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.

What is the Vertex Strategy? 

We have a unique way of building our drug discovery programs to maximize their chances of creating therapies that may dramatically improve patients’ lives. We focus on serious diseases where we can have a transformative impact for patients, not just an incremental benefit. We work only on projects where we have a deep understanding of the underlying cause of disease in humans. Then we research and develop therapeutic approaches that are most likely to succeed. Rather than looking for problems we can solve with only the tools we’ve used before, we figure out the problems that need to be solved for the diseases we’re going after and invent the tools to potentially fix them.

Cystic Fibrosis (CF)

Ivacaftor
Phase 4

For information about ongoing clinical studies, visit clinicaltrials.gov.

 

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Lumacaftor/Ivacaftor
Phase 4

For information about ongoing clinical studies, visit clinicaltrials.gov.

 

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Tezacaftor/Ivacaftor + Ivacaftor
Phase 4

For information about ongoing clinical studies, visit clinicaltrials.gov.

 

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ELEXACAFTOR/TEZACAFTOR/IVACAFTOR + IVACAFTOR
Phase 4

For information about ongoing clinical studies, visit clinicaltrials.gov

 

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VX-561
Phase 2

We are investigating medicines aimed at treating the underlying cause of cystic fibrosis. VX-561 (deutivacaftor) is an investigational once daily potentiator 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. 

To learn about our Phase 2 study to evaluate the efficacy and safety of VX-561 in subjects aged 18 years and older with cystic fibrosis, visit clinicaltrials.gov.

 

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VX-121 + TEZACAFTOR + VX-561 
Phase 2

We are investigating medicines aimed at treating the underlying cause of cystic fibrosis. In people with certain types of mutations in the CFTR gene, the CFTR protein is not processed and cannot move through the cell normally. This results in little to no protein at the cell surface. VX-121 and tezacaftor are designed to increase the amount of mature protein at the cell surface by targeting the processing and trafficking defect of the CFTR protein. VX-561 (deutivacaftor) 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.  

To learn about our Phase 2 study to evaluate the safety and efficacy of VX-121 combination therapy in subjects aged 18 years and older with cystic fibrosis, visit clinicaltrials.gov.

 

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Pain

VX-150
Phase 2

We are focused on researching and discovering medicines aimed at the underlying cause of pain. Although the function of voltage-gated sodium channels like NaV 1.7 and 1.8 as related to pain sensation has been understood since 2006, no one has successfully developed a treatment that uses these mechanisms to relieve pain. Because of the significant prevalence of pain across the world and health complications it may cause, we believe that there is a significant unmet need for potential new treatments.

We are investigating an approach to treating pain, targeting and potentially interrupting pain signaling at its root. We are investigating VX-150 and other molecules as potential non-opioid medicines for the treatment of both acute and chronic pain, including chronic neuropathic and chronic musculoskeletal pain. VX-150 is designed to block pain sensation by selectively inhibiting the voltage-gated sodium channel 1.8 (NaV 1.8). We are continuing to discover, research and develop additional small molecule inhibitors for the potential treatment of acute and chronic pain.

 

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Sickle Cell Disease (SCD)

CTX001
Phase 1/2

We are investigating the use of genetic therapies aimed at the underlying cause of SCD. The cause of SCD has been known since Linus Pauling described the "first molecular disease" in 1949, but no one has successfully developed a therapeutic approach that gets at the underlying cause of disease. With the discovery of tools like CRISPR gene editing, we now potentially have an opportunity to address diseases at their root cause. 

We are collaborating with CRISPR Therapeutics to investigate the use of gene-editing technology, known as CRISPR-Cas9, to discover and develop a new one-time treatment for SCD. CTX001 is an investigational ex-vivo CRISPR gene-edited therapy, which aims to edit a person’s hematopoietic stem cells to produce fetal hemoglobin (HbF; hemoglobin F) in red blood cells. The aim of using the body's own machinery to switch red blood cells back to fetal hemoglobin production is a significant reduction or elimination of symptoms associated with the disease.

 

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Beta Thalassemia

CTX001
Phase 1/2

We are investigating the use of genetic therapies aimed at the underlying cause of transfusion-dependent beta thalassemia. The cause of transfusion-dependent beta thalassemia has been known for decades, but no one has developed a therapeutic approach that gets at the underlying cause of disease. With the discovery of tools like CRISPR gene editing, we now potentially have an opportunity to address diseases at their root cause.

We are collaborating with CRISPR Therapeutics to investigate the use of gene-editing technology, known as CRISPR-Cas9, to discover and develop a new one-time treatment for transfusion-dependent beta thalassemia. CTX001 is an investigational ex-vivo CRISPR gene-edited therapy, which aims to edit a person’s hematopoietic stem cells to produce fetal hemoglobin (HbF; hemoglobin F) in red blood cells. The aim of using the body's own machinery to switch red blood cells back to fetal hemoglobin production is a significant reduction or elimination of symptoms associated with the disease.

 

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Alpha-1 Antitrypsin Deficiency (AATD)

VX-814
Phase 2

We are focused on discovering and investigating medicines aimed at treating the underlying cause of alpha-1 antitrypsin deficiency. Although the protein folding defect that causes AATD has been known since 1963, there are still no effective treatments for the multiple manifestations of the disease. We are building on our pioneering research with protein folding correction in cystic fibrosis to develop medicines with the potential to treat people with AATD.

We are investigating the use of small molecules, which have the potential to address the underlying cause of disease and impact the lung and liver disease associated with AATD. We are currently investigating two candidate medicines, VX-814 and VX-864, and continue to discover and develop a portfolio of medicines for the potential treatment of AATD.

 

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VX-864
Phase 2

We are focused on discovering and investigating medicines aimed at treating the underlying cause of alpha-1 antitrypsin deficiency. Although the protein folding defect that causes AATD has been known since 1963, there are still no effective treatments for the multiple manifestations of the disease. We are building on our pioneering research with protein folding correction in cystic fibrosis to develop medicines with the potential to treat people with AATD.

We are investigating the use of small molecules, which have the potential to address the underlying cause of disease and impact the lung and liver disease associated with AATD. We are currently investigating two candidate medicines, VX-814 and VX-864, and continue to discover and develop a portfolio of medicines for the potential treatment of AATD.

 

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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.

 

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Duchenne Muscular Dystrophy (DMD)

DUCHENNE MUSCULAR DYSTROPHY

We are focused on inventing and researching genetic therapies aimed at treating the underlying cause of DMD. The cause of DMD has been known for decades and with tools like CRISPR gene editing, we now have a potential opportunity to address this devastating disease at its root cause.

We are investigating multiple approaches to treating DMD by delivering CRISPR gene-editing technology to the muscles with a virus, AAV9, in order to achieve the precise changes in the targeted DNA. Specifically, we will investigate CRISPR gene-editing technology to restore dystrophin protein expression by reframing the mutated DMD gene that causes the disease. Due to the number of mutations that can cause DMD, our research consists of multiple different gene-editing programs to potentially address many of the disease-causing mutations.

 

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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.

 

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Outlicensed

VX-970 (OUTLICENSED TO MERCK KGAA, DARMSTADT, GERMANY)
Phase 2

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 immune-oncology program and a program against a completely novel target. Learn more.

VX-803 (OUTLICENSED TO MERCK KGAA, DARMSTADT, GERMANY)
Phase 1

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 immune-oncology program and a program against a completely novel target. Learn more.

VX-984 (OUTLICENSED TO MERCK KGAA, DARMSTADT, GERMANY)
Phase 1

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 immune-oncology program and a program against a completely novel target. Learn more.