Prof Krystof Bankiewicz. Overview of AADC Deficiency - AAV2-hAADC Gene Therapy Trials.
Prof Krystof Bankiewicz of University of California, San Francisco, USA presents an overview of AADC Deficiency and AAV2-hAADC Gene Therapy Trials. He describes the technique and background...
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Prof Krystof Bankiewicz. Overview of AADC Deficiency - AAV2-hAADC Gene Therapy Trials. - Video
AADC Gene Therapy Trial Natural History Study. Dr Toni Pearson
Dr Toni Pearson, Mount Sinai Medical Center - USA Gene Therapy Trial Natural History Study Summary: AADC Gene Therapy Trial. An update on the planning of the trial, overview of the trial,...
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AADC Gene Therapy Trial & Natural History Study. Dr Toni Pearson - Video
Surgical Technique for AADC Gene Therapy. Prof John Heiss
Prof. John Heiss, National Institute of Health NINDS - USA Surgical Technique for AADC Gene Therapy Summary: The history of delivering gene therapy to the central nervous system will be...
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Surgical Technique for AADC Gene Therapy. Prof John Heiss - Video
Bioethics on Gene Therapy
Talks about gene therapy the costs and benefits.
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Gene Therapy AN NM Biology 1233
By: Angelica Nino
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In an interesting move, Pfizer ( PFE ) has struck a deal with Spark Therapeutics to establish a gene therapy platform. King's College Professor Michael Linden, who is an expert in gene therapy research, will lead the effort. Under the agreement, Pfizer will make an upfront payment of $20 million to Spark Therapeutics and $260 million in additional milestone-based payments. In return, Pfizer will handle late stage clinical trials, approval and commercialization of the product. Spark is currently investigating the efficacy of gene therapy forhemophilia B, and its program will enter early phase trials next year. Gene therapy has been under scientific research for over 2 decades, but viable therapies have yet to gain commercial acceptance due to safety and delivery-related issues. However, Pfizer's move and some other recent developments in the industry suggest that the therapy may be coming off age. There is another implication of this move. Even if successful, Pfizer is still a long way away from a commercialized gene-derived therapy. It is thus just a single step in its program to reverse its revenue decline in coming years. Other actions are possible, or even likely, including an outright acquisition of a substantially larger company, considering that most deals announced thus far are focused on early stage compounds.
Our price estimate for Pfizer stands at $35 , implying a premium of about 20% to the market price.
See Full Analysis For Pfizer
Establishing a gene therapy platform is a welcome move, and points towards Pfizer's willingness to innovate and take risks. Gene therapy involves treating a disease through modification of defective or absent gene. Such modification can include replacing, altering or supplementing existing genetic material. The approach offers the potential to treat rare hereditary diseases and open new doors in curing cancer. Although it is too early to estimate the revenue potential of this industry, a successful launch can pave way for big pharmaceutical firms to revive their declining businesses provided they jump on the bandwagon at the right time. Although Spark's gene therapy platform initially will focus on treatment of hemophilia B, there exists the potential for developing similar therapies for the treatment of cancer. This is where Pfizer is showing special interest. Its oncology drug sales jumped 16% globally in Q3 2014, sustaining the growth rate observed in the second quarter and representing a growth acceleration compared to the first quarter. For the first nine months, the segment's revenue growth stood at roughly 13%. The figure is the highest among the company's primary business segments, with vaccine sales racing past that of oncology drugs only in the third quarter. We expect the company to continue to push for deals and possibly, acquisitions, that can strengthen its oncology pipeline.
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Scientists at Indiana University and colleagues at Stanford and the University of Texas have demonstrated a technique for "editing" the genome in sperm-producing adult stem cells, a result with powerful potential for basic research and for gene therapy.
The researchers completed a "proof of concept" experiment in which they created a break in the DNA strands of a mutant gene in mouse cells, then repaired the DNA through a process called homologous recombination, replacing flawed segments with correct ones.
The study involved spermatogonial stem cells, which are the foundation for the production of sperm and are the only adult stem cells that contribute genetic information to the next generation. Repairing flaws in the cells could thus prevent mutations from being passed to future generations.
"We showed a way to introduce genetic material into spermatogonial stem cells that was greatly improved from what had been previously demonstrated," said Christina Dann, associate scientist in the Department of Chemistry at IU Bloomington and a co-author of the study. "This technique corrects the mutation, theoretically leaving no other mark on the genome."
The paper, "Genome Editing in Mouse Spermatogonial Stem/Progenitor Cells Using Engineered Nucleases," was published in the online science journal PLOS-ONE.
The lead author, Danielle Fanslow, carried out the research as an IU research associate and is now a doctoral student at Northwestern University. Additional co-authors are from the Stanford School of Medicine and the University of Texas Southwestern Medical Center.
A challenge to the research was the fact that spermatogonial stem cells, like many types of adult stem cells, are notoriously difficult to isolate, culture and work with. It took years of intensive effort by multiple laboratories before conditions were created a decade ago to maintain and propagate the cells.
For the IU research, a primary hurdle was to find a way to make specific, targeted modifications to the mutant mouse gene without the risk of disease caused by random introduction of genetic material. The researchers used specially designed enzymes, called zinc finger nucleases and transcription activator-like effector nucleases, to create a double strand break in the DNA and bring about the repair of the gene.
Stem cells that had been modified in the lab were then transplanted into the testes of sterile mice. The transplanted cells grew or colonized within the mouse testes, indicating the stem cells were viable. However, attempts to breed the mice were not successful.
"Whether the failure to produce sperm was a result of abnormalities in the transplanted cells or the recipient testes was unclear," the researchers write.
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'Genome editing' could correct genetic mutations for future generations
ANN ARBOR (PRWEB) December 15, 2014
Gene therapy pioneer and longtime Veterans Affairs researcher Dr. David Fink received the 2014 Paul B. Magnuson Award from VA in a ceremony at the VA Ann Arbor Healthcare System on Dec. 15, 2014.
Dr. Fink is a staff neurologist and an investigator with the Geriatric Research, Education and Clinical Center at the Ann Arbor VA. He is also the Robert Brear Professor and Chair of Neurology at the University of Michigan. He has been with VA since 1982.
A Harvard Medical School graduate, Fink has pioneered methods to introduce genes into the body to treat chronic pain and other nervous-system diseases. His team led the first human clinical trial of gene therapy for pain. The phase 1 trial, published in the Annals of Internal Medicine in 2011, involved 10 cancer patients with severe pain who had failed to respond even to high doses of morphine or other pain drugs. Finks group gave them skin injections of an inactive form of the herpes simplex virus as a means to deliver a gene known as PENK. The gene helps the body produce an opioid-like molecule called proenkephalin.
The gene treatment, based on years of research, is safe in humans and led to pain reduction. A larger phase 2 clinical trial of the approach is now being planned.
Besides cancer pain, Finks work focuses on Veterans and others with nerve-related conditions such as spinal cord injury and diabetic neuropathy. The team is developing non-replicating viral vectors, similar to the one used in the 2011 human trial, to ferry genes into the nervous system that code for the production of the bodys own pain relievers. A related approach, now being funded by VA, is to use the vectors to bring about the continuous release of proteins that protect nerve cells from dying. This could help prevent neuropathy and the sharp chronic pain it entails.
Dr. Finks work holds tremendous potential for treating Veterans with chronic neurological disease, said Robert McDivitt, an Army Veteran and director of the VA Ann Arbor Healthcare System.
Fink was presented the award by Dr. Carolyn Clancy, VAs Interim Undersecretary for Health. Also attending the ceremony was Dr. Patricia Dorn, director of VA Rehabilitation Research and Development, which each year presents the Magnuson Award as the highest honor for VA rehabilitation investigators.
The award is named for Paul B. Magnuson, a bone and joint surgeon who was a key figure in the expansion of the VA research program after World War II. He was known for his dedication to finding new treatments and devices to help Veterans cope with their disabilities, and, as he put it, to restoring each patient to his family, his job, and his life. Established in 1998, the Magnuson Award consists of a plaque, a one-time award of $5,000, and $50,000 per year for up to three years to supplement ongoing peer-reviewed research.
About the VA Ann Arbor Healthcare System Since 1953, VA Ann Arbor Healthcare System which includes the VA Ann Arbor Medical Center, the VA Toledo Community Based Outpatient Clinic [CBOC], the VA Flint CBOC, and the VA Jackson CBOC, as well as the VA Center for Clinical Management Research, an HSR&D Center of Innovation, has provided state-of-the-art healthcare services to the men and women who have proudly served our nation. More than 65,000 Veterans living in a 15-county area of Michigan and Northwest Ohio utilized VAAAHS in fiscal year 2014. The Ann Arbor Medical Center also serves as a referral center for specialty care.
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Gene Therapy and Micro-needle Based Protein Therapy for RDEB - 2014 PCC
The 2014 Patient Care Conference was held at the Gaylord Opryland Resort in Nashville, TN. DebRA invited those with EB as well as their families, advocates, ...
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Rationale for Using Gene Therapy in B-Thalassemia Major and Sickle Cell Anemia
In this exclusive interview, Marina Cavazzana, MD, PhD, of Hpital Universitaire Necker -Enfants Malades, Paris, France, talks about the logic reasoning behi...
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Rationale for Using Gene Therapy in B-Thalassemia Major and Sickle Cell Anemia - Video
Muscular Dystrophy
Video 1: In this film from 1910, a boy demonstrates clinical maneuvers that are still used today in gene-therapy trials for Duchenne #39;s muscular dystrophy. The boy has pseudohypertrophy of...
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By Estel Grace Masangkay
Pfizer announced two major moves that will expand its rare disease R&D activities. First, the company said it will establish a gene therapy platform through its newly inked agreement with Spark Therapeutics. Secondly, it has appointed Dr. Michael Linden, Director of the University College London Gene Therapy Consortium, as head of Pfizers gene therapy research in rare diseases area.
Late-stage gene therapy firm Spark Therapeutics will collaborate with Pfizer to develop SPK-FIX, a program incorporating a bio-engineered AAV vector as potential treatment for Hemophilia B. SPK-FIX is set to begin Phase 1/2 clinical trials in the first half of 2015. Under the terms of the agreement, Spark Therapeutics will stay at the helm of the products Phase 1/2 clinical development studies. Pfizer will take over for pivotal studies, regulatory approvals, and potential commercialization of SPK-FIX worldwide. Pfizer will pay Spark an upfront fee of $20 million with up to $260 million in milestone payments for several hemophilia B product leads that may result from the collaboration. Spark will also be eligible to receive royalties based on global sales of the resulting products.
Geno Germano, group president of Global Innovative Pharma Business at Pfizer, said, We believe the SPK-FIX program could add to our existing portfolio of hemophilia products and could pioneer a potential new treatment technology for patients with bleeding disorders.
We are excited to announce our collaboration with Pfizer, as we believe it marks an important step towards bringing a potentially life-altering therapeutic to patients with hemophilia B, said Jeffrey D. Marrazzo, co-founder and CEO of Spark. The company recently announced that it has received Breakthrough Therapy Designation from the FDA for its lead drug candidate SPK-RPE65 as treatment for nyctalopia in certain patients.
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Pfizer Inks Gene Therapy Partnership With Spark Therapeutics
Gerson: Novel Gene Therapy for Glioma
Stanton Gerson, MD, discusses study findings that looked into a novel genetic therapy for patients with glioma. He presented his study at the American Society of Hematology 2014 meeting in...
By: Cancer Therapy Advisor
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Ethical questions are crucial, but they shouldnt stall the progress of this promising branch of medicine
In late November, Reuters reported a milestone in medical history: a gene therapy drug could go on sale in Germany next year, after winning the approval of European regulators two years ago. The drug, Glybera, by a Dutch firm called UniQure currently being scrutinised by Germanys federal joint committee would be the first commercial use of gene therapy in the Western world (China has had a gene therapy drug for a specific form of cancer in the market since 2004). This marks a potential turning point in an area of medicine that has been the subject of highs and lows over more than two decades of clinical trials.
Gene therapy which can involve a number of things, including replacing a malfunctioning gene or introducing a new gene with the ability to fight a disease has been in conceptual development for far longer. Its origins could be said to go back as early as the 1920s, well before the discovery of the structure of DNA, when a British scientist, Frederick Griffith, put forward what he described as the transforming principle; he successfully converted a non-virulent strain of bacteria into a virulent one, after injecting mice with both.
From the late 1960s, when the concept of gene therapy began to involve, it took several decades for the first clinical trial to take place in 1990. A young girl in the US with a genetic defect that had left her with a severely weakened immune system was successfully injected with her own white blood cells containing a corrected form of the malfunctioning gene.
However, the boost gene therapy got following that first successful trial was soon tarnished, in the view of the public, by a tragedy in 1999; an 18-year-old American boy, who had a mild version of a liver condition, which meant his body couldnt process ammonia, died during a gene therapy treatment. This was after a massive response by his immune system to the vector or carrier used to introduce the corrected gene.
The episode raised a number of issues including that of informed consent of those participating in clinical trials as well as the fact that identifying and correcting a defective gene was far from the only challenge facing gene therapy. Selecting the appropriate vector was also vital and not without risks.
Despite predictions that gene therapy would be lastingly damaged by the tragedy, research and trials continued with many promising results for a range of conditions ranging from immune system conditions to cancer, cystic fibrosis, Parkinsons disease and hemophilia.
The renewed confidence in gene therapy is highlighted by the fact that the worlds largest pharmaceutical companies have also entered the market (earlier this week, Pfizer announced collaboration with Spark Therapeutics, a Philadelphia based company on the development of a hemophilia B treatment).
Over 1,700 approved gene therapy trials have taken place in the past two decades, estimated an article on the history of gene therapy in Gene magazine last year with many successes and a few hits. Among the latter were trials conducted in France in 2001 on Severe Combined Immunodeficiency, a condition where the immune system is so crippled that in one case it required a boy to live in a germ-free bubble. Several infants involved in the trial subsequently developed leukemia, though other clinical trials for gene therapy since have been successful.
There have been some understandable public concerns about gene therapy and its impact on the one hand it offers that tantalising potential of curing some of the most lethal conditions while on the other, tampering with genetic makeup is something that has long conjured up fears in the public imagination of genetic engineering and exacerbating discrimination against those with disabilities and disease.
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Treatment allows scientists to remodel eye cells into light receptors It uses a gene that alters eye cells and an injected chemical 'photoswitch' The photoswitch works with the gene to turn light sensitivity on in cell Blind rescue dogs could see flashing lights after treatment, study says Blind mice became as good at navigating a water maze as normal mice The treatment could be used to help people with retinitis pigmentosa -an inherited condition resulting in progressive loss of sight
By Ellie Zolfagharifard for MailOnline and Press Association
Published: 08:31 EST, 9 December 2014 | Updated: 10:03 EST, 9 December 2014
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A radical form of gene therapy that remodels eye cells into light receptors has allowed scientists to partially restore the sight of animals with inherited blindness.
Scientists say the same technique could one day be used to treat people with retinitis pigmentosa - an inherited condition resulting in progressive loss of sight.
In early tests on blind rescue dogs with a similar condition, showed they could restore sufficient light sensitivity for the animals to distinguish between flashing and non-flashing lights.
In normal mice (left), stimulating the retina produced a variety of responses, as shown by the colours. A similar response was achieved using the radical new therapy in blind mice - as shown in the colourful centre square. The right image reveals the blind mice who had the therapy in different retinal ganglion cells. The results in these types of cells were less dramatic
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Gene therapy that restores sight in mice and dogs could be used on humans
SAN FRANCISCO (TheStreet) -- Bluebird Bio (BLUE) has now treated seven beta-thalassemia patients with its experimental, one-time gene therapy. Four of the patients -- all followed for longer than three months -- are producing enough oxygen-carrying hemoglobin on their own to eliminate the need for chronic blood transfusions.
Two of these super-responding beta-thalassemia patients -- followed for a year and nine months, respectively -- have hemoglobin levels of healthy adults. At this point, a single infusion of Bluebird's gene therapy has essentially cured them of this serious, inherited blood disease.
The remaining three beta-thalassemia patients were infused with Bluebird's gene therapy around one month ago so it's too early to assess their response. A single patient with sickle cell disease was also just treated within the past month.
It's still unreasonable to expect an equivalent strong response in every patient, but Bluebird is learning that as its therapy replaces the defective gene causing beta-thalassemia (or sickle cell disease) with a gene that is fully functional, the patient's ability to produce normal-functioning hemoglobin improves over time, said CEO Nick Leschly.
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Bluebird Gene Therapy Inducing Durable Cures in Blood Disorder Patients
It's been an absolute banner year so far for gene therapy developer bluebird bio Inc. (BLUE: Quote) whose share price has surged more than 130 percent year-to-date.
Before we discuss what's making news at bluebird bio today, here's what "gene therapy" means in simple terms.
Gene therapy is a technique that uses genes to treat or prevent diseases caused by defective or missing genes. This approach helps to address the underlying cause of the disease, rather than offering solutions that focus only on the disease symptoms. Though not a new field, gene therapy is still considered experimental as it faces many technical challenges and concerns regarding its safety.
Did you know the world's first commercial gene therapy was approved in China?
Gendicine, developed by SiBiono GeneTech Co., Ltd., for head and neck squamous cell carcinoma was launched in China in 2004. A year later, another gene therapy product - Oncorine for the treatment of head and neck cancer - was launched, in China, by Shanghai Sunway Biotech Co. Ltd.
Russia launched its first gene therapy drug, Neovasculgen, to treat Peripheral Arterial Disease on the market in 2011.
In the Western world too, there is one approved gene therapy - Glybera - developed by uniQure N.V. (QURE) for adult patients diagnosed with familial lipoprotein lipase deficiency. Approved by the European Commission in November of 2012, Glybera is expected to be launched in Europe this quarter or in Q1, 2015. It is currently not approved for use outside of the European Union.
Now, coming back to today's topic under consideration - bluebird bio - its stock was up more than 43% in Monday's extended trading (Dec.8, 2014), following promising results of its investigational gene therapy LentiGlobin BB305 in patients with beta-thalassemia major, a severe form of beta-thalassemia. The results were presented at the Annual Meeting of the American Society of Hematology yesterday.
Beta thalassemia is an inherited blood disease that reduces the production of hemoglobin. Abnormalities in a gene that is responsible for the production of beta globin (beta-T87Q-globin) causes sickle-cell disease and beta thalassemia. Depending on the severity of symptoms, beta thalassemia is classified as thalassemia major (also known as Cooley's anemia) and thalassemia intermedia.
Beta globin is one of the proteins that make up hemoglobin. Treating beta-thalassemia includes frequent and lifelong blood transfusions, which deliver red blood cells to the body to correct the anemia.
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Gene Therapy a new tool to cure human disease
All the videos uploaded here are collected from different platforms and portals and websites. All these videos are purely for educational purposes. If anyone has problem regarding ownership...
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Research in action at Marquette University | Spinal cord injuries
Dr. Murray Blackmore is an assistant professor of biomedical sciences in the College of Health Sciences at Marquette University. Dr. Blackmore #39;s research focuses on the use of gene therapy...
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Research in action at Marquette University | Spinal cord injuries - Video
LONDON Pfizer Inc. is moving into the gene therapy space in the latest sign that the technology for fixing faulty genes may finally be ready for prime time, following earlier setbacks.
The U.S. drugmaker said on Monday it was establishing a gene therapy platform to study potential treatments, led by a top UK expert, and had struck a deal with privately owned U.S. biotech firm Spark Therapeutics to develop a treatment for hemophilia.
The Spark program is expected to enter early-stage clinical trials for hemophilia B in the first half of 2015. Spark will be responsible for the early Phase I/II tests, with Pfizer taking over late-stage studies, any regulatory approvals and potential commercialization.
Spark will get $20 million upfront and be eligible for additional payments based on product success worth up to $260 million.
Pfizer's research effort in gene therapy will be led by Michael Linden, a professor from King's College London and director of the University College London Gene Therapy Consortium. Linden is joining Pfizer on a two-year secondment.
Gene therapy has seen more than 20 years of experiments but research has been dogged by a series of disappointments and safety concerns.
Now, however, scientists have solved some of the earlier problems and treatments are starting to reach the clinic, with a the Western world's first gene therapy set to go on sale in Germany to treat and ultra-rare blood disease.
"The fundamental understanding of the biology of hereditary rare diseases, coupled with advances in the technology to harness disarmed viruses as gene delivery vehicles, provide a ripe opportunity to investigate the next wave of potential life-changing therapies for patients," said Pfizer research head Mikael Dolsten.
Among other major pharmaceutical companies, Bayer AG struck a gene therapy deal with Dimension Therapeutics in June, while Novartis AG recently established a new cell and gene therapies unit, and Sanofi SA has a long-standing tie-up with Oxford BioMedica.
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