surabgene lomparvovec (ABBV-RGX-314) / REGENXBIO, AbbVie - LARVOL DELTA

Study to Assess the Injection Burden, Adverse Events, Change in Disease Activity, and Long-Term Preservation of Visual Acuity of Surabgene Lomparvovec in Adult Participants With Neovascular Age-Related Macular Degeneration (nAMD) (clinicaltrials.gov) - P3 | N=561 | Recruiting | Sponsor: AbbVie | Not yet recruiting ➔ Recruiting

Adverse events • Enrollment open • Real-world evidence • Age-related Macular Degeneration • Macular Degeneration • Ophthalmology • Retinal Disorders • Wet Age-related Macular Degeneration

AAVIATE: RGX-314 Gene Therapy Administered in the Suprachoroidal Space for Participants With Neovascular Age-Related Macular Degeneration (nAMD) (clinicaltrials.gov) - P2 | N=140 | Recruiting | Sponsor: AbbVie | Trial completion date: Aug 2026 ➔ Feb 2027 | Trial primary completion date: Oct 2025 ➔ Aug 2026

Trial completion date • Trial primary completion date • Age-related Macular Degeneration • Gene Therapies • Macular Degeneration • Ophthalmology • Retinal Disorders • Wet Age-related Macular Degeneration

REGENXBIO Inc....announced that it will present interim data from the Phase II ALTITUDE trial evaluating suprachoroidal delivery of surabgene lomparvovec (ABBV-RGX-314, sura-vec) for the treatment of diabetic retinopathy (DR) at the American Academy of Ophthalmology 2025 Annual Meeting. (Regenxbio Press Release)

P2 data • Diabetic Retinopathy

REGENXBIO Announces Completion of Enrollment in Pivotal Trials of Subretinal Surabgene Lomparvovec for Wet AMD (PRNewswire) - "Over 1,200 participants enrolled in ATMOSPHERE and ASCENT pivotal trials, representing largest global gene therapy program ever conducted....Topline pivotal data expected in Q4 2026."

Clinical data • Enrollment closed • Wet Age-related Macular Degeneration

Computational Fluid Dynamics Modeling of Intravitreal Ranibizumab Bolus Versus Subretinal ABBV-RGX-314 Transgene Product in Human Eyes. (PubMed, Transl Vis Sci Technol) - "The current study results predict that the 2 pivotal trial ABBV-RGX-314 doses (6.4E10 and 1.3E11 genome copies/eye) are expected to achieve and maintain sufficient retinal ABBV-RGX-314 TP levels for the treatment of nAMD. CFD modeling effectively bridges limited human ocular PK data with rich preclinical data, supporting model-informed drug development (MIDD) for clinical dose selection."

Journal • Age-related Macular Degeneration • Macular Degeneration • Ophthalmology • Retinal Disorders • Wet Age-related Macular Degeneration

ALTITUDE: RGX-314 Gene Therapy Administered in the Suprachoroidal Space for Participants With Diabetic Retinopathy (DR) With and Without Center Involved-Diabetic Macular Edema (CI-DME) (clinicaltrials.gov) - P2 | N=139 | Active, not recruiting | Sponsor: AbbVie | Trial completion date: Sep 2026 ➔ Dec 2026 | Trial primary completion date: Feb 2026 ➔ Jun 2026

Trial completion date • Trial primary completion date • Diabetic Macular Edema • Diabetic Retinopathy • Gene Therapies • Ophthalmology • Retinal Disorders

ATMOSPHERE: Pivotal 1 Study of ABBV-RGX-314 (Also Known as RGX-314) Gene Therapy Administered Via Subretinal Delivery One Time in Participants With nAMD (clinicaltrials.gov) - P2/3 | N=630 | Recruiting | Sponsor: AbbVie | Trial completion date: May 2026 ➔ Nov 2027 | Trial primary completion date: May 2025 ➔ Dec 2026

Trial completion date • Trial primary completion date • Age-related Macular Degeneration • Gene Therapies • Macular Degeneration • Ophthalmology • Retinal Disorders • Wet Age-related Macular Degeneration

ALTITUDE: RGX-314 Gene Therapy Administered in the Suprachoroidal Space for Participants With Diabetic Retinopathy (DR) With and Without Center Involved-Diabetic Macular Edema (CI-DME) (clinicaltrials.gov) - P2 | N=139 | Active, not recruiting | Sponsor: AbbVie | Recruiting ➔ Active, not recruiting

Enrollment closed • Diabetic Macular Edema • Diabetic Retinopathy • Gene Therapies • Ophthalmology • Retinal Disorders

ASCENT: Pivotal 2 Study of RGX-314 Gene Therapy in Participants With nAMD (clinicaltrials.gov) - P3 | N=660 | Recruiting | Sponsor: AbbVie | Trial completion date: Nov 2026 ➔ Nov 2027 | Trial primary completion date: Nov 2025 ➔ Oct 2026

Trial completion date • Trial primary completion date • Age-related Macular Degeneration • Gene Therapies • Macular Degeneration • Ophthalmology • Retinal Disorders • Wet Age-related Macular Degeneration

REGENXBIO Announces Pivotal Program for Surabgene Lomparvovec in Diabetic Retinopathy (PRNewswire) - P2 | N=139 | ALTITUDE (NCT04567550) | Sponsor: AbbVie | "REGENXBIO...announced it will initiate a pivotal Phase IIb/III clinical trial for investigational surabgene lomparvovec...in diabetic retinopathy (DR) using suprachoroidal delivery and a corresponding amendment to its eyecare collaboration with AbbVie...In the Phase II ALTITUDE trial, sura-vec was well tolerated in subjects with non-proliferative diabetic retinopathy (NPDR) at dose levels 1, 2, and 3. As of June 9, 2025, there were no drug-related serious adverse events. No intraocular inflammation was observed through two years at dose level 3 (1.0x1012 GC/eye) (n = 15) with short-course topical prophylactic steroids...Updated Phase II ALTITUDE results will be presented at a future medical meeting....AbbVie will pay REGENXBIO $100 million upon first subject dosed in the Phase IIb/III trial and an additional $100 million upon first subject dosed in a second Phase III clinical trial. REGENXBIO will pay for all costs..."

Financing • New P2/3 trial • P2 data • Diabetic Retinopathy

Gene Therapy in Age-related Macular Degeneration. (PubMed, Int Ophthalmol Clin) - "Current trials in wet AMD include ADVM-022 and RGX-314. Current trials in dry AMD include GT-005 and JNJ-1887."

Journal • Review • Age-related Macular Degeneration • Dry Age-related Macular Degeneration • Gene Therapies • Macular Degeneration • Ophthalmology • Retinal Disorders • Wet Age-related Macular Degeneration

Clearside Biomedical Announces Multiple Presentations to be Featured at the Clinical Trials at the Summit Meeting (GlobeNewswire) - "Clearside Biomedical, Inc...announced today that the use of its SCS delivery platform will be featured in multiple presentations at the Clinical Trials at the Summit (CTS) Meeting on June 21, 2025 in Las Vegas, Nevada."

Clinical data • Age-related Macular Degeneration • Choroidal Melanomas • Diabetic Retinopathy

Study to Assess the Injection Burden, Adverse Events, Change in Disease Activity, and Long-Term Preservation of Visual Acuity of Surabgene Lomparvovec in Adult Participants With Neovascular Age-Related Macular Degeneration (nAMD) (clinicaltrials.gov) - P3 | N=561 | Not yet recruiting | Sponsor: AbbVie

Adverse events • New P3 trial • Real-world evidence • Age-related Macular Degeneration • Macular Degeneration • Ophthalmology • Retinal Disorders • Wet Age-related Macular Degeneration

Beyond the injection: delivery systems reshaping retinal disease management. (PubMed, Expert Opin Pharmacother) - "Specific innovations discussed include the ranibizumab port delivery system, EYP-1901 (Duravyu, vorolanib implant), KSI-301 (tarcocimab tedromer), KSI-501, OTX-TKI (Axpaxli, axitinib implant), 4D-150, revakinagene taroretcel-lwey (Encelto, NT-501, encapsulated cell therapy), Xipere (triamcinolone acetonide injectable suspension), AU-011 (belzupacap sarotalocan targeted delivery), ABBV-RGX-314, elamipretide, and OCS-01 (high concentration dexamethasone)...Challenges include overcoming the blood-retinal barrier, surgical complications with implantable devices, and ensuring patient adherence. Advances in smart delivery systems, drug formulations, and predictive models, alongside interdisciplinary collaboration, will be crucial in achieving personalized, effective, and sustainable retinal therapies."

Journal • Review • Age-related Macular Degeneration • Diabetic Macular Edema • Diabetic Retinopathy • Gene Therapies • Macular Degeneration • Ophthalmology • Retinal Disorders

REGENXBIO Announces Strategic Royalty Monetization Agreement for Up to $250 Million (PRNewswire) - "REGENXBIO...announced the closure of a non-dilutive, limited recourse royalty bond agreement of up to $250 million with Healthcare Royalty (HCRx). This agreement monetizes select anticipated royalties and milestones, and provides both immediate and expected future, non-dilutive capital. REGENXBIO received $150 million at closing, extending the Company's expected cash runway into early 2027. 'This strategic financing brings future potential funds forward and extends our runway beyond multiple meaningful milestones, including...top-line data readouts for two pivotal studies of subretinal ABBV-RGX-314 for wet AMD,' said Mitchell Chan Chief Financial Officer of REGENXBIO."

Financing • Wet Age-related Macular Degeneration

Gene Therapy in Diabetic Retinopathy and Diabetic Macular Edema: An Update. (PubMed, J Clin Med) - "Preliminary results from the SPECTRA (4D-150) and ALTITUDE (ABBV-RGX-314) studies are promising, demonstrating an improvement in the diabetic retinopathy severity score and a reduction in the treatment burden. In contrast, the INFINITY (ADVM-022) trial was complicated by several cases of severe inflammation and hypotony that led the sponsor to discontinue further development of this product for DME."

Journal • Review • Diabetic Macular Edema • Diabetic Retinopathy • Gene Therapies • Glaucoma • Inflammation • Ocular Inflammation • Ophthalmology • Retinal Disorders • Uveitis

REGENXBIO Reports First Quarter 2025 Financial Results and Recent Operational Highlights (PRNewswire) - "Sura-vec for the Treatment of Wet AMD (Subretinal Delivery): Enrollment is ongoing in the ATMOSPHERE and ASCENT pivotal trials. REGENXBIO and AbbVie expect to share topline results in 2026."

Clinical data • Enrollment status • Wet Age-related Macular Degeneration

Gene therapy in neovascular age related macular degeneration: an update. (PubMed, Graefes Arch Clin Exp Ophthalmol) - "Ixoberogene soroparvovec, RGX-314 and 4D-150 are the leading NV-AMD genetic treatment programs. Pre-clinical models suggest that genome surgery with clustered regularly interspaced short palindromic repeats (CRISPR) may be another option in the future."

Journal • Review • Age-related Macular Degeneration • Gene Therapies • Macular Degeneration • Oncology • Ophthalmology • Retinal Disorders • Wet Age-related Macular Degeneration

ELAAVATE: Phase 2 Trial of RGX-314 in Adults With Center Involved - Diabetic Macular Edema (CI - DME) (clinicaltrials.gov) - P2 | N=18 | Recruiting | Sponsor: Sierra Eye Associates

New P2 trial • Diabetic Macular Edema • Gene Therapies • Ophthalmology

Subretinal Delivery of Investigational ABBV-RGX-314 as a Gene Therapy for Neovascular Age-Related Macular Degeneration (nAMD): Interim Results of Bilateral Dosing from a Fellow Eye Study (ARVO 2025) - "Layman Abstract (optional): Provide a 50-200 word description of your work that non-scientists can understand. Describe the big picture and the implications of your findings, not the study itself and the associated details."

Gene therapy • Age-related Macular Degeneration • Macular Degeneration • Ocular Inflammation • Ophthalmology • Retinal Disorders • Uveitis • Wet Age-related Macular Degeneration

ALTITUDE: RGX-314 Gene Therapy Administered in the Suprachoroidal Space for Participants With Diabetic Retinopathy (DR) With and Without Center Involved-Diabetic Macular Edema (CI-DME) (clinicaltrials.gov) - P2 | N=130 | Recruiting | Sponsor: AbbVie | N=100 ➔ 130 | Trial completion date: Oct 2024 ➔ Sep 2026 | Trial primary completion date: May 2024 ➔ Feb 2026

Enrollment change • Trial completion date • Trial primary completion date • Diabetic Macular Edema • Diabetic Retinopathy • Gene Therapies • Ophthalmology • Retinal Disorders

ATMOSPHERE: Pivotal 1 Study of ABBV-RGX-314 (Also Known as RGX-314) Gene Therapy Administered Via Subretinal Delivery One Time in Participants With nAMD (clinicaltrials.gov) - P2/3 | N=540 | Recruiting | Sponsor: AbbVie | N=300 ➔ 540

Enrollment change • Age-related Macular Degeneration • Gene Therapies • Macular Degeneration • Ophthalmology • Retinal Disorders • Wet Age-related Macular Degeneration

AbbVie and REGENXBIO Announce Updates on the ABBV-RGX-314 Clinical Program (PRNewswire) - "AbbVie...and REGENXBIO Inc...today announced updates to the ABBV-RGX-314 clinical program....Data from the ATMOSPHERE and ASCENT pivotal trials evaluating the safety and efficacy of the subretinal delivery of ABBV-RGX-314 in patients with wet AMD are expected in 2026."

P2/3 data • P3 data • Age-related Macular Degeneration

Subretinal Gene Therapy for Treatment of Retinal and Choroidal Vascular Diseases. (PubMed, Am J Ophthalmol) - "This review article discusses investigational subretinal gene therapies for retinal vascular diseases, including AVA-101, an adeno-associated viral (AAV) 2 vector expressing soluble vascular endothelial growth factor (VEGF) receptor 1, ABBV-RGX-314, an AAV8 vector expressing an anti-VEGF-A antibody fragment, and EXG102-031, an AAV8 vector expressing a recombinant protein that blocks VEGF family members and angiopoietin 2. In contrast, subretinal injection of some doses of ABBV-RGX-314 have shown evidence of controlling exudation and the maintaining vision, as well as safety and tolerability, leading to two ongoing pivotal trials comparing subretinal delivery of two different doses of ABBV-RGX-314 versus intravitreal injections of 0.5mg ranibizumab or 2mg aflibercept. These preliminary results are an encouraging and welcome development in the search for efficacious, long-duration treatments for retinal vascular diseases."

Gene therapy • Journal • Age-related Macular Degeneration • Gene Therapies • Inflammation • Macular Degeneration • Ophthalmology • Retinal Disorders

Suprachoroidal drug delivery: a versatile therapeutic platform. (PubMed, Expert Opin Drug Deliv) - No abstract available

Journal • Age-related Macular Degeneration • Diabetic Retinopathy • Gene Therapies • Macular Degeneration • Macular Edema • Ocular Inflammation • Ophthalmology • Retinal Disorders • Uveitis