Aptadir Ce-49 / Aptadir Therapeutics - LARVOL DELTA

RNA-GUIDED SELECTIVE DNMT1 INHIBITION RESTORES MYELOID DIFFERENTIATION AND OUTPERFORMS AZACITIDINE IN TET2-MUTANT MYELODYSPLASTIC SYNDROMES (EHA 2026) - "Patients with TET2-mutant MDS commonly receive hypomethylating agents (HMAs), such as azacitidine and decitabine, which broadly inhibit DNMT activity...Results Selective DNMT1 inhibition by aptaDiR_Ce-49_sh_v1 induced robust granulocytic maturation and restored myeloid differentiation in TET2-deficient hematopoiesis, exceeding the effects observed with azacitidine ( Fig.1 )...Data are presented as mean ± SD (n=3). Statistical analysis was performed using one-way ANOVA (**p<0.01, ***p<0.001, ****p<0.0001)."

Hematological Disorders • Hematological Malignancies • Myelodysplastic Syndrome • ASXL1 • ITGAM • PTPRC • TET2

Launch of Aptadir Therapeutics With a Novel Class of RNA Inhibitors (Businesswire) - "Aptadir Therapeutics...announces its launch and the appointment of Giovanni Amabile as Executive Chairman and acting CEO...Aptadir was founded by Dr Annalisa Di Ruscio and Dr Vittorio De Franciscis, both leaders in the field of RNA biology and DNA methylation, alongside Prof. Daniel Tenen, an expert in MDS and leukaemia, and Prof. Marcin Kortylewski, an expert in oligonucleotide immunotherapeutics...The first programme, Aptadir Ce-49 is being developed in MDS and the aim is to enter the clinic by the end of 2025....Aptadir has received $1.6m in pre-seed funding from the EXTEND initiative to optimize its lead asset, Aptadir Ce-49. The Company is the first to receive funding from EXTEND, which is the Italian National Technology Transfer Hub wholly dedicated to the biopharmaceutical sector for the development of new therapeutic approaches."

Financing • New molecule • New trial • Hematological Malignancies • Myelodysplastic Syndrome • Oncology