Research

Our pipeline relies on groundbreaking scientific research, physiologically relevant animal models and state-of-the-art delivery systems to enable our therapeutic.

Halt degeneration at its root

Improved animal models

Neurodegenerative diseases are notoriously difficult to model in animals because most species fail to recapitulate the full complexity of these disorders. Prion diseases are a notable exception. When mice or other susceptible species are infected with prions, they develop the same disease seen in humans, with highly reproducible disease onset and progression. This predictable time-course provides a powerful readout for determining whether a therapeutic meaningfully delays or prevents disease. Prion diseases are also biologically simpler than other dementias, as the prion protein is the sole known risk factor driving pathology. At Heprion, we are refining and expanding these unique animal models, allowing us to harness their reliability to accelerate the development and validation of our lead therapeutics.

Targeted therapeutic delivery

Since the cellular prion protein is expressed across nearly all neurons, any therapeutic for prion disease must reach broadly throughout the brain. This challenge is not unique to prion diseases as most neurodegenerative conditions require delivery methods capable of accessing large, distributed neuronal populations. At Heprion, we address this by using state-of-the-art viral delivery tools to package and transport our therapeutic to the brain. Through focused viral engineer - optimizing vector design, regulatory elements, purification and packaging procedures - we have achieved robust, brain-wide delivery that ensures our therapeutic reaches the neuronal populations most affected by these diseases.

Prion diseases are a group of invariably fatal neurodegenerative diseases with no approved treatments and a rapid decline measured in months following diagnosis. The underlying paradigm for these diseases is the misfolding of an endogenously expressed healthy protein, called the prion protein or PrPC , into an aberrant and neurotoxic conformer referred to as scrapie or PrPSc. At Heprion we leverage fundamental principles in prion biology refined through evolution, to engineer protective "rescue molecules" that directly target the root mechanism of prion diseases, thereby halting disease progression and the spread of infectious prions throughout the brain.