What side effects are associated with this type of intervention?

Common treatments for Age-Related Macular Degeneration (AMD) include anti-VEGF injections, photodynamic therapy, and laser therapy. Known side effects of these treatments can include eye pain, increased intraocular pressure, inflammation, and in rare cases, retinal detachment or infection. Gene therapy treatments, such as those being studied in the GT005 trial, aim to modify or correct genetic factors contributing to AMD. Potential side effects of gene therapy may include immune reactions, inflammation, and off-target genetic effects, although long-term safety data is still being gathered.

What other types of research exist?

Promising alternatives to GT005 for AMD treatment in 2024 include: 1) RNA interference therapies targeting specific genetic factors involved in AMD, such as those using siRNA or antisense oligonucleotides. 2) CRISPR-Cas9 based gene editing therapies aimed at correcting genetic mutations associated with AMD. 3) Novel pharmacological agents that target the underlying molecular pathways of AMD, such as inhibitors of complement factor D or other components of the complement system. These approaches are in various stages of clinical development and offer potential new avenues for treating AMD.

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Gene Therapy

GT005 for Dry Age-Related Macular Degeneration

Q: What prior FDA approvals exist?

As of now, the FDA has approved several treatments for Age-Related Macular Degeneration (AMD), primarily focusing on anti-VEGF (vascular endothelial growth factor) therapies such as ranibizumab (Lucentis), aflibercept (Eylea), and bevacizumab (Avastin). These treatments are designed to inhibit abnormal blood vessel growth and leakage in the retina. However, gene therapy approaches like GT005, which aim to modify or correct genetic factors contributing to Macular Atrophy in AMD, are still in the experimental stages. GT005 represents a novel approach in this field, and ongoing clinical trials are evaluating its safety and efficacy.

Phase 1 & 2

Waitlist Available

Research Sponsored by Gyroscope Therapeutics Limited

Eligibility Criteria Checklist

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

Must have

Have a clinical diagnosis of GA secondary to AMD in the study eye, as determined by the Investigator, and a diagnosis of AMD in the contralateral eye (except if the subject is monocular)

Cohorts 1 to 3: the GA lesion in the treatment eye must reside completely within the FAF fundus image

Must not have

Have clinically significant cataract that may require surgery during the study period in the study eye

Have received any investigational product for the treatment of GA within the past 6 months or 5 half-lives (whichever is longer), other than nutritional supplements such as the Age-Related Eye Disease Study (AREDS) formula

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 240 weeks

Awards & highlights

No Placebo-Only Group

Summary

This trial tested an eye injection treatment called GT005 for people with vision loss from Macular Atrophy due to AMD. The goal was to improve or maintain their vision. The trial was stopped because the treatment was not effective, but it was not due to safety issues.

Who is the study for?

This trial is for adults aged 55+ with Dry Age-Related Macular Degeneration (AMD) causing macular atrophy. Participants must have specific sizes of GA lesions in their eyes and meet certain vision acuity requirements. Women capable of childbearing need a negative pregnancy test, and all participants should be able to attend study visits.

What is being tested?

The FOCUS trial is testing GT005, delivered using the Orbit Subretinal Delivery System, on people with Dry AMD to see if it's safe and effective in treating macular atrophy. It's an open-label Phase I/II multicentre study where everyone receives the treatment.

What are the potential side effects?

Potential side effects are not specified but may include typical risks associated with eye injections such as discomfort, redness, infection risk or changes in vision. As this is a first-in-human study for GT005, close monitoring will occur for any unexpected side effects.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I have been diagnosed with GA due to AMD in one eye and AMD in the other.

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My eye condition is fully visible in a specific eye test image.

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I can attend all required study visits and complete the procedures.

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I am 55 years old or older.

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I have AMD in both eyes, confirmed by color photos.

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My eye condition affects an area between 1.25mm2 and 17.5mm2.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I might need cataract surgery in the eye being studied during the trial.

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I haven't taken experimental treatments for GA, except for AREDS, in the last 6 months.

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I have undergone gene or cell therapy.

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My eye condition is due to diabetes and is moderate to severe.

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I have had eye surgery or treatment for my retina in the study eye.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 240 weeks

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~240 weeks

This trial's timeline: 3 weeks for screening, Varies for treatment, and 240 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

Incidence of ocular and non-ocular Treatment Emergent AEs (TEAEs) and Treatment-Emergent Serious AEs (TESAEs) Severe Adverse Events (TEAE/TESAE)

Secondary study objectives

Geographic Atrophy

Incidence of device-related AEs and SAEs (US only)

Long-term safety of GT005 at 3 doses

+4 more

Awards & Highlights

No Placebo-Only Group

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

Trial Design

7Treatment groups

Experimental Treatment

Group I: GT005 Dose 3 with Orbit Subretinal Delivery SystemExperimental Treatment2 Interventions

A single dose of GT005 will be administered with subretinal injection via suprachoroidal cannulation approach

Group II: GT005 Dose 3 with Orbit Subretinal Delivery SysemExperimental Treatment1 Intervention

A single dose of GT005 will be administered with subretinal injection via suprachoroidal cannulation approach

Group III: GT005 Dose 3Experimental Treatment1 Intervention

A single dose of GT005 will be administered via subretinal injection

Group IV: GT005 Dose 2 with Orbit Subretinal Delivery SystemExperimental Treatment2 Interventions

A single dose of GT005 will be administered with subretinal injection via suprachoroidal cannulation approach

Group V: GT005 Dose 2Experimental Treatment1 Intervention

A single dose of GT005 will be administered via subretinal injection

Group VI: GT005 Dose 1, 2 or 3Experimental Treatment1 Intervention

A single dose of GT005 will be administered via subretinal injection. This dose will be determined by dose levels determined to be tolerable in Arms 1,2 and 3

Group VII: GT005 Dose 1Experimental Treatment1 Intervention

A single dose of GT005 will be administered via subretinal injection

0 / 11Eligibility criteria met

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

Common treatments for Age-Related Macular Degeneration (AMD) include anti-VEGF (vascular endothelial growth factor) injections, which reduce abnormal blood vessel growth and leakage in the retina, and photodynamic therapy, which uses light-activated drugs to destroy abnormal blood vessels. Gene therapy, such as the GT005 trial, aims to modify or correct genetic factors contributing to Macular Atrophy in AMD by delivering therapeutic genes to retinal cells. This approach can potentially provide a long-term solution by addressing the underlying genetic causes of the disease, offering hope for more sustained and effective treatment outcomes for AMD patients.