Category Archives: Gene Therapy

HGF properties gene therapy By: Sumin Sin … Continue reading →

Contact Information Available for logged-in reporters only Newswise Study results of CD19-directed chimeric antigen receptor (CAR) therapy using the Sleeping Beauty non-viral transduction system to modify T cells has demonstrated further promise in patients with advanced hematologic malignancies. Patients who had acute lymphocytic leukemia (ALL), non-Hodgkin lymphoma (NHL) or chronic lymphocytic leukemia (CLL) were part of clinical trials at The University of Texas MD Anderson Cancer Center, which used the Sleeping Beauty gene transfer system initially discovered at the University of Minnesota. Results from the study were presented at the 56th Annual Meeting of the American Society of Hematology (ASH) annual conference in San Francisco and were published in the Dec. 5 issue of the ASH journal Blood. The Sleeping Beauty gene was named for its ability to awaken an extinct transposon DNA that can replicate itself and insert the copy back into the genome. This allows a gene to be transferred into a DNA molecule known as a plasmid. An enzyme called a transposase binds to the plasmid, cuts the transposon and gene out of the plasmid and pastes it into the target DNA sequence. This gene transfer system was the basis for the MD Anderson clinical trials. Using … Continue reading →

Gene Therapy: A Misunderstood Science Blue 4's Breaking Boundaries final submission. 4 min video about gene therapy. By: Bethany M Slingsby … Continue reading →

EPIGENETIC GENE THERAPY ON THE KETOGENIC DIET How to get ripped by changing your genes! WEBSITE: http://www.stephanieperson.com FB: https://www.facebook.com/pages/Stephanie-The-Business-Person/604522012970143?ref=hl INSTAGRAM: ... By: stephanie Keto person … Continue reading →

HOW TO LOSE WEIGHT: by using your emotions the ketogenic diet Strengthen you genetics to lose weight through epigenetic gene therapy WEBSITE: http://www.stephanieperson.com FB: ... By: stephanie Keto person … Continue reading →

#NotImmune - Alliance for Cancer Gene Therapy Alliance for Cancer Gene Therapy funds research to: Attack the cancer, not the patient. http://www.acgtfoundation.org/notimmune ACGT is the nation's only nonprofit dedicated exclusively to funding... By: ACGTFoundation … Continue reading →

A gene therapy spinout from Childrens Hospital of Philadelphia has added a head of regulatory affairs as it continues Phase 3 clinical trials for its treatment to reverse the effects of inherited retinal degeneration caused by a mutation in the RPE65 gene. It is the latest in a series of additions to the company following a $72.8 million Series B fundraise earlier this year. Daniel Takefman previously worked for eight years as chief of the gene therapy branch at the Center for Biologics Evaluation and Research at the U.S. Food and Drug Administration, according to a company statement. Spark Therapeutics, headed by CEO Jeffrey Marrazzo, has been bulking up its management muscle this year. Dr. Katherine High, the former founding director of the Center for Cellular and Molecular Therapeutics at CHOP and a pediatrician at the Perelman School of Medicine, became president and Chief Scientific Officer and has a board seat. It added Stephen Webster as CFO from Optimer Pharmaceuticals and Adolor Corp, whic Cubist Pharmaceuticals acquired in 2011. Steven Gelone joined as head of clinical research and development. Gelone had previously worked for ViroPharma in a similar role Shire acquired the company last year. Diane Blumenthal, another industry veteran, … Continue reading →

Meet Shoukhrat Mitalipov He's using breakthrough gene therapy techniques to cure disease. Learn more at http://ohsu.info/LNCf. By: OHSUvideo … Continue reading →

The western worlds first gene therapy drug is expected to go on sale in Germany next year. Photograph: Eliseo Fernandez/Reuters The western worlds first gene therapy drug is set to go on sale in Germany, with a price tag that could amount to an 870,000 cost to treat a single patient. Glybera, a treatment for the rare genetic condition lipoprotein lipase deficiency (LPLD), which clogs the blood with fat, has been developed by Dutch biotech firm UniQure and Italian marketing marketing partner Chiesi. It is undergoing an assessment of benefits by Germanys federal joint committee, which will report by April 2015. But the company is seeking a retail price of 53,000 (42,000) per phial, which equates to 1.1m (870,000) for a course of treatment for a typical LPLD patient. This price will be subject to a discount under Germanys drug pricing system. A Chiesi spokeswoman confirmed the launch price and added that a final figure would be set after the German authorities gave their verdict and negotiations are held with health insurance funds. First commercial treatments are expected in the first half of 2015, she said. UniQure, which will get a net royalty of between 23% and 30% on sales, … Continue reading →

London - The Western world's first gene therapy drug is set to go on sale in Germany with a 1.1m ($1.4m) price tag, a new record for a medicine to treat a rare disease. The sky-high cost of Glybera, from Dutch biotech firm UniQure and its unlisted Italian marketing partner Chiesi, shows how single curative therapies to fix faulty genes may upend the conventional pharmaceutical business model. After a quarter century of experiments and several setbacks, gene therapy is finally throwing a life-line to patients by inserting corrective genes into malfunctioning cells - but paying for it poses a challenge. The new drug fights an ultra-rare genetic disease called lipoprotein lipase deficiency (LPLD) that clogs the blood with fat. The medicine was approved in Europe two years ago but its launch was delayed to allow for the collection of six-year follow-up data on its benefits. Now Chiesi has filed a pricing dossier with Germany's Federal Joint Committee, or G-BA, which will issue an assessment of the drug's benefits by the end of April 2015. The company is seeking a retail price of 53 000 per vial, or 43 870 ex-factory. That equates to 1.11m for an typical LPLD patient, averaging … Continue reading →