To read more, please follow this link.

The company then announced that it had received authorization from the U.S. Food and Drug Administration (FDA) for a Phase I/II clinical trial of the company’s proprietary HuCNS-SCr product candidate (purified human neural stem cells) in dry age-related macular degeneration (AMD), the most common form of AMD.

We believe that at least one of the sites for the clinical trial will be the Casey Eye Institute at Oregon Health and Science University.

To read the complete story, please follow this link.

The results have been published in the journal, Proceedings of the National Academy of Sciences.

To read the full story, please follow this link.

The trial is aimed at patients with Usher Syndrome Type 1B, a form or retinitis pigmentosa that leads to blindness and hearing loss.

There is currently no treatment for this disease.

Based on laboratory pre-clinical studies, the researchers believe that a single UshStat treatment may last for several years, and perhaps for a lifetime.

In addition, Oxford BioMedica issued an update on all of its ophthalmic gene therapy programs, as part of its year-end report.

To read the full article, please follow this link.

A new start-up company is looking into the possibility of certain compounds that appear to boost mitochondrial function to provide “energy” to “reduced capacity” mitochondria in retinal cells that are possibly at the root of retinal degenerations in such diseases as retinitis pigmentosa and macular degeneration.

Two scientists, Drs. Rohrer and Beeson, of the Ophthalmology department at the Medical University of South Carolina have started a new company, MitoChem Therapeutics, that has just received $2 million from the Foundation Fighting Blindness to do just that. After screening a library of 50,000 drug compounds, they have apparently identified three compounds that appear to boost mitochondrial function, and will now attempt to identify which one will work best in people as an eye drop, and move it into a clinical trial.

To read more, please follow this link.

Here is how I commented about this breakthrough in my writeup:

“If this approach is successful, it could result in a “game change” in the way wet AMD is treated. Instead of multiple injections to gain control of neovascularization, one injection at the first signs of neovascularization could stop it dead in its tracks and the effect could last, potentially, for several years.

In addition, it is conceivable that patients at risk of developing wet AMD could receive a therapeutic injection of AVA-101 to prevent the occurrence of wet AMD. This would be a definite paradigm shift in the treatment of AMD.

With the human clinical testing now underway, we should have an indication of initial results with this unique approach within about a year.

Stay tuned!”

In a nutshell, Avalanche has developed a proprietary drug delivery system, known as Ocular BioFactoryTM, that continuously secretes a therapeutic protein over an extended period following a single subretinal injection, thus avoiding the need for frequent intraocular injections of recombinant anti-VEGF protein.

To read more about this potential “game changing” breakthrough in the future treatment of wet AMD, please follow this link.

As noted by both Drs. Phil Rosenfeld and Carmen Puliafito, who have used the new software, this new diagnostic tool will play an important role in assessing and speeding the development of  new treatments for both dry and wet AMD, that are being researched and brought to the market.

To read the full story, please follow this link.

First, the company said that a peer-reviewed publication of clinical results from its first patients treated at UCLA Jules Stein Eye Institute had been published online by the UK’s The Lancet. The study reported on the four-month results of a safety study initiated in human patients last July. In that study, one eye of a patient with Stargardt’s macular dystrophy (SMD), and another with the dry form of AMD were given doses of human embryonic stem cell-derived retinal pigment epithelial (RPE) cells. After four months, no safety problems had been observed and vision improvements were noted in both patients.

The second event was the announcement that the first patient had been treated with stem cells in the UK arm of the Stargardt’s study, at Moorfields Eye Hospital on January 20th.

For the whole story, please follow this link.

I have put together a table in which I list the fourteen clinical trials that I know about in the use of gene therapy in treating ophthalmic disease. Half of the trials are aimed at treating Leber’s Congenital Amaurosis (LCA), while three are for treating the wet form of AMD; one is underway for treating Choroideremia; one for Stargardt’s Disease; and two are aimed at different forms of retinitis pigmentosa (Autosomal Recessive RP and Usher Syndrome 1b).

In addition, I show at least twenty four clinical trials in either the pre-clinical (animal study) mode, or a couple in the IND-preparation mode. That is close to forty clinical trials using gene therapy to treat ophthalmic diseases.

To read more about this important method for treating ophthalmic diseases, please follow this link.

Cenecor (J&J)  has an active trial for Dry AMD underway at both Retina Institute of CA and at Wills Eye.

There are several trials looking at the corneal surface – one by CellSeed France (being done in Germany); one by a Spanish Clinic at three hospitals in Spain; and two at Japanese University clinics. And finally, there is one trial underway looking at optic nerve atrophy in China.

To see the complete list please follow this link.