COMPANY PROFILE / iPS CELLS & REGENERATIVE MEDICINE
CRISPR Therapeutics
A company that owns the genome-editing "tool" and is reaching for iPS-cell-derived islet cells edited to hide from the immune system
CRISPR Therapeutics is a genome-editing company, not an iPS cell company. Together with Vertex it brought the world's first approved CRISPR therapy, CASGEVY, to market, and the two split the profits and losses 40:60. What matters in this field is CTX213 (preclinical), islet cells made from iPS cells edited at six sites. Rather than relying on a device, the idea is to edit the cells themselves so that they hide from the immune system, and the company's 10-K says so explicitly. This profile concentrates on what the company invests in, who it works with and what it is aiming for.
- CRISPR Therapeutics 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
- 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. CRISPR Therapeutics in 30 seconds, and where it stands in iPS cells and regenerative medicine
Boston, U.S. (research)Research is based in the U.S. The manufacturing site is in Framingham, Massachusetts
iPS cell & cell therapy (secondary)One preclinical iPS cell programme, for type 1 diabetes
(both outside and inside the body)Plus iPS-cell-derived islet cells that have been edited
(preclinical)Islet cells made from edited iPS cells. Given without a device
CRISPR Therapeutics is a genome-editing company through and through. It holds rights to use part of the core CRISPR/Cas9 patents and, with Vertex, brought the world's first approved CRISPR therapy, CASGEVY, to market Sourced. Vertex, the large drugmaker, handles sales, and CRISPR Therapeutics shares 40% of the profits and losses.
Its link to iPS cells is CTX213, islet cells made from iPS cells in which six genes have been edited (preclinical) Sourced. In its 10-K the company says that every stem-cell-derived islet candidate from the companies working on them requires long-term immunosuppression, and it aims to get past that by making cells that hide from the immune system through gene editing. No clinical trial has started yet, and this is not the core of the business.
2. The logo and how the name is written
A note on names: the legal entity is CRISPR Therapeutics AG (AG, short for Aktiengesellschaft, is the Swiss form of a joint-stock company). "CRISPR" is both the name of a technology and part of the company's name, so this article writes "CRISPR/Cas9" for the technology and "CRISPR Therapeutics" for the company. Vertex Pharmaceuticals, which sells CASGEVY, is a separate company (it has its own profile in this series).
Official site: https://crisprtx.com/3. Where the company sits in iPS cells and regenerative medicine
| Therapeutic approach | Where CRISPR Therapeutics 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, Canada, Switzerland and parts of the Middle East. In the U.S., for ages 2 and older | Sourced |
| Genome editing (directly inside the body) | CTX310 (ANGPTL3), CTX340, CTX460, CTX321. Delivered to the liver in lipid nanoparticles | CTX310 is in Phase 1b, CTX340 and CTX460 in Phase 1, and CTX321 is preclinical | Sourced |
| Allogeneic cell therapy (genome-edited CAR-T) | zugo-cel (formerly CTX112). T cells from healthy donors edited at five sites | Phase 1/2. Autoimmune diseases and B-cell malignancies. RMAT designation | Sourced |
| iPS-cell-derived cells | CTX213 (progenitor islet cells derived from edited iPS cells) | Preclinical. The Phase 1 trial of its predecessor, CTX211, ended in August 2025 | Sourced |
| Gene delivery (AAV, LNP and similar) | LNPs (lipid nanoparticles) carry the editing payload for in vivo editing. No AAV product is described in this field | LNP programmes are in the clinic | Sourced |
| Mesenchymal stem cells and tissue engineering | Not described (the predecessor, CTX211, used a cell-containing device) | Could not be confirmed | Not yet confirmed |
Our explainers on genome editing, off-target effects and safety assessment, lipid nanoparticles, MHC and universal donors, genome-editing therapies for blood disorders, and cell therapy for diabetes give the background to each row of the table.
4. Company scale
Figures are in U.S. dollars and the fiscal year ends in December. CRISPR Therapeutics is a development-stage company with almost no revenue, so we look at the scale of its expenses and cash Sourced.
| Measure (US$ millions) | 2024 | 2025 | First half of 2026 |
|---|---|---|---|
| Total revenue | 37.3 | 3.5 | 11.6 |
| Research and development expense | 310.2 | 284.8 | 135.7 |
| In-process R&D (acquisitions and licences) | - | 96.3 | 2.5 |
| Collaboration expense, net | 120.7 | 213.5 | 86.2 |
| Net loss | 366.3 | 581.6 | 214.1 |
| Cash and marketable securities (period end) | 1,903.8 | 1,975.8 | 2,364.4 |
"Collaboration expense, net" is the share CRISPR Therapeutics bears after CASGEVY's costs and revenues are split under its contract with Vertex. It rose in 2025 from the previous year, which the 10-K explains by the end of the cost deferral that had been allowed up to 2024. In the first half of 2026 it fell because the company's share of CASGEVY sales grew Sourced.
We could not confirm the number of employees from the body text of the 10-K that we used Not yet confirmed. One reason given for lower R&D expense in 2025 was "a US$25.8 million decrease in personnel costs from reduced headcount", which shows the company has been trimming its size Sourced. The size of total revenue says nothing about the strength of the business; this is a company whose development is supported by a thick cushion of cash.
5. Resources committed to iPS cells and regenerative medicine
No figures are disclosed for iPS cells and regenerative medicine alone. We show the parts of the company's overall resources that bear on this field.
| Resource | Details | Category |
|---|---|---|
| People (company-wide) | The number of employees could not be confirmed in the body of the 10-K. Personnel costs fell in 2025 because of reduced headcount | Could not be confirmed / Sourced |
| People (iPS cells and regenerative medicine) | The staff assigned to CTX213 are not disclosed | Could not be confirmed |
| Sites | Zug (headquarters). A research building of about 263,500 sq ft in Boston (leased to October 2034). A cell therapy manufacturing building of about 50,249 sq ft in Framingham, Massachusetts (leased to March 2036). A laboratory in San Francisco as well | Sourced |
| Money (R&D expense) | US$284.8 million company-wide (2025). The part for regenerative medicine is not disclosed | Sourced / could not be confirmed |
| Money (cash) | US$2,364.4 million (30 June 2026). The company says it covers more than 24 months of operating expenses and capital expenditure | Sourced |
| Investments and acquisitions | Collaboration with Sirius Therapeutics (May 2025; in-process R&D of US$96.3 million). This is in siRNA and separate from regenerative medicine | Sourced |
| In-licensing | Exclusive licence to the core patents from Dr. Charpentier (2014). The University of California and the University of Vienna share the rights | Sourced |
| Out-licensing | To Vertex: US$75.0 million upfront and a US$30.0 million equity investment in 2015, US$30.0 million on option exercise in 2019, a US$900 million upfront payment in 2021, and a US$200.0 million milestone in January-March 2024. For the non-exclusive type 1 diabetes licence (2023), US$100.0 million upfront plus US$105.0 million in milestones received in total, with up to a further US$125.0 million | Sourced |
| Universities, research institutes and public funding | The universities that share the patent rights (above). The 10-K mentions two grants for HIV gene editing, but we could not confirm the source or amount. No university collaboration on iPS cells is described | Sourced / could not be confirmed |
What stands out in this table is that CRISPR Therapeutics is on the receiving end rather than the paying end. Simply adding up the amounts received from Vertex shown above (US$75.0 million in 2015, US$30.0 million in 2019, US$900.0 million in 2021, US$200.0 million in 2024, and the type 1 diabetes licence's US$100.0 million upfront and US$105.0 million milestones) gives US$1,410.0 million Our calculation (the US$30.0 million equity investment is not included; the sum is ours). This is the foundation of its thick cash cushion.
6. Track record so far
| When | What happened | What it means |
|---|---|---|
| April 2014 | Exclusive licence agreement with Dr. Charpentier for the core CRISPR/Cas9 patents | Secured the rights to the editing tool |
| 2015 | Research collaboration agreement with Vertex (US$75.0 million upfront, US$30.0 million equity investment) | Found a partner with funds and development capability |
| December 2017 | Joint development agreement with Vertex. Treatments for sickle cell disease and beta thalassemia to be developed and sold jointly | The contract that became the basis of CASGEVY |
| End of 2018 | Starts its first clinical trial | Entered the clinical stage |
| 2021 | Vertex agreement amended (US$900 million upfront; CASGEVY profits and losses now CRISPR 40%, Vertex 60%). Diabetes co-development agreement with ViaCyte | Handed the lead on development, manufacturing and sales to Vertex |
| 2022 | Vertex acquires ViaCyte | The diabetes partner changed |
| January 2023 | Phase 1 trial of VCTX211 (which we take to be what later became CTX211) begins (ClinicalTrials.gov start date: 20 January 2023) | An attempt to transplant edited stem-cell-derived islet cells inside a device |
| March 2023 | Non-exclusive licence to Vertex for diabetes (US$100.0 million upfront). ViaCyte agreement amended | Licensed the editing rights to Vertex as well |
| 8 December 2023 | The FDA approves CASGEVY for sickle cell disease (the first FDA-approved therapy to use CRISPR/Cas9). US$200.0 million milestone received from Vertex in January-March 2024 | Approval translated directly into revenue |
| February 2024 | ViaCyte withdraws from the co-development agreement. CRISPR Therapeutics takes full ownership of the programmes in progress (with a royalty on future sales to ViaCyte) | Laid the ground for pursuing iPS-cell-derived islet cells on its own |
| May 2025 | Collaboration with Sirius Therapeutics (siRNA; in-process R&D of US$96.3 million recorded) | Directed funds to an area unrelated to regenerative medicine |
| August 2025 | The VCTX211 Phase 1 trial ends (per ClinicalTrials.gov; 5 enrolled) | The device-based trial came to a close |
| November 2025 | Phase 1 results for CTX310 (in vivo liver editing) published in NEJM | Human data for in vivo editing became available |
| October-December 2025 | ToolGen files suit, claiming that CASGEVY infringes its patents | The patent fights go on |
| July 2026 | The FDA extends CASGEVY's approval to ages 2 and older | The approved population widened by age |
The 10-K says that in the Phase 1 trial of the predecessor, CTX211, persistent C-peptide production 12 months after transplant, and survival of the transplanted islet cells in tissue, were confirmed Sourced. It also says there was fibrosis of the device and infiltration by immune cells. That point leads into the "no device" approach described next.
7. Plans ahead
UK and Saudi ArabiaThe FDA already extended approval to ages 2 and older in July 2026
(moving to Phase 2)Rests on positive data through December 2025
Of the items in the table, only two bear directly on iPS cells and regenerative medicine: CTX213 and the further milestones from Vertex. The rest are plans to widen the genome-editing technology base Sourced. There is no guarantee that any of this goes to plan, and the outlook is Not yet confirmed.
None of the following appears in the material we used Not yet confirmed.
When the clinical trial of CTX213 will start / how many people it will enrol / its contract manufacturer / when the further milestones from Vertex will arise / the source of the iPS cell line used for CTX213. Dates and amounts 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 |
|---|---|---|
| The core of its cell therapy is "editing to hide from the immune system" | The 10-K states that stem-cell-derived islet cells all require chronic immunosuppression and that it aims to protect cells through editing. CTX213 is edited at six sites: deletion of B2M and TXNIP, and insertion of PD-L1, HLA-E, MANF and A20 | Sourced |
| For islet cells, it has shifted from a device-based approach to a device-free one | It records fibrosis and immune-cell infiltration with the CTX211 device and says its next candidate, CTX213, is "device-free". No reason is stated | Inference |
| It intends to run its iPS-cell type 1 diabetes work in parallel with Vertex | The licence to Vertex is non-exclusive, and CRISPR Therapeutics keeps its own CTX213. If Vertex makes progress, milestones also come in | Inference |
| It is shifting its weight to in vivo editing | Several in vivo programmes are in the clinic (CTX310 in Phase 1b, CTX340 and CTX460 in Phase 1). Regenerative medicine is still preclinical, and R&D expense was also cut in 2025 | Inference |
| The cost burden of CASGEVY is heavy | Cost deferral ended from 2025, and collaboration expense, net rose from US$120.7 million to US$213.5 million. The company says costs will exceed its share of revenue for the time being | Sourced |
We read the move to a device-free approach as significant from a materials point of view too Inference. Putting cells in a membrane or device keeps them physically apart from the immune system, but as the 10-K says, once fibrosis forms around the device, oxygen and nutrients struggle to get through Sourced. If instead the cells themselves are edited so the immune system cannot find them, the "materials problem" of the device can be replaced by a "genetic problem" of cell design. That is how we read the thinking behind CTX213. The more edits are made, however, the more safety endpoints have to be assessed. CTX213 is still preclinical and has no human results Not yet confirmed.
9. Partnership map for cell and gene therapy
The thing to notice in this map is that the partnerships are concentrated on a single company, Vertex. The 10-K itself lists as a risk that Vertex has significant control over CASGEVY Sourced. For islet cells, the only partnerships we could confirm in primary sources were those with Vertex and ViaCyte.
10. Technologies and products on offer
| Product | How it works | Target | Status |
|---|---|---|---|
| CASGEVY | 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). Non-viral. Vertex leads development, manufacturing and sales | Sickle cell disease, transfusion-dependent beta thalassemia | Approved (in the U.S., ages 2 and older) |
| CTX213 | Immature islet cells made from iPS cells edited at six genes. Given directly, without a device | Type 1 diabetes | Preclinical (the company states that improved blood glucose and C-peptide production were seen in rats) |
| CTX211 (VCTX211) | Edited stem-cell-derived islet cells, transplanted together with a cell-containing device | Type 1 diabetes | Phase 1 ended in August 2025 |
| zugo-cel (CTX112) | Allogeneic CAR-T made from healthy donors' T cells edited at five sites | Autoimmune diseases, B-cell malignancies | Phase 1/2. RMAT designation (FDA) |
| CTX310 and others | Cas9 mRNA and guide RNA packaged in LNPs and infused intravenously to edit a gene in the liver | Hypercholesterolemia and others | Phase 1b |
| SyNTase | A new gene-correction method that combines a compact Cas9 with a modified enzyme | - (platform technology) | Research stage |
Trial results (figures from papers or the 10-K, with comparator and time point)
| Trial | Population and design | Result | Time point and caveats |
|---|---|---|---|
| CTX211 Phase 1 (as described in the 10-K) | Type 1 diabetes. A cell-containing device was implanted | Persistent C-peptide production 12 months after transplant, and survival of the transplanted islet cells in tissue | Fibrosis of the device and infiltration by immune cells. Participant numbers and individual values are not given in the 10-K |
| CTX310 Phase 1 (NEJM) | Adults with hypercholesterolemia and other conditions. Dose escalation. 15 people received a single dose (followed for 60 days or more) | Blood ANGPTL3 fell by a mean of 79.7% at 0.7 mg/kg and 73.2% at 0.8 mg/kg (no decrease at 0.1 and 0.3 mg/kg, where the mean rose slightly) | No comparison group. One person died suddenly 179 days after dosing, and another had a herniated disc. No dose-limiting toxicity |
| CASGEVY, sickle cell disease (NEJM, with Vertex) | 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 |
CTX310, the in vivo editing programme, works by packaging Cas9 mRNA and guide RNA in lipid nanoparticles (LNPs) and infusing them Sourced. For this approach, the lipid composition, the mixing process that keeps particle size uniform, and the synthetic purity of the mRNA and guide RNA determine quality. The 10-K says the company has its own LNP delivery platform, which shows that materials and manufacturing know-how are a core asset Sourced.
For CTX213, the iPS-cell programme, the key is whether a cell line edited at six sites can be expanded reliably. Quality control of a repeatedly edited cell line (no unintended cuts, no change in properties when scaled up) and the reproducibility of culture medium, scaffold and cryopreservation medium are likely to be the barriers to volume production Inference. As for the cell-containing device that proved a problem with the predecessor, CTX211, the challenge was how the membrane material interacts with fibrosis around the cells Sourced.
11. The risks this company carries
| Risk | Details | Category |
|---|---|---|
| Dependence on Vertex | The 10-K lists as a risk that Vertex has significant control over CASGEVY. The partner holds the initiative on sales and manufacturing | Sourced |
| Costs exceed revenue | The company states that its CASGEVY cost burden will exceed its share of revenue for the time being. Net loss for 2025 was US$581.6 million | Sourced |
| Patents | The core patents are shared among Dr. Charpentier, the University of California and the University of Vienna, so CRISPR Therapeutics is not in a position to hold them exclusively on its own. Patent fights (interferences) between the CVC group, the Broad Institute side and others continue. In the fourth quarter of 2025, ToolGen filed suit over CASGEVY | Sourced |
| iPS-cell-derived islet cells are preclinical | CTX213 has not yet been given to a person and no timing is published. The predecessor device-based approach (CTX211) had its trial end in August 2025 | Sourced |
| Safety of in vivo editing | In the CTX310 Phase 1 trial, one person died suddenly 179 days after dosing (reported in the paper). An edit made inside the body cannot be reversed | Sourced |
| Use of funds | The US$2,364.4 million of cash at the end of June 2026 is a thick cushion, but the net loss for the first half alone was US$214.1 million | Sourced |
| Burden of conditioning (CASGEVY) | The company states that CASGEVY's safety profile is generally consistent with autologous stem cell transplant and myeloablative conditioning. Research on lighter conditioning is preclinical | Sourced |
12. Glossary
- CRISPR/Cas9
- A tool that cuts DNA at a chosen spot. A guide RNA specifies the location and Cas9 makes the cut.
- Guide RNA (gRNA)
- The RNA that tells Cas9 where to cut.
- Ex vivo / in vivo editing
- Ex vivo means cells are taken out, edited and returned. In vivo means a drug is infused and edits genes directly inside the body.
- LNP (lipid nanoparticle)
- A tiny particle made of lipids that wraps nucleic acids and delivers them into cells.
- SyNTase
- A new gene-correction method under development at CRISPR Therapeutics.
- Allogeneic CAR-T
- A cell therapy in which a healthy donor's T cells are given a receptor that targets cancer or other cells, so that one batch can be used for many patients.
- Hypoimmune (low immunogenicity)
- Cells edited so that the immune system has a harder time detecting them. The aim is to avoid rejection after transplant.
- C-peptide
- A substance made together with insulin. It is used as a marker of whether transplanted islet cells are working.
- Preclinical
- Research before a drug is given to people: experiments in animals or cells.
- Milestone
- A lump-sum payment from a partner when development reaches a set stage.
13. References
- CRISPR Therapeutics Form 10-K for the fiscal year ended December 31, 2025 (filed with the U.S. SEC on 12 February 2026). Business overview, terms of the Vertex agreements, regenerative medicine (CTX213 and CTX211), patents, sites and financials. https://www.sec.gov/Archives/edgar/data/1674416/000119312526048957/crsp-20251231.htm
- CRISPR Therapeutics Form 10-Q for the quarterly period ended June 30, 2026 (filed with the U.S. SEC on 3 August 2026). First-half 2026 financials, the expanded approval of CASGEVY to ages 2 and older, and the current status of each programme. https://www.sec.gov/Archives/edgar/data/1674416/000119312526330672/crsp-20260630.htm
- Vertex Pharmaceuticals Form 10-K for the fiscal year ended December 31, 2025 (filed with the U.S. SEC). Used to check the split of CASGEVY profits and losses (Vertex 60%, CRISPR 40%) and the payments under the type 1 diabetes licence with CRISPR. 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). Applications for CASGEVY in ages 5 to 11 in the UK and Saudi Arabia. https://www.sec.gov/Archives/edgar/data/875320/000087532026000259/vrtx-20260630.htm
- U.S. FDA (CBER) Approved product page for CASGEVY (exagamglogene autotemcel). Carries approval letters including one dated 8 December 2023. 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 NCT05565248, a Phase 1 study evaluating the VCTX211 combination product in type 1 diabetes (sponsor: CRISPR Therapeutics AG). Start date 20 January 2023, end date 8 August 2025, 5 enrolled. https://clinicaltrials.gov/study/NCT05565248
- N Engl J Med (indexed in PubMed) Laffin LJ et al., "Phase 1 Trial of CRISPR-Cas9 Gene Editing Targeting ANGPTL3," N Engl J Med 2025;393 (issue of 27 November 2025). Phase 1 trial of CTX310. https://pubmed.ncbi.nlm.nih.gov/41211945/
- N Engl J Med (indexed in PubMed) Frangoul H et al., "Exagamglogene Autotemcel for Severe Sickle Cell Disease," N Engl J Med 2024;390:1649-1662. Single-arm Phase 3 trial in ages 12 to 35. https://pubmed.ncbi.nlm.nih.gov/38661449/
- CRISPR Therapeutics Official website (used to check the company overview and the logo). https://crisprtx.com/
14. Claim-to-source audit
| Claim in the article | Category | Source |
|---|---|---|
| Legal name (CRISPR Therapeutics AG), Zug headquarters, Boston research building, Nasdaq: CRSP | Sourced | References 1 and 10 https://www.sec.gov/Archives/edgar/data/1674416/000119312526048957/crsp-20251231.htm https://crisprtx.com/ |
| Total revenue, R&D expense, net loss and cash (2024 and 2025) | Sourced | Reference 1 https://www.sec.gov/Archives/edgar/data/1674416/000119312526048957/crsp-20251231.htm |
| First-half 2026 total revenue, R&D expense, net loss and cash (US$2,364.4 million), and cash for more than 24 months of operations | Sourced | Reference 2 https://www.sec.gov/Archives/edgar/data/1674416/000119312526330672/crsp-20260630.htm |
| The rise in collaboration expense, net (end of the deferral cap) and its fall in 2026 (a larger share of CASGEVY sales) | Sourced | References 1 and 2 https://www.sec.gov/Archives/edgar/data/1674416/000119312526048957/crsp-20251231.htm https://www.sec.gov/Archives/edgar/data/1674416/000119312526330672/crsp-20260630.htm |
| Lower personnel costs in 2025 (reduced headcount). Number of employees not stated in the body text | Sourced | Reference 1 https://www.sec.gov/Archives/edgar/data/1674416/000119312526048957/crsp-20251231.htm |
| Boston building of about 263,500 sq ft and Framingham cell therapy manufacturing building of about 50,249 sq ft | Sourced | Reference 1 https://www.sec.gov/Archives/edgar/data/1674416/000119312526048957/crsp-20251231.htm |
| How CASGEVY works, countries and regions of approval (U.S., EU, UK, Canada, Switzerland, Middle East), expansion to ages 2 and older in the U.S. in July 2026 | Sourced | References 1 and 2 https://www.sec.gov/Archives/edgar/data/1674416/000119312526048957/crsp-20251231.htm https://www.sec.gov/Archives/edgar/data/1674416/000119312526330672/crsp-20260630.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 |
| The Vertex agreements (US$75.0 million and a US$30.0 million equity investment in 2015, US$30.0 million in 2019, US$900 million in 2021, US$200.0 million in 2024; 40:60 profit and loss split) | Sourced | References 1 and 3 https://www.sec.gov/Archives/edgar/data/1674416/000119312526048957/crsp-20251231.htm https://www.sec.gov/Archives/edgar/data/875320/000087532026000056/vrtx-20251231.htm |
| Non-exclusive type 1 diabetes licence (US$100.0 million upfront, US$105.0 million milestones received, up to a further US$125.0 million, royalty in the low to mid single digits) | Sourced | References 1 and 3 https://www.sec.gov/Archives/edgar/data/1674416/000119312526048957/crsp-20251231.htm https://www.sec.gov/Archives/edgar/data/875320/000087532026000056/vrtx-20251231.htm |
| The ViaCyte co-development agreement, Vertex's acquisition (2022), the February 2024 withdrawal and CRISPR Therapeutics' full ownership of the assets | Sourced | Reference 1 https://www.sec.gov/Archives/edgar/data/1674416/000119312526048957/crsp-20251231.htm |
| CTX213 (progenitor islet cells from edited iPS cells, six edits, preclinical) | Sourced | References 1 and 2 https://www.sec.gov/Archives/edgar/data/1674416/000119312526048957/crsp-20251231.htm https://www.sec.gov/Archives/edgar/data/1674416/000119312526330672/crsp-20260630.htm |
| CTX211 Phase 1: persistent C-peptide production and cell survival, device fibrosis and immune-cell infiltration | Sourced | Reference 1 https://www.sec.gov/Archives/edgar/data/1674416/000119312526048957/crsp-20251231.htm |
| VCTX211 Phase 1 (start 20 January 2023, end 8 August 2025, 5 enrolled) | Sourced | Reference 7 https://clinicaltrials.gov/study/NCT05565248 |
| Exclusive licence from Dr. Charpentier (2014), rights shared with the University of California and the University of Vienna, and the shared, non-exclusive nature of the patents | Sourced | Reference 1 https://www.sec.gov/Archives/edgar/data/1674416/000119312526048957/crsp-20251231.htm |
| ToolGen's lawsuit (fourth quarter of 2025) | Sourced | Reference 1 https://www.sec.gov/Archives/edgar/data/1674416/000119312526048957/crsp-20251231.htm |
| CTX310 Phase 1 (15 people, mean ANGPTL3 reductions of 79.7% and 73.2%, one sudden death) and the LNP approach | Sourced | References 8 and 1 https://pubmed.ncbi.nlm.nih.gov/41211945/ https://www.sec.gov/Archives/edgar/data/1674416/000119312526048957/crsp-20251231.htm |
| CTX310 in Phase 1b, CTX340 and CTX460 in Phase 1, CTX321 preclinical | Sourced | Reference 2 https://www.sec.gov/Archives/edgar/data/1674416/000119312526330672/crsp-20260630.htm |
| zugo-cel (five edits, Phase 1/2, RMAT designation, combination study and clinical supply with Eli Lilly) | Sourced | References 1 and 2 https://www.sec.gov/Archives/edgar/data/1674416/000119312526048957/crsp-20251231.htm https://www.sec.gov/Archives/edgar/data/1674416/000119312526330672/crsp-20260630.htm |
| Phase 3 results in sickle cell disease (29 of 30 evaluable, median 19.3 months) | Sourced | Reference 9 https://pubmed.ncbi.nlm.nih.gov/38661449/ |
| The risk statement that Vertex has significant control over CASGEVY, and the continuing excess of costs over revenue | Sourced | Reference 1 https://www.sec.gov/Archives/edgar/data/1674416/000119312526048957/crsp-20251231.htm |
| The company's statement that CASGEVY's safety profile is generally consistent with autologous stem cell transplant and myeloablative conditioning | Sourced | Reference 2 https://www.sec.gov/Archives/edgar/data/1674416/000119312526330672/crsp-20260630.htm |
| CASGEVY applications for ages 5 to 11 under review in the UK and Saudi Arabia (applications complete) | Sourced | Reference 4 https://www.sec.gov/Archives/edgar/data/875320/000087532026000259/vrtx-20260630.htm |
| Collaboration with Sirius Therapeutics (May 2025, in-process R&D of US$96.3 million) | Sourced | Reference 1 https://www.sec.gov/Archives/edgar/data/1674416/000119312526048957/crsp-20251231.htm |
| Total received from Vertex (US$1,410.0 million) | Our calculation | Reference 1 https://www.sec.gov/Archives/edgar/data/1674416/000119312526048957/crsp-20251231.htm Addition performed in this article (2015, 2019, 2021, 2024 and the 2023 diabetes licence) |
| 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 |
| Staff numbers, R&D expense and capital expenditure for iPS cells and regenerative medicine alone | Not yet confirmed | Reference 1 https://www.sec.gov/Archives/edgar/data/1674416/000119312526048957/crsp-20251231.htm Not stated in the 10-K. This article does not estimate them |
| CTX213 trial start date, number of participants, contract manufacturer, and the source of the iPS cell line | Not yet confirmed | References 1 and 2 https://www.sec.gov/Archives/edgar/data/1674416/000119312526048957/crsp-20251231.htm https://www.sec.gov/Archives/edgar/data/1674416/000119312526330672/crsp-20260630.htm Not stated in the 10-K or 10-Q |
| Source and amount of the two grants for HIV gene editing | Not yet confirmed | Reference 1 https://www.sec.gov/Archives/edgar/data/1674416/000119312526048957/crsp-20251231.htm The 10-K gives only the number of grants. Source and amount could not be confirmed |
| The reading that VCTX211 and CTX211 are the same product | Inference | References 1 and 7 https://www.sec.gov/Archives/edgar/data/1674416/000119312526048957/crsp-20251231.htm https://clinicaltrials.gov/study/NCT05565248 The 10-K does not say they are identical. This article's interpretation |
| The reading that it has moved from a device-based to a device-free approach | Inference | Reference 1 https://www.sec.gov/Archives/edgar/data/1674416/000119312526048957/crsp-20251231.htm The 10-K gives no reason. This article's interpretation |
| The reading that it intends to run its type 1 diabetes work in parallel with Vertex | Inference | Reference 1 https://www.sec.gov/Archives/edgar/data/1674416/000119312526048957/crsp-20251231.htm This article's interpretation |
| The reading that it is shifting its weight to in vivo editing | Inference | References 1 and 2 https://www.sec.gov/Archives/edgar/data/1674416/000119312526048957/crsp-20251231.htm https://www.sec.gov/Archives/edgar/data/1674416/000119312526330672/crsp-20260630.htm This article's interpretation |
| The reading that quality control of a cell line edited at six sites will be the barrier to volume production | Inference | This article's interpretation (from a materials and process viewpoint) |
Last updated 1 October 2026 / Troy Technical
Every figure in this article comes from the statutory filings CRISPR Therapeutics and Vertex have made with the U.S. Securities and Exchange Commission (SEC), 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.