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Category Archives: Gene Therapy

New Gene Therapy Treatment Helps Fight Prostate Cancer, Asons Solicitors Comment

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Bolton, UK (PRWEB UK) 29 October 2013

According to the BBC, the University Hospital in Birmingham are trialing a new gene therapy hospital treatment, aiding in the fight against prostate cancer (1). The pioneering treatment could help prevent the incidence of surgical errors, increasing the success rate associated with treating the disease.

It is believed, that if the treatment works, surgery for prostate cancer will become a thing of the past, eliminating the surgical errors associated with the procedure.

According to the BBC, the University Hospital in Birmingham is among the first to trial the new treatment, stimulating the bodys own immune system to attack the tumor (1). With studies on mice showing complete eradication of the cancer, hopes are high for the imminent human trials.

A new form of gene therapy, the treatment requires a virus to be injected directly into the prostate cancer tumour; a gene attached to the virus (GM-CSF,) is then released, activating the body's own immune system, attracting white blood cells to attack the cancer (1). As reported by the BBC, the virus also carries an enzyme - nitroreductase - which sits inside the cancer cell.

Two days after the injection, the patient in question will be put on a drip, which contains a cancer drug (CB1954), which is initially inactive. When the drug comes into contact with the enzyme, it reacts and starts killing the cancer cells. The inactive drug, CB1954, does not harm cells which do not have the enzyme inside, protecting the healthy tissue surrounding the tumour (1).

Urology Specialist, Prashant Patel, is hopeful that gene therapy could provide real hope for patients who are running out of treatment options (1).

"I have to stress that this is only a phase one safety trial to test that there are no side effects. However, we are excited by this."

"If this works, 15 to 20 years from now, we could be using the patient's own immune system in this way to fight early onset prostate cancer so that patients won't need painful treatments or even surgery."

Considering the procedure, Asons Executive, Laura Williams

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New Gene Therapy Treatment Helps Fight Prostate Cancer, Asons Solicitors Comment

Researchers identify way to increase gene therapy success

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PUBLIC RELEASE DATE:

29-Oct-2013

Contact: Mary Ellen Peacock maryellen.peacock@nationwidechildrens.org 614-355-0495 Nationwide Children's Hospital

Scientists in The Research Institute at Nationwide Children's Hospital have found a way to overcome one of the biggest obstacles to using viruses to deliver therapeutic genes: how to keep the immune system from neutralizing the virus before it can deliver its genetic payload. In a study published recently in Molecular Therapy, researchers found that giving subjects a treatment to temporarily rid the body of antibodies provides the virus safe passage to targeted cells, allowing it to release a corrective or replacement gene to treat disease.

Gene therapy is among the most promising treatment options for such genetic disorders as muscular dystrophy, congenital blindness and hemophilia. Scientists also are investigating gene therapy as a cure for some cancers, neurodegenerative diseases, viral infections and other acquired illnesses. To get the therapeutic gene into cells, researchers have turned to viruses, which deliver their genetic material into cells as part of their normal replication process. Time and time again, these efforts have been thwarted by the body's own immune system, which attacks the viral vector. The therapeutic genes aren't delivered and disease rages on.

Now, a team led by Louis G. Chicoine, MD, Louise Rodino-Klapac, PhD, and Jerry R. Mendell, MD, principal investigators in the Center for Gene Therapy at Nationwide Children's, has shown for the first time that using a process called plasmapheresis just before delivering a virus-packed gene therapy protects the virus long enough for it to enter the cell and deliver the gene.

Plasmapheresis, widely used to treat patients with autoimmune disorders, removes blood from the body, separates the plasma and cells, filters out antibodies, and returns the blood to the patient. The antibody loss is temporary; the body begins producing new antibodies within a few hours of the procedure.

In a study of a gene therapy designed to treat Duchenne muscular dystrophy (DMD), Drs. Chicoine and Rodino-Klapac used plasmapheresis in a large animal model, then injected a virus packed with a micro-dystrophin gene. When they examined the levels of micro-dystrophin gene expression in the animals, they found a 500 percent percent increase over gene expression in animals that did not receive plasmapheresis. Dr. Mendell, director of the Center for Gene Therapy, helped conceive of this treatment for DMD patients based on experience with autoimmune diseases such as myasthenia gravis and inflammatory nerve diseases.

"Right now, gene therapy seems to work best in patients who have no antibodies for the virus being used to deliver the gene," Dr. Mendell says. "That limits the number of patients who can benefit from gene therapy."

Using plasmapheresis would increase the potential for gene therapy, Dr. Chicoine adds, by eliminating one obstacle of immune reaction.

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Researchers identify way to increase gene therapy success

Pluristem to Present at Omics Group's 2nd International Conference and Exhibition on Cell & Gene Therapy

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HAIFA, Israel, Oct. 23, 2013 (GLOBE NEWSWIRE) -- Pluristem Therapeutics Inc. (PSTI) (TASE:PLTR), a leading developer of placenta-based cell therapies, announced today that Pluristem's VP of Development, Ohad Karnieli PhD will present at Omics Group's 2nd International Conference and Exhibition on Cell & Gene Therapy in Orlando, Florida on October 25, 2013 at 12:50pm. Dr. Karnieli's presentation will focus on Pluristem's approach on industrialized cell therapy manufacturing.

As previously announced, Pluristem's 3-dimensional cell production is manufactured in its state-of-the-art 40,000 square foot facility.

Omics Group's 2nd International Conference and Exhibition on Cell & Gene Therapy 2013 is an event which brings together a unique and international mix of leading universities and cell therapy institutions making the congress a perfect platform to share experience. It paves a way to gather visionaries through the research talks and presentations and put forward many thought provoking strategies in emerging cell & gene therapies. The conference aims to serve as a catalyst for the advancement in cell & gene therapies by connecting scientists within and across disciplines at conferences held under a single roof that create an environment conducive to information exchange, generation of new ideas and acceleration of applications that benefit society.

About Pluristem Therapeutics

Pluristem Therapeutics Inc. is a leading developer of placenta-based cell therapies. The Company's patented PLX (PLacental eXpanded) cells are a drug delivery platform that releases a cocktail of therapeutic proteins in response to a host of local and systemic inflammatory and ischemic diseases. PLX cells are grown using the company's proprietary 3D micro-environmental technology and are an "off-the-shelf" product that requires no tissue matching prior to administration.

Pluristem has a strong intellectual property position, company-owned GMP certified manufacturing and research facilities, strategic relationships with major research institutions and a seasoned management team. For more information visit http://www.pluristem.com, the content of which is not part of this press release.

The Pluristem Therapeutics Inc. logo is available at http://www.globenewswire.com/newsroom/prs/?pkgid=6882

Safe Harbor Statement

This press release contains forward-looking statements within the meaning of the "safe harbor" provisions of the Private Securities Litigation Reform Act of 1995 and federal securities laws. These forward-looking statements and their implications are based on the current expectations of the management of Pluristem only, and are subject to a number of factors and uncertainties that could cause actual results to differ materially from those described in the forward-looking statements. The following factors, among others, could cause actual results to differ materially from those described in the forward-looking statements: changes in technology and market requirements; we may encounter delays or obstacles in launching and/or successfully completing our clinical trials; our products may not be approved by regulatory agencies, our technology may not be validated as we progress further and our methods may not be accepted by the scientific community; we may be unable to retain or attract key employees whose knowledge is essential to the development of our products; unforeseen scientific difficulties may develop with our process; our products may wind up being more expensive than we anticipate; results in the laboratory may not translate to equally good results in real surgical settings; results of preclinical studies may not correlate with the results of human clinical trials; our patents may not be sufficient; our products may harm recipients; changes in legislation; inability to timely develop and introduce new technologies, products and applications; loss of market share and pressure on pricing resulting from competition, which could cause the actual results or performance of Pluristem to differ materially from those contemplated in such forward-looking statements. Except as otherwise required by law, Pluristem undertakes no obligation to publicly release any revisions to these forward-looking statements to reflect events or circumstances after the date hereof or to reflect the occurrence of unanticipated events. For a more detailed description of the risks and uncertainties affecting Pluristem, reference is made to Pluristem's reports filed from time to time with the Securities and Exchange Commission.

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Pluristem to Present at Omics Group's 2nd International Conference and Exhibition on Cell & Gene Therapy

Late stage gene therapy for inherited blindness part of Children’s Hospital spinout

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A Children Hospital of Philadelphia gene therapy spinout is taking over development of a Phase 3 gene therapy program to treat inherited blindness by counteracting retinal degeneration. Its one of the most common causes of blindness in children.

Spark Therapeutics is advancing the work of CHOPs Center for Cellular and Molecular Therapeutics. The center was set up in 2004 as a center for gene therapy translational research and manufacturing. CHOP is giving it $50 million to advance its genetic therapies.

Inherited blindness is caused by mutations in the RPE65 gene. There is currently no drug or therapeutic treatment for this form of inherited retinal degeneration, according to the hospital statement. It ultimately causes irreversible blindness.

The treatment has produced some encouraging results. A clinical study of 12 patients with RPE65-related blindness demonstrated notable improvement in visual function. In some cases, children who were profoundly blind were able to recognize faces and move independently, according to the statement.All school-age patients enrolled in the trial were able to transfer from Braille classrooms to sighted classrooms. The next step is a Phase 3 open-label, randomized, controlled study that will expand on the study.

The gene mutation in one of 14 genes that cause Lebers congenital amaurosis. It disrupts development of the retina, causing people with the disease to have severe vision deficits from birth that progress slowly over time to total blindness.

Many of the centers leaders will take on management roles in Spark or work with the company as scientific advisers. Among those advisers is Dr. Katherine A. High, a gene therapy pioneer who has worked as the director of the center from the start.

The group is also developing a gene therapy for hemophilia B. The goal is to eliminate or reduce the need for regular infusions of clotting factor. It might even be able to help hemophilia B patients with inhibiting antibodies.

The company is also advancing toward the clinic with gene therapy programs to address neurodegenerative diseases and additional hematologic disorders and other forms of inherited blindness.

The company was co-founded by CEO Jeffrey D. Marrazzo, who has served as an entrepreneurial consultant to the hospital for the past three years. He said: The creation of Spark is the culmination of a decade-long commitment by CHOP and our founding team to drive the field of gene therapy forward during a time when many in the industry had moved away.

Last month, CHOP formed a partnership with Osage University Partners in a move to develop more partnership opportunities. One spin-off company CHOP has produced is Vascular Magnetics to treat peripheral artery disease. A rotovirus vaccine called RotaTeq produced through joint research between CHOP and Wistar Institute is now sold by Merck.

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Late stage gene therapy for inherited blindness part of Children’s Hospital spinout

New Gene Therapy Company Launches

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Spark Therapeutics hopes to commercialize multiple gene-based treatments developed at the Childrens Hospital of Philadelphia.

A new biotechnology company will take over human trials of two gene therapies that could offer one-time treatments for a form of childhood blindness and hemophilia B.

The gene therapies were developed by researchers at the Childrens Hospital of Philadelphia, which has committed $50 million to the new company called Spark Therapeutics. The launch is the latest hint that after decades of research and some early setbacks, gene therapy may be on its way to realizing its potential as a powerful treatment for inherited disease.

In December 2012, the European Union gave permission to Dutch company Uniqure to sell its gene therapy for a fat-processing disorder, making Glybera the first gene therapy to make its way into a Western market (see Gene Therapy on the Mend as Treatment Gets Western Approval). However, Glybera has not been approved by the U.S., nor has any other gene therapy.

Spark has a chance to be the first gene-therapy company to see FDA approval. Results for a late-stage trial of a gene therapy for Lebers Congenital Amaurosis, an inherited condition that leads to a loss of vision and eventually blindness, are expected by mid-2015. That treatment is one of several gene therapies in or nearing late-stage testing contending to be the first gene therapy approved by the FDA for sale in the U.S. (see When Will Gene Therapy Come to the U.S.).

In addition to taking the reins for two-ongoing human trials, Spark will also work on gene therapies for other eye and blood conditions as well as neurodegenerative diseases, says CEO Jeff Marrazzo. The gene therapy technology developed at the Childrens Hospital has been speeding down the tracks, he says, and the company will provide the vehicle to get these therapies to the people who need them.

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New Gene Therapy Company Launches

Novel gene therapy enables persistent anti-tumor immune response

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Public release date: 9-Oct-2013 [ | E-mail | Share ]

Contact: Vicki Cohn vcohn@liebertpub.com 914-740-2100 x2156 Mary Ann Liebert, Inc./Genetic Engineering News

New Rochelle, NY, October 9, 2013Cancer immunotherapy can successfully use the body's own immune system to kill tumor cells. But some current approaches to stimulate an antitumor immune response are short-lived, with limited clinical effectiveness. A new gene transfer strategy that introduces modified, immune-stimulating human stem cells is both feasible and effective for achieving persistent immunotherapy to treat leukemias and lymophomas, according to a study published in Human Gene Therapy, a peer-reviewed journal from Mary Ann Liebert, Inc., publishers. The article is available on the Human Gene Therapy website.

Satiro Nakamura De Oliveira and coauthors from the David Geffen School of Medicine, University of California, Los Angeles and University of Texas MD Anderson Cancer Center, Houston, describe the gene transfer method they developed to deliver chimeric antigen receptors, or CARS, that direct the immune system to target tumor cells derived from B-lymphocytes.

In the article "Modification of Hematopoietic Stem/Progenitor Cells with CD19-specific Chimeric Antigen Receptors as a Novel Approach for Cancer Immunotherapy" the authors show that by packaging the CARS in human hematopoietic stem cells, the immunotherapeutic receptors will be produced in the bloodstream for a long period of time. This persistent expression should improve their effectiveness in the treatment of blood cancers such as leukemia and lymphoma.

"This study represents an interesting new direction for an approach that has generated substantial interest," says Dr. Wilson, Director of the Gene Therapy Program, Department of Pathology and Laboratory Medicine, University of Pennsylvania Perelman School of Medicine, Philadelphia.

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About the Journal

Human Gene Therapy, the official journal of the European Society of Gene and Cell Therapy, British Society for Gene and Cell Therapy, French Society of Cell and Gene Therapy, German Society of Gene Therapy, and five other gene therapy societies, is an authoritative peer-reviewed journal published monthly in print and online. Human Gene Therapy presents reports on the transfer and expression of genes in mammals, including humans. Related topics include improvements in vector development, delivery systems, and animal models, particularly in the areas of cancer, heart disease, viral disease, genetic disease, and neurological disease, as well as ethical, legal, and regulatory issues related to the gene transfer in humans. Its sister journal, Human Gene Therapy Methods, published bimonthly, focuses on the application of gene therapy to product testing and development, and Human Gene Therapy Clinical Development, new in 2013, publishes data relevant to the regulatory review and commercial development of cell and gene therapy products. Complete tables of content for all three publications and a free sample issue may be viewed on the Human Gene Therapy website.

About the Publisher

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Novel gene therapy enables persistent anti-tumor immune response

OncoSec Medical to Present at the Cancer Vaccines and Gene Therapy Meeting

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SAN DIEGO--(BUSINESS WIRE)--

OncoSec Medical Inc. (ONCS), a company developing its advanced-stage ImmunoPulse DNA-based immunotherapy and NeoPulse therapy to treat solid tumors, has announced it will be presenting at the Cancer Vaccines and Gene Therapy Meeting at The Desmond Malvern Hotel in Philadelphia, Pennsylvania.

Dr. Richard Heller, Professor at Old Dominion University, will be presenting pre-clinical data on the use of electroporation to deliver plasmid IL-12 in solid tumors in a presentation titled Gene Electrotransfer as an Effective Delivery Tool. This presentation will take place on Thursday, October 3 at 3:30 p.m. EST.

Dr. Adil Daud, principal investigator and co-director of melanoma research at the University of California San Francisco School of Medicine, will be presenting an update to OncoSecs Phase II melanoma immune response data, which will include IL-12 expression findings in a presentation titled Gene Electrotransfer in Solid Tumors. This presentation will take place on Friday, October 4 at 1:00 p.m. EST.

About the Cancer Vaccines and Gene Therapy Meeting

The Cancer Vaccines and Gene Therapy Meeting will bring together important leaders to present and discuss the latest approaches and technologies used to develop promising anti-cancer therapeutics.

About OncoSec Medical Inc.

OncoSec Medical Inc. isa biopharmaceutical companydeveloping its advanced-stage ImmunoPulse DNA-based immunotherapy and NeoPulse therapy to treat solid tumors.ImmunoPulse and NeoPulse therapiesaddress an unmet medical needandrepresenta potential solution for less invasive and less expensive therapies that are able to minimize detrimental effects resulting from currently available cancer treatments such as surgery, systemic chemotherapy or immunotherapy, and other treatment alternatives. OncoSec Medical's core technology is based upon its proprietaryuse of anelectroporation platform to enhancethedelivery and uptake of a locally delivered DNA-based immunocytokine (ImmunoPulse) or chemotherapeutic agent(NeoPulse). Treatment ofvarious solid cancersusing these targetedanti-cancer agentshas demonstratedselective destruction of cancerous cellswhile potentially sparing healthy normal tissues during early and late stage clinical trials. OncoSec's clinical programs include three Phase II clinical trials for ImmunoPulse targeting lethal skin cancers. More information is available athttp://www.oncosec.com/.

This press release contains forward-looking statements within the meaning of the U.S. Private Securities Litigation Reform Act of 1995. Any statements in this release that are not historical facts may be considered such "forward-looking statements." Forward-looking statements are based on management's current preliminary expectations and are subject to risks and uncertainties, which may cause our results to differ materially and adversely from the statements contained herein. Some of the potential risks and uncertainties that could cause actual results to differ from those predicted include our ability to raise additional funding, our ability to acquire, develop or commercialize new products, uncertainties inherent in pre-clinical studies and clinical trials, unexpected new data, safety and technical issues, competition, and market conditions. These and additional risks and uncertainties are more fully described in OncoSec Medical's filings with the Securities and Exchange Commission. Undue reliance should not be placed on forward-looking statements, which speak only as of the date they are made. OncoSec Medical disclaims any obligation to update any forward-looking statements to reflect new information, events or circumstances after the date they are made, or to reflect the occurrence of unanticipated events.

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OncoSec Medical to Present at the Cancer Vaccines and Gene Therapy Meeting

Research and Markets: Gene Therapy Drug Pipeline Analysis Report 2013

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DUBLIN--(BUSINESS WIRE)--

Research and Markets (http://www.researchandmarkets.com/research/nnxfrm/gene_therapy_drug) has announced the addition of the "Gene Therapy Drug Pipeline Analysis" report to their offering.

Gene Therapy Drug Pipeline Analysis gives a comprehensive insight on the various drugs being developed for the treatment of multiple disease based upon Gene Therapy. Research report covers all the ongoing drug development in various development phases.

Each drug profiles include detailed information like: Originator, Owner, Collaborator, Technology Provider, Licensee, Development Phase, Development Indications, Mechanism of Action, Country of Development and detailed analysis on the development process.

Insight for each drug profile in development phase enables the reader to identify and understand the Gene Therapy associated with the various diseases.

This report enables pharmaceutical companies, collaborators and other associated stake holders to identify and analyze the available investment opportunity in the Gene Therapy based drug development process.

Following parameters for each drug profile in development phase are covered in Gene Therapy Drug Pipeline Analysis research report:

- Drug Profile Overview

- Alternate Names for Drug

- Active Indication

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Research and Markets: Gene Therapy Drug Pipeline Analysis Report 2013


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