A new era of rna therapeutics
At Chimerna, we are leveraging our Tornado platform to develop therapies against previously untreatable diseases.
Autosomal-dominant polycystic kidney disease (ADPKD)
ADPKD is among the most common inherited form of kidney disease affecting as many as 1 in 500 people. The disease is characterized by the growth of multiple cysts in the kidney eventually leading to kidney failure. ADPKD is caused by mutations in the gene Pkd1 or Pkd2, which leads to upregulation of c-Myc, which in turn induces expression of the miR-17~92 locus. Inhibition of these microRNAs reduces cyst formation and improves renal function. We have developed Tornado-based microRNA sponges that can be specifically delivered to the kidney as a therapy to halt the formation of cysts and prevent kidney failure.
Alzheimer's disease is a progressive neurodegenerative brain disorder resulting in worsening memory, thinking, and behavior. The death rates from Alzheimer's has been steadily climbing over the decades and yet there remains no effective treatment. While the factors that cause Alzheimer's disease are still unclear, one protein, Tau, is known to form neurofibrillary tangles (NFTs) in neurons that can result in neuronal death. Additionally, these tangles can be spread from neuron to neuron through the cerebrospinal fluid. We are designing aptamers to prevent the formation of Tau NFTs and inhibit their spread to other neurons.
Parkinson's disease is a neurodegenerative disorder that primarily affects dopaminergic neurons affecting fine muscle movements. Like Alzheimer's disease, Parkinson's is thought to be caused by the formation of fibrils of alpha-synuclein that make up Lewy bodies and eventually lead to death of the dopaminergic neurons. Also similar to Alzheimer's disease, these fibrils can spread through the cerebrospinal fluid. Alpha-synuclein plays an important role in healthy neurons so Chimerna scientists are designing aptamers to specifically inhibit the fibril form, which takes on an entirely different 3D structure from the healthy protein.