Cell and gene therapy takeaways from ASCO 2026

While Revolution Medicines' plenary presentation on daraxonrasib's survival benefit in metastatic pancreatic cancer was among ASCO's biggest stories, several promising cell and gene therapy programs also generated attention with new clinical data.

In vivo and allogenic CAR-T

Kelonia Therapeutics — acquired by Eli Lilly in April in a potential $7 billion deal —  shared positive updated results from its ongoing first-in-human phase 1 study of its in vivo CAR-T, KLN-1010, in patients with relapsed and refractory multiple myeloma.

Fourteen additional patients have been dosed in the inMMyCAR study since first clinical data were reported at the ASH 2025 Annual Meeting, bringing the total number of patients dosed to 18. The interim data, presented at ASCO 2026, demonstrate a 100% overall response rate and minimal residual disease-negative bone marrow at one month post treatment in all evaluable patients. Importantly, the data demonstrated robust generation and sustained persistence of CAR-T cells in the peripheral blood and bone marrow exceeding what is typically seen with ex vivo CAR-T therapies.

Strand Therapeutics presented promising preclinical data from its in vivo CAR-T program. The Boston-based biotech's Signal Stack platform combines EverScript circRNA, an enhanced CAR design, and targeted lipid nanoparticle (LNP) delivery to generate CAR-T cells directly in the body, potentially eliminating the need for complex ex vivo manufacturing. In preclinical studies, the approach produced functional CAR-T cells after intravenous administration and achieved robust target-cell elimination in humanized mouse models and non-human primates.

Imugene Limited presented new phase 1b data for its off-the-shelf, allogenic CAR-T cell therapy, with responses recorded across six different blood cancer subtypes in CAR-T naïve patients.

Azer-cel demonstrated promising clinical activity across a broad range of CD19+ B-cell malignancies. A total of 25 patients received azer-cel in combination with low-dose interleukin-2 with 24 evaluable for response after their first disease assessment at day 28. Responses were observed across all cancer subtypes included in the analysis:

• Diffuse large B-cell lymphoma (DLBCL): 67% response rate
• Marginal zone lymphoma (MZL): 83% response rate
• Chronic lymphocytic leukemia (CLL): 100% response rate
• Primary central nervous system lymphoma (PCNSL): 50% response rate
• Follicular lymphoma (FL): 100% response rate
• Waldenström macroglobulinaemia (WM): 100% response rate

Solid tumors

While ASCO 2026 did not deliver a practice-changing solid-tumor cell therapy breakthrough, it did showcase encouraging early clinical evidence that next-generation CAR-T approaches may be beginning to overcome some of the barriers that have historically limited success in solid tumors.

Legend Biotech announced first-in-human clinical data for LB2102, its investigational autologous CAR-T cell therapy for patients with relapsed or refractory small cell lung cancer or large-cell neuroendocrine carcinoma — cancers with very limited treatment options and poor outcomes.

Early phase 1 results demonstrate evidence of clinical activity and a manageable safety profile. At higher dose levels, an objective response rate of 28.6% and disease control rate of 78.6% were observed, including durable responses in some heavily pretreated patients.

LB2102 is a DLL3-targeted CAR-T cell therapy with dnTGFBR2 Armor engineered to enhance activity by overcoming immunosuppressive signaling within the tumor microenvironment. The therapy is part of Legend Biotech's effort to expand CAR-T beyond blood cancers and into solid tumors. Under a 2023 deal, Novartis acquired exclusive worldwide rights to the DLL3-targeted CAR-T program, with Legend leading the ongoing U.S. phase 1 trial and Novartis responsible for broader development and commercialization.

Gilead's Kite also presented early data aimed at extending engineered cell therapies into solid tumors. Researchers from the University of Pennsylvania Perelman School of Medicine delivered an oral presentation featuring updated results from a phase 1 trial evaluating a bivalent CAR-T therapy in patients with recurrent glioblastoma.

The update included longer-term overall survival, safety, and neurologic function data from 18 patients who received a single intracerebroventricular dose of CART-EGFR-IL13Rα2 cells. After a median follow-up of 18.5 months, median overall survival was 12 months, with three patients surviving longer than 18 months. The therapy continued to demonstrate a favorable safety profile. Aside from one previously reported case of prolonged Grade 1 neurotoxicity, investigators observed no prolonged, late-onset, or unexpected toxicities during the extended follow-up period.

Shanghai-based Oricell Therapeutics gave an oral presentation highlighting a potential new benchmark for liver cancer patients who have exhausted standard therapies. 

The phase 1b BEACON trial evaluated the company’s lead asset, Ori-C101, a GPC3-targeted CAR-T therapy, in patients with advanced, heavily pretreated hepatocellular carcinoma. As of April 3, 2026, among 18 efficacy-evaluable patients, overall objective response rate (ORR) reached 50%. At the recommended phase 2 dose, ORR surged to 66.7%, with a disease control rate nearing 90%. patient achieved a complete response lasting 24 months.

On the gene therapy side, Genprex presented biomarker data from patients treated with its Reqorsa gene therapy showing that higher Trop-2 levels and lower PTEN levels were associated with longer progression-free survival in non-small cell lung cancer, supporting the potential use of these biomarkers for patient selection and precision medicine approaches. The clinical validation was derived from patients in the company’s Acclaim clinical trials.

Reqorsa, Genprex’s lead drug candidate, is designed to deliver a plasmid coding for the TUSC2 tumor suppressor gene to lung cancer cells, as >80% of lung cancers have been shown to have decreased or absent TUSC2 protein.