Gyala Therapeutics’ CAR-T Leukemia Therapy Shows Promise, Heads for Clinical Trial

Destruction of lymphoblasts. Conceptual 3D illustration of treatment of acute lymphoblastic leukemia | Image Credit: © Dr_Microbe - stock.adobe.com

Preclinical results have been published in the journal Leukemia that show the efficacy of a chimeric antigen receptor T cell (CAR-T) therapy developed by Gyala Therapeutics, GYA-01, against acute myeloid leukemia (AML) and T-cell acute lymphoblastic leukemia (T-ALL) (1,2). According to Gyala Therapeutics, the published study is the first that presents preclinical data with CAR-T cells targeting the CD84 protein, highlighting that protein as a broad hematological target that may expand CAR-T therapy opportunities beyond B-cell malignancies.

What did the study reveal?

Some of the main findings of the study included GYA-01’s reported specific, potent cytotoxicity against AML, T-ALL, and aggressive B-cell lymphoma cell lines; a significant efficacy of CAR-T cells in animal models, using both cell lines and leukemic cells from patients; and successful expansion of CAR-T cells from both healthy donor and patient samples, without the need for gene editing (1).

A press release from Gyala Therapeutics said that AML accounts for approximately 30% of leukemias diagnosed in adults—equating to more than 145,000 new cases worldwide per year—with T-ALL, while comparatively rare, accounting for up to 25% of acute lymphoblastic leukemia (ALL) diagnoses in adults and 15% in children (1).

Why is this therapy potentially important?

Few therapeutic options are available. In the case of AML, many patients initially achieve remission with chemotherapy, but almost half of those relapse following first-line treatment (1). Regarding T-ALL, more than 90% of pediatric patients survive with chemotherapy, but long-term survival in adults is much lower, at approximately 40%, according to Gyala Therapeutics.

“The preclinical evidence obtained confirms that GYA-01 is a highly promising CAR-T therapy, supports CD84 as a pan-hematological target, and establishes a solid foundation for advancing to clinical trials in patients with AML and T-ALL,” Nela Klein-Gonzalez, MD, PhD, medical director of Gyala Therapeutics and author of the Leukemia article, said in the press release (1). “In these indications, the main challenge for CAR-T therapies is the lack of specific tumor targets.”

Are other treatments available?

Another treatment being developed for T-ALL, March Biosciences’ MB-105, was granted orphan drug designation by FDA in January 2025 for treating relapsed/refractory CD5-positive T-cell lymphoma (3).

In BioPharm International®’s Next-Generation Biotherapeutics eBook, published in April 2025, CAR T-cell therapies were spotlighted as a major area of focus for cancer treatment, but primarily in the relatively small sector of blood cancers (4).

What’s next for CAR-T therapies?

According to Shada Warreth, Global Partnerships Implementation senior manager at NIBRT and PhD Candidate at TU Dublin, Ireland, the next impactful innovation in this area may be allogeneic CAR-T therapies. These utilize T cells from healthy donors or such sources as umbilical cord blood or induced pluripotent stem cells to provide scalable, off-the-shelf treatments with broader applicability and reduced waiting times compared to autologous CAR-T therapies (5).

In the Gyala Therapeutics press release, the company said its next goal is to start a clinical trial for GYA-01 before the end of 2025 in two hospitals in Spain (1). To launch that trial, the company is backed by a €3 million (US$3.5 million) investment round with participation from Invivo Partners, Nara Capital, and CDTI Innovación.

References

1. Gyala Therapeutics. Gyala Therapeutics Unveils First-in-Class CAR-T Therapy Targeting CD84 Showing Strong Preclinical Efficacy in Hard-to-Treat Leukemias. Press Release. Sept. 15, 2025.
2. Pérez-Amill, L.; Armand-Ugón, M.; Val-Casals, M.; et al. A Novel Chimeric Antigen Receptor T-Cell Therapy Targeting CD84 for the Treatment of Acute Myeloid and T-Cell Lymphoblastic Leukemias. Leukemia 2025 (open access). DOI: 10.1038/s41375-025-02705-4
3. March Biosciences. March Biosciences Receives FDA Orphan Drug Designation for MB-105, a First-in-Class CD5 CAR-T Cell Therapy, for T-Cell Lymphoma. Press Release. Jan. 28, 2025.
4. Haigney, S. Emerging Treatments for Cancer. BioPharm International Next-Generation Biotherapeutics eBook, 2025 April.
5. Warreth, S. The Manufacturing Journey of CAR-T Cellular Therapy—An Overview. BioPharmInternational.com, Sept. 8, 2025.