The inherited retinal disease (IRD) community closely monitors advancements in ophthalmology, especially new therapies that hold promise for preserving or restoring vision. Recent news from Novartis, a major pharmaceutical company, indicates a shift in its ophthalmology strategy, including the discontinuation of a gene therapy program for geographic atrophy (GA). This development, reported by Fierce Biotech, highlights the complex and often challenging path of drug development and its direct impact on patient communities.

Novartis Halts GT005 Gene Therapy Development

Novartis has announced the discontinuation of its gene therapy candidate, GT005, which was under development for geographic atrophy (GA) secondary to dry age-related macular degeneration (AMD). The decision was made following a recommendation from an independent data monitoring committee, which conducted a benefit-risk assessment of the available data from the Phase 2 HORIZON program. The committee concluded that the overall data did not support the continuation of the program, as futility criteria had been met. Notably, no new safety signals were identified during the trials.

Novartis acquired GT005 through its 2021 acquisition of Gyroscope Therapeutics for up to $1.5 billion, which included an $800 million upfront payment and potential milestone payments. With the discontinuation of GT005, these milestone payments will not be realized.

Broader Ophthalmology Restructuring at Novartis

The discontinuation of GT005 is part of a larger restructuring within Novartis's ophthalmology division. The company is reportedly looking to offload its broader eye disease unit as part of a major restructuring effort that also involves spinning off its generics unit, Sandoz. Novartis had previously divested its