|
Gyala Therapeutics, a biotechnology company that specializes in developing next-generation immunotherapies to treat haematological malignancies, has announced the publication of preclinical results demonstrating the efficacy of its CAR-T therapy, GYA01, in acute myeloid leukaemia (AML) and T-cell acute lymphoblastic leukaemia (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 haematological target, opening new opportunities to expand CAR-T therapies beyond B-cell malignancies.
AML accounts for approximately 30% of all leukaemias 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 leukaemia that accounts for about 15% of paediatric 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 favourable memory phenotype and without the need for gene editing to avoid fratricide.
The study has shown that CD84 is consistently overexpressed in acute myeloid leukaemia and T-cell acute lymphoblastic leukaemia, even in high-risk subtypes, such as AML with TP53 mutation or MECOM gene rearrangement. In addition, this protein is highly expressed in other haematological cancers, such as chronic lymphocytic leukaemia and aggressive B-cell lymphomas, while its presence in non-haematological 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-haematological 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 tumour 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 Clinic in Barcelona and Hospital La Fe in Valencia, to get this therapy to patients facing aggressive types of leukaemia with few alternatives as soon as possible.”
The GYA01 programme 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 Innovación 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.
Founded in 2020 as a spin-off of Hospital Clinic Barcelona and the August Pi i Sunyer Biomedical Research Institute (IDIBAPS), Gyala Therapeutics is building on the extensive experience of these pioneering institutions in CAR-T therapies for cancer, having treated more than 500 patients with these therapies. This track record stems from the ARI Project, which gave rise to CAR-T ARI-0001, approved in Spain under hospital exemption to treat acute lymphoblastic leukaemia in adults, and CAR-T ARI-0002h, also approved under this framework to treat multiple myeloma. In addition, the scientific development of Gyala Therapeutics’ lead program, GYA01, has benefited from more than a decade of research in this field led by Dr. Manel Juan, head of the Immunology Service at Hospital Clinic Barcelona and head of the Immunogenetics and Immunotherapy of Autoinflammatory and Immune Responses Research Group at IDIBAPS.
Gyala Therapeutics is a biotechnology company focused on developing next-generation immunotherapies to treat haematological cancers with unmet medical needs.
|