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Virus Therapy

Gene Therapy for Retinitis Pigmentosa

Phase 1 & 2
Recruiting
Research Sponsored by Bionic Sight LLC
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Confirmed diagnosis of retinitis pigmentosa
Be older than 18 years old
Must not have
Prior receipt of any AAV gene therapy product
Large amplitude nystagmus
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 12 months
Awards & highlights
No Placebo-Only Group

Summary

This trial tests a modified virus that carries a light-sensitive gene to help people with vision problems. The virus delivers this gene to eye cells, making them respond to light and potentially improving vision.

Who is the study for?
This trial is for individuals with confirmed retinitis pigmentosa who have very limited vision, described as 'bare light perception', in at least one eye. It's not open to those who've previously received any AAV gene therapy or have large amplitude nystagmus, which is a condition where the eyes make repetitive, uncontrolled movements.
What is being tested?
The study involves BS01, a genetic treatment using a modified virus to deliver genes into cells of the eye. This early-phase trial will test different doses to see how safe it is and what effects it has on patients' vision.
What are the potential side effects?
As this is an early-stage trial for BS01, specific side effects are being studied; however, potential risks may include immune reactions to the viral vector or inflammation in the eye.

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 retinitis pigmentosa.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:
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I have never received AAV gene therapy before.
Select...
I experience severe involuntary eye movements.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~12 months
This trial's timeline: 3 weeks for screening, Varies for treatment, and 12 months for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
Measure
Secondary study objectives
Measures

Awards & Highlights

No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.

Trial Design

4Treatment groups
Experimental Treatment
Group I: Cohort 4Experimental Treatment1 Intervention
BS01 Cohort4 dose
Group II: Cohort 3Experimental Treatment1 Intervention
BS01 Cohort 3 dose
Group III: Cohort 2Experimental Treatment1 Intervention
BS01 Cohort 2 dose
Group IV: Cohort 1Experimental Treatment1 Intervention
BSO1 Cohort 1 dose

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 Retinitis Pigmentosa (RP) often involves using adeno-associated virus (AAV) vectors to deliver functional genes to retinal cells. In the BS01 trial, the AAV vector delivers the ChronosFP gene, which encodes a light-sensitive channelrhodopsin, to retinal cells. This approach aims to restore light sensitivity and improve visual function by compensating for the defective or missing genes responsible for RP. This matters for RP patients because it offers a potential to halt or reverse the progression of vision loss, providing a more targeted and long-lasting treatment compared to traditional methods. Other common treatments, such as vitamin A supplementation and retinal implants, aim to slow disease progression or replace lost function but do not address the underlying genetic causes as directly as gene therapy.
Gene therapy for Leber congenital amaurosis: advances and future directions.AAV-mediated gene therapy for retinal degeneration in the rd10 mouse containing a recessive PDEbeta mutation.

Find a Location

Who is running the clinical trial?

Bionic Sight LLCLead Sponsor
Sheila Nirenberg, PhDStudy DirectorBionic Sight LLC

Media Library

BS01 (Virus Therapy) Clinical Trial Eligibility Overview. Trial Name: NCT04278131 — Phase 1 & 2
Retinitis Pigmentosa Research Study Groups: Cohort 2, Cohort 1, Cohort 3, Cohort 4
Retinitis Pigmentosa Clinical Trial 2023: BS01 Highlights & Side Effects. Trial Name: NCT04278131 — Phase 1 & 2
BS01 (Virus Therapy) 2023 Treatment Timeline for Medical Study. Trial Name: NCT04278131 — Phase 1 & 2
Retinitis Pigmentosa Patient Testimony for trial: Trial Name: NCT04278131 — Phase 1 & 2
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