Ikarovec to Advance Pipeline With Exclusive Worldwide Option to VectorBuilder’s Intravitreal Capsid Technology

Novel capsid technology to enable convenient delivery in the doctor’s office of IKAR-003 for intermediate AMD, designed to prevent progression to sight-threatening disease

NORWICH, England & CHICAGO–(BUSINESS WIRE)–#BioTechNews–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 $1bn.

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.

‘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,’ 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.’

‘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,’ he added.

‘We have worked with Ikarovec for several years and have developed a deep understanding of the company’s approach and technical requirements,’ said Dr. Bruce Lahn, Founder and Chief Scientist at VectorBuilder. ‘By utilizing 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.’

Notes for Editors

About Ikarovec

Ikarovec Limited is a UK-based, near clinical stage biotechnology company pioneering dual-pathway gene therapies for vision-threatening retinal diseases. Founded in 2020 as a spin-out from Quethera (acquired by Astellas Pharmaceuticals in 2018), the company is headquartered at Norwich Research Park in Norfolk, United Kingdom. Ikarovec’s proprietary bicistronic gene therapy platform builds on validated therapeutic approaches and adds synergistic second mechanisms, addressing what current single-pathway therapies miss with reduced development risk. The company’s lead programmes address large market opportunities including geographic atrophy and retinitis pigmentosa (using validated subretinal delivery) plus intermediate AMD and wet AMD (via doctor’s office-based intravitreal delivery). Ikarovec is led by an experienced team with expertise in bicistronic gene therapies and direct involvement in major ophthalmology products and exits totaling over $13 billion including Spark, Iveric and EyeBio. For more information, visit www.ikarovec.com and follow us on LinkedIn.

About VectorBuilder

VectorBuilder is a global leader in gene delivery technologies. As a trusted partner for thousands of labs and biotech/pharma companies across the globe, VectorBuilder offers a full spectrum of gene delivery solutions covering virtually all research and clinical needs from bench to bedside. The company’s R&D programs have focused on enhancing the functionality, safety and manufacturability of genetic medicines by solving major pain points in current gene delivery technologies such as low tissue specificity, immunogenicity, limited cargo size, and low manufacturing yield. VectorBuilder’s proprietary DeepCap platform combines machine learning and rational design with massively parallel ultradeep search of distributed sequence space to power the engineering of novel AAV capsids with best-in-class transduction efficiency and tissue specificity. Utilizing this platform, VectorBuilder has built a large panel of capsids targeting a wide range of therapeutically important tissues including CNS, PNS, muscle, heart, eye and ear. The company also operates about 10,000 m² of modern GMP facilities for genetic medicine manufacturing, fully compliant with ICH guidelines and US and EU regulatory standards. VectorBuilder has provided IND-enabling vectors to a global client base across the US, Europe, Australia, Japan, China, and South Korea. The company has been honored with numerous awards such as BioTech Breakthrough Award, PharmaVoice 100 Award, Bioz Rapid Star Award, and CDMO Leadership Award. For more information, visit www.vectorbuilder.com.

Contacts

For further information, please contact
Emma Palmer Foster, Communications + 44 (0)7880 787185

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