AGTC Leverages Funding from Foundation Fighting Blindness to Garner Major Investments

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 — 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.

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 to advance promising retinal-disease gene therapies into clinical trials, including approximately $1.6M 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.

For example, during the time period when the Foundation was investing in its programs, AGTC raised $11.8 million in additional Series A venture capital and secured $37.5 million in Series B funding. In 2014, the company closed its initial public offering, which raised $57.5 million, and a follow-on offering raising $34.5 million. Most impressive, in 2015, AGTC closed a strategic collaboration with Biogen, which included an upfront payment of $124 million, with AGTC eligible to receive more than one billion dollars in future payments.

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.


































7168 Columbia Gateway Drive


Suite 100

Columbia, MD 21046