Antisense Restoration of ELAVL3 for Neurological Diseases

Application

Antisense therapeutics targeting ELAVL3 expression for neurodegenerative disorders.

Key Benefits

Antisense approach for restoring protein levels and function of ELAVL3 and TDP-43.
Preclinical data show the ability of several ELAVL3-targeting ASOs to restore protein expression via in vitro experiments.
Potential to be a new medicine for many neurodegenerative conditions without effective treatments.

Market Summary

More than a billion people worldwide suffer from some form of neurological disorder, such as Alzheimer's (AD), Parkinson's (PD), multiple sclerosis, epilepsy, stroke, or autism. These conditions result in more than 7 million deaths, with associated costs exceeding $1.5 trillion annually. It's estimated that less than 10 percent of those suffering from neurological discords in developing countries go untreated due to the limited availability of adequate and low-cost treatments. The underlying causes of these diseases are unknown, but genetic factors and exposure to environmental insults are believed to be involved in their pathogenesis. Most neurological diseases cannot be cured, and available drugs have limited efficacy and severe side effects. Hence, there is a significant need to develop new treatment options for these devastating conditions.

Technical Summary

Emory researchers are developing a new therapeutic modality using antisense oligonucleotides (ASOs) to restore ELAVL3, a protein that has been shown to be involved in many neurological conditions. ELAVL3 is a transcript that modulates TDP-43, which is depleted in diseased cells. Thus far, the inventors have developed several ELAVL3 ASO-based drug candidates within the laboratory. Through several in vitro disease models, the inventors showed the ability of their ASOs to restore ELAVL3 protein levels associated with TPD-43 loss and may be a viable treatment option for neurodegenerative conditions.