Powerful science. Purposeful
design.
Powerful science. Purposeful design.
LYFGENIATM enables patients to produce a form of anti-sickling adult hemoglobin (HbAT87Q)1
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
Overview of SCD and Prevalence of VOEs
Hear from clinical experts as they contextualize the burden of disease and discuss why VOE frequency remains central to treatment decision-making
Overview of SCD and Prevalence of VOEs
Hear from clinical experts as they contextualize the burden of disease and discuss why VOE frequency remains central to treatment decision-making
How LYFGENIA works to address HbS levels without electroporation of cells
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 pair with α-globin to produce functional HbA (HbAT87Q)1
ENGRAFTMENT1
Following successful engraftment, red blood cells containing
βA-T87Q-globin pair 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 pair
with α-globin to produce
functional
HbA
(HbAT87Q)1
ENGRAFTMENT1
Following successful engraftment, red blood cells
containing βA-T87Q-globin pair 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.
Transduction introduces genetic material via common cell receptors, whereas electroporation creates temporary holes in the cell membrane to enable drug entry1,4,5
How LYFGENIA Works
Practicing clinicians explain the underlying science behind LYFGENIA, including its mechanism of action
How LYFGENIA Works
Practicing clinicians explain the underlying science behind LYFGENIA, including its mechanism of action
Confidence in every cell
LYFGENIA USES A WELL-STUDIED AND TRACEABLE VECTOR6
LYFGENIA is powered by a traceable, third-generation LVV5-7
The longest-studied viral vector platform in SCD:
Viral vectors are the most commonly used vehicle in FDA-approved cell and gene therapies, representing the predominant platform with the longest-standing safety data.8
A traceable vector:
A gene-addition therapy, LYFGENIA was engineered on a well-studied and traceable LVV platform that does not include the harmful viral genes that are required to cause HIV infection.5-7,9
Patients who have received LYFGENIA should not be screened for HIV infection using a PCR-based assay because they may have a false-positive test result if tested using a PCR assay.1,10 Other tests such as antigen tests or nucleic acid tests may be able to accurately assess a person’s HIV status.10
Understanding Viral Vectors
Thought leaders address common questions and discuss viral vectors
Understanding Viral Vectors
Thought leaders address common questions and discuss viral vectors
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