Ikarovec Limited,a UK-based company pioneering dual-pathway gene therapies for retinal diseases, has entered into an exclusive agreement with VectorBuilder to use the company’s novel AAV capsid technology in combination with Ikarovec’s gene therapy candidate IKAR-003 for intermediate age-related macular degeneration (AMD).
The development could lead to eye disease gene therapy being used in a doctor’s office.
Announced on 6 January 2026, the agreement covers VectorBuilder’s proprietary intravitreal capsid technology and paves the way for a potential strategic partnership, under which Ikarovec would take responsibility for the clinical development and commercialisation of IKAR-003.
Based on the program’s potential in intermediate AMD, the proposed deal is expected to be worth more than US$1 billion (AU$1.48 billion).
The capsid technology is designed to enable minimally invasive intravitreal delivery of IKAR-003 in a doctor’s office, improving accessibility for patients at the intermediate stage of AMD.
This patient group, which numbers in the millions globally, currently has no approved drug treatments and is at significant risk of progressing to geographic atrophy or wet AMD, both of which lead to irreversible vision loss.
IKAR-003 is intended as a one-time, AAV-delivered dual-pathway gene therapy that combines neuroprotection and complement modulation to preserve visual function and prevent disease progression.
Ikarovec president and CEO Dr Thomas Ciulla said the agreement strengthened the company’s pipeline by supporting an office-based treatment approach suited to large-scale adoption.
“We have assessed other intravitreal capsid options and are confident that VectorBuilder’s technology will provide superior efficacy,” he said.
Dr Ciulla added that IKAR-003 shares the same dual-pathway strategy as Ikarovec’s lead programme, IKAR-001, which is on track to enter clinical trials in late 2026 for geographic atrophy using subretinal delivery.
“We are therefore addressing two distinct patient populations with a delivery approach tailored to each,” he said.
VectorBuilder founder and chief scientist Dr Bruce Lahn said the companies’ long-standing collaboration had enabled close alignment on technical requirements.
He highlighted the role of VectorBuilder’s AI-powered DeepCap platform in engineering ocular capsids with broad retinal coverage.
According to the company, non-human primate studies have shown that the intravitreally administered capsids can target wide areas of the retina and transduce virtually all cells of the macula, with broader and more robust transduction than current clinical intravitreal capsids.
Dr Lahn said the combination of VectorBuilder’s capsid technology with Ikarovec’s dual-pathway approach and leadership team provided a strong foundation for developing new therapies for vision-threatening retinal diseases.
