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 FFB 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Again, this table is currently out-of-date. See the offer to receive current versions of these tables 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 in the offer made on the Stem Cell W\writeup posting just above.)
A report on the progress being made in treating Leber’s using gene therapy, as reported by the Foundation Fighting Blindness.
Miscellaneous
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.
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.)
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. (Fourteen active clinical trials are listed, with live links.)
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
