Gene Therapy in Ophthalmology Update 11: Clinical Trial Details

I have found fifteen gene therapy clinical trials underway to treat various eye disorders. In thinking about this, I wondered how many patients had been treated to date in these clinical trials. Not able to find this information, I decided to undertake the task of obtaining the information by asking the people running the trials.

This resulted in this new table, Table 3 Gene Therapy in Ophthalmologly - Ongoing Clinical Trial Details.

I have visited each National Clinical Trial (NCT) site, derived the pertinent information, and placed it into the table.

I then requested the number of patients treated to date in each trial from the principal investigator or sponsor. I was able to get information from nine of the fifteen trials – either from the principal source or from a reliable secondary source.

It turns out that since the first gene therapy clinical trial for Leber’s Congenital Amaurosis by University College London and Moorfields Hospital in February 2007, more than 50 patients and 54 eyes have received gene therapy treatment (at least 4 second eyes have been treated by Children’s Hospital in Philadelphia in their Leber’s trial).

As new information is obtained, I will continue to issue updates.

Here then, is the initial version of the table:

Irv Arons May 2012 (Version 1, 5/18/12)

NYR - Not yet recruiting
R - Recruiting

1. Reported at ARVO 2012

A downloadable pdf file of the above table is available by request.

To obtain copies of the current versions of the first two tables, listed below, please send me an email.

Table 1. Gene Therapy Companies/Institutions Active in Ophthalmology

Table 2. Gene Therapy in Ophthalmology by Application

Editors Note:

Two quick corrections in the published table:

1. Oxford Biomedica should read Oxford BioMedica.

2. King Khaled Eye Hospital is treating MERTK-related autosomal recessive RP, and not autosomal-dominent RP as listed.

Both of these corrections will appear in future updates.

Version 2 (May 22, 2012), containing several data updates will be available on May 22nd by email request.