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 Type 1 Diabetes

What is type 1 diabetes? Type 1 diabetes is a chronic, metabolic disorder caused by an insulin deficiency in the pancreas. Insulin is a hormone that the body needs to process energy. The absence of insulin leads to abnormalities in how the body processes nutrients, leading to high blood sugar levels. Symptoms of high blood sugar levels include increased thirst or hunger, frequent urination and weight loss. Over time, high blood sugar can lead to complications such as kidney disease, eye disease (including vision loss), nerve damage and even death. There are currently limited treatment options for the management of type 1 diabetes, and they require near-constant monitoring. Today, people with type 1 diabetes need to give themselves multiple injections of insulin per day or wear an insulin pump at all times, check their blood sugar regularly and follow a strict diet. A transplant of insulin-producing cells (beta cells) can eliminate the need for insulin but is only available for 100-150 people each year and requires lifelong treatment to prevent rejection of the donor tissue.  

How is type 1 diabetes diagnosed? People are often diagnosed during infancy or childhood. Type 1 diabetes was previously known as juvenile-onset diabetes or insulin-dependent diabetes. Blood tests are used to assess blood sugar levels and confirm the diagnosis. 

What is the underlying cause of disease? In most cases of type 1 diabetes, the inability to make insulin is immune-mediated, resulting in the body destroying its own beta cells in the pancreas. When beta cells function normally, they produce insulin to respond to increases in blood sugar (e.g., after eating). Without enough beta cells, and therefore insufficient insulin, blood sugar levels are not regulated, and this leads to symptoms and long-term complications. Genetic and environmental factors are thought to play a role in diabetes, but the exact mechanisms are not known.  

The Vertex Approach 

Vertex is investigating and advancing cell therapies aimed at treating the underlying cause of type 1 diabetes. In the 1970s, scientists developed an understanding that type 1 diabetes is caused by the destruction of insulin-producing beta cells by the body’s own immune system. Since then, many new medicines and technologies for disease management have become available. However, there are still no treatments that address the root cause of type 1 diabetes.  

Our investigational approach, aimed at the underlying cause of type 1 diabetes, is to use transplant technology to replace the insulin-producing cells that are destroyed in people with type 1 diabetes. We are evaluating approaches to deliver the insulin-producing cells, including a transplant approach that would require immunosuppression similar to an organ transplant, and a device approach that would protect the transplanted cells from the immune system. Proof-of-concept data in animals has indicated that there may be an opportunity to combine the ability to make large quantities of stem-cell derived human insulin-producing cells and transplant technology to initiate clinical research with a new investigative treatment for people with type 1 diabetes.  

Pipeline

Type 1 Diabetes

Type 1 Diabetes

Vertex is investigating and advancing cell therapies aimed at treating the underlying cause of type 1 diabetes. In the 1970s, scientists developed an understanding that type 1 diabetes is caused by the destruction of insulin-producing beta cells by the body's own immune system. Since then, many new medicines and technologies for disease management have become available. However, there are still no treatments that address the root cause of type 1 diabetes.

Our investigational approach, aimed at the underlying cause of type 1 diabetes, is to use transplant technology to replace the insulin-producing cells that are destroyed in people with type 1 diabetes. We are evaluating approaches to deliver the insulin-producing cells, including a transplant approach that would require immunosuppression similar to an organ transplant, and a device approach that would protect the transplanted cells from the immune system. Proof-of-concept data in animals has indicated that there may be an opportunity to combine the ability to make large quantities of stem-cell derived human insulin-producing cells and transplant technology to initiate clinical research with a new investigative treatment for people with type 1 diabetes.

 

LEARN MORE ABOUT TYPE 1 DIABETES

News 

For more information from Vertex on type 1 diabetes, please visit the Newsroom.