What side effects are associated with this type of intervention?

Gene therapies using AAV vectors, such as the one being studied in the BS01 trial for Retinitis Pigmentosa, generally have a favorable safety profile. Common side effects reported in similar treatments include mild procedure-related adverse events such as inflammation or irritation at the injection site. Serious adverse events are rare but can include immune responses to the vector or the transgene, which may lead to inflammation or damage to the retinal cells. Overall, these treatments are well-tolerated, but long-term safety and efficacy studies are still needed.

What prior FDA approvals exist?

As of now, there are no FDA-approved treatments specifically for Retinitis Pigmentosa that use an AAV vector to deliver a gene for light-sensitive channelrhodopsin expression, like the BS01 trial. However, the FDA has approved Luxturna (voretigene neparvovec), an AAV vector-based gene therapy for RPE65-mediated Leber congenital amaurosis, a different genetic eye disease. This approval marks a significant advancement in gene therapy for inherited retinal diseases and provides a foundation for future treatments targeting conditions like Retinitis Pigmentosa.

What other types of research exist?

Promising novel alternatives to BS01 for Retinitis Pigmentosa patients include: 1) CRISPR-Cas9 based gene editing therapies, which aim to correct specific genetic mutations causing RP; 2) Stem cell therapies, such as human embryonic stem cell-derived retinal pigment epithelium (RPE) cell transplantation, which have shown potential in restoring visual function; 3) Pharmacological treatments like neuroprotective agents and small molecules that aim to slow disease progression and preserve retinal cells. These alternatives are in various stages of clinical development and may offer new hope for RP patients in 2024.

← Back to Search

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

Select...

I have been diagnosed with retinitis pigmentosa.

Exclusion Criteria

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

Select...

I have never received AAV gene therapy before.

Select...

I experience severe involuntary eye movements.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 12 months

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

0 / 3Eligibility 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

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.