Early Data Show Dual-Targeting CAR-T Agent CTA313 Stabilizes Progressive Multiple Sclerosis

Early phase 1 data presented at the 10th Joint ACTRIMS-ECTRIMS Meeting (MSToronto2026), held October 21-23, 2026, in Toronto, Ontario, Canada, show that CTA313, an allogeneic, dual-targeting CAR-T cell therapy, was associated with manageable safety and early signs of clinical stabilization in patients with refractory progressive multiple sclerosis (MS). The findings come from the first 3 patients treated in an ongoing, open-label trial.¹

CTA313 was developed by Imviva Biotech using the company's proprietary ANSWER platform, an allogeneic, or “off-the-shelf,” CAR-T approach derived from healthy donor cells rather than a patient's own T cells. Jan Davidson-Moncada, MD, PhD, Imviva Biotech's chief medical officer and a co-author on the abstract, said when the FDA cleared the company's broader autoimmune disease trial in June 2026 that an off-the-shelf approach “means eligible patients could potentially receive treatment more quickly, without the delays that can be life-threatening for those facing rapidly progressing disease, while also reducing their burden by eliminating the need for apheresis.”^1,2

The ongoing phase 1 study (NCT07337785) is evaluating CTA313 in patients with refractory progressive MS who had already failed other disease-modifying therapies, including anti-CD20 antibodies and S1P modulators. Led by Dai-Shi Tian, MD, PhD, a professor and neurologist in the Department of Neurology at Tongji Hospital, patients receive a single infusion of CTA313, dosed at 7 to 10x10⁶ CAR-positive cells/kg, following fludarabine and cyclophosphamide lymphodepletion. Key endpoints include safety, cellular kinetics, and clinical measures such as the Expanded Disability Status Scale (EDSS), the Timed 25-Foot Walk Test, the 9-Hole Peg Test, and cerebrospinal fluid oligoclonal bands.

As of April 30, 2026, 3 patients, 2 with secondary progressive MS and 1 with primary progressive MS, had evaluable data. Their median age was 39 years, and median baseline EDSS was 6.0. At month 3, 2 patients showed an EDSS improvement from 6.0 to 5.5, while the third remained stable at 6.0.¹

Timed functional tests improved in all 3 patients: the Timed 25-Foot Walk Test improved by 36.0% and 36.9% in the 2 patients with secondary progressive MS, and 9-Hole Peg Test completion times for the dominant hand improved by up to 30.6%. Cerebrospinal fluid oligoclonal bands converted to negative by day 28 in 2 of the 3 patients.

Grade 1 cytokine release syndrome occurred in all 3 patients, and no cases of neurotoxicity or graft-versus-host disease were observed. CAR-T cell expansion was detected by quantitative PCR, with a median peak concentration of 16,445 copies per microgram of genomic DNA between days 7 and 14.

Unlike relapsing MS, which is driven largely by peripheral immune activation, progressive MS is associated with inflammation confined within the central nervous system, including long-lived plasma cells that existing anti-CD20 therapies are thought to reach poorly. Researchers have proposed that CAR-T cells, which can cross the blood-brain barrier, may reach these compartmentalized immune cells in ways that current disease-modifying therapies cannot.³

CTA313 joins a growing field of CAR-T approaches under investigation for progressive MS, including Kyverna Therapeutics' autologous CD19-directed KYV-101 and IASO Biotechnology's BCMA-directed CT103A, both of which have reported early case-level data in small cohorts. CTA313 differs from those programs by targeting both CD19 and BCMA simultaneously and by using donor-derived, rather than patient-derived, T cells.

The investigators cautioned that the findings are preliminary, based on only 3 patients and a 3-month observation period, and that longer follow-up in a larger cohort is needed to evaluate how durable and consistent these early clinical observations are.¹

For more of NeurologyLive's coverage from the 10th Joint ACTRIMS-ECTRIMS Meeting (MSToronto2026), click here.

REFERENCES
1. Shang K, Qin C, Kangqi B, et al. Safety and preliminary efficacy of CTA313 (CD19/BCMA UCAR-T) in patients with progressive multiple sclerosis: early results from a phase 1 study. Abstract 38. Presented at: 10th Joint ACTRIMS-ECTRIMS Meeting (MSToronto2026); October 21-23, 2026; Toronto, Ontario, Canada.
2. Imviva Biotech receives FDA investigational new drug approval for CTA313, a dual-targeted CD19/BCMA allogeneic CAR-T cell therapy for autoimmune diseases. News release. Imviva Biotech. Published June 9, 2026. Accessed October 20, 2026. https://www.biospace.com/press-releases/imviva-biotech-receives-fda-investigational-new-drug-approval-for-cta313-a-dual-targeted-cd19-bcma-allogeneic-car-t-cell-therapy-for-autoimmune-diseases
3. Conway S, Galetta K. Chimeric antigen receptor T-cell therapy for progressive multiple sclerosis: rationale, evidence, and therapeutic potential. Practical Neurology. 2026.