Our Science

We utilize patient biology, biomarker-based screening, and advanced data analytics to de-risk clinical translation.

Patient Inspired,
ML/AI-Powered

A groundbreaking discovery engine to facilitate clinical translation and accelerate CNS drug development.

Our proprietary platform combines human patient iPSC-derived models of brain disease, large-scale biology,  and the computational tools of deep-learning and network medicine to identify therapeutic candidates that halt, prevent, or reverse critical molecular events that lead to synaptic network dysfunction and
neurodegeneration.

Patient-derived iPSC Organoids

Leverage an understanding of human disease biology to identify clinically translatable disease phenotypes.

Biomarker-based Screening

Identify, validate, and de-risk
targets and hits across multiple
biological endpoints.

Value creation

Discover and develop best-in-class novel and repurposed drugs.

AI/ML Powered

Integrating deep learning and network medicine to usher in a new era in CNS drug discovery.

Features

/Platform Features

Reproducibly capture human disease pathophysiology in a high throughput format.

Amenable to both phenotypic and target-based screening.

Drive compound discovery with clinically translatable disease endpoints.

Rapid discovery of first and/or best in class therapeutic candidates.

Patient Inspired

Patient-inspired drug discovery and development involves actively engaging and incorporating patient perspectives throughout the process, aiming to create more effective and patient-centric treatments that address real-world needs and challenges.

AI/ML-Powered

AI/ML powered drug discovery and development involves using artificial intelligence and machine learning to analyze vast amounts of data, predict drug efficacy, identify potential candidates, and optimize clinical trials, ultimately accelerating the process of finding new and effective treatments for improved patient outcomes.

Proprietary

Revolutionary

Efficient

Effective

Currently focused on drug discovery for Parkinson’s disease (PD)

01

PD Pathophysiology

Human PD patient derived midbrain organoids recapitulate key elements of PD pathophysiology
including dopamine neuron degeneration and synaptic dysfunction.

02

Genetic Entry Points

Utilize familial PD genetic entry points into dysfunctional autophagy/lysosomal and
mitochondrial/oxidative stress pathways for expansion into the sporadic PD population.

03

Underlying Disease Processes

Phenotypic screening of targeted small molecule libraries with clinically translatable endpoints to
identify compounds that ameliorate the underlying disease processes.

04

Design better clinical trials

Identify and define key molecular signatures to refine clinical trial endpoints and patient selection.

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