Ikarovec and VectorBuilder enter into exclusive worldwide option agreement
Ikarovec Ltd, which is developing dual-pathway gene therapies for vision-threatening retinal diseases, and VectorBuilder, a global leader in gene delivery technologies, have entered into an exclusive worldwide option agreement for VectorBuilder’s novel AAV capsid technology to be used in combination with Ikarovec’s gene therapy candidate IKAR-003 for intermediate age-related macular degeneration (AMD).
Following successful further evaluation of the technology, the companies will engage in a strategic partnership under which Ikarovec will be responsible for clinical development and commercialisation of IKAR-003. Based on the potential of IKAR-003 in intermediate AMD, the proposed deal is expected to be worth in excess of $1 billion.
VectorBuilder’s proprietary capsid technology will allow IKAR-003 to be delivered to the eye via the minimally invasive intravitreal route in a doctor’s office. This will enhance access to the treatment for the target patient population - those at the earlier, intermediate AMD stage for whom prevention of disease progression is a priority. IKAR-003 is designed to prevent this progression through a one-time injection of an AAV-delivered dual-pathway gene therapy combining neuroprotection and complement modulation to preserve visual function.
Patients with intermediate AMD, a condition that affects millions worldwide and currently has no approved drug treatments, are at significant risk of progressing to geographic atrophy or wet AMD, both of which cause irreversible vision loss.
Thomas Ciulla, MD, MBA, President and CEO of Ikarovec, said:
Partnering with VectorBuilder on IKAR-003 increases the value of Ikarovec’s pipeline by creating an office-based intravitreally delivered product, ideal for large-scale adoption in the prevention of disease progression setting. We have assessed other intravitreal capsid options and are confident that VectorBuilder’s technology will provide superior efficacy. IKAR-003 uses the same dual-pathway approach as our lead programme, IKAR-001, which remains on track for clinical trial initiation in late 2026 in geographic atrophy, using validated subretinal delivery in this advanced stage of disease. We are therefore addressing two distinct patient populations with a delivery approach tailored to each.
Dr. Bruce Lahn, Founder and Chief Scientist at VectorBuilder, said:
We have worked with Ikarovec for several years and have developed a deep understanding of the company’s approach and technical requirements. By utilising our AI-powered DeepCap platform that integrates rational design with ultradeep search of AAV sequence space, we were able to engineer best-in-class ocular capsids. In non-human primate studies, our intravitreally administered capsids can target wide areas of the retina and successfully transduce virtually all cells of the macula, demonstrating broader and more robust transduction than current clinical intravitreal capsids. We are confident that our capsid technology, combined with Ikarovec's innovative dual-pathway therapeutic approach and experienced leadership team, will create a strong foundation for developing transformative therapies for patients with vision-threatening retinal diseases.
