Category Archives: Gene Therapy

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... By: Rare Disease Report … Continue reading →

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 … Continue reading →

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 … Continue reading →

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 … Continue reading →

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 … Continue reading →

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 22 shares 11 View comments 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 … Continue reading →

Gene Therapy Infomercial Song- Take The Plunge Heinz Ketchup ops I mean kiessling Voiced by Jesse W., Nestor F., Zachary E., and Zachary P. By: Nestor Falcon … Continue reading →

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's research focuses on the use of gene therapy... By: MarquetteU … Continue reading →

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. More: Bluebird Gene Therapy Inducing Durable Cures in Blood Disorder Patients … Continue reading →

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 … Continue reading →