Research and Pipeline

Vertex is focused on discovering, developing and commercializing innovative medicines so people with serious diseases can lead better lives. Our scientists don’t see the impossible as an obstacle; they see it as a good place to start.

These studies are investigating treatments or outcomes that have not all received approval from a health authority. The information presented is not intended to convey conclusions of safety or efficacy. There is no guarantee that the outcome of these studies will result in approval by a health authority. 

About APOL1-Mediated Kidney Diseases

The APOL1 gene is part of the APOL gene family, which plays a role in innate immunity, our body’s built-in system to fight threats. It is present in many tissues, including the kidneys. Certain variants in the APOL1 gene cause damage to the parts of the kidney that filter blood, which can lead to kidney disease or even kidney failure. Focal segmental glomerulosclerosis is one type of kidney disease that can be caused by APOL1 gene variants.

APOL1-Mediated Focal Segmental Glomerulosclerosis (FSGS)

What is APOL1-mediated focal segmental glomerulosclerosis? APOL1-mediated FSGS is a severe kidney disorder that results in high levels of protein in the urine, or proteinuria. This excess protein can lead to symptoms, including swelling in the legs and feet and weight gain. The disease may eventually lead to kidney failure, which requires dialysis or a transplant. Current treatments do not address the underlying cause of the disease. High-dose steroids for a short period of time are often used, but they do not always work and can have severe side effects. Even with treatment, people with APOL1-mediated FSGS may progress to kidney failure. Kidney failure is treated with regular, long-term dialysis or a kidney transplant. Both treatments require lifelong treatment and follow-up. 

How is APOL1-mediated FSGS diagnosed? Tests to diagnose APOL1-mediated FSGS usually include blood and urine tests, a kidney biopsy and genetic tests. Other tests may be needed to exclude other causes of FSGS or other types of kidney disease. 

What is the underlying cause of disease? In people living with APOL1-mediated FSGS, variants in the APOL1 gene can lead to cell death in the kidneys and scarring in the glomeruli, which filter blood in the kidney. This leads to abnormal amounts of protein in the urine and decreased ability of the kidney to function. The disease may eventually lead to kidney failure.  

The Vertex Approach 

We are focused on discovering and researching medicines aimed at treating the underlying cause of APOL1-mediated kidney diseases. The genetic link between APOL1 gene variants and severe kidney diseases was only identified in 2010. That insight led our team to work to invent new investigational medicines to target the underlying cause of APOL1-mediated kidney diseases, including FSGS.  

We are investigating VX-147 and other small molecules aimed at inhibiting APOL1. We continue to discover, research and develop a portfolio of small molecule inhibitors for the potential treatment of APOL1-mediated kidney diseases.  

Pipeline

APOL1-Mediated Kidney Diseases

VX-147
Phase 2

We are focused on discovering and researching medicines aimed at treating the underlying cause of APOL1-mediated kidney diseases. The genetic link between APOL1 gene variants and severe kidney diseases was only identified in 2010. That insight led our team to work to invent new investigational medicines to target the underlying cause of APOL1-mediated kidney diseases, including FSGS. 

We are investigating VX-147 and other small molecules aimed at inhibiting APOL1. We continue to discover, research and develop a portfolio of small molecule inhibitors for the potential treatment of APOL1-mediated kidney diseases.

 

LEARN MORE ABOUT APOL1-MEDIATED KIDNEY DISEASES

News 

For more information from Vertex on APOL1-mediated kidney diseases, please visit the Newsroom