Recent presentations at ARVO and ASGCT highlighted several important developments in the treatment landscape for geographic atrophy (GA) and inherited retinal diseases, reinforcing growing industry interest in durable gene therapy and approaches that combine neuroprotection with complement pathway modulation.

1. The Field Is Moving Beyond Complement Monotherapy

      Multiple real-world studies presented at ARVO highlighted the challenges and limitations associated with complement inhibitor monotherapies. Real world patient compliance is relatively poor due to the low visual benefit and risk of new-onset wet AMD. There is a growing consensus on the importance of a durable treatment and the ability to address multiple drivers of GA and retinal degeneration.

      2. Growing Validation of Neuroprotection and PEDF Biology

      There was convergence of evidence supporting pigment epithelium-derived factor (PEDF) as a neuroprotective strategy for retinal disease. New basic science presentations further elucidated PEDF receptor biology and downstream signaling pathways associated with photoreceptor and retinal pigment epithelium (RPE) protection. Complementary translational studies demonstrated photoreceptor rescue and preservation across multiple disease models and delivery approaches. Importantly, clinical data presented from an investigator-sponsored study of a PEDF gene therapy demonstrated encouraging long-term visual outcomes, supporting the clinical therapeutic potential of PEDF-mediated neuroprotection in retinal degeneration.

      3. Independent Validation of Dual-Pathway Therapeutic Design

      Other scientific presentations independently converged on therapeutic architectures combining multiple mechanisms of action to target complex disease pathologies including addition neurotrophic or neuroprotective factors to treat GA. These programs provide additional validation for the broader scientific rationale that addressing both inflammatory and degenerative pathways may be necessary to achieve meaningful clinical benefit in retinal disease.

      4. Benefits of Gene Therapy Durability

      Gene therapy offers a durable and steady approach compared to frequent eye injections that cause drug levels to rise and fall over time. These fluctuations, along with the challenge of keeping up with repeated treatments, can reduce real-world effectiveness. In eyes with an inherited retinal dystrophy, it was shown that a single subretinal dose of an approved AAV gene therapy improved visual function over 5 years. Another Phase 2 study found that patients receiving an anti-VEGF subretinal gene therapy had a more stable retinal thickness that tended to achieve better vision outcomes while requiring fewer supplemental injections.

      As a result, the value of gene therapy may extend beyond reducing treatment burden. By delivering a steady, long-lasting therapeutic effect, it may also help improve visual outcomes compared with treatments that require frequent repeat dosing.

      5. Ikarovec’s IKAR-001 gene therapy for Geographic Atrophy

      IKAR-001 brings together all four key themes. It is a one-time subretinal gene therapy for geographic atrophy that goes beyond complement inhibition alone. The therapy combines PEDF-driven neuroprotection with complement regulation in a single construct, and is designed to preserve visual function, which current approved therapies do not. This dual approach reflects the direction the field is increasingly taking. By using sustained gene expression, IKAR-001 is designed to provide long-lasting, steady treatment without the fixed, frequent injections that current approved therapies require. In many ways, IKAR-001 was built around the same principles now being highlighted by ARVO and ASGCT.

      CSO Katie Binley, PhD to showcase dual-pathway gene therapy approach for Geographic Atrophy

      Norwich, UK – [Date] – Ikarovec Limited today announced that Chief Scientific Officer Katie Binley, PhD will present at the LSX Investival Showcase on Monday, November 17th, 2025, during London Life Sciences Week (November 16-21).

      Dr. Binley will deliver a showcase presentation titled “Dual-Pathway Gene Therapy for Geographic Atrophy” during the LSX Biotech session from 11:50 AM to 12:00 PM GMT. The presentation will highlight IKAR-001’s differentiated approach combining neuroprotection with complement modulation to preserve vision in patients with geographic atrophy.

      “We’re excited to share our progress with the global investment community at this premier life sciences event,” said Thomas Ciulla, MD, MBA, President and CEO of Ikarovec. “LSX Investival provides an excellent platform to engage with investors and partners who share our vision of transforming treatment for retinal degenerative diseases.”

      About LSX Investival

      LSX Investival is Europe’s leading biotech partnering and investment event, bringing together visionary leaders, investors, and innovators across the life sciences sector during London Life Sciences Week. The event facilitates strategic connections between emerging biotech companies and the global investment community.

      Both Dr. Ciulla and Dr. Binley will be available for one-on-one meetings throughout the conference. Investors and partners interested in learning more about Ikarovec’s dual-pathway gene therapy platform are encouraged to reach out to schedule a meeting.

      About Ikarovec

      Ikarovec is pioneering dual-pathway gene therapies for retinal degenerative diseases. The company’s proprietary bicistronic platform enables the expression of two synergistic therapeutic proteins from a single gene therapy vector, addressing multiple disease pathways simultaneously.

      Led by an experienced management team with proven exits including Luxturna (Spark Therapeutics, $4.8B to Roche), Izervay (Iveric Bio, $5.9B to Astellas), and EyeBio ($3.0B to Merck), Ikarovec is backed by LifeArc Ventures, Parkwalk Advisors, and UKI2S.

      For more information, visit www.ikarovec.com or follow us on LinkedIn.

      Ikarovec s advancing IKAR-001 with regulatory guidance supporting dual-pathway gene therapy approach

      Norwich, UK – [Date] – Ikarovec Limited today announced positive feedback from recent interactions with the U.S. Food and Drug Administration (FDA) regarding the development pathway for IKAR-001, the company’s first-in-class dual-pathway gene therapy for geographic atrophy (GA).

      The FDA feedback validated Ikarovec’s clinical development strategy and provided helpful guidance on the regulatory pathway as the company advances toward Investigational New Drug (IND) application submission.

      “The positive FDA feedback reinforces our confidence in IKAR-001’s differentiated approach,” said Thomas Ciulla, MD, MBA, President and CEO of Ikarovec. “Our dual-pathway strategy—combining neuroprotection with complement modulation—is designed to preserve vision, not just slow disease progression. This represents a significant advancement over current single-pathway therapies.”

      About IKAR-001

      IKAR-001 uniquely combines neuroprotection with complement modulation in a single bicistronic AAV8 gene therapy. This dual-pathway approach builds on the validated complement pathway demonstrated by recently approved GA therapies while adding neuroprotection to address functional vision benefit—what current approved therapies miss.

      Preclinical studies demonstrate IKAR-001’s superiority versus complement-only approaches. The gene-agnostic platform targets 5.0M GA patients worldwide with potential extension to 1.5M retinitis pigmentosa (RP) patients.

      Regulatory and Clinical Progress

      In addition to the positive FDA feedback, Ikarovec is advancing its Australian Clinical Trial Notification (CTN) application for Phase I/IIa trial initiation. The company has also:

      About Ikarovec

      Ikarovec is pioneering dual-pathway gene therapies for retinal degenerative diseases. The company’s proprietary bicistronic platform enables the expression of two synergistic therapeutic proteins from a single gene therapy vector, addressing multiple disease pathways simultaneously.

      Led by an experienced management team with proven exits including Luxturna (Spark Therapeutics, $4.8B to Roche), Izervay (Iveric Bio, $5.9B to Astellas), and EyeBio ($3.0B to Merck), Ikarovec is backed by LifeArc Ventures, Parkwalk Advisors, and UKI2S.

      Beyond IKAR-001, the company is developing office-based gene therapies for wet AMD (anti-angiogenic + anti-fibrotic) and intermediate AMD (neuroprotection + complement modulation).

      For more information, visit www.ikarovec.com or follow us on LinkedIn.

      Company releases updated corporate deck highlighting preclinical superiority, regulatory progress, and compelling investment opportunity

      Norwich, UK – 22/10/2025 – Ikarovec Limited today announced the release of its updated corporate presentation in conjunction with the company’s ongoing Series A fundraising round. The comprehensive deck showcases recent milestones, preclinical data demonstrating superiority versus complement-only approaches, and the company’s clinical development strategy for IKAR-001.

      “We’ve made substantial progress since initiating our Series A process,” said Thomas Ciulla, MD, MBA, President and CEO of Ikarovec. “With 25% of our $46 million round already verbally committed, positive FDA feedback, manufacturing underway, and our Australian clinical trial application advancing, we’re well-positioned to bring our differentiated dual-pathway gene therapy to patients with geographic atrophy.”

      Recent Milestones:

      The updated corporate presentation is available at [link].

      Investment Opportunity

      Ikarovec is advancing a de-risked dual-pathway gene therapy platform with near-term value inflection points, including IND approval and Phase I/IIa trial initiation. The company’s approach builds on validated complement modulation pathways while adding neuroprotection—designed to preserve vision, not just slow disease progression.

      With a large addressable market (5M GA patients, gene-agnostic extension to 1.5M RP patients), proven management team, and platform expansion opportunities in wet AMD and intermediate AMD, Ikarovec offers investors multiple value creation pathways.

      Connect with Ikarovec

      Investors interested in learning more about the Series A opportunity can meet with the Ikarovec team at LSX Investival on November 17th or throughout London Life Sciences Week (November 16-21). To schedule a meeting or request additional information, please contact [email/contact info].

      About Ikarovec

      Ikarovec is pioneering dual-pathway gene therapies for retinal degenerative diseases. The company’s proprietary bicistronic platform enables the expression of two synergistic therapeutic proteins from a single gene therapy vector, addressing multiple disease pathways simultaneously.

      Led by an experienced management team with proven exits including Luxturna (Spark Therapeutics, $4.8B to Roche), Izervay (Iveric Bio, $5.9B to Astellas), and EyeBio ($3.0B to Merck), Ikarovec is backed by LifeArc Ventures, Parkwalk Advisors, and UKI2S.

      For more information, visit www.ikarovec.com or follow us on LinkedIn.