Gene Therapy in Ophthalmology Update 13: New Clinical Site for Usher Syndrome Clinical Trials

In joint news releases, both the Foudation Fighting Blindness (FFB) and Oxford BioMedica announced that FFB had recently awarded $125,000 to the Centre Hospitalier National d'Ophtalmologie des Quinze-Vingts, a clinical trial site for UshStat, a gene therapy developed by Oxford BioMedica for people with Usher syndrome type 1B (USH1B). The funding level is the amount needed for the site, located in Paris, France, to take part in the study.

Usher syndrome is the world's leading cause of combined blindness and deafness. The condition affects approximately 45,000 people in the United States. USH1B is a form of the disease caused by defects in the gene MY07A. The UshStat treatment replaces copies of the defective MY07A gene in the retina with normal copies. Researchers believe a single treatment may halt the disease for several years, perhaps a lifetime. There are currently no therapies available for USH1B.

UshStat is a novel gene-based treatment for Usher syndrome type 1B, designed and developed by Oxford BioMedica using its proprietary LentiVector platform technology, and is the third ocular product to enter clinical development under the Company's collaboration with Sanofi. In February 2012, Oxford BioMedica initiated an open label, dose escalation Phase I/IIa study in the US led by Professor Richard Weleber at the Oregon Health & Science University's Casey Eye Institute.  The ongoing study will enroll up to 18 patients and will evaluate three dose levels for safety, tolerability and aspects of biological activity. Initial safety data from this study are expected in H2 2012.

Oxford BioMedica plans to open the second clinical site at Centre Hospitalier National d'Ophtalmologie des Quinze-Vingts in Paris, France with Professor José-Alain Sahel as Principal Investigator. Subject to receiving regulatory approval, the second clinical site is anticipated to open in early 2013, from which point patients could be treated in parallel at both sites.

The study is scheduled to run through December 2014. Evaluating safety is the primary goal of the trial, though investigators will also be looking at the treatment's effect on the retina and vision.

Oxford BioMedica is also conducting clinical trials of RetinoStat, a gene therapy for wet age-related macular degeneration, and StarGen, a gene therapy for Stargardt disease. The latter clinical trial is also being conducted at both OHSU and at Centre Hospitalier National d'Ophtalmologie des Quinze-Vingts in Paris.

"We are very pleased to be supporting the UshStat trial in Paris," said Dr. Stephen Rose, chief research officer, Foundation Fighting Blindness. "Led by Dr. José Sahel, the Paris team is one of the best retinal research groups in the world. They will do an outstanding job evaluating UshStat's potential for saving vision in people affected by a particularly challenging condition."

Professor José-Alain Sahel of Centre Hospitalier National d'Ophtalmologie des Quinze-Vingts Paris, France commented: "We are very grateful to FFB for this support and are committed to testing this innovative approach with our partners, such as Pr Christine Petit (Pasteur Institute) who first identified disease-causing mutations in this gene. Our collaboration with Oxford BioMedica, the Portland team and FFB, already implemented with the ongoing StarGen trial, holds major promise for our patients."

Stuart Naylor, Chief Scientific Officer of Oxford BioMedica, said: "Having worked with FFB since 2003, our strong relationship has underpinned the success we have had in bringing novel ocular gene therapies into clinical development. With no available treatments for patients with Usher syndrome type 1B, we are grateful for FFB's support to boost this program with an additional clinical site."

Gene Therapy in Ophthalmology Update 12: First Gene Therapy Approval on the Horizon

As Andrew Pollack writes in today’s NYTimes, “After more than two decades of dashed expectations, the field of gene therapy appears close to reaching a milestone: a regulatory approval. The European Medicines Agency has recommended approval of a gene therapy to treat a rare genetic disease.”

The therapy recommended for approval in Europe, called Glybera, was developed by uniQure, a Dutch company. It treats lipoprotein lipase deficiency, a disease that affects only several hundred people in the European Union and a similar number in North America.

People with the disease have a genetic mutation that prevents them from producing an enzyme needed to break down certain fat-carrying particles that circulate in the bloodstream after meals. Without the enzyme, so much fat can accumulate that the blood looks white rather than red.

Glybera provides correct copies of the lipoprotein lipase gene, which allows patients to make some of the needed enzyme. A single treatment, consisting of injections into multiple spots on the leg muscles on the same day, is expected to last for several years, if not longer, said Jörn Aldag, chief executive of uniQure.

The reason I believe that this is important is because it brings “legitimacy” to the whole field of regenerative medicine. As readers of this online Journal are aware, my interest is in the field of ophthalmology. As you may be further aware, I am currently tracking eleven clinical trials involving the use of stem cells to treat ophthalmic disorders and sixteen gene therapy clinical trials. Several of these are showing promising results and the above approval, when it comes, will bring increased attention to the whole of this field, including the ophthalmic trials.

Anyone interested in obtaining my latest tables of ongoing clinical trials in either or both areas, please use the “Email Me” link to the right to request copies.