Evaluating the Safety and Preliminary Efficacy of EXG202 Gene Therapy for Neovascular AMD

NCT07178249 | Not Yet Recruiting Phase 1 DMC

• 1 other identifier: EXG202-111
• Study Type: interventional
• Target: 60
• Locations: 0 countries
• Sites: N/A

Brief Summary

VEGF inhibitors (anti-VEGF),such as aflibercept has been shown to be safe and effective for treating nAMD and have demonstrated improvement in vision. However, anti-VEGF therapy is administered frequently via intravitreal injection and can be a significant burden to the patients. EXG 202 is a recombinant adeno-associated virus (rAAV) gene therapy vector carrying a coding sequence for a soluble anti-VEGF protein. The long-term, stable delivery of this therapeutic protein following a 1 time gene therapy treatment for nAMD could potentially reduce the treatment burden of currently available therapies while maintaining vision with a favorable benefit.

Conditions

Wet Age-related Macular Degeneration

Outcome Measures

Primary Outcomes (2)

• Safety and tolerability after EXG202 injection

  Type, severity, and incidence of adverse events (AEs) and serious adverse events (SAEs) from Week 0 to 52, and dose limited toxicity

  Up to 52 weeks after treatment

• Preliminary Efficancy after EXG202 injection

  The Best Corrected Visual Acuity (BCVA) change from baseline

  Up to 52 weeks after treatment

Secondary Outcomes (1)

• Safety and tolerability after EXG202 injection

  Up to 24 weeks after treatment

Study Arms (4)

Dose escalation-Cohort 1

EXPERIMENTAL

Dose 1 :Administered via intravitreal injection

Biological: EXG202 injection

Dose escalation-Cohort 2

EXPERIMENTAL

Dose 2 :Administered via intravitreal injection

Biological: EXG202 injection

Dose escalation-Cohort 3

EXPERIMENTAL

Dose 3 :Administered via intravitreal injection

Biological: EXG202 injection

Dose escalation-Cohort 4

EXPERIMENTAL

Dose 4 :Administered via intravitreal injection

Biological: EXG202 injection

Interventions

EXG202 injection BIOLOGICAL

EXG202 injection is a gene therapy product for the treatment of wet (neovascular) Age-related Macular Degeneration（wAMD） with a single intravitreal injection and administration.

Dose escalation-Cohort 1; Dose escalation-Cohort 2; Dose escalation-Cohort 3; Dose escalation-Cohort 4

Eligibility Criteria

• Age: 50 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Male or female, aged ≥50 years old;
• The study eye must be diagnosed of wAMD and current active lesions;
• Subjects have clear refractive media and sufficient pupil dilation at the time of screening to obtain high-quality retinal images for confirmation of diagnosis;
• Subjects (including male subjects) have no pregnancy plans during the screening period and the entire trial period and voluntarily take effective contraceptive measures and have no sperm or egg donation plans;
• Subjects are Voluntarily participate in this clinical trial, understand the research procedures and sign the informed consent form before screening; subjects have good compliance and are willing to abide by the research procedures.

You may not qualify if:

• The study eye has any eye disease other than wAMD that may affect central vision and/or macular detection ;
• The study eye has a history of retinal detachment or retinal detachment during the screening period;
• The study eye has MNV caused by reasons other than wAMD (such as diabetic retinopathy, pathological myopia, retinal vein occlusion, angioid streak disease, ocular histoplasmosis, trauma, etc.), and a history of macular pathology unrelated to wAMD;
• Any intraocular surgery is planned for the study eye during the study period;
• The study eye currently has retinal angiomatous proliferation (RAP), central serous chorioretinopathy or symptomatic vitreomacular traction syndrome;
• Presence of glaucoma or optic neuropathy that involves or compromises the central visual field of the study eye or presence of uncontrolled high intraocular pressure in the study eye；
• The fellow eye meets the definition of legal blindness；
• Hormone-induced increased intraocular pressure in any eye；
• Active infection in any eye this trial (such as those who cannot understand and comply with the trial requirements or are deemed unsuitable for safety reasons).

Sponsors & Collaborators

• Hangzhou Jiayin Biotech Ltd: lead

Study Officials

• Mingwei ZHAO, PhD

  Peking University People's Hospital

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Sara Yang

CONTACT

13957164092; sarayang@exegenesisbio.com

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 10, 2025
• First Posted: September 17, 2025
• Study Start: October 16, 2025
• Primary Completion (Estimated): February 28, 2028
• Study Completion (Estimated): December 30, 2032
• Last Updated: September 17, 2025

Record last verified: 2025-09

Data Sharing

• IPD Sharing: Will not share