Gyala Therapeutics unveils first-in-class CAR-T therapy targeting CD84 showing strong preclinical efficacy in hard-to-treat leukemias

Gyala Therapeutics has announced the publication of preclinical results demonstrating the efficacy of its CAR-T therapy, GYA01, in acute myeloid leukemia (AML) and T-cell acute lymphoblastic leukemia (T-ALL). The study, published in Leukemia, is the first to present preclinical data with CAR-T cells targeting the CD84 protein. The findings validate GYA01 as a promising novel therapy and highlight CD84 as a broad hematological target, opening new opportunities to expand CAR-T therapies beyond B-cell malignancies.

AML accounts for approximately 30% of all leukemias diagnosed in adults, with roughly 145,000 new cases per year worldwide. Although many patients achieve initial remission with chemotherapy, nearly half relapse after first-line treatment. T-ALL is a very rare subtype of lymphoblastic leukemia that accounts for about 15% of pediatric ALL cases and up to 25% of ALL in adults. In children, survival rates exceed 90% with chemotherapy, but in adults long-term survival is much lower, at around 40%. Currently, for both these rare and very aggressive cancers, patients who relapse or do not respond to initial treatment have very few therapeutic options.

The main findings of the study on GYA01, the CAR-T therapy targeting CD84, are:

• Specific potent cytotoxicity against AML, T-ALL and aggressive B-cell lymphoma cell lines, as well as primary leukemic cells from patients.
• Significant efficacy of CAR-T cells in animal models using both cell lines and leukemic cells from patients, achieving disease eradication and prolonging survival.
• A successful expansion of CAR-T cells from both healthy donor and patient samples, with a favorable memory phenotype and without the need for gene editing to avoid fratricide.

The study has shown that CD84 is consistently overexpressed in acute myeloid leukemia and T-cell acute lymphoblastic leukemia, even in high-risk subtypes, such as AML with TP53 mutation or MECOM gene rearrangement. In addition, this protein is highly expressed in other hematological cancers, such as chronic lymphocytic leukemia and aggressive B-cell lymphomas, while its presence in non-hematological healthy tissues is limited. This expression profile positions CD84 as a target with high potential for expanding the use of CAR-T therapies beyond the context of B-cell neoplasms, where approved treatments already exist.

“The preclinical evidence obtained confirms that GYA01 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. In these indications, the main challenge for CAR-T therapies is the lack of specific tumor targets,” explains Dr. Nela Klein-Gonzalez, medical director of Gyala Therapeutics and author of the article.

Claudio Santos, PhD, CEO of Gyala Therapeutics, comments that the company’s next goal “is to start a clinical trial with GYA01 in the second half of 2025 at Hospital Clínic de Barcelona and Hospital La Fe in Valencia, to get this therapy to patients facing aggressive types of leukemia with few alternatives as soon as possible.”

The GYA01 program builds on the expertise of Hospital Clinic Barcelona, a pioneering institution in the CAR-T field, and the leadership of Dr. Manel Juan, a driving force in CAR-T development in Spain, as well as the important contribution of Hospital La Fe, which conducted key expression analyses of CD84 in AML patient and healthy bone marrow samples.

To launch the clinical trial, Gyala Therapeutics has the backing of a €3 million investment round with the participation of Invivo Partners (the company’s founding venture capital), Nara Capital and CDTI Innovation though the Innvierte program). Gyala exemplifies how company creation by experienced investors, building on Spain’s academic excellence, can translate breakthrough science into clinical innovation.