CAR-T cell therapy

01Overview and value chain

Markers: [EC: ATMP Regulation (EC No 1394/2007) + FDA CTLA guidance | OECD: Bio-pharma | Regulator: FDA (USA), EMA (European Union), NMPA (China)]

CAR-T cell therapy collects a patient’s T lymphocytes, engineers them ex vivo with a chimeric antigen receptor (CAR) using a lentiviral or retroviral vector, expands and re-infuses them as a one-time living drug that recognises and kills cancer. It is the flagship advanced therapy medicinal product (ATMP) class, with multiple approved CD19 products (Novartis Kymriah, Kite Yescarta/Tecartus, Bristol Myers Squibb Breyanzi) and BCMA multiple-myeloma products (BMS/2seventy Abecma, Janssen/Legend Carvykti). Kite secured an FDA label update for Yescarta in February 2026, BMS’s Breyanzi monitoring update was approved in Canada in June 2026, Janssen’s Carvykti continues to read out from the LEGEND programme, and Cellectis (France) is advancing off-the-shelf allogeneic CAR-T (UCART) built on its TALEN gene-editing platform.

The key directions of CAR-T cell therapy are:

1. Autologous CD19 CAR-T (CD19 CAR-T): patient-specific CD19-directed CAR-T for B-cell lymphoma and leukemia — Novartis (Kymriah), Kite (Yescarta, Tecartus), Bristol Myers Squibb (Breyanzi).
2. BCMA CAR-T for multiple myeloma (BCMA CAR-T): B-cell-maturation-antigen-directed CAR-T for relapsed myeloma — Bristol Myers Squibb/2seventy (Abecma), Janssen/Legend (Carvykti).
3. Allogeneic off-the-shelf CAR-T (Allogeneic CAR-T): TALEN- or CRISPR-edited donor T cells intended as an immediate, inventory-managed product — Cellectis (UCART).
4. Manufacturing and vein-to-vein access (Manufacturing): apheresis, lentiviral transduction, automated GMP expansion, cryopreservation and lymphodepletion that determine the ~2-4 week vein-to-vein time and scalability.

Sectoral value chain

[patient T cells (apheresis)] ──> [lentiviral CAR transduction + expansion] ──> [cryopreserved CAR-T product]
                                            │
                                   (lymphodepletion conditioning)
                                            │
                                            ▼
[cancer remission] <─── [CAR-T infusion + CRS/ICANS management] <─────┘

Value chain levels

Cross-cutting technologies of the sector:

• Lentiviral and retroviral vectors (Lentiviral Vector): all autologous CAR-T products use integrating viral vectors to insert the CAR transgene, and vector supply is a structural manufacturing bottleneck.
• Automated cell processing (Cell Manufacturing): closed systems (e.g. CliniMACS) and single-use bioreactors standardise vein-to-vein time and underpin the CDMO tier.
• Target antigen selection (Target Selection): CD19 (lymphoma/leukemia) and BCMA (myeloma) anchor the approved class, with the allogeneic frontier using gene editing to prevent graft-versus-host and rejection.

02US

The US is the centre of gravity for CAR-T — Kite, Bristol Myers Squibb and Janssen (with its Legend partner) are US-based, and the FDA has approved the most CAR-T products globally.

Kite, BMS, Janssen, FDA

• Kite Pharma (a Gilead company): the FDA approved a label update for Yescarta (axicabtagene ciloleucel) in February 2026, reinforcing its lead in CD19 CAR-T for large B-cell lymphoma, alongside Tecartus for mantle-cell and ALL.
• Bristol Myers Squibb: Breyanzi (lisocabtagene maraleucel, CD19) had its monitoring and activity update approved in Canada in June 2026, and BMS co-develops Abecma (idecabtagene vicleucel, BCMA) with 2seventy bio for multiple myeloma.
• Janssen (with Legend Biotechnology): Carvykti (ciltacabtagene autoleucel) is a BCMA CAR-T for relapsed/refractory multiple myeloma, advanced through the LEGEND-2 programme and a growing real-world evidence base.
• FDA framework: CAR-T products are approved as biologics with boxed warnings for cytokine release syndrome (CRS) and neurotoxicity (ICANS), Risk Evaluation and Mitigation Strategies (REMS) and long-term follow-up requirements.

03CN

China’s CAR-T field is the largest clinical pipeline globally and the origin of a marketed BCMA product, led by Legend Biotechnology as the global Carvykti co-originator.

Legend, NMPA, pipeline depth

• Legend Biotechnology: the Nanjing-headquartered CAR-T pioneer that co-developed and co-commercialises Carvykti (ciltacabtagene autoleucel) with Janssen, making it the first China-origin CAR-T to reach the US and EU markets; Q1 2026 losses narrowed as Carvykti revenue scaled.
• Domestic pipeline: China runs the deepest CAR-T clinical pipeline worldwide, with multiple CD19 and BCMA candidates from domestic biotechs under NMPA review, several approved for domestic use.
• NMPA framework: the National Medical Products Administration has approved CAR-T products for the Chinese market and aligns manufacturing and pharmacovigilance expectations with the FDA and EMA.

04EU

Europe contributes Cellectis’s allogeneic CAR-T platform, Novartis’s Kymriah franchise and the EMA’s centralised ATMP authorisation route.

Novartis, Cellectis, EMA, ATMP

• Novartis (Switzerland): Kymriah (tisagenlecleucel) was the first FDA-approved CAR-T (2017, CD19) for pediatric ALL and diffuse large B-cell lymphoma, anchoring the autologous class.
• Cellectis (France): a clinical-stage biotechnology company using its TALEN gene-editing platform to develop allogeneic, off-the-shelf CAR-T (UCART); it presented final Phase 1 results for lasme-cel and preliminary data in June 2026.
• EMA framework: CAR-T are advanced therapy medicinal products centrally authorised by the European Medicines Agency under Regulation (EC) No 1394/2007, with PRIME and conditional approval pathways supporting early access.
• European CDMO: European cell-therapy CDMOs and vector manufacturers participate in the GMP supply chain that underpins the vein-to-vein manufacturing model.

05Leading companies and research institutes

06Tech stack and innovations

The stack pairs CAR transgene design with integrating-vector delivery, closed-system GMP manufacturing, and structured clinical management of the CAR-T unique toxicities.

1. Autologous CD19 CAR-T (CD19 CAR-T):
   • Novartis’s Kymriah was the first approved CAR-T (CD19, pediatric ALL and DLBCL), and Kite’s Yescarta won a February 2026 FDA label update reinforcing its DLBCL lead, with Tecartus extending the franchise.
   • Bristol Myers Squibb’s Breyanzi (CD19) had a monitoring update approved in Canada in June 2026, broadening its clinical use.
2. BCMA CAR-T for myeloma (BCMA CAR-T):
   • Janssen and Legend’s Carvykti (ciltacabtagene autoleucel) and BMS/2seventy’s Abecma (idecabtagene vicleucel) target BCMA in relapsed multiple myeloma, with Carvykti scaling on the LEGEND-2 evidence base.
   • Legend Biotechnology (Nanjing) is the first China-origin CAR-T developer to reach US and EU markets, with Q1 2026 losses narrowing as revenue scaled.
3. Allogeneic off-the-shelf CAR-T (Allogeneic CAR-T):
   • Cellectis uses its TALEN gene-editing platform to build donor-derived UCART products that could be manufactured as inventory, presenting final Phase 1 lasme-cel results in June 2026.
   • Gene editing to knock out the T-cell receptor and HLA is what enables an off-the-shelf product while controlling graft-versus-host and rejection.

07Value chains and production pipelines

Industrial pipeline of an autologous CAR-T product (ATMP Regulation, REMS)

┌───────────────────────────┐      ┌───────────────────────────┐
│ 1. Leukapheresis          │ ───> │ 2. Vector engineering     │
└───────────────────────────┘      └───────────────────────────┘
                                                   │
                                                   ▼
┌───────────────────────────┐      ┌───────────────────────────┐
│ 4. Lymphodepletion        │ <─── │ 3. Cell manufacturing     │
└───────────────────────────┘      └───────────────────────────┘
              │
              ▼
┌───────────────────────────┐      ┌───────────────────────────┐
│ 5. Infusion & monitoring  │ ───> │ 6. Follow-up & PV         │
└───────────────────────────┘      └───────────────────────────┘

Stage 1: Leukapheresis

The patient’s T cells are collected by leukapheresis at a qualified treatment centre and cryopreserved or shipped fresh under chain-of-identity to the manufacturing site.

Stage 2: Vector engineering

T cells are activated and transduced with a lentiviral or retroviral vector carrying the CAR transgene (CD19 or BCMA scFv plus costimulatory domains), inserting the CAR into the T-cell genome.

Stage 3: Cell manufacturing

Transduced CAR-T are expanded in closed, automated single-use bioreactor systems (e.g. CliniMACS), formulated, fill-and-finished, cryopreserved and released under GMP, setting the ~2-4 week vein-to-vein time.

Stage 4: Lymphodepletion

The patient receives lymphodepleting chemotherapy (typically fludarabine/cyclophosphamide) to create immunological space for CAR-T expansion and persistence.

Stage 5: Infusion and monitoring

The CAR-T product is re-infused, and the patient is monitored and managed for cytokine release syndrome (CRS) and immune effector cell-associated neurotoxicity syndrome (ICANS), with tocilizumab and steroids on standby under REMS.

Stage 6: Follow-up and pharmacovigilance

Long-term follow-up tracks CAR-T persistence, relapse, late cytopenias and secondary malignancies, feeding the pharmacovigilance record that supports label updates such as Kite’s February 2026 Yescarta revision.