COMPANY PROFILE / iPS CELLS & REGENERATIVE MEDICINE
Vertex Pharmaceuticals
A large drugmaker redirecting its cystic fibrosis profits into genome editing and stem-cell-derived cell therapy
Vertex is not an iPS cell company. It appears in this field for two reasons: it sells CASGEVY, a cell therapy edited with CRISPR/Cas9, and it is running late-stage trials of zimislecel, a therapy that transplants islet cells grown from donor-derived stem cells into people with type 1 diabetes. On 1 July 2026 the U.S. FDA extended the approval of CASGEVY to patients aged 2 and older. This profile keeps the company-wide picture to a minimum and concentrates on what Vertex invests in cell and gene therapy, who it works with and what it is aiming for.
What this article covers
- Vertex in 30 seconds, and where it stands in iPS cells and regenerative medicine
- The logo and how the name is written
- Where the company sits in iPS cells and regenerative medicine
- Company scale (kept brief for a large drugmaker)
- Resources committed to iPS cells and regenerative medicine
- Track record so far
- Plans ahead
- What the company is aiming for
- Partnership map for cell and gene therapy
- Technologies and products on offer
- The risks this company carries
- Glossary, references and claim-to-source audit
1. Vertex in 30 seconds, and where it stands in iPS cells and regenerative medicine
LEGAL NAMEVertex Pharmaceuticals IncorporatedA Massachusetts corporation. No official Japanese-language rendering of the name could be confirmed
HEADQUARTERSBoston,
Massachusetts, U.S.50 Northern Avenue (cover of the 10-K)
Massachusetts, U.S.50 Northern Avenue (cover of the 10-K)
LISTINGNasdaq: VRTXA U.S.-listed company
CATEGORY IN THIS FIELDLarge pharma (main)
Genome-editing products (secondary)The editing tools are licensed in from partners
Genome-editing products (secondary)The editing tools are licensed in from partners
HOW THE THERAPIES WORKGenome editing (autologous)
Stem-cell-derived islet cells (allogeneic)Use of iPS cells could not be confirmed
Stem-cell-derived islet cells (allogeneic)Use of iPS cells could not be confirmed
APPROVED PRODUCTCASGEVYExpanded in the U.S. to ages 2 and older on 1 July 2026
CASGEVY SALES (2025)US$115.8 millionTotal revenue that year was US$12.0 billion (about 1%)
CELL THERAPIES IN DEVELOPMENTzimislecel (Phase 1/2/3)
VX-017 (Phase 1/2 planned)Type 1 diabetes. Approval timing not confirmed
VX-017 (Phase 1/2 planned)Type 1 diabetes. Approval timing not confirmed
The one thing to grasp first
Vertex is a large drugmaker that booked US$12.0 billion in total revenue in 2025, mostly from small-molecule drugs for cystic fibrosis (CF); it is not an iPS cell company. It still cannot be left out of this field, for two reasons: it sells a genome-edited therapy that has won regulatory approval (CASGEVY), and it is running late-stage trials of a therapy that transplants islet cells made from donor-derived stem cells into people with type 1 diabetes (zimislecel) Sourced.
One caution. CASGEVY is an "autologous" therapy: the patient's own blood stem cells are removed, edited and returned, and no iPS cells are involved. For zimislecel the sources say only "stem-cell-derived", so we could not confirm from the material we used whether the cells come from iPS cells or embryonic stem cells Not yet confirmed. We therefore read this article not as the profile of an iPS cell company but as a case study of what happens when a large drugmaker turns genome editing and cell transplantation into products.
2. The logo and how the name is written
Vertex
A note on names: the legal entity is Vertex Pharmaceuticals Incorporated (registered with the SEC as VERTEX PHARMACEUTICALS INC / MA). We could not confirm an official Japanese-language rendering in the material we used, so this article uses the English name throughout. CRISPR Therapeutics AG, a Swiss company, is a separate company that co-developed CASGEVY; it has its own profile in this series. The logo image on the official website could not be retrieved automatically, so the name is shown as text.
Official site: https://www.vrtx.com/3. Where the company sits in iPS cells and regenerative medicine
| Therapeutic approach | Where Vertex fits | Status | Category |
|---|---|---|---|
| Genome editing (CRISPR/Cas9) | CASGEVY (the patient's own blood stem cells are edited outside the body) | Approved in the U.S., EU, UK and elsewhere. In the U.S., for ages 2 and older | Sourced |
| Genome editing (directly inside the body) | "Being investigated" for sickle cell disease and beta thalassemia | Described in the 10-K as research stage. No clinical trial could be confirmed | Sourced |
| Allogeneic cell therapy (stem-cell-derived) | zimislecel (type 1 diabetes) and VX-017 | Zimislecel is enrolling and dosing in Phase 1/2/3. VX-017 is due to start Phase 1/2 | Sourced |
| iPS-cell-derived cells | No mention of "iPS" in the sources | Could not be confirmed | Not yet confirmed |
| Gene delivery (AAV, LNP and similar) | Not described as a product in this field (the CF mRNA programme with Moderna is outside it) | Could not be confirmed | Not yet confirmed |
| Mesenchymal stem cells and tissue engineering | Not described. VX-264, an encapsulated-cell device, missed its efficacy endpoint and was stopped | Development ended | Sourced |
Our explainers on genome editing, genome-editing therapies for blood disorders, cell therapy for diabetes, and autologous versus allogeneic therapy make it easier to see what each row refers to.
4. Company scale (kept brief for a large drugmaker)
Vertex is a large drugmaker, so we limit the company-wide picture to a single table and give the cell and gene therapy side more room in the chapters that follow. Figures are in U.S. dollars and the fiscal year ends in December Sourced.
| Measure (US$ millions) | 2023 | 2024 | 2025 |
|---|---|---|---|
| Total revenue | 9,869.2 | 11,020.1 | 12,001.3 |
| of which TRIKAFTA/KAFTRIO (CF) | 8,944.7 | 10,238.6 | 10,312.7 |
| of which CASGEVY | Not confirmed in the sources used | 10.0 | 115.8 |
| Research and development expense | 3,162.9 | 3,630.3 | 3,909.5 |
| First half of 2026 (US$ millions) | January-June 2025 | January-June 2026 | April-June 2026 |
|---|---|---|---|
| Total revenue | 5,734.9 | 6,320.8 | 3,333.9 |
| of which CASGEVY | 44.6 | 119.3 | 76.4 |
Vertex had about 6,400 employees at 31 December 2025 (about 5,200 in the U.S. and about 1,200 elsewhere). It does not disclose how many work on cell and gene therapy alone.
CASGEVY accounted for about 0.96% of total revenue in 2025 (115.8 / 12,001.3) and about 1.9% in the first half of 2026 (119.3 / 6,320.8) Our calculation. TRIKAFTA/KAFTRIO alone made up about 85.9% of 2025 total revenue (10,312.7 / 12,001.3) Our calculation.
A word of caution
The figures above are for the whole company. They do not measure the size of any iPS cell or regenerative medicine business. Apart from CASGEVY, the cell and gene therapies (zimislecel and others) have no sales yet, and the share of R&D spending going to cell and gene therapy is not disclosed Not yet confirmed.
5. Resources committed to iPS cells and regenerative medicine
We look at four areas (people, money, external relationships, and universities or public funding), separating what could be confirmed from what could not.
| Resource | Details | Category |
|---|---|---|
| People (company-wide) | About 6,400 (31 December 2025). Sites include the Boston headquarters and the nearby "Jeffrey Leiden Center for Biologics, Cell and Genetic Therapies" campus | Sourced |
| People (cell and gene therapy) | The number of dedicated researchers and manufacturing staff is not disclosed | Could not be confirmed |
| Money (R&D expense) | US$3,909.5 million company-wide (2025). The part spent on cell and gene therapy is not disclosed | Sourced / could not be confirmed |
| Money (manufacturing facilities) | Cell therapy manufacturing capacity is being built up in the Boston area, and the company plans to use its own site for additional CASGEVY supply. It signed with Lonza for a dedicated facility of about 130,000 sq ft in New Hampshire, to be operated by Lonza; the lease began in January-March 2026 and a right-of-use asset of US$95.8 million was recorded | Sourced |
| Acquisitions and investments | Built its type 1 diabetes business by acquiring Semma Therapeutics (2019) and ViaCyte (2022). The purchase prices could not be confirmed in the sources used | Sourced |
| In-licensing | Joint development agreement with CRISPR Therapeutics (2017, amended in 2021). A non-exclusive licence to gene-edited, low-immunogenicity cells for type 1 diabetes (2023: US$100.0 million upfront plus a US$70.0 million research milestone, then US$35.0 million in 2024; up to a further US$125.0 million). A non-exclusive sublicence from Editas (December 2023) | Sourced |
| Contract manufacturing | Lonza (type 1 diabetes cell therapy). For CASGEVY, outside manufacturers also supply cell editing and reagents | Sourced |
| Universities and research institutes | Authors of the peer-reviewed papers include clinicians at Toronto General Hospital, the University of Pennsylvania, the University of Wisconsin, the University of Chicago and Leiden University Medical Center, among others (clinical trial sites). The 10-K names no specific university research agreements | Sourced / could not be confirmed |
| Public funding | The 10-K mentions no grants or public funding for cell and gene therapy | Could not be confirmed |
The only amounts we can pin down are the payments to CRISPR Therapeutics and the leased asset at the Lonza facility. Vertex's refusal to break out R&D by modality is the big difference from specialist companies such as CRISPR Therapeutics, covered next in this series.
6. Track record so far
| When | What happened | What it means |
|---|---|---|
| 2015 | Research collaboration agreement with CRISPR Therapeutics (CF and several other targets) | The starting point for bringing in editing technology from outside |
| 2017 | Joint development and commercialization agreement (JDCA) with CRISPR Therapeutics. Sickle cell disease and beta thalassemia developed jointly | The contract that became the basis of CASGEVY |
| 2019 | Acquired Semma Therapeutics, starting its type 1 diabetes business based on stem-cell-derived islet cells | Brought cell therapy in through an acquisition |
| 2021 | JDCA amended (CASGEVY profits and losses split 60% Vertex, 40% CRISPR). Trial of zimislecel (then VX-880) begins (ClinicalTrials.gov start date: 29 March 2021) | Vertex took the lead on development, manufacturing and sales |
| 2022 | Acquired ViaCyte | Accelerated its type 1 diabetes cell therapy |
| 2023 | Manufacturing agreement with Lonza for the type 1 diabetes cell therapy. Non-exclusive licence from CRISPR Therapeutics for gene-edited, low-immunogenicity cells. Non-exclusive sublicence from Editas in December | Strengthened both manufacturing and editing-tool rights |
| 8 December 2023 | The FDA approves CASGEVY for sickle cell disease (ages 12 and older). It is the first FDA-approved therapy to use CRISPR/Cas9 | The first genome-editing product from a large drugmaker |
| 16 January 2024 | The FDA's CASGEVY approval page carries an approval letter dated 16 January 2024 (transfusion-dependent beta thalassemia) | A second indication |
| March 2025 | VX-264, an encapsulated-cell therapy, fails to meet its efficacy endpoints in Phase 1/2 and is discontinued. US$379.0 million impairment in January-March 2025 | One route to avoiding immunosuppression hit a dead end |
| June 2025 | Interim analysis of the zimislecel Phase 1/2 trial published in the New England Journal of Medicine (NEJM) | Peer-reviewed human data became available |
| December 2025 | Announces positive results from the pivotal trials of CASGEVY in children aged 5 to 11 | The basis for extending the label to younger patients |
| February 2026 | At the time of the 10-K filing, completion of zimislecel dosing is temporarily paused (for in-house manufacturing analysis) | A manufacturing check was needed |
| 1 July 2026 | The FDA extends CASGEVY's approval to ages 2 and older (53 days after the application). About 5,500 more people in the U.S. become eligible | The approved population widened by age |
| August 2026 | The 10-Q says zimislecel is "continuing to enroll and dose". The IND (investigational new drug application) for VX-017 is accepted by the FDA | From a pause back to resuming |
| September 2026 | Results of the trials in children aged 5 to 11 published in NEJM | Peer-reviewed paediatric data |
7. Plans ahead
ZIMISLECELUpdated plan to be
announced in 2026The company's statement in the 10-Q. Approval timing not confirmed
announced in 2026The company's statement in the 10-Q. Approval timing not confirmed
VX-017Phase 1/2 to
start soonDescribed as designed to work regardless of blood type
start soonDescribed as designed to work regardless of blood type
CASGEVY (AGES 5 TO 11)Under review in the
UK and Saudi ArabiaApplications complete. Timing of decisions not confirmed
UK and Saudi ArabiaApplications complete. Timing of decisions not confirmed
IMPROVED CONDITIONING AGENTPreclinicalAims to ease the burden of clearing bone marrow and widen the eligible population
IN VIVO EDITINGResearch stageBeing investigated for sickle cell disease and beta thalassemia
LEIDEN II (NEW BUILDING)Due to open at
the end of 2026About 348,000 sq ft of research and laboratory space
the end of 2026About 348,000 sq ft of research and laboratory space
NON-CF PRODUCTS IN 2026US$500 million+ in salesThe company's outlook for CASGEVY and JOURNAVX combined
AUTHORIZED TREATMENT CENTERSMore than 75 in the U.S.Number active as of 1 July 2026
In the 10-K the company says that for CASGEVY it will "reach more eligible patients in 2026 and increase the number of people dosed through the global network of authorized treatment centers" Sourced. The "non-CF products in 2026" figure in the table is the company's own outlook Not yet confirmed.
What could not be confirmed
None of the following appears in the material we used Not yet confirmed.
When zimislecel will be submitted for approval / the expected approval date / its price / whether VX-017 uses gene editing / the price per patient for CASGEVY and the target for the number of patients dosed in 2026. Prices and patient numbers that appear in press reports cannot be verified against primary sources, so this article does not use them.
8. What the company is aiming for
This section includes things the company does not say directly. We set them out as inferences within what the primary sources support, with the evidence for each.
| What the evidence suggests | Primary information it rests on | Category |
|---|---|---|
| In type 1 diabetes, the end goal is cells that can be used without immunosuppression | The 10-K says it is exploring, at the research stage, ways to reduce or eliminate standard immunosuppression. It has also acquired rights to gene-edited, low-immunogenicity cells from CRISPR | Sourced |
| VX-017 may be a cell product that uses low-immunogenicity editing | VX-017 is described as designed to work regardless of blood type, and there is a separate agreement with CRISPR on low-immunogenicity edited cells. The sources do not say that VX-017 uses editing | Inference |
| In genome editing, the company is looking ahead to a shift from editing cells outside the body to editing directly inside it | The 10-K says it is investigating in vivo gene editing, and an agent to ease the burden of conditioning is in preclinical work | Inference |
| For cell therapy manufacturing, it is trying to secure two routes to volume: its own site and Lonza | It plans to use its Boston site for additional CASGEVY supply, and is setting up a dedicated Lonza facility for type 1 diabetes | Inference |
| A policy of not betting on a single technology but lining up several mechanisms | The 10-K says it pursues "multiple modalities", running small molecules, antibody-like molecules, mRNA and cell therapy in parallel | Sourced |
One piece of evidence in more detail
We read the "small" look of CASGEVY sales as the result of a long gap between starting treatment and being dosed Inference. According to the 10-K, in 2025 about 300 people worldwide started treatment, 147 had their first cell collection and 64 were dosed Sourced. Between collection and dosing come editing outside the body, quality checks, frozen transport and conditioning, so many of those who start treatment are not dosed within the year. In the fourth quarter of 2025 alone, 30 people were dosed, so dosing grows in the later quarters. The 78% rise in second-quarter 2026 sales (US$76.4 million) over the first quarter fits this reading Sourced. Neither the timing of revenue recognition nor the price is disclosed, however, so we cannot be sure Not yet confirmed.
9. Partnership map for cell and gene therapy
The thing to notice in this map is that Vertex owns no editing tools of its own and turns them into products through a mix of joint development and non-exclusive licences. It hands 40% of CASGEVY's profits and losses to its partner, but Vertex leads development, manufacturing and sales Sourced.
10. Technologies and products on offer
| Product | How it works | Target | Status |
|---|---|---|---|
| CASGEVY (exagamglogene autotemcel) | The patient's own blood stem cells are edited outside the body with CRISPR/Cas9 (at the erythroid-specific enhancer region of the BCL11A gene), which raises fetal hemoglobin. Non-viral. A conditioning step to clear bone marrow is given before the cells are returned | Sickle cell disease (with recurrent vaso-occlusive crises) and transfusion-dependent beta thalassemia | Approved (in the U.S., ages 2 and older) |
| zimislecel (VX-880) | Fully differentiated, insulin-producing islet cells made from donor-derived (allogeneic) stem cells. Infused into the hepatic portal vein and protected with standard immunosuppression | Type 1 diabetes (with severe hypoglycemia) | In development (Phase 1/2/3). RMAT and Fast Track (FDA), PRIME (EMA). The company estimates the initial eligible population at about 65,000 people (those who experience severe hypoglycemia) |
| VX-017 | Stem-cell-derived islet cells designed to work regardless of blood type | Type 1 diabetes | In development (IND accepted; Phase 1/2 to start) |
| VX-264 | Zimislecel enclosed in a device that shields the cells from the immune system | Type 1 diabetes | Missed its efficacy endpoint in March 2025 and was discontinued |
| Improved conditioning agent / in vivo editing | An agent to lighten the burden of clearing bone marrow / a method that edits directly inside the body | Sickle cell disease and beta thalassemia | Research and preclinical |
Trial results (figures from the paper abstracts, with comparator and time point)
| Trial | Population and design | Result | Time point and caveats |
|---|---|---|---|
| CASGEVY, sickle cell disease (NCT03745287) | Ages 12 to 35. Single-arm, open-label Phase 3. 44 people dosed | Of 30 people who could be evaluated, 29 (97%) had no severe vaso-occlusive crises for at least 12 consecutive months | Median follow-up 19.3 months. No comparison group (the null hypothesis was 50%) |
| CASGEVY, transfusion-dependent beta thalassemia (NCT03655678) | Ages 12 to 35. Single-arm, open-label Phase 3. 52 people dosed | Of 35 people who could be evaluated, 32 (91%) needed no transfusions for at least 12 months | Median follow-up 20.4 months. No comparison group |
| CASGEVY, ages 5 to 11 (NCT05356195, NCT05329649) | 15 people with beta thalassemia and 11 with sickle cell disease dosed | All 8 people in each group followed for 16 months or more were transfusion-free or free of crises. The rest had not yet reached evaluation | Grade 3 or 4 adverse events in all participants. Two people with beta thalassemia had serious liver injury linked to conditioning, and one died |
| zimislecel (NCT04786262) | Phase 1/2. 14 adults (2 in Part A, 12 in Parts B and C) followed for 12 months or more | All 12 people in Parts B and C had no severe hypoglycemia and HbA1c below 7%. 10 of the 12 (83%) were insulin-independent at day 365 | Interim analysis not prespecified; small and short. No comparison group. Two deaths (meningitis and progression of dementia) |
A materials engineer's view: what a product made of "frozen living cells" requires
According to the 10-K, CASGEVY works like this: the patient's cells are carried from the treatment centre to a manufacturing site, where the stem cells are purified and edited, then frozen, returned to the treatment centre, stored and finally infused Sourced. Because living cells are frozen and thawed, the composition of the cryopreservation medium, the container material and the temperature control of cold-chain transport bear directly on quality. The 10-K also states plainly that the company relies on outside manufacturers for cell culture reagents and for editing components such as Cas9 protein and guide RNA Sourced. From the materials side, we read this as meaning that the synthesis quality of the editing enzyme and guide RNA, and lot-to-lot variation in media and reagents, could become the rate-limiting step for supply Inference.
The other point is the kind of membrane or device that shields cells from the immune system, as in VX-264. That approach was stopped after it missed its efficacy endpoint Sourced, but the 10-K does not give the reason, and no materials-related cause has been identified Not yet confirmed. Designing a material that lets oxygen and nutrients through while keeping immune cells out remains homework for cell therapy as a whole.
11. The risks this company carries
| Risk | Details | Category |
|---|---|---|
| Dependence on CF drugs | TRIKAFTA/KAFTRIO alone account for about 85.9% of 2025 total revenue. Sales of cell and gene therapy are still small | Our calculation |
| Growth of CASGEVY sales | The 10-K says there is no assurance that CASGEVY sales can be increased or sustained. The cost of manufacturing as a share of sales is far higher than for the CF drugs | Sourced |
| Burden of conditioning | CASGEVY requires a conditioning step to clear bone marrow, and the 10-K says market acceptance depends on how severe the side effects of conditioning are. In the paediatric trial, serious liver injury linked to conditioning occurred in two people, and one died (paper abstract) | Sourced |
| Patent disputes | ToolGen claims that CASGEVY's manufacturing method infringes its patents, and the matter is in dispute in the U.S., UK and Netherlands. Fights over the basic CRISPR/Cas9 patents also continue in several jurisdictions | Sourced |
| Uncertainty in type 1 diabetes cell therapy | Zimislecel dosing completion was paused for a time, and VX-264 was stopped with a US$379.0 million impairment. Standard immunosuppression is required, and the published results are small and short | Sourced |
| Scaling up manufacturing | CASGEVY needs coordination across several sites. For zimislecel, approval of Lonza's new facility is a precondition for commercial production | Sourced |
| Allocation of resources | In July 2026 it agreed to buy Crinetics for about US$10.0 billion, completing the deal on 1 September. Large sums are also going to areas outside cell therapy | Sourced / Inference |
12. Glossary
- CRISPR/Cas9
- A tool that cuts DNA at a chosen spot. A guide RNA specifies the location and Cas9 makes the cut.
- BCL11A
- A gene that suppresses fetal hemoglobin. CASGEVY edits the part of this gene that is active in red blood cell precursors.
- Fetal hemoglobin (HbF)
- An oxygen-carrying protein made before birth. The FDA explains that higher levels prevent red blood cells from sickling.
- Myeloablative conditioning
- Using drugs before treatment to reduce the cells in the bone marrow and make room for the edited cells. It places a heavy burden on the body.
- Autologous / allogeneic
- Autologous means using the patient's own cells; allogeneic means using cells from another person. Allogeneic therapy has to deal with immune rejection.
- Islet cells
- The clusters of cells in the pancreas that make insulin. In type 1 diabetes they are destroyed.
- Immunosuppression
- Using drugs to dampen the immune system so that transplanted cells are not rejected.
- Authorized treatment center (ATC)
- An independently run medical institution that collects cells for CASGEVY and carries out the infusion.
- JDCA
- Joint development and commercialization agreement. Here, the contract between Vertex and CRISPR Therapeutics.
- RMAT
- Regenerative Medicine Advanced Therapy designation, an FDA programme that speeds up the development of regenerative medicine. One of the expedited programmes.
13. References
- Vertex Pharmaceuticals Form 10-K for the fiscal year ended December 31, 2025 (filed with the U.S. SEC on 13 February 2026). Descriptions of CASGEVY, zimislecel and VX-264, the agreements with CRISPR Therapeutics, the Semma and ViaCyte acquisitions, the Lonza agreement, headcount, revenue and R&D expense, manufacturing, and patent disputes. https://www.sec.gov/Archives/edgar/data/875320/000087532026000056/vrtx-20251231.htm
- Vertex Pharmaceuticals Form 10-Q for the quarterly period ended June 30, 2026 (filed with the U.S. SEC on 4 August 2026). CASGEVY sales for April-June and the first half of 2026, the FDA's paediatric label expansion, the current status of zimislecel and VX-017, and the start of the Lonza facility lease. https://www.sec.gov/Archives/edgar/data/875320/000087532026000259/vrtx-20260630.htm
- Vertex Pharmaceuticals Press release, "Vertex Announces US FDA Approval for Expanded Use of CASGEVY for the Treatment of People Ages 2 Years and Older" (1 July 2026). Expanded approval to ages 2 and older, and more than 75 authorized treatment centers. https://news.vrtx.com/news-releases/news-release-details/vertex-announces-us-fda-approval-expanded-use-casgevyr-treatment
- Vertex Pharmaceuticals Press release, "Vertex Reports Second Quarter 2026 Financial Results" (3 August 2026). Number of countries approving CASGEVY, 53 days from application to approval, the current status of zimislecel and VX-017, and the 2026 outlook for non-CF product sales. https://news.vrtx.com/news-releases/news-release-details/vertex-reports-second-quarter-2026-financial-results
- U.S. FDA (CBER) Approved product page for CASGEVY (exagamglogene autotemcel). Carries approval letters dated 8 December 2023, 16 January 2024, 18 March 2026, 15 June 2026 and 1 July 2026. https://www.fda.gov/vaccines-blood-biologics/casgevy
- U.S. FDA FDA News Release, "FDA Approves First Gene Therapies to Treat Patients with Sickle Cell Disease" (8 December 2023). CASGEVY is the first FDA-approved therapy to use CRISPR/Cas9. https://www.fda.gov/news-events/press-announcements/fda-approves-first-gene-therapies-treat-patients-sickle-cell-disease
- ClinicalTrials.gov NCT04786262, a study evaluating the safety, tolerability and efficacy of VX-880 and VX-017 (type 1 diabetes). Start date 29 March 2021, Phase 3, estimated enrolment of 57, infused into the hepatic portal vein. https://clinicaltrials.gov/study/NCT04786262
- ClinicalTrials.gov NCT03745287, a Phase 1/2/3 study evaluating CTX001 in severe sickle cell disease (sponsor: Vertex). The primary endpoint is freedom from severe vaso-occlusive crises for at least 12 months. https://clinicaltrials.gov/study/NCT03745287
- ClinicalTrials.gov NCT03655678, a Phase 1/2/3 study evaluating CTX001 in transfusion-dependent beta thalassemia. The primary endpoint is transfusion independence for at least 12 months. https://clinicaltrials.gov/study/NCT03655678
- N Engl J Med (indexed in PubMed) Reichman TW et al., "Stem Cell-Derived, Fully Differentiated Islets for Type 1 Diabetes," N Engl J Med 2025;393:858-868 (published online 20 June 2025). Interim analysis of the zimislecel Phase 1/2 trial. https://pubmed.ncbi.nlm.nih.gov/40544428/
- N Engl J Med (indexed in PubMed) Frangoul H et al., "Exagamglogene Autotemcel for Severe Sickle Cell Disease," N Engl J Med 2024 (issue of 9 May 2024). Single-arm Phase 3 trial in ages 12 to 35. https://pubmed.ncbi.nlm.nih.gov/38661449/
- N Engl J Med (indexed in PubMed) Locatelli F et al., "Exagamglogene Autotemcel for Transfusion-Dependent β-Thalassemia," N Engl J Med 2024 (issue of 9 May 2024). Single-arm Phase 3 trial in ages 12 to 35. https://pubmed.ncbi.nlm.nih.gov/38657265/
- N Engl J Med (indexed in PubMed) Frangoul H et al., "Exa-cel in Children with Transfusion-Dependent β-Thalassemia or Sickle Cell Disease," N Engl J Med 2026 (issue of 10 September 2026, published online 11 June 2026). Phase 3 trial in ages 5 to 11. https://pubmed.ncbi.nlm.nih.gov/42274009/
- Vertex Pharmaceuticals Press release, "Vertex Completes Acquisition of Crinetics Pharmaceuticals and Announces Expansion of Executive Leadership Team" (1 September 2026). https://news.vrtx.com/news-releases/news-release-details/vertex-completes-acquisition-crinetics-pharmaceuticals-and
- Vertex Pharmaceuticals Official website (used to check the company overview). https://www.vrtx.com/
14. Claim-to-source audit
| Claim in the article | Category | Source |
|---|---|---|
| Legal name, headquarters address, listing venue (Nasdaq: VRTX) | Sourced | References 1 and 15 https://www.sec.gov/Archives/edgar/data/875320/000087532026000056/vrtx-20251231.htm https://www.vrtx.com/ |
| Total revenue (2023 to 2025), TRIKAFTA/KAFTRIO sales, CASGEVY sales (US$10.0 million in 2024, US$115.8 million in 2025), R&D expense | Sourced | Reference 1 https://www.sec.gov/Archives/edgar/data/875320/000087532026000056/vrtx-20251231.htm |
| Total revenue and CASGEVY sales for the first half and April-June 2026 (US$119.3 million, US$76.4 million) | Sourced | References 2 and 4 https://www.sec.gov/Archives/edgar/data/875320/000087532026000259/vrtx-20260630.htm https://news.vrtx.com/news-releases/news-release-details/vertex-reports-second-quarter-2026-financial-results |
| About 6,400 employees (about 5,200 in the U.S., about 1,200 elsewhere) | Sourced | Reference 1 https://www.sec.gov/Archives/edgar/data/875320/000087532026000056/vrtx-20251231.htm |
| How CASGEVY works (non-viral, outside the body, CRISPR/Cas9, BCL11A), frozen return to the treatment centre, conditioning | Sourced | References 1 and 3 https://www.sec.gov/Archives/edgar/data/875320/000087532026000056/vrtx-20251231.htm https://news.vrtx.com/news-releases/news-release-details/vertex-announces-us-fda-approval-expanded-use-casgevyr-treatment |
| About 300 people started CASGEVY treatment in 2025, 147 had a first cell collection and 64 were dosed (30 in the fourth quarter) | Sourced | Reference 1 https://www.sec.gov/Archives/edgar/data/875320/000087532026000056/vrtx-20251231.htm |
| FDA approval of CASGEVY for sickle cell disease on 8 December 2023 (the first FDA-approved therapy to use CRISPR/Cas9) | Sourced | References 6 and 5 https://www.fda.gov/news-events/press-announcements/fda-approves-first-gene-therapies-treat-patients-sickle-cell-disease https://www.fda.gov/vaccines-blood-biologics/casgevy |
| Approval letters on the FDA's CASGEVY page dated 16 January 2024, 18 March 2026, 15 June 2026 and 1 July 2026 | Sourced | Reference 5 https://www.fda.gov/vaccines-blood-biologics/casgevy |
| FDA expansion to ages 2 and older on 1 July 2026, about 5,500 more eligible people, 53 days from application, more than 75 authorized treatment centers | Sourced | References 3, 4 and 2 https://news.vrtx.com/news-releases/news-release-details/vertex-announces-us-fda-approval-expanded-use-casgevyr-treatment https://news.vrtx.com/news-releases/news-release-details/vertex-reports-second-quarter-2026-financial-results https://www.sec.gov/Archives/edgar/data/875320/000087532026000259/vrtx-20260630.htm |
| Countries and regions approving CASGEVY (U.S., EU, UK, Saudi Arabia, Bahrain, Qatar, UAE, Kuwait, Switzerland, Canada) and the statement that it is approved in 39 countries worldwide | Sourced | References 1 and 4 https://www.sec.gov/Archives/edgar/data/875320/000087532026000056/vrtx-20251231.htm https://news.vrtx.com/news-releases/news-release-details/vertex-reports-second-quarter-2026-financial-results |
| Phase 3 results in sickle cell disease (29 of 30 evaluable, median 19.3 months) | Sourced | References 11 and 8 https://pubmed.ncbi.nlm.nih.gov/38661449/ https://clinicaltrials.gov/study/NCT03745287 |
| Phase 3 results in transfusion-dependent beta thalassemia (32 of 35 evaluable, median 20.4 months) | Sourced | References 12 and 9 https://pubmed.ncbi.nlm.nih.gov/38657265/ https://clinicaltrials.gov/study/NCT03655678 |
| Results in ages 5 to 11 (15 and 11 dosed; all 8 in each group followed for 16 months or more transfusion-free or crisis-free; serious liver injury in 2 people with beta thalassemia, 1 death) | Sourced | Reference 13 https://pubmed.ncbi.nlm.nih.gov/42274009/ |
| Zimislecel is an allogeneic, stem-cell-derived, fully differentiated islet cell product used with standard immunosuppression, enrolling and dosing in Phase 1/2/3 | Sourced | References 1, 2 and 7 https://www.sec.gov/Archives/edgar/data/875320/000087532026000056/vrtx-20251231.htm https://www.sec.gov/Archives/edgar/data/875320/000087532026000259/vrtx-20260630.htm https://clinicaltrials.gov/study/NCT04786262 |
| Zimislecel interim analysis (14 people followed for 12 months or more; all 12 free of severe hypoglycemia with HbA1c below 7%; 10 insulin-independent; 2 deaths) | Sourced | Reference 10 https://pubmed.ncbi.nlm.nih.gov/40544428/ |
| Temporary pause in completing zimislecel dosing (10-K) and continued enrolment and dosing (10-Q, August 2026) | Sourced | References 1, 2 and 4 https://www.sec.gov/Archives/edgar/data/875320/000087532026000056/vrtx-20251231.htm https://www.sec.gov/Archives/edgar/data/875320/000087532026000259/vrtx-20260630.htm https://news.vrtx.com/news-releases/news-release-details/vertex-reports-second-quarter-2026-financial-results |
| Zimislecel's RMAT, Fast Track, PRIME and other designations, and the company's estimate of about 65,000 people in the initial eligible population | Sourced | Reference 1 https://www.sec.gov/Archives/edgar/data/875320/000087532026000056/vrtx-20251231.htm |
| VX-017 IND accepted, Phase 1/2 to start soon, updated plan to be announced in 2026 | Sourced | References 2, 4 and 7 https://www.sec.gov/Archives/edgar/data/875320/000087532026000259/vrtx-20260630.htm https://news.vrtx.com/news-releases/news-release-details/vertex-reports-second-quarter-2026-financial-results https://clinicaltrials.gov/study/NCT04786262 |
| VX-264 missed its efficacy endpoint, was discontinued, US$379.0 million impairment | Sourced | Reference 1 https://www.sec.gov/Archives/edgar/data/875320/000087532026000056/vrtx-20251231.htm |
| Joint development agreement with CRISPR Therapeutics (2017, amended 2021, CASGEVY profits and losses split 60:40) | Sourced | Reference 1 https://www.sec.gov/Archives/edgar/data/875320/000087532026000056/vrtx-20251231.htm |
| Non-exclusive CRISPR licence for type 1 diabetes (US$100.0 million upfront, US$70.0 million and US$35.0 million milestones, up to a further US$125.0 million) | Sourced | Reference 1 https://www.sec.gov/Archives/edgar/data/875320/000087532026000056/vrtx-20251231.htm |
| Non-exclusive sublicence from Editas (December 2023) | Sourced | Reference 1 https://www.sec.gov/Archives/edgar/data/875320/000087532026000056/vrtx-20251231.htm |
| Acquisitions of Semma (2019) and ViaCyte (2022) | Sourced | Reference 1 https://www.sec.gov/Archives/edgar/data/875320/000087532026000056/vrtx-20251231.htm |
| Lonza agreement, the dedicated facility of about 130,000 sq ft in New Hampshire, lease start and the US$95.8 million right-of-use asset | Sourced | References 1 and 2 https://www.sec.gov/Archives/edgar/data/875320/000087532026000056/vrtx-20251231.htm https://www.sec.gov/Archives/edgar/data/875320/000087532026000259/vrtx-20260630.htm |
| Plan to use the Boston site for additional CASGEVY supply, and reliance on outside manufacturers for Cas9 and guide RNA | Sourced | Reference 1 https://www.sec.gov/Archives/edgar/data/875320/000087532026000056/vrtx-20251231.htm |
| Improved conditioning agent (preclinical), investigation of in vivo editing, Leiden II (due to open at the end of 2026) | Sourced | Reference 1 https://www.sec.gov/Archives/edgar/data/875320/000087532026000056/vrtx-20251231.htm |
| Patent dispute with ToolGen (U.S., UK, Netherlands) | Sourced | Reference 1 https://www.sec.gov/Archives/edgar/data/875320/000087532026000056/vrtx-20251231.htm |
| Agreement to acquire Crinetics (about US$10.0 billion) | Sourced | References 2 and 14 https://www.sec.gov/Archives/edgar/data/875320/000087532026000259/vrtx-20260630.htm https://news.vrtx.com/news-releases/news-release-details/vertex-completes-acquisition-crinetics-pharmaceuticals-and |
| CASGEVY's share of total revenue (about 0.96% and about 1.9%) and TRIKAFTA/KAFTRIO's share (about 85.9%) | Our calculation | References 1 and 2 https://www.sec.gov/Archives/edgar/data/875320/000087532026000056/vrtx-20251231.htm https://www.sec.gov/Archives/edgar/data/875320/000087532026000259/vrtx-20260630.htm Division performed in this article |
| The three types of company (large pharma and strategic partners / genome editing and gene therapy / iPS cell and cell therapy) | Our calculation | This article's own classification. Example companies are drawn from those covered in this series |
| Use of iPS cells, AAV or LNP gene delivery in this field, mesenchymal stem cells | Not yet confirmed | Reference 1 https://www.sec.gov/Archives/edgar/data/875320/000087532026000056/vrtx-20251231.htm Not stated in the 10-K. This article does not conclude that they do not exist either |
| Number of staff dedicated to cell and gene therapy, breakdown of R&D expense, grants and public funding | Not yet confirmed | Reference 1 https://www.sec.gov/Archives/edgar/data/875320/000087532026000056/vrtx-20251231.htm Not stated in the 10-K |
| Timing of a zimislecel filing, its price, whether VX-017 uses editing, CASGEVY's price and the 2026 target for patients dosed | Not yet confirmed | Reference 2 https://www.sec.gov/Archives/edgar/data/875320/000087532026000259/vrtx-20260630.htm Not stated in the 10-Q or the earnings release. Figures from press reports are not used |
| Whether zimislecel's cells are derived from iPS cells or embryonic stem cells | Not yet confirmed | References 1 and 10 https://www.sec.gov/Archives/edgar/data/875320/000087532026000056/vrtx-20251231.htm https://pubmed.ncbi.nlm.nih.gov/40544428/ The 10-K and the paper abstract say only "stem-cell-derived" |
| VX-017 may use low-immunogenicity editing | Inference | This article's interpretation (reading the blood-type-independent design together with the CRISPR agreement) |
| The reading that the end goal is cells usable without immunosuppression | Inference | Reference 1 https://www.sec.gov/Archives/edgar/data/875320/000087532026000056/vrtx-20251231.htm This article's interpretation of the 10-K's research-stage description |
| The reading that it is looking ahead from editing outside the body to editing inside it | Inference | Reference 1 https://www.sec.gov/Archives/edgar/data/875320/000087532026000056/vrtx-20251231.htm This article's interpretation of the investigation of in vivo editing and the preclinical conditioning agent |
| The reading that cell therapy manufacturing runs on two tracks, its own site and Lonza | Inference | Reference 1 https://www.sec.gov/Archives/edgar/data/875320/000087532026000056/vrtx-20251231.htm This article's interpretation |
| The reading that CASGEVY sales look small because of the long gap between collection and dosing | Inference | References 1 and 2 https://www.sec.gov/Archives/edgar/data/875320/000087532026000056/vrtx-20251231.htm https://www.sec.gov/Archives/edgar/data/875320/000087532026000259/vrtx-20260630.htm This article's interpretation. Timing of revenue recognition is not disclosed |
| The reading that the quality of Cas9, guide RNA and reagents could limit supply, and that the Crinetics acquisition affects resource allocation | Inference | This article's interpretation (from a materials viewpoint) |
Last updated 1 October 2026 / Troy Technical
Every figure in this article comes from the statutory filings Vertex has made with the U.S. Securities and Exchange Commission (SEC), the company's official press releases, public information from the U.S. FDA, ClinicalTrials.gov and peer-reviewed papers. No research-firm estimates or press-based figures have been used. Passages marked "Inference" are this article's interpretation of the primary sources, not statements made by the company. This article summarises a company's activities; it does not recommend any treatment and is not medical advice.