Ikarovec and VectorBuilder Enter Global Option Agreement to Advance Gene Therapy for Intermediate AMD

Ikarovec has entered into an exclusive worldwide option agreement with VectorBuilder, a provider of gene delivery technologies. The agreement grants Ikarovec access to VectorBuilder’s novel adeno-associated virus (AAV) capsid technology for use in combination with Ikarovec’s gene therapy candidate, IKAR-003, for the treatment of intermediate age-related macular degeneration (AMD).

Under the terms of the agreement, Ikarovec will evaluate VectorBuilder’s proprietary capsid technology for further development of IKAR-003. Upon successful completion of this evaluation, the companies plan to enter into a strategic partnership in which Ikarovec will assume responsibility for the clinical development and commercialisation of the program. Based on the potential of IKAR-003 in intermediate AMD, the proposed transaction is expected to be valued at more than $1 billion.

VectorBuilder’s capsid technology is designed to enable delivery of IKAR-003 via a minimally invasive intravitreal injection that can be administered in a doctor’s office. According to Ikarovec, this approach is expected to enhance patient access, particularly for individuals with intermediate AMD. IKAR-003 is intended to halt or slow disease progression through a one-time injection of an AAV-delivered dual-pathway gene therapy that combines neuroprotection and complement modulation to preserve visual function.

“Partnering with VectorBuilder on IKAR-003 increases the value of Ikarovec’s pipeline by creating an office-based, intravitreally delivered product that is ideal for large-scale adoption in a disease prevention setting,” said Thomas Ciulla, MD, MBA, President and CEO of Ikarovec. “We have assessed other intravitreal capsid options and are confident that VectorBuilder’s technology will provide superior efficacy.”

Dr. Ciulla added that IKAR-003 shares the same dual-pathway therapeutic strategy as Ikarovec’s lead program, IKAR-001, which is on track to enter clinical trials in late 2026 for GA using a validated subretinal delivery approach. “With these programs, we are addressing two distinct patient populations with delivery methods tailored to the stage of disease,” he said.

According to VectorBuilder, non-human primate studies have shown that its intravitreally administered capsids can target wide areas of the retina and transduce virtually all cells of the macula, demonstrating broader and more robust retinal transduction than current intravitreal capsids in clinical use.