Hematological malignancies are cancers that affect blood, bone marrow, and lymph nodes, and include leukemias and lymphomas. They are also referred to as blood cancers or liquid tumors. Hematological malignancies result from improper production, function, and proliferation of blood cell including lymphocytes. Depending on the specific disease and its severity, treatment of hematological malignancies may involve chemotherapy, radiotherapy, or bone marrow transplant.
miRagen’s lead program in hematological malignancy targets miR-155, a microRNA that has key roles in the differentiation, function and proliferation of blood and lymph cells. Therapeutic inhibition of miR-155 in lymphoma cells restores normal function and reduces the aberrant cell proliferation that is characteristic of cancerous cells.
Tissues and organs in the body respond to injury through a wound-healing response involving formation of fibrous scar tissue which is characterized by deposition of extracellular matrix proteins like collagen and elastin. In the setting of chronic stress however, progressive accumulation of fibrotic tissue impairs the function of vital organs like the heart, lungs, liver and kidney and is a major (and largely untreatable) contributor to morbidity and mortality.
Discoveries by miRagen researchers revealed that miR-29 is a powerful regulator of extracellular matrix production and is an attractive therapeutic target for the treatment of cutaneous and pathological fibrosis
Our Neuro-Inflammation program is initially focused on Amyotrophic Lateral Sclerosis (ALS), or Lou Gehrig’s disease. ALS is a neurodegenerative disease characterized by rapidly progressive muscle weakness and wasting throughout the body. Patients experience difficulty moving, speaking, swallowing and breathing. ALS is a severe disease and the majority of patients ultimately succumb to breathing problems, typically within 3-5 years of initial symptoms. Improvements in survival with existing drug treatments are modest and new therapeutic approaches are urgently needed.
miRagen is targeting microRNA miR-155 as a novel therapy for ALS. miR-155 is inappropriately increased in the spinal cord of patients with ALS. We believe it plays key roles in the inflammation and immune mechanisms implicated in ALS pathology and that inhibition of miR-155 has the potential to alleviate symptoms and extend survival.
miRagen’s program targets miR-92c, a microRNA linked to the regulation of blood vessel growth. Increasing blood vessel growth is an attractive revascularization approach for the treatment for chronic ischemic disorders. antimiR-92a offers a potential therapeutic to accelerate the healing process.
Target and potential indications not disclosed.
We are pursuing a diverse set of disease targets identified as having both high unmet medical need and scientific evidence of benefit from microRNA modulation. Within that context, we prioritize programs on our ability to rapidly translate the pre-clinical science into a mechanistic proof-of-concept trial in patients. We emphasize target validation in human-derived in vitro systems and industry standard models of disease. Programs that progress into human trials are designed to be accompanied by a validated set of PD biomarkers that allow us to stratify and enrich the study population. Through this approach, we seek to progressively reduce the risk of the programs by quantifying target engagement and identifying the efficacious dose prior to progression to Phase 2 trials.