Ophthalmology Breaking News: Weekly Roundup (July 22, 2025)

Sanofi’s Intravitreal Gene Therapy Receives FDA Fast Track Designation for Geographic Atrophy

📢 The FDA has granted Fast Track designation to Sanofi’s SAR446597, an investigational gene therapy aimed at treating geographic atrophy (GA) secondary to age-related macular degeneration (AMD).

SAR446597 is a one-time intravitreal gene therapy designed to deliver two therapeutic antibody fragments that inhibit key complement system components: C1s (classical pathway) and factor Bb (alternative pathway). This dual-targeting approach is intended to suppress complement activation more comprehensively, potentially slowing GA progression more effectively than current options.

Sanofi plans to begin a phase 1/2 trial to assess SAR446597’s safety and efficacy in GA patients. The Fast Track status provides potential for accelerated development and regulatory engagement. Sanofi is also advancing SAR402663 in a separate phase 1/2 trial for wet AMD.

🔗Explore how SAR446597’s complement inhibition strategy could transform management of GA and reduce treatment burden for patients.

Nanoscope Begins Rolling BLA Submission for MCO-010: First Gene-Agnostic RP Therapy

📢 Nanoscope Therapeutics has initiated a rolling biologics license application (BLA) submission for MCO-010, the first gene-agnostic gene therapy targeting retinitis pigmentosa (RP), a genetically heterogeneous blinding disorder.

MCO-010 uses Nanoscope’s multi-characteristic opsin (MCO) platform to convert surviving bipolar cells into light-sensitive cells via a single intravitreal injection. It bypasses the need for genetic testing or surgery, enabling potential application across all RP patients regardless of genotype. The therapy has Fast Track designation and qualifies for priority review.

“We’ve seen this investigational therapy surpass our expectations in the lab and in patients,” said Samarendra Mohanty, PhD. The phase 2b RESTORE trial showed significant visual acuity improvements sustained for up to three years, with no serious adverse events in treated eyes.

🔗 Learn how MCO-010 is redefining RP treatment by eliminating genetic barriers and delivering long-term vision gains.

FDA Clears AAVantgarde to Begin Human Trials for Stargardt Gene Therapy AAVB-039

📢 AAVantgarde Bio has received FDA clearance to initiate its first-in-human trial (CELESTE) for AAVB-039, a gene therapy for Stargardt disease using the company’s proprietary dual AAV intein platform.

AAVB-039 aims to restore the full-length ABCA4 protein by delivering large gene constructs that exceed the capacity of standard AAV vectors. CELESTE will evaluate safety, tolerability, and initial efficacy, while a parallel natural history study (STELLA) informs its design.

“This FDA clearance marks a pivotal milestone,” said CEO Natalia Misciattelli, PhD. The company is also advancing AAVB-081 for RP due to Usher syndrome type 1B, the first dual AAV therapy tested clinically in an eye disorder.

🔗 See how AAVantgarde’s gene delivery innovations may expand treatment possibilities for inherited retinal diseases.

Krystal Biotech Doses First Patient in EMERALD-1 Trial for Neurotrophic Keratitis Gene Therapy

📢 Krystal Biotech has dosed the first patient in its EMERALD-1 trial evaluating KB801, a redosable topical gene therapy for neurotrophic keratitis (NK)—a rare corneal disease linked to loss of corneal nerve function.

The trial will enroll up to 27 patients with stage 2 or 3 NK. KB801 is administered twice weekly as an eye drop and delivers sustained expression of nerve growth factor (NGF), aiming to reduce the dosing burden compared to current recombinant NGF therapies that require frequent daily application.

“Until now, rapid protein clearance and high cell turnover in the front of the eye have severely limited the therapeutic potential of biologics and gene therapies,” said Suma Krishnan, President of R&D at Krystal Biotech.

🔗 Explore how KB801 may overcome delivery challenges and provide a new model for treating front-of-eye diseases.

FDA Accepts Aldeyra’s Resubmitted NDA for Reproxalap in Dry Eye Disease

📢 The FDA has accepted Aldeyra Therapeutics’ resubmitted NDA for reproxalap, a reactive aldehyde species modulator for dry eye disease (DED), with a PDUFA target action date of December 16, 2025.

The NDA resubmission includes data from a new phase 3 trial designed to address concerns raised in a prior complete response letter. The study demonstrated a statistically significant reduction in ocular discomfort compared to vehicle between 80 and 100 minutes in a dry eye chamber model (P=0.002).

“Reproxalap... remains the only dry eye disease investigational therapy to have demonstrated acute activity in reducing ocular discomfort and redness in pivotal trials,” said Dr. Todd C. Brady, CEO of Aldeyra.

🔗 Get insights into how Aldeyra’s updated trial results could bring a new option to DED patients facing flare-related symptoms.

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