This page shows what was measured, who it was measured in, and what that does not settle.
What Lovotibeglogene autotemcel does in the body
One deliberate change in the added gene, at position 87, blocks the stacking reaction that makes cells sickle.
Your beta-globin gene has one wrong letter, and rewriting it is hard. So this treatment does not rewrite it — it adds a second, redesigned copy. Your own blood stem cells are collected and a disabled virus carries the new gene into their DNA, where it stays. Chemotherapy clears your marrow, the modified cells go back, and every red cell they make afterwards carries the new haemoglobin alongside the old one.
Why people take it. Used for sickle cell disease with repeated severe pain crises.
What happened in people
All 25 people followed long enough stopped having severe pain crises during the measured period.
✓ Reviewed first-read answer
Where this came from
A person wrote this and a reviewer approved it against this exact record. It carries no effect size.
A reviewer approved this against this record. The reviewed-claim record carrying the exact population and effect size does not exist yet.
No source is stored against this line.
The limit that matters most
The result came from an early, unfinished study with no untreated comparison group.
Where it acts
Bone marrow — autologous CD34+ cells carrying an integrated lentiviral transgene
Kind of result
Symptoms and quality of life
Supervision
Professional supervision is normally required. A regulator classifies this substance in a way that restricts how it is supplied.
What the registries record it as
The substance registry classes this as structurallydiverse.
FDA substance registry · 2C6A9NH2Z8 · read 2026-08-29
It is recorded as coming from HOMO SAPIENS WHOLE. The part recorded is mobilised peripheral blood, from patients with sickle cell disease.
FDA substance registry · 2C6A9NH2Z8 · read 2026-08-29
Where each sentence above came from
A person wrote this explanation into the record, with the studies named in the path below.
A reviewer approved this sentence against this exact record and its sources.
The limit a reviewer approved as the one that matters most here.
The four opening statements run to 113 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.
Biomarker
A biomarker is a number from a test that stands in for something about health.
A picture of it, and where the picture fails
A biomarker is like a fuel gauge.
Where that stops being true. A gauge is wired to the tank. Many biomarkers are only loosely tied to health.
What people get wrong. A better number is read as a better life. Several medicines improved a number and helped nobody.
A measurable indicator used as a substitute for a clinical outcome of interest.
Placebo
A placebo is a dummy treatment given so the real one can be compared with it.
A picture of it, and where the picture fails
A placebo is like a blank control in an experiment.
Where that stops being true. A blank does nothing. People given a placebo often do get better.
What people get wrong. A placebo effect is read as imaginary. The improvement is measured and real.
An inactive intervention matched in appearance to the test intervention, used to control for non-specific effects.
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.
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.
Complete resolution of severe vaso-occlusive events after infusion, in patients with at least four such events in the preceding 24 months
✓ The study showed what it set out to show
Who was studied
HGB-206 Group C (NCT02140554)
How many people
35
Study design
Phase 1-2, single arm, unprespecified interim analysis
Compared against
Not recorded for this study
Kind of result
A number that stands in for health
What was found
Not reported as a p-value — a single-arm within-patient comparison against each patient's own pre-enrolment event rate
Repeated elsewhere
Unreplicated
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 haematologic cancer was observed during up to 37.6 months of follow-up in Group C at the time of this publication. Cases in Group A and a later Group C myelodysplastic syndrome are what produced the boxed warning, and they sit outside this analysis window.
How far it carries. This study is recorded from the curated record, not from the registry match.
Form and route. Ex vivo lentiviral-transduced autologous CD34+ cell suspension, single intravenous infusion
Interval reported. Not recorded in this summary
Written into the record, not signed off as a reviewed claim.
HGB-206: A Study Evaluating the Safety and Efficacy of Lovo-cel in Severe Sickle Cell Disease (NCT02140554) · a recorded source, not a stored snapshot
What we know
RNAWiki holds 1 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
Lovotibeglogene autotemcel
What a person takes: Ex vivo lentiviral-transduced autologous CD34+ cell suspension, single intravenous infusion.
The measurement behind this step
One intravenous infusion of the patient's own CD34+ cells carrying an integrated beta-A-T87Q globin transgene, given after pharmacokinetically dose-adjusted myeloablative busulfan conditioning. Manufacture requires multiple apheresis cycles and transfusion support beforehand.
Getting in
Collect the patient's own blood stem cells
Plerixafor pushes stem cells out of the marrow into the blood, and a machine collects them over several sessions. Patients are transfused first so the collection itself does not trigger a crisis.
╌╌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
Plerixafor-mobilised autologous CD34+ haematopoietic stem cells are collected by apheresis and immunomagnetically selected, after transfusion to a target haemoglobin to suppress sickling during mobilisation.
A disabled virus carries a redesigned gene into the DNA
A virus stripped of everything that lets it replicate delivers a new beta-globin gene, which becomes a permanent part of the cell's own DNA.
╌╌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 BB305 self-inactivating lentiviral vector integrates the beta-A-T87Q globin transgene, with erythroid-specific promoter and locus control region elements, into the host genome at a vector copy number measured at release. Integration is semi-random and permanent.
One designed substitution blocks the stacking reaction
The added gene is not a plain copy. A single deliberate change at position 87 puts a bulky amino acid exactly where sickle haemoglobin molecules would otherwise lock together.
╌╌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 T87Q substitution replaces threonine with glutamine at beta-globin position 87, mimicking the corresponding residue of gamma-globin and sterically interfering with the lateral contact that nucleates HbS polymerisation. The transgene product is therefore not merely additional haemoglobin, it is actively anti-sickling.
Conditioning, engraftment, and a new erythroid output
Chemotherapy empties the marrow so the modified cells can take root, then those cells rebuild the blood system from scratch.
╌╌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
Pharmacokinetically dose-adjusted myeloablative busulfan clears the niche; transduced CD34+ cells home to the marrow and reconstitute haematopoiesis, with the erythroid-specific promoter restricting transgene expression to the red-cell lineage.
The new haemoglobin makes up a large share of the total, in most red cells, and the pain crises stop.
──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
In HGB-206 Group C, HbAT87Q contributed at least 40% of total haemoglobin across a mean 85 ± 8% of red cells, median total haemoglobin reached 11 g/dL or more from month 6 through month 36, haemolysis markers fell, and all 25 evaluable patients had complete resolution of severe vaso-occlusive events.
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.
Patients aged 12 and older with sickle cell disease and a history of vaso-occlusive events, who can tolerate myeloablative conditioning and accept lifelong malignancy surveillance.
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: “The safety and efficacy of LYFGENIA in children less than 12 years of age have not been established.”
US prescribing information · 0d1b475e-5781-2bd1-e063-6294a90a7311 · read 2026-08-30
On older people, the label states: “LYFGENIA has not been studied in patients 65 years of age and older.”
US prescribing information · 0d1b475e-5781-2bd1-e063-6294a90a7311 · read 2026-08-30
On people who are pregnant, the label states: “Risk Summary There are no available data on LYFGENIA administration in pregnant women.”
US prescribing information · 0d1b475e-5781-2bd1-e063-6294a90a7311 · read 2026-08-30
On people who are breastfeeding, the label states: “Risk Summary There is no information regarding the presence of LYFGENIA in human milk, the effect on the breastfed infant, and the effects on milk production.”
US prescribing information · 0d1b475e-5781-2bd1-e063-6294a90a7311 · read 2026-08-30
On people with reduced liver function, the label states: “LYFGENIA has not been studied in patients with advanced hepatic disease.”
US prescribing information · 0d1b475e-5781-2bd1-e063-6294a90a7311 · read 2026-08-30
On people with reduced kidney function, the label states: “LYFGENIA has not been studied in patients with renal impairment (defined as creatinine clearance ≤ 70 mL/min/1.73 m 2 ).”
US prescribing information · 0d1b475e-5781-2bd1-e063-6294a90a7311 · read 2026-08-30
Where the result stopped carrying
Groups A and B of HGB-206 produced insufficient transgene expression and were used to rebuild the manufacturing and transplant process before Group C
Two acute myeloid leukaemias in Group A and one myelodysplastic syndrome in Group C, which together produced the boxed warning
Commercially: a $3.1 million price against a competitor at $2.2 million, and a company taken private at $3.00 per share eighteen months after approval
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
Ex vivo lentiviral-transduced autologous CD34+ cell suspension, single intravenous infusion
✎ 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. A regulator classifies this substance in a way that restricts how it is supplied.
❝ Quoted from a source
Where this came from
Copied from a source RNAWiki stored, with the source named.
Suppression classes recorded: S6, S7.
No source is stored against this line.
What is in the pack
One intravenous infusion of the patient's own CD34+ cells carrying an integrated beta-A-T87Q globin transgene, given after pharmacokinetically dose-adjusted myeloablative busulfan conditioning. Manufacture requires multiple apheresis cycles and transfusion support beforehand.
✎ 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
Not recorded.
∅ Nothing recorded
Where this came from
RNAWiki holds nothing here and says so rather than guessing.
No register entry is recorded.
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
Boxed warning for haematologic malignancy, with mandated complete blood counts at least every six months and integration site analysis at months 6 and 12 and as warranted. Additional labelled risks include delayed platelet engraftment, infusion reactions, and the toxicities of myeloablative busulfan — cytopenias, mucositis, febrile neutropenia and infertility. Anti-retroviral medicines must be stopped before mobilisation and apheresis because they interfere with lentiviral transduction.
Nobody counted how many people took this and were fine, so this cannot be turned into a rate.
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◇Read from sources, not yet reviewed
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
Ex vivo lentiviral-transduced autologous CD34+ cell suspension, single intravenous infusion
✎ 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
Manufacture requires multiple apheresis cycles and transfusion support beforehand.
✎ 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.
What is recorded as being sold
2 products list this as an active ingredient in the United States drug directory. 2 of them contain it and nothing else.
FDA National Drug Code directory · 84146-0001 · read 2026-08-29
They are sold as injection, suspension and suspension, taken intravenous.
FDA National Drug Code directory · 84146-0001 · read 2026-08-29
1 published label names it as an active ingredient. 1 of them describe this substance alone, which is where its own label text on this page comes from.
US prescribing information · 0d1b475e-5781-2bd1-e063-6294a90a7311 · read 2026-08-29
Those labels are classed as cellular therapy.
US prescribing information · 0d1b475e-5781-2bd1-e063-6294a90a7311 · read 2026-08-29
Lyfgenia is intravenous at 3 DOSAGE FORMS AND STRENGTHS LYFGENIA is a cell suspension for intravenous infusion., recorded as fda label in effect 2026-07-21 in the United States.
US prescribing information · 0d1b475e-5781-2bd1-e063-6294a90a7311 · read 2026-08-30
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 Lovotibeglogene autotemcel 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 the leukaemia risk has been excluded — the label's boxed warning and the required lifelong surveillance say the opposite
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 2022 causality verdict on the first leukaemia case generalises, when the sister lentiviral product reported 7 haematologic cancers in 67 patients two years later
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 an unprespecified interim analysis of 25 patients in a phase 1-2 group is equivalent to a randomised pivotal result
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 resolving crises has been shown to prevent stroke, nephropathy or organ failure; none was measured
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.
Formulation uncertainty
Several salts, forms or products of Lovotibeglogene autotemcel are recorded. Results from one form may not transfer to another.
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.
HGB-206 Group C: complete resolution of severe vaso-occlusive events in all 25 evaluable patients
In plain words
Thirty-five people were treated. Of the twenty-five followed long enough to judge, every one stopped having severe pain crises, against a median of 3.5 a year beforehand.
What was measured
Severe vaso-occlusive events after infusion: 0 in 25 of 25 evaluable patients
Effect estimate
No reviewed effect estimate exists for this record.
Limits
See the technical detail.
From the source
Phase 1-2, unprespecified interim analysis. As of February 2021, cell collection had begun in 43 patients in Group C and 35 received an infusion, with median follow-up 17.3 months (range 3.7 to 37.6). Engraftment occurred in all 35. Median total haemoglobin rose from 8.5 g/dL at baseline to 11 g/dL or more from month 6 through month 36. HbAT87Q contributed at least 40% of total haemoglobin and was distributed across a mean 85 ± 8% of red cells. Among 25 evaluable patients, all had resolution of severe vaso-occlusive events versus a median of 3.5 events per year (range 2.0 to 13.5) in the 24 months before enrolment.
Written into the record, not signed off as a reviewed claim
A boxed warning for blood cancer, written out of the product's own development programme
In plain words
Two patients in an early group developed acute myeloid leukaemia and one in the pivotal group developed myelodysplastic syndrome. The label now warns about blood cancer and requires monitoring for the rest of the patient's life.
What was measured
Not recorded
Effect estimate
No reviewed effect estimate exists for this record.
Limits
This entry records a failure.
From the source
The US prescribing information carries a boxed warning: haematologic malignancy has occurred in patients treated with Lyfgenia, and patients must be monitored by complete blood count at least every six months and by integration site analysis at months 6 and 12 and as warranted. At approval, two patients treated in Study 1 Group A — an earlier manufacturing process and transplant procedure — had developed acute myeloid leukaemia, and one patient in Group C with alpha-thalassemia trait had been diagnosed with myelodysplastic syndrome. The mechanism is inherent to the platform: a lentiviral vector integrates semi-randomly and permanently into the genome of a self-renewing stem cell.
Source
LYFGENIA US prescribing information, Boxed Warning and Warnings and Precautions
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 first leukaemia case was investigated and attributed to the disease, not the vector
In plain words
When the first patient developed leukaemia five and a half years after treatment, the obvious suspect was the inserted gene. A detailed investigation concluded the insertion was probably not the cause — and that sickle cell disease plus transplant is itself a cancer risk.
What was measured
Not recorded
Effect estimate
No reviewed effect estimate exists for this record.
Limits
See the technical detail.
From the source
Acute myeloid leukaemia developed in a woman approximately 5.5 years after LentiGlobin for sickle cell disease in the initial Group A cohort of HGB-206. Blast cells did contain a BB305 vector insertion site, but the causality investigation found the leukaemia unlikely to be vector-related given the insertion site's location, very low transgene expression in blasts, and no effect on neighbouring gene expression. Several somatic mutations predisposing to acute myeloid leukaemia were present at diagnosis. The published conclusion was that patients with sickle cell disease carry an elevated baseline risk of haematologic malignancy after transplantation, from the disease, the procedure and inadequate prior disease control combined.
Written into the record, not signed off as a reviewed claim
The reassuring verdict on lentiviral insertion did not survive the sister product
In plain words
Two years later, a different bluebird lentiviral therapy for a brain disease reported seven blood cancers in sixty-seven patients, with the vector sitting in a known leukaemia gene in most of them. That is not the same vector, but it is the same principle.
What was measured
That modern self-inactivating lentiviral vectors have eliminated insertional oncogenesis
Effect estimate
No reviewed effect estimate exists for this record.
Limits
See the technical detail.
From the source
Elivaldogene autotemcel (Skysona), which uses the Lenti-D vector rather than BB305, produced haematologic cancer in 7 of 67 patients across ALD-102, ALD-104 and the LTF-304 follow-up: six myelodysplastic syndromes and one acute myeloid leukaemia, at 14 to 92 months. In six patients with available data, predominant clones carried vector insertions at MECOM-EVI1 in five and PRDM16 in one — both canonical insertional-oncogenesis loci. This does not transfer directly to Lyfgenia, whose vector, promoter and transgene differ, but it removes the option of treating lentiviral insertional oncogenesis as a solved historical problem.
Written into the record, not signed off as a reviewed claim
The pivotal analysis was an unprespecified interim look at a phase 1-2 study
In plain words
The evidence behind this approval is an interim analysis that was not planned in advance, in an early-phase single-arm study, in 25 patients.
What was measured
That an unprespecified interim analysis of a single-arm phase 1-2 group carries the evidential weight of a randomised pivotal trial
Effect estimate
No reviewed effect estimate exists for this record.
Limits
See the technical detail.
From the source
The New England Journal publication describes itself as an unprespecified interim analysis of Group C of the ongoing phase 1-2 HGB-206 study. Group C was created mid-study with a more stringent entry criterion of at least four severe vaso-occlusive events in the preceding 24 months, after Groups A and B were used to optimise the manufacturing and transplant process. There is no control arm; the comparator is each patient's own pre-enrolment event rate. Median follow-up was 17.3 months.
Written into the record, not signed off as a reviewed claim
A $3.1 million price, and a company that no longer trades publicly
In plain words
Lyfgenia launched at $3.1 million, $900,000 above the therapy approved the same week. Eighteen months later the company was taken private for $3.00 a share.
What was measured
Not recorded
Effect estimate
No reviewed effect estimate exists for this record.
Limits
This entry records a failure.
From the source
bluebird bio set a US list price of $3.1 million in December 2023 against Vertex's $2.2 million for Casgevy. In February 2025 bluebird agreed to be acquired by funds managed by Carlyle and SK Capital at $3.00 per share in cash plus a contingent value right of $6.84 per share tied to a net sales milestone; the acquisition completed on 2 June 2025 and the common stock ceased trading. The clinical result and the commercial result of this product point in opposite directions, and only one of them is on the label.
Source
Reuters, 8 December 2023 (list prices); Carlyle press release, 2 June 2025 (completion of acquisition)
Role in the trial
Not matched to a registered study
Identity check
no open identity hold
Audit mark
verified
Review state
Written into the record, not signed off as a reviewed claim
Where else this substance is registered
FDA substance identifier (UNII)
2C6A9NH2Z8
RxNorm concept
2672691
Checks this page had to pass
✓ Passed
Identity resolved
no open identity hold
✓ Passed
No unresolved merge across substance families
no quarantine open
✓ Passed
Every public sentence names a source
The opening statement carries the origin: Written into the record, not signed off.
✗ Not passed
Trial roles classified for highlighted evidence
No registered study is classified as testing this substance.
✓ Passed
No internal keys in reader text
enforced by the copy-contract test over the rendered page
✓ Passed
Safety mode resolved
Suppression classes recorded: S6, S7.
✓ Passed
Canonical metadata present
slug and display name present
What is missing or unclear◇Read from sources, not yet reviewed
How this medicine reached us
Kept near the foot of the page. A historical event moves up only when it changes something about this substance today.
What the approval register records
The earliest marketing start date recorded for a listed product is 20231208.
FDA National Drug Code directory · 84146-0001 · read 2026-08-29
What is missing or unclear◇Read from sources, not yet reviewed
What to learn next
Ordered by what would most change how you read this page, starting with what is easiest to misunderstand.
This order is fixed in code and does not count clicks or time on the page.
What is not here
7 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.
What changed on this page — found nothing in the sources checked.
The record as stored
The full record, for auditing
Everything above is built from what is below. This layer keeps the technical vocabulary, record identifiers and every stored row, so a reader who wants to check the page can.
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.
A lentiviral gene addition that gives red cells a redesigned, sickling-resistant haemoglobin: all 25 evaluable patients in the pivotal group had complete resolution of severe vaso-occlusive events, and the same permanent DNA insertion that makes it work is why the label carries a boxed warning for blood cancer.
Recorded evidence blocks (3)
Q2
On the Lovotibeglogene autotemcel label: indicated for what?
"LYFGENIA is indicated for the treatment of patients 12 years of age or older with sickle cell disease and a history of vaso-occlusive events. LYFGENIA is an autologous hematopoietic stem cell-based gene therapy indicated for the treatment of patients 12 years of age or older with sickle cell disease and a history of…": indications and usage on Lovotibeglogene autotemcel's label. DailyMed label · 0d1b475e-5781-2bd1-e063-6294a90a7311 · 2026-07-21
Q3
2 registered trials of Lovotibeglogene autotemcel — at which phases?
✗ required summary fields resolved: 4 required field(s) not terminal: Why people use it, Best-supported result, Biggest unanswered question, Human evidence
✓ public claims reviewed: 0 reviewed claim(s); drafts are never rendered
✓ source coverage passed: 3 source rows
✓ no critical contamination: no quarantine open
✓ canonical metadata passed: slug and display name present
✓ no raw internal fields: enforced by the copy-contract test over the rendered page
This is a record of evidence. It is not medical advice, and it does not say this substance suits you. Nothing here says any substance on RNAWiki is appropriate for a child.