This page shows what was measured, who it was measured in, and what that does not settle.
What Voretigene neparvovec does in the body
An inherited form of childhood blindness caused by two faulty RPE65 genes
The layer of cells behind your retina runs a chemical recycling plant that resets the molecule your eye uses to detect light. Both your copies of the gene for the key enzyme are broken, so the plant has stopped. A surgeon lifts the retina slightly and injects a fluid containing a harmless virus shell carrying a working copy of that gene into the cells underneath. Those cells start making the enzyme again, the recycling restarts, and the photoreceptors that are still alive can respond to much dimmer light than before.
What happened in people
Bilateral MLMT change at 1 year: 1.8 light levels treated versus 0.2 control, difference 1.6 (95% CI 0.72 to 2.41; P=0.0013)
✎ Source-linked record
Where this came from
A person wrote this into the record with the study named beside it. No reviewer has signed it off.
The recorded finding that sits closest to something a person would notice. Written into the record, not signed off, and it carries no population or interval.
Safety and Efficacy Study in Subjects With Leber Congenital Amaurosis (Study 302) (NCT00999609) · a recorded source, not a stored snapshot
The limit that matters most
That vision was restored — visual acuity did not differ significantly from control at one year, per the FDA label
Where it acts
Retinal pigment epithelium, reached by subretinal injection
Kind of result
A number that stands in for health
Supervision
Professional supervision is normally required. It is a prescription or clinician-administered medicine where it is approved.
What the registries record it as
The substance registry classes this as structurallydiverse.
FDA substance registry · 2SPI046IKD · read 2026-08-29
The material is recorded as coming from virus.
FDA substance registry · 2SPI046IKD · read 2026-08-29
Where each sentence above came from
No recorded sentence describes the change this makes in a way that stands on its own. The page opens with what it is taken for instead, and the recorded explanation follows below.
Shown as the opening line on this page.
A limit recorded against this substance. Not signed off as a reviewed claim.
The four opening statements run to 132 words.
Words this page uses
Four words worth knowing first
Chosen from what this page shows, with each one explained before the word it depends on.
Comparator
A comparator is whatever the treatment was measured against.
A picture of it, and where the picture fails
It is like the other runner in a race.
Where that stops being true. A race has one winner. A study can show both arms improved.
What people get wrong. Results are read without asking what the other group got. Beating nothing is not beating a treatment.
The control condition against which the experimental intervention is assessed.
Randomisation
Randomisation means chance decides who gets which treatment.
A picture of it, and where the picture fails
It is like a coin toss deciding the groups.
Where that stops being true. A coin toss is fair once. Fairness here comes from doing it for everyone.
What people get wrong. It is thought to make groups identical. It makes them similar on average, including on things nobody measured.
Allocation of participants to arms by a chance process, so that unmeasured factors are balanced in expectation.
Absolute difference
An absolute difference is how many more people in a hundred were affected.
A picture of it, and where the picture fails
It is like counting heads in two rooms of a hundred.
Where that stops being true. Heads are easy to count. Study results carry a margin of error too.
What people get wrong. It is confused with a percentage change, which can look far larger.
The arithmetic difference in event rates between arms.
Confidence interval
A confidence interval is the range the true answer is likely to sit in.
A picture of it, and where the picture fails
It is like a weather forecast giving a range rather than one number.
Where that stops being true. A forecast range covers tomorrow. This one covers what the study could resolve.
What people get wrong. The middle number is read as the answer. A range crossing zero means no change is still possible.
An interval estimate that would contain the true parameter in a stated proportion of repeated studies.
What happened in people◇Read from sources, not yet reviewed
What happened in people
Each card is one study: the exact question it asked, who was in it, and whether it showed what it set out to show.
One-year change in bilateral multi-luminance mobility test performance
✓ The study showed what it set out to show
Who was studied
Study 302 (NCT00999609)
How many people
31
Study design
Phase 3, randomised, controlled, open label with masked grading
Compared against
Not recorded for this study
Kind of result
A number that stands in for health
What was found
P = 0.0013 (difference 1.6 light levels, 95% CI 0.72 to 2.41)
Repeated elsewhere
Partially Replicated
What this does not prove. Meeting one endpoint in one population does not carry to other goals or other people.
The limits of this study, and where it came from
Main limit. No product-related serious adverse events or deleterious immune responses in the trial. The chorioretinal atrophy signal emerged later, in post-authorisation registry data.
How far it carries. This study is recorded from the curated record, not from the registry match.
Form and route. Recombinant AAV2 vector, subretinal injection, one eye per procedure
Interval reported. 95% CI 0
Written into the record, not signed off as a reviewed claim.
Safety and Efficacy Study in Subjects With Leber Congenital Amaurosis (Study 302) (NCT00999609) · a recorded source, not a stored snapshot
FDA LUXTURNA product page and package insert (https://www.fda.gov/vaccines-blood-biologics/cellular-gene-therapy-products/luxturna) · a recorded source, not a stored snapshot
Real-world safety and effectiveness up to 2 years
✓ The study showed what it set out to show
Who was studied
PERCEIVE post-authorisation registry
How many people
103
Study design
Prospective, multicentre, registry-based observational study
Compared against
Not recorded for this study
Kind of result
A number that stands in for health
What was found
Descriptive; no hypothesis test — an observational registry with no control arm
Repeated elsewhere
Partially Replicated
What this does not prove. Meeting one endpoint in one population does not carry to other goals or other people.
The limits of this study, and where it came from
Main limit. Ocular treatment-emergent adverse events in 34% of patients, chorioretinal atrophy in 12.6%, procedure-related intraocular inflammation or infection in 7.
How far it carries. This study is recorded from the curated record, not from the registry match.
Form and route. Recombinant AAV2 vector, subretinal injection, one eye per procedure
Interval reported. Not recorded in this summary
Written into the record, not signed off as a reviewed claim.
Safety and Efficacy Study in Subjects With Leber Congenital Amaurosis (Study 302) (NCT00999609) · a recorded source, not a stored snapshot
FDA LUXTURNA product page and package insert (https://www.fda.gov/vaccines-blood-biologics/cellular-gene-therapy-products/luxturna) · a recorded source, not a stored snapshot
What we know
RNAWiki holds 2 written-up human studies for this substance, each with the question it asked.
How we know it
Each card names the study, the number of people and what the study set out to measure.
What this does not prove
These summaries were written by a person and have not been signed off as reviewed claims.
Why it matters
A study that failed is as informative as one that worked, and both are here.
What we still do not know
No reviewed claim exists, so no exact population, effect size or interval is published yet.
What it changes in the body◇Read from sources, not yet reviewed
The path through the body
From what a person takes to what changes. A solid line is a step measured in people. A dashed line is a step nobody has measured that way.
Start
Voretigene neparvovec
What a person takes: Recombinant AAV2 vector, subretinal injection, one eye per procedure.
The measurement behind this step
A single subretinal injection of 1.5 x 10^11 vector genomes in 0.3 mL per eye, delivered by vitrectomy and subretinal bleb, with the second eye treated in a separate procedure. Systemic corticosteroid brackets each surgery. Air travel and scuba diving are not recommended until any intraocular air bubble has been absorbed.
Getting in
Surgery lifts the retina and places the dose underneath it
The gel inside the eye is removed, a tiny blister of fluid is raised under the retina away from the centre of vision, and the treatment is injected into it. Each eye is done separately.
╌╌Described, with no measurement named. No measurement is named for this step, so it is drawn as an unconfirmed link.
The measurement behind this step
Pars plana vitrectomy followed by subretinal injection of 1.5 x 10^11 vector genomes in 0.3 mL, deliberately away from the fovea because injection in its immediate vicinity risks macular abnormalities. The second eye follows in a separate procedure.
Safety and Efficacy Study in Subjects With Leber Congenital Amaurosis (Study 302) (NCT00999609) · a recorded source, not a stored snapshot
Reaching the cell
AAV2 enters the retinal pigment epithelium
The virus shell is taken up by the layer of cells directly behind the retina — the exact cells that run the recycling plant.
╌╌Described, with no measurement named. No measurement is named for this step, so it is drawn as an unconfirmed link.
The measurement behind this step
AAV2 binds heparan sulphate proteoglycan and co-receptors on retinal pigment epithelial cells and is endocytosed. The subretinal space places the vector in direct contact with the target layer, which is why 1.5 x 10^11 vg suffices where a systemic AAV dose runs to 10^14.
Safety and Efficacy Study in Subjects With Leber Congenital Amaurosis (Study 302) (NCT00999609) · a recorded source, not a stored snapshot
What it acts on
The gene stays as a loop in the nucleus and is read continuously
The delivered gene does not join the cell's chromosomes. It sits alongside them and is read, and because these cells barely divide, it can stay for years.
╌╌Described, with no measurement named. No measurement is named for this step, so it is drawn as an unconfirmed link.
The measurement behind this step
The RPE65 cassette persists as a non-integrating episome under a hybrid chicken beta-actin promoter. Retinal pigment epithelial cells are essentially post-mitotic, so episomal dilution is minimal — the structural reason ocular AAV durability outperforms hepatic AAV durability.
Safety and Efficacy Study in Subjects With Leber Congenital Amaurosis (Study 302) (NCT00999609) · a recorded source, not a stored snapshot
The change it makes
The visual cycle restarts
The enzyme bends the light-detecting molecule back into its usable shape, so photoreceptors can be reloaded and fire again.
╌╌Described, with no measurement named. No measurement is named for this step, so it is drawn as an unconfirmed link.
The measurement behind this step
RPE65 isomerohydrolase converts all-trans-retinyl ester to 11-cis-retinol, which is oxidised to 11-cis-retinal and returned to photoreceptors to regenerate rhodopsin and cone opsins. Without it the chromophore supply collapses, which is why night vision fails first.
Safety and Efficacy Study in Subjects With Leber Congenital Amaurosis (Study 302) (NCT00999609) · a recorded source, not a stored snapshot
What that does for a person
Navigation in dimmer light — but not a better eye chart
Patients could walk a course at light levels they could not manage before, some of them down to near-darkness. Reading letters on a chart did not improve significantly.
──Measured in people. Written by a person from the study named beside the step. Not signed off as a reviewed claim.
The measurement behind this step
Bilateral MLMT change at 1 year was 1.8 light levels versus 0.2 for control, difference 1.6 (95% CI 0.72 to 2.41; P=0.0013), with 13 of 20 treated participants passing at 1 lux. Full-field light sensitivity threshold improved significantly; visual acuity did not differ significantly between groups.
Safety and Efficacy Study in Subjects With Leber Congenital Amaurosis (Study 302) (NCT00999609) · a recorded source, not a stored snapshot
No suggested links are held for this record, so nothing is hidden from this path.
What we know
The record describes 5 steps from taking it to an effect in a person.
How we know it
Each step names the study behind it in the technical detail beside it.
What this does not prove
A change inside the body is a reason to look. It is not a result in a person.
Why it matters
A reader can see exactly where the chain stops being measured in people.
What we still do not know
No reviewed claim binds any of these steps to a named population.
What is missing or unclear◇Read from sources, not yet reviewed
Were people like you studied?
RNAWiki cannot tell whether a study fits you. It can show who was in it, and where the result stops carrying.
Who was studied
People similar to this profile were included.
Adults and children with genetically confirmed biallelic RPE65 mutations, provided enough viable retina remains for the injected cells to be worth rescuing.
Who is missing from the studies
Which groups were under-represented in the studies has not been recorded for this substance. Pregnancy, breastfeeding, children, older adults, and people with liver or kidney conditions are commonly excluded from trials; nothing here says whether they were.
What the label states about particular groups
On pediatric, the label states: “Use of LUXTURNA is supported by Study 1 and Study 2 [ see Clinical Studies ( 14 ) ] that included 25 pediatric patients with biallelic RPE65 mutation-associated retinal dystrophy in the following age groups: 21 children (age 4 years to less than 12 years) and 4 adolescents (age 12 years to less than 17 years).”
US prescribing information · 08313a24-e7ce-457a-bb3f-161bc45517ee · read 2026-08-30
On older people, the label states: “The safety and effectiveness of LUXTURNA have not been established in geriatric patients.”
US prescribing information · 08313a24-e7ce-457a-bb3f-161bc45517ee · read 2026-08-30
On people who are pregnant, the label states: “Risk Summary Adequate and well-controlled studies with LUXTURNA have not been conducted in pregnant women.”
US prescribing information · 08313a24-e7ce-457a-bb3f-161bc45517ee · read 2026-08-30
On people who are breastfeeding, the label states: “Risk Summary There is no information regarding the presence of LUXTURNA in human milk, the effects on the breastfed infant, or the effects on milk production.”
US prescribing information · 08313a24-e7ce-457a-bb3f-161bc45517ee · read 2026-08-30
Where the result stopped carrying
Visual acuity, the pre-existing standard measure, showed no significant between-group difference at one year
Chorioretinal atrophy after treatment became the most frequent real-world ocular adverse event and a labelled risk
This is a scope explorer, not a diagnosis engine.
It shows who was studied so you can see whether people similar to you were included.
RNAWiki cannot tell whether a study fits you. It can show who was in it.
What it would be like to take◇Read from sources, not yet reviewed
Why it might seem to do nothing
A real effect and a noticed effect are different things. These are the recorded reasons the two come apart.
It was studied in different people
The people in the studies were not much like the reader.
On this record: Which groups were under-represented in the studies has not been recorded for this substance. Pregnancy, breastfeeding, children, older adults, and people with liver or kidney conditions are commonly excluded from trials; nothing here says whether they were.
The evidence may simply be wrong
Small early studies often do not hold up.
On this record: RNAWiki has not yet published a reviewed conclusion for this use.
Other reasons RNAWiki checked and found nothing for (11)
It was studied for a different goal
The studies measured something else entirely. Nothing in this record points to this reason.
A different form was studied
The studied form is not the form on the shelf. Nothing in this record points to this reason.
There was nothing to correct
Where a level is already normal, topping it up may change nothing. Nothing in this record points to this reason.
Something else had to happen too
In the studies it was paired with training, a diet or another treatment. Nothing in this record points to this reason.
The change is too small to feel
A real change can still sit below what a person notices. Nothing in this record points to this reason.
Day-to-day swing hides it
Sleep, food, stress and time of day move most numbers more than this would. Nothing in this record points to this reason.
Not enough time yet
The studies ran longer than the person has waited. Nothing in this record points to this reason.
It was not taken as studied
Missed days change the result, and studies count them. Nothing in this record points to this reason.
Something else changed at the same time
Two changes at once cannot be told apart afterwards. Nothing in this record points to this reason.
The product may not be what it says
Contents of a sold product are not always what the label states. Nothing in this record points to this reason.
No recorded reason
RNAWiki has nothing stored that would explain it. Nothing in this record points to this reason.
None of these is a reason to take more. Taking more is not a step this page ever suggests.
What it would be like to take◇Read from sources, not yet reviewed
What taking it involves
The recorded facts about getting hold of it and using it. Nothing here is a suggestion about what you should do.
How it is supplied
Given by a clinician
❝ Quoted from a source
Where this came from
Copied from a source RNAWiki stored, with the source named.
Read from the recorded approval status.
No source is stored against this line.
Form and route
Recombinant AAV2 vector, subretinal injection, one eye per procedure
✎ Source-linked record
Where this came from
A person wrote this into the record with the study named beside it. No reviewer has signed it off.
The form and route recorded on this substance.
No source is stored against this line.
Who oversees it
Professional supervision is normally required. It is a prescription or clinician-administered medicine where it is approved.
❝ Quoted from a source
Where this came from
Copied from a source RNAWiki stored, with the source named.
The identity record classes it as CRISPR / Gene Therapy.
No source is stored against this line.
What is in the pack
3 mL per eye, delivered by vitrectomy and subretinal bleb, with the second eye treated in a separate procedure. Systemic corticosteroid brackets each surgery. Air travel and scuba diving are not recommended until any intraocular air bubble has been absorbed.
✎ Source-linked record
Where this came from
A person wrote this into the record with the study named beside it. No reviewer has signed it off.
Recorded notes on how this is sold and what that means for what is in the pack.
No source is stored against this line.
Where it is registered
Regulatory records are listed in the technical disclosure at the foot of this page.
# Counted from records
Where this came from
A count of rows RNAWiki holds. It describes our records, not your body.
Register entries are stored per jurisdiction and shown with their dates.
No source is stored against this line.
Why people stop
Not recorded.
∅ Nothing recorded
Where this came from
RNAWiki holds nothing here and says so rather than guessing.
No record of why people stopped taking it is stored for this substance.
No source is stored against this line.
What it would be like to take◇Read from sources, not yet reviewed
What can go wrong
Sorted by where each line came from. A regulator's label and a report somebody sent in are not the same kind of fact.
·Worth asking a clinician aboutWritten into the record from the studies named on this page
No boxed warning. Labelled warnings are endophthalmitis, permanent decline in visual acuity, retinal abnormalities including macular changes, retinal tears and chorioretinal atrophy, increased intraocular pressure, expansion of intraocular air bubbles, and cataract formation or accelerated progression. Injection in the immediate vicinity of the fovea is specifically to be avoided.
Nobody counted how many people took this and were fine, so this cannot be turned into a rate.
Safety and Efficacy Study in Subjects With Leber Congenital Amaurosis (Study 302) (NCT00999609) · a recorded source, not a stored snapshot
Over a long time. No completed tested study window is recorded, so long-term effects are not established by the registry record.
Groups the studies covered thinly. Which groups were under-represented in the studies has not been recorded for this substance. Pregnancy, breastfeeding, children, older adults, and people with liver or kidney conditions are commonly excluded from trials; nothing here says whether they were.
What is missing or unclear∅Nothing found in the sources checked
Does the exact form matter?
Evidence belongs to the exact thing that was tested. A different salt, a different mixture or a different preparation is a different question.
The form that was studied
Recombinant AAV2 vector, subretinal injection, one eye per procedure
✎ Source-linked record
Where this came from
A person wrote this into the record with the study named beside it. No reviewer has signed it off.
The form used in the studies cited on this page.
No source is stored against this line.
What is sold
etinal bleb, with the second eye treated in a separate procedure. Systemic corticosteroid brackets each surgery. Air travel and scuba diving are not recommended until any intraocular air bubble has been absorbed.
✎ Source-linked record
Where this came from
A person wrote this into the record with the study named beside it. No reviewer has signed it off.
Recorded notes on which forms are sold and how they compare.
No source is stored against this line.
Evidence carries across two names for one substance. It does not carry across a salt, a mirror form or a mixture.
What it would be like to take◇Read from sources, not yet reviewed
What you could measure
This one is decided with a clinician, so this section holds questions to ask rather than a plan to run.
Self-experiment planning is not offered for a prescription or clinician-supervised medicine. The questions below are for a clinician or pharmacist.
Questions worth asking
Which of the trials of Voretigene neparvovec studied people like me?
What was measured, and for how long?
Was the result a laboratory value or a health outcome?
What would we watch for, and when would we stop?
Tracking can show whether something changed for you. It cannot show what caused it.
RNAWiki records evidence. It does not say whether this substance is right for you.
What is missing or unclear◇Read from sources, not yet reviewed
Claims that go past the evidence
Things said about this substance that sound reasonable, and the exact step that is missing between the evidence and the claim.
✗Goes past the evidence
That vision was restored — visual acuity did not differ significantly from control at one year, per the FDA label
Why it sounds right. It follows from something real on this page: a body step, an animal result or a related finding.
The missing step. Nobody measured this in people for this use, or the study that did measure it did not show it.
What would change this
A study in people, for this exact use, measuring this exact thing, that a reviewer signs off.
RNAWiki has not yet published a reviewed conclusion for this use.
✗Goes past the evidence
That the degeneration is halted; the therapy replaces a metabolic enzyme and the indication itself requires viable retina remaining
Why it sounds right. It follows from something real on this page: a body step, an animal result or a related finding.
The missing step. Nobody measured this in people for this use, or the study that did measure it did not show it.
What would change this
A study in people, for this exact use, measuring this exact thing, that a reviewer signs off.
RNAWiki has not yet published a reviewed conclusion for this use.
✗Goes past the evidence
That the mobility gain is comparable to any other product's endpoint, when the endpoint was created for this trial and is licensed to its sponsor
Why it sounds right. It follows from something real on this page: a body step, an animal result or a related finding.
The missing step. Nobody measured this in people for this use, or the study that did measure it did not show it.
What would change this
A study in people, for this exact use, measuring this exact thing, that a reviewer signs off.
RNAWiki has not yet published a reviewed conclusion for this use.
✗Goes past the evidence
That two-year registry light-sensitivity numbers establish durability, when they come from 13 eyes
Why it sounds right. It follows from something real on this page: a body step, an animal result or a related finding.
The missing step. Nobody measured this in people for this use, or the study that did measure it did not show it.
What would change this
A study in people, for this exact use, measuring this exact thing, that a reviewer signs off.
RNAWiki has not yet published a reviewed conclusion for this use.
What is missing or unclear◇Read from sources, not yet reviewed
What nobody knows yet
Open questions, each with why it is open and what would close it.
How much did people take in the studies?
The sources RNAWiki checked hold nothing for this field.
Why it matters. A result belongs to an amount. Without the amount the result floats free.
What would answer it
A stored source that records it, and a reviewer to sign it off.
Last checked 2026-09-04. Searched: The sources listed in the receipts at the foot of this page.
Missing populations
Which groups were under-represented in the studies has not been recorded.
Why it matters. Recorded by the evidence model as a gap on this record.
What would answer it
A study that measures it, and a reviewer to sign it off.
Last checked 2026-09-04. Searched: The sources listed in the receipts at the foot of this page.
Missing long-term data
No completed tested study window is recorded.
Why it matters. Recorded by the evidence model as a gap on this record.
What would answer it
A study that measures it, and a reviewer to sign it off.
Last checked 2026-09-04. Searched: The sources listed in the receipts at the foot of this page.
No reviewed conclusion
RNAWiki has not yet published a reviewed conclusion for this use.
Why it matters. Recorded by the evidence model as a gap on this record.
What would answer it
A study that measures it, and a reviewer to sign it off.
Last checked 2026-09-04. Searched: The sources listed in the receipts at the foot of this page.
Missing interaction studies
No interaction was found in the registers checked. Not finding one is not the same as showing there is none.
Why it matters. Recorded by the evidence model as a gap on this record.
What would answer it
A study that measures it, and a reviewer to sign it off.
Last checked 2026-09-04. Searched: The sources listed in the receipts at the foot of this page.
Mechanism not reviewed
No reviewed mechanism story exists for this substance.
Why it matters. Recorded by the evidence model as a gap on this record.
What would answer it
A study that measures it, and a reviewer to sign it off.
Last checked 2026-09-04. Searched: The sources listed in the receipts at the foot of this page.
What happened in people◇Read from sources, not yet reviewed
Check any of this yourself
Every line above traced back to the study it came from. This is the technical layer, and the vocabulary changes here.
Every line above can be traced to the study named beside it. Follow the link and read it.
A randomised, controlled trial — the rarest thing in this modality
In plain words
Thirty-one people were randomly assigned, two to one, to treatment or no treatment, and independent graders who did not know which was which scored the results. Almost no gene therapy has this design.
What was measured
Randomised 2:1, masked independent grading of the primary endpoint
Effect estimate
No reviewed effect estimate exists for this record.
Limits
See the technical detail.
From the source
Open-label, randomised, controlled phase 3 trial at two US sites. 31 individuals aged 3 or older with biallelic RPE65 mutations, best-corrected visual acuity 20/60 or worse or visual field under 20 degrees, randomised 2:1 to bilateral subretinal voretigene neparvovec (n=21) or control (n=10), stratified by age and baseline mobility level. One participant withdrew from each group, leaving a modified intention-to-treat population of 20 and 9. Graders assessing the primary outcome were masked to treatment group.
Written into the record, not signed off as a reviewed claim
A 1.6 light-level advantage on the mobility test at one year
In plain words
Treated participants could complete a navigation course at much dimmer light than before. Thirteen of twenty passed at the dimmest level tested, roughly a moonless night, where none of the control group did.
What was measured
Bilateral MLMT change at 1 year: 1.8 versus 0.2 light levels, difference 1.6
Effect estimate
No reviewed effect estimate exists for this record.
Limits
See the technical detail.
From the source
Mean bilateral MLMT change at 1 year was 1.8 light levels (SD 1.1) with treatment versus 0.2 (SD 1.0) with control, a difference of 1.6 (95% CI 0.72 to 2.41; P=0.0013). 13 of 20 treated but no control participants passed at 1 lux, the lowest level tested. The FDA label reports the same study by median: MLMT score change 2 (min 0, max 4) treated versus 0 (min -1, max 2) control, P=0.001 bilaterally and P=0.003 for the first-treated eye, with 11 of 21 treated versus 1 of 10 control achieving a change of two or more, the threshold the label calls clinically meaningful.
Written into the record, not signed off as a reviewed claim
Visual acuity — the standard measure of sight — did not differ significantly
In plain words
The eye chart did not improve significantly. The measure that did improve was a walking test through an obstacle course, which is a real ability but not the same claim as better vision.
What was measured
That improved navigation in dim light means restored or improved sight in the ordinary sense of the word
Effect estimate
No reviewed effect estimate exists for this record.
Limits
See the technical detail.
From the source
The FDA label states directly: "The change in visual acuity from Baseline to Year 1 was not significantly different between the LUXTURNA and control groups." Full-field light sensitivity threshold testing did improve significantly. The approval therefore rests on functional vision in dim light, not on acuity, and the distinction is load-bearing: a patient can gain two MLMT light levels and read no further down the chart.
Source
LUXTURNA US prescribing information, Section 14 Clinical Studies
Role in the trial
Not matched to a registered study
Identity check
no open identity hold
Audit mark
caution
Review state
Written into the record, not signed off as a reviewed claim
The primary endpoint was invented for this product, and patented
In plain words
The obstacle course used to prove the drug works did not exist before this trial. The sponsor developed it, and the trial's own disclosures record patent applications on it licensed to the sponsor.
What was measured
That a sponsor-developed and sponsor-licensed endpoint carries the same independence as an established clinical measure
Effect estimate
No reviewed effect estimate exists for this record.
Limits
See the technical detail.
From the source
Multi-luminance mobility testing scores a videotaped navigation course at seven illumination levels from 400 lux to 1 lux, with a score of -1 for failure at 400 lux. The Lancet paper's declaration of interests records that several authors, including sponsor employees, hold a patent pending pertaining to the primary endpoint measure licensed to Spark Therapeutics. A sponsor-developed, sponsor-licensed endpoint is not disqualifying — no pre-existing instrument measured what this therapy was meant to change — but it removes the independence a standard endpoint would carry, and it makes cross-product comparison impossible.
Written into the record, not signed off as a reviewed claim
Chorioretinal atrophy is the most common real-world adverse event
In plain words
In a registry of 103 treated patients, one in eight developed patches of atrophy in the treated retina. Retinal thinning after this injection is now a labelled risk.
What was measured
Chorioretinal atrophy in 13 of 103 registry patients (12.6%)
Effect estimate
No reviewed effect estimate exists for this record.
Limits
This entry records a failure.
From the source
PERCEIVE, a prospective post-authorisation registry study, reported outcomes in 103 patients with a mean follow-up of 0.8 years (maximum 2.3). Thirty-five patients (34%) had ocular treatment-emergent adverse events, most frequently related to chorioretinal atrophy in 13 (12.6%). Eighteen patients (17.5%; 24 eyes) had ocular events of special interest, including procedure-related intraocular inflammation or infection in 7. The label separately warns of endophthalmitis, permanent decline in visual acuity, retinal abnormalities including chorioretinal atrophy, raised intraocular pressure, expansion of intraocular air bubbles and cataract.
Written into the record, not signed off as a reviewed claim
The therapy replaces an enzyme; it does not stop the retina degenerating
In plain words
The treatment restarts a chemical process in one layer of cells. The photoreceptors that were dying before the injection go on dying afterwards.
What was measured
That a one-off enzyme replacement halts a degenerative retinal disease rather than improving function in the tissue that survives
Effect estimate
No reviewed effect estimate exists for this record.
Limits
See the technical detail.
From the source
RPE65 encodes the isomerohydrolase of the visual cycle in the retinal pigment epithelium. Restoring it restores retinoid recycling; it does not address the photoreceptor degeneration that the deficiency has already caused, which is why the indication requires viable retinal cells and why earlier treatment is expected to yield more. In PERCEIVE the mean change in full-field light sensitivity threshold with white light was -16.59 dB at month 1 and -13.67 dB at year 2 — a large gain that is smaller at two years than at one month, in a small and shrinking number of eyes. Whether that is regression to a durable plateau or the leading edge of a decline is not resolved by the published follow-up, and long-term studies run to fifteen years.
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What is not here
8 questions this page could not answer
These sections were prepared and left out because there was nothing to put in them. They are listed rather than hidden, so the gaps in this record are visible.
What was measured, goal by goal — found nothing in the sources checked.
How close this is to real life — found nothing in the sources checked.
Felt, measured, or meaningful — found nothing in the sources checked.
How long anything takes — found nothing in the sources checked.
What it may clash with — found nothing in the sources checked.
Other ways to the same goal — found nothing in the sources checked.
How this medicine reached us — found nothing in the sources checked.
What changed on this page — found nothing in the sources checked.
The record as stored
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The older medicine-wide conclusion held in this record
Written for a clinical reader, and about the medicine as a whole rather than about one indication, population and set of trials. RNAWiki does not treat it as a programme conclusion and no reviewer has signed it off in that form. It is here word for word because it is what the record holds.
The first in vivo gene therapy approved in the United States: subretinal AAV2 carrying a working RPE65 gene improved navigation of a dim obstacle course by a median of two light levels versus none in controls, while visual acuity, the standard measure of sight, did not differ significantly from control.
Recorded evidence blocks (6)
Q1
On the Voretigene neparvovec label: indicated for what?
"LUXTURNA (voretigene neparvovec-rzyl) is an adeno-associated virus vector-based gene therapy indicated for the treatment of patients with confirmed biallelic RPE65 mutation-associated retinal dystrophy. Patients must have viable retinal cells as determined by the treating physician(s).": indications and usage on Voretigene neparvovec's label. DailyMed label · 08313a24-e7ce-457a-bb3f-161bc45517ee · 2022-05-01
Q2
6 registered trials of Voretigene neparvovec — at which phases?
Which one trial of Voretigene neparvovec posted no result?
Posted no result
1 of 1 completed trials
Registrations
NCT00516477
Completion dates
oldest 2018-03-20
Show the evidence
TrialNCT00516477
2018-03-20
Q4
At the median, Voretigene neparvovec's trials enrolled 21.5 people — anything larger?
Median enrolment
21.5
Largest enrolment
87
Registered trials counted
6
Q5
What do 20 spontaneous reports say about Voretigene neparvovec — and not say?
These are reports people sent to a regulator. They do not show the medicine caused the reaction. Nobody counted how many people took the medicine and reported nothing. The same event can be reported more than once, and many reports are incomplete. News coverage, lawsuits and new warnings change how often people report. A count is not a rate and not a risk.
Voretigene neparvovec appears in spontaneous reports to regulators. Across the 4 most-reported reaction terms, 20 reaction mentions were counted: intraocular pressure increased 6; retinal tear 6; intraocular pressure decreased 4; vitreous opacities 4. FAERS via Open Targets · CHEMBL3707275 · 2026-06-24
Show the evidence
intraocular pressure increased
6
retinal tear
6
intraocular pressure decreased
4
vitreous opacities
4
recorded 2026-06-24 · last checked 2026-09-04
Q6
Which 4 reactions does Voretigene neparvovec's label not list?
ChEMBL 37 — CC BY-SA 3.0 Unported · ChEMBL ATC CC BY-SA · ClinicalTrials.gov — US Government work · Open Targets 26.06 — CC0 · mixed register set; per-register licences in docs/specs/corpus-20k-sources.md · openFDA / DailyMed — US Government work
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✓ public claims reviewed: 0 reviewed claim(s); drafts are never rendered
✓ source coverage passed: 5 source rows
✓ no critical contamination: no quarantine open
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