Gene Therapy in Ophthalmology Update 7 2012 the Year for Gene Therapy

While I have previously written about the progress being made in the use of stem cells in ophthalmology (see Stem Cell Update 13) and described the 9-10 clinical trials currently underway or about to start (see Stem Cell Update 14), recent events point to 2012 becoming a breakthrough year for the use of gene therapy to overcome genetic defects that cause several ophthalmic diseases.

In the accompanying table, 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.

The treatment of Leber’s using gene therapy has been ongoing for at least three years and, as I will show in the next update (Gene Therapy Update 8), those trials are going quite well, with many of the patients showing improved vision.

Finally, as another indicator that gene therapy will play an important role in ophthalmology in this year, Ocular Surgery News is about to begin a special section, OSN Retina, to be part of it’s coverage of the ophthalmic scene. The January 25 issue of Ocular Surgery News will include OSN Retina - a leading destination that will provide retina specialists with more relevant information specific to their field.. The premiere issue will include a feature on how  “Retinal gene therapy may pave the way for attempts to reverse genetic disease: Advancements in retinal gene therapy have prompted a collaborative effort to attain FDA approval.”

For those of you who wish a better understanding of how gene therapy works, and until I write the Primer on the Use of Gene Therapy in Ophthalmology, which I have threatened to write for the past year and a half, you can gain an understanding by reading my first article about gene therapy, written back in November 2010, The Use of Gene Therapy in Treating Retinitis Pigmentosa and Dry AMD by Retrosense.

Here then is my latest version of Gene Therapy in Ophthalmology by Application:

A pdf file of the table is available by email request.