PIPELINE
We develop treatments applicable across multiple neurodegenerative diseases to address urgent unmet need.
ALS = Amyotrophic lateral sclerosis, FTD = Frontotemporal Dementia, AD = Alzheimer's Disease, PN = Peripheral Neuropathy, PD = Parkinson's DiseasePROGRAMS
APRTX-001: a first-in-class molecule for targeted suppression of microglial-driven neuroinflammation.
CD33 is a critical regulator of inflammatory activity in the brain and spinal cord, modulating molecular signaling pathways that lead to suppression of microglial phagocytosis and induction of a pro-inflammatory glial phenotype. Its level is consistently elevated in tissues from patients with neurodegenerative diseases, including ALS, FTD, PD, and AD.
Inhibition of CD33 in human cells and naturally occurring loss-of-function variants in the general population provide greater resilience to neurodegenerative diseases, reduced neuroinflammation, and lower levels of key pathological biomarkers linked to neural aging and degeneration.
Aperture has identified a genetic link between CD33 and progression of severe neurodegenerative diseases, established novel humanized rodent models of CD33, and developed APRTX-001 as a potent modulator of CD33 activity.
APRTX-003: a first-in-class-molecule for targeted rescue of motor neuron vulnerability and microglial-driven neurotoxicity.
MMP9 is a key regulator of neuroinflammatory activity in the brain and spinal cord, integrating extracellular matrix remodeling with microglial- and neuron-driven pathways that promote synaptic dysfunction, blood–brain barrier disruption, and motor neuron vulnerability in ALS and related disorders. Its expression and activity are consistently elevated in neurodegenerative conditions, including ALS and other disorders marked by microgliosis and matrix remodeling, where MMP9-positive microglia and neurons drive loss of perineuronal nets, axonal die-back, and neuronal apoptosis.
Genetic and pharmacologic reduction of MMP9 in preclinical models protects motor units, delays muscle denervation, reduces neuroinflammatory damage, and improves survival, establishing MMP9 as a tractable upstream driver of selective neurodegeneration rather than a mere bystander marker.
Aperture has identified a mechanistic link between MMP9-dependent microglial dysfunction and progression of severe neurodegenerative disease, established novel humanized rodent model of MMP9, and APRTX-003 as a first-in-class, selective modulator of MMP9 activity.
APRTX-002 and APRTX-005: Undisclosed
PARTNERING
We are partnering to advance new medicines through clinical development, and unlock new genetic targets for neurological and inflammatory diseases.
Pharma partnerships to advance assets through clinical trials
Biotech partnerships to unlock delivery routes and disease indications
For licensing and partnership opportunities, please contact partnering@aperturetx.com