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AGTC Leverages Funding from the Foundation to Move Promising Treatments into Clinical Trials

Seed becoming a plantCompany Builds on FFB’s Initial Investment to Garner $265 Million in Therapy Development Funding

In the early 1990s, scientists began discovering the genetic defects causing blinding, inherited retinal diseases and saw a unique opportunity to overcome them. They envisioned gene therapy — delivering healthy genes to the retina to replace the bad ones — as an elegant approach to saving and restoring vision. Furthermore, a single injection of gene therapy would likely halt or reverse the disease process and work effectively for several years, perhaps the patients’ lifetimes.

The Foundation Fighting Blindness, the world’s leading private, nonprofit retinal research organization, funded most of these genetic discoveries for retinal diseases and immediately recognized the enormous opportunity for gene therapy to beat blindness.

“Twenty years ago, gene therapy was considered quite cutting edge, but we saw great potential for using it to treat genetic diseases of the retina,” says William T. Schmidt, the Foundation’s chief executive officer. “We felt it was incumbent upon us to try to move the field forward.”

In 1997, the Foundation began funding gene-therapy visionary Dr. William Hauswirth at the University of Florida to develop retinal treatments. Early successes in the lab led to investments in earnest in Dr. Hauswirth and several other gene-therapy developers around the world.

In 1999, Dr. Hauswirth and other leading researchers and executives founded Applied Genetic Technologies Corporation (AGTC) to advance gene therapies for retinal diseases and other conditions into the marketplace.

Between 2009 and 2016, the Foundation invested more than $8.5 million in lab studies that ultimately helped AGTC advance promising retinal-disease gene therapies into clinical trials. Nearly $7 million funded related lab studies at academic research centers. Approximately $1.6 million in support went to AGTC. These investments targeted treatments for three retinal diseases: achromatopsia (day blindness); X-linked retinoschisis or XLRS (splitting of the retina); and X-linked retinitis pigmentosa or XLRP, a leading cause of inherited blindness in males.

Thanks in part to the Foundation’s investments in lab research for AGTC, gene-therapy clinical trials were recently launched for achromatopsia (2016) and XLRS (2015). The XLRP effort continues to progress well.

The research advancements made possible by the Foundation’s support are no doubt impressive, but AGTC has been able to leverage the nonprofit’s funding to attract significant investment from the commercial sector.

Ultimately, an $8.5 million investment from the Foundation Fighting Blindness helped AGTC garner investments of at least $265 million to date, with the further potential for as much as an additional $1 billion from a variety of private, public, and industry sources. This is a great example of financial leverage. But if the company can bring products to market that save and restore sight, the investment payoff will truly be remarkable and everlasting.

“The Foundation plays a unique, invaluable role in advancing emerging treatments to the point where we, and our partners, can attract commercial investments,” says Mr. Schmidt. “AGTC is one of several companies that has been able to leverage our support in a big way.”

7 Responses to 'AGTC Leverages Funding from the Foundation to Move Promising Treatments into Clinical Trials'

  1. Glenn says:

    I am interested in knowing if RPE stem cell treatment is being done with one’s own cells, as to avoid the use of immune suppressant drugs. I have RP, and I would wish to not inhibit my immune system for eyesight.
    Thanks for any information.

    • Dr. Tim Schoen says:

      Dear Glenn, Because of the high cost and low return for “personalized” medicine, it is unlikely that pharmaceutical companies will pursue this approach and I am not aware of any clinical trials that are using a patient’s own stem cells to generate autologous RPE cells. However, several groups are using “banks” of stem cells that are immunologically similar. The human leukocyte antigen (HLA) system is a family of proteins that are differentially expressed on the surface of all cells that enables the body’s immune system to distinguish self from non-self and react accordingly. FFB-supported researcher, Dr. David Gamm and his colleagues have developed an IPS stem cell bank containing over 200 lines of human IPS stem cells. Although not yet in human clinical trials, when the time comes, Dr. Gamm hopes to match each recipient with a donor stem cell line that has similar HLA markers, thus eliminating the need for immune suppressants. To read more about Dr. Gamm’s studies, please see the following web link:

  2. Nicole Reed says:

    I am writing you today because i have a 4 year old grandaughter Marlaya ,
    who was diagnosed with L.C.A RPE65. Dr. Duncan of UCSF told us that Marlaya would be a good canadate for these studies. We are willing to do anything to help her, She is a brite beautiful little girl with no other health or developmental ssues. Can you help us give her sight. please contact me anytime day or night by phone or Email. Thank you for your time, Nicole Reed
    Dear Ms. Reed,

    Thanks for your message. We’re sorry to hear of your granddaughter’s diagnosis.

    Please see:

    There has been research using gene therapy for LCA patients. Please see this from our National Eye Institute (NEI):

    To search for clinical research trials around the United States, please visit: You can narrow your search by additional symptoms, conditions, geographic area, etc. If interested in any of these studies, scroll down to the section about Eligibility to see the age range for study enrollment, then scroll to the section titled “Location and Contacts” to see who you can talk to for more information.

    To search for clinical research trials at the NIH Clinical Center in Bethesda, Maryland, please visit: If interested in any of these studies, please contact NIH Patient Recruitment by or by calling 1-800-411-1222.

    You can also sign up at, a national registry of clinical trials that might be able to pair you with a relevant study when one becomes available.

    For additional information, please contact NEI:


    NIH INFO – D

    From: Nicole Reed []
    Sent: Sunday, December 18, 2016 10:51 AM
    To: NIHInfo (NIH/OD)
    Subject: L.C.A. RPE65

    Show quoted text

    Dear Ms. Reed – Thank you for contacting me. AGTC is no longer conducting clinical trials of treatments for LCA RPE65, but there is a company called Spark Therapeutics that is working on a treatment for this condition. If you click on the link below you can find information on how to contact them.

    I wish you and your granddaughter success in seeking out treatment.

    Jeffrey D. Chulay, MD

    386.462.2204, ext. 7205

    Description: cid:image001.png@01CE992A.9C139BD0

    From: Nicole Reed []
    Sent: Monday, December 19, 2016 5:56 PM
    To: Jeff Chulay
    Subject: NCT00749957, AGTC-RPE65-002, Phase 1/2 Safety and Efficacy Study of AAV-RPE65 Vector to Treat Leber Congenital Amaurosis

    Show quoted text

  3. Alieu Kamara says:

    Very exciting news from AGTC. This is a step in the right direction. However, I would like to know if there are plans on the way to introduce a clinical trial for GRPA1? I hope I got it right. lol I was informed by a genetic counselor from Information DNA that it is the form of RP I have after undergoing genetic testing. If so I would appreciate if FFB would send me an email about how I could become a participant.


  4. Donald Weston says:

    What is the current status of Fenretinide for the treatment of AMD? It has been in clinical trials for many years with promising results but apparently is still not available. Why?

    • Dr. Tim Schoen says:

      Dear Donald, It is my understanding that the company Acucela acquired the rights to fenretinide. Fenretinide has a number of side effects and this may be why there is no current trial.

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