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Ophthotech Launching Human Study of Emerging Therapy for Stargardt Disease

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Ophthotech, a biopharmaceutical company developing therapies for eye diseases, has enrolled the first patient in its Phase 2b clinical trial of Zimura® for people with Stargardt disease caused by mutations in the gene ABCA4. The 120-participant study will be taking place at more than 30 sites. Data and knowledge gleaned from ProgStar, a natural history study for people with Stargardt disease funded by the Foundation Fighting Blindness, was used in the design of the clinical trial.

Zimura is also in human studies for the treatment of the dry and wet forms of age-related macular degeneration (AMD) and idiopathic polypoidal choroidal vasculopathy (IPCV), an age-related retinal disease.

Zimura is administered by an intravitreal injection — an injection into the soft gel in the middle of the eye. Intravitreal injections are commonly performed in a doctor’s office.

Ophthotech’s emerging therapy is designed to inhibit activity of the complement system. An overactive complement system has been implicated in the development of retinal conditions such as AMD and Stargardt disease. The complement system is a part of the immune system that normally protects the body from harmful, invading bacteria. However, it can also attack healthy cells leading to vision-robbing retinal diseases and other conditions. Zimura blocks a complement protein known as C5. In lab studies, inhibiting C5 prevented retinal degeneration.

“Recent scientific literature emphasizes the role of complement in Stargardt disease, supporting our strategy for the development of Zimura in this devastating orphan retinal condition,” said Kourous A. Rezaei, MD, senior vice president and chief medical officer at Ophthotech, in a press release. “Our work with the highly distinguished organization, Foundation Fighting Blindness, provided us access to publicly available data from ProgStar, the largest natural history study in autosomal recessive Stargardt disease to date, playing an integral role in the design of our clinical trial.”

“Stargardt disease is a devastating orphan retinal disease for which there is currently no treatment,” said Benjamin Yerxa, PhD, FFB’s chief executive officer. “We are excited that Ophthotech has joined the mission to fight against orphan degenerative retinal diseases as both organizations share the common goal of transforming the lives of these patients and their families by developing potential new treatments.”

Stargardt disease is the leading cause of inherited macular degeneration, affecting 30,000 people in the U.S. and tens of thousands more around the world. The condition, usually diagnosed in childhood or adolescence, leads to loss of central vision.

5 Responses to 'Ophthotech Launching Human Study of Emerging Therapy for Stargardt Disease'

  1. Diane Franklin Dunbar says:

    Thank you for your consideration in informing me on what my niece is going through. Through it all she is a trooper full of courage and strength.

  2. Jill says:

    thanks I was diagnosed with Stargardts at age 8. I am now 54. I remain open and hopeful. I would like to see “normally” in my lifetime. While it has been a funfilled journey so far, patience is not my best quality. My faith in a “Stargardts miracle “ holds on.

  3. Amelia Todd says:

    This is exciting news for those of us afflicted with Stargardt’s,. While reading this, 2 questions come to mind: 1) how can one participate in this study? 2) are there research studies testing the affects of nutrition on this disease? With current research showing a link between inflammation and neurological diseases such as Parkinson’s and dementia I am very Interested in finding out if this type of research is being conducted.

    • EyeOnTheCure says:

      Hi Amelia, if you’d like to participate in this study, you can enroll on here:

      So far, we are not involved in any studies related to nutrition and Stargardt disease, as it’s an inherited disease. But an FFB-funded study recently suggests that vitamin A palmitate supplementation may slow the decline of cone function in children with retinitis pigmentosa (RP). Although this is a different disease, this will help open the door for other similar development.

  4. Wanda hedrick says:

    Would love for someone to find a cute for stargodt disease. Have suffered many years with it.

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