MiNA Therapeutics Limited has announced breakthrough pre-clinical results for its small activating RNA (RNAa) therapy targeting sickle cell disease, with data showing unprecedented levels of fetal hemoglobin induction that could transform treatment approaches for this genetic disorder.
The company's lead candidate, MTL-HBG, demonstrated remarkable efficacy in pre-clinical studies using erythroid progenitor cells derived from four patients with sickle cell disease. The therapy induced fetal hemoglobin (HbF) to 62% as a proportion of total hemoglobin, representing an absolute increase of 45% HbF compared to negative controls.
Novel RNA Activation Approach
MTL-HBG represents a revolutionary approach to treating sickle cell disease by directly targeting the gamma globin gene (HBG) to increase transcription and enable enhanced production of fetal hemoglobin. This compensatory form of hemoglobin, when induced to sufficient levels, protects patients from recurrent vaso-occlusive crises and progressive organ damage associated with sickle cell disease.
"The extraordinary levels of fetal hemoglobin induced by MTL-HBG underscore its transformational therapeutic potential," said Robert Habib, CEO of MiNA Therapeutics. "This new pre-clinical data further increases our confidence of translating the potential therapeutic benefits of MTL-HBG to patients with sickle cell disease."
Pancellular Activity and Clinical Advantages
The studies revealed that MTL-HBG's therapeutic effect was pancellular, with 82% of erythroid progenitor cells expressing HbF. This broad cellular response suggests the potential for comprehensive therapeutic benefit across the patient's red blood cell population.
Significantly, MTL-HBG is administered in vivo without requiring harmful pre-conditioning treatments or complex cell engineering procedures that characterize current gene therapy approaches. The drug comprises an RNAa payload that directly targets the HBG gene, encapsulated in NOV340 liposomes for targeted delivery.
Translational Potential
The pre-clinical studies were conducted using reduced drug doses designed to replicate the level of cellular uptake anticipated in patients following systemic administration, enhancing the translational relevance of the findings. MTL-HBG has previously demonstrated promising biodistribution and in vivo activity in non-human primates and is currently advancing through IND-enabling studies.
Conference Presentation
The comprehensive data will be presented at the European Hematology Association 2025 Congress on June 14, 2025, at 5:30 p.m. CEST in an oral presentation titled "Small activating RNA-mediated induction of HBG via liposome delivery for in vivo treatment of sickle cell disease." The presentation will be available on the MiNA website following the congress.
MiNA Therapeutics positions itself as the leader in small activating RNA therapeutics, developing a proprietary pipeline of medicines for genetic diseases while collaborating with pharmaceutical companies to expand RNA-based medicine applications across therapeutic areas.
