The LentiVector® Gene Therapy Platform for Ocular Disease: A Clinical Update Yatish Lad, Kyriacos Mitrophanous, Scott Ellis, James Miskin, Katie Binley, Michelle Kelleher, Cherry Lucas, Stuart Naylor. Oxford BioMedica (UK) Ltd, Oxford, United Kingdom Oxford BioMedica has developed ocular gene therapies using its proprietary LentiVector® platform which is based on recombinant Equine Infectious Anaemia Virus (EIAV). Currently we have three ocular therapies in clinical development: RetinoStat®, StarGen™ and UshStat®, for the treatment of age-related macular degeneration, Stargardt macula dystrophy and Usher Syndrome 1B respectively. Clinical evaluations are underway in the US for a Phase I RetinoStat®, US/France for a Phase I/IIa StarGen™ and the US for a Phase I/IIa UshStat® study. The RetinoStat®, StarGen™ and UshStat® trials consist of a dose-escalation phase followed by an expanded final cohort at the highest safe and tolerated dose. Safety and signs of clinical benefit will be assessed throughout these trials. The dose-escalation phase of the RetinoStat® trial is complete whilst this phase is in progress for StarGen™ and UshStat®. Subretinal administration of all three products has been safe and well tolerated, causing no ocular inflammation or immune responses in any patients and there have been no SAEs related to the products. In the RetinoStat® trial, the secreted nature of the endostatin and angiostatin transgenes has meant that expression of these proteins could be quantified over time in each patient from aqueous tap samples. Transduction of the retina following subretinal injection of RetinoStat® produced both proteins that were detectable in the aqueous humour at a dose dependent level that increased over time in each patient. In summary, ocular gene therapies based on the LentiVector® platform continue to show good safety following subretinal delivery into patients for three different ocular indications. The platform has proved to be a highly effective delivery system for relatively large genes into target retinal cells, resulting in stable and long-term expression. Keywords: Eye Diseases; Clinical Gene Therapy; Lentivirus Vectors Session: Poster Session: Sensory (Ophthalmic and Auditory) Gene & Cell Therapy (4:00 PM-6:00 PM) Date/Time: Thursday, May 16, 2013 - 4:00 PM Room: Exhibit Hall C/D http://www.abstracts2view.com/asgct/view.php?nu=ASGCT13L1_232&terms= Good luck and GOD bless, George
http://www.abstracts2view.com/asgct/view.php?nu=ASGCT13L1_56 Glaucoma-GT, a Novel Gene Therapy Treatment for Primary Open-Angle Glaucoma Katie Binley, Scott Ellis, Vicky Scripps, Sharifah Iqball, Stuart Naylor, Kyriacos Mitrophanous. Oxford BioMedica (UK) Ltd, Oxford, United Kingdom Glaucoma is the second leading cause of blindness worldwide, affecting around 70 million people, over 2.5 million people in the USA alone. It is characterized by irreversible degeneration of the optic nerve, usually associated with an elevated intraocular pressure (IOP). Prostenoid analogues such as Latanoprost are a first-line treatment for glaucoma due to their high level of efficacy and low risk of side-effects, but fail to halt disease progression in many patients due to non-adherence, which can be > 50%. Surgery is often required, which is expensive and only partially effective. There is therefore a real medical need for a novel drug that overcomes this issue of poor compliance. Initial proof-of-concept for this approach has been demonstrated (Barraza et al, 2010), and we are now moving this into a translational gene therapy platform for clinical evaluation. Glaucoma-GT is a gene therapy product aimed at lowering IOP via the prostenoid pathway: a single transcorneal administration leads to the expression of human cyclooxygenase-2 (COX-2) and prostaglandin F (FP) receptor in the front of the eye. COX-2 is a rate-limiting enzyme in the biosynthesis of prostaglandins, and both COX-2 and the FP receptor are down-regulated in glaucomatous eyes. Expression of both genes increases both prostaglandin 2α (PGF2α) biosynthesis and signaling, increasing aqueous outflow and reducing longterm IOP. Gene transfer following transcorneal administration of EIAV-GFP vector was characterised in animal models. Various vector genome configurations were assessed for the production of PGF2α and activation of the FP receptor in in vitro assays. Further preclinical studies are currently ongoing and data from these will be presented. Transcorneal injection of EIAV vector led to the transduction of cells of the trabecular meshwork and corneal endothelium in vivo. Different configurations of expression cassette gave a broad range of PGF2α and FP activation, with CMV-COX-2-IRES-FP having the highest activity. We have demonstrated significant gene transfer following transcorneal administration of EIAV-GFP vector in animal models, and have optimised the therapeutic expression cassette to produce high levels of prostaglandin 2α and enhanced FP activation in in vitro studies. These vector configurations will be assessed for IOP lowering in a relevant animal model. Keywords: Eye Diseases; Lentivirus Vectors; Gene Correction/Modification Session: Simultaneous Oral Abstract Sessions: Sensory (Ophthalmic and Auditory) Gene & Cell Therapy (4:20 PM-5:20 PM) Date/Time: Wednesday, May 15, 2013 - 5:05 PM Room: 150 ABC Good luck and GOD b
Yet again , many thanks for all your hard work and hopefully the future looks better for all the sufferers out there.
2013 Annual General Meeting (‘..
http://www.oxfordbiomedica.co.uk/uploads/meetings/2013-oxb-notice-of-agm-2013.pdf Good luck and GOD bless, George
Does OXB's gene therapy work?
Watch these three different videos and tell me if you think that Oxford Biomedica's gene therapy works http://www.youtube.com/watch?v=pTQuZ2hWa-0 http://www.youtube.com/watch?v=yOoO2mwE8ro http://news.sky.com/story/9567/revolutionary-new-treatment-for-parkinsons Good luck and GOD bless, George
What is all this about ? George has been posting and giving all the latest information so why as you put it is " all this school yard name calling " going on ? You are new to this board and it is thanks to George that this board is " alive ". I clicked on the link you gave and it does not take much to realise why George did not answer to whoever wrote that post. God bless................ ( are you going to put the same question to me ?
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