June 2013
Volume 54, Issue 15
ARVO Annual Meeting Abstract  |   June 2013
The LentiVector® Gene Therapy Platform for Ocular Disease: a clinical update
Author Affiliations & Notes
  • Kyriacos Mitrophanous
    Oxford BioMedia (UK) Ltd, Oxford, United Kingdom
  • Scott Ellis
    Oxford BioMedia (UK) Ltd, Oxford, United Kingdom
  • James Miskin
    Oxford BioMedia (UK) Ltd, Oxford, United Kingdom
  • Katie Binley
    Oxford BioMedia (UK) Ltd, Oxford, United Kingdom
  • Michelle Kelleher
    Oxford BioMedia (UK) Ltd, Oxford, United Kingdom
  • Cherry Lucas
    Oxford BioMedia (UK) Ltd, Oxford, United Kingdom
  • Stuart Naylor
    Oxford BioMedia (UK) Ltd, Oxford, United Kingdom
  • Footnotes
    Commercial Relationships Kyriacos Mitrophanous, Oxford BioMedica (UK) Ltd (E); Scott Ellis, Oxford BioMedica (E); James Miskin, Oxford BioMedica (UK) Ltd (E); Katie Binley, Oxford BioMedica (UK) Ltd (E); Michelle Kelleher, Oxford BioMedica (UK) Ltd (E); Cherry Lucas, Oxford BIoMedica Ltd (C); Stuart Naylor, Oxford BioMedica (UK) Ltd (E)
  • Footnotes
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Investigative Ophthalmology & Visual Science June 2013, Vol.54, 2744. doi:
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      Kyriacos Mitrophanous, Scott Ellis, James Miskin, Katie Binley, Michelle Kelleher, Cherry Lucas, Stuart Naylor; The LentiVector® Gene Therapy Platform for Ocular Disease: a clinical update. Invest. Ophthalmol. Vis. Sci. 2013;54(15):2744.

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      © ARVO (1962-2015); The Authors (2016-present)

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Purpose: 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. Regulatory approval in the US and France has been received for RetinoStat® and StarGen™, and UshStat® has been approved in the US. Phase I (RetinoStat®) and Phase I/IIa (StarGen™ and UshStat®) clinical evaluations are currently underway.

Methods: 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. Additionally, the secreted nature of the transgenes in the RetinoStat® trial has meant that transgene expression from aqueous tap samples can also be quantified over time at each dose.

Results: The dose-escalation phase of the RetinoStat® trial has been completed. For StarGen™ and UshStat® the dose-escalation phase is in progress. Subretinal administration of all three products has been well tolerated, causing no ocular inflammation or immune responses in any patients. There have been no SAEs related to product. All three products have been safe and well tolerated thus far. Transduction of the retina following subretinal injection of RetinoStat® rapidly produced high levels of both transgenes detectable in the aqueous. These levels are substantial, show dose dependence.

Conclusions: 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: 466 clinical (human) or epidemiologic studies: treatment/prevention assessment/controlled clinical trials • 494 degenerations/dystrophies • 538 gene transfer/gene therapy  

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