Latest Gene therapy Stories
VANCOUVER, British Columbia, Feb. 5, 2013 /PRNewswire/ -- Xenon, a clinical stage biopharmaceutical company developing novel therapeutics for rare diseases, today announced that it has received a milestone payment from uniQure BV for the European Commission marketing approval of Glybera®, a novel gene therapy treatment for the orphan disease lipoprotein lipase deficiency (LPLD), and the first gene therapy approved in the Western world. Xenon exclusively licensed to uniQure BV its...
CINCINNATI, Feb. 4, 2013 /PRNewswire-USNewswire/ -- Researchers have overcome a major challenge to treating brain diseases by engineering an experimental molecular therapy that crosses the blood-brain barrier to reverse neurological lysosomal storage disease in mice. (Logo: http://photos.prnewswire.com/prnh/20110406/MM79025LOGO) Posted online in PNAS Early Edition on Feb. 4, the study was led by scientists at Cincinnati Children's Hospital Medical Center. "This study provides a...
AMSTERDAM, January 22, 2013 /PRNewswire/ -- uniQure B.V., the leader in the field of human gene therapy, today announced the appointment of Philip Astley-Sparke, former President and CEO of BioVex, as President US to provide strategic leadership and help build uniQure's clinical, regulatory, and commercial infrastructure in the US. Since May 2012, Philip is a venture partner at Forbion Capital Partners, uniQure's largest investor. As a venture partner he works with...
April Flowers for redOrbit.com - Your Universe Online Jumping genes, or transposable elements, are strange rogue sequences from which most of our DNA can be traced, but are largely idle in mammals. Johns Hopkins researchers report, however, that they have identified a new DNA sequence moving around in bats. This is the first member of the transposable elements class found to be active in mammals. The findings of this study, published online at the Proceedings of the National Academy of...
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...
University of Pennsylvania Researchers Report on Results of Trial in 12 Patients, Including Two Children ATLANTA, Dec. 9, 2012 /PRNewswire-USNewswire/ -- Nine of twelve leukemia patients who received infusions of their own T cells after the cells had been genetically engineered to attack the patients' tumors responded to the therapy, which was pioneered by scientists in the Perelman School of Medicine at the University of Pennsylvania. Penn Medicine researchers will present the...
AMSTERDAM, The Netherlands, December 6, 2012 /PRNewswire/ -- uniQure B.V., a leader in the field of human gene therapy, today announced the appointment of Hans Christian Rohde as Chief Commercial Officer. Mr. Rohde joins uniQure from Basilea Pharmaceutica (SIX: BSLN) where from 2007 he was Chief Commercial Officer and member of the company's executive management committee with responsibility for global commercial operations, marketing, supply chain, medical affairs,...
AMSTERDAM, The Netherlands, December 5, 2012 /PRNewswire/ -- uniQure B.V., a leader in the field of human gene therapy, today announced a non-exclusive cross-licensing agreement with Benitec Biopharma Ltd. (ASX: BLT) giving uniQure access to Benitec's proprietary DNA-directed RNA interference (ddRNAi) technology in Huntington's disease. In return, uniQure granted Benitec non-exclusive access to the Company's AAV5 delivery technology for the development of a ddRNAi therapy for...
GULF SHORES, Ala., Dec. 4, 2012 /PRNewswire/ -- More than 75 percent of amyotrophic lateral sclerosis (ALS) patients who received neural reprogrammed stem cell therapy have shown a positive response to the procedure. The groundbreaking technique was introduced to the United States this year by Precision StemCell (http://www.precisionstemcell.com), an outpatient imaging and image-guided treatment facility located in Gulf Shores, Ala. The procedure is performed by Dr. Jason R....
SUNNYVALE, Calif., Nov. 27, 2012 /PRNewswire/ -- Amarantus BioSciences, Inc. (OTCQB: AMBS), a biotechnology company developing new disease-modifying treatments and diagnostics for Parkinson's disease and Traumatic Brain Injury centered on its proprietary anti-apoptosis therapeutic protein MANF, today announced that the Company has successfully completed experiments demonstrating that MANF has an excellent diffusion profile in the striatum of rat brains. The striatum is located in the...
