Technology

Key Mechanisms to Halt Degeneration

Calcium imbalance and oxidative stress are major contributors to cell death in neurodegenerative diseases. Stable calcium levels inside cells are crucial for healthy cellular function, controlling important processes like muscle contraction and how nerve cells communicate. Disturbances in calcium regulation have been linked to the progression of several disorders.

Oxidative stress and the excessive production of reactive oxygen species are closely linked to the biological processes that drive neurodegenerative diseases such as Amyotrophic Lateral Sclerosis, Alzheimer’s, and Parkinson’s, as well as cardiovascular disease, cancer, and diabetes. 

Addressing these core disease mechanisms offers a promising way to slow down or stop the progression of degenerative conditions that currently have very limited treatment options.

Dual mechanism of action:
Ryanodine Receptor Stabilization & ROS Scavenger

Ryanodine Receptor Stabilization

The ryanodine receptor (RyR) is a key channel inside cells that helps release calcium from an internal storage area called the endoplasmic reticulum. This process is essential for many cell functions, especially in active cells such as nerve cells, photoreceptors, and muscle cells.

To work properly, the RyR channel needs to be well controlled. The protein FKBP12 plays an important role in this balance — it helps keep the channel stable and prevents calcium from leaking out when it shouldn’t.

ROS Scavenger

MP compounds naturally help neutralize harmful reactive oxygen species (ROS), highly unstable molecules that can damage cells.

An imbalance between calcium inside the cell and oxidative stress creates a vicious cycle that drives neurodegenerative diseases.

By preventing calcium leaks from RyR channels—often caused by oxidation under pathological conditions—MP molecules restore healthier cell function.

FKBP12 acts like a molecular switch, controlling RyR activity by changing its structure through reversible, non-covalent interactions.

Dysfunctions in RyR-mediated calcium regulation have been linked to the development of several diseases affecting the heart, muscles, brain, eyes, lungs, and pancreas.

MP molecules are innovative FKBP12 ligands designed to prevent harmful calcium leaks from RyR channels, which may result from mutations, abnormal phosphorylation, oxidative stress, or reactive oxygen species. By restoring the FKBP–RyR interaction, these compounds stabilize the closed state of the channel and help re-establish healthy intracellular calcium balance.

Through RyR channel stabilization and ROS inhibition, MP compounds counteract pathological processes, restoring cellular balance and preventing cell death.

MP compounds

MP compounds are a patented innovative family of molecules designed with advanced computational tools to target FKBP12 and stabilize its interaction with the ryanodine receptor. They have been optimized for drug development, showing excellent properties such as stability, safety, and the ability to cross the blood-brain and blood-retinal barriers.

From over 35,000 molecules designed in silico, 141 were synthesized, and 20 have already been tested. Miramoon has identified three lead candidates, now advancing in ophthalmology, muscle and cardiac disorders, and central nervous system diseases.