Category Archives: Gene Therapy

Gene therapy (4 of 5) Created using PowToon -- Free sign up at http://www.powtoon.com/ . Make your own animated videos and animated presentations for free. PowToon is a free to... By: Beau Johanan dela Cruz … Continue reading →

Gene therapy (1 of 5) Created using PowToon -- Free sign up at http://www.powtoon.com/ . Make your own animated videos and animated presentations for free. PowToon is a free to... By: Beau Johanan dela Cruz … Continue reading →

Gene therapy (1 of 4) Created using PowToon -- Free sign up at http://www.powtoon.com/ . Make your own animated videos and animated presentations for free. PowToon is a free tool that allows you to develop... By: Beau Johanan dela Cruz … Continue reading →

Xenon Pharmaceuticals, an early-stage biotech with a gene therapy discovery platform for rare diseases, announced terms for its IPO on Monday. The Burnaby, Canada-based company plans to raise $44 million by offering 4.0 million shares at a price range of $10 to $12. At the midpoint of the proposed range, Xenon Pharmaceuticals would command a market value of $149 million. Xenon has a number of collaboration and licensing agreements with large pharmaceuticals including Teva, Genentech and Merck for its preclinical research.The company's discovery platform was used to develop uniQure's Glybera treatment for orphan disease lipoprotein lipase deficiency, the first gene therapy approved in the EU. February IPO uniQure ( QURE ) priced above its range but ended the first day down 14% and now trades 40% below the IPO price.Xenon is eligible to receive mid single-digit royalties on net sales of Glybera. Teva is in Phase 2 trials for a gene therapy discovered by Xenon that is being developed to treat osteoarthritis. Genentech was cleared to begin a Phase 1 trial for Xenon's pain treatment and Merck is in preclinical development for cardiovascular disease. Xenon also has a variety of preclinical therapies for both orphan diseases (such as Dravet Syndrome) … Continue reading →

Jorge Santiago - UC Berkeley Medical Researcher Jorge works in the Schaffer Lab at UC Berkeley. Studying genetics, he is curious about engineering and modifying genes. Jorge's research is working towards creating vehicles for gene therapy. By: LabTV … Continue reading →

Day 8 Video 6 Gene Therapy By: Amy B Hollingsworth … Continue reading →

Anna David (Prenatal Cell and Gene Therapy, IfWH) UCLTEDx Anna David (Prenatal Cell and Gene Therapy, Research Department of Maternal and Fetal Medicine, IfWH) gives UCLTEDx Women talk. By: UCL EGA Institute for Women's Health … Continue reading →

Gene therapy information By: Alexandra Cook … Continue reading →

PHILADELPHIA In the second of two papers outlining new gene-therapy approaches to treat a rare disease called MPS I, researchers from Perelman School of Medicine at the University of Pennsylvania examined systemic delivery of a vector to replace the enzyme IDUA, which is deficient in patients with this disorder. The second paper, which is published online in the Proceedings of the National Academy of Sciences this week, describes how an injection of a vector expressing the IDUA enzyme to the liver can prevent most of the systemic manifestations of the disease, including those found in the heart. The first paper, published in Molecular Therapy, describes the use of an adeno-associated viral (AAV) vector to introduce normal IDUA to glial and neuronal cells in the brain and spinal cord in a feline model. The aim of that study was to directly treat the central nervous system manifestations of MPS while the more recent study aims to treat all other manifestations of the disease outside of the nervous system. This family of diseases comprises about 50 rare inherited disorders marked by defects in the lysosomes, compartments within cells filled with enzymes to digest large molecules. If one of these enzymes is mutated, … Continue reading →

PUBLIC RELEASE DATE: 29-Sep-2014 Contact: Karen Kreeger karen.kreeger@uphs.upenn.edu 215-349-5658 University of Pennsylvania School of Medicine @PennMedNews PHILADELPHIA In the second of two papers outlining new gene-therapy approaches to treat a rare disease called MPS I, researchers from Perelman School of Medicine at the University of Pennsylvania examined systemic delivery of a vector to replace the enzyme IDUA, which is deficient in patients with this disorder. The second paper, which is published online in the Proceedings of the National Academy of Sciences this week, describes how an injection of a vector expressing the IDUA enzyme to the liver can prevent most of the systemic manifestations of the disease, including those found in the heart. The first paper, published in Molecular Therapy, describes the use of an adeno-associated viral (AAV) vector to introduce normal IDUA to glial and neuronal cells in the brain and spinal cord in a feline model. The aim of that study was to directly treat the central nervous system manifestations of MPS while the more recent study aims to treat all other manifestations of the disease outside of the nervous system. This family of diseases comprises about 50 rare inherited disorders marked by defects in the lysosomes, compartments … Continue reading →