Week in review: Tocilizumab, fast track, retinas in a dish
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--Tocilizumab shows promise for patients suffering from COVID-19–associated pneumonia. A new phase 3 results reveal that patients receiving the drug—an interleukin-6 inhibitor approved for giant cell arteritis—along with standard care were 44% less likely to progress to mechanical ventilation or death by day 28 compared with those receiving placebo. Moreover, this global phase 3 trial is the first to primarily enroll underserved and minority patient populations.

--A gene therapy for dry AMD has been fast-tracked by the FDA. Gyroscope Therapeutics’ investigational AAV-based gene therapy is delivered under the retina of people with geographic atrophy who have a specific mutation in complement factor I (CFI).

--A novel gene therapy approach for retinal diseases may bypass the need for invasive surgery. The key Microneedles. Unlike current gene therapies that require more invasive, complex surgeries, the new method uses needles less than 1 mm in length to inject viral particles into the suprachoroidal space and is simple enough to perform in an office setting. Experiments in rhesus monkeys demonstrated that these suprachoroidal injections can produce diffuse, peripheral expression in retinal pigment epithelial cells but does induce localized inflammation—a side effect that will need further investigation.

--Production of personalized, functional retinas in the lab may soon be a reality, thanks to a team of Swiss scientists. The group has developed a model in which stem cells can self-organize into light-sensitive, multilayered retinal organoids with functional synapses and can give rise to most retinal cell types within 38 weeks. Even more impressive is that the approach enables researchers to generate thousands of highly uniform retinal organoids. "The research addresses a fundamental unmet need, which is to develop model retinas that closely resemble the real organ," said study investigator Cameron Cowan, PhD. “It opens up the possibility of developing treatments in a dish tailored to individual patients." Institute of Molecular and Clinical Ophthalmology Basel, Cell