Ex vivo autologous cell and gene therapy has more than a decade of clinical results behind it; Europe approved the first ex vivo gene therapy in 2016, and the US approved the first CAR T-cell therapy a year later.
Since then, the modality has built a long, consistent clinical record. Some of the first patients treated with CAR-T cells for leukemia are still in remission more than ten years on. Approved therapies span blood cancers, inherited blood disorders and rare immune diseases.
This year we’ve seen more clinical results add to the category’s evidence base. Satri-cel became the first CAR T-cell therapy approved for a solid tumor. Kyverna’s miv-cel showed that a single infusion can reverse disability in stiff person syndrome, with responses in myasthenia gravis holding for up to 18 months. In multiple myeloma, CAR-T continues to move into earlier lines of treatment, and major pharma companies have reinforced their commitment to the modality.
Recent news articles have raised questions about where ex-vivo autologous therapy goes next and how it integrates with ex-vivo allogenic and in-vivo opportunities. Progress in a field is rarely linear, and the clinical record speaks for itself. Ex-vivo autologous therapies have approved products, long-term follow-up data and pipelines moving into new indications and it’s exciting to think about how they will continue to deliver meaningful impact to patients globally. We know it will take time for ex-vivo allogenic and in-vivo approaches to catch up and we are excited to see how these options will provide more and more choices for patients in the future.
While the biology of ex-vivo autologous therapy has been proven, the next hurdles continue to include manufacturing at scale and reduced cost, which limit how widely these therapies are offered. The field is maturing to meet these challenges, and the FDA created its Advanced Manufacturing Technology designation in 2025 to speed up the shift.
The highlights below show data reported or validated through 2026, covering approved medicines and first-in-human studies, blood cancers and solid tumors, rare inherited disease and autoimmunity.
Search the 2026 highlights by drug, company or indication
This listing may not be comprehensive and should not be used as such. It reflects information available as of October 2026, may omit items or contain errors, and is subject to change without notice. Users should verify the information independently before relying on it.
Scope
This listing covers ex vivo autologous cell and gene therapies with new clinical data, approvals or deals made public between January 1 and October 29, 2026.
Included: therapies made from the patient’s own cells and processed outside the body: CAR-T, TCR-T and mRNA CAR-T, tumor-infiltrating lymphocyte (TIL) therapies, and gene-modified or gene-edited stem cells (HSC gene therapy).
Excluded: donor-derived (allogeneic) products and in vivo CAR-T.
Dating: a result counts if it was first presented in 2026. Data presented in 2025 and published in a journal this year is left out, as is Waskyra®, which the FDA approved on December 10, 2025.
Sources: each drug name links to its main source.
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Approved Therapies
| Drug | Therapy area | Company | Modality | Lead setting | Where approved | Defining clinical achievement | Why it matters |
|---|---|---|---|---|---|---|---|
| Satri-cel | Oncology | CARsgen Therapeutics | Autologous Claudin18.2 CAR-T | Claudin18.2-positive, HER2-negative advanced stomach and gastroesophageal cancer after two or more prior treatments | China (June 2026) | First CAR-T approved for a solid tumor. Two patients from an earlier study alive more than four years after starting treatment; no relapses so far in 10 patients treated after surgery | Shows CAR-T can work in solid tumors, and it is already being tested earlier in treatment |
| Carvykti® | Oncology | Johnson & Johnson and Legend Biotech | Autologous BCMA CAR-T | Multiple myeloma after at least one prior treatment | US, EU and Japan, among others; Japan extended it to earlier lines in July 2026 | Half of patients treated after one to three prior treatments alive and progression-free at five years with no further therapy; survival benefit held in high-risk genetic subgroups; in smoldering myeloma, every patient had undetectable disease by month two | Points to long treatment-free remissions when CAR-T is used earlier, and to its use before myeloma becomes active |
| Kymriah® | Oncology | Novartis | Autologous CD19 CAR-T | Relapsed B-cell leukemia and lymphoma; first-line use in high-risk childhood leukemia under study | US, EU and others | In 121 children and young adults whose leukemia persisted after chemotherapy, 85% alive at four years and 64% disease-free at five years | Moves CAR-T into first-line care for children with high-risk leukemia |
| Yescarta® | Oncology | Kite (Gilead) | Autologous CD19 CAR-T | Large B-cell and other lymphomas, now including primary central nervous system lymphoma | US, EU and others; US label extended in February 2026 | FDA removed the label restriction on primary central nervous system lymphoma, based on an investigator-led phase 1 study | Extends CAR-T to lymphoma in the brain |
| Rimqarto® | Oncology | Curocell | Autologous CD19 CAR-T | Relapsed large B-cell lymphoma | South Korea (April 2026) | 67% complete response rate in its pivotal trial | First CAR-T developed in Korea |
| Amtagvi® | Oncology | Iovance Biotherapeutics | Autologous TIL (unmodified) | Advanced melanoma after checkpoint inhibitors; first-line melanoma, sarcoma and endometrial cancer under study | US | First-line melanoma with pembrolizumab: 62.5% responded and 29% completely (24 patients). Sarcoma: 4 of 8 responded, all still responding. Endometrial cancer: 40% responded (5 patients) | Takes TIL beyond its approved use; the phase 3 TILVANCE-301 trial and a registrational sarcoma trial are enrolling |
| Tecelra® | Oncology | US WorldMeds | Autologous MAGE-A4 TCR-T | Advanced synovial sarcoma after chemotherapy, in HLA-A*02-positive patients whose tumor expresses MAGE-A4 | US (full approval June 2026, extended to age 12 and older; accelerated approval 2024) | In 137 patients, 43.8% responded and 3.6% completely; responses lasted a median of 5.3 months, and 31.9% of responders were still responding at two years or more | The first engineered T-cell therapy for a solid tumor now has full US approval, and is open to teenagers |
| Aucatzyl® | Autoimmune | Autolus | Autologous CD19 CAR-T | Adult relapsed B-cell acute lymphoblastic leukemia; severe lupus under study | US and EU (for leukemia) | In severe, treatment-resistant lupus, 5 of 9 patients in remission, with no neurotoxicity or significant cytokine release | FDA Regenerative Medicine Advanced Therapy designation in lupus; phase 2 LUMINA trial in lupus nephritis under way |
| Casgevy® | Rare inherited disease | Vertex and CRISPR Therapeutics | Autologous CRISPR-edited stem-cell (HSC) gene therapy | Sickle cell disease and transfusion-dependent beta-thalassemia | US, EU, UK and others | In children aged 5 to 11, all 8 evaluable sickle cell patients free of pain crises and all 8 evaluable thalassemia patients transfusion-free for at least 12 months; one death from conditioning chemotherapy | Supports extending Casgevy to children aged 5 to 11; under US review |
| Kresladi® | Rare inherited disease | Rocket Pharmaceuticals | Autologous lentiviral stem-cell (HSC) gene therapy | Severe leukocyte adhesion deficiency type I in children | US (accelerated approval, March 2026) | Approved on a biomarker (CD18 and CD11a expression); clinical benefit still to be confirmed | The second US-approved stem-cell gene therapy for an inherited immune deficiency, less than four months after Waskyra® |
Investigational Therapies
| Drug | Therapy area | Company | Modality | Lead setting | Development stage | Defining clinical achievement | Why it matters |
|---|---|---|---|---|---|---|---|
| Anito-cel | Oncology | Gilead (Kite), which acquired Arcellx | Autologous BCMA CAR-T | Relapsed or refractory multiple myeloma | Under FDA review; decision due December 23, 2026 | Final phase 1 results: all 38 patients responded and 79% had a complete response, with a median follow-up of 38 months | Gilead paid $7.8 billion for Arcellx to own it outright |
| Arlo-cel | Oncology | Bristol Myers Squibb | Autologous GPRC5D CAR-T | Multiple myeloma after four or more prior treatments, including a BCMA therapy | Registrational phase 2 met its primary endpoint (September 2026) | Met its primary endpoint (response rate) and key secondary endpoint; figures not yet released. Used earlier, in phase 1, 94% responded and 71% had a complete response or better | A new myeloma target beyond BCMA; the phase 3 QUINTESSENTIAL-2 trial is under way |
| Anzu-cel (IMA203) | Oncology | Immatics | Autologous PRAME TCR-T | Advanced melanoma after checkpoint inhibitors, including eye (uveal) melanoma | Phase 3 (SUPRAME); results due in the first half of 2027 | 56% confirmed response rate (50% skin, 67% uveal); responses lasted a median of 14.6 months | Immatics plans a US filing in 2027 if the phase 3 succeeds |
| IMA203CD8 | Oncology | Immatics | Autologous next-generation PRAME TCR-T | Advanced solid tumors, including gynecological cancers, melanoma and synovial sarcoma | Phase 1 | 51% responded across 90 patients (41% confirmed); 60% in gynecological cancers at the higher doses; some responses ongoing beyond 2.5 years | Responses across several solid tumor types |
| C-CAR031 | Oncology | AstraZeneca, with AbelZeta | Autologous armored GPC3 CAR-T | Advanced liver cancer (hepatocellular carcinoma) | Phase 1 | 44% responded and 92% had disease control; median survival 18 months (36 patients) | AstraZeneca paid $630 million in January for full global rights |
| Ori-C101 | Oncology | OriCell Therapeutics | Autologous armored GPC3 CAR-T | Heavily pretreated advanced liver cancer (hepatocellular carcinoma) | Confirmatory phase 2 cleared in China (June 2026) | 50% responded overall and 66.7% at the recommended dose; one complete response lasting 24 months | Moving into a randomized confirmatory trial |
| LB2102 | Oncology | Legend Biotech | Autologous armored DLL3 CAR-T | Relapsed small-cell lung cancer and large-cell neuroendocrine carcinoma | Phase 1 | At the higher doses, 29% responded and 79% had disease control (20 patients) | Extends CAR-T to small-cell lung cancer |
| Dual-target EGFR/IL13Rα2 CAR-T | Oncology | University of Pennsylvania, with Kite | Autologous CAR-T delivered into the brain fluid | Recurrent glioblastoma | Phase 1 | Median survival of 12 months at 19 months’ follow-up, with no late side effects | Supports delivering CAR-T directly into the brain in glioblastoma |
| SynKIR-110 | Oncology | Verismo Therapeutics | Autologous mesothelin KIR-CAR T | Advanced ovarian cancer, mesothelioma and cholangiocarcinoma | Phase 1 | One partial response among the 3 patients at the highest dose; no severe cytokine release and no neurotoxicity | Early data for a new CAR design in solid tumors |
| OBX-115 | Oncology | Obsidian Therapeutics | Autologous TIL, engineered to make adjustable interleukin-15 | Advanced melanoma, mostly after two checkpoint inhibitors | Phase 1/2; registration-enabling cohort starting | At the recommended dose, 67% responded, including 2 complete responses (15 patients) | Obsidian agreed in April to merge with Galera Therapeutics |
| TIL for uveal melanoma | Oncology | University of Pittsburgh Medical Center (UPMC) Hillman Cancer Center | Autologous TIL (unmodified) | Metastatic eye (uveal) melanoma | Phase 2 | 37% responded when their TIL reacted against the tumor, and none when they did not; responses lasted a median of 17.3 months | Points to a test for picking patients whose TIL are likely to work |
| Miv-cel (KYV-101) | Autoimmune | Kyverna Therapeutics | Autologous fully human CD19 CAR-T | Stiff person syndrome and generalized myasthenia gravis; also studied in rheumatoid arthritis and progressive multiple sclerosis | Registrational; US filing for stiff person syndrome due to complete by end of 2026 | Stiff person syndrome at one year: walking speed improved by a median of 49% and 24 of 26 patients off long-term immune therapies, with no severe cytokine release or neurotoxicity. Myasthenia gravis responses followed for up to 1.5 years | Could become the first CAR-T approved for an autoimmune disease |
| Rese-cel | Autoimmune | Cabaletta Bio | Autologous CD19 CAR-T | Myositis, lupus, systemic sclerosis and pemphigus vulgaris | Registrational cohort in myositis; US filing planned for 2027 | 8 of 10 patients with dermatomyositis or antisynthetase syndrome would have met the registrational endpoint; 6 of 8 lupus patients in remission at 12 months without immune-suppressing drugs | Also testing treatment without chemotherapy beforehand, which could make it simpler to give |
| Zola-cel | Autoimmune | Bristol Myers Squibb | Autologous CD19 CAR-T, made in five days | Multiple sclerosis, lupus, systemic sclerosis, myositis and rheumatoid arthritis | Phase 1 | In 66 multiple sclerosis patients, 6 of 7 with relapsing disease showed no disease activity and 22 of 24 with progressive disease were stable or improved | A large CAR-T dataset in multiple sclerosis |
| Rap-cel | Autoimmune | Novartis | Autologous CD19 CAR-T, made in about two days | Lupus, myositis and systemic sclerosis | Phase 1/2 | Two-year follow-up in 21 lupus patients and first myositis and systemic sclerosis results presented at EULAR | Made in about two days, against roughly 14 days for standard processes |
| Descartes-08 | Autoimmune | Cartesian Therapeutics | Autologous BCMA mRNA CAR-T, given without chemotherapy | Generalized myasthenia gravis | Phase 3 (AURORA); results due in the first quarter of 2027 | Patients retreated after symptoms returned improved by 6.6 points on average on a symptom score at 12 months | Given without chemotherapy, and patients can be retreated |
| CD19 CAR-T (Erlangen) | Autoimmune | Erlangen University Hospital | Autologous CD19 CAR-T | Lupus | Academic, four-year follow-up | All eight of the first patients treated at Erlangen still in remission without steroids or immunosuppressants | Four years of drug-free remission in lupus |
| Risto-cel (BEAM-101) | Rare inherited disease | Beam Therapeutics | Autologous base-edited stem-cell (HSC) gene therapy | Sickle cell disease | Phase 1/2 | Fetal hemoglobin above 60% and no severe pain crises after engraftment (31 patients); median 2.9 months from cell collection to product release | Uses base editing, a newer gene-editing method that avoids cutting both strands of DNA |
Abbreviations
EULAR: European Alliance of Associations for Rheumatology congress
FDA: US Food and Drug Administration
CAR-T: chimeric antigen receptor T cells
TCR-T: T-cell receptor engineered T cells
TIL: tumor-infiltrating lymphocytes
HSC: hematopoietic (blood-forming) stem cells