Ikarovec Ltd and VectorBuilder have entered into an exclusive worldwide option agreement covering VectorBuilder’s adeno-associated virus capsid technology for use with Ikarovec’s gene therapy candidate, IKAR-003, aimed at intermediate age-related macular degeneration. Under the proposed structure, Ikarovec would assume responsibility for clinical development and commercialization if the option is exercised. The companies said the potential value of the deal exceeds $1 billion, based on expectations for the program.

IKAR-003 is designed as a one-time, intravitreally delivered gene therapy that combines neuroprotection with complement modulation to slow disease progression. The use of an intravitreal route would allow administration in an office setting, expanding access for patients with intermediate AMD, a population that currently has no approved pharmacologic treatments and faces a risk of progression to geographic atrophy or neovascular disease.

The program builds on Ikarovec’s dual-pathway platform, which is also being used in its lead candidate, IKAR-001, planned for subretinal delivery in geographic atrophy, with clinical trial initiation expected in late 2026. VectorBuilder’s capsid technology, developed using its AI-based DeepCap platform, has demonstrated broad retinal transduction in nonhuman primate studies following intravitreal injection.

Ikarovec, a UK-based biotechnology company, is advancing multiple retinal gene therapy programs targeting both early and advanced stages of disease, with delivery strategies tailored to disease severity. RP