Our Science
Our Focus
Neurodegenerative diseases are relentless, often fatal, and affect a range of cognitive, behavioral, and motor functions as basic as memory, thinking, speaking, walking, and breathing. A lack of accurate diagnostics has challenged therapeutic development for these conditions because our understanding of the underlying disease pathophysiology remains incomplete and there are no easily measured biomarkers that can yet predict or track the rate of progression in individual patients.
Despite significant advances in neuroscience and neurology, there is a growing need for better treatments for neurodegenerative diseases.
Approximately
An estimated
Approximately
Beyond AD and PD, there are more than 20 clinicopathologies that result in neurodegeneration.
Neurodegenerative diseases continue to burden and devastate patients, families, and society at large.
At APRINOIA, we target the respective pathological protein aggregates for 2 types of neurodegenerative diseases—tauopathies, including AD and progressive supranuclear palsy (PSP), and α-synucleinopathies, including PD and Lewy body dementia (LBD).
Tauopathies
Tauopathies are neurodegenerative diseases caused mainly by aggregated tau proteins. There are native forms of tau, which are essential in maintaining microtubule stability in neurons and brain health, and toxic forms, which accumulate in tauopathies and contribute to neuronal death.
Additionally, toxic tau proteins can further “infect” its healthy counterparts. As a result, toxic tau can be transmitted between neurons and spread across brain regions, causing progressive brain degeneration. Depending on the tauopathy, patients may experience cognitive and behavioral deficits along with motor impairments in later disease phases.
Major tauopathies include AD, PSP, and frontotemporal
dementia (FTD).
α-synucleinopathies
Alpha-synucleinopathies are neurodegenerative disorders caused by aggregation of abnormal α-synuclein proteins (αSyn) with formations of insoluble fibrils in neurons and glia.
Pathogenetically, these diseases are usually accompanied by disturbances in αSyn metabolism, causing high αSyn levels and αSyn accumulations.
Abnormal αSyn species can be associated with degeneration in multiple systems, causing a broad spectrum of clinical syndromes, including dementia and movement disorders. Major alpha-synucleinopathies include PD, LBD, and multiple system atrophy (MSA).
PDD=Parkinson’s disease Dementia // ALS=amyotrophic lateral sclerosis // CBD=Corticobasal degeneration
// Pick/bvFTD=Pick’s Disease, behavior variant frontotemporal demential.
Our Solution
In harnessing the power of precision neuroscience, we are developing several different diagnostic and therapeutic platforms to detect and target neurodegenerative disease pathologies, leveraging both small molecules and biologics.
Diagnostics
APRINOIA provides first-in-class solutions in noninvasive precision diagnostics for neurodegenerative diseases. Our positron emission tomography (PET) tracers target pathological protein aggregates and reveal their distribution in the brain. This will provide key information for clinicians in the differential diagnosis of distinct neurodegenerative diseases. Our PET tracers also provide a quantifiable way of monitoring disease progression and thus can be applied in personalized investigations on the effectiveness of current and novel treatments.
Therapeutics
Lead candidates arise through our screening platforms to precisely neutralize the pathological protein aggregates of the underlying diseases. Our small molecule portfolio tackles the diseases from inside the neurons by removing distinct protein aggregates through proteasomal or autophagic degradation (degraders). Meanwhile, our antibodies protect the extracellular spaces and stop the “spreading” of pathological protein aggregates to slow or even prevent disease progression.
