Invention Title:

Universal mRNA Vaccine to Treat Dementia-Causing Neurodegenerative Disorders

Publication number:

US20240293519

Publication date:
Section:

Human necessities

Class:

A61K39/0007

Inventors:

Assignee:

Applicants:

Smart overview of the Invention

A novel approach to combat neurodegenerative disorders (NDs) such as Alzheimer's Disease (AD), Amyotrophic Lateral Sclerosis (ALS), and others involves the use of mRNA coding vaccines. These vaccines target improperly folded or aggregated proteins, including amyloid-beta and tau proteins, by generating specific antibodies. The method utilizes epitopes from these proteins to create open reading frames for the mRNA vaccines, enhancing the immune response against these harmful aggregates.

Targeted Proteins and Their Role in Neurodegeneration

Key proteins implicated in neurodegenerative diseases include:

  • Aβ42: Associated with amyloid plaques in Alzheimer's Disease.
  • Tau Protein: Forms tangles that disrupt neuronal function.
  • α-Synuclein: Linked to Lewy Body Dementia and Parkinson's Disease.
  • TDP-43 and FUS: Involved in ALS and other disorders.

The mRNA vaccines aim to produce antibodies that recognize these proteins as neoantigens, facilitating their clearance from the nervous system.

Mechanism of Action and Formulation

The mRNA is encapsulated in lipid nanoparticles designed to effectively cross the blood-brain barrier (BBB). This formulation enhances the delivery of the vaccine components directly to the central nervous system (CNS), promoting a localized immune response. By targeting misfolded proteins, the vaccine seeks to mitigate the progression of neurodegeneration and restore normal cellular function.

Understanding Neurodegenerative Diseases

Neurodegenerative diseases are characterized by progressive neuronal loss leading to cognitive decline and motor dysfunction. Factors contributing to these conditions include genetic predispositions, environmental exposures, and traumatic injuries. The aggregation of proteins like Aβ42 and tau disrupts neural communication and triggers inflammatory responses, exacerbating disease progression.

Implications for Future Treatments

This innovative mRNA vaccine strategy presents a promising avenue for treating various neurodegenerative disorders. By targeting the underlying protein aggregates that contribute to cognitive decline, it has the potential to alter disease trajectories significantly. Continued research and clinical development may lead to effective therapeutic options for patients suffering from these debilitating conditions.