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

 

Lumacaftor/Ivacaftor
Phase 4

For information about ongoing clinical studies, visit clinicaltrials.gov

Tezacaftor/Ivacaftor + Ivacaftor
Phase 4

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

ELEXACAFTOR/TEZACAFTOR/IVACAFTOR + IVACAFTOR
Phase 4

For information about ongoing clinical studies, visit clinicaltrials.gov

VX-561
Phase 2

We’re investigating other potential small molecule medicines aimed at treating the underlying cause of CF. 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.

VX-121 + TEZACAFTOR + VX-561 
Phase 2

We’re investigating other potential small molecule medicines aimed at treating the underlying cause of CF. 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 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 safety and efficacy of VX-121 combination therapy in subjects aged 18 years and older with cystic fibrosis, visit clinicaltrials.gov.

ADDITIONAL SMALL MOLECULES
Preclinical

We’re investigating a portfolio of other small molecule medicines aimed at treating the underlying cause of CF. This includes CFTR potentiators, which are designed to keep CFTR proteins at the cell surface open longer to improve the flow of salt and water across cell membranes, helping to hydrate and clear mucus from the airways. We’re also researching additional small molecules to address the trafficking and processing defect of the CFTR protein to enable it to move through cells and reach the surface. 

CRISPR/CAS9
Preclinical

We’re investigating potential treatments for people with CF who do not make any CFTR protein at all. We are investing in our own science and with our external partners, CRISPR TherapeuticsArbor Biotechnologies and Affinia Therapeutics to develop other potential approaches to treat the approximately 10% of people who are not expected to respond to CFTR modulator medicines. Our efforts to discover and develop therapies to potentially reach all people with CF through alternative investigational technologies, like gene editing, have been underway for years.

MRNA THERAPEUTICS
Preclinical

We’re investigating potential treatments for people with CF who do not make any CFTR protein at all. We are investing in our own science and with our external partner, Moderna, Inc., to develop other potential approaches to treat the approximately 10% of people who are not expected to respond to CFTR modulator medicines. Our efforts to discover and develop therapies to potentially reach all people with CF through alternative investigational technologies, like mRNA therapies, have been underway for years.

Pain

VX-548
Phase 2

We continue to discover, research and develop a portfolio of small molecule medicines as potential non-opioid medicines for the treatment of both acute and neuropathic pain. Our approach is to inhibit specific channels validated by human biology with the aim of alleviating pain.

ADDITIONAL SMALL MOLECULES
Preclinical

We continue to discover, research and develop a portfolio of small molecule medicines as potential non-opioid medicines for the treatment of both acute and neuropathic pain. Our approach is to inhibit specific channels validated by human biology with the aim of alleviating pain.

Sickle Cell Disease (SCD)

CTX001
Phase 1/2

We are collaborating with CRISPR Therapeutics to investigate the use of a gene-editing technology, known as CRISPR/Cas9, to discover and develop a potential one-time treatment for SCD. CTX001™ is an investigational, autologous, ex vivo CRISPR/Cas9 gene-edited therapy which aims to edit a person’s hematopoietic stem cells to produce fetal hemoglobin (HbF; hemoglobin F) in red blood cells. HbF is a form of the oxygen-carrying hemoglobin that is naturally present at birth, which then switches to the adult form of hemoglobin. The aim of using the body’s own machinery to switch red blood cells back to producing fetal hemoglobin is to address symptoms associated with the disease.  

SMALL MOLECULE
Preclinical

We’re investigating small molecule medicines aimed at the underlying cause of SCD.  

Beta Thalassemia

CTX001
Phase 1/2

We are collaborating with CRISPR Therapeutics to investigate the use of a gene-editing technology, known as CRISPR/Cas9, to discover and develop a potential one-time treatment for transfusion-dependent beta thalassemia. CTX001™ is an investigational, autologous, ex vivo CRISPR/Cas9 gene-edited therapy which aims to edit a person’s hematopoietic stem cells to produce fetal hemoglobin (HbF; hemoglobin F) in red blood cells. HbF is a form of the oxygen-carrying hemoglobin that is naturally present at birth, which then switches to the adult form of hemoglobin. The aim of using the body’s own machinery to switch red blood cells back to producing HbF is to address symptoms associated with the disease.

SMALL MOLECULE
Preclinical

We’re investigating small molecule medicines aimed at the underlying cause of transfusion-dependent beta thalassemia (TDT).  

Alpha-1 Antitrypsin Deficiency (AATD)

SMALL MOLECULES
Preclinical

We continue to research a portfolio of small molecules as potential medicines for the treatment of AATD.  

APOL1-Mediated Kidney Diseases

VX-147
Phase 2

We are investigating VX-147 aimed at inhibiting variants of APOL1

ADDITIONAL SMALL MOLECULES
Preclinical

In addition to investigating candidate medicine VX-147, we continue to research and develop a portfolio of small molecule inhibitors for the potential treatment of APOL1-mediated kidney diseases.

Duchenne Muscular Dystrophy (DMD)

DUCHENNE MUSCULAR DYSTROPHY
Preclinical

We are investigating a novel approach to treating DMD by delivering CRISPR/Cas9 gene-editing technology to the muscle cells with a virus, AAV9, to achieve precise changes in the targeted DNA sequence. Specifically, we’re researching CRISPR/Cas9 gene-editing technology with the goal of restoring near-full length dystrophin protein expression by targeting certain mutations in the dystrophin gene that cause 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. 

 

Type 1 Diabetes

VX-880
Phase 1/2

VX-880, formerly known as STx-02, is an investigational allogeneic human stem cell-derived islet cell therapy that is being evaluated for patients who have T1D with impaired hypoglycemic awareness and severe hypoglycemia.   

 

VX-880 involves an infusion of fully differentiated, functional islet cells, as well as the chronic administration of concomitant immunosuppressive therapy, to protect the islet cells from immune rejection.   

CELLS IN DEVICE
Preclinical

In addition, we are researching a second approach that would use a device to potentially protect the transplanted cells from the immune system.

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