COMPANY PROFILE / iPS CELLS & REGENERATIVE MEDICINE
Novartis
A major drugmaker that actually sells CAR-T (Kymriah) and AAV gene therapy (Zolgensma). Its next pillar is a new CAR-T for autoimmune disease
Novartis is not an iPS cell company. It is, however, one of the few large pharmaceutical companies selling both cell therapy and gene therapy as products around the world. Its net sales in 2025 were about US$54.5 billion, of which the Zolgensma gene therapy (spinal muscular atrophy) brought in US$1.232 billion and the Kymriah CAR-T cell therapy US$381 million. In its Form 20-F the company names "cell and gene therapy" as one of three advanced platforms, alongside radioligand therapy and xRNA. The cell therapy in development is a CD19 CAR-T for autoimmune disease (rapcabtagene autoleucel, YTB323), with several Phase 2 studies under way. The term "iPS cell", on the other hand, does not appear in the 20-F. This article keeps the company-wide picture brief and focuses on its cell and gene therapy assets and investments. It is not a recommendation of any treatment, only an organisation of the company's disclosures.
- Novartis 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 the iPS cell and regenerative medicine field
- Scale
- Resources committed to cell and gene therapy
- What it has done so far
- What comes next
- What the company is aiming for
- Map of relationships
- Technologies, products and services on offer
- The risks this company carries
- Glossary, references and claim-to-source audit
1. Novartis in 30 seconds, and where it stands in iPS cells and regenerative medicine
NYSE: NVS (ADS)75,267 employees (FTE, end of 2025)
Autologous CAR-T (Kymriah)The 20-F does not mention iPS cells
Kymriah US$381 millionTotal net sales: about US$54.5 billion
(YTB323)A CD19 CAR-T. Phase 2 in autoimmune disease
(AAV, October 2024)US$427 million in cash plus milestones of up to US$700 million
What sets Novartis apart among the large companies in this field is that it has cell and gene therapies that are already selling Sourced. The momentum, though, points in opposite directions. In 2025 Kymriah sales fell 14% (which the company attributes to competition) while the Zolgensma franchise was flat (the company says the intravenous version already reaches a high share of new patients, leaving little room for growth). The company is therefore shifting its weight to Itvisma, the intrathecal version (approved in the U.S. and the UAE in the fourth quarter of 2025), and a new CAR-T for autoimmune disease. It is a front-runner in cell and gene therapy, then, but its sales there are not growing, and the success of its next moves is the point to watch. For more, see our explainers on CAR-T cells, viral vectors, and autologous versus allogeneic cells.
2. The logo and how the name is written
A note on names: the legal entity is Novartis AG (a Swiss stock corporation). This article covers the Swiss parent company, not its Japanese subsidiary. In the restructuring since 2023 the company separated its generics business, Sandoz, and the 20-F now describes Novartis as focused on innovative medicines. Zolgensma is a product obtained through the acquisition of AveXis (20-F).
Official site: https://www.novartis.com/3. Where the company sits in the iPS cell and regenerative medicine field
Novartis is placed mainly on the left of Fig. 1 (large pharma & strategic partners), with the middle (genome editing & gene therapy) highlighted as secondary. Its mechanisms of treatment break down as follows Sourced.
| Mechanism | What Novartis has | Status |
|---|---|---|
| Gene delivery with AAV | Zolgensma (intravenous), Itvisma (intrathecal). Preclinical products from Kate Therapeutics (muscle and heart disease) | Two products on sale or approved. Kate's are preclinical |
| Autologous CAR-T | Kymriah (on sale), rapcabtagene autoleucel / YTB323 (CD19, in development) | Kymriah sales are falling. YTB323 is in Phase 2 for autoimmune disease |
| Genome editing (CRISPR, base editing, prime editing) | - | Not described in the 20-F |
| iPS cell-derived cells | - | Not described in the 20-F |
| xRNA (a separate advanced platform) | Agreed in October 2025 to acquire Avidity Biosciences (RNA medicines) | Not yet completed at the time of the 20-F. A separate category from cell and gene therapy |
4. Scale
Novartis is a large pharmaceutical company, so the whole-company picture is limited to one table. Amounts are in US$ million (IFRS) on a continuing-operations basis, and the fiscal year ends in December Sourced.
| Measure (US$ million) | 2023 | 2024 | 2025 |
|---|---|---|---|
| Net sales (continuing operations) | - | - | about 54,500 (up 8% on the previous year) |
| R&D expenses | - | 10,022 | 11,200 |
| Operating income (continuing operations) | - | 14,544 | 17,644 |
| Zolgensma franchise sales | - | - | 1,232 (up 1% on the previous year) |
| Kymriah sales | 508 | 443 | 381 |
| Employees (FTE, end of 2025) | - | - | 75,267 (of whom 20,015 in R&D) |
The figures above are for the company as a whole. Of the US$11.2 billion of R&D expenses, the amount spent on cell and gene therapy is not disclosed. The number of researchers and manufacturing staff in cell and gene therapy is not disclosed either Not yet confirmed. The Zolgensma franchise and Kymriah together (about US$1.6 billion) come to about 3% of net sales Our calculation ((1,232 + 381) / about 54,500).
5. Resources committed to cell and gene therapy
| Resource | What it is | Category |
|---|---|---|
| People (sites and staff) | 75,267 staff company-wide (FTE), 20,015 of them in R&D. The 20-F names research sites such as East Hanover in the U.S., but the number of staff dedicated to cell and gene therapy is not disclosed | Sourced / dedicated headcount not confirmed |
| Money (manufacturing facilities) | Cell and gene therapy is manufactured at Stein in Switzerland (sterile products, drug substance, and cell and gene therapy) and at Durham, North Carolina (commercial production of Zolgensma). The company says it is expanding cell culture and radioligand therapy capacity. Investment in cell and gene therapy is not disclosed | Sourced / amount not confirmed |
| Outside investment and acquisitions | Kate Therapeutics (31 October 2024: US$427 million in cash, contingent consideration with a fair value of US$91 million, total consideration with a fair value of US$518 million, and milestones of up to US$700 million). Avidity (RNA medicines, about US$12 billion, agreed in October 2025, not completed at the time of the 20-F) | Sourced |
| In-licensing and collaborations | Only a general statement that it works closely with outside researchers, biotechnology companies and universities. No individual cell and gene therapy collaboration is described in the 20-F | Not confirmed |
| Universities, research institutions and public funding | Apart from the general statement above, nothing is described individually. ClinicalTrials.gov registers a study that uses YTB323 and is sponsored by Stanford University (NCT07443137, due to start in September 2026) | Not confirmed (20-F) |
Kate Therapeutics is a preclinical-stage AAV gene therapy company. The purchase consideration was allocated to in-process R&D (IPR&D) of US$135 million, a "scientific platform" of US$135 million and goodwill of US$284 million Sourced. The "up to US$700 million" in milestones is a ceiling that applies if development advances, not a confirmed payment. The high share of goodwill suggests that the deal was less about a specific medicine than about acquiring a technology platform and a team Not yet confirmed.
6. What it has done so far
| When | What happened | Why it matters |
|---|---|---|
| (Date not given in the 20-F) | Acquires AveXis (the origin of Zolgensma) | Obtains an AAV gene therapy product |
| June 2019 | Start of the Phase 1/2 study of rapcabtagene autoleucel (YTB323) (NCT03960840), in CLL, DLBCL, ALL and first-line high-risk LBCL | Begins as a CD19 CAR-T for tumours |
| February 2023 | Start of the YTB323 study in autoimmune disease (severe refractory SLE, NCT05798117) | CAR-T moves into immune disease |
| September 2024 to June 2025 | Studies start one after another in SLE and lupus nephritis (Phase 2, NCT06581198), systemic sclerosis (NCT06655896), myositis (NCT06665256), ANCA-associated vasculitis (NCT06868290) and rheumatoid arthritis (NCT07048197) | The range of indications widens |
| 31 October 2024 | Acquires Kate Therapeutics (AAV for muscle and heart disease) | Strengthens its AAV technology platform |
| 2025 | Kymriah sales are US$381 million (down 14%). The Zolgensma franchise is flat | The existing cell and gene therapies are stalling |
| Fourth quarter of 2025 | Itvisma (intrathecal) is approved in the U.S. and the UAE, for adults and children aged 2 and over with SMA (with a confirmed SMN1 mutation) | Extends Zolgensma to older patients |
| 25 October 2025 | Agrees to acquire Avidity Biosciences (US$72 per share, about US$12 billion) | Strengthens RNA medicines. A pillar separate from cell and gene therapy |
A caution on reading CAR-T results
Most of the YTB323 autoimmune studies on ClinicalTrials.gov are open-label, early-stage or Phase 2 studies. Novartis's 20-F gives no figures for these clinical results. This article does not discuss efficacy. Please consult a physician about any individual treatment choice.
7. What comes next
The "main development projects" table in the 20-F lists the planned filing year and current phase of each project. For YTB323 it reads as follows Sourced.
| YTB323 target | What the 20-F says (year development began / planned filing and current phase) | Studies on ClinicalTrials.gov (examples) |
|---|---|---|
| Severe refractory SLE and lupus nephritis | 2023 / 2028, Phase 2 | NCT06581198 (Phase 2, estimated 179 participants, primary endpoints efficacy at weeks 24 and 52, estimated primary completion February 2028) |
| Systemic sclerosis | 2024 / 2029 or later, Phase 2 | NCT06655896 (Phase 2, estimated 96 participants, rCRISS50 response at week 52, estimated primary completion November 2028) |
| Myositis, ANCA-associated vasculitis | 2024 and 2025 / 2029 or later, Phase 2 | NCT06665256 (myositis), NCT06868290 (vasculitis) |
| High-risk large B-cell lymphoma (first-line) | 2023 / 2029 or later, Phase 2 | NCT03960840 (Phase 1/2, 217 participants, estimated primary completion June 2027) |
All are in development (Phase 2), and the prospects for approval are Not yet confirmed.
None of the following appears in the 20-F Not yet confirmed.
Any in-house plan using iPS cells; any in-house genome editing programme; dedicated R&D spending and headcount for cell and gene therapy; the timing of entering the clinic for Kate-derived products; and measures to respond to the fall in Kymriah sales.
8. What the company is aiming for
This section goes beyond what the company says directly. It sets out inferences that can be drawn from primary sources, each with its basis.
| What can be read | Primary evidence behind it | Category |
|---|---|---|
| It is shifting the centre of gravity of CAR-T from cancer to autoimmune disease | The 20-F development table lists four autoimmune and one oncology project for YTB323. Most of the studies on ClinicalTrials.gov are in autoimmune disease | Inference (Sourced: the distribution of projects) |
| In AAV it is running two tracks: widening the use of an existing product (intrathecal dosing) and new targets (Kate) | The Itvisma approval (fourth quarter of 2025) and the Kate acquisition (October 2024) | Inference |
| It is not currently pursuing iPS cells or genome editing outside the body | Not described in the 20-F | Inference (Sourced: the absence of any mention) |
| It wants to move cell and gene therapy towards low-volume, individualised manufacturing | The 20-F says the company is moving from high-volume products to low-volume, customised, individualised medicines | Sourced (the statement) / Inference (the implication) |
On manufacturing, the 20-F says the company is moving from high-volume products to low-volume, customised, individualised medicines, and also states that it has closed, consolidated or sold six manufacturing sites Sourced. For patient-by-patient therapies such as CAR-T, lots are small and where the manufacturing sites are placed decides whether the business works. Yet Kymriah sales are falling, and whether this move towards individualisation translates into revenue depends on future studies and on the market Not yet confirmed.
9. Map of relationships
10. Technologies, products and services on offer
| Item | What the 20-F says |
|---|---|
| Zolgensma (onasemnogene abeparvovec) | A gene therapy that, in a single intravenous infusion, supplies the function of the missing or non-working SMN1 gene. In the U.S., the EU and elsewhere it is used for infants with SMA who have mutations in both copies of the SMN1 gene. 2025 sales of the Zolgensma franchise were US$1.232 billion |
| Itvisma (onasemnogene abeparvovec-brve) | The intrathecal version of the same gene therapy. In the U.S. and elsewhere it is for adults and children aged 2 and over with a confirmed SMN1 mutation. Approved in the U.S. and the UAE in the fourth quarter of 2025 |
| Kymriah | A CAR-T cell therapy (the 20-F gives no description of the product, only sales). US$381 million in 2025 (down 14%) |
| rapcabtagene autoleucel (YTB323) | A CD19 CAR-T. In development for SLE, systemic sclerosis, myositis and ANCA-associated vasculitis (autoimmune diseases) and for large B-cell lymphoma |
| Manufacturing | Manufacturing including cell and gene therapy at Stein (Switzerland), and manufacture and shipment of Zolgensma at Durham (U.S.). Sales of contract manufacturing (including cell and gene therapy) are also recorded |
Within the same "cell and gene therapy" platform, Novartis holds two entirely different lines in terms of materials Sourced. (1) AAV (Zolgensma, Itvisma and Kate): the viral vector is the product itself. The virus is made in cultured cells, and manufacturing runs through purification, titre measurement and filling. The materials that matter are culture media and serum substitutes, the plasmids that carry the gene, purification resins and containers for cryopreservation (see our explainers on viral vectors, bioreactors and GMP). With Zolgensma moving from an intravenous to an intrathecal version (Itvisma), the dose and the form of the formulation change, and so do the manufacturing conditions. (2) Autologous CAR-T (Kymriah and YTB323): the patient's cells are collected, given a gene, expanded, frozen and returned. Here patient-by-patient lot control, closed-system culture equipment, cryopreservation media and shipping containers matter (see our explainers on CAR-T cells and on autologous versus allogeneic cells). The 20-F speaks of moving to patient-by-patient, low-volume manufacturing, but does not disclose specific materials, specifications or suppliers Not yet confirmed.
11. The risks this company carries
| Risk | Substance | Category |
|---|---|---|
| Falling Kymriah sales | US$508 million in 2023, US$443 million in 2024 and US$381 million in 2025. The company attributes this to competition | Sourced |
| The Zolgensma franchise struggles to grow | The intravenous version already reaches a high share of new patients, and sales were flat in 2025. Growth depends on Itvisma | Sourced (sales) / Inference (the dependence) |
| CAR-T for autoimmune disease is early | Filing for the main projects is planned for 2028 or later, and they are now in Phase 2. Results are not yet established | Sourced |
| Manufacturing complexity | The 20-F says complex manufacturing processes raise the risk of production failure and product recalls. A cell or gene therapy given only once differs in its sales profile from a medicine for a chronic disease | Sourced |
| Cell and gene therapy is about 3% of sales | The Zolgensma franchise and Kymriah together are about 3% of net sales. The success of the whole company does not hinge on them | Sourced (the ratio is our calculation) / Inference (the implication) |
| The burden of a large acquisition | The roughly US$12 billion for Avidity is to be paid from cash on hand and outside borrowing (20-F). This may affect its capacity to invest in cell and gene therapy | Sourced (the financing) / Inference (the effect) |
| No mention of iPS cells or genome editing | For now, bringing in new mechanisms depends on acquisitions | Inference |
12. Glossary
- AAV
- Adeno-associated virus. Used as a vector to deliver genes into cells.
- SMA (spinal muscular atrophy)
- An inherited neuromuscular disease in which motor neurons are lost because of mutations in the SMN1 gene.
- Intrathecal administration
- A route in which the medicine is injected directly into the space where the fluid around the spinal cord (cerebrospinal fluid) flows.
- CAR-T cell therapy
- A cell therapy in which T cells are given a gene so that they carry a receptor (CAR) that recognises a target.
- Autologous
- Using the patient's own cells. Each product is made for one person.
- xRNA
- One of the advanced platforms Novartis names. Medicines that use RNA (such as siRNA).
- Acquired in-process R&D (IPR&D)
- The price paid for an acquired asset still in development.
- FTE
- Full-time equivalent. A headcount converted to full-time staff.
- Form 20-F
- The annual report that a foreign company listed in the United States files with the SEC.
13. References
- Novartis AG Form 20-F for the fiscal year ended December 31, 2025 (filed with the U.S. SEC on 4 February 2026). Business, products, sales, R&D expenses, employees, the development table, manufacturing sites and the Kate and Avidity acquisitions. https://www.sec.gov/Archives/edgar/data/1114448/000111444826000004/nvs-20251231.htm
- ClinicalTrials.gov NCT06581198 (rapcabtagene autoleucel, SLE / lupus nephritis, Phase 2). https://clinicaltrials.gov/study/NCT06581198
- ClinicalTrials.gov NCT06655896 (rapcabtagene autoleucel, diffuse cutaneous systemic sclerosis, Phase 2). https://clinicaltrials.gov/study/NCT06655896
- ClinicalTrials.gov NCT03960840 (rapcabtagene autoleucel, CLL, DLBCL, ALL and first-line high-risk LBCL, Phase 1/2). https://clinicaltrials.gov/study/NCT03960840
- ClinicalTrials.gov NCT05798117 (YTB323, severe refractory SLE, Phase 1/2). https://clinicaltrials.gov/study/NCT05798117
- ClinicalTrials.gov NCT06665256 (rapcabtagene autoleucel, myositis, Phase 2). https://clinicaltrials.gov/study/NCT06665256
- ClinicalTrials.gov NCT06868290 (rapcabtagene autoleucel, ANCA-associated vasculitis [GPA / MPA], Phase 2). https://clinicaltrials.gov/study/NCT06868290
- ClinicalTrials.gov NCT07048197 (rapcabtagene autoleucel, rheumatoid arthritis, Phase 1/2). https://clinicaltrials.gov/study/NCT07048197
- ClinicalTrials.gov NCT07443137 (a study sponsored by Stanford University that uses rapcabtagene autoleucel). https://clinicaltrials.gov/study/NCT07443137
- Novartis Official website, used to check the company name and logo. https://www.novartis.com/
14. Claim-to-source audit
| Claim in the article | Category | Source |
|---|---|---|
| Legal name, head office (Basel), listings (NYSE: NVS, SIX), 75,267 employees (20,015 in R&D) | Sourced | Reference 1 https://www.sec.gov/Archives/edgar/data/1114448/000111444826000004/nvs-20251231.htm |
| Net sales (about US$54.5 billion), R&D expenses (US$11.2 billion / US$10.0 billion), operating income | Sourced | Reference 1 https://www.sec.gov/Archives/edgar/data/1114448/000111444826000004/nvs-20251231.htm |
| Zolgensma franchise sales of US$1.232 billion, Kymriah sales (2023 to 2025), approval of Itvisma (fourth quarter of 2025, U.S. and UAE) | Sourced | Reference 1 https://www.sec.gov/Archives/edgar/data/1114448/000111444826000004/nvs-20251231.htm |
| That cell and gene therapy is named as one of three advanced platforms | Sourced | Reference 1 https://www.sec.gov/Archives/edgar/data/1114448/000111444826000004/nvs-20251231.htm |
| Acquisition of Kate Therapeutics (31 October 2024, US$427 million in cash, milestones of up to US$700 million, breakdown of the consideration) | Sourced | Reference 1 https://www.sec.gov/Archives/edgar/data/1114448/000111444826000004/nvs-20251231.htm |
| Agreement to acquire Avidity Biosciences (25 October 2025, US$72 per share, about US$12 billion, not completed at the time of the 20-F) | Sourced | Reference 1 https://www.sec.gov/Archives/edgar/data/1114448/000111444826000004/nvs-20251231.htm |
| Planned filing years and current phases for YTB323 (SLE in 2028, others in 2029 or later) | Sourced | Reference 1 https://www.sec.gov/Archives/edgar/data/1114448/000111444826000004/nvs-20251231.htm |
| Manufacturing sites (Stein, Durham), the move to low-volume, individualised manufacturing, and the closing, consolidation or sale of six sites | Sourced | Reference 1 https://www.sec.gov/Archives/edgar/data/1114448/000111444826000004/nvs-20251231.htm |
| Phase 2 study in SLE / lupus nephritis (NCT06581198) | Sourced | Reference 2 https://clinicaltrials.gov/study/NCT06581198 |
| Phase 2 study in systemic sclerosis (NCT06655896) | Sourced | Reference 3 https://clinicaltrials.gov/study/NCT06655896 |
| Phase 1/2 study of YTB323 in tumours (NCT03960840) | Sourced | Reference 4 https://clinicaltrials.gov/study/NCT03960840 |
| Early YTB323 study in autoimmune disease (NCT05798117, started February 2023) | Sourced | Reference 5 https://clinicaltrials.gov/study/NCT05798117 |
| YTB323 studies in myositis (NCT06665256) and vasculitis (NCT06868290) | Sourced | References 6 and 7 https://clinicaltrials.gov/study/NCT06665256 |
| YTB323 study in rheumatoid arthritis (NCT07048197, started June 2025) | Sourced | Reference 8 https://clinicaltrials.gov/study/NCT07048197 |
| YTB323 study sponsored by Stanford University (NCT07443137) | Sourced | Reference 9 https://clinicaltrials.gov/study/NCT07443137 |
| Confirmation of the logo and the way the name is written | Sourced | Reference 10 https://www.novartis.com/ |
| The three types of company in Fig. 1 | Our calculation | Our own classification. Companies shown are examples from those covered in this series |
| Cell and gene therapy (Zolgensma franchise and Kymriah) at about 3% of net sales | Our calculation | Our own calculation, from the figures in reference 1 |
| Dedicated R&D spending, headcount and capital expenditure for cell and gene therapy, timing of clinical entry for Kate-derived products, and any in-house plan for iPS cells or genome editing | Not yet confirmed | Not in the 20-F. This article makes no estimate https://www.sec.gov/Archives/edgar/data/1114448/000111444826000004/nvs-20251231.htm |
| The reading that the centre of gravity of CAR-T is shifting from cancer to autoimmune disease | Inference | This article's interpretation of the distribution of YTB323 studies |
| The reading that AAV runs on two tracks: an existing product and new targets | Inference | This article's interpretation of Itvisma and Kate |
| The reading that it is not pursuing iPS cells or genome editing outside the body | Inference | This article's interpretation of the absence of any mention in the 20-F |
| The reading that it wants to move to low-volume, individualised manufacturing, the view that growth of the Zolgensma franchise depends on Itvisma, and the view that a large acquisition may affect its capacity to invest | Inference | This article's interpretation of the manufacturing policy statement, sales trends and the financing of the acquisition |
Last updated 1 October 2026 / Troy Technical
Every figure in this article comes from the statutory filing (Form 20-F) that Novartis AG made with the U.S. Securities and Exchange Commission, and from ClinicalTrials.gov. No research-firm estimates or press-based figures have been used. Passages marked "Inference" are this article's interpretation of primary sources, not statements made by the company. This article is not medical advice. It does not recommend any particular treatment or rank it against others.