ABBV-RGX-314 Subretinal Gene Therapy — PatSnap Eureka
ABBV-RGX-314 & Subretinal Gene Therapy in Wet AMD and Diabetic Retinopathy
rAAV-based anti-VEGF gene therapy is reshaping treatment of neovascular AMD and diabetic retinopathy. Explore the patent landscape behind ABBV-RGX-314, competing AAV platforms, and emerging delivery innovations — all analysed through PatSnap Eureka.
VEGF-Driven Neovascularization: The Central Therapeutic Target
Neovascular age-related macular degeneration (wet AMD) and diabetic retinopathy (DR)/diabetic macular edema (DME) represent the leading causes of vision loss in adults globally, driven pathologically by VEGF-mediated choroidal and retinal neovascularization. According to the World Health Organization, diabetic retinopathy alone affects hundreds of millions worldwide, underscoring the scale of unmet need.
Retrieved patent results converge on VEGF (vascular endothelial growth factor) as the dominant molecular driver of pathological neovascularization across wet AMD, DR, and DME. Multiple patent families explicitly name VEGF-A — and in some cases VEGF-C — as primary targets. The VEGF receptor fusion protein aflibercept — a chimeric decoy receptor capturing VEGF-A, VEGF-B, and placental growth factor (PlGF) — appears in the largest patent cluster, assigned to Adverum Biotechnologies.
Beyond VEGF, the dataset signals secondary targets including complement component 3 (C3), the transcription factor APE1/Ref-1, IRAK-M (Interleukin-1 receptor-associated kinase M) in RPE cells, VEGF-C co-targeted alongside VEGF-A by 4D Molecular Therapeutics, and the CBS (cystathionine beta-synthase) gene as a novel H2S-pathway-based intervention for DR. The rAAV-based gene therapy approach — delivering sustained anti-VEGF transgene expression — has emerged as a transformative alternative to chronic intravitreal injection burden. Learn more about PatSnap's life sciences intelligence solutions for tracking these emerging programs.
Seven Distinct Approaches Across the Wet AMD & DR Pipeline
Patent signals reveal a competitive landscape spanning AAV gene therapy, high-dose protein drugs, novel capsid engineering, complement inhibition, small molecules, cell therapy, and suprachoroidal delivery formulations.
AAV2-Variant + Aflibercept Transgene (Adverum / Ixo-vec)
Adverum Biotechnologies holds the largest and most geographically distributed patent family in this dataset, centered on a modified AAV2 capsid with heptapeptide insertion LGETTRP (SEQ ID NO:14) between positions 587 and 588 of the AAV2 VP1 capsid. The transgene encodes a polypeptide with ≥95% identity to aflibercept (SEQ ID NO:35), flanked by AAV2 ITRs. Filings span WO, EP, JP, KR, SG, MX, BR, CA (2021–2026). Clinical endpoints including 2-step or 3-step DRSS improvement and CST/CRT fluctuation reduction are explicitly claimed. This is the same LGETTRP capsid engineering associated with ixoberogene soroparvovec (ixo-vec).
Active EP grant (2025) · Multi-jurisdictionHuPTM Anti-VEGF Fab via rAAV (REGENXBIO — RGX-314 / ABBV-RGX-314)
REGENXBIO covers delivery of a fully human, glycosylated (HuGly) anti-hVEGF antigen-binding Fab fragment via rAAV to the retina/vitreous humor. Filings explicitly reference subretinal, suprachoroidal, and extrascleral administration routes. The retinal depot produces α2,6-sialylated glycan-containing Fab. Indications include wet AMD, dry AMD, DME, DR, and RVO, with the target population specifically identified as patients previously responsive to ranibizumab, aflibercept, or bevacizumab. Co-developed with AbbVie as ABBV-RGX-314. Explore the full patent analytics platform to map REGENXBIO's IP moat.
HuGly Fab · α2,6-sialylated glycanHigh-Dose Extended-Interval Protein Anti-VEGF (Bayer — 8 mg Aflibercept)
Bayer Healthcare LLC holds a large multinational patent family covering high-dose (8 mg) aflibercept intravitreal injection regimens for DR, DME, and nAMD — characterized by extended dosing intervals (HDq12, HDq16, HDq20; every 12, 16, or 20 weeks). EP filing references specific BCVA letter gain thresholds (≥7–9 ETDRS letters by week 60, baseline BCVA ~61–63 letters). This represents the incumbent protein drug's IP strategy for extending the dosing interval, competing directly with gene therapy's durable expression rationale. Filings span US, WO, EP, CA, KR, AU, CN, ID, NZ (2023–2026).
HDq16 / HDq20 · ≥7–9 ETDRS letter gainNovel AAV Capsid Engineering — Dual Payload (4D Molecular Therapeutics)
4D Molecular Therapeutics discloses variant AAV capsid proteins with enhanced infectivity of retinal cell types relative to parental serotypes. Their platform targets wet AMD through intravitreal delivery of rAAV encoding both aflibercept and a VEGF-C-targeting miRNA — a dual-payload approach. Preclinical non-human primate (NHP) data are referenced, including GFP/CD31 co-localization in retinal endothelial cells after intravitreal injection, and in vivo aflibercept expression measured in aqueous humor, vitreous, and retina+choroid. NHP choroidal neovascularization (CNV) models showed protective effects versus vehicle control.
NHP CNV model data · Dual VEGF-A/VEGF-CAAV + Complement Inhibition (Adverum; Gilot Ocup Therapeutics)
Adverum Biotechnologies filed a patent covering rAAV-mediated expression of anti-complement component 3 (anti-C3) molecules to block C3 activity in the eye, relevant to geographic atrophy and the complement-driven component of AMD pathology. Gilot Ocup Therapeutics covers dual-payload nucleic acid constructs encoding both anti-VEGF entities and complement negative regulators (including C3b inactivation/iC3b degradation in RPE and vitreous), with explicit coverage of AMD and DR. Two independent assignees converging on this combination rationale suggests IP race conditions may be forming around dual-payload AAV constructs for AMD.
Anti-C3 · C3b/iC3b inactivation in RPESuprachoroidal Delivery Formulations (REGENXBIO)
REGENXBIO's Chinese filings (2023) describe pharmaceutical compositions — including aggregate-forming and high-viscosity formulations — specifically optimized for suprachoroidal space (SCS) administration of rAAV encoding transgenes. Retrieved data claim that the effective dose required for therapeutic response via SCS delivery is lower than that required by subretinal or intravitreal routes, a meaningful practical advantage for manufacturability and therapeutic index. This route innovation is part of REGENXBIO's multi-dimensional IP moat for the ABBV-RGX-314 program.
Lower effective dose vs. subretinal · SCS routePipeline Intelligence: Modality Mix & Jurisdictional Coverage
Patent signal analysis from PatSnap Eureka reveals the relative weight of therapeutic modalities and the geographic footprint of leading programs in wet AMD and diabetic retinopathy.
Therapeutic Modality Distribution in Wet AMD & DR Patent Dataset
AAV-mediated anti-VEGF gene therapy dominates the patent dataset, with high-dose protein anti-VEGF and complement/novel targets representing significant secondary clusters.
Jurisdictional Filing Breadth by Leading Program
REGENXBIO and Adverum Biotechnologies lead on geographic patent coverage, with Bayer's high-dose aflibercept program showing strong multi-national registration-stage filing activity across 10+ jurisdictions.
Key Patent Holders: Programs, Approaches, and Filing Scope
Activity in this dataset is overwhelmingly patent-driven. The following organisations generate the most retrieved records, with distinct IP strategies across vector, transgene, and delivery dimensions.
| Assignee | Program / Technology | Key Jurisdictions | Filing Period | Modality Tag |
|---|---|---|---|---|
| REGENXBIO INC. | HuPTM HuGly anti-VEGF Fab via rAAV (RGX-314 / ABBV-RGX-314); subretinal, suprachoroidal, extrascleral routes; broad eye pathology platform | WO, AU, IL, MX, CA, US, KR, JP, CN, BR, SG | 2020–2025 | AAV Gene Therapy |
| Adverum Biotechnologies | AAV2 LGETTRP capsid variant + aflibercept transgene (ixo-vec); anti-C3 complement program | WO, EP, JP, KR, SG, MX, BR, CA | 2021–2026 | AAV Gene Therapy |
| Bayer Healthcare LLC | High-dose 8 mg aflibercept intravitreal injection; HDq12/q16/q20 extended dosing regimens for DR, DME, nAMD | US, WO, EP, CA, KR, AU, CN, ID, NZ | 2023–2026 | Protein Anti-VEGF |
| 4D Molecular Therapeutics | Variant AAV capsid + dual payload (aflibercept + VEGF-C miRNA); NHP CNV model data | CN, BR | 2022–2024 | Novel Capsid |
Track Every New Filing in This Space
PatSnap Eureka monitors global patent databases so you never miss a competitive signal in wet AMD or DR gene therapy.
Five Strategic Implications from the Patent Landscape
Patent signals across assignees, jurisdictions, and claim types reveal actionable intelligence for R&D strategy, business development, and competitive positioning in wet AMD and DR gene therapy.
AAV2-Variant Aflibercept IP Is Contested and Broad
Adverum Biotechnologies holds the deepest cluster of filings covering the specific LGETTRP capsid variant with aflibercept transgene for wet AMD/DME/DR across global jurisdictions. Any competitor entering this space with intravitreally delivered aflibercept-encoding AAV2 constructs faces substantial freedom-to-operate considerations around both the capsid engineering and the clinical indication claims.
REGENXBIO/AbbVie Holds a Multi-Dimensional IP Moat
The fully human post-translational modification (HuPTM/HuGly) claim on the anti-VEGF Fab product, combined with suprachoroidal and subretinal delivery route patents (including vitrectomy-free subretinal access), represents a multi-dimensional IP moat distinct from Adverum's capsid-centered claims. Track this via PatSnap's IP analytics platform.
Patent Language as a Proxy for Clinical Stage
Retrieved results contain several signals of clinical translation, though none include explicit clinical trial outcome data within the patent text. Multiple Adverum Biotechnologies filings explicitly claim 2-step or 3-step DRSS improvements following rAAV particle administration — an endpoint used in clinical trials for DR severity staging — suggesting the filings are anchored in or anticipate clinical study design rather than purely preclinical claims.
The Bayer high-dose aflibercept EP filing references specific BCVA letter gain thresholds (≥7–9 ETDRS letters by week 60, baseline BCVA approximately 61–63 letters) and maintenance BCVA targets (≥69 letters by weeks 48–60) — language characteristic of clinical trial result capture. The scope of international filings across more than 10 jurisdictions for the 8 mg aflibercept extended dosing program is consistent with registration-stage IP protection. The ClinicalTrials.gov registry provides complementary clinical stage data for these programs.
For 4D Molecular Therapeutics, the Chinese filing references NHP laser-induced CNV model experiments with quantitative OCT and histopathology, and in vivo aqueous humor/vitreous/retinal+choroidal aflibercept expression measurements — representing IND-enabling study level data. A 2025 REGENXBIO JP filing explicitly claims a subretinal administration method without vitrectomy, a clinically significant procedural refinement implying real-world surgical development activity. The European Medicines Agency and US FDA regulatory pathways are critical context for interpreting these filing signals. Discover how PatSnap customers use patent signals to anticipate clinical milestones.
ABBV-RGX-314 & Subretinal Gene Therapy — Key Questions Answered
ABBV-RGX-314 is a co-development between REGENXBIO and AbbVie. REGENXBIO's portfolio covers delivery of a fully human, glycosylated (HuGly) anti-hVEGF antigen-binding Fab fragment via rAAV to the retina/vitreous humor, with explicit reference to subretinal, suprachoroidal, and extrascleral administration routes. The technology exploits the immunological privilege of the subretinal space and the long-lived transduction capacity of AAV to create a sustained drug-delivery depot.
Adverum Biotechnologies holds the largest cluster of filings in this dataset, all centered on a modified AAV2 capsid containing the heptapeptide insertion LGETTRP (SEQ ID NO:14) between positions 587 and 588 of the AAV2 VP1 capsid — a variant that enhances retinal tropism. The transgene encodes a polypeptide with ≥95% identity to aflibercept (SEQ ID NO:35), flanked by AAV2 inverted terminal repeats (ITRs).
Bayer Healthcare LLC holds a large multinational patent family covering high-dose (8 mg) aflibercept intravitreal injection regimens for DR, DME, and nAMD — characterized by extended dosing intervals (HDq12, HDq16, HDq20; i.e., every 12, 16, or 20 weeks). These represent the protein drug incumbent's IP strategy for extending the dosing interval and reducing injection burden, competing directly with gene therapy's durable expression rationale.
A 2025 REGENXBIO JP filing explicitly claims a subretinal administration method without vitrectomy — a clinically significant procedural refinement that implies real-world surgical development activity. If clinically validated, this would significantly reduce procedural risk and lower the barrier to widespread adoption of subretinal gene therapy.
Novel targets include: complement component 3 (C3), with Adverum Biotechnologies filing covering anti-C3 antibody expression via rAAV; VEGF-C co-targeted alongside VEGF-A by 4D Molecular Therapeutics; IRAK-M expressed by AAV5 in retinal pigment epithelium (RPE) cells (University of Bristol); CBS (cystathionine beta-synthase) in diabetic retinopathy (Chengnuo Regenerative Medicine); and APE1/Ref-1 inhibitors (APX2009, APX2014) targeting NF-κB and HIF-1α transcription factors (Indiana University Research and Technology Corporation).
REGENXBIO's Chinese filings (2023) describe pharmaceutical compositions — including aggregate-forming and high-viscosity formulations — specifically optimized for suprachoroidal space (SCS) administration of rAAV encoding transgenes. Retrieved data claim that the effective dose required for therapeutic response via SCS delivery is lower than that required by subretinal or intravitreal routes, a meaningful practical advantage.
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References
- Gene therapy for eye pathologies (subretinal without vitrectomy) — REGENXBIO INC., 2025, JP [Patent]
- Gene therapy for eye pathologies — REGENXBIO INC., 2021, WO [Patent]
- Treatment of diabetic retinopathy with fully-human post-translationally modified anti-VEGF Fab — REGENXBIO INC., 2022, SG [Patent]
- Treatment of ocular diseases with fully-human post-translationally modified anti-VEGF Fab — REGENXBIO INC., 2020, IL [Patent]
- Gene therapy for eye conditions — REGENXBIO INC., 2022, JP [Patent]
- Subretinal administration method, suprachoroidal administration method, external scleral space administration method — REGENXBIO INC., 2021, BR [Patent]
- Formulations for suprachoroidal administration, such as formulations with aggregate formation — REGENXBIO INC., 2023, CN [Patent]
- Formulations for suprachoroidal administration, such as high-viscosity formulations — REGENXBIO INC., 2023, CN [Patent]
- Methods of treating ocular neovascular diseases using AAV2 variants encoding aflibercept — ADVERUM BIOTECHNOLOGIES, INC., 2025, EP [Patent]
- Methods of treating ocular neovascular diseases using AAV2 variants encoding aflibercept — ADVERUM BIOTECHNOLOGIES, INC., 2025, WO [Patent]
- Method for reducing CST fluctuation in neovascular AMD caused by a recombinant adeno-associated virus — ADVERUM BIOTECHNOLOGIES, INC., 2024, BR [Patent]
- Cassettes of anti-complement component 3 antibody, vectorization and therapeutic application — ADVERUM BIOTECHNOLOGIES, INC., 2023, CA [Patent]
- Extended, high dose VEGF antagonist regimens for treatment of angiogenic eye disorders — BAYER HEALTHCARE LLC, 2023, EP [Patent]
- Extended, high dose VEGF antagonist regimens for treatment of angiogenic eye disorders — BAYER HEALTHCARE LLC, 2023, US [Patent]
- Extended, high dose VEGF antagonist for treatment of angiogenic eye disorder — BAYER HEALTHCARE LLC, 2025, KR [Patent]
- Compositions and methods for treating eye disease associated with angiogenesis — 4D MOLECULAR THERAPEUTICS INC., 2024, BR [Patent]
- Variant AAV capsid proteins and methods of use — 4D Molecular Therapeutics Inc., 2022, CN [Patent]
- Nucleic acids encoding anti-VEGF entities and complement negative regulators — Gilot Ocup Therapeutics Ltd., 2025, CN [Patent]
- A combination for treating a retinal disease — HADASIT MEDICAL RESEARCH SERVICES AND DEVELOPMENT LTD., 2025, IL [Patent]
- World Health Organization — Blindness and Vision Impairment
- ClinicalTrials.gov — Gene Therapy for Wet AMD and Diabetic Retinopathy
- European Medicines Agency — Advanced Therapy Medicinal Products
- US Food and Drug Administration — Gene Therapy Guidance
All data and statistics on this page are sourced from the references above and from PatSnap's proprietary innovation intelligence platform. This report is derived from a limited set of patent and literature records retrieved across targeted searches and represents a snapshot of innovation signals within this dataset only. It should not be interpreted as a comprehensive view of the full field, clinical pipeline, or regulatory landscape.
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