Stress granules facilitate the formation of biomolecular condensates which have attracted immense excitement from the scientific community and drug companies over recent years because they have the potential to completely revolutionize drug discovery. Biomolecular condensates, a general term reflecting compartments within a cell that dynamically form without any encapsulating membranes, are involved in almost every aspect of cellular physiology and human health.  

Stress Granules are a special type of biomolecular condensate that form when the cell is stressed. Stress granules cause the cell to halt its normal function by directing its natural resources to combat the immediate insult or stressor. While this is beneficial strategy in the short term, persistent stress granules cause disease. This occurs in part because persistent stress granules promote formation of insoluble “sticky” protein aggregates that act as a trap to sequester multiple biologically important proteins. These protein aggregates interfere with normal cellular processes and eventually lead to toxicity and killing of the cell. By preventing and removing these pathological protein aggregates Aquinnah technology can halt and even reverse the disease processes.

The location of “sticky” protein aggregates determines the symptoms. Accumulation in the spinal cord results in motor neuron dysfunction and ALS, while accumulation in the hippocampus causes memory loss and Alzheimer’s disease.

The ‘holy grail’ in harnessing biomolecular condensates for drug development is to identify small molecules that can be delivered as a pill and selectively interfere with the formation of pathological biomolecular condensates to prevent neuronal death and dysfunction.

Aquinnah Pharmaceuticals, the first company to realize the potential of stress granules for drug development, has years of experience developing small compounds which selectively target various pathologic biomolecular condensates.

Our platform has proven immensely successful – where we are able to show in vivo efficacy for our compounds in some of the most aggressive animal models of ALS and Alzheimer’s disease. These successes are driving the advancement of our programs towards clinical trials.

Aquinnah’s unique scientific approach has been recognized nationally by receiving nine highly competitive peer-reviewed grants, two from the ALS Association, four from the National Institute of Health, two from the Alzheimer’s Association and Rainwater Foundation Tau Consortium and one from the Massachusetts Life Science Center.

Aquinnah's therapeutic drug development compounds adhere to rigorous standards, demonstrating potency, oral (pill) bioavailability, brain uptake and demonstrated efficacy in animal models of neurodegenerative diseases, where our compounds exhibit robust and beneficial effects. Patient clinical trials will be forthcoming, following successful completion of studies required by the FDA.

These outcomes serve as compelling validation for Aquinnah's drug discovery pipeline. Our deep understanding of the molecular processes driving neurodegenerative diseases, coupled with our globally acclaimed chemistry, biology, and development team, which has empowered Aquinnah to progress towards the clinic with first in class compounds directed against these devastating diseases.

Both Dr. Larsen and Dr. Wolozin are very optimistic about the future of our pipeline to imminently demonstrate therapeutic utility in our lead programs for ALS and Alzheimer’s diseases.

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