Company Profile
Vertex Pharmaceuticals
— A cross-modality drug developer funded by cystic-fibrosis small molecules and expanding into gene editing, stem cells, and inhaled mRNA, while outsourcing much of its manufacturing
Vertex Pharmaceuticals is a pharmaceutical company headquartered in Boston, U.S.A., with a product sales of 119,7 million in 2025.TRIKAFTA/KAFTRIOis occupied (cal ating this article). CRISPR The euticsThe world's first CRISPR/Cas9 gene editing therapy CASGEVYapproved in the United States. The current product is composed of pancreatic cells (type 1 diabetes), andererna.Inhalation mRNA therapyCo-product with Entrada, which has technology to deliver nucleic acid to the core of cells, and protein drugs obtained by the acquisition of Alpine are arranged. In this article, the size of the company is concise,What do you have for each modality?Filter by
- Bartex in 30 seconds and standing position in drug discovery and DDS
- About logo and company name
- Where is this company in the world of drug discovery and DDS?
- Company Size (Concise)
- Drug discovery and DDS resources
- Past Initiatives
- Future plans
- What is DDS?
- Collaboration mapping of drug discovery and DDS
- Technologies, Products and Services
- The risk of this company
- Glossary/Source/Ins ment and Source Response Table
1. Bartex in 30 seconds and standing position in drug discovery and DDS
Boston50 Northern Avenue
(Up to sales)The characteristics of pharmaceutical companies
Bartex departs from the main body that causes certain diseasesChoose your modalitiesPharmaceutical company 10-K explains the strategy ‘common of human psychology that causes the disease and the advances in science of the means of treatment’Candidates of multiple modalities simultaneously clinicallyI wrote FactsHome Cystic fibrosis for patients who cannot treat low- drugsInhalation mRNA therapy (VX-522)Moderna In sickle red blood cell diseaseCASGEVY to regenerate and restore the patient's own cells outside the bodyOn the other hand, most of the products are outsourced and third parties produce Cas9 proteins and guide RNAs used for CASGEVY. "Research and sales are the combination of our own and manufacturing with outside"This is the starting point when you see this company in drug discovery and DDS.
2. Logo and company name
Bartex Japan Ltd. is a trademark of the company. In this article, we do not reproduce the generated image,
Logo image on the company's official websiteHome img/vertex_logo.png Contact Us 182×96 pixels on the official website.
Note: Corporate name Vertex Pharmaceuticals IncorporatedHome CASGEVY CRISPR Therapeutics is a Swiss-based company, not a subsidiary of Vertex. Product name (TRIKAFTA, KAFTRIO in Europe)
Official Site:https://www.vrtx.com/3. Where is this company in the world of drug discovery and DDS?
Most of the sales areLowPhekta/Kaftrio, Alyftrek, etc. FactsHome Now,Intracellular gene-edited cell therapy（CASGEVY）、Pancreatic cells from stem cells(DiRe, VX-017),Inhalation mRNA（VX-522）、 Oligonucleotides delivered in cellsVX-670Protein Medicine(Duplex inhibition of Pavetacinate, BAFF and APRIL) is added. The mechanisms of each technology are handled in the technical commentary of this series: Drug discovery modalities, mRNA, nucleic acid drugs, CAR-T cells, and CDMO.
4. Company Scope (Concise)
Press only the scale of the company. The amount is US dollars (million), and the year is the calendar year. Facts。
| Index | FY2024 | FY2025 | January to June 2026 |
|---|---|---|---|
| Total sales (US$ million) | 11,020.1 | 12,001.3 | 6,320.8 |
| KAFTA/KAFTRIO | 10,238.6 | 10,312.7 | 4,851.9 |
| ALYFTREK | ― | 837.8 | 998.0 |
| CASGEVY | 10.0 | 115.8 | 119.3 |
| JOURNAVX | ― | 59.6 | 78.6 |
| R&D expenses (US$ million) | 3,630.3 | 3,909.5 | 1,955.4 |
| Costs of acquired products (acquired IPR&D) | 4,628.4 | 133.0 | ― |
| Capital investment (acquiring tangible fixed assets) | 297.7 | 437.6 | ― |
The acquisition of Alpine Immune Sciences "-" indicates that there is no value of the same category in the document confirmed in this article.
The 2025 Sales Forecast for KAFTRIO/HAFTAabout86.2% The calculation of this paperHome CASGEVY of genetic editing did not meet 1% of sales, and 64 people were actually given in 2025 Facts。 Low drugs that are installed on the company's scaleThe new modality is still not a scale of business. Research and development costs for modalities are not disclosed Undetermined / Future。
5. Drug discovery and DDS resources
| Resource Types | ||
|---|---|---|
| Home | Approx. 6,400 employees Non-dis sure of R&D | Facts |
| Location | Lease of office and research center in Boston. 2.3 million square feet in Massachusettsproduction equipmentLeiden II (approximately 34.8 million sq. ft.) near the head office will be launched in late 2026. | Facts |
| Gold (R&D) | R&D expenses US$3,909.5 million (2025). Approx. 32.6% of total sales | Facts |
| Gold (Manufacturing Equipment) | Capital investment US$437.6 million (2025). On the basis of the production of biologics and cell and gene therapy, “Co e investing in large amounts of resources” and 10-K | Facts |
| Production | Boston KAFTRIO Cell Therapy Production Capacity Development and Commercial Supply Planning for CASGEVY | Facts |
| Manufacture of outsour | Most of the commercial supply and considerable parts of clinical supply depend on third parties. CASGEVY’s Cas9 protein, guide RNA, and povetacinate are also manufactured by third parties. Type 1 Diabetes Cell Therapy Strategic Agreement with Lonza | Facts |
| Concert (FY2017, CF asset), Semma (FY2019, stem cell-derived pancreatic island), Alpine (FY2024, $50 billion), Crinetics (FY2026, $100 billion) | Facts | |
| Contact Us | CRISPR The。eutics CASGEVY’s profit is 60%, CRISPR 40%),ererna(2016, CF mRNA), Entrada (2022, cell delivery) | Facts |
| Collaboration with universities and research institutions | 10-K is generally described as "Cooperate with universities, government laboratories, foundations, etc.", and does not include individual opponents and amounts | Not confirmed |
| Public Funds | Acceptance of government research grants related to drug discovery and DDS is not listed in the primary document confirmed | Not confirmed |
6. Past Initiatives
| Time | Home | The meaning of drug discovery and DDS |
|---|---|---|
| Year 1989 | Established in Massachusetts | ― |
| Year 2015 | Joint Research Initiated with CRISPR Technology | Entering genetic editing |
| FY2016 | Moderna and CF mRNA treatment | Development of mRNA to code CFTR |
| Year 2017 | Co-development and Sales Agreement with CRISPR (Revised in 2021 and led by our company to develop, manufacture and sell). Get CF assets from Concert | System to commercialize gene editing treatment |
| Year 2019 | Semma Therapeutics | Entered type 1 diabetes with stem cell-derived pancreatic cells |
| 2022 year | Partner with Entrada | Technology that efficiently delivers oligonucleotides to the cells for mus dy phy type 1 |
| December 8, 2023 | FDACASGEVYApproval of sickle red blood cell disease (over 12 years old) | First CRISPR Therapy Approved in the United States |
| 2024 views | Alpine Immune Sciencesacquires $50 billion. FDA Approved ALYFTREK | To kidney disease with protein medicine (povetaside) |
| January 30, 2025 | FDAJOURNAVXApprove with moderate to severe acute pain (US/New approval) | NaV1.8 Selectively Inhibits Oral Low Blood Pressure |
| March 2025 | VX-264 enclosed in a device that protects pancreatic cells from immunity does not achieve validity evaluation items and disco e development | DDS-type approach |
| September 1, 2026 | Crinetics PharmaceuticalsCompleted acquisition of shares ($100 billion) | Adds oral low- endocrine drugs |
Major clinical trials (new modalities)
| Test name (NCT number) | Phase | Main evaluation items | Contact |
|---|---|---|---|
| VX-522（NCT05668741） | Phase 1/2. CF patients with CFTR protein. Inhalation mRNA | H)dous events up to 8 weeks (safety and tolerance) | RECRUIT Repetitive dose part data disclosure targets late 2026 Undetermined / Future |
| Dimithre (VX-880)・VX-017(NCT04786262) | Phase 1/2/3. Type 1 diabetes | Insulin-free and severe hypo)emia (part B・C), safety | 10-K temporary postponement of administration for in-house analysis of manufacturing. 10-Q is registered and administered. Revised Schedule will be presented during 2026 Undetermined / Future |
| RAINIER | Phase 3. IgA renal disease, placebo contrast | (Interim analysis for quick approval) | FDA receives fast approval applications. November 30, 2026 Undetermined / Future |
The test information is based on the company’s legal disclosure and the content of ClinicalTrials.gov. The efficacy value does not appear because it does not include the scope of the primary document. This article does not recommend a specific drug, but does not inferior to other drugs. Please contact your doctor for treatment.
7. Future plans
November 30, 2026Assuming self-injection subcutaneous administration once every four weeks
Data Disclosure TargetPhase 1/2 iteration part
Early 2027A1Re Renal Disease, Assuming Rapid Approval
In addition, Phase 3 in Diabetic peri al neuro of szetryzine (to be registered until the end of 2026), Phase 1/2 of next-generation CFTR regulator (VX-828, VX-581), pancreatic cell VX-017 that can be used for blood type is scheduled to start FactsHome Both approval periods Undetermined / Future Comment
None of the following documents have been listed Undetermined / Future。
VX-522Formulation (lipids and other components of mRNA)/CASGEVYCompany name and ability/ MoreR&D expenses/Research & DevelopmentNumber of employees/Manufacturing analysis of dimethyltheone。
8. What is this company aiming for drug discovery and DDS?
This includes parts that the company does not directly state.Calculation in the range that can be read from primary informationand write with the basis.
| Read | Primary Information | |
|---|---|---|
| CFRemaining patients who cannot be cured by low genetic sCover | "Research mRNA and gene editing for those who cannot make CFTR protein" and 10-K. CRISPR acquires CF rights | Facts |
| Cell TherapySpeed of productionWhen it comes to the company itself | T postponement for manufacturing analysis, investment in CASGEVY manufacturing process improvement and ensuring capacity, manufacturing contract with Lonza | |
| In type 1 diabetes, it is not enclosed on the deviceImproving immunity suppression and cell modificationContact Us | Disco、ation of VX-264 (enclosed type), improvement of immunity suppression and research of low immunogenic cells (acquisition of rights from CRISPR) | |
| to lower CF dependenciesIncrease area by buying late assets | Acquisition of Alpine and Crinetics Explanation of the company’s acquisition of Crinetics “addition to s and accelerate sales growth” | Facts |
| InhalationDeliver mRNA to lungs with local administrationbet on | VX-522 is a nebulizer-type mRNA treatment and description that creates a CFTR that acts on lung cells |
10-K indicates that the production of biologics and cell and gene therapy is more complex than low、s and requires separate systems, equipment, and expertise, and there is no single process available for all, and it is necessary to create each program. FactsHome In fact, dimithre is temporarily suspended for in-house analysis of the production of clinical trials, and CASGEVY is investing in improving the manufacturing process and ensuring its ability, 1-type diabetes cell therapy is formed with Lonza. A company with its own continuous production equipment in low、s.Steps to determine the success of your businessRead this article Undetermined / Future。
9. Collaboration mapping of drug discovery and DDS
10. Technologies, Products and Services
| Technology | Typical Products and Initiatives | Stage |
|---|---|---|
| Low) (CFTR regulator) | 。KAFTA/KAFTRIO, ALYFTREK, KALYDECO, etc. Next Generation VX-828・VX-581 | Approved / Clinical Trial |
| Low genetic s (pain relievers, kidneys, endocrine systems) | JAVNAX (NaV1.8 inhibition), INAKISAPRINE (A2A inhibition), PALSONIFY · MERMENTO (from Crinetics) | Approved/Phase 2/3 |
| Cell therapy for extracellular gene editing | CASGEVY Edit and return BCL11A gene introns in CRISPR/Cas9. How to use a virus vector | Approved (December 8, 2023) |
| Cell therapy from stem cells | Dimithre , VX-017 (Design without blood type) | Phase 1/2/3/Phase 1/2 |
| Inhalation mRNA | VX-522 | Phase 1/2 |
| Intracellular delivery of oligonucleotide | VX-670 Muscle Strength Dy phy Type 1) | Clinical Development |
| Protein Medicine | Double inhibition of BAFF and APRIL. A small amount is injected at home once every 4 weeks | Undergraduate |
Bartex is about 21.3 million square feet in Massachusettsproduction equipmentKAFTA FactsHome Manufacturing is a method to continue to produce raw materials and finish them into tablets, maintaining a constant supply, mixing, and tableting of solventsManagement of material properties (fluidity and particle size)is the element that determines the quality. Non-dis sure of the company’s process details.
In contrast, CASGEVY purifies and edits cells collected by patients at manufacturing sites,Freeze and send to the treatment facilityProcess FactsHome This is the material of Cas9 protein and guide RNA, cell culture reagents, cryopreservation and transportation produced by third parties. VX-264 was canceled in 2025.Enclosed on devices that protect from immunityIt was a method. Biological conformity and material permeability of enclosed materials is a field that remains as a difficult task in the DDS of cell therapy (introductory).
11. Risks that this company has
| Risks | ||
|---|---|---|
| Focus on CF drugs | Approximately 86% of product sales by PRODUCT KAFTRA/KAFTRIO (2025) | Facts |
| Dependency on external manufacturing | Most of the commercial supply depends on a third party. Including contract manufacturers based in China and listed in 10-K | Facts |
| The difficulty of manufacturing cells and gene therapy | The process differs depending on the program and cannot use a single process. Dimithre temporarily postponed for manufacturing analysis | Facts |
| CASGEVY delay | In 2025, about 300 people started treatment and 64 people received the treatment. Accompaniment of certification treatment facilities, collection, manufacturing, and transportation | Facts |
| New modalities failure | E ted VX-264 can not achieve validity evaluation items | Facts |
| Integration of large acquisitions | Integration of Crinetics (approximately $100 billion) and borrowings for that purpose |
12. Glossary
- Cystic fibrosis (CF)
- The mutations of the CFTR gene, the mucus of the lungs, etc. increases the viscous.
- CFTR Regulator
- A low- drug that corrects the shape and working of mutated CFTR protein.
- CRISPR/Cas9
- A technology that guides Cas9 proteins to the desired location in the guide RNA and cuts and edits DNA.
- Ex vivo gene editing
- How to remove cells from the patient and edit them outside the body and return them to the body.
- Hematopoietic Stem Cell (HSPC)
- A cell that is also intended to make cells of blood. CASGEVY
- Pancreatic cells
- A collection of cells that make insulin by the panc . Lost in type 1 diabetes.
- Nebulizer
- A device that inhales the chemical liquid into a fog.
- Origonucleotide
- A short chain that connects dozens of nucleic acids. Nucleic acid drugs
- production
- Manufacturing method to finish products while continuously flowing raw materials. Comparison with b production.
- Quick approval
- US FDA system. Quickly approve in alternative evaluation items and check clinical benefits later.
- acquired IPR&D
- The cost of the drug being acquired and introduced is calculated as a cost.
- Form 10-K／10-Q
- Annual reports and quarterly reports submitted by U.S. listed companies to SEC.
Concept diagram of AI generation13. Source
- Vertex Pharmaceuticals Incorporated Form 10-K for the) year ended December 31, 2025 Business overview, strategy, product and development, CASGEVY manufacturing process and 2025 results, manufacturing system and outsour , contract with Lonza, acquisition (Concert, Semma, Alpine), contract with CRISPR,ererna, Entrada, sales by product, R&D, capital investment, number of employees, base. https://www.sec.gov/Archives/edgar/data/875320/000087532026000056/vrtx-20251231.htm
- Vertex Pharmaceuticals Incorporated Form 10-Q for the quarterly period ended June 30, 2026 Agree to acquisition of Crinetics, scheduled for Inakisapur Gimisre・ VX-017, the product sales and R&D expenses, the judgement goal of Povetacicept. https://www.sec.gov/Archives/edgar/data/875320/000087532026000259/vrtx-20260630.htm
- Vertex Pharmaceuticals・CRISPR Therapeutics FDA Approval of CASGEVY (December 8, 2023) https://news.vrtx.com/news-releases/news-release-details/vertex-and-crispr-therapeutics-announce-us-fda-approval
- Vertex Pharmaceuticals Vertex Announces FDA Approval of JOURNAVX (suzetrigine) https://news.vrtx.com/news-releases/news-release-details/vertex-announces-fda-approval-journavxtm-suzetrigine-first-class
- Vertex Pharmaceuticals Vertex Completes Acquisition of Crinetics Pharmaceuticals https://news.vrtx.com/news-releases/news-release-details/vertex-completes-acquisition-crinetics-pharmaceuticals-and
- ClinicalTrials.gov NCT05668741(VX-522 Phase 1/2). Target, number of registrations and major evaluation items. https://clinicaltrials.gov/study/NCT05668741
- ClinicalTrials.gov NCT04786262(VX-880)・VX-017) Phase, Key Evaluation Items. https://clinicaltrials.gov/study/NCT04786262
- Vertex Pharmaceuticals Official website (for confirmation of company profile and logo) https://www.vrtx.com/
14. Response table of claims and sources
| Content | ||
|---|---|---|
| Established in 1989, head office location, listed market, and 6,400 employees | Facts | Source 1 https://www.sec.gov/Archives/edgar/data/875320/000087532026000056/vrtx-20251231.htm |
| Total sales, product sales, R&D expenses, acquired IPR & D investment | Facts | Source 1 https://www.sec.gov/Archives/edgar/data/875320/000087532026000056/vrtx-20251231.htm |
| KAKAFTA/KAFTRIO is about 86.2% of product sales and about 32.6% of total R&D costs | The calculation of this paper | )ulation of this paper (cal)d from the number of source 1) https://www.sec.gov/Archives/edgar/data/875320/000087532026000056/vrtx-20251231.htm |
| Sales and R&D expenses for January to June 2026 | Facts | Source 2 https://www.sec.gov/Archives/edgar/data/875320/000087532026000259/vrtx-20260630.htm |
| Researching mRNA and genetic editing in strategy (origin point, multiple modalities) and CF | Facts | Source 1 https://www.sec.gov/Archives/edgar/data/875320/000087532026000056/vrtx-20251231.htm |
| CASGEVY manufacturing process, external production of Cas9 and guide RNA, and the number of people in 2025 | Facts | Source 1 https://www.sec.gov/Archives/edgar/data/875320/000087532026000056/vrtx-20251231.htm |
| Production System (In-house low- manufacturing, continuous production equipment, cell therapy manufacturing capacity, outsour , contract with Lonza), Base and Leiden II | Facts | Source 1 https://www.sec.gov/Archives/edgar/data/875320/000087532026000056/vrtx-20251231.htm |
| CRISPR (2015, 2017, 2021, 60:40),cererna (2016), Entrada (2022), Concert, Semma, and Alpine | Facts | Source 1 https://www.sec.gov/Archives/edgar/data/875320/000087532026000056/vrtx-20251231.htm |
| Discovery of VX-264 (discovered in March 2025), temporary postponement of dimethyltheone | Facts | Source 1 https://www.sec.gov/Archives/edgar/data/875320/000087532026000056/vrtx-20251231.htm |
| Agree to the acquisition of Crinetics, the status of Zimiresel VX-017, the appointment of Inakisaprin on the date of review of Poveta Silent | Facts | Source 2 https://www.sec.gov/Archives/edgar/data/875320/000087532026000259/vrtx-20260630.htm |
| CASGEVY USA Approval (December 8, 2023) | Facts | Source 3 https://news.vrtx.com/news-releases/news-release-details/vertex-and-crispr-therapeutics-announce-us-fda-approval |
| JOURNAVX USA Approval (January 30, 2025) | Facts | Source 4 https://news.vrtx.com/news-releases/news-release-details/vertex-announces-fda-approval-journavxtm-suzetrigine-first-class |
| Completion of Acquisition of Crinetics (September 1, 2026) | Facts | Source 5 https://news.vrtx.com/news-releases/news-release-details/vertex-completes-acquisition-crinetics-pharmaceuticals-and |
| VX-522 | Facts | Source 6・7 https://clinicaltrials.gov/study/NCT05668741 |
| Positioning of three types of drug discovery and DDS, as well as two types | The calculation of this paper | This article does not specify the company name. The company name is an example from the series of companies in this article. |
| VX-522 formulation, CASGEVY contract destination, modalities R&D expenses, R&D personnel, production analysis | Undetermined / Future | Not specified in the primary document. Don't guess https://www.sec.gov/Archives/edgar/data/875320/000087532026000056/vrtx-20251231.htm |
| Individual collaborative research with universities and receipt of public funds | Undetermined / Future | No individual description in Source 1. This article does not break even if it is not present https://www.sec.gov/Archives/edgar/data/875320/000087532026000056/vrtx-20251231.htm |
| Explanation of biocompatible properties of continuous production and enclosed materials | Undetermined / Future | It is a general technical commentary, not disclosure of the company's process |
| Cell therapy reads that manufacturing capacity becomes the norm, and reads that how to make it is the stage of determining the success of business | Interpre of this paper from the description of the production of source 1 | |
| Type 1 diabetes reads improvement of immunity suppression and cell modification rather than enclosed | Disco ed VX-264 of Source 1 and interpretation of this paper from CRISPR contract | |
| Reads to bet on local administration by inhalation, and integrated risk of large acquisitions | Interpre of this article from Source 1・2・5 |
Last Updated:September 26, 2026/Troy Technical
All figures in this document are based on the legal disclosure document submitted by Bartex to the US Securities and Exchange Commission (SEC), the official release of the company, and the information provided by ClinicalTrials.gov. We do not use estimates and media-based numbers of survey companies. The "、ulation" section is an interpretation that the article reads from the primary information and is not an announcement of the company. This article is not medical advice, but is not recommended to use certain drugs.