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鈥檛 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鈥 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鈥檝e used before, we figure out the problems that need to be solved for the diseases we鈥檙e going after and invent the tools to potentially fix them.

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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鈥檛 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鈥 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鈥檝e used before, we figure out the problems that need to be solved for the diseases we鈥檙e 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 in the U.S., visit聽the . For information about non-U.S. sites, visit .

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 in the U.S., visit聽the . For information about non-U.S. sites, visit .

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 in the U.S., visit聽the . For information about non-U.S. sites, visit .

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 in the U.S., visit聽the . For information about non-U.S. sites, visit .

False
Phase 1
Phase 2
Phase 3Phase 3
Phase 4

We鈥檙e 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. Vanzacaftor, formerly known as 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. 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 vanzacaftor/tezacaftor/deutivacaftor is being developed as an investigational once-daily treatment for people with CF with certain mutations in the CFTR gene.

For information about ongoing clinical studies in the U.S., visit聽the . For information about non-U.S. sites, visit .

False
Phase 1Phase 1
Phase 2
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Phase 4

We are investigating VX-522, a CFTR mRNA that can be delivered to the lung by lipid nanoparticles to address the underlying cause of CF lung disease in approximately 5,000 people with CF who do not make any CFTR protein that responds to a CFTR modulator therapy.

For information about ongoing clinical studies in the U.S., visit聽the . For information about non-U.S. sites, visit .

False
Research
Phase 1
Phase 2
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Phase 4

We鈥檙e investigating a portfolio of other small molecule medicines targeting the underlying cause of cystic fibrosis with the aim of achieving carrier levels of CFTR function. 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鈥檙e 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
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Phase 4

We鈥檙e investigating potential treatments for people with cystic fibrosis who do not make any CFTR protein. We are investing in our own science and with our external partners CRISPR, Arbor and Moderna to develop other potential approaches with an aim to treat approximately 5,000 people who do not make any CFTR protein that responds to a CFTR modulator therapy. Our efforts to discover and develop therapies to reach as many people as possible with cystic fibrosis through alternative investigational technologies, like mRNA therapies, have been underway for years.

False
icon graphic for pain
Phase 1
Phase 2
Phase 3Phase 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.听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.听Our approach is to inhibit specific channels validated by human biology with the aim of alleviating pain.听聽

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

We are submitting regulatory filings globally for geographic expansion. For information about ongoing clinical studies in the U.S., visit the . For information about non-U.S. sites, visit .听

True
Research
Phase 1
Phase 2
Phase 3
Phase 4

We鈥檙e鈥痠nvestigating鈥痵mall molecule鈥痬edicines聽aimed at the underlying cause of sickle cell disease.

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

We are submitting regulatory filings globally for geographic expansion. For information about ongoing clinical studies in the U.S., visit the . For information about non-U.S. sites, visit .听

False
Research
Phase 1
Phase 2
Phase 3
Phase 4

We鈥檙e鈥痠nvestigating鈥痵mall molecule鈥痬edicines聽aimed at the underlying cause of鈥痶ransfusion-dependent beta thalassemia.鈥

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

We are investigating VX-634, a small molecule that aims to treat the underlying cause of alpha-1 antitrypsin deficiency.

False
Phase 1Phase 1
Phase 2
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Phase 4

We are investigating VX-668, a small molecule that aims to treat the underlying cause of alpha-1 antitrypsin deficiency.

False
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
Phase 3Phase 3
Phase 4

We are investigating inaxaplin, formerly known as VX-147, aimed at inhibiting鈥痟igh-risk variants of鈥 APOL1.听

False
Research
Phase 1
Phase 2
Phase 3
Phase 4

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

False
An icon of a pair of lungs, including one with one cysts in it, to represent ADPKD
Phase 1Phase 1
Phase 2
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Phase 4

We are investigating VX-407 for the treatment of ADPKD in patients with a subset of PKD1 genetic variants, with the aim of restoring function to the polycystin 1 (PC1) protein.

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 muscle鈥痗ells to achieve precise changes in the targeted DNA sequence. Specifically, we鈥檙e鈥痳esearching鈥疌RISPR/Cas9 gene-editing technology with the goal of restoring near-full length dystrophin protein expression by鈥痶argeting鈥痗ertain鈥痬utations in the鈥痙ystrophin鈥痝ene 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
A purple icon of a muscle representing myotonic dystrophy type 1 inside of a circle with a purple border
Phase 1
Phase 1/2
Phase 2
Phase 3
Phase 4

We are investigating VX-670, an oligonucleotide approach using an intracellular Endosomal Escape Vehicle (EEV鈩) aimed at targeting the underlying cause of myotonic dystrophy type 1.

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
Phase 1
Phase 1/2
Phase 2
Phase 3
Phase 4

VX-264 is an investigational approach encapsulating cells in a protective device to be surgically implanted in the body. VX-264 is being evaluated without the use of immunosuppressive therapy as the devices are designed to shield the cells from the body's 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.听.

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.听.

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.听.

False
True
False