Jul 11, 2022

Stargardt Disease Research Advances

Retinal Disease Research Advances

Recent developments in research on Stargardt disease

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Kubota Conducting Clinical Trial of Drug for Stargardt Disease

Emixustat, an oral drug which originally targeted the dry form of age-related macular degeneration (AMD), is conducting a Phase 3 clinical trial for Stargardt disease. While the drug did not meet desired outcomes in the AMD clinical trial, experts believe it may work well for people with Stargardt disease. Developed by Acucela, the drug works by slowing the buildup of toxic waste products that lead to retinal degeneration in a number of retinal conditions, including Stargardt disease.

Alkeus Launches Clinical Trial for Drug Designed to Reduce Toxins in Retina

The biotech company Alkeus is conducting a multi-center Phase 2 clinical trial for a drug (ALK-001) that targets the toxic build-up in the retina that is thought to cause degeneration and vision loss. The emerging therapy is a modified form of vitamin A, which when metabolized in the retina, results in much less waste. Scientists developed ALK-001 by replacing hydrogen atoms in vitamin A with deuterium. Known as deuterated vitamin A, it “burns cleaner” than the natural form. Deuterium is a safe, naturally occurring, stable form of hydrogen, which is present in the human body.

Stargardt Disease Natural History Study Will Help Prepare for Future Clinical Trials

The Foundation Fighting Blindness Clinical Research Institute is completing a $6 million natural history study for people affected by Stargardt disease. Known as ProgSTAR, the study has three primary goals: 1) Determine the best outcome measures to accelerate evaluation of emerging treatments in clinical trials, 2) better understand disease progression for selecting future clinical trial participants, and 3) identify potential participants for forthcoming clinical trials. The study has enrolled 365 patients in 10 international clinical centers.

Research Team Is Creating Patch Derived from Stem Cells to Restore Vision

David Gamm, MD, PhD, at the University of Wisconsin–Madison, is leading a team of experts to create a patch composed of retinal cells derived from stem cells as a vision-restoring therapy for people with macular conditions such as Stargardt disease. The company Opsis Therapeutics was recently formed to advance the therapy into and through a clinical trial and out to the patients who need it. The patch consists of two layers. One layer will serve as a precursor to vision-enabling photoreceptors. Once transplanted, the stem cells of this layer will mature into photoreceptors. The other layer will consist of mature retinal pigment epithelial (RPE) cells, which provide waste disposal and nutrition for photoreceptors. A thin plastic film will serve as a structural backbone for the patch. A biodegradable gel will protect the cells and hold the layers together. The Foundation Fighting Blindness currently funds the project, and has funded Dr. Gamm for more than a decade to advance this therapeutic approach.

Gene Therapies in Development for Stargradt Disease

Applied Genetics Technology Corporation, Iveric bio, and Coave are among the biotech companies developing gene therapies for Stargardt disease. Their projects are currently in preclinical development and involve augmenting mutated copies of the ABCA4 gene with normal copies.

Iveric Bio Conducting Clinical Trial of Complement Inhibitor for Stargardt Disease

Iveric Bio is conducting a Phase 2 clinical trial for Zimura, a C5 complement inhibitor designed to slow the progression of Stargardt disease. In many retinal diseases, the complement system (part of the innate immune system) is overactive, thereby exacerbating retinal degeneration and accelerating vision loss.


JULY 2022