Through transcriptional activation, RNAa therapeutics promise a revolution in our ability to modulate previously undruggable targets.
Through transcriptional activation, RNAa therapeutics promise a revolution in our ability to modulate previously undruggable targets.
DEVELOPMENT PROGRAMS
HbF Program
Sickle Cell Disease
Sickle Cell Disease (SCD) is a severe blood disorder that affects more than 5 million people around the world. SCD is an inherited disease caused by a single mutation in the HBB gene, which is responsible for producing a key component of hemoglobin. Without this component, red blood cells become misshapen and are less efficient at transporting oxygen around the body. SCD patients experience painful symptoms, require chronic hospitalisation and face early mortality. While SCD patients can be fully protected from disease symptoms, currently this is only possible by stem cell transplant which entails severe risks and to which few patients have access.
MTL-HBG
MTL-HBG is an investigational in vivo therapy for the treatment of SCD. MiNA’s treatment approach is to increase transcription of the HBG gene with an RNAa medicine, enabling patients to produce enhanced levels of fetal hemoglobin (HbF). HbF is a redundant form of hemoglobin which, when produced at sufficient levels, has been demonstrated to protect SCD patients from disease symptoms. MTL-HBG is administered in vivo and enables specific induction of HbF without the need for harmful preconditioning or complex cell engineering. MTL-HBG is currently in pre-clinical development, in which it has demonstrated induction of HbF to levels associated with protection from symptoms of SCD.
Clinical Proof of Platform
MiNA demonstrated clinical proof of platform with MTL-CEBPA, the first RNAa medicine to have entered into clinical development.
MTL-CEBPA is designed to reduce immune suppression of myeloid cells by restoring C/EBP-α protein to normal levels and has been developed as a combination therapy in cancer.
MTL-CEBPA has been evaluated in clinical trials in more than 130 subjects including 80 patients with advanced liver cancer and 50 patients with advanced solid tumor malignancies.