R&D Pipeline

These programs 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.

Vertex is focused on discovering, developing and producing 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.

We have a unique way of building our drug discovery programs to maximize their chances of creating therapies that may dramatically improve patients’ lives. Our focus is on serious diseases where we could truly 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.

Read more

Line drawing of a scientist adding liquid to a test tube

These programs 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.

Vertex is focused on discovering, developing and producing 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.

We have a unique way of building our drug discovery programs to maximize their chances of creating therapies that may dramatically improve patients’ lives. Our focus is on serious diseases where we could truly 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.

Read more

icon graphic for cystic fibrosis
Phase 1
Phase 2
Phase 3
Phase 4Phase 4

We are submitting regulatory filings globally for geographic expansion and/or label expansions. For information about ongoing clinical studies, visit clinicaltrials.gov or EU Clinical Trials Register.

False
Phase 1
Phase 2
Phase 3
Phase 4Phase 4

We are submitting regulatory filings globally for geographic expansion and/or label expansions. For information about ongoing clinical studies, visit clinicaltrials.gov or EU Clinical Trials Register.

False
Phase 1
Phase 2
Phase 3
Phase 4Phase 4

We are submitting regulatory filings globally for geographic expansion and/or label expansions. For information about ongoing clinical studies, visit clinicaltrials.gov or EU Clinical Trials Register.

False
Phase 1
Phase 2
Phase 3
Phase 4Phase 4

We are submitting regulatory filings globally for geographic expansion and/or label expansions. For information about ongoing clinical studies, visit clinicaltrials.gov or EU Clinical Trials Register.

False
Phase 1
Phase 2
Phase 3Phase 3
Phase 4

We’re investigating other potential small molecule medicines targeting the underlying cause of CF. In people with certain types of mutations in the cystic fibrosis transmembrane conductance regulator (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 a 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. The triple combination of VX-121/tezacaftor/VX-561 is being developed as an investigational once-daily treatment for people with CF with certain mutations in the CFTR gene. 

To learn about our Phase 3 study to evaluate the safety and efficacy of VX-121 combination therapy in eligible subjects aged 12 years and older with CF, visit clinicaltrials.gov or EU Clinical Trials Register

False
Research
Phase 1
Phase 2
Phase 3
Phase 4

We’re investigating a portfolio of other small molecule medicines targeting the underlying cause of cystic fibrosis. 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.

False
Research
Phase 1
Phase 2
Phase 3
Phase 4

We’re investigating potential treatments for people with cystic fibrosis who do not make any CFTR protein at all. We are investing in our own science and with our external partners, CRISPR Therapeutics, Arbor 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 cystic fibrosis through alternative investigational technologies, like gene editing, have been underway for years.

False
Research
Phase 1
Phase 2
Phase 3
Phase 4

We’re investigating potential treatments for people with cystic fibrosis 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 cystic fibrosis through alternative investigational technologies, like mRNA therapies, have been underway for years.

False
icon graphic for pain
Phase 1
Phase 2Phase 2
Phase 3
Phase 4

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.

True
Research
Phase 1
Phase 2
Phase 3
Phase 4

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.  

False
icon graphic for sickle cell disease
Phase 1
Phase 2/3
Phase 2
Phase 3
Phase 4

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 sickle cell disease. 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). 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 is to use the body’s own machinery to switch back to producing fetal hemoglobin.   

True
Research
Phase 1
Phase 2
Phase 3
Phase 4

We’re investigating small molecule medicines aimed at the underlying cause of sickle cell disease.

False
icon graphic for beta thalassemia
Phase 1
Phase 2/3
Phase 2
Phase 3
Phase 4

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). 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 is to use the body’s own machinery to switch back to producing fetal hemoglobin.

False
Research
Phase 1
Phase 2
Phase 3
Phase 4

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

False
icon graphic for alpha-1 antitrypsin deficiency
Research
Phase 1
Phase 2
Phase 3
Phase 4

We continue to research a portfolio of small molecules as potential medicines for the treatment of alpha-1 antitrypsin deficiency.  

False
icon graphic for apol-1 mediated kidney disease
Phase 1
Phase 2/3
Phase 2
Phase 3
Phase 4

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

False
Research
Phase 1
Phase 2
Phase 3
Phase 4

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 disease.

False
icon graphic for duchenne muscular dystrophy
Research
Phase 1
Phase 2
Phase 3
Phase 4

We are investigating a novel approach to treating Duchenne muscular dystrophy 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 Duchenne muscular dystrophy, our research consists of multiple different gene-editing programs to potentially address many of the disease-causing mutations.   

False
icon graphic for type 1 diabetes
Phase 1
Phase 1/2
Phase 2
Phase 3
Phase 4

VX-880 is an investigational allogeneic stem cell-derived, fully differentiated, insulin-producing islet cell therapy manufactured using proprietary technology. It is being evaluated for patients who have type 1 diabetes with impaired hypoglycemic awareness and severe hypoglycemia. VX-880 is being investigated as an infusion into the hepatic portal vein and requires immunosuppressive therapy to protect the islet cells from immune rejection.

False
Research
Phase 1
Phase 2
Phase 3
Phase 4

We are researching an encapsulated islet cell program, which would potentially eliminate the requirement for immunosuppression by protecting the cells from the immune system.

False
icon graphic for outlicensed investigational medicines
(Outlicensed to Merck KGaA, Darmstadt, Germany)
Phase 1
Phase 2Phase 2
Phase 3
Phase 4

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.

False
(Outlicensed to Merck KGaA, Darmstadt, Germany)
Phase 1Phase 1
Phase 2
Phase 3
Phase 4

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.

False
(Outlicensed to Merck KGaA, Darmstadt, Germany)
Phase 1Phase 1
Phase 2
Phase 3
Phase 4

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.

False
True
False