Stargardt Disease Research Advances

SpliceBio testing Stargardt disease protein splicing therapy in Phase 1/2 clinical trial

SpliceBio, a genetic medicines company developing protein splicing therapies for inherited retinal diseases and other conditions, has moved into the dose-expansion phase of their Phase 1/2 clinical trial for SB-007, an emerging protein splicing therapy for Stargardt disease caused by ABCA4 mutations. ASTRA is the first FDA-authorized clinical trial for a protein splicing therapy. The primary goal of the study is to determine whether SB-007 is safe and tolerable. It will also examine whether SB-007 can preserve or improve vision. SB-007 is designed to be a one-time treatment administered via subretinal injection.

 

VeonGen launches clinical trial for mRNA trans-splicing gene therapy for Stargardt disease

VeonGen, a company developing genetic therapies for eye diseases, has received authorization from the US Food & Drug Administration (FDA) to launch a Phase 1/2 clinical trial for VG801, an emerging mRNA trans-splicing gene therapy for people with Stargardt disease and other retinal diseases caused by mutations in the ABCA4 gene. The company will also seek authorization from the European Medicines Agency to conduct the clinical trial at sites in the EU. VG801 is currently being tested in a Phase 1/2 trial in Shanghai, China.

 

Alkeus reports encouraging results in clinical trials for drug designed to reduce toxins in retina

The biotech company Alkeus has conducted multiple Phase 2 clinical trials for a drug (ALK-001) that targets the toxic build-up in the retina that is thought to cause degeneration and vision loss. The therapy slowed lesion growth by about 30 percent. 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.

 

Belite Bio to apply for FDA approval for Tinlarebant

The San Diego-based biopharmaceutical company Belite Bio reported that Tinlarebant slowed the growth of atrophic lesions (areas of cell death) by 36% compared to placebo in their Phase 3 DRAGON trial. Tinlarebant is an oral drug designed to inhibit a protein known as retinol binding protein 4 (RBP4) to reduce the uptake of vitamin A to the retina, thereby decreasing the production and accumulation of toxic vitamin A byproducts, which are the hallmark of Stargardt disease. Based on these results, Belite Bio plans to seek FDA approval in the first half of 2026.

 

Ascidian launches clinical trial for RNA editing treatment for Stargardt disease

Ascidian Therapeutics has launched a Phase 1/2 clinical trial for ACDN-01, the company’s RNA editing therapy for people with Stargardt disease, an inherited form of macular degeneration caused by mutations in the ABCA4 gene.

Unlike genetic therapies that deliver an entire healthy gene (DNA) to replace the mutated gene, or which edit DNA, ACDN-01 rewrites RNA, the genetic message derived from DNA that cells read to make proteins. Healthy proteins are essential to the survival and function of all cells in the body, including those of the retina. ACDN-01 specifically rewrites RNA exons, the regions where mutations are most likely to occur for the ABCA4 gene. Ascidian estimates that ACDN-01 can address mutations in ABCA4 for a significant percentage of people with Stargardt disease because the editing replaces a large number of exons, all at once.

 

Nanoscope to launch Phase 3 clinical trial of optogenetic therapy for Stargardt disease

Nanoscope Therapeutics, a biotechnology company developing gene therapies for retinal degenerative diseases, has received authorization from the FDA to launch a Phase 3 clinical trial of its optogenetic therapy for people with advanced Stargardt disease. Some patients in Phase 2 had rudimentary vision improvements after receiving the emerging treatment. Known as MCO-010, the optogenetic therapy is designed for  people who have lost most or all of their photoreceptors, the cells that make vision possible. The treatment, a small drop of liquid delivered by an intravitreal injection, uses a human-engineered virus to deliver copies of the Multi-Characteristic Opsin (MCO) gene to bipolar cells — cells that don’t normally sense light but often survive after photoreceptors are lost to advanced retinal disease.

 

Ocugen launches Phase 2/3 clinical trial for its modifier gene therapy for Stargardt disease

Ocugen has launched its Phase 2/3 GARDian3 clinical trial for OCU410ST, a modifier gene therapy candidate being developed for Stargardt disease, as a one-time treatment for life. The study plans to enroll 51 patients, who will be randomized 2:1 to the treatment vs. placebo group. Slowing of disease legions (areas of cell loss) were reported in the Phase 1/2 clinical trial for the treatment.

 

Stargardt disease natural history study helping prepare for future clinical trials

The Foundation Fighting Blindness completed 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. Multiple companies developing therapies for Stargardt disease have utilized ProgSTAR data to inform their clinical trial design.

 

 

FEBRUARY 2026