April 2013 Update From Dr. Klassen

This is our first monthly update and I want to thank all of you who have provided help, encouragement, and feedback related to our stem cell project for retinal diseases.

In March, our RP Disease Team successfully manufactured two additional lots of our particular stem cells (retinal progenitors) at the GMP Facility at UC Davis Medical Center. These cells are intended for future use in patients. The cells have been frozen in liquid nitrogen and will be stored until needed. Further testing will also be carried out to verify that the new cells qualify for clinical use.

In March, I served as Co-Chair of the Retinal Degeneration Session at the International Symposium in Ocular Pharmacology and Therapeutics (ISOPT) Clinical, in Paris, where I updated the ophthalmology community on progress towards treatment for RP and related conditions. I also spoke at the Southern California Vision Seminar in Long Beach, hosted by the Foundation Fighting Blindness, where I had a chance to meet many friends and interested parties. I also traveled to Rockville, Maryland and met with representatives of the Therapeutics for Rare and Neglected Diseases (TRND) Program of the National Institutes of Health, who were interested in hearing more about this project.

We were happy to recently receive our Notice of Grant Award from CIRM, officially authorizing the transfer of funds to support the clinical development activities of the project. As a result, a “kick off” meeting as held at UCI to coordinate the many offices supporting our Disease Team effort at the institutional level. This major grant is critical and will provide the funding “backbone” for our project, however, it is restricted in scope and therefore additional financial resources are needed to bridge specific gaps and accelerate clinical implementation of this technology. Your contribution in helping us reach our 2013-2014 funding initiative of $1 million is greatly appreciated. If you know of potential donors or corporate parties interested sponsorship, please contact Terry O’Neal at: terry.oneal@tffrr.org

Warmest Regards,

Henry Klassen, MD., PhD.

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