Saturday, September 15, 2012

Menu 21: A List of Writeups on Gene Therapy Used in Ophthalmology

As with my menu on stem cells used in ophthalmology (Menu 20), here is one for the current articles on the use of gene therapy in ophthalmology, with links to the full writeups.

I have repeated the offer for my tables showing the companies and institutions involved, as well as all of the current clinical trials, at the end.

Gene Thearpy

After several discussions with Sean Ainsworth, the founder of RetroSense, and much online research, I think I have learned a little about what gene therapy is about, and its application in ophthalmology, especially in the possible restoration of vision in those who suffer from retinitis pigmentosa (RP). Thanks to Sean for whetting my appetite -- here is what I have learned.

In an announcement today, Oxford BioMedica said that it had gained approval from the FDA to begin a Phase I/IIa Clinical Trial for a form of Usher’s Syndrome, Type 1B, which leads to progressive retinitis pigmentosa combined with a congenital hearing defect.

In a news release that I found on the net, I learned that the Foundation Fighting Blindness was going to put $8.25 million into six gene therapy projects, either already underway or about to start. The release contains good information about several projects that I knew about, and others that I did not.

In  another of the presentations made during the Retina SubSpecialty Day Meeting, Dr. Stephen Tsang presented on factors and the genetics of retinitis pigmentosa. His paper was based on the article previously published by he and his co-author, Kyle Wolpert, that appeared in the November 2010 issue of Retinal Physician.

Gene Therapy in Ophthalmology Update 4: Table of Companies and Institutions Participating

Again, this table is currently out-of-date. Contact me via email to receive current versions of the tables listed at the end of this menu.

A writeup about a start-up company, Hemera Biosciences, with a gene therapy approach to treating dry AMD.

Thanks to my friends at the Foundation Fighting Blindness, I learned about this first human clinical trial using gene therapy for treating recessive retinitis pigmentosa.

In this opus, I discuss my reasons why I think 2012 is going to be the year for gene therapy and also presented my table of current clinical trials underway. (Again, note there is now an updated table available via email.)

A report on the progress being made in treating Leber’s using gene therapy, as reported by the Foundation Fighting Blindness.

Gene Therapy in Ophthalmology Update 9: Oxford BioMedica/OHSU Preparing to Treat First Usher Syndrome Patient & Oxford BioMedica Ophthalmic Program Update

A report on the start of the program to treat Usher Syndrome patients at OSHU, and an update on other ophthalmic programs underway by Oxford BioMedica.

Gene Therapy in Ophthalmology Update 10: Gene Therapy Research in Dogs Cures X-Linked Retinitis Pigmentosa – Paves the Way for Similar Treatment in Humans

Researchers at several universities and laboratories collaborated to treat dogs afflicted with the x-linked form of retinitis pigmentosa, to deliver the therapeutic RPGR gene specifically to the diseased rods and cones, which led to functional and structural recovery. This is the first proof that this condition is treatable in an animal model and the researchers feel the results are promising and relevant for translation to humans afflicted with this disease.

Gene Therapy in Ophthalmology Update 11: Clinical Trial Details
In attempting to determine how many patients had been treated with gene therapy for eye disorders, I quickly found that no one was keeping track – at least no one that I could find.

So, I decided to try and get this data. I have now found reliable data for more than two-thirds of the 16 clinical trials underway and present this information in my new table. My latest table (by email request) contains all of the newest data.

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.

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.

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

The Foundation Fighting Blindness and Oxford BioMedica announced funding for a second clinical site to conduct a gene therapy trial for Ushers Syndrome. The site will be the Centre Hospitalier National d'Ophtalmologie des Quinze-Vingts in Paris, and will join the ongoing clinical trial being held at the Oregon Health & Science University's Casey Eye Institute.

Gene Therapy in Ophthalmology Update 14: Early Positive Results in Ongoing Gene Therapy Wet AMD and Stargardt’s Disease Studies

Last week, Oxford BioMedica and its partner Sanofi announced positive results in their ongoing gene therapy clinical trials for wet AMD and Stargardt’s disease. In an interim review of their Phase I (RetinoStat) and Phase I/IIa (StarGen) trials, the Data Safetly Monitoring Board (DSMB), an independent panel of specialists in the fields of ophthalmology, virology and vectorology, gave the go ahead to proceed to a final patient cohort in the Phase I study in the case of the RetinoStat trial, and to a third patient cohort in the Phase I/IIa study of the StarGen trial.


Recently, I encountered a unique referral source, goldenretrevor/pra-research. This piqued my curiosity and I went to the site and took a look. It turns out that the site is run by the owner of a Golden Retriever, named Trevor, along with two Labrador Retriever siblings. It seems that Trevor had been diagnosed with photo receptor cone disease (prcd), associated with progressive retinal atrophy (PRA). This was discovered when the dog was a puppy and the owner decided to look into this disease to see if there was anything that could be done to prevent him from going blind.

In doing extensive research, the owner, Katie McCormick, discovered that there was little research being done in the field of PRA in animals, but that PRA is genetically similar to retinitis pigmentosa (RP) in humans, as one study noted, "Identical mutation in a novel retinal gene causes progressive rod-cone degeneration (prcd) in dogs, and retinitis pigmentosa in man." And, there was lots of research being done on RP.

In her blog entry on PRA Research, Katie describes how she set up a “Google Alert” using the terms “progressive retinal atrophy” and “retinitis pigmentosa” – which is how she found my Journal article on The Use of Gene Therapy in Treating RP and Dry AMD.

A Novel Gene Therapy Approach to Treating the Wet Form of AMD: The BioFactoryTM From Avalanche Biotech

I originally contacted this company in November 2010, when they were still in “stealth mode” and weren't able to share details about what they were doing. Recently, the company got back in touch to provide an update, having announced, in December 2011, a clinical trial of their gene therapy approach to treating the wet form of AMD.

Since their approach is unique, and possibly “game changing” for the treatment of the wet form of AMD, I asked if I could prepare a writeup about the company and its technology for publication in my online Journal, and the co-founder and CEO Tom Chalberg agreed to answer my questions, as best as he could.

So, here in their own words is what Avalanche Biotech is all about.

And, finally, the blog entry on how to obtain the current, up-to-date tables of both stem cell companies and clinical trials underway, and the same for gene therapy companies and trials.

Because I have been updating this information almost on a daily basis, I’ve decided to no longer post the information on this Journal (Irv Arons’ Journal), as it is practically out-of-date almost as soon as I post it. Therefore, I will only offer my tables of information to those who are interested in receiving them, and put notices on the appropriate LinkedIn Forums and Twitter (and this forum) when there is substantial new information to report.

Here is what is currently available:

Stem Cells

Stem Cell Companies (and Institutions) Active in Ophthalmology

A list of seventeen companies and institutions working with stem cells for ophthalmic applications. The table lists collaborators, the cell type being used, and the applications against which the cells will be applied.

Stem Cell Therapy in Ophthalmology by Applications

A list of six ophthalmic applications being tested in clinical trials. The table includes which companies/institutions are involved, the clinical trial status, and the clinical trial number for those which are currently active. (Nine active clinical trials are listed, with live links.)

Stem Cell Therapy in Ophthalmology – Ongoing Clinical Trial Details

More detailed information about the ongoing clinical trials for the six ophthalmic applications noted above. This table includes detailed information about the clinical sites and provides the latest information about how many patients have been treated in each clinical trial.

Gene Therapy

Gene Therapy Companies/Institutions Active in Ophthalmology

The table lists more than twenty-five companies and institutions actively pursuing gene therapy solutions to ophthalmic diseases. The table shows the delivery viral platform, the gene type being used (where known), the application, and clinical status.

Gene Therapy in Ophthalmology by Application

This table, like the one for stem cells, lists the ophthalmic indication, the company/institutions involved, the clinical status, and the clinical trial number. (Fifteen active clinical trials are listed, with live links.)

Gene Therapy in Ophthalmology – Ongoing Clinical Trial Details

As with the Stem Cell table above, this table provides detailed information about the sixteen ongoing or completed clinical trials. It provides information about the clinical sites, the status of each clinical trial and the number of patients (or eyes) that have been treated in each trial.

Anyone interested in receiving a copy of any or all of the above tables, please contact me via email (see the link in the side bar) and let me know which ones, or all four, that you wish to obtain. The tables are in pdf format, and as mentioned, contain live links to all of the clinical trials listed.

Irv Arons


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