What is the mechanism of action of this treatment?

The most common treatments for Age-Related Macular Degeneration (AMD) include anti-VEGF (vascular endothelial growth factor) therapies, which work by inhibiting the growth of abnormal blood vessels in the retina that can lead to vision loss. These treatments, such as ranibizumab and aflibercept, are typically administered through regular intraocular injections. Gene therapy approaches, like KH631, aim to deliver a protein that blocks VEGF using an adeno-associated viral vector, potentially offering a one-time treatment that reduces the need for frequent injections. This is significant for AMD patients as it can improve adherence to treatment, reduce the burden of regular medical visits, and potentially provide more sustained protection against vision loss.

What side effects are associated with this type of intervention?

Common treatments for Age-Related Macular Degeneration (AMD) include anti-VEGF therapies, which are known to cause side effects such as eye pain, increased intraocular pressure, vitreous floaters, and inflammation. Gene therapies like KH631, which deliver proteins to block VEGF, may also carry risks of immune reactions, retinal detachment, and potential long-term effects on retinal cells. Regular monitoring by an ophthalmologist is essential to manage these side effects effectively.

What prior FDA approvals exist?

The FDA has approved several treatments for Age-Related Macular Degeneration (AMD) that target vascular endothelial growth factor (VEGF) to reduce abnormal blood vessel growth and fluid leakage. Notable drugs include ranibizumab (Lucentis), aflibercept (Eylea), and bevacizumab (Avastin), which are anti-VEGF therapies administered via intraocular injections. These treatments have shown significant efficacy in improving vision and reducing fluid in patients with neovascular AMD. While gene therapies like KH631, which use adeno-associated viral vectors to deliver VEGF-blocking proteins, are still under investigation, they hold promise for reducing the frequency of treatments by providing a more sustained therapeutic effect.

What other types of research exist?

Promising novel alternatives to KH631 for AMD treatment include: 1) Faricimab, a bispecific antibody targeting both VEGF-A and Ang-2, which has shown efficacy in reducing retinal fluid and improving vision. 2) RGX-314, another gene therapy using an adeno-associated viral vector to deliver an anti-VEGF protein, currently in advanced clinical trials. 3) KSI-301, an anti-VEGF antibody biopolymer conjugate designed for extended durability, reducing the frequency of injections.

← Back to Search

Gene Therapy

Gene Therapy for Age-Related Macular Degeneration

Phase 1

Recruiting

Research Sponsored by Chengdu Origen Biotechnology Co., Ltd.

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Be older than 18 years old

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 104 weeks

Awards & highlights

No Placebo-Only Group

Summary

This trial is testing a new gene therapy called KH631 for patients with a severe eye condition known as neovascular AMD. The therapy uses a harmless virus to deliver a protein that blocks a harmful substance in the eye. This could help improve vision and reduce the need for frequent eye injections.

Who is the study for?

This trial is for men and women aged 50 to 85 with neovascular Age-related Macular Degeneration (AMD) who've had some improvement from previous anti-VEGF treatments. They must have a certain level of vision in the affected eye and be able to undergo high-quality imaging. Those with recent eye surgery, other causes of CNV, or long-term intraocular steroid use can't participate.

What is being tested?

KH631 gene therapy is being tested for safety and tolerability in this Phase I trial. It's designed as a one-time treatment delivering a protein that blocks VEGF, which could potentially reduce the need for regular eye injections currently used to treat neovascular AMD.

What are the potential side effects?

As this is an early-stage trial focusing on safety, specific side effects are not listed but may include typical risks associated with gene therapy such as immune reactions, inflammation at the injection site, or changes in vision.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 104 weeks

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~104 weeks

This trial's timeline: 3 weeks for screening, Varies for treatment, and 104 weeks for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Safety (Type, Severity and Incidence of ocular and systemic AEs and SAEs)

Secondary study objectives

Best Corrected Visual Acuity

Rescue Injections

Safety (type, severity and incidence of ocular and systemic AEs and SAEs)

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

5Treatment groups

Experimental Treatment

Group I: KH631 Dose 5Experimental Treatment1 Intervention

KH631 One-Time Intraocular Injection Dose Level 5

Group II: KH631 Dose 4Experimental Treatment1 Intervention

KH631 One-Time Intraocular Injection Dose Level 4

Group III: KH631 Dose 3Experimental Treatment1 Intervention

KH631 One-Time Intraocular Injection Dose Level 3

Group IV: KH631 Dose 2Experimental Treatment1 Intervention

KH631 One-Time Intraocular Injection Dose Level 2

Group V: KH631 Dose 1Experimental Treatment1 Intervention

KH631 One-Time Intraocular Injection Dose Level 1

Do I qualify?Apply below to find out

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

The most common treatments for Age-Related Macular Degeneration (AMD) include anti-VEGF (vascular endothelial growth factor) therapies, which work by inhibiting the growth of abnormal blood vessels in the retina that can lead to vision loss. These treatments, such as ranibizumab and aflibercept, are typically administered through regular intraocular injections. Gene therapy approaches, like KH631, aim to deliver a protein that blocks VEGF using an adeno-associated viral vector, potentially offering a one-time treatment that reduces the need for frequent injections. This is significant for AMD patients as it can improve adherence to treatment, reduce the burden of regular medical visits, and potentially provide more sustained protection against vision loss.