lyf_hcp_icon_MOA

Powerful science. 
Purposeful design.

LYFGENIATM enables patients to produce a form of anti-sickling adult hemoglobin (HbAT87Q)1

sickled-red-blood-cell-combo

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

SICKLED RED BLOOD CELL2,3

sickled-red-blood-cell

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-beta-globin-gene

Mutated
β-globin gene

sickled-hemoglobin

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

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

lyf_hcp_icon_Vector

Confidence in every cell

LYFGENIA USES A WELL-STUDIED AND TRACEABLE VECTOR6
LYFGENIA is powered by a traceable, third-generation LVV5-7
viral-vector

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

traceable-vector

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

Take a closer look at LYFGENIA

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