Background

Project Transfidelity is developing a novel approach to target the root cause of neurodegenerative diseases like Alzheimer’s. Instead of treating symptoms, we aim to improve protein production accuracy and prevent toxic aggregates from forming in the first place.

Led by Dr. Dimitri Scherbakov and Dr. Rashid Akbergenov, with over 50 years of combined experience, the team has identified two molecules with the potential to protect against protein misfolding and cognitive decline. Cerebrum DAO will support the development of near- and long-term solutions for neurodegeneration and aging.
Project Transfidelity has the potential to lead in this fast-growing market by focusing on prevention rather than treatment. This IPT represents a new opportunity to create novel IP around protein fidelity and drive future studies aimed at addressing the underlying causes of aging and dementia.

Commercial Potential

There is a high unmet need for effective treatments for neurodegenerative diseases and the growing prevalence of these conditions. Alzheimer's disease alone affects approximately 55 million people worldwide, with annual healthcare costs reaching $1.3 trillion. The final product of this research will be a small molecule to treat age-related neurodegenerative disorders like Alzheimer’s and Parkinson’s that would benefit from enhanced translation fidelity.

Milestones

Milestone 1: Reduction in protein aggregates in human-origin cell lines.

The two compounds will be tested in disease-relevant human cells, and their impact on protein aggregation will be measured.

Required Funding: $29,000
Duration: 8 Months

Milestone 2: Identification of impacted biological pathways and their cellular counterparts.

To determine the mechanism of action of the compounds, various proteomics and transcriptomics assays will be performed. These assays give information on which systems the compounds interact with, which is critical in understanding how to develop the therapeutic and will aid in prioritizing the hits.

Required Funding: $18,980
Duration: 5 Months

Milestone 3: Establishment of a clear target product profile.

We will consult a medicinal chemist to assist us in creating a target product profile as well as provide recommendations for therapeutic development. They will check for any red flags in the current hits and check the patent landscape.

Required Funding: $20,000
Duration: 3 Months

Senior Review

All reviewers acknowledged the strong scientific foundation behind Project Transfidelity and its innovative approach of improving protein translation accuracy to address neurodegenerative diseases. They support the project but highlight that it is still early stage, with major risks around proving effectiveness in disease models, refining the drug candidates, and securing strong IP. Reviewers noted the need for more validation linking translational fidelity directly to human disease, concerns about off-target effects, and challenges in fundraising due to the project's deviation from more established hypotheses like amyloid. Despite these risks, they believe the project is fundable with expert guidance, a clear proof-of-concept path, and a focus on building drug development expertise early.