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OUR PIPELINE

Program Target Discovery Pre-clinical Phase I Phase II Phase III More Info
CB-01 Lysosomal Storage Disease with High Unmet Need    
More Info
CB-01 is a discovery-stage program focused on a lysosomal storage disorder with significant unmet medical need.

CB-01 Program
Program Target Discovery Pre-clinical Phase I Phase II Phase III More Info
CB-01 Lysosomal Storage Disease with High Unmet Need    
More Info
CB-01 is a discovery-stage program focused on a lysosomal storage disorder with significant unmet medical need.

CB-01 Program
Program Target Discovery Pre-clinical Phase I Phase II Phase III
CB001 Lysosomal Storage Disease with High Unmet Need    

CB001 Program Overview

CB001 is a discovery-stage program focused on a lysosomal storage disorder with significant unmet medical need. The program aims to address disease progression at its molecular root cause.

Program Target Discovery Pre-clinical Phase I Phase II Phase III
CB001 Neuronopathic Gaucher Disease (Type II and III)    
CB001 Program Overview

CB001 is a pre-clinical stage program focused on a neuronopathic Gaucher disease and GBA-Parkinson’s with significant unmet medical need. The program aims to address disease progression at its molecular root cause.

Program Target Discovery Pre-clinical Phase I Phase II Phase III
CB001 Neuronopathic Gaucher Disease (Type II and III)    
GBA-Parkinson's Disease    
CB001 Program Overview

CB001 is a pre-clinical stage program focused on a neuronopathic Gaucher disease and GBA-Parkinson’s with significant unmet medical need. The program aims to address disease progression at its molecular root cause.

Gaucher is a protein folding disease with deep biological connections to Parkinson’s

GBA1 mutations produce misfolded GCase protein that is degraded before reaching the lysosome. The resulting loss of enzyme activity drives both lysosomal storage dysfunction and the alpha-synuclein aggregation loop implicated in Parkinson’s pathology.
The reality for Gaucher and Parkinson’s patients reveals an urgent unmet need.

6,000

GD1 patients in the US managed by ERT, which does not address neurodegeneration risk

~10x​

Increased risk of Parkinson’s disease for GD1 patients vs. general population (~5x for carriers)

$2.1B → $3.5B​

Global Gaucher disease market size, 2025 forecast to 2032

No treatments exist for neuronopathic GD2 or GD3 (several hundred US patients each). GD2 patients die in infancy; GD3 patients can live into midlife with ERT but face inevitable cognitive decline.

ERT: enzyme replacement therapy. Source: coherentmarketinsights.com, Gaucher Disease Treatment Market, Dec 2025.

Why our solution? Non-inhibitory small molecule chaperone that enables both CNS efficacy and convenience

CB-001 binds an allosteric site to stabilize mutant GCase without inhibiting its active site, restoring proper protein folding and function. It is designed for CNS penetration and delivered as an oral, once-daily dose, eliminating the infusion burden of current therapies.

Increased Success Rates

By optimizing trial enrollment to patients predicted to respond, StratiVar increases the chance of a successful trial and FDA approval.

Accelerated Development

StratiVar streamlines the process of clinical trial design through the application of high-quality functional data.

Personalized patient population

By establishing the probability of drug-sensitivity and drug-resistance for all tested variants, StratiVar ensures that only the most-likely to respond patients are enrolled for clinical trials.
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