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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 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.

Cystic Fibrosis

Ivacaftor

Cystic Fibrosis
Clinical studies in currently marketed product

A Phase 3 clinical study is underway in children up to 24 months of age, who have one of the following mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene: G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, G1349D, or R117H (in the U.S. only).

Clinical studies in currently marketed product
Ivacaftor
Ivacaftor

How It Works

Ivacaftor is designed to keep CFTR proteins at the cell surface open longer to improve the flow of salt and water across the cell membrane, which helps hydrate and clear mucus from the airways.
 

Our Studies

A Phase 3 clinical study is underway in children up to 24 months of age, who have one of the following mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene: G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, G1349D, or R117H (in the U.S. only). To learn more, visit clinicaltrials.gov

About Cystic Fibrosis

Cystic fibrosis (CF) is a rare, life-threatening genetic disease affecting approximately 75,000 people in North America, Europe and Australia.

CF is caused by a defective or missing CFTR protein resulting from mutations in the CFTR gene. Children must inherit two defective CFTR genes — one from each parent — to have CF. There are approximately 2,000 known mutations in the CFTR gene. Some of these mutations, which can be determined by a genetic test, lead to CF by creating defective or too few CFTR proteins at the cell surface. The defective or missing CFTR protein results in poor flow of salt and water into or out of the cell in a number of organs, including the lungs. This leads to the buildup of abnormally thick, sticky mucus that can cause chronic lung infections and progressive lung damage.

Lumacaftor/Ivacaftor

Cystic Fibrosis
Clinical studies in currently marketed product

A Phase 3 clinical study was completed in children ages 2 through 5 with cystic fibrosis who have two copies of the F508del mutation in the CFTR gene.

A Phase 3, rollover long-term safety study is underway in children aged 2 years and older with cystic fibrosis who have two copies of the F508del mutation in the CFTR gene.

Clinical studies in currently marketed product
Lumacaftor/Ivacaftor
Lumacaftor/Ivacaftor

How It Works

In people with certain types of mutations in the CFTR gene, the CFTR protein is not processed and moved through the cell normally, resulting in little to no protein at the cell surface. Lumacaftor is designed to increase the amount of mature protein at the cell surface by targeting the processing and trafficking defect of the F508del CFTR protein, and ivacaftor is designed to enhance the function of the CFTR protein once it reaches the cell surface. This helps improve the flow of salt and water in to and out of cells.
 

Our Studies

Vertex has completed a Phase 3 clinical study of lumacaftor/ivacaftor in children ages 2 through 5 with cystic fibrosis who have two copies of the F508del mutation.

A Phase 3, rollover long-term safety study is underway in children aged 2 years and older with cystic fibrosis who have two copies of the F508del mutation in the CFTR gene. To learn more, visit clinicaltrials.gov.

About Cystic Fibrosis

Cystic fibrosis (CF) is a rare, life-threatening genetic disease affecting approximately 75,000 people in North America, Europe and Australia.

CF is caused by a defective or missing CFTR protein resulting from mutations in the CFTR gene. Children must inherit two defective CFTR genes — one from each parent — to have CF. There are approximately 2,000 known mutations in the CFTR gene. Some of these mutations, which can be determined by a genetic test, lead to CF by creating defective or too few CFTR proteins at the cell surface. The defective or missing CFTR protein results in poor flow of salt and water into or out of the cell in a number of organs, including the lungs. This leads to the buildup of abnormally thick, sticky mucus that can cause chronic lung infections and progressive lung damage

Tezacaftor/Ivacaftor + Ivacaftor

Tezacaftor + Ivacaftor

Cystic Fibrosis
Clinical studies in currently marketed product

A Phase 3 clinical study is underway in children ages 6 through 11 who have two copies of the F508del mutation or who have one copy of the F508del mutation and a second mutation that is expected to be responsive to tezacaftor/ivacaftor.

We have completed other Phase 3 clinical studies in people with these mutations ages 12 and older.

Clinical studies in currently marketed product
Tezacaftor/Ivacaftor + Ivacaftor

Tezacaftor + Ivacaftor

How It Works

Tezacaftor is designed to increase the amount of mature protein at the cell surface by aiding the processing and trafficking of the CFTR protein, and ivacaftor is designed to enhance the channel open probability (or gating) of the CFTR protein once it reaches the cell surface. The goal is to improve the flow of salt and water into and out of cells.

 

Our Studies

A Phase 3 clinical study is underway in children ages 6 through 11 who have two copies of the F508del mutation or who have one copy of the F508del mutation and a second mutation that is expected to be responsive to tezacaftor/ivacaftor. To learn more, visit clinicaltrials.gov.

We have completed other Phase 3 clinical studies in people with these mutations ages 12 and older.

About Cystic Fibrosis

Cystic fibrosis (CF) is a rare, life-threatening genetic disease affecting approximately 75,000 people in North America, Europe and Australia.

CF is caused by a defective or missing CFTR protein resulting from mutations in the CFTR gene. Children must inherit two defective CFTR genes — one from each parent — to have CF. There are approximately 2,000 known mutations in the CFTR gene. Some of these mutations, which can be determined by a genetic test, lead to CF by creating defective or too few CFTR proteins at the cell surface. The defective or missing CFTR protein results in poor flow of salt and water into or out of the cell in a number of organs, including the lungs. This leads to the buildup of abnormally thick, sticky mucus that can cause chronic lung infections and progressive lung damage.

VX-659 + Tezacaftor + Ivacaftor

VX-659 + Tezacaftor + Ivacaftor

Cystic Fibrosis
Phase 3

Two Phase 3 studies of VX-659, tezacaftor and ivacaftor as an investigational triple combination regimen are underway in patients ages 12 and older; one study in patients who have one F508del mutation and one minimal function mutation not likely to respond to tezacaftor and/or ivacaftor, and a second study in patients who have two copies of the F508del mutation.

Phase 3
VX-659 + Tezacaftor + Ivacaftor

How It Works

In people with some types of mutations to the CFTR gene, the CFTR protein is not processed and moved through the cell normally, resulting in little to no CFTR protein at the cell surface. VX-659 and tezacaftor (VX-661) are designed to increase the amount of mature protein at the cell surface by targeting the processing and trafficking defect of the F508del CFTR protein. Ivacaftor is designed to enhance the function of the CFTR protein once it reaches the cell surface.
 

Our Studies

Two Phase 3 studies of VX-659, tezacaftor and ivacaftor as an investigational triple combination regimen are underway in people with CF ages 12 and older.

One study is underway in patients who have one F508del mutation and one minimal function mutation not likely to respond to tezacaftor and/or ivacaftor. For a list of the minimal function mutations currently included in the study, click here.

To learn more, visit ClinicalTrials.gov.

A second Phase 3 study is underway in patients who have two copies of the F508del mutation.

To learn more, visit ClinicalTrials.gov

About Cystic Fibrosis

Cystic fibrosis (CF) is a rare, life-threatening genetic disease affecting approximately 75,000 people in North America, Europe and Australia.

CF is caused by a defective or missing CFTR protein resulting from mutations in the CFTR gene. Children must inherit two defective CFTR genes — one from each parent — to have CF. There are approximately 2,000 known mutations in the CFTR gene. Some of these mutations, which can be determined by a genetic test, lead to CF by creating defective or too few CFTR proteins at the cell surface. The defective or missing CFTR protein results in poor flow of salt and water into or out of the cell in a number of organs, including the lungs. This leads to the buildup of abnormally thick, sticky mucus that can cause chronic lung infections and progressive lung damage.

VX-445 + Tezacaftor + Ivacaftor

VX-445 + Tezacaftor + Ivacaftor

Cystic Fibrosis
Phase 3

Two Phase 3 studies of VX-445, tezacaftor and ivacaftor as an investigational triple combination regimen are underway in patients 12 and older; one study in patients who have one F508del mutation and one minimal function mutation not likely to respond to tezacaftor and/or ivacaftor, and a second study in patients with two copies of the F508del mutation.

Phase 3
VX-445 + Tezacaftor + Ivacaftor

How It Works

In people with some types of mutations to the CFTR gene, the CFTR protein is not processed and moved through the cell normally, resulting in little to no CFTR protein at the cell surface. VX-445 and tezacaftor (VX-661) are designed to increase the amount of mature protein at the cell surface by targeting the processing and trafficking defect of the F508del CFTR protein. Ivacaftor is designed to enhance the function of the CFTR protein once it reaches the cell surface. The goal is to improve the flow of salt and water into and out of cells.
 

Our Studies

Two Phase 3 studies of VX-445, tezacaftor and ivacaftor as an investigational triple combination regimen are underway in people with CF ages 12 and older.

One study is underway in patients who have one F508del mutation and one minimal function mutation not likely to respond to tezacaftor and/or ivacaftor. For a list of the minimal function mutations currently included in the study, click here. To learn more, visit ClinicalTrials.gov.

A second Phase 3 study is underway in patients who have two copies of the F508del mutation. To learn more, visit ClinicalTrials.gov

About Cystic Fibrosis

Cystic fibrosis (CF) is a rare, life-threatening genetic disease affecting approximately 75,000 people in North America, Europe and Australia.

CF is caused by a defective or missing CFTR protein resulting from mutations in the CFTR gene. Children must inherit two defective CFTR genes — one from each parent — to have CF. There are approximately 2,000 known mutations in the CFTR gene. Some of these mutations, which can be determined by a genetic test, lead to CF by creating defective or too few CFTR proteins at the cell surface. The defective or missing CFTR protein results in poor flow of salt and water into or out of the cell in a number of organs, including the lungs. This leads to the buildup of abnormally thick, sticky mucus that can cause chronic lung infections and progressive lung damage.

VX-152 + Tezacaftor + Ivacaftor

VX-152 + Tezacaftor + Ivacaftor

Cystic Fibrosis
Phase 2

Vertex has completed a Phase 2 clinical investigational study in people ages 18 and older who have one copy of the F508del mutation and a minimal function CFTR mutation not likely to respond to tezacaftor and/or ivacaftor therapy, or people who have two copies of the F508del mutation. 

Phase 2
VX-152 + Tezacaftor + Ivacaftor

How It Works

In people with some types of mutations to the CFTR gene, the CFTR protein is not processed and moved through the cell normally, resulting in little to no CFTR protein at the cell surface. VX-152 and tezacaftor (VX-661) are designed to increase the amount of mature protein at the cell surface by targeting the processing and trafficking defect of the F508del CFTR protein. Ivacaftor is designed to enhance the function of the CFTR protein once it reaches the cell surface. The goal is to improve the flow of salt and water into and out of cells.
 

Our Studies

Vertex has completed a Phase 2 clinical investigational study of VX-152 in triple combination with tezacaftor and ivacaftor in people ages 18 and older who have one copy of the F508del mutation and a minimal function CFTR mutation not likely to respond to tezacaftor and/or ivacaftor therapy, or people who have two copies of the F508del mutation.

About Cystic Fibrosis

Cystic fibrosis (CF) is a rare, life-threatening genetic disease affecting approximately 75,000 people in North America, Europe and Australia.

CF is caused by a defective or missing CFTR protein resulting from mutations in the CFTR gene. Children must inherit two defective CFTR genes — one from each parent — to have CF. There are approximately 2,000 known mutations in the CFTR gene. Some of these mutations, which can be determined by a genetic test, lead to CF by creating defective or too few CFTR proteins at the cell surface. The defective or missing CFTR protein results in poor flow of salt and water into or out of the cell in a number of organs, including the lungs. This leads to the buildup of abnormally thick, sticky mucus that can cause chronic lung infections and progressive lung damage.

VX-440 + Tezacaftor + Ivacaftor

VX-440 + Tezacaftor + Ivacaftor

Cystic Fibrosis
Phase 2

Vertex has completed a Phase 2 clinical investigational study in people ages 12 and older who have two copies of the F508del mutation or one copy of the F508del mutation and a minimal function CFTR mutation not likely to respond to tezacaftor and/or ivacaftor therapy.

Phase 2
VX-440 + Tezacaftor + Ivacaftor

How It Works

In people with certain types of mutations to the CFTR gene, the CFTR protein is not processed and moved through the cell normally, resulting in little to no CFTR protein at the cell surface. VX-440 and tezacaftor (VX-661) are designed to increase the amount of mature protein at the cell surface by targeting the processing and trafficking defect of the F508del CFTR protein. Ivacaftor is designed to enhance the function of the CFTR protein once it reaches the cell surface. The goal is to improve the flow of salt and water into and out of cells.
 

Our Studies

Vertex has completed a Phase 2 clinical investigational study of VX-440 in triple combinations with tezacaftor and ivacaftor in people ages 12 and older who have two copies of the F508del mutation or who have one copy of the F508del mutation and a minimal function CFTR mutation that is not likely to respond to tezacaftor and/or ivacaftor therapy.

About Cystic Fibrosis

Cystic fibrosis (CF) is a rare, life-threatening genetic disease affecting approximately 75,000 people in North America, Europe and Australia.

CF is caused by a defective or missing CFTR protein resulting from mutations in the CFTR gene. Children must inherit two defective CFTR genes — one from each parent — to have CF. There are approximately 2,000 known mutations in the CFTR gene. Some of these mutations, which can be determined by a genetic test, lead to CF by creating defective or too few CFTR proteins at the cell surface. The defective or missing CFTR protein results in poor flow of salt and water into or out of the cell in a number of organs, including the lungs. This leads to the buildup of abnormally thick, sticky mucus that can cause chronic lung infections and progressive lung damage.

VX-371 + Lumacaftor + Ivacaftor

VX-371 + Lumacaftor + Ivacaftor

Cystic Fibrosis
Phase 2

Vertex has completed a Phase 2a clinical investigational study in people ages 12 and older who have two copies of the F508del mutation who are also being treated with lumacaftor/ivacaftor.

There is also a Phase 2 clinical investigational study of VX-371 in people ages 12 and older who have primary ciliary dyskinesia (PCD).

Phase 2
VX-371 + Lumacaftor + Ivacaftor

How It Works

In partnership with Parion Sciences, our VX-371 program is investigating the role of sodium epithelial channel (ENaC) inhibitors in people with CF. Learn more here.

 

Our Studies

Vertex has completed a Phase 2a clinical investigational study of VX-371 in people ages 12 and older who have two copies of the F508del mutation who are also being treated with lumacaftor/ivacaftor.

There is also a Phase 2 clinical investigational study of VX-371 in people ages 12 and older who have primary ciliary dyskinesia (PCD), a rare genetic disease that results in a loss of function in key ciliary proteins. The defective proteins lead to dysfunctional beating of cilia on the surface of cells, especially in the lungs, where the accumulation of mucus can lead to chronic lung infections, bronchiectasis and progressive lung function decline. To learn more, visit clinicaltrials.gov.

About Cystic Fibrosis

Cystic fibrosis (CF) is a rare, life-threatening genetic disease affecting approximately 75,000 people in North America, Europe and Australia.

CF is caused by a defective or missing CFTR protein resulting from mutations in the CFTR gene. Children must inherit two defective CFTR genes — one from each parent — to have CF. There are approximately 2,000 known mutations in the CFTR gene. Some of these mutations, which can be determined by a genetic test, lead to CF by creating defective or too few CFTR proteins at the cell surface. The defective or missing CFTR protein results in poor flow of salt and water into or out of the cell in a number of organs, including the lungs. This leads to the buildup of abnormally thick, sticky mucus that can cause chronic lung infections and progressive lung damage.

VX-659 + Tezacaftor + Ivacaftor

VX-659 + Tezacaftor + Ivacaftor

VX-659 + Tezacaftor + VX-561

Cystic Fibrosis
Phase 2

Vertex has completed a Phase 2 clinical investigational study of VX-659 in triple combination with tezacaftor and ivacaftor, and in triple combination with tezacaftor and VX-561, in people with CF ages 18 and older who have certain mutations.

 

Phase 2
VX-659 + Tezacaftor + Ivacaftor
VX-659 + Tezacaftor + VX-561

How It Works

In people with some types of mutations to the CFTR gene, the CFTR protein is not processed and moved through the cell normally, resulting in little to no CFTR protein at the cell surface. VX-659 and tezacaftor (VX-661) are designed to increase the amount of mature protein at the cell surface by targeting the processing and trafficking defect of the F508del CFTR protein. Ivacaftor is designed to enhance the function of the CFTR protein once it reaches the cell surface.
 

Our Studies

Vertex has completed a Phase 2 clinical investigational study of VX-659 in triple combination with tezacaftor and ivacaftor in people with CF ages 18 and older who have one copy of the F508del mutation and one minimal function mutation, and also in people with CF ages 18 and older who have two copies of the F508del mutation. The study also evaluated VX-659 in triple combination with tezacaftor and VX-561 (formerly CTP-656) in people with CF ages 18 and older who have one copy of the F508del mutation and one minimal function mutation. 

About Cystic Fibrosis

Cystic fibrosis (CF) is a rare, life-threatening genetic disease affecting approximately 75,000 people in North America, Europe and Australia.

CF is caused by a defective or missing CFTR protein resulting from mutations in the CFTR gene. Children must inherit two defective CFTR genes — one from each parent — to have CF. There are approximately 2,000 known mutations in the CFTR gene. Some of these mutations, which can be determined by a genetic test, lead to CF by creating defective or too few CFTR proteins at the cell surface. The defective or missing CFTR protein results in poor flow of salt and water into or out of the cell in a number of organs, including the lungs. This leads to the buildup of abnormally thick, sticky mucus that can cause chronic lung infections and progressive lung damage.

VX-445 + Tezacaftor + Ivacaftor

VX-445 + Tezacaftor + Ivacaftor

VX-445 + Tezacaftor + VX-561

Cystic Fibrosis
Phase 2

Vertex has completed a Phase 1/2 study of VX-445 alone, in triple combination with tezacaftor and ivacaftor, and in triple combination with tezacaftor and VX-561 in healthy volunteers and in people with CF ages 18 and older who have certain mutations. 

 

Phase 2
VX-445 + Tezacaftor + Ivacaftor
VX-445 + Tezacaftor + VX-561

How It Works

In people with some types of mutations to the CFTR gene, the CFTR protein is not processed and moved through the cell normally, resulting in little to no CFTR protein at the cell surface. VX-445 and tezacaftor (VX-661) are designed to increase the amount of mature protein at the cell surface by targeting the processing and trafficking defect of the F508del CFTR protein. Ivacaftor is designed to enhance the function of the CFTR protein once it reaches the cell surface. The goal is to improve the flow of salt and water into and out of cells.
 

Our Studies

Vertex has completed a Phase 1/2 study of VX-445 alone, and in triple combination with tezacaftor and ivacaftor, in people with CF ages 18 and older who have one copy of the F508del mutation and one minimal function mutation, and in people with CF ages 18 and older who have two copies of the F508del mutation. The study also evaluated VX-445 in triple combination with tezacaftor and VX-561 (formerly CTP-656) in people with CF ages 18 and older who have one copy of the F508del mutation and one minimal function mutation. 

About Cystic Fibrosis

Cystic fibrosis (CF) is a rare, life-threatening genetic disease affecting approximately 75,000 people in North America, Europe and Australia.

CF is caused by a defective or missing CFTR protein resulting from mutations in the CFTR gene. Children must inherit two defective CFTR genes — one from each parent — to have CF. There are approximately 2,000 known mutations in the CFTR gene. Some of these mutations, which can be determined by a genetic test, lead to CF by creating defective or too few CFTR proteins at the cell surface. The defective or missing CFTR protein results in poor flow of salt and water into or out of the cell in a number of organs, including the lungs. This leads to the buildup of abnormally thick, sticky mucus that can cause chronic lung infections and progressive lung damage.

Gene Editing

Cystic Fibrosis
Research & Investigation
Research & Investigation
Gene Editing
Gene Editing

Gene Editing

Vertex is collaborating with CRISPR Therapeutics to investigate the use of CRISPR's gene editing technology, known as CRISPR-Cas9, to discover and develop potential new treatments aimed at the underlying genetic causes of human disease. As part of the collaboration, Vertex and CRISPR will evaluate the use of CRISPR-Cas9 to potentially correct the mutations in the CFTR gene known to result in the defective protein that causes CF and to edit other genes that contribute to the disease. Check back for future updates.

About Cystic Fibrosis

Cystic fibrosis (CF) is a rare, life-threatening genetic disease affecting approximately 75,000 people in North America, Europe and Australia.

CF is caused by a defective or missing CFTR protein resulting from mutations in the CFTR gene. Children must inherit two defective CFTR genes — one from each parent — to have CF. There are approximately 2,000 known mutations in the CFTR gene. Some of these mutations, which can be determined by a genetic test, lead to CF by creating defective or too few CFTR proteins at the cell surface. The defective or missing CFTR protein results in poor flow of salt and water into or out of the cell in a number of organs, including the lungs. This leads to the buildup of abnormally thick, sticky mucus that can cause chronic lung infections and progressive lung damage.

Messenger Ribonucleic Acid (mRNA) Therapies

Cystic Fibrosis
Research & Investigation
Research & Investigation
Messenger Ribonucleic Acid (mRNA) Therapies
Messenger Ribonucleic Acid (mRNA) Therapies

Messenger Ribonucleic Acid (mRNA) Therapies

Vertex is collaborating with Moderna Therapeutics in an exclusive research collaboration and licensing agreement aimed at the discovery and development of messenger Ribonucleic Acid (mRNA) Therapeutics™ for the treatment of cystic fibrosis (CF). The research collaboration focuses on the use of mRNA therapies to treat the underlying cause of CF by enabling cells in the lungs to produce functional copies of the cystic fibrosis transmembrane conductance regulator (CFTR) protein, which is known to be defective in people with CF. Through the collaboration, the companies will explore the potential utilization of pulmonary mRNA delivery. Check back for future updates.

About Cystic Fibrosis

Cystic fibrosis (CF) is a rare, life-threatening genetic disease affecting approximately 75,000 people in North America, Europe and Australia.

CF is caused by a defective or missing CFTR protein resulting from mutations in the CFTR gene. Children must inherit two defective CFTR genes — one from each parent — to have CF. There are approximately 2,000 known mutations in the CFTR gene. Some of these mutations, which can be determined by a genetic test, lead to CF by creating defective or too few CFTR proteins at the cell surface. The defective or missing CFTR protein results in poor flow of salt and water into or out of the cell in a number of organs, including the lungs. This leads to the buildup of abnormally thick, sticky mucus that can cause chronic lung infections and progressive lung damage.

Pain

VX-150

VX-150

Pain
Phase 2

Vertex has completed Phase 2 proof-of-concept studies in osteoarthritis and acute pain and is currently conducting an additional Phase 2 proof-of-concept study in neuropathic pain.

Phase 2
VX-150

How it Works

Vertex is investigating VX-150 as a potential medicine for the treatment of acute and neuropathic pain. VX-150 is designed to block pain sensation through inhibition of NaV1.8, a voltage-gated sodium channel expressed in peripheral neurons that transmit pain.

Our Studies

In 2016, Vertex completed a Phase 2 proof-of-concept study in osteoarthritis. To learn more, visit clinicaltrials.gov.

In 2018, Vertex completed a Phase 2 proof-of-concept study in acute pain. To learn more, visit clinicaltrials.gov.

To learn more about the ongoing Phase 2 proof-of-concept study in neuropathic pain, visit clinicaltrials.gov.

   

Hemoglobinopathies

Hemoglobinopathies
Phase 1/2
Phase 1/2
CTX001

Our Studies

Vertex is collaborating with CRISPR Therapeutics to investigate the use of CRISPR's gene editing technology, known as CRISPR-Cas9, to discover and develop potential new treatments aimed at the underlying genetic causes of human disease. CRISPR Therapeutics and Vertex will co-develop and co-commercialize CTX001 for the treatment of hemoglobinopathies, including β-thalassemia and sickle cell disease. CTX001 is an investigational ex vivo CRISPR gene-edited therapy for patients suffering from β-thalassemia and sickle cell disease in which a patient's hematopoietic stem cells are engineered to produce high levels of fetal hemoglobin (HbF; hemoglobin F) in red blood cells.

About Hemoglobinopathies

Hemoglobinopathies are blood disorders that are caused by changes in the genes that encode the hemoglobin protein. Hemoglobin is a protein in red blood cells that carries oxygen to all organs of the body. Vertex is currently investigating CTX001 for the treatment of two hemoglobinopathies: Sickle Cell Disease (SCD) and Beta-Thalassemia.

Beta-Thalassemia is caused by mutations in the HBB gene that results in low or no beta-globin production, which is an important building block of hemoglobin. Patients with severe β-thalassemia suffer from anemia and are dependent on blood transfusions which can lead to iron accumulation and complications that damage organs and shorten life-span.

Sickle Cell Disease (SCD) is caused by mutations in the HBB gene that lead to an abnormal hemoglobin, called sickle hemoglobin (HbS). Because of this abnormal hemoglobin, red blood cells can become rigid and block small blood vessels. Patients with SCD suffer from pain, organ damage, and shortened life span due the abnormal properties of HbS.

Alpha-1 Antitrypsin Deficiency

Alpha-1 Antitrypsin Deficiency
Research & Investigation
Research & Investigation
Alpha-1 Antitrypsin Deficiency

How It Works

Vertex is investigating the use of small molecule correctors to potentially treat AATD.

About Alpha-1 Antitrypsin Deficiency

Alpha-1 Antitrypsin Deficiency, or AATD, is a genetic disorder caused by mutations in the gene that codes for the AAT protein. Some of these mutations lead to defective AAT that becomes trapped in the liver, where most of the body’s AAT is synthesized. Since AAT plays an important role regulating inflammation in the body, low levels of AAT circulating in the blood allows inflammation to proceed unchecked and can result in damage to healthy tissues. The impact is greatest in the lung and liver. In the lung, tissue damage can lead to emphysema, resulting in difficulty breathing and problems with mucus clearance. In the liver, scarring and damage can occur, causing tiredness, weight loss and jaundice. This can progress to irreversible liver damage or failure and increases a patient's risk of liver cancer.

Oncology (Outlicensed to Merck KGaA, Darmstadt, Germany)

VX-970

VX-970

Oncology (Outlicensed to Merck KGaA, Darmstadt, Germany)
Phase 2
Phase 2
VX-970

In January 2017, Vertex entered into a licensing agreement with Merck KGaA, Darmstadt, Germany for the worldwide development and commercialization of four promising research and development programs for the treatment of cancer. As part of the agreement, Merck KGaA, Darmstadt, Germany licensed two clinical-stage programs comprised of the compounds VX-970, VX-803 and VX-984, targeting DNA damage and repair, along with two additional novel research programs that include one immuno-oncology program and a program against a completely novel target. Learn more.

   

VX-803

VX-803

Oncology (Outlicensed to Merck KGaA, Darmstadt, Germany)
Phase 1
Phase 1
VX-803

In January 2017, Vertex entered into a licensing agreement with Merck KGaA, Darmstadt, Germany for the worldwide development and commercialization of four promising research and development programs for the treatment of cancer. As part of the agreement, Merck KGaA, Darmstadt, Germany licensed two clinical-stage programs comprised of the compounds VX-970, VX-803 and VX-984, targeting DNA damage and repair, along with two additional novel research programs that include one immuno-oncology program and a program against a completely novel target. Learn more.

   

VX-984

VX-984

Oncology (Outlicensed to Merck KGaA, Darmstadt, Germany)
Phase 1
Phase 1
VX-984

In January 2017, Vertex entered into a licensing agreement with Merck KGaA, Darmstadt, Germany for the worldwide development and commercialization of four promising research and development programs for the treatment of cancer. As part of the agreement, Merck KGaA, Darmstadt, Germany licensed two clinical-stage programs comprised of the compounds VX-970, VX-803 and VX-984, targeting DNA damage and repair, along with two additional novel research programs that include one immuno-oncology program and a program against a completely novel target. Learn more.

   

Influenza (Outlicensed to Janssen Pharmaceuticals, Inc.)

VX-787

VX-787

Influenza (Outlicensed to Janssen Pharmaceuticals, Inc.)
Phase 2
Phase 2
VX-787

In June 2014, Vertex entered into a licensing agreement with Janssen Pharmaceuticals, Inc. for the worldwide development and commercialization of VX-787, a novel medicine discovered by Vertex for the treatment of influenza. Learn more.