LYFGENIA™ enables patients to produce a form of anti-sickling adult hemoglobin (HbAT87Q)1
LYFGENIA™ enables patients to produce a form of anti-sickling adult hemoglobin (HbAT87Q)1
A single mutation in the beta-globin gene leads to the production of sickled hemoglobin (HbS) rather than adult hemoglobin (HbA)2
SICKLED RED BLOOD CELL2,3
Mutated
β-globin gene
Sickled
hemoglobin
SICKLED RED BLOOD CELL2,3
A single mutation in the β-globin gene leads to the production of sickled hemoglobin (HbS) rather than adult hemoglobin (HbA).2
Red blood cells that contain high levels of HbS can undergo polymerization, become rigid, and become sickle shaped, leading to vaso-occlusive events, the hallmark of SCD.2,3
Mutated
β-globin gene
Sickled
hemoglobin
How LYFGENIA works to address HbS levels
LYFGENIA adds functional copies of a modified form of the β-globin gene (βA-T87Q-globin gene) through gene addition.1
TRANSDUCTION1
LYFGENIA is manufactured by
transducing autologous CD34+ cells with the BB305 lentiviral vector (LVV), which carries the βA-T87Q-globin gene1
Mutated β-globin gene
BB305 LVV
TRANSDUCTION1
LYFGENIA is manufactured by
transducing autologous CD34+ cells with the BB305 lentiviral vector (LVV), which carries the βA-T87Q-globin gene1
Mutated β-globin gene
BB305 LVV
ENGRAFTMENT1
Following successful engraftment, red
blood cells containing βA-T87Q-globin pairs
with α-globin to produce functional HbA
(HbAT87Q)1
ENGRAFTMENT1
Following successful engraftment, red blood cells containing βA-T87Q-globin pairs with α-globin to produce functional
HbA (HbAT87Q)1
FUNCTIONING RED BLOOD CELL1
HbAT87Q reduces intracellular and total
HbS levels and is designed to sterically
inhibit polymerization of HbS, thereby
limiting the sickling of red blood cells1
βA-T87Q-globin
gene
Functioning
HbA (HbAT87Q)
Mutated
β-globin gene
Sickled
hemoglobin
FUNCTIONING RED BLOOD CELL1
HbAT87Q reduces intracellular and total HbS levels and is designed to sterically inhibit polymerization of HbS, thereby limiting the sickling of red blood cells1
βA-T87Q-globin
gene
Functioning
HbA (HbAT87Q)
Mutated
β-globin gene
Sickled
hemoglobin
TRANSDUCTION1
LYFGENIA is manufactured by
transducing autologous CD34+ cells with the BB305 lentiviral vector (LVV), which carries the βA-T87Q-globin gene1
Mutated β-globin gene
BB305 LVV
TRANSDUCTION1
LYFGENIA is manufactured by
transducing autologous CD34+ cells with the BB305 lentiviral vector (LVV), which carries the βA-T87Q-globin gene1
Mutated β-globin gene
BB305 LVV
ENGRAFTMENT1
Following successful engraftment, red
blood cells containing βA-T87Q-globin pairs
with α-globin to produce functional HbA
(HbAT87Q)1
ENGRAFTMENT1
Following successful engraftment, red blood cells containing βA-T87Q-globin pairs with α-globin to produce functional
HbA (HbAT87Q)1
FUNCTIONING RED BLOOD CELL1
HbAT87Q reduces intracellular and total
HbS levels and is designed to sterically
inhibit polymerization of HbS, thereby
limiting the sickling of red blood cells1
βA-T87Q-globin
gene
Functioning
HbA (HbAT87Q)
Mutated
β-globin gene
Sickled
hemoglobin
FUNCTIONING RED BLOOD CELL1
HbAT87Q reduces intracellular and total HbS levels and is designed to sterically inhibit polymerization of HbS, thereby limiting the sickling of red blood cells1
βA-T87Q-globin
gene
Functioning
HbA (HbAT87Q)
Mutated
β-globin gene
Sickled
hemoglobin
TRANSDUCTION1
LYFGENIA is manufactured by
transducing autologous CD34+ cells with the BB305 lentiviral vector (LVV), which carries the βA-T87Q-globin gene1
Mutated β-globin gene
BB305 LVV
TRANSDUCTION1
LYFGENIA is manufactured by
transducing autologous CD34+ cells with the BB305 lentiviral vector (LVV), which carries the βA-T87Q-globin gene1
Mutated β-globin gene
BB305 LVV
ENGRAFTMENT1
Following successful engraftment, red
blood cells containing βA-T87Q-globin pairs
with α-globin to produce functional HbA
(HbAT87Q)1
ENGRAFTMENT1
Following successful engraftment, red blood cells containing βA-T87Q-globin pairs with α-globin to produce functional
HbA (HbAT87Q)1
FUNCTIONING RED BLOOD CELL1
HbAT87Q reduces intracellular and total
HbS levels and is designed to sterically
inhibit polymerization of HbS, thereby
limiting the sickling of red blood cells1
βA-T87Q-globin
gene
Functioning
HbA (HbAT87Q)
Mutated
β-globin gene
Sickled
hemoglobin
FUNCTIONING RED BLOOD CELL1
HbAT87Q reduces intracellular and total HbS levels and is designed to sterically inhibit polymerization of HbS, thereby limiting the sickling of red blood cells1
βA-T87Q-globin
gene
Functioning
HbA (HbAT87Q)
Mutated
β-globin gene
Sickled
hemoglobin
HbAT87Q is nearly identical to natural HbA1
HbAT87Q has similar oxygen-binding affinity and oxygen hemoglobin dissociation curve to wild-type HbA.
HbAT87Q inhibits polymerization of HbS1
HbAT87Q reduces intracellular and total HbS levels and is designed to sterically inhibit polymerization of HbS, thereby limiting the sickling of red blood cells.
LYFGENIA USES A WELL-STUDIED AND TRACEABLE VECTOR5
LYFGENIA is powered by a third-generation LVV6
Your patients may have important questions about the LVV used in LYFGENIA. Currently, viral vectors are the most common vehicle used in FDA-approved cell and gene therapies.7
A gene-addition therapy, LYFGENIA was engineered on a well-studied and traceable LVV platform that does not include the harmful viral genes needed to cause HIV infection.5,8,9
Patients may have a false-positive test for HIV if tested using a PCR-based assay after being treated with LYFGENIA. Advise patients that other tests such as antigen tests or nucleic acid tests may be able to accurately assess a person’s HIV status.1,10
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