BIOTECH GENE THERAPY
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Category Archives: Gene Therapy
Producing Viral Vectors for Clinical Trials With On-Site Clinical Manufacturing Facility – Video
Producing Viral Vectors for Clinical Trials With On-Site Clinical Manufacturing Facility
Viral gene therapy is being tested as treatment for numerous diseases, including cancer, cystic fibrosis, heart disease, muscle diseases, diabetes and neurodegenerative disease like Parkinson #39;s. While gene therapy is still available only as part of a clinical trial, scientists, clinicians and families hold hope that it will eventually become a fundamental part of modern medicine and provide treatment for the currently untreatable. Modified viruses used to deliver genetic material into cells, known as viral vectors, have become the go-to delivery capsule in gene therapy. Investigators use viral vectors to efficiently target certain cells and deliver genetic material into the cell nucleus where native genes reside. Nationwide Children #39;s has a unique facility that specializes in creating these viral vectors - a rare, incredibly beneficial asset to our Research Institute - called the Clinical Manufacturing Cleanroom Facility. So far, the Cleanroom Facility has produced several vectors for use in investigational new drug studies, and products for phase I clinical trials for the congenital blindness disorder, choroideremia, muscular dystrophy and inclusion body myositis. Additional trials focused on spinal muscular atrophy and Duchenne and limb girdle muscular dystrophies are in the works. Having this facility on-site allows for expedited clinical trials at a much more affordable price, which all comes back to being able to offer the best possible care for all of our patients ...From:NationwideChildrensViews:5 0ratingsTime:01:33More inNonprofits Activism
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Producing Viral Vectors for Clinical Trials With On-Site Clinical Manufacturing Facility - Video
Breakthrough gene therapy at WSU
Gene therapy is the key that unlocked treatment for a disorder that attacks the pancreas at a laboratory at Wright State University.
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Ramsey Majzoub explains Gene Therapy Research conducted in the Safinya Group at UCSB – Video
Ramsey Majzoub explains Gene Therapy Research conducted in the Safinya Group at UCSB
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Ramsey Majzoub explains Gene Therapy Research conducted in the Safinya Group at UCSB - Video
Help Hemophilia of Georgia fund research for a cure – Video
Help Hemophilia of Georgia fund research for a cure
Families affected by hemophilia are anxiously waiting to find out if current gene therapy research will result in a cure. In this video, two fathers share what a cure would mean to their familiesFrom:HemophiliaOfGeorgiaViews:1 0ratingsTime:01:23More inNonprofits Activism
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Melanoma, how can we have an earlier diagnosis? – Profesor Dréno – Video
Melanoma, how can we have an earlier diagnosis? - Profesor Drno
Interview of Professor Brigitte Drno Vice Dean in charge of Research Faculty of Medicines Department of Dermato Cancerology Director Cell and Gene Therapy Unit Nantes Hospital University Giving her opinion on how to have an earlier diagnosis for melanoma.From:UVDAMAGELRPViews:2 0ratingsTime:03:45More inEducation
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Melanoma, how can we have an earlier diagnosis? - Profesor Dréno - Video
Gene Therapy Second Period Mr. Bell – Video
Gene Therapy Second Period Mr. Bell
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New follistatin gene therapy for muscular dystrophy gets orphan drug status
An experimental drug being developed by Milo Biotechnology for the treatment of certain neuromuscular diseases has been given orphan drug designation by the U.S. Food and Drug Administration. The Cleveland company’s drug candidate, AAV1-FS344, is a gene delivery therapy that’s designed to produce follistatin, a protein associated with muscle size and strength, in patients with [...]
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New follistatin gene therapy for muscular dystrophy gets orphan drug status
Dr. Ted Ross Joins the Vaccine & Gene Therapy Institute of Florida as Program Director
PORT ST. LUCIE, Fla.--(BUSINESS WIRE)--
The Vaccine and Gene Therapy Institute of Florida (VGTI Florida), a leading nonprofit immunological research institute, is pleased to announce the appointment of Ted M. Ross, Ph.D., as Program Director. Dr. Ross will explore new vaccine technologies intended to protect against all strains of seasonal influenza. Such a vaccine could potentially eliminate the need for seasonal flu shots.
Traditionally, the flu vaccine is a mixture of inactive influenza viruses. However, Dr. Ross is taking a novel approach by examining the use of a synthetic vaccine created from genetic sequences of many different flu viruses. Dr. Ross previously used this method to produce a vaccine against the H5N1 virus, commonly known as the bird flu. The vaccine, well-tolerated in pre-clinical trials, successfully protects against known strains of bird flu. Dr. Ross and his VGTI Florida colleagues are applying similar strategies to fight other serious viruses such as West Nile Virus, Dengue and HIV Type 1 (HIV-1).
The holy grail of influenza vaccine research is a vaccine that protects against all influenza strains, avoiding the need for annual vaccinations, said Jay Nelson, Ph.D., founder and Executive Director, VGTI Florida. Were genuinely excited that Dr. Ross has joined us. His extensive background in immunology and microbiology will further benefit our efforts to develop vaccines that can help people with influenza, especially the elderly, added Dr. Rafick Skaly, Chief Scientific Officer and Co-Director of the institute.
Most recently, Dr. Ross was an Associate Professor, University of Pittsburgh School of Medicine, where he served in the Microbiology and Molecular Genetics department and was a full member of the Center for Vaccine Research. Prior to that, he was an Assistant Professor in the Department of Microbiology and Immunology at East Carolina University, School of Medicine in North Carolina.
Dr. Ross has co-authored numerous scientific articles published in journals such as Nature Immunology, The Journal of Virology, Journal of Infectious Disease, Vaccine, and the Proceedings of the National Academy of Sciences. He is a member of the American Society for Virology, American Society of Microbiology, Sigma Xi, International Society of Vaccines, and others. He holds B.S. and M.S. degrees from the University of Arkansas in Zoology and Microbiology, respectively. Dr. Ross earned his Ph.D. from Vanderbilt University in Microbiology & Immunology. Additionally, he was a Postdoctoral Fellow in HIV Research at Duke University and a Senior Research Associate in HIV Vaccine Research at Emory University.
VGTI Florida
VGTI Florida is a leading immunological research institute that is on an urgent mission to transform scientific discoveries into novel treatments and cures for devastating chronic illnesses such as cancer and HIV/AIDS. VGTI Florida is an independent non-profit 501 (c)(3) organization located in the Tradition Center for Innovation in Port St. Lucie, Florida. For more information, please visit http://www.VGTIFL.org.
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Dr. Ted Ross Joins the Vaccine & Gene Therapy Institute of Florida as Program Director
Innovation Presentation – Cancer Gene Therapy Project – Video
Innovation Presentation - Cancer Gene Therapy Project
NSW Health:bit.ly Media Release:bit.ly The Minister for Health, Jillian Skinner, hosted a community health forum in Parramatta on Wednesday 26 September 2012 to give Greater Western Sydney residents the chance to have their say on the future of health services in their community.From:NewSouthWalesHealthViews:0 0ratingsTime:06:48More inNonprofits Activism
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Innovation Presentation - Cancer Gene Therapy Project - Video
Gene therapy at St. Jude bringing relief to hemophilia patients
Derek Houser ate a quick lunch, settled into a hospital bed and waited for his life to change.
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Gene therapy at St. Jude bringing relief to hemophilia patients
Gene Therapy Using T-Cells Helps Child Beat Leukemia
Ground-breaking therapy is helping kids beat cancer. Doctors at Children's Hospital of Philadelphia are using gene therapy to help children battle leukemia.
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uniQure Initiates Phase I in Acute Intermittent Porphyria
AMSTERDAM, December 11, 2012 /PRNewswire/ --
uniQure B.V., a leader in the field of human gene therapy, today announced the start of its Phase I clinical trial in acute intermittent porphyria (AIP) with the treatment of the first patient. The study is conducted under the aegis of the AIPGENE consortium, a pan-European collaboration funded in part by the European Commission's Seventh Framework Program with the aim to develop a gene therapy for the treatment of AIP, a rare and devastating disease caused by mutations in the porphobilinogen deaminase gene (PBGD). AIP can be life-threatening and the long-term effects include irreversible nerve damage, liver cancer and kidney failure. uniQure was granted orphan drug designation for the treatment of AIP in 2009 from the European Medicines Agency.
"The start of the AIP Phase I study marks the first of four programs that will enter clinical trials over the next 12 months," says Jrn Aldag, CEO of uniQure. "After AIP we expect clinical trials to be initiated in Parkinson's disease, hemophilia B, and Sanfilippo B. After many years of building and developing our capabilities and competencies, and the approval in November of Glybera for LPLD as the first gene therapy in the Western world, we are highly motivated to expedite the clinical development of our other advanced gene therapies."
About the AIP Phase I study
The Phase I will enroll eight patients with severe AIP at two centers: the Clinical University of Navarra, Pamplona, Spain, and the 12 de Octubre University Hospital,, Madrid, Spain. The study's primary objective is the assessment of safety and determination of the maximum tolerated dose. Secondary objectives include tolerability of treatment, pharmacokinetics, changes in the levels of surrogate markers of activity including porphobilinogen (PBG) and delta-aminolevulinic acid (ALA), and assessment of symptom control, neuro-psychological changes and quality of life. All patients will be followed for one year, and the interim results of the Phase I are expected in Q3 2013.
About acute intermittent porphyria
Acute Intermittent porphyria (AIP) is a rare genetic disease which is caused by mutations in the porphobilinogen deaminase (PBGD) gene; one of the enzymes of the heme biosynthesis pathway. Mutations in this gene cause insufficient activity of the protein resulting in partially disruption of heme synthesis. This in turn leads to accumulation of toxic intermediates (ALA and PBG) giving rise to a wide variety of problems including acute, severe abdominal pains, psychiatric and neurological disorders, and muscular weakness. Acute porphyric attacks can be life-threatening and the long-term consequences include irreversible nerve damage, liver cancer and kidney failure. Currently, the only curative therapy is liver transplantation and thus, new curative options are urgently needed. Severe AIP patients are suffering poor quality of life with palliative treatments for the different symptoms including glucose or heme infusions for metabolic replacement and inhibition of toxic metabolic production.
About AIPGENE
AIPGENE is a European Commission Framework Programme 7-funded consortium (Grant Agreement number 261506) which was put together with the aim to develop the orphan gene therapy drug AAV5-AAT-PBGD (AMT-021) for the treatment of Acute Intermittent porphyria (AIP). The consortium's objective is to contribute to alleviating the negative impact of this disease on the quality of life of the patients and their families. Overall coordinator of the project is the Centre for Applied Medical Research (CIMA) at the University of Navarra, Pamplona, Spain. Apart from uniQure, other members of the consortium are the Clinical University of Navarra, Pamplona, Spain; Karolinska University Hospital, Stockholm, Sweden; German Cancer Research Center (NCT-DKFZ), Heidelberg, Germany; DIGNA Biotech, Pamplona, Spain; Servicio Madrileno de Salud, Madrid, Spain.
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Alliance for Cancer Gene Therapy Funds Promising Leukemia Study – Patients in Remission More Than Two Years after Gene …
STAMFORD, Conn.--(BUSINESS WIRE)--
Alliance for Cancer Gene Therapy (ACGT), the nations only non-profit organization dedicated exclusively to funding cell and gene therapy research for cancer, is excited to play a major role in the recent leukemia study pioneered by scientists at the Perelman School of Medicine at the University of Pennsylvania. ACGT was the initial funding arm for the study using immune-mediated gene therapy for leukemia and lymphoma. This study illustrates the successful and sustained demonstration of how gene therapy uses the bodys own T-cells and turns them into weapons aimed directly at cancer.
Initial ACGT grants were awarded in 2004 to Dr. Carl June of the Abramson Family Cancer Research Institute at the University of Pennsylvania, and to Dr. Michel Sadelain, of Memorial Sloan-Kettering Cancer Center, Gene Therapy & Gene Expression Laboratory in New York City. Preliminary results were issued by Dr. Carl June and the University of Pennsylvania in August 2011, with additional results released this week and presented by Dr. Carl June at the American Society of Hematologys Annual Meeting and Exposition being held in Atlanta, Georgia.
The clinical trial participants, all of whom had advanced cancers, included ten adult patients with chronic lymphocytic leukemia, were treated at the Hospital of the University of Pennsylvania (HUP) and two children with acute lymphoblastic leukemia were treated at the Childrens Hospital of Philadelphia (CHOP). Two of the first three patients treated with the protocol at HUP whose cases were detailed in The New England Journal of Medicine and Science Translational Medicine in August 2011 remain healthy and in full remission more than two years after their treatment, with the engineered cells still circulating in their bodies. Currently, nine out of 12 of the participants show their disease in remission.
The discovery of successful cancer gene therapy treatments are what my husband and I hoped for when we founded ACGT a decade ago, noted Barbara Netter, president and co-founder of the Alliance for Cancer Gene Therapy. We knew it would be an uphill battle. ACGT was the only organization willing to take the risk when others were not. With federal funds decreasing, and the realization that pharmaceutical companies will not participate in the research phase until marketable and mass-produced treatments are created, we seized the chance to make a difference. My late husband Edward Netter (1932-2011), was a true visionary in the field of medical research. He would be so thrilled by the progress ACGT has made possible.
ACGT is currently funding 17 clinical trials in cancer cell and gene therapy targeting numerous types of cancers. Since its inception, ACGT has awarded more than $23 million in grants to 39 investigators to treat 11 different types of cancer. In 2012, ACGT also awarded a $500,000 grant to a clinical translational study on pancreatic cancer and has great expectations for its outcome. In ACGTs 2011 grant cycle, 87 scientists from throughout the U.S. responded with grant applications for ACGTs Young Investigator Grants, making 2011 one of the most sought-after funding year in ACGT history. The ACGT funded clinical studies are already showing promise, especially in the treatment of leukemia, lung, melanoma and prostate cancers.
It is so exciting that the pathfinder role ACGT played by provided the seed money for the University of Pennsylvania trial has led to these stunning successes for leukemia treatment, said Dr. Savio Woo, chairman of ACGTs Scientific Advisory Council, and founding Chair of the Department of Gene and Cell Medicine at Mt. Sinai School of Medicine in New York City.
Woo noted that 100 percent of all funds donated to ACGT go directly toward funding innovative cancer gene therapy research grants. ACGTs Scientific Advisory Council, which comprises some of the best scientific minds and thought leaders with major U.S. medical institutions, oversee all phases of the ACGT grant process. Through this rigorous review, ACGT is able to identify and fund studies with the most potential for positive and innovative outcomes for treating cancer using cell and gene therapy.
The University of Pennsylvanias initial study was funded primarily by ACGT. The most recent clinical trial was also supported by ACGT, the Leukemia & Lymphoma Society (Dr. June is the leader of one of the LLSs grants), and the National Institutes of Health. In addition, Novartis announced this summer that it would fund additional research at the University of Pennsylvania to further study the immunotherapies and has acquired exclusive rights to market the treatment.
Alliance for Cancer Gene Therapy (ACGT) is the nations only non-profit dedicated exclusively to cell and gene cancer therapy research. One hundred percent of all contributions to ACGT go directly to research and fund grants with leading scientists in the U.S., representing such institutions as Harvard Medical School, Johns Hopkins University School of Medicine, Mayo Clinic, St. Judes Childrens Hospital, Duke University, The Salk Institute, University of Pennsylvania, Memorial Sloan-Kettering, Stanford University, Dana Farber Cancer Center, University of California San Diego, University of Pittsburgh, and the University of Chicago. A rigorous grant review by Scientific Advisory Council ensures the most promising projects are rewarded. To learn more about the leukemia study at the University of Pennsylvania, and about the Alliance for Cancer Gene Therapy (ACGT), visit http://www.acgtfoundation.org or call 203.358.8000.
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Gene therapy shows promise in eradicating blood cancers
A 7-year-old pixie named Emily Whitehead has erased any remaining doubts about the power of a University of Pennsylvania gene therapy to eradicate certain blood cancers.
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Gene therapy saves cancer-stricken 7-year-old
Dr. Stephan Grupp, of The Children's Hospital of Philadelphia examines first pediatric patient Emma Whitehead six months after T cell therapy. (Courtesy The Children's Hospital of Philadelphia)
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University of Pennsylvania gene therapy shows promise in eradicating some blood cancers
A 7-year-old pixie named Emily Whitehead has erased any remaining doubts about the power of a University of Pennsylvania gene therapy to eradicate certain blood cancers.
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University of Pennsylvania gene therapy shows promise in eradicating some blood cancers
NATHAN KLEIN- FIRST MAN GENE THERAPY FOR PARKINSONS DISEASE – Video
NATHAN KLEIN- FIRST MAN GENE THERAPY FOR PARKINSONS DISEASE
FIRST MAN TO RECIEVE GROUND BREAKING GENE THERAPY FOR PARKINSONS DISEASEFrom:venture1webViews:12 0ratingsTime:03:27More inScience Technology
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NATHAN KLEIN- FIRST MAN GENE THERAPY FOR PARKINSONS DISEASE - Video
Investigational agent targets gene signaling pathways to improve response for patients with CLL
( American Society of Hematology ) The promising investigational targeted therapy ibrutinib and its mechanism of silencing gene communication pathways critical to the development of cancer may be an effective way to combat chronic lymphocytic leukemia, according to studies presented today at the 54th Annual Meeting of the American Society of Hematology.
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Investigational agent targets gene signaling pathways to improve response for patients with CLL
Gene Therapy (Advanced Biotechnology Project) – Video
Gene Therapy (Advanced Biotechnology Project)
james73394 #39;s shared video file.From:James ReisViews:0 0ratingsTime:03:01More inScience Technology
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