GSK is developing a bone marrow-derived stem cell gene therapy through late phase development and has a small number of collaborations in that field

Uniqure BV, of Amsterdam, the Netherlands, reported that the consortium it coordinates will receive a $3.2 million Eurostars grant to develop an RNA interference gene therapy for Huntington's disease...The goals of the consortium, which includes three European university medical centers, are to develop a regulated gene expression system for glial cell-derived neurotrophic factor to improve the maintenance and survival of neurons as a Huntington's gene therapy and to develop regulated expression of

Earlier, gene therapy firm bluebird bio Inc

The Advanced Therapy Access Program for experimental treatments with the company's oncolytic viruses was made possible through European Union (EU) regulation EC/1394/2007, designed to improve safe access by patients across the EU to gene therapy, somatic cell therapy and tissue-engineered products that were not yet commercially available. (See BioWorld International, Sept. 6, 2006, and Nov. 1, 2006.) Under the experimental program, 290 patients from 20 countries were treated with the company's

On Thursday, two more firms filed S-1s: gene therapy firm bluebird bio Inc...Cambridge, Mass.-based bluebird is looking to raise $86.3 million in its proposed IPO, with proceeds going to fund upcoming trials of its lead gene therapy candidates

Cardium has a Phase III trial under way with the gene therapy Generx (alferminogene tadenovec, Ad5FGF-4) in myocardial ischemia. (See BioWorld Today, March 21, 2012

The RAC review is a required step for a product involving gene therapy vectors, and most of the material will be used in the subsequent investigational new drug application filing

Ceregene Inc., of San Diego, reported top-line data from its double-blind, randomized, controlled Phase IIb study of CERE-120 (AAV-neurturin), a gene therapy designed to deliver the neurotrophic factor neurturin, for Parkinson's disease

The RAC review is a required step for a product involving gene therapy vectors, and most of the material will be used in the subsequent investigational new drug application filing

Proceeds will be used to advance its gene therapy product for congestive heart failure into clinical development

Eyevensys is developing a process for nonviral gene therapy using electrotransfer to enable the prolonged production of therapeutic proteins for ocular disease

Gene therapy Alzheimer's disease Completed enrollment in its Phase II study 3/14/13 Convergence Pharmaceuticals Ltd. (Cambridge, UK) CNV1014802 Sodium channel blocker Trigeminal neuralgia Interim Phase II data showed that in the open-label portion a total of 70% had a successful response and were subsequently randomized into the double-blind treatment period 3/28/13 Lightlake Therapeutics Inc. (London) Intranasal naloxone Opioid antagonist nasal spray Binge eating Phase II data showed a

Neuralgene, of Bogota, Columbia, will begin animal studies of PRCN-829, a gene therapy agent for amyotrophic lateral sclerosis, in May...Early animal studies have shown safety of the AAV9 viral gene therapy platform

Eyevensys is developing a process for nonviral gene therapy using electrotransfer to enable the prolonged production of therapeutic proteins for ocular disease

Gene Therapy – Derisked One of the backers, Genghis Lloyd-Harris, partner at Abingworth in London, who is joining the board of GenSight, agreed with Gilly's assessment that gene therapy in general – and in the eye in particular – has been de-risked

Gene Therapy – Derisked One of the backers, Genghis Lloyd-Harris, partner at Abingworth in London, who is joining the board of GenSight, agreed with Gilly's assessment that gene therapy in general – and in the eye in particular – has been de-risked

had a major setback in the area three years ago with a gene therapy treatment, called Temusi (riferminogen pecaplasmid), which was based on a plasmid encoding fibroblast growth factor 1...In complex indications, such as CLI, cell therapy has theoretical advantages over gene therapy, given its greater biological sophistication

In November 2012, Glybera, developed by Amsterdam, the Netherlands-based uniQure BV, became the first gene therapy approved in a regulated market, for the ultra-rare inherited disorder lipoprotein lipase deficiency. (See BioWorld Today, Nov. 5, 2012.) Glybera is "a gene therapy, it's the same vector we're using and it's the same route of administration," Hawkins pointed out