Fast Track designation granted to one-time intravitreal gene therapy for geographic atrophy

Sanofi announced that its investigational intravitreal gene therapy for geographic atrophy due to AMD has received a Fast Track designation from the FDA. SAR446597, according to the company’s press release, delivers two antibody fragments for a dual-targeting approach that could provide sustained complement pathway suppression. The company plans to begin a Phase 1/2 study for this indication soon, evaluating safety, tolerability, and efficacy. The company is currently studying SAR402663, also a single intravitreal gene therapy injection, for treatment of neovascular wet AMD in a Phase 1/2 study.

Back to top

FDA clears IND for Stargardt gene therapy to move forward into a Phase 1/2 clinical trial

AAVantgarde Bio received clearance for its Investigational New Drug (IND) application for AAVB-039. This investigational gene therapy for Stargardt disease will now enter a Phase 1/2 first-in-human clinical trial to assess safety, tolerability, and efficacy. According to the company’s press release, the therapy uses a proprietary dual AAV intein platform, which in preclinical animal models has shown high transduction, protein expression, and safety. AAVB-039 will attempt to restore the full length of the ABCA4 protein.

Back to top

BLA submission for retinitis pigmentosa gene therapy begins

Nanoscope Therapeutics submitted a Biologics License Application (BLA) to the FDA for MCO-010, a gene-agnostic gene therapy for treatment of vision loss due to retinitis pigmentosa. According to the company’s press release, if approved, this therapy could help restore vision in patients with retinitis pigmentosa, regardless of the underlying mutation that caused the condition. The company noted that the FDA granted a rolling review of the BLA, and complete submission is expected by early 2026. It will receive priority review due to its Fast Track designation.

Back to top

First patient dosed in Phase 1/2 trial evaluating NK therapy

Krystal Biotech announced that the first patient was dosed in its Phase 1/2, 2:1 randomized, double-masked, multicenter, placebo-controlled study evaluating KB801 for treatment of neurotrophic keratitis (NK). According to the company’s press release, KB801 is a “redosable eye drop gene therapy.” The study will enroll up to 27 adults with stage 2 or 3 NK. The primary endpoint is safety and tolerability; the secondary objective is efficacy defined as 0 mm of corneal fluorescein staining at the corneal lesion site at week 8 and week 10. Other efficacy measures being assessed are change in corneal lesion size, corneal sensation, and patient-reported symptom burden.

Back to top

Enrollment complete in Phase 2b trial for PCED therapy

Kala Bio’s enrollment is complete for its Phase 2b trial evaluating KPI-012 for treatment of persistent corneal epithelial defect (PCED). KPI-012 is described as a human mesenchymal stem cell secretome. The study enrolled and randomized 79 patients who received one of two doses of KPI-012 or vehicle QID for 56 days. The study’s primary endpoint is complete PCED healing. Topline data is expected in the third quarter of 2025. Depending on the results from this study, the company thinks it could serve as one of the pivotal trials for BLA submission.

Back to top

ASCRS news and events

• Business of Refractive Cataract Surgery Summit: This course, taking place October 3–5, in Chicago, Illinois, along with its extensive, take-home Resource Library, will help practices gain the navigational tools needed to increase premium IOL adoption and lead patients effectively on their refractive surgery journey. Learn more.
• ASCRS Live!: This educational dinner series is heading to several cities across the U.S. in 2025. Registration is open for the next event in Seattle, Washington, on July 24.
• 2026 ASCRS Annual Meeting: Hotel room blocks are open for the 2026 ASCRS Annual Meeting, taking place in Washington, D.C., April 10–13, 2026. The call for submissions is also ongoing until September 1. Submit an abstract and get involved in the 2026 ASCRS Annual Meeting.

Back to top

Research highlights

• The impact of using a visual simulation tool preoperatively (the SimVis Gekko) on predicting postop visual performance for patients having bilateral cataract surgery with trifocal or EDOF lenses was the subject of a single-center, prospective, observational cohort study published in the Journal of Cataract & Refractive Surgery. The study enrolled 51 patients with mild to moderate cataract (44 were included in the final analysis), simulating a trifocal, EDOF, and a monofocal IOL with the SimVis. Of these, 26 received a trifocal IOL and 18 an EDOF. The visual acuity curve for both IOLs was comparable between the preop simulation and the actual postop visual acuity. Stereopsis was also similar with and without the simulation. The subjective visual performance score improved postop in both IOL groups but was higher for the trifocal group. The simulation tool preoperatively was helpful to 92.2% of patients. The authors concluded that “this instrument seemed to be very useful for the patient’s decision-making process and may reduce dissatisfaction after surgery.”
• The outcomes after use of a non-cavitating lensectomy device (MICOR, Zeiss) vs. phacoemulsification were compared in a two-arm, prospective, open-label clinical trial conducted at a single center between March and December 2023. The study included 34 eyes of 23 patients in the MICOR arm and 50 eyes of 32 patients in the phaco arm. There was no difference in logMAR BCVA postoperatively in the two arms. There was no significant difference in adverse event rates in either arm. The authors reporter a faster lens removal time with MICOR and less irrigation volume in this group. They concluded that MICOR use resulted in “similar visual outcomes and adverse event rates while requiring less time, irrigation fluid, and ultrasound energy, suggesting MICOR is a viable alternative to phacoemulsification.” The study is published in the journal Clinical Ophthalmology.

Back to top

Product news

• The RainDrop Dispenser Aid (Rain Eye Drops) was launched as a device exclusively licensed from the U.S. Department of Veterans Affairs to help dispense drops from monodose eye drop vials. According to the company’s press release, the RainDrop dispenser is free with any Rain purchase.

Back to top

---