Retinal Gene Therapy Developer AGTC Receives $37.5 Million in Venture Capital

November 19, 2012

Applied Genetic Technologies Corporation (AGTC), a leading developer of gene therapies, has received $37.5 million in Series B (second round) venture capital funding to advance potential gene therapies for the retinal degenerative diseases achromatopsia and X-linked retinoschisis (XLRS) toward human studies. The investment will also enable AGTC to continue its Phase II clinical trial for alpha-1 antitrypsin deficiency, a condition which causes serious lung and liver dysfunction.

The Foundation Fighting Blindness is currently funding translational research in support of AGTC’s emerging gene therapies for achromatopsia and XLRS. Dr. Jeff Chulay is leading the preclinical effort for XLRS at Oregon Health & Science University, and Dr. William Hauswirth is conducting achromatopsia research at the University of Florida. By demonstrating safety and efficacy in the laboratory, AGTC will be well positioned to move the potential treatments into clinical trials.

“We are delighted by this major boost in funding for these two important gene therapy projects,” says Dr. Stephen Rose, chief research officer of the Foundation Fighting Blindness. “The investment provides us critical momentum in moving these potentially sight-saving treatments into human studies and out to the people who need them. We have the opportunity to save vision for tens of thousands of people.”

AGTC’s gene therapies work by delivering copies of healthy genes to the retina to replace the defective copies that are causing vision loss. A human-engineered virus known as an adeno-associated virus, or AAV, is used to deliver copies of the corrective genes to the patients’ retinal cells. Researchers believe that a single ocular injection of the treatment will halt vision loss for several years or perhaps a lifetime.

Often referred to as day blindness, achromatopsia is caused by genetic defects that affect cones, the cells in the retina that provide central and daytime vision, as well as the ability to perceive colors and detail. Approximately 22,000 people in the United States and Europe, and tens of thousands more around the world, are affected by achromatopsia.

Affecting males, XLRS is a genetic condition causing progressive and severe loss of central vision due to splitting of the layers of the retina. XLRS affects tens of thousands of people all over the world. Approximately 35,000 have the condition in the United States and Europe.

The second round of venture capital investing in AGTC’s gene therapies is being led by Alta Partners. Additional investors include: SROne, Osage University Partners, InterWest, Intersouth and MedImmune Ventures.

“It is critical that our research attracts outside investment to move treatments into clinical trials. These human studies cost tens and sometimes hundreds of millions of dollars,” says William Schmidt, chief executive officer of the Foundation. “It is imperative that we provide compelling evidence for safety and effectiveness in the lab to build investor confidence in their potential for saving vision in people.”