Recent developments in the CAR-T field have been significant, with several in vivo CAR-T therapies reporting encouraging data and domestic pipelines entering initial validation phases. Future focus will be on larger sample sizes, AUC, durability of efficacy, and long-term safety data. Key milestones include the first global submission of an autologous CAR-T for an autoimmune disease and the first approval for a solid tumor indication.
In Vivo CAR-T Data Shows Promise, Domestic Pipelines Begin Validation
At the ASCO 2026 and EHA 2026 conferences, Kelonia's KLN-1010 and Legend Biotech's LB2501 presented updated results, both demonstrating excellent efficacy. For KLN-1010 in MM patients with over 4 months of follow-up (n=6), the ORR was 100%, with 67% achieving sCR and a 100% MRD-negative rate. LB2501 at DL2 (n=6) in BNHL patients showed a 100% ORR and 83% CR, with no grade 3 or higher CRS observed. Pharmacokinetically, the Cmax for KLN-1010 and LB2501 reached 0.7-1.1×10^5 and 1.1×10^5, with Tmax at 18 and 15 days, respectively. Based on short-term efficacy and PK data, both KLN-1010 and LB2501 have reached or even surpassed levels comparable to autologous CAR-T therapies targeting the same antigens. Further data on larger cohorts, AUC, sustained efficacy, and long-term safety are awaited. Additionally, recent positive early-stage data has been disclosed for several other domestic candidates, including Gracell Biotech's GT801, WestGene's WGb-0301, Qihan Biotech's dual-target in vivo CAR-T, JiuKe RunJia's Arnovie101, and JiaChen XiHai's JCXH-213, marking the entry of domestic pipelines into the preliminary validation stage.
KYV-101 Initiates Rolling BLA Submission, Signaling Autoimmune Disease Breakthrough
On May 12, Kyverna Therapeutics initiated a rolling Biologics License Application (BLA) submission to the FDA for KYV-101 for the treatment of Stiff Person Syndrome (SPS). This represents the first global CAR-T therapy submission for an autoimmune disease. KYV-101 is a fully human, autologous CD19-targeted CAR-T. In April 2026, Kyverna presented primary results from the pivotal KYSA-8 clinical trial at the American Academy of Neurology annual meeting, showing that KYV-101 demonstrated statistically significant and durable clinical benefit across all primary and secondary endpoints at 16 weeks, with reversal of disability scores following a single infusion. Through week 16 and the last follow-up, all SPS patients remained off other immunotherapies, and the therapy was well-tolerated. Furthermore, Cartesian Therapeutics' Descartes-08 (targeting BCMA) and Bristol Myers Squibb's Zola-cel (targeting CD19) are both in Phase III clinical trials. Descartes-08 is expected to submit a BLA for myasthenia gravis next year, while pivotal clinical data for Zola-cel in systemic lupus erythematosus (SLE) is anticipated next year.
CT041 Approved in China, Filling CAR-T Void in Solid Tumors
On June 22, the NMPA approved the marketing of CARsgen Therapeutics' Zevor-cel (CT041) for the treatment of CLDN18.2-positive, HER2-negative advanced gastric/gastroesophageal junction adenocarcinoma after failure of at least two prior lines of therapy. CT041 has become the world's first CAR-T therapy approved for a solid tumor indication. CT041 is an autologous, humanized CAR-T targeting CLDN18.2. The approval was based on positive results from the confirmatory Phase II study (CT041-ST-01) conducted in China for advanced gastric/gastroesophageal junction adenocarcinoma. In the mITT (modified intent-to-treat) population, the CT041 group showed a median progression-free survival (mPFS) of 4.37 months (vs. 1.84 months, HR=0.304) and a median overall survival (mOS) of 8.61 months (vs. 5.49 months, HR=0.601). The therapy also demonstrated a favorable safety profile, with no ICANS or grade 4/5 CRS observed.
Potential risks include the failure of innovative drug development, intensifying market competition, and challenges in the commercialization of novel therapeutics.
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