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UCL Gene Therapy Trial shows Benefit for up to Three Years After Treatment

Tue, 05 May 2015 14:44:39 +0000

We are delighted to be able to announce that yesterday, Monday 4th May, the long-term results of our RPE65 gene therapy trial for Leber Congenital Amaurosis Type 2 (LCA2) were published in the prestigious New England Journal of Medicine. Begun in 2007, this was the world’s first-in-human trial of gene therapy to treat an inherited […]

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UCL researchers solve a major riddle of retinal degeneration research!

Mon, 26 Jan 2015 10:11:36 +0000

Today a paper published in Nature Communications from the Gene and Cell Therapy Group at the UCL Institute of Ophthalmology has shed light on why, until now, it has not been possible to effectively restore vision in rd1 mice – the world’s major model for retinitis pigmentosa (RP). The rd1 mouse is a model of […]

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Professor Ali honoured for his contribution to research into retinal disease

Mon, 08 Sep 2014 12:27:36 +0000

              Professor Robin Ali, PhD, Professor of Human Molecular Genetics and Head of the Department of Genetics, UCL Institute of Opthalmology has been awarded the Pioneer Award for his work in proof-of-concept studies that have demonstrated the feasibility of using gene therapy and cell transplantation to treat dysfunction and degeneration of the cells […]

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Gene and Cell Therapy Group

Developing gene and cell therapy for sight loss by translating basic research into new treatments


UCL Gene Therapy Trial shows Benefit for up to Three Years After Treatment

NEJM 2015 COver

We are delighted to be able to announce that yesterday, Monday 4th May, the long-term results of our RPE65 gene therapy trial for Leber Congenital Amaurosis Type 2 (LCA2) were published in the prestigious New England Journal of Medicine.

Begun in 2007, this was the world's first-in-human trial of gene therapy to treat an inherited form of blindness. Twelve patients were enrolled in the trial over the course of six years and followed up over a three year period to assess the long-term safety and benefit of treatment with gene therapy in this Phase I/II clinical trial.

A number of patients enrolled in the trial experienced gains in night vision for a period of two to three years with greatest improvements seen in the first 6 to 12 months after treatment. This is consistent with the published results and interim findings of other studies of RPE65 gene therapy.

This study confirms our preliminary findings (published in NEJM, 2008) that gene therapy can improve night vision, providing further evidence of benefit in inherited blindness.

Professor James Bainbridge, lead clinician for the trial

Our latest results provide confirmation of efficacy but the data, together with results of a parallel study in dogs, indicate that the demand for RPE65 is not fully met with the current generation of vectors. We have concluded that early intervention using a more potent vector, expressing higher levels of RPE65 is likely to provide greater benefit and protection against progressive degeneration.

Professor Robin Ali, lead for the research group

The group has now developed a new, more powerful gene therapy vector and is aiming to test this in a second clinical trial funded by The UK Medical Research Council.


Video of trial participant with improved night vision as a result of gene therapy


Key Media Coverage

BBC

Link to article

New NH Voice

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MedPageToday

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Science aaas

Link to article


Data

  • The full results from this study can be found in the NEJM:
  1. Bainbridge, JWB, Mehat MS, Sundaram V, et al. Long-term Effect of Gene Therapy on Leber Congenital Amaurosis. New England Journal of Medicine. 2015;10.1056/NEJMoa1414221
  2. Bainbridge, JWB, Smith AJ, Barker SS, et al. Effect of Gene Therapy on Visual Function in Leber's Congenital Amaurosis. New England Journal of Medicine. 2008; 358: 2231-9



We are also conducting a second clinical trial:

Phase I trial to determine the safety of transplanting stem cell-derived retinal cells

We are conducting Europe's first clinical trial looking at the safety of injecting retinal cells, derived from embryonic stem cells, into the eyes of patients with Stargardt macular dystrophy.

Stargardt Disease
White flecks as seen in Stargardt disease


The cells being injected are retinal pigment epithelium (RPE) cells that were grown from human embryonic stem cells in a dish, by the American company Advanced Cell Technology Inc.

This trial involves patients who are already severely visually impaired and is designed to show whether the injection of these cells is safe - future studies will be required to determine if cell transplantation can restore sight.


Other research highlights

UCL researchers solve a major riddle in retinal degeneration research


Rd1 mouse retina treated with PDE6B gene therapy

Today a paper published in Nature Communications from the Gene and Cell Therapy Group at the UCL Institute of Ophthalmology has shed light on why until now it has not been possible to effectively restore vision in rd1 mice, the world’s major model for retinitis pigmentosa.


Page last modified on 05 may 15 12:08