Application:

  • Around 55 million people globally are currently living with Alzheimer’s disease or other forms of dementia. In 2024, Alzheimer’s and other dementias are estimated to cost about $360 billion in the U.S., with expenses projected to reach $1.1 trillion by 2050.
  • Post-translational modification by the ubiquitin-like protein UFM1 has been extensively shown to be essential for brain development. Recently, alterations in this modification have been observed in patients with Alzheimer’s disease (AD). Specifically, hyperufmylation, an abnormal increase in ufmylation activity, has been detected in AD brains and is significantly associated with pathological tau, a protein that plays a key role in the development of neurofibrillary tangles, a hallmark of AD. Given the absence of a cure for AD, targeting UFMylation presents a promising new avenue for treatment exploration.

Our innovation:

  • A novel method for detecting ufmylation, making it compatible with high-throughput screening for drug discovery.
  • Our technology addresses the challenge of detecting post-translational modifications by UFM1(ufmylation), which are difficult to identify and can impede high-throughput screening. This challenge limits the potential of UFM1 as a novel drug target.

Advantages:

  • Simple Assay with Recombinant Proteins: The assay is based on recombinant proteins, making it straightforward to detect small molecules that affect ufmylation.
  • Modular Design: This assay can be adapted for use with other ubiquitin-like proteins, enhancing its versatility.
  • Screening Capabilities: It allows for the screening of not only inhibitors but also accelerators, providing a broader range of potential compounds.
  • Counter-Screening: The technology can be easily modified for counter-screening to address false positive hits, improving the accuracy of the results.

Commercial Opportunity:

Our method will bring to the development of new drugs for Alzheimer’s Disease, since the UFMylation pathway regulates protein homeostasis and ER stress response, key factors in neurodegeneration and amyloid accumulation.

The same approach can be applied to breast and lung cancers, where UFMylation controls cell survival pathways and stress responses, making it a promising target for therapy-resistant tumors. Additionally, rare blood disorders and developmental conditions caused by UFMylation defects represent significant opportunities for targeted therapeutics, as these currently lack effective treatments.