What side effects are associated with this type of intervention?

Common treatments for Retinal Dystrophy, such as gene therapy and intravitreal injections, can have several side effects. Gene therapy targeting specific mutations like PRPF31 may cause immune reactions, inflammation, and potential retinal damage. Intravitreal injections, often used to deliver therapeutic agents, can lead to side effects such as eye pain, increased intraocular pressure, cataracts, and retinal detachment. It is crucial for patients to discuss these potential risks with their healthcare provider to make an informed decision.

What prior FDA approvals exist?

The FDA has approved several gene therapy treatments for retinal dystrophies. One notable example is Luxturna (voretigene neparvovec-rzyl), which targets RPE65 mutation-associated retinal dystrophy. Luxturna was the first in vivo gene therapy approved by the FDA for a genetic disease, providing a precedent for treatments like VP-001. While specific FDA approvals for PRPF31 mutation-associated retinal dystrophy are not mentioned, the approval of Luxturna highlights the potential for gene therapy in treating various forms of retinal dystrophy.

What other types of research exist?

Promising novel alternatives to VP-001 for Retinal Dystrophy patients include: 1) CRISPR-Cas9 based gene editing therapies, which are being explored for precise genetic corrections. 2) RNA interference (RNAi) therapies, such as those targeting specific gene expressions involved in retinal dystrophy. 3) Stem cell-based therapies, which aim to replace damaged retinal cells with healthy ones. 4) Other gene therapies targeting different retinal dystrophy-related genes, such as RPE65 or CEP290, which have shown efficacy in clinical trials.

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Virus Therapy

VP-001 for Retinal Dystrophy (Platypus Trial)

Phase 1

Recruiting

Research Sponsored by PYC Therapeutics

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 up to 5 years

Awards & highlights

No Placebo-Only Group

Summary

This trial tests a new eye injection treatment called VP-001 for people with a genetic condition that affects their vision. The goal is to see if it is safe and can help the damaged cells in their eyes.

Who is the study for?

Adults with a genetic diagnosis of PRPF31 mutation-associated retinal dystrophy (RP11) can join this trial. They must be willing to follow the study plan and attend all visits, use effective birth control if they can have children, and not be pregnant or breastfeeding. People with uncontrolled diseases, recent certain eye treatments or surgeries, other gene therapy for retinal conditions, or excessive drug/alcohol use cannot participate.

What is being tested?

The trial is testing VP-001 given as an injection into the eye to see if it's safe for people with RP11-related vision loss. It's an early-phase study where everyone gets the treatment but at different doses to find out which one is safest.

What are the potential side effects?

Since this is a Phase 1 trial primarily focused on safety and tolerability, specific side effects are being investigated but may include typical reactions related to eye injections such as discomfort, redness, swelling around the eyes and potential visual disturbances.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to 5 years

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to 5 years

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 5 years for reporting.

Treatment Details

Awards & Highlights

No Placebo-Only Group

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

Trial Design

1Treatment groups

Experimental Treatment

Group I: Single arm dose escalation study of VP-001Experimental Treatment1 Intervention

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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

Gene therapy for retinal dystrophy, such as the treatment being studied in the VP-001 trial targeting the PRPF31 mutation, works by delivering a functional copy of the defective gene directly into the retinal cells. This approach aims to restore the normal function of the retinal pigment epithelium (RPE) and photoreceptors, thereby slowing or halting the progression of vision loss. This is crucial for patients as it addresses the root cause of the disease at the genetic level, potentially offering a long-term solution and preserving vision. Other treatments, like subthreshold laser therapy, focus on modulating cellular responses to reduce edema and inflammation, but do not correct the underlying genetic defect. Gene therapy's ability to target the genetic basis of retinal dystrophy represents a significant advancement in treatment, offering hope for more effective and lasting outcomes.

Real-life outcomes of subthreshold laser therapy for diabetic macular edema.Functional Rescue of Retinal Degeneration-Associated Mutant RPE65 Proteins.Gene therapy for leber congenital amaurosis caused by RPE65 mutations: safety and efficacy in 15 children and adults followed up to 3 years.