Finding Cures For Neurodegeneration and Cystic Fibrosis

In the realm of ALS, Alzheimer's disease, frontotemporal lobar degeneration, and cystic fibrosis, the lack of effective treatments presents a dire need. These incurable diseases, affecting countless lives, underscore the urgency of our mission to develop groundbreaking therapies that can bring hope and relief to patients worldwide.

TDP-43 accumulation, prevalent in various neurodegenerative diseases, shifts from being essential in early development to highly toxic in adult neurons. This toxicity, causing dysfunction across different models, highlights the importance of targeting TDP-43 to combat the underlying cause of these debilitating conditions.

Our strategic innovation lies in addressing neurodegeneration at its source - TDP-43. This protein's late-life toxicity is a major contributor to neuronal damage. By inhibiting its nucleic acid binding, our approach offers a multifaceted solution, potentially halting or reversing multiple neurodegenerative processes.

The ALTA drug series marks a therapeutic breakthrough in treating neurodegenerative diseases. By specifically targeting the guanine pocket of TDP-43, ALTA compounds disrupt its detrimental interactions, offering a unique and promising avenue in the quest to alleviate these complex conditions.