CRISPR gene-editing trial shows ‘exciting potential’

A Phase 1/2 trial using CRISPR-based gene editing to treat patients with Leber congenital amaurosis type 10 due to a mutation in their CEP290 gene shows “exciting potential” of the gene therapy, according to a news article from Oregon Health & Science University. According to the article, 79% of clinical trial participants had a measurable improvement after receiving EDIT-101. That is, 11 participants showed improvement in at least one of four measured outcomes, 6 (43%) showed improvement in two or more outcomes, and 6 (43%) reported improved vision-related quality of life. Twenty-nine percent (4 participants) showed clinically meaningful improvements in visual acuity, and there were no serious adverse treatment-related events. When the trial began in 2020, the article noted, it was the first time CRISPR had been used to edit genes in vivo. The paper describing these findings is published in the New England Journal of Medicine.

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Study: laser vision correction clinics missing opportunities

A study, sponsored by Carl Zeiss Meditec, has found that many laser vision correction clinics are missing out on patient leads. Among the notable observations in the study were that 17.8% of clinics approached by patients had no means of communication through their websites; the lead response rate was 73% within 3 days of inquiry, and 79 of 279 leads didn’t receive engagement from the practice within the 3-day window; 46% of practices included in the study did not offer online booking for in-person appointments. The study quantifies the potential revenue loss from missed laser vision correction leads and offers recommendations for practice enhancement to increase web lead conversions.

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Broad-based protection observed with investigational geographic atrophy therapy

Annexon announced positive neuroprotection data observed with ANX007, a therapy being investigated for the treatment of geographic atrophy. According to the company’s press release detailing new analysis from its Phase 2 trial, “significant, broad-based protection from vision loss in the foveal and non-foveal patients, and in low-light settings” was observed. The company reported that there was a significant reduction in photoreceptor cell loss and a meaningful slowing of lesion growth in the fovea. The researchers found that the treatment protected photoreceptor synapses and preserved retinal function. The company expects to begin two Phase 3 trials this year. ANX007 is described as a “first-in-class, non-pegylated antigen-binding fragment designed to block C1q and activation of the classical complement cascade.” It is delivered via intravitreal injection.

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FDA accepts IND for diabetic macular edema therapy

Eluminex Biosciences announced that the FDA accepted its IND for EB-105, a “novel trispecific fusion antibody targeting VEGF-A (and isomers), VEGF-B, placental growth factor (PlGF), angiopoetin-2 (Ang-2), and interleukin-6 receptor.”

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Phase 2 trial for non-proliferative diabetic retinopathy therapy does not meet primary endpoint

EyePoint Pharmaceuticals’ DURAVYU (vorolanib intravitreal insert, previously EYP-1901) did not meet its primary endpoint in the Phase 2 clinical trial, though the company noted it did have a biologic effect for patients with non-proliferative diabetic retinopathy and a favorable safety/tolerability profile. The company will provide its plan forward with the possible treatment after its full 12-month data review.

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ASCRS news and events

• ASCRS Live!: This new dinner series, making its way to nine cities, is continuing to bring ASCRS education around the U.S. Next week is New York! See where it’s headed.
• ASCRS 50th Anniversary: Review historical videos, watch member accounts, and scroll through the timeline—all brought together on these webpages to celebrate the Society’s historic anniversary.
• ASCRS Annual Meeting: Hotel blocks are open for the 2025 ASCRS Annual Meeting in Los Angeles, California, April 25–28, 2025.

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Research highlights

• A prospective consecutive case series published in the Journal of Cataract & Refractive Surgery compared three capsulotomy centration methods. The study included 180 eyes that prior to capsulotomy creation had the anterior capsule stained with trypan blue. Group 1 had mydriatic dilated pupil-centered capsulotomies. Group 2 were centered on the trypan blue central landmark. Group 3 were centered on the patient-fixated coaxial Purkinje reflex (CPR). The researchers observed “noticeably decentered” capsulotomies in Group 1 by 0.3±0.2 mm. Group 2, they wrote, showed a “symmetrical IOL relationship”; decentration was 0.15 ± 0.1 mm. Group 3 had decentration of 0.15±0.1 mm. The authors wrote that a clearly visible trypan blue central landmark was an alternative landmark to patient-fixated CPR. They also wrote that being on the anterior capsule was not sensitive to tilt and patient compliance wasn’t required. They think this has applications for both capsulotomy and capsulorhexis creation.
• A retrospective cohort study with case control analysis published in the journal Eye has associated ADHD medication therapies with an increased risk of glaucoma. According to the paper, these medications include atomoxetine, methylphenidate, and amphetamines and are some of the “most prescribed medications in North America.” The study included 240,257 patients who were new to ADHD medications. Patients using atomoxetine and amphetamines had a higher adjusted incidence-rate-ratio (aIRR) for developing angle closure glaucoma, while methylphenidate users had a higher aIRR for developing open-angle glaucoma. With the high prevalence of ADHD medication use, the authors wrote that this data regarding glaucoma risk “have profound public health implications.”

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