Ikarovec Enters $1bn-Plus Option Deal with VectorBuilder for AMD Gene Therapy

COMPANY PROFILE
  • Ikarovec and VectorBuilder have signed an exclusive worldwide option agreement covering VectorBuilder’s AAV capsid technology for use with Ikarovec’s gene therapy candidate IKAR-003 in intermediate age-related macular degeneration.
  • Subject to further evaluation, the proposed strategic partnership is expected to be worth in excess of $1bn, with Ikarovec responsible for clinical development and commercialisation.

Ikarovec Ltd and VectorBuilder have entered into an exclusive worldwide option agreement for VectorBuilder’s novel adeno-associated virus (AAV) capsid technology. The technology will be evaluated for use with Ikarovec’s gene therapy candidate IKAR-003, which is being developed for the treatment of intermediate age-related macular degeneration (AMD).

Under the terms outlined, the companies plan to progress to a strategic partnership following successful further evaluation of the capsid technology. Ikarovec would then take responsibility for the clinical development and commercialisation of IKAR-003. Based on the potential of the programme in intermediate AMD, the proposed deal is expected to be worth more than $1bn.

VectorBuilder’s proprietary capsid technology is designed to enable delivery of IKAR-003 via an intravitreal injection administered in a doctor’s office. According to the companies, this minimally invasive route is intended to improve access for patients at the intermediate stage of AMD, where preventing disease progression is a clinical priority. IKAR-003 is a one-time AAV-delivered dual-pathway gene therapy combining 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, ideal for large-scale adoption in the prevention of disease progression setting,”

Thomas Ciulla, MD, MBA, President and CEO of Ikarovec

Ikarovec stated that IKAR-003 uses the same dual-pathway approach as its lead programme, IKAR-001, which is on track for clinical trial initiation in late 2026 in geographic atrophy using subretinal delivery. The company said this strategy allows it to address two distinct patient populations using delivery approaches tailored to disease stage.

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