A Pipeline of First-in-Class
Medicines for Neurodegeneration
We are systematically applying the power of the Cognate-AI™ platform to create a portfolio of transformative therapies for diseases of the central nervous system. Our focus is on validated targets that have historically been considered "undruggable" with conventional biologics due to the blood-brain barrier. Each program in our pipeline represents a potential breakthrough for patients and a significant value creation opportunity.
Interactive Pipeline Overview
A comprehensive view of our systematic approach to developing transformative CNS therapeutics
Development Stages
| Program | Target(s) | Indication | Development Stage | Details |
|---|---|---|---|---|
| AET-101 | Pathogenic α-Synuclein & Transferrin Receptor | Parkinson's Disease | Preclinical (IND-Enabling) | |
| AET-102 | Undisclosed CNS Target & BBB Receptor | Undisclosed Neurodegenerative Disease | Discovery |
AET-101
First-in-class bispecific antibody designed to cross the blood-brain barrier and neutralize toxic alpha-synuclein oligomers.
Timeline: IND filing targeted for 2025
Indication: Parkinson's Disease
Key Features
Lead Program Deep Dive: AET-101
A Novel Approach to Parkinson's Disease
AET-101
First-in-Class Bispecific Antibody
The Unmet Need
Parkinson's Disease (PD) is a progressive neurodegenerative disorder that affects millions of people worldwide, with prevalence expected to double in the coming decades. There are currently no available treatments that can halt or reverse the underlying progression of the disease.
A primary pathological driver of PD is the misfolding, aggregation, and cell-to-cell spread of the protein alpha-synuclein (α-synuclein). Toxic oligomeric forms of this protein are believed to cause synaptic dysfunction and neuronal death, leading to the motor and non-motor symptoms of the disease.
Our Solution
AET-101 is a first-in-class, humanized, bispecific IgG1 antibody designed entirely by the Cognate-AI™ platform. It is engineered to perform two critical functions: first, to efficiently cross the blood-brain barrier by targeting the transferrin receptor, and second, to selectively bind and neutralize the toxic oligomeric forms of α-synuclein that propagate disease within the brain.
Key Preclinical Data Highlights
Brain Exposure
Increase vs. non-BBB-crossing antibody
Target Engagement
Reduction in α-synuclein pathology
Safety Profile
No adverse findings in tox studies
Motor Function
Significant improvement in PD models
Superior Brain Exposure
Demonstrated a greater than 30-fold increase in brain exposure compared to a non-BBB-crossing monospecific anti-α-synuclein antibody in non-human primate studies. This level of brain penetration is critical for achieving a therapeutic effect.
Robust Target Engagement
Effectively engaged and reduced soluble and insoluble α-synuclein pathologyin the brains of a transgenic mouse model of Parkinson's Disease, correlating with improved motor function.
Current Status & Timeline
Preclinical Studies Completed
Efficacy and safety demonstrated in multiple animal models
IND-Enabling Studies (Current)
Formal toxicology and CMC studies in progress
75% Complete
IND Filing Targeted
FDA submission planned within 12-18 months
AET-101 Key Advantages
Brain-Penetrant
Engineered for efficient BBB crossing
Selective Targeting
Binds pathogenic α-synuclein forms
Favorable Safety
Designed for low immunogenicity
Disease Modifying
Targets root cause, not just symptoms
Our Discovery Engine
Building the Future of Neuroscience Therapeutics
AET-101 is the first of many candidates to emerge from our discovery engine. The modularity, speed, and precision of the Cognate-AI™ platform allow us to rapidly prosecute new targets for a range of proteinopathies and other neurological disorders.
We are actively expanding our internal pipeline to address the root causes of diseases like Alzheimer's, Amyotrophic Lateral Sclerosis (ALS), and Huntington's Disease.
Our systematic approach transforms validated but "undruggable" targets into accessible therapeutic opportunities.
Expanding Our Therapeutic Reach
Alzheimer's Disease
Target: Amyloid-β & Tau
Targeting multiple pathological proteins simultaneously
ALS
Target: TDP-43 & SOD1
Addressing protein aggregation in motor neurons
Huntington's Disease
Target: Huntingtin Protein
Preventing toxic protein accumulation
Strategic Partnerships
We believe that collaboration is key to maximizing the impact of our technology. We are actively seeking strategic partnerships with global pharmaceutical leaders to accelerate the development of our internal programs and apply our platform to partner-nominated targets.
This dual strategy of internal development and external collaboration is designed to bring a new generation of medicines to patients as quickly as possible.
Global Reach
Partnering with leading pharma companies worldwide
Target Flexibility
Platform adaptable to partner-nominated targets
Shared Expertise
Combining AI innovation with clinical development experience