11 percent of patients, the cancer completely disappeared, which suggests that the therapy spreads throughout the body, targeting tumors that werent initially injected. Overall survival rates for cancer patients in the trials are expected to be reported in the first half of 2014.

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When Will Gene Therapy Come to the U.S.?

When Will Gene Therapy Come to the United States?

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Susan Young for MIT Technology Review 2013-09-30 14:11:37 UTC

Though many gene therapies have been tested in patients around the world in hopes of curing hereditary diseases, few governments have approved their sale, and none has been approved in the United States. That could change in coming years as several therapies enter advanced trials.

A big step forward already came in November 2012, when the European Medicines Agency gave the Dutch biotech startup UniQure permission to sell its treatment. That approval came as a relief to many in the field, who had been waiting for a break in the clouds hanging over the technology since failed and fatal trials in the 1990s. You see a resurgence in terms of investors, and in truth, a number of problems have been solved, says Katherine High, a medical researcher at Childrens Hospital of Philadelphia, who is overseeing a late-stage clinical trial for a different gene therapy.

Still, experts say it is likely to be a few years before a treatment is approved in the U.S. With its European approval in hand, UniQure may have a good chance of also getting the first U.S. approval, but the company says it has not yet submitted an application to the FDA.

Like most gene therapies, UniQures treatment uses a modified virus to deliver a working copy of a gene to patients who lack a healthy version. In this case, the gene is needed for the body to break down fats; without it, patients can develop painful and even fatal inflammation of the pancreas. UniQure uses a modified version of a virus that most of us already carry. The choice of virus used to deliver a gene therapy depends in part on where the treatment needs to go in the body and whether the viruses are intended to replicate themselves. Some viruses, for instance, are designed to spread throughout the body to kill cancer cells.

There are several groups that could be the first to develop a U.S.-approved gene therapy (see table). Highs team is one; they are enrolling patients in a late-stage trial of a treatment for a disorder that causes blindness at an early age. The patients in this trial have previously been given the gene therapy in one eye, and now the other will be tested.

In the experimental treatment, doctors inject a virus-based particle just behind a patients retina. The treatment improved some patients vision to the point that they were no longer legally blind. Some patients have been stable for nearly six years. The trial is scheduled to end in April 2015.

Another possibility comes from Massachusetts-based Bluebird Bio, which has published results from patients who have seemingly been cured of a genetic blood disease. The company is about to start testing its approach in a hereditary neurological disorder that is often fatal in young boys.

SEE ALSO: Gene Therapy Combats Hereditary Blood Disease

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When Will Gene Therapy Come to the United States?

Gene therapy relieves pain in dogs

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A revolutionary gene therapy developed in Colorado could relieve people's chronic pain, such as arthritis or fibromyalgia but first, it is being tested on pets with incredible results.

A Lafayette veterinarian is using cutting-edge research to heal dogs.

It may not look like it when 9-year-old Amos, a Labrador Retriever mix, is running happily after the ball, but he has arthritis, bad.

His owner, Vicki Riedel knew immediately.

"He is always by my side," said Riedel. "When I would go outside or downstairs,and he wouldn't come with me, I knew he was really hurting."

Their vet tried one medication after the next.

"And they all helped a little bit, but none of them really helped a lot, and he seemed to be getting worse and worse," said Riedel.

But a few weeks ago, Amos met pet pain specialist Dr. Rob Landry.

"Amos has a history of chronic pain in both his elbows," said Landry, a Lafayette veterinarian who has been testing a breakthrough gene therapy for chronic pain for the last two years.

"It's amazing," said Laundry with a smile.

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Gene therapy relieves pain in dogs

First Human Application of SDF1 Gene Therapy to promote healing of open heart surgery. – Video

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First Human Application of SDF1 Gene Therapy to promote healing of open heart surgery.
September 12, 2012 Stewart Manning was the first person in the world to receive SDF1 Gene Therapy. It was used to promote healing after Dr. Amit Patel MD, MS...

By: Schmocter MED

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First Human Application of SDF1 Gene Therapy to promote healing of open heart surgery. - Video

Vincent Mauro – Codon optimization new safety concerns for gene therapy and genetic vaccines – Video

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Vincent Mauro - Codon optimization new safety concerns for gene therapy and genetic vaccines
Codon-optimization describes gene engineering approaches that use synonymous codon changes to increase protein production. It is used extensively for express...

By: LabRoots

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Vincent Mauro - Codon optimization new safety concerns for gene therapy and genetic vaccines - Video

$1.5 Million Gift To Penn Medicine Establishes Associate Professorship In Cancer Gene Therapy

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PHILADELPHIA A $1.5 million gift to the University of Pennsylvania has established the Barbara and Edward Netter Associate Professorship in Cancer Gene Therapy at the Abramson Cancer Center.Bruce Levine, PhD, a faculty member in the department of Pathology and Laboratory Medicine in the Perelman School of Medicine and the director of the Clinical Cell and Vaccine Production Facility, has been appointed to this new associate professorship.

Barbara Netter and her late husband, Penn alumnus Edward Netter (C53), have supported Penn since the early 1980s. Their contributions created the Netter Center for Community Partnerships on campus, and they have been longtime champions for research to advance gene therapy, having founded the Alliance for Cancer Gene Therapy in 2001 following the loss of their daughter-in-law to breast cancer. The foundation has provided funding to numerous Penn Medicine scientists, including the team Levine is part of, led by Carl June, MD, that has conducted trials demonstrating the first successful and sustained demonstration of the use of gene transfer therapy to turn the bodys own immune cells into weapons aimed at cancerous tumors. This new personal gift from Mrs. Netter will further this breakthrough research.

Dr. Levine is an alumnus of the University of Pennsylvania (C84), where he earned a bachelors degree in biology. He began his scientific career at the Wistar Institute during summers in high school and as an undergraduate at Penn, and in the division of Infectious Diseases at the Children's Hospital of Philadelphia examining immune responses following Varicella vaccination. Dr. Levine received his PhD in Immunology and Infectious Diseases from the Johns Hopkins University in 1992, and served as a post-doctoral fellow with Dr. Carl June at the Naval Medical Research Institute in Bethesda, MD, and later as an Investigator at NMRI and a Research Assistant Professor of Medicine at the Uniformed Services University for the Health Sciences. He joined returned to Philadelphia to join the Penn Medicine faculty in 1999. The Clinical Cell and Vaccine Production Facility at the Hospital of the University of Pennsylvania, which Dr. Levine directs, is charged with developing, manufacturing, and testing novel cell, gene, and biologic therapies being investigated in clinical trials at Penn, the Childrens Hospital of Philadelphia, and collaborating institutions.

This appointment and professorship establishment will be celebrated by Penn Medicine in January 2014.

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Penn Medicine is one of the world's leading academic medical centers, dedicated to the related missions of medical education, biomedical research, and excellence in patient care. Penn Medicine consists of the Raymond and Ruth Perelman School of Medicine at the University of Pennsylvania (founded in 1765 as the nation's first medical school) and the University of Pennsylvania Health System, which together form a $4.3 billion enterprise.

The Perelman School of Medicine has been ranked among the top five medical schools in the United States for the past 16 years, according to U.S. News & World Report's survey of research-oriented medical schools. The School is consistently among the nation's top recipients of funding from the National Institutes of Health, with $398 million awarded in the 2012 fiscal year.

The University of Pennsylvania Health System's patient care facilities include: The Hospital of the University of Pennsylvania -- recognized as one of the nation's top "Honor Roll" hospitals by U.S. News & World Report; Penn Presbyterian Medical Center; and Pennsylvania Hospital -- the nation's first hospital, founded in 1751. Penn Medicine also includes additional patient care facilities and services throughout the Philadelphia region.

Penn Medicine is committed to improving lives and health through a variety of community-based programs and activities. In fiscal year 2012, Penn Medicine provided $827 million to benefit our community.

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$1.5 Million Gift To Penn Medicine Establishes Associate Professorship In Cancer Gene Therapy

Heart gene therapy trial begins

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5 September 2013 Last updated at 13:15 ET

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Carol Gedda: 'It could improve the heart muscle. I'm really pleased to be part of it.'

It is 18 months since Carol Gedda suffered a massive heart attack. It left her with just 20% of her heart functioning.

"I have a lot of trouble with stairs, and sometimes I can even run out of breath in a conversation", says Mrs Gedda, who is 65.

She is one of at least 750,000 people in the UK with heart failure. It occurs when the heart is damaged and becomes unable to pump blood adequately.

There are treatments for the condition but nothing so far that can reverse the damage.

Mrs Gedda, from Essex, is among 200 patients being enrolled on a gene therapy trial to test whether introducing genetic material into damaged heart cells can improve their function.

Researchers at Imperial College London found that levels of the protein SERCA2a are lower in patients with heart failure.

Royal Brompton Hospital in London, where Mrs Gedda is being treated, is one of only two British centres taking part in the international study, The Golden Jubilee National Hospital in Glasgow is also involved.

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Heart gene therapy trial begins

A Man and His Virus: Gene Therapy Emerges From Disgrace to Be the Next Big Thing, Again

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In 1980, though, he opened up the journal Science and suddenly understood how doctors might someday cure Lesch-Nyhan, along with thousands of other genetic disorders that had once seemed incurable. Two Stanford biologists, Richard Mulligan and Paul Berg, had figured out a way to transplant genes into cells, effectively rewriting their DNA. The phrase gene therapy had been floating around medical circles for decades, but Wilson realized that its time had come. As soon as he finished his degrees, he and his wife moved to Boston so he could learn about gene transplantation from Mulligan, now at MIT. After nearly three years under Mulligans tutelage, he headed back to Michigan to set up his own lab.

The first disease that Wilson targeted was called familial hypercholesterolemia, in which the patient lacks the gene that produces receptors for grabbing bad cholesterol, or LDL, from the blood, which the liver normally filters out. Vessels become so badly clogged that many sufferers have heart attacks in their forties and fifties, and sometimes even before age 30.

Wilson figured out how to make a vector to attack the conditiona virus with a working version of the gene loaded on it. He first tested it on a type of rabbit genetically prone to high levels of LDL, and the gene therapy lowered those levels considerably. For a human trial in 1992, he and his colleagues chose a 28-year-old woman from Canada. Surgeons removed part of her liver, and then Wilson and his colleagues infected its cells with the virus, which delivered a working version of the defective gene. Finally, Wilson and his colleagues injected those cells back into the womans liver, where they took hold and grew. The womans LDL levels dropped by 23 percent.

The result, published in 1994, was a milestone in the young field. Gene Experiment to Reverse Inherited Disease is Working, The New York Times reported, noting that Wilsons paper was the first to report any therapeutic benefits of human gene therapy. Thanks to this study and others, the FDA gave the green light to more clinical trials every year, jumping from zero in 1989 to 91 in 1999. Universities set up gene therapy programs to stake a claim in the new field.

One of those was the Institute for Human Gene Therapy at the University of Pennsylvania. At age 38, Wilson became the institutes head, overseeing a staff that soon grew to more than 200. They launched new clinical trials, including a sequel to Wilsons study on familial hypercholesterolemia and on another genetic disorder in the liver: OTCD. Wilson now wanted to take the surgery out of gene therapy, so he and his colleagues searched the scientific literature for a virus that could seek out liver cells in the body.

They settled on a virus known as an adenovirus. Adenoviruses are best known for causing the common cold, but other scientists had found that they were very good at delivering genes into cells. Everything seemed to be moving forward nicelyuntil Jesse Gelsinger checked into Childrens Hospital of Philadelphia.

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A Man and His Virus: Gene Therapy Emerges From Disgrace to Be the Next Big Thing, Again

Research and Markets: Gene Therapy in Oncology Drug Pipeline Update 2013

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DUBLIN--(BUSINESS WIRE)--

Research and Markets (http://www.researchandmarkets.com/research/l9rw32/gene_therapy_in) has announced the addition of the "Gene Therapy in Oncology Drug Pipeline Update 2013" report to their offering.

There are today 120 companies plus partners developing 108 gene therapy drugs in 263 developmental projects in cancer. In addition, there are 4 suspended drugs and the accumulated number of ceased drugs over the last years amount to another 96 drugs. Gene Therapy In Oncology Drug Pipeline Update lists all drugs and gives you a progress analysis on each one of them. Identified drugs are linked to 102 different targets. All included targets have been cross-referenced for the presence of mutations associated with human cancer. To date 92 out of the 92 studied drug targets so far have been recorded with somatic mutations. The software application lets you narrow in on these mutations and links out to the mutational analysis for each of the drug targets for detailed information. All drugs targets are further categorized on in the software application by 44 classifications of molecular function and with pathway referrals to BioCarta, KEGG, NCI-Nature and NetPath.

Reasons To Buy

- Show investors/board/management that you are right on top of drug development progress in your therapeutic area.

- Find competitors, collaborations partners, M&A candidates etc.

- Jump start competitive drug intelligence operations

- Excellent starting point for world wide benchmarking

- Compare portfolio and therapy focus with your peers

- Speed up pro-active in-/out licensing strategy work

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Research and Markets: Gene Therapy in Oncology Drug Pipeline Update 2013

Research and Markets: Gene Therapy – Technologies, Markets and Companies – Updated 2013 Report with 180 Company Profiles

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DUBLIN--(BUSINESS WIRE)--

Research and Markets (http://www.researchandmarkets.com/research/g2hdqj/gene_therapy) has announced the addition of Jain PharmaBiotech's new report "Gene Therapy - Technologies, Markets and Companies" to their offering.

Gene therapy can be broadly defined as the transfer of defined genetic material to specific target cells of a patient for the ultimate purpose of preventing or altering a particular disease state. Genes and DNA are now being introduced without the use of vectors and various techniques are being used to modify the function of genes in vivo without gene transfer. If one adds to this the cell therapy particularly with use of genetically modified cells, the scope of gene therapy becomes much broader. Gene therapy can now combined with antisense techniques such as RNA interference (RNAi), further increasing the therapeutic applications. This report takes broad overview of gene therapy and is the most up-to-date presentation from the author on this topic built-up from a series of gene therapy report written by him during the past decade including a textbook of gene therapy and a book on gene therapy companies. This report describes the setbacks of gene therapy and renewed interest in the topic

Profiles of 180 companies involved in developing gene therapy are presented along with 202 collaborations. There were only 44 companies involved in this area in 1995. In spite of some failures and mergers, the number of companies has increased more than 4-fold within a decade. These companies have been followed up since they were the topic of a book on gene therapy companies by the author of this report. John Wiley & Sons published the book in 2000 and from 2001 to 2003, updated versions of these companies (approximately 160 at mid-2003) were available on Wiley's web site. Since that free service was discontinued and the rights reverted to the author, this report remains the only authorized continuously updated version on gene therapy companies.

Benefits of this report

- Up-to-date on-stop information on gene therapy with 73 tables and 15 figures

- Evaluation of gene therapy technologies

- 740 selected references from the literature

- Estimates of gene therapy markets from 2012-2022

- Profiles of 180 companies involved and collaborations in this area

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Research and Markets: Gene Therapy - Technologies, Markets and Companies - Updated 2013 Report with 180 Company Profiles

Gene Therapy Partnering Deals & Agreements By Healthcare Companies Analyzed in New Research Report at ReportsnReports …

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Dallas, Texas (PRWEB) July 07, 2013

The Gene Therapy Partnering Agreements report provides an understanding and access to the gene therapy partnering deals and agreements entered into by the world's leading healthcare companies:

Trends in gene therapy partnering deals Disclosed headlines, upfronts, milestones and royalties by stage of development Gene therapy partnering contract documents Top gene therapy deals by value

The report provides a detailed understanding and analysis of how and why companies enter gene therapy partnering deals (http://www.reportsnreports.com/reports/262461-gene-therapy-partnering-terms-and-agreements.html). The majority of deals are early development stage whereby the licensee obtains a right or an option right to license the licensors gene therapytechnology 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.

Understanding the flexibility of a prospective partners negotiated deals terms provides critical insight into the negotiation process in terms of what you can expect to achieve during the negotiation of terms. Whilst many smaller companies will be seeking details of the payments clauses, the devil is in the detail in terms of how payments are triggered contract documents provide this insight where press releases and databases do not.

This report contains a comprehensive listing of all gene therapy partnering deals announced since 2007 including financial terms where available including over 300 links to online deal records of actual gene therapy partnering deals as disclosed by the deal parties. In addition, where available, records include contract documents as submitted to the Securities Exchange Commission by companies and their partners.

Contract documents provide the answers to numerous questions about a prospective partners flexibility on a wide range of important issues, many of which will have a significant impact on each partys ability to derive value from the deal.

For example, analyzing actual company deals and agreements allows assessment of the following: What is actually granted by the agreement to the partner company? What exclusivity is granted? What are the precise rights granted or optioned? What is the payment structure for the deal? How are sales and payments audited? What is the deal term? How are the key terms of the agreement defined? How are IPRs handled and owned? Who is responsible for commercialization? Who is responsible for development, supply, and manufacture? How is confidentiality and publication managed? How are disputes to be resolved? Under what conditions can the deal be terminated? What happens when there is a change of ownership? What sublicensing and subcontracting provisions have been agreed? Which boilerplate clauses does the company insist upon? Which boilerplate clauses appear to differ from partner to partner or deal type to deal type? Which jurisdiction does the company insist upon for agreement law?

The report also includes numerous tables and figures that illustrate the trends and activities in gene therapy partnering and dealmaking since 2007.

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Gene Therapy Partnering Deals & Agreements By Healthcare Companies Analyzed in New Research Report at ReportsnReports ...


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