Gyala Therapeutics today announced the initiation of a Phase I/IIa clinical trial of GYA01 following authorization from the Spanish Agency of Medicines and Medical Devices (AEMPS).
In this trial, GYA01 will be evaluated in patients with relapsed or refractory (R/R) acute myeloid leukemia (AML) or T-cell acute lymphoblastic leukemia (T-ALL), two diseases associated with a poor prognosis and limited therapeutic options in this clinical setting.
GYA01 is a CAR-T cell therapy, being developed to address this critical unmet medical need. With GYA01, a patient’s T lymphocytes are genetically modified to recognize and eliminate leukemic cells expressing CD84, in contrast to currently approved CAR-T therapies that target CD19 (B-cell malignancies) or BCMA (multiple myeloma). GYA01 has shown strong activity in preclinical models of AML and T-ALL, with results published in the journal Leukemia in 2025. Currently, there are no approved CAR-T therapies for either of these indications.
The study will be conducted at two leading hospital centers with extensive experience in the clinical management of acute leukemias: Hospital La Fe in Valencia, with Dr. Pau Montesinos serving as Principal Investigator and Study Coordinator, and Hospital Clínic Barcelona, a center with a long-standing track record in advanced cell therapies, with Dr. Jordi Esteve as Principal Investigator.
GYA01 will be manufactured in a decentralized manner at both sites, following the recent GMP certification of the Advanced Therapies Unit at Hospital La Fe in Valencia by AEMPS.
This is an interventional, open-label, single-arm clinical trial designed to evaluate the safety, tolerability, and preliminary efficacy of GYA01 in patients with AML and T-ALL. The study includes a dose-escalation phase followed by an expansion phase and has already begun recruiting patients.
This milestone represents a significant step in a technology transfer process that has enabled Gyala Therapeutics, as a spin-off of Hospital Clínic Barcelona and the Fundació de Recerca Clínic Barcelona–Institut d’Investigacions Biomèdiques August Pi i Sunyer (IDIBAPS), to capitalize on the experience of these institutions in the development and manufacturing of CAR-T therapies.
Gyala Therapeutics and Hospital La Fe have received €3.7 million in funding from Spain’s Ministry of Science, Innovation and Universities through the CPP 2024 program to conduct the study.
Dr. Pau Montesinos highlights the clinical relevance of the study: “This clinical trial represents a meaningful opportunity to offer new therapeutic alternatives to patients with acute leukemias who do not respond to conventional treatments. The identification of CD84 as a therapeutic target opens an innovative pathway that could extend the reach of CAR-T therapies to diseases for which effective options have not previously existed. In addition, this trial represents a new challenge for Hospital La Fe, as we have had to implement a complex accreditation process to enable manufacturing of this medicinal product at our facilities.”
Dr. Manuel Guerreiro, from Hospital La Fe and the trial’s Clinical Specialist, emphasizes that “this study is an example of cooperation between public hospitals and private companies to bring innovative therapies to patients with AML and T-ALL—very severe diseases for which there is an urgent need of new treatment strategies, such as CAR-T cells.”
Dr. Nela Klein-González, Medical Director of Gyala Therapeutics and who led the preclinical development of GYA01, notes: “The authorization to initiate this trial reflects the robustness of a preclinical program developed with a high level of scientific rigor and oriented from the outset toward future clinical evaluation. The initiation of the study will allow us to explore its therapeutic potential in a setting of high unmet medical need.”
Claudio Santos, PhD, CEO of Gyala Therapeutics, states: “The initiation of the first clinical trial of GYA01 represents a decisive step in our strategy to develop innovative cellular therapies for patients with aggressive leukemias who lack effective therapeutic alternatives. This milestone marks the company’s transition into the clinical stage and the beginning of a phase focused on the rigorous and efficient execution of the study, with the objective of generating early clinical data.”
“This clinical trial represents a meaningful opportunity to offer new therapeutic alternatives to patients with acute leukemias who do not respond to conventional treatments”