Application:          

Bipolar Disorder (BD) affects millions worldwide, with a high heritability of 0.70. Current treatments, like lithium, are not effective for all patients. BD is genetically complex, with no single causal variant identified, and there are no good animal models due to this complexity. Existing treatments often have limited efficacy and significant side effects, leaving a substantial unmet need in the market for more effective and personalized treatment options.

Our innovation:

Our groundbreaking approach introduces two molecules, HU-40 and HU-41, targeting key pathways in BD.

  • HU-40 inhibits a crucial regulator of neuronal development and function.
  • HU-41 prevents mitochondrial dysfunction.

This innovative treatment utilizes human induced pluripotent stem cell (iPSC)-derived neurons from treatment resistant patients as a DNA-precise model for BD, ensuring genetic accuracy. Our comprehensive methodology combines proteomics, bioinformatics, biochemical, pharmacological, electrophysiological, and molecular biology tools to develop and refine these promising therapeutic candidates.

Advantages:

  • Precision Medicine Approach: Uses patient-specific iPSC-derived neurons, ensuring genetic accuracy, and allows for personalized treatment strategies based on individual patient profiles.
  • Dual-Action Treatment: Targets multiple pathways implicated in BD pathology and holds potential for synergistic effects, improving overall efficacy.
  • Mechanism of Action: Addresses underlying cellular and molecular dysfunctions in BD and holds potential to treat both lithium-responsive and non-responsive patients.
  • Improved Safety Profile: A targeted approach may reduce off-target effects and side effects.
  • Bridging the Translation Gap: Direct testing on human neurons reduces risks associated with animal-to-human translation.

Opportunity:

The global BD therapeutics market is projected to reach $6.9 billion by 2027, with a CAGR of 2.6%. There is a high unmet need, as 30-50% of BD patients do not respond adequately to current treatments.

Our innovation presents potential as a first-in-class therapy for treatment-resistant BD, a combination therapy with existing treatments, and possible expansion into other mood disorders and neurodegenerative diseases.

We are seeking partnerships with major pharmaceutical companies for development and commercialization, positioning our treatment to make a significant impact in the BD therapeutics market.